Summit Therapeutics PLC (NASDAQ:SMMT LON:SUMM) will receive a US$22mln milestone payment from Sarepta Therapeutics (NASDAQ:SRPT) after the final patient was enrolled in a dosing trial of utrophin modulator, ezutromid.
Summit hopes the trial will identify utrophin modulation as a potential disease-modifying treatment for all patients suffering Duchenne Muscular Dystrophy (DMD), a progressive and fatal muscle wasting disease that affects 50,000 boys and young men globally.
The endpoints for PhaseOut DMD, a phase II trial of ezutromid, are muscle structure, health and function improvements.
Dosing of the last patient triggered a US$22mln payment for Summit through its licence and collaboration agreement with Sarepta (NASDAQ:SRPT).
David Roblin, chief operating officer and Summit’s president of R&D said: "Utrophin modulation has potential as a universal treatment option for patients with DMD, and we look forward to the 24-week data readout expected in the first quarter of 2018."
The PhaseOut DMD will monitor 40 patients at sites in the UK and the US and take 48-weeks overall to complete.
As part of the trial, each patient undergoes two biopsies, a baseline biopsy on enrolment and a second either at 24 or 48 weeks.
In the first quarter of 2018, Summit expects to report 24-week biopsy analysis from approximately 20 patients, as well as 24-week MRI and functional data from all 40 patients enrolled in the trial.
Top-line data from the complete 48-week trial is expected in the third quarter of 2018.
Shares in Summit rose by 5% to 187p.