Viridian Therapeutics, Inc. announced positive preliminary data from its ongoing Phase 1/2 clinical trial of VRDN-001, an investigational full antagonist antibody to the insulin-like growth factor 1 receptor (IGF-1R), in patients with chronic thyroid eye disease (TED). The Company also announced an amendment to its ongoing THRIVE Phase 3 trial design and provided an update on recent progress of its SC program candidates in TED. VRDN-001 – Phase 1/2 proof-of-concept trial in chronic TED: The proof-of-concept portion of the double-masked, placebo-controlled Phase 1/2 trial evaluated two infusions of VRDN-001 administered intravenously (IV), three weeks apart, with clinical activity endpoints measured six weeks after the first infusion.

VRDN-001 was evaluated at doses of 10 and 3 mg/kg, with each cohort designed to include six patients randomized to drug, and two patients randomized to placebo. Participant eligibility criteria included: chronic TED with documented evidence of ocular symptoms or signs that began more than one year prior to screening (mean duration of 7.8 years), and proptosis of =3 mm above normal values for gender and race. Any clinical activity score (CAS, 0 – 7) was allowed for randomization (mean CAS was 3.3).

VRDN-001 – Safety data: VRDN-001 was generally well tolerated by all drug treated patients in both dose cohorts. There were no reported serious adverse events (SAEs), including no hearing impairment or hyperglycemia events, in patients with chronic TED treated with VRDN-001 as of May 30, 2023, the most recent cutoff date for follow-up observation. The safety and tolerability profile was generally consistent with previously reported results in patients with active TED treated with VRDN-001.

VRDN-001 – Clinical activity data in chronic TED: Patients in the 10 mg/kg (n=6) and 3 mg/kg (n=6) cohorts who were treated with two doses of VRDN-001 were evaluated for changes in proptosis, CAS, and diplopia at week 6. Proptosis changes from baseline were measured both by exophthalmometry and magnetic resonance imaging (MRI, an exploratory measure). Viridian believes exophthalmometry and MRI together provide a robust assessment of changes in proptosis. Diplopia: Five out of the twelve VRDN-001 treated patients across both dose cohorts had diplopia (double vision) at baseline.

None of the patients treated with VRDN-001 achieved complete resolution of diplopia at week 6, defined as patients with baseline diplopia who achieved a score of 0 on the Gorman subjective diplopia scale.