Health Care Professionals


Santhera is passionate about developing new treatments for patients with mitochondrial diseases. Our focus is on the development of treatments for neuromuscular and neuro-ophthalmological diseases that currently lack treatment options.

Our pipeline: Raxone in PPMS

Multiple sclerosis (MS) is an inflammatory and demyelinating disorder of the central nervous system.1 About 10–15% of all MS patients suffer primary progressive MS (PPMS).1,2 This sub-type is characterized by a gradual progression of the neurological impairment from its onset with no superimposed relapses or remissions.1,2

Ppms Deut
Rrms Deut

While the cause of MS remains unclear, accumulating data indicate that – especially in progressive stages – mitochondrial dysfunction and oxidative stress may play a major role, particularly in the pathogenesis of PPMS.3

There is still an urgent medical need for effective therapies in PPMS to treat patients with this debilitating disease.1,2

Raxone clinical development in PPMS

Raxone is being studied in a Phase I/II trial (IPPoMS) in patients with PPMS.4,5
IPPoMS (Idebenone in Patients with Primary Progressive Multiple Sclerosis) is a Phase I/II trial with a 12-month pre-treatment baseline period followed by a double-blind, randomized, placebo-controlled treatment of 24 months duration investigating the safety and efficacy of high dose Raxone versus placebo in patients with PPMS.4 Patients can enroll in a 12 month extension study.5

These studies are sponsored and conducted by the National Institute of Neurological Disorders and Stroke (NINDS) and the IPPoMS trial is expected to complete in 2018.4,5

For more information about the IPPoMS study and extension, please visit (trial identifiers: NCT00950248 and NCT01854359).4,5



EMA Guideline on clinical investigation of medicinal products for the treatment of Multiple Sclerosis, March 2015; Available here. Accessed December 2015.


Miller DH and Leary SM. Lancet Neurol 2007; 6: 903–912.


Lassmann, H et al. Nat Rev Neurol 2012; 8:647–656.


Clinical Trials Identifier NCT00950248. Available here. Accessed December 2015.


Clinical Trials Identifier NCT01854359. Available here. Accessed December 2015.