Mainstay Announces the Partial Exercise of the Over-Allotment Option, Increasing the Gross Proceeds of the Offer to Approximately €18.8M

Mainstay Announces the Partial Exercise of the Over-Allotment Option, Increasing the Gross Proceeds of the Offer to Approximately €18.8M

Not for distribution, directly or indirectly, in the United States of America, Canada, Australia and Japan

Mainstay Medical International plc (“Mainstay”) (Paris:MSTY) announces that, in connection with its initial public offering on the regulated market of Euronext Paris and on the Enterprise Securities Market of the Irish Stock Exchange, the Over-allotment Option granted to Kempen & Co and Société Générale, in their role as Joint Global Coordinators and Joint Bookrunners, was partially exercised on 28 May 2014. The partial exercise of the Over-allotment Option resulted in the allotment and issue by Mainstay to Société Générale as Stabilising Manager of 38,264 new Ordinary Shares at the Offer Price of €21.15 per share, giving additional gross proceeds of approximately €0.8m. As a result, the total number of Ordinary Shares allotted and issued by Mainstay in connection with the Offer amounted to 889,439 new Ordinary Shares, increasing the total gross proceeds of the Offer to approximately €18.8m.

Application has been made for listing and admission to trading of the 38,264 new Ordinary Shares on Euronext Paris and on the ESM. It is expected that listing and admission to trading of the 38,264 new Ordinary Shares on Euronext Paris will take place at 9.00 am CET on 30 May 2014 and on the ESM will take place at 8.00 am (Dublin time) on 4 June 2014.

Total Voting Rights

In conformity with Regulation 20 of the Transparency (Directive 2004/109/EC) Regulations 2007 of Ireland, Mainstay confirms that, as at 29 May 2014, the issued share capital of Mainstay consists of 4,294,141 Ordinary Shares (which carry voting rights) and 40,000 deferred shares of €1 each (which do not carry voting rights). Therefore, the figure that may be used by shareholders as the denominator for the calculations by which they will determine if they are required to notify their interest in, or a change to their interest in, Mainstay under the Transparency (Directive 2004/109/EC) Regulations 2007 of Ireland and the Transparency Rules of the Central Bank of Ireland is 4,294,141.

In accordance, with the provisions of Article 631-10 of the Règlement général of the French Autorité des marchés financiers, Société Générale, acting as Stabilising Manager, declares that:

It has conducted stabilization transactions on Mainstay’s Ordinary Shares (IE00BJYS1G50);

The stabilization period started on 29 April, 2014;

The stabilization period closed on 28 May, 2014.

Pricing information is set forth below:

Date                  Low price              High price

29/04/2014       20.900                  21.150

30/04/2014       20.800                  21.150

02/05/2014       21.150                  21.150

05/05/2014       20.930                  21.150

06/05/2014       21.150                  21.150

07/05/2014       20.930                  21.150

08/05/2014       20.800                  21.150

09/05/2014       21.150                  21.150

12/05/2014       21.150                  21.150

13/05/2014       20.910                  21.150

14/05/2014       21.140                  21.140

15/05/2014       21.140                  21.140

16/05/2014       20.800                  21.140

FOR FURTHER DETAILS, CONTACT:

Joint Global Coordinators and Joint Bookrunners
Kempen & Co N.V.
Beethovenstraat 300
1077 WZ Amsterdam
The Netherlands

Société Générale
Tour Société Générale
17, Cours Valmy,
92972 Paris La Défense Cedex
France

Prospectus Adviser, ESM Adviser and Co-Lead Manager
Davy
Davy House
49 Dawson Street
Dublin 2, Ireland

Media & Investor Relations Adviser

FTI Consulting (Dublin)
10 Merrion Square
Dublin 2, Ireland
+353 1 663 3600 or mainstay@fticonsulting.com
Eilish Joyce/Jonathan Neilan

FTI Consulting (Paris)
5, Rue Scribe
Paris, 75009, France
+33 1 47 03 68 63 or mainstay@fticonsulting.com
Jeanne Bariller

DISCLAIMERS

This document does not constitute and shall not be considered as constituting a public offer, an offer to purchase or as an intention to solicit the interest of the public for a public offering of securities.

