Santhera Showcases Potential and Progress of its Clinical Development Pipeline

 

Commenting on the progress the Company has made since its inception in 2004, Klaus Schollmeier, Chief Executive Officer of Santhera, says: "During this relatively short period of time, we were able to build a very promising mid- to late-stage development pipeline for orphan, neuromuscular diseases. We basically started with an idea and one early stage clinical compound in one single neuromuscular indication. Today, our pipeline is comprised of three distinct molecules in five indications. Meanwhile, the vision from 2004 begins to turn into reality as our lead compound SNT-MC17 for the treatment of Friedreich's Ataxia is currently under review for market authorization in the EU and Switzerland and soon in Canada."

 

Thomas Meier, Chief Scientific Officer of Santhera, adds: "We are building on a clear strategy in R&D. Focusing on our key area of expertise, we strive to identify novel compounds from within our research team or from external sources for development in neuromuscular diseases. Furthermore, we are constantly re-profiling existing compounds for their potential therapeutic use in neuromuscular indications. By leveraging our internal know-how of compounds, diseases and medical needs, we intend to fully optimize the medical and commercial potential of our product candidates."

 

During the R&D Day, Santhera will also be presenting the following update on its clinical development portfolio:

 

SNT-MC17 (INN: idebenone) in Friedreich's Ataxia (FRDA): The product's filing for marketing authorization is currently under review in the EU, in Switzerland, and in due course in Canada. In the US, preparations to initiate patient recruitment for the 6 month pivotal trial are almost completed at the two study centers in Los Angeles and Philadelphia. Santhera is currently waiting for the final approval from the respective ethic boards. Results from the study are expected for late 2008 or early 2009. In Europe, recruitment for the on-going 12-month Phase III trial recently passed the 50 percent recruitment level. Results of this trial are expected for the second-half of 2009. Meanwhile, the first completers of this European trial enrolled into the open-label extension study on high dose of SNT-MC17.

 

SNT-MC17 in Duchenne Muscular Dystrophy (DMD): Recently reported positive, first results from a 12 month Phase II clinical trial show a trend to improve on cardiac parameters after treatment with SNT-MC17. Furthermore, DMD patients receiving SNT-MC17 improved in their lung function. Most striking and statistically significant was the improvement upon SNT-MC17 in peak flow while patients on placebo deteriorated over the study period. The study again confirmed the excellent safety and tolerability of SNT-MC17. Santhera will seek protocol advice from the regulatory authorities and expects to start the pivotal clinical development program in the second-half of 2008. Meanwhile, the Company is preparing a long-term, open-label extension study for the participants of the recent trial to collect additional efficacy data.

 

SNT-MC17 in Leber's Hereditary Optic Neuropathy (LHON): A Phase II trial to assess the efficacy of one dose of SNT-MC17 (900 mg/day) in the treatment and prevention of vision loss against placebo is on-going at two study centers in the Munich and Newcastle. The study will enroll 84 patients with LHON for treatment duration of 6 months. Results of this proof-of-concept study are expected for the second-half of 2009.

 

JP-1730 (INN: fipamezole) in Dyskinesia in Parkinson's Disease (DPD): Santhera and its collaboration partner Juvantia Pharma recently enrolled the first patient into their Phase IIb clinical trial. The study is designed to confirm the dual efficacy of JP-1730, the capacity to reduce dyskinetic movements and to extend the anti-parkinsonian action of levodopa, observed previously in a successful proof-of-concept trial. The Phase IIb study evaluates the safety and efficacy of three escalating doses of JP-1730 compared to placebo over a treatment period of 28 days. A total of 152 patients with advanced PD will be enrolled in 30 study centers in the US and India. Results are expected for the second half of 2008.

 

SNT-317 (INN: omigapil) in Congenital Muscular Dystrophies (CMD): In-licensed from Novartis in June 2007, Santhera started the production of study medication and initiated a juvenile tox program requested for the intended use in pediatric patients. Santhera expects to initiate the clinical development program under Novartis' open Investigational New Drug (IND) status in the second half of 2008. The Company currently estimates the market for a pharmaceutical product that could treat CMD to exceed EUR 100 million, based on disease prevalence, severity of the disease and the expected reimbursement levels for such a product.

 

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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. 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 five clinical-stage development programs, three of which are investigating its lead compound, SNT-MC17 (INN: idebenone), for the treatment of Friedreich's Ataxia (FRDA), Duchenne Muscular Dystrophy (DMD) and Leber's Hereditary Optic Neuropathy (LHON). Another clinical program is investigating JP-1730 (INN: fipamezole) for the treatment of Dyskinesia in Parkinson's Disease (DPD) in cooperation with Juvantia, the compound's owner. The fifth program comprises SNT-317 (INN: omigapil) in Congenital Muscular Dystrophies (CMD), a compound in-licensed from Novartis. For the most advanced program, SNT-MC17 in FRDA, the Company has applied for marketing authorization in Europe and will be submitted shortly in Canada. The compound is also in Phase III clinical development for FRDA in the US while the other clinical programs are in Phase II. For further information, please visit www.santhera.com.

 

 

 

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

 

 

 

This news release is not and under no circumstances to be construed as a solicitation, offer, or recommendation, to buy or sell securities issued by Santhera Pharmaceuticals Holding AG. Santhera Pharmaceuticals Holding AG 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 Pharmaceuticals Holding AG 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.

 

This news release expressly or implicitly contains certain forward-looking statements concerning Santhera Pharmaceuticals Holding AG and its business. 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. Such statements involve certain known and unknown risks, uncertainties and other factors, which could cause the actual results, financial condition, performance or achievements of Santhera Pharmaceuticals Holding AG to be materially different from any expected results, performance or achievements expressed or implied by such forward-looking statements. There can be no guarantee that any of the research and/or development projects described will succeed or that any new products or indications will be brought to market. Similarly, there can be no guarantee that Santhera Pharmaceuticals Holding AG or any future product or indication will achieve any particular level of revenue. In particular, management's expectations could be affected by, among other things, uncertainties involved in the development of new pharmaceutical products, including unexpected preclinical and clinical trial results; unexpected regulatory actions or delays or government regulation generally; the Company's ability to obtain or maintain patent or other proprietary intellectual property protection; competition in general; government, industry, and general public pricing and other political pressures. Santhera Pharmaceuticals Holding AG is providing the information in this new release as of the date of the publication, and does not undertake any obligation to update any forward-looking statements contained herein as a result of new information, future events or otherwise.