Petition update

Why we believe Accelerated Approval is the better option for GM604.

Nicholas Grillo
San Francisco, CA, United States

Apr 6, 2015 — While researchers are calling for more clinical trials for Genervon’s GM604, we recognize that the FDA’s Accelerated Approval Program (AAP) is a preferable and an appropriate alternative. Let's look at why that is true:

1) There is an urgent and unmet need to get a viable treatment to People with ALS (PALS). The only option at this time is Rilutek, which extends life expectancy by a few months with no improvement to quality of life. The average life expectancy for PALS is 2-5 years. So by the time another clinical trial for GM604 is completed, the majority of the current generation of PALS will be dead. AAP was designed to meet the needs of populations just like the ALS Community.

2) The common clinical trial design limits patient inclusion to those diagnosed within 2-3 years of trial commencement and those exhibiting an "average" disease progression. In ALS, the notion of an "average" patient is scientifically invalid and indeterminable. This gap in knowledge precludes determining the number of patients to recruit in order to create a valid sample population. This common trial design yields no data on efficacy in late or early stages of the disease.

3) For GM604, AAP is a superior choice over a Phase 3 trial because AAP has the ability to yield more data sooner, across a broader spectrum of patient profile, and at far lower cost. Post-approval studies could include focused single arm trials designed to ask specific questions regarding correlation of clinical outcomes and biomarker responses, and a patient registry to capture more limited but still meaningful data from a larger population of patients (but not necessarily all) receiving the drug as an approved medicine.


Why is the ALS Community so excited about GM604? It's safe, we like the data (see links below), and we believe in Genervon's innovative approach.

1) GM604 is a 6-amino acid endogenous peptide. It is very safe and tolerable as shown in Phase 1 (32 subjects), ALS Phase 2A (12 subjects), Parkinson’s Disease Phase 2A (6 subjects), Stroke (28 of 36 subjects, not yet un-blinded). Adverse events and serious adverse events were comparable to placebo, with no reported drug related clinically serious adverse events.

2) In the 1990s, Genervon realized that the many failures of clinical trials for central nervous system diseases can be attributed to the fact that the classic drug development paradigm - designing single-target drugs - simply cannot account for the multifactorial nature of these complex diseases. Instead, Genervon's drug development strategy was to find the endogenous regulator(s) that control the development and function of the nervous system by modulating the expression of multiple genes through different pathways, thus bringing homeostasis and health to the biological systems. Genervon has received both fast track and orphan drug designations for GM604 in the treatment of ALS.

3) Bottom line: a Phase 3 trial for GM604 would result in the same data, more slowly, at higher cost, while AAP with required patient data surveillance could be used to not only broaden experimental drug access to patients in need of medical options, but also to gather data on a spectrum of patients that is more representational of the whole population.

Whatever you do, don’t rely on anyone else’s opinion. Educate yourself about GM604 by reading the information in the links below and consider our position. Then, decide for yourself if ALS patients and researchers will both benefit from an FDA program that is already in place to meet urgent and unmet needs of the terminally ill.

http://www.genervon.com/genervon/medicines_trialdata.php
https://docs.google.com/viewer?a=v&pid=sites&srcid=ZGVmYXVsdGRvbWFpbnxhYXA0Z202fGd4OmRjMThlZmVmN2Y1NGI0OA
https://docs.google.com/viewer?a=v&pid=sites&srcid=ZGVmYXVsdGRvbWFpbnxhYXA0Z202fGd4OjE2ZDZmOGQ2MjYwMmFiNQ
https://www.youtube.com/watch?v=45vVnjKlFxM

Our grassroots campaign has adopted the moniker, "Hope NOW for ALS" and our mission, in addition to gaining access to GM604, is to gain access to ALL promising treatments for ALS, and help to redesign the clinical trial process for ALS treatments.

Please join us at the next Washington Rally, May 11. We need ALL of you! Together we can change the status quo and start saving the current generation of PALS. http://www.meetup.com/ALS-Faster-Drug-Approval/events/221529700/

We’re in this together,
Anthony Carbajal, Bethany Wolfe, the Torrino Family, Nick Grillo (gm6nick@gmail.com), and Jehad Majed (jmajed@mac.com)


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