• FDA confirms single pivotal trial pathway for Elate Ocular, reducing development cost and accelerating timeline
• Increased probability of BLA success by concentrating statistical risk into one well-powered study
• Elate Ocular holds FDA Fast Track Designation, enabling rolling BLA submission

Cambium Bio Limited (ASX:CMB), a clinical-stage regenerative medicine company, announced that the U.S. Food and Drug Administration (FDA) has confirmed that a single adequate and well-controlled pivotal clinical study, together with confirmatory evidence, is a reasonable approach to support a Biologics License Application (BLA) for Elate Ocular for the treatment of moderate-to-severe dry eye disease. This confirmation aligns the Elate Ocular development programme with the FDA's updated regulatory policy, which established a single pivotal clinical trial plus confirmatory evidence as the new default standard for marketing authorisation. The FDA also confirmed the previously agreed randomised, double-masked, vehicle-controlled pivotal study design, including the co-primary sign and symptom endpoints, n=400 evaluable subjects, and 9-week masked treatment period. The single-trial pathway materially reduces the capital required to progress Elate Ocular to BLA submission, accelerates the timeline to potential approval by consolidating clinical sites and patient recruitment, and improves the overall probability of generating a successful BLA data package. Cambium Bio's immediate priority is to initiate the single n=400 pivotal Phase 3 study as soon as operationally feasible, with First Patient In expected later in 2026. The specific confirmatory evidence package to be submitted as part of the BLA will be determined in consultation with the FDA following Phase 3 topline data.

Cambium Bio's operational focus is now to initiate the single pivotal Phase 3 study as soon as operationally feasible, with First Patient In expected later in 2026, and to drive the programme efficiently toward topline readout and BLA submission.