veligrotug (VRDN-001) / Sanofi, Viridian Therap, Zenas BioPharma, AbbVie, ZAI Lab, Kissei - LARVOL DELTA

Preclinical pharmacology, pharmacokinetics, and pharmacodynamics of veligrotug, a full antagonist antibody to the IGF-1 receptor in development for thyroid eye disease. (PubMed, MAbs) - "Binding epitope and antagonist properties were compared to teprotumumab (Tepezza®), a marketed anti-IGF-1R antibody. Veligrotug's pharmacologic, pharmacokinetic, and pharmacodynamic characteristics make it a good candidate for clinical development. Indeed, efficacy data at week 15 from two Phase 3 pivotal studies of veligrotug, THRIVE and THRIVE-2, showed statistically significant improvements in TED symptoms based on primary and secondary outcomes."

Journal • PK/PD data • Preclinical • Endocrine Disorders • Grave’s Disease • Inflammation • Ocular Inflammation • Ophthalmology • Thyroid Eye Disease • IGF1 • IR

Viridian Therapeutics Announces Successful October Submission of Biologics License Application (BLA) to U.S. FDA for Veligrotug in Thyroid Eye Disease (Businesswire) - "The BLA submission is supported by data from two pivotal phase 3 clinical trials, THRIVE and THRIVE-2, evaluating the efficacy and safety of veligrotug in patients with active and chronic TED, respectively....Viridian’s BLA includes a request for Priority Review, which if granted, could accelerate FDA's review timing for a potential mid-2026 veligrotug commercial launch, if approved."

FDA filing • Launch US • Thyroid Eye Disease

An Efficacy, Safety, and Tolerability Study of Veligrotug (VRDN-001), in Participants With Chronic Thyroid Eye Disease (TED) (THRIVE-2) (clinicaltrials.gov) - P3 | N=188 | Completed | Sponsor: Viridian Therapeutics, Inc. | Active, not recruiting ➔ Completed

Trial completion • Ophthalmology • Thyroid Eye Disease

VRDN-001-302: An Open-label Study (OLE) for Non-responders of VRDN-001-101 and VRDN-001-301 (clinicaltrials.gov) - P3 | N=143 | Completed | Sponsor: Viridian Therapeutics, Inc. | Active, not recruiting ➔ Completed

Trial completion • Ophthalmology • Thyroid Eye Disease

THRIVE-2 Phase 3 Trial of Veligrotug (VRDN-001), a Full Antagonist Humanized Monoclonal Antibody to IGF-1R: Efficacy, Safety, and Quality of Life at 15 Weeks in Chronic Thyroid Eye Disease (TED) (ATA 2025) - P3 | "Primary results from THRIVE-2 in chronic TED show veligrotug was generally well-tolerated, with rapid onset and significant treatment effect in proptosis, diplopia, and CAS and clinically meaningful improvement in GO-QoL outcomes at 15 weeks. Results suggest the promising potential of veligrotug in chronic TED. Follow-up through 52 weeks is ongoing."

Clinical • HEOR • P3 data • Ophthalmology • Thyroid Eye Disease

Viridian Therapeutics Announces Collaboration and License Agreement with Kissei Pharmaceutical to Develop and Commercialize Veligrotug and VRDN-003 in Japan with an Upfront Payment of $70 Million and up to $315 Million in Milestone Payments (Viridian Therap Press Release) - "Viridian Therapeutics...announced that it has entered into an exclusive collaboration and license agreement with Kissei Pharmaceutical Co., Ltd. ('Kissei') to develop and commercialize veligrotug and VRDN-003 in Japan. Both molecules are anti-insulin-like growth factor-1 receptor (IGF-1R) antibodies for the potential treatment of patients with thyroid eye disease (TED)....Under the terms of the agreement, Viridian will grant Kissei an exclusive license to develop and commercialize veligrotug and VRDN-003 in Japan. Viridian will receive an upfront cash payment of $70 million, with the potential to receive an additional $315 million in development, regulatory, and commercial milestone payments, as well as tiered royalties on net sales in Japan with percentages ranging from the 20s to mid-30s. Kissei will be responsible for all development, regulatory, and commercialization activities, and associated costs, in Japan."

Licensing / partnership • Thyroid Eye Disease

THRIVE-2 Phase 3 Trial of Veligrotug (VRDN-001) in Chronic Thyroid Eye Disease (TED): Efficacy and Safety at 15 Weeks (ENDO 2025) - P3 | "THRIVE-2, which assessed 5 IV infusions of veli vs placebo, is the first RCT in chronic TED to show statistically significant improvement not only in proptosis, but also in subjective diplopia, with a generally well-tolerated safety profile. Results suggest the promising potential of veli in chronic TED. Follow-up through 52 weeks is ongoing."