The information in this announcement is for background purposes only and does not purport to be accurate, full or complete. It is given at the date of its publication (unless otherwise marked) and is subject to updating, revision and amendment and no reliance may be placed for any purpose on it.

The prospectus (the "Prospectus") of Mainstay Medical International Plc (the "Company") was approved on 9 April 2014 by The Central Bank of Ireland, the Irish competent authority, and notified to the French Autorité des Marchés Financiers ("AMF") for passporting in connection with the Company’s application for listing its ordinary shares on Euronext Paris and the Enterprise Securities Market operated by the Irish Stock Exchange, and the public offering of its ordinary shares in France. The Prospectus and the French translation of the summary are available on the Company’s website at www.mainstay-medical.com; the French translation of the summary is also available on the AMF's website at www.amf-france.org. The Company draws the attention of the public in France to Part 2 "Risk Factors" of the Prospectus and their summary in the French translation of the Prospectus summary. These risks may have a material adverse effect on the Company and its subsidiaries, their business, financial condition, results of operations or growth prospects as well as on the market price of Mainstay Medical International’s ordinary shares on Euronext Paris and the ESM.

No reliance may or should be placed by any person for any purposes whatsoever on the information contained in this announcement or on its completeness, accuracy or fairness.

The distribution of this document in certain countries may be subject to specific regulations. Persons who come into possession of this press release must inform themselves of and comply with these restrictions.

In particular:

This document does not constitute an offer to sell or a solicitation of an offer to buy any securities, nor shall there be any sale of securities in the United States of America (the "United States"), or in any other jurisdiction in which such an offer, solicitation, or sale would be unlawful prior to registration or qualification under the securities laws of the United States or any such jurisdiction. Securities may not be offered or sold in the United States absent registration or an exemption from registration under the U.S. Securities Act of 1933, as amended. The Company has not registered, and does not intend to register, any portion of any offering of its securities in the United States, and does not intend to conduct a public offering of any of its securities in the United States.

This announcement includes statements that are, or may be deemed to be, forward looking statements. These forward looking statements can be identified by the use of forward looking terminology, including the terms “anticipates”, “believes”, “estimates”, “expects”, “intends”, “may”, “plans”, “projects”, “should” or “will”, or, in each case, their negative or other variations or comparable terminology, or by discussions of strategy, plans, objectives, goals, future events or intentions. These forward looking statements include all matters that are not historical facts. They appear throughout this announcement and include, but are not limited to, statements regarding the Company’s intentions, beliefs or current expectations concerning, among other things, the Company’s results of operations, financial position, prospects, financing strategies, expectations for product design and development, regulatory approvals, reimbursement arrangements, costs of sales and market penetration.

By their nature, forward looking statements involve risk and uncertainty because they relate to future events and circumstances. Forward looking statements are not guarantees of future performance and the actual results of the Company’s operations, and the development of the markets and the industry in which the Company operates, may differ materially from those described in, or suggested by, the forward looking statements contained in this announcement. In addition, even if the Company’s results of operations, financial position and growth, and the development of the markets and the industry in which the Company operates, are consistent with the forward looking statements contained in this announcement, those results or developments may not be indicative of results or developments in subsequent periods. A number of factors could cause results and developments of the Company to differ materially from those expressed or implied by the forward looking statements including, without limitation, general economic and business conditions, the global medical device market conditions, industry trends, competition, changes in law or regulation, changes in taxation regimes, the availability and cost of capital, currency fluctuations, changes in its business strategy, political and economic uncertainty and other factors to be disclosed in the Prospectus. The forward-looking statements therein speak only at the date of this announcement.

Media & Investor Relations Adviser

FTI Consulting (Dublin)
Eilish Joyce/Jonathan Neilan, +353 1 663 3600
mainstay@fticonsulting.com

or

FTI Consulting (Paris)
Jeanne Bariller, +33 1 47 03 68 63
mainstay@fticonsulting.com

Palyon Medical and CORE Manufacturing Announce Strategic Partnership

Valencia, CA - Palyon Medical Corporation and CORE Manufacturing LLC have entered into a strategic partnership for the manufacturing of Palyon’s line of implantable drug delivery pumps.

