Controversy over Genervon’s ALS drug GM604 study

A very small study for privately-held US firm Genervon Biopharmaceuticals’ amyotrophic lateral sclerosis (ALS) drug GM604 has raised questions about the data that were provided by the company, which has aroused an angry campaign by ALS patients to gain an accelerated FDA approval.

Genervon's 12-person study, with four people assigned to a placebo arm, assessed the drug's performance on several biomarkers for the disease, which would not be sufficient to gain an accelerated approval.

The company said that, despite the fact that Genervon's Phase IIa trial of GM604 for ALS was conducted by respected, independent professionals at two of the leading medical facilities in the country, some have questioned the integrity of the data collected by Genervon in those trials and previously. However, Genervon has complied with all US Food and Drug Administration requirements regarding data collection, management, and analysis, a process that involves many independent contractors and personnel precisely for that reason - to ensure the integrity of preclinical and clinical data.