BEAM's IND for BEAM-302 in Genetic Disorder Study Gets FDA Clearance
Beam Therapeutics Inc. BEAM announced that the FDA has cleared the investigational new drug (IND) application to begin clinical studies on its pipeline candidate, BEAM-302, for the treatment of alpha-1 antitrypsin deficiency (AATD) in the United States.
BEAM-302 is a liver-targeting lipid-nanoparticle (LNP) formulation of base editing reagents designed to correct the disease-causing PiZ mutation.
AATD is an inherited genetic disorder that can cause early onset of emphysema and liver disease. Currently, there are no approved curative treatments for the given indication.
In June 2024, Beam Therapeutics dosed the first patient in a phase I/II study on BEAM-302 for the treatment of AATD in the United Kingdom. The candidate has also received clinical trial authorization from New Zealand, Australia, the Netherlands and Ireland.
Year to date, shares of Beam Therapeutics have declined 10.6% against the industry's increase of 3.3%.
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Earlier this month, Beam Therapeutics announced positive initial safety and efficacy data from the phase I/II study evaluating BEAM-302 for treating patients with AATD.
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.
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.
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 the candidate 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 Therapeutics currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Jazz Pharmaceuticals, Inc JAZZ, Dynavax Technologies Corporation DVAX and Krystal Biotech, Inc. KRYS, 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 Jazz Pharmaceuticals' earnings per share have increased from $22.06 to $23.33 for 2025. During the same time, earnings per share estimates for 2026 have increased from $23.13 to $23.35. Year to date, shares of JAZZ have risen 2.6%.
JAZZ's earnings beat estimates in three of the trailing four quarters while missing the same on the remaining occasion, the average surprise being 3.20%.
In the past 60 days, estimates for Dynavax's earnings per share have increased from 32 cents to 33 cents for 2025. During the same time, earnings per share have increased from 49 cents to 57 cents for 2026. Year to date, shares of DVAX have risen 7.4%.
DVAX's earnings beat estimates in three of the trailing four quarters while missing the same on the remaining occasion, the average surprise being 9.58%.
In the past 60 days, estimates for Krystal Biotech's earnings per share have increased from $5.50 to $7.06 for 2025. During the same time, earnings per share estimates for 2026 have increased from $9.15 to $11. Year to date, shares of KRYS have rallied 16.7%.
KRYS' earnings beat estimates in three of the trailing four quarters while missing the same on the remaining occasion, the average surprise being 3.29%.
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