UK drug discovery firm Summit Pharmaceuticals (AIM: SUMM) is an Oxford-based drug discovery and development company targeting high-value areas of unmet medical need including Duchenne Muscular Dystrophy (DMD) and C. difficile infection.

For DMD, Summit’'s utrophin modulation program uses oral small molecules to increase the production of a protein called utrophin. This scientific approach for the treatment of DMD aims to use 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’s novel antibiotic programme for C.diff is being developed as a treatment for initial CDI and the prevention of recurrent disease.

SMT 19969 is a novel antibiotic that works via a new mechanism of action and it is differentiated to existing marketed drugs or those in development. It combines excellent potency with unprecedented selectivity for C. difficile meaning it does not disrupt the healthy gut bacteria. This is expected to be important in naturally preventing recurrent CDI and so improve the prognosis for patients.