Helixmith Planning Phase 2 Trial of Engensis, Potential Gene Therapy for ALS

Helixmith Planning Phase 2 Trial of Engensis, Potential Gene Therapy for ALS
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Helixmith has selected Worldwide Clinical Trials to run a planned Phase 2 study of Engensis (VM202), its potential gene therapy for amyotrophic lateral sclerosis (ALS), according to a company press release.

Engensis consists of a circular piece of DNA called a plasmid that carries instructions for the hepatocyte growth factor (HGF) protein.

HGF promotes the formation of new blood vessels (angiogenesis), prevents muscle atrophy, and participates in the growth and survival of nerve cells, potentially promoting nerve regeneration. In a past study in people with diabetic neuropathy, injections of Engensis into muscles led to the production of HGF within the body.

As ALS is characterized by progressive damage to motor neurons, leading to reduced muscle control, boosting HGF production in the body might slow or stop the disorder’s progression.

An earlier Phase 1/2 trial (NCT02039401) found weekly intramuscular injections of Engensis to be safe and well-tolerated among 18 ALS patients, and to potentially slow disease progression. This led the U.S. Food and Drug Administration to grant the medication orphan drug and fast track designations for ALS.

“In our Phase 1 trial, patients’ disease progression showed trends toward improvement for two to three months after Engensis injection. We hope to replicate these results in our upcoming Phase 2 [trial],” said Sunyoung Kim, CEO of Helixmith.

Helixmith, formerly known as ViroMed, signed a master service agreement appointing Worldwide to manage the trial’s clinical operations on Aug. 7. The trial is due to take place in the United States and to begin late this year.

“Helixmith made the decision to conduct the upcoming trial with Worldwide because of their expertise in ALS clinical studies,” Kim said.

Worldwide is a global contract research organization with niche expertise in operating clinical trials for rare diseases and those of the central nervous system (the brain and spinal cord), such as ALS. Other areas of Worldwide’s focus include cardiovascular and metabolic disorders, general medicine, and oncology.

Headquartered in the U.S., Worldwide maintains networks in over 60 countries.

“The collaborative engagement demonstrated between … Helixmith and Worldwide has been exceptional,” said Michael F. Murphy, chief medical and scientific officer for Worldwide.

“Both the trial design and operational concepts which have been developed will assure an efficient and informative clinical evaluation of this unique therapeutic approach in a neuromuscular disorder with exceptional unmet clinical need,” he added.

Forest Ray received his PhD in systems biology from Columbia University, where he developed tools to match drug side effects to other diseases. He has since worked as a journalist and science writer, covering topics from rare diseases to the intersection between environmental science and social justice. He currently lives in Long Beach, California.
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Inês holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in blood vessel biology, blood stem cells, and cancer. Before that, she studied Cell and Molecular Biology at Universidade Nova de Lisboa and worked as a research fellow at Faculdade de Ciências e Tecnologias and Instituto Gulbenkian de Ciência. Inês currently works as a Managing Science Editor, striving to deliver the latest scientific advances to patient communities in a clear and accurate manner.
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Forest Ray received his PhD in systems biology from Columbia University, where he developed tools to match drug side effects to other diseases. He has since worked as a journalist and science writer, covering topics from rare diseases to the intersection between environmental science and social justice. He currently lives in Long Beach, California.
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