Benitec Biopharma Inc. (NASDAQ: BNTC) announced on October 5, 2026, that it has received formal feedback from the U.S. Food and Drug Administration (FDA) following a Type C meeting held in the third quarter of 2026. The meeting concerned the pivotal study design for the company's investigational gene therapy, BB-301.
The FDA has requested additional data to support the proposed open-label, single-arm pivotal study for BB-301. Specifically, the Agency has asked for information from the Benitec-sponsored OPMD Natural History Study. Benitec stated that the FDA agreed to review this information on a timeline that would allow the company to confirm the final pivotal study design in the first quarter of 2027.
Despite the request for additional data, Benitec confirmed that the pivotal trial remains on track. Patient enrollment is planned to begin in mid-2027. The company noted that the OPMD natural history study was designed to provide a rigorous foundation for the clinical development of BB-301.
In other company news, Benitec announced that it will present interim clinical results from the ongoing BB-301 Phase 1b/2a study as a late-breaking poster presentation at the European Society of Gene and Cell Therapy (ESGCT) Annual Congress in Hamburg, Germany, scheduled for October 27-30, 2026. The presentation will include 12-month follow-up data for all six patients in Cohort 1 and interim follow-up data for all three patients in Cohort 2.
