Beam Therapeutics Inc. BEAM announced positive initial safety and efficacy data from a phase I/II study, which is evaluating its pipeline candidate, BEAM-302, for treating patients with alpha-1 antitrypsin deficiency (AATD).

Currently, there are no approved curative treatments for the given indication.

Data from the study showed that treatment with a single dose of BEAM-302 demonstrated durable, dose-dependent increases in total and functional alpha-1 antitrypsin (AAT). Also, new production of corrected protein (M-AAT) and decreases in toxic mutant Z-AAT were observed in circulation across the initial three doses.

All three patients treated with BEAM-302 (60 mg) achieved a mean total AAT of 12.4µM at day 28, exceeding the 11µM protective therapeutic threshold. Also, treatment with BEAM-302 (60 mg) reduced mutant Z-AAT up to 78%.

The initial safety finding suggests that treatment with BEAM-302 was well tolerated across all dose levels, leading to durable dose-dependent correction of the disease-causing mutation.

Despite the initial positive data, shares of Beam Therapeutics were down 9.8% on March 10 following the news announcement.

In the past year, shares of Beam Therapeutics have plunged 28.2% compared with the industry’s decline of 8.1.

Image Source: Zacks Investment Research

More on BEAM's Phase I/II Study on BEAM-302

The open-label, dose expansion phase I/II study evaluates the safety, tolerability, pharmacodynamics, pharmacokinetics and efficacy of BEAM-302 for treating AATD, an inherited genetic disorder that can cause early onset of emphysema and liver disease.

Per the company, nine patients were administered a single-ascending fixed dose of BEAM-302 (15 mg, 30 mg and 60 mg) in part A of the phase I/II study. The initial safety and efficacy data announced are from all nine patients as of the data cut-off date of Feb. 26, 2025.

The phase I/II study consists of two parts. Part A of the study will investigate BEAM-302 in AATD patients with lung disease, while part B of the study will investigate BEAM-302 in AATD patients with mild-to-moderate liver disease with or without lung disease.

The company plans to dose the first patient in part B of the phase I/II study in the second half of 2025.

Beam Therapeutics expects to enroll additional patients in a fourth-dose cohort in the ongoing phase I/II study with data from the same expected in the second half of 2025.

BEAM-302 is a liver-targeting lipid-nanoparticle (LNP) formulation of base editing reagents designed to correct the disease-causing PiZ mutation.

BEAM's Zacks Rank & Stocks to Consider

Beam Therapeutics currently carries a Zacks Rank #3 (Hold).

Some better-ranked stocks in the drug/biotech sector are Xencor, Inc. XNCR, Anixa Biosciences, Inc. ANIX and BioMarin Pharmaceutical Inc. BMRN, each carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

In the past 60 days, estimates for Xencor’s loss per share have narrowed from $3.64 to $2.87 for 2025. In the past year, shares of XNCR have lost 40.2%.

XNCR’s earnings beat estimates in two of the trailing four quarters while missing the same on the remaining two occasions, the average negative surprise being 4.77%.

In the past 60 days, estimates for Anixa Biosciences’ loss per share have narrowed from 46 cents to 41 cents for 2025. In the past year, shares of ANIX have decreased 14.6%.

ANIX’s earnings beat estimates in three of the trailing four quarters while missing the same on the remaining occasion, the average surprise being 6.31%.

In the past 60 days, estimates for BioMarin’s earnings per share have increased from $4.01 to $4.25 for 2025. In the past year, shares of BMRN have declined 15.4%.

BMRN’s earnings beat estimates in each of the trailing four quarters, the average surprise being 32.36%.

Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report

BioMarin Pharmaceutical Inc. (BMRN) : Free Stock Analysis Report

Beam Therapeutics Inc. (BEAM) : Free Stock Analysis Report

Xencor, Inc. (XNCR) : Free Stock Analysis Report

ANIXA BIOSCIENCES INC (ANIX) : Free Stock Analysis Report

This article originally published on Zacks Investment Research (zacks.com).

Zacks Investment Research