Last October, Genervon Biopharmaceuticals announced promising results in its Phase IIa trial in ALS of GM6, a peptide-based drug that modulates multiple pathways involved in inflammation, apoptosis and hypoxia. Now, the company is submitting additional data to the US Food and Drug Administration from a compassionate use trial of a single ALS patient in late stages of the disease. The patient, who was diagnosed with ALS 10 years ago, was given 6 doses of GM6 and followed for 12 weeks. The company reported small but significant improvement relative to baseline, including an increase in swallow volume and oral volume consumption. In addition, biomarker data on SOD1, Cystatin and total tau revealed changes toward the normal values. Although these data result from a single patient, they support further evaluation in larger cohorts of late-stage patients.
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