Mainstay Medical Provides Company Update and Publishes 2018 Full Year Results

  • ReActiv8-B study results received; pre-PMA submission meeting with FDA expected during the second quarter

  • European commercialization continues to advance

  • Cash on hand of $15.5m at 31 December 2018

Dublin,Ireland – Mainstay Medical International plc (“Mainstay” or the “Company”, Euronext Paris: MSTY.PA and Euronext Growth operated by Euronext Dublin (MSTY.IE), a medical device company focused on bringing to market ReActiv8, an implantable restorative neurostimulation system to treat disabling Chronic Low Back Pain, today provides a company update and announces the publication of its 2018 Full Year results and Annual Report.

Jason Hannon, CEO of Mainstay, said: “I believe fully in ReActiv8 as a therapeutic option for patients suffering from disabling Chronic Low Back Pain, and I believe that the value we can deliver to both our physician customers and their patients, as well as the investing market, will be demonstrated in the months and years to come. The primary endpoint of our ReActiv8-B clinical trial was not achieved due to a higher response (at 120 days) in the control (active sham) patient group than was expected. However, we believe the overall results from the trial represent solid evidence of the efficacy and safety of ReActiv8. This belief is based on, among other things:

  • A pre-specified analysis of the primary endpoint where we adjusted for patients who increased their pain medications for reasons unrelated to their back pain. As a reminder, to be considered a “Responder” in the clinical trial, a patient must have reported 30% or greater pain relief on the VAS scale and not increased their pain medications leading up to the 120 day measurement point;

  • Statistically significant differences between the treatment and control groups on key secondary efficacy endpoints at 120 days;

  • A responder rate of 72% in the patients in the treatment and control groups combined that have reached one year since implantation; and

  • A significant reduction in the use of pain medications (including opioids) by patients at one year.

We believe these results will support sales growth in Germany and other markets under our existing CE Mark, as well as our plan to file a Pre-Market Approval Application with the U.S. Food and Drug Administration. We plan to submit a PMA to the FDA in mid-2019, with a decision on approval expected in late 2020.”

Business Update

  • In November 2018, Mainstay announced top line results from the ReActiv8-B clinical study, its international, multi-center, prospective, randomized, active sham-controlled, blinded trial with one-way cross-over, conducted under an Investigational Device Exemption (IDE) from the U.S. Food & Drug Administration (FDA). A total of 204 patients with chronic low back pain refractory to physical therapy were implanted with ReActiv8 at leading clinical sites in the U.S., Europe and Australia and randomized 1:1 to therapy or control. The average duration of symptoms was 14 years. In the treatment group, the ReActiv8 pulse generator was programmed to deliver electrical stimulation expected to elicit contractions of the multifidus muscle. In the control group, the ReActiv8 device was programmed to provide a low level of electrical stimulation. Following assessment of the primary endpoint at 120 days, patients in the control group crossed-over to receive levels of electrical stimulation similar to those in the treatment group. A summary of the clinical trial results is as follows:

    • The primary efficacy endpoint of the study was a comparison of responder rates between the treatment and control groups as measured on the visual analog scale (VAS) of pain, with responders defined as having a 30% or greater improvement on this measure between baseline and 120 days after randomization, without any increase in pain medication and/or muscle relaxants taken in the two weeks prior to the primary endpoint assessment visit. In the treatment group the responder rate at 120 days was 57%, compared to 47% in the control group, resulting in a difference that is not statistically significant.

    • Statistically significant differences on a number of key secondary endpoints were observed in the treatment group as compared to the control group at 120 days, including change from baseline in disability measured by the Oswestry Disability Index (ODI), change from baseline in quality of life measured by the European Quality of Life Score on Five Dimensions (EQ-5D), percent pain relief (PPR) compared to baseline and subject global impression of global change (SGIC).

    • Improvements in the percentage of patients reporting pain reduction continued beyond the 120-day assessment through one year for both groups. The percentage of the 116 patients in the treatment group and control groups that had completed the one-year assessment having a 30% or greater reduction in low back pain VAS at that assessment without a significant increase in pain medication was 72%. These data are subject to change as the remaining patients reach the one-year assessment.

    • The protocol permitted patients to adjust their back pain medication usage after the 120-day assessment point. At one year, 44% of the 50 patients in both groups combined who were on opioids at baseline had voluntarily eliminated (28%) or significantly reduced (16%) their use of opioids. These results are subject to change as the remaining patients reach the one-year assessment.

    • The incidence and type of adverse events (AEs), including serious AEs, were comparable to AEs in clinical trials reported for other neurostimulation devices, with no unanticipated AEs related to the device, procedure or stimulation.

  • Mainstay is preparing to submit a PMA application to the FDA based upon the totality of its clinical data for ReActiv8. The Company expects to have a pre-PMA submission meeting with the FDA during the second quarter to obtain feedback on its filing content and strategy. A PMA application filing is expected in mid-2019 after that meeting, with a decision on approval expected in late 2020. The FDA’s review of the PMA may result in the FDA not agreeing with Mainstay’s interpretation of its clinical data, including whether statistical significance was achieved for one or more endpoints.

  • In Germany, Mainstay’s initial European market, the commercial team was repositioned in order to better focus efforts on key physician targets. Commercialization efforts in line with this strategy began in earnest in late-2018. We are beginning to see the early fruits of these efforts as we regularly onboard new implanting physicians. We expect this early momentum to continue, leading to meaningful sales and revenue by the end of this year and the beginning of 2020.

Financial Update

  • Since the beginning of 2018, Mainstay has conducted significant financing activities:

    • On 18 April 2019, Mainstay and its subsidiary, Mainstay Medical Limited, entered into an amendment to its agreement with IPF Partners relating to their existing debt facility. Pursuant to the amendment:

      • The repayment schedule for the three existing tranches drawn under the debt facility was amended such that no principal or interest will be repaid until 2021, with the principal and accrued interest to be amortized over the period from January 1, 2021 through September 30, 2023.

      • A new tranche of €3.0 million (approximately $3.34 million) was made available to Mainstay, conditioned upon Mainstay raising at least $10 million in gross proceeds from one or more offerings of equity prior to June 30, 2019. The repayment schedule for the new tranche will be the same as the amended repayment schedule for the three existing tranches.

      • The interest rate for all tranches will be 8% per annum, with interest accruing but capitalized prior to January 1, 2021.

      • The 5% repayment fee applicable to each existing tranche was eliminated.

      • All principal and accrued interest from all tranches will automatically convert into ordinary shares of the Company at a price per share of €8 upon the earlier of (a) FDA approval of Mainstay’s PMA application for ReActiv8, (b) the date by which at least 900,000 ordinary shares of the Company are publicly sold on-market by non-affiliates of Mainstay since 18 April 2019 at a price per share of at least €8, or (c) IPF Partners’ election to undertake such conversion, in each case unless the Company elects to satisfy such obligation in whole or in part in cash.

      • The minimum cash covenant was amended so that Mainstay is required to hold cash at least equal to its projected cash expenditures for operations and debt repayment for the next three months, and the covenant relating to the achievement of commercial milestones was eliminated.

      • Mainstay issued to IPF Partners warrants to purchase 1.5 million of its ordinary shares at a price per share of €6 at any time prior to the 6th anniversary of the amendment date. Mainstay has issued further conditional warrants to IPF Partners that will become exercisable only to the extent Mainstay elects to repay the debt in cash rather than issue ordinary shares when a conversion of the debt is triggered. As such, the conditional warrants are intended to ensure that, notwithstanding any such election to repay in cash, IPF Partners retains the right to subscribe for ordinary shares of the Company on the terms and conditions that would otherwise have applied.

    • On 15 February 2018, the Company announced the completion of a €30.1 million financing (approximately $37.5 million) through a placement of 2,151,332 new ordinary shares to new and existing shareholders.

  • Revenue during the year ended 31 December 2018 was $0.7 million (2017: $0.3 million).

  • Operating expenses related to on-going activities were $29.6 million during the year ended 31 December 2018 (2017: $27.9 million).

  • Cash on hand as at 31 December 2018 was $15.5 million (2017: $10 million).

About Mainstay

Mainstay is a medical device company focused on commercializing an innovative implantable restorative neurostimulation system, ReActiv8®, for people with disabling Chronic Low Back Pain (CLBP). The Company is headquartered in Dublin, Ireland. It has subsidiaries operating in Ireland, the United States, Australia, Germany and the Netherlands, and is listed on the regulated market of Euronext Paris (MSTY.PA) and the ESM of Euronext Dublin (MSTY.IE).

About Chronic Low Back Pain

One of the root causes of CLBP is impaired control by the nervous system of the muscles that dynamically stabilize the spine. ReActiv8 is designed to electrically stimulate the nerves responsible for contracting these muscles to improve dynamic spine stability, allowing the body to recover from CLBP.

People with CLBP usually have a greatly reduced quality of life and score significantly higher on scales for pain, disability, depression, anxiety and sleep disorders. Their pain and disability can persist despite the best available medical treatments, and only a small percentage of cases result from an identified pathological condition or anatomical defect that may be correctable with spine surgery. Their ability to work or be productive is seriously affected by the condition and the resulting days lost from work, disability benefits and health resource utilization put a significant burden on individuals, families, communities, industry and governments.

Further information can be found at www.mainstay-medical.com

CAUTION – in the United States, ReActiv8 is limited by federal law to investigational use only.

PR and IR Enquiries:

Investor Relations:

LifeSci Advisors, LLC

Brian Ritchie

Tel: + 1 (212) 915-2578

Email: britchie@lifesciadvisors.com

Euronext Advisers:

Davy

Fergal Meegan or Barry Murphy

Tel: +353 1 679 6363

Email: fergal.meegan@davy.ie or barry.murphy2@davy.ie

Velicept Therapeutics Announces Positive Top-Line Phase 2b Results for Solabegron in the Treatment of Overactive Bladder (OAB)

  • Study VEL-2002 further validates solabegron’s potential for best-in-class efficacy across multiple primary and secondary endpoints

Wayne, PA - Velicept Therapeutics, a privately-held specialty pharmaceutical company dedicated to the development of best-in-class compounds for the treatment of urological and gastrointestinal disorders, announced that its next generation beta-3 adrenoceptor agonist solabegron met the primary endpoint in VEL-2002, a Phase 2b study in patients with overactive bladder (OAB). In the study, twice-daily administration of solabegron demonstrated a statistically significant improvement compared to placebo at week 12, as measured by the mean change in number of micturitions per day, the study’s primary endpoint. Solabegron also demonstrated statistical significance across multiple secondary endpoints including percent reduction of urge urinary incontinence episodes, dry rate, and urgency episodes.


The 12-week placebo-controlled VEL-2002 study enrolled 435 women ages 18 to 80 suffering from OAB. Solabegron was generally well-tolerated. Treatment emergent adverse events and serious adverse events were infrequent and comparable between the solabegron and placebo-treated groups. Velicept plans to submit the data from the VEL-2002 study for presentation at upcoming scientific meetings.


“We are encouraged by these positive top-line results for VEL-2002 as they are consistent with what we’ve seen in a prior study with solabegron undertaken by GSK and further validate the potential for best-in-class efficacy and improved safety for patients with OAB,” said James Walker, President and Chief Executive Officer, Velicept Therapeutics. “We look forward to the results of our second study, which will help us finalize our plans for moving forward with a pivotal Phase 3 program early next year.”


Velicept has also developed a novel, once-daily formulation of solabegron which is being evaluated in an ongoing Phase 2b study (VEL-2001). VEL-2001 has completed enrollment and the company expects to announce top-line results in the second quarter of this year.


About Solabegron

Solabegron is a highly potent and selective beta-3 adrenoceptor agonist being investigated for overactive bladder (OAB) and irritable bowel syndrome (IBS). A Phase 2 study of solabegron in OAB, previously conducted by GlaxoSmithKline, evaluated a twice-daily dose in 258 patients with moderate to severe incontinence experiencing an average of 4.5 wet episodes per day. Results demonstrated a statistically significant improvement with solabegron as compared to placebo. Furthermore, the Phase 2 study also indicated a safety and tolerability profile for solabegron that was similar to placebo.


About Velicept

Velicept Therapeutics, Inc. is a privately-held, clinical development company focused on advancing best-in-class compounds for the treatment of urological and gastrointestinal disorders. Its lead product, solabegron, is a highly potent and selective beta-3 adrenoceptor agonist being investigated for overactive bladder (OAB) and irritable bowel syndrome (IBS). For more information, visit www.velicept.com.


Contacts

Media:

W2Otwist

Sheryl Seapy, (213) 262-9390

sseapy@twistmktg.com

Inflazome receives funding from The Michael J. Fox Foundation for Parkinson’s Research

  • Funding in excess of US$1 million to support the development of a brain imaging probe for patient diagnosis and the clinical development of drugs to treat neurodegenerative diseases

  • Inflazome is developing orally available drugs to address clinical unmet needs in inflammatory diseases by targeting the NLRP3 inflammasome

  • NLRP3 activation is now associated with the progression of Parkinson’s Disease

Dublin (IE), Cambridge (UK): Inflazome (inflazome.com), the pioneering biotech company developing several small molecule drugs that inhibit harmful inflammation, today announces it has been awarded funding in excess of US$1 million by The Michael J. Fox Foundation for Parkinson’s Research (MJFF). The grant will fund the development of a NLRP3-specific Positron Emission Tomography (PET) tracer to allow non-invasive imaging of inflammasome-driven inflammation in the brain.

