Leading medical journal publishes treatment effect of Santhera's Raxone on inspiratory function in patients with Duchenne muscular dystrophy (DMD)
(Thomson Reuters ONE) -
Santhera Pharmaceuticals Holding AG /
Leading medical journal publishes treatment effect of Santhera's Raxone on
inspiratory function in patients with Duchenne muscular dystrophy (DMD)
. Processed and transmitted by Nasdaq Corporate Solutions.
The issuer is solely responsible for the content of this announcement.
Liestal, Switzerland, September 13, 2016 - Santhera Pharmaceuticals (SIX: SANN)
announces that additional data from the pivotal phase III trial (DELOS)
demonstrating efficacy of Raxone (idebenone) on inspiratory function in patients
with DMD were published online in the medical journal Pediatric Pulmonology
(http://dx.doi.org/10.1002/ppul.23547).
The assessment of dynamic inspiratory function provides valuable information
about the degree and progression of pulmonary involvement in patients with DMD.
The data now published in Pediatric Pulmonology evaluated the effect of Raxone
on the highest inspiratory flow generated during a forced vital capacity (FVC)
maneuver (maximum inspiratory flow; V'I,max(FVC)). DMD patients in both
treatment groups of the DELOS trial (Raxone, n=31; placebo: n=33) had comparable
and abnormally low V'I,max(FVC) at baseline. During the study period,
V'I,max(FVC) further declined by -0.29 L/s (liters/second) in patients on
placebo (95% CI: -0.51, -0.08; p=0.008 at Week 52), whereas it remained stable
in patients on Raxone (change from baseline to Week 52: 0.01 L/s; 95% CI: -0.22,
0.24; p=0.950). The between-group difference demonstrated a positive treatment
effect for Raxone by 0.27 L/s (p=0.043) at Week 26 and 0.30 L/s (p=0.061) at
Week 52. In addition, during the study period, the fraction of the maximum flow
that is not used during tidal breathing, called Inspiratory Flow Reserve (IFR),
improved by 2.8% in patients receiving Raxone and worsened by -3.0% among
patients on placebo (between-group difference 5.8% at Week 52; p=0.040).
"These new data expand previously published results with Raxone from the
successful phase III DELOS trial which enrolled patients not taking concomitant
glucocorticoids," commented Thomas Meier PhD, CEO of Santhera Pharmaceuticals.
"Since its completion, the DELOS Study Group has published data demonstrating a
statistically significant and clinically relevant benefit of Raxone treatment on
expiratory and inspiratory function, a reduced risk of patients treated with
Raxone to experience bronchopulmonary complications and a reduced need for
systemic antibiotics. These results clearly demonstrate an overall benefit for
patients receiving Raxone on multiple pulmonary function outcomes, which is of
relevance for patients with DMD."
About Raxone (idebenone) in Duchenne muscular dystrophy
Duchenne muscular dystrophy (DMD) is one of the most common and devastating
types of muscle degeneration and results in rapidly progressive muscle weakness.
DMD is characterized by a loss of the protein dystrophin, leading to cell
damage, impaired calcium homeostasis, elevated oxidative stress and reduced
energy production in muscle cells. This results in progressive muscle weakness
and wasting and early morbidity and mortality due to respiratory failure.
Idebenone is a synthetic short-chain benzoquinone and a cofactor for the enzyme
NAD(P)H:quinone oxidoreductase (NQO1) capable of stimulating mitochondrial
electron transport, reducing and scavenging reactive oxygen species (ROS) and
supplementing cellular energy levels.
Following an exploratory Phase II trial (DELPHI), the safety and efficacy of
Raxone (idebenone) was investigated in the confirmatory phase III, double-blind,
placebo-controlled DELOS trial. DELOS randomized 64 patients, not taking
concomitant glucocorticoids, to receive either Raxone (900 mg/day) or matching
placebo. The trial met its primary endpoint and demonstrated that Raxone can
slow the loss of respiratory function and reduces bronchopulmonary
complications. The positive outcome of the Phase III DELOS study was so far
published by Buyse et al., The Lancet 2015, 385:1748-1757 and McDonald et al.,
Neuromuscular Disorders 2016, 26: 473-480.
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 Christoph Rentsch, Chief Financial
Officer 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 press release 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 Pediatric Pulmonology:
http://hugin.info/137261/R/2041648/761884.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: 13.09.2016 - 07:00 Uhr
Sprache: Deutsch
News-ID 494285
Anzahl Zeichen: 7797
contact information:
Town:
Liestal
Kategorie:
Business News
Diese Pressemitteilung wurde bisher 380 mal aufgerufen.
Die Pressemitteilung mit dem Titel:
"Leading medical journal publishes treatment effect of Santhera's Raxone on inspiratory function in patients with Duchenne muscular dystrophy (DMD)"
steht unter der journalistisch-redaktionellen Verantwortung von
Santhera Pharmaceuticals Holding AG (Nachricht senden)
Beachten Sie bitte die weiteren Informationen zum Haftungsauschluß (gemäß TMG - TeleMedianGesetz) und dem Datenschutz (gemäß der DSGVO).
