Elegrobart boosts Viridian (NASDAQ: VRDN) with strong Phase 3 TED trial win
Rhea-AI Filing Summary
Viridian Therapeutics reported positive phase 3 REVEAL-2 topline results for elegrobart, a subcutaneous anti‑IGF‑1R antibody in chronic thyroid eye disease (TED). Elegrobart achieved proptosis responder rates of 50% with every‑4‑week dosing and 54% with every‑8‑week dosing at week 24, versus 15% on placebo, with high statistical significance (p < 0.0001).
The Q4W arm also showed a 61% diplopia responder rate at week 24 compared with 38% on placebo (p = 0.0118). Across both dosing regimens, elegrobart was generally well tolerated, with low placebo‑adjusted rates of hearing impairment and 91% of treated patients completing the full course.
Viridian plans a Biologics License Application (BLA) submission for elegrobart in Q1 2027 and highlights that it is the only subcutaneous program with positive phase 3 data in both active and chronic TED. Veligrotug, its intravenous IGF‑1R antibody, has a PDUFA target action date of June 30, 2026, under Priority Review and Breakthrough Therapy Designation, while cash of $762 million as of March 31, 2026 is described as sufficient, together with anticipated milestones and revenues if approvals occur, to fund current plans through profitability.
Positive
- REVEAL-2 achieved strong efficacy in chronic TED: Elegrobart Q4W and Q8W produced 50% and 54% proptosis responder rates at week 24 versus 15% on placebo (p < 0.0001), and Q4W showed a 61% diplopia responder rate versus 38% placebo (p = 0.0118).
- Multiple late-stage catalysts and funding runway: Veligrotug has a Priority Review PDUFA date of June 30, 2026, elegrobart BLA submission is planned for Q1 2027, and Viridian reports $762M cash as of March 31, 2026, which it expects, with milestones and potential revenues, to fund plans through profitability.
Negative
- None.
Insights
Positive phase 3 TED data strengthens Viridian’s late‑stage pipeline and potential future revenue base.
Viridian now has four positive phase 3 TED trials across veligrotug and elegrobart. REVEAL‑2 in chronic TED showed proptosis responder rates of 50% and 54% for Q4W and Q8W elegrobart versus 15% on placebo, with p < 0.0001. Diplopia response in the Q4W arm reached 61% versus 38% on placebo.
Safety was generally favorable, with low placebo‑adjusted hearing‑impairment rates (4.1% and 8.8% in Q4W and Q8W) and 91% of elegrobart‑treated patients completing therapy. This supports chronic use in a population that may need well‑tolerated long‑term options.
The company plans an elegrobart BLA in Q1 2027 and is already under Priority Review for veligrotug with a June 30, 2026 PDUFA date. With $762M in cash as of March 31, 2026 and an expected $75M DRI milestone on potential U.S. veligrotug approval, Viridian states that cash, potential milestones, and future revenues, if both products are approved, are expected to fund current plans through profitability. Actual impact will depend on regulatory outcomes and market uptake once detailed data and labels are available.
8-K Event Classification
Key Figures
Key Terms
proptosis responder rate medical
diplopia responder rate medical
Biologics License Application regulatory
Priority Review regulatory
PDUFA target action date regulatory
FcRn inhibitors medical
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| Item 7.01 | Regulation FD Disclosure. |
On May 5, 2026, Viridian Therapeutics, Inc. (the “Company”) issued a press release announcing topline data from a phase 3 clinical trial of elegrobart, a subcutaneously delivered anti-insulin-like growth factor-1 receptor antibody, in patients with chronic thyroid eye disease, which the Company refers to as the REVEAL-2 trial. The Company also began using an updated corporate presentation, which includes the REVEAL-2 topline data.
A copy of the press release and the corporate presentation are furnished as Exhibit 99.1 and Exhibit 99.2, respectively, to this Current Report on Form 8-K and are incorporated herein by reference.
The information in this Item 7.01 and in Exhibits 99.1 and 99.2 to this Current Report on Form 8-K is being furnished and shall not be deemed to be “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Exchange Act or the Securities Act of 1933, as amended, regardless of any general incorporation language in such filing, except as shall be expressly set forth by specific reference in such filing.
| Item 9.01 | Financial Statements and Exhibits. |
(d) Exhibits.
| 99.1 | Press Release, dated May 5, 2026 | |
| 99.2 | Corporate Presentation, dated May 5, 2026 | |
| 104 | Cover Page Interactive Data File (embedded within the Inline XBRL document) | |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the Registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| Viridian Therapeutics, Inc. | ||||||
| Date: May 5, 2026 | By: | /s/ Stephen Mahoney | ||||
| Stephen Mahoney | ||||||
| President and Chief Executive Officer | ||||||
Exhibit 99.1
Viridian Therapeutics Announces Positive Topline Results from Elegrobart Phase 3 REVEAL-2 Clinical
Trial in Chronic Thyroid Eye Disease
- REVEAL-2 met its primary endpoint with a highly statistically significant treatment effect -
- Elegrobart Q4W and Q8W achieved 50% and 54% proptosis responder rates (PRR) at week 24, respectively, versus 15% placebo, both highly statistically significant results (p < 0.0001) -
- Elegrobart Q4W achieved a statistically significant 61% diplopia responder rate at week 24, versus 38% placebo (p = 0.0118) -
- Elegrobart was generally well tolerated in both dose groups, with a safety profile consistent with REVEAL-1 and low rates of hearing impairment -
- Elegrobart is the only subcutaneous program to demonstrate positive phase 3 data in both active and chronic TED pivotal clinical trials and has the potential to be the first autoinjector treatment for TED; BLA submission on track for Q1 2027 -
- Conference call and webcast to be held today, May 5th at 8:00 a.m. ET -
WALTHAM, Mass. – (BUSINESS WIRE) – Viridian Therapeutics, Inc. (Nasdaq: VRDN), a biotechnology company focused on discovering, developing, and commercializing potentially best-in-class medicines for autoimmune and rare diseases, today announced positive topline data from the REVEAL-2 phase 3 clinical trial of elegrobart in patients with chronic thyroid eye disease (TED). Elegrobart is a subcutaneously delivered, half-life-extended monoclonal antibody targeting the insulin-like growth factor-1 receptor (IGF-1R). REVEAL-2 evaluated two dosing regimens, every four weeks (Q4W) and every eight weeks (Q8W), compared with placebo.
“We are excited by today’s positive REVEAL-2 results and view these data as a major step forward for the chronic TED patient population. Given the IV-like proptosis response and our plans to launch with an at-home autoinjector, we believe elegrobart can meaningfully attract chronic patients to seek treatment. Elegrobart’s unmatched simplicity and convenience could uniquely drive expansion of the large and underserved chronic TED market,” said Steve Mahoney, President and Chief Executive Officer of Viridian Therapeutics. “With our anticipated launch of veligrotug, which is a short IV infusion course, and two positive phase 3 REVEAL pivotal clinical trials supporting both Q4 weekly and Q8 weekly subcutaneous dosing for elegrobart, our portfolio has the potential to offer anti-IGF-1R efficacy and safety in convenient treatment regimens for TED patients with active or chronic disease.”
“Chronic TED remains a challenging condition. Many patients have been living with this disease for years or decades and would benefit from an effective and convenient treatment option,” said John Mandeville, MD, PhD, an oculoplastic surgeon at Ophthalmic Consultants of Boston and who is also a clinical associate at the Massachusetts General Hospital. “These REVEAL 2 results demonstrate the potential for elegrobart to provide meaningful improvement in the signs and symptoms of TED in as few as three doses. What’s more, a simple autoinjector that patients can use at home could be an attractive option for many patients living with chronic disease.”
