New Therapy for AATD Could be Best in Class
Research Report

Beam Therapeutics Inc.'s (BEAM:NASDAQ) early results from its ongoing Phase 1/2 trial of BEAM-302 in patients with alpha-1 antitrypsin deficiency (AATD) are "impressive," reported Patrick Trucchio, H.C. Wainwright & Co. analyst, in an April 7 research note. The biotech presented these updated data at the 2025 Alpha-1 Foundation Global Research Conference earlier this month.

"This [clinical] program represents a potential best-in-class solution in a field where current options are limited to chronic protein augmentation therapy for lung disease and no approved therapies for liver disease," Trucchio wrote.

424% Potential Gain

H.C. Wainwright maintained its $80 per share price target on the precision genetic therapy developer, now trading at about $15.27 per share, noted Trucchio. The implied return for investors is 424%.

Beam remains a Buy.

One Therapy, Two Actions

BEAM-302 is the biotech's vivo lipid nanoparticle liver-targeted adenine base editor designed to correct the disease-causing PiZ point mutation in the SERPINA1 gene, the root cause of severe AATD, explained Trucchio. By directly rewriting the A-to-G mutation, BEAM-302 aims to restore functional M-AAT production while reducing the accumulation of toxic Z-AAT aggregates in the liver and circulation. The therapy potentially addresses both pulmonary and hepatic manifestations of the disease with a one-time treatment.

Trucchio reported that new trial results showed that in all three patients of the 60 milligram (60 mg) cohort, after a single infusion, the corrected M-AAT protein reached a mean 91% of total AAT in circulation by Day 28 and lowered mutant Z-AAT by 79% from baseline. Importantly, the entire cohort achieved total AAT levels that surpassed the protective threshold of 11 microns.

[This marks] the first clinical demonstration of durable, dose-dependent correction of the PiZ mutation," Trucchio wrote.

Beam has begun dosing the fourth cohort, which is receiving 75 mg of BEAM-302.

Shown Safe and Efficacious

Cohorts 1 through 3 demonstrated favorable safety, dose-dependent efficacy and durability, and updated data reinforced these results, Trucchio pointed out. As such, BEAM-302 could be the first curative treatment for AATD and also establish clinical proof-of-concept for Beam's in vivo base editing platform. Therefore, currently this therapy "is underappreciated," Trucchio purported.

As for safety, in all three initial dose levels, 15 mg, 30 mg and 60 mg, no serious adverse events, dose-limiting toxicities or grade 3 or higher treatment-emergent adverse events (TEAEs) occurred. Only mild to moderate TEAEs were observed. They included transient infusion-related reactions and asymptomatic grade 1 elevations of ALT/AST, which resolved on their own.

Regarding efficacy, a single dose of BEAM-302 led to increases in total and functional AAT, with the 60 mg cohort achieving a mean of 12.4 microns by Day 28. Importantly, corrected M-AAT reached up to 91% of total circulating AAT, and ZAAT was reduced by 79%.

This supports the dual mechanism of increasing functional protein and decreasing the toxic mutant form. This outcome is consistent with the "80/20" M/Z AAT profile (80% M-AAT and 20% Z-AAT) associated with no disease, the goal for BEAM-302 treatment according to the management team.

"These effects were not only rapid, observed as early as Day 7, but were also sustained, with durability demonstrated out to six months in earlier cohorts," Trucchio highlighted.

Near-Term Catalysts

The analyst relayed that two potential share price-moving events are slated to happen in H2/25.

Beam will announce more data pertaining to Part A, including longer-term follow-up. The company will dose the first patient for Part B, which will consist of AATD patients with mild to moderate liver disease.

Share Structure

As for its stock, Trucchio reported Beam has 99.8 million shares outstanding.

The company's market cap is $1.525 billion. Its 52-week range is $14.72–35.25.