To enhance the accuracy and probability of success of a clinical trial in neurodegenerative disease, it is important to select suitable patients at the appropriate staging of the disease. It is also essential to determine whether the biological target of interest is being engaged by the drug in the brain. One approach to achieve this in the central nervous system (CNS) is by using an NLRP3-specific tracer during a PET scan. The tracer could quickly, accurately and non-invasively produce images showing the drug binding to target inflammasomes in the brain.

The NLRP3 inflammasome is believed to drive chronic inflammation associated with the progression of many neurodegenerative diseases, including Parkinson’s Disease. The PET tracer will also help Inflazome to determine what doses are needed for patients in larger clinical trials in the future. The Principal Investigator on this project is Prof. Matthew Cooper, CEO and co-founder of Inflazome, who is assisted by Co-Investigator Dr David Miller, Head of Medicinal Chemistry.

Inflazome is developing orally available drugs to address clinical unmet needs in inflammatory diseases by targeting the NLRP3 inflammasome, which is now understood to drive many chronic and acute inflammatory conditions. The NLRP3 inflammasome was recently shown to be associated with the progression of Parkinson’s Disease in humans and in non-clinical models, in research published on 31 October 2018 in Science Translational Medicine (Link).

This innovative research was co-authored by Prof. Cooper with research teams at The University of Queensland, Australia, led by A/Prof. Trent Woodruff and funded by The Michael J. Fox Foundation for Parkinson’s Research and the Shake It Up Australia Foundation.

Prof Matt Cooper, Co-Founder and CEO of Inflazome, commented: “The Michael J. Fox Foundation is a fantastic organisation with a passionate commitment to new science, science translation and candidate therapies for Parkinson’s. We are fully aligned in our shared goal to help patients with Parkinson’s and other debilitating neurodegenerative diseases, for which there are inadequate therapies and no cures. Their support will help us advance and hopefully validate our disruptive approach to diagnose and then treat patients by focusing on neuroinflammation.”

Dr Jamie Eberling, Director of Research Programs at MJFF, said, “An imaging tool to visualize neuroinflammation may help investigate Parkinson’s onset and progression as well as evaluate new treatments that could alter the course of the disease. Our Foundation is investing in this research due to the significant potential impact on drug development and patient lives.”


About Parkinson’s Disease

Parkinson’s disease is the second most common neurodegenerative disorder worldwide, affecting more than 10 million people. It is characterised by the loss of dopamine-producing neurons, accompanied by chronic inflammation in the brain. Inflazome has identified drugs to stop the chronic cycle of inflammation in the brain. Research published in Science Translational Medicine on 31 October 2018 found that the tool compound MCC950, a potent inhibitor of the NLRP3 inflammasome, given orally once a day could stop neuroinflammation. MCC950 arrested the effects of Parkinson’s in several animal models of the disease, leading to reduced brain neuron loss and higher levels of dopamine.

A link to a Parkinson’s Disease explainer video can be found here


About Inflazome

Utilizing the scientific expertise of our founders and advisors, Inflazome is leading the way in developing rally available drugs to address clinical unmet needs in inflammatory diseases by targeting the NLRP3 inflammasome, which is now understood to drive many chronic inflammatory conditions. Headquartered in Dublin, Ireland, Inflazome was founded in 2016 by leading academics Professor Matt Cooper, The University of Queensland (Australia) and Professor Luke O’Neill, Trinity College Dublin (Ireland), following a highly productive joint collaboration.

To learn more visit: inflazome.com


About The Michael J. Fox Foundation for Parkinson's Research

As the world's largest nonprofit funder of Parkinson's research, The Michael J. Fox Foundation is dedicated to accelerating a cure for Parkinson's disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research program coupled with active global engagement of scientists, Parkinson's patients, business leaders, clinical trial participants, donors and volunteers. In addition to funding more than $800 million in research to date, the Foundation has fundamentally altered the trajectory of progress toward a cure. Operating at the hub of worldwide Parkinson's research, the Foundation forges ground-breaking collaborations with industry leaders, academic scientists and government research funders; increases the flow of participants into Parkinson's disease clinical trials with its online tool, Fox Trial Finder; promotes Parkinson's awareness through high-profile advocacy, events and outreach; and coordinates the grassroots involvement of thousands of Team Fox members around the world.

Syndesi Therapeutics awarded non-dilutive funding of up to €3.2M for development of its lead program, SDI-118, aimed at treating cognitive impairment

Syndesi Therapeutics SA, a biotechnology company developing novel SV2A modulators for cognitive impairment, today announced it has been granted up to EUR 3.2 M in non-dilutive funding from the Walloon Region. The funding is provided in the form of an ‘avance récupérable’ (recoverable loan), repayable under certain conditions with the majority of the repayment linked to the success of the programme.

Jonathan Savidge, CEO of Syndesi, said “We very much appreciate the Walloon Region’s ongoing support for the continued development of this program. This is an area of high unmet need where it has proven challenging to develop new therapeutics. This additional support will enable us to leverage our investor financing to expand the clinical development program for SDI-118 and maximize the chance of bringing this promising molecule to patients.”

The funding will support the development of SDI-118 through Phase I clinical studies, which will include PET imaging to directly measure the binding of SDI-118 to its target in the brain, providing key information for subsequent dose selection.

About Syndesi Therapeutics

Syndesi Therapeutics was established to develop a series of novel, pro-cognitive small molecule SV2A modulators licensed from UCB, and in February 2018 announced €17M in Series A funding from a syndicate of Belgium and international investors. Syndesi is investigating the potential of these novel SV2A modulators to improve cognition in diseases such as Alzheimer’s Disease and other dementias, as well as cognitive impairment associated with schizophrenia and other conditions. The discovery of the lead molecule, SDI-118, at UCB benefitted from prior support of the Walloon Region. For more information please visit www.syndesitherapeutics.com

Prof. Marco Colonna M.D. Join's Inflazome's Scientific Advisory Board

Distinguished scientist with deep expertise in innate immunity and its role in neurodegenerative diseases

Dublin (IE), Cambridge (UK): Inflazome (inflazome.com), the pioneering inflammasome company, announces the appointment of distinguished scientist, Prof. Marco Colonna M.D. to its Scientific Advisory Board. Prof. Colonna has deep expertise and extensive experience in innate immunity. His team is responsible for the innovative discovery of Triggering Receptors Expressed on Myeloid cells (TREM), important regulators of the innate immune response. Prof. Colonna is currently investigating TREM’s link to Alzheimer’s Disease at the Washington University School of Medicine in St. Louis, MO, where he has been a Professor of Pathology and Immunology since 2001.

With the appointment of Prof. Colonna, Inflazome continues to strengthen its Scientific Advisory Board, a collaboration of eminent, world renowned scientists who will work closely with the management team as Inflazome prepares to advance its lead product candidates into clinical studies later this year.

On his appointment Prof. Colonna commented: “Neurodegenerative diseases are growing at an alarming rate. A small molecule that crosses the blood brain barrier and inhibits NLRP3 in the brain has the potential to open a new exciting avenue for therapeutic intervention. I am delighted to join the Inflazome Scientific Advisory Board to help advance this potential”.

Prof Luke O’Neill, Co-Founder and CSO of Inflazome, commented: “I am delighted to welcome Marco to Inflazome’s SAB. He brings a wealth of experience in the molecular and cellular basis of innate immunity, especially in the Central Nervous System. He will bring tremendous new insights into our research and clinical programs”.

About Prof. Marco Colonna

Prof. Marco Colonna, born in Parma, Italy, received his medical degree at Parma University and completed his postdoctoral training at Harvard Medical School (Cambridge, MA, USA). He became a scientific member of the Basel Institute for Immunology in Basel, Switzerland. Since 2001 he has been a Professor of Pathology & Immunology at Washington University School of Medicine in St. Louis, MO. Prof. Colonna’s research focuses on immunoreceptors. In this field, his accomplishments include identification and characterization of the Killer cell Ig-like receptors and HLA-C polymorphisms as their inhibitory ligands, as well as the discovery of the LILR and TREM inhibitory and activating receptor families.

Through analysis of the cellular distribution of these receptors, Prof. Colonna identified plasmacytoid dendritic cells as source of IFNa/b in anti-viral responses and innate lymphoid cells that produce IL-22 in mucosae. His current areas of research include:

1) TREM2 and innate immunoreceptors in Alzheimer's disease.

2) Innate lymphoid cells in mucosal immunity.

3) Plasmacytoid dendritic cells in host defense and autoimmunity.


Prof. Colonna has published over 80 primary last-author studies in peer-reviewed journals, and holds editorial appointments for many publications, including European Journal of Immunology, Immunity, Journal of Experimental Medicine, Blood, Journal of Clinical Investigation, and Human Immunology.

Members of the Inflazome’s Scientific Advisory Board include:

- Prof. Luke O’Neill, Trinity College Dublin

- Prof. Marco Colonna, MD, Washington University School of Medicine

- Prof. Veit Hornung, Ludwig Maximilian University of Munich

- Dr. David Morris, MD, Novartis Venture Fund

- Prof. Mihai G. Netea, Nijmegen University

- Prof. Paul M. Ridker, MD, Brigham and Women’s Hospital

About Inflazome

Utilizing the scientific expertise of our founders and advisors, Inflazome is leading the way in developing orally available drugs to address clinical unmet needs in inflammatory diseases by targeting the NLRP3 inflammasome, which is now understood to drive many chronic and acute inflammatory conditions from Alzheimer’s and Parkinson’s to cardiovascular, liver, kidney and inflammatory bowel diseases.

Headquartered in Dublin, Ireland, Inflazome was founded in 2016 by leading academics Prof. Matt Cooper, The University of Queensland (Australia) and Prof. Luke O’Neill Trinity College Dublin (Ireland), following a highly productive joint collaboration.

To advance its programs to clinical development, Inflazome recently closed a successful Series B financing of €40M / US$46M led by Forbion Capital Partners with Longitude Capital, with Series A investors, Fountain Healthcare Partners and Novartis Venture Fund, also participating.

To learn more visit: inflazome.com

KaNDy Therapeutics announces initiation of Phase 2b trial of NT-814 for the treatment of troublesome post-menopausal symptoms

Stevenage, UK - KaNDy Therapeutics, a clinical-stage company developing a potential breakthrough non-hormonal treatment for multiple debilitating symptoms of the menopause, today announces initiation of the Phase 2b clinical trial evaluating NT-814 in women with troublesome symptoms of the menopause. The study (called SWITCH-1) will evaluate the effect of treatment with NT-814 on hot flashes and other symptoms of the menopause.

Dr. Steve Pawsey, Chief Medical Officer of KaNDy Therapeutics, said: “We are very pleased to have initiated the Phase 2b, SWITCH-1 study, on schedule with our previous guidance. We anticipate that results from this trial will validate the Phase 2a results and position NT-814 as a novel, non-hormonal treatment for women suffering from troublesome symptoms of the menopause. We will continue to enrol patients over the coming months and look forward to reporting results in late 2019. We expect NT-814 to be fully Phase 3 ready in H2 2020”

Prof. James A. Simon, Coordinating Principal Investigator on the SWITCH-1 Study and Scientific and Clinical Advisor to KaNDy Therapeutics said: “There are millions of women worldwide suffering from debilitating symptoms of the menopause and a non-hormonal treatment that could offer relief would be extremely valuable to this population. NT-814 has delivered very encouraging results to date and I am hopeful that findings from this trial will be equally promising.”

SWITCH-1 is a Phase 2b, double-blind, randomised, placebo-controlled, adaptive study designed to determine the effectiveness and safety of NT-814, taken once daily, for the treatment of troublesome post-menopausal symptoms. Four doses of NT-814 (40 mg once a day, 80 mg once a day, 120 mg once a day and 160 mg once a day) will be investigated and compared to placebo, in five parallel groups. The study is anticipated to enrol approximately 165 postmenopausal women aged 40 to 65 years although the exact number will be determined during the adaptive design reviews. Subjects will participate in the study for a total of approximately 19 weeks, comprising a screening period of three weeks, a 12-week treatment period and then a final follow up visit four weeks after the end of the treatment period. The co-primary efficacy endpoints will be the change from baseline in frequency and severity of moderate and severe hot flashes at Weeks 4 and 12 of treatment.

More information can be found at www.clinicaltrials.gov, identifier: NCT03596762

For more information, please contact:

KaNDy Therapeutics

Mary Kerr, Chief Executive Officer

Tel: +44 1438 906960

Email: info@kandytherapeutics.com

Consilium Strategic Communications

Mary-Jane Elliott/ Lindsey Neville/ Carina Jurs

Tel: +44 (0) 20 3709 5700

KaNDyTherapeutics@consilium-comms.com

KaNDy Therapeutics is a clinical-stage company developing a potential breakthrough non-hormonal treatment for multiple debilitating symptoms of the menopause including hot flashes and sleep disturbance due to night sweats. KaNDy’s drug in development, NT-814 is a unique dual mechanism NK(neurokinin)-1,3 receptor antagonist with the potential to ‘switch off’ multiple debilitating symptoms of the menopause without the need for oestrogen exposure.