Palyon, a developmental stage company, has a pump portfolio that includes programmable devices for maximum clinical flexibility and constant flow devices for lower cost steady drug delivery. CORE is an ISO 13485, ISO 9001 and FDA approved manufacturer of implantable devices. Under this new partnership, CORE Manufacturing will provide manufacturing services, procurement coordination and dual quality control oversight, greatly reducing Palyon’s time to market introduction.

“CORE brings a level of clinical manufacturing experience that will greatly streamline our process and allow our researchers and engineers to focus on developing the next generation of devices, such as an implantable insulin pump,” said Michael Sember, Palyon CEO. “Having CORE’s production facility in such close proximity to us will allow for enhanced efficiency, reduced manufacturing coordination costs and speed our path to market.”

Palyon’s inventive implantable infusion pump platform will offer highly accurate drug delivery rates and incorporate unique safety features including the only sensor-based feedback flow control, refill error detection capability, a non-motorized pumping mechanism that is less susceptible to wear and tear, catheter occlusion detection and a highly favorable MRI profile.

"CORE Manufacturing is honored to partner with Palyon. CORE is uniquely qualified to develop and manufacture the lineup of Palyon’s active implantable devices. This partnership will allow both companies to leverage their core competencies," said Joel Hirsch, President of CORE Manufacturing.

About Palyon

Palyon Medical Corporation, headquartered in Southern California, is a privately held medical device company focused on creating innovative treatment systems to alleviate symptoms of chronic pain, spasticity, diseases of the central nervous system and chronic metabolic diseases. Palyon’s mission is to provide the market with the broadest range of implantable infusion devices that offer an unparalleled feature set to address a broad range of unmet medical needs. For more information visit www.palyonmedical.com.

About CORE Manufacturing, LLC

Located in Valencia, CA, CORE Manufacturing is an ISO 13485, ISO 9001, and FDA approved manufacturer of implantable, minimally invasive, and peripheral medical devices. Capabilities include: rapid-turn prototyping, full turnkey device-level assembly, sterilization and testing.

Media Contact:

Palyon Medical Corp: Michael Sember msember@palyoncorp.com, (661) 705-5601

CORE Manufacturing LLC: Joel Hirsch jhirsch@core-manufacturing.com, (661) 621-6200

Civitas Therapeutics Announces Positive Phase 2b Results for CVT-301, Inhaled Levodopa for the Treatment of Parkinson’s Disease

- CVT-301 shown to rapidly treat debilitating OFF episodes associated with Parkinson’s disease -

- Met primary endpoint with clinically important and statistically-significant reduction in average UPDRS III motor score versus placebo -

- Phase 2 study results to be presented at American Academy of Neurology 2014 Annual Meeting -

Chelsea, MA -  Civitas Therapeutics, Inc., a privately-held pharmaceutical company developing and commercializing transformative therapeutics using its proprietary ARCUSTM technology, today announced positive results from a Phase 2b clinical trial of CVT-301, an inhaled formulation of levodopa (L-dopa). Study results from Phase 2 studies will be presented at the 66th Annual American Academy of Neurology Meeting on April 29, 2014 at 2:30pm ET in Philadelphia, PA.

“We are extremely pleased that we met our primary endpoints in our Phase 2b trial with statistical significance, demonstrating that CVT-301 was safe and well tolerated and provides patients with a rapid, clinically important improvement in motor function. CVT-301 provided onset of action by 10 minutes with durable effects lasting for at least 60 minutes,” said Mark Iwicki, President and CEO of Civitas. “CVT-301 is being developed to address a significant unmet need facing Parkinson’s disease patients today, and these results, particularly the efficacy and ease-of-use seen in the outpatient setting, give us confidence that CVT-301 can have a transformative impact on the daily lives of patients.”

CVT-301 is being developed as an adjunctive, as-needed therapy to provide rapid and reliable relief from intermittent debilitating motor fluctuations, known as OFF episodes, suffered by Parkinson’s disease patients. OFF episodes, which result from the unpredictable nature of oral baseline therapies, affect over half of all patients on an oral L-dopa treatment regimen and are considered to be one of the most important issues facing Parkinson’s disease patients today.

