January 31, 2007: Santhera Intends Early Filing in Europe for SNT-MC17/idebenone in Friedreich's Ataxia

 

 

Santhera's early filing strategy takes advantage of new EU regulatory guidelines covering drug development for small patient populations. These guidelines aim to address patient needs by facilitating the approval of product candidates developed to treat diseases affecting only a small number of patients in the EU.

 

Santhera currently intends to file an MAA for SNT-MC17/idebenone for the treatment of FRDA during summer 2007. The filing, under the new EU regulatory guideline, is based on the positive data obtained in a clinical trial conducted in collaboration with the NIH, the results of which were presented at the 3rd International Friedreich's Ataxia Scientific Conference in Bethesda, Maryland, from November 10 to 12, 2006. Santhera believes that SNT-MC17/idebenone has the potential to be granted European marketing approval for the treatment of FRDA in the second half of 2008, approximately half a year earlier than originally communicated.

 

Despite the early filing strategy, Santhera will continue its ongoing Phase III clinical trial with SNT-MC17/idebenone in Europe to collect additional safety and efficacy data particularly for the high dose in a wider population of FRDA patients. The Company will amend the study protocol based on the findings of the NIH study and will focus more on evaluating the benefits of SNT-MC17/idebenone on the neurological aspects of FRDA. Consequently, cardiomyopathy will be dropped as an inclusion criterion for the study. Submission for Ethics approval of the amendments to the protocol is expected shortly.

 

Klaus Schollmeier, Santhera's CEO, commenting on today's announcement said: "If everything goes according to plan with our revised regulatory strategy, the chances are good that our marketing partner Takeda could launch our first product on the market in Europe in the second half of 2008. This is good news both for Santhera and for FRDA patients who are in great need of the first approved treatment for this devastating disease. I am glad that we have the full support for our accelerated regulatory strategy from Takeda, our European marketing partner for SNT-MC17/idebenone in Friedreich's Ataxia."

 

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Note to Editor

 

For more details regarding the EU "Guideline on Clinical Trials in Small Populations", see www.emea.eu.int/pdfs/human/ewp/8356105en.pdf. For an update on the Company's development strategy in the US, please refer to today's news release "Santhera Updates Development Strategy for SNT-MC17/idebenone in Friedreich's Ataxia in the US".

 

 

Santhera Pharmaceuticals (SWX:SANN) 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 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 compound's owner. The most advanced program, SNT-MC17/idebenone in FRDA, is in Phase III clinical development while the other clinical programs are in Phase II. For further information, please visit www.santhera.com.

 

For Further Information, Contact

 

Santhera Pharmaceuticals

Klaus Schollmeier, Chief Executive Officer

phone: +41 (0)61 906 89 52

 

Barbara Heller, Chief Financial Officer

phone: +41 (0)61 906 89 54

 

Thomas Staffelbach, VP Public & Investor Relations

phone: +41 (0)61 906 89 47

 

Media Contacts: Citigate Dewe Rogerson

David Dible

phone: +44 207 638 95 71

 

 

This news release is not and under no circumstances is to be construed as a solicitation, offer, or recommendation, to buy or sell securities issued by Santhera. Santhera makes no representation (either express or implied) that the information and opinions expressed in this news release are accurate, complete or up to date. Santhera disclaims, without limitation, all liability for any loss or damage of any kind, including any direct, indirect or consequential damages, which might be incurred in connection with the information contained in this news release.

 

Forward-looking statements and other information contained in this release involve risks and uncertainties. Such statements reflect the current views, intentions and estimates of the Company. They are based on assumptions that may be inaccurate. Results could differ materially from those anticipated. Certain of these forward-looking statements can be identified by the use of forward-looking terminology such as "believe", "expect", "may", "are expected to", "will", "will continue", "should", "would be", "seek" or "anticipate" or by discussions of strategy, plans or intentions. Furthermore, the Company does not assume any obligation to update these forward-looking statements.