Data from a Phase 1b/2a study in post-menopausal women with troublesome hot flashes showed NT-814 was able to rapidly and profoundly reduce both the frequency and severity of hot flashes as well as sleep disturbances due to night sweats. NT-814, is now being evaluated in a Phase 2b clinical trial in postmenopausal women with moderate to severe vasomotor symptoms. Headline results are expected at the end of 2019 and NT-814 will be fully Phase 3 ready by H2 2020.

For more information please visit https://www.kandytherapeutics.com/

Mainstay Medical to Showcase ReActiv8 and Key Clinical Data at the 13th German Spine Congress of the DWG

Mainstay Medical International plc (“Mainstay” or the “Company”, Euronext Paris: MSTY.PA and Euronext Dublin: MSTY.IE), a medical device company focused on commercializing ReActiv8®, an implantable restorative neurostimulation system designed to treat an underlying cause of disabling Chronic Low Back Pain, today announces that it will participate in the 13th German Spine Congress of the Deutsche Wirbelsäulengesellschaft (DWG), taking place in Wiesbaden from December 6-8. DWG will be the first medical meeting at which pivotal clinical data from the Company’s ReActiv8-B clinical study will be discussed.

A distinguished faculty of physicians, each of whom has deep experience with Reactiv8, will present the clinical data and the scientific background of the therapy, and describe commercial treatment of patients in Germany:

  • Dr. Jörg Franke, Chief, Department of Orthopedics, Klinikum Magdeburg, will chair the symposium where the results from the ReActiv8-B study will be presented, and also provide his experience with ReActiv8 in Germany;

  • Dr. Chris Gilligan, Chief, Division of Pain Medicine, Department of Anaesthesiology, Perioperative and Pain Medicine Brigham & Women’s Hospital, Assistant Professor of Anaesthesia, Harvard Medical and Principal investigator of the study, will present the pivotal clinical data from the ReActiv8-B study;

  • Dr. Jan Schilling, Chief of Spine and Neurosurgery, Tabea Hospital in Hamburg, will introduce the scientific background and the underlying physiological mechanisms of this new restorative treatment for chronic low back pain; and

  • Dr. Ardeshir Ardeshiri, Chief of Spine Surgery at Klinikum Itzehoe, will present his “real world” experience with ReActiv8 in Germany via his initial series of patient outcomes.

“As Germany is our first commercial market for ReActiv8, I am excited to be unveiling the data from our ReActiv8-B clinical study to the scientific community at the German Spine Congress,” said Jason Hannon, Chief Executive Officer of Mainstay. “We believe the long-term clinical results demonstrated in the study are compelling, particularly that we are seeing more than 60% of the study patients reporting greater than 50% pain relief at one year. We look forward to discussing the results with the attending physicians, and we plan to leverage the study results in continuing to drive our commercial business in Germany and more broadly in Europe.”

“The data from the ReActiv8-B clinical study through one year, and the favourable safety profile demonstrate that ReActiv8 is a viable restorative treatment option for patients with chronic low back pain,” said Dr. Franke. “ReActiv8 has the potential to provide long-term pain relief to this patient population, which suffers from a lack of available treatment alternatives.”

The ReActiv8 B clinical study is an international, multi-center, prospective, randomized, active-controlled, blinded trial with one-way cross-over, conducted under an Investigational Device Exemption (IDE) from the U.S. Food & Drug Administration (FDA). A total of 204 patients were implanted with ReActiv8 at leading study centers in the U.S., Europe and Australia and randomized 1:1 to therapy or control 14 days after implant. The initial results from this clinical study were announced by Mainstay on November 19, 2018.

“The results from this study confirm the results we have seen in our own experience with chronic mechanical low back pain patients with ReActiv8,” said Dr. Schilling. “There is an urgent need for new, effective therapies to treat long-term, mechanical low back pain, and the data from the studies demonstrate that ReActiv8 can provide sustained pain reduction over time.”

Mainstay will hold a ReActiv8-B symposium at which the physician faculty will make their presentations. The symposium will take place on Friday, December 7 from 13:00 - 14:30 in Studio 1.2 A & B at the Rhein Main Congress Center. Seating is limited. Pre-registration is available through www.reactiv8-B.de.

The Company will also have a booth at the DWG Congress, location number 26, and Mainstay leadership and the symposium faculty will be available for further discussions throughout the conference.

About Mainstay

Mainstay is a medical device company focused on commercializing an innovative implantable restorative neurostimulation system, ReActiv8®, for people with disabling Chronic Low Back Pain (CLBP). The Company is headquartered in Dublin, Ireland. It has subsidiaries operating in Ireland, the United States, Australia, Germany and the Netherlands, and is listed on the regulated market of Euronext Paris (MSTY.PA) and the ESM of Euronext Dublin (MSTY.IE).

About the ReActiv8-B Study

The ReActiv8-B Study is an international, multi-center, prospective, randomized, sham-controlled, blinded trial with one-way crossover conducted under an Investigational Device Exemption (IDE). In summary, this means that eligible patients had baseline data collected and then following verification that the enrollment criteria were met, ReActiv8 was implanted. At the 14-day post implant follow up visit, half the patients were randomized to receive appropriately programmed stimulation (the treatment arm), and half were randomized to receive sham stimulation/low stimulation (the control arm). Information about the study can be found at https://clinicaltrials.gov/ct2/show/study/NCT02577354.

About Chronic Low Back Pain

One of the recognized root causes of CLBP is impaired control by the nervous system of the muscles that dynamically stabilize the spine in the low back, and an unstable spine can lead to back pain. ReActiv8 is designed to electrically stimulate the nerves responsible for contracting these muscles and thereby help to restore muscle control and improve dynamic spine stability, allowing the body to recover from CLBP.

People with CLBP usually have a greatly reduced quality of life and score significantly higher on scales for pain, disability, depression, anxiety and sleep disorders. Their pain and disability can persist despite the best available medical treatments, and only a small percentage of cases result from an identified pathological condition or anatomical defect that may be correctable with spine surgery. Their ability to work or be productive is seriously affected by the condition, and the resulting days lost from work, disability benefits and health resource utilization put a significant burden on individuals, families, communities, industry and governments.

Further information can be found at www.mainstay-medical.com

CAUTION – in the United States, ReActiv8 is limited by federal law to investigational use only.

Appointment of Dr Thomas Jung as Chief Medical Officer of Inflazome

  • Industry veteran clinician to lead the clinical development of Inflazome’s pioneering NLRP3 inflammasome inhibitors to treat inflammatory diseases

  • Appointment follows Inflazome’s recent successful €40m Series B financing

  • The NLRP3 inflammasome is now associated with a broad range of serious medical conditions driven by harmful inflammation; from neurological disorders such as Alzheimer’s and Parkinson’s, to cardiovascular, liver, kidney and inflammatory bowel diseases.

Dublin, Ireland and Cambridge, UK : Inflazome (inflazome.com), the pioneering inflammasome company, announces the appointment of Dr Thomas Jung M.D., Ph.D., as its Chief Medical Officer.

Dr Jung joins Inflazome’s senior management team to lead the clinical development of its potent and selective small-molecule inhibitors of the NLRP3 inflammasome. Dr Jung will oversee the company's global operations related to the development of Inflazome’s lead and next-generation compounds across a range of therapeutic indications; and will work collaboratively with Dr Jeff Thompson, Inflazome’s Head of Clinical Operations and Joanna Tufnell, Head of Regulatory Affairs.

Dr Matt Cooper, Co-Founder and CEO of Inflazome, commented: “Dr Jung has a long and successful track record in guiding pioneering medicines to the market. He brings significant breadth and depth in clinical immunology to the team. We look forward to working together to drive our novel immunotherapies through clinical trials to benefit patients affected by harmful inflammation.”

On his appointment Dr Jung commented: “I am delighted to join the Inflazome team. I see huge potential in Inflazome’s novel small molecules and their ability to transform patients’ lives in a variety of clinical indications. I am looking forward to working with physicians, scientists and patients to develop these new drugs and to deliver on the promise to modify inflammation for patients’ benefit”.

Dr Thomas Jung

Dr Jung is a M.D., Ph.D. physician-scientist with extensive experience in Translational Sciences, Preclinical and Clinical Development of small molecules and biologics.

During a distinguished career at Novartis, Dr Jung was a member of the Novartis Institute of Biomedical Research and Novartis Pharma for 13 years. His primary focus of immunological research was the study of innovative anti-inflammatory principles. He led the Canakinumab (Illaris®) Team in the US and EU, from bench to first approval for CAPS (Cryopyrin-Associated Periodic Syndromes, a rare monogenetic disease). Dr Jung was also central to its expansion strategy into indications such as systemic juvenile idiopathic arthritis, gout and other rare fever syndromes. He made a significant contribution to the exploration of Canakinumab in cardiovascular diseases and served as the Development Head for Immunology products and, subsequently, as Head of the Translational Medicine Group for Novartis Europe. Thereafter, Dr Jung served as Chief Development Officer and Chief Medical Officer for two biotechnology companies in Switzerland: Auris Medical and Delenex. Dr Jung is a board-certified Dermatologist and holds an Associate Professor position for Dermatology at the University of Göttingen, Germany.

About Inflazome

Utilizing the scientific expertise of our founders and advisors, Inflazome is leading the way in developing orally available drugs to address clinical unmet needs in inflammatory diseases by targeting the NLRP3 inflammasome, which is now understood to drive many chronic and acute inflammatory conditions from Alzheimer’s and Parkinson’s to cardiovascular, liver, kidney and inflammatory bowel diseases.

Headquartered in Dublin, Ireland, Inflazome was founded in 2016 by leading academics Prof. Matt Cooper, The University of Queensland (Australia) and Prof. Luke O’Neill Trinity College Dublin (Ireland), following a highly productive joint collaboration.

To advance its programs to clinical development, Inflazome recently closed a Series B financing of €40M / US$46M led by Forbion Capital Partners with Longitude Capital and Series A investors, Fountain Healthcare Partners and Novartis Venture Fund, also participating.

To learn more visit: inflazome.com

Neuromod Appoints Leading Tinnitus Expert Professor Richard S. Tyler, PhD to its Clinical Advisory Board

Dublin, Ireland | Neuromod Devices Limited (“Neuromod”), an Irish medical technology company specialising in non-invasive neuromodulation technologies, is delighted to announce that Professor Richard S. Tyler has agreed to join Neuromod’s Clinical Advisory Board. A clinical expert in tinnitus and audiology, Professor Tyler is a renowned pioneer in the advancement of tinnitus research and treatment, founding the annual International Conference on Management of Tinnitus and Hyperacusis more than 26 years ago. In addition to his roles as Professor in the Department of Otolaryngology, Head & Neck Surgery and in the Department of Speech Pathology and Audiology at the University of Iowa, Professor Tyler continues to see and treat a large number of US tinnitus patients.

Professor Tyler’s appointment to the Clinical Advisory Board follows a number of other exciting appointments by the Company this year. In June, Mr. Christopher M. Smith, a global leader in the hearing and medical device industries agreed to join the Board of Directors and in January, Professor Hubert Lim, a world-renowned scientist and thought leader in auditory neuroscience was appointed as Chief Scientific Officer.

Commenting on the appointment, Dr. Ross O’Neill, CEO of Neuromod said: “We’re delighted to welcome Prof. Rich Tyler to Neuromod’s Clinical Advisory Board. Prof. Tyler is a world-renowned audiologist who has advanced our understanding of tinnitus and pioneered research into the development of treatments for tinnitus throughout his illustrious career. As a practising audiologist who continues to see and help a large number of tinnitus patients, Prof. Tyler’s insight and guidance will be invaluable as we prepare for the commercial roll out of our ground-breaking, home-use tinnitus treatment device.”

Prof. Tyler commented: “There are estimates of up to 30% of the population suffering from tinnitus and no one seems to want to help them. Many of these patients cannot sleep at night and take medications for anxiety and depression. I am excited to join Neuromod’s Clinical Advisory Board. Neuromod are working with leading tinnitus scientists and are taking an evidence-based approach to develop treatments for this large unmet clinical need. They have conducted some of the largest and most rigorous clinical trials to date in tinnitus. I am glad to play a part in helping them develop viable treatment options for the millions of patients living with this debilitating condition.”

About Professor Richard S. Tyler

Richard S. Tyler, Ph.D., M.Sc., was trained in Audiology and Psychoacoustics at the University of Western Ontario and the University of Iowa. He worked initially at the Institute of Hearing Research in the United Kingdom and is currently a Professor in both the Department of Otolaryngology – Head & Neck Surgery and in the Department of Speech Pathology and Audiology at the University of Iowa. Prof. Tyler has been a visiting scholar in China, South Africa, Australia, Sweden, Poland, Germany and France. He became interested in tinnitus early in his career while working with Professor Ross Coles. His scientific work includes the quantification of tinnitus, necessary for its investigation, as well as the investigation of different treatments. Prof Tyler has published over 270 articles and edited five books, including three books on tinnitus; the first textbook on tinnitus, a treatment protocol book for clinicians and a self-help book for patients.

