Fitusiran

At the 66th ASH Annual Meeting, Steven W. Pipe, MD, presented ATLAS-OLE trial data on fitusiran, an siRNA therapeutic targeting antithrombin, showing its effectiveness as a prophylactic agent for hemophilia A and B, reducing breakthrough bleeds by 70% and factor consumption by 64-98%.

At ASH 2024, physicians discussed advancements in hemophilia care, including Roche's Hemlibra and curative AAV gene therapies like CSL Behring's Hemgenix, Biomarin's Roctavian, and Pfizer's Beqvez and Hympavzi. Despite progress, unmet needs remain, with concerns over long-term risks of gene therapies and limited access. Pfizer presented Phase III AFFINE trial data for giroctocogene fitelparvovec, showing comparable efficacy to Roctavian with better safety. Sanofi's fitusiran, a siRNA, demonstrated efficacy similar to clotting factor replacement therapy. Staidson's bemiltenase alfa, a fusion protein, showed favorable bleed clearance rates. Challenges persist, but advancements in curative, prophylaxis, and on-demand treatments offer more options for patients.

DelveInsight's 'Hemophilia B Pipeline Insight 2024' report details 20+ pipeline drugs from 15+ companies, focusing on mechanism of action, route of administration, and molecule types. Key companies include Belief Biomed, TiumBio, and Pfizer, with therapies in various clinical stages. Notable recent developments include Novo Nordisk and ApcinteX trials in May 2024.

Hemophilia B market to grow significantly by 2034, driven by gene therapies like HEMGENIX and BEQVEZ, priced at $3.5 million each. Key companies include Centessa Pharmaceuticals, Sanofi, Pfizer, and Novo Nordisk. Market dynamics influenced by increasing prevalence, prophylactic treatment focus, and pipeline advancements.

Gene therapy fidanacogene elaparvovec outperforms prophylaxis factor IX concentrate in reducing bleeding and stabilizing factor IX in hemophilia B patients, suggesting a more durable treatment. Published in the New England Journal of Medicine, the phase 3 study involved 45 patients, showing reduced annualized bleeding rates and mean factor IX activity at 15 months. Despite some resuming prophylaxis, the therapy demonstrated a favorable benefit-risk profile.

Sanofi reports 15.7% Q3 sales growth, driven by vaccine sales and strong business performance, raising 2024 EPS guidance. Dupixent sales up 23.8%, with full-year target confirmed at €13 billion. Pharma launches grew 67.1%, led by ALTUVIIIO, Nexviazyme, and Rezurock. Vaccines sales increased 25.5%, boosted by flu sales and Beyfortus. Opella sales rose 7.9%, supported by Qunol acquisition. R&D expenses grew 12.7%, while SG&A expenses grew 6.4%. Business EPS at €2.86, up 12.2% reported and 17.6% at CER. Four regulatory approvals and four positive phase 3 data readouts achieved. Negotiations underway to transfer a controlling stake of Opella to CD&R. 2024 business EPS guidance upgraded to low single-digit growth at CER.