Enlaza Therapeutics Inc. will take charge of research through nominating candidates in its potentially $2 billion-plus deal with Vertex Pharmaceuticals Inc. For its efforts, Enlaza is getting $45 million in an up-front payment and equity investment, plus the opportunity to bring in more than $2 billion in research, development, regulatory and commercial milestones and tiered royalties on net sales.
The FDA has granted orphan drug designation to Vanda Pharmaceuticals Inc.’s VGT-1849B, a selective peptide nucleic acid-based JAK2 inhibitor for the treatment of polycythemia vera (PV).
BMS-986470 is a potentially first-in-class molecular glue dual degrader, targeting zinc finger and BTB domain containing 7A (ZBTB7A) and widely interspaced zinc finger protein (WIZ). Bristol Myers Squibb Inc. recently presented details on the discovery of BMS-986470, which is now in phase I studies for the treatment of sickle cell disease (SCD) (NCT06481306).
CSL Ltd.’s stock tumbled 16.88% on the news that it plans to cut 3,000 jobs and to hive off its Seqirus vaccine unit in a cost-cutting measure to save more than $500 million per year over the next three years.
Syntara Ltd.’s stock plummeted 52% on news that the FDA recommended an additional phase IIb trial following a type C meeting about the company’s clinical development plan for lysyl oxidase inhibitor amsulostat (SNT-5505, formerly PXS-5505) in myelofibrosis.
Syntara Ltd.’s stock plummeted 52% on news that the FDA recommended an additional phase IIb trial following a type C meeting about the company’s clinical development plan for lysyl oxidase inhibitor amsulostat (SNT-5505, formerly PXS-5505) in myelofibrosis.
Hebei Yiling Medicine Research Institute Co. Ltd. has described piperidine indole derivatives targeting complement factor B (CFB) reported to be useful for the treatment of inflammatory disorders, and hematological, neurodegenerative and autoimmune diseases.
Agios Pharmaceuticals Inc. said the patients’ deaths tied to anemia therapy Pyrukynd (mitapivat) that were announced in a securities analyst’s Aug. 4 note to investors hasn’t changed the drug’s already established benefit-risk profile on the drug’s U.S. prescribing information.
A total of 33 states, plus the District of Columbia and Puerto Rico, agreed to participate in the U.S. CMS’ voluntary, outcomes-based program aimed at helping state Medicaid programs cover high-priced cell and gene therapies, starting with therapies for sickle cell disease.
It may be a year later than originally planned, but Regeneron Pharmaceuticals Inc. is set to commercialize Lynozyfic (linvoseltamab) in the U.S. following FDA approval for use in adults with relapsed and refractory multiple myeloma. A bispecific antibody targeting B-cell maturation antigen (BCMA) and CD3, Lynozyfic was cleared under the accelerated approval pathway, with continued approval contingent upon a confirmatory trial.
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