PASADENA, CA – 12/11/2015 (PRESS RELEASE JET) — While our colleagues are gathering at the ALS International Symposium in Orlando Florida, Genervon is overwhelmed by many deserving ALS patients’ requests to access GM604 now to save their lives. However, Genervon cannot grant access until we receive an accelerated/conditional approval with Phase 4 surveillance from the FDA or conduct a Phase 3 clinical trial. Most of the 30,000 ALS patients in the US and the 350,000 around the world will not survive the time it will take to finish the trial.
Genervon cannot offer expanded access (compassionate) or right-to-try use in the US due to cost and liability. Under various special access programs for ALS Genervon has been legally exporting GM604 internationally to physicians in other countries to treat their local ALS patients. Patients and physicians continue to collect data, evaluate and report positive results with not one reported adverse effect.
Before we present some of the clinical data we collected from GM604 compassionate treatments from around the world, the following is a recap of GM604 Phase 2A clinical trial results submitted to FDA, excluding the results of significant changes in biomarkers. Treated patients receive 320 mg GM604 by intravenous bolus injection three time a week for two weeks only. Then compare clinical endpoint at baseline with week 12 which has a 10-week period of no treatment.
1. The Japanese Health Department approved Edaravone as an ALS drug by a Japanese Company in June 2015 based on its less functional loss compare to placebo in its confirmatory trial. GM604 in Phase 2A trial has less functional loss than Edaravone: a difference of 0.8 (dosed for 2 weeks) by GM604 as compare to a difference of 0.65 (dosed for 24 weeks) by Edaravone in ALSFRS-R change.
2. GM604 treated group had a 30% decrease in ALSFRS-R slope when compared to before treatment achieving a positive trend of slowing down disease progression.
3. GM604 treated definite ALS patients showed less functional loss with statistical significance, p=0.0047, when compare with historical Ceftriaxone trial placebo data in ALSFRS-R.
4. GM604 treatment reduced the decline in FVC in ALL treated patients from screening to week 12 with statistical significance favoring the treated group, p=0.0476.
Patient 202 is a 30-year-old famous female from US 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. Since then, her FVC improved from baseline to the 8th dose, and as reported by her physician, “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 104 is a 44-year-old famous male from South Africa. His ALS onset was more than 5 years ago, and he was diagnosed with MND in May 2011. Patient 104 exhibited speech difficulty as early as May 2011, which is a sign of bulbar, cervical, and lumbosacral motor neuron deterioration. Patient 104 had some mobility in his legs, but could not stand up or walk. He was able to move his fingers slightly. He had very little strength in his arms and hands. He could not hold or pick up objects. His speech was barely audible. He had difficulty chewing and swallowing. His neck was extremely weak and he was unable to hold it up for any length of time.
After his 5th dose, Patient 104 said he is able to swallow a little bit better than he could before. His doctor observed a marginal improvement in arm strength. Friends thought his speech is slightly better. Patient 104 did not experience any adverse side effect. After his 7th dose, Patient 104 reports that his speech is slightly easier, his ability to swallow is better, and sucking through a straw is “a lot” better. He says his legs are slightly stronger and that he is drooling less. He has gained 2 kilograms weight and believes something is working. His changes have been slight and gradual. He says he definitely hasn’t deteriorated since the start of his treatment program.
Patient 101 is a 32-year-old female from Australia who had ALS onset in November 2014. Her progressive bulbar weakness and left arm weakness began in late 2014, and her diagnosis was confirmed on April 1st 2015. Her ALSFRS-R score dropped from 43 on January 15, 2015 to 37 on May 15, 2015. Her disease experienced rapid deterioration before she received GM604. Under the Special Access Scheme, Patient 101 received GM604 from June 14 to September 8, 2015.
After treatment, it was observed that Patient 101 experienced not only an improvement in her outlook and mood, but she has shown an improvement in her range of movement (ROM) and an increase in energy as well. Her therapist reports that Patient 101 “is now unassisted in turning and has greater ease of movement, her upper strength has improved and is now not using a cane to assist with walking as previously noted…she is able to slowly shower, to dress herself, and hang laundry on clothes lines. She has gained greater independence over her life and disease.” Patient 101’s physician notes, “I monitored Patient 101 for thirty minutes after each dose for any adverse reactions or side effects, fortunately the treatment was safe and well tolerated. I personally observed progression of the disease slowing and even a mild improvement after treatment, Patient 101 seemed to walk and speak with more ease and displayed more energy.”
Patient 106 is a 74-year-old famous male from Argentina and a Professor Emeritus. He has been on a ventilator, quadriplegic, and communicates through an eye-tracking computer. In addition, his respiratory symptoms and bulbar symptoms had worsened. Patient 106 received his first dose of GM604 right after several weeks in the intensive care for aspiration pneumonia – a perilous situation for someone with a compromised respiratory system. He is being treated with GM604 under a clinical trial approved by the Ethics Committee of FLENI and approved by ANMAT, the FDA of Argentina. After his 3rd dose, Patient 106 was able to move his legs. His kinesiologist reported that Patient 106 has much more strength in his right leg and can stand with assistance now.
After two months of treatment with GM604, Patient 106 shows significant improvement in every region – legs, torso, neck control and face (no more facial distortion). He exhibits greater overall improvement in energy working/writing for 8-9 hours each day and has gained weight. His drooling was drastically reduced. He can now sit on a chair unassisted and holds his head quite upright, improving his writing speed and precision. His ALSFRS-R improved one point from baseline and his vital capacity was raised from 300 to 320 and 360 in the last measurement.
Patient 110 is a 47-year-old Italian man who has suffered ALS since 2009. He and several ALS patients in Italy hired an experienced lawyer with the help of a noted neurologist in Rome to file lawsuits against the government public health system, winning ex parte decisions quickly. The Italian Health Authority has paid for and imported GM604 per court orders. Patient 110 posted the news in the newspaper and in social media, which is how Genervon learned of the court victory.
Since he began treatment with GM604 he could sense a trend reversal and reports (direct translation from Italian), “The symptoms, such as stiffness akinetic, dysarthria, the lack of muscle strength, the involuntary twitching etc. etc. decreased. After 5 doses in 10 days, he and the physical therapist and the social and health workers have observed small improvements. In particular, he noticed: A decrease in both the stiffness in the legs (feet, ankles and say included), the annoyance he felt during manipulation or massage in stretching. Spasms do not go wild as before and do not last long, they are very manageable and allow him to finally sit with his feet near the body. It is no longer impossible to bend them. Almost no involuntary contractions during the day, significantly improved at night. A little better range of motion on all four limbs. All voluntary movements are easier, more fluid, less spastic, faster. Muscle strength improved slightly in all muscles. Unchanged for those atrophic total as the deltoids and the flexors of the feet. Fasciculation involuntary regressed as the sensation of breathlessness or ‘shortness of breath’. Bowel regularity as more have become easier the steps of position from lying/sitting and bed/chair/WC and vice versa.”
It seems that some common improvements in compassionate patients treated with GM604 are increase in energy and strength, improved swallowing and weight, improved range of motion and breathing and improvement in mood with GM604 treatments.
Distributed by Press Release Jet
Company Name: Genervon Biopharmaceuticals
Contact Person: Emily Wang
Country: United States