Amylyx ALS Drug: ICER Revised Evidence Report Casts Doubt On Firm’s New Data Analyses

The Institute for Clinical and Economic Review (ICER) opted not to include new data analyses by Amylyx Pharmaceuticals, Inc. in its revised evidence report on the company’s AMX0035 (sodium phenylbutyrate/taurursodiol) candidate for amyotrophic lateral sclerosis due to concerns with missing data and other aspects of the post hoc studies.

As a result, ICER’s latest report reiterates the conclusion from its draft report that the clinical evidence “suggests AMX0035 provides a small to substantial benefit in ALS, but there remains substantial uncertainty about this estimate