April 28, 2014
Genervon Biopharmaceuticals LLC (“Genervon”) today announced that it has successfully completed its Phase 2a clinical trial for amyotrophic lateral sclerosis (“ALS”) disease modification. A full analysis of the trial’s results is expected to be completed in the third quarter of 2014, but preliminary data suggests that Genervon’s novel, proprietary, multi-target biological drug candidate, GM604, shows significant promise for treating ALS.
The randomized, double-blinded trial was conducted at two sites with six ALS patients participating at each site. Each patient received six intravenous doses of either GM604 (eight patients) or placebo (four patients) over two weeks and was then evaluated at three time points over the following ten weeks. Disease progression was measured by ALS Functional Rating Scale –Revised (ALSFRS-R), Time Up and Go (TUG), and Forced Vital Capacity (FVC).
According to the preliminary data, ten weeks following completion of dosing without further treatment, clinical measurements of ALS disease progression remained the same and unchanged from the baseline in two of the trial’s eight patients treated with GM604, while the rates of degradation of those clinical measurements had slowed in five of the remaining six treated patients.
Additional information regarding the trial is available on request, and a final analysis of the preliminary data will be presented later this year.
In the interim Genervon is planning the next trial for GM604. GM 604 has already received orphan drug and fast track designation from FDA for ALS indication. Genervon intends to partner with one or more pharmaceutical companies in connection with this effort.
Discussions with prospective partner candidates are expected to occur at the American Academy of Neurology Convention in Philadelphia from April 26th through May 3rd; interested partner firms are encouraged to contact Genervon to schedule a discussion.
Genervon is a privately held, clinical-stage biopharmaceutical company in California developing breakthrough multi-target biological drugs to address the world’s critical unmet medical needs.