On May 24, 2022 / ENB Therapeutics, Inc., a clinical stage biotechnology company pioneering a new and differentiated class of therapeutics targeting the endothelin B receptor (ETBR) inhibitor, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for its lead product candidate, ENB-003, for the treatment of pancreatic cancer (Press release, ENB Therapeutics, MAY 24, 2022, View Source [SID1234634068]). This is the second Orphan Drug Designation award for ENB-003, which has also been granted Orphan Drug Designation for melanoma.

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"Immune checkpoint therapies have significantly improved the survival of patients with certain cancers; however, there remain a significant percentage of patients that exhibit no response to these therapies," said Sumayah Jamal, MD-PhD, President, Co-founder and CSO of ENB Therapeutics. "This is especially true for pancreatic cancer patients, where only about 3% of tumors are responsive to the leading anti-PD-1 therapy KEYTRUDA (pembrolizumab). We believe that combining our ETRB inhibitor, ENB-003, with pembrolizumab, has the potential to overcome this resistance, and provide much needed additional treatment options for patients in a broader range of pancreatic tumors."

"Our Phase 1/2 trial will exclude patients with pancreatic tumors that have the molecular alterations known as high microsatellite instability (MSI-H) or defective DNA mismatch repair (dMMR). Thus, we are targeting truly pembrolizumab resistant patients to evaluate the potential for a ENB-003 + pembrolizumab combination as a potential treatment for this population with clear and significant unmet need," said Dr. Jamal.

FDA Orphan Drug Designation is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States. This designation acts as a stimulus for the development of drugs for rare diseases through several incentives, including assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and the potential for seven years of post-approval marketing exclusivity after FDA approval.

About ENB-003

ENB-003 is a selective endothelin B receptor (ETBR) inhibitor that, in preclinical studies, enhanced the efficacy of immunotherapies such as anti-PD-1, anti-CTLA-4 and CAR T across multiple cancer types in preclinical studies. In an ongoing multi-center Phase 1/2 clinical trial, early efficacy signals suggest that ENB-003 overcomes resistance to the anti-PD-1 therapy KEYTRUDA (pembrolizumab) in heavily pre-treated drug resistant cancer patients. The Phase 2 portion of the ENB-003 + pembrolizumab combination study is expected to start in the first quarter of 2023. The trial will enroll melanoma patients with innate resistance to anti-PD-1 based immunotherapies, platinum refractory and platinum resistant ovarian cancer patients, as well pancreatic cancer patients that have failed standard of care.