MedPath

SerpinPC

Generic Name
SerpinPC
Drug Type
Biotech
Background

SerpinPC is a mutated alpha-1-antitrypsin (α1AT) and serine protease inhibitor being investigated for the treatment of hemophilia.

Acquired Hemophilia A Pipeline Expands with 10+ Novel Therapies in Development

• Global Acquired Hemophilia A pipeline comprises over 10 companies developing innovative therapies across various clinical stages, according to DelveInsight's 2025 report. • Recent clinical advances include promising results for rituximab as a potential first-line therapy and Pfizer's positive Phase 3 AFFINE trial results for giroctocogene fitelparvovec gene therapy. • Key pharmaceutical players including Novo Nordisk, Sanofi, Pfizer, and Belief Biomed are advancing treatments ranging from gene therapies to monoclonal antibodies targeting this rare autoimmune bleeding disorder.

Centessa Pharmaceuticals Appoints Stephen Kanes as Chief Medical Officer to Advance Orexin Agonist Program

• Centessa Pharmaceuticals appointed Stephen Kanes as Chief Medical Officer, leveraging his 30+ years of experience in neuroscience and drug development. • Dr. Kanes will focus on advancing Centessa's orexin receptor 2 (OX2R) agonist program, including ORX750, currently in Phase 2 trials for sleep-wake disorders. • Centessa's ORX750 has shown promise in Phase 1 trials, restoring wakefulness in sleep-deprived individuals, with Phase 2a studies underway for narcolepsy and idiopathic hypersomnia. • Kanes previously led the development of ZULRESSO® for postpartum depression at Sage Therapeutics, bringing valuable expertise to Centessa's neurology and psychiatry pipeline.

Hemophilia B Therapeutics: Advances in Clinical Trials and Emerging Therapies

• Several companies are actively involved in developing therapies for Hemophilia B, with Belief Biomed's drug candidates reaching Phase III clinical trials. • Recent trials include Novo Nordisk's assessment of Nonacog Beta Pegol in Chinese patients and ApcinteX Ltd's study of SerpinPC in severe Hemophilia A or B. • Emerging therapies like BBM-H901 (Belief Biomed) and Fidanacogene elaparvovec (Pfizer/Spark Therapeutics) are under investigation across various clinical stages. • Hemophilia B therapies are being developed using various routes of administration, including oral, intravenous, and subcutaneous, and molecule types, such as gene therapy and recombinant fusion proteins.

Hemophilia A and B Pipeline Advances with Novel Therapies in Clinical Trials

• Several companies are actively developing novel therapies for Hemophilia A and B, with many in various phases of clinical trials, showing promise for improving treatment options. • TiumBio Co., Ltd. has submitted a Clinical Trial Application to initiate a Phase 1b investigation of TU7710, a new recombinant activated factor VII for hemophilia patients with inhibitors. • The FDA approved ALTUVIIIO (efanesoctocog alfa) in February 2023, a factor VIII replacement therapy for adults and children with Hemophilia A, for routine prophylaxis and on-demand treatment. • BioMarin received FDA approval for Roctavian in June 2023, a single-dose gene therapy for severe Hemophilia A, marking a significant advancement in treatment.
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