Swiss Specialty Pharma Company Santhera Announces Opening Of U.S. Office In Burlington, MA

Published: Feb 24, 2017

BURLINGTON, Mass. & LIESTAL, Switzerland--(BUSINESS WIRE)--Santhera Pharmaceuticals, a Swiss specialty pharmaceutical company focused on the development of innovative treatments for rare mitochondrial and neuromuscular diseases, announced that it will be establishing U.S. operations in Burlington, Mass.

“A patient and caregiver survey conducted by Parent Project Muscular Dystrophy clearly demonstrated that the DMD community highly values treatment options for respiratory complications.”

Santhera Establishing U.S. Operations

Santhera’s U.S. operations are being led by Todd Bazemore, Chief Operating Officer of Santhera Pharmaceuticals (USA), Inc. The U.S. operations will initially be staffed to focus on regulatory and clinical operations support, medical affairs, patient advocacy liaison and commercial strategy.

“A strong presence in the U.S. is an important next step in our mission to advance novel treatments for Duchenne muscular dystrophy and other rare neuromuscular and mitochondrial diseases,” said Todd Bazemore, COO of Santhera U.S. “We chose the metropolitan Boston area for our U.S. operations because it is one of the main centers for the biotech and pharma business globally with a unique confluence of academia, life science companies, and clinical expertise.”

Idebenone in DMD

Duchenne muscular dystrophy (DMD) is one of the most common and devastating types of muscle degeneration and leads to progressive muscle weakness starting at an early age. DMD is a genetic, degenerative disease that occurs almost exclusively in males with an incidence of up to 1 in 3,500 live male births worldwide.1

DMD is characterized by a loss of the protein dystrophin, ultimately resulting in muscle weakness and wasting and early morbidity and mortality due to respiratory failure. In DMD, the progressive respiratory muscle weakness results in respiratory symptoms such as inability to cough and clear mucus, frequent airway infections, sleep-disordered breathing and the need for assisted ventilation. Addressing the progressive loss of respiratory function is a hallmark in DMD and an urgent unmet medical need for these patients.

In a Phase III clinical trial (DELOS) conducted in DMD patients not taking glucocorticoids, Santhera’s lead therapeutic candidate, idebenone, demonstrated a clinically significant slowing in the loss of respiratory function, regardless of the underlying genetic mutation. Santhera is now enrolling patients in a new Phase III trial (SIDEROS) in 60 centers across the United States and Europe to confirm the efficacy of idebenone in 266 patients who are currently taking a stable dose of glucocorticoids. The first patient was enrolled at the University of Kansas Medical Center (KUMC), Department of Neurology in September with Jeffrey Statland, MD. In the U.S., the SIDEROS trial will be conducted in 24 study centers with the goal of having this trial fully enrolled by end of 2017. If successful, this study will provide data that supports the use of idebenone in all DMD patients experiencing respiratory decline irrespective of their glucocorticoid use or genetic mutation.

“Idebenone is generating a significant interest among researchers, physicians and the patient community. In slowing the loss of respiratory function in all patients irrespective of their mutational status, this drug candidate has the potential to expand the current treatment paradigm for DMD,” said Thomas Meier, PhD, CEO of Santhera. “Our presence here in the U.S. will enable us to expand our engagement with physicians, their patients, and families as the SIDEROS trial moves forward while we continue our efforts to find a regulatory path for early approval.”

“There is high interest from investigators and the patient community in therapies that hold the promise of maintaining respiratory function in patients with DMD,” added Oscar Henry Mayer, MD, Medical Director of the Pulmonary Function Testing Laboratory at the Children’s Hospital of Philadelphia and Lead SIDEROS Investigator for the U.S. “A patient and caregiver survey conducted by Parent Project Muscular Dystrophy clearly demonstrated that the DMD community highly values treatment options for respiratory complications.”

About the SIDEROS Trial

SIDEROS is a phase III, double-blind, randomized, placebo-controlled trial with idebenone in approximately 266 DMD patients receiving concomitant glucocorticoids. Patients with declining respiratory function on any stable glucocorticoid treatment scheme and irrespective of the underlying dystrophin mutation or ambulatory status will be eligible. Study participants will receive either idebenone (900 mg/day; given as 2 tablets 3 times a day with meals) or placebo for 78 weeks (18 months). The primary endpoint of the trial is the change from baseline to week 78 in forced vital capacity % predicted (FVC%p). Secondary endpoints include changes from baseline in % predicted peak expiratory flow (PEF%p), time to first 10% decline in FVC and change from baseline in inspiratory flow reserve. Patients completing the trial will be offered the opportunity to enroll in an open label extension study in which all patients receive idebenone.

The study will be conducted at about 60 centers in the United States and Europe. Patients wishing to enroll in the study should contact their neuromuscular clinic physician. Further information about the study is available under

About Idebenone in Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is one of the most common and devastating types of muscle degeneration and results in rapidly progressive muscle weakness. DMD is characterized by a loss of the protein dystrophin, leading to cell damage, impaired calcium homeostasis, elevated oxidative stress and reduced energy production in muscle cells. This results in progressive muscle weakness and wasting and early morbidity and mortality due to respiratory failure.

Idebenone is a synthetic short-chain benzoquinone and a cofactor for the enzyme NAD(P)H:quinone oxidoreductase (NQO1) capable of stimulating mitochondrial electron transport, reducing and scavenging reactive oxygen species (ROS) and supplementing cellular energy levels.

DELOS was a phase III, double-blind, placebo-controlled trial which randomized 64 patients, not taking concomitant glucocorticoids, to receive either idebenone (900 mg/day) or matching placebo. The trial met its primary endpoint and demonstrated that idebenone can slow the loss of respiratory function and reduces bronchopulmonary complications.

The statistically significant and clinically relevant outcomes of the phase III DELOS study were published: Buyse et al., The Lancet 2015, 385:1748-1757; McDonald et al., Neuromuscular Disorders 2016, 26: 473–480 and Buyse et al., Pediatric Pulmonology 2016:

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) and the Swiss regulatory authorities Swissmedic are currently assessing a Marketing Authorization Application (MAA) for idebenone under the name RAXONE® in DMD patients with respiratory function decline who are not taking concomitant glucocorticoids. The indication would include patients who previously were treated with glucocorticoids or in whom glucocorticoid treatment is not desired, not tolerated or is contraindicated.

About Santhera

Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative pharmaceutical products for the treatment of orphan mitochondrial and neuromuscular diseases. Santhera's lead product RAXONE® (idebenone) is authorized in the European Union, Norway, Iceland and Liechtenstein for the treatment of Leber's hereditary optic neuropathy (LHON). For Duchenne muscular dystrophy (DMD), the second indication for RAXONE®, Santhera has filed a Marketing Authorization Application (MAA) in the European Union and Switzerland. In collaboration with the U.S. National Institute of Neurological Disorders and Stroke (NINDS) Santhera is developing RAXONE® in a third indication, primary progressive multiple sclerosis (PPMS), and omigapil for congenital muscular dystrophy (CMD), all areas of high unmet medical need. For further information, please visit the Company's website

RAXONE® is a trademark of Santhera Pharmaceuticals.

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. These statements could be affected by, among other things, risks and factors referred to in the Risk Factors section on Santhera’s web site ( 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.

1 National Institutes of Health, U.S. National Library of Medicine, Genetics Home Reference, Duchenne and Becker muscular dystrophy,

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