About Neuromod Devices Limited

Neuromod, headquartered in the Digital Hub, Dublin, Ireland, is an emerging medical technology company specialising in the design and development of home-use, non-invasive neuromodulation technologies to address the clinical needs of underserved patient populations who live with chronic and debilitating conditions. The company was founded in 2010, by Dr. Ross O’Neill, as a spin-out from National University of Ireland, Maynooth. The lead application of Neuromod’s technology is in the field of tinnitus, where Neuromod has conducted extensive clinical trials to confirm the efficacy of its non-invasive neuromodulation platform in this common disorder for which no standard of care has yet been established. Tinnitus affects between 10 and 15% of the global population, and the lives of at least 1 in every 100 people worldwide are severely compromised because of the illusory sound that is often described as a ringing or buzzing in the ears.

www.NeuromodDevices.com

Inflazome Completes €40m ($46m) Series B Financing to Develop NLRP3 Inflammasome Inhibitors to Clinical Proof-of-Concept

  • Financing led by Forbion, with Longitude Capital and founding investors, Novartis Venture Fund and Fountain Healthcare Partners, also participating

  • Funding to advance clinical trials of Inflazome’s pioneering orally-available NLRP3 inflammasome inhibitor compounds in several inflammatory diseases

  • NLRP3 is associated with harmful inflammation in a broad range of serious medical conditions including Alzheimer’s, Parkinson’s, inflammatory bowel disease, gout, osteoarthritis, liver, kidney and cardiovascular diseases

Dublin, Ireland and Cambridge, U.K. : Inflazome, a pioneering inflammasome company developing small molecule drugs that block harmful inflammation, today announces the successful completion of a Series B financing round of €40 million ($46 million). The financing round was led by Forbion, with Longitude Capital and founding investors, Novartis Venture Fund and Fountain Healthcare Partners, also participating.

Clinical and scientific data indicates that the NLRP3 inflammasome, a compelling biological target that regulates our innate immune response, is overactive in a broad range of serious medical conditions driven by harmful inflammation. These include neurological disorders such as Alzheimer’s and Parkinson’s as well as inflammatory bowel disease, gout, osteoarthritis, liver, kidney and cardiovascular diseases; conditions often inadequately treated by current therapies.

Inflazome is developing potent and selective small-molecule inhibitors of the NLRP3 inflammasome, to stop the cycle of chronic inflammation that drives such diseases. The Series B proceeds will be used to advance the Company’s first-in-class NLRP3 inflammasome inhibitors into multiple clinical trials in 2019, with next-generation compounds following thereafter.

Following the completion of the financing round, Dr Marco Boorsma, a General Partner at Forbion, and Dr David Hirsch, a Managing Director and Founder of Longitude Capital, have joined Inflazome’s Board of Directors.

Dr Matt Cooper, Co-Founder and CEO of Inflazome, commented: “We are very pleased to have secured a partnership with leading EU and US investors Forbion and Longitude and additional support from our founding investors, Novartis Venture Fund and Fountain Healthcare Partners. Forbion and Longitude have excellent track records of working closely with biotechs to drive R&D programs to successful clinical outcomes.”

Dr Marco Boorsma, General Partner at Forbion Capital Partners, commented: “Recognizing the important role of the inflammasome in many major diseases, we intensively screened the universe of companies active in this space. We decided to back Inflazome because of their leading position, deep understanding of inflammasome biology, foundational IP position and advanced and diversified pipeline of NLRP3 modulators. We were also highly impressed with the experience and knowledge of the Company’s Management Team and Board.”

Dr David Hirsch, Managing Director and Founder of Longitude Capital, added: “We’re excited by the potential of NLRP3 inhibitors and look forward to generating robust human proof of concept data with this financing.”

Inflazome Chairman, Dr Manus Rogan and Co-Founder and Managing Partner of Fountain Healthcare Partners, commented: “Due to the breadth of therapeutic applications, the market potential for a successful small molecule inhibitor of NLRP3 is clearly very significant. It has been exciting to have been involved in the Inflazome story since its inception and to follow our investment in one of the largest European venture capital backed financing rounds in the biotech sector this year.”

About Inflazome

Utilizing the scientific expertise of our founders and advisors, Inflazome is leading the way in developing rally available drugs to address clinical unmet needs in inflammatory diseases by targeting the NLRP3 inflammasome, which is now understood to drive many chronic inflammatory conditions. Headquartered in Dublin, Ireland, Inflazome was founded in 2016 by leading academics Prof. Matt Cooper, The University of Queensland (Australia) and Professor Luke O’Neill, Trinity College Dublin (Ireland), following a highly productive joint collaboration. For more information, please visit: inflazome.com

Mainstay Medical Announces Headline Results from ReActiv8-B Clinical Study

  • Responder rates at 120 days for treatment and active control groups were 56% vs. 47%; statistically significant difference on primary endpoint not achieved

  • Responder rates in both groups rose steadily from 120 days to one year; preliminary data for the 116 patients that have completed the one-year assessment, including those from the cross-over group, show:

  • 72% of patients achieved a 30% or greater reduction from baseline in low back pain VAS without increase in pain medications

  • 60% of patients achieved a 50% or greater reduction from baseline in low back pain VAS without increase in pain medications

  • 44% of the 50 patients that have completed the one-year assessment and were on opioids at baseline had voluntarily eliminated (28%) or significantly reduced (16%) their use of opioids

  • Incidence and type of adverse events (AEs) similar to AEs in clinical trials reported for other neurostimulation devices

Dublin, Ireland– Mainstay Medical International plc (“Mainstay” or the “Company”, Euronext Paris: MSTY.PA and Euronext Dublin: MSTY.IE), a medical device company focused on commercializing ReActiv8®, an implantable restorative neurostimulation system designed to treat an underlying cause of disabling Chronic Low Back Pain, today announces headline results from its ReActiv8-B study.

The ReActiv8 B clinical study is an international, multi-center, prospective, randomized, active-controlled, blinded trial with one-way cross-over, conducted under an Investigational Device Exemption (IDE) from the U.S. Food & Drug Administration (FDA). A total of 204 patients were implanted with ReActiv8 at leading study centers in the U.S., Europe and Australia and randomized 1:1 to therapy or control 14 days after implant. In the treatment group, the ReActiv8 pulse generator was programmed to deliver electrical stimulation expected to elicit contractions of the multifidus muscle. In the control group, the ReActiv8 device was programmed to provide a low level of electrical stimulation. Following assessment of the primary endpoint at 120 days, patients in the control group crossed-over to receive levels of electrical stimulation as in the treatment group.

The patients in the study had an average age of 46, and an average duration of chronic low back pain of 14 years. This patient population has tried many other treatment alternatives with limited success, and 80% of the patients were on pain medication at baseline.

Efficacy Outcomes

The primary efficacy endpoint of the study was a comparison of responder rates between the treatment and control groups as measured on the visual analog scale (VAS) of pain, with responders defined as having a 30% or greater improvement on this measure between baseline and 120 days after randomization, without any increase in pain medication taken in the two weeks prior to the primary endpoint assessment visit. In the treatment group the responder rate at 120 days was 56%, compared to 47% in the control group, resulting in a difference that is not statistically significant.

ReActiv8-B is the first ever active sham-controlled clinical trial of an implantable neurostimulator for chronic low back pain. The literature from other sham or placebo-controlled studies, involving drugs, device implants or other interventions, suggested that a responder rate of 20% or more could have been expected in the control group. The study design assumed a 25% responder rate in the control group.

“Our study involved surgically implanting a device into patients who had not previously received surgical implants, and further required the patient to activate the device twice daily to administer the therapy,” said Jason Hannon, Chief Executive Officer of Mainstay. “The study design underestimated the amount and duration of the sham effect under these conditions.”

“The overall results of this study, however, are a solid endorsement of the efficacy and safety of ReActiv8,” continued Mr. Hannon. “We saw very high responder rates in the patients in the treatment group that have reached one year of therapy, and substantial improvement in the patients that were crossed-over to treatment from the control group after 120 days. We believe these long-term results represent the most important clinical factors to physicians, and we plan to leverage these results in continuing to drive our commercial business under our existing CE Mark in Europe.”

Responder Rates Grow to 1 Year

Improvements in the percentage of patients reporting pain reduction continued beyond the 120-day assessment through one year for both groups. The percentage of the 56 patients in the treatment group that have completed the one-year assessment having a 30% or greater reduction in low back pain VAS at that assessment without an increase in pain medication was 75%. The percentage of the 60 patients in the cross-over group that have completed the one-year assessment having a 30% or greater reduction in low back pain VAS at that assessment without an increase in pain medication was 68%. The percentage of the 116 patients in the both groups that have completed the one-year assessment having a 50% or greater reduction in low back pain VAS at one year without an increase in pain medication was 60%. Substantial improvements in disability as measured by the Oswestry Disability Index (ODI) were also observed at one year. These data are subject to change as the remaining patients reach the one-year assessment.

“The totality of this data is encouraging for this large group of patients with limited treatment alternatives,” said Dr. Chris Gilligan, Chief, Division of Pain Medicine, Department of Anesthesiology, Perioperative and Pain Medicine, Brigham & Women’s Hospital, Assistant Professor of Anaesthesia, Harvard Medical School, and Principal Investigator for the study. “The data on the patients that have completed one year of therapy are important, particularly because these patients have not experienced meaningful pain relief from existing treatment options. The data showing 60% of patients experiencing a 50% or greater pain reduction at one year surpasses what many implanting physicians expect from implantable neurostimulation devices.”

Results Demonstrate Voluntary Decrease in Back Pain Medication Use

The protocol permitted patients to adjust their back pain medication usage after the 120-day assessment point. At one year, 44% of the 50 patients who were on opioids at baseline had voluntarily eliminated (28%) or significantly reduced (16%) their use of opioids. These results are subject to change as the remaining patients reach the one-year assessment.

Secondary Endpoints

For secondary endpoints, such as disability measured by the ODI and quality of life measured by the EQ-5D quality of life instrument, numerical improvements in the treatment group as compared to the control group were observed at 120 days. The Company is evaluating the clinical significance of those findings. The Company continues to analyze the efficacy data for the purpose of better understanding the performance of the device.

Device-Related Adverse Events in Line With Other Studies

ReActiv8 is implanted in a minimally invasive procedure utilizing techniques commonly used by our physician customer base in other procedures. The incidence and type of adverse events (AEs), including serious AEs, were comparable to AEs in clinical trials reported for other neurostimulation devices, with no unanticipated AEs related to the device, procedure or stimulation.

“The study showed a comparable safety profile to other implantable neurostimulation devices, including no migration of leads,” said Dr. Richard Rauck, President and Founder, Carolinas Pain Institute, Medical Director for The Center for Clinical Research, Pain Fellowship Director at Wake Forest University School of Medicine and Chairman of the DMC. “This safety profile, combined with the efficacy data from this study and the lack of available alternatives for this patient population, suggest that ReActiv8 can be a promising therapy for patients with chronic low back pain.”

PMA Plan

“In summary, we believe the evidence of effectiveness and the favorable safety profile of ReActiv8 show considerable promise, particularly when compared to the response rates and safety profiles of alternative treatments,” continued Mr. Hannon. “We believe that the totality of the data will support the submission of a PMA application for ReActiv8 to the FDA. We plan to seek a pre-PMA meeting with the FDA in the coming months to obtain guidance on our filing content and strategy.”

Investor Conference Call & Webcast

Jason Hannon, Chief Executive Officer, will host a conference call and webcast with Q&A for analysts and investors to discuss the results from the study at 8:00am GMT (9:00am CET) on 19 November 2018. The call will be conducted in English. The webcast will be made available on Mainstay’s website at www.mainstay-medical.com on the News & Events tab. The presentation and webcast will be available on the Mainstay Medical website one hour before the call, and a replay will be available for 30 days.

About Mainstay

Mainstay is a medical device company focused on commercializing an innovative implantable restorative neurostimulation system, ReActiv8®, for people with disabling Chronic Low Back Pain (CLBP). The Company is headquartered in Dublin, Ireland. It has subsidiaries operating in Ireland, the United States, Australia, Germany and the Netherlands, and is listed on the regulated market of Euronext Paris (MSTY.PA) and the ESM of Euronext Dublin (MSTY.IE).

About the ReActiv8-B Study

The ReActiv8-B Study is an international, multi-center, prospective, randomized, sham-controlled, blinded trial with one-way crossover conducted under an Investigational Device Exemption (IDE). In summary, this means that eligible patients had baseline data collected and then following verification that the enrollment criteria were met, ReActiv8 was implanted. At the 14-day post implant follow up visit, half the patients were randomized to receive appropriately programmed stimulation (the treatment arm), and half were randomized to receive sham stimulation/low stimulation (the control arm). Information about the study can be found at https://clinicaltrials.gov/ct2/show/study/NCT02577354.

