Spark Therapeutics and Genable Technologies Announce Collaboration to Advance a Gene Therapy Treatment for a Rare Form of Retinitis Pigmentosa

PHILADELPHIA -  Spark Therapeutics and Genable Technologies announced today that they have entered into a collaboration agreement for Genable's lead therapeutic to treat rhodopsin-linked autosomal dominant retinitis pigmentosa (RHO adRP), GT038. Under the terms of the collaboration, Genable will license certain adeno-associated virus (AAV) vector manufacturing patents from Spark. The parties have entered into a broad agreement in which Spark will be the exclusive manufacturer of the product and provide development advice and expertise to Genable to help in the ongoing development of GT038. Spark will receive milestone payments and royalties on future sales of GT038, as well as near-term revenue from the manufacture and supply of product.

"We are excited to apply our deep expertise in AAV clinical development and manufacturing to augment Genable's great work, and expand the number of debilitating diseases of the eye that can be addressed through gene therapy," said Jeffrey D. Marrazzo, Spark Therapeutics co-founder, president and CEO.

Dr. Jason Loveridge, CEO of Genable Technologies commented, "We have chosen Spark as our partner to advise, lend their experience and manufacture GT038 based on their broad expertise in gene therapy. We see them as a world-class organization, and we are excited to be advancing our novel therapy GT038 into the clinic."

"The collaboration with Spark provides an exciting opportunity to greatly expedite development of Genable's novel therapy targeted towards RHO-adRP," said Professor Jane Farrar, founder and director, Genable Technologies; professor, Trinity College (Dublin).

GT038 is a potential treatment for rhodopsin (RHO)-linked autosomal dominant retinitis pigmentosa (adRP), an inherited retinal dystrophy that leads to blindness in most cases. There is currently no approved pharmacologic treatment for adRP, which affects an estimated 30,000 patients worldwide. GT038 utilizes AAV vectors with an established safety and efficacy profile to deliver RNA interference (RNAi) molecules to suppress the expression of faulty and normal copies of RHO and restore normal gene expression. GT038 has been granted Orphan Drug Designation in both the U.S. and Europe.

About Genable Technologies

Genable Technologies Ltd. is a privately held, venture-capital-backed, Dublin (Ireland)- based bio-pharmaceutical company. The company is developing new gene therapies to treat "dominant" genetic diseases. The company has received significant support and investment form Fountain Healthcare Partners, Delta Partners, Fighting Blindness Ireland, Foundation Fighting Blindness Clinical Research Institute (U.S.) and Enterprise Ireland. To learn more please visit www.genable.net

About Spark Therapeutics

Spark Therapeutics is developing potentially curative, one-time gene therapy products to transform the lives of patients and re-imagine the treatment of debilitating diseases. Spark's lead gene therapy candidate, for RPE65-related blindness, is currently in Phase 3 clinical trials with the potential to be the first approved gene therapy in the U.S., and the first treatment to address the significant unmet needs of patients living with blindness due to inherited retinal dystrophies.

Spark's founding team includes scientists who led the movement to develop gene therapy as a new treatment paradigm, establishing clinical proof of concept in the eye and liver and contributing key insights to the field that have resulted in a resurgence of industry interest in gene-based medicines. In addition to the Phase 3 program in RPE65-related blindness, the company has a Phase 1/2 program in hemophilia B, and preclinical programs to address neurodegenerative diseases and other inherited retinal dystrophies and hematologic disorders. Spark has rights to a proprietary manufacturing platform that has an unparalleled track record of success in supporting clinical studies across diverse therapeutic areas and routes of administration. The company's expertise across research, clinical, regulatory and manufacturing builds on a legacy of innovation and excellence in gene therapy established by Spark's team while at The Children's Hospital of Philadelphia Center for Cellular and Molecular Therapeutics. To learn more visit www.sparktx.com.

Media Inquiries:

Jessica Rowlands, 202-729-4089, jessica.rowlands@fkhealth.com

Dr. Jason Loveridge + 33 674177812, jloveridge@genable.net

Mainstay Medical Begins Clinical Trial of ReActiv8® for People with Chronic Low Back Pain

Dublin based medical device company hits key milestone.

Dublin, Ireland – Mainstay Medical today announced that it has secured approval from Ethics Committees in Australia to start a clinical trial of ReActiv8, its innovative implantable neurostimulation device for the treatment of people with Chronic Low Back Pain (CLBP). Recruitment of subjects for the trial has commenced at three clinical sites in Australia.

The purpose of the clinical trial is to investigate ReActiv8 as a treatment for adults with debilitating Chronic Low Back Pain for whom surgery is not indicated.

One of the root causes of CLBP is impaired control by the nervous system of the muscles that stabilize the spine in the lower 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 spine stability, allowing the body to recover from chronic low back pain.