Elegrobart REVEAL-2 Phase 3 Topline Results
REVEAL-2 assessed the efficacy and safety of subcutaneous Q4W or Q8W elegrobart versus placebo in patients with chronic TED. The clinical trial enrolled 204 patients, randomized 1:1:1 to elegrobart Q4W (n = 70), elegrobart Q8W (n = 68), and placebo (n = 66).
REVEAL-2 Efficacy
REVEAL-2 met its primary endpoint for both the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) with high statistical significance (p < 0.0001). In addition, REVEAL-2 met all its proptosis key secondary endpoints in the Q4W and Q8W treatment arms with high statistical significance, and the Q4W treatment arm showed a statistically significant diplopia responder rate at week 24. Efficacy was generally consistent regardless of baseline Clinical Activity Score (CAS). Results from primary and all key secondary endpoints at week 24 are presented below:
| Elegrobart Q4W |
Elegrobart Q8W (n = 68) |
Placebo (n = 66) | ||||||
| Proptosis | Proptosis responder rate (exophthalmometry) | 50% (FDA Primary Endpoint) |
54% | 15% | ||||
| p-value | p < 0.0001 | p < 0.0001 | n/a | |||||
|
Overall responder rate (ORR)1 |
47% (EMA Primary Endpoint) |
54% | 15% | |||||
| p-value | p < 0.0001 | p < 0.0001 | n/a | |||||
| Proptosis mean change from baseline (exophthalmometry) | -1.9 mm | -2.1 mm | -0.5 mm | |||||
| p-value | p < 0.0001 | p < 0.0001 | n/a | |||||
| Diplopia | Diplopia responder rate | 61% | 55% | 38% | ||||
| p-value | p = 0.0118 | p = 0.0419 | n/a | |||||
| Diplopia complete resolution | 44% | 36% | 25% | |||||
| p-value | p = 0.0295 | p = 0.1304 | n/a | |||||
Results with p < 0.025 are statistically significant.
| 1 | Participants with both proptosis and CAS response; CAS response defined as no worsening in CAS from baseline in study eye, without deterioration in fellow eye (≥2-point increase) |
REVEAL-2 Safety
Elegrobart was generally well tolerated in REVEAL-2 with a safety profile consisting of adverse events generally expected from the anti-IGF-1R class, the vast majority of which were mild. Rates of hearing impairment were low in both the Q4W and Q8W treatment arms (4.1% and 8.8% placebo-adjusted rates, respectively). 91% of elegrobart-treated patients completed the full course of treatment, and there were no treatment-related serious adverse events (SAEs).
Elegrobart BLA Submission Expected in Q1 2027
| • | REVEAL-2 is the second successful pivotal phase 3 clinical trial for elegrobart, following positive results from REVEAL-1 in active TED. |
| • | Viridian remains on track to submit a Biologics License Application (BLA) to the U.S. FDA for elegrobart in Q1 2027. |
| • | If approved, elegrobart has the potential to offer a convenient, at-home treatment in as few as three doses for both active and chronic patients. |
Veligrotug on Track with a PDUFA Target Action Date of June 30, 2026
| • | Viridian is prepared for the planned U.S. commercial launch for veligrotug, its lead program for TED. Viridian anticipates the veligrotug commercial and medical affairs infrastructure will support a potential elegrobart launch, if approved, with limited incremental investment. |
| • | Veligrotug was granted Breakthrough Therapy Designation from the FDA, and its BLA is under Priority Review at the FDA with a Prescription Drug User Fee Act (PDUFA) target action date of June 30, 2026. |
Conference call and webcast information
Viridian will host a conference call today at 8:00 a.m. ET to discuss the REVEAL-2 topline data.
| • | Dial-in (U.S.): (800) 715-9871 |
| • | Dial-in (International): +1 (646) 307-1963 |
| • | Conference ID: 7373356 |
A live webcast of the conference call can be accessed through the “Events” section in the Investors page of the Viridian Therapeutics website. Following the live webcast, an archived version of the call will also be available on the website.
About Viridian Therapeutics
Viridian is a biotechnology company focused on discovering, developing, and commercializing potential best-in-class medicines for patients with autoimmune and rare diseases. Viridian’s expertise in antibody discovery and protein engineering enables the development of differentiated therapeutic candidates for validated drug targets 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 the clinic for the treatment of patients with thyroid eye disease (TED). The company conducted a pivotal program for veligrotug, including two global phase 3 clinical trials, THRIVE and THRIVE-2, to evaluate its efficacy and safety in patients with active and chronic TED. THRIVE and THRIVE-2 reported positive topline data, meeting their primary endpoints and all secondary endpoints. Viridian is also advancing elegrobart as the potential first subcutaneous autoinjector for the treatment of TED. Viridian is conducting an ongoing pivotal program for elegrobart, including two global phase 3 pivotal clinical trials, REVEAL-1 and REVEAL-2, to evaluate the efficacy and safety of elegrobart in patients with active and chronic TED. REVEAL-1 and REVEAL-2 reported positive topline data, meeting their primary endpoints and multiple secondary endpoints.
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 and VRDN-008, which have the potential to be developed in multiple autoimmune diseases.
Viridian is based in Waltham, Massachusetts. For more information, please visit www.viridiantherapeutics.com. Follow Viridian on LinkedIn and X.
Forward Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of words such as, but not limited to, “anticipate,” “believe,” “become,” “continue,” “could,” “design,” “estimate,” “expect,” “intend,” “may,” “might,” “on track,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” or “would” or other similar terms or expressions that concern our expectations, plans and intentions. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on our current beliefs, expectations, and assumptions. Forward-looking statements include, without limitation, statements regarding: preclinical development, clinical development, and anticipated commercialization of Viridian’s product candidates; Viridian’s expectations regarding the anticipated timing or likelihood of regulatory submissions and approvals, including the anticipated approval of the BLA for veligrotug and the anticipated submission of a BLA for elegrobart in Q1 2027; elegrobart’s potential to be the potential first subcutaneous therapy for the treatment of TED and its potential to launch commercially with an at-home autoinjector; the potential benefits of elegrobart for patients, including its viability as a compelling solution for patients living with both active and chronic disease and provide meaningful improvement in the signs and symptoms of TED in as few as three doses; Viridian’s expectations with the market size and position; that the veligrotug commercial infrastructure will support a potential elegrobart launch, if approved, with limited incremental investment; elegrobart’s potential to expand the market for products in TED, if approved; and Viridian’s product candidates potentially being best-in-class.
New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. Such forward-looking statements are subject to a number of material risks and uncertainties including but not limited to: potential utility, efficacy, potency, safety, clinical benefits, clinical response, and convenience of Viridian’s product candidates; that results or data from completed or ongoing clinical trials may not be representative of the results of ongoing or future clinical trials; that the results of ongoing or future clinical trials may not support submission for regulatory approvals; the timing, progress and plans for our ongoing or future research, preclinical, and clinical development programs; changes to trial protocols for ongoing or new clinical trials; expectations and changes regarding the timing for regulatory filings; expectations and changes regarding the timing for enrollment and data; uncertainty and potential delays related to clinical drug development; the duration and impact of regulatory delays in our clinical programs; the timing of and our ability to obtain and maintain regulatory approvals for our therapeutic candidates; manufacturing risks; competition from other therapies or products; estimates of market size; other matters that could affect the sufficiency of existing cash, cash equivalents, and short-term investments to fund operations; our financial position; our future operating results and financial performance; Viridian’s intellectual property position; that our product candidates may not be commercially successful, if approved; and other risks described from time to time in the “Risk Factors” section of our filings with the Securities and Exchange Commission (SEC), including those described in our most recent Annual Report on Form 10-K or Quarterly Report on Form 10-Q, as applicable, and supplemented from time to time by our Current Reports on Form 8-K. Any forward-looking statement speaks only as of the date on which it was made. Neither the company, nor its affiliates, advisors, or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. These forward-looking statements should not be relied upon as representing the company’s views as of any date subsequent to the date hereof.
