Pasadena, CA - 5/19/16 - Genervon Biopharmaceuticals has continued to provide GM604 to Doctors of named ALS patients around the world since our December 10, 2015 Press Release.
Genervon has the freedom to enroll a wide spectrum of heterogeneous ALS patients ranging from the severely advanced to the newly diagnosed, contrary to the very narrow and restrictive inclusion and exclusion criteria of a standard clinical trial. Observations are being recorded by physicians before, during and after GM604 treatment, and personal assessments are being requested of the treated patients. While it needs to be stressed that this protocol does not have the rigor of a clinical trial these evaluations are useful in building up a better understanding of GM604's tolerability and efficacy across diverse ALS patients. Below are updated reports from patients and/or their doctors;
Patient Number 111 (Italy):
Note: ALS patients in Italy sought the help of an experienced lawyer and a noted neurologist. The Italian Ministry of Health has since paid for and imported GM604 per court orders for named ALS patients. Patient #111 from Italy was the first to be treated with Genervon's patent pending protocol, Plan E. The Italian Ministry of Health has placed an order to purchase a third treatment for Patient #111.
Housewife, born in 1945. Since 2013 she had some accidental falls and progressive lower extremity weakness. As a result of a traumatic fracture to her L1, she underwent vertebroplasty surgery in 2014. In May 2015 she was hospitalized for respiratory problems and received a diagnosis of OSAS and a prescription for nocturnal NIV. She experienced progressive, generalized weakness and received the diagnosis of ALS in August 2015: signs of moderate to severe neurogenic pain were active and widespread. Received riluzole 1/2 cpr die, Dobetin, Carnitine.
Neurological examination: Dyspnoeic (shortness of breath) at rest in a chair. Moderate dysphonia and dysphagia. Reduction in the tropism of the tongue with fibrillation. Tetraipostenia more pronounced lower limb dive will not hold a position against gravity. Ipereflessia osteotendinous. No Babinski. widespread muscle atrophy with fasciculations to quadrcipite femoral.
GM604 treatment and results
First dosing January 20, 2016 to 12th dosing February 15, 2016.
"Mrs. [Xxxxxxxx], 70 years old. Today, after a careful examination, I, [Xxxxxxx Xxxxxx] M.D., can say that the patient condition is improved. The breathing and the swallowing are better. The patient can move alone in her house and can take care of herself."
Patient #111 improved by 3 points in ALSFRS-R from a score of 30 at baseline/visit 1 to a score of 33 at visit 12 and maintained a score of 33 after 4 weeks without treatment as measured during her follow up visit. Her FVC also improved from 23% at baseline to 26% at her follow up visit 4 weeks after her last dosing.
Comparing ALSFRS-R from baseline to 2 completed treatments (12 doses, 4 weeks) of GM604
- Stopped ALS progression in 8 categories.
- Reversed ALS progression in 3 categories by a total of 3 points: improved swallowing; "cannot cut food" improved to "cutting most food"; and "slow/clumsy" improved to "normal turning in bed".
Updated Summary Report of Patient #202 (USA, FDA approved conditional use), March 31, 2016
Genervon received an update from Patient #202 who had positive results from two GM604 treatments that began September 8, 2015 and one treatment that began September 23, 2015.
She participated in a separate research project at Cedars-Sinai Medical Center, Los Angeles, which is tracking the disease progression of PALS by measuring muscle strength. Data was collected 6/24/15, 8/19/15, 11/18/15, and the latest report is from 3/16/16.
Patient #202 wrote on 3/16/16 "I actually have incredible news, according to my strength tests, I haven't lost any strength since SEPTEMBER (six months ago), which is when I got my first dose of GM6. I also gained a few more points of FVC. I just visited the ALS clinic at Cedar Sinai, those updates are all based on that. I'm super excited, honestly!" Left and right grip strength slope reversed direction after GM604 treatment: LEFT -0.11 reversed to +0.20; RIGHT -0.15 reversed to +0.06.
Patient 202 is a 30-year-old female from the U.S. who had ALS onset in July 2013 and was diagnosed in 2014. The patient experienced rapid disease progression in the summer of 2015 while waiting for FDA compassionate use approval and suffered serious falls requiring trips to the emergency room and stitches on her face.
She started receiving GM604 compassionate treatment on September 8, 2015. Her FVC improved from baseline to the 8th dose, and her physician reports: "After reviewing [Patient 202]'s past medical history, it is in [both treating doctors'] opinion that the patient has greatly benefited from GM604 over the past six weeks when the drug was administered three times a week. During our initial assessment, the patient had rapid tongue fasciculation which developed over July and August but decreased dramatically with the medication. The patient has stopped biting her tongue, which were also reflected in her previous medical records. This is a reversal and an unexpected benefit from the medication. Moreover, the patient's limb progression has completely plateaued."
Patient #202 participated in a research project tracking muscle groups strength deterioration of ALS patients at Cedars-Sinai LA before GM604 treatment.
GM604 is an endogenous embryonic stage signaling master regulator of the human nervous system. It is novel and may be curative for ALS. The potential benefits of GM604 include reduced inflammation and apoptosis, increased expression of kinesins and dynactins leading to improved axonal transport efficiency, modulation of undesirable gene expressions, and reduction of toxic protein aggregates. These beneficial impacts lead ultimately to attenuation of ALS disease progression, improvement in clinical outcomes, and reduction in disability caused by neurological deficit.
Safety & Tolerability and Efficacy:
GM6 is a small, endogenous regulatory signaling peptide that has been shown to be safe and tolerable in intravenous injections of six doses of 320 mg each given over a two-week period. Phase 1 and Phase 2A trials showed no clinically significant shift in ECG readings, neurological indicators, hematology, or clinical chemistry. There were no reported deaths or withdrawals due to Adverse Events (AE) or any reported clinically Serious Adverse Events (SAE).
Since June 2015, Genervon Biopharmaceuticals has been providing GM604 upon the request of individual ALS patients in 5 countries across 5 continents under their government health agencies' special access schemes for individual, named patients. Genervon has completed Phase 2A randomized double-blinded GALS-001 trial with GM604 in ALS patients and submitted all clinical trial reports to the FDA. While a phase 3 trial is being planned, Genervon remains absolutely committed to finding a faster way of demonstrating the safety and effectiveness of GM604 and getting it to ALS patients who simply can't wait-out the 3 to 5 years needed for Phase 3 trials.
No one can access GM604 in the U.S. until FDA approval is received. Genervon can legally export the investigational drug GM604 to physicians treating ALS patients in 35 different countries under various special access programs in those countries, treating named patients or under compassionate use for patients suffering fatal diseases without other treatment options. Genervon receives many deserving requests for compassionate or RTT (right to try) use from ALS patients in the U.S. every day, but we cannot afford the risk and cost of agreeing to the requests.
Genervon Biopharmaceuticals LLC
Dorothy Ko, 323-721-5500