“Our novel approach of electrical stimulation to help restore the muscle control system is based on published scientific research, and the performance of the therapy was demonstrated in the recently completed European Feasibility Study.” said Peter Crosby, the CEO of Mainstay Medical. “The energy and experience of the Mainstay Medical team has enabled us to complete the development of our innovative, therapy-specific device and obtain approval to start the ReActiv8 clinical trial within a year after the Feasibility Study results.”

People with debilitating CLBP usually have a greatly reduced quality of life and score significantly higher on scales for disability, depression, anxiety and sleep disorders. Their pain and disability can persist despite the best available medical treatments, and most of these people have no indications for 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 utilisation put a significant burden on economies.

“Back pain specialists from all over the world have told us that they need a new approach to help the large group of people with CLBP who are stranded without a viable alternative. We believe, based on published research, that there are millions of such people in Europe and the USA today. The results of the European Feasibility Study encouraged us to believe that ReActiv8 can play an important role in helping these people,” Crosby mentioned.

Results from Mainstay Medical’s European Feasibility Study were presented at the meeting of the International Neuromodulation Society in Berlin in June 2013. Results showed a statistically significant and clinically important improvement in key outcome measures, including reduction in pain and disability from CLBP and an improved quality of life.

About Mainstay Medical Ltd

Mainstay Medical Limited is a medical device company which is developing an innovative implantable neurostimulation medical device, ReActiv8, for people with debilitating Chronic Low Back Pain (CLBP). Low Back Pain is the leading cause of activity limitation and work absence throughout much of the developed world, imposing a high economic burden on individuals, families, communities, industry, and governments.

The Company is headquartered in Dublin, Ireland and has subsidiaries operating in the United States and Australia. Mainstay Medical is backed by investors including Sofinnova Partners (France), Fountain Healthcare Partners (Ireland), Medtronic (US), Capricorn Venture Partners (Belgium), Seventure Partners (France) and Twin Cities Angels (Minneapolis, USA).

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

Media queries to:

Eilish Joyce, FTI Consulting
Tel: +353 1 663 3609 / +353 87 7914641
Email: eilish.joyce@fticonsulting.com

Paul McSharry, FTI Consulting
Tel: +353 1 663 3609 / +353 87 240 6642
Email: paul.mcsharry@fticonsutling.com

Jeanne Bariller, FTI Consulting
Tel: +33 1 47 03 6863 / +33 67 412 4452
Email: jeanne.bariller@fticonsulting.com

Mainstay Medical Strengthens Board

Dublin, Ireland – Mainstay Medical Ltd, a medical device company headquartered in Dublin, today announced the appointment of Mr David Brabazon to the Board of Directors of the company. Mr Brabazon brings a wealth of international industry knowledge and commercial expertise garnered from a twenty year career in life sciences.

Mr Brabazon’s appointment to the Board comes as Mainstay Medical continues to advance towards regulatory approval and commercialisation of its innovative medical device for the treatment of chronic low back pain. Mainstay’s ReActiv8 is a small, implantable neurostimulation device that is designed to restore spine stability to ease chronic low back pain.1

The clinical results from a European Feasibility Study were presented at the meeting of the International Neuromodulation Society in Berlin in June 2013, and showed that the therapy delivered by ReActiv8 improves symptoms of back pain, reduces disability from back pain and improves quality of life.2

Commenting on his appointment Mr Brabazon said, “I look forward to working with Mainstay Medical’s board and management team to continue the fast and efficient progress the company has made to date. More than half of us will experience back pain at some point in our lives and for many people, the disabling pain will continue for long periods. We are optimistic that ReActiv8 will make a real difference to people with chronic low back pain, and also ease the economic and social impacts that can stem from this debilitating condition”.

Welcoming Mr Brabazon’s appointment, Mainstay Medical CEO Peter Crosby said, “We are delighted to have David join our Board. He brings a wealth of commercialisation and business development experience as well as knowledge of relevant therapeutic areas. We are at a very exciting stage in our development, we continue to add to our intellectual property portfolio and we are making progress towards regulatory approval for the ReActiv8”.

About David Brabazon

A co-founder of Adapt Pharma Limited in November 2013, a US focused specialty pharmaceuticals company, Mr Brabazon currently serves as the company’s Chief Financial Officer. He previously co-founded Azur Pharma plc in 2005, where he also held the role of Chief Financial Officer. Azur Pharma was a specialty pharmaceuticals business focused on diseases of the central nervous system, pain and women’s health that was acquired by Jazz Pharmaceuticals in January 2012. After the business merged with Jazz Pharmaceuticals, Mr Brabazon served with the new entity as Senior Vice President of Finance and Group Company Secretary. Prior to Azur Pharma, he served as Vice President and Group Financial Controller at Elan Corporation plc.

