FDA halts testing of Beam’s base editing cancer therapy

The Food and Drug Administration has halted testing of a preclinical cancer medicine from Beam Therapeutics, the biotechnology company announced Monday.

Beam, a high-profile developer of a gene editing technique known as base editing, said in a short statement that the FDA put its request to start human trials of the experimental treatment on clinical hold.

Beam didn’t say why the FDA paused its application. The biotech was informed of the agency’s decision via an email on Friday, and expects to provide an update “pending discussion” with the FDA. The regulator will provide Beam with a formal letter within 30 days.

Company shares fell by more than 10% in pre-market trading Monday.

Beam is the leading developer of base editing, an approach borne out of research from the labs of Harvard University gene editing specialist David Liu. Unlike the first generation of CRISPR editing, which cuts both strands of DNA, base editing is designed to change single DNA “letters” without causing a double-stranded break, a method that’s thought to carry fewer risks.

Beam was formed five years ago to turn the approach into human medicines and has since received significant financial support. The company raised $180 million in an initial public offering in February 2020 and in January got $300 million upfront from Pfizer in a wide-ranging research deal. The biotech had $1.2 billion in cash on its balance sheet at the end of the first quarter.

The company has already been cleared by U.S. regulators to start a study of BEAM-101, a drug for sickle cell disease, and expects to start enrolling patients in that trial later this year. Verve Therapeutics also recently began clinical testing of a heart disease drug that uses Beam’s base editing technology.

BEAM-201, an experimental treatment for leukemia and lymphoma, was expected to follow this year along with a second sickle cell drug called BEAM-102.

Verve’s treatment is an infusion of a drug that performs base editing inside the body. Beam’s two most advanced programs, including the cancer drug now on hold, genetically modify cells outside the body.

BEAM-201 is meant to overcome some of the limitations of personalized cancer cell therapies from Bristol Myers Squibb, Novartis and Gilead, which are approved to treat certain leukemias and lymphomas. The treatment uses cells from donors, rather than patients themselves, and silences multiple genes simultaneously — an approach Beam claims could make those cells more durable. Several developers of so-called off-the-shelf cell therapies have struggled to prove their drugs are as long-lasting as personalized treatments, however.

The drug is the latest gene-based medicine, meanwhile, to be slowed by regulators. The FDA has recently paused testing of a number of gene replacement or gene editing therapies, wary of potential safety concerns.

“This is obviously negative for the stock and reiterates a high level of scrutiny from the regulators on novel technologies like gene/base editing,” wrote RBC Capital Markets analyst Luca Issi in a research note. “However, we also note that the [application] was submitted at the end of June, so we assume no patient has been dosed, and it is possible that the hold is simply procedural in nature.”

Beam is seeking to treat patients with either relapsed or refractory T cell acute lymphoblastic leukemia or T cell lymphoblastic lymphoma.

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