Independent DSMB Recommends Continuation of Santhera's Phase III Trial of Catena® in Duchenne Muscular Dystrophy
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Santhera Pharmaceuticals Holding AG /
Independent DSMB Recommends Continuation of Santhera's Phase III Trial of
Catena® in Duchenne Muscular Dystrophy
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The issuer is solely responsible for the content of this announcement.
Liestal, Switzerland, April 16, 2013 - 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.
Catena(®) is a trademark of Santhera Pharmaceuticals.
For further information, contact
Thomas Meier, Chief Executive Officer
Phone: +41 61 906 89 64
thomas.meier(at)santhera.com
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. 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.
News release DELOS:
http://hugin.info/137261/R/1693291/556555.pdf
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Source: Santhera Pharmaceuticals Holding AG via Thomson Reuters ONE
[HUG#1693291]
Unternehmensinformation / Kurzprofil:
Datum: 16.04.2013 - 07:15 Uhr
Sprache: Deutsch
News-ID 249240
Anzahl Zeichen: 7793
contact information:
Town:
Liestal
Kategorie:
Business News
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