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news.US-based Genervon Biopharmaceuticals (Genervon) has completed its Phase IIa clinical trial of its novel, proprietary, multi-target biological drug candidate, GM604, for amyotrophic lateral sclerosis (ALS) disease modification.
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A full analysis of the trial’s results is scheduled to be completed in the third quarter of 2014, but preliminary data suggests that GM604 shows significant promise for treating ALS.
The randomized, double-blinded trial was carried out at two sites with six ALS patients participating at each site.
In the trial, 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.
The company said that disease progression was measured by ALS Functional Rating Scale -Revised (ALSFRS-R), Time Up and Go (TUG), and Forced Vital Capacity (FVC).
The preliminary data showed that, ten weeks after 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.
In the interim the company is planning the next trial for GM604, which has already secured orphan drug and fast track designation from FDA for ALS indication.
The company plans to partner with one or more pharmaceutical companies in connection with this effort.