Santhera Reports Encouraging, First Data from Phase IIa Clinical Trial with SNT-MC17 in Duchenne Muscular Dystrophy
LIESTAL, Switzerland, Oct. 31, 2007-Santhera Pharmaceuticals
(SWX:SANN), a Swiss specialty pharmaceutical company focused on
neuromuscular diseases, announces today positive, first results
from a 12 month Phase IIa clinical trial with SNT-MC17 (INN:
idebenone) in Duchenne Muscular Dystrophy (DMD) as measured by
cardiac and respiratory parameters. Santhera therefore is committed
to further clinical development of SNT-MC17 in DMD.
This exploratory Phase IIa trial was a 12-month double-blind,
randomized, placebo-controlled study conducted at the University of
Leuven, Belgium. In total 21 DMD patients between the age of 8 and
16 years were enrolled to assess the efficacy and tolerability of
one dose level of SNT-MC17 (450 mg/day) compared to placebo.
Thirteen patients were receiving SNT-MC17 while 8 patients were
randomized to the placebo group. There were no drop-outs in the
study and the compliance was very good. Importantly, there was no
difference in the safety and tolerability of SNT-MC17 compared to
placebo underlining again the excellent safety profile of SNT-MC17
also in this pediatric population.
The primary objective was to assess whether SNT-MC17 improves or
slows the decline in cardiac function in DMD patients, applying a
comprehensive echocardiographic approach that included cardiac
tissue Doppler and strain rate imaging technology. The primary
endpoint was an assessment of the change in contractility of the
region of the heart muscle that is affected early and most severely
in DMD patients, measured by the peak systolic radial strain of the
left ventricular inferolateral wall. After treatment for twelve
months with SNT-MC17, DMD patients showed a trend to improve on
this functional cardiac parameter compared to placebo
(P=0.098).
In addition to these data, patients on SNT-MC17 improved also on
certain respiratory parameters. Most striking and statistically
significant was the improvement of DMD patients' lung function
measured by peak flow (P=0.042). Patients treated with SNT-MC17
ameliorated on this parameter, while patients on placebo
deteriorated over the study period.
These findings are even more interesting as the majority of the
older and more affected patients happened to be randomized to the
SNT-MC17 treatment arm while the majority of the younger, less
affected patients were randomized to the placebo arm. Although this
led to differences in baseline which complicate interpretation of
some of the findings, it is encouraging that even patients more
severely affected appeared to show improvements.
"Despite the small sample size of this pilot study and the
resulting limitations, we saw several interesting efficacy trends
with SNT-MC17 that encourage further investigation of the drug in
DMD. Particularly encouraging are changes in cardiac and more so
respiratory parameters, as these represent life threatening
complications of this disease. We would like to thank the patients
and their parents who participated in this long-term randomized
controlled pilot trial allowing us to test for the first time
whether SNT-MC17 and its unique mode of action could potentially
provide a therapeutic benefit for this devastating disease", said
Gunnar Buyse, associate professor of child neurology at the
University of Leuven and principal investigator of the study.
"We were very fortunate for the opportunity in collaborating with
the group in Leuven for this study. This team has great clinical
and research experience in the neurological and cardiological
assessment of DMD patients. Based on the promising data the study
team has been able to analyze so far, we at Santhera are excited to
proceed with the clinical development of SNT-MC17 for DMD. In the
forthcoming weeks, the study team will further analyze the data and
we expect that the findings will provide important guidance for the
planning and execution of the subsequent clinical development",
added Thomas Meier, Chief Scientific Officer of Santhera.
The scientific rationale for this trial was based on promising data
obtained in a collaborative study between the University of Leuven
and Santhera. In a well established mouse model for DMD, it was
demonstrated that early initiated and long-term administration of
SNT-MC17 showed cardioprotective efficacy and improved exercise
performance. These data were presented at the Annual Meeting of the
American Academy of Neurology in Boston, USA, earlier this
year.
Following further analyses of the data obtained in the Phase IIa
trial reported today, Santhera intends to seek protocol advice from
the European Medicines Agency (EMEA) and the US Food and Drug
Administration (FDA) in preparation of the further clinical
development of SNT-MC17. The compound has already been granted
orphan drug designation in DMD in both the EU and the US. In August
2007, Santhera and Takeda Pharmaceutical Company Limited (TSE:4502)
announced an agreement under which Santhera granted exclusive
marketing rights for SNT-MC17 in DMD in the EU and in Switzerland
to Takeda.
About Duchenne Muscular Dystrophy (DMD)
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
recessive mode. DMD affects approximately 30,000 patients in the
USA, EU, and Japan and its incidence is approximately 1 in 3,500
live born males. Women can be carriers of DMD but usually exhibit
no symptoms. DMD is characterized by a complete loss of the protein
dystrophin, leading to progressive muscle weakness and wasting
through a complex cascade that involves impaired calcium
homeostasis 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 DMD patients, frequently in late teens -
early twenties.
* * *
About Santhera
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.
Conference call
At 15.00 CET / 14.00 UKT / 09:00 EST today October 29, 2007,
Santhera will host a conference call. People interested in
participating may join the teleconference facility using the
following dial-in in Switzerland +41 52 267 07 36 (PIN code
274915). The conference call will be recorded for playback and is
available one hour after the conference call ends and for 5 days
under +41 52 267 07 00 (reference 559357#).
For further information, contact
Klaus Schollmeier, Chief Executive Officer
Phone: +41 (0)61 906 89 52
klaus.schollmeier@santhera.com
Barbara Heller, Chief Financial Officer
Phone: +41 (0)61 906 89 54
barbara.heller@santhera.com
Thomas Staffelbach, VP Public & Investor Relations
Phone: +41 (0)61 906 89 47
thomas.staffelbach@santhera.com
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Posted: October 2007
