Thyroid Eye Disease Treatment Shows Positive Results in Phase 3 Trial
Viridian Therapeutics also said it has completed enrollment for a second phase 3 trial of veligrotug, THRIVE-2, and expects topline data to be available by the end of this year.
Positive results have been announced from a phase 3 trial evaluating the safety, tolerability and efficacy of veligrotug for the treatment of active thyroid eye disease (TED) in adults, Viridian Therapeutics announced in a release.1 The therapy, which was previously known as VRDN-001, is an intravenously delivered anti-insulin-like growth factor-1 receptor (IGF-1R) antibody.
Thyroid Eye Disease Treatment Shows Positive Results in Phase 3 Trial / Andriy Blokhin - stock.adobe.com
Data from the THRIVE (NCT05176639) study showed veligrotug met both primary endpoints of number of participants with treatment-emergent adverse events and proptosis responder rate. The therapy also met all of its key secondary endpoints, including change in measurement of proptosis, change in volume of orbital fat, change in volume of extraocular muscles, and change in facial fat volume.
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“These phase 3 clinical data demonstrate the robust clinical activity of veligrotug,” Michael Yen, MD, an investigator on the study and a professor of Oculoplastic Surgery and Ophthalmology at Baylor College of Medicine, said in a release.1 “The data showed substantial improvements in proptosis, clinical activity score, and diplopia in patients with TED after just five infusions, and these strong results are consistent with the clinical outcomes of IGF-1R antagonism in TED. Veligrotug also showed a rapid onset of action which I believe is meaningful for patients.”
THRIVE is a randomized, parallel assignment, multiple ascending dose phase 3 study assessing the safety, tolerability and efficacy of veligrotug. The study cohort included 113 patients, of which 75 received the therapy and 38 received placebo. Patients received either 5 infusions of veligrotug 10 mg/kg or 5 infusions of placebo over 15 weeks. Patients were included if they had moderate to severe active TED with documented evidence of ocular symptoms or signs that began within 15 months prior to screening.
The study found that patients who received veligrotug had a 70% proptosis responder rate and a 2.9 mm mean reduction in proptosis from baseline. There was a 54% complete resolution of diplopia in patients receiving veligrotug and 64% of patients achieved maximal or near-maximal therapeutic effect on the clinical activity score. Additionally, 67% of patients in the treatment arm achieved an overall response, compared to 5% in the placebo arms.
Veligrotug was seen to be generally well tolerated, with the majority of adverse events being mild in severity. There was also a low rate of hearing impairment: 16% of patients receiving veligrotug had hearing impairment, compared with 10.5% in patients receiving placebo.
Viridian said it has completed enrollment for a second phase 3 trial of veligrotug, THRIVE-2, and expects topline data to be available by the end of this year. In addition to the THRIVE trials, the company also initiated 2 global phase 3 clinical trials of veligrotug in August, REVEAL-1 and REVEAL-2. Viridian said it anticipates submitting a biologics license application for veligrotug for the treatment of TED in the second half of 2025.
“We are thrilled to see these exciting THRIVE topline results which met our high bars for both efficacy and safety,” Steve Mahoney, president and CEO of Viridian, said in a release.1 “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 utilizes a differentiated, abbreviated five infusion dosing regimen that has the potential to bring a more convenient treatment option with lower IV burden for patients, compared to the current standard of care.”
READ MORE: Eye Health Resource Center
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