Viridian Therapeutics Inc 2025年度报告

2025Annual ReportFraming the future ofautoimmune diseases. About ViridianTherapeutics Viridian is a biopharmaceutical company focused on discovering, developing, and commercializing potentialbest-in-class medicines for patients with serious and rare diseases. Viridian’s expertise in antibody discoveryand protein engineering enables the development of differentiated therapeutic candidates for validated drugtargets and disease-driving mechanisms in autoimmune and rare diseases. Viridian is advancing multiple late-stage, anti-insulin-like growth factor-1 receptor (IGF-1R) candidates in theclinic for the treatment of patients with thyroid eye disease (TED). The company conducted a pivotal programfor veligrotug, including two global phase 3 clinical trials (THRIVE and THRIVE-2), to evaluate its efficacyand safety in patients with active and chronic TED. Both THRIVE and THRIVE-2 reported positive topline data,meeting the primary and all secondary endpoints of each study. Viridian is also advancing elegrobart as thepotential first subcutaneous autoinjector for the treatment of TED, including two ongoing global phase 3pivotal clinical trials, REVEAL-1 and REVEAL-2, to evaluate the efficacy and safety of elegrobart in patientswith active and chronic TED. REVEAL-1 met its primary endpoint with a highly statistically significant treatmenteffect, and REVEAL-2 is on track to report topline data in Q2 2026. In addition to its IGF-1R inhibitor portfolio, Viridian is developing an anti–thyroid-stimulating hormone receptor(TSHR) program designed as a potential therapy for TED and Graves’ disease. Viridian is also advancing a novel portfolio of neonatal Fc receptor (FcRn) inhibitors, including VRDN-006 andVRDN-008, which have the potential to be developed in multiple autoimmune diseases. DIFFERENTIATED PIPELINE: TED portfolio moving towards commercial andFcRn inhibitor portfolio moving through the clinic Letter to Shareholders DEAR SHAREHOLDER, Our ambition at Viridian is to be a leader in bringing therapies to patients for thetreatment of thyroid and autoimmune diseases. We are establishing a strong foundationin thyroid eye disease (TED), while continuing to advance pipeline programs to addresspatients’ needs in other autoimmune diseases. Advancing Next-Gen Treatments for Thyroid Eye Disease Veligrotug: approaching anticipated commercial launchVeligrotug is our intravenous, monoclonal antibody that acts as a full antagonist of IGF-1R for the treatment of TED. In 2025, we advanced veligrotug toward potential regulatory approval in both the US and Europe following thestrong results we reported from the THRIVE and THRIVE-2 pivotal trials. In October 2025, we submitted ourBiologics License Application (BLA), and in December 2025, the FDA granted us Priority Review with a PDUFAtarget action date of June 30, 2026. We also submitted a Marketing Authorization Application to the EuropeanMedicines Agency in January 2026.et action date of Junicines Agency in Jan We are proud of the recognitions that veligrotug received from the FDA in 2025, including Breakthrough TherapyDesignation and Priority Review, which reflects the strength of the veligrotug clinical data. As we approach theJune PDUFA date, and in anticipation of our commercial launch, our commercial and medical affairs organizationsare ready to deliver with our experienced teams in place.cts thour commenced teams ine proud of the recation and PriorUFA date, anto deliver wWDeJuneare rea We are incredibly proud of what the Viridian team hasaccomplished in 2025 and are excited for what 2026and beyond has in store. Elegrobart: phase 3 topline results and a potential simple at-home autoinjector treatment for TEDElegrobart, previously called VRDN-003, is our next-generation, half-life extended, monoclonal antibody that acts as a full antagonist of IGF-1R for TED. Elegrobart is designed to be self-administered subcutaneously in an autoinjector. We have made significant progress to advance elegrobart as a potentially differentiated treatment option in TED.In March 2026, we reported positive topline results from REVEAL-1, our phase 3 pivotal study of elegrobart in activeTED where the trial met its primary endpoint with high statistical significance with a safety profile that indicates thatthe drug was generally well-tolerated. We believe that elegrobart has the potential to be the first subcutaneous autoinjector in TED, enabling patientsto self-administer at home while delivering clinically meaningful outcomes. If approved, we believe at-homeadministration could broaden access and expand the treated TED population. Looking ahead, we anticipate announcing topline data from REVEAL-2, our phase 3 study in chronic TED, in thesecond quarter of 2026 and submitting a Biologics License Application to the FDA for elegrobart in the first quarterof 2027. Developing Additional Treatments for Autoimmune Diseases We are building on our veligrotug and elegrobart foundation in TED