FDA halts testing of Beam’s core-edition cancer therapy

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

Beam, a top developer of a gene-editing technique known as base-editing, said in a short statement that the FDA has placed its application to start human trials of the investigational treatment on clinical hold.

Beam did not explain why the FDA suspended its application. The biotech was notified of the agency’s decision via email Friday and plans to provide an update “pending discussion” with the FDA. The regulator will provide Beam with an official letter within 30 days.

Shares of the company fell more than 10% in premarket trading on Monday.

Beam is the lead developer of base editing, an approach born out of research from the labs of Harvard University gene editing specialist David Liu. Unlike first-generation CRISPR editing, which cuts both strands of DNA, base-editing is designed to modify single DNA “letters” without causing double-strand breaks, a method believed to be less risky .

Beam was created five years ago to translate 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 secured $300 million upfront from Pfizer in a high-profile research deal. Biotechnology had $1.2 billion in cash on its balance sheet at the end of the first quarter.

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

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

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

BEAM-201 is intended 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 donor cells, rather than patients themselves, and silences multiple genes simultaneously – an approach Beam says could make these cells more durable. However, several developers of so-called off-the-shelf cell therapies have struggled to prove that their drugs are as durable as personalized treatments.

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

“This is obviously negative for the stock and reiterates a high level of scrutiny from regulators on new technologies such as gene/base editing,” RBC Capital Markets analyst Luca Issi wrote in a statement. research note. “However, we also note that the [application] was submitted at the end of June, so we assume that no patients received a dose, and it is possible that the suspension was merely procedural in nature.

Beam seeks to treat patients with relapsed or refractory T-cell acute lymphoblastic leukemia or T-cell lymphoblastic lymphoma.

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