Santhera Secures Exclusive Rights from NIH for a Novel Approach for the Treatment of Primary Progressive MS

Santhera Pharmaceuticals Holding AG / Santhera Secures Exclusive Rights from NIH for a Novel Approach for the Treatment of Primary Progressive MS . Processed and transmitted by Thomson Reuters ONE. The issuer is solely responsible for the content of this announcement.

Liestal, Switzerland, June6, 2013 -Santhera Pharmaceuticals (SIX:SANN) announced today 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 PhaseII 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 PhaseII 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. "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.

About primary progressive MS Multiple Sclerosis (MS) is a chronic neurological disease of the central nervous system affecting around 2.5 million people worldwide. Notable differences in MRI findings can distinguish clinical subtypes of MS. The relapsing-remitting form of MS (rrMS) is the most common form of the disease. Patients affected by rrMS experience attacks of clinical worsening followed by periods of clinical stability with partial or even complete recovery. So far, effective treatments for rrMS are limited to immune-modulatory therapies which have not been proven to be effective in ppMS patients. This subtype develops much slower than rrMS but presents a steady functional decline without any distinct episodes of regeneration or acute relapses. According to the MS Association of America (MSAA) there are about 40,000 patients with ppMS living in the United States.

About idebenone and rationale for use in ppMS Mitochondrial dysfunction together with aberrant formation of reactive oxygen species are believed to underlie at least partially the development of progressive CNS tissue destruction in ppMS. Idebenone, a synthetic short-chain benzoquinone and a cofactor for the enzyme NAD(P)H:quinoneoxidoreductase (NQO1) is capable of transferring electrons directly onto complex III of the mitochondrial electron transport chain, thereby restoring cellular energy levels. In addition, idebenone is a powerful anti-oxidant. In view of the hypothesized involvement of mitochondrial dysfunction, and particularly the reduced activity of the mitochondrial electron transport chain in patients affected by ppMS, idebenone is a rational treatment choice for this disease based on its pharmacological properties including its ability to cross the blood-brain barrier.

About the IPPoMS trial Adult ppMS patients up to 65 years of age with disability ranging from none to moderately severe are eligible to enroll in the trial. This is a PhaseI/II safety/efficacy trial with an adaptive trial design: one year of pretreatment baseline period serves the dual purpose of collecting patient-specific biomarkers of disease progression and collecting longitudinal neuroimaging and clinical data for selection of primary outcome measures. This baseline period is then followed by a double-blind, idebenone (2250mg/day) versus placebo treatment phase for a total of 2 years. Additional information can be obtained under http://www.clinicaltrials.gov (NCT00950248).

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About the NINDS The National Institute of Neurological Disorders and Stroke (www.ninds.nih.gov) is the nation's leading funder of research on the brain and nervous system. The NINDS mission is to reduce the burden of neurological disease - a burden borne by every age group, by every segment of society, by people all over the world.

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Santhera Secures Exclusive Rights from NIH for a Novel Approach for the Treatment of Primary Progressive MS