Thyroid eye disease candidate proves promising in phase 1/2 trial

Viridian Therapeutics unveils positive data from its ongoing Phase 1/2 trial evaluating low dose VRDN-001 in patients diagnosed with TED.

Viridian Therapeutics Inc. recently announced positive results from the first two dose cohorts, which demonstrated a favorable safety profile.

Viridian Therapeutics Inc. this week announced positive topline clinical data from the third, low dose cohort in its ongoing Phase 1/2 clinical trial of VRDN-001, an anti-insulin-like growth factor 1 receptor (IGF-1R) antibody, in patients with active thyroid eye disease (TED).

According to the company, the data validate the clinical activity of VRDN-001 and also supports the proposed dosing interval for its VRDN-002 and VRDN-003 subcutaneous programs of up to once monthly.

“The rapid and meaningful improvements in signs and symptoms of TED observed with a low dose of VRDN-001 reinforce previously reported findings in this trial, and suggest that VRDN-001 may offer a differentiated efficacy profile,” Roger Turbin, MD, professor of Ophthalmology and Visual Science within the Department of Ophthalmology of Rutgers New Jersey Medical School, and an investigator on the VRDN-001 trial. Said in the news release. “The data also support development of VRDN-001 as a patient-friendly low volume subcutaneous injection, which could reduce the burden of care for patients suffering from TED.”

The ongoing trial examined two intravenous infusions of VRDN-001 administered intravenously, three weeks apart, with efficacy measured six weeks after the first dose. VRDN-001 was evaluated at doses of 3, 10, and 20 mg/kg, with each cohort designed to include six patients randomized to drug, and two patients randomized to placebo.

The company recently announced positive results from the first two dose cohorts, which demonstrated a favorable safety profile. A third cohort evaluated a VRDN-001 dose of 3 mg/kg with 6-week data announced this week.

According to the news release, in the 3 mg/kg dose cohort, nine patients were randomized to receive VRDN-001 to enable all consented patients who were eligible following screening to participate in the trial, and two patients were randomized to receive placebo. One patient receiving placebo discontinued in the trial prior to the 6-week evaluation.

The company also noted in its news release that the trial showed VRDN-001 was “generally safe and well-tolerated by all patients treated in the three dose cohorts.”

Moreover, the trial showed that there were no reported serious adverse events (SAEs), no discontinuations, and no infusion reactions in patients treated with VRDN-001 as of December 19, 2022, the most recent cut-off date for follow-up observation. The safety and tolerability profile at the 3 mg/kg dose level was generally consistent with previously reported results.

The company noted that VRDN-001 is also being evaluated in Phase 2 trial cohorts in patients with chronic TED. Pending positive results, the company plans to start its THRIVE-2 Phase 3 trial in patients with chronic TED.

According to the news release, the company is advancing VRDN-002, a distinct anti-IGF-1R antibody incorporating half-life extension technology, and VRDN-003, a half-life extended version of VRDN-001. Both VRDN-002 and VRDN-003 are designed for administration as convenient, low-volume, subcutaneous injections.

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