Centessa Pharmaceuticals Announces Orphan Drug Designation Granted to SerpinPC for the Treatment of Hemophilia B
- Registrational studies planned to start in 4Q 2022 -
- Two-year data from Phase 2a Open Label Extension study expected in 4Q 2022 -
BOSTON and LONDON, Sept. 14, 2022 (GLOBE NEWSWIRE) -- Centessa Pharmaceuticals plc (Nasdaq: CNTA), a clinical-stage pharmaceutical company with a Research & Development (“R&D”) innovation engine that aims to discover, develop, and ultimately deliver impactful medicines to patients, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to SerpinPC, a novel inhibitor of activated protein C (“APC”), for the treatment of hemophilia B. Centessa plans to begin registrational studies of SerpinPC in the fourth quarter of 2022.
SerpinPC is a biological drug candidate, based on the serpin family of proteins that is designed to allow more thrombin to be generated by inhibiting APC, thus rebalancing coagulation in hemophilia patients. In September 2021, Centessa announced positive topline data from its proof-of-concept Phase 2a study of SerpinPC in severe hemophilia A and B patients not on prophylaxis. In the highest dose cohort, SerpinPC demonstrated an 88% reduction in median Annualized Bleeding Rate (ABR) for all bleeds and a 94% reduction in median ABR for spontaneous joint bleeds. SerpinPC was observed to be well-tolerated with no thrombosis and no instances of sustained D-dimer elevations.
“We believe SerpinPC has the potential to offer patients with hemophilia B a convenient subcutaneous option that is designed to prevent and reduce bleeds without the risk of thrombosis,” said Saurabh Saha, MD, PhD, Chief Executive Officer of Centessa. “This designation from the FDA is an important milestone in the development of SerpinPC and underscores the need for new, innovative treatment options for patients with hemophilia B. We look forward to initiating registrational studies for SerpinPC later this year, as well as reporting the two-year follow-up data from the SerpinPC Phase 2a open label extension study in the fourth quarter.”
Orphan Drug Designation is granted by the FDA to drugs or biologics intended to treat a rare disease or condition, defined as one that affects fewer than 200,000 people in the U.S. Orphan Drug Designation provides certain financial incentives to support clinical development, and the potential for up to seven years of marketing exclusivity for the product for the designated orphan indication in the U.S. if the product is ultimately approved for its designated indication.
About Centessa Pharmaceuticals
Centessa Pharmaceuticals plc is a clinical-stage pharmaceutical company with an R&D innovation engine that aims to discover, develop, and ultimately deliver impactful medicines to patients. Our programs span discovery-stage to late-stage development and cover a range of high-value indications in rare diseases and immuno-oncology. We are led by a management team with extensive R&D experience, providing direct guidance to our program teams to rapidly advance our candidates from research through all stages of development. For more information, visit www.centessa.com, which does not form part of this release.
Forward Looking Statements
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Kristen K. Sheppard, Esq.
SVP of Investor Relations