نئون مرحلو 1 ڪلينڪل آزمائشي ٻارن جي سي اين ايس ٽيمر لاء

EpicentRx, Inc., a clinical stage drug and device company, in collaboration with the Texas Children’s Cancer Center in Houston, today announced the start of a Phase 1 study to examine the safety and benefit of its lead small molecule, RRx-001, plus irinotecan and temozolomide for pediatric patients with recurrent or progressive malignant solid and central nervous system tumors. The study, PIRATE (NCT04525014) was conceived and developed by doctors Holly Lindsay and Patricia Baxter at the Texas Children’s Cancer Center and will enroll patients 1 to 21 years of age with recurrent or progressive malignant primary brain or spinal cord tumors and solid tumors, excluding lymphomas.        

The genesis for the PIRATE trial was based on several preceding clinical trials which have established potential evidence that RRx-001 increases chemotherapy delivery and uptake in tumors. A Phase 1 clinical trial called G-FORCE (NCT02871843) and a Phase 1/2 clinical trial called BRAINSTORM (NCT02215512) demonstrated the safety of RRx-001 as well as potential evidence of clinical benefit in adult patients with glioblastoma or GBM and with brain metastases, respectively.

Cancer cells are programmed to travel to different tissues in the body, where they establish a barrier to prevent drug delivery. One of the hypotheses of this trial is that RRx-001 may “normalize” the tumor microenvironment, making the tumor vasculature more efficient for the delivery of irinotecan and temozolomide so that their efficacy is increased while overstimulating immune cells like macrophages to go on the attack through its antagonism of the “do not eat me” CD47 signal. Another hypothesis is that RRx-001 will protect normal tissues, but not tumors, from the toxicities of irinotecan and temozolomide.

“We are excited to launch this study with RRx-001 and to establish its role as a potential treatment for pediatric CNS tumors,” said Tony R. Reid, M.D., Ph.D., Chief Executive Officer of EpicentRx. “This trial would not have been possible without the enthusiastic support of Texas Children’s Cancer Center. As clinical trials in adult CNS tumors suggest that RRx-001 may provide benefit in a pediatric population, our collaboration with them is a testament to the commitment of this company to follow the data wherever it leads.”

“There is compelling preclinical and clinical rationale for the safety and potential efficacy of RRx-001 in CNS tumors,” said Dr. Holly Lindsay, M.D., Assistant Professor, Department of Pediatrics, Section of Hematology-Oncology, Baylor College of Medicine and lead Investigator. “The incidence of CNS tumors in children is particularly difficult to treat, and we expect that the PIRATE study will generate valuable data to help inform future treatment decisions.”