Utrophin upregulation by ezutromid (SMT C1100) [terminated]

Ricotti V, Spinty S, Roper H, et al. Safety, Tolerability, and Pharmacokinetics of SMT C1100, a 2-Arylbenzoxazole Utrophin Modulator, following Single- and Multiple-Dose Administration to Pediatric Patients with Duchenne Muscular Dystrophy. PloS one 2016;11:e0152840

Ricotti_2016_SMT C1000 phase 1

Muntoni F, Tejura B, Spinty S, et al. A Phase 1b Trial to Assess the Pharmacokinetics of Ezutromid in Pediatric Duchenne Muscular Dystrophy Patients on a Balanced Diet. Clinical pharmacology in drug development

Muntoni_2019_Clin Pharmacol Drug Dev. Jan 16

In a first phase I trial (Ricotti et al., 2016) into the utrophin upregulator exutromid (SMT C1100) the compound was found to be save; however plasma levels were lower than expected and very variable between patients. They hypothesized that this could be due to the diet. In the following phase Ib study (Muntoni et al., 2019) controlling the diet resulted in improved uptake of the compound. They suggest this information is valuable for future trials. They, however, do not mention that the sponsor (Summit) has recently decided to stop the development of ezutromid, since the phase II trial did not show positive results:

27-06-2018_RNS_30-PhaseOut-Full-Results-FINAL

https://www.summitplc.com/app/uploads/2018/09/27-06-2018_RNS_30-PhaseOut-Full-Results-FINAL.pdf

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