Viridian Therapeutics Reports Positive Phase 3 Results for Veligrotug in Thyroid Eye Disease
Viridian Therapeutics, Inc., a biopharmaceutical company specializing in innovative treatments for serious and rare diseases, announced promising results from its THRIVE phase 3 clinical trial for VRDN-001, now named veligrotug. This drug, delivered intravenously, targets the IGF-1R antibody in patients suffering from active thyroid eye disease (TED), an autoimmune condition causing inflammation and tissue damage around the eyes.
Steve Mahoney, President and CEO of Viridian, expressed enthusiasm over the trial's outcomes. He highlighted the drug's efficacy and safety, noting the rapid onset of positive responses. Veligrotug’s five-infusion regimen offers a more convenient option for patients, reducing the burden compared to current treatments. Mahoney also mentioned that Viridian has initiated phase 3 trials for VRDN-003, administered subcutaneously, which they believe will complement veligrotug and potentially become a preferred treatment for TED.
Michael Yen, M.D., a trial investigator from Baylor College of Medicine, emphasized the robust clinical activity shown by veligrotug. The data revealed significant improvements in proptosis, clinical activity score, and diplopia just after five infusions. Yen also noted the favorable safety profile, which included monitoring for potential hearing impairment.
The THRIVE trial enrolled 113 patients, who were randomized to receive either veligrotug or a placebo. The drug met the primary and all secondary endpoints at 15 weeks, showing statistically significant improvements in all measured signs and symptoms of TED. Key findings include:
1. Proptosis:
- 70% of patients receiving veligrotug showed at least a 2mm reduction in eye bulging compared to 5% in the placebo group.
- A mean reduction of 2.9mm in proptosis was observed in the veligrotug group versus a 0.5mm reduction in the placebo group.
2. Diplopia (double vision):
- 54% of patients on veligrotug experienced a complete resolution of diplopia, compared to 12% on placebo.
- 63% of patients showed a positive response in diplopia symptoms against 20% in the placebo group.
3. Clinical Activity Score (CAS):
- 64% of patients achieved a CAS reduction to 0 or 1, compared to 18% in the placebo group.
- The mean reduction in CAS was 3.4 points for veligrotug recipients versus a 1.7-point reduction for placebo recipients.
Veligrotug was well-tolerated, with most adverse events being mild and a discontinuation rate of only 4%. There were no treatment-related serious adverse events. The trial also noted a 5.5% placebo-adjusted rate of hearing impairment events.
Viridian aims to submit a Biologics License Application (BLA) for veligrotug in the second half of 2025, with the second phase 3 trial, THRIVE-2, ongoing and completion expected by the end of 2024. Additionally, Viridian initiated two global phase 3 trials for VRDN-003 in August, called REVEAL-1 and REVEAL-2, to evaluate its efficacy as a subcutaneous treatment for TED. These trials will provide data by the first half of 2026, with a BLA submission projected by the end of the same year.
Viridian Therapeutics continues to advance its portfolio, which includes FcRn inhibitors like VRDN-006 and VRDN-008, aimed at treating various autoimmune diseases. The company is committed to developing best-in-class treatments and expanding its market reach for the benefit of patients with serious and rare conditions. Viridian is headquartered in Waltham, Massachusetts.
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