Swiss specialty pharma company Santhera Pharmaceuticals announces opening of U.S. office in Burlington, MA.
Santhera Pharmaceuticals, a Swiss specialty pharmaceutical company focused on the development of treatments for rare mitochondrial and neuromuscular diseases, announced that it will be establishing U.S. operations in Burlington, MA.
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 Bazemore. “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, M.D. 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, Ph.D., 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, M.D., 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.”
1 National Institutes of Health, U.S. National Library of Medicine, Genetics Home Reference, Duchenne and Becker muscular dystrophy, https://ghr.nlm.nih.gov/condition/duchenne-and-becker-muscular-dystrophy#statistics
(Source: Business Wire)