Herantis Pharma receives orphan designation in USA for CDNF for treatment of ALS

Herantis Pharma receives orphan designation in USA for CDNF for treatment of ALS

Herantis Pharma Plc
Company release 11 July 2016 at 9:00 am

The United States Food and Drug Administration FDA has granted orphan designation for Herantis Pharma Plc's ("Herantis") CDNF for treatment of amyotrophic lateral sclerosis (ALS). Earlier this year the European Medicines Agency EMA issued a corresponding positive decision.

"We are very pleased with the world's leading medicines agencies reaching independently the same conclusion on the strength of the preclinical evidence", says Dr. Henri Huttunen, Chief Scientific Officer of Herantis.

"This positive decision by the FDA greatly encourages us in the planning of a clinical development program of CDNF for the treatment of ALS in addition to the treatment of Parkinson's disease", adds Pekka Simula, Herantis' CEO.

Orphan drug designation can be granted by the FDA for drugs intended to treat diseases or disorders affecting no more than 200,000 patients in the US. In addition a medically plausible basis must be established for expecting the drug to be effective. Orphan drug designation does not exempt the company from clinical studies; however the designation provides several benefits and incentives.

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 25 380 246

ALS (Amyotrophic Lateral Sclerosis)

ALS, also known as Motor Neurone Disease or Lou Gehrig's disease, is a fatal motoneuron disease. First symptom of the disease is typically weakness of limb muscles. With disease progression the patient loses ability to control muscles causing difficulty in moving, speaking, swallowing and breathing. Average lifetime expectation from diagnosis is between two and five years. ALS cannot be cured and available treatments are essentially symptomatic. Estimated 140,000 new cases are diagnosed annually.

About CDNF

CDNF, or Cerebral Dopamine Neurotrophic Factor, is a neuroprotective and neurotrophic protein patented worldwide by Herantis.  Following a preclinical development program, which has showed it as efficacious in several preclinical models of Parkinson's disease (PD), Herantis is preparing for a first-in-human clinical study of CDNF in the treatment of PD and has a preclinical development program for the treatment of ALS.

In preclinical studies including chronic toxicology studies, CDNF administration has been safe; CDNF has protected and regenerated 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 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 a drug development company focused on early clinical development of innovative drugs in unmet clinical needs. Our special emphasis is in regenerative medicine where the company has two first-in-class assets based on globally leading science in their fields: CDNF for neurodegenerative diseases, primarily Parkinson's and ALS; and Lymfactin® for breast cancer associated lymphedema, with potential also in primary lymphedema. The shares of Herantis are listed on the First North Finland marketplace run by Nasdaq Helsinki stock exchange.


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