Helixmith presented three study results of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, at the 2020 Northeast Amyotrophic Lateral Sclerosis Consortium (NEALS) held for two days from Sept. 30.

Engensis (VM202), developed by Helixmith, has received orphan drug status and fast track designation for ALS by the U.S. Food and Drug Administration. VM202 produces a hepatocyte growth factor (HGF) and helps nerve regeneration, blood vessel generation, and muscle atrophy prevention.

Helixmith presented three study results of amyotrophic lateral sclerosis at the 2020 Northeast Amyotrophic Lateral Sclerosis Consortium (NEALS) for two days from Sept. 30. (Helixmith)
Helixmith presented three study results of amyotrophic lateral sclerosis at the 2020 Northeast Amyotrophic Lateral Sclerosis Consortium (NEALS) for two days from Sept. 30. (Helixmith)

NEALS is an academic organization to lead the scientific achievements made in the motor neuron disease field to develop treatments. It also provides various methodologies necessary for ALS research.

At the consortium, Helixmith CEO Kim Sun-young, Dr. Nho Bo-ram, and Professor John A. Kessler of the Northwestern University Medical School presented the study results.

Kim introduced a drug development platform for ALS, “Defeating ALS (DART),” and Nho explained the effects of recombinant AAV1 expressing HGF on motor function and neuromuscular system. Kessler presented a phase 1 and 2 study of VM202 and a plasmid deoxynucleic acid revealing HGF and the plan for phase 2 trials.

“Presenting all three papers at the NEALS meeting shows that the experts have shown great interest in our research and development on ALS,” Kim said. “We expect to report the results of our phase 2 study, which will begin soon, at next year’s meeting.”

ALS destroys motor neurons that control muscle movement, making all kinds of voluntary motions impossible, and eventually, the muscle involved in breathing stops and leads the patient to death.

About 30,000 ALS patients are in the U.S. ALS is a special disease of great social and political interest. Even the FDA has lowered the regulatory bar to facilitate the development of ALS treatments.

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