Patients with active thyroid eye disease (TED) could benefit from rapid and significant improvements after positive data emerged from a clinical trial.
Viridian Therapeutics made the announcement yesterday (August 15) that the 10mg/kg cohort in its ongoing phase 1/2 trial of VRDN-001, an anti-IGF-1R antibody TED – an autoimmune disease where the body’s own immune system attacks the tissues around and behind the eyes causing inflammation, swelling and damage.
Intravenously
This can develop into signs and symptoms of double vision, bulging eyes and ocular pain. The double-blind, placebo controlled trial is evaluating two infusions of the drug administered intravenously.
The inclusion and exclusion criteria and the baseline patient characteristics for this trial are similar to prior thyroid eye disease clinical trials. Efficacy measurements include proptosis (bulging eyes), Clinical Activity Score (CAS), and diplopia (double vision), which are the same endpoints as measured in the clinical development of Tepezza, the only approved therapy targeting IGF-1R in patients with TED.
Raymond Douglas director of the Orbital and Thyroid Eye Disease Program, Cedars-Sinai Medical Center, said: “TED is a severe, debilitating eye disease that threatens patients’ vision and overall quality of life.
Significant improvement
“In this trial, rapid and significant improvement in a broad set of efficacy measures suggests that VRDN-001 may have a differentiated profile that could offer meaningful benefits to patients.”
The company said the ongoing trial is evaluating two infusions of VRDN-001, three weeks apart, with efficacy measured six weeks after the first dose. Each dose is evaluated in a cohort of eight patients, randomized so that six patients receive VRDN-001 and two patients receive placebo.
The first cohort evaluated a dose of 10mg/kg, with initial clinical data being reported today. The second cohort is evaluating a dose of 20mg/kg and enrollment is nearly complete, and the company plans to report results at an upcoming medical meeting in the fourth quarter of 2022. A third cohort will evaluate a dose of 3mg/kg, with data anticipated in the fourth quarter of 2022.
Hyperglycemia
VRDN-001 was well-tolerated by all patients treated at the 10mg/kg dose. There were no reported serious adverse events (SAEs), no patient discontinuations, and no hyperglycemia or infusion reactions as of August 9, 2022, the cutoff date for follow up observation. Two cases of mild muscle spasm were reported and did not require intervention. There was one report of ringing in the ears which resolved within two weeks without intervention.
In the ongoing second TED cohort, which is evaluating two infusions of 20mg/kg of VRDN-001, no adverse events of hyperglycemia, muscle spasm, hearing impairment, infusion reactions, or any serious adverse events were reported as of the cutoff date of August 9, 2022.