Shire and Parion Sciences Enter into a Collaborative License Agreement to Advance P-321 for Ophthalmic Indications

LEXINGTON, Massachusetts and DURHAM, North Carolina, May 1, 2017 /PRNewswire/ --

Demonstrates ongoing commitment to innovation in ophthalmics for Shire

P-321 is a Phase 2 investigational topical treatment for dry eye disease  

Shire plc (LSE: SHP, NASDAQ: SHPG) and Parion Sciences, Inc. today announced they have entered into an agreement granting Shire exclusive worldwide rights to develop and commercialize P-321. Shire will lead development of P-321, an investigational epithelial sodium channel (ENaC) inhibitor for the potential treatment of dry eye disease in adults, with the opportunity for Parion to co-fund.  

P-321 is a Phase 2 compound being developed to address tear volume deficiency and promote ocular surface healing. While further clinical trials are needed to fully evaluate its safety profile and efficacy, P-321 is a novel molecule that is believed to address dry eye disease by inhibiting ENaC, which is thought to block the absorption of tears, and help keep the ocular surface hydrated; current prescription treatments target the effects of ocular inflammation.  

"Ophthalmics is a continued focus for Shire, and the program for P-321 will benefit from our development and commercial infrastructure and expertise," said Flemming Ornskov, M.D., M.P.H., Chief Executive Officer, Shire. "This is an opportunity to apply our knowledge and experience from ophthalmics and dry eye disease for further innovation in this space. If approved, P-321 would expand our eye care portfolio."

"Advancing P-321 with Shire, an emerging global leader in ophthalmics, offers the expertise and resources to move this promising potential therapy for dry eye sufferers forward," said Paul Boucher, President and Chief Executive Officer of Parion. "This collaborative license agreement enables us the opportunity to contribute and participate in P-321's success, while continuing our drive to progress Parion's pipeline of novel therapies."

While specific terms of the deal were not disclosed, Shire will make an initial $20 million upfront license payment with an additional $20 million payment based on the achievement of a near term development milestone. Parion will be entitled to receive additional potential milestone payments, with a total potential deal value of up to $535 million. Parion has the option to co-fund through additional stages of development in exchange for enhanced tiered double-digit royalties. In addition, Parion has the option to co-fund commercialization activities and participate in the financial outcome from those activities.

P-321 and Clinical Development Program 

P-321 is a novel small molecule inhibitor of ENaC, which is thought to block the absorption of tears, and help keep the ocular surface hydrated. A Phase 1/2a placebo-controlled, dose escalation clinical study in 53 patients has been completed, which evaluated the safety and tolerability of P-321 in patients with mild to moderate dry eye disease. Although the study -
P-321-101 - was not powered to assess efficacy signals, positive trends were observed in improvement of signs and symptoms of dry eye disease in subsets of patients, compared to placebo. At the highest strength studied (0.01%), no discomfort or instillation irritation was reported with P-321. The adverse events seen in the patients assigned to P-321 were generally similar to or fewer than those that occurred in the patients assigned to placebo, and none of the adverse events were considered serious. A Phase 2b study is expected to begin after consultation with health authorities.

Based on pre-clinical models of dry eye disease, ENaC inhibitors are believed to have the potential to restore or improve tear volume on the ocular surface. Further clinical trials are needed to evaluate the safety profile, efficacy and mechanism of action of P-321.

About Chronic Dry Eye Disease 

The prevalence of dry eye disease ranges from 6-34% in adults globally. Dry eye disease is a multifactorial disease of the ocular surface that is often chronic and may be progressive. The disease is most commonly associated with eye dryness and overall eye discomfort, as well as stinging, burning, a gritty feeling or fluctuating blurry vision.

Eye care professionals can diagnose dry eye disease based on patient reported symptoms as well as signs, which can be objectively evaluated through various tests. Management options may include the use of non-prescription and prescription treatments.

Aging and gender are recognized as traditional risk factors of chronic dry eye disease while modern risk factors include prolonged digital/computer screen time, contact lens wear and cataract or refractive surgery. Dry eye is a common complaint to eye care professionals.

About Parion Sciences 

Parion Sciences is a development stage biopharmaceutical company dedicated to research, development and commercialization of treatments to improve and extend the lives of patients with innate mucosal surface defense deficiencies of the eye or airway. Parion has a diverse pipeline of pre-clinical and clinical candidates for the treatment of these diseases via distinctive mechanisms of action and approaches. Parion is at the forefront of ENaC development and is leveraging our scientific expertise in epithelial biology to expand our platforms and novel chemical compounds into new indications to treat mucosal defects. Parion has received support and grant funding from the National Institutes of Health and the Cystic Fibrosis Foundation Therapeutics, Inc. For more information, please see our website at http://www.Parion.com .

