Demonstrates ongoing commitment to innovation in ophthalmics for Shire
P-321 is a Phase 2 investigational topical treatment for dry eye disease
Lexington, Massachusetts, and Durham, North Carolina, US – May 1, 2017 – 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 www.Parion.com.
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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.
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- 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;
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- 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.
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