“OFF episodes are debilitating events for Parkinson’s disease patients. A rapid and reliable therapy that can address these episodes would be a major advancement in treatment," said Dr. Todd Sherer, CEO of The Michael J. Fox Foundation for Parkinson's Research, which provided supportive funding for the study. “These results suggest that CVT-301 could have a transformative impact on patients’ lives,” said Dr. Sherer. “As supporters of this program from its early days, we are pleased with its performance thus far and look forward to continued success in Phase 3 trials.”

The Phase 2b study was designed to assess the on-demand use of CVT-301 in Parkinson’s disease patients experiencing motor fluctuations over a one-month period. The trial evaluated the efficacy, safety and tolerability of two doses of CVT-301. Motor responses were evaluated during regularly- scheduled clinical visits using the Unified Parkinson’s Disease Rating Scale Part 3 (UPDRS III). In addition, efficacy was also evaluated during out-patient use with commonly used diary-based outcomes measures.

The primary endpoint was successfully met with CVT-301 achieving a clinically important and statistically significant reduction in average UPDRS III motor score versus placebo at time points ranging from 10 to 60 minutes post-administration (p < 0.001). Furthermore, clinically important and statistically significant improvements in UPDRS III were seen at every time point including 10 minutes, the earliest time point tested, for both tested doses.

Over the course of the study, patients self-administered CVT-301 as-needed to treat approximately 4,500 OFF episodes, at an average of approximately 2 treatments per day. With self-administration during at-home use, CVT-301 use was not associated with any increase in either non-troublesome or troublesome dyskinesia. All doses of CVT-301 were safe and well-tolerated, and the CVT-301 inhaler was also shown to be easily utilized by Parkinson’s disease patients in the OFF state.

“Patients today live with the reality that their oral medications could fail them at any moment,” said Dr. Karl Kieburtz, the Robert J. Joynt Professor of Neurology, University of Rochester, president of Clintrex LLC and a member of the Civitas Scientific Advisory Board. “By providing rapid onset and reliable relief using levodopa, the standard of care and backbone of patients’ therapy, in a simple patient-friendly device, CVT-301 has the potential to usher in a new paradigm in the treatment of Parkinson’s disease.”

Trial Design

The Phase 2b trial (CVT-301-003) was a randomized, double-blind, placebo-controlled, multicenter study of inhaled CVT-301 or placebo for the treatment of up to 3 OFF episodes per day in Parkinson’s disease subjects experiencing intermittent OFF episodes. The 86 patients treated in this study underwent an initial screen and run-in period of 2-4 weeks during which baseline motor assessments, measured using the Unified Parkinson’s Disease Rating Scale Part 3 (UPDRS III), as well as other baseline efficacy and safety/pulmonary function assessments, were conducted. Following this phase, patients were randomized to receive CVT-301 or inhaled placebo in a double-blinded fashion. During the first 2 weeks of treatment, patients self-administered an inhaled levodopa dose of approximately 35 mg or placebo. During the final 2 treatment weeks, patients were dose-escalated to an inhaled levodopa dose of approximately 50 mg or placebo. In-office assessments of UPDRS III were performed at week 1, week 2 and week 4. The primary endpoint was defined as the mean change from pre-dose in average UPDRS III score (10-60 minutes post dose) after 4 weeks of treatment. Patients were also instructed to complete a Parkinson’s disease diary for three days prior to the office visits, recording daily OFF time and ON time without and with dyskinesias, as well as a daily treatment log. Safety parameters measured included pulmonary function, ECGs and vital signs (blood pressure, heart rate and orthostatic blood pressure).