About Mainstay Medical Ltd

Mainstay Medical Ltd. is a medical device company which is developing innovative neurostimulation therapies for the population of people with debilitating Chronic Low Back Pain. The Company is focused on the development of ReActiv8, an active implantable medical device designed to treat people with Chronic Low Back Pain. Low Back Pain is the leading cause of activity limitation and work absence throughout much of the developed world, imposing a high economic burden on individuals, families, communities, industry, and governments.

The Company is headquartered in Dublin, Ireland and has subsidiaries operating in the United States and Australia. Mainstay Medical Inc. was founded in 2008 in Minnesota, United States, and the headquarters of the business moved to Dublin in 2012. Mainstay Medical is backed by investors including Sofinnova Partners (France), Fountain Healthcare Partners (Ireland), Medtronic (US), Capricorn Venture Partners (Belgium), Seventure Partners (France) and Twin Cities Angels (Minneapolis, USA). Further information can be found at www.mainstay-medical.com

Media queries to:

Eilish Joyce, FTI Consulting
Tel: +353 1 663 3609 / +353 87 7914641
Email: eilish.joyce@fticonsulting.com

Paul McSharry, FTI Consulting
Tel: +353 1 663 3609 / +353 87 240 6642
Email: paul.mcsharry@fticonsutling.com

Jeanne Bariller
Tel: +33 1 47 03 6863 / +33 67 412 4452
Email: jeanne.bariller@fticonsulting.com

Civitas Therapeutics Appoints Mark Iwicki President and Chief Executive Officer

-- Company prepares for late stage development and commercialization of CVT-301--

CHELSEA, Mass. – January 29, 2014 – Civitas Therapeutics, Inc., a biopharmaceutical company with a lead program in Parkinson’s disease that utilizes the proprietary ARCUS(TM) respiratory delivery platform, today announced the appointment of Mark Iwicki as President and Chief Executive Officer. Mr. Iwicki is

an accomplished pharmaceutical executive with a successful track record leading fully integrated organizations, developing therapeutics across all stages and launching multiple successful products. Mr. Iwicki has also been elected to the Board of Directors. Mr. Iwicki succeeds Glenn Batchelder, co- founder and CEO from the company’s inception, who will remain an active member of the Board of Directors.

“Having successfully launched numerous products in the pulmonary and central nervous system (CNS) therapeutic areas, Mark is uniquely qualified to direct the advancement of Civitas’ lead program CVT- 301, for Parkinson’s disease, into late stage clinical development,” said Tim Nelson, Chairman of the Board of Directors. “Mark’s proven ability to lead high performance teams from early development through commercialization will help achieve the Civitas mission to build a robust and sustainable pipeline around the ARCUS(TM) platform. The Board would like to thank Glenn Batchelder for his leadership and contributions to Civitas’ remarkable success to date.”

Mr. Iwicki has more than 24 years of experience as a pharmaceutical industry leader across multiple therapeutic areas. He has extensive experience building brands and has been instrumental in the success of a number of drugs, including Prilosec®, Diovan®, Zelnorm®, Lunesta®, and Latuda®. Prior to joining Civitas, Mr. Iwicki was CEO of Blend Therapeutics and CEO of Sunovion Pharmaceuticals, which was created after the acquisition of Sepracor by Dainippon Sumitomo Pharmaceuticals. At Sepracor, Mr. Iwicki served as Chief Commercial Officer for the company, launching three products and managing all aspects of the company’s portfolio. Prior to joining Sepracor in 2007, he was the senior vice president and head of the cardiovascular business unit at Novartis Pharmaceuticals. He began his career with management positions of increasing responsibility at Astra Merck and Merck.

“It is a great privilege to join the Civitas team during this pivotal period and fully capitalize on the unique capabilities of the ARCUS(TM) technology," said Mr. Iwicki. “Civitas unites a highly differentiated and clinically validated platform, a fully-operational commercial manufacturing facility, a lead program addressing a significant unmet need for Parkinson’s patients and an opportunity to produce transformative therapies.”

"Mark shares the Civitas commitment to make a difference in patients' lives," said Mr. Batchelder. "He is dedicated to helping Civitas become a leading biopharmaceutical company and is the ideal candidate to direct the company during this next phase of success."

