Santhera Targets Third Indication - Leber's Hereditary Optic Neuropathy (LHON) - with its Lead Compound SNT-MC17/idebenone

 

Liestal, Switzerland - Santhera Pharmaceuticals, a Swiss specialty pharmaceutical company with a focus on neuromuscular diseases, announced today that it is developing its lead product SNT-MC17/idebenone in a third indication, Leber's Hereditary Optic Neuropathy (LHON). LHON is a rare genetic disease that results in the degeneration of nerve cells in the retina and optic nerve leading to a rapid loss of central vision and blindness within a period of months. The disease predominantly affects young men and presently there is no cure or therapeutic treatment. In addition to LHON, Santhera is currently developing SNT-MC17/idebenone for two life-threatening neuromuscular diseases, Friedreich's Ataxia (FRDA), where it is in Phase III clinical development and Duchenne Muscular Dystrophy (DMD) where a Phase IIa clinical study is underway.

 

There is a compelling scientific rationale that suggests that SNT-MC17/idebenone may delay, lessen or protect from vision loss in LHON. This is based on the principal mode of action of
SNT-MC17/idebenone as an enhancer of mitochondrial electron flux and energy production as well as the molecule's antioxidant properties which may protect the retinal and optic nerve cells in LHON patients.

 

Santhera has received regulatory approvals for the study in Germany and the United Kingdom and will begin to recruit newly diagnosed LHON patients for the Phase IIa clinical trial within the forthcoming weeks. The study, which is a double-blind, randomized and placebo-controlled trial, is designed to assess the efficacy of SNT-MC17/idebenone on the progression of vision loss in symptomatic LHON patients. Up to sixty LHON patients will be recruited for the study and they will be treated for a period of nine months. The LHON study's principal investigators are Prof. Patrick Chinnery (University of Newcastle upon Tyne, United Kingdom) and PD Dr. Thomas Klopstock (University of Munich, Germany), both distinguished physicians in the field of neurological and mitochondrial diseases.

 

Klaus Schollmeier, Ph.D., Chief Executive Officer of Santhera, stated: "Central to Santhera's strategy is to investigate and capture the full medical and commercial potential of SNT-MC17/idebenone. LHON is the third indication for our lead compound and complements our current clinical development program with SNT-MC17/idebenone in another rare disease with a high unmet medical need."

 

Thomas Meier, Ph.D., Chief Scientific Officer of Santhera, commented: "LHON is a mitochondrial disorder, which strikes predominantly otherwise healthy young men. Encouraged by preliminary clinical evidence and our understanding of the mode of action of SNT-MC17/idebenone, we have now initiated this proof-of-concept trial. We are pleased to collaborate with distinguished experts in this field from Newcastle and Munich."

 

- Ends -

LHON is a genetic disease that results in the degeneration of nerve cells in the retina and optic nerve, which leads to the rapid loss of central vision and blindness. The symptoms of the disease predominantly affect young adult males. LHON is due to mutations of the genetic code within the mitochondria, the energy production centers of a cell. These mutations adversely affect the biochemical processes that ensure the cellular production of energy from nutrients, which can ultimately lead to cell damage and cell death. The effects of LHON are rapid and severe, with the damage to retinal and optic neurons cells leading to blindness within a few months after the onset of symptoms. The symptomatic phase of the disease begins with blurring of central vision. Both eyes are usually affected within several months of symptom onset in the first eye. Within 12 months over 97% of patients will experience vision loss in the second eye, most often leaving them severely visually impaired. The prevalence of LHON was found in a recent study to be ~3 per 100,000.

 

Santhera Pharmaceuticals is a Swiss specialty pharmaceutical company focusing on the discovery, development and marketing of small molecule pharmaceutical products for the treatment of severe neuromuscular diseases. Santhera's vision is to become a leading specialty pharmaceutical company offering therapies for a number of indications in this area of high unmet medical need which includes many orphan indications with no current therapy. Santhera has a proven track record in discovering and developing drug candidates that address severe neuromuscular disorders in orphan and ultra-orphan diseases.

 

Santhera currently has four clinical stage development programs, three of which are investigating its lead compound, SNT-MC17/idebenone, in the treatment of Friedreich's Ataxia (FRDA), Duchenne Muscular Dystrophy (DMD) and Leber's Hereditary Optic Neuropathy (LHON). The fourth clinical program is investigating JP-1730/fipamezole for the treatment of Dyskinesia in Parkinson's Disease (DPD) in cooperation with Juvantia. The most advanced program, SNT-MC17/idebenone in FRDA, has entered pivotal Phase III clinical trials; the other clinical programs are in Phase II. There are currently no effective treatments available for FRDA. Treatments currently in use for DMD and DPD are generally viewed by the medical community as inadequate. Santhera's drug pipeline comprises another three preclinical programs in cancer cachexia, DMD and diabetes (out licensed to Biovitrum)

 

Santhera was formed in 2004 through a business combination of MyoContract AG and Graffinity Pharmaceuticals AG. The Company is based in Liestal, Switzerland. Santhera has attracted investment from leading global industry investors including NGN Capital, Merlin Biosciences Limited, 3i Group plc, Varuma AG, Oxford Bioscience Partners, the Novartis Venture Fund, Heidelberg Innovation, GIMV, Carnegie Asset Management, TechnoStart, Clariden Biotechnology Fund, the Swiss Foundation for Research on Muscle Diseases, The Dow Chemical Company, Altana Innovationsfonds, tbg, and private investors.

 

For further information on Santhera, please visit www.santhera.com.

 

 

Klaus Schollmeier, Chief Executive Officer

Phone: +41 (0) 61 906 89 52

 

Thomas Meier, Chief Scientific Officer

Phone: +41 (0) 61 906 89 87

 

Thomas Staffelbach, VP Public & Investor Relations

Phone: +41 (0) 61 906 89 47

 

David Dible

Phone: +44 (0) 207 638 9571

 

Chris Gardner

Phone: +44 (0) 207 638 9571