Contacts
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Greg Rossino
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Lisa Lopez
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Source: Viridian Therapeutics, Inc.
Exhibit 99.2 Corporate Presentation May 2026
Cautionary note regarding forward-looking statements This presentation contains forward-looking statements. These statements may be identified by the use of words such as, but not limited to, “anticipate,” “believe,” “become,” “continue,” “could,” “design,” “estimate,” “expect,” “intend,” “may,” “might,” “on track,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” or “would” or other similar terms or expressions that concern our expectations, plans and intentions. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on our current beliefs, expectations, and assumptions. Forward-looking statements include, without limitation, statements regarding: preclinical development, clinical development, and anticipated commercialization of Viridian’s product candidates veligrotug, elegrobart, VRDN-006, and VRDN-008, including Viridian's view that the THRIVE and THRIVE-2 data provides support for ongoing elegrobart development; anticipated start dates of studies; anticipated data results and timing of their disclosure, including the anticipated VRDN-008 healthy volunteer clinical data; plans to communicate development plans for our product candidates; Viridian’s expectations regarding the anticipated timing or likelihood of regulatory submissions and approvals, including the anticipated approval of the BLA for veligrotug, BLA submission for elegrobart in Q1 2027, MAA submission for veligrotug, and IND submission for an anti-TSHR product candidate in Q4 2026; that Viridian plans to submit a BLA for elegrobart with both dosing regimens; that a treatment course of elegrobart could be a few as three doses, if approved; the impact of Priority Review, including the potential commercial launch of veligrotug in mid-2026, if approved; clinical trial designs; the potential utility, efficacy, potency, safety, clinical benefits, clinical response, convenience and number of indications of veligrotug, elegrobart, VRDN-006, VRDN-008 and Viridian’s anti-TSHR product candidate; the potential benefits of elegrobart for patients, including its potential to transform the treatment of patients with TED; Viridian’s expectations with respect to the market size and position, including with respect to patient adoption, of its product candidates; Viridian’s view of the strength of the THRIVE durability data and veligrotug’s robust clinical profile; Viridian’s expectations regarding the potential commercialization of veligrotug and elegrobart, if approved, including plans to launch elegrobart with a low-volume autoinjector; the potential for elegrobart to be first subcutaneous autoinjector in TED; Viridian’s ability to receive milestone payments pursuant to the 2025 royalty agreement with DRI; the potential for veligrotug and elegrobart to transform the treatment for thyroid eye disease (TED); the potential for elegrobart to be a treatment-of-choice in TED; elegrobart’s potential to expand the market for products in TED, if approved; potential market sizes and market opportunities for Viridian’s product candidates, including Viridian’s belief that veligrotug is well-positioned to become a leading product in the TED market and its FcRn portfolio has the potential to capture significant market share in autoimmune indications; Viridian’s product candidates potentially being best-in-TED; Viridian’s anticipated pipeline expansion; Viridian’s ability to expand to autoimmune disease beyond TED; and Viridian’s expectations regarding its ability to fund its current business through break-even. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. Such forward-looking statements are subject to a number of material risks and uncertainties including but not limited to: potential utility, efficacy, potency, safety, clinical benefits, clinical response, and convenience of Viridian’s product candidates; that results or data from completed or ongoing clinical trials may not be representative of the results of ongoing or future clinical trials; that preliminary data may not be representative of final data; the timing, progress, and plans for our ongoing or future research, preclinical and clinical development programs; changes to trial protocols for ongoing or new clinical trials; expectations and changes regarding the timing for regulatory filings; regulatory interactions; expectations and changes regarding the timing for enrollment and data; uncertainty and potential delays related to clinical drug development; the duration and impact of regulatory delays in our clinical programs; the timing of and our ability to obtain and maintain regulatory approvals for our therapeutic candidates; manufacturing risks; competition from other therapies or products; estimates of market size; other matters that could affect the sufficiency of existing cash, cash equivalents, and short- term investments to fund operations; our future operating results and financial performance; Viridian’s intellectual property position; the timing of preclinical and clinical trial activities and reporting results from the same; and those risks described from time to time under the caption “Risk Factors” in our filings with the Securities and Exchange Commission, including those described in our most recent Annual Report on Form 10-K or Quarterly Report on Form 10-Q, as applicable, and supplemented from time to time by our Current Reports on Form 8-K. The forward-looking statements in this presentation represent our views as of the date of this presentation. Neither we, nor our affiliates, advisors, or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this presentation. This presentation also contains estimates and other statistical data made by independent parties and by us relating to market size and other data about our industry. This data involves a number of assumptions and limitations, and you are cautioned not to give undue weight to such estimates. In addition, projections, assumptions and estimates of our future performance and the future performance of the markets in which we operate are necessarily subject to a high degree of uncertainty and risk. Trademarks used herein are the property of their respective owners. 2
Viridian aspires to be a leading autoimmune company… … starting with Best in TED In thyroid eye disease (TED), we aim to bring new treatment options to patients that address unmet needs and expand the number of treated patients 3 3
Viridian is building a portfolio to address patient needs in TED with veligrotug, elegrobart, and targeted pipeline expansion Current TED TSHR Inhibitor v Veligrotug Elegrobart Market & Pipeline Launch-ready; Potential to be first Primed for new granted Breakthrough Innovate for the subcutaneous entrants and growth Therapy Designation and future of TED autoinjector in TED Priority Review • PDUFA target: June 30, 2026 • Transformative convenience • TSHR product candidate Annualized 1 of at-home autoinjector every 4 designed to be best-in-class: ~$2B • Robust and consistent 5 TED market or 8 weeks half-life extended to support clinical responses in active extended dosing intervals in 3,4 and chronic TED • Potential to greatly expand • Low penetration with currently an autoinjector TED market, if approved • Rapid onset of treatment approved product 3,4 effect • Met primary endpoint with • Potential in TED and Graves’ • No subcutaneous option high statistical significance disease • First statistically significant available commercially in REVEAL-1 and REVEAL-2, demonstration of diplopia • Recent WW approvals are pivotal phase 3 clinical trials in resolution and response in a • Anticipated IND Q4 2026 2 expanding the global market 6 active and chronic TED global chronic TED phase 3 4 • New-start market dynamic study • Evaluating novel treatments 6 • Generally well-tolerated for the future of TED 3,4 • Limited competitive • Generally well-tolerated • BLA submission anticipated development landscape with • 12-week treatment course in Q1 2027 high bar set by IGF-1R inhibitors Veligrotug and elegrobart are investigational products that have not been approved by any regulatory authority; the safety and efficacy have not been established. 1 2 3 Source: Annualized TEPEZZA sales based on Amgen Q1 2026 Earnings, Amgen Press Release “AMGEN REPORTS FIRST QUARTER 2026 FINANCIAL RESULTS,” Viridian THRIVE data on file, 4 5 6 4 Viridian THRIVE-2 data on file, Planned product profile with commercial autoinjector format, Viridian REVEAL-1 and REVEAL-2 data on file (studies conducted with vial & syringe). BLA = Biologics License Application, IGF-1R = insulin-like growth factor-1 receptor, IND = Investigational New Drug application, TED = thyroid eye disease, TSHR = thyroid stimulating hormone receptor, WW = worldwide.
Viridian has a proven track record of execution Granted 2 >1200 TED veligrotug regulatory BLA submitted designations in patients enrolled in during the U.S. Viridian clinical 2025: 1200 2 1 government trials since January Breakthrough shutdown 2024 Therapy and Priority Review Secured access to 4 positive phase 3 Advanced FcRn up to $685M in non- TED pivotal trials portfolio: 2 INDs dilutive capital in between veligrotug $685 2 4 submitted in two 2025 between Kissei and elegrobart years Japan license and DRI royalty deals 5 Source: Viridian press releases and internal data on file. BLA = Biologics License Application, FcRn = neonatal Fc receptor, IND = Investigational New Drug application, TED = thyroid eye disease.
