Santhera Receives FDA Grant in Support of its Ongoing Phase I Trial with Omigapil in Congenital Muscular Dystrophy
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Santhera Pharmaceuticals Holding AG /
Santhera Receives FDA Grant in Support of its Ongoing Phase I Trial with
Omigapil in Congenital Muscular Dystrophy
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Liestal, Switzerland, August 30, 2016 - Santhera Pharmaceuticals (SIX: SANN)
announces that the Office of Orphan Products Development (OOPD) at the US Food
and Drug Administration (FDA) has granted Santhera an award of USD 246,000 in
support of its ongoing Phase I trial with omigapil (CALLISTO) in patients with
congenital muscular dystrophy (CMD). Santhera is conducting CALLISTO in
collaboration with the US National Institutes of Health (NIH). The FDA awards
grants through the Orphan Products Grants Program to support the clinical
development of products for use in rare diseases where no current therapy
exists.
"We are delighted that the FDA has awarded us this prestigious and highly
competitive grant in support of our CALLISTO trial in CMD," commented Thomas
Meier, PhD, CEO of Santhera. "With this award the FDA emphasizes the need for a
therapy for CMD and the contribution of the CALLISTO trial to the development of
an effective treatment. After Raxone, omigapil is our second pipeline product
which further demonstrates our dedication to developing effective medicines for
the treatment of mitochondrial and neuromuscular diseases."
"As the leading organization representing the interests of patients with CMD, we
are very pleased that the FDA recognizes the need to advance medical research in
this orphan disease. With currently no effective treatment available for
patients, the CALLISTO trial offers hope to patients with this devastating
disease," added Patrick May, President of CureCMD.
Orphan Products Grants are intended for clinical studies evaluating the safety
and/or effectiveness of products that could either result in, or substantially
contribute to, market approval of these products. Santhera, in collaboration
with the US National Institutes of Health (NIH), is currently conducting
CALLISTO under the leadership of Prof. Carsten Bönnemann and Dr. Reghan Foley at
the National Institute of Neurological Disorders and Stroke (NINDS) in Bethesda,
Maryland. CALLISTO assesses the pharmacokinetics, safety and tolerability of
omigapil in ambulatory and non-ambulatory children affected by either of two
subtypes of CMD (COL6-RD or LAMA2-RD). The study is expected to be completed in
the first half of 2017. The CALLISTO trial was previously supported by a public-
private partnership including two patient organizations, the US-based Cure CMD
and the Swiss Foundation for Research on Muscle Diseases, and EndoStem, an EU
7th Framework program. More details on the study are available from
www.clinicaltrials.gov (Identifier NCT01805024).
In May 2016, Santhera received Fast Track Designation from the FDA for omigapil
for the treatment of CMD. Omigapil was previously granted Orphan Drug
Designation for CMD in both the EU and the US.
About Congenital Muscular Dystrophy
Congenital muscular dystrophy (CMD) is a heterogeneous and clinically distinct
group of inherited neuromuscular diseases including the subtypes LAMA2-RD and
COL6-RD and manifests with an early onset of symptoms that may include weakness,
contractures, elevated creatine kinase levels, dystrophic changes on muscle
histology, loss of ambulation or even inability to stand or walk, respiratory
insufficiency, feeding difficulties and early death. Severe forms can affect
newborns or young children with life-threatening progressive muscle weakness
("floppy infant syndrome"). A contributing pathway and factor in disease burden
and muscle fibrosis is apoptosis through a cascade of cellular events involving
the glyceraldehyde-3-phosphate dehydrogenase (GAPDH) pathways for transcription
of pro-apoptotic genes. No pharmacological therapy is currently available or in
advanced clinical development. Treatment options are limited to respiratory
support and orthopedic surgery for scoliosis as well as supplementary nutrition
to avoid malnutrition.
About Omigapil
Omigapil is a deprenyl-analog with anti-apoptotic properties, originally
developed by Novartis. Santhera obtained an exclusive, worldwide license for
omigapil for the development in congenital muscular dystrophy (CMD). Omigapil
binds to glyceraldehyde-3-phosphate dehydrogenase (GAPDH) and this interaction
leads to its inhibition and is thought to underlie the compound's observed anti-
apoptotic effect. This mechanism of action provides the rationale for the
clinical development of omigapil in CMD and anti-apoptotic effects were
demonstrated in animal models of CMD where omigapil inhibited cell death and
reduced body weight loss and skeletal deformation, while increasing locomotive
activity and protecting from early mortality.
About CureCMD
Cure CMD's mission is to bring research, treatments and in the future, a cure
for congenital muscular dystrophy. Cure CMD will achieve this mission by working
globally together with dedicated parent, government and research advocates. By
focusing on this mission, Cure CMD will find and fund high potential research
and clinical trials. Success will be determined by clinical applications that
improve the lives of those afflicted with CMD's. For further information, please
visit www.curecmd.org.
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(®) 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. In collaboration with the US 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 www.santhera.com.
Raxone® is a trademark of Santhera Pharmaceuticals.
For further information, contact:
Thomas Meier, PhD, Chief Executive Officer Christoph Rentsch, Chief
Financial Officer
Phone +41 61 906 89 64 Phone
+41 61 906 89 65
thomas.meier(at)santhera.com
christoph.rentsch(at)santhera.com
Daniel Piller, Head Communications
Phone +41 61 906 89 26
daniel.piller(at)santhera.com
US investor contact US Public Relations
contact
Hans Vitzthum, LifeSci Advisors, LLC Deanne Eagle, Planet
Communications
Phone +1 212 915 2568 Phone
+1 917 837 5866
hans(at)lifesciadvisors.com
deanne(at)planetcommunications.nyc
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.
# # #
Press Release FDA Grant Omigapil:
http://hugin.info/137261/R/2038077/759510.pdf
This announcement is distributed by Nasdaq Corporate Solutions on behalf of Nasdaq Corporate Solutions clients.
The issuer of this announcement warrants that they are solely responsible for the content, accuracy and originality of the information contained therein.
Source: Santhera Pharmaceuticals Holding AG via GlobeNewswire
Unternehmensinformation / Kurzprofil:
Datum: 30.08.2016 - 07:00 Uhr
Sprache: Deutsch
News-ID 491650
Anzahl Zeichen: 9488
contact information:
Town:
Liestal
Kategorie:
Business News
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