Topline results of Phase 1-2 CDNF trial

Herantis Pharma Plc announces topline results of Phase 1-2 CDNF trial

Topline analysis confirms positive safety and tolerability of CDNF in advanced-stage Parkinson’s disease patients, with encouraging biological responses as measured by PET imaging in some patients.

Results from the second part of trial, in which all patients will receive CDNF for an additional six months, are expected in Q3 2020

Company release 25 Feb 2020 at 9:00 am: Herantis Pharma Plc

Herantis Pharma Plc (“Herantis” or “Company”) has announced the topline results from the ongoing Phase 1-2 clinical trial examining Herantis’ proprietary neuroprotective factor and novel drug candidate, CDNF, in patients with Parkinson’s disease. Herantis is developing CDNF as a disease-modifying treatment with the objective of introducing a significant breakthrough to current standard-of-care therapies for Parkinson’s disease. 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.

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.

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-characterised 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.

Study Design

The randomised, placebo-controlled Phase 1-2 trial is a first-in-human study that evaluates the safety and tolerability of CDNF, a novel experimental therapy, in a total of 17 patients with advanced Parkinson’s disease. In the main study, the patients received an implant of the Renishaw plc investigational drug delivery system. The patients were then randomised to receive either six monthly doses of placebo, or six increasing doses of CDNF, in a blinded manner. All patients who completed the main study volunteered to continue in an extension study, in which all patients, including those who previously received placebo doses, receive either lower or higher CDNF doses for another six months. After the extension study, which is expected to be completed in Q2/2020, the Phase 1-2 trial continues with a long-term follow-up study.

Study Objectives

The primary endpoints of the study will evaluate safety and tolerability of CDNF as well as of the drug delivery device, and accuracy of surgical placement of the device in the study patients. Secondary and exploratory endpoints include initial signs of efficacy of CDNF treatment, e.g. Unified Parkinson’s Disease Rating Scale (UPDRS), motor score evaluation, patient diary, dopamine transporter PET imaging, and the levels of different forms of alpha synuclein in serum and CSF.

Further information:

Herantis Pharma Plc, Pekka Simula, CEO, +358 40 7300 445

About CDNF

CDNF is a novel neuroprotective and neurorestorative factor highly distinct from conventional neurotrophic factors. An innovative drug candidate for the treatment of neurodegenerative diseases, CDNF, is patented internationally by Herantis. Supported by a strong preclinical proof-of-concept Herantis launched a first-in-human, randomized Phase 1-2 clinical study with CDNF in the treatment of Parkinson’s disease (PD). The clinical study has received funding from the European Union’s research and innovation program Horizon 2020 under the grant agreement number 732386.

CDNF has been safe in preclinical studies and in the completed parts of the ongoing Phase 1-2 clinical study. In disease models, CDNF has protected and regenerated dopamine-generating cells in the midbrain suggesting potential for disease modification of PD. It has also shown efficacy in non-motor symptoms of PD.

About Herantis Pharma Plc

Herantis Pharma Plc is an innovative drug development company breaking the boundaries of standard therapeutic approaches. Our regenerative medicine drug candidates, CDNF and Lymfactin®, aim to revolutionise the treatment of Parkinson’s disease and other neurodegenerative diseases, and of secondary lymphedema. The shares of Herantis are listed on the Nasdaq First North Growth Market Finland and Nasdaq First North Growth Market Sweden.