EpicentRx and Texas Children's Cancer Center Announce the Initiation of a Phase 1 Clinical Trial with RRx-001 for Pediatric CNS Tumors

TORREY PINES, Calif., Feb. 23, 2022 /PRNewswire/ -- 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."

For more information about PIRATE please visit https://clinicaltrials.gov/ct2/show/NCT04525014   

About EpicentRx, Inc.
EpicentRx is a leading-edge biopharmaceutical company with a complementary pipeline of small molecule and cancer-targeting virus platforms that represent the next frontier in treating patients with diseases of significant unmet need. With two platforms, CyNRGY and AdAPT, EpicentRx has developed novel therapies and drug delivery devices with emphasis on not just treating the disease but improving quality of life. For more information, visit www.epicentrx.com.

About Texas Children's Hospital
Texas Children's Hospital, a not-for-profit health care organization, is committed to creating a healthier future for children and women throughout the global community by leading in patient care, education and research. Consistently ranked as the best children's hospital in Texas, and among the top in the nation, Texas Children's has garnered widespread recognition for its expertise and breakthroughs in pediatric and women's health. The hospital includes the Jan and Dan Duncan Neurological Research Institute; the Feigin Tower for Pediatric Research; Texas Children's Pavilion for Women, a comprehensive obstetrics/gynecology facility focusing on high-risk births; Texas Children's Hospital West Campus, a community hospital in suburban West Houston; and Texas Children's Hospital The Woodlands, the first hospital devoted to children's care for communities north of Houston. The organization also created Texas Children's Health Plan, the nation's first HMO for children; Texas Children's Pediatrics, the largest pediatric primary care network in the country; Texas Children's Urgent Care clinics that specialize in after-hours care tailored specifically for children; and a global health program that's channeling care to children and women all over the world. Texas Children's Hospital is affiliated with Baylor College of Medicine. For more information, go to www.texaschildrens.org. Get the latest news by visiting the online newsroom and Twitter at twitter.com/texaschildrens.

SOURCE EpicentRx, Inc.