NeuroVive pipeline rebuild continues with Karolinska pact

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The NeuroVive-Karolinska collaboration builds on research into cyclosporine A

NeuroVive Pharmaceutical has entered into a research collaboration with Karolinska Institutet. The deal will see Karolinska test mitochondrial myopathy candidate NV556, a cyclophilin inhibitor that has grown in importance to NeuroVive as other programs have flopped in the clinic.

Lund, Sweden-based NeuroVive did see NV556 primarily as a NASH candidate that it wanted to outlicense. But the drug developer has reassessed its pipeline in light of recent events, including the publication of a paper showing cyclosporine A slows progression of mitochondrial myopathy in mice.

Professor Håkan Westerblad, one of the authors of that paper, will lead research into the effects of NV556 in experimental models of the group of mitochondria-driven muscles diseases.


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NV556, like cyclosporine A, targets cyclophilin D, a mitochondrial matrix protein that is found in high levels in patients with mitochondrial myopathy. Westerblad and his team found targeting the protein with cyclosporine A improved muscle fiber Ca(2+) handling and acted against development of muscle weakness. Mice that received the treatment lived longer.

NeuroVive is hoping NV556 will yield similar outcomes while also being more tolerable than cyclosporine A. NV556 is seen as having higher specificity than cyclosporine A.

Generating data to back up these hopes would be a rare, if minor, filip for NeuroVive, a company that has suffered two big setbacks over the past 18 months. The run began in June 2015 when a phase 3 acute myocardial infarction trial of NeuroVive’s cyclosporine formulation missed its primary endpoint. Then, in October 2016, a phase 2 trial of the same formulation in acute kidney injury also missed its primary endpoint.

NeuroVive is waiting on data from a trial of the formulation in severe traumatic brain injury. If the 20-person phase 2 delivers positive data, the formulation could still have a future. But if the brain injury trial continues NeuroVive’s losing streak, the likelihood of the asset delivering a return for the company will shrink further still.

Either way, the future of NeuroVive is increasingly tied to NV556 and other preclinical programs the company is developing. Mitochondrial disorders have emerged as a cornerstone of the rebooted pipeline, with NV556 sitting alongside NVP015 on the list of NeuroVive programs targeting the field.

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