BEAM - Beam drops plans to submit IND for sickle cell disease candidate in 2022

Beam Therapeutics ( NASDAQ: BEAM ), a biotech focused on genetic medicines, announced Monday that the company will not file an investigational new drug (IND) for base editing candidate BEAM-102 targeting sickle cell disease in 2022. The shares dropped ~5% in reaction.

The Cambridge, Massachusetts-based biotech has decided to postpone plans for BEAM-102 IND in favor of BEAM-101, the lead program in its Wave 1 strategy for SCD.

BEAM-101 is designed to upregulate the expression of fetal hemoglobin (HbF) as opposed to BEAM-102, which directly edits a defective mutation.

The company is on track to enroll the first patient in the Phase 1/2 clinical trial for BEAM-101 called BEACON later this year.

Beam ( BEAM ) is speeding up the “overall BEAM-101 development program, where we see an opportunity to potentially seek regulatory approval on data generated from the BEACON trial,” Chief Executive John Evans noted.

In conjunction with the disclosure, Beam ( BEAM ) announced its Q3 results for 2022, which exceeded Street forecasts for revenue but fell short of the consensus for earnings.

While revenue for the quarter climbed to $15.8M from $8.6M in the prior year period, the company’s net loss nearly quadrupled to $109.6M as R&D expenses jumped ~56% YoY to $85.3M. However, Beam’s ( BEAM ) cash and equivalents stood at $1.1B at the end of the quarter compared to $956.5M in 2021 year-end.

Verve Therapeutics ( VERV ), which has a licensing deal with Beam ( BEAM ) for base editing, also announced on Monday that the FDA placed its IND application for lead asset VERVE-101 on hold.

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