Strong progress across TED and FcRn inhibitor portfolios DISCOVERY PRECLINICAL PHASE 1 PHASE 2 PHASE 3 Anticipated Milestones PDUFA target date Veligrotug June 30, 2026 Anti-IGF-1R; Intravenous Thyroid Eye BLA submission Elegrobart Disease Q1 2027 Anti-IGF-1R; Subcutaneous Portfolio Anti-TSHR IND Q4 2026 Potential in TED and Graves’ disease Subcutaneous VRDN-006 Communicate dev FcRn-targeting plan in 2026 FcRn- Fc fragment Targeting Autoimmune Portfolio VRDN-008 HV data Bispecific, extended 2H 2026 half-life FcRn inhibitor 6 BLA = Biologics License Application, Fc = fragment crystallizable, FcRn = neonatal Fc receptor, HV = healthy volunteer, IGF-1R = insulin-like growth factor-1 receptor, IND = Investigational New Drug application, PDUFA = Prescription Drug User Fee Act, TED = thyroid eye disease, TSHR = thyroid-stimulating hormone receptor.
th Veligrotug PDUFA target date of June 30 and multiple additional anticipated value-creating catalysts portfolio-wide in 2026 2026 2027 MAA Submission PDUFA target date Veligrotug ü Intravenous Jan 2026 June 30, 2026 REVEAL-1 Topline REVEAL-2 Topline BLA Submission Elegrobart üü Subcutaneous Q1 2026 Q2 2026 Q1 2027 IND Anti-TSHR Subcutaneous Q4 2026 FcRn inhibitor VRDN-008 HV Data: 2H 2026 portfolio Subcutaneous Strong balance sheet: $762M cash as of Mar 31, 2026; cash, anticipated near-term DRI milestone ($75M on veli U.S. approval), and future revenues, if both veligrotug and elegrobart are approved, are expected to fund Viridian’s current business plans through profitability 7 BLA = Biologics License Application, FcRn = neonatal Fc receptor, HV = healthy volunteer, IND = Investigational New Drug application, IV = intravenous, MAA = Marketing Authorization Application, PDUFA = Prescription Drug User Fee Act, TED = thyroid eye disease, TSHR = thyroid-stimulating hormone receptor.
Viridian is building towards a leadership position in TED, enabling our expansion to other autoimmune diseases Revenue Funded Growth Autoimmune Thyroid 5 Yrs Commercial Company New Pipeline TED INDs and POCs* 2 Yrs FcRn Expansion* TSHR POC* IGF-1R FcRn POC* Ele Launch** TSHR IND* Veli Launch** Ele Topline 2026 Continue to Advance and Expand Pipeline * Planned; ** If approved. 8 Ele = Elegrobart, FcRn = neonatal Fc receptor, IGF-1R = insulin-like growth factor-1 receptor, IND = Investigational New Drug application, POC = proof of concept, TED = thyroid eye disease, TSHR = thyroid stimulating hormone receptor, Veli = Veligrotug.
Veligrotug Intravenous anti–IGF-1R 9
TED is an autoimmune condition characterized by inflammation, growth, and damage to tissues around and behind the eyes Normal Eye Anatomy Thyroid Eye Disease (TED) 1 Autoantibodies trigger IGF-1R/TSHR pathway Bulging Eyes Heterogeneous autoimmune disease with clinical signs and symptoms that can vary or modulate following onset, 2,3 in some cases for the rest of a patient’s life Main signs include proptosis (eye bulging), redness, 2,3 Optic Nerve Enlargement of extraocular muscles swelling, diplopia (double vision), and lid retraction People living with TED experience proptosis, redness, Severe cases can cause sight-threatening optic swelling, diplopia, and lid retraction 4 nerve compression An estimated 190K people in the US alone have 5 moderate to severe TED 1 2 3 Sources: George A et al. Front Endocrinol (Lausanne). 2021;11:629925., Smith TJ et al. NEJM. 2016;375(16):1552–1565., Bahn RS. NEJM. 2010; 4 5 362(8): 726–738., Bartley GB et al. Am J Ophthalmol 1996;121(3):284–290., Viridian-sponsored market research, includes active and chronic TED. TED patient images are from Bahn RS. NEJM. 2010; 362(8): 726–738. Copyright © (2010) Massachusetts Medical Society. Reprinted with permission 10 from Massachusetts Medical Society. IGF-1R = insulin-like growth factor-1 receptor, TED = thyroid eye disease, TSHR = thyroid stimulating hormone receptor.
Veligrotug PDUFA target date of June 30, 2026 Veligrotug in Active TED Veligrotug in Chronic TED ü THRIVE met its primary and all secondary ü THRIVE-2 met its primary and all secondary endpoints endpoints ü Demonstrated a rapid onset of treatment effect ü Demonstrated a rapid onset of treatment effect in in as few as 3 weeks as few as 3 weeks ü Generally well-tolerated, with a low rate of ü Generally well-tolerated, with a low rate of hearing hearing impairment impairment ü Strong durability of proptosis response: 70% of ü First pivotal phase 3 clinical trial to show topline proptosis responders maintained statistically significant diplopia response & response at week 52 resolution in chronic TED Detailed data can be found in appendix starting on slide 48 Detailed data can be found in appendix starting on slide 58 Veligrotug BLA is under Priority Review and was granted Breakthrough Therapy Designation by the FDA Veligrotug is an investigational product that has not been approved by any regulatory authority; the safety and efficacy have not been established. 11 Source: Viridian THRIVE & THRIVE-2 data on file. BLA = Biologics License Application, FDA = U.S. Food and Drug Administration, PDUFA = Prescription Drug User Fee Act, TED = thyroid eye disease.
Veligrotug’s robust clinical profile expected to drive rapid commercial adoption in TED, if approved Large & Growing Market Focused Footprint 1 • Narrow and well-defined call point supports • ~$2B single-product market in U.S. small, efficient sales force • Teprotumumab launch as first entrant: $166M net 7 sales in first full quarter of launch (2Q 2020), and • Estimated ~2,000 core prescribers in the U.S. 2 $820M in launch year • Teprotumumab launched with field force of <100 sales 8 • Over 25k patients treated to date among estimated reps 3,4,5 US prevalence of ~190K moderate to severe TED • Established market price and reimbursement • New-start market dynamic enables potential pathway rapid uptake for new entrant • Current WAC price for teprotumumab: ~$525K per 9 complete treatment course in the U.S. • Strong patient demand for new options • >1,200 TED patients enrolled in Viridian clinical trials • Established strong & deep KOL relationships 6 since January 2024 • Investigators have experience with veligrotug, one of the largest TED clinical programs to date 1 2 3 Sources: Annualized teprotumumab sales based on Amgen Q1 2026 earnings, Horizon 2Q 2020 and full-year 2020 earnings, TEPEZZA® (teprotumumab-trbw) Patient Website, 4 5 6 7 Viridian-sponsored market research, includes active and chronic TED, Amgen Q4 2025 earnings, Viridian data on file, Viridian internal claims analysis on file, 8 9 12 FiercePharma, “Horizon bulks up sales force ahead of $750M inflammatory eye drug launch,” published: June 25, 2019, Internal estimate, based on 80 kg patient. KOL = key opinion leader, TED = thyroid eye disease, Tepro = teprotumumab, WAC = wholesale acquisition cost.
