Beam Therapeutics BEAM announced positive updated safety and efficacy data from a phase I/II study evaluating its pipeline candidate, BEAM-302, for the treatment of patients with alpha-1 antitrypsin deficiency (AATD) across multiple dose levels. Currently, there are no approved curative treatments for this indication.
BEAM-302 is a liver-targeting lipid-nanoparticle (LNP) formulation of base editing reagents designed to correct the disease-causing PiZ mutation.
Year to date, shares of Beam Therapeutics have plunged 13.9% against the industry’s 1.9% rise.
Key Updated Data From BEAM’s Phase I/II Study on BEAM-302
BEAM-302 is currently being evaluated in an ongoing phase I/II open-label study assessing its safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy across dose-escalation and expansion cohorts in 29 patients. The study comprises two parts: part A, which enrolls patients with AATD-related lung disease and part B, which includes patients with varying degrees of liver disease, with or without lung involvement.
Safety data from 26 patients receiving a single dose of BEAM-302 demonstrated a favorable and consistent tolerability profile across doses up to 75 mg in both study parts, with no serious adverse events or dose-limiting toxicities reported.
In the multi-dose cohort, following the second dose of BEAM-302, three patients experienced higher-grade liver enzyme elevations and Grade 2 infusion-related reactions. All liver enzyme elevations were asymptomatic, resolved without intervention and no bilirubin elevations were observed.
Following a single dose of BEAM-302 in part A, patients achieved sustained increases in total AAT levels, with mean concentrations of 16.1 µM in the 60 mg cohort and 14.4 µM in the 75 mg cohort. In the multi-dose cohort, AAT levels reached 16.5 µM after the second 60 mg dose, indicating that a single 60 mg dose is sufficient to achieve near-maximal editing efficiency.
Treatment with BEAM-302 resulted in substantial and sustained reductions in mutant Z-AAT levels across all cohorts, with mean decreases of approximately 84% and 79% at the 60 mg and 75 mg doses, respectively, alongside an 80% reduction observed in the multi-dose cohort.
Following BEAM-302 treatment, corrected M-AAT became the predominant circulating form of AAT, accounting for approximately 94% in the 60 mg cohort and 91% in the 75 mg cohort, with similarly high levels of 93% observed in the multi-dose group.
In Part B, patients with AATD-related liver disease demonstrated consistent efficacy with single doses of 30 mg and 60 mg BEAM-302.
This updated data further supports the potential of BEAM-302 to emerge as a first- and best-in-class, one-time treatment for patients with AATD.
Strategic Development Path for BEAM-302
BEAM plans to advance BEAM-302 via an accelerated approval pathway, based on a primary endpoint of AAT biomarkers evaluated more than 12 months, with 60 mg selected as the optimal biological dose for further development.
To support a future biologics license application, the company anticipates enrolling approximately 50 additional patients with AATD-related lung disease, with or without liver involvement, by expanding its ongoing open-label phase I/II study. BEAM expects to initiate the global cohort in the second half of 2026.
BEAM’s Zacks Rank & Stocks to Consider
Beam Therapeutics currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Catalyst Pharmaceuticals CPRX, ADMA Biologics ADMA and Inovio Pharmaceuticals INO. While CPRX sports a Zacks Rank #1 (Strong Buy), ADMA and INO carry a Zacks Rank #2 (Buy) each at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 60 days, estimates for Catalyst Pharmaceuticals’ 2026 earnings per share (EPS) have risen from $2.55 to $2.87. CPRX shares have gained 21.7% over the past six months.
Catalyst Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 35.19%.
Over the past 60 days, estimates for ADMA Biologics’ 2026 EPS have increased from 85 cents to 96 cents. ADMA shares have lost 34.5% over the past six months.
ADMA Biologics’ earnings beat estimates in one of the trailing three quarters, matched once and missed on the remaining occasion, with the average negative surprise being 1.79%.
Over the past 60 days, estimates for Inovio Pharmaceuticals’ 2026 loss per share have narrowed from $1.26 to $1.12. INO shares have plunged 36.9% over the past six months.
Inovio Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 57.94%.
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