Civitas Therapeutics Receives Second Grant for Their Lead Product CVT-301 from The Michael J. Fox Foundation for Parkinson’s Research

Chelsea, MA – Civitas Therapeutics, Inc., a biopharmaceutical company developing transformative therapeutics using the ARCUS(TM) respiratory delivery platform, today announced it has been selected to receive a second grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF). The $1 million grant will support a Phase 2b clinical trial of CVT-301, an inhaled formulation of levodopa (L-dopa). CVT-301 is being developed as an adjunct PRN (as needed) therapy to provide rapid and reliable relief from intermittent debilitating motor fluctuations (OFF episodes) that impact a large proportion of Parkinson’s disease patients.

“The enthusiasm and support of MJFF for CVT-301 has been extraordinarily valuable in our efforts. In addition to its financial contributions, the Foundation’s ability to help connect us with patients, thought leaders and other important stakeholders has contributed to our rapid progress,” said Rick Batycky, Ph.D., Founder and Chief Scientific Officer of Civitas. “We receive consistent feedback that our simple product configuration is uniquely suited for this patient population and this furthers our confidence that CVT-301 has the potential to provide a transformative benefit to those suffering from this devastating disease.”

Civitas recently announced positive top-line results from a Phase 2a clinical trial that showed that CVT-301 administered in the OFF state produced a rapid and durable improvement in motor function in study participants. All doses of CVT-301 tested were generally safe and well tolerated. These results have also provided the basis for dose selection for the Phase 2b trial. Civitas intends to present the comprehensive data from the Phase 2a study at a future scientific meeting.

“We are encouraged by the progress achieved to date by Civitas in their effort to address the unpredictable and debilitating OFF episodes associated with the variability of oral L-dopa administration,” said Alison Urkowitz, Vice President of Research Programs at MJFF. “We are hopeful that the Civitas therapy can play a key role in addressing this unmet need for Parkinson’s patients and are pleased to continue our support of CVT-301 as the therapy undergoes continued human clinical trials.”

About CVT-301

Civitas’ lead program, CVT-301, is an inhaled formulation of L-dopa being developed for the rapid and reliable relief from debilitating motor fluctuations (OFF episodes) associated with Parkinson’s disease. For chronic symptomatic management, oral L-dopa is administered to maintain dopamine levels in the brain above the therapeutic threshold; yet the reliability of oral L-dopa is significantly compromised by delayed and unpredictable absorption and excessive variability in the circulating plasma drug concentrations inherent to the oral delivery route. Oral L-dopa remains widely recognized as the most efficacious treatment of Parkinson’s disease symptoms in spite of this intrinsic unreliability and the resulting OFF episodes. CVT-301 is being developed as an adjunct PRN therapy to standard oral L-dopa therapy to address the OFF episodes as they emerge and enable patients to reliably control their symptoms. CVT-301 is an ARCUS(TM) therapeutic that incorporates L-dopa and is optimized to deliver a precise dose to the deep lung for rapid and predictable L-dopa absorption. The ARCUS(TM) platform is uniquely able to deliver the necessary L-dopa dose with the required precision. A Phase 1 study in healthy volunteers showed that CVT-301 rapidly achieved target L-dopa plasma levels with a pharmacokinetic (PK) profile supportive of its therapeutic potential. The Phase 2a double blind placebo controlled dose finding study (CVT-301-002) recapitulated the PK profile in patients, produced rapid and durable improvement in motor function when administered to patients in the OFF state, and was generally safe and well tolerated in all doses tested.

About Parkinson’s Disease

Over one million people in the US and six million people worldwide suffer from Parkinson’s disease, a neurodegenerative disorder caused by the diminished production of dopamine, resulting in progressive impairment of motor function including tremors, rigidity, and difficulty in moving. The unreliability of available medications for symptomatic treatment of Parkinson’s disease remains a significant unmet need. Even when treated with the current standard of care, the majority of Parkinson’s patients continue to experience motor fluctuations. These unpredictable OFF episodes reduce patients’ ability to lead productive, independent lives and are recognized by patients, care givers, and healthcare professionals as one of the most troubling and debilitating issues associated with the disease.

About ARCUS(TM) Platform

The ARCUS(TM) platform is a proprietary dry powder and device combination with a unique ability to deliver a large, precise dose independent of inspiratory flow rate from a simple, breath actuated device. The platform is protected by a large intellectual property estate including over 130 issued patents. The technology has successfully delivered more than one million doses to patients and the manufacturing technology has been scaled to accommodate a significant commercial launch.

About Civitas Therapeutics

Civitas is a privately-held biopharmaceutical company focused on developing a robust pipeline of inhaled therapeutics with the clinically proven ARCUS(TM) dry powder pulmonary delivery platform. In addition to the lead program, CVT-301 for treating Parkinson’s disease, other programs encompass respiratory disease, central nervous system disorders, and infectious disease. The company is headquartered in Chelsea, MA in a facility that includes both development and commercial scale GMP manufacturing capabilities. The Company is financed by leading investors including Canaan Partners, Fountain Healthcare Partners, Longitude Capital and Alkermes plc. For further information on Civitas, please visit www.civitastherapeutics.com.

About the Michael J. Fox Foundation for Parkinson’s Research

As the world’s largest private 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 $325 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 groundbreaking 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.

Trino Therapeutics Secures Series A Investment Of Over €9M ($12M) For A Novel Oral Drug Therapy For Mild To Moderate Ulcerative Colitis

• Ireland’s Trino Therapeutics secures major additional investment from new investors Fountain Healthcare Partners and founding investor the Wellcome Trust;
• Wellcome Trust will invest through the prestigious internationally competitive Strategic Translation Award;
• New funding to progress the lead candidate, PH46A, to clinical studies in healthy volunteers and patients with mild to moderate ulcerative colitis;
• Accelerates development with international development partners and major clinical centres

DUBLIN, IRELAND: May 28th 2013, TRINO THERAPEUTICS LTD (‘Trino’) a drug discovery and early drug development company focused on anti-inflammatory therapeutics, today announced that it has raised more than €9 million (US $ 12million) in Series A financing from news investors Fountain Healthcare Partners and founding investor, the Wellcome Trust. Other investors in Trino include Enterprise Ireland and Growcorp. Both the Wellcome Trust and Fountain Healthcare Partners will be represented on the board of directors.

The company is developing PH46A, the lead candidate from a novel, proprietary, class of drugs which was inspired by the indane scaffold molecule derived from a Taiwanese fern. PH46A is a potential first-in-class drug oral small molecule for the treatment of inflammatory bowel disease (IBD), which could be used in both ulcerative colitis (UC) and Crohn’s disease (CD).

“Our commitment to Trino is a strong validation of the quality of the investment opportunity in Irish biomedical research, particularly in areas such as immunology and gastroenterology”, noted Dr. Ena Prosser, Fountain Healthcare Partners.

Other molecules in Trino’s drug class show promise with broad anti-inflammatory activity that could be suitable for applications in dermatology, pulmonary and auto-immune disease and the company will work to develop these compounds internally and in partnership with major international research centres.

The additional funding from the Wellcome Trust is in the form of a prestigious, international and highly competitive Strategic Translation Award.

Dr Richard Seabrook, Head of Business Development at the Wellcome Trust commented, “Current treatments for inflammatory bowel diseases often have significant side effects and patients are faced with tough decisions in how to manage their condition. We are pleased to extend our successful partnership with Trino to support the development of PH46A as a potential new therapy for these debilitating disorders.”

The company was founded by pharmacologist Prof Neil Frankish and medicinal chemist Prof Helen Sheridan centred on their highly innovative work on pharmaceutical grade drugs based on the indane skeleton as derived from a Taiwanese fern, used historically in plant-based medicine.

Commenting on this investment on behalf of the company Prof Frankish said, “This significant investment validates our research, enabling us to expand the Trino team and develop our clinical partnerships so that we can investigate the effectiveness of our research where it is needed - in patients with inflammatory diseases and ineffective drugs”.

Initial funding of €2.2M from this syndicate was used to complete GMP manufacture and pre-clinical testing. The additional €7M investment will be used to bring the drug through formal First in Man testing in healthy volunteers, to identify the international clinical investigator network to run the patient studies and to complete initial Phase II testing of PH46A in mild to moderate ulcerative colitis.

For further information:

Eilish Joyce, FTI Consulting, +353 1 663 3609 / eilish.joyce@fticonsulting.com

Genable Technologies Ltd appoints Annette Clancy as Chairman of the Board

DUBLIN, Ireland, 13th May 2013: Genable Technologies Ltd., a bio-pharmaceutical company developing new gene medicines to treat ‘dominant’ genetic diseases, today announced the appointment of Annette Clancy as Chairman of the Board. Annette graduated in Pharmacology from Bath University UK and also holds a series of American Management Diplomas. She has had a celebrated 30-year career in the pharmaceutical/biotechnology industry, predominantly at GlaxoSmithKline, where she has worked on a variety of R&D and commercial functions

Ms Clancy was Head of Transactions and Alliance Management at GlaxoSmithKline (GSK) where she was responsible for executing innovative deals ranging from early drug discovery partnerships, to global commercial alliances and Mergers and Acquisitions.

