Newron Pharmaceuticals is about to initiate its Phase II clinical trial, designed to evaluate novel molecule sNN0029 in patients who suffer from Amyotrophic Lateral Sclerosis (ALS), according to information provided by the company in a press release. The company, which has been dedicated to providing CNS and pain therapies, will conduct the Phase II study to assess sNN0029’s safety and preliminary evidence of efficacy, with support from the Wellcome Trust – a biomedical research charity based in London, United Kingdom.
The therapy developed by Newron is a recombinant human vascular endothelial growth factor (rhVEGF-165) to be administered intracerebroventricularly (ICV). Preclinical examination in animal models of ALS, revealed the sNN0029’s capacity of improving motor neurons’ survival, which the disease causes to degenerate, eventually leading to disability and death due to heart or respiratory failure.
The phase 2 study to be initiated by Newron is a randomized, placebo-controlled double blind study, in which 18 ALS patients will be administrated continuously with 4 µg per day of sNN0029. After previously receiving approval from the Health authorities in the country, Newron is now recruiting patients to enroll in the study, which is set to take place in clinical sites across Belgium and the Netherlands.
Over a span of three months, the scientists will analyze the patients’ disease progress in order to evaluate the safety and efficacy of sNN0029, according to a series of clinical safety and efficacy rating scales, as well as biochemical analyses and neurophysiological exams. If the patients meet the protocol criteria, they will be able to continue on a long-term treatment, following the end of the study.
The early animal studies conducted were focused on the most common ALS mutation in humans, the G93A-SOD1 mutation, and revealed that the participants who were treated with the drug not only improved their survival time, but also their muscular strength, when compared to the control group. Therefore, the Newron scientists believe that, given its mechanism of action, sNN0029 may be a unique option of treatment for patients who suffer from ALS.
In addition, the Phase 1/2 safety and tolerability studies conducted in ALS patients also revealed encouraging results, with better results associated to higher doses on multiple efficacy measures. The Wellcome Trust granted the company a EUR 2.5 million research grant, to be invested in the Phase 1/2 trials, last February 2013 and has continued to support the development of the compound.