Beam Therapeutics shares were down after the company shared updated data for its trial of a treatment for the genetic condition alpha-1 antitrypsin deficiency.
Shares declined 9% to $26.88 on Tuesday. The stock is roughly flat this year.
In a Phase 1/2 trial evaluating BEAM-302 for AATD, the treatment was well tolerated with an acceptable safety profile, the company said.
The updated results were not worse than previous data, but were not numerically better, either, Evercore ISI analysts led by Cory Kasimov said.
At the 12-month mark, total alpha-1 antitrypsin in treated patients came down to 13.5 uM to 14.4 uM, compared with 16.1 uM at the nine-month mark. The treatment is supposed to increase alpha-1 antitrypsin, a protein made by the liver.
About 41% of patients experienced adverse events, with the most common being mild-to-moderate infusion-related reactions.
Based on the feedback from the Food and Drug Administration, Beam plans to pursue an accelerated approval pathway for the treatment. Beam plans to enroll 50 additional patients to support a future biologics license application, the company said.