Launch-ready for June 30 PDUFA target action date under Priority Review Veligrotug is well positioned to be Viridian’s Launch-ready with experienced team first commercial launch in TED Medical Affairs Field Sales Diplopia Resolution & Response: Significant impact on diplopia in chronic TED Full team engaging in Fully hired; in field scientific exchange Rapid Onset: Significant proptosis response demonstrated in as few as 3 weeks Generally Well Tolerated: Consistent profile across studies; low rate of hearing impairment AEs Patient Services Market Access Actively engaging offices Payer engagements Reduced Treatment Burden: ~70% shorter and infusion centers in ongoing with positive 1 infusion time and shorter course of therapy introductory conversations feedback on veli value Veligrotug received Breakthrough Therapy Viridian go-to-market approach is grounded in Designation (BTD) and Priority Review from FDA comprehensive understanding of the TED market Veligrotug is an investigational product that has not been approved by any regulatory authority; the safety and efficacy have not been established. 13 1 Sources: Compared with current available anti-IGF-1R treatment option. AE = adverse event, BLA = Biologic License Application, FDA = U.S. Food and Drug Administration, HCP = Health Care Professional, IV = intravenous, PDUFA = Prescription Drug User Fee Act, TED = thyroid eye disease.
Elegrobart (VRDN-003) Subcutaneous half-life extended anti–IGF-1R 14
Positive topline results from REVEAL-1 & REVEAL-2 pivotal trials in active and chronic TED for elegrobart ü Achieved primary endpoint with high statistical ü Achieved primary endpoint with high statistical significance significance ü Clinically meaningful outcomes on multiple ü Statistically significant and IV-like proptosis benefit secondary endpoints, across both Q4W and achieved in both Q4W and Q8W treatment arms Q8W treatment arms ü Meaningful benefit on diplopia in Q4W treatment arm ü Rapid onset of treatment effect ü First and only subcutaneous treatment with positive ü Generally well-tolerated with low rates of data in a pivotal chronic TED clinical trial hearing impairment ü Generally well-tolerated with low rates of hearing impairment Anticipated BLA submission in Q1 2027 for both Q4W and Q8W dosing regimens Elegrobart is an investigational product that has not been approved by any regulatory authority; the safety and efficacy have not been established. 15 Source: Viridian REVEAL-1 & REVEAL-2 data on file. BLA = Biologics License Application, IV = intravenous, Q4W = every 4 weeks, Q8W = every 8 weeks, TED = thyroid eye disease.
Both veligrotug and elegrobart offer potential for significantly improved anti-IGF-1R dosing profiles Weeks 0 1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 17 18 19 20 21 22 23 24 teprotumumab IV 60-90 min infusion Veligrotug IV 30-45 min infusion teprotumumab OBI Up to 30 min on-body infusion Elegrobart Q4W Dosed in seconds Elegrobart Q8W Dosed in seconds Comparison based on dosing or proposed dosing regimens only. No head-to-head studies have been conducted. Veligrotug and elegrobart are investigational products that have not been approved by any regulatory authority; the safety and efficacy have not been established. Source: Teprotumumab U.S. Prescribing Information; Amgen Teprotumumab OBI Topline Data Press Release on April 6, 2026; Jefferies VRDN equity research report on September 25, 2024 1 16 Planned product profile with commercial autoinjector format. IGF-1R = insulin-like growth factor-1 receptor, IV = intravenous, Q4W = every 4 weeks, Q8W = every 8 weeks. Wearable 1 Autoinjector Pen IV Infusions Pump
Elegrobart topline data & profile support its potential to transform TED treatment with BLA submission anticipated in Q1 2027 Potential to be the first Only SC program with positive data in both subcutaneous autoinjector in TED active and chronic TED pivotal clinical trials Planned simple, one-step autoinjector with each Elegrobart’s two pivotal trials met their primary dose delivered in just seconds and multiple secondary endpoints, and elegrobart was generally well-tolerated Full treatment course as few as 3 doses Uniquely positioned to expand the Potential to be treatment-of-choice TED market for TED patients Compelling proptosis & diplopia benefit Anticipated to attract new patients, underserved by with the potential to be the most convenient today’s therapies, with a simple, convenient anti- treatment in TED IGF-1R, planned for at-home self-administration Elegrobart is an investigational product that has not been approved by any regulatory authority; the safety and efficacy have not been established. 17 Source: Viridian REVEAL-1 & REVEAL-2 week 24 topline data on file (interim topline database lock). BLA = Biologics License Application, IGF-1R = insulin-like growth factor-1 receptor, SC = subcutaneous, TED = thyroid eye disease.
REVEAL-1 Topline Data in Active TED Potential first subcutaneous autoinjector for TED 18
REVEAL-1 in active TED patients met primary endpoint and elegrobart was generally well tolerated Achieved the primary endpoint with high statistical significance (p < 0.0001) – 54% of Q4W patients achieved a proptosis response versus 18% placebo at week 24 Achieved clinically meaningful outcomes on multiple secondary endpoints – 63% PRR in the Q8W arm versus 18% placebo at week 24 – 51% diplopia complete resolution in the Q4W arm versus 16% placebo, all at week 24 Rapid onset of treatment effect in as few as 4 weeks Generally well tolerated in both dose groups, with low rate of hearing impairment AEs through week 24 Elegrobart is an investigational product that has not been approved by any regulatory authority; the safety and efficacy have not been established. 19 Source: Viridian REVEAL-1 week 24 topline data on file (interim topline database lock). AE = adverse event, IGF-1R = insulin-like growth factor-1 receptor, PRR = proptosis responder rate, Q4W = every 4 weeks, Q8W = every 8 weeks, TED = thyroid eye disease.
REVEAL-1 is a phase 3 randomized, controlled, double-masked trial of elegrobart in active TED Treatment Phase (20 weeks treatment with primary endpoint at 24 weeks) 1 D1 W4 W8 W12 W16 W20 W24 Treatment Arms Follow-up through W52 (1:1:1) Primary Endpoint 1 Elegrobart Q4W Analysis Primary efficacy endpoint: Key Inclusion Proptosis responder rate (PRR) Criteria in Q4W arm Additional efficacy • CAS ≥3 1, 2 Elegrobart Q8W & safety • Onset of TED Key secondary endpoints: follow-up at: symptoms within • Proptosis mean change from • Week 36 15 months baseline • Week 52 • Proptosis of ≥3 mm • Clinical Activity Score (CAS) Placebo reduction to 0 or 1 • Diplopia responder rate • Diplopia complete resolution Elegrobart Key: Placebo • Q8W endpoints 300 mg 1 2 20 600 mg loading dose given as two 300 mg injections; Placebo injections administered at alternating study visits to maintain study masking across arms. D = day, mm = millimeter, Q4W = every 4 weeks, Q8W = every 8 weeks, TED = thyroid eye disease, W = week.
REVEAL-1 is the largest pivotal clinical trial conducted in active TED to date Randomized Patients (n = 132) Elegrobart Q4W Elegrobart Q8W Placebo (n = 44) (n = 44) (n = 44) Discontinued • 1 TEAE (Gr1 amylase / Discontinued Discontinued lipase elevation) • 2 withdrawal of consent • 1 TEAE (Gr3 swollen tongue, • 2 withdrawal of consent Gr2 chest pain & Gr2 IRR) • 1 lost to follow-up • 2 lost to follow-up • 2 failure to follow protocol • 2 participant decision Completed Randomized Completed Randomized Completed Randomized Treatment Treatment Treatment (n = 35) (n = 41) (n = 43) 21 Source: Viridian REVEAL-1 week 24 topline data on file (interim topline database lock). Q4W = every 4 weeks, Q8W = every 8 weeks. Gr = grade, TEAE = treatment-emergent adverse event, IRR = infusion related reaction.
