Viridian Therapeutics, Inc. announced positive topline data from the elegrobart REVEAL-1 phase 3 clinical trial in patients with active thyroid eye disease (TED). Elegrobart is a subcutaneously delivered, half-life-extended monoclonal antibody targeting the insulin-like growth factor-1 receptor (IGF-1R). REVEAL-1 evaluated 2 dosing regimens, every 4 weeks (Q4W) and every 8 weeks (Q8W), and compared the results with placebo, the company said in a press release.
REVEAL-1 assessed the efficacy and safety of subcutaneous Q4W or Q8W elegrobart vs placebo in patients with active TED. The study enrolled 132 patients, randomized 1:1:1 to elegrobart Q4W (n=44), elegrobart Q8W (n=44), and placebo (n=44).
According to the company, REVEAL-1 was highly statistically significant on its primary endpoint of Q4W proptosis responder rate and the key secondary endpoint of Q4W proptosis mean change from baseline, each at week 24 as measured by exophthalmometry. REVEAL-1 also assessed elegrobart Q8W vs placebo and additional clinically relevant endpoints for both the Q4W and Q8W dosing arms.
Elegrobart was generally well-tolerated, with a safety profile consisting of generally expected adverse events from the anti-IGF-1R class, the vast majority of which were mild. Rates of hearing impairment were low in both the Q4W and Q8W treatment arms (11.3% and 2.3% placebo-adjusted rates, respectively), and all reports were of tinnitus, none of which were associated with reductions in hearing, the company said in the press release.
According to Viridian Therapeutics, topline data from REVEAL-2, the second pivotal phase 3 clinical trial of elegrobart for patients with chronic TED, remains on track to read out in Q2 2026. The company anticipates submitting a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for elegrobart in Q1 2027.
In addition, Viridian's veligrotug BLA is under Priority Review at the FDA with a Prescription Drug User Fee Act (PDUFA) target action date of June 30, 2026. Veligrotug also received both Breakthrough Therapy Designation and Priority Review from the FDA in 2025.
Finally, Viridian Therapeutics said it is approaching full launch-readiness and is on track to support the PDUFA action date next quarter, with experienced teams in place across field sales and medical affairs, market access, and patient services. The company also said it anticipates the veligrotug commercial and medical affairs infrastructure will support a potential elegrobart launch, if approved, with limited incremental investment, providing an infrastructure to support multiple treatment options for patients within the Viridian TED portfolio.