FDA Grants "Orphan Drug" Designation for Genervon's Breakthrough Multi-Target ALS Bio-Drug GM604

Genervon expects to present the analysis of the unblinded results of ALS, PD and Ischemic Stroke trials by the Fall of 2014.

PASADENA, Calif.--(BUSINESS WIRE)--Genervon Biopharmaceuticals announced today that the U.S. Food and Drug Administration (FDA) has granted an "orphan drug" designation to Genervon's GM604 (a.k.a. GM6) bio-drug for the treatment of Amyotrophic Lateral Sclerosis (ALS). The FDA also granted GM604 a "fast-track" designation for ALS in 2013.

In addition, the U.S. Patent and Trademark Office recently notified Genervon that it will be issued U.S. patent #8673852 on March 18, 2014 for its novel proprietary peptide GM6. GM6 is Genervon's first-in-class, multi-target, master regulator bio-drug that has shown itself to be highly efficacious in a number of therapeutic applications. This brings a total of 53 U.S. and international patents issued to Genervon.

The following lists the clinical trials involving GM6 currently sponsored by Genervon:

ALS Phase 2a Clinical Trial with GM604 (a.k.a. GM6, clinicaltrials.gov NCT01854294) Genervon finished enrolling ALS patients in January 2014 for its ALS Phase 2a trial under the direction of Dr. Hiroshi Mitsumoto (Columbia University Medical Center in New York) and Drs. Nazem Atassi and Merit Cudkowicz (Massachusetts General Hospital in Boston). Twelve ALS patients, including four in a placebo group, have completed two weeks of dosing.

Parkinson's disease Phase 2a Clinical Trial with GM608 (a.k.a. GM6, clinicaltrials.gov NCT01859381) Genervon will finish enrolling all six Parkinson's disease (PD) patients this month for its PD Phase 2a trial at Columbia University Medical Center in New York under the direction of Drs. Stanley Fahn and Cheryl Waters.

Ischemic Stroke Phase 2 Clinical Trial with GM602 (a.k.a. GM6, clinicaltrials.gov NCT01221246) Genervon will finish enrolling ischemic stroke (IS) patients next month for its IS Phase 2 trial at eight sites across the country.

Each of these three Phase 2 trials is a double-blinded, randomized, placebo-controlled trial. GM6 has been safe and well-tolerated in each trial. Genervon expects to present the analysis of the unblinded results of all three trials by the third quarter of 2014.

About GM6

In the 1990s, Genervon hypothesized that neurological and neurodegenerative diseases involve the interplay of multiple targets and processes in an interactive dynamic mechanism and network. Most researchers in the field have by now confirmed that ALS and other neurological disorders are remarkably complex, involving many different biological systems and potential therapeutic targets. Genervon believes that this is one reason why single-target drugs have uniformly failed in previous clinical trials of ALS.

Using breakthrough proprietary biotechnologies, Genervon scientists discovered GM6, an endogenous, multi-target, master regulator signaling peptide. Highly expressed in week 9 of human embryonic/fetal gestation, GM6 plays a significant role in nervous system development, protection, and correction.

About Genervon

Genervon is a privately funded clinical stage biopharmaceutical company in California developing breakthrough multi-target bio-drugs to address the world's critical unmet medical needs.

Genervon Biopharmaceuticals LLC
Dorothy Ko, 323-721-5500