Viridian Therapeutics has announced positive topline clinical data from its ongoing Phase 1/2 clinical trial evaluating VRDN-001, an anti-IGF-1R antibody, in patients with active thyroid eye disease (TED). The data, from the first two cohorts of the trial, showed significant and rapid improvement in both signs and symptoms of TED after two infusions of 20 mg/kg VRDN-001, generally consistent with 10 mg/kg results at week 6. These findings support the potential of VRDN-001 as a new treatment option for patients with TED, a rare autoimmune disease characterized by inflammation and damage to the tissues around the eyes.
VRDN-001 Phase 1/2 Trial Results
The Phase 1/2 trial is a double-blind, placebo-controlled study evaluating two intravenous infusions of VRDN-001 administered three weeks apart, with efficacy measured six weeks after the first dose. The trial includes cohorts receiving 10 mg/kg and 20 mg/kg doses of VRDN-001, compared to placebo. Key efficacy measurements include proptosis (bulging eyes), Clinical Activity Score (CAS), and diplopia (double vision), aligning with endpoints used in the clinical development of Tepezza, the only approved therapy targeting IGF-1R in TED.
Across all VRDN-001 treated patients to date:
- 75% were proptosis responders, defined as a ≥2mm reduction in proptosis from baseline as measured by exophthalmometry.
- 75% were overall responders, defined as a ≥2mm reduction in proptosis and a ≥2 point reduction in CAS.
- 58% achieved a Clinical Activity Score (CAS) of 0 or 1.
- 75% had complete resolution of their diplopia.
The company also reported 12-week data from the 10 mg/kg cohort, showing that after two infusions, VRDN-001 maintained efficacy for six additional weeks through week 12.
Safety and Tolerability
VRDN-001 was well-tolerated at the 20 mg/kg dose, with a safety profile consistent with the 10 mg/kg dose. There were no reported serious adverse events (SAEs), no patient discontinuations, no hearing impairment, no drug-related hyperglycemia, and no infusion reactions.
VRDN-002 Subcutaneous Program
Viridian is also developing VRDN-002, a novel monoclonal antibody with half-life extension technology designed for subcutaneous (SC) administration. Phase 1 data demonstrated a half-life of up to 43 days, supporting less frequent dosing. Topline SC proof-of-concept data for VRDN-002 in TED patients is expected in the second half of 2023.
Ongoing and Upcoming Milestones
Viridian has initiated a global Phase 3 THRIVE clinical trial in active TED patients. Topline data from the 3 mg/kg cohort of active TED patients is expected in early January 2023. The company anticipates enrolling the first patient in a chronic TED proof-of-concept study in December 2022, with preliminary data expected in the first half of 2023.