About Chronic Low Back Pain

One of the recognized root causes of CLBP is impaired control by the nervous system of the muscles that dynamically stabilize the spine in the low back, and an unstable spine can lead to back pain. ReActiv8 is designed to electrically stimulate the nerves responsible for contracting these muscles and thereby help to restore muscle control and improve dynamic spine stability, allowing the body to recover from CLBP.

People with CLBP usually have a greatly reduced quality of life and score significantly higher on scales for pain, disability, depression, anxiety and sleep disorders. Their pain and disability can persist despite the best available medical treatments, and only a small percentage of cases result from an identified pathological condition or anatomical defect that may be correctable with spine surgery. Their ability to work or be productive is seriously affected by the condition, and the resulting days lost from work, disability benefits and health resource utilization put a significant burden on individuals, families, communities, industry and governments.

Further information can be found at www.mainstay-medical.com

Vivasure Medical Announces Launch of the PerQseal® Large-Bore Closure Device

The world’s first fully absorbable, patch-based large-bore percutaneous closure device is now available to European physicians and patients

GALWAY, Ireland - Vivasure Medical®, a company pioneering novel fully absorbable technology for percutaneous vessel closure, today announced the European launch of the PerQseal® closure device for large-bore transcatheter procedures.

Novel transcatheter endovascular procedures, including transcatheter aortic valve replacement (TAVR), thoracic endovascular aneurysm repair (TEVAR), and endovascular abdominal aneurysm repair (EVAR), require large-bore femoral artery access. Closure of these large-bore access sites is challenging and has been associated with significant vascular and bleeding complications.

PerQseal is the first sutureless, fully absorbable synthetic implant for large-bore arterial punctures. The PerQseal technology consists of an intravascular patch that seals the vessel from the inside, returning the artery to its natural state.

“Closing the artery has been a concern since we started using transcatheter techniques for valve implantation,” says Prof. Horst Sievert, of the CardioVascular Center in Frankfurt, Germany. “The PerQseal device is a very innovative solution for closing large holes, and we are enthusiastic to make it part of our armamentarium.”

“We’re excited that the PerQseal device is now widely available to European physicians, providing a safe and simple option for the closure of large puncture holes that leaves behind no sutures, metal implants, or collagen,” said Gerard Brett, co-founder and CEO of Vivasure Medical. “This commercial launch is a key milestone for Vivasure as we journey to enable better patient outcomes in fully percutaneous large-bore endovascular procedures.”

“In my first clinical experience with PerQseal, I found the device intuitive and well controlled, which helped me quickly learn how to use the technology safely and successfully,” said Dr. Saib Khogali, Heart & Lung Centre, New Cross Hospital Wolverhampton, United Kingdom. “I believe the PerQseal has the potential to be an important large-hole closure device in many TAVR and EVAR patients.”

About Vivasure Medical

Based in Galway, Ireland, Vivasure is focused on the development of advanced polymer implants and delivery systems, primarily focused on minimally invasive vessel closure in cardiology, interventional radiology and vascular surgery. Vivasure operates a fully integrated R&D and ISO 13485 certified manufacturing facility and is backed by leading international medtech investors. For more information, please visit www.vivasuremedical.com.

The PerQseal® is not available for sale in the United States.

Contacts

Vivasure Medical

Sierra Smith, 408-540-4296

sierra@healthandcommerce.com

Inflazome: Validation of a new target in Parkinson’s Disease

Innovative research was funded by The Michael J. Fox Foundation for Parkinson’s Research and the Shake It Up Australia Foundation 

Dublin (IE), Cambridge (UK): Inflazome Ltd, (“Inflazome”) is a pioneering biotech company developing small molecule drugs that stop harmful inflammation by targeting inflammasomes. Research leading towards a potential approach to treat Parkinson’s patients was published today in Science Translational Medicine. The study was co-authored by Inflazome Co-founder and Chief Executive Officer, Prof. Matt Cooper with research teams at The University of Queensland, Australia, led by A/Prof. Trent Woodruff. Funding for these studies was provided by The Michael J. Fox Foundation for Parkinson’s Research and the Shake It Up Australia Foundation.

Parkinson’s disease is the second most common neurodegenerative disorder worldwide, affecting more than 10 million people. It is characterised by the loss of dopamine-producing neurons, accompanied by chronic inflammation in the brain. Inflazome has identified drugs to stop the chronic cycle of inflammation in the brain. The researchers found that the tool compound MCC950, a potent inhibitor of the NLRP3 inflammasome, given orally once a day could stop neuroinflammation. MCC950 arrested the effects of Parkinson’s in several animal models of the disease, leading to reduced brain neuron loss and higher levels of dopamine.

NLRP3 inflammasome activation drives chronic inflammation and is implicated in many diseases - from Parkinson’s and Alzheimer’s to Asthma, Inflammatory Bowel Disease, Chronic Kidney Disease, Cardiovascular Disease, Arthritis and the liver disease, NASH. Inflazome is developing orally available drugs to address clinical unmet needs in these inflammatory diseases by blocking the activation of inflammasomes in our immune system.

Prof. Matt Cooper, Co-founder and CEO of Inflazome, commented: “We are indebted to The Michael J. Fox and Shake It Up Australia Foundations for supporting this work. We also thank those generous individuals affected by Parkinson’s who donated samples to aid medical research. Drug companies have tried for decades to develop medicines to combat neurodegenerative diseases by blocking the many different neurotoxins and amyloids that build up in the brain as we age.”

“The problem is that if one toxin is blocked by a drug, another may still build up and cause disease. In contrast, our line of research focuses not on individual toxins, but instead on the immune cells in our brains that clear amyloid and other neurotoxins. These cells, called microglia, normally protect us from infections that can lead to encephalitis and meningitis. However, as we age our immune system can become over-activated, which leads to neuroinflammation. Over-active microglia can no longer function as efficient ‘cleaners’ of neurotoxins, but instead contribute to long-term damage in the brain.”

The progression of Parkinson’s disease was studied using brain and blood samples donated by patients for medical research. The study demonstrated a key target in microglia, called the NLRP3 inflammasome, is highly activated in Parkinson’s. The same signals produced by inflammasome activation in human brains were seen in different pre-clinical models of Parkinson’s disease. A small molecule, MCC950, prevented the progression of Parkinson’s in these animal models, leading to reduced brain cell loss, and higher levels of dopamine and motor function to effectively ‘cool the brain on fire’.

Prof. Cooper added “We look forward to progressing this research through to clinical trials using our proprietary, improved drug candidates”.

Kuldip Dave, PhD, Director of Research Programs at The Michael J. Fox Foundation said, “Inflazome has validated a promising new target for Parkinson’s therapeutics and translated that finding into a potential drug to treat Parkinson’s disease. This is a key aspect of The Michael J. Fox Foundation’s research strategy, and we look forward to their candidate drug’s continued development.”

“Shake It Up is proud and excited to be a part of the collaboration’s funding team, allowing highly-talented researchers an opportunity to create world leading breakthroughs that have the opportunity to be a game changer for people with Parkinson’s,” said Clyde Campbell, Founder and CEO of the Shake It Up Foundation.

The Science Translational Medicine paper can be found here

A link to an explainer video can be found here

About Inflazome

Utilizing the scientific expertise of our founders and advisors, Inflazome is leading the way in developing orally available drugs to address clinical unmet needs in inflammatory diseases by targeting the NLRP3 inflammasome, which is now understood to drive many chronic and acute inflammatory conditions from Parkinson’s and Alzheimer’s to Cardiovascular Diseases.

Headquartered in Dublin, Ireland, Inflazome was founded in 2016 by leading academics Prof. Matt Cooper, The University of Queensland (Australia) and Prof. Luke O’Neill, Trinity College Dublin (Ireland), following a highly productive joint collaboration.

To learn more visit: inflazome.com

Contacts

Inflazome:

Dr Jeremy Skillington | VP Business Development

E: j.skillington@inflazome.com

Media:

Jonathan Neilan | FTI Consulting

E: jonathan.neilan@fticonsulting.com

Paddy Berkery | FTI Consulting

E: patrick.berkery@fticonsulting.com


Opsona Therapeutics Ltd. to present results on Tomaralimab study at the 60th Annual Meeting of the American Society of Hematology (ASH)

Opsona Therapeutics Ltd (‘Opsona’), today announces that it will give an oral presentation on results from its ongoing prospective, open label Phase I/II study being conducted with Tomaralimab (OPN-305), its novel proprietary humanized IgG4 monoclonal antibody (MAb) against Toll-Like Receptor 2 (TLR2), in second and third-line lower (Low and intermediate-1) risk myelodysplastic syndrome (MDS). The presentation will take place on Dec 03rd 2018 at the 60th Annual Meeting of the American Society of Hematology (ASH) in San Diego.

The study in transfusion-dependent patients with lower risk MDS who have failed hypomethylating agents is ongoing in collaboration with the MD Anderson Cancer Center in Houston, Moffitt Cancer Center in Florida, Weill Cornell in New York and Montefiore in the Bronx USA.

The lead principal investigator Professor Guillermo Garcia-Manero will present the data at ASH and commenting on today’s announcement said “Tomaralimab therapy presents a potential safe and efficacious therapeutic option for heavily pre-treated low risk patients that have failed HMA therapy.”

Myelodysplastic syndromes are a complex and heterogeneous group of bone marrow failure disorders characterized by ineffective hematopoiesis and poor prognosis. Transfusion dependent patients who have failed on HMA have a worse prognosis in terms of survival. The best standard of care for patients in the USA who have failed on HMA is repeated red blood cell transfusions which are associated with reduced Quality of Life outcomes.

There is an urgent need for the development of novel therapies in the treatment of MDS in patients who have exhausted other therapies and which can eliminate the need for red blood cell transfusions, delay progression, improve patient survival and overall quality of life.

Details of the presentation are as follows:

TITLE: A Clinical Study of Tomaralimab (OPN-305), a Toll-like Receptor 2 (TLR-2) Antibody, in Heavily Pre-Treated Transfusion Dependent Patients with Lower Risk Myelodysplastic Syndromes (MDS) That Have Received and Failed on Prior Hypomethylating Agent (HMA) Therapy

Session Name: 637. Myelodysplastic Syndromes—Clinical Studies: Prognosis and Prediction

Session Date: Monday, December 3, 2018

Session Time: 2:45 PM - 4:15 PM

Presentation Time: 4:00 PM

Room: Manchester Grand Hyatt San Diego, Grand Hall A

First publication of the abstract will be in the ASH online meeting program on November 1, 2018, at 9 a.m. EDT.

About Opsona Therapeutics

Opsona is a leading immunology drug development company, focused on novel therapeutic approaches to key targets of the innate immune system associated with a wide range of major human diseases, including cancer, autoimmune and other inflammatory diseases. The company was founded in 2004 by three world-renowned immunologists at Trinity College, Dublin. Opsona has a strong international investor consortium including: Amgen Ventures, BB Biotech Ventures, EMBL Ventures, Enterprise Ireland, Fountain Healthcare Partners, Inventages Venture Capital, Novartis Venture Fund, Omnes Capital, Roche Venture Fund, Seroba Life Sciences, Shire and Sunstone Capital.

Contacts

Opsona Therapeutics

Mary Reilly (COO)

+ 353 16770223

mreilly@opsona.com

Velicept Therapeutics Raises an Additional $15 Million in Series B Round

  • Additional Funding to Advance Phase 2b Development Program in OAB-

  • Enrollment for Second Phase 2 Study Well Underway-

  • Issuance of U.S. Patent No. 10,065,922 Further Expands Velicept’s Portfolio Covering Solabegron

MALVERN, Pa. - Velicept Therapeutics, a privately-held clinical stage pharmaceutical company dedicated to the development of best-in-class compounds for the treatment of urological and gastrointestinal disorders, announced today it has closed on the expansion of a Series B financing bringing an additional $15 million to support completion of the two ongoing Phase 2b studies, as well as Phase 3 enabling work. The financing led by Samsara BioCapital, included existing investors CDK Associates, LLC; Fountain Healthcare Partners Fund II, L.P.; Longitude Venture Partners II, L.P; and founding investor Becker Ventures. Cory Freedland, a Principal with Samsara BioCapital, has joined the Company’s Board of Directors.

“We are pleased with our progress in advancing the development of solabegron. This financing provides us the capital necessary to complete the robust Phase 2 program and ready the program to enter Phase 3,” said Dr. James Walker, President and Chief Executive Officer, Velicept Therapeutics. “These are important steps as we continue to execute on our plan to deliver an improved next generation therapy to women and men suffering from overactive bladder.”

Velicept also announced that VEL2001, the second of two Phase 2b clinical studies designed to evaluate two doses of a novel once daily time-dependent release formulation of solabegron in patients with overactive bladder (OAB), was initiated in July with enrollment progressing to plan. The primary objective of the study is to evaluate the mean change in number of micturitions per day as measured by patients in an e-diary. The formulation was engineered to optimize efficacy in a convenient once daily dose. Together with the BID study currently underway (VEL2002), the comprehensive Phase 2 program will explore the full dose range for solabegron with the goal of delivering best in class efficacy in the treatment of OAB.

