Herantis Pharma Plc Announces CDNF Phase I-II Study Met Primary Endpoints of Safety and Tolerability at 12 months
Data on exploratory efficacy endpoints continue to show a promising biological signal in some patients
Herantis Pharma Plc
Company release August 27, 2020 at 9:00 AM Eastern European Summer Time
Herantis Pharma Plc (“Herantis” or “Company”) announced today 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.
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.
“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,” said 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.”
“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,” said 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.
Herantis Pharma Plc, Craig Cook, CEO, telephone: +358 9 222 119
Company web site: www.herantis.com
Certified Advisor: UB Securities Ltd, telephone: +358 9 25 380 225
Cerebral Dopamine Neurotrophic Factor (CDNF) is a promising innovative drug candidate for the treatment of neurodegenerative diseases. This unique compound is a member of a novel family of unconventional neurotrophic factors which is structurally and mechanistically very distinct from all known neurotrophic factors. Specifically, CDNF acts via a multimodal mechanism that involves modulation of endoplasmic reticulum (ER) stress, a-synuclein aggregation and toxicity and reduced neuroinflammation. CDNF is patented internationally by Herantis.
In preclinical studies it has definitively been shown to be safe and effective. In particular, it protects and regenerates dopamine-generating cells in the midbrain, suggesting potential for disease modification of Parkinson’s disease. It has shown efficacy in both motor and non-motor symptoms in rodent and primate models of Parkinson’s disease.
CDNF has entered the clinic where its profile is being evaluated for safety and efficacy. A Phase I-II study was successfully completed which has established safety of the product and preliminary indications of efficacy in patients.
About Parkinson’s disease
Parkinson’s disease (PD) is a slowly progressing, incurable neurodegenerative disease caused by the loss of dopaminergic neurons in the midbrain. Common first symptoms of the disease include tremors, rigidity, and slowness of movement. While the motor symptoms can be treated with medication, the progression of the disease cannot be prevented. The benefits of medication may be lost with disease progression or their side effects can become unmanageable. The progressing disease is also associated with non-motor symptoms such as sleep problems, depression, and anxiety, which are not alleviated by current Parkinson’s drugs. It is estimated that 7-10 million people worldwide suffer from Parkinson’s disease.
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 revolutionize 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.