Liestal, Switzerland

Santhera Pharmaceuticals Holding

santhera.com
Liestal, Switzerland
SEARCH FILTERS
Time filter
Source Type

Liestal, Switzerland, May 19, 2017 - Santhera Pharmaceuticals, a specialty pharmaceutical company focused on the development of innovative treatments for rare mitochondrial and neuromuscular diseases, announces an updated timeline for the ongoing assessment by the Committee for Medicinal Products for Human Use (CHMP) of its extension application for Raxone® (idebenone) in Duchenne muscular dystrophy (DMD). "Santhera is in ongoing, constructive discussions with the CHMP, and we are now expecting to receive a request for supplementary information to further support the clinical relevance of our data. We are working closely with the CHMP to conclude the application process and anticipate an opinion in Q3 2017," said Thomas Meier, PhD, CEO of Santhera. The application was filed as a Type II Variation of the existing marketing authorization and is based on data from Santhera's phase II (DELPHI) study and the successful pivotal phase III (DELOS) study, the latter in patients not taking concomitant glucocorticoids. This data demonstrated a statistically significant and clinically relevant benefit of Raxone in preserving respiratory function compared to placebo. This result is further substantiated by a natural history study, which shows that the benefits observed in the group treated with Raxone would not have been expected from the natural course of the disease. The intended indication for Raxone is to slow the loss of respiratory function in patients with DMD who are currently not taking glucocorticoids. The indication would include patients who were previously treated with glucocorticoids or in whom glucocorticoid treatment is not desired, not tolerated, or is contraindicated. About Duchenne Muscular Dystrophy DMD is one of the most common and devastating types of muscle degeneration and leads to progressive muscle weakness starting at an early age. DMD is a genetic, degenerative disease that occurs almost exclusively in males with an incidence of up to 1 in 3,500 live male births worldwide. About Idebenone in Duchenne Muscular Dystrophy 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, muscle wasting, 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. DELOS was a phase III, double-blind, placebo-controlled 52-week study which randomized 64 patients, not taking concomitant steroids, to receive either idebenone (900 mg/day) or matching placebo. The study met its primary endpoint, the change from baseline in Peak Expiratory Flow (PEF), which demonstrated that idebenone can slow the loss of respiratory function. Idebenone was well tolerated in the DELOS study, with overall incidence of adverse events being similar to placebo. The statistically significant and clinically relevant outcomes of the phase III DELOS study were published: Buyse et al., The Lancet 2015, 385:1748-1757; McDonald et al., Neuromuscular Disorders 2016, 26:473-480 and Buyse et al., Pediatric Pulmonology 2017, 52:580-515. The European Medicines Agency's CHMP and the Swiss regulatory authority Swissmedic are currently assessing a Marketing Authorization Application for idebenone under the name Raxone in patients with DMD with respiratory function decline who are not taking glucocorticoids. 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® (idebenone) 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), Santhera has filed a Marketing Authorization Application in the European Union and Switzerland for DMD patients with respiratory function decline who are not taking glucocorticoids. In collaboration with the U.S. 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. 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@santhera.com                                christoph.rentsch@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.


Liestal, Switzerland, May 16, 2017 - Santhera Pharmaceuticals, a specialty pharmaceutical company focused on the development of innovative treatments for rare mitochondrial and neuromuscular diseases, announces that two posters with data from the positive phase III DELOS trial in patients with Duchenne muscular dystrophy (DMD) will be presented at the 113th American Thoracic Society (ATS) International Conference in Washington D.C. Throughout the conference, which runs May 19-24, 2017, Santhera will host a clinical trial booth to inform clinicians on the new SIDEROS trial. The SIDEROS trial is a phase III, double-blind, randomized, placebo-controlled trial that evaluates the efficacy, safety and tolerability of idebenone in slowing the rate of respiratory function decline in DMD patients. Patients with reduced respiratory function on any stable glucocorticoid treatment regimen are eligible, regardless of their underlying dystrophin mutation or ambulatory status. More information is available on the new SIDEROS study website at www.siderosdmd.com. Santhera will also facilitate a non-CME symposium featuring an expert panel consisting of: Topics of the symposium include the pathophysiology of DMD and data around the importance of preserving respiratory function in DMD. "Santhera is pleased to have the opportunity to showcase our focus on research to address pulmonary function decline in DMD patients at this year's ATS conference," said Thomas Meier, PhD, CEO of Santhera. "Pulmonary function decline is a serious medical complication