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Duchenne Muscular Dystrophy: Summit plc Business Developments

December 21, 2013

This is the fourth Blog Post in a series examining Duchenne Muscular Dystrophy (DMD) in the rare disease and orphan drug space. Recent Summit plc business developments for DMD are reviewed.

Summit plc, a spin-out from the University of Oxford (UK), is a drug discovery and development company advancing therapies for DMD and C. difficile infection. The company is strategically moving forward with their DMD program. The company initiates a clinical and biomarker development program for DMD, forms strategic alliances with an academic center and a patient advocacy organization, and doses the first patient in a Phase Ib clinical trial for SMT C1100. All of these activities occur during the past few months :

• In October 2013, at the International World Muscle Society Congress in California, presents the DMD biomarker program that aims to develop new clinical endpoints to help evaluate benefits of therapies

• In November 2013, enters into a funding agreement with the UK patient advocacy organization, Joining Jack, to support the development of novel biomarkers for DMD

• In November 2013, enters into an alliance with the University of Oxford (UK) to develop treatments for DMD. The company acquires exclusive commercial rights to a pipeline of novel, early-stage utrophin modulators and core biological screening technology

• On December 9th announces that the first DMD patient is enrolled and dosed in a Phase Ib clinical trial of the oral, small molecule utrophin modulator SMT C1100.

SMT C1100 is the first utrophin modulator to enter clinical trials for DMD. The Phase Ib clinical trial is a dose-escalating, open-label study that is being conducted in pediatric patients with DMD. Endpoint of the clinical trial is the safety and tolerability of SMT C1100. The trial is enrolling 12 patients, 5 – 11 years of age, divided into three dose cohorts.

SMT C1100 receives orphan drug designation from both the FDA (November 2011) and the European Medicines Agency (EMA). Summit plc’s utrophin modulation program uses oral small molecules to increase the production of a protein called utrophin. The objective of the utrophin modulation program is to use the:

” … naturally occurring and functionally similar utrophin protein to replace the missing dystrophin protein. Utrophin is produced during fetal and regenerating muscle development but is then switched off in mature muscle fibers. If production could be maintained, utrophin has the potential to substitute for the missing dystrophin to restore and maintain the healthy function of muscles. A significant advantage of increasing or upregulating utrophin is that it will benefit all DMD patients, regardless of the specific genetic mutation that is causing the underlying disease…”.


Summit plc DMD Program.

Please Note: “Technician Transfer Liquid To Test Tube” courtesy of US National Institute of Mental Health.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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