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March 21, 2015

Update for ALS Patients and Caregivers who have contacted Genervon

Dear ALS patients and caregivers,

After our February meeting with the FDA, we have filed a formal request for the Accelerated Approval (AA) Program and are now waiting for a final decision. Genervon would like to take time to address some frequently asked questions:

a.In the U.S., it is illegal to access GM604 without FDA approval or outside a clinical trial.

b.If the FDA decides we must continue with a Phase 3 trial, Genervon will not be able to offer compassionate use or expanded access. These programs are expensive and time consuming, which would only delay a future FDA decision and force us to choose between patients.

c.Even though the FDA has promised to help Genervon expedite the approval process for a Phase 3 trial, it would still take at least 3 years for GM604 to reach NDA. This means the majority of this generation of ALS patients would not survive to try GM604.

d.Phase 3 trials are very restrictive in enrollment, with a limited number of patients and 1/3 of those patients are randomized to placebo treatment.

e.Alternately, if AA with post-marketing Phase 4 requirements is granted, all ALS patients can have legal access to GM604 now and the health insurance will pay for the treatment minus co-pays. In our opinion, this is the best option for all ALS patients.

f.Although the Food and Drug Administration Safety and Innovation Act (FDASIA) of 2012 is quite clear in its intent and the FDA is strongly encouraged by Congress to grant AA to treatments that benefit all serious and life threatening diseases, the chance of approval is still quite small.

g.GM604 met the AA criteria of FDA (http://youtu.be/fzlePvW-Dg4) with FVC as the surrogate end point. Clinical end point in forced vital capacity (FVC) is an indicator of clinical survival and disease progression. Most ALS deaths are due to a decline in pulmonary involvement function.

h.There are statistically significant differences in the change and percentage change of FVC between the treated and placebo group from screening to week 12 for all patients in our small Phase 2a trial. The fact that these p-values are significant with so few patients is remarkable and speak volumes to the effect size of GM604. (see Statistical Report Addendum table 3, page 9 below)

i.This clinical data correlated very well in terms of achieving statistical significance between the treated and placebo groups with the three important ALS biomarker data, TDP43, SOD1 and Tau. We are able to provide more information upon request via an Asahi TV ALS Special, which includes a GM604 treated patient. Asahi TV owns the copyright and gave Genervon the right to private sharing (i.e. this video is not allowed to be posted on the internet). Please honor our promise. If you cannot ensure confidentiality (that the video will not be posted on the internet), please do not ask for the private link. (Please email info@genervon.com). You will be held to the legal implications of breach of contract.

Statistical Report shows significance of ALS/Alzheimer biomarker TDP43 for disease modulation by GM604:http://www.genervon.com/genervon/medicines_20150202.php

Statistical expert, Dr. Paul J. Lupinacci, addresses the significance of small clinical trials:http://www.genervon.com/genervon/images/data20150203.pdf

Links below may answer some of your questions. Please read all links carefully to minimize repeat of asked and answered questions. http://www.genervon.com/genervon/medicines_trialdata.php http://www.genervon.com/genervon/about_pressreleases.php

Contacts:
Genervon Biopharmaceuticals LLC
Dorothy Ko, 323-721-5500
Chief Operating Officer
info@genervon.com
www.genervon.com

 

 


 
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