TRACON Pharmaceuticals announces orphan drug designation for envafolimab in soft tissue sarcoma

TRACON Pharmaceuticals Inc (NASDAQ:TCON) has announced that the US Food and Drug Administration (FDA) has granted orphan drug designation to envafolimab, a novel, single-domain antibody against PD-L1. Envafolimab is the first subcutaneously injected PD-L1 inhibitor to be studied in pivotal trials. The antibody gained orphan drug status from the FDA following TRACON's submission of an amended application that included Phase 1 clinical trial data from sarcoma patients treated with single-agent envafolimab. Clinical trial data were submitted in response to an FDA request to provide data using envafolimab to treat patients with soft tissue sarcoma that demonstrated a therapeutic effect, the company added. READ: TRACON Pharmaceuticals presents Phase 1 study data of uliledlimab and atezolizumab at American Society of Clinical Oncology meeting Orphan drug designation is granted by the FDA to drugs or biologics intended to treat a rare disease or condition, defined as one that affects fewer than 200,000 people in the US. Programs with orphan drug status receive partial tax credit for clinical trial expenditures, waived user fees and eligibility for seven years of marketing exclusivity. “The receipt of orphan drug designation is one of multiple milestones we expect this year for envafolimab, including interim efficacy data from the pivotal ENVASARC trial in the second half of 2021,” TRACON president and CEO Dr Charles Theuer said in a statement. “The 40% response rate demonstrated by envafolimab in ASPS patients is similar to the 42% response rate reported by the National Cancer Institute in ASPS patients treated with the PD-L1 antibody Tecentriq, which is consistent with data in MSI-H colorectal cancer, suggesting that subcutaneously administered envafolimab is as active as approved intravenously administered PD-1 antibodies.” The orphan drug designation submission for envafolimab in sarcoma included clinical data demonstrating confirmed objective partial responses by RECIST with duration of response in excess of six months, in two of five patients with refractory metastatic alveolar soft part sarcoma (ASPS) who received single-agent envafolimab in Phase 1 clinical trials conducted by TRACON’s partners 3D Medicines and Alphamab Oncology. Patients with undifferentiated pleomorphic sarcoma (UPS) or myxofibrosarcoma (MFS) were not treated as part of Phase 1 trials. TRACON develops targeted therapies for cancer utilizing a capital-efficient, CRO-independent, product development platform. The company’s clinical-stage pipeline includes Envafolimab; TRC102, a Phase 2 small molecule drug candidate for the treatment of lung cancer; and TJ004309, a CD73 antibody in Phase 1 development for the treatment of advanced solid tumors. Contact the author at stephen.gunnion@proactiveinvestors.com