Herantis Pharma receives orphan designation of CDNF for treatment of ALS
Herantis Pharma Plc
Company release 29 March 2016 at 9:00 am
The European Medicines Agency's Committee for Orphan Medicinal Products (COMP) has issued a positive opinion on the application of Herantis Pharma Plc ("Herantis") for orphan drug designation of recombinant human Cerebral Dopamine Neurotrophic Factor (CDNF) for treatment of amyotrophic lateral sclerosis (ALS).
"This positive opinion underlines two things", explains Dr. Henri Huttunen, Chief Scientific Officer of Herantis. "First, there is a dire need for novel treatments for ALS, which is a truly devastating disease. Second, based on scientific data we have justified the assumption that CDNF will be of significant benefit to ALS patients. While years of hard work remains, this is an important milestone for us developing a novel treatment for ALS."
ALS is caused by the progressive loss of neurons that control voluntary muscles. This leads to stiffness, weakness, difficulty in speaking, swallowing, and eventually breathing. Most patients die from respiratory failure. The underlying cause of the disease can be genetic but in most cases remains unknown. No efficacious treatments are known.
"Even though an orphan indication, ALS is a major societal challenge", reminds Pekka Simula, Herantis' CEO. "140,000 new cases are diagnosed every year and the financial burden of motoneuron diseases in Europe is about €8 billion every year. 140,000 people a year are basically given a slow death sentence until we develop effective treatments. We will do our best to make CDNF available in the future as a new treatment option for ALS patients."
Orphan drug designation can be provided in the European Union to drugs intended to treat diseases or disorders affecting no more than five persons per 10,000. In addition at least early preclinical data are required, which suggest that the drug could provide patients with benefits that improve over currently available therapies. Orphan drug designation does not exempt the company from clinical studies for establishing the safety and efficacy of the drug. The designation however provides benefits and incentives such as fee waivers, scientific advice, and an extended marketing exclusivity period against competition.
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.
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.