Since her retirement from GSK in 2008, Ms Clancy has been appointed as Non-Executive Board Director to Silence Therapies plc. (2008-2012) and Clavis Pharma in Norway (2008 ongoing). Ms Clancy also currently serves as Senior European Advisor to the biopharmaceutical team of Frazier Healthcare Ventures, for whom she provides strategic advice on both new and existing investments.

As a Non-Executive Board Member to Genable Technologies Ltd., Ms Clancy will also provide strategic consultancy advice to the company.

Ms Clancy said, “This is a very exciting time for the company and I am looking forward to working with the Genable Technologies team as they continue to make advances for suffers of genetic diseases.”

Welcoming Ms Clancy’s appointment, Genable’s outgoing Chairman Dr John Monahan said, “Annette has extensive and impressive experience in this industry and we are delighted that she will bring some of that experience to Genable Technologies as Chairman. We have seen significant advances in our products and research recently and we are certain that Annette’s proficiency and skill will enhance the expertise already within the company.” The company are also delighted to note Dr Monahan will remain on the Genable Board as a Non-Executive Director.

Genable Technologies Ltd gene therapy product, GT038, has recently been granted Orphan Drug Designation by the Federal Drug Administration (FDA) in the USA for the treatment of Retinitis Pigmentosa, complimenting the previous orphan drug designation granted by the European Commission. This is a significant milestone as it provides Genable Technologies with seven years market exclusivity once GT308 has secured regulatory approval.

GT038 is an adeno-associated viral vector containing DNA encoding an RNAi targeting rhodopsin in combination with an adeno-associated viral vector containing a rhodopsin gene for the treatment of rhodopsin-linked Retinitis Pigmentosa. The product is currently undergoing formal pre-clinical assessments prior to commencing clinical studies in patients.

About Genable Technologies Ltd

Genable Technologies Ltd. is a privately held, venture capital backed Dublin (Ireland) based bio-pharmaceutical company and secured financing from Fountain Healthcare Partners and Delta Partners in 2011. The company is developing new gene therapies to treat "dominant" genetic diseases based on the pioneering work of Professor Jane Farrar, Dr Paul Kenna & Professor Peter Humphries of Trinity College Dublin. The background research has been supported by Fighting Blindness Ireland, Science Foundation Ireland, Foundation Fighting Blindness-National Neurovision Research Institute (USA), Enterprise Ireland & EVI-GenoRet (EU FP6-funded). www.genable.ie

For additional information:

Eilish Joyce
FTI Consulting
Tel: +353 1 663 3609 / +353 87 791 4641
Email: eilish.joyce@fticonsulting.com

Opsona Therapeutics Ltd. Initiates a Phase II Study with OPN-305

Opsona Therapeutics Ltd. Initiates a Phase II Study with OPN-305, a First-in Class Monoclonal Antibody that Blocks Toll-Like Receptor 2, in Renal Transplant Patients at High Risk of Delayed Graft Function

DUBLIN, May 07, 2013.  Opsona Therapeutics Ltd ('Opsona'), the innate immune drug development company focused on novel therapeutic approaches to treat autoimmune and inflammatory diseases, today announced that it has initiated a phase II clinical trial in renal transplant patients at high risk of delayed graft function with its lead drug candidate OPN-305.

OPN-305 has already been administered to a number of transplant patients at high risk of delayed graft function (DGF) as part of a Pilot study before initiation of this multi-centre, randomized, double-blind, placebo-controlled, parallel group, sequential adaptive phase II trial. This pilot clearly demonstrated that 100% receptor occupancy of TLR2 on circulating monocytes was achievable and durable for the period of ischemia/reperfusion risk. It provided evidence that there was up-regulation of TLR2 in patients and provided the range of doses to be tested versus placebo in the double-blind trial. OPN-305 was well tolerated with no related adverse events. The phase II study which is expected to enrol 278 patients is an adaptive design powered to show a 15-20% absolute benefit of OPN-305 over placebo in reducing the incidence of DGF.

OPN-305 is a novel proprietary humanized IgG4 monoclonal antibody (MAb) against Toll-Like Receptor 2 (TLR2), a target within the innate immune system, and is under development as a treatment for the prevention of DGF following renal transplantation. OPN-305 has orphan status in the EU and USA for solid organ transplantation. Other therapeutic indications are being explored in addition.

Opsona has identified the prevention of DGF following renal transplantation as the first clinical indication for the development of OPN-305. Delayed Graft Function is a serious complication that can increase the risk of organ rejection in the immediate post-operative period of kidney transplants and can range from 45-60% in high risk donor kidneys. Opsona believes that OPN-305 has the potential to be first and best in class in the prevention of DGF and will also provide a novel treatment option for a much wider variety of human diseases, including acute kidney injury, transplantation of other organs, cancer, cardiovascular disease and others.

Commenting on today's announcement, Mary Reilly VP Pharmaceutical Development and Operations of Opsona Therapeutics said, "It's exciting to see cutting-edge science offering the prospect of a real breakthrough in life expectancy and quality post transplantation. OPN-305 was granted 'orphan' status for solid organ transplantation, in recognition of its rarity and the fact that there are no other comparable treatments being developed. OPN-305 could be a potential first and best in class candidate to reduce this complication. Developing a novel product for an unmet clinical need and working with world class global key opinion leaders in the transplant community is highly motivating and gives all the project partners a sense of urgency about this collaboration."

Dr. Robert Miller Chief Medical Officer of Opsona commented, "This study represents a real opportunity to improve early graft function in patients offered kidneys from older donors and enlarging the pool of potential organs for patients with end-stage renal disease. We are very pleased with the enthusiasm of the transplant community who have readily agreed to participate in this process."

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 autoimmune and inflammatory diseases, solid organ transplantation, cancer, diabetes, Alzheimer's disease and others. The company was founded in 2004 by three world-renowned immunologists at Trinity College in Dublin. Opsona's lead product, a fully humanized monoclonal IgG4 antibody (OPN-305) targeting Toll-like-receptor-2 (TLR2) has demonstrated activity in a number of preclinical models and has been tested in healthy volunteers and recently in a Pilot study in transplant patients. Opsona was awarded EUR5.9 million from the European Commission in 2010 to lead a European framework 7 (FP7) consortium of research and clinical groups (termed MABSOT*) to advance OPN-305 through clinical development. Opsona has recently completed a EUR33 million Series C financing round with an international investor consortium including Novartis Venture Fund, Fountain Healthcare Partners, Roche Venture Fund, Seroba Kernel Life Sciences, BB Biotech Ventures, Sunstone Capital, Baxter Ventures, Amgen Ventures and EMBL Ventures.

Note

*Monoclonal antibody solid organ transplantation

Opsona Therapeutics Limited raises €33 million (US$43 million) oversubscribed Series C equity financing to advance clinical development of its lead product OPN-305

April 29th, 2013, Dublin, Ireland – Opsona Therapeutics Limited (‘Opsona’), the innate immune drug development company, today announced that it has raised €33 million (US$43 million) in an oversubscribed Series C financing. The participants in this Series C financing include existing investors, Novartis Venture Fund, Fountain Healthcare Partners, Roche Venture Fund and Seroba Kernel Life Sciences. The new investors joining the consortium are BB Biotech Ventures, Sunstone Capital, Baxter Ventures, Amgen Ventures, and EMBL Ventures. BB Biotech Ventures and Novartis Venture Fund led the Series C financing round. BB Biotech Ventures, Sunstone Capital and Baxter Ventures will be joining the board of directors.

Opsona is developing new treatments for inflammatory diseases and will use the proceeds to conduct a two-part multi-centered, double blinded and placebo controlled clinical study to evaluate the safety, tolerability and efficacy of its lead product OPN-305 in renal transplant patients at high risk of Delayed Graft Function (DGF). This is the first clinical indication for OPN-305, a fully human monoclonal IgG4 antibody targeting Toll-like-receptor-2 (TLR2). Opsona recently completed a successful Phase I clinical trial in healthy human volunteers and has also demonstrated activity in preclinical animal models and ex-vivo studies. This first-in-class inhibitor of TLR2 has the advantage of inhibiting multiple cytokines leading to the pathogenesis of the complex inflammatory response in various diseases (ischemia/reperfusion injuries, rheumatoid arthritis, diabetes, lupus, nephritis and various cancers) and has therefore a potentially broad application potential.

Dr. Martin Welschof, CEO of Opsona, commented: “The innate immune system represents a new frontier in targeting inflammatory diseases, and the quality of venture and corporate investors in this funding round is a demonstration of Opsona's expertise and capabilities in this highly promising field. With their repeat investment, our existing investors have clearly indicated their long-term commitment to Opsona, while the new investment from BB Biotech Ventures, Sunstone Capital, Baxter Ventures, Amgen Ventures and EMBL Ventures is a further endorsement of Opsona’s future potential.”