in patients with DMD. However, it's often overlooked, particularly in the early stage of the disease. Our placebo-controlled phase III DELOS trial in 10 to 18 year old patients with respiratory function decline and not on steroids, met its primary endpoint, demonstrating that idebenone treatment slows pulmonary function decline. Santhera is proud to be leader in this important aspect of clinical DMD research." Date: Tuesday, May 23, 6:30 - 9:30 p.m. Location: Constitution Ballroom C-E (Constitution Level 3B) Title: Duchenne Muscular Dystrophy (DMD): Importance of Preserving Pulmonary Function and Impact of Idebenone (An Investigational Treatment) in Boys with DMD Speakers: Oscar H. Mayer, MD; Erik Henricson, PhD, MPH; Hemant Sawnani, MD About: Please join us for an informative and interactive session on one of the most common and devastating types of muscle degeneration, Duchenne muscular dystrophy (DMD). This expert panel will review the pathophysiology of DMD, data from a DMD natural history study, and data around the importance of preserving pulmonary function in DMD. Date: May 22, 9:15 - 4:15 p.m. Session: B69 - WHEN KIDS CANNOT SLEEP (OR BREATHE) Title: A4125 - Correlation Between Volume- and Flow-Related Pulmonary Function Outcomes in Patients with DMD Location: Walter E. Washington Convention Center, Area F, Hall B-C Session Label: Thematic Poster Session; Poster Board Number: P676 Authors: O. H. Mayer, MD; E. Henricson, PhD; M. Leinonen, MS; J. Karafilidis, PharmD; C. McDonald, MD; G. Buyse, MD, PhD Date: May 23, 2:15 - 4:15 p.m. Session: C105 - DISORDERS OF RESPIRATORY PHYSIOLOGY AND SLEEP IN CHILDREN Title: A6875 - Effect of Idebenone on Bronchopulmonary Adverse Events and Hospitalizations in Patients with Duchenne Muscular Dystrophy (DMD) Location: Walter E. Washington Convention Center, Room 201 (South Building, Level 2) Session Label: Poster Discussion Session; Poster Board Number: 502 Authors: O. H. Mayer, MD; C. McDonald, MD; T. Meier, PhD; M. Leinonen, MS; G. Buyse, MD, PhD About the SIDEROS Trial SIDEROS is a phase III, double-blind, randomized, placebo-controlled trial with idebenone in 266 DMD patients receiving concomitant glucocorticoid steroids. Patients with declining respiratory function on any stable steroid treatment scheme and irrespective of the underlying dystrophin mutation or ambulatory status are eligible for enrolment. Study participants will receive either idebenone (900 mg/day; given as 2 tablets 3 times a day with meals) or placebo for 78 weeks (18 months). The primary endpoint of the trial is change from baseline to week 78 in forced vital capacity % predicted (FVC%p). Secondary endpoints include changes from baseline in % predicted peak expiratory flow (PEF%p), time to first 10% decline in FVC and change from baseline in inspiratory flow reserve. Patients completing the trial will be offered the opportunity to enroll in an open label extension study where all patients receive idebenone. The study will be conducted in 63 centers in the United States, Europe and Israel. Further information about the study is available under www.clinicaltrials.gov and www.siderosdmd.com. About Duchenne Muscular Dystrophy Duchenne muscular dystrophy (DMD) is one of the most common and devastating types of muscle degeneration and leads to progressive muscle weakness starting at an early age. DMD is a genetic, degenerative disease that occurs almost exclusively in males with an incidence of up to 1 in 3,500 live male births worldwide. About Idebenone in Duchenne Muscular Dystrophy 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. DELOS was a phase III, double-blind, placebo-controlled 52-week trial which randomized 64 patients, not taking concomitant steroids, to receive either idebenone (900 mg/day) or matching placebo. The trial met its primary endpoint which was a change from baseline in Peak Expiratory Flow (PEF) demonstrating that idebenone can slow the loss of respiratory function. Idebenone was well tolerated in the DELOS trial with overall incidence of adverse events being similar to placebo. Most commonly reported adverse events were: diarrhea (idebenone 25% vs. 12% for placebo), constipation (idebenone 9% vs. 18% for placebo) and abdominal pain (idebenone 9% and 9% for placebo). The statistically significant and clinically relevant outcomes of the phase III DELOS study were published: Buyse et al., The Lancet 2015, 385:1748-1757; McDonald et al., Neuromuscular Disorders 2016, 26: 473-480 and Buyse et al., Pediatric Pulmonology 2016: http://dx.doi.org/10.1002/ppul.23547. The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) and the Swiss regulatory authority Swissmedic are currently assessing a Marketing Authorization Application (MAA) for idebenone under the name RAXONE® in DMD patients with respiratory function decline who are not taking concomitant steroids. 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® (idebenone) 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), Santhera has filed a Marketing Authorization Application (MAA) in the European Union and Switzerland for DMD patients with respiratory function decline who are not taking concomitant steroids. In collaboration with the U.S. 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. 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@santhera.com                                christoph.rentsch@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.


