PASADENA, Calif.--(BUSINESS WIRE)--Genervon Biopharmaceuticals (“Genervon”) today announced that its lawyers have filed a patent for legal protection of its intellectual properties based on the proprietary data from its Phase 2A clinical trial with GM604 for ALS (GALS001 trial) and in single ALS compassionate patient study (GALS-C). GM604 modulated ALS disease biomarkers showing homeostasis, leading to prognosis and therapeutic treatment for ALS disease. To date, Genervon’s innovative discoveries and development of a multiple target master regulator drug of the human nervous system have resulted in the ownership of 66 patents.
Classic single target drugs are mono-directional: agonist or antagonist. Genervon's hypothesis is that the endogenous signaling master regulator GM604 can restore health by regulating/modifying multiple genes and their protein expressions in the correct direction when they are above or below the normal range. This hypothesis has been difficult to prove, as most ALS patients included in a trial are uniformly above or below the normal ranges at baseline for most CSF and plasma biomarkers. Comparing modulating effect by GM604 in patients of very different baseline may provide the necessary data to show homeostasis.
The GALS-C study was approved by FDA, IND#120052 (http://www.ericvalor.org/condition-green/). Genervon’s GALS-C patient was diagnosed with ALS 10 years ago, and has been quadriplegic and on ventilator for 8 years. His CSF biomarkers SOD1, Cystatin C and total TAU results all were below the normal range at baseline. At week 2, after a six-dose treatment of GM604, all three biomarkers were upregulated substantially towards the normal range showing disease modification.
In the GALS001 trial, these same biomarkers of the definite ALS patients with onset within 2 years were all above the normal ranges and six-dose treatment with GM604 down regulated them towards the normal ranges. GM604 regulated the same disease related biomarkers of both GALS-C patient and the GALS001 patients in the correct but opposite direction towards the normal ranges/levels, the hallmark of homeostatic processes in healthy living organisms. "This is positive indication for GM604 as a potent master regulator of neuronal homeostasis," said Dorothy Ko, COO of Genervon.
Last month Genervon received the latest TDP43 biomarker data analysis for its GALS-C patient from the Biomarker CRO. Genervon was surprised that the GALS-C patient’s plasma TDP43 level of 144.54 pg/ml at baseline over 10 years after ALS onset - was as high as all the definite ALS patients in the phase 2A trial, a mean of 138.88 pg/ml at baseline within 2 years of ALS onset. The normal range of TDP43 in plasma is 0-50 pg/ml. At the end of two weeks (6 doses) GALS-C patient’s data was reduced to 92.59 pg/ml and at the end of 12 weeks it was reduced to 52.53 pg/ml, very close to normal range. The percentage change of GALS-C patient’s TDP43 by GM604 from baseline was -63% in 12 weeks. The mean percentage change in TDP43 of the treated GALS001 patient group was -34% and the mean of percentage change in the placebo patient group was +6% in 12 weeks. The slope in plasma TDP43 through week 12 in treated (-3.513 pg/mL/wk) is lower than in placebo (0.493 pg/mL/wk), p=0.0078.
According to the Kyoto University research results, TDP43 spreads out from the nucleus to the outside and becomes toxic, causing the death of other motor neurons. In ALS patients TDP43 has shortened axons in motor neurons than in healthy subjects. As TDP43 decreased the death of neurons were prevented and the previously shortened projection of nerves became longer. Therefore a slowing of ALS disease progression is expected. TDP43 and TAU are major disease proteins found in the brains of ALS and Alzheimer patients.
Top line clinical data are available on Genervon’s website, June 29, 2015, http://www.genervon.com/genervon/medicines_trialdata.php. A confidential video featuring a GM604 treated ALS patient in the Phase 2A trial and an explanation of the scientific rationale behind treating ALS by lowering TDP43 over-expression is available upon request by email to email@example.com.