Dr. Martin Muenchbach, Managing Director at BB Biotech Ventures, added: “We are delighted to be working with Opsona Therapeutics and the other investors. We are excited to further advance Opsona’s lead product OPN-305 into a well-designed Phase II efficacy study to improve post-operative complications in renal transplantation, an indication with major unmet medical need and attractive commercial potential. This high-quality study with clinically meaningful endpoints will also be of relevance for a subsequent Phase III registration study.”

-ends-

For further information please contact:

Martin Welschof (CEO), telephone: + 35316770223, e-mail: mwelschof@opsona.com

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 autoimmune and inflammatory diseases, transplant rejection, cancer, diabetes, Alzheimer's disease and atherosclerosis. The company was founded in 2004 by three world-renowned immunologists at Trinity College in Dublin. Opsona's lead product, a fully human monoclonal IgG4 antibody (OPN-305) targeting Toll-like-receptor-2 (TLR2) has demonstrated activity in a number of animal models and was recently tested in a Phase 1 clinical trial in healthy volunteers. The company has initiated a two-part multi-centered, double blinded and placebo controlled clinical study to evaluate the safety, tolerability and efficacy of OPN 305 in renal transplant patients at high risk of Delayed Graft Function (DGF) as the first clinical target indication for the development of OPN-305. The company was awarded a EUR 5.9 million non-dilutive grant by the European Union for clinical development of its anti-TLR2 antibody in solid organ transplantation including renal transplantation and the program has recently obtained EMA and FDA orphan drug status. Additional indications are currently being explored.

Further information is available at http://www.opsona.com/.

Civitas Therapeutics Announces Positive Phase 2 Clinical Results for CVT-301, an Inhaled L-dopa for Parkinson’s Disease

CVT-301 shown to treat debilitating OFF Episodes Associated with Parkinson’s Disease

Chelsea, MA – April 19, 2013 – Civitas Therapeutics, Inc., a privately-held pharmaceutical company developing transformative therapeutics using the ARCUS(TM) respiratory delivery platform, today announced positive topline results from a Phase 2 clinical trial of CVT-301, an inhaled formulation of levodopa (L-dopa). CVT-301 is being developed as an adjunct therapy to provide rapid and reliable relief from intermittent debilitating motor fluctuations (OFF episodes) that impact a large proportion of Parkinson’s disease patients.

The study used a randomized, placebo-controlled design to evaluate L-dopa pharmacokinetics and pharmacodynamic effects following administration of CVT-301 to Parkinson’s disease patients experiencing motor fluctuations. Administering CVT-301 to patients in the OFF state produced a rapid and durable improvement in motor function. The pharmacokinetic data recapitulated the Phase 1 study results showing CVT-301 provided immediate L-dopa absorption and consistent increases in plasma concentrations in marked contrast to the delayed and variable L-dopa levels seen with Sinemet® (oral L-dopa/carbidopa). All doses of CVT-301 were safe and well tolerated with no increase in the frequency or severity of dyskinesias relative to oral. Civitas plans to present the comprehensive data from the study at a future scientific meeting.

“The unpredictable wearing off of oral L-dopa and the dyskinetic side effects are among the most significant challenges with the current management of Parkinson's disease," said Dr. Todd Sherer, CEO of The Michael J. Fox Foundation for Parkinson's Research. "There remains a critical unmet need for therapies that increase the reliability of L-dopa while not exacerbating the side effects."

“The significant inherent variability of oral L-dopa absorption is known to be a major contributor to the development of debilitating OFF episodes,” said Dr. Martin Freed, Chief Medical Officer and co-founder of Civitas. “CVT-301 has the potential to provide a transformative benefit to patients by enabling more predictable and effective symptomatic relief without worsening side effects such as dyskinesia, thereby allowing them to regain control of their lives.”

“The results of this study represent proof-of-concept of CVT-301 as a therapy to provide rapid and precise control of patients’ L-dopa levels enabling better management of their intermittent motor fluctuations. This has the potential to be a very meaningful advancement in the symptomatic treatment of Parkinson’s disease,” said Dr. Karl Kieburtz, the Robert J. Joynt Professor of Neurology, University of Rochester, and a member of the Civitas Scientific Advisory Board.

This Phase 2a study of CVT-301 was funded in part by a grant from The Michael J. Fox Foundation for Parkinson’s Research.

Trial Design

The Phase 2 study (CVT-301-002) was a multicenter, randomized, double blind, placebo-controlled, single dose, cross-over design with three arms (placebo, 25mg and 50mg) and included an “open label” oral Sinemet arm. The twenty four (24) patients treated in this study underwent serial evaluations of L-dopa plasma levels, motor response, and safety at each visit. The patients were administered the study drug in the OFF state with the serial evaluations starting prior to dosing and continuing for up to 180 minutes post-dose. Motor function was measured using a tapping test, the Unified Parkinson’s Disease

Rating Scale Part III (UPDRS III), and subjective evaluation of “meaningful” ON and OFF. Safety parameters monitored included pulmonary function, clinical laboratory data, EGCs, and vital signs (blood pressure, heart rate, and orthostatic blood pressure). This study was designed to measure the time, magnitude, and durability of CVT-301’s effect on motor function, to evaluate the safety and tolerability of CVT-301 in Parkinson’s disease patients, to confirm the results from a CVT-301 Phase 1 healthy volunteer study (CVT-301-001), and to establish the dose for future clinical trials with CVT-301.

About CVT-301

Civitas’ lead program, CVT-301, is an inhaled formulation of L-dopa for the rapid and reliable relief from debilitating motor fluctuations associated with Parkinson’s disease. For symptomatic relief, oral L-dopa is administered to maintain dopamine levels in the brain above the therapeutic threshold; yet the efficacy of oral L-dopa is significantly compromised by delayed and unpredictable absorption and excessive variability in the circulating plasma drug concentrations inherent to the oral delivery route. CVT-301 is an ARCUS(TM) therapeutic that incorporates L-dopa and is optimized to deliver a precise dose to the deep lung for rapid and predictable L-dopa absorption. The ARCUS(TM) platform is able to uniquely deliver the necessary L-dopa dose with the required precision. CVT-301 is being developed as an adjunct to standard oral L-dopa therapy to enable patients to manage motor fluctuations caused in part by the inter-dose variability of oral L-dopa. In preclinical models, CVT-301 has demonstrated immediate and consistent increases in L-dopa peak plasma concentration providing rapid, durable symptomatic relief, even when compared to larger doses of oral L-dopa. A Phase 1 study in healthy volunteers showed that CVT-301 rapidly achieved target L-dopa plasma levels with a pharmacokinetic profile supportive of its therapeutic potential.

About Parkinson’s Disease

Over one million people in the US and six million people worldwide suffer from Parkinson’s disease, a neurodegenerative disorder caused by the diminished production of dopamine, resulting in progressive impairment of motor function including tremors, rigidity, and difficulty in moving. Even when treated with the current standard of care, the majority of Parkinson’s patients continue to experience motor fluctuations. These unpredictable OFF episodes reduce patients’ ability to lead productive, independent lives and are recognized by patients, care givers, and healthcare professionals as one of the most troubling and debilitating issues associated with the disease.

About ARCUS(TM) Platform

The ARCUS(TM) inhalation technology delivers a reliable and consistent drug dose with a compact, breath actuated inhaler. The ARCUS(TM) platform uses a proprietary dry powder and inhaler combination that is unique in its ability to deliver a large, precise dose independent of inspiratory flow rate from a simple, easy-to-use device suitable for convenient self-administration. The platform has successfully delivered more than one million doses to patients incorporating active agents ranging from small molecules to large proteins and has been scaled up to accommodate a commercial product launch.

About Civitas Therapeutics

Civitas is a privately-held pharmaceutical company focused on developing a robust pipeline of inhaled therapeutics with the clinically proven ARCUS(TM) dry powder pulmonary delivery platform. Additional programs encompass respiratory disease, central nervous system disorders, and infectious disease. Civitas exclusively licensed and purchased the technology and assets underlying the ARCUS(TM) platform from Alkermes plc, including a large intellectual property estate, a set of development stage pipeline assets, specialized equipment for respiratory products, and the commercial scale GMP manufacturing facility. Civitas was launched at the beginning of 2011 with Canaan Partners, Fountain Healthcare Partners, Longitude Capital, and Alkermes as investors.

For additional information contact:

Stephanie Gillis
Civitas Therapeutics

Ireland’s Genable Technologies Ltd secures Orphan Drug Designation by FDA for gene therapy product GT308

DUBLIN, Ireland, 12th April, 2013 - Genable Technologies Ltd gene therapy product, GT038, has been granted Orphan Drug Designation by the Federal Drug Administration (FDA) in the USA for the treatment of Retinitis Pigmentosa. This is a significant milestone as it provides Genable Technologies with seven years market exclusivity once GT308 has secured regulatory approval.

GT038 is an adeno-associated viral vector containing DNA encoding an RNAi targeting rhodopsin in combination with an adeno-associated viral vector containing a rhodopsin gene for the treatment of rhodopsin-linked Retinitis Pigmentosa. The product is currently undergoing formal pre-clinical assessments prior to commencing clinical studies in patients.

