December 4, 2015 -­‐ FOR IMMEDIATE RELEASE

NIJMEGEN – Khondrion, the Dutch biopharmaceutical company focusing on small molecule therapeutics for mitochondrial diseases, announces today the successful completion of its Phase 1 Clinical trials with its lead compound KH176. KH176 is an orally bio-­‐available small molecule developed by Khondrion for the treatment of mitochondrial (-­‐related) diseases. The compound is a member of a new class of Khondrion drugs essential for the control of oxidative and redox pathologies. The Phase 1 Clinical trials, performed in healthy male volunteers, consisted of single ascending and multiple dosing arms both randomized, placebo controlled and double-­‐blinded. The study deemed that KH176 is well tolerated and has an excellent pharmacokinetic profile.

Jan Smeitink, Khondrion’s CEO and Professor of Mitochondrial Medicine at the Radboud University Medical Center, Nijmegen, The Netherlands, said “With the successful outcome of the KH176 Phase I Clinical trials we have set an important step to accomplish our mission – making a substantial contribution to the development of drugs for patients suffering from mitochondrial diseases".

In July this year three German speaking countries, Germany, Austria and Switzerland, came together to share knowledge on the latest developments in research, diagnostics and therapy in mitochondrial medicine. Special attention was paid to the broad circle of treating clinicians who wish to learn more about mitochondrial disease. The congress was supported in different ways by the german DGM, represented by the special Mito-Team. DGM is also a member of IMP and as such on the forefront of the latest developments in mitochondrial medicine.

After learning about all new research developments the conclusion was drawn that there is light at the end of the tunnel (finding a cure) but there is still a long way to go. And in the meantime patients and their representatives can undertake many activities to support and stimulate further research. For further information you may contact Carsten Gamroth of the DGM and member of the board of IMP: This email address is being protected from spambots. You need JavaScript enabled to view it.. The entire report is in the German language and can be read here:

Liestal, Switzerland, October 1, 2015

Santhera Pharmaceuticals (SIX: SANN) announces that it is today launching Raxone® for the treatment of Leber's Hereditary Optic Neuropathy (LHON) in Germany, its first and largest EU market. LHON is a rare inherited mitochondrial disease that usually leads rapidly to profound and permanent blindness.

 "The introduction of Raxone in Germany is a major step forward for LHON patients who now have an effective treatment option" said Giovanni Stropoli, Santhera's Chief Commercial Officer for Europe. "We are particularly proud as Raxone is also the first approved treatment for any mitochondrial disease, a therapeutic area which is in the focus of our Company. Germany is our first and largest EU market but product availability is anticipated in additional countries in the immediate future."

MEETers run for IMP


Patients with mitochondrial diseases suffer: headaches, neuropathic pain, abdominal pain, muscle pain and aches. Indeed, kids and adults with mitochondrial disorders endure various types of torments and in many cases no obvious cause for pain can be found and those patients frequently feel much worse than they look. That a look at the youtube clip:

It is all the more regrettable that we sadly record a lack of general awareness about these diseases. Until relatively recently, mitochondrial disorders were considered obscure, exceptionally rare diseases, but they actually are amongst the most common inherited human diseases.

Statins are a popular and easy-to-swallow option for people looking to lower their cholesterol. But for a quarter of patients, statins come with muscle pain, stiffness, cramps, or weakness without any clear signs of muscle damage. These symptoms may affect daily activities so much that people stop using the drugs. In Cell Metabolism on September 1, Dutch researchers show, in mice and humans, that statins yield an off-target reaction that disrupts muscle mitochondria function, possibly causing the side-effects.

“Adverse drug-effects, like those of statins and many other drugs, have been linked to mitochondria—the cell’s powerhouses—though the exact mechanisms are often unknown,” says co-senior study author Frans Russel, of the Nijmegen Center for Mitochondrial Disorders at the Radboud University Medical Center in the Netherlands. “This research leads to several opportunities to synthesize new classes of cholesterol-lowering drugs without the unwanted muscle effects, as well as the development of new avenues to counteract these effects, both of which we are currently investigating.”

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