European Medicines Agency scientists conclude that there is a wealth of data to demonstrate that GM604 "improves motor function, breathing and disability progression."
PASADENA, CA - 6 June, 2016 - On May 30, 2016 European Commission approved and implemented the decision relating to the designation of "GM604" as an orphan medicinal product under Regulation (EC) No 141/2000 of the European Parliament and of the Council.
The conclusion in COMP/EMA summary of positive opinion in April is that "...the sponsor provides a wealth of data, which the COMP considers to be sufficient to support medical plausibility for the purpose of orphan designation. Firstly motor-function outcome and survival data in relevant preclinical disease models, secondly preliminary clinical data that demonstrate positive trends and statistically relevant improvements in FVC and ALSFRS-R decrease. Supportive data include anecdotal clinical data from compassionate use and clinical biomarkers data." COMP Scientists from every member state are represented and voted for the positive opinion. The positive opinion was adopted at the first COMP discussion without having to supply any additional information.
GM604 is an endogenous embryonic stage signalling master regulator of the human nervous system and its mechanism of action (MOA) is by binding specifically to the beta subunit of the tyrosine kinase of the Insulin Receptor. IGF1R and IGF2R have the identical beta subunits. The binding activates the monitoring of the distress signals from the nervous system and response through multiply pathways by modulating the defective genes and misfolded proteins thus bringing homeostasis to the biologic systems.
COMP Summary Report on the conclusion of "how is the medicine is expected to work... [GM604] is made of 6 amino acids. The amino acids are arranged in the same order as those in a part of MNTF gene." The gene encoding MNTF is located in the reverse strand on a specific human chromosome location. "[GM604] regulates the development of the nervous system in a fetus. Like MTNF, the medicine is expected to act on genes that help to regenerate and repair nerve cells. In this way, the medicine is expected to help reduce damage to nerve cells caused by ALS."
Genervon had previously submitted the same set of its confidential data for review by FDA which has also granted the orphan drug status to GM604. The European Medicines Agency's committee of independent experts reviewed the same confidential data as submitted to FDA. Commenting on the report Dorothy Ko, COO at Genervon said: "We are very pleased by this positive opinion which we consider as a form of peer review from scientists representing all member states of the EU who carefully scrutinized a very large volume of data."
Genervon has also been encouraged that the EMA welcomed the use of anecdotal information from Compassionate Use patients treated by GM604 from around the world. "With ALS, the independent regulators like the EMA usually only get to see early clinical results from a narrow band of patients that fit the restrictive drug trial requirements of being recently diagnosed and in relatively good health. We are pleased that the EMA has recognized that there is some learning value in treating a broad range of heterogeneous ALS patients who are in different stages of disease progression." The opinion did not identify any safety concerns about GM604.
Editor's Notes:
1. Genervon Pharmaceuticals: is a clinical stage Biopharmaceutical company. Genervon has discovered and developed new classes of novel master regulators bio-drugs. For more information, visit: http://www.genervon.com
2. GM604 is an endogenous embryonic stage signaling master regulator of the human nervous system. It is novel and may be curative for ALS. Genervon's innovative hypothesis, drug discovery, drug development and clinical trials for GM604 have been in progress for the last 20 years.
3. ALS: Amyotrophic lateral sclerosis (ALS), sometimes called Lou Gehrig's disease or Motor Neuron Disease (MND), is a rapidly progressive, invariably fatal neurological disease that attacks the nerve cells (neurons) responsible for controlling voluntary muscles (muscle action we are able to control, such as those in the arms, legs, and face). The disease belongs to a group of disorders known as motor neuron diseases, which are characterized by the degeneration and death of motor neurons. The only licensed treatment for ALS is R3.
4. Phase 3 Trials: Phase 3 studies are randomized controlled multicenter trials on large patient groups depending upon the disease/medical condition studied, and are aimed at being the definitive assessment of how effective the drug is, in comparison with current 'gold standard' treatment. Because of their size and comparatively long duration, Phase 3 trials are the most expensive, time-consuming and difficult trials to design and run, especially in therapies for chronic medical conditions.
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