Date: 2013-06-14
Type of information:
phase:
Announcement: presentation of results at the 17th International Congress of Parkinson’s disease and Movement Disorders, at the Sydney Convention and Exhibition Centre, Sydney, Australia
Abstract no. 487, “Safety and efficacy of recombinant platelet-derived growth factor BB (rhPDGF-BB) in Parkinson’s disease”, has been accepted for presentation as part of Poster Session “Parkinson’s disease: Clinical Trials” in Exhibition Hall 5, from 12:30-14:00 pm local time on June 18, 2013
Company: Newron Pharmaceuticals (Italy)
Product: sNN0031
Action
mechanism: sNN0031 is a novel drug candidate for the treatment of Parkinson’s disease that is designed to act on neural stem and progenitor cells in the brain. In animal models of Parkinson’s disease, treatment with sNN0031 restores motor function and improves neurochemical deficits. In a Phase I/II trial in patients with PD, it was well tolerated and demonstrated preliminary beneficial effects on biochemical markers of the degenerating dopamine system in PD patients. The product is comprised of the naturally occurring protein PDGF-BB (platelet-derived growth factor BB) formulated for intracerebroventricular (ICV) delivery. The intended therapy involves short-term continuous infusion of sNN0031 into the ventricular lumen, in order to optimize PDGF-BB access to the lateral ventricular walls of the brain where the targeted stem and progenitor cells reside. sNN0031 holds the potential to halt and even reverse disease progression, a much-needed improvement over currently available treatments, which only address the symptoms of Parkinson’s disease.
Disease: Parkinson’s disease
Therapeutic area: Neurodegenerative diseases - CNS diseases
Country:
Trial details: In the study, 12 patients with Idiopathic Parkinson's disease (PD) of moderate severity were randomized in a double-blind, placebo-controlled safety, tolerability and preliminary efficacy study. Three increasing doses of sNN0031 (containing human platelet derived growth factor rhPDGF-BB) were delivered by intracerebroventricular administration, using an implantable infusion system. Patients received the drug or placebo for a 2 week period and were followed up to 12 weeks for initial analyses. Safety and tolerability was continuously monitored and efficacy was assessed before and after the 12 weeks using various clinical rating scales and brain imaging (PET-technology). Patients have now been followed since 2009.
Latest
news: * On June 14, 2013, Newron Pharmaceuticals, a research and development company focused on novel CNS and pain therapies, has announced that results from a first Phase I/II safety and efficacy study with sNN0031, a recombinant human platelet-derived growth factor BB, in Parkinson’s disease, will be presented at the 17th International Congress of Parkinson’s disease and Movement Disorders, taking place from June 16 to 20, at the Sydney Convention and Exhibition Centre, Sydney, Australia. The compound was part of the acquisition of NeuroNova AB by Newron in December 2012. Data from animal models indicate that new and maturing brain cells can restore the dopamine system in a Parkinsonian brain. In this study in PD patients, i.c.v. administration of sNN0031 was well-tolerated with no drop-outs, treatment related serious adverse events or severe side-effects. Dopamine transporter activity, assessed using PET, indicated a dose-dependent increase in the substantia nigra and putamen, areas of the brain damaged by PD. Regulatory meetings in Europe and the US have been initiated to seek input and agreement on a proposed Phase II/III pivotal placebo-controlled study in severe PD patients who do not benefit from treatment with optimized standard of care using oral therapies.