The multicenter, randomized, double-blind, vehicle-controlled Phase 2b study will enroll 375 adult women ages 18 to 80 with signs and symptoms of overactive bladder from 90 centers across North America. Patients will receive either low or high dose of solabegron once daily or placebo for 12 weeks.

Additionally, the United States Patent and Trademark Office (USPTO) has issued U.S. Patent No. 10,065,922 with 126 claims, directed to solid solabegron zwitterion. The patent will expire February 23, 2037, inclusive of patent term adjustment. This issued U.S. Patent is part of an intellectual property portfolio, which includes other issued patents, and numerous patent applications in the U.S. and major international markets directed to solabegron for the treatment of OAB.

About Solabegron

Solabegron is a highly potent and selective beta-3 adrenoceptor agonist being investigated for overactive bladder (OAB) and irritable bowel syndrome (IBS). A Phase 2 study of solabegron in OAB, previously conducted by GlaxoSmithKline, evaluated a twice-daily dose in 258 patients with moderate to severe incontinence experiencing an average of 4.5 wet episodes per day. Results demonstrated a statistically significant improvement with solabegron as compared to placebo. Furthermore, the Phase 2 study also indicated a safety and tolerability profile for solabegron that was similar to placebo.

About Velicept

Velicept Therapeutics, Inc. is a privately held, clinical stage pharmaceutical company focused on advancing best-in-class compounds for the treatment of urological and gastrointestinal disorders. Its lead product, solabegron, is a highly potent and selective beta-3 adrenoceptor agonist being investigated for overactive bladder (OAB) and irritable bowel syndrome (IBS). For more information, visit www.velicept.com.

Contacts

Media:

Sam Brown, Inc.

Mike Beyer, 312-961-2502

mikebeyer@sambrown.com

Syndesi Therapeutics announces appointment of Roy Twyman MD to its Board of Directors

Syndesi Therapeutics SA, a biotech company developing novel treatments for cognitive impairment, today announced the appointment of Dr Roy Twyman to its Board as an independent non-executive Director.

Dr Twyman spent over 19 years at Janssen Research & Development (part of the Janssen Pharmaceutical Companies of Johnson & Johnson) in CNS development, holding roles of increasing responsibility. Most recently, Dr Twyman was Senior Vice President in the Neuroscience Therapeutic Area at Janssen. In this role, he had responsibility to oversee the Alzheimer’s Disease Area across all stages of R&D, including oversight of Neuroscience Experimental Medicine group covering clinical development from first time in human to proof of concept. Prior to joining Janssen in 1998, Dr Twyman held professorships in neurology at University of Michigan and University of Utah and gained his MD from University of Kentucky.

“I am delighted to welcome Roy onto Syndesi’s Board,” commented Jean Combalbert, Chair of the Board. “The fact that Syndesi has convinced an acknowledged world leader in CNS such as Roy to join the board reflects the promise of the pro-cognitive SV2A modulators for the treatment of Alzheimer's disease and other conditions. Roy’s contribution will definitively be a major asset for the company.”

Jonathan Savidge, CEO of Syndesi said “With his tremendous experience in CNS drug development, Roy brings complimentary expertise to the Board and his appointment is a major value add for the company as we prepare for Phase I entry with our lead molecule SDI-118, a small molecule modulator of SV2A that acts in a distinct way to the approved anti-epileptic compounds acting on this target.”

Commenting on his appointment, Dr Twyman noted “There is a need to explore new mechanisms that could help alleviate symptoms of cognitive impairment in dementia and other neuropsychiatric disorders. Modulation of SV2A, an important protein involved in synaptic communication between neurons, has been shown to be a successful therapeutic strategy in epilepsy. Syndesi are developing an intriguing lead molecule that modulates SV2A in a distinct way. By directly targeting synaptic dysfunction, Syndesi’s research and development effort represents a promising approach for the treatment of cognitive impairment in conditions such as Alzheimer’s Disease.”

About Syndesi Therapeutics

Syndesi Therapeutics was established to develop a series of novel, pro-cognitive small molecule SV2A modulators licensed from UCB. Syndesi is investigating the potential of these molecule to improve cognition in diseases such as Alzheimer’s Disease, other dementias and cognitive impairment associated with schizophrenia. The lead molecule, SDI-118 is anticipated to commence first in human Phase I studies in H1 2019. Syndesi announced a Series A financing round of €17M in February 2018, led by Novo Seeds and Fountain Healthcare with participation from Johnson & Johnson Innovation – JJDC, SRIW (Société Régionale d’Investissement de Wallonie), V-Bio Ventures and Vives Fund. Syndesi has been established at the Centre d’Entreprises et d’Innovation (CEI) in Louvain-la-Neuve, Belgium and is a resident company at Johnson & Johnson Innovation, JLABS (JLABS @ BE) in Beerse, Belgium.

For more information please visit www.syndesitherapeutics.com

KaNDy Therapeutics presents new compelling data with lead candidate NT-814 at The North American Menopause Society 2018 Annual Meeting

~ Treatment with NT-814 resulted in immediate improvements in bothersome post-menopausal symptoms~

~ Phase Ib/IIa data showed rapid and significant reductions in the frequency and severity of hot flashes and the number of night time awakenings~

STEVENAGE, England -- KaNDy Therapeutics, a UK clinical-stage biotech company, announces the presentation of new clinical data from the Phase Ib/IIa proof of concept clinical trial with its lead non-hormonal drug candidate, NT-814, at The North American Menopause Society 2018 Annual Meeting taking place from 3-6 October 2018 in San Diego, US.

Data from the Phase Ib/IIa RELENT-1 study, presented by Dr Steve Pawsey, Chief Medical Officer at KaNDy Therapeutics, demonstrate compelling evidence that NT-814, a novel, once-daily, oral neurokinin-1,3 receptor antagonist, being developed to provide a safe and effective alternative to hormone replacement therapy (HRT), produces a rapid and profound reduction in debilitating symptoms of the menopause including vasomotor symptoms (VMS), also known as hot flush or flash (HF), and night time awakenings.

The effects of treatment with NT-814 were immediate in onset, being both statistically significant and clinically relevant as soon as the first day of treatment. In contrast, studies with HRT have shown onset is within weeks or months.

The Phase Ib/IIa data also demonstrated that treatment with NT-814, taken once daily for two weeks at the most effective dose evaluated, resulted in rapid and significant reductions in:

  • the frequency of HF, with a reduction of 62% from baseline in the number of moderate and severe HF vs 24% for placebo in Week 1 (p<0.0014) and an 84% reduction from baseline vs 37% for placebo in week 2 (p<0.0002)

  • the severity of HF, with a reduction of 23% from baseline in average HF severity vs 9% for placebo in week 1 (p<0.015) and a 50% reduction from baseline vs 16% for placebo in Week 2 (p<0.0004)

  • the number of night time awakenings, with a reduction of 58% from baseline vs 17% for placebo in Week 1 (p< 0.0022) and an 81% reduction from baseline vs 32% for placebo in Week 2 (p< 0.0005).

NT-814 was found to be well-tolerated and no safety concerns were identified in the study.

Commenting on the data, Dr Steve Pawsey, Chief Medical Officer at KaNDy Therapeutics said: “These data presented today provide very compelling evidence to support the use of NT-814 to treat bothersome symptoms of the menopause. The fact that the effects of treatment are seen from the first day is extremely promising and positions NT-814 as a real alternative to HRT which can take weeks or months to be fully effective. We are looking forward to progressing this candidate through clinical development, in order to make this treatment commercially available as soon as possible.”

Dr Hadine Joffe, Paula A. Johnson Associate Professor of Psychiatry in the Field of Women’s Health, Harvard Medical School, Executive Director of the Connors Center for Women’s Health, Brigham and Women's Hospital, and Scientific and Clinical Advisor to KaNDy Therapeutics added: “The results generated thus far are indeed promising. HRT is effective and has a number of safety concerns and is slow in onset, so there is a real need for a safe, non-hormonal treatment option which acts quickly to relieve symptoms. This study has shown that NT-814 not only results in rapid and significant reductions in both hot flashes and night-time awakenings, but also that the effects of treatment are immediate. I believe NT-814 could be a potentially breakthrough non-hormonal option for the millions of women who suffer with debilitating symptoms related to menopause.”

Based on these results, KaNDy Therapeutics intends to advance NT-814 into a fully powered Phase IIb definitive dose-ranging study in patients suffering from debilitating symptoms of the menopause. The Phase IIb trial is expected to begin later this year.

The full abstract can be viewed on The NAMS website: https://www.menopause.org/annual-meetings/2018-meeting/meeting-abstracts

About the RELENT-1 Study: The Phase Ib/IIa RELENT-1 study was a randomised, double-blind, placebo-controlled study conducted at three clinical pharmacology units (CPU) in the US. Seventy-six women aged 41 to 64 years experiencing 7-20 moderate or severe HFs per day were recruited into the study and randomized to receive one of four escalating doses of NT-814 or placebo in four cohorts. Study drug was taken once daily in the morning for 14 days, the first seven of which were resident in the CPU. Subjects completed diaries twice daily for the two weeks before and throughout treatment and underwent routine safety assessments periodically throughout the trial. Further information on the study design can be found on www.clinicaltrials.gov and full results of the study will be published at scientific congresses and in peer-reviewed journals over the coming months.

NT-814 is an orally administered, potent and selective small molecule dual antagonist of both the neurokinin-1 and 3 receptors under development by KaNDy as a therapy for a range of Women’s Health conditions. NT-814 addresses VMS by modulating a group of oestrogen sensitive neurones in the hypothalamus in the brain (the KNDy neurones), that, because of the absence of oestrogen in menopausal women, become hyperactive and consequently disrupt body heat control mechanisms resulting in the debilitating vasomotor symptoms of HF.

KaNDy Therapeutics is a clinical-stage company focused on optimizing the potential of NT-814 in the treatment of common, chronic debilitating female sex-hormone related conditions. These conditions, such as post-menopausal VMS, are debilitating for women often over many years and associated with significant healthcare and economic costs.

Contacts

KaNDy Therapeutics

info@kandytherapeutics.com

or

Consilium Strategic Communications

Mary-Jane Elliott/ Lindsey Neville/ Carina Jurs

Tel: +44 (0) 20 3709 5700

KaNDyTherapeutics@consilium-comms.com

REDUCE-IT Cardiovascular Outcomes Study of Vascepa® (icosapent ethyl) Capsules Met Primary Endpoint

REDUCE-IT Is First Outcomes Study to Assess Treatment of Patients with LDL-C Controlled by Statin Therapy, Persistent Elevated Triglycerides and Other Cardiovascular Risk Factors

Results Specific to Pure EPA Vascepa at 4 Grams Daily

BEDMINSTER, N.J. and DUBLIN, Ireland -- Amarin Corporation plc (NASDAQ:AMRN), announced today topline results from the Vascepa® cardiovascular (CV) outcomes trial, REDUCE-ITTM, a global study of 8,179 statin-treated adults with elevated CV risk. REDUCE-IT met its primary endpoint demonstrating an approximately 25% relative risk reduction, to a high degree of statistical significance (p<0.001), in major adverse CV events (MACE) in the intent-to-treat patient population with use of Vascepa 4 grams/day as compared to placebo.

Patients enrolled in REDUCE-IT had LDL-C between 41-100 mg/dL (median baseline LDL-C 75 mg/dL) controlled by statin therapy and various cardiovascular risk factors including persistent elevated triglycerides (TGs) between 150-499 mg/dL (median baseline 216 mg/dL) and either established cardiovascular disease (secondary prevention cohort) or diabetes mellitus and at least one other CV risk factor (primary prevention cohort).

Key topline results include:

  • Efficacy: Approximately 25% relative risk reduction, demonstrated to a high degree of statistical significance (p<0.001), in the primary endpoint composite of the first occurrence of MACE, including cardiovascular death, nonfatal myocardial infarction (MI), nonfatal stroke, coronary revascularization, or unstable angina requiring hospitalization. This result was supported by robust demonstrations of efficacy across multiple secondary endpoints.

  • Safety: Vascepa was well tolerated with a safety profile consistent with clinical experience associated with omega-3 fatty acids and current FDA-approved labeling. The proportions of patients experiencing adverse events and serious adverse events in REDUCE-IT were similar between the active and placebo treatment groups. Median follow-up time in REDUCE-IT was 4.9 years.

Amarin is eager to share REDUCE-IT data in greater detail with both the medical community and regulatory authorities. REDUCE-IT results have been accepted for presentation at the 2018 Scientific Sessions of the American Heart Association (AHA) on November 10, 2018 in Chicago, Illinois. The presentation, classified as late breaking clinical trial results, is scheduled to commence at 2:16 pm Central Time and listed as Main Event 1 for the time frame. This acceptance as a presentation of late-breaking clinical trial results was granted based on the ability of REDUCE-IT to address a critical question in cardiovascular prevention.