Zu den Themen dieses Symposiums gehören die Pathophysiologie von DMD und Daten zur Bedeutung des Erhalts der Atmungsfunktion bei DMD. "Santhera freut sich über die Gelegenheit, unseren Fokus bei der Erforschung der Atmungsfunktionsverringerung bei DMD-Patienten anlässlich der diesjährigen ATS-Konferenz zu präsentieren", sagte Thomas Meier, PhD, CEO von Santhera. "Die Abnahme der Atmungsfunktion ist eine ernsthafte medizinische Komplikation bei Patienten mit DMD. Allerdings wird dies vor allem in der frühen Phase der Krankheit oft übersehen. Unsere Plazebo-kontrollierte Phase-III-DELOS-Studie bei 10 bis 18 Jahre alten Patienten mit abnehmender Atmungsfunktion welche nicht mit Steroiden behandelt wurden, erreichte den primären Endpunkt und zeigte, dass Idebenon den Atmungsfunktionsverlust verlangsamt. Santhera ist stolz darauf, in diesem wichtigen Aspekt der DMD-Forschung führend zu sein." Datum: Dienstag, 23. Mai, 18:30 - 21:30 Uhr Ort: Constitution Ballroom C-E (Constitution Level 3B) Titel: Duchenne Muscular Dystrophy (DMD): Importance of Preserving Pulmonary Function and Impact of Idebenone (An Investigational Treatment) in Boys with DMD Referenten: Oscar H. Mayer, MD; Erik Henricson, PhD, MPH; Hemant Sawnani, MD Über: Wir laden Sie ein, unsere informative und interaktive Veranstaltung zu Duchenne-Muskeldystrophie (DMD), eine der häufigsten und schwersten Arten von Muskelschwund, zu besuchen. Dieses Expertengremium wird die Pathophysiologie von DMD, Daten zum natürlichen Krankheitsverlauf von DMD sowie Daten zur Bedeutung des Erhalts der Atmungsfunktion bei DMD erörtern. Datum: 22. Mai, 9:15 - 16:15 Uhr Session: B69 - WHEN KIDS CANNOT SLEEP (OR BREATHE) Titel: A4125 - Correlation Between Volume- and Flow-Related Pulmonary Function Outcomes in Patients with DMD Ort: Walter E. Washington Convention Center, Area F, Hall B-C Bezeichnung: Thematic Poster Session; Posterwand: P676 Autoren: O. H. Mayer, MD; E. Henricson, PhD; M. Leinonen, MS; J. Karafilidis, PharmD; C. McDonald, MD; G. Buyse, MD, PhD Datum: 23. Mai, 14:15 - 16:15 Uhr Session: C105 - DISORDERS OF RESPIRATORY PHYSIOLOGY AND SLEEP IN CHILDREN Titel: A6875 - Effect of Idebenone on Bronchopulmonary Adverse Events and Hospitalizations in Patients with Duchenne Muscular Dystrophy (DMD) Ort: Walter E. Washington Convention Center, Room 201 (South Building, Level 2) Bezeichnung: Poster Discussion Session; Posterwand: 502 Autoren: O. H. Mayer, MD; C. McDonald, MD; T. Meier, PhD; M. Leinonen, MS; G. Buyse, MD, PhD Über die SIDEROS-Studie SIDEROS ist eine Phase-III, doppelblinde, randomisierte, Plazebo-kontrollierte Studie mit Idebenon bei 266 DMD-Patienten, welche gleichzeitig mit Glukokortikoiden behandelt werden. Patienten mit abnehmender Atmungsfunktion unter stabiler Behandlung mit Glukokortikoiden können, unabhängig von der zugrundeliegenden Dystrophin-Mutation oder Gehfähigkeit, in die Studie aufgenommen werden. Die Studienteilnehmer erhalten entweder Idebenon (900 mg/Tag; Einnahme von zwei Tabletten drei Mal täglich mit den Mahlzeiten) oder Plazebo während 78 Wochen (18 Monaten). Der primäre Endpunkt der Studie ist die Veränderung der forcierten Vitalkapazität (Forced Vital Capacity % predicted, FVC%p) vom Ausgangswert bis Woche 78. Sekundäre Endpunkte sind unter anderem Veränderungen der maximalen exspiratorischen Atemflussrate (Peak Expiratory Flow % predicted, PEF%p), die Dauer bis zur erstmaligen 10-prozentigen Abnahme der FVC und die Veränderung des inspiratorischen Reservevolumens vom Ausgangswert. Patienten, welche die Studie zu Ende führen, können an einer offenen Anschluss-Studie teilnehmen, bei der alle Patienten Idebenon erhalten. Die Studie wird in 63 Zentren in den USA, Europa und Israel durchgeführt. Weitere Informationen zur Studie sind verfügbar unter www.clinicaltrials.gov und www.siderosdmd.com. Über Duchenne-Muskeldystrophie