Patients with rhodopsin-linked Retinitis Pigmentosa have a mutation in the rhodopsin gene which causes a patient's sight to worsen, eventually leading to blindness. Almost 300,000 people worldwide have Retinitis Pigmentosa (RP) and of these, approximately 30% will have rhodopsin- linked (RP). GT038 is a novel and unique therapy for rhodopsin-linked Retinitis Pigmentosa that utilizes AAV vectors to obtain expression of RNA interference molecules, which suppress the expression of the faulty gene, and replaces this with a gene encoding a functioning protein. This simple combination overcomes the significant hurdle in diseases such as rhodopsin-linked Retinitis Pigmentosa of mutation variability by eliminating the need to target specific mutations.

Professor Alan Boyd MD of Genable Technologies Ltd said, ‘‘Retinitis Pigmentosa is a serious condition that leads to blindness in young people. There are currently no approved treatments for this condition and having orphan designation for GT038 in the US as well as Europe, is fundamental to the future development of our product that could have a significant impact on patients’ lives.’’

This orphan drug designation for GT038 by the FDA compliments the previous orphan drug designation granted by the European Commission to Genable Technologies Ltd, (Orphan Designation EU/3/10/817) for the treatment of rhodopsin-linked retinitis pigmentosa.

About Genable Technologies Ltd

Genable Technologies Ltd. is a privately held, venture backed, Dublin (Ireland) based bio-pharmaceutical company developing new gene medicines to treat "dominant" genetic diseases based on the pioneering work of Professor Jane Farrar, Dr Paul Kenna & Professor Peter Humphries of Trinity College Dublin. The company has secured financing from Fountain Healthcare Partners and Delta Partners. The background research has been supported by Fighting Blindness Ireland, Science Foundation Ireland, Foundation Fighting Blindness-National Neurovision Research Institute (USA), Enterprise Ireland & EVI-GenoRet (EU FP6-funded). www.genable.ie/

For additional information:

Eilish Joyce
FTI Consulting
Tel: +353 1 663 3609 / +353 87 791 4641
Email: eilish.joyce@fticonsulting.com

Mainstay Medical Appoints Oern R. Stuge MD as Chairman of the Board

Dublin, Ireland, 31st January, 2013 –Mainstay Medical Ltd., an Irish medical device company developing a disruptive new therapy for patients with chronic non-specific low back pain, today announced the appointment of Oern R. Stuge MD as Independent Chairman of the Board. After training and practicing as an internal medicine physician in Norway, Dr Stuge embarked on a distinguished career with extensive international strategic and operational experience in market leading medical device, diagnostics and pharmaceutical companies.

Dr Stuge currently leads Orsco Life Science, a Swiss based management consulting firm that specialises in advising companies in the Medtech sector, and holds several executive and non-executive board memberships or advisory positions.

During his tenure as a senior executive at Medtronic, Dr Stuge served as Vice President of EMEA Neurological & Spinal Division which manufactured and sold spinal implants, neurostimulators, implantable drug pumps, powered surgery systems, microendoscopy instruments and navigation equipment. He served as Senior Vice-President & President, Europe and Central Asia, managing all of Medtronic’s operations in these territories. He also held the roles of Senior Vice President & President, Cardiac Surgery; VP Cardiac Rhythm Management; Chairman of the Board & VP Medtronic Functional Diagnostics. Dr Stuge served on the Executive & Operating Committee of Medtronic.

Prior to Medtronic, Dr Stuge worked in senior positions at Abbott Laboratories and was CEO of Medinor A/S.

Dr Stuge said, “I’m looking forward to working with the board and leadership of Mainstay Medical at this very exciting time for the company. Mainstay’s products hold real promise for the millions of people who suffer from chronic low back pain, and who have few other options.”

Welcoming Dr Stuge’s appointment, Mainstay Medical CEO Peter Crosby said, “We are pleased that Oern will bring his extensive experience to Mainstay Medical as our Chairman that will add to the expertise already within the company. We are making great progress in advancing our products from concept to reality and are delighted to attract such expertise to our growing company.”

Mainstay Medical has a novel approach for treating chronic low back pain using an implantable device like a “pacemaker for the back” to restore spine stability to ameliorate pain and allow a return to normal daily life.

Mainstay Medical has conducted a European Feasibility Study and in parallel advanced the development of its own proprietary products, and added to its intellectual property portfolio. Mainstay will continue to expand the team as it moves towards its goals of achieving regulatory approval in Europe and USA setting the stage for building an exciting business.

The company has also moved into new offices in Swords, Co. Dublin.

About Mainstay Medical Ltd

Mainstay Medical Ltd. is a privately held, venture backed, Dublin (Ireland) based medical device company. The company develops products for patients with chronic low back pain. In September 2012, Mainstay Medical completed an oversubscribed Series B financing round for $20.0M (€15.3M) led by Fountain Healthcare Partners (Ireland). Other new investors in the Series B round included Medtronic (US), Capricorn Venture Partners (Belgium) and Seventure Partners (France). Existing Series A investors Sofinnova Partners (France), and Twin City Angels (Minneapolis, USA) also participated.

Civitas Therapeutics to Present Positive Clinical Study Results for CVT-301 at Sixth Annual Parkinson’s Disease Therapeutics Conference

Civitas Therapeutics to Present Positive Clinical Study Results for CVT-301 at Sixth Annual Parkinson’s Disease Therapeutics Conference, Sponsored by The Michael J. Fox Foundation for Parkinson’s Research

Company’s first public presentation will feature results from Phase I study demonstrating pharmacokinetic clinical proof-of-concept

Company continues progress with ongoing Phase 2 clinical study

Chelsea, MA – October 15, 2012 – Civitas Therapeutics, Inc., a privately-held pharmaceutical company developing transformative therapeutics using the ARCUS(TM) respiratory delivery platform, announced today that the Company will make its first public presentation at the Sixth Annual Parkinson’s Disease Therapeutics Conference, sponsored by The Michael J. Fox Foundation for Parkinson’s Research, to be held on October 24th in New York City. Martin Freed, MD, Chief Medical Officer and co-founder of Civitas, will present the CVT-301 Phase 1 study results. CVT-301 is an inhaled levodopa (L-dopa) formulation, being developed to provide rapid alleviation of intermittent motor fluctuations (“off” episodes) associated with Parkinson’s disease.

“While L-dopa remains the most effective and widely used symptomatic agent for Parkinson’s disease, the significant intrinsic variability of the conventional oral administration of L-dopa contributes to unpredictable ‘off’ episodes that have a devastating impact on patients’ lives,” said Dr. Freed. “By enabling patients to rapidly and consistently increase their L-dopa plasma levels as they sense ‘off’ symptoms, CVT-301 has the potential to dramatically improve their ability to reliably manage their symptoms and regain control of their daily lives.”

The Phase 1 study in healthy volunteers evaluated the safety, tolerability and L-dopa pharmacokinetic profile across a range of doses of CVT-301 delivered using Civitas’ proprietary, simple handheld breath- actuated inhaler. The study showed that inhaled delivery of CVT-301 achieved therapeutic L-dopa plasma levels within five minutes of administration with unprecedented precision. Consistent and dose proportional pharmacokinetics were seen across all doses tested. In addition, all doses tested of CVT-301 were observed to be safe and well tolerated. In the context of extensive clinical experience correlating L-dopa plasma levels to symptomatic relief, the CVT-301 Phase 1 clinical study results represent pharmacokinetic clinical proof of concept.

CVT-301 is being developed as an adjunct to standard oral L-dopa therapy to enable patients to manage motor fluctuations caused in part by the inter-dose variability of oral L-dopa. Civitas anticipates reporting topline results from the ongoing CVT-301 Phase 2a double blind placebo controlled study in Parkinson’s disease patients in early 2013. Both the Phase 1 and Phase 2a study were funded in part by a grant from The Michael J. Fox Foundation for Parkinson’s Research.

About CVT-301

CVT-301 is an inhaled levodopa (L-dopa) formulation, being developed as a therapy to provide rapid alleviation of intermittent motor fluctuations (“off” episodes) associated with Parkinson’s disease. Conventional oral L-dopa is administered at regularly scheduled intervals to provide symptomatic relief by maintaining dopamine levels in the CNS above the therapeutic threshold. However, for many patients the efficacy of oral L-dopa is significantly compromised by delayed absorption and excessive variability in the circulating plasma drug concentrations inherent to the oral delivery route. CVT-301 is an inhaled L-dopa formulation that utilizes the ARCUS(TM) platform to deliver a precise dose to the deep lung for rapid and predictable L-dopa absorption. The ARCUS platform is uniquely able to deliver the necessary L-dopa dose with the required precision with a simple breath- actuated device. In preclinical models, CVT-301 has demonstrated rapid, durable symptomatic relief, even when compared to larger doses of oral L-dopa.

The Phase 1 clinical study of CVT-301 was completed in 2011 and demonstrated pharmacokinetic proof-of-concept. Therapeutic plasma levels of L-dopa were achieved within five minutes of inhalation dosing with unprecedented precision. Dose proportional pharmacokinetics were seen across all doses tested. In addition, all doses tested of CVT-301 were safe and well tolerated. A Phase 2a study of CVT-301 is currently underway.

