Beam Therapeutics Inc. BEAM on Saturday offered extra information from the Part 1/2 medical trial of BEAM-302 in sufferers with alpha-1 antitrypsin deficiency (AATD) on the 2025 Alpha-1 Basis seventh World Analysis Convention and tenth Affected person Congress.
AATD is an inherited genetic dysfunction that may trigger early onset emphysema and liver illness.
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Preliminary security and efficacy information from the Part 1/2 trial of BEAM-302 had been beforehand reported in March 2025, establishing medical proof of idea as a possible therapy for AATD and in vivo base enhancing.
Preliminary outcomes from the primary three single-ascending dose cohorts demonstrated that BEAM-302 was properly tolerated.
Single doses of BEAM-302 led to sturdy dose-dependent correction of the disease-causing mutation and whole AAT protein ranges above the therapeutic threshold within the 60 mg dose cohort.
These beforehand reported information had been included within the presentation, alongside up to date biomarker information from the 60 mg cohort displaying ranges of corrected protein (M-AAT) and the mutant type of alpha-1 antitrypsin protein (Z-AAT) out to Day 28 for all three sufferers.
At Day 28, the proportion of corrected M-AAT reached a imply of 91% of whole AAT in circulation, surpassing ranges noticed in sufferers with the MZ genotype the place circulating M-AAT is often ~80%.
As well as, therapy with BEAM-302 led to a imply lower of 79% of circulating mutant Z-AAT from baseline as of Day 28.
Beam plans to proceed the dose-escalation portion of Half A of the continued Part 1/2 trial, together with enrolling and dosing a fourth dose cohort of 75 mg, and expects to report additional information at a medical convention within the second half of 2025.
As well as, the corporate plans to dose the primary affected person in Half B, together with AATD sufferers with delicate to average liver illness, within the second half of 2025.
The U.S. Meals and Drug Administration not too long ago cleared Beam Therapeutics’ investigational drug utility for BEAM-302, enabling the corporate to activate websites within the U.S. for its ongoing Part 1/2 trial.
Worth Motion: BEAM inventory is down 6.75% at $14.24 on the final test on Monday.
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