Duchenne-Muskeldystrophie (DMD) ist eine der am weitesten verbreiteten und schwerwiegendsten Formen von rasch fortschreitendem Muskelschwund. DMD ist eine genetische, degenerative Erkrankung, die fast ausschliesslich Knaben betrifft und weltweit mit einer Inzidenz von bis zu 1 in 3'500 männlichen Lebendgeburten auftritt. Über Idebenon bei Duchenne-Muskeldystrophie Charakteristisch für Duchenne-Muskeldystrophie (DMD) ist der Verlust des Proteins Dystrophin, der zu Zellschädigung, gestörtem zellulärem Kalziumhaushalt, erhöhtem oxidativem Stress und verringerter zellulärer Energieproduktion in Muskelzellen führt. Diese zellulären Schäden resultieren in fortschreitender Muskelschwäche und Muskelschwund und führen aufgrund von Atmungsversagen zu früher Morbidität und Mortalität. Idebenon ist ein synthetisches Benzoquinone und Kofaktor für das zelluläre Enzym NAD(P)H:quinone oxidoreductase (NQO1). Der Wirkstoff kann den mitochondrialen Elektronentransport stimulieren, den oxidativen Stress vermindern und die zelluläre Energieversorgung verbessern. DELOS war eine Phase-III, doppelblinde, Plazebo-kontrollierte Studie über 52 Wochen mit 64 Patienten ohne Glukokortikoid-Begleittherapie, welche entweder Idebenon (900 mg/Tag) oder entsprechendes Plazebo erhielten. Die Studie erreichte den primären Endpunkt, eine Veränderung der maximalen exspiratorischen Atemflussrate (Peak Expiratory Flow, PEF) und zeigte, dass Idebenon den Verlust der Atmungsfunktion verzögern kann. Idebenon war in der DELOS-Studie gut verträglich und die Gesamthäufigkeit von Nebenwirkungen vergleichbar zu Plazebo. Die am häufigsten beobachteten Nebenwirkungen waren Durchfall (Idebenon 25% gegenüber 12% für Plazebo), Verstopfung (Idebenon 9% gegenüber 18% für Plazebo) und Bauchschmerzen (Idebenon 9% und 9% für Plazebo). Die statistisch signifikanten und klinisch relevanten Resultate der Phase-III-DELOS-Studie wurden veröffentlicht von Buyse et al., The Lancet 2015, 385:1748-1757; McDonald et al., Neuromuscular Disorders 2016, 26: 473-480 und Buyse et al., Pediatric Pulmonology 2016: http://dx.doi.org/10.1002/ppul.23547. Der Ausschuss für Humanarzneimittel (CHMP) der Europäischen Arzneimittelbehörde (EMA) und das Schweizerische Heilmittelinstitut Swissmedic prüfen derzeit einen Zulassungsantrag (Marketing Authorization Application, MAA) für Idebenon unter der Marke RAXONE® bei DMD-Patienten mit abnehmender Atmungsfunktion und ohne Glukokortikoid-Begleittherapie.   Über Santhera Santhera Pharmaceuticals (SIX: SANN) ist ein auf die Entwicklung und Vermarktung innovativer Medikamente zur Behandlung seltener neuromuskulärer und mitochondrialer Krankheiten fokussiertes Schweizer Spezialitätenpharmaunternehmen. Das erste Produkt von Santhera, RAXONE® (Idebenon), ist in der Europäischen Union, Norwegen, Island und Liechtenstein zur Behandlung von Leber hereditärer Optikusneuropathie (LHON) zugelassen. Für Duchenne-Muskeldystrophie (DMD) bei Patienten mit abnehmender Atmungsfunktion und ohne Glukokortikoid-Begleittherapie hat Santhera in der Europäischen Union und der Schweiz einen Antrag auf Marktzulassung gestellt. In Zusammenarbeit mit dem US National Institute of Neurological Disorders and Stroke (NINDS) ent-wickelt Santhera RAXONE® in einer dritten Indikation, primär progredienter Multipler Sklerose (PPMS), sowie Omigapil für Patienten mit kongenitaler Muskeldystrophie (CMD). Für alle diese Krankheiten besteht ein sehr hoher medizinischer Bedarf. Weitere Informationen zu Santhera sind verfügbar unter www.santhera.com. RAXONE® ist eine eingetragene Marke von Santhera Pharmaceuticals.    