About CVT-301

CVT-301 is being developed as a self-administered, as needed, inhaled L-dopa therapy for OFF episodes, providing rapid delivery of L-dopa to the brain without altering a patient’s individually optimized oral L-dopa regimen. 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 drug concentrations in the bloodstream 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 as- needed 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 Civitas’ proprietary ARCUSTM technology to optimally deliver a precise dose to the lung for rapid and predictable L-dopa absorption. The ARCUSTM platform is uniquely able to deliver the necessary L-dopa dose with the required precision in a convenient, non-invasive manner. 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) confirmed 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 has recently completed a successful 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 over seven 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 at rest, rigidity and impaired movement. Even when treated with the current standard of care, the majority of Parkinson’s patients continue to experience OFF periods. 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 ARCUSTM Technology

The ARCUSTM inhalation technology delivers a reliable and consistent drug dose with a compact, breath-actuated inhaler. The ARCUSTM platform uses a proprietary dry powder and inhaler combination that is unique in its ability to deliver a large, precise dose independent of inspiratory flow rate from a simple, easy-to-use device suitable for convenient self-administration. The technology 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 biopharmaceutical company focused on developing and commercializing transformative therapeutics using its proprietary ARCUSTM technology, with an initial focus on treating debilitating OFF episodes in patients with Parkinson’s disease. Civitas is financed by leading investors including Alkermes plc, Bay City Capital, Canaan Partners, Fountain Healthcare Partners, Longitude Capital and RA Capital.

For additional information contact:

Stephanie Gillis Civitas Therapeutics
sgillis@civitastherapeutics.com

Mainstay Medical International Plc: Announcement of Offer Price and Number of Issued Ordinary Shares on Admission To Euronext Paris and ESM

Further to the announcement on 9 April, 2014 of its intention to raise funds through an initial public offering, Mainstay Medical International plc (“Mainstay” or the “Company”) announces the successful pricing of its initial public offering of new ordinary shares by way of a public offering in France and of an international private placement to institutional investors (the “Offer”), the number of new ordinary shares comprised in the offer and the timing for admission of its existing and new ordinary shares (“Ordinary Shares”) to trading on the regulated market of Euronext Paris and on the Enterprise Securities Market (“ESM”) of the Irish Stock Exchange.

Mainstay is an Irish medical device company with operations in Ireland, Australia and the United States. Mainstay is focused on the development of ReActiv8®, an innovative implantable medical device designed to treat people with Chronic Low Back Pain (CLBP).

HIGHLIGHTS

• The Offer comprises 851,175 new Ordinary Shares representing gross proceeds of an amount of €18.0 million, excluding any Ordinary Shares that may be issued under the Over-allotment Option.
• The Offer price has been set at €21.15 per new Ordinary Share (“Offer Price”).
• The size of the Offer may be increased by up to 45,953 new Ordinary Shares issued in the case of exercise, in part or in full, of the Over-allotment Option within 30 days after the Offer Price is made public.
• On commencement of unconditional dealings, the Company will have a market capitalisation, based on the Offer Price, of approximately €90.0 million.
• The gross proceeds will be used to conduct clinical trials, initially in Australia and then additionally in Europe, to submit an application for CE mark approval and for general corporate purposes.
• Retail investors who applied for Ordinary Shares under the retail offer have been allocated 16,783 Ordinary Shares, corresponding to €0.4 million, or 2.0% of the new Ordinary Shares.
• Institutional investors who applied for Ordinary Shares under the institutional private placement have been allocated 834,392 Ordinary Shares, corresponding to €17.6 million, or 98.0% of the new Ordinary Shares. Of the Ordinary Shares allocated to institutional investors, 355,791 Ordinary Shares were allocated to US Qualified Institutional Buyers (QIBs), corresponding to €7.5 million, or 41.8% of the new Ordinary Shares.
• Mainstay’s major institutional shareholders (Sofinnova Partners, Fountain Healthcare Partners, Medtronic, Inc., Capricorn Venture Partners and Seventure Partners Managed Funds) invested €8 million in the Offer. David Brabazon, a director of Mainstay, invested €0.1 million in the Offer.
• Following commencement of unconditional dealings:
  • Existing shareholders will hold 83.3% of the Ordinary Shares; and
  • Directors will hold 14.1% of the Ordinary Shares.
• Conditional dealings in the Ordinary Shares on Euronext Paris (under the “if and when issued” product line: MAINSTAY AIW) and the ESM are expected to commence at 9:00 a.m. (CET) on 29 April 2014.
• Settlement and delivery is expected to occur on 2 May 2014, with unconditional dealings in Ordinary Shares on the ESM expected to start at 9:00 a.m. (CET) on 2 May 2014 and on 5 May 2014 on Euronext Paris.

