Genervon Biopharmaceuticals announced its future goals for investigational therapy GM6 in a recent presentation on its multi-target drug development strategy to tackle neurodegenerative diseases, including Parkinson’s disease.
Genervon presented its hypothesis that a multi-target drug approach is the key to curing complex neurological disorders at the 2018 BIO CEO & Investor Conference on Feb. 12, 2018, at the New York Marriott Marquis.
Following this strategy, researchers at Genervon previously discovered what they believe is the regulator of the development of the human nervous system, called the motoneuronotrophic factor (MNTF).
The company then developed GM6, a synthetic equivalent of MNTF, and launched a comprehensive clinical program to test GM6 as a potential treatment for Parkinson’s disease and other central nervous system (CNS) diseases.
Phase 2 trials are now being planned to test GM6 in Alzheimer’s disease, Parkinson’s disease, Huntington’s disease, and multiple sclerosis.
MNTF is an endogenous neurotrophin — a growth factor that induces the survival, development, and function of neurons — highly specific of the central nervous system and expressed rapidly during the first three months of human embryonic development of the complete nervous system, peaking at week nine and remaining detectable in adult tissue.
Previous studies showed that MNTF regulates embryonic stem cell differentiation into motor neurons and helps maintain these nerve cells. Additionally, MNTF helps motor axon regeneration and restoration of nerve function in target muscles and organs.
GM6 controls the development, monitoring, and correction of the human nervous system. It can traverse the blood-brain barrier — a highly selective membrane that helps shield the brain and central nervous system from blood circulation — and activate an embryonic program to help detect and repair damages to the central nervous system, including those triggered in Parkinson’s and other neurodegenerative diseases.
Genervon’s pre-clinical studies identified six potential mechanisms of action of GM6 in Parkinson’s disease, including the down-regulation or up-regulation of six genes involved in signalling, growth factor generation, or oxidative stress defense.
In 2013, Genervon launched a Phase 2a clinical trial (NCT01850381) to assess GM6’s safety and tolerability in Parkinson’s disease patients.
The pilot study enrolled six patients, who were randomized to receive either intravenous GM6 and a placebo. They received six doses of GM6 or placebo over two weeks. Researchers measured the levels of brain-derived neurotrophic factor (BDNF), a blood marker for Parkinson’s disease, at the start (visit one), after four doses (visit four), after six doses (visit six) and 10 weeks post-treatment (visit eight).
Changes in BDNF blood levels for each patient relative to baseline were analyzed at visits four, six, and eight. The results showed that GM6 was well-tolerated and linked to favorable changes in BDNF.
GM6 has also been granted orphan drug status and a fast-track designation by the U.S. Food and Drug Administration to treat amyotrophic lateral sclerosis (ALS), and a Phase 3 trial is being planned to test GM6 in ALS.