Für weitere Auskünfte wenden Sie sich bitte an: Thomas Meier, PhD, Chief Executive Officer            Christoph Rentsch, Chief Financial Officer Telefon +41 61 906 89 64                                         Telefon +41 61 906 89 65 thomas.meier@santhera.com                                   christoph.rentsch@santhera.com Sue Schneidhorst, Head Group Communications Telefon +41 61 906 89 26 sue.schneidhorst@santhera.com Medienkontakt (deutschsprachige Länder) Eva Kalias, Vio Consult Telefon +41 78 671 98 86 kalias@vioconsult.com Disclaimer / Forward-looking statements Diese Mitteilung stellt weder ein Angebot noch eine Aufforderung zur Zeichnung oder zum Kauf von Wertpapieren der Santhera Pharmaceuticals Holding AG dar. Diese Publikation kann bestimmte zukunftsgerichtete Aussagen über das Unternehmen und seine Geschäftsaktivitäten enthalten. Solche Aussagen beinhalten bestimmte Risiken, Unsicherheiten und andere Faktoren, die dazu führen können, dass die tatsächlichen Ergebnisse, die finanzielle Lage, der Leistungsausweis oder die Zielerreichung des Unternehmens wesentlich von den in diesen Aussagen ausgedrückten oder implizierten Erwartungen abweichen. Die Leser sollten sich daher nicht in unangemessener Weise auf diese Aussagen verlassen, insbesondere nicht im Zusammenhang mit einer Vertrags- oder Investitionsentscheidung. Das Unternehmen lehnt jede Verpflichtung zur Aktualisierung dieser Aussagen ab.  # # #


Liestal, Schweiz, 19. Mai 2017 - Santhera Pharmaceuticals, ein auf die Entwicklung innovativer Medikamente zur Behandlung seltener neuromuskulärer und mitochondrialer Krankheiten fokussiertes Spezialitätenpharmaunternehmen, gibt einen aktualisierten Zeitrahmen für die derzeit laufende Beurteilung ihres Erweiterungsantrags für Raxone® (Idebenon) für Duchenne-Muskeldystrophie (DMD) durch den Ausschuss für Humanarzneimittel (CHMP) bekannt. "Wir sind in konstruktiven Gesprächen mit Vertretern des CHMP und erwarten demnächst eine Aufforderung zur Einreichung ergänzender Informationen, um die klinische Bedeutung unserer Daten weiter zu unterstützen. Wir arbeiten eng mit dem CHMP zusammen, um den Antragsprozess zügig abzuschliessen und gehen jetzt von einem Entscheid im dritten Quartal 2017 aus", sagte Thomas Meier, PhD, CEO von Santhera. Der Antrag wurde als Typ-II-Variation zur bestehenden Marktzulassung eingereicht und stützt sich auf Daten von Santheras Phase-II-Studie (DELPHI) und der erfolgreichen Phase-III-Studie (DELOS), letztere bei Patienten ohne Glukokortikoid-Begleittherapie. Diese Daten wiesen einen statistisch signifikanten und klinisch relevanten Nutzen von Raxone auf den Erhalt der Atmungsfunktion gegenüber Plazebo nach. Die Resultate wurden zudem durch eine Studie zum natürlichen Krankheitsverlauf erhärtet, die zeigte, dass der mit Raxone-Behandlung beobachtete Krankheitsverlauf beim natürlichen Fortschreiten der Erkrankung nicht zu erwarten gewesen wäre. Die beantragte Indikation für Raxone ist die Verlangsamung des Atmungsfunktionsverlusts bei DMD-Patienten ohne Glukokortikoid-Begleittherapie. Die Indikation schliesst Patienten mit ein, die früher mit Glukokortikoiden behandelt wurden oder bei denen diese unerwünscht, nicht verträglich oder kontraindiziert sind. Über Duchenne-Muskeldystrophie Duchenne-Muskeldystrophie (DMD) ist eine der am weitesten verbreiteten und schwerwiegendsten Formen von rasch fortschreitendem Muskelschwund. DMD ist eine genetische, degenerative Erkrankung, die fast ausschliesslich Knaben betrifft und weltweit mit einer Inzidenz von bis zu 1 in 3'500 männlichen Lebendgeburten auftritt. Über Idebenon bei Duchenne-Muskeldystrophie Charakteristisch für Duchenne-Muskeldystrophie (DMD) ist der Verlust des Proteins Dystrophin, der zu Zellschädigung, gestörtem zellulärem Kalziumhaushalt, erhöhtem oxidativem Stress und verringerter zellulärer Energieproduktion in Muskelzellen führt. Diese zellulären Schäden resultieren in fortschreitender Muskelschwäche und Muskelschwund und führen aufgrund von Atmungsversagen zu früher Morbidität und Mortalität. Idebenon ist ein synthetisches Benzoquinone und Kofaktor für das zelluläre Enzym NAD(P)H:quinone oxidoreductase (NQO1). Der