LIESTAL, Switzerland I April 16, 2013 I Santhera Pharmaceuticals (SIX: SANN) announced today that the Phase III DELOS study of orally administered Catena® in patients with Duchenne Muscular Dystrophy (DMD) successfully passed a planned futility and safety analysis. The independent Data Safety Monitoring Board (DSMB) for DELOS informed Santhera that the study has a reasonable chance of achieving its primary endpoint for improving or delaying the loss of respiratory function in Duchenne patients not using corticosteroids and since no safety issues were detected, recommended that the study should continue as planned.
“I am very enthusiastic about this news, which is important for the DMD community as it indicates that Catena® eventually may evolve into a real treatment for all patients with this devastating disease. Importantly it suggests that the DELOS data are consistent with the outcome of the Phase II DELPHI study, which showed that treatment with Catena® resulted in a significant increase in peak expiratory flow and may improve early signs of respiratory weakness and dysfunction” commented Gunnar Buyse M.D., Ph.D., Professor of Child Neurology at the University Hospitals Leuven (Belgium) and Principal Investigator for the DELOS study. “Some promising therapies for DMD have failed to demonstrate clinical benefit in late-stage development, so it is encouraging to see that we now may be on track to clear this hurdle for this patient group”.
“This futility analysis is particularly relevant since it included primary efficacy variable data after at least 6 months of treatment from 80%, and final outcome data after 12 months of treatment from over 60% of the planned total number of patients in the corticosteroid non-using cohort. This increases the probability of a positive outcome in the final analysis of this cohort”, said Dr. Nick Coppard, Santhera’s Senior VP Development. “Since DELOS is not restricted to patients with a specific mutational or ambulatory status, the potential benefit in respiratory function may extend to all Duchenne patients”.
Santhera has previously announced that it explores strategic and financing options including product licensing and the possibility of a merger or acquisition. The Company’s ability to complete the DELOS program is contingent upon the availability of the financial resources required. To this end, Santhera has started the process to seek a development or licensing partner for Catena® in DMD outside Europe, where commercialization rights have been licensed to Takeda.
The European Medicines Agency (EMA) and US Food and Drug Administration (FDA) have both granted orphan drug status to Santhera’s program with Catena® in DMD in the European Union and the United States of America, respectively.
About the DELOS study
DELOS (DuchEnne Muscular Dystrophy Long-term IdebenOne Study) is a Phase III randomized, double-blind study in patients aged 10-18 years with DMD of any mutational etiolology, ambulatory or non-ambulatory, recruited in Europe and in North America. The study is designed to assess the efficacy of orally administered Catena® (900 mg/day), compared to placebo, in improving or delaying the loss of respiratory function in Duchenne patients. DELOS uses the change from baseline to 12 months in per cent predicted Peak Expiratory Flow (PEF) as the primary endpoint. The study design was agreed with both the EMA and the FDA. The study plans to sequentially enroll two cohorts of patients, 60 corticosteroid non-using and 200 corticosteroid using patients, to allow these sub-groups to be independently powered for efficacy and analyzed separately in the final primary endpoint analysis. The study is powered to detect a 10.3% difference in the change from baseline to 12 months in per cent predicted PEF between the Catena® and placebo groups. As was agreed with both the US and European regulatory agencies, DELOS is intended to provide pivotal efficacy data for regulatory filings for Catena® in the treatment of patients with DMD in these territories.
About Duchenne Muscular Dystrophy
DMD is the most common and a devastating type of muscular degeneration and results in rapidly progressive muscle weakness. It is a genetic, degenerative disease that is inherited in an X-linked mode. DMD is characterized by a loss of the protein dystrophin, leading to progressive muscle weakness and wasting through a complex cascade that involves impaired calcium homeostasis, mitochondrial dysfunction and oxidative stress. The average age of onset is between 3 and 5 years of age with a loss of ambulation in teenage patients. Dilated cardiomyopathy and respiratory failure are commonly associated with this chronic disease leading to early morbidity and mortality in Duchenne patients.
* * *
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 neuromuscular and mitochondrial diseases, areas of high unmet medical need with no current therapies. For further information, please visit www.santhera.com.
