PASADENA, Calif.--(BUSINESS WIRE)--Genervon Biopharmaceuticals LLC ("Genervon") reported today that Genervon has published in F1000Research the GM604 (aka GM6) clinical trial Phase 2A data. Confidential data were published for open peer review. The data showed favorable shifts in ALS biomarkers and improved clinical and functional measures in ALSFRS-R and FVC during the Phase 2A clinical trial as well as in an advanced ALS patient. The biomarker results in GALS-001 suggests that GM6 modulates ALS disease through multiple pathways.
Our findings suggest a tentative tripartate mechanism of action (MOA) by which GM6 could prolong motor neuron survival in ALS patients. First, by reducing SOD1 expression, GM6 may block accumulation of pathologic SOD1 aggregates in motor neurons. Second, by reducing mitochondrial gene expression and potentially mitochondrial abundance (decreasing total tau), GM6 may disrupt the mitochondrial (intrinsic) apoptotic pathway. Third, GM6 appears to activate developmental/mitotic pathways (Cystatin C), which may promote cellular repair, axonogenesis, and neuron projection.
GM6 is not a cocktail of different molecules. It is an endogenous embryonic stage tyrosine kinase motoneuronotrophic factor regulator. GM6 binds to the insulin receptors, IGF1 receptors, and IGF2 receptors of the human nervous system.
Although a larger trial is needed to confirm these findings, the present data are encouraging and support GM604 as an ALS drug candidate. Genervon is planning a Phase 3 ALS trial in 2017 in the US. Enrollment detail will be announced later. DOI: http://dx.doi.org/10.12688/f1000research.10519.1
Linked below is a one page Executive Summary of Genervon GM604 which contains 10 links covering the discovery and history of GM604 as well as patients' reports and videos. https://www.dropbox.com/s/c5yg6nsp91lvs65/Genervon%20Executive%20Summary%2020170320.pdf?dl=0
Genervon discovered the endogenous embryonic stage multi-target regulator GM6. GM6 modulates 89 ALS, 48 AD, 46 PD and 9 MS (FDR < 0.10, FC > 2) associated defective genes to bring homeostasis for the respective patient groups of these progressive, incurable and fatal neurodegenerative diseases. The uniform failure of the curative ALS trials and 99.6% failure rate of AD trials is a testimony that the pervasive and dominating drug development paradigm of single gene/target reductionism cannot cure ALS and other neurological and neurodegenerative multifactorial diseases.