Wirkstoff kann den mitochondrialen Elektronentransport stimulieren, den oxidativen Stress vermindern und die zelluläre Energieversorgung verbessern. DELOS war eine Phase-III, doppelblinde, Plazebo-kontrollierte Studie über 52 Wochen mit 64 Patienten ohne Glukokortikoid-Begleittherapie, welche entweder Idebenon (900 mg/Tag) oder entsprechendes Plazebo erhielten. Die Studie erreichte den primären Endpunkt, eine Veränderung der maximalen exspiratorischen Atemflussrate (Peak Expiratory Flow, PEF) und zeigte, dass Idebenon den Verlust der Atmungsfunktion verzögern kann. Idebenon war in der DELOS-Studie gut verträglich und die Gesamthäufigkeit von Nebenwirkungen vergleichbar zu Plazebo. Die statistisch signifikanten und klinisch relevanten Resultate der Phase-III-DELOS-Studie wurden veröffentlicht von Buyse et al., The Lancet 2015, 385:1748-1757; McDonald et al., Neuromuscular Disorders 2016, 26: 473-480 und Buyse et al., Pediatric Pulmonology 2017, 52:580-515. Der Ausschuss für Humanarzneimittel (CHMP) der Europäischen Arzneimittelbehörde (EMA) und das Schweizerische Heilmittelinstitut Swissmedic prüfen derzeit einen Zulassungsantrag (Marketing Authorization Application) für Idebenon unter der Marke Raxone® bei DMD-Patienten mit abnehmender Atmungsfunktion und ohne Glukokortikoid-Begleittherapie.    Über Santhera Santhera Pharmaceuticals (SIX: SANN) ist ein auf die Entwicklung und Vermarktung innovativer Medikamente zur Behandlung seltener neuromuskulärer und mitochondrialer Krankheiten fokussiertes Schweizer Spezialitätenpharmaunternehmen. Das erste Produkt von Santhera, Raxone® (Idebenon), ist in der Europäischen Union, Norwegen, Island und Liechtenstein zur Behandlung von Leber hereditärer Optikusneuropathie (LHON) zugelassen. Für Duchenne-Muskeldystrophie (DMD) bei Patienten mit abnehmender Atmungsfunktion und ohne Glukokortikoid-Begleittherapie hat Santhera in der Europäischen Union und der Schweiz einen Antrag auf Marktzulassung gestellt. In Zusammenarbeit mit dem US National Institute of Neurological Disorders and Stroke (NINDS) entwickelt Santhera Raxone® in einer dritten Indikation, primär progredienter Multipler Sklerose (PPMS), sowie Omigapil für Patienten mit kongenitaler Muskeldystrophie (CMD). Für alle diese Krankheiten besteht ein sehr hoher medizinischer Bedarf. Weitere Informationen zu Santhera sind verfügbar unter www.santhera.com. Raxone® ist eine eingetragene Marke von Santhera Pharmaceuticals. Für weitere Auskünfte wenden Sie sich bitte an: Thomas Meier, PhD, Chief Executive Officer            Christoph Rentsch, Chief Financial Officer Telefon +41 61 906 89 64                                         Telefon +41 61 906 89 65 thomas.meier@santhera.com                                   christoph.rentsch@santhera.com Sue Schneidhorst, Head Group Communications Telefon +41 61 906 89 26 sue.schneidhorst@santhera.com Medienkontakt (deutschsprachige Länder) Eva Kalias, Vio Consult Telefon +41 78 671 98 86 kalias@vioconsult.com Disclaimer / Forward-looking statements Diese Mitteilung stellt weder ein Angebot noch eine Aufforderung zur Zeichnung oder zum Kauf von Wertpapieren der Santhera Pharmaceuticals Holding AG dar. Diese Publikation kann bestimmte zukunftsgerichtete Aussagen über das Unternehmen und seine Geschäftsaktivitäten enthalten. Solche Aussagen beinhalten bestimmte Risiken, Unsicherheiten und andere Faktoren, die dazu führen können, dass die tatsächlichen Ergebnisse, die finanzielle Lage, der Leistungsausweis oder die Zielerreichung des Unternehmens wesentlich von den in diesen Aussagen ausgedrückten oder implizierten Erwartungen abweichen. Die Leser sollten sich daher nicht in unangemessener Weise auf diese Aussagen verlassen, insbesondere nicht im Zusammenhang mit einer Vertrags- oder Investitionsentscheidung. Das Unternehmen lehnt jede Verpflichtung zur Aktualisierung dieser Aussagen ab.


