Date: 2015-10-28
Type of information: discontinuation of development
phase: 2
Announcement: discontinuation of development
Company: Newron Pharmaceuticals (Italy)
Product: sNN0031
Action
mechanism: protein. sNN0031 is a recombinant human platelet-derived growth factor-BB (rhPDGF-BB) that is designed to act on neural stem and progenitor cells in the brain. It is administered intracerebroventricularly (ICV). Administration of sNN0031 in animal models of PD has resulted in a dose-dependent proliferation of subventricular and striatal cells and also counteracted the behavioral and/or tissue effects of prior exposure to neurotoxins for dopaminergic neurons. In animal models a 14 day treatment resulted in a long-lasting improvement in Parkinsonian symptoms and dopamine transporter (DAT) -binding, a marker of dopamine system integrity and function. Based on its mechanism of stimulating existing progenitor cells to enhance dopaminergic activity, rhPDGF-BB may offer a new therapeutic option for patients with PD who do not benefit from treatment with optimized standard of care using oral therapies.
Disease: Parkinson’s disease
Therapeutic area: Neurodegenerative diseases
Country:
Trial details:
Latest
news: * On October 28, 2015, Newron Pharmaceuticals announced that the company has completed its review of the early-stage development programs for sNN0031 and sNN0029 and decided to terminate both development programs. The compounds are delivered into the brain with an investigational drug delivery catheter from a third-party supplier, who entered into a consent decree with. FDA in April/May 2015, preventing it from commercializing the catheter or engaging in new manufacturing of the catheter until previously identified quality system issues are resolved. This information was highlighted by Newron on 15 September 2015. The issues raised by the FDA relating to the supplier’s quality system led Newron to temporarily interrupt any further patient screening activities, surgical implantation of medical device, or randomization of patients to enable Newron to perform a benefit-risk assessment for sNN0029. Newron's assessment, together with the continuing delays and information relating to the inability of the supplier to manufacture new catheters to replace the current ones that expire on 27 February 2016, has led the company to the decision to discontinue the development of both programs. The Company will now inform investigators and help find appropriate solutions for the patients still on treatment. The decision will lead to a restructuring of Newron’s operations in Sweden, affecting up to six employees. * On January 14, 2015, Newron Pharmaceuticals, a research and development company focused on novel CNS and pain therapies, announced the initiation of a Phase II study of its novel molecule sNN0031 in patients with Parkinson’s disease. The sNN0031 Phase II safety and preliminary evidence of efficacy trial is supported by funding from the European Commission (FP7 Framework Program).
sNN0031 is a recombinant human platelet-derived growth factor-BB (rhPDGF-BB) that is administered intracerebroventricularly (ICV).
Previous studies of sNN0031 in PD patients showed that the ICV administration of sNN0031 was well tolerated. DAT, assessed using brain imaging, indicated a dose-dependent positive effect in brain regions damaged in PD. The study will include patients with advanced PD on standard of care oral medication who will receive 6 µg/day in two cycles of 14 days each, separated by three months, in a randomized, placebo-controlled double blind design using an implantable pump and brain catheter. The trial has received approvals from Health authorities in the UK, Germany and Sweden, as well as from the ethic committees of the centres involved in the trial. The study will enroll 20 patients who will be assessed for safety and efficacy for nine months as part of this study. Multiple clinical safety and efficacy rating scales will be used in combination with biochemical analyses and brain imaging.
