Herantis Pharma has announced the results from the ongoing Phase 1-2 clinical trial examining its neuroprotective factor and novel drug candidate, CDNF, in patients with Parkinson’s disease.
Herantis is developing CDNF as a disease-modifying treatment. As a novel neuroprotective and neurorestorative factor, CDNF acts on several mechanisms relevant to Parkinson’s disease and has been shown to protect neurons from degeneration and to restore the function of already degenerating neurons in preclinical studies, states the company.
The first and second part of the study
The first part of the Phase 1-2 study, in which patients first received a surgically implanted dose delivery system provided by Renishaw plc, and then repeated and increased CDNF or placebo dosing for 6 months, met its primary endpoint of safety and tolerability. The recorded Serious Adverse Events (SAEs) were considered unlikely by the treating physicians to be related to CDNF and the affected patients fully recovered. Certain SAEs were considered to be probably related to device surgery and the drug administration process. The surgical and infusion procedures were improved to avoid any such incidents in the future.
The secondary and exploratory endpoints of the study evaluate initial signs of efficacy, including assessments based on the Unified Parkinson’s Disease Rating Scale (UPDRS), dopamine transporter PET imaging, actigraphy measurements and alpha-synuclein levels. At this early stage of data review, the company observed promising signals in some patients, for instance in dopamine transporter PET imaging, which is an indirect measure of the dopaminergic function. As the trial is a first-in-human study involving a small number of patients at an advanced disease stage, this is an encouraging initial outcome. The company will continue to assess the results through the extension part of the study which will last six months.
Initiate patient enrolment in 2021
All patients who completed the first part of the trial volunteered to participate in the extension study in which every patient, including those previously randomly assigned to the placebo group, will receive one of the two dose levels of CDNF on a monthly basis. Herantis expects to announce the next set of results, including details on the exploratory endpoints, in Q3/2020.
“This first set of topline data provides a solid basis for the next part of the study and confirms the positive safety and tolerability profile of CDNF,” commented Pekka Simula, CEO of Herantis. “Building on the established safety profile and encouraging observations, we have initiated the planning for a Phase 2 study with a longer treatment period that will assess the efficacy of CDNF in earlier-stage, well-characterized Parkinson’s patients. We currently expect to initiate patient enrolment in 2021. We extend our thanks to the clinical study sites and the patients who contributed to the advancement of this very important and challenging trial.”