The Phase Ib trial is designed to evaluate the safety, pharmacokinetics, and pharmacodynamics of escalating doses of EPZ-5676 in patients between the ages of three months and 18 years and to provide a preliminary assessment of efficacy.
In addition, the company now has ongoing assessments of proof-of-concept (POC) in three genetically defined cancer patient groups in the EPZ-5676 clinical program (MLL-r adults, MLL-PTD, and MLL-r pediatrics) with data expected to be announced reported in the second half of 2014.
Epizyme chief medical officer Eric Hedrick said acute leukemias with MLL-r affect young children and are one of the last remaining forms of childhood leukemia with inadequate treatment options.
"Children who are afflicted with this genetically defined cancer have an extremely poor prognosis, especially in comparison to pediatric leukemias without the MLL rearrangement," Hedrick said.
"This pediatric clinical study of EPZ-5676 is an important complement to our ongoing adult studies and reflects our commitment to developing personalized treatments for patients with genetically defined cancers."
At present, the company is enrolling adult MLL-r and MLL-PTD acute leukemia patients in the expansion stage of the adult EPZ-5676 Phase I trial.
In January 2014, the company had secured a $25m clinical proof-of-concept milestone in their Celgene collaboration with objective responses observed in two adult MLL-r patients in the fourth cohort of the dose escalation stage of the Phase I trial, which completed enrolment in December 2013.
The company intends to release data from the adult Phase I dose escalation and expansion stage trial for EPZ-5676 in the second half of 2014.