CSL Behring

uniQure's stock surged due to FDA's willingness to review AMT-130, a Huntington's disease gene therapy. Despite challenges, AMT-130 shows potential, with strong evidence of slowing disease progression. The stock's modest market cap could increase significantly if AMT-130 is approved, making it a high-risk, high-reward investment.

CSL announced EMA's CHMP recommended garadacimab for HAE treatment, a first once-monthly factor XIIa inhibitor. The decision is based on Phase 3 VANGUARD trial data showing 62% attack-free patients and 86.5% reduction in attacks. The EC decision is expected in Q1 2025.

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 'Graft versus host disease Pipeline Insight 2024' covers 60+ companies and 65+ pipeline drugs, including clinical and nonclinical stage products, with a focus on therapeutics assessment by product type, stage, route of administration, and molecule type. Key companies and promising therapies in development are highlighted.

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.

The 'Non-cystic fibrosis bronchiectasis - Pipeline Insight, 2024' report highlights emerging therapies like Brensocatib and HSK31858, which aim to address the underlying mechanisms of NCFB, potentially improving patient quality of life. Key industry players are actively developing these therapies, with significant implications for managing NCFB.

Pfizer's Hympavzi (marstacimab) receives EU marketing authorization for hemophilia A/B prophylaxis, becoming the first U.S.-approved hemophilia treatment with a pre-filled auto-injector pen. Based on the phase III BASIS study, Hympavzi significantly reduces annualized bleeding rates. This follows earlier approvals for Pfizer's gene therapies Durveqtix and Beqvez, and ongoing development of hemophilia A gene therapy giroctocogene fitelparvovec.

FDA approves PTC Therapeutics' eladocagene exuparvovec for AADC deficiency, marking the first brain-administered gene therapy in the U.S. Abeona Therapeutics' BLA for EB-101 accepted by FDA. Neurogene's NGN-401 trial for Rett syndrome faces critical immune response issue. Adicet Bio initiates phase 1 trial for ADI-100 in lupus nephritis. CSL Behring to close Pasadena R&D facility, shifting focus from ex vivo gene therapy. Anixa Biosciences and Moffitt Cancer Center dose first patient in phase 1 trial for ovarian cancer therapy. Answer ALS and Cedars-Sinai release ALS data repository. MyoGene receives FDA ODD and RPD designations for MyoDys45-55 for Duchenne muscular dystrophy.

Fanaroff: Research grants from American Heart Association and National Institutes of Health; consulting fees from Abbott Laboratories. Vora: Consulting fees from Medtronic. Mehran: Institutional research grants from AstraZeneca, Bayer, Beth Israel Deaconess, Bristol Myers Squibb/Sanofi, CSL Behring, Eli Lilly/Daiichi Sankyo, Medtronic, Novartis, OrbusNeich; consulting fees from Boston Scientific, Abbott Vascular, Medscape, Siemens Medical Solutions, Roivant Sciences Inc, Sanofi; consulting (no fees) for Regeneron Pharmaceuticals Inc; institutional consulting fees from Abbott Vascular, Spectranetics/Phillips/Volcano Corporation, Bristol Myers Squibb, Novartis, Watermark Research; Executive committee member for Janssen Pharmaceuticals and Bristol Myers Squibb; <1% equity in Claret Medical and Elixir Medical. Granger: Research grants from Bayer, Boehringer Ingelheim, Bristol Myers Squibb, Daiichi Sankyo, Janssen, Pfizer, Armetheon, AstraZeneca, US Food and Drug Administration, GlaxoSmithKline, The Medicines Company, Medtronic Foundation, Medtronic Inc, Novartis; consulting fees from Bayer, Boehringer Ingelheim, Boston Scientific, Bristol Myers Squibb, Daiichi Sankyo, Janssen, Pfizer, Abbvie, Armetheon, AstraZeneca, Eli Lilly, Gilead, GlaxoSmithKline, Hoffmann-La Roche, The Medicines Company, National Institutes of Health, Novartis, Sirtex, Verseon, Apple, Medscape, LLC, Merck, Novo Nordisk, Roche Diagnostics, Rho Pharmaceuticals. Goodman: Research grant support and/or speaker/consulting honoraria from Amgen, AstraZeneca, Bayer, Boehringer Ingelheim, Bristol Myers Squibb, CSL Behring, Daiichi-Sankyo, Eli Lilly, Esperion, Fenix Group International, Ferring Pharmaceuticals, GlaxoSmithKline, HLS Therapeutics, Janssen/Johnson & Johnson, Luitpold Pharmaceuticals, Matrizyme, Merck, Novartis, Novo Nordisk A/C, Pfizer, Regeneron, Sanofi, Servier, Tenax Therapeutics, Heart and Stroke Foundation of Ontario/University of Toronto, Canadian Heart Research Centre and MD Primer, Canadian VIGOUR Centre, Duke Clinical Research Institute, PERFUSE. Aronson: Employee of Bristol Myers Squibb. Windecker: Institutional research and educational grants from Abbott, Amgen, Bayer, BMS, CSL Behring, Boston Scientific, Biotronik, Edwards Lifesciences, Medtronic, Polares, Sinomed. Alexander: Research grants from Bristol Myers Squibb, Boehringer Ingelheim, AstraZeneca, CryoLife, CSL Behring, US Food and Drug Administration, National Institutes of Health, Sanofi, VoluMetrix; consulting fees from Pfizer, Bristol Myers Squibb, AbbVie Pharmaceuticals, CSL Behring, Novo Nordisk, Portola Pharmaceuticals, Quantum Genomics, Teikoku Pharmaceuticals, VA Cooperative Studies, Zafgen. Lopes: Research grants from Bristol Myers Squibb, Pfizer, Amgen, Inc, GlaxoSmithKline, Medtronic PLC, Sanofi Aventis; consulting fees from Bristol Myers Squibb, Pfizer, Boehringer Ingelheim, Bayer AG. The other authors report no conflicts.

Non-cystic fibrosis bronchiectasis market driven by rising prevalence, early diagnosis, and drug development. Over 15 companies, including Zambon SpA, Insmed, and AstraZeneca, are developing 15+ pipeline therapies. Key drugs in trials include Colistimethate sodium, Benralizumab, and Brensocatib.