PASADENA, Calif. (Business Wire)—Winston Ko, founder and chairman of Genervon Biopharmaceuticals LLC (“Genervon”), presented the company’s history, research, and discoveries at the 2018 BIO CEO & Investor Conference at the New York Marriott Marquis on February 12, 2018.
Mr. Ko introduced Genervon’s guiding hypothesis that multi-target drug development is the key to curing complex neurological disorders. Following this insight, Genervon searched for and discovered what it believes is the regulator of the development of the human nervous system, called the “motoneuronotrophic factor,” or MNTF.
MNTF, which is expressed at its highest levels during week 9 of human gestation and remains detectable in adult tissue, has been shown to have both a trophic effect and a tropic effect on neuronal growth. In addition, extensive preclinical testing suggests that MNTF regulates multiple key CNS-related biological functions, including neuronal differentiation, axonal regeneration, reinnervation, and inflammation and apoptosis. Because MNTF is endogenous to the body, its active analog GM6 has been shown to be safe and tolerable in Phase 1 and 2A clinical trials.
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. GM6 is a small, cationic peptide that crosses the blood-brain barrier and activates regulatory pathways to induce an embryonic-like neuroprotective microenvironment that aids in the detection and self-correction of CNS- and neurodegenerative-related pathophysiology.
Genervon has completed a Phase 1 safety trial as well as Phase 2A trials in ALS and Parkinson’s disease patients. ALS patients treated with GM6 experienced a significant reduction in ALS-related disease biomarkers (e.g. tau, SOD1, and TDP-43), as well as a positive signal in clinical benefit as evidenced by a site-specific, significantly slower decline in Forced Vital Capacity (FVC) between placebo and treatment groups over a 12-week period, as well as improvement in treated patients’ ALSFRS-R scores when compared to a historical placebo group.
Genervon holds more than 80 patents worldwide for MNTF, GM6, and related compounds and methods and uses. GM6 has obtained orphan-drug designation from both FDA and the European Medicines Agency, as well as a fast-track designation to treat ALS from FDA.
Genervon is currently planning Phase 3 clinical trials for GM6 for ALS, and Phase 2 trials for Alzheimer’s disease, Parkinson’s disease, Huntington’s disease, and multiple sclerosis indications. Genervon is now inviting pharmaceutical partners to help advance GM6 from a development-stage drug candidate to a marketable therapy for the many victims of neurodegenerative diseases and disorders.