Liestal, Switzerland, May 9, 2017 - Santhera Pharmaceuticals (SIX: SANN) announces approval by the Scottish Medicines Consortium for restricted use of Raxone® (idebenone) in patients with LHON, with effect May 8, 2017. Patients with Leber's hereditary optic neuropathy (LHON), an extremely rare, inherited cause of blindness, could benefit from the only licensed treatment to date, following advice issued by the Scottish Medicines Consortium (SMC) under the ultra-orphan medicine process. The SMC accepted Raxone® (idebenone) for restricted use by NHS Scotland in the treatment of visual impairment in adolescent and adult patients with LHON who are not yet blind (i.e., they do not meet the UK criteria to be registered as severely sight impaired). Raxone was accepted following consideration through SMC's Patient and Clinician Engagement (PACE) process, for medicines used at the end of life and for very rare conditions. Scotland is the first country in the UK to make Raxone available for the treatment of LHON. The SMC advice takes into account the benefits of a Patient Access Scheme (PAS), which improves the cost-effectiveness of Raxone. LHON is a devastating disease that causes blindness mainly in young men during their late teens/early adulthood. Patients suddenly lose central vision and can no longer see fine details, read print or recognize faces. Within 12 months from onset, about 80% of untreated patients will have become legally blind. This is exceptionally debilitating and isolating in young adults, affecting their education, career plans and overall quality of life. Russell Wheeler, Trustee of the UK based LHON Society said: "Despite LHON being identified almost 150 years ago, the lack of any approved treatment for this devastating condition until now has led to many patients feeling helpless and abandoned by the health service. We applaud the pragmatism of the SMC's decision following a rigorous and transparent process. The availability of Raxone to affected families in Scotland is a welcome step towards avoiding preventable blindness in patients with LHON and we hope it will be followed by similar availability in other parts of the UK." Raxone treatment is now available to LHON patients under different reimbursement models in several European countries. Giovanni Stropoli, EVP, Chief Commercial Officer Europe & ROW commented, "We are grateful to the SMC for their comprehensive evaluation of our dossier for Raxone. As we continue working with the health services in other parts of the UK we hope that this positive advice to NHS Scotland will be followed soon by similar availability for LHON patients across the United Kingdom." The full SMC advice can be reviewed at the following link: http://www.scottishmedicines.org.uk/SMC_Advice/Advice/1226_17_idebenone_Raxone/idebenone_Raxone. Please also refer to the full Summary of Product Characteristics at: http://www.medicines.org.uk/emc/medicine/32655. About Leber's Hereditary Optic Neuropathy and the Therapeutic Use of Raxone LHON is a heritable genetic disease causing blindness. The disease presents predominantly in young, otherwise healthy adult males as rapid, painless loss of central vision, usually leading to permanent bilateral blindness within a few months of the onset of symptoms. About 95% of patients harbor one of three pathogenic mutations of the mitochondrial DNA, which cause a defect in the complex I subunit of the mitochondrial respiratory chain. This defect leads to decreased cellular energy (ATP) production, increased reactive oxygen species (ROS) production and retinal ganglion cell dysfunction, which cause progressive loss of visual acuity and blindness. Raxone (idebenone), a synthetic short-chain benzoquinone and a cofactor for the enzyme NAD(P)H:quinone oxidoreductase (NQO1), circumvents the complex I defect, reduces and scavenges ROS, restores cellular energy levels in retinal ganglion cells and promotes recovery of visual acuity. Raxone is an oral medication, authorized at a daily dose of 900 mg (given as 2 tablets three times a day with food), for the treatment of visual impairment in adolescent and adult patients with LHON. Efficacy data come from Santhera's randomized, placebo-controlled 24-week RHODOS trial, from the open label Expanded Access Program of 'real-life' usage and from a natural history study of untreated patients, which together have demonstrated that vision loss can be mitigated or reversed in patients treated with Raxone. Treatment should be initiated and supervised by a physician with experience in LHON. 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 and Switzerland. 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. 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@santhera.com                                christoph.rentsch@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 Article | November 22, 2016
Site: www.prnewswire.co.uk

ReportsnReports.com adds "Muscular Dystrophy - Pipeline Review, H2 2016" to its store, providing comprehensive information on the therapeutics under development for Muscular Dystrophy, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Muscular Dystrophy and features dormant and discontinued projects. Muscular dystrophy is a group of diseases in which muscle fibers are unusually susceptible to damage. These damaged muscles become progressively weaker. Symptoms usually appear before age 6 and may appear as early as infancy. They may include fatigue, learning difficulties, intellectual disability, muscle weakness and progressive difficulty walking. Complete report on Global Muscular Dystrophy Market Research with 44 market data tables and 15 figures, spread across 144 pages is available at http://www.reportsnreports.com/reports/755879-muscular-dystrophy-pipeline-review-h2-2016.html . Company Analysis and Positioning discussed in this research are Acceleron Pharma, Inc., AMO Pharma Limited, Asahi Kasei Pharma Corp., aTyr Pharma, Inc., Benitec Biopharma Limited, Bio Blast Pharma Ltd., Biophytis SAS, Corcept Therapeutics Incorporated, Evotec AG, F. Hoffmann-La Roche Ltd., Genethon, Ionis Pharmaceuticals, Inc., Marina