New collaboration to support omigapil clinical trial for congenital muscular dystrophy

Published Date
Julia Ambler

Santhera Pharmaceuticals have announced a collaboration that will help to facilitate the smooth transition of omigapil from the laboratory through to the clinic. The pharmaceutical company has partnered with European consortium EndoStem for the upcoming clinical trial of omigapil.

EndoStem, a Europe-wide consortium of clinicians, scientists, biotech and pharmaceutical companies, is to collaborate with Santhera Pharmaceuticals on the phase 1 clinical trial of omigapil which is expected to be started early next year. Omigapil, which was originally developed to treat neurological conditions such as Parkinson’s disease and amyotrophic lateral sclerosis, will be tested for use in Ullrich congenital muscular dystrophy, Bethlem myopathy and merosin-deficient congenital muscular dystrophy (MDC1A). 

EndoStem, which is co-funded by the European Union, aims to conduct clinical trials in muscular dystrophies using innovative new drugs and therapeutic approaches. Their focus is on regenerative medicine and involves trying to understand and harness the factors that control how muscles can repair and regenerate themselves following injury, in disease and in aging.

The consortium’s research involves studying stem cells from muscle and blood vessels, inflammatory processes that could be causing damage or preventing tissue regeneration, and investigations into the environment around the muscle that could help or hinder regeneration. Together, the consortium partners all have expertise that represents each step needed to get a drug or technology from the “bench to the bedside”. 

It is hoped that should the clinical trials provide a positive outcome, the collaboration between EndoStem and Santhera will ensure a commercialisation pathway for omigapil so that it could become widely available for patients with these conditions.

The phase 1 clinical trial of omigapil is due to take place in two centres: one in the UK, at Great Ormond Street Hospital (London)and the other in the USA, at the National Institute of Neurological Disorders and Stroke/National Institute of Health (Bethesda, Maryland). Santhera hopes to start enrolling patients in the second half of 2012. 

Support from the Muscular Dystrophy Campaign has been vital for Great Ormond Street Hospital’s participation since charity-funded clinical fellow Dr Reghan Foley has worked closely with Prof Francesco Muntoni on the development of the protocol for the clinical trial. We also fund a Clinical Trials Co-ordinator, Hinal Patel, who has been instrumental in co-ordinating Great Ormond Street Hospital’s participation in the trial and will facilitate the future recruitment of study participants.

Prof Francesco Muntoni, clinical lead for the trial at Great Ormond Street Hospital said:

The main focus of this trial will be that of demonstrating safety and assessing if the drug is metabolised in children at the same speed as in adults. This information is crucial before committing to a long term study. We will nevertheless also assess the feasibility of performing accurate clinical and physiotherapy assessments for children with these disorders. A lot of discussion has been held between our team of doctors and physiotherapists, and the team of Dr Bonnemann in UK. Input from families and charities, including Cure CMD and MDC have helped to shape the final study protocol. 

Further information and links

Read Santhera’s press release 

Find out more about EndoStem 

Visit the congenital muscular dystrophy international registry 

Read more about the planned phase 1 clinical trial 

Find out more about omigapil 

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