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Herantis Pharma announces Phase I-II study results

Parkinsons disease

Herantis Pharma has announced that its novel drug candidate, Cerebral Dopamine Neurotrophic Factor (CDNF) for the treatment of Parkinson’s disease (PD), has successfully achieved its primary endpoint of safety and tolerability in a 12 month Phase I-II study in patients with moderate disease.

CDNF is a multi- modal, natural protein that has shown potential to slow or even reverse loss of dopaminergic neurons, the main catalyst in PD.

Promising results

The two-part study in 17 patients comprised of an initial 6-month period in which all patients were assigned to either placebo or CDNF at one of two dose levels, followed by a 6-month extension period, in which all patients received one of the two dose levels of CDNF on a monthly basis, including the previous placebo group patients. Treatment was administered via a dose delivery system provided by Renishaw Neuro Solutions Ltd, that was implanted into the brain at the beginning of the study. Topline data from the initial 6-month period was reported in February 2020.

Treatment Emergent Adverse Events (TEAE) were generally mild and transient, and reduced compared to the first period. Similarly, Serious Adverse Events (SAEs) were also less frequent during the second treatment period, and all patients who previously experienced SAEs have since recovered.

The study also gathered preliminary non-statistical data to support an exploratory assessment of the product’s potential efficacy. This included measuring the severity of motor symptoms using the Unified Parkinson’s Disease Rating Scale (UPDRS). Although patient responses varied, at the 12-month mark the data suggests an overall improvement in their motor symptoms as measured by UPDRS. This may suggest a potential slowing of disease progression in some patients.

An additional exploratory assessment evaluated biological signals in the brain, as measured by Dopamine Transporter Positron Emission Tomography (DAT PET) imaging which provides an indirect measure of dopaminergic function in the brain.  Responses again varied, but a promising biological signal was seen in some patients where significant increases in DAT PET signaling was observed in the target infusion area of CDNF.

Read more: Interview with Pekka Simula, Senior Advisor, Herantis Pharma

“In addition to the study’s favorable safety profile, it is encouraging to observe an improvement in the UPDRS motor score as well as an indication of a biological response compared to baseline, which may be clinically relevant. Considering that patients with moderate Parkinson’s disease are not an optimal cohort for studying effects of neuroprotective therapeutics, this is especially promising,” says Professor Per Svenningsson from Karolinska University Hospital, Principal Investigator of this clinical trial. “In chronic diseases, such as Parkinson’s, where patients continue to decline over time despite receiving treatment, the differences observed in the treatment and control groups in this study present a potential opportunity for the development of meaningful therapeutic effects.”

Further development

“As this is the first clinical study of CDNF and was therefore designed primarily to examine the safety and dosing of our drug candidate, we are encouraged by the safety and tolerability we have seen so far as well as the observed improvements in some patients as measured by UPDRS and DAT PET imaging,” says Craig Cook, CEO of Herantis Pharma. “Based on the outcome of this trial, we continue to be optimistic regarding the ongoing development of CDNF and intend to further develop and demonstrate its neuroprotective effects to make a difference in the lives of patients living with PD.”

Analyses of important additional exploratory outcome measures, such as cerebrospinal fluid alpha-synuclein and proteomics studies, are ongoing and expected to be completed in Q4 2020. Further development of CDNF by Herantis includes potential follow on clinical studies and additional optimization of dosing, delivery and patient selection.

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