About Parkinson’s Disease

Over one million people in the US suffer from Parkinson’s disease, a neurodegenerative disorder caused by the diminished production of dopamine, a key neurotransmitter, resulting in progressive impairment of motor function including tremors, rigidity and difficulty in moving. Even when treated with the current standard of care, the majority of Parkinson’s patients continue to experience motor fluctuations. These motor fluctuations reduce patients’ ability to lead productive, independent lives and are recognized by patients, care givers and healthcare professionals as one of the most troubling and debilitating issues associated with the disease.

About Civitas Therapeutics

Civitas is a privately-held pharmaceutical company focused on developing a robust pipeline of inhaled therapeutics with the clinically proven ARCUS(TM) dry powder pulmonary delivery platform. The company’s lead program for Parkinson’s disease is intended to treat intermittent and debilitating motor fluctuations resulting from an inadequate response to their standard oral medications. Additional programs encompass respiratory disease, central nervous system disorders and infectious disease.  Civitas exclusively licensed and purchased the technology and assets underlying the ARCUS platform from Alkermes plc, including a large intellectual property estate, a set of development stage pipeline assets, specialized equipment for respiratory products and the commercial scale GMP manufacturing facility. Civitas was launched at the beginning of 2011 with Canaan Partners, Fountain Healthcare Partners, Longitude Capital and Alkermes as investors.

Mainstay Medical Completes $20.0M (€15.3M) Oversubscribed Series B Venture Financing

Fountain Healthcare Partners (Ireland) lead Series B Financing

Series A Financing led by Sofinnova Partners (France) in 2010

Proceeds will advance product development and clinical studies towards regulatory approval for market release in the US and Europe.

Dublin, Ireland, 26th September, 2012 – Medical device company Mainstay Medical Ltd. today announced that is has successfully completed an oversubscribed Series B financing round for $20.0M (€15.3M) led by Fountain Healthcare Partners (Ireland - “Fountain”). Other new investors in the Series B round include Medtronic (US - “Medtronic”), Capricorn Venture Partners (Belgium - “Capricorn”) and Seventure Partners (France - “Seventure”). Existing Series A investors Sofinnova Partners (Sofinnova - France), and Twin City Angels (TCA – Minneapolis, USA) also participated. This funding will advance the on-going development of Mainstay Medical’s disruptive new medical device for the treatment of patients with chronic non-specific low back pain. As part of the financing, Mainstay Medical relocated its head office and executive leadership to Dublin, Ireland from Minneapolis, Minnesota US.

The World Health Organization reports that low back pain is the most prevalent of musculoskeletal conditions, and affects nearly everyone at some point in time. Low back pain is the number one cause of lost working days in the developed world, with a staggering 7.5 Million people in the US with chronic back pain at any time, and similar numbers for Europe. The cost to society of lost working days and health care treatment is enormous. For many of these people, low back pain results from malfunction of muscles responsible for spine stability. Mainstay Medical has developed a novel approach using an implantable device like a “pacemaker for the back” to restore spine stability to ameliorate pain and allow return to work.

Mainstay was founded in 2008 by serial entrepreneur Dr. Danny Sachs, and then under the leadership of CEO Peter Crosby, Mainstay conducted a European Feasibility Study across four leading clinics in Europe. The promise of the new therapy was clearly demonstrated in that Feasibility Study and in parallel the Company also advanced the development of its own proprietary products, and added to its intellectual property portfolio. Mainstay will use the proceeds of the Series B financing to expand the team for product development, quality, clinical, regulatory and administration, as it moves towards its goals of achieving regulatory approval in Europe and running a global clinical trial leading to a PMA submission to the FDA in the US.

Dr. Manus Rogan, Co-Founder and Managing Partner at Fountain Healthcare Partners said, “We are excited about Mainstay, and the potential to relieve the back pain suffered by millions of people worldwide. We are particularly pleased that Mainstay and the syndicate of international investors has recognised the economic value and attractiveness of Ireland as a location to build an innovative medical device company.”

Mainstay Medical Chief Executive Peter Crosby commented, “Our early clinical results are very encouraging, and with this new investment we will be able to make great progress in advancing Mainstay’s products from concept to reality, and start to build our business.” Mr. Crosby added, “The Mainstay approach has the potential to save billions of dollars on health care expenditure, while also creating a multi-billion market for us, so it is a winning situation all around.”

Antoine Papiernik, Managing Partner at Sofinnova Partners remarked, “We saw the vision of Mainstay when it was presented to us as just an idea, and we took the risk to lead the first investment. It is great to see that the company has advanced so far, so quickly, and so efficiently. We are pleased to add such quality new investors to the syndicate.”

As part of the financing Dr. Manus Rogan (Fountain) will join the Board of Directors, and Dr. Stephen Oesterle (Medtronic), Dr. Frank Bulens (Capricorn) and Iain Wilcock (Seventure) will become Observers. Existing independent director Andrew Weiss (CEO Coaxia, past head of Medtronic Neuromodulation business) will continue.

New Chairman for Irish Venture Capital Association

Dublin, 7th September, 2012: Dr Manus Rogan, managing partner and co-founder of Fountain Healthcare Partners, has been elected chairman of the Irish Venture Capital Association (IVCA). He succeeds Maurice Roche, partner in Delta Partners.

The Irish Venture Capital Association is the representative organisation for venture capital firms in Ireland. In the last ten years Irish VCs have invested €1.5bn into Irish SMEs and have attracted an additional €1.5bn from international VCs through syndication.

Dr Rogan has over 23 years investment and operating experience in the life science sector in the US, Europe and Japan. He began his career at GlaxoSmithKline in the UK. In 1996 he joined Elan Corporation where he concluded over 25 investment and technology licensing transactions globally. He currently serves on the board of Opsona Therapeutics and until recently represented Fountain Healthcare Partners on the Amarin Corporation board.

Dr Rogan holds a PhD in chemistry from the University of York (sponsored by GlaxoSmithKline) and an MBA from Trinity College Dublin.

Civitas Therapeutics Appoints Bryan E. Stuart Chief Business Officer

Industry Veteran Brings Diverse Strategic and Transaction Experience, Further Strengthens Management Team

Chelsea, Mass. – September 5, 2012 – Civitas Therapeutics, a privately-held pharmaceutical company developing inhaled therapeutics with the clinically-proven ARCUS(TM) dry powder platform, today announced that Bryan Stuart has been appointed Chief Business Officer. In this role Mr. Stuart will lead the company’s corporate development and business development efforts, and he will serve as a member of the company’s executive leadership team.

“We are very pleased to have Bryan join our team,” said Glenn Batchelder, Chief Executive Officer of Civitas Therapeutics. “His strong track record in building valuable product portfolios and establishing successful partnerships through creative transactions will accelerate our efforts to develop important therapies for patients.”

“The transformative therapeutic potential of Civitas’ lead program, CVT-301, for Parkinson’s disease illustrates the power and capabilities of the ARCUS(TM) platform as a product engine,” said Mr. Stuart. “The clinical validation of this technology combined with the existing commercial manufacturing infrastructure uniquely positions Civitas to develop multiple highly differentiated products in a rapid and capital efficient way. I am very excited to join this team in their commitment to improving patients’ lives.”

Prior to joining Civitas, Mr. Stuart led the business and corporate development activities for Ovation Pharmaceuticals and EKR Therapeutics. While at Ovation, Mr. Stuart oversaw numerous transactions involving specialty CNS therapies used for disorders such as Huntington’s disease, refractory epilepsy and infantile spasms. Ovation was sold to H. Lundbeck A/S in 2009 and EKR was sold to Cornerstone Therapeutics earlier this year. He began his career as a healthcare investment banker with William Blair & Company. Mr. Stuart holds a B.S. degree in Finance from the University of Illinois at Urbana-Champaign and an MBA from Northwestern University's Kellogg School of Management.

About Civitas Therapeutics

Civitas is a privately-held pharmaceutical company focused on developing a robust pipeline of inhaled therapeutics with the clinically proven ARCUS(TM) dry powder platform. The company’s lead program for Parkinson’s disease, CVT-301, is intended to treat intermittent and debilitating motor fluctuations resulting from an inadequate response to patients’ standard oral medications. CVT-301 is currently enrolling a Phase 2 dose ranging study following the completion of a successful Phase 1 study which established clinical pharmacokinetic proof-of-concept at end of 2011.The product portfolio includes additional programs in respiratory disease, CNS disorders and infectious disease.

Civitas acquired exclusive rights to the technology and assets underlying the ARCUS(TM) platform from Alkermes plc, including a large intellectual property estate, a set of development stage pipeline assets, specialized equipment for respiratory products and the commercial scale GMP manufacturing facility. Civitas was launched at the beginning of 2011 with Canaan Partners, Fountain Healthcare Partners, Longitude Capital and Alkermes as investors and has additionally been awarded a grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF).

