VM Biopharma’s Gene Therapy to Treat ALS Approved for Fast Track Development by FDA

The U.S. Food and Drug Administration (FDA) has given Fast Track designation to VM BioPharma’s lead investigational drug candidate, VM202, a potential gene therapy for the treatment of amyotrophic lateral sclerosis (ALS).

VM BioPharm is planning to publish data from its Phase 1/2, open-label trial assessing the safety and tolerability of VM202 (primary endpoints) in people with ALS later this year. The trial’s secondary endpoints included measures of the ALS Functional Rating Scale (ALSFRS-r), an efficacy parameter.

“We are very pleased to announce that the FDA has granted Fast Track designation for VM202 in the potential treatment of ALS. This status underscores the need to expedite potentially new and important treatment options for the ALS community,” said Seungshin Yu, head of new business development of ViroMed Co., VM BioPharma’s parent company, in a press release. “The Fast Track designation, coupled with the recent Orphan Drug designation from the FDA, provides even more momentum in our work to address the urgent and significant unmet medical need that remains in the treatment of people with ALS.”

VM202 is a DNA-based drug intended to improve microvasculature and regenerate nerve cells. When VM202 is injected into patients, it produces a protein called hepatocyte growth factor (HGF), which is to induce angiogenesis — the formation of new blood vessels — and acts as a neurotrophic factor.

The drug is also in clinical testing as a potential treatment for diabetic neuropathy and limb ischemia.

ALS, a disorder that involves the death of neurons, is characterized by stiff muscles, muscle twitching, and gradually increasing muscle weakness that results in difficulty speaking, swallowing, and eventually breathing. The disease’s cause  is not known in 90% to 95% of cases. In others it is an inherited disease, and about half of these genetic cases are due to one of two specific genes.

Fast Track designation is intended to speed the development and review of drugs under study to treat a serious or life-threatening condition and fill an unmet medical need.