About Civitas Therapeutics

Civitas Therapeutics 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. The company’s lead program, CVT-301, is being developed as adjunctive, on-demand (PRN) therapy to provide Parkinson’s disease patients with rapid and reliable relief from intermittent debilitating motor fluctuations (OFF episodes). OFF episodes result from the unreliability of current medications -- a problem impacting about half of all Parkinson’s disease patients. In April 2013, Civitas announced positive results for CVT-301 in a Phase 2 dose-ranging study. The company has completed enrollment of a Phase 2b study to evaluate the efficacy and safety of CVT-301 in treating emergent OFF episodes during one month of continued use, with data expected by the end of the first quarter of this year. The Company’s pipeline also includes products for respiratory disease, central nervous system disorders and infectious disease.

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 and is protected by a large intellectual property estate. 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 Company is financed by leading investors including Alkermes plc, Bay City Capital, Canaan Partners, Fountain Healthcare Partners, Longitude Capital and RA Capital.

For further information on Civitas, please visit www.civitastherapeutics.com.

For additional information contact:

Stephanie Gillis Maureen L. Suda (Media)
Civitas Therapeutics Suda Communications LLC
sgillis@civitastherapeutics.com 585-387-9248

Civitas Therapeutics Appoints Timothy S. Nelson as Chairman of the Board of Directors

-Strengthens team as CVT-301 Phase 2b trial completes patient enrollment -

CHELSEA, Mass. – January 8, 2014 – Civitas Therapeutics, Inc., a biopharmaceutical company with a lead program in Parkinson’s disease that utilizes the proprietary ARCUS(TM) respiratory delivery platform, today announced the appointment of Timothy S. Nelson as Chairman of the Board of Directors. Civitas also announced today that it has completed enrollment in its ongoing Phase 2b clinical study for its lead compound CVT-301, with data expected by the end of the first quarter of this year. CVT-301 is being developed as an adjunct, as needed (PRN) therapy to potentially provide rapid and reliable relief from intermittent debilitating motor fluctuations (OFF episodes) that affect many Parkinson’s disease patients.

“Tim brings an exceptional track record of transformative leadership and company building across a broad range of therapeutic and medical device domains. His recent experience leading MAP Pharmaceuticals, initially as a privately-held platform company and ultimately as a publicly-traded late stage development company will be particularly relevant in helping to guide Civitas’ continued growth,” said Glenn Batchelder, Chief Executive Officer and Co-Founder of Civitas. “With our shared focus on improving patients’ lives and his proven talent for building shareholder value, we are pleased to welcome Tim as Chairman of the Board.”

“Civitas has a highly-validated drug inhalation platform, a potentially transformative Parkinson’s therapy with important upcoming clinical data and a management team with an exceptional record of execution. This unique combination provides a strong foundation to build a company with a robust portfolio of differentiated therapies," said Mr. Nelson. “I am delighted to be joining the Civitas Board and look forward to supporting the team as they deliver on the company’s remarkable promise.”

Mr. Nelson has more than 20 years of experience with drug delivery, medical devices and drug device combinations. Most recently he was Chief Executive Officer of MAP Pharmaceuticals, where he led the company through its initial public offering and developed LEVADEX, inhaled dihydoergotamine for migraine, through NDA filing (Allergan acquired MAP in March 2013). Prior to MAP, he served as Senior Vice President of Business and Commercial Development of Durect Corporation and previously held various positions at Medtronic including Business Director of Neurological Division for Europe, Middle East and Africa, and Manager of Drug Delivery Ventures and Business Development where he implemented growth initiatives for innovative therapies treating a variety of medical disorders. Mr. Nelson holds a bachelor’s degree in chemical engineering from the University of Minnesota and a master of management degree with distinction from the J.L. Kellogg Graduate School of Management, Northwestern University.

About Civitas Therapeutics

Civitas Therapeutics 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. The company’s lead program, CVT-301, is being developed as adjunctive PRN therapy to provide Parkinson’s disease patients with rapid and reliable relief from intermittent debilitating motor fluctuations (OFF episodes). OFF episodes result from the unreliability of current medications -- a problem impacting about half of all Parkinson’s disease patients. In April 2013, Civitas announced positive results for CVT-301 in a Phase 2 dose-ranging study. The company is currently conducting a Phase 2b study to evaluate the efficacy and safety of CVT-301 in treating emergent OFF episodes during one month of continued use. The Company’s pipeline also includes products for respiratory disease, central nervous system disorders and infectious disease.

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 and is protected by a large intellectual property estate. 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 Company is financed by leading investors including Alkermes plc, Bay City Capital, Canaan Partners, Fountain Healthcare Partners, Longitude Capital and RA Capital. For further information on Civitas, please visit www.civitastherapeutics.com.

