Genervon is a closely held, clinical-stage biopharmaceutical company focused on novel treatments for neurodegenerative diseases and disorders.

To date, no effective therapy has been developed for such conditions, but Genervon believes that its new paradigm of drug discovery has unlocked the potential for a wide range of therapeutic and even curative drugs.

It is well-known by now that neurodegenerative diseases and disorders are complex, multifactorial conditions, and that multiple biologic targets must be modulated to affect the disease in question. It is not surprising, then, that the current paradigm of single-target drug development continues to result in clinical trial failures with respect to neurodegenerative conditions. And yet for all this complexity, there is a considerable overlap of common pathways and targets across neurodegenerative diseases. It therefore stands to reason that something that could regulate these common underlying pathways and targets could have dramatic effects across a range of neurodegenerative conditions.

Building on this core hypothesis, in 1996 Genervon discovered what it believes is the naturally occurring regulator of the development of the human nervous system, called the “motoneuronotrophic factor” (MNTF), which is endogenously expressed at its highest levels at week nine of fetal gestation and remains detectable at very low levels in adult tissue. Extensive preclinical testing over many years suggests that MNTF regulates multiple key CNS-related biological functions—including neuronal differentiation, axonal regeneration, reinnervation, and inflammation and apoptosis—providing both neuroprotective and neuro-regenerative therapeutic effects. 

Following a multi-year research and drug development effort, Genervon has advanced GM6, a 6-amino-acid active analog peptide of MNTF, into Phase 2 clinical development. Preclinical and clinical data have shown that GM6, which can pass through the blood-brain barrier, is safe and well-tolerated—as one would expect from a compound endogenous to human biology. Moreover, in Phase 2A trials for amyotrophic lateral sclerosis (ALS) conducted at leading research institutions (Massachusetts General and Columbia Presbyterian), as well as in more than two dozen compassionate use or expanded access applications around the world, GM6 has been shown to modulate key biomarkers associated with neurodegenerative disorders and to improve clinical outcomes for patients. Further, bioinformatic analyses have shown that GM6 promotes differential expression of numerous disease-associated genes related to ALS, as well as those related to Parkinson’s Disease (PD), Alzheimer’s Disease (AD) and Multiple Sclerosis (MS), suggesting that GM6 may effectively target the common pathophysiology underlying these conditions. (Click here for a recent press release on Genervon’s ALS strategy

Genervon holds more than 80 patents worldwide for MNTF, GM6 and related compounds and processes and has obtained an orphan-drug designation and a fast-track designation for GM6 to treat ALS. Genervon is currently planning late-stage clinical trials for GM6 for ALS, PD, AD and MS indications. After 20 years of funding its own research and operations, Genervon is now actively seeking partners to help advance GM6 from a development-stage drug candidate to a marketable therapy for the many victims of neurodegenerative diseases and disorders that desperately need help.