Santhera Provides Update on Clinical/Regulatory Processes with SNT-MC17/Idebenone for the Treatment of Friedreich's Ataxia

LONDON, Aug. 8, 2008-Santhera Pharmaceuticals (SWX:SANN), a Swiss specialty pharmaceutical company focused on neuromuscular diseases, today gave an update on the clinical and regulatory processes surrounding the commercialization of SNT-MC17/idebenone for the treatment of Friedreich's Ataxia. The Company requested reexamination of the negative opinion by the CHMP regarding Sovrima® (idebenone), despite the progress of its ongoing Phase III trials. As previously announced, the advanced status of Santhera's Phase III trials appeared to have prevented a review by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMEA) on the Company's Marketing Authorization Application (MAA) under the EMEA Guideline on Clinical Trials in Small Populations, as requested upon filing of the MAA in 2007. Meanwhile, Health Canada has published the product monograph of Catena® (idebenone), subsequent to the July 24 approval of the drug to treat symptoms of Friedreich's Ataxia under a Notice of Compliance with Conditions.

Despite the good progress of the ongoing phase III studies, Santhera requested reassessment of the CHMP's initial negative opinion on an EU approval. The Company has 60 days starting from the day of the CHMP opinion to forward detailed grounds for requesting reexamination. For the procedure, the CHMP will appoint a different rapporteur and, as requested by Santhera, consult with a specialist Scientific Advisory Group for specific questions.

In Europe, the MICONOS (Mitochondrial Protection With Idebenone In Cardiac Or Neurological Outcome Study) Phase III trial has currently enrolled 182 patients, i.e. approx. 90% of the patients needed to complete recruitment and is on track for full enrollment in 2008.

In the United States, the IONIA (Idebenone effects On Neurological ICARS Assessments) Phase III trial has currently enrolled 48 patients. It was agreed with the US Food and Drug Administration under a Special Protocol Assessment process to recruit a minimum of 51 patients but to include more patients if available. Given the current prospects for patient availability, Santhera and its US clinical investigators believe that the study can enroll about 60 to 65 patients by early Q4 2008.

Klaus Schollmeier, Chief Executive Officer of Santhera, commented: "Together with our European marketing partner Takeda, we concluded that requesting reexamination of the initial CHMP opinion is a feasible option for making this important drug available as a controlled pharmaceutical product to patients throughout Europe as we are allowed to do in Canada following the recent marketing authorization under conditions for Catena® in that country. We are aware of the challenges of such reexamination processes, but we believe that the clinical benefit afforded to Friedreich's Ataxia patients was underestimated in the initial assessment."

About Friedreich's Ataxia

Friedreich's Ataxia is a rare but severe genetic neuromuscular disorder that results in the degeneration of an individual's nerve and muscle tissue. This disorder causes loss of muscle control, uncoordinated movements, muscle wasting and thickening of heart walls which frequently leads to a shortened life span. Friedreich's Ataxia affects both Caucasian males and females equally and it is estimated that about 20,000 patients suffer from the disease in North America and Europe. Average life expectancy for Friedreich's Ataxia patients is limited to approximately 35 to 50 years.

The disorder results from a genetic defect in the gene encoding for frataxin. Reduced levels of this protein ultimately result in impaired energy production in mitochondria, the cells' energy production centers, and elevated oxidative stress. Tissues that have the highest need for energy, in particular nerve and cardiac tissues, are primarily affected by frataxin deficiency resulting in pathological changes in heart muscle anatomy and function and loss of nerve cells.

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About Santhera

Santhera Pharmaceuticals (SWX:SANN) is a Swiss specialty pharmaceutical company focused on the discovery, development and marketing of small-molecule pharmaceutical products for the treatment of severe neuromuscular diseases, an area of high unmet medical need which includes many orphan indications with no current therapy. Santhera currently investigates three compounds in five clinical-stage development programs. The Company's first product, SNT-MC17/idebenone, has received a marketing approval with conditions from Health Canada to treat Friedreich's Ataxia and will be marketed under its brand name Catena®. The product is also under review by health authorities in the EU and in Switzerland, while in the United States, a pivotal Phase III trial is recruiting patients. The compound has also shown efficacy in a Phase II clinical trial as a potential treatment for Duchenne Muscular Dystrophy. For further information, please visit the Company's website www.santhera.com.

Sovrima® is a trademark of Takeda Pharmaceuticals in the European Union and Switzerland. Catena® is a trademark of Santhera Pharmaceuticals, registered in Canada and the United States.

Please check also www.emea.europa.eu/pdfs/human/euleg/5074505en.pdf for further details on such reexamination procedures.

For Further Information, Contact

Klaus Schollmeier, Chief Executive Officer Phone: +41 (0)61 906 89 52 klaus.schollmeier@santhera.com

Barbara Heller, Chief Financial Officer Phone: +41 (0)61 906 89 54 barbara.heller@santhera.com

Thomas Staffelbach, Head Public & Investor Relations Phone: +41 (0)61 906 89 47 thomas.staffelbach@santhera.com

Disclaimer/Forward-looking statements

This communication does not constitute an offer or invitation to subscribe for or purchase any securities of Santhera Pharmaceuticals Holding AG. This publication may contain certain forward-looking statements concerning the company and its business. Such statements involve certain risks, uncertainties and other factors which could cause the actual results, financial condition, performance or achievements of the company to be materially different from those expressed or implied by such statements. Readers should therefore not place undue reliance on these statements, particularly not in connection with any contract or investment decision. The company disclaims any obligation to update these forward-looking statements.

 

 

Posted: August 2008

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