With approval in Europe due shortly for Xadago, Newron Pharmaceuticals has begun mid-stage trials of another Parkinson’s therapy, which the Italian firm hopes could halt or even reverse disease progression.
The molecule in question, called sNN0031, is a recombinant human platelet-derived growth factor-BB that is administered intracerebroventricularly (ICV). In a Phase I/II trial, it was well tolerated and demonstrated preliminary beneficial effects on biochemical markers of the degenerating dopamine system in PD patients.
Treatment involves short-term continuous infusion of sNN0031 into the ventricular lumen, using an implantable pump and brain catheter. Newron says that based on its mechanism of stimulating existing progenitor cells to enhance dopaminergic activity, sNN0031 may offer a new option for PD patients who do not benefit from standard oral therapies, which only address the symptoms of the disease.
The trial, which is supported by funding from the European Commission, has received approvals from health authorities in the UK, Germany and Sweden. It will enroll 20 patients.
Things are going well for Newron at the moment. Shares soared to a six-year high at the end of December when advisors to the European Medicines Agency issued a positive opinion for Xadago (safinamide), the first new PD drug to get one in 10 years. It will be marketed by fellow Italian group Zambon.
Safinamide was resubmitted in the USA just after Christmas, in response to a ‘refusal to file’ letter from the Food and Drug Administration issued after a review of the first filing made in May 2014. The letter highlighted “organisational and navigational problems’, largely due to the hyperlinking of tables and folders in the submission.