VM BioPharma Announces FDA Fast Track Designation Granted for Investigational Gene Therapy VM202 for Patients with Amyotrophic Lateral Sclerosis (ALS)
VM BioPharma, the United States division of ViroMed Co., Ltd. in Seoul, South Korea (KOSDAQ:084990), today announced that the U.S. Food and Drug Administration (“FDA”) has granted Fast Track designation for the Company’s lead investigational drug, VM202, a Phase 2 novel gene therapy for the potential treatment of Amyotrophic Lateral Sclerosis (ALS). ViroMed is anticipating the publication of data from the Phase 1/2 trial of VM202 in ALS in the second half of the year. The primary endpoint of the trial was the safety and tolerability of VM202, and measures of the ALS Functional Rating Scale (ALSFRS-r) and other efficacy parameters were studied as secondary endpoints.
“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 Dr. Seungshin Yu, head of new business development of ViroMed Co., Ltd. “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.”
The FDA established the Fast Track designation process to facilitate the development and expedite the review of drugs intended to treat serious or life-threatening conditions, and that demonstrate the potential to address unmet medical needs. Through the Fast Track program, a product may be eligible for priority review at the time of a new drug application (NDA) filing and may also be eligible to submit completed sections of the NDA on a rolling basis before the complete application is submitted. These expedited processes can significantly cut down the development time and cost associated with bringing a drug to market, and the orphan-drug designation also allows for 7 years of market exclusivity even after expiration of related patents.
About Amyotrophic Lateral Sclerosis
Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is a rare neurodegenerative disease of unknown causes where the motor neurons necessary to move muscles in the body are destroyed, leading to paralyzation of all muscles, such as those in the tongue, neck, and the limbs. The disease occurs mostly in adults and average life expectancy is 2-5 years of symptoms onset. According to the National Institutes of Health (NIH), prevalence of ALS in the U.S. is 3.9 cases per 100,000 persons, and there are currently an estimated of 450,000 patients around the world[i]. With roughly 20,000-30,000 patients in the U.S currently, there are an estimated 6,400 new patients are diagnosed with the diseased every year. (ref: ALS TDI, ALS Association).
VM202 is a proprietary gene therapy from VM BioPhrama targeting four different indications. When injected into patients, VM202 produces hepatocyte growth factor (HGF) protein, which induces angiogenesis (formation of new blood vessels) and acts as a neurotrophic factor to stimulate regeneration of damaged nerve cells. VM202 successfully completed a Phase 1/2 study for amyotrophic lateral sclerosis (ALS) in the U.S. and the drug was granted orphan drug status by the U.S. FDA on February 6, 2014.
In addition to ALS, ViroMed is advancing the study of VM202 and enrolling patients in a trial for painful diabetic neuropathy (DPN) after successful Phase 2 results. The Company also completed a Phase 2 clinical study of VM202 for the treatment of critical limb ischemia, and has received an investigational new drug (IND) approval from the FDA to initiate a Phase 3 study targeting chronic non-healing ischemic foot ulcer in diabetic patients. A Phase 2 trial of VM202 is also planned for coronary artery disease (CAD) in Korea.
About VM BioPharma and ViroMed Co., Ltd.
VM BioPharma is a U.S. division of ViroMed Co., Ltd., an R&D focused biopharmaceutical company founded in 1996 and based in Seoul, Korea. ViroMed is developing new and innovative biopharmaceuticals for the treatment of currently untreatable diseases. The current development focus is on the proprietary plasmid DNA-based drug VM202 in cardiovascular and neurological diseases at various clinical stages in U.S., Korea, and China.
ViroMed has assembled a diverse yet technologically and conceptually linked pipeline. Other research areas include antibody-based cancer therapeutics, immune disorders, recombinant protein-based thrombocytopenia treatment, and CAR-T technology. With constant track record of clinical efficacy and quality molecular biological research, ViroMed aims to become a trailblazer in the field of gene therapy.