HELIXMITH

Helixmith Submits Engensis ALS Phase 2a Protocol to FDA

작성자 helixmith 작성일 20-10-06 09:03

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Helixmith Co., Ltd. has announced that the company has submitted a Phase 2a clinical protocol (REViVALS-1A) for amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s disease) using its flagship gene therapy Engensis (VM202) to the US Food and Drug Administration (FDA). The FDA granted Engensis Orphan Drug designation in 2014 and Fast Track designation in 2016 for the ALS indication.

The Phase 1 study supported the apparent safety of Engensis, as no serious adverse events were attributed to the study drug. In addition, ALSFRS-R sub-scores showed that change from baseline in bulbar, gross motor, and respiratory functions was stable for three months after Engensis treatment.

Following a successful Phase 1 trial, Helixmith will further investigate the safety of Engensis in this Phase 2a trial. The primary safety endpoint will be evaluated by the occurrence of serious adverse events during the trial period. Several exploratory endpoints for efficacy include change from baseline in: (1) patients’ muscle functions as measured by ALSFRS-R, (2) muscle strength as assessed by hand-held dynamometry, (3) change in quality of life as measured by Patient Global Impression of Change (PGIC), (4) effect on respiratory function and survival rate improvement, and (5) changes in biomarkers for muscle atrophy.

The study is especially significant in that the gene expression level will be measured by collecting a small amount of muscle tissue from the patients after treatment . In order to conduct this biomolecular analysis, the small-scale Phase 2a trial will be conducted in 18 ALS patients who will be treated and monitored over 180 days. The results are expected to play a major role in understanding the drug’s mechanism of action in ALS treatment.

After completing REViVALS-1A, Helixmith plans to immediately launch a second larger-scale Phase 2 trial (REViVALS-2A) with over 100 participants. Dr. Sunyoung Kim, CEO, commented, “ALS is the indication that attracts the most interest when we discuss licensing possibilities with other pharmaceutical companies. Favorable safety and efficacy signals from the trials could have important business implications.”


[About amyotrophic lateral sclerosis (ALS)]
Also called Lou Gehrig’s disease, ALS is a fatal neuromuscular disease characterized by the progressive deterioration of motor neurons. Death usually occurs within 2 to 5 years of symptom onset, most commonly due to respiratory failure, following the loss of the ability to carry out all voluntary movements. The incidence of the disease is 1 per 20,000 people, and about 30,000 Americans are estimated to have ALS. The specific causes of the disease are yet unknown, however, and no approved curative treatments have yet been approved.

[About Engensis (VM202)]
Engensis (VM202) is a gene therapy that uses plasmid DNA to express hepatocyte growth factor (HGF). Data from 15 years of research and clinical trials, have shown that simple intramuscular injections of Engensis can produce HGF proteins in the body, which could potentially be effective in nerve regeneration, angiogenesis, and muscle atrophy prevention. Engensis was granted Orphan Drug designation in 2014 and Fast Track designation in 2016 for the ALS indication by the FDA .