“I look forward to the publication of these detailed REDUCE-IT results in a major peer-reviewed journal and to presenting them at the AHA in November,” stated Deepak L. Bhatt, MD, MPH, Professor of Medicine at Harvard Medical School, Executive Director of Interventional Cardiovascular Programs in the Heart and Vascular Center at Brigham and Women's Hospital, and the Principal Investigator and Steering Committee Chair for REDUCE-IT.

“Amarin expresses its great appreciation for all the people that brought REDUCE-IT to completion, especially the patients and investigators and their colleagues at clinical sites that participated in this study for many years,” stated Steven Ketchum, PhD, president of research and development and chief scientific officer of Amarin. “Amarin is also grateful to the U.S. Food and Drug Administration (FDA) for its continued encouragement and support toward study design and completion. REDUCE-IT was conducted under a special protocol assessment agreement with FDA that was re-affirmed in 2016.”

“We are delighted with these topline study results,” said John F. Thero, president and CEO of Amarin. “Given Vascepa is affordably priced, orally administered and has a favorable safety profile, REDUCE-IT results could lead to a new paradigm in treatment to further reduce the significant cardiovascular risk that remains in millions of patients with LDL-C controlled by statin therapy, as studied in REDUCE-IT.”

“Considered against the backdrop of multiple unsuccessful cardiovascular outcomes studies of earlier generation drug therapies, including multiple recent failed cardiovascular studies of omega-3 mixture products that contain the omega-3 acid DHA, REDUCE-IT topline results stand alone as positive and confirm our hypothesis that pure EPA Vascepa at 4 grams/day can provide additional cardiovascular risk reduction benefit on top of LDL-C control with standard of care statin therapy in studied patients,” added Craig Granowitz, MD, PhD, senior vice president and chief medical officer of Amarin. “REDUCE-IT results cannot be generalized to fenofibrate, fish oil or omega-3 mixture products that contain DHA. The most relevant comparator study to REDUCE-IT is the Japan EPA lipid intervention study (JELIS), the 18,645 patient, open label, blinded endpoint outcomes study of EPA added to low-dose statin therapy, which showed cardiovascular event reduction in Japanese hypercholesterolemic patients of 19% in the overall population and 53% in a subgroup of patients with elevated TG levels and low HDL-C.”1, 2, 3

Commercial Expansion and Next Steps

As previously described, given the successful topline results of REDUCE-IT, Amarin is in the process of increasing the number of company sales representatives promoting Vascepa to over 400 people in the United States. This will provide a greater concentration of coverage in current sales territories and provide new coverage where Amarin currently does not have sales representatives.

In addition to sales force expansion in the United States, Amarin plans to work with its international partners to support regulatory efforts outside the United States based on REDUCE-IT results. As previously described in the months leading up to REDUCE-IT results, Amarin increased its Vascepa inventory levels in preparation for positive results.

Overall managed care insurance coverage for Vascepa has been broad. Amarin looks forward to working with insurance carriers to increase understanding of REDUCE-IT results and the potential benefits Vascepa could bring to many millions of patients.

REDUCE-IT Study Background

The REDUCE-IT cardiovascular outcomes study commenced in 2011, enrolled and followed 8,179 randomized patients, and was conducted based on a special protocol assessment agreement with FDA.

REDUCE-IT is the first global cardiovascular outcomes study to prospectively evaluate the effect of Vascepa, or any therapy, in adult patients with LDL-C controlled to between 41-100 mg/dL (median baseline 75 mg/dL) by statin therapy and various cardiovascular risk factors including persistent elevated TGs between 150-499 mg/dL (median baseline 216 mg/dL) and either established cardiovascular disease (secondary prevention cohort) or diabetes mellitus and at least one other CV risk factor (primary prevention cohort). The design of the REDUCE-IT cardiovascular outcomes study was published in March 2017 in Clinical Cardiology4 and can be found in the R&D section on the company’s website at www.amarincorp.com.

The REDUCE-IT hypothesis tested whether additional cardiovascular risk reduction beyond LDL-C controlled with statin therapy could be achieved in high risk patients with the putative cardioprotective effects of Vascepa 4 grams/day. Independent of REDUCE-IT, Amarin has worked to further support the REDUCE-IT hypothesis with published scientific findings based on various degrees of evidence that show EPA may interrupt the atherosclerotic process (e.g., plaque formation and instability) by beneficially affecting cellular functions thought to contribute to atherosclerosis and cardiovascular events and by beneficially affecting lipid, lipoprotein and inflammation biomarkers.5, 6, 7, 8, 9

Financial Disclosure

Funding from Amarin was provided to Brigham and Women’s Hospital for Dr. Deepak L. Bhatt’s work as the REDUCE-IT study chair and international principal investigator.

Conference Call and Webcast Information

Amarin will host a conference call at 8:00 a.m. ET, September 24, 2018 to discuss this information. The call will be accessible through the investor relations section of the company’s website at www.amarincorp.com. The call can also be heard via telephone by dialing 877-407-8033. A replay of the call will be made available for a period of two weeks following the conference call. To hear a replay of the call, dial 877-481-4010 (inside the United States) or 919-882-2331 (outside the United States). A replay of the call will also be available through the company's website shortly after the call. For both dial-in numbers please use conference ID 37638.

About Amarin

Amarin Corporation plc. is a rapidly growing, innovative pharmaceutical company focused on developing therapeutics to improve cardiovascular health. Amarin’s product development program leverages its extensive experience in lipid science and the potential therapeutic benefits of polyunsaturated fatty acids. Vascepa (icosapent ethyl) is Amarin's first FDA-approved drug and is available by prescription in the United States, Lebanon and the United Arab Emirates. Amarin’s commercial partners are pursuing additional regulatory approvals for Vascepa in Canada, China and the Middle East. For more information about Amarin, visit www.amarincorp.com.

About Cardiovascular Disease

Worldwide, cardiovascular disease (CVD) remains the #1 killer of men and women. In the United States CVD leads to one in every three deaths – one death approximately every 38 seconds – with annual treatment cost in excess of $500 billion.10, 11

Multiple primary and secondary prevention trials have shown a significant reduction of 25% to 35% in the risk of cardiovascular events with statin therapy, leaving significant persistent residual risk despite the achievement of target LDL-C levels.5

Beyond the cardiovascular risk associated with LDL-C, genetic, epidemiologic, clinical and real-world data suggest that patients with elevated triglycerides (TG) (fats in the blood), and TG-rich lipoproteins, are at increased risk for cardiovascular disease. 12, 13, 14, 15


About VASCEPA® (icosapent ethyl) Capsules

Vascepa® (icosapent ethyl) capsules are a single-molecule prescription product consisting of the omega-3 acid commonly known as EPA in ethyl-ester form. Vascepa is not fish oil, but is derived from fish through a stringent and complex FDA-regulated manufacturing process designed to effectively eliminate impurities and isolate and protect the single molecule active ingredient. Vascepa, known in scientific literature as AMR101, has been designated a new chemical entity by the FDA. Amarin has been issued multiple patents internationally based on the unique clinical profile of Vascepa, including the drug’s ability to lower triglyceride levels in relevant patient populations without raising LDL-cholesterol levels.

Indication and Usage Based on Current FDA-Approved Label (not including REDUCE-IT results)

  • Vascepa (icosapent ethyl) is indicated as an adjunct to diet to reduce triglyceride (TG) levels in adult patients with severe (≥500 mg/dL) hypertriglyceridemia.

  • The effect of Vascepa on the risk for pancreatitis and cardiovascular mortality and morbidity in patients with severe hypertriglyceridemia has not been determined.

Important Safety Information for Vascepa Based on Current FDA-Approved Label (not including REDUCE-IT results) (Includes Data from Two 12-Week Studies (n=622) (MARINE and ANCHOR) of Patients with Triglycerides Values of 200 to 2000 mg/dL)

  • Vascepa is contraindicated in patients with known hypersensitivity (e.g., anaphylactic reaction) to Vascepa or any of its components.

  • In patients with hepatic impairment, monitor ALT and AST levels periodically during therapy.

  • Use with caution in patients with known hypersensitivity to fish and/or shellfish.

  • The most common reported adverse reaction (incidence >2% and greater than placebo) was arthralgia (2.3% for Vascepa, 1.0% for placebo). There was no reported adverse reaction >3% and greater than placebo.

  • Adverse events and product complaints may be reported by calling 1-855-VASCEPA or the FDA at 1-800-FDA-1088.

  • Patients receiving treatment with Vascepa and other drugs affecting coagulation (e.g., anti-platelet agents) should be monitored periodically.

  • Patients should be advised to swallow Vascepa capsules whole; not to break open, crush, dissolve, or chew Vascepa.

FULL VASCEPA PRESCRIBING INFORMATION CAN BE FOUND AT WWW.VASCEPA.COM.

Vascepa has been approved for use by the United States Food and Drug Administration (FDA) as an adjunct to diet to reduce triglyceride levels in adult patients with severe (≥500 mg/dL) hypertriglyceridemia. Nothing in this press release should be construed as promoting the use of Vascepa in any indication that has not been approved by the FDA.

Important Cautionary Information About Topline Results

Existing and prospective investors are cautioned not to place undue reliance on topline results. As with any topline cardiovascular outcomes study result, further REDUCE-IT data assessment and data release will yield additional useful information to inform greater understanding of the study outcome. Aspects that could change and impact the final evaluation of the totality of the efficacy/safety data from REDUCE-IT may include: the magnitude of the treatment benefit on the primary composite endpoint, its components, secondary endpoints and the primary and secondary risk prevention cohorts; consideration of which components of the composite or secondary endpoints have the most clinical significance; the consistency of the primary and secondary endpoints; the consistency of findings across cohorts and subgroups; tolerability and safety considerations and risk/benefit considerations; consideration of REDUCE-IT results in the context of other clinical studies; and study conduct and data quality, integrity and consistency.

Forward-Looking Statements

This press release contains forward-looking statements, including expectations regarding planned publication, scientific presentation, regulatory review and related timing thereof; expectations that REDUCE-IT results could lead to a new treatment paradigm in the patient population studied; plans for sales force, international and insurance coverage expansion. These forward-looking statements are not promises or guarantees and involve substantial risks and uncertainties. In addition, Amarin's ability to effectively commercialize Vascepa will depend in part on its ability to continue to effectively finance its business, efforts of third parties, its ability to create market demand for Vascepa through education, marketing and sales activities, to achieve market acceptance of Vascepa, to receive adequate levels of reimbursement from third-party payers, to develop and maintain a consistent source of commercial supply at a competitive price, to comply with legal and regulatory requirements in connection with the sale and promotion of Vascepa and to maintain patent protection for Vascepa. Among the factors that could cause actual results to differ materially from those described or projected herein include the following: uncertainties associated generally with research and development, clinical trials and related regulatory approvals; the risk that sales may not meet expectations and related cost may increase beyond expectations; the risk that patents may not be upheld in patent litigation and applications may not result in issued patents sufficient to protect the Vascepa franchise. A further list and description of these risks, uncertainties and other risks associated with an investment in Amarin can be found in Amarin's filings with the U.S. Securities and Exchange Commission, including its most recent quarterly report on Form 10-Q. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. Amarin undertakes no obligation to update or revise the information contained in this press release, whether as a result of new information, future events or circumstances or otherwise.

Availability of Other Information About Amarin

Investors and others should note that Amarin communicates with its investors and the public using the company website http://www.amarincorp.com/), the investor relations website (http://investor.amarincorp.com/), including but not limited to investor presentations and investor FAQs, Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Amarin posts on these channels and websites could be deemed to be material information. As a result, Amarin encourages investors, the media, and others interested in Amarin to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Amarin’s investor relations website and may include social media channels. The contents of Amarin’s website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.


References

1 Yokoyama M, Origasa H, Matsuzaki M, et al. Effects of eicosapentaenoic acid on major coronary events in hypercholesterolaemic patients(JELIS): a randomised open-label, blinded endpoint analysis. Lancet. 2007;369(9567):1090-1098.

2 Saito Y, Yokoyama M, Origasa H, et al. Effects of EPA on coronary artery disease in hypercholesterolemic patients with multiple risk factors: sub-analysis of primary prevention cases from the Japan EPA Lipid Intervention Study (JELIS). Atherosclerosis. 2008;200(1):135-140.

3 Matsuzaki M, Yokoyama M, Saito Y, et al. Incremental effects of eicosapentaenoic acid on cardiovascular events in statin-treated patients with coronary artery disease. Circ J. 2009;73(7):1283-1290.

4 Bhatt DL, Steg G, Brinton EA, et al. Rationale and design of REDUCE-IT: Reduction of Cardiovascular Events with Icosapent Ethyl–Intervention Trial. Clinical Cardiology. 2017;40:138-148.

5 Ganda OP, Bhatt DL, Mason RP, et al. Unmet need for adjunctive dyslipidemia therapy in hypertriglyceridemia management. J Am Coll Cardiol. 2018;72(3):330-343.

6 Borow KM, Nelson JR, Mason RP. Biologic plausibility, cellular effects, and molecular mechanisms of eicosapentaenoic acid (EPA) in atherosclerosis. Atherosclerosis. 2015;242(1):357-366.

