Date: 2015-10-28
Type of information: discontinuation of development
phase: 2
Announcement: discontinuation of development
Company: Newron Pharmaceuticals (Italy)
Product: sNN0029
Action
mechanism: protein. sNN0029 is a recombinant human vascular endothelial growth factor (rhVEGF-165) that is administered intracerebroventricularly. This is the first treatment to target motor neurons by blocking the activity of genes causing cell death. In preclinical in vivo studies, animals with a defective VEGF-gene displayed motor neuron death in parallel with muscle weakness and atrophy, pointing to the role of VEGF for motor neuron survival. In ALS model animals, VEGF treatment slowed disease progression and increased life span. sNN0029 has also been successfully tested in a three month Phase I/II safety and tolerability study in ALS patients. In February 2013, the Wellcome Trust granted an award of up to € 2.5 million to support a Phase I/II clinical trial to evaluate the safety and efficacy of higher doses of sNN0029 in patients with ALS. sNN0029 has been developed by Neuronova that Newron has acquired in 2012.
Disease: amyotrophic lateral sclerosis
Therapeutic area: Neurodegenerative diseases - Rare diseases
Country: Belgium, The Netherlands
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 15, 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 sNN0029 in patients with Amyotrophic Lateral Sclerosis (ALS). This safety and preliminary evidence of efficacy Phase II trial of sNN0029 is supported by funding from the Wellcome Trust.
An earlier study of sNN0029 in ALS patients at doses up to and including 2 µg/day indicated in preliminary analyses evidence of significant benefit especially in patients at the highest dose on multiple efficacy measures, compared to placebo. Treatment with sNN0029 was well tolerated and many patients have received treatment for up to two years and more.
The new study will include patients with ALS who will receive 4 µg/day as a continuous treatment in a randomized, placebo-controlled double blind fashion using an implantable pump and brain catheter. The trial has been approved by Health authorities and is currently recruiting patients to clinical sites in Belgium and the Netherlands. It will enroll 18 patients who will be assessed for safety and efficacy for three months as part of this study. Multiple clinical safety and efficacy rating scales will be used in combination with biochemical analyses and neurophysiological examination. Patients will be able to receive long term treatment after the study is completed, provided they meet protocol criteria.
