Agios Pharmaceuticals Inc. has synthesized phenylalanine hydroxylase (PAH) R408W mutant stabilizers reported to be useful for the treatment of phenylketonuria.
Agios Pharmaceuticals Inc. is learning the hard way the downside of having a pipeline in a product. When a side effect crops up, investors are likely to worry that it may affect the potential of the drug in the numerous diseases the drug could potentially treat.
The BioWorld Drug Developers Index (BDDI) declined in August, ending the month down 1.11% for the year and diverging from its previous trend of mirroring the Nasdaq Biotechnology Index (NBI) and the Dow Jones Industrial Average (DJIA). By the close of August, the BDDI had slipped, while the NBI gained 11.7% year-to-date and the DJIA rose 7.05%.
In December 2020, Servier Pharmaceuticals LLC bought Agios Pharmaceuticals Inc.’s approved acute myeloid leukemia drug, Tibsovo (ivosidenib), and the rest of its oncology business in a deal valued at up to $2 billion. Now, the U.S. FDA has approved one of those assets, with the brand name Voranigo (vorasidenib), for treating gliomas.
While Servier Pharmaceuticals LLC awaits this summer’s PDUFA date for the IDH-mutant glioma drug vorasidenib, acquired in the buyout of oncology assets from Agios Pharmaceuticals Inc., the latter unveiled positive data from a global phase III study with oral mitapivat in adults with transfusion-dependent alpha- or beta-thalassemia.
Vorasidenib, a glioma candidate under U.S. FDA review, may bring in additional cash for Agios Pharmaceuticals Inc., this time via a deal with Royalty Pharma, which agreed to pay $905 million to Agios in exchange for the 15% royalty arrangement stemming from an earlier collaboration with Servier Pharmaceuticals LLC.
Agios Pharmaceuticals Inc.’s fourth-quarter and full-year 2023 financial report in mid-February highlighted further prospects with Pyrukynd (mitapivat), the pyruvate kinase activator for hemolytic anemia in adults with pyruvate kinase deficiency (PKD).
Agios Pharmaceuticals Inc. and Alnylam Pharmaceuticals Inc. have entered into an exclusive worldwide license agreement under which Agios will acquire the rights to develop and commercialize Alnylam’s novel preclinical siRNA targeting TMPRSS6, as a potential disease-modifying treatment for patients with polycythemia vera (PV).
An Agios Pharmaceuticals Inc. patent describes branched-chain-amino-acid aminotransferase, mitochondrial (BCAT2) inhibitors reported to be useful for the treatment of isovaleric acidemia, maple syrup urine disease, methylmalonic acidemia and propionic acidemia.
The positive opinion Jan. 27 from the EMA’s Committee for Medicinal Products for Human Use regarding Reblozyl (luspatercept) from Bristol Myers Squibb Co. to treat adults with non‑transfusion-dependent beta-thalassemia marked an advance in the space, where several developers are jockeying for position. Reblozyl, a first-in-class erythroid maturation agent, was first approved in November 2019 in the hands of Celgene Corp., acquired by Princeton, N.J.-based BMS the same year.
