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Moderator emeritus
Jan 29, 2013
Colorado Springs
Odd that we've not mentioned this since a year ago.
It appears That Genervon was quite pleased with the recent Phase 2a results, and the 'orphan drug' classification is, hopefully, hopeful! ;)

June 30, 2014, 6:01 a.m. EDT
Genervon Announces Biomarker Data from GALS-001 Clinical Trial for ALS

PASADENA, Calif., Jun 30, 2014 (BUSINESS WIRE) -- Genervon Biopharmaceuticals LLC (“Genervon”) today announced that it has analyzed and generated a Biomarker Data Report for its recent Phase 2a clinical trial for ALS disease modification. A full analysis of the trial’s results is expected to be completed in the third quarter of 2014, but preliminary clinical data and this biomarker data suggest that GM604—Genervon’s novel, proprietary, multi-target biological drug candidate—shows significant promise for treating ALS.

Of the eight biomarkers measured in the trial, GM604 modulated six in a manner that suggests disease modification: one “target” biomarker, two “efficacy” biomarkers, two “target/efficacy” biomarkers, and one “prognostic” biomarker. Despite the small size of the trial (twelve patients, of whom eight received GM604 and four received a placebo) and its short duration (a two-week treatment period followed by a ten-week observation period), the modulations of two of the biomarkers were shown to be statistically significant between the treated and placebo groups. These results are consistent with the previously reported clinical trial results indicated that 7 of the 8 treated definite ALS patients had their disease progression slowed or stopped when compared to the historical ALSFRS-R scores of definite ALS patients over the course of the trial.

Genervon is extremely encouraged by these results, particularly given the challenge of detecting even one biomarker signature over such a short trial period, and believes that they support Genervon’s hypothesis that GM604 is a potent “master regulator” drug that modifies ALS by working across multiple genes in multiple pathways through multiple biological processes. Genervon believes that GM604 operates by responding to distress signals from affected genes and regulating those genes bi-directionally to their normal functional ranges, thereby bringing homeostasis to affected biological systems.

Genervon further believes that GM604’s multi-target efficacy sets it apart from the single-target strategies that have failed to translate into effective therapies for neurodegenerative and nervous system disorders. As of 2013, 62 companies have a product in development for ALS. More than 160 ALS clinical trials had failed. It is increasingly apparent that single-target drugs cannot cure most neurological diseases. This is not surprising given that the pathogenesis of those diseases is highly interactive and multifactorial, involving multiple biological targets and processes. Genervon believes that because GM604 is an endogenous human peptide that regulates overall neurological development at the embryonic stage, it has the potential to modify neurological diseases where single-target therapies have failed.

ALS patients lost the ability to initiate and control muscle movement, which often leads to total paralysis and death within two to five years of diagnosis. There is no cure and no life-prolonging treatments for the disease. GM604 has received orphan drug and fast track designation from FDA to treat ALS.


Part of a 2012 press release:

PASADENA, Calif. --

Genervon scientists have confirmed that the target of GM604 regulates over 4,000 genes. More significantly, it modifies ALS disease progression by modulating at least 30 significant ALS related genes. It does this through at least 8 pathways, potentially more, and up to 22 biological processes.
I was disappointed that my sister did not qualify for this trial. It seems like a promising approach and the early reports are encouraging. Hope they get another trial going soon. Thanks for sharing
this looks really promising. what is their next step?!
Hopefully another trial soon. Unfortunately sometimes things look good in the phase 2 and then bomb in the larger phase 3. Remember Dex in 2012 anyone? But I do think their approach is promising ( fingers crossed)
found nothing on alstdi or untangled ...
You won't find als untangled looking into drugs in legitimate clinical trials i think. They are more geared toward the alternative treatments.
I wonder what would happened if I tried one of each at the same time ... still wouldn't know if progression was faster or slower :)

ok, cynic mode off ....
This is the most exciting press release since, well maybe lithium. Hope there is more to it than turned with lithium though. I'm also surprised it hasn't received more attention. Thanks for posting it.
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