Alnylam switching to backup AAT molecule after safety signal

Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) said it will discontinue development of ALN-AAT for alpha-1 antitrypsin (AAT; A1AT; SERPINA1) deficiency-associated liver disease due to a low incidence of elevated liver enzymes observed in a Phase I/II trial. The biotech intends to select a follow-on candidate, for which it plans to submit a CTA next year.

In a presentation at the Oligonucleotide Therapeutics Society meeting in Montreal, Alnylam's data showed that ALN-AAT led to "dose-dependent, and durable knockdown of serum AAT." The company said a single dose of ALN-AAT led to three instances of asymptomatic, transient elevations of liver enzymes among 15 healthy volunteers. ...