Independent Data Monitoring Committee Recommendation was Based on Review of Safety and Efficacy; Enrollment Expected to Complete in 2H 2026
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Vedanta Biosciences, a late clinical-stage biopharmaceutical company developing microbiome-based oral therapies for gastrointestinal diseases, today announced that the independent Data Monitoring Committee (DMC) overseeing RESTORATiVE303, the pivotal Phase 3 trial of VE303 for prevention of recurrent Clostridioides difficile infection (CDI), has completed its first prespecified interim analysis and recommended that the study continue without modification.
The interim analysis was triggered when 50% of the planned enrollment of individuals with one or more recurrences of CDI at baseline had reached the Week 8 primary efficacy timepoint. Following an unblinded review of the accumulated safety and efficacy data, the DMC determined that efficacy to date has exceeded the prespecified futility threshold and that no significant adverse events or new safety signals had emerged. The DMC therefore recommended that the study continue as planned.
Upcoming RESTORATiVE303 Study Milestones
Vedanta anticipates completing enrollment of the RESTORATiVE303 study in the second half of 2026. A second interim analysis is also planned for earlier in the second half of 2026. The study is recruiting patients who have had one or more recurrences of CDI at baseline across more than 150 sites in approximately 20 countries.
About RESTORATiVE303
RESTORATiVE303 is a randomized, double-blind, placebo-controlled, multinational Phase 3 clinical trial evaluating the safety and efficacy of VE303 in patients at high risk for recurrence of Clostridioides difficile infection (CDI) following a course of antibiotics for a prior CDI episode. The primary endpoint is the CDI recurrence rate at Week 8 in the VE303 and placebo groups. Participants are randomized 2:1 to receive a 14-day course of VE303 or matching placebo. The study is enrolling patients who are experiencing recurrent CDI, including many with first recurrence and a significant proportion receiving fidaxomicin for their qualifying CDI episode, reflecting the evolving standard of care in CDI management. The RESTORATiVE303 trial is intended to form the basis for a Biologics License Application (BLA) to be submitted to the U.S. Food and Drug Administration (FDA).
The Data Monitoring Committee (DMC) is an independent committee of clinical, scientific, and biostatistical experts who have no affiliation with Vedanta Biosciences. Consistent with standard practice for blinded, controlled trials, the DMC is the only body with access to unblinded interim data. Its mandate is to review accumulating safety and efficacy data at prespecified intervals and to recommend to the sponsor whether the trial should continue as planned, be modified, or be stopped. A recommendation to continue the study as planned indicates that the data reviewed are within the prespecified statistical boundaries required to complete the trial with the protocol-defined sample size and power to detect the primary endpoint.
About VE303
VE303 is an orally administered, potential first-in-class live biotherapeutic product (LBP) for the prevention of recurrent Clostridioides difficile infection (CDI). It consists of a defined bacterial consortium of eight strains that were rationally selected using Vedanta’s product engine. VE303 is produced from pure, clonal bacterial cell banks, which yield a standardized drug product in powdered form and bypass the need to rely on donor fecal material of inconsistent composition. In the Phase 2 CONSORTiUM study, high-dose VE303 achieved a 30.5% adjusted absolute risk reduction in CDI recurrence versus placebo, representing a greater than 80% reduction in the odds of recurrence. VE303 has received Orphan Drug Designation (2017) and Fast Track Designation (2023) from the FDA. This project has been funded in part with federal funds from the U.S. Department of Health and Human Services (HHS); Administration for Strategic Preparedness and Response; Biomedical Advanced Research and Development Authority, under contract number 75A5012C00177.
About Recurrent C. difficile Infection
Clostridioides difficile infection is a potentially life-threatening gastrointestinal infection affecting up to 175,000 patients annually in the United States, resulting in approximately 20,000 deaths each year. Patients who experience an initial CDI episode are at significant risk of recurrence, with each recurrence increasing the likelihood of subsequent episodes. Recurrent CDI is associated with substantial morbidity, prolonged hospitalization, and diminished quality of life. There remains a significant unmet need for safe and effective therapies that address the underlying dysbiosis driving recurrence.
About Vedanta Biosciences
Vedanta Biosciences is a late clinical-stage biopharmaceutical company developing a new category of microbiome-based oral therapies for gastrointestinal diseases. Vedanta’s lead program, VE303, is currently being evaluated in the global Phase 3 RESTORATiVE303 registrational study for the prevention of recurrent C. difficile infection.
Contacts
Media
Nichole Bobbyn
Ten Bridge Communications
[email protected]
Investors and Media
Chris Brinzey
+1 617-835-9304
[email protected]