7 Nelson JR, Wani O, May HT, et al. Potential benefits of eicosapentaenoic acid on atherosclerotic plaques. Vascul Pharmacol. 2017;91:1–9.

8 Mason RP, Dawoud H, Jacob RF, et al. Eicosapentaenoic acid improves endothelial function and nitric oxide bioavailability in a manner that is enhanced in combination with a statin. Biomed Pharmacother. 2018;103:1231-1237.

9 Takamura M, Kurokawa K, Ootsuji H, et al. Long-term administration of eicosapentaenoic acid improves post-myocardial infarction cardiac remodeling in mice by regulating macrophage polarization. J Am Heart Assoc. 2017;6(2). pii: e004560.

10 American Heart Association. 2018. Disease and Stroke Statistics-2018 Update.

11American Heart Association. 2017. Cardiovascular disease: A costly burden for America projections through 2035.

12 Budoff M. Triglycerides and triglyceride-rich lipoproteins in the causal pathway of cardiovascular disease. Am J Cardiol. 2016;118:138-145.

13 Toth PP, Granowitz C, Hull M, et al. High triglycerides are associated with increased cardiovascular events, medical costs, and resource use: A real-world administrative claims analysis of statin-treated patients with high residual cardiovascular risk. J Am Heart Assoc. 2018;7(15):e008740.

14 Nordestgaard BG. Triglyceride-rich lipoproteins and atherosclerotic cardiovascular disease - New insights from epidemiology, genetics, and biology. Circ Res. 2016;118:547-563.

15 Nordestgaard BG, Varbo A. Triglycerides and cardiovascular disease. Lancet. 2014;384:626–635.

Amarin Contact Information

Investor Relations:

Elisabeth Schwartz

Investor Relations and Corporate Communications Amarin Corporation plc

In U.S.: +1 (908) 719-1315 investor.relations@amarincorp.com

Lee M. Stern

Trout Group

In U.S.: +1 (646) 378-2992 lstern@troutgroup.com

Media Inquiries:

Christy Maginn Burson-Marsteller

In U.S.: +1 (646) 280-5210 Christy.Maginn@bm.com

Mainstay Medical Announces 2018 Half Year Financial Results and Business Update

  • ReActiv8-B study on track – full data readout expected towards the end of 2018

  • Successfully completed financing of $37.5m (€30m) to fund completion of ReActiv8-B study and advance European commercialization

  • Cash on hand of $29.7m at 30 June 2018

Mainstay Medical International plc, a medical device company focused on commercializing ReActiv8®, an implantable restorative neurostimulation system designed to treat an underlying cause of disabling Chronic Low Back Pain, today announces the publication of its results for the Half Year ended 30 June 2018 and provides a company update.

Jason Hannon, CEO of Mainstay, said: “The recent period has seen us make significant progress across the business. We announced completion of all implants in the ReActiv8-B study and remain on track to have full data towards the end of 2018. This is a significant step as we continue to build momentum in our efforts to bring ReActiv8 to patients in the U.S.

“We are dedicated to making rapid progress in Germany, our first commercial market. Starting in March of this year we refined our commercial strategy, repositioned our sales team, focused the training of our new and existing sales team members, and increased our communication to potential implanting physicians. We also appointed a new Managing Director with responsibility for growing the commercial business in Germany, building relationships with key implanting physicians, and growing our team with experienced sales people who are capable of rapidly expanding ReActiv8 in the market.

In recent months we have seen encouraging signs that these initiatives are working. Importantly, we remain on track to meet our target of having 10 or more physician partners performing multiple implants by the end of this year. We believe this momentum will set us up for more meaningful commercial expansion in 2019 as more customers begin to adopt and get comfortable with selecting the appropriate patients for the therapy.”

Business Update

  • During the first half of 2018, significant further progress was made in Mainstay’s pivotal IDE clinical study, ReActiv8-B, which is intended to gather data in support of a pre-market approval (PMA) application to the FDA, a key step towards the commercialization of ReActiv8 in the U.S. Completion of all implants was announced at the start of the third quarter of 2018. A total of 204 patients were implanted in the study, reflecting the strength of interest in study participation. The completion of implants in the clinical study means the Company remains on track to announce full data towards the end of 2018.

  • In Germany, Mainstay’s initial European market, the commercial team was repositioned in order to better focus efforts on key physician targets. Commercialization efforts in line with this strategy began in earnest in March 2018, post our financing announced in February 2018, and are gaining traction. The rates of implants and new implanting sites have increased sharply in July and August 2018 as compared to the first six months of the year.

  • Wolfgang Frisch was appointed VP and Managing Director for Germany on 20 June 2018. Mr. Frisch has over 30 years’ experience in the medical technology industry and will be instrumental in continuing to drive the commercial strategy forward, with a focus on adoption in a select number of high volume spine care centers.

  • Matthew Onaitis was appointed Chief Financial Officer, effective 20 August 2018, bringing with him more than 20 years of experience working with dynamic healthcare businesses and deep knowledge of financing innovative growth companies like Mainstay.

Financial Update

  • On 15 February 2018, the Company announced the completion of a €30.1 million financing (approximately $37.5 million) through a placement of 2,151,332 new ordinary shares to new and existing shareholders. The funds are being used to complete the ReActiv8-B clinical study, advance the initial commercialization of ReActiv8 in Germany and other markets and invest in early commercial activities in preparation for launch in the U.S.

  • Revenue during the six-month period ending 30 June 2018 was $0.4 million ($0.3 million in 1H17).

  • Operating expenses were $15.8 million ($12.3 million in 1H17). This increase was driven primarily by costs relating to the ReActiv8-B study and associated with commercialization efforts.

  • Cash on hand at 30 June 2018 was $29.7 million (31 December 2017: $10 million).

About Mainstay

Mainstay is a medical device company focused on commercializing an innovative implantable restorative neurostimulation system, ReActiv8®, for people with disabling Chronic Low Back Pain (CLBP). The Company is headquartered in Dublin, Ireland. It has subsidiaries operating in Ireland, the United States, Australia, Germany and the Netherlands, and is listed on the regulated market of Euronext Paris (MSTY.PA) and the ESM of Euronext Dublin (MSTY.IE).

About Chronic Low Back Pain

One of the recognized root causes of CLBP is impaired control by the nervous system of the muscles that dynamically stabilize the spine in the low back, and an unstable spine can lead to back pain. ReActiv8 is designed to electrically stimulate the nerves responsible for contracting these muscles and thereby help to restore muscle control and improve dynamic spine stability, allowing the body to recover from CLBP.

People with CLBP usually have a greatly reduced quality of life and score significantly higher on scales for pain, disability, depression, anxiety and sleep disorders. Their pain and disability can persist despite the best available medical treatments, and only a small percentage of cases result from an identified pathological condition or anatomical defect that may be correctable with spine surgery. Their ability to work or be productive is seriously affected by the condition and the resulting days lost from work, disability benefits and health resource utilization put a significant burden on individuals, families, communities, industry and governments.

Further information can be found at www.mainstay-medical.com

KaNDy Therapeutics successfully raises £25 million in a Series C financing

~Funding to advance a breakthrough non-hormonal treatment for symptoms of the menopause, into a Phase 2b study in Q4 2018~

Stevenage, UK - KaNDy Therapeutics, a clinical-stage Women’s Health company, today announces it has successfully closed a Series C financing round, raising £25 million from new US investor Longitude Capital, and existing internationally recognised life sciences investors Advent Life Sciences, Fountain Healthcare Partners, Forbion Capital Partners and OrbiMed.

The proceeds will enable KaNDy Therapeutics to advance its breakthrough non-hormonal drug candidate, NT-814, for treatment of multiple symptoms of the menopause, through a multi-country Phase 2b dose-ranging study due to start recruiting patients in Q4 2018 with headline results expected in late 2019.

Commenting on the financing round, Mary Kerr CEO of KaNDy Therapeutics, said:"We are delighted by the level of enthusiasm and financial support we have received from our investors and would like to welcome Longitude Capital into the syndicate and the board of directors. Our investors and the KaNDy management team are united by the common belief that NT-814 has the potential to be a transformational treatment for the millions of women worldwide who suffer debilitating symptoms of the menopause.”

NT-814 is an orally administered once daily, potent and selective small molecule dual antagonist of both the neurokinin-1 and 3 receptors. It is being developed by KaNDy Therapeutics to provide a viable alternative to hormone replacement therapy. In June 2018, the Company announced positive data from the Phase Ib/IIa proof of concept clinical trial which showed that women who were treated with NT-814 once daily for two weeks at the most effective doses evaluated, experienced a rapid and profound reduction in two key symptoms of the menopause, namely frequency and severity of hot flashes and the number of night time awakenings

Josh Richardson, M.D., Managing Director of Longitude Capital said: “We were very pleased to participate in this funding round. We have been impressed with the data announced thus far, by KaNDy’s business strategy and the strong and experienced management team. We believe NT-814 has the potential to greatly improve the quality of life of millions of women worldwide and we look forward to supporting the Company as it continues to progress this potentially transformational candidate through the clinic towards commercialization.”

A Phase 2b study to further evaluate the safety and efficacy of NT-814 in women with bothersome post-menopausal symptoms, and to establish the optimum dose to take forward into Phase 3, is anticipated to start recruiting patients in the US, Canada and the UK in Q4 2018, with headline results expected in late 2019.

For more information, please contact:

KaNDy Therapeutics
Mary Kerr, Managing Director of KaNDy Therapeutics
Tel: +44 1438 906960
Email: info@kandytherapeutics.com

Consilium Strategic Communications
Mary-Jane Elliott/ Lindsey Neville/ Carina Jurs
Tel: +44 (0) 20 3709 5700
KaNDyTherapeutics@consilium-comms.com

About KaNDy Therapeutics
KaNDy Therapeutics is a clinical-stage company focused on optimizing the potential of NT-814 as a non-hormonal treatment for common, chronic debilitating female sex-hormone related conditions. These conditions, such as post-menopausal hot flashes, can impact womens’ quality of life for 10 years or more and are associated with significant social, healthcare and economic costs.

NT-814 is a once-a-day oral, potent and selective small molecule dual antagonist of both the neurokinin-1 and 3 receptors under development by KaNDy as a therapy for a range of Women’s Health conditions. NT-814 addresses hot flashes by modulating a group of oestrogen sensitive neurones in the hypothalamus in the brain (the KNDy neurones), that in menopausal women due to the absence of oestrogen, become hyperactive and consequently disrupt body heat control mechanisms resulting in the debilitating vasomotor symptoms of HF.

About Advent Life Sciences
Advent Life Sciences is one of Europe’s leading venture teams investing in life sciences businesses. The team consists of professionals with extensive scientific, medical and operational experience, and a long-standing track record of entrepreneurial and investment success across the UK, Europe and the US. The firm invests in a range of sectors within life sciences, principally in new drug discovery, enabling technologies and med tech. Realizations include Algeta, Avila, CardiAQ, CN Creative, EUSA and Micromet. Current investments include Acutus, Arrakis, Aura, Axonics, GMPO and NeRRe. For more information, please visit www.adventls.com.

About Forbion Capital Partners
Forbion is a dedicated life sciences venture capital firm with offices in The Netherlands and Germany. Forbion invests in life sciences companies that are active in the pharmaceutical, as well as the medical device space. Forbion’s investment team has built an impressive performance track record since the late nineties with successful investments in over 50 companies. Forbion manages well over EUR 1 billion across ten funds. Its investors include the EIF, through its European Recovery Programme (ERP), LfA and Dutch Venture Initiative (DVI) facilities and the KFW through the ERP – Venture Capital Fondsfinanzierung facility. Forbion also operates a joint venture with BGV, the manager of seed and early stage funds focused on Benelux and Germany. www.forbion.com

About Fountain Healthcare Partners
Fountain Healthcare Partners is a life science focused venture capital fund with €176 million ($200 million) under management. Within the life science sector, specific areas of interest to Fountain include specialty pharma, medical devices, biotechnology and diagnostics. The firm deploys the majority of its capital in Europe, with the balance in the United States. Fountain’s main office is in Dublin, Ireland, with a second office in New York. For more information, please visit www.fh-partners.com.

About Longitude Capital
Longitude Capital is a private investment firm that makes venture growth investments in biotechnology and medical technology companies that seek to improve clinical outcomes, enhance quality of life and/or reduce system costs. Longitude Capital invests in both privately held and publicly traded life science companies through a variety of investment approaches. Since 2006, Longitude Capital has raised over $1.2 billion across three funds and has offices in Menlo Park, CA and Greenwich, CT. For more information, please visit www.longitudecapital.com.

About OrbiMed
OrbiMed is a leading healthcare investment firm, with $14 billion in assets under management. OrbiMed invests globally across the healthcare industry, from start-ups to large multinational corporations, utilizing a range of private equity funds, public equity funds, and royalty/credit funds. OrbiMed maintains offices in New York City, San Francisco, Shanghai, Hong Kong, Mumbai and Herzliya. OrbiMed seeks to be a capital provider of choice, providing tailored financing solutions and global team resources and support to help build world-class healthcare companies.