For additional information contact:

Stephanie Gillis Maureen L. Suda (Media)
Civitas Therapeutics Suda Communications LLC
617-660-4121 585-387-9248
sgillis@civitastherapeutics.com

Leading International Blindness Charities Welcome Genable’s Enhanced Patent Portfolio

Genable Technologies Ltd., Dublin, Ireland, has recently expanded its intellectual property portfolio having obtained grants for two additional US patent applications on its novel suppression and replacement technology. The first of Genable’s recently granted US patents encompasses aspects of RNA interference (RNAi) mediated suppression and replacement (12/710343), while the second US patent (13/539,835) is focused on the generation of optimized replacement vectors for incorporation in innovative therapeutic strategies such as suppression and replacement therapies.

Avril Daly, CEO of Ireland’s Fighting Blindness commented “As a patient-led research charity the development of appropriate therapies for unmet medical needs is paramount therefore our organization was a founding investor in Genable Technologies. Having these newly granted patents improves the likelihood that the products will become viable therapies for patients with genetic blindness and we congratulate the inventors and the Company.”

Dr Stephen Rose, Chief Research Officer at US charity Foundation Fighting Blindness added “our key focus is to enable effective therapies get to patients and we recognize that securing IPR is a very important step on this journey. As such, we continue to support Genable as they bring their lead therapy GT038 to the clinic”.

Suppression and replacement technology represents a unique solution that enables treatment of dominantly inherited genetic disorders. The Company’s lead product, GT038, is focused on an ocular indication, Rhodopsin-linked autosomal dominant Retinitis Pigmentosa. GT038 has been granted orphan drug status and is currently in late preclinical development.

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.net

About Fighting Blindness

Irish research funded by Fighting Blindness has made a global impact in the search for therapies and cures for conditions causing sight loss. Since 1983, the patients who founded the organisation met regularly to help each other cope with the reality of degenerating vision. There was no treatment, nor was there real hope of one. Now not only do we understand how the conditions develop, but we also know what the treatments will look like. Since its founding, Fighting Blindness has invested over €10 million in Irish research, and our goal of cures and treatments is in sight. Our research is also contributing to international discoveries and developments and many clinical trials are being undertaken in both genetically inherited and age related conditions.

http://www.fightingblindness.ie

About Foundation Fighting Blindness

Foundation Fighting Blindness is a US-based non-profit organization driving the research that will lead to preventions, treatments and cures for retinitis pigmentosa, macular degeneration, Usher syndrome and the entire spectrum of retinal degenerative diseases that affects more than 10 million Americans. Since 1971, the Foundation has raised more than $550 million as the leading non-governmental funder of inherited retinal research. Breakthrough Foundation-funded studies using gene therapy have restored significant vision in children and young adults who were previously blind, paving the way for additional clinical trials to treat a variety of retinal degenerative diseases. With a coveted four-star rating from Charity Navigator, the Foundation also has nearly 50 chapters that provide support, information and resources to affected individuals and their families in communities across the country. http://www.fightblindness.org/

For more information please contact:

Dr. Jason Loveridge,
CEO Genable Technologies Ltd.
c/o Delta Partners
Media House South County Business Park
Leopardstown Dublin 18 Ireland
Ph. +33 674177812

www.genable.net

Civitas Therapeutics Extends CVT-301 Patent Protection into 2032

New US Patent Covering CVT-301 Composition Expands Extensive Patent Estate

CHELSEA, Mass.  – Civitas Therapeutics, Inc., a biopharmaceutical company with a lead program in Parkinson’s disease that leverages the ARCUS® respiratory delivery platform, today announced the Unites States Patent and Trademark Office (USPTO) has issued US Patent 8,545,878 entitled “Capsules containing high doses of levodopa for pulmonary use.” This newly issued patent covers the pharmaceutical composition of CVT-301 and has an expiration date of November 16, 2032. US 8,545,878 is one of over 115 issued patents worldwide protecting CVT-301.

“This newly issued patent further fortifies and extends our robust patent estate covering CVT-301,” said Glenn Batchelder, Chief Executive Officer and Co-founder of Civitas. “The ARCUS® platform’s unique capabilities and versatility provide a wealth of opportunities for continued innovation thereby broadening and deepening our patent estate around both the platform and CVT-301.”

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. Oral L-dopa, used for chronic symptom management, is administered to maintain dopamine levels in the brain above the therapeutic threshold; however, the reliability of oral L-dopa formulations is significantly compromised by delayed and unpredictable absorption and excessive variability in circulating plasma drug concentrations inherent to the oral delivery route. L-dopa remains widely recognized as the most efficacious treatment for Parkinson’s disease symptoms in spite of this intrinsic unreliability, which results in OFF episodes. CVT-301 is being developed as an adjunct PRN therapy to standard oral L-dopa therapy to address OFF episodes as they emerge and enable patients to reliably manage their symptoms.

