Phase 3 trial: Positive topline results for elegrobart for the treatment of TED

Viridian Therapeutics recently announced the phase 3 REVEAL-2 clinical trial results achieved by elegrobart for treating chronic thyroid eye disease (TED). Elegrobart is a subcutaneously delivered, half‑life‑extended monoclonal antibody targeting the insulin-like growth factor-1 receptor (IGF-1R), for the treatment of chronic TED, according to the company.

The phase 3 trial met its primary endpoint, ie, the proptosis responder rate, with a significant treatment effect that indicated that elegrobart delivers rapid and clinically meaningful benefits for patients with chronic TED compared with placebo. The study evaluated 2 dosing regimens: treatment every 4 weeks and treatment every 8 weeks.

A total of 204 patients were enrolled in the clinical trial: 70 patients were randomized to every-4-week dosing, 68 patients to every-8-week dosing, and 66 patients to placebo.

Key takeaways from the phase 3 REVEAL-2 clinical trial

The every-4-week and every-8-week dosing regimens achieved respective proptosis responder rates of 50% and 54% at week 24, in contrast to the 15% placebo responder (P < 0.0001 for both comparisons).

The every-4-week regimen achieved a 61% diplopia responder rate at week 24 compared with 38% for placebo (P = 0.0118).

The safety profile of elegrobart, which showed that patients receiving both dosing regimens generally tolerated the drug well, was consistent with the findings of the REVEAL-1 study and showed low rates of hearing impairment.

Elegrobart is the only subcutaneously delivered treatment that achieved positive phase 3 data in clinical trials that evaluated the effects in patients with active and chronic TED and has the potential to be the first autoinjector treatment for TED. Biologics License Application (BLA) submission is on track for the first quarter of 2027, according to a press release.

John Mandeville, MD, PhD, an oculoplastic surgeon at Ophthalmic Consultants of Boston and a clinical associate at the Massachusetts General Hospital, Boston, commented, “Chronic TED remains a challenging condition. Many patients have been living with this disease for years or decades and would benefit from an effective and convenient treatment option. These REVEAL 2 results demonstrate the potential for elegrobart to provide meaningful improvement in the signs and symptoms of TED in as few as three doses. What’s more, a simple autoinjector that patients can use at home could be an attractive option for many patients living with chronic disease.”

Additional research into TED

Veligotug, another product of Viridian Therapeutics considered its lead program for TED, is described as a potential intravenously delivered, anti-IGF-1R antibody for the treatment of active and chronic TED.

The press release also announced that veligrotug is on Track with a Prescription Drug User Fee Act target action date of June 30, 2026.

The company anticipates the veligrotug commercial and medical affairs infrastructure will support a potential elegrobart launch, if approved, with limited incremental investment, according to the press release.

The FDA granted veligrotug Breakthrough Therapy Designation, and its BLA is under Priority Review at the FDA.