Clinical • P3 data • Inflammation • Ocular Inflammation • Ophthalmology • Thyroid Eye Disease

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 (ENDO 2025) - P3 | "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."

Clinical • P3 data • P3 data: top line • Inflammation • Ocular Inflammation • Ophthalmology • Thyroid Eye Disease • IGF1

A Safety, Tolerability and Efficacy Study of Veligrotug (VRDN 001) in Healthy Volunteers and Participants With Thyroid Eye Disease (TED) ( THRIVE ) (clinicaltrials.gov) - P3 | N=113 | Completed | Sponsor: Viridian Therapeutics, Inc. | Active, not recruiting ➔ Completed

Trial completion • Ophthalmology • Thyroid Eye Disease

THRIVE Phase 3 Trial of Veligrotug (VRDN-001), a Full Antagonist Humanized Monoclonal Antibody to IGF-1 Receptor: Efficacy, Safety, and Quality of Life in Active Thyroid Eye Disease (TED) (ATA 2025) - P3 | "Primary results from THRIVE in active TED show veligrotug was generally well tolerated, with rapid onset and significant treatment effect in proptosis, diplopia, and CAS and clinically meaningful improvement in GO-QoL outcomes at 15 weeks. Results suggest the promising potential of veligrotug in active TED. Follow-up through 52 weeks is ongoing."

Clinical • HEOR • P3 data • Ophthalmology • Thyroid Eye Disease • IGF1

THRIVE-2 phase 3 trial of veligrotug (VRDN-001) in chronic thyroid eye disease (TED): efficacy and safety at 15 weeks (SOE 2025) - P3 | "THRIVE-2, assessing 5 IV infusions of veli in chronic TED, is the first RCT to show statistically significant improvement not only in proptosis, but also in subjective diplopia, with a generally well-tolerated safety profile. These results show the potential of veli in chronic TED. Follow-up through 52 weeks is ongoing."

Clinical • P3 data • Ophthalmology

THRIVE-2 phase 3 trial of veligrotug (VRDN-001) in chronic thyroid eye disease (TED): efficacy and safety at 15 weeks (SOE 2025) - P3 | "THRIVE-2, assessing 5 IV infusions of veli in chronic TED, is the first RCT to show statistically significant improvement not only in proptosis, but also in subjective diplopia, with a generally well-tolerated safety profile. These results show the potential of veli in chronic TED. Follow-up through 52 weeks is ongoing."

Clinical • P3 data • Ophthalmology

THRIVE phase 3 topline results: efficacy and safety of veligrotug (VRDN-001), a full antagonist humanized monoclonal antibody to IGF-1 receptor, in active thyroid eye disease (TED) (SOE 2025) - P3 | "Topline results from the THRIVE phase 3 trial show 5 IV infusions of 10 mg/kg veli 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."

Clinical • P3 data • P3 data: top line • Ophthalmology • IGF1

THRIVE-2 Phase 3 Trial of Veligrotug (VRDN-001) in Chronic Thyroid Eye Disease (TED): Efficacy and Safety at 15 Weeks (AACE 2025) - "TED remains a condition with significant unmet needs, particularly in the chronic phase, where treatment options are limited. IGF-1R antagonism has emerged as a key therapeutic approach, addressing inflammation and proptosis."

Clinical • P3 data • Inflammation • Ocular Inflammation • Ophthalmology • Thyroid Eye Disease

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 (AACE 2025) - "Veligrotug, a full antagonist 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."

Clinical • P3 data • P3 data: top line • Inflammation • Ocular Inflammation • Ophthalmology • Thyroid Eye Disease • IGF1

Viridian Therapeutics Announces Positive Long-Term Durability Data from the Veligrotug Phase 3 THRIVE Clinical Trial in Patients with Active Thyroid Eye Disease (TED) (Businesswire) - P3 | N=154 | THRIVE (NCT05176639) | Sponsor: Viridian Therapeutics, Inc. | "70% of veligrotug patients (21/30) in THRIVE, who were proptosis responders at week 15 and continued follow-up to the end of the study at week 52, maintained their proptosis response. Maintenance of response is defined as responders at week 15 who still had at least a 2-millimeter (mm) reduction in proptosis compared to baseline at week 52, without worsening in the fellow eye (≥2 mm increase), as measured by exophthalmometry. There were no changes to the safety profile in the follow-up period. The vast majority of adverse events reported at the week 15 primary analysis had resolved by week 52....We continue to make great progress towards submitting the BLA in the second half of this year and preparing for a potential launch in 2026."