Dr Oern Stuge, Chairman, said:

“The successful initial public offering marks a major milestone in the company’s progress and we are now ready to move on to the next stage of our development as a listed company. We are very pleased that our IPO has been so well received by institutional and retail investors, who have recognised that Mainstay’s unique and innovative approach to the treatment of debilitating CLBP not only addresses an important clinical need, but moreover creates an interesting investment opportunity. We have recently commenced clinical trials of ReActiv8 and we continue to diligently execute our plans to obtain regulatory approval and subsequent commercialisation.”

Further Information

• Kempen & Co and Société Générale Corporate & Investment Banking are acting as Joint Bookrunners in connection with the Offer. Davy is acting as Prospectus adviser, ESM adviser and Co-Lead Manager.
• This Pricing Statement relating to the Offer will be made available on the Company’s website at www.mainstay-medical.com.
• Capitalised terms used but not defined herein shall have the meaning given to those terms in the Prospectus.

OFFER STATISTICS

Price (per Ordinary Share) €21.15

Number of Ordinary Shares that will be in issue immediately prior to the commencement of unconditional dealings 3,404,702

Number of Ordinary Shares issued in the Offer (1) 851,175

Maximum number of Ordinary Shares that may be issued under the Over- allotment Option 45,953

Estimated gross proceeds of the Offer receivable by the Company (2) €18.0 million

Estimated net proceeds of the Offer receivable by the Company (2) €14.5 million

Market Capitalisation of the Company at the Offer Price on the commencement of unconditional dealings €90.0 million

Euronext / ESM ticker MSTY

ISIN Code IE00BJYS1G50

(1) The Extension Clause was not exercised, and assuming no exercise of the Over-allotment Option.

(2) Assumes no exercise of the Over-allotment Option.

About Mainstay Medical

Mainstay Medical is an Irish medical device company that is developing an innovative implantable neurostimulation device, ReActiv8, for people with debilitating Chronic Low Back Pain.

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

Chronic Low Back Pain

Chronic Low Back Pain is generally defined as Low Back Pain where the pain persists for more than three months. Low Back Pain is a 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 estimates that in approximately 7% of all cases of Low Back Pain, the pain persists for more than three months.

About ReActiv8

ReActiv8 represents a new approach to the treatment of Chronic Low Back Pain. ReActiv8 is an implantable neurostimulation device which applies electrical stimulation to nerves that supply one of the key stabilising muscles in the back, the lumbar multifidus muscle. The hypothesis on which ReActiv8 is based is that electrical stimulation of the nerve that innervates the lumbar multifidus muscle to cause contraction of the muscle can help reactivate the muscle control system, thereby leading to improved spine stability and a reduction in the effects of CLBP.

Further information is available at www.mainstay-medical.com

Mainstay Medical Announces Intention To Launch Initial Public Offering And List On Euronext Paris And On The Enterprise Securities Market Of Irish Stock Exchange

Mainstay Medical International plc (“Mainstay”), a medical device company that is developing innovative therapies for people with debilitating Chronic Low Back Pain (“CLBP”), today announces its intention to raise funds through an initial public offering of new ordinary shares (the “Offer”) and to seek admission of its shares to trading on Euronext Paris and the Enterprise Securities Market of the Irish Stock Exchange.

Mainstay is an Irish medical device company with operations in Ireland, Australia and the United States. Mainstay is focused on the development of ReActiv8®, an innovative implantable medical device designed to treat people with Chronic Low Back Pain.

Mainstay is led by an experienced Board and management team with a strong track record in the medical device industry. Mainstay has a committed shareholder base, including experienced venture capital investors in France, Ireland, Belgium and the United States.