CVT-301 leverages the ARCUS® platform to optimally 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. 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 recently completed 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 at all doses tested. Civitas is currently conducting a Phase 2b study to evaluate the efficacy and safety of CVT-301 in treating emergent OFF episodes during one month of continued use. CVT-301 clinical studies conducted to date have been funded in part by grants from The Michael J. Fox Foundation for Parkinson’s Research.

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 diminished production of dopamine in the substantia nigra area of the brain, 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® Platform

The ARCUS® 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® 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, Mass. in a facility that includes both development and commercial scale GMP manufacturing capabilities. The Company is financed by leading investors including Alkermes plc, Bay City Capital, Canaan Partners, Fountain Healthcare Partners, Longitude Capital and RA Capital. For further information on Civitas, please visit www.civitastherapeutics.com.

For additional information contact:

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

Maureen L. Suda (Media)
Suda Communications LLC 585-387-9248

Opsona Therapeutics Limited raises an additional €3 million (USD 4 million) from Omnes Capital in Series C extension, resulting in a total financing round of € 36 million (USD 48.6 million)

Opsona Therapeutics Limited (Opsona), the innate immune drug development company, today announced that it has raised an additional €3 million (US $4 million) in a second closing of its previously announced Series C equity financing from new investor Omnes Capital. The extension brings the total raised by Opsona in this Series C financing to €36 million (US $48.6 million). On April 25th, 2013, Opsona raised €33 million (US $43 million) from 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.

The company will use the proceeds of this Series C financing to supplement funding a three-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) as the first clinical indication for the development of OPN-305. The clinical study has already commenced with successful recruitment of patients underway. Opsona's lead product is a humanized monoclonal IgG4 antibody targeting Toll-like-receptor-2 (TLR2) and has demonstrated activity in a number of animal models and was recently tested successfully in a phase I clinical trial in healthy volunteers and in a pilot cohort of renal transplant recipients.

Dr. Martin Welschof, CEO of Opsona, commented: “I am delighted Omnes Capital is joining the exceptional Series C consortium of venture captial and corporate venture firms. The second closing of our Series C with Omnes Capital further validates the great medical and commercial potential of our drug candidate OPN-305.” Dr. Bruno Montanari, Life Sciences Director, Omnes Capital, added: “I am looking forward to progressing Opsona’s lead product OPN-305 through the well-designed Phase II efficacy study to prevent delayed graft function (DGF) in renal transplantation, an indication with major unmet medical need and attractive commercial potential. In addition, I am interested in exploring the full potential of OPN-305 in additional disease indications.”

-ends-

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. The company was awarded a EUR 6 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 explored.

Further information is available at http://www.opsona.com/. About Omnes Capital (formerly Crédit Agricole Private Equity)

Omnes Capital is a major player in private equity, with a commitment to financing SMEs. With €1.8 billion in assets under management, Omnes capital provides companies with the capital needed to finance their growth and with key expertise in a number of areas: Mid and Small Cap Buyout & Growth Capital, Venture Capital in technology and life sciences, Renewable Energy, Mezzanine, Secondary Funds of Funds, Co-Investment. Omnes Capital, formerly Crédit Agricole Private Equity, was a subsidiary of Crédit Agricole until March 2012 when the company gained its independence. Omnes Capital is a signatory to the United Nations Principles for Responsible Investment (PRI).

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

For further information please contact:

Martin Welschof (CEO) - mwelschof@opsona.com or + 353 1 6770223

Martine Sessin-Caracci - martine.sessincaracci@omnescapital.com or +33 1 80 48 79 15

Caroline Babouillard (Shan) - caroline.babouillard@shan.fr or +33 1 44 50 58 72

Civitas Therapeutics Secures $38 Million in Financing

-- To complete Phase 2b and support Phase 3 initiation for Parkinson’s disease program and pipeline expansion --

CHELSEA, Mass. September 11, 2013 – Civitas Therapeutics, Inc., a biopharmaceutical company with a lead program in Parkinson’s disease that leverages the ARCUS® respiratory delivery platform, today announced the successful completion of a $38 million Series B financing. Bay City Capital led the round and was joined by new investors RA Capital and another undisclosed blue chip public investment fund with participation from all existing investors including Alkermes plc, Canaan Partners, Fountain Healthcare Partners, and Longitude Capital.

“The strength and diversity of this investor group provides a robust foundation for achieving our vision of developing proprietary ARCUS®-based therapeutics to improve outcomes for patients,” said Glenn Batchelder, Chief Executive Officer and Co-founder of Civitas. “We are pleased that our new investors recognized the significant value that has been created since our initial financing, and we are excited to have their input and support as we further develop our lead program along with the broader pipeline.”

