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Duchenne Muscular Dystrophy: Business Developments (09.02.13)

September 2, 2013

This is the second Blog Post in a series that examines Duchenne Muscular Dystrophy (DMD) in the rare disease and orphan drug space.

This Blog Post discusses and reviews recent Summit plc business developments.

Summit plc, a drug discovery and development UK-based company, reports on August 22, 2013, interim financial results for the first six months of 2013. Some of the DMD product highlights from Summit plc’s 6-month financial review:

  1. Establishment of a Program Advisory Board to support scientific and clinical development of the company’s DMD Program
  2. Initiation of a Phase Ib safety and dose-finding clinical trial of SMT C1100 to start in the second half of 2013
  3. Initiation of a novel biomarker program
  4. A grant of £2.4 million (approximately US $3.7 million), from the UK Biomedical Catalyst Fund, is awarded for the support of the clinical development of SMT C1100.

Summit’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…”.

Per Summit plc, some of the strengths of the company’s DMD Program:

  1. Utrophin modulation program is a new potential treatment paradigm for DMD
  2. Phase Ib clinical rial of SMT C1100 is the 1st to evaluate a utrophin therapy in patients
  3. Novel biomarker program to develop techniques for evaluating SMT C1100 in future clinical trials.

Summit plc announces on August 28, 2013, that the company receives an US “composition of matter patent” that covers its DMD product SMT C1100. This “cornerstone patent” provides exclusivity for SMT C1100 to at least November 2018. Also, in August 2013, a US patent is issued to protect the use of SMT C1100 in combination with steroids, such as orphan drug Deflazacort and Prednisolone.  


Review of Phase II and Phase III clinical trials for Duchenne Muscular Dystrophy. Scully M, Pandya S, Moxley R. Expert Opinion on Orphan Drugs. Jan 2013; 1(1): 33-46

Summit plc DMD Program.

Please Note: “Spinning Device To Mix Tissue Culture Medium” by National Cancer Institute [Public domain] | Wikimedia Commons.

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

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