April 21, 2015
By Mark Terry, BioSpace.com Breaking News Staff
In an unusual move, the U.S. Food and Drug Administration (FDA) made a request on April 17 for Pasadena, Calif.-based Genervon Biopharmaceutical to publicly release data from a recent clinical trial. By law the FDA cannot release data about experimental drugs, but it has requested that Genervon do so in order to facilitate assessments of the drug by other parties.
BioSpace reached out to Genervon for a statement, but did not receive a response by deadline.
GM604 is a potential treatment for amyotrophic lateral sclerosis (ALS), also commonly known as Lou Gehrig’s disease. The company initiated Phase II trials of GM6 in 2013 for Ischemic Stroke, ALS and Parkinson’s disease, as well as a Phase I trial for safety in humans. More recently Genervon completed a Phase IIa clinical trial, which was a six-dose treatment of GM604 for ALS.
The compound has received both Fast Track and Orphan Drug designations from the FDA for the treatment of ALS. The Phase IIa trial was designed to determine if the six-dose treatment, over a two-week period, would start to modify the disease. Over a 12-week period various points were measured and for an additional 10 weeks after the patients stopped taking the drug. There were a total of 12 patients in the study.
Genervon indicates that the drug slowed the patients’ forced vital capacity (FVC) decline versus those taking placebo. It apparently also showed slower disease progression. The company then filed a request for the Accelerated Approval program with the FDA in March 2015.
other factors that are causing controversy are patient pressure with an online petition and a rally at Capitol hill in Washington, D.C. Then Steven Perrin, chief executive officer of the ALS Therapy Development Institute, wrote a critical blog post, suggesting that the size of the trial was too small and the complexity and individual patient progress of the disease makes the study problematic.
The Perrin blog pointed out that the public has rallied around the Genervon study and GM604, which has a relatively small collection of data behind it, compared to NP001, being studied by Neuraltus Pharmaceuticals and Tirasemtiv, being developed by Cytokinetics. He also cites other potential ALS compounds, including Retigabine (GlaxoSmithKline), memantine (developed by Eli Lilly and marketed by several companies, including Merz, Forest and Unipharm), Rasagaline (Teva Neuroscience) and others.
“The ALS community may not appreciate this opinion,” concluded Perrin in his blog, “but each and every patient should consider that GM604 has not been adequately tested in patients as of today. There is still evidence to be collected on long term safety. There is still data to be collected on if it actually makes disease progression worse and does indeed provide therapeutic benefit.”
Genervon shot back with a press release on April 20 aimed at responding to Perrin’s blog post. The press release made several points, including questioning if Perrin had any conflicts of interest, questioning his assumptions and saying that “his confusing analysis is misleading and puzzling.” The company also argues that there is great risk in release of all of their data.
“Through APP [accelerated approval program], the FDA can get the large population data they could never get from Phase III trials while at the same time today’s 30,000 ALS patients in the U.S. will have access to treatment options,” the company wrote in a statement. “It takes a lot more courage for Genervon to allow GM604 to be exposed to a full spectrum of heterogeneous ALS patients through AAP with Phase 4 surveillance requirements than pursuing the much safer route of a very narrowly defined, controllable, small number of ALS patients in a Phase III trial.”