Civitas plans to use the proceeds from this financing for late stage clinical development of the company’s lead program, CVT-301, an inhaled formulation of levodopa (L-dopa) being developed for the rapid and reliable relief from debilitating motor fluctuations (OFF episodes) associated with Parkinson’s disease. Civitas recently initiated a Phase 2b clinical study to evaluate the efficacy and safety of CVT-301, self-administered by patients, in treating emergent OFF episodes during one month of continued use. The company will report preliminary data from this study in the first half of 2014. In addition, Civitas will explore additional opportunities to leverage the ARCUS® platform for other disease states where the potential to deliver a large, precise dose of a drug -- independent of inspiratory flow rate -- from a simple, breath actuated device, would provide a significant clinical advantage.

“As we surveyed the Parkinson’s disease landscape, we found CVT-301 to be a particularly compelling opportunity that uniquely addresses a significant unmet need,” said Rajeev Shah, Partner at RA Capital. “CVT-301 represents a rare combination of a large commercial opportunity for a severe disease coupled with a highly de-risked, capital efficient development path.”

“We are very pleased to join the Civitas investor syndicate,” said Rob Hopfner, Partner at Bay City Capital. “Civitas, along with its scientific and patient foundation collaborators, has made impressive achievements in rapidly advancing the development of CVT-301 since the company’s launch in 2011. The company’s track record of outstanding execution gives us confidence that they are destined to deliver important new therapies to patients.”

As part of this financing, Mr. Shah and Dr. Hopfner will join Civitas’ Board of Directors.

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. Oral L-dopa, used for chronic symptom management, is administered to maintain dopamine levels in the brain above the therapeutic threshold; however, the reliability of oral L-dopa formulations is significantly compromised by delayed and unpredictable absorption and excessive variability in circulating plasma drug concentrations inherent to the oral delivery route. L-dopa remains widely recognized as the most efficacious treatment for Parkinson’s disease symptoms in spite of this intrinsic unreliability, which results in OFF episodes. CVT-301 is being developed as an adjunct PRN therapy to standard oral L-dopa therapy to address OFF episodes as they emerge and enable patients to reliably manage their symptoms.

CVT-301 leverages the ARCUS® platform to optimally 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. 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 recently completed 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 at all doses tested. Civitas is currently conducting a Phase 2b study to evaluate the efficacy and safety of CVT-301 in treating emergent OFF episodes during one month of continued use. CVT-301 clinical studies conducted to date have been funded in part by grants from The Michael J. Fox Foundation for Parkinson’s Research.

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 diminished production of dopamine in the substantia nigra area of the brain, 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® Platform

The ARCUS® 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® 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, Mass. in a facility that includes both development and commercial scale GMP manufacturing capabilities. The Company is financed by leading investors including Alkermes plc, Bay City Capital, Canaan Partners, Fountain Healthcare Partners, Longitude Capital and RA Capital. For further information on Civitas, please visit www.civitastherapeutics.com.

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

-- Potential therapy for treating intermittent OFF episodes associated with Parkinson’s disease --

CHELSEA, Mass. – Civitas Therapeutics, Inc., a biopharmaceutical company developing transformative therapeutics using the ARCUS® respiratory delivery platform, today announced the initiation of a Phase 2b clinical study of CVT-301, an inhaled formulation of levodopa (L-dopa). CVT-301 is being developed as an adjunct, as needed (PRN) therapy that will provide rapid and reliable relief from intermittent debilitating motor fluctuations (OFF episodes) that affect many Parkinson’s disease patients. This Phase 2 study will evaluate the efficacy and safety of CVT-301 in treating emergent OFF episodes during one month of continued use.

The Phase 2b study follows a successful Phase 2a study for CVT-301, which demonstrated that the administration of 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.

“We are excited to move to this next stage of development in which patients will self-administer CVT-301 upon the emergence of their OFF symptoms as they go about their normal daily activities, exactly aligned with the intended treatment paradigm,” said Martin Freed, M.D., Chief Medical Officer of Civitas. “The ease of use of the simple ARCUS® inhaler allows patients to use CVT-301 wherever and whenever their oral Parkinson’s medications begin to fail them in between their regularly scheduled doses.”

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

About the Phase 2b Study (CVT-301-003)

CVT-301-003 is a randomized, double blind, placebo controlled, parallel group trial that is being conducted in the United States and Europe. Patients will use CVT-301 to treat emerging OFF episodes over a 28 day study period. The study is intended to test the efficacy, safety and tolerability of CVT-301 in the treatment of intermittent OFF episodes. Objective motor responses will be evaluated during regularly schedule clinic visits using the Unified Parkinson’s Disease Rating Scale (UPDRS) Part 3. In addition, efficacy will also be evaluated during out-patient use with diary based outcomes measures. Eighty (80) patients will be enrolled in the study. The company will report preliminary data from this study in the first half of 2014.