FDA filing • Launch US • P3 data • Thyroid Eye Disease

Viridian Therapeutics Receives FDA Breakthrough Therapy Designation for Veligrotug for the Treatment of Thyroid Eye Disease (TED) (Businesswire) - "Viridian Therapeutics...announced that the United States Food and Drug Administration (FDA) granted Breakthrough Therapy Designation to veligrotug ('veli'), the company’s lead anti-insulin-like growth factor-1 receptor (IGF-1R) drug candidate for the treatment of TED....Breakthrough Therapy Designation request based on veligrotug’s (i) consistent and robust improvement and resolution of diplopia in chronic TED, and (ii) rapid onset of proptosis response. Veligrotug met all of its primary and secondary endpoints in the pivotal THRIVE and THRIVE-2 clinical trials in active and chronic TED."

Breakthrough therapy • Thyroid Eye Disease

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 (ESPE-ESE 2025) - P3 | "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."

Clinical • P3 data • P3 data: top line • Ocular Inflammation • Ophthalmology • Thyroid Eye Disease • IGF1

Viridian Therapeutics Highlights Recent Progress and Reports First Quarter 2025 Financial Results (Businesswire) - "Biologics License Application (BLA) submission for veligrotug on track for second half 2025 with potential for U.S. launch in 2026; preparatory commercial activities underway; REVEAL-1 and REVEAL-2, phase 3 clinical trials assessing VRDN-003 in active and chronic thyroid eye disease (TED), are on track for topline data in the first half of 2026....VRDN-008, a bispecific neonatal Fc receptor (FcRn) inhibitor with an extended half-life, on track for an Investigational New Drug (IND) submission for year-end 2025."

FDA filing • IND • Launch US • P3 data: top line • Ophthalmology • Thyroid Eye Disease

THRIVE-2 Phase 3 Trial of Veligrotug (VRDN-001) in Chronic Thyroid Eye Disease (TED): Efficacy and Safety at 15 Weeks (ESPE-ESE 2025) - P3 | "THRIVE-2, which assessed 5 IV infusions of veli vs placebo, is the first RCT in chronic TED to show statistically significant improvement not only in proptosis, but also in diplopia, with a generally well-tolerated safety profile. Results suggest the promising potential of veli in chronic TED. Follow-up through 52 weeks is ongoing."

Clinical • P3 data • Ocular Inflammation • Ophthalmology • Thyroid Eye Disease

Study of ZB001 in Chinese Patients With Thyroid Eye Disease (clinicaltrials.gov) - P1 | N=17 | Completed | Sponsor: Zenas BioPharma (USA), LLC | Active, not recruiting ➔ Completed | Trial completion date: Oct 2024 ➔ Apr 2024 | Trial primary completion date: Oct 2024 ➔ Apr 2024

Trial completion • Trial completion date • Trial primary completion date • Ophthalmology • Thyroid Eye Disease • IGF1

VRDN-001-302: An Open-label Study for Non-responders of VRDN-001-101 and VRDN-001-301 (OLE) (clinicaltrials.gov) - P3 | N=143 | Active, not recruiting | Sponsor: Viridian Therapeutics, Inc. | Recruiting ➔ Active, not recruiting | Trial completion date: Mar 2026 ➔ Jul 2025

Enrollment closed • Trial completion date • Ophthalmology • Thyroid Eye Disease

STRIVE: A Randomized, Controlled, Safety and Tolerability Study of VRDN-001 in Participants with Thyroid Eye Disease (TED) (clinicaltrials.gov) - P3 | N=231 | Active, not recruiting | Sponsor: Viridian Therapeutics, Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • Ophthalmology • Thyroid Eye Disease

Veligrotug (VRDN-001), a Full Antagonist Monoclonal Antibody to IGF-1 Receptor, in Active Thyroid Eye Disease (TED): THRIVE Phase 3 Efficacy and Safety Topline Results (ARVO 2025) - P3 | "Layman Abstract (optional): Provide a 50-200 word description of your work that non-scientists can understand. Describe the big picture and the implications of your findings, not the study itself and the associated details."

Clinical • P3 data • P3 data: top line • Ocular Inflammation • Ophthalmology • IGF1

Viridian Therapeutics Highlights Recent Progress and Reports Fourth Quarter and Full Year 2024 Financial Results (Businesswire) - "TED Portfolio Progress: Veligrotug:...BLA submission is on track for the second half of 2025, with an MAA submission to the European Medicines Agency expected in the first half of 2026."

EMA filing • FDA filing • Thyroid Eye Disease