Contacts

Civitas Therapeutics
Stephanie Gillis, 617-660-4121
sgillis@civitastherapeutics.com

Irish Medical Device Company, Neuravi Completes €5.2m Series A Financing

Galway, Ireland, 11th July, 2012 – Neuravi Limited today announced that is has successfully completed a Series A financing round for €5.2m (US$6.5m) led by Fountain Healthcare Partners and Delta Partners. This funding will accelerate the on-going development of Neuravi’s ground breaking clot retrieval device for the treatment of acute ischemic stroke and will enable Neuravi to undertake clinical evaluation in patients. Established in 2009, Neuravi has assembled a large body of intellectual property in the field of mechanical retrieval devices for stroke and has positioned itself to be a major strategic player in this fast developing market.

Patients suffer an estimated 700,000 ischemic strokes in the US and a further 950,000 in Europe each year. Neuravi is developing a medical device to help stroke doctors treat patients very quickly after stroke onset by removing the lodged blood clot from the brain with its proprietary Stent Basket technology. A staggering 95% of patients currently receive no acute intervention for this often deadly or very debilitating condition, relying on rehabilitation only. The potential market could be as high as $2 billion per year following successful clinical trial completion and the products being approved for sale in major markets.

Commenting on the new funding, Dr Joe Mason at Delta Partners said, “Stroke is devastating to patients and their families. Its results impose huge burdens on society. Neuravi’s therapeutic device will potentially offer significant benefits to patients, clinicians and payors by improving treatment outcomes for large numbers of people who suffer from the condition.”

Commenting on their investment in Neuravi, Justin Lynch, Partner at Fountain Healthcare Partners said, “This talented Galway team has a successful track record in miniature endovascular product development from concept design to clinical approval and international commercialization. The neuro-vasculature is especially challenging because brain arteries are miniscule, delicate and tortuous and this team is on a path to developing the best in class thrombectomy device addressing what is set to become a multi-billion dollar market worldwide”.

Neuravi Chief Executive Eamon Brady commented, “We are delighted to welcome Fountain Healthcare Partners and Delta Partners as investors and appreciate the extensive industry expertise they bring onto the Board. We would also like to take the opportunity to thank Enterprise Ireland, PanEuro Technology Ventures and The Western Development Commission for the support they have provided Neuravi to date and their on-going commitment to the Company and its development of a novel therapeutic device for Ischemic Stroke”.

Neuravi expects to create 25 new jobs over the next three years as a result of this investment. This includes a series of both senior management and technical/functional hires over the coming months to fulfill the Research & Development, Quality Assurance, Regulatory Assurance, Manufacturing, Marketing and Finance functions of the company.

Civitas Therapeutics Initiates Phase 2a Clinical Study of CVT-301, an Inhaled L-dopa for Parkinson’s Disease

Chelsea, MA – June 15, 2012 – Civitas Therapeutics, Inc., a privately-held pharmaceutical company developing transformative therapeutics using the ARCUS(TM) respiratory delivery platform, announced today the initiation of a Phase 2a clinical trial in Parkinson’s disease patients evaluating CVT-301, an inhaled formulation of levodopa (L-dopa), for the rapid relief from motor fluctuations.  CVT-301 provides immediate onset of a large and precise dose of L-dopa. 

“Consistent with our commitment to rapidly develop important new therapies for patients, we demonstrated pharmacokinetic proof-of-concept for CVT-301 in less than 12 months from launching Civitas and are now initiating this Phase 2a study in Parkinson’s patients,” said Dr. Martin Freed, Chief Medical Officer and co-founder of Civitas. “Leveraging the ARCUS platform along with the 40 years of existing L-dopa clinical experience we hope to provide Parkinson’s patients with a new therapy enabling improved management of their motor fluctuations.”   

The Phase 2a study is a randomized, placebo-controlled, single dose, cross-over design that will characterize the safety and tolerability of CVT-301 and evaluate pharmacodynamic effects and L-dopa pharmacokinetics in patients with Parkinson’s disease with motor fluctuations (“off episodes”).  This Phase 2a study is designed to establish the dose for future clinical trials with CVT-301. Patients will receive oral Sinemet®, inhaled placebo and CVT-301 which will be followed by serial evaluations of L-dopa pharmacokinetics, motor response and safety at each visit. Twenty-four (24) patients will be enrolled. 

This Phase 2a study of CVT-301 is funded in part by a grant from The Michael J. Fox Foundation for Parkinson’s Research. 

About CVT-301

Civitas’ lead program, CVT-301, is an inhaled formulation of L-dopa for the immediate relief from debilitating motor fluctuations associated with Parkinson’s disease.  For symptomatic relief, oral L-dopa is administered to maintain dopamine levels in the brain above the therapeutic threshold; yet the efficacy of oral L-dopa is significantly compromised by delayed absorption and excessive variability in the circulating plasma drug concentrations inherent to the oral delivery route.  CVT-301 is an ARCUS(TM) therapeutic that incorporates L-dopa and is optimized to deliver a precise dose to the deep lung for rapid and predictable L-dopa absorption.   The ARCUS(TM)  platform is uniquely able to deliver the necessary L-dopa dose with the required precision.  CVT-301 is being developed as an adjunct to standard oral L-dopa therapy to enable patients to manage motor fluctuations caused in part by the inter-dose variability of oral L-dopa.  In preclinical models, CVT-301 has demonstrated rapid, durable symptomatic relief, even when compared to larger doses of oral L-dopa.   The Phase 1 clinical study is complete, and pharmacokinetic proof-of-concept was demonstrated. Therapeutic plasma levels of L-dopa were achieved within five minutes of inhalation dosing with unprecedented precision. Dose proportional pharmacokinetics were seen across all doses tested. In addition, all doses tested of CVT-301 were safe and well tolerated.  

About Parkinson’s Disease

Over one million people in the US suffer from Parkinson’s disease, a neurodegenerative disorder caused by the diminished production of dopamine, a key neurotransmitter, resulting in progressive impairment of motor function including tremors, rigidity and difficulty in moving. Even when treated with the current standard of care, the majority of Parkinson’s patients continue to experience motor fluctuations.  These motor fluctuations reduce patients’ ability to lead productive, independent lives and are recognized by patients, care givers and healthcare professionals as one of the most troubling and debilitating issues associated with the disease.   

About Civitas Therapeutics

Civitas is a privately-held pharmaceutical company focused on developing a robust pipeline of inhaled therapeutics with the clinically proven ARCUS(TM)  dry powder pulmonary delivery platform.  The company’s lead program for Parkinson’s disease is intended to treat intermittent and debilitating motor fluctuations resulting from an inadequate response to their standard oral medications.  Additional programs encompass respiratory disease, central nervous system disorders and infectious disease.  Civitas exclusively licensed and purchased the technology and assets underlying the ARCUS(TM) platform from Alkermes plc, including a large intellectual property estate, a set of development stage 

pipeline assets, specialized equipment for respiratory products and the commercial scale GMP manufacturing facility.  Civitas was launched at the beginning of 2011 with Canaan Partners, Fountain Healthcare Partners, Longitude Capital and Alkermes as investors.

Opsona Therapeutics announces the issuance of new patent from the European Patent Office (EPO) covering the development and use of an antibody directed against Toll-like Receptor TLR-2

April 17th, 2012, Dublin, Ireland - Opsona Therapeutics, the innate immune drug development company, today announced that the European Patent Office has issued EP Patent 1,664,118 which covers an antibody directed against Toll-like Receptor-2 (TLR-2) and the use and development thereof.

TLR-2 plays an important role in the induction and progression of a number of non-pathogen associated inflammatory conditions including ischemia reperfusion injury (delayed graft function in renal transplantation, myocardial infarct), certain cancer, autoimmune diseases, diabetes, Alzheimer's disease and atherosclerosis.

TLR-2 is one of the key structures of the innate immune system and is part of the first line defense against microbial organisms. Upon stimulation it induces and propagates inflammation. TLR-2 is activated through so called external danger signals (microbial cell wall components) as well as through so called internal danger signals resulting from tissue injury.

This patent describes a cross reactive antibody which specifically blocks mammalian TLR-2 and further provides for a pharmaceutical composition for the treatment of various inflammatory conditions. The recently issued patent is assigned to the Technische Universitat Munchen (TUM) and Amgen Inc, and is exclusively licensed by Opsona Therapeutics.

Using the TUM/Amgen license, Opsona has developed a clinical anti-TLR-2 antibody candidate, termed ‘OPN-305'. OPN-305 is a humanised IgG4 monoclonal antibody (MAb) antagonizing TLR-2 and is under development as a treatment for the prevention of Delayed Graft Function (DGF) following renal transplantation, in addition to other therapeutic indications.

Opsona has successfully conducted a phase 1 clinical trial in healthy volunteers with its lead drug candidate OPN-305. This is the first-in-human study with OPN-305 and also represents the first clinical study for an anti-TLR-2 drug candidate.

Following successful completion of the phase 1 trial, the company plans to conduct a two-part multi-centered, double blinded and placebo controlled clinical study to evaluate the safety, tolerability and efficacy of OPN-305 in renal transplant patients at high risk of Delayed Graft Function (DGF) as the first clinical target indication for the development of OPN-305 to be initiated in 2012.

Commenting on today's announcement, Mary Reilly VP Pharmaceutical Development and Operations of Opsona Therapeutics said, "The issuance of this patent is an important milestone in the development of Opsona's TLR2 intellectual property portfolio and will facilitate market exclusivity for the use of OPN-305 in the ever expanding area of TLR2 mediated diseases."