REVEAL-1 baseline characteristics were well-balanced between arms 7.8 Elegrobart Q4W Elegrobart Q8W Placebo (n = 44) (n = 44) (n = 44) Age in years, mean (SD) 52.6 (12.1) 48.1 (12.4) 48.5 (12.9) Participant Female sex, n (%) 35 (80%) 34 (77%) 35 (80%) Demographics White race, n (%) 36 (82%) 36 (82%) 35 (80%) Months since TED onset, mean (SD) 6.6 (4.4) 7.7 (4.3) 8.3 (5.2) Baseline proptosis by exophthalmometry (mm), mean (SD) 22.3 (2.6) 22.7 (3.3) 21.8 (2.5) Disease Baseline CAS, mean (SD) 4.3 (1.0) 4.2 (1.0) 4.0 (0.9) Characteristics Participants with diplopia, n (%) 28 (64%) 27 (61%) 31 (70%) 1 Diplopia (Gorman Score), mean (SD) 1.8 (0.8) 1.8 (0.8) 1.8 (0.7) Source: Viridian REVEAL-1 week 24 topline data on file (interim topline database lock). Note: all proptosis & CAS reported values and endpoints in the data analysis are based on study eye (defined as eye with greater proptosis at baseline). 1 22 Of patients with diplopia at baseline. CAS = clinical activity score, mm = millimeter, SD = standard deviation, TED = thyroid eye disease.
REVEAL-1 achieved high statistical significance on primary endpoint at 24 weeks Elegrobart Placebo p-value (n = 44 per arm) (n = 44) 1 FDA: Proptosis responder rate (exophthalmometry) 54% 18% p < 0.0001* Primary Q4W Endpoint 2 EMA: Overall responder rate (ORR) 51% 16% p = 0.0001* Proptosis mean change from baseline (exophthalmometry) -2.33 mm -0.81 mm p < 0.0001* Clinical activity score (CAS) reduction to 0 or 1 57% 50% p = 0.24 Q4W 3 Diplopia responder rate 71% 32% p = 0.0009 4 Diplopia complete resolution 51% 16% p = 0.0013 1 Key Proptosis responder rate (exophthalmometry) 63% 18% p < 0.0001 Secondary 2 EMA: Overall responder rate (ORR) 58% 16% p < 0.0001 Endpoints Proptosis mean change from baseline (exophthalmometry) -2.50 mm -0.81 mm p < 0.0001 Q8W Clinical activity score (CAS) reduction to 0 or 1 69% 50% p = 0.03 3 Diplopia responder rate 54% 32% p = 0.05 4 Diplopia complete resolution 28% 16% p = 0.14 1 Proptosis responder rate (MRI) 50% 2% p < 0.0001 Q4W Other Proptosis mean change from baseline (MRI) -2.04 mm -0.22 mm p < 0.0001 Secondary 1 Proptosis responder rate (MRI) 36% 2% p < 0.0001 Endpoints Q8W Proptosis mean change from baseline (MRI) -1.99 mm -0.22 mm p < 0.0001 Source: Viridian REVEAL-1 week 24 topline data on file (interim topline database lock). * Statistically significant. Key secondary endpoints below Q4W “CAS Reduction to 0 or 1” in the prespecified testing hierarchy and other secondary endpoints are nominally significant if below the statistically significant threshold of 0.025. 1 2 Participants with ≥2 mm reduction in proptosis from baseline in study eye, without deterioration in fellow eye (≥2 mm increas e), Participants with both proptosis and CAS response; CAS response defined as ≥2- 23 3 point reduction in CAS from baseline in study eye, without deterioration in fellow eye (≥2poi - nt increase), Participants with reduction of ≥1 on Gorman Score at week 24, among patients with diplopia at baseline, 4 Participants with baseline diplopia (Gorman Score >0) and a score of 0 at week 24. CAS = clinical activity score, mm = millimeter, MRI = magnetic resonance imaging, Q4W = every 4 weeks, Q8W = every 8 weeks.
Significant proptosis responder rate as early as 4 weeks after just one dose and across all time points in both arms Proptosis Responder Rate – Q4W Proptosis Responder Rate – Q8W Primary 100 100 endpoint: p < 0.0001 80 80 63% 63% 62% 61% 57% 54% 60 60 45% 43% 43% 37% 31% 30% 40 40 21% 21% 18% 18% 14% 14% 20 20 10% 10% 10% 10% 8% 8% 0 0 Baseline Week 4 Week 8 Week 12 Week 16 Week 20 Week 24 Baseline Week 4 Week 8 Week 12 Week 16 Week 20 Week 24 Analysis Visits Analysis Visits Placebo Elegrobart Q4W Placebo Elegrobart Q8W Rapid onset of treatment effect: proptosis response in patients receiving elegrobart was observed as early as week 4, after just one dose Source: Viridian REVEAL-1 week 24 topline data on file (interim topline database lock). Primary endpoint was statistically significant. PRR at time points prior to week 24 were prespecified exploratory endpoints. Results at all time points and 24 across both treatment arms prior to week 24 were nominally significant (p < 0.025). PRR = proptosis responder rate, Q4W = every 4 weeks, Q8W = every 8 weeks. Proptosis Responder Rate (%) Proptosis Responder Rate (%)
Significant proptosis mean change from baseline at all time points across both treatment arms, including at week 4 Mean Change from Baseline – Q4W Mean Change from Baseline – Q8W Key secondary -0.21 -0.21 -0.30 -0.30 0 0 endpoint: -0.49 -0.49 p < 0.0001 -0.52 -0.52 -0.81 -0.81 -0.81 -0.81 -1 -1 -1.07 -1.16 -1.37 -2 -1.65 -2 -1.66 -1.76 -2.10 -2.24 -2.30 -2.33 -2.41 -2.50 -3 -3 Baseline Week 4 Week 8 Week 12 Week 16 Week 20 Week 24 Baseline Week 4 Week 8 Week 12 Week 16 Week 20 Week 24 Analysis Visits Analysis Visits Placebo Elegrobart Q4W Placebo Elegrobart Q8W Source: Viridian REVEAL-1 week 24 topline data on file (interim topline database lock). The key secondary endpoint of mean change from baseline at week 24 for Q4W arm was statistically significant. Proptosis mean change from baseline at time points prior to week 24 25 were prespecified exploratory endpoints. Results at all time points and across both treatment arms prior to week 24 were nominally significant (p < 0.025). mm = millimeter, Q4W = every 4 weeks, Q8W = every 8 weeks. Proptosis Mean Reduction (mm) Proptosis Mean Reduction (mm)
Proptosis endpoints as measured by MRI were consistent with exophthalmometer, and significant at all time points Proptosis Responder Rate (MRI) Proptosis Mean Change from Baseline (MRI) -0.13 -0.22 100 0 90 80 70 -1 50% 60 43% -1.69 50 -1.99 40 -2 30 41% -1.87 36% -2.04 20 5% 10 2% 0 -3 Baseline Week 12 Week 24 Baseline Week 12 Week 24 Analysis Visits Analysis Visits Placebo Elegrobart Q4W Elegrobart Q8W Placebo Elegrobart Q4W Elegrobart Q8W Source: Viridian REVEAL-1 week 24 topline data on file (interim topline database lock). PRR and proptosis mean change from baseline at all time points prior to week 24 were prespecified exploratory endpoints. Results at all time points and 26 across both treatment arms were nominally significant (p < 0.025). MRI assessment was only conducted at baseline, week 12, and week 24. mm = millimeter, Q4W = every 4 weeks, Q8W = every 8 weeks. Proptosis Responder Rate (%) Proptosis Mean Reduction (mm)