SOURCE: Santhera
Post Views: 117
LIESTAL, Switzerland I April 16, 2013 I Santhera Pharmaceuticals (SIX: SANN) announced today that the Phase III DELOS study of orally administered Catena® in patients with Duchenne Muscular Dystrophy (DMD) successfully passed a planned futility and safety analysis. The independent Data Safety Monitoring Board (DSMB) for DELOS informed Santhera that the study has a reasonable chance of achieving its primary endpoint for improving or delaying the loss of respiratory function in Duchenne patients not using corticosteroids and since no safety issues were detected, recommended that the study should continue as planned.
“I am very enthusiastic about this news, which is important for the DMD community as it indicates that Catena® eventually may evolve into a real treatment for all patients with this devastating disease. Importantly it suggests that the DELOS data are consistent with the outcome of the Phase II DELPHI study, which showed that treatment with Catena® resulted in a significant increase in peak expiratory flow and may improve early signs of respiratory weakness and dysfunction” commented Gunnar Buyse M.D., Ph.D., Professor of Child Neurology at the University Hospitals Leuven (Belgium) and Principal Investigator for the DELOS study. “Some promising therapies for DMD have failed to demonstrate clinical benefit in late-stage development, so it is encouraging to see that we now may be on track to clear this hurdle for this patient group”.
“This futility analysis is particularly relevant since it included primary efficacy variable data after at least 6 months of treatment from 80%, and final outcome data after 12 months of treatment from over 60% of the planned total number of patients in the corticosteroid non-using cohort. This increases the probability of a positive outcome in the final analysis of this cohort”, said Dr. Nick Coppard, Santhera’s Senior VP Development. “Since DELOS is not restricted to patients with a specific mutational or ambulatory status, the potential benefit in respiratory function may extend to all Duchenne patients”.
Santhera has previously announced that it explores strategic and financing options including product licensing and the possibility of a merger or acquisition. The Company’s ability to complete the DELOS program is contingent upon the availability of the financial resources required. To this end, Santhera has started the process to seek a development or licensing partner for Catena® in DMD outside Europe, where commercialization rights have been licensed to Takeda.
The European Medicines Agency (EMA) and US Food and Drug Administration (FDA) have both granted orphan drug status to Santhera’s program with Catena® in DMD in the European Union and the United States of America, respectively.
About the DELOS study
DELOS (DuchEnne Muscular Dystrophy Long-term IdebenOne Study) is a Phase III randomized, double-blind study in patients aged 10-18 years with DMD of any mutational etiolology, ambulatory or non-ambulatory, recruited in Europe and in North America. The study is designed to assess the efficacy of orally administered Catena® (900 mg/day), compared to placebo, in improving or delaying the loss of respiratory function in Duchenne patients. DELOS uses the change from baseline to 12 months in per cent predicted Peak Expiratory Flow (PEF) as the primary endpoint. The study design was agreed with both the EMA and the FDA. The study plans to sequentially enroll two cohorts of patients, 60 corticosteroid non-using and 200 corticosteroid using patients, to allow these sub-groups to be independently powered for efficacy and analyzed separately in the final primary endpoint analysis. The study is powered to detect a 10.3% difference in the change from baseline to 12 months in per cent predicted PEF between the Catena® and placebo groups. As was agreed with both the US and European regulatory agencies, DELOS is intended to provide pivotal efficacy data for regulatory filings for Catena® in the treatment of patients with DMD in these territories.
About Duchenne Muscular Dystrophy
DMD is the most common and a devastating type of muscular degeneration and results in rapidly progressive muscle weakness. It is a genetic, degenerative disease that is inherited in an X-linked mode. DMD is characterized by a loss of the protein dystrophin, leading to progressive muscle weakness and wasting through a complex cascade that involves impaired calcium homeostasis, mitochondrial dysfunction and oxidative stress. The average age of onset is between 3 and 5 years of age with a loss of ambulation in teenage patients. Dilated cardiomyopathy and respiratory failure are commonly associated with this chronic disease leading to early morbidity and mortality in Duchenne patients.
* * *
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 neuromuscular and mitochondrial diseases, areas of high unmet medical need with no current therapies. For further information, please visit www.santhera.com.
SOURCE: Santhera
Post Views: 117
