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.
Shares in Forma Therapeutics Holdings Inc. gained 51% as Novo Nordisk A/S made a $20-per-share offer that values the firm’s equity at $1.1 billion. The stock (NASDA:FMTX) had closed at $13.40 prior to the disclosure of the bid on Sept. 1. It closed the day at $20.24, up $6.84, suggesting at least some optimistic investors believe the final price could go higher.
There was no slowing of biopharma innovation in 2021, even as industry directed significant resources to, while feeling the impact of, the ongoing COVID-19 pandemic. The year saw big wins for developers of DNA vaccines and biosimilars, while CAR T expanded its reach and a drug target once considered undruggable was finally conquered. And as 2021 gives way to 2022, other potentially game-changing technologies and therapeutics are waiting in the wings.
It has been a challenging opening first quarter for companies developing new therapeutics, with the BioWorld Drug Developers index recording a 9% drop in value, representing a stark contrast to its more than 35% growth last year.
Agios Pharmaceuticals Inc., a company that has until now revolved primarily around its FDA-approved acute myeloid leukemia drug, Tibsovo (ivosidenib), is selling that drug and the rest of its oncology business to Servier Pharmaceuticals LLC in a deal valued at up to $2 billion, plus royalties. The agreement includes $1.8 billion cash up front for the portfolio and as much as $200 million in regulatory milestone payments for the investigational glioma treatment vorasidenib.
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.
Investors appear to be maintaining a bullish outlook on biopharma companies developing innovative medicines, with the BioWorld Drug Developers index currently tracking up over 5% in value so far this month heading into third-quarter earnings season.
The CDC estimates that sickle cell disease affects well over 100,000 Americans, with the disease occurring most often in African Americans. September has been designated as National Sickle Cell Awareness month designed to focus attention on the ongoing research in this field and the need for new treatments.