Wall Street’s measure of how Cogent Biosciences Inc.’s KIT D816V inhibitor bezuclastinib (often shortened to bezu) might fare in mastocytosis against U.S. FDA cleared Ayvakit (avapritinib), the tyrosine kinase inhibitor from Blueprint Medicine Corp., caused the former’s stock (NASDAQ:COGT) to tumble, closing Dec. 11 at $4.06, down $4.58, or 53%. Data from the ongoing phase II Summit trial testing bezu in non-advanced systemic mastocytosis rolled out during the American Society of Hematology (ASH) meeting in San Diego. Waltham, Mass.-based Cogent’s prospect turned up a rapid and continuing improvement in patient symptoms, with a 57% median best improvement on Mast Cell Quality-of-Life.
Spirits were high at the 2023 Annual Meeting of the American Society of Hematology (ASH), buoyed by U.S. FDA approval of the first two gene therapies for sickle cell disease (SCD) the day before the conference kicked off in San Diego.
Following a strategic transaction with Graphite Bio Inc., Kamau Therapeutics is emerging from stealth with sickle cell treatment nulabeglogene autogedtemcel (nula-cel). Kamau received an option to acquire all of Graphite’s genome editing assets, including a platform technology that integrates precision DNA repair using homology directed repair and CRISPR/Cas9, as well as the autologous CRISPR/Cas9 gene corrected CD34+ cell product nula-cel, which offers a potential cure for sickle cell disease derived from the patient's cells.
Both Vertex Pharmaceuticals Inc.’s Casgevy (exagamglogene autotemcel, exa-cel) and Bluebird Bio Inc.’s Lyfgenia (lovotibeglogene autotemcel, lovo-cel) received U.S. FDA approval Dec. 8, providing 16,000 American sickle cell patients who have recurring vaso-occlusive events with access to the first cell-based gene therapies.
The U.S. FDA has approved Novartis AG’s Fabhalta (iptacopan) as the first oral monotherapy for adults with paroxysmal nocturnal hemoglobinuria, a rare blood disease that impairs blood cell production. This is the only factor B inhibitor of the immune system’s complement pathway and is expected to be on the market before December ends. Fabhalta has plenty of competition from already-approved therapies and more treatments are in development.
Cereno Scientific AB has completed the preclinical safety program for CS-014, a histone deacetylase (HDAC) inhibitor in development for arterial and venous thrombosis prevention.
Argenx SE’s surprise phase III blowup with subcutaneous Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) in the platelet disorder primary immune thrombocytopenia (ITP) – blamed on a higher-than-expected placebo response – had investors speculating about possible read-throughs to other indications.
Sernova Corp. has announced that its hemophilia A program, combining the Sernova Cell Pouch with a patient’s own cells corrected for the production of factor VIII (FVIII), has been awarded U.S. orphan drug and rare pediatric disease designations.
Bayer AG’s asundexian, considered one of the next generation class of anticoagulants and a potential blockbuster, has failed a phase III clinical trial in atrial fibrillation. The factor XIa inhibitor is one of three with a similar mechanism of action in late stage development by big companies.
With a landmark U.K. approval in hand for Casgevy (exagamglogene autotemcel [exa-cel]) to treat sickle cell disease and transfusion-dependent beta thalassemia, Crispr Therapeutics AG and partner Vertex Therapeutics Inc. are turning their attention to the PDUFA dates set by the U.S. FDA for the treatment in both conditions.
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