Herantis Pharma is launching a development program for non-invasive cerebral dopamine neurotrophic factor (CDNF) as a treatment candidate for patients with Parkinson’s disease, the company recently announced.
Currently, the investigational treatment needs to be administered directly into the brain using an implanted delivery system suited for Parkinson’s patients. Under the new program, Herantis will seek new methods of delivering the therapy to patients.
A new licensing agreement with the University of Helsinki will grant the company worldwide, exclusive rights for a non-invasive CDNF-based treatment.
“Non-invasive CDNF is an important expansion to our existing patent estate and strengthens the profile and value of our CDNF program,” Pekka Simula, Herantis’ CEO, said in a press release. “We believe CDNF can make a significant difference in the treatment of Parkinson’s disease and we look forward to exploring the new possibilities a non-invasive administration could offer to provide the best product for the benefit of patients.”
CDNF is based on a protein naturally present in blood and cerebrospinal fluid — the liquid filling the brain and spinal cord. Preclinical studies showed that it has neuroprotective and neurorestorative properties in brain cells that generate the neurotransmitter dopamine, suggesting its potential to help Parkinson’s patients.
In 2017, Herantis began a Phase 1/2 clinical trial testing the safety and tolerability of CDNF. In this study, the therapy cannot be delivered as a pill or injection because the body will not transport it to the brain. So a neurosurgeon needs to implant a drug delivery system in patients’ brains.
In the randomized, double-blind trial (NCT03295786), participants will receive monthly infusions of either CDNF (mid- or high-dose) or placebo for six months. Herantis is recruiting 18 Parkinson’s patients for the trial.
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The study is being conducted at the Karolinska University Hospital and the Lund University Hospital, both in Sweden, and the Helsinki University Hospital in Finland.
“We have previously shown that CDNF protects and recovers neurons from degeneration, neuroinflammation and endoplasmic reticulum stress, critical contributors to many neurodegenerative diseases,” said Mart Saarma, PhD, who has been leading the research into CDNF at the University of Helsinki.
Saarma considers the development of CDNF into an investigational product “exciting” and says he is looking forward to supporting Herantis in the process. “Our recent discoveries provide an opportunity to target numerous indications beyond Parkinson’s with simpler administration and broader distribution while maintaining the full potential of CDNF,” he said.
Besides Parkinson’s, Herantis is also developing CDNF as a potential treatment for patients with amyotrophic lateral sclerosis.