Company: Genervon Biopharmaceuticals
Drug Type: Protein Biologic
Conditions: ALS, stroke, Parkinson’s disease, Alzheimer’s disease, Huntington’s disease, multiple sclerosis
Mechanism Type: Neuroprotection
Mechanism: GM604 is based on motorneuronotrophic factor (MNTF), a fetally expressed six amino acid endogenous neurotrophin that exhibits neuroprotective, anti-apoptotic, anti-oxidative and anti-inflammatory effects. It acts according to Genervon by modulating 8 or more key pathways that are disrupted in ALS.
U.S. Status for ALS: Phase IIA
 A Phase 2A randomized, double-blind, placebo-controlled pilot trial of GM604
in patients with Amyotrophic Lateral Sclerosis (ALS Protocol GALS-001) and a single compassionate patient treatment (Protocol
GALS-C) [version 1; referees: 1 approved with reservations]. Kindy M et al. F1000Research 2017, 6:230.
 Genervon. Therapeutic Applications ALS. Genervon, 2016. Accessed 9 Mar 2016 from http://www.genervon.com/genervon/science_development.php.
 Genervon GM604 Reduced TDP-43 Protein Aggregates to Homeostasis Normal Range and thus Slowed Down Heterogeneous ALS Progression. Genervon, 22 Sep 2015. Accessed 11 Mar 2016 from http://www.genervon.com/genervon/PR20150922.php.
 Genervon Announces ALS and PD Phase 2a Trial Results Businesswire, 19 Oct 2014. Accessed 11 Mar 2016 from http://www.businesswire.com/news/home/20141019005074/en/Genervon-Announces-ALS-PD-Phase-2a-Trial.
 Experimental ALS Drug Triggers Social Media Hype, But Doctors Say Facts about GM604 Are Slim. Fitzgerald, Susan. Neurology Today, 2 Jul 2015 Vol.15(13):20–22. Accessed 9 Mar 2016 from http://journals.lww.com/neurotodayonline/Fulltext/2015/07020/Experimental_ALS_Drug_Triggers_Social_Media_Hype,.8.aspx.
Last updated March 9th, 2019