Gibson’s Novel Anti-cancer Drug, LMP-400, Receives FDA Rare Pediatric Disease Designation for the Treatment of Ewing Sarcoma

On December 12, 2018 Gibson Oncology, LLC ("Gibson"), a privately held clinical stage company developing a novel class of oncology drugs for treating adult and pediatric cancers resistant to traditional cancer drugs, reported that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to LMP-400, Gibson’s novel small molecule topoisomerase 1inhibitor for the treatment of Ewing sarcoma, a rare pediatric cancer (Press release, Gibson Oncology, DEC 12, 2018, View Source [SID1234532034]). LMP-400 has successfully completed multiple phase I human clinical trials as a single agent in advanced stage cancer patients with demonstrated anti-cancer activity and improved safety attributes over first generation topoisomerase inhibitors.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Mr. Randall Riggs, President & CEO of Gibson Oncology, stated that "Gibson Oncology is excited that the U.S. FDA has granted LMP-400 RPDD, which has the potential to greatly benefit kids diagnosed with Ewing Sarcoma. We intend to conduct phase 2 trials with LMP-400 that will employ a recently discovered biomarker called Schlafen11 to select pediatric patients most likely to respond."

In a previous study performed at the National Cancer Institute (NCI) by Dr. Pommier et. al., cancer cells expressing high levels of Schlafen11 were hypersensitive to LMP-400. Ewing Sarcoma patients commonly have very high expression levels of Schlafen11 and are therefore excellent candidates for treatment with LMP-400.

The FDA grants RPDD for diseases that primarily affect children from birth to age 18, and affect fewer than 200,000 persons in the U.S. This program is intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.

About Ewing Sarcoma

Ewing sarcoma is the second most common bone malignancy among children and adolescents. According to a study published in the Journal of Hematology/Oncology, the incidence is about 3 cases per 1 million per year in children younger than age 20. Despite the favorable prognosis, an American Cancer Society study showed that approximately 30-40% of patients develop metastases or local recurrence, and the long-term survival rate for refractory or recurrent disease is only 22-24%. The relapsed and refractory statistics underscore the need for new treatment options.