The Food and Drug Administration (FDA) issued a statement acknowledging the concerns about Genervon’s investigational drug, GM604, for the treatment of amyotrophic lateral sclerosis (ALS).

The FDA is prohibited by law from releasing confidential information about experimental drugs. However, the Agency requests Genervon to release data from their recently completed trial to allow a more thorough discussion of study findings among the ALS stakeholders. The released information should include the pre-specified clinical outcome measures as assessed by change from baseline observations obtained just before randomization to trial drug or placebo.

GM604 is a novel endogenous, multi-target, master regulator signaling peptide that has received both Fast Track and Orphan Drug designations from the FDA for the treatment of ALS. A Phase 2a trial for GM604 was designed to assess whether a 6-dose treatment in two weeks will start ALS disease modification process. The results were measured from baseline to various time points within 12 weeks or after dose cessation for 10 weeks by clinical outcome and biological effects. Data from the 12 patients showed statistical significance in disease modification. GM604 reduced the decline in patients’ forced vital capacity (FVC) vs. placebo from screening to Week 12 (FVC% change vs. -5.60 vs. -22.61, P=0.0359). Also, when compared to placebo, the GM604 treated group had slower disease progression from before treatment initiation as measured by ALSFRS-R slope. A further analysis using historical data in ALS patients showed statistical significance with GM604 treatment vs. placebo (P=0.0047).

In March 2015, Genervon filed a formal request for the Accelerated Approval program to the FDA and is awaiting a final decision. The FDA stated that it will provide more advice and support to Genervon as they further explore the safety and efficacy of GM604 in the treatment of ALS.

FDA Addresses Investigational ALS Drug Concerns

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