Diplopia responder rate and complete resolution for patients receiving elegrobart Q4W improved throughout treatment period Diplopia Responder Rate – Q4W Diplopia Complete Resolution – Q4W 100 100 74% 71% 80 80 58% 54% 51% 50% 50% 60 60 43% 33% 33% 32% 40 27% 40 35% 20 20 32% 29% 29% 26% 19% 19% 16% 16% 13% 13% 0 0 6% Baseline Week 4 Week 8 Week 12 Week 16 Week 20 Week 24 Baseline Week 4 Week 8 Week 12 Week 16 Week 20 Week 24 Analysis Visits Analysis Visits Placebo Elegrobart Q4W Placebo Elegrobart Q4W Note: diplopia time course data not shown for Q8W treatment arm given week 24 endpoints did not meet nominal significance threshold Source: Viridian REVEAL-1 week 24 topline data on file (interim topline database lock). 27 Diplopia responder rate and diplopia complete resolution at time points prior to week 24 were prespecified exploratory endpoints. Q4W = every 4 weeks, Q8W = every 8 weeks. Diplopia Responder Rate (%) Diplopia Resolution Rate (%)
Elegrobart was generally well tolerated through week 24 Elegrobart Q4W Elegrobart Q8W Placebo N=44 N=44 N=44 n (%) n (%) n (%) Participants with any treatment-emergent 40 (91%) 31 (70%) 24 (55%) adverse event (TEAE) 1 2 Participants with any serious AE (SAE) 2 (5%) 2 (5%) 0 Participants with any treatment-related TEAE 32 (73%) 22 (50%) 12 (27%) 1 Participants with any treatment-related SAE 1 (2%) 0 0 • Vast majority of TEAEs in both treatment arms were mild • Only 2 treatment discontinuations due to TEAEs 3 ‒ 1 in placebo arm (related TEAE) 4 ‒ 1 in elegrobart Q4W arm (unrelated TEAE) Source: Viridian REVEAL-1 week 24 topline data on file (interim topline database lock). 1 2 2 participants with 3 Gr3 SAEs: dehydration due to norovirus (unrelated), headache with left-ear tinnitus (related); 2 participants with 4 Gr3 SAEs: three abscesses (unrelated), pulmonary 3 4 embolism (unrelated); Related TEAE discontinuation in placebo arm was due to Gr3 swollen tongue, Gr2 chest pain, & Gr2 IRR; Unrelated TEAE discontinuation in Q4W arm was due to 28 Gr1 amylase increase & Gr1 lipase increase. AE = adverse event, MedDRA= medical dictionary for regulatory activities, SAE = serious adverse event, TEAE = treatment-emergent adverse event, Gr = grade, IRR = infusion related reaction.
AE categories for elegrobart in REVEAL-1 were consistent with those generally expected from the anti-IGF-1R class Elegrobart Q4W Elegrobart Q8W Placebo AEs occurring at ≥10% frequency in any arm N=44 N=44 N=44 n (%) n (%) n (%) Muscle spasms 18 (41%) 16 (36%) 3 (7%) 1,2 Injection site reactions (ISR) 15 (34%) 9 (21%) 7 (16%) Headache 7 (16%) 3 (7%) 3 (7%) Ear discomfort 7 (16%) 3 (7%) 1 (2%) Alopecia 7 (16%) 3 (7%) 1 (2%) Diarrhea 6 (14%) 4 (9%) 2 (5%) 1,3 Hearing impairment 6 (14%) 2 (5%) 1 (2%) 1 Hyperglycemia 5 (11%) 5 (11%) 1 (2%) 1 Injection related reactions (IRR) 5 (11%) 2 (5%) 2 (5%) 1,4 Menstrual disorders 5 / 17 (29%) 6 / 23 (26%) 1 / 20 (5%) Source: Viridian REVEAL-1 week 24 topline data on file (interim topline database lock). 1 2 3 Includes multiple terms aggregated using standard sets of MedDRA terms; All ISRs were Grade 1 except for one Grade 2 in Q8W arm (erythema), and majority of ISRs were erythema; All 4 29 hearing impairment events in the treatment arms were tinnitus with no reductions in hearing. There was one hypoacusis event in placebo arm; Reported as percentage of menstruating women. AE = adverse event, MedDRA = medical dictionary for regulatory activities.
REVEAL-2 Topline Data in Chronic TED Potential first subcutaneous autoinjector for TED 30
REVEAL-2 in chronic TED patients met primary and multiple secondary endpoints and elegrobart was generally well tolerated Achieved the primary endpoint with high statistical significance (p < 0.0001), with IV-like proptosis response – 50% of Q4W and 54% of Q8W achieved a proptosis response vs 15% placebo at week 24 (p < 0.0001 for both arms) Meaningful benefit on diplopia – 61% of Q4W elegrobart achieved diplopia response vs 38% placebo at week 24 (p = 0.0118) – 44% of Q4W elegrobart achieved diplopia complete resolution vs 25% placebo at week 24 (p = 0.0295) Generally well tolerated in both dose groups, with low rates of hearing impairment through week 24 Elegrobart is the first & only subcutaneous program with positive data in a pivotal chronic TED trial Elegrobart is an investigational product that has not been approved by any regulatory authority; the safety and efficacy have not been established. Source: Viridian REVEAL-2 week 24 topline data on file (interim topline database lock). 31 P-values below 0.025 are statistically significant. AE = adverse event, IGF-1R = insulin-like growth factor-1 receptor, PRR = proptosis responder rate, Q4W = every 4 weeks, Q8W = every 8 weeks, TED = thyroid eye disease.
REVEAL-2 is a phase 3 randomized, controlled, double-masked trial of elegrobart in chronic TED Treatment Phase (20 weeks treatment with primary endpoint at 24 weeks) 1 D1 W4 W8 W12 W16 W20 W24 Treatment Arms Follow-up through W52 (1:1:1) Primary Endpoint 1 Elegrobart Q4W Analysis Primary efficacy endpoint: Key Inclusion Proptosis responder rate Criteria in Q4W arm Additional efficacy • Any CAS (0–7) 1, 2 Elegrobart Q8W & safety • Onset of TED Key secondary endpoints: follow-up at: symptoms • Proptosis responder rate • Week 36 >15 months in Q8W arm • Week 52 • Proptosis of ≥3 mm • Proptosis mean change Placebo from baseline • Diplopia responder rate • Diplopia complete Elegrobart Key: Placebo resolution rate 300 mg 1 2 32 600 mg loading dose given as two 300 mg injections; Placebo injections administered at alternating study visits to maintain study masking across arms. D = day, mm = millimeter, Q4W = every 4 weeks, Q8W = every 8 weeks, TED = thyroid eye disease, W = week.
REVEAL-2 is the largest pivotal clinical trial conducted in chronic TED to date Randomized Patients (n = 204) Elegrobart Q4W Elegrobart Q8W Placebo (n = 70) (n = 68) (n = 66) Discontinued Discontinued Discontinued • 3 withdrawal of consent • 1 withdrawal of consent • 2 withdrawal of consent • 2 TEAEs • 2 TEAEs • 1 TEAE • 1 failure to follow protocol • 2 failure to follow protocol • 1 failure to follow protocol • 1 lost to follow-up • 1 lost to follow-up Completed Randomized Completed Randomized Completed Randomized Treatment Treatment Treatment (n = 63) (n = 62) (n = 62) 33 Source: Viridian REVEAL-2 week 24 topline data on file (interim topline database lock). Q4W = every 4 weeks, Q8W = every 8 weeks. Gr = grade, TEAE = treatment-emergent adverse event, IRR = infusion related reaction.