Highlights

• ReActiv8 is targeted at people with debilitating Chronic Low Back Pain. Low Back Pain is a 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.
• Mainstay estimates that the initial target market for ReActiv8 is approximately two million people in the US and Europe.
• ReActiv8 is an innovative implantable neurostimulation medical device. The design of ReActiv8 is based on experience gained in a European Feasibility Study conducted in 2011 and 2012, which showed the therapy provided by ReActiv8 yielded improvements in pain and disability of people with Chronic Low Back Pain.
• Product development of ReActiv8 is complete, and enrolment in clinical trials in Australia commenced in March 2014.
• Mainstay intends to progress clinical trials in Australia; obtain CE Mark in Europe; obtain initial approvals for US clinical trials; and subject to regulatory approval, advance the commercialisation of ReActiv8.
• Strong and experienced Board, management team and existing investor base to continue the commercialisation of ReActiv8.
• Existing shareholders have committed to make a substantial investment in the Offer.

Dr Oern Stuge, Chairman, commented:

“We believe that ReActiv8 offers an innovative therapy for the millions of people who suffer from Chronic Low Back Pain. We further believe there is a significant market opportunity for ReActiv8 and the results of the recent Feasibility Study demonstrate the premise of our unique therapeutic approach. The Board is confident that we have the right management team, led by Peter Crosby, to commercialise ReActiv8 and is excited about the prospects for Mainstay.”

Peter Crosby, Mainstay CEO, added:

“Back pain specialists from all over the world have indicated the need for a new approach to help people with Chronic Low Back Pain. We believe our approach of using electrical stimulation to help restore control to the key stabilizing muscles in the lower back can play an important role in helping these people.”

“The therapy was investigated in a recently completed European Feasibility Study. The results presented in mid-2013 showed improvement in back pain and the disabling effects of back pain.”

“The energy and experience of the Mainstay team has enabled us to complete the development of ReActiv8 and obtain approval to start the clinical trials within a year of the publication of Feasibility Study results, and we have now commenced clinical trials and have a clear plan to obtain regulatory approval and the commercialisation of ReActiv8.”

About Mainstay Medical

Mainstay is an Irish medical device company that is developing an innovative implantable neurostimulation medical device, ReActiv8™, for people with debilitating Chronic Low Back Pain.

Mainstay is headquartered in Dublin, Ireland and has subsidiaries in Australia and the United States. Mainstay 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).

Chronic Low Back Pain

Chronic Low Back Pain is generally defined as Low Back Pain where the pain persists for more than three months.

Low Back Pain is a 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 estimates that in approximately 7% of all cases of Low Back Pain, the pain persists for more than three months.

ReActiv8

ReActiv8 represents a new approach to the treatment of Chronic Low Back Pain. ReActiv8 is an implantable device which applies electrical stimulation to nerves that supply one of the key stabilising muscles in the back, the lumbar multifidus muscle. The hypothesis on which ReActiv8 is based is that electrical stimulation of the nerve that innervates the lumbar multifidus muscle to cause contraction of the muscle can help reactivate the muscle control system, thereby leading to improved spine stability and a reduction in the effects of Chronic Low Back Pain.

Kempen & Co and Société Générale are acting as Joint Global Coordinators and Joint Bookrunners in connection with the Initial Public Offering. Davy has been appointed as Co-Lead Manager and ESM advisor.

FOR FURTHER DETAILS, CONTACT:

Joint Global Coordinators and Joint Bookrunners

Kempen & Co N.V.
Beethovenstraat 300
1077 WZ Amsterdam
Postbus 75666
1070 AR Amsterdam
The Netherlands

Société Générale
Tour Société Générale
17, Cours Valmy,
92972 Paris La Défense Cedex
France

ESM Adviser and Co-Lead Manager

Davy
Davy House
49 Dawson Street
Dublin 2, Ireland

Media & Investor Relations Adviser

FTI Consulting (Dublin)
10 Merrion Square
Dublin 2, Ireland
+353 1 663 3600 or mainstay@fticonsulting.com
Eilish Joyce/Jonathan Neilan

FTI Consulting (Paris)
5, Rue Scribe
Paris, 75009, France
+33 1 47 03 68 10 or mainstay@fticonsulting.com
Arnaud de Cheffontaines/ Stephan Dubosq

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