At the recent 39th J.P. Morgan Healthcare Conference, Biomarin Pharmaceutical Inc. popped the lid off top-line results from its ongoing phase III GENEr8-1 study with valoctocogene roxaparvovec – also known as valrox, now commonly called Roctavian. Data, though encouraging, may not have quelled controversy around the prospect.
Imara Inc. reported a mixed bag of phase IIa study data for its lead candidate, IMR-687, for treating sickle cell disease in adults, bringing an end to a study that the company found cumbersome and that eventually stung its stock.
Amsterdam-based Uniqure NV’s regulatory hitch with its highly regarded hemophilia B program had analysts questioning during a conference call what the development might mean for the company’s deal with CSL Ltd.
New and updated preclinical and clinical data presented by biopharma firms at the American Society of Hematology’s annual meeting, Dec. 5-8, including: Company List: Gamida, Glycomimetics, Janssen, Jasper, Kadmon, Keros, Kiadis, Kleo, Maat, Macrogenics, Medigene, Mesoblast, Mustang, Novartis, Oncopeptides, Oncternal, Oryzon, Pfizer, Pharmaessentia, Protagonist, Sangamo, Roche, Rocket, Schrodinger, Seagen, Sierra Oncology, Sumitomo, Sutro, Synthekine, Teneobio, TG, Treadwell, Uniqure, Xencor.
Questions of durability came up regarding Curis Inc.’s latest data with CA-4948, but that didn’t stop shares from soaring to $6.55 by day’s end, an increase of $5.11, or 355%. The ride came after Lexington, Mass.-based Curis rolled out positive preliminary data from the firm’s ongoing open-label, single-arm phase I dose-escalation study with the compound.
New data on a variety of blood disorder therapies announced at the 62nd American Society of Hematology (ASH) annual meeting moved company shares on Dec. 7. Shares of Fate Therapeutics Inc. (NASDAQ:FATE) hit a record high, rising 37.8% to $83.77 by day's end on news of clinical activity for one candidate in refractory diffuse large B-cell lymphoma.
There will be plenty of data from clinical trials testing treatments for various lymphomas at the 62nd American Society of Hematology (ASH) annual meeting this weekend. While CAR T cells have changed the landscape in the relapsed and refractory space for large B-cell lymphoma patients with two FDA approved medications, Kymriah (tisagenlecleucel, Novartis AG) and Yescarta (axicabtagene ciloleucel, Gilead Sciences), "the uptake has been good but not dominant. There's still plenty of space. There's still plenty of need," Jason Westin, leader of the diffuse large B-cell lymphoma research team at the University of Texas MD Anderson Cancer Center in the Department of Lymphoma and Myeloma, said on a call with clients from Raymond James.
New and updated preclinical and clinical data presented by biopharma firms at the American Society of Hematology’s annual meeting, Dec. 5-8, including: In8bio, Incyte, Jannsen, Rigel.
Despite challenges tossed at the phase III study of mitapivat from Agios Pharmaceuticals Inc. by COVID-19, top-line data showed the oral therapy hit its primary endpoint in treating adults with pyruvate kinase (PK) deficiency who don’t receive regular transfusions.
RSS