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 autoimmune and inflammatory diseases, transplant rejection, cancer, diabetes, Alzheimer's disease and atherosclerosis. The company was founded in 2004 by three world-renowned immunologists at Trinity College in Dublin. Opsona's lead product, a fully human monoclonal IgG4 antibody (OPN-305) targeting Toll-like-receptor-2 (TLR-2) has demonstrated activity in a number of animal models and was recently tested in a phase 1 clinical trial in healthy volunteers. Following successful completion of the phase 1 trial, the company plans to conduct a two-part multi-centered, double blinded and placebo controlled clinical study to evaluate the safety, tolerability and efficacy of OPN 305 in renal transplant patients at high risk of Delayed Graft Function (DGF) as the first clinical target indication for the development of OPN-305 to be initiated in 2012. In May 2009 the company announced the completion of a € 21.3 million equity funding with an international investor consortium including: Inventages Venture Capital, Novartis Venture Fund, Roche Venture Fund, Seroba Kernel Life Sciences, Fountain Healthcare Partners and Enterprise Ireland. Further information is available at http://www.opsona.com/.

For further information please contact:

Martin Welschof (CEO)
Telephone: + 35316770223
E-mail: mwelschof@opsona.com

Civitas Therapeutics Announces Award of Michael J. Fox Foundation Grant and Lead Drug Candidate for Parkinson’s Disease

CVT-301 Offers Inhaled Administration of Levodopa for More Rapid and Consistent Dosing to Treat Motor Fluctuations in Parkinson’s Disease

Company Plans to Initiate Clinical Study of CVT-301 by End of 2011

Chelsea, MA - November 29, 2011 – Civitas Therapeutics, Inc., a privately-held pharmaceutical company developing transformative therapeutics using the ARCUSTM respiratory delivery platform, announced today the award of a grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF).  In addition, Civitas revealed today that the company’s lead drug candidate for Parkinson’s disease is CVT-301, an inhaled formulation of levodopa (L-dopa).  The grant from MJFF will support the clinical development of CVT-301, which has the potential to produce rapid, consistent and durable relief from debilitating motor fluctuations associated with Parkinson’s disease.

“We are proud to be recognized by The Michael J. Fox Foundation with this award,” said Glenn Batchelder, Chief Executive Officer of Civitas. “This provides important validation of our vision to improve Parkinson’s patients’ lives with an inhaled L-dopa therapeutic by overcoming the historical challenges in developing a better way to administer L-dopa.” 

“Our Foundation believes the challenges associated with L-dopa delivery represent a critical unmet need in Parkinson’s disease,” said Todd Sherer, Ph.D., Chief Executive Officer of MJFF. “We are optimistic that CVT-301’s novel approach could provide an opportunity to improve the standard of care for those living with the disease.”

Civitas has conducted a range of preclinical studies demonstrating CVT-301’s ability to deliver more rapid and consistent systemic exposure of L-dopa compared to oral administration.  CVT-301 was also shown to achieve more rapid, durable motor function restoration in animal models of Parkinson’s disease in comparative studies with oral L-dopa, providing evidence that CVT-301 has the potential to effectively address motor fluctuations in patients.

“Patients with Parkinson’s disease face the constant challenge of maintaining adequate therapeutic L-dopa levels which is difficult using the existing oral L-dopa regimens. CVT-301 shows promise as an important new treatment option for patients managing their symptoms,” said Matthew Stern, M.D., Director, Parkinson's Disease and Movement Disorders Center, University of Pennsylvania Health System, and a member of the Civitas Scientific Advisory Board.

The grant from MJFF provides support for CVT-301 clinical studies through proof-of-concept which is anticipated to be complete by the end of 2012.  Clinical trials of CVT-301 are planned to begin in 2011.

About CVT-301

Civitas’ lead program, CVT-301, is an inhaled formulation of L-dopa for the treatment of debilitating motor fluctuations associated with Parkinson’s disease.  For symptomatic relief, oral L-dopa is administered to maintain dopamine levels in the brain above the therapeutic threshold; yet the efficacy of oral L-dopa is significantly compromised by delayed absorption and excessive variability in the circulating plasma drug concentrations inherent to the oral delivery route.  CVT-301 is an ARCUSTM therapeutic that incorporates L-dopa and is optimized to deliver a precise dose to the deep lung for rapid, predictable and consistent L-dopa absorption.   The ARCUSTM platform is uniquely able to deliver the necessary L-dopa dose with the required precision.  CVT-301 is being developed as an adjunct to standard oral L-dopa therapy to enable patients to manage motor fluctuations caused by the inter-dose variability of oral L-dopa.  In preclinical models, CVT-301 has demonstrated immediate and consistent increases in L-dopa peak plasma concentration providing rapid, durable symptomatic relief, even when compared to larger doses of oral L-dopa.  

About Parkinson’s Disease

Over one million people in the US suffer from Parkinson’s disease, a neurodegenerative disorder caused by the diminished production of dopamine, a key neurotransmitter, resulting in progressive impairment of motor function including tremors, rigidity and difficulty in moving. Even when treated with the current standard of care the majority of Parkinson’s patients still experience motor fluctuations.  These motor fluctuations reduce patients’ ability to lead productive, independent lives and are recognized by patients, care givers and healthcare professionals as one of the most troubling and debilitating issues associated with the disease.  

About ARCUSTM Platform

The ARCUSTM inhalation technology delivers a reliable and consistent drug dose with a compact, breath actuated inhaler.  The ARCUSTM platform uses a proprietary dry powder and inhaler combination that is unique in its ability to deliver a large, precise dose independent of inspiratory flow rate from a simple, easy-to-use device suitable for convenient self-administration.  The platform has successfully delivered more than one million doses to patients incorporating active agents ranging from small molecules to large proteins, and has been scaled up to accommodate a commercial product launch. 

About Civitas Therapeutics

Civitas is a privately-held pharmaceutical company focused on developing a robust pipeline of inhaled therapeutics with the clinically proven ARCUSTM dry powder pulmonary delivery platform.  The company’s lead program is for Parkinson’s disease with clinical proof of concept anticipated to be complete in 2012.  Additional programs encompass respiratory disease, central nervous system disorders, and infectious disease.  Civitas exclusively licensed and purchased the technology and assets underlying the ARCUSTM platform from Alkermes, including a large intellectual property estate, a set of development stage pipeline assets, the specialized pulmonary equipment and the commercial scale GMP manufacturing facility.  Civitas’ investors are Canaan Partners, Fountain Healthcare Partners, Longitude Capital, and Alkermes.

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

As the world’s largest private 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 $270 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 groundbreaking 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. Now through December 31, 2012, all new and increased giving to The Michael J. Fox Foundation, as well as gifts from donors who have not given since 2009 or earlier, will be matched on a dollar-for-dollar basis with the $50-million Brin Wojcicki Challenge, launched by Sergey Brin and Anne Wojcicki.

For additional information contact:

Stephanie Gillis
Civitas Therapeutics
sgillis@civitastherapeutics.com

Ireland’s Genable Technologies Completes €5M Series B Financing

DUBLIN, Ireland, 11th November, 2011 - Genable Technologies (Dublin, Ireland), today announced that is has successfully completed a new financing round for €5m led by new investors Fountain Healthcare Partners alongside existing investors Delta Partners.  This funding will support the on-going development of Genable’s suppression/replacement gene therapy technology and specifically progress Genable’s lead product GT038 for Retinitis Pigmentosa (RP) forward into clinical evaluation in man.

Patients with rhodopsin-linked Retinitis Pigmentosa have a mutation in the rhodopsin gene, which causes a patient’s sight to worsen over time, eventually leading to blindness.  There are currently no available therapies for Retinitis Pigmentosa.  GT038 is a pioneering and unique therapy to treat rhodopsin-linked Retinitis Pigmentosa that utilizes AAV vectors to obtain expression of RNA interference molecules, which suppress the expression of the faulty gene, and replaces this with a gene encoding a functioning protein. This simple combination represents a new paradigm in medicine with the potential to cure this debilitating disease and not just treat the symptoms. Genable Technologies Ltd, was granted orphan drug designation for GT038 by the European Medicines Agency in December 2010.

Commenting on the new funding Dr. Ena Prosser, Partner at Fountain Healthcare Partners said, “This renowned Trinity College-based team, along with the dedication and support of Irish families who carry genes which can lead to inheritable blindness, has pioneered research into this disease over several years. We believe that GT038 offers a significant technological breakthrough to address Retinitis Pigmentosa and we look forward to working closely with the Company to accelerate the development of GT038 and other products to the market.”

Genable Chairman Dr. Geoffrey Vernon commented, “We would like to welcome Fountain Healthcare Partners as an investor with extensive industry expertise onto the Board. We would also like to take the opportunity to thank Delta Partners, Fighting Blindness Ireland, Foundation Fighting Blindness-National Neurovision Research Institute (USA) and Enterprise Ireland for the support they have provided Genable to date and their on-going commitment to the Company and its development of novel therapies for serious ophthalmic diseases.”

