GEN Exclusives

More »

GEN News Highlights

More »
Dec 20, 2011

Summit Gets $0.75M from Muscular Dystrophy Association

  • U.K. Biotech Summit received a $750,000 grant from the Muscular Dystrophy Association (MDA) to help fund early clinical development of the firm’s SMT C110 candidate, a small molecule utrophin upregulator, which is in development for the treatment of Duchenne muscular dystrophy (DMD).

    SMT C110 was originally discovered and developed by Summit, and has previously been evaluated in a healthy volunteer study by a U.S. pharmaceutical company. While the drug was found to be safe and well tolerated, administration led to variable blood concentrations, and therapeutic blood plasma levels were only achieved in some patients. Summit believes part of this problem was due to the formulation used in the trial, and aims to develop the SMT C1100 based on alternative formulations.

    SMT C1100 has been granted orphan drug status in the U.S. and Europe. Previous preclinical studies indicated that treatment increases utrophin protein levels in dystrophin-deficient muscle cells from DMD patients, to levels expected to be of significant therapeutic benefit. In vivo studies also found the drug significantly increased utrophin levels, improved whole-muscle function, and reduced muscle degeneration, fibrosis, and chronic inflammation. 


Add a comment

  • You must be signed in to perform this action.
    Click here to Login or Register for free.
    You will be taken back to your selected item after Login/Registration.

Related content

Jobs

GEN Jobs powered by HireLifeScience.com connects you directly to employers in pharma, biotech, and the life sciences. View 40 to 50 fresh job postings daily or search for employment opportunities including those in R&D, clinical research, QA/QC, biomanufacturing, and regulatory affairs.
 Searching...
More »

GEN Poll

More » Poll Results »

Connectomics Advocacy

How might connectomics maintain lasting support?