Herantis Pharma opening recruitment in Parkinson’s disease study in Helsinki and Lund based on favorable first CDNF safety assessment

Herantis Pharma opening recruitment in Parkinson's disease study in Helsinki and Lund based on favorable first CDNF safety assessment

Herantis Pharma Plc
Company release 14 February 2018 at 9:00 am

Herantis Pharma Plc's ("Herantis") first-in-human clinical study with the company's investigational product CDNF for the treatment of Parkinson's disease has proceeded to its first CDNF safety assessment by an independent Data Safety Monitoring Board ("DSMB"). Following the DSMB's recommendation based on safety data on the first patients, the study continues as planned. Subsequently patient recruitment is opening at two new study sites: Helsinki University Hospital in Finland, and Skåne University Hospital in Lund, Sweden. The first study site, Karolinska University Hospital in Stockholm, Sweden continues to recruit additional patients.

"Patient safety is always our first priority", comments Sigrid Booms, Herantis' Director of Clinical Development. "Clinical dosing of CDNF for the first time in the world is a major milestone for our company. We are very pleased with the progress and the collaboration with the top-level neurological and neurosurgical teams of these three hospitals, and aim to have all patients recruited in the study by the end of 2018."

The randomized, placebo-controlled clinical study of CDNF intends to recruit in total 18 patients with Parkinson's disease. The participating patients will also be offered a possibility to participate in an extension study in which all patients will receive CDNF treatment, subject to regulatory approval of the extension study.

More detailed information on the CDNF clinical study is available on the EU project's website at https://treater.eu/.

Further information:

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 (PD) is a slowly progressing, incurable neurodegenerative disease caused by the death of dopaminergic neurons in the midbrain. 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, PD may cause non-motor symptoms such as sleep problems, depression, and anxiety, which are not alleviated by current Parkinson's drugs. It is estimated that 7 million people worldwide suffer from Parkinson's disease.

About CDNF

CDNF is a novel drug candidate for the treatment of neurodegenerative diseases. Naturally present in the human blood circulation and cerebrospinal fluid, CDNF is a protein with neuroprotective and neurorestorative properties, patented internationally by Herantis. Encouraged by strong preclinical proof-of-concept Herantis has launched a first-in-human, randomized Phase 1-2 clinical study with CDNF for 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 grant agreement number 732386.

CDNF administration has been safe in preclinical studies including chronic toxicology studies; CDNF has protected and regenerated midbrain dopamine-generating cells suggesting potential for disease modification of PD. It has also shown efficacy in non-motor symptoms of 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 University of Helsinki, led 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.


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