Biotech, Inc., Medestea Research & Production S.p.A., Novogen Limited, Pfizer Inc., Prothelia, Inc., SanBio, Inc., Santhera Pharmaceuticals Holding AG, Sarepta Therapeutics, Inc., Selecta Biosciences, Inc., Strykagen Corporation, Takeda Pharmaceutical Company Limited and WAVE Life Sciences Ltd. Drug Profiles mentioned in this research are  ACE-083, Antisense Oligonucleotide to Inhibit DM1 Protein Kinase for Myotonic Dystrophy, Antisense RNAi Oligonucleotides for Myotonic Dystrophy, ATYR-1940, baliforsen, BIO-103, domagrozumab, Drugs for Merosin-Deficient Congenital Muscular Dystrophy Type 1A, elcatonin, Gene Therapy for Muscular Dystrophy and Liver Diseases, Gene Therapy to Activate Dysferlin for Duchenne and Limb Girdle Muscular Dystrophies, Gene Therapy to Activate Dysferlin for Dysferlinopathies, Gene Therapy to Activate Dystrophin for Muscular Dystrophy, IUCT-169, IUCT-290, IUCT-309, ketoprofen, LR-08, MED-1101, mexiletine hydrochloride, Oligonucleotide 1 to Target Dystrophia Myotonica Protein Kinase for Myotonic Dystrophy, Oligonucleotides to Inhibit DM1 Protein Kinase for Myotonic Dystrophy, omigapil, Pabparna, poloxamer, PRT-01, Recombinant Protein to Activate Utrophin for Muscular Dystrophies, RNAi Gene Therapy to Inhibit Myotilin for LGMD, RP-33, SB-308, SIWA-318, Small Molecule to Inhibit DUX4 for Muscular Dystrophy, Small Molecule to Target CUG RNA for Myotonic Dystrophy 1, Small Molecule to Target RNA for Myotonic Dystrophy, Small Molecules for Dysferlinopathies, Small Molecules for Facioscapulohumeral Muscular Dystrophy, Small Molecules for Myotonic Dystrophy, Small Molecules for Myotonic Dystrophy Type 1, Small Molecules to Activate SMCHD1 for Facioscapulohumeral Dystrophy, Small Molecules to Antagonize Glucocorticoid Receptor II for Muscular Dystrophy, Small Molecules to Inhibit MBNL1 for Myotonic Dystrophy Type I, Small Molecules to Target RNA for Myotonic Dystrophy, SRT-149, SRT-152, Stem Cell Therapy for Muscular Dystrophy, Stryka-232, Stryka-234, Stryka-425, Stryka-533, Stryka-978, tideglusib, trehalose, TXA-127, VAL-0411 and VAL-1205. The Muscular Dystrophy (Musculoskeletal Disorders) pipeline guide also reviews of key players involved in therapeutic development for Muscular Dystrophy and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 5, 2, 24 and 14 respectively. Similarly, the Universities portfolio in Phase II, Phase I, Preclinical and Discovery stages comprises 1, 1, 3 and 4 molecules, respectively.Muscular Dystrophy. Muscular Dystrophy (Musculoskeletal Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis. ReportsnReports.com is your single source for all market research needs. Our database includes 500,000+ market research reports from over 100+ leading global publishers & in-depth market research studies of over 5000 micro markets. With comprehensive information about the publishers and the industries for which they publish market research reports, we help you in your purchase decision by mapping your information needs with our huge collection of reports. Connect With Us on:


Liestal, Switzerland, December 23, 2016 - Santhera Pharmaceuticals (SIX: SANN) announces that it has been informed that the UK's Medicines and Healthcare Products Regulatory Agency (MHRA) designated Raxone® (idebenone) for the treatment of Duchenne muscular dystrophy (DMD) in patients with respiratory function decline not taking concomitant glucocorticoids as Promising Innovative Medicine (PIM) and as a suitable candidate for entry into Step II of the EAMS process. In the UK the Early Access to Medicines Scheme aims to give patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorization when there is a clear unmet medical need. "We are delighted about MHRA's decision designating Raxone as a candidate for further consideration under the Early Access to Medicines Scheme as this underscores the high unmet medical need in DMD and acknowledges the innovative treatment approach of Raxone," said Thomas Meier, PhD, CEO of Santhera. Nic Bungay, Director of Campaigns, Care and Information at Muscular Dystrophy UK, commented: "We welcome this very encouraging news that Raxone has been considered suitable for the second step of the EAMS process, especially as this is the first muscular dystrophy drug to reach this stage. This demonstrates that the introduction of the EAMS, which Muscular Dystrophy UK had called for, could help to fast track emerging treatments. We urge the Government to continue to support the use of EAMS by pharmaceutical companies to bring through potentially promising treatments." The European Medicines Agency (EMA) and Swissmedic (the Swiss Agency for Therapeutic Products) are currently reviewing marketing authorization applications for Raxone for DMD patients in whom respiratory function has started to decline and who are not taking concomitant glucocorticoids. This indication would include patients who were previously treated with glucocorticoids or in whom glucocorticoid treatment is not desired, not tolerated or is contraindicated. About the UK Early Access to Medicines Scheme (EAMS) The UK's industry-sponsored EAMS aims to give patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorization when there is a clear unmet medical need. The EAMS is a two-step process: Step I is the Designation as a Promising Innovation Medicine (PIM). The PIM designation is an early indication that a medicinal product is a promising candidate for EAMS and gives reassurance that its clinical development is on track by having an early review of its data by the medicines regulator. Step II is the Scientific Opinion by the Medicines and Healthcare products Regulatory Agency (MHRA, UK regulatory agency). The Scientific Opinion describes the benefits and risks of the medicine and supports the prescriber and patient to make a decision on using the medicine before its license is approved. 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 and Switzerland. 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. 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.

Loading Santhera Pharmaceuticals Holding collaborators
Loading Santhera Pharmaceuticals Holding collaborators