Clinigen and Santhera launch US Expanded Access Program with Idebenone for patients with Duchenne Muscular Dystrophy

Clinigen Group plc (AIM: CLIN, ‘Clinigen’), the global pharmaceutical and services company, has initiated an Expanded Access Program (‘EAP’) in the US with Santhera Pharmaceuticals (SIX: SANN, ‘Santhera’) for idebenone in patients with Duchenne Muscular Dystrophy (‘DMD’) who are in respiratory decline.

The EAP (also known as a Managed Access Program), referred to as BreatheDMD, provides a mechanism through which eligible patients in the US with DMD can obtain access to investigational idebenone, through a network of research centres across the US.

EAPs such as BreatheDMD allow eligible patients with serious or life-threatening conditions to access an investigational medicinal product, upon physician request, before it is approved by regulatory authorities where there is no suitable alternative treatment for the medical condition.

Idebenone is a medicine that is currently under investigation for the treatment of DMD in patients who are in respiratory decline.

John Lagus, Head of Managed Access, Clinigen, said:

“There is a huge unmet clinical need for patients with DMD and we are pleased to be working in partnership with Santhera to help eligible patients in the US gain access to this important medicine.

“This partnership aligns with our mission of getting the right medicines to the right patient at the right time.”

Thomas Meier, PhD, CEO, Santhera, said:

“Santhera’s mission remains to develop and bring new treatment options for patients with DMD. Feedback from the DMD patient community highlights the need for access to additional treatment options. Santhera is proud to be launching this EAP, allowing eligible patients to obtain access to investigational idebenone.”

Details regarding this EAP program, including eligibility requirements, can be obtained by visiting the website www.breatheDMD.com.

Healthcare professionals can obtain details about the idebenone EAP by calling Clinigen at
Tel: +1 877 768 4303, or emailing idebenone@clinigengroup.com.

CONTACT DETAILS

Clinigen Group plc
Shaun Chilton, Group Chief Executive Officer
John Lagus, Head of Managed Access
Tel: +44 (0) 1283 495010

Instinctif Partners (media relations - Clinigen)
Melanie Toyne-Sewell / Alex Shaw / Deborah Bell
Tel: +44 (0) 20 7457 2020
Email: clinigen@instinctif.com

Notes to Editors

About Duchenne Muscular Dystrophy

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.

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.

With age, progressive respiratory muscle weakness affecting thoracic accessory muscles and the diaphragm causes respiratory disease, impaired clearance of airway secretions, recurrent pulmonary infections due to ineffective cough, and eventually respiratory failure. There is currently no treatment approved for slowing loss of respiratory function in patients with DMD.

About Idebenone in Duchenne Muscular Dystrophy

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. Data from the DELOS trial are published in peer-reviewed medical journals: Buyse et al., The Lancet 2015, 385:1748-1757; McDonald et al., Neuromuscular Disorders 2016, 26:473-480; Buyse et al., Pediatric Pulmonology 2017, 52:580-515; Mayer et al., Journal of Neuromuscular Diseases 2017, 4:189-198; Meier et al., Neuromuscular Disorders 2017, 27:307-314.

About Clinigen Group

Clinigen Group plc (AIM: CLIN) is a global pharmaceutical and services company with a unique combination of businesses focused on providing ethical access to medicines. Its mission is to deliver the right medicine to the right patient at the right time through three areas of global medicine supply; clinical trial, unlicensed and licensed medicines. Clinigen acquired Quantum Pharma in November 2017.

For more information, please visit www.clinigengroup.com

About Santhera

Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative medicines for orphan and other diseases with high unmet medical needs. The portfolio comprises clinical stage and marketed treatments for neuro-ophthalmologic, neuromuscular and pulmonary diseases. Santhera's lead product Raxone® (idebenone) is authorized in the European Union, Norway, Iceland, Liechtenstein and Israel for the treatment of Leber's hereditary optic neuropathy (LHON) and currently commercialized in 20 countries. Santhera is currently conducting the Phase III SIDEROS trial with idebenone in patients with Duchenne muscular dystrophy (DMD) in respiratory function decline and using concomitant glucocorticoids. In collaboration with the U.S. National Institute of Neurological Disorders and Stroke (NINDS) Santhera is developing idebenone in a third indication, primary progressive multiple sclerosis (PPMS), and another product – omigapil – for congenital muscular dystrophy (CMD), both also areas of high unmet medical need. For further information, please visit the Company's website: www.santhera.com