ECEESPE2025 Oral Communications Oral Communications 11: Thyroid Part 1 (6 abstracts)
Efficacy and safety of veligrotug (VRDN-001), a full antagonist monoclonal antibody to IGF-1 receptor, in active thyroid eye disease (TED): THRIVE phase 3 topline results
Patrice Rodien 1 , Antonio Manuel Garrido Hermosilla 2 , Peerooz Saeed 3 , Michael Schittkowski 4 , Vickie Lee 5 , Julian Bermúdez Pío-Rendón 6 , Eckart Bertelmann 7 , Edwina Eade 8 , Marta Pérez-López 9 , Marco Sales Sanz 10 , Cathy Michalsky 11 , Abhijit Narvekar 12 & Thomas Ciulla 12
1Centre Hospitalier Universitaire d’Angers - Hématologie, Angers, France; 2Hospital Universitario Virgen Macarena, Universidad de Sevilla, Seville, Spain; 3Amsterdam UMC location AMC, Amsterdam, Netherlands; 4Universitätsmedizin Göttingen, Göttingen, Germany; 5Imperial College Healthcare NHS Trust - Western eye Hospital, London, United Kingdom; 6Metavisión Arruzafa (Hospital La Arruzafa), Cordoba, Spain; 7Department of Ophthalmology, Charité University Medicine Berlin, Berlin, Germany; 8North Shore Private Hospital, New South Wales, Australia; 9Hospital Universitario Y Politécnico La Fe, Valencia, Spain; 10Hospital Universitario Ramón y Cajal, Madrid, Spain; 11Viridian Therapeutics, Inc. at the time the work was completed, Waltham, United States; 12Viridian Therapeutics, Inc., Waltham, United States
JOINT196
Purpose: Veligrotug (VRDN-001), a full antagonist humanized monoclonal antibody to the IGF-1 receptor (IGF-1R), is an investigational treatment for thyroid eye disease (TED). Clinical and preclinical evidence indicate a central role for IGF-1R antagonism in reducing the inflammation and proptosis that occur in TED. Topline efficacy and safety results at 15 weeks were assessed from an ongoing phase 3 randomized double-masked placebo-controlled trial of veligrotug vs placebo in patients with active TED (THRIVE, NCT05176639).
Methods: Adults with moderate-to-severe active TED (onset ≤15 months, proptosis ≥3 mm, and clinical activity score [CAS] ≥3) were randomized to receive 5 IV infusions 3 weeks apart of either 10 mg/kg veligrotug or placebo. Outcomes included proptosis responder rate (PRR), defined as ≥2-mm reduction vs baseline by Hertel exophthalmometry, PRR by MRI/CT, complete resolution of diplopia, and mean changes from baseline in proptosis and CAS. Treatment-emergent adverse events (AEs) were assessed through 15 weeks, with follow-up ongoing through 52 weeks.
Results: A total of 113 patients were randomized to veligrotug (n=75) or placebo (n=38) and included in the intent-to-treat population. At baseline, mean proptosis was 23.2 mm in each group; CAS was 4.5 vs 4.8 and diplopia was present in 67% vs 68% of patients for veligrotug vs placebo. At 15 weeks, PRR by Hertel was 70% vs 5% (P<0.0001) for veligrotug vs placebo, with a mean proptosis reduction of 2.9 mm vs 0.5 mm (P<0.0001). PRR by MRI/CT was 69% vs 9% (P<0.0001) for veligrotug vs placebo, with a mean proptosis reduction of 2.9 mm vs 0.6 mm (P<0.0001). Mean CAS decreased by 3.4 vs 1.7 (P<0.0001) for veligrotug vs placebo. In patients with diplopia, complete resolution of diplopia occurred in 54% (27/50) vs 12% (3/26) (P<0.0001) for veligrotug vs placebo. AEs occurred for 66 (88%) veligrotug vs 24 (63%) placebo patients, and most were mild; 4 patients (veligrotug) had serious AEs (all unrelated to treatment). Hearing impairment AEs were reported for 12 (16%) veligrotug vs 4 (11%) placebo patients.
Conclusions: Topline results from the THRIVE phase 3 trial show 5 IV infusions of 10 mg/kg veligrotug were well tolerated and led to significant and clinically meaningful improvements in proptosis, CAS, and diplopia at 15 weeks. Additional follow-up through 52 weeks is ongoing.
Volume 110
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Endocrine Abstracts
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