Four months after the FDA slapped a clinical hold on Beam Therapeutics’ phase 1 ready off-the-shelf CAR-T, the biotech is now free to proceed.
The agency’s concerns about the therapy, dubbed BEAM-201, were outlined in a letter in August along with questions about the company’s gene editing technology and request for more control data for a “cytokine-independent growth assay.” The hold was originally placed in late July.
Beam announced today that the agency has lifted the hold and cleared the CAR-T to enter the clinic for T-cell acute lymphoblastic leukemia and lymphoma.
All approved CAR-Ts to date are autologous, meaning they use T cells from the patient’s own body. A number of Big Pharmas and biotechs are now investigating allogeneic, or “off-the-shelf” CAR-Ts, which use donor cells. The furthest along is Allogene, which entered phase 2 in October.
With the lifting of the clinical hold, Beam is now back in the race. The company’s take on the allogeneic model is to use base editing technology to alter one of the four main bases that make up the human genome. That contrasts with the more popularized gene editing method, including CRISPR, which opens up the DNA helix to make larger edits.
In a Dec. 2 release, Beam CEO John Evans heralded the FDA’s decision as “an exciting moment for Beam and for the field of gene editing, as it represents the first IND clearance for a multiplex-base edited investigational drug.
“We believe the future of cell therapy involves high levels of cell engineering, enabled by multiplex base editing technology,” Evans added. “Combining four unique edits with high efficiency, BEAM-201 has the potential to make a substantial impact for patients diagnosed with these challenging T-cell cancers, who lack innovative, new treatment options for their disease.”
The clinical hold hasn’t been the only bump in the road for Beam this year. The company reported in August that more edits than expected would be necessary to make its cell therapies fully allogeneic. The revelation meant the company no longer expected to nominate a second CAR-T candidate in 2022, Beam said at the time.
Elsewhere, the company has had more positive news, with Pfizer signing on to a four-year, $1.35 billion pact in January covering three in vivo programs targeting rare diseases in the liver, muscle and central nervous system. Beam also linked up with Apellis Pharmaceuticals in 2021 in a $75 million collaboration.
Last month, the company enrolled the first patient in a phase 1/2 trial of its BEAM-101 autologous hematopoietic stem cell therapy in patients with sickle cell disease. Days earlier, the company announced that a focus on BEAM-101 meant it was no longer planning to apply this year for another potential sickle cell disease treatment called BEAM-102 to enter human trials.