Genervon Biopharmaceuticals says its efforts to develop treatments for ALS and other neurodegenerative diseases continue to focus on a factor that regulates stem cells’ evolution into nerve cells.
It continues working on Phase 3 clinical trial plans, it added.
The company outlined its treatment-development strategy at the 2018 BIO CEO & Investor Conference in New York City in February.
In addition to regulating stem cells’ evolution into nerve cells, motoneuronotrophic factor, or MNTF, helps regenerates electricity-conducting nerve cells known as axons. And it regenerates nerve cells connected with muscles and organs.
Scientists have detected the factor in human embryos’ nervous systems when they are three months old. Although it reaches its highest levels in the ninth week of embryo development, its levels remain detectable in adults.
Genervon researchers developed a version of MNTF called GM6 that they are testing in clinical trials. The studies are looking at its safety and effectiveness as a neurodegenerative disorder treatment.
GM6 regulates nervous system development and repair. It can do this because it is able to penetrate the brain, a task that many brain-targeting therapies are unable to pull off. The blood-brain barrier, which shields the brain and central nervous system from blood, is what blocks many treatments.
Once it reaches the brain, GM6 can help correct nervous system damage.
A preclinical-trial study showed that GM6 delayed the start of ALS in mice and improved their survival rate. It also led to improvements in function.
Genervon conducted a Phase 2a trial (NCT01854294) in which researchers randomized 12 patients with rapidly progressing ALS to receive either GM6 or a placebo. Eight received GM6 and four the placebo through intraveneous injections six times over two weeks.
Researchers assessed the therapy’s safety and effectiveness, compared with the placebo, at weeks two, six and 12.
Their measuring tools included changes in patients’ ALSFRS-R and forced vital capacity scores and levels of biomarkers in their cerebrospinal fluid. GM6 improved both scores and led to a significant reduction in ALS-related biomarkers such as tau, SOD1, and TDP-43.
The company is working on a Phase 3 trial of GM6 as an ALS therapy. The U.S. Food and Drug Administration granted it both orphan-drug and fast-track designations as a potential treatment for the disease.
Genervon is seeking partners to help it continue the GM6 trials process.