Tell me about VRDN-001.
VRDN-001 is a monoclonal antibody that blocks cell surface receptor activity known as insulin-like growth factor-1 receptor (IGF-1R), leading to a potential reduction in tissue swelling and inflammation associated with TED. (via)
What’s the goal of the trial?
The phase 1 proof-of-concept study consists of multiple randomized, placebo-controlled cohorts assessing the potential for VRDN-001 to deliver rapid improvement of TED symptoms—particularly proptosis.
I need more details.
The phase 1/2 trial is analyzing two injection infusions of VRDN-001 given three weeks apart. Efficacy is measured 6 weeks following the first dose and includes: proptosis, clinical activity score (CAS), and diplopia.
The first cohort evaluated a 10 mg/kg dose of VRDN-001 and the second cohort evaluated a 20 mg/kg dosage. (via)
What is the data showing?
VRN-001 was found to be well-tolerated among all patients treated with the 20 mg/kg dose; safety data for 20 mg/kg was also consistent with the 10 mg/kg dose.
No serious adverse reactions, patient discontinuations, hearing impairment, drug-related hyperglycemia, or infusion reactions were reported. Improvements in proptosis, CAS, and diplopia were also generally consistent between the cohorts.
Give me some numbers.
Overall, researchers reported a 75% overall responder rate between the two cohorts. Activity recorded for all patients at week 6 included:
- An average proptosis responder rate of 75% and a 2.04 mm mean reduction average from baseline.
- A 4-point mean reduction in CAS from baseline on a 7-point measure of TED symptoms, with 58% of patients achieving maximal or near-maximal therapeutic effect on CAS.
- A 75% complete resolution of diplopia, defined as patients with baseline diplopia achieving a score of 1 on the Gorman subjective diplopia scale. (via)
Anything else?
Additional results included 12-week data from the 10 mg/kg cohort, which showed VRDN–001 remained effective after two infusions for 6 additional weeks.
What’s next?
Viridian expects to enroll the first patients in two studies next month: the chronic TED proof-of-concept study, with preliminary data by the first half of 2023; and the phase 3 THRIVE study, with results expected by 2024.
The company also plans to release topline data from its 3 mg/kg cohort of active TED patients by January 2023.