Viridian Therapeutics, Inc. (VRDN) has announced positive topline data from its THRIVE Phase 3 clinical trial for VRDN-001 (veligrotug), an intravenously delivered anti-insulin-like growth factor-1 receptor (IGF-1R) antibody, for active thyroid eye disease (TED). This news has sent VRDN stock soaring in pre-market trading.
TED is an autoimmune condition that causes inflammation, growth, and tissue damage around and behind the eyes. The THRIVE trial, which involved patients with active TED, achieved all primary and secondary endpoints at 15 weeks after five infusions of veligrotug.
This promising data demonstrates the potential of veligrotug as a treatment option for TED. The study showed that veligrotug achieved a rapid onset of action, with a significant number of patients experiencing improvement in their proptosis (bulging eyes) after just one infusion.
Here’s a breakdown of the key findings from the THRIVE trial at the 15-week mark:
*
Proptosis:
A remarkable 70% of patients receiving veligrotug showed a positive response in proptosis compared to only 5% of those receiving placebo (a 64% placebo-adjusted difference). Additionally, proptosis decreased by an average of 2.9 mm from baseline in the veligrotug group compared to 0.5 mm in the placebo group (a 2.4 mm placebo-adjusted difference).*
Diplopia (Double Vision):
54% of patients treated with veligrotug experienced complete resolution of diplopia, compared to 12% in the placebo group (a 43% placebo-adjusted difference). Furthermore, 63% of veligrotug-treated patients showed a positive response in diplopia compared to 20% in the placebo group (a 43% placebo-adjusted difference).*
Clinical Activity Score (CAS):
64% of patients receiving veligrotug achieved a maximal or near-maximal therapeutic effect on CAS, which measures inflammatory signs and symptoms of TED. This is compared to only 18% of patients receiving placebo (a 46% placebo-adjusted difference). CAS showed a 3.4-point mean reduction from baseline in the veligrotug group compared to a 1.7-point reduction in the placebo group (a 1.7-point placebo-adjusted difference).Veligrotug was generally well-tolerated in the trial, with no treatment-related serious adverse events (SAEs) reported. The most common side effect was hearing impairment, occurring in 5.5% of patients compared to placebo.
Viridian Therapeutics is currently conducting a second Phase 3 trial, THRIVE-2, for patients with chronic TED. The topline data from this trial is expected by the end of 2024. Based on the positive results from the THRIVE trial, Viridian anticipates submitting a marketing application to the FDA for veligrotug for TED in the second half of 2025.
These findings highlight the potential of veligrotug as a game-changing treatment for TED. The drug’s rapid onset of action, significant efficacy across multiple endpoints, and favorable safety profile provide compelling evidence for its potential to improve the lives of patients suffering from this debilitating condition.
