On Monday, Beam Therapeutics Inc. ( BEAM ) announced initial safety and efficacy data from its Phase 1/2 trial of BEAM-302, establishing clinical proof-of-concept as a potential treatment for alpha-1 antitrypsin deficiency (AATD) and for in vivo base editing.

The initial safety and efficacy data reported are from all nine patients as of February 26, the data cut-off date.

Preliminary results from the first three single-ascending dose cohorts demonstrated that BEAM-302 was well tolerated, with single doses of BEAM-302 leading to durable dose-dependent correction of the disease-causing mutation.

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Alpha-1 antitrypsin (AAT) deficiency is a genetic disorder that can cause lung disease and liver disease. It occurs when the body doesn’t produce enough AAT, a protein that protects the lungs and liver. 

Following a single infusion of BEAM-302, rapid, durable and dose-dependent increases in total AAT, a new production of corrected M-AAT and decreases in mutant Z-AAT were observed in circulation.

Changes in total AAT were observed as early as Day 7, plateaued around Day 21 and were maintained for the duration of follow-up (up to Month 6 in the 15 mg cohort, Month two in the 30 mg cohort and Day 28 in the 60 mg cohort).

Increased total AAT was functional.

Beam plans to continue the dose-escalation portion of Part A of the ongoing Phase 1/2 trial, including enrolling and dosing a fourth dose cohort. It also expects to report further data at a medical conference in the second half of 2025.

In addition, the company plans to dose the first patient in Part B, including AATD patients with mild to moderate liver disease, in the second half of 2025.

Concurrently, Beam Therapeutics ( BEAM ) priced the underwritten offering of 16.15 million shares at $28.48 per share and pre-funded warrants to purchase 1.4 million at $28.47 per pre-funded warrant, with approximately $500 million gross proceeds.

Price Action: BEAM stock is down 3.44% at $27.50 during the premarket session at the last check Monday.

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