Herantis Pharma's clinical study with CDNF in Parkinson's disease authorized in Finland, company expands on development plan
Herantis Pharma Plc
Company release 12 June 2017 at 9:00 am
Finnish Medicines Agency Fimea has authorized Herantis Pharma Plc's ("Herantis") first-in-human clinical study of the company's investigational product CDNF for the treatment of Parkinson's disease. The company announced earlier this year having secured approvals for the same clinical study in Sweden. The first study site to start patient recruitment will be the Karolinska University Hospital in Stockholm, Sweden.
At the same time Herantis has decided to postpone the launch of patient recruitment to the third quarter of 2017. The company has previously estimated that the study would be started in the first half of 2017. The company considers it justified to postpone patient recruitment until after the local holiday period to secure optimal resources for the study and to ensure the safety of the patients. Consequently the expansion of the study into further study centers including Finland is postponed to 2018.
The clinical study intends to recruit in total 18 patients with Parkinson's disease. A subset of the patients will be administered a placebo infusion instead of CDNF, in a blinded manner. This study setup supports a more robust initial assessment of the effects of CDNF. The participating patients will also be offered the possibility to participate in an extension study in which all patients will receive CDNF, subject to regulatory approvals of the extension study.
Herantis Pharma Plc, Pekka Simula, CEO, telephone: +358 40 7300 445
Company web site: www.herantis.com
Certified Advisor: UB Securities Ltd, telephone: +358 9 25 380 225
About Parkinson's disease
Parkinson's disease is a slowly progressing neurodegenerative disease caused by the death of dopaminergic neurons in the midbrains. Common first motor symptoms of the disease include tremors, rigidity and slowness of movement. While the motor symptoms can be treated with medication the disease progression cannot be prevented, and the benefits of medication may be lost with disease progression or side effects can become unmanageable. In addition, Parkinson's disease may cause non-motor symptoms such as sleep problems, depression, and anxiety, which are not alleviated by current Parkinson's drugs. Estimated 7 million people worldwide have Parkinson's disease.
CDNF, or Cerebral Dopamine Neurotrophic Factor, is an endoplasmic reticulum located and secreted protein with neuroprotective and neurorestorative properties, patented worldwide by Herantis. Encouraged by strong preclinical proof-of-concept Herantis is launching a first-in-human, randomized Phase 1-2 clinical study with CDNF for the treatment of Parkinson's disease in Q3/2017. The clinical study has received funding from the European Union's research and innovation program Horizon 2020 under grant agreement number 732386.
In preclinical studies including chronic toxicology studies, CDNF administration has been safe; CDNF has protected and regenerated midbrain dopamine-generating cells suggesting a potential for disease modification of PD; it has also shown efficacy in non-motor symptoms in PD. In an ALS disease model CDNF has significantly increased survival and reduced symptoms. This suggests the potential to address unmet clinical needs in both PD and ALS. CDNF drug development is based on research at the Institute of Biotechnology at the University of Helsinki, lead by professor Mart Saarma.
About drug development in general
Drug development projects can usually be divided in two stages: The preclinical stage, and the clinical stage involving human subjects. The clinical stage can be further broken in three formal phases. Phase 1 clinical studies assess the safety of a drug candidate in human subjects. In Phase 2, the optimal dosing and possible efficacy in the treatment of a particular disease is studied. Phase 3 studies finally aim to establish a statistical proof of safety and efficacy of the drug candidate in typically hundreds or thousands of patients for market approval. Drug development can take 10-15 years from the first preclinical studies to market approval.
About Herantis Pharma Plc
Herantis Pharma Plc is an innovative drug development company focused on regenerative medicine for breakthrough in unmet clinical needs. Our first-in-class assets are based on globally leading scientific research in their fields: CDNF for disease modification in neurodegenerative diseases, primarily Parkinson's and ALS; and Lymfactin® for breast cancer associated lymphedema, with potential also in other lymphedemas. The shares of Herantis are listed on the First North Finland marketplace run by Nasdaq Helsinki stock exchange.