Genervon Announces Phase 2a Trial Results in ALS

Genervon Biopharmaceuticals LLC announced the results of their Phase 2a trial of GM604 today. The trial was designed to determine whether a six-dose treatment of GM604 would begin the process of disease modification. The Phase 1 study began midway through 2013, and according to its public protocol, enrolled 12 patients in a double blind, placebo controlled clinical trial. According to the press release dated October 19, 2014, results suggest GM604 was shown to be safe and tolerable when delivered intravenously, “GM604 significantly reduced the decline in ALSFRS-R and slowed or reversed decline in forced air capacity (FVC).” Genervon is also reporting statistically significant data for target, efficacy, prognostic biomarkers as well as TDP-43 and SOD1 genes in ALS. Results from a Phase 2a in Parkinson’s disease was also shown to be statistically significant. Genervon has submitted the results of these trials to the FDA for guidance on how to make GM604 available as a therapeutic drug.
 
While ALS TDI is encouraged to hear these results, we cannot comment further until the full datasets from the clinical trial are available for review.  ALS TDI cautions that the press release results not be over-interpreted.  A Phase 1 study is used to develop data in support of establishing the safe use of a new potential medicine for any disease.  With such a small number of people involved (12) and with such a short period of time (12 weeks, six doses) this study was not designed or powered to effectively determine any impact of the progression rate of ALS in general.  It would be challenging to prove efficacy of a compound as an effective treatment of ALS with such a small cohort, especially given the heterogeneity of ALS.   Other than the press release, no further information is available on the demographics of the cohort. However, there are several upcoming scientific conferences in 2014, and we hope that more information will be made available during one or more of those meetings, such as the International ALS/MND Research Symposium in Belgium of which ALS TDI is a proud sponsor.
 
 
BrainStorm Cell Therapeutics Inc. announced today that the FDA has designated NurOwn™ as a Fast Track product for the treatment of ALS. The FDA’s Fast Track program is designed to expedite the review of drugs and biologics intended to treat serious conditions and meet unmet medical needs through increased meetings and written communications with the FDA. A Phase 2 clinical trial of NurOwn™ is still recruiting participants in the US. This Phase 2 study should provide greater understanding of whether or not this therapeutic is an effective approach to treating ALS.  ALS TDI hosted a webinar with Brainstorm at the end of its Phase 1 clinical trial and in preparation of its Phase 2 trial launch in the US. We encourage people to view that conversation to learn more about this approach.
 
 
Cytokinetics provided a program update relating to tirasemtiv, announcing it has completed its review of the Phase 2b trial of BENEFIT-ALS (Blinded Evaluation of Neuromuscular Effects and Functional Improvement with Tirasemtiv ALS) and has concluded that effects observed on Slow Vital Capacity (SVC) in patients treated with tirasemtiv are robust and potentially clinically meaningful. Cytokinetics believes the data supports a Phase 3 clinical trial that could begin in 2015. The company has begun regulatory interactions with the FDA to explore registration of tirasemtiv as a potential treatment for patients with ALS. ALS TDI is excited to hear that the analysis of the Phase 2 clinical trial has been completed, and is encouraged by these results. The Institute is in close communication with Cytokinetics and has participated in a webinar on the initial results and hosts its CEO Robert Blum as keynote speaker at its 2014 Leadership Summit, which will be open to the public for comment and viewable in full via live webcast.