Beam Therapeutics Inc. (BEAM:NASDAQ) delivered follow-up data for BEAM-302, an investigational in vivo gene therapy, or base editing medicine, intended to correct the underlying cause of alpha-1 antitrypsin deficiency (AATD), reported Wedbush Analyst Dr. David Nierengarten in an April 6 research note.
"With a clear pathway toward commercial entry in sickle cell disease and continuing room for differentiation in AATD, we anticipate considerable share price movement in the next six months," Nierengarten wrote.
273% Return Potential
Following the recent news, Wedbush reiterated its $57 per share target price on the biotech, noted Nierengarten. In comparison, Beam now is trading at about $15.27 per share. Thus, the implied return for investors is 273%.
"[We] see recent price action as a buying opportunity," Nierengarten wrote.
Beam is rated Outperform.
New Info Provided
According to the life sciences firm, updated biomarker results from the 60 milligram (60 mg) cohort showed decreasing levels of mutant Z-AAT and a high proportion of corrected M-AAT (mean 91%) at 28 days. The mean percent change in Z-AAT at Day 28 was 11% among patients on the 15 mg dose of BEAM-302, 38% for those on 30 mg and 79% for individuals receiving 60 mg. Beam reported other data last month.
The biotech also indicated that 75 mg will be the final dose evaluated, the others having been 15, 30 and 60 mg.
"We continue to regard 11 microns of serum AAT, already achieved at the 60 mg dose of BEAM-302 but not by competing gene therapies, as the threshold necessary for regulatory approval," Nierengarten wrote. "We view increases from these levels as providing upside, noting the existing benchmark does not consider proportion of corrected M-AAT."
Events to Watch For
Beam has multiple catalysts slated for this year, the analyst pointed out and listed them. Launch of a Phase 1/2 trial of BEAM-301 in GSD1a, an autosomal recessive disorder, is expected by this quarter's end. Updated results are due out midyear from the Phase 1/2 BEACON trial evaluating BEAM-101, an investigational, base-edited cell therapy, in sickle cell disease.
Anticipated events in H2/25 include the release of updated results from the Phase 1/2 trial of BEAM-302 in AATD and the start of patient enrollment for this study's liver disease cohort.
Late this year, the trial of BEAM-103 in healthy volunteers will be launched. BEAM-103 is an anti-CD117 monoclonal antibody designed to suppress hematopoietic stem and progenitor cells expressing CD117, part of the biotech's ESCAPE technology. Also coming up later in 2025 is the release of further data on nonhuman primates for ESCAPE.
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