Professor Jane Farrar of Trinity College Dublin and co-Founder of Genable Technologies Limited concluded, “We are extremely pleased to see GT038 raise the necessary finance to translate basic research performed at TCD into the clinic.  It will help raise awareness of Retinitis Pigmentosa as a serious disease and ultimately help more patients receive therapy for their disease”.

Amarin Announces NDA Submission for AMR101 for the Treatment of Patients with Very High Triglycerides

BEDMINSTER, N.J., and DUBLIN, Ireland, Sept. 26, 2011 – Amarin Corporation plc (NASDAQ: AMRN), a clinical-stage biopharmaceutical company with a focus on cardiovascular disease, today announced that it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking approval for the marketing and sale of AMR101 for treatment of patients with very high triglycerides. The submission is based on the entire data set from the Company’s AMR101 development program, including safety and efficacy data from the Phase 3 MARINE and ANCHOR studies.  

“This is another significant milestone achieved for Amarin. Data from our two pivotal Phase 3 studies show that, unlike other triglyceride-lowering therapies, AMR101 does not increase LDL-cholesterol and, in certain cases, significantly decreases it,” said Joseph S. Zakrzewski, Chairman and Chief Executive Officer of Amarin. “The submission of this NDA moves AMR101 one step closer to commercial launch. If AMR101 is approved, we believe it can play a significant role in cardiovascular health management.” 

It is estimated that 75 million people in U.S. alone have triglyceride levels greater than 150mg/dL, including 4 million people with very high triglyceride levels (the triglyceride range studied in the MARINE trial) and 36 million people with high triglyceride levels (the triglyceride range studied in the ANCHOR trial).  Elevated triglycerides are clinically stratified into three groups: very high triglycerides (>500 mg/dL), high triglycerides (>200 and <500 mg/dL) and borderline high triglycerides (>150 and <200 mg/dL).  Clinical treatment guidelines include recommendations for triglyceride reductions in each of these groups and each group represents a multi-billion dollar market opportunity.  In the top seven world markets it is estimated that the number of people with elevated triglyceride levels is at least two times that of the U.S. alone. 

The treatment of patients with very high triglycerides was studied in the Company’s MARINE trial.  The treatment of patients with high triglycerides on statin therapy was studied in the Company’s ANCHOR trial.  Amarin plans to separately seek approval for the treatment of high triglycerides in patients on statin therapy (the population studied in the ANCHOR trial) after its REDUCE-IT cardiovascular outcomes trial is substantially underway, which the Company expects will occur before the end of 2012 (final results of the REDUCE-IT outcomes study are not required for approval of the very high triglyceride indication). 

In both the MARINE and ANCHOR trials, AMR101 achieved all primary endpoints and was well tolerated with a safety profile comparable to placebo. Each trial was conducted under a Special Protocol Assessment (SPA) agreement from the FDA.  As recently announced, an SPA agreement was also reached for the REDUCE-IT cardiovascular outcomes study. 

About AMR101

AMR101 is a prescription-grade omega-3 fatty acid, comprising not less than 96% ultra pure EPA(icosapent ethyl), that Amarin is developing as a potentially best-in-class prescription medicine for the treatment of patients with very high triglyceride levels (>500 mg/dL) and as a potentially first-in-class therapy for patients with high triglyceride levels (>200 and <500mg/dL) who are also on statin therapy for elevated LDL-cholesterol levels (which we refer to as mixed dyslipidemia). Triglycerides are fats in the blood. Significant scientific and clinical evidence support the efficacy and safety of ethyl-EPA in reducing triglyceride levels and other important lipid and inflammation biomarkers, including Apo-B, non-HDL-C, Total-Cholesterol, VLDL-C, Lp-PLA2, and hs-CRP without increasing LDL-C. AMR101 demonstrated a safety profile comparable to placebo in two complete Phase 3 clinical trials.

Amarin Announces Agreement From FDA On Special Protocol Assessment For Amr101 Outcomes Study

Study Positions AMR101 to Potentially Address Patient Populations of More Than 70 Million in the U.S. Alone

MYSTIC, Conn. and DUBLIN, Ireland, Aug. 10, 2011 (GLOBE NEWSWIRE) -- Amarin Corporation plc (Nasdaq:AMRN), a clinical-stage biopharmaceutical company with a focus on cardiovascular disease, today announced that it has reached agreement with the U.S. Food and Drug Administration (FDA) on a Special Protocol Assessment (SPA) agreement for the design of the previously described cardiovascular outcomes study of AMR101 formally titled REDUCE-IT (Reduction of Cardiovascular Events with EPA - Intervention Trial). Amarin previously announced that it achieved the primary endpoints of two Phase 3 studies of AMR101, both of which were conducted under separate SPAs.

In REDUCE-IT, Amarin will evaluate the effectiveness of AMR101 in reducing the first major cardiovascular events in an at-risk patient population. The control arm of the study will be patients on optimized statin therapy. The active arm of the study will be patients on optimized statin therapy plus AMR101. All subjects enrolled in the study will have elevated triglyceride levels and either coronary heart disease or risk factors for coronary heart disease. Amarin will be responsible for the study which will be conducted internationally. The Company will use an experienced clinical research organization (CRO) to help manage the study and is in the late stages of contract negotiations with a leading CRO for that purpose.

Consistent with prior comments, Amarin estimates that the study will require approximately 8,000 patients and take approximately 6 years for completion. The Company anticipates that if, as intended, it commences Outcomes study activities in 2011 that it will be positioned to achieve approximately 50% enrollment before the end of 2012.

Once REDUCE-IT is substantially underway, the Company believes that it will have met all of the requirements to request approval of AMR101 for treating the mixed dyslipidemia patient population studied in the ANCHOR trial. AMR101 is positioned to be the first drug in its class approved for treatment of this indication. Upon completing REDUCE-IT, and assuming a successful result, Amarin anticipates being able to pursue an indication for the prevention of cardiovascular events; this population is estimated to be greater than twice the size of the combined indications studied in the MARINE and ANCHOR trials. The Company also anticipates that, similar to ANCHOR, a significant number of the patients in REDUCE-IT will have diabetes.

"We are delighted to have finalized the protocol for REDUCE-IT and to have the FDA agree to this via a Special Protocol Assessment, our third SPA for AMR101, which is remarkable," stated Joseph Zakrzewski, Amarin's Executive Chairman and CEO. "Based on the strong safety profile of AMR101, our positive Phase 3 results for AMR101 and success in Japan with an outcomes study of highly pure EPA, we believe that REDUCE-IT is positioned for success." Mr. Zakrzewski added, "The design of REDUCE-IT reflects the diligent evaluation of numerous other outcome studies by our clinical team, advisors and other interested parties all of whom are commended and thanked for their contributions to the very direct and efficient design of this study."

Civitas Therapeutics Secures $5M in Financing - Accelerating Parkinson’s disease program

Chelsea, MA - July 12, 2011 – Civitas Therapeutics, Inc., a privately held biopharmaceutical company focused on developing transformative pulmonary delivery therapies, announced today that it has secured $5 million of financing from Fountain Healthcare Partners. This additional capital will be primarily used to accelerate Civitas’ lead therapeutic program for Parkinson’s disease as well as to begin advancing additional drug development programs.

“We are excited to add Fountain Healthcare Partners as an investor with their extensive drug delivery technology experience and global industry expertise,” said Glenn Batchelder, Chief Executive Officer of Civitas. “The additional capital will allow us to more rapidly advance this important therapy for Parkinson’s patients.”

“We believe Civitas’ validated and highly differentiated pulmonary delivery technology represents a unique platform to develop transformative therapies for a number of unmet medical needs, currently un- addressable with existing platforms. The company’s initial Parkinson’s program is a prime example of this potential.” said Aidan King, Founding and Managing Partner of Fountain Healthcare. “The remarkable progress made by the company in its initial six months of operation is testament to the quality of the Civitas team and augers well for their ability to deliver these products to the market place.”

Aidan King will join Glenn Batchelder, David Hirsch of Longitude Capital, Blair Jackson of Alkermes, and Tim Shannon of Canaan Partners on the Board of Directors for Civitas.

About Civitas Therapeutics

Civitas is a privately held pharmaceutical company focused on developing products with a clinically proven dry powder pulmonary delivery platform. The proprietary dry powder and device combination is unique in its ability to deliver a large, precise dose independent of inspiratory flow rate from a simple, passive device. The technology has successfully delivered more than one million doses to patients and the manufacturing technology has been scaled to accommodate a significant commercial launch. The lead program is for Parkinson’s disease with clinical proof of concept anticipated to be complete in 2012. Additional programs encompass pulmonary disease, allergy, and pain.

Civitas licensed and purchased the technology and assets from Alkermes which includes a large IP estate, a set of development stage pipeline assets, the specialized pulmonary equipment and the commercial scale GMP manufacturing facility. Civitas’ investors are Canaan Partners, Fountain Healthcare Partners, Longitude Capital, and Alkermes.

For additional information contact:

Stephanie Gillis
sgillis@civitastherapeutics.com

Website
http://www.civitastherapeutics.com