Viridian Therapeutics announced the release of positive topline, 6-week data from the third, low-dose cohort in the ongoing phase 1/2 clinical trial evaluating VRDN-001 in patients with active thyroid eye disease (TED).
Tell me about VRDN-001.
VRDN-001 is an anti-insulin-like growth factor 1 receptor (IGF-1R) antibody that blocks cell surface receptor activity, leading to a potential reduction in tissue swelling and inflammation associated with TED. (via)
What is this trial?
The double-blind, placebo-controlled, proof-of-concept study (NCT05176639) consists of multiple randomized, placebo-controlled cohorts assessing the potential for VRDN-001 to deliver rapid improvement of TED symptoms—particularly proptosis.
Two infusions of VRDN-001 were evaluated after being intravenously administered, 3 weeks apart. Efficacy is measured 6 weeks following the first dose and VRDN-001 is evaluated at doses 3, 10, and 20 mg/kg. Each cohort includes six participants randomized to drug and two participants randomized to placebo. (via)
What happened in cohort 3?
VRDN-001 was evaluated at a dosage of 3 mg/kg, with nine patients randomized to receive VRDN-001 in order for all patients to be eligible post-screening to participate in the trial. Two patients were randomized to receive the placebo. Before the 6-week evaluation, one patient who received placebo discontinued the trial.
What does the data show?
VRDN-001 was found to be generally well-tolerated among all patients treated within the three doses. No serious adverse events (SAEs), no hearing impairment, no drug-related hyperglycemia, no patient discontinuations, and no infusion reactions in patients treated with VRDN-001 were reported as of the most recent cut-off date (December 19, 2022) for follow-up observation.
Improvements in proptosis, CAS, and diplopia were also generally consistent between the cohorts. (via)
See here for some stats.
What’s the significance of this data?
Viridian reports that the overall data from cohorts 1, 2, and 3 points to VRDN-001 as a patient-friendly, low-volume intravenous injection, and supports the company’s plans to launch a dosing interval for VRDN-002 and VRDN-003—its half-life extended anti-IGF-14R antibodies— subcutaneous programs of up to once monthly.
What’s next?
Viridian expects to report initial VRDN-001 results from the proof-of-concept study of patients with chronic TED in the first half of 2023. It also plans to file an investigational new drug application with the FDA for VRDN-003 in Q2 of 2023—with phase 1 results expected in Q4 of 2023—and to report VRDN-002 results from patients with TED in the second half of 2023. (via)