On June 29, 2021 TRACON Pharmaceuticals (NASDAQ: TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted cancer therapeutics and utilizing a cost efficient, CRO-independent product development platform to partner with ex-U.S. companies to develop and commercialize innovative products in the U.S., reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to envafolimab, a novel, single-domain antibody against PD-L1, for the treatment of patients with soft tissue sarcoma following submission of an amended application that included Phase 1 clinical trial data from sarcoma patients treated with single agent envafolimab (Press release, Tracon Pharmaceuticals, JUN 29, 2021, View Source [SID1234584469]). 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.
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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.
"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," said Charles Theuer, M.D., Ph.D., President and CEO of TRACON. "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."
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 United States. Programs with Orphan Drug status receive partial tax credit for clinical trial expenditures, waived user fees and eligibility for seven years of marketing exclusivity.
About Envafolimab
Envafolimab (KN035), a novel, single-domain antibody against PD-L1, is the first subcutaneously injected PD-(L)1 inhibitor to be studied in pivotal trials. Envafolimab is currently being studied in the ENVASARC Phase 2 pivotal trial in the U.S. sponsored by TRACON, has been studied in a completed Phase 2 pivotal trial as a single agent in MSI-H/dMMR advanced solid tumor patients in China and is being studied in an ongoing Phase 3 pivotal trial in combination with gemcitabine and oxaliplatin in advanced biliary tract cancer patients in China, with both Chinese trials sponsored by TRACON’s corporate partners, Alphamab Oncology and 3D Medicines. Alphamab Oncology and 3D Medicines submitted an NDA to the NMPA in China for envafolimab in MSI-H/dMMR cancer that was accepted for review in December 2020 and granted priority review in January 2021. In the Phase 2 MSI-H/dMMR advanced solid tumor trial, the confirmed objective response rate (ORR) by blinded independent central review in MSI-H/dMMR colorectal cancer (CRC) patients treated with envafolimab who failed a fluoropyrimidine, oxaliplatin and irinotecan was 32%, which was similar to the 28% confirmed ORR reported in the Opdivo package insert in MSI-H/dMMR CRC patients who failed a fluoropyrimidine, oxaliplatin, and irinotecan and the 33% confirmed ORR reported for Keytruda in MSI-H/dMMR CRC patients who failed a fluoropyrimidine, oxaliplatin and irinotecan in cohort A of the KEYNOTE-164 clinical trial.
About ENVASARC (NCT04480502)
The ENVASARC pivotal trial is a multi-center, open label, randomized, non-comparative, parallel cohort study at approximately 25 top cancer centers in the United States that began dosing in December 2020. TRACON expects the trial to enroll 160 patients with UPS or MFS who have progressed following one or two lines of prior treatment and have not received an immune checkpoint inhibitor, with 80 patients enrolled into cohort A of treatment with single agent envafolimab and 80 patients enrolled in cohort B of treatment with envafolimab and Yervoy. The primary endpoint is ORR by blinded independent central review with duration of response a key secondary endpoint.