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December 10, 2015

Genervon Case Report Summary from International ALS Patients treated by GM604

Pasadena, CA - 12/10/15 5:00AM PST 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 complete 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 no reported adverse effect.

Before we present some of the clinical data we have collected from GM604 treatments from around the world, the following is a recap of GM604 Phase 2A clinical trial results submitted to the FDA. Treated patients received 320 mg GM604 by intravenous bolus injection three times a week for two weeks with evaluations performed at baseline and at 12 weeks.

  1. The GM604 treated group had a 30% decrease in their ALSFRS-R slope when compared to before treatment achieving a positive trend of slowing down disease progression.
  2. The definite ALS patients treated with GM604 showed less functional loss with statistical significance, p=0.0047, when compare with historical Ceftriaxone trial placebo data in ALSFRS-R.
  3. 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.

The following patients from around the world were selected to receive GM604 treatment first because they are all influential, vocal, and strong advocates for curing ALS. Some of these patients are celebrities in their field. We want all of them to experience first-hand the curing power of GM604 to strengthen their conviction.

Patient 202 is a 30-year-old female from the 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 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 104 is a 44-year-old male from South Africa. His ALS onset was more than 5 years ago, and he was diagnosed with MND in May 2011. He 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 he could not stand up or walk. He was able to move his fingers slightly. He had very little strength in his arms and hands, and he could not hold or pick up objects. His speech was barely audible, and he had difficulty chewing and swallowing. His neck was extremely weak, and he was unable to hold his head up for any length of time.

After his 5th dose the patient's Progress Report notes:

"Patient says [he is] able to swallow a little bit better. Doctor observes what seems to be a marginal improvement in [arm] strength. Friends think his speech is slightly better. Patient had not experience any adverse side effects."

After his 7th dose, the patient's Progress Report notes:

"Patient reports that: his speech is slightly easier... his ability to swallow is better and sucking through a straw is, he says, 'a lot' better. He says his legs are slightly stronger and that he is drooling less. He has not experienced any adverse side effects. He has gained 2 kgs and believes something is working. The changes have been slight and gradual. He says he definitely hasn't deteriorated since the start of the 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, her therapist 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:

"there has been a visible improvement in [Patient 101's] symptoms... [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 male from Argentina and a Professor Emeritus of an American university. He has been on a ventilator, quadriplegic, and communicates through TOBII, an eye-tracking computer. In addition, his respiratory symptoms and bulbar symptoms had worsened. Patient 106 received his first dose of GM604 in September 2015 right after several weeks in an intensive care unit for aspiration pneumonia - a perilous situation for someone with a compromised respiratory system. He is being treated with GM604 under compassionate use with clinical trial protocols approved by the Ethics Committee of FLENI. Argentina's National Administration of Drugs, Food and Medical Technology (ANMAT) approved the importation of GM604. 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 (with 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. Using his TOBII (transcribed by his assistant), he writes to his wife this week:

"I am feeling better, with much energy and less phlegm. Movement of my legs are better and get better all the time. I hope I will be better with my lung capacity and be able to breath without the tube and then be able to talk. I have the illusion of recovering the use of the right hand to be able to read and write by myself, but the process is slow. Of course I would very much like to walk and go back to the world and I do everything possible to get better. At times my communication is not very clear because sometimes it is a little difficult for me to [wink]. I am in a very good mood and I am optimist."

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. Patient 110 posted the news in the newspaper and in social media, which is how Genervon first learned of the court victory. The Italian Health Authority has paid for and imported GM604 per court orders.

Since he began treatment with GM604, Patient 110 could sense a trend reversal and reports (direct translation from Italian, unedited):

"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."

Some common improvements reported by patients treated with GM604 are an increase in energy and strength, improved swallowing and body weight, improved range of motion and breathing, and improvement in mood with GM604 treatments.

Media Contact:
Company Name: Genervon Biopharmaceuticals
Name: Emily Wang
Email Address: ewang@genervon.com
Website: www.genervon.com



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