About Shire 

Shire is the leading global biotechnology company focused on serving people with rare diseases and other highly specialized conditions. We strive to develop best-in-class products, many of which are available in more than 100 countries, across core therapeutic areas including Hematology, Immunology, Neuroscience, Ophthalmics, Lysosomal Storage Disorders, Gastrointestinal / Internal Medicine / Endocrine and Hereditary Angioedema; and a growing franchise in Oncology.

Our employees come to work every day with a shared mission: to develop and deliver breakthrough therapies for the hundreds of millions of people in the world affected by rare diseases and other high-need conditions, and who lack effective therapies to live their lives to the fullest.

http://www.shire.com

Forward-Looking Statements 

Statements included herein that are not historical facts, including without limitation statements concerning future strategy, plans, objectives, expectations and intentions, the anticipated timing of clinical trials and approvals for, and the commercial potential of, inline or pipeline products, are forward-looking statements. Such forward-looking statements involve a number of risks and uncertainties and are subject to change at any time. In the event such risks or uncertainties materialize, Shire's results could be materially adversely affected. The risks and uncertainties include, but are not limited to, the following:

• Shire's products may not be a commercial success;
• increased pricing pressures and limits on patient access as a result of governmental regulations and market developments may affect Shire's future revenues, financial condition and results of operations;
• Shire conducts its own manufacturing operations for certain of its products and is reliant on third party contract manufacturers to manufacture other products and to provide goods and services. Some of Shire's products or ingredients are only available from a single approved source for manufacture. Any disruption to the supply chain for any of Shire's products may result in Shire being unable to continue marketing or developing a product or may result in Shire being unable to do so on a commercially viable basis for some period of time;
• the manufacture of Shire's products is subject to extensive oversight by various regulatory agencies. Regulatory approvals or interventions associated with changes to manufacturing sites, ingredients or manufacturing processes could lead to, among other things, significant delays, an increase in operating costs, lost product sales, an interruption of research activities or the delay of new product launches;
• certain of Shire's therapies involve lengthy and complex processes, which may prevent Shire from timely responding to market forces and effectively managing its production capacity;
• Shire has a portfolio of products in various stages of research and development. The successful development of these products is highly uncertain and requires significant expenditures and time, and there is no guarantee that these products will receive regulatory approval;
• the actions of certain customers could affect Shire's ability to sell or market products profitably. Fluctuations in buying or distribution patterns by such customers can adversely affect Shire's revenues, financial conditions or results of operations;
• Shire's products and product candidates face substantial competition in the product markets in which it operates, including competition from generics;
• adverse outcomes in legal matters, tax audits and other disputes, including Shire's ability to enforce and defend patents and other intellectual property rights required for its business, could have a material adverse effect on the Company's revenues, financial condition or results of operations;
• inability to successfully compete for highly qualified personnel from other companies and organizations;
• failure to achieve the strategic objectives, including expected operating efficiencies, cost savings, revenue enhancements, synergies or other benefits at the time anticipated or at all with respect to Shire's acquisitions, including NPS Pharmaceuticals Inc., Dyax Corp. or Baxalta Incorporated may adversely affect Shire's financial condition and results of operations;
• Shire's growth strategy depends in part upon its ability to expand its product portfolio through external collaborations, which, if unsuccessful, may adversely affect the development and sale of its products;
• a slowdown of global economic growth, or economic instability of countries in which Shire does business, as well as changes in foreign currency exchange rates and interest rates, that adversely impact the availability and cost of credit and customer purchasing and payment patterns, including the collectability of customer accounts receivable;
• failure of a marketed product to work effectively or if such a product is the cause of adverse side effects could result in damage to Shire's reputation, the withdrawal of the product and legal action against Shire;
• investigations or enforcement action by regulatory authorities or law enforcement agencies relating to Shire's activities in the highly regulated markets in which it operates may result in significant legal costs and the payment of substantial compensation or fines;
• Shire is dependent on information technology and its systems and infrastructure face certain risks, including from service disruptions, the loss of sensitive or confidential information, cyber-attacks and other security breaches or data leakages that could have a material adverse effect on Shire's revenues, financial condition or results of operations;
• Shire incurred substantial additional indebtedness to finance the Baxalta acquisition, which may decrease its business flexibility and increase borrowing costs; and

a further list and description of risks, uncertainties and other matters can be found in Shire's most recent Annual Report on Form 10-K and in Shire's subsequent Quarterly Reports on Form 10-Q, in each case including those risks outlined in "ITEM 1A: Risk Factors", and in Shire's subsequent reports on Form 8-K and other Securities and Exchange Commission filings, all of which are available on Shire's website.

All forward-looking statements attributable to us or any person acting on our behalf are expressly qualified in their entirety by this cautionary statement. Readers are cautioned not to place undue reliance on these forward-looking statements that speak only as of the date hereof. Except to the extent otherwise required by applicable law, we do not undertake any obligation to update or revise forward-looking statements, whether as a result of new information, future events or otherwise.

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SOURCE Shire plc