Viridian Therapeutics’s veligrotug has met all primary and secondary endpoints in a Phase III trial in active thyroid eye disease (TED).
Previously known as VRDN-001, veligrotug won in the Phase III THRIVE trial (NCT05176639). It also boasted positive safety data in patients with TED, which is also known as Graves’ disease.
The news saw the Massachusetts-based company’s stock price rise by 32% on 10 September, having closed at $14.19 on 9 September and $18.75 on 10 September.
The trial met the primary and all secondary endpoints at 15 weeks after five infusions of veligrotug, showing highly statistically significant improvements in all the measured signs and symptoms of TED. Veligrotug additionally showed a rapid onset of action, with more than half of veligrotug-treated patients achieving a proptosis response after just one infusion.
After 15 weeks, the treatment group saw a 70% proptosis responder rate (PRR) compared to 5% in the placebo arm. There was a 2.9mm mean reduction in proptosis from baseline in patients receiving veligrotug, compared with a 0.5mm reduction in patients receiving a placebo.
Patients treated with veligrotug saw a 54% complete resolution of diplopia compared with 12% of patients receiving a placebo. With diplopia response, 63% achieved response in the treatment group while 20% of patients in the placebo group achieved diplopia response.
There was an overall response rate (ORR) of 67% of patients being treated with veligrotug compared with 5% of placebo patients. ORR is defined as achieving a proptosis response and a reduction in clinical activity score (CAS).
Veligrotug remained generally well-tolerated with most adverse events (AEs) being mild. There were no treatment-related serious AEs.
Viridian’s president and CEO Steve Mahoney said: “We were particularly pleased to observe the rapid onset of clinically meaningful responses across all endpoints, and veligrotug’s safety profile exceeded our expectations.”
Veligrotug is an intravenously delivered anti-insulin-like growth factor-1 receptor (IGF-1R) antibody.
The THRIVE-2 study (NCT06021054) of veligrotug in patients with chronic TED is fully enrolled with topline data expected before the end of this year. The company will then look ahead to submit a biologics licence application to the US Food and Drug Administration in H2 2025.
TED is an autoimmune condition characterised by inflammation, growth, and damage to tissues around and behind the eyes. The current standard of care is Amgen’s Tepezza (teprotumumab-trbw).
The company has also initiated two Phase III trials, REVEAL-1 and REVEAL-2, of VRDN-003 in August. VRDN-003 is a subcutaneous, half-life-extended anti-IGF-1R antibody with the same binding domain as veligrotug.
