Investing.com -- Shares of Beam Therapeutics Inc. (Nasdaq: BEAM) surged 15% following the announcement of encouraging initial data from the Phase 1/2 trial of its BEAM-302 treatment for Alpha-1 Antitrypsin Deficiency (AATD). The company reported that the single dose of BEAM-302 led to durable, dose-dependent increases in total and functional Alpha-1 Antitrypsin (AAT), production of corrected M-AAT protein, and notable decreases in mutant Z-AAT in circulation across the initial three dose levels.

The data, which was released on March 10, 2025, indicated that the third dose level of BEAM-302 achieved a mean total AAT of 12.4µM at Day 28, surpassing the protective therapeutic threshold, and reduced mutant Z-AAT by up to 78%. Additionally, the treatment was well tolerated at all dose levels with no serious adverse events or dose-limiting toxicities observed.

Beam Therapeutics’ BEAM-302 is a liver-targeting lipid-nanoparticle (LNP) formulation designed to correct the disease-causing PiZ mutation associated with AATD. The positive trial results offer hope for a potential one-time therapy that addresses the underlying pathophysiology of both liver and lung disease associated with AATD.

Professor Noel "Gerry" McElvaney, a specialist in the field, highlighted the breakthrough nature of the data, stating that the direct correction of the PiZ mutation could simultaneously treat lung and liver disease by targeting the root cause, offering the potential for a cure from a single therapeutic administration.

John Evans, chief executive officer of Beam, expressed confidence in the transformative potential of BEAM-302, citing the promising safety profile and sustainable increase of total AAT above the therapeutic threshold. He also emphasized the importance of these results for Beam’s liver genetic disease franchise and the company’s in vivo delivery capabilities.

Beam plans to continue dose escalation in the Phase 1/2 trial and expects to present updated data at a medical conference in the second half of 2025. The company also intends to initiate Part B of the trial, which will include AATD patients with mild to moderate liver disease.

BMO analyst Kostas Biliouris provided a positive outlook on the trial data, saying, "Today’s data suggest that there is a clear path forward for BEAM-302, falling under our best-case scenario." He anticipates a significant uptick in Beam’s stock in response to the news.

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