REVEAL-2 baseline characteristics were well-balanced between arms Elegrobart Q4W Elegrobart Q8W Placebo 7.8 (n = 70) (n = 68) (n = 66) Age in years, mean (SD) 50.1 (11.3) 52.0 (11.2) 53.3 (11.2) Participant Female sex, n (%) 60 (86%) 57 (84%) 53 (80%) Demographics White race, n (%) 54 (77%) 52 (76%) 51 (77%) Months since TED onset, mean (SD) 78.9 (73.4) 75.0 (72.1) 95.8 (108.6) 1 Baseline proptosis (mm), mean (SD) 22.7 (2.9) 22.6 (2.9) 22.7 (2.7) Baseline CAS, mean (SD) 2.7 (1.7) 3.0 (1.6) 2.8 (1.7) Disease Baseline CAS ≤1, n (%) 17 (24.3) 15 (22.1) 16 (24.2) Characteristics Baseline CAS ≥3, n (%) 38 (54.3) 43 (63.2) 34 (51.5) Participants with diplopia, n (%) 47 (67%) 54 (79%) 47 (71%) 2 Diplopia (Gorman Score), mean (SD) 1.8 (0.7) 1.9 (0.7) 1.8 (0.7) Source: Viridian REVEAL-2 week 24 topline data on file (interim topline database lock). Note: all proptosis & CAS reported values and endpoints in the data analysis are based on study eye (defined as eye with greater proptosis at baseline). 1 2 34 Measured by exophthalmometry, Of patients with diplopia at baseline. CAS = clinical activity score, mm = millimeter, SD = standard deviation, TED = thyroid eye disease.
REVEAL-2 achieved high statistical significance on primary endpoint and multiple secondary endpoints at 24 weeks Elegrobart Elegrobart Placebo Q4W Q8W (n = 66) (n = 70) (n = 68) FDA Primary 50% 54% 1,2 Endpoint Proptosis responder rate (PRR) 15% (p < 0.0001) (p < 0.0001) EMA Primary 47% 54% 3 Endpoint Proptosis Overall responder rate (ORR) 15% (p < 0.0001) (p < 0.0001) -1.88 mm -2.08 mm 1 Proptosis mean change from baseline -0.52 mm (p < 0.0001) (p < 0.0001) 61% 55% 4 Diplopia responder rate 38% (p = 0.0118) (p = 0.0419) Diplopia 44% 36% 5 Diplopia complete resolution 25% (p = 0.0295) (p = 0.1304) Source: Viridian REVEAL-2 week 24 topline data on file (interim topline database lock). P-values below 0.025 are statistically significant. 1 2 3 Measured by exophthalmometry, Participants with ≥2 mm reduction in proptosis from baseline in study eye, without deterioration in fellow eye (≥2 mm increas e), Participants with 4 both proptosis and CAS response; CAS response defined as no worsening in CAS from baseline in study eye, without deterioration in fellow eye (≥2-point increase), Participants with 35 5 reduction of ≥1 on Gorman Score at week 24, among patients with diplopia at baseline (Gorman Score >0), Participants with diplopia at baseline and a score of 0 at week 24. CAS = clinical activity score, mm = millimeter, Q4W = every 4 weeks, Q8W = every 8 weeks.
Significant proptosis responder rate at all time points after week 4 in both treatment arms Proptosis Responder Rate – Q4W Proptosis Responder Rate – Q8W Primary Key secondary endpoint: endpoint: 100 100 p < 0.0001 p < 0.0001 80 80 54% 50% 60 60 44% 42% 41% 38% 34% 36% 40 40 30% 22% 19% 18% 18% 19% 16% 15% 15% 20 20 11% 11% 9% 9% 6% 6% 5% 0 0 Baseline Week 4 Week 8 Week 12 Week 16 Week 20 Week 24 Baseline Week 4 Week 8 Week 12 Week 16 Week 20 Week 24 Analysis Visits Analysis Visits Placebo Elegrobart Q4W Placebo Elegrobart Q8W Source: Viridian REVEAL-2 week 24 topline data on file (interim topline database lock). PRR at time points prior to week 24 were prespecified exploratory endpoints. 36 P-values below 0.025 at week 24 are statistically significant. Q4W = every 4 weeks, Q8W = every 8 weeks. Proptosis Responder Rate (%) Proptosis Responder Rate (%)
Significant proptosis mean change from baseline at all time points across both treatment arms, including at week 4 Mean Change from Baseline – Q4W Mean Change from Baseline – Q8W Key secondary Key secondary endpoint: endpoint: p < 0.0001 p < 0.0001 0 0 -0.27 -0.27 -0.52 -0.52 -0.56 -0.56 -0.61 -0.61 -0.65 -0.65 -0.68 -0.68 -0.63 -1 -1 -0.82 -1.10 -1.26 -1.44 -1.45 -1.66 -2 -2 -1.77 -1.79 -1.84 -1.88 -2.08 -3 -3 Baseline Week 4 Week 8 Week 12 Week 16 Week 20 Week 24 Baseline Week 4 Week 8 Week 12 Week 16 Week 20 Week 24 Analysis Visits Analysis Visits Placebo Elegrobart Q4W Placebo Elegrobart Q8W Source: Viridian REVEAL-2 week 24 topline data on file (interim topline database lock). Proptosis mean change from baseline at time points prior to week 24 were prespecified exploratory endpoints. 37 P-values below 0.025 at week 24 are statistically significant. mm = millimeter, Q4W = every 4 weeks, Q8W = every 8 weeks. Proptosis Mean Reduction (mm) Proptosis Mean Reduction (mm)
First demonstration of statistically significant diplopia response for a subcutaneous treatment in chronic TED Diplopia Responder Rate – Q4W Diplopia Complete Resolution – Q4W Key secondary Key secondary 100 100 endpoint: endpoint: p = 0.0118 p = 0.0295 80 80 61% 58% 49% 60 60 44% 42% 38% 32% 40 40 27% 25% 21% 21% 38% 36% 13% 32% 20 31% 20 26% 25% 23% 23% 20% 17% 16% 13% 0 0 Baseline Week 4 Week 8 Week 12 Week 16 Week 20 Week 24 Baseline Week 4 Week 8 Week 12 Week 16 Week 20 Week 24 Analysis Visits Analysis Visits Placebo Elegrobart Q4W Placebo Elegrobart Q4W Source: Viridian REVEAL-2 week 24 topline data on file (interim topline database lock). Diplopia responder rate and diplopia complete resolution at time points prior to week 24 were prespecified exploratory endpoints. 38 P-values below 0.025 at week 24 are statistically significant. Q4W = every 4 weeks, Q8W = every 8 weeks. Diplopia Responder Rate (%) Diplopia Resolution Rate (%)
Proptosis & diplopia benefit demonstrated in low-CAS subgroup Key efficacy endpoints in subgroup of patients with Elegrobart Elegrobart Placebo low CAS (CAS ≤ 1) at baseline Q4W Q8W (n = 16) (n = 17) (n = 15) 1 Same CAS inclusion criteria as teprotumumab chronic TED phase 4 study 54% 55% 2 Proptosis responder rate (PRR) 6% (p = 0.0002) (p = 0.0004) Proptosis -2.07 mm -2.31 mm 2 Proptosis mean change from baseline -0.05 mm (p = 0.0002) (p < 0.0001) 57% 73% Diplopia responder rate 30% Diplopia (p = 0.1057) (p = 0.0153) (Q4W: n=10; Q8W: n=11; 33% 46% placebo: n=10) Diplopia complete resolution 10% (p = 0.0497) (p = 0.0067) Overall 42% 54% Overall responder rate (ORR) 6% Response (p = 0.0063) (p = 0.0001) Elegrobart demonstrated consistent, IV-like clinical activity in chronic TED patients regardless of baseline disease activity, in the largest and broadest TED phase 3 study completed to date Source: Viridian REVEAL-2 week 24 topline data on file (interim topline database lock). CAS subgroup analyses were prespecified exploratory endpoints. P-values below 0.025 are nominally significant. 1 2 39 Douglas RS et al., J Clin Endocrinol Metab. 2023; 109(1):25–35, Measured by exophthalmometry. CAS = clinical activity score, mm = millimeter, ORR = overall responder rate, Q4W = every 4 weeