Beam Therapeutics announced on Mar. 26 that its investigational base editor, BEAM-302, has shown promising results in increasing the functional levels of a key enzyme in patients with alpha-1 antitrypsin deficiency (AATD). The company plans to move forward with pivotal development later this year.
AATD is a genetic disorder caused by mutations in the SERPINA1 gene, leading to low levels of the AAT enzyme. This can result in progressive lung damage, cirrhosis, and sometimes liver failure. Beam’s therapy aims to correct the underlying genetic mutation and boost production of functional AAT.
Phase 1/2 trial data showed that after a single dose of BEAM-302 at 60 mg, 94% of circulating AAT was correctly folded and faulty enzyme concentration dropped by 84%. The average level of circulating AAT reached 16.1 micromolar. William Blair analysts said, “This is the highest mean serum AAT level we have clinically seen to date from SERPINA1 correction approaches,” describing the data as “impressive.”
Initial safety findings indicated that most adverse events were mild or moderate and there were no dose-limiting toxicities. One case of grade 4 liver enzyme elevation was reported but it was asymptomatic and did not require medical intervention. William Blair stated, “Overall, today’s results reinforce BEAM-302’s best-in-class profile and place Beam in the strongest position among emerging genetic approaches to AATD.”
Beam also announced it will seek an accelerated approval pathway from the Food and Drug Administration based on these results. The pivotal expansion portion of its ongoing Phase 1/2 study is expected to begin later this year.
Analysts from H.C. Wainwright said Beam’s latest readout "reinforces our conviction in BEAM-302’s best- and first-in-class potential." If approved, they project annual peak revenues for BEAM-302 could reach approximately $4 billion.