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 manage their symptoms.

CVT-301 is an ARCUS® 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® 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 at 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® Platform

The ARCUS(R) 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® 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, Mass. 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 $350 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.

For additional information contact:

Stephanie Gillis Maureen L. Suda (Media)
Civitas Therapeutics Suda Communications LLC
617-660-4121 585-387-9248
sgillis@civitastherapeutics.co
 

Amarin Informed by FDA of October 16th Advisory Committee Date in Connection With Supplemental New Drug Application (sNDA) for Vascepa(R)

Amarin Informed by FDA of October 16th Advisory Committee Date in Connection With Supplemental New Drug Application (sNDA) for Vascepa(R) in the Treatment of Patients With High Triglycerides (TG ≥200 mg/dL and < 500 mg/dL) with mixed dyslipidemia

BEDMINSTER, N.J. and DUBLIN, Ireland, - Amarin Corporation plc (Nasdaq:AMRN), a biopharmaceutical company focused on the commercialization and development of therapeutics to improve cardiovascular health, announced today that it was informed yesterday by the U.S. Food and Drug Administration (FDA) that the FDA will convene an advisory committee on October 16, 2013 in connection with the FDA's review of the Supplemental New Drug Application (sNDA) seeking approval for the use of Vascepa® (icosapent ethyl) capsules as an adjunct to diet in the treatment of adult patients with high triglycerides (TG ≥200 mg/dL and < 500 mg/dL) with mixed dyslipidemia. The FDA has previously assigned a Prescription Drug User Fee Act (PDUFA) date of December 20, 2013 for completion of its review of the sNDA. The FDA will consider the recommendation of the advisory committee, but the final decision regarding the approval of the sNDA will be made by the FDA.

"ANCHOR clinical trial results demonstrated the important role EPA-only omega-3 can play in helping adult patients with high triglycerides and mixed dyslipidemia," said Christie M. Ballantyne, M.D., Baylor College of Medicine and the Methodist DeBakey Heart and Vascular Center, Houston, Texas, and principal investigator of the ANCHOR trial. "In the ANCHOR trial, Vascepa lowered high triglyceride levels and showed robust reductions in a broad range of other lipid parameters on top of optimized statin therapy compared to placebo. Importantly, these beneficial effects were seen with a safety and tolerability profile comparable to placebo."

Vascepa was approved in 2012 as an adjunct to diet to reduce triglyceride levels in adult patients with severe (≥500 mg/dL) hypertriglyceridemia, based on the results of Amarin's MARINE clinical trial. The safety data from both the MARINE and ANCHOR clinical trials were reviewed by the FDA as part of the approval of Vascepa in the MARINE indication and are reflected in the approved product labeling for Vascepa. As is customary practice at FDA, yesterday's notification from the FDA is the only communication confirming the need for such a meeting. The FDA advisory committee topics and questions to the committee are anticipated to be made public by the FDA on its website in the week preceding the meeting, consistent with FDA procedure.

"For key first-in-class indications, an FDA advisory committee meeting is expected, and this public forum will be an important opportunity to discuss the ANCHOR data, which demonstrated Vascepa's unique potential as an adjunct to diet in the treatment of adult patients with high triglycerides (TG 200-499 mg/dL) and mixed dyslipidemia," said Eliot A. Brinton, MD, FAHA, FNLA, Director of Atherometabolic Research, Utah Foundation for Biomedical Research, and President, American Board of Clinical Lipidology. "Currently, many of these patients are receiving another prescription omega-3 which is not indicated for this disorder. Having instead an omega-3 product which lowers LDL-cholesterol in addition to triglycerides, has tolerability comparable to placebo, and is FDA-approved for use on top of statin therapy would be a welcome addition to the physician's armamentarium for comprehensive lipid management."

"With the support of the Amarin team, including our outside consultants, such as Christie and Eliot, we look forward to the advisory panel and working with the FDA to obtain regulatory approval of Vascepa for ANCHOR this year," said Joseph Zakrzewski, Chairman and Chief Executive Officer of Amarin.

Currently, Amarin only markets Vascepa for the MARINE indication, for which it was approved in 2012, and which includes approximately 4 million patients in the United States. The proposed ANCHOR indication would expand the Vascepa patient population to include adult patients on statin therapy with triglyceride levels ranging from 200 to 499 mg/dL (and mixed dyslipidemia). Amarin estimates that one in five, or nearly 40 million, U.S. adults have triglyceride levels in this range. Amarin began selling Vascepa on January 28, 2013. Amarin believes that many of the same clinicians who have prescribed Vascepa for its currently approved indication will prescribe Vascepa for the much broader indication, based on the ANCHOR study now under formal FDA review, if approved.

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.”

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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.