Beam Therapeutics’ leukemia and lymphoma CAR-T therapy has been stopped in its tracks by the FDA after a clinical hold was placed on the new drug application.
In a very brief update posted Monday morning, Beam said that the FDA informed the base editing company via email that the new drug application for BEAM-201 has been placed on a clinical hold. No further details on the nature of the hold were provided. The FDA will be providing additional details within 30 days, and Beam promised an update when more details were available.
Shares of Beam fell over 10% in premarket trading to $56.25, compared to a close on Friday of $62.98.
Beam is trying to move BEAM-201 into human study for relapsed/refractory T-cell acute lymphoblastic leukemia and T-cell lymphoblastic lymphoma. The therapy is an anti-CD7, multiplex-edited, allogeneic CAR-T. The company has highlighted this therapy from its ex vivo T-cell program as a priority for 2022.
The request for phase 1 study was submitted at the end of June, Beam said.
Beam signed a massive licensing deal with Pfizer in January to work on gene editing therapies, with $300 million upfront and $1.35 billion possibly available down the line in milestones. The companies are working on three candidates for rare genetic diseases of the liver, muscle and central nervous system.
The biotech is taking a slightly different approach to gene editing than peers like Intellia and Editas with a more precise class of genetic medicines. Beam's base editing technique targets a single base in the genome, rather than creating a double-stranded break in the DNA. In slightly plainer English, that means Beam is attempting to develop therapies that do not create unwanted DNA modifications.
Beam also signed a $75 million partnership with Apellis Pharmaceuticals in 2021.