Fennec Inc. received the complete response letter (CRL) it had expected from the FDA, sidelining U.S. development of Pedmark, a formulation of sodium thiosulfate for preventing ototoxicity associated with cisplatin chemotherapy in pediatric patients older than 1 month to those 18 years of age with localized, non-metastatic, solid tumors.
While the world grapples for a clear picture of the Omicron variant and how to handle it, Moderna Inc., Biontech SE and Adagio Therapeutics Inc. stepped out with stock advances, building on momentum from the end of last week, while eyeing 2022 as a launch date against the variant.
The first approval for treating advanced malignant perivascular epithelioid cell tumor (PEComa) in adults has been awarded by the FDA to Aadi Bioscience Inc.’s Fyarro. The number of patients is low, from 100 to 300 patients annually in the U.S., but the geography the company hopes to sell to is wide as it takes in Europe and China.
Biomarin Pharmaceutical Inc.’s data supporting the use of Voxzogo (vosoritide) in children with the most common form of dwarfism proved compelling for the FDA, which cleared the modified C-type natriuretic peptide as the first treatment for the rare genetic disease affecting bone growth.
Protego Biopharma Inc. raised $51 million in a series A financing to advance the targeting of protein misfolding diseases. The fundraising is a few years on from 2017, when the idea underscoring the company’s birth was to build a platform based on small molecules that could stabilize proteins and restore their functionality.
Roche Holding AG is walking away from a deal with Atea Pharmaceuticals Inc. to co-develop the COVID-19 oral antiviral treatment AT-527 after the mid-October failure of a global phase II study testing the medicine in non-hospitalized adults with mild or moderate disease missed its primary endpoint. Roche’s decision tosses the rights and licenses back to Atea so it can continue its development and commercialization efforts.
A futility analysis of Molecular Partners AG’s ensovibep for treating hospitalized COVID-19 adults prompted an independent data safety monitoring board to recommend discontinuing recruitment of patients in the phase III ACTIV-3 study.
Catalyst Biosciences Inc. decided to stop developing marzeptacog alfa (MarzAA), a recombinant human coagulation factor VIIa variant for treating factor VII deficiency, and sell its hemophilia assets to focus on its complement therapeutics and protease medicines platform.