Santhera Secures Exclusive Rights from NIH

Santhera Pharmaceuticals has announced that it has obtained an exclusive license from the National Institutes of Health (NIH) to its rights on a patent granted in the USA for the use of idebenone for the treatment of primary progressive Multiple Sclerosis (ppMS), a currently untreatable disease affecting about 40,000 patients in the Unites States.

The NIH is investigating the efficacy of Catena® (idebenone) in ppMS in a placebo-controlled Phase II clinical trial.

Lead by the Neuroimmunology Branch of the National Institute of Neurological Disorders and Stroke (NINDS), the NIH is investigating the efficacy of Catena® (idebenone) in patients with ppMS in a double-blind, placebo-controlled Phase II clinical trial (IPPoMS trial).

Santhera is providing study medication under a clinical trial agreement which gives Santhera the rights to the results. Santhera has now obtained the exclusive rights to the use patent for idebenone in ppMS granted in the USA.

Patients who complete the IPPoMS trial can enter into a 12-months open-label extension trial for which Santhera and the NIH recently signed a Materials Cooperative Research and Development Agreement (M-CRADA).

"Patients with ppMS do not respond to immunomodulatory therapies with proven efficacy in relapsing remitting MS," said Bibiana Bielekova, M.D, the principal investigator of the IPPoMS trial and Chief of the Neuroimmunology Disease Unit at the NINDS.

Bielekova continued, "Accumulating data indicate that mitochondrial dysfunction and related oxidative stress may play a major role in the pathogenesis of progressive MS. Idebenone enhances mitochondrial function and acts as an anti-oxidant against membrane damage in laboratory models and is a rational treatment choice in ppMS based on these pharmacological properties. Recruitment is proceeding well in the IPPoMS trial and we are excited about the opportunity to study the potential of idebenone in this currently untreatable disease."

"We are very enthusiastic about this collaboration and about the progress currently being made. ppMS is a disease with high medical need. In contrast to other forms of MS, there is currently no treatment option available for the 10-15 % of MS patients affected by this primary progressive subtype," commented Nick Coppard, Santhera's Head of Development.