Beam Therapeutics lost more than 10% on Tuesday after releasing updated Phase 1/2 trial data for BEAM-302, its gene-editing therapy for alpha-1 antitrypsin deficiency. The company reported that the treatment was well tolerated but noted elevations in liver enzymes, including alanine transaminase and aspartate aminotransferase. The data cut from June 24 covered the dose-escalation portion of the study, which tested doses up to 75 mg in Part A and Part B cohorts, but excluded the multi-dose cohort and Part A expansion cohort. One patient with underlying AATD-linked liver disease in Part B experienced a brief Grade 3 case of ALT/AST elevations after receiving 60 mg, the dose selected for an accelerated approval pathway. J.P. Morgan analyst Brian Cheng, who rates Beam Overweight, downplayed the safety event, saying it is not a major overhang but could affect perception of the drug's prospects. The company plans to expand the study globally, targeting 50 additional patients with AATD-associated lung disease, with dosing of this registrational cohort starting in July.
Beam Therapeutics IncUpdated Phase 1/2 data for BEAM-302 showed liver enzyme elevations including a Grade 3 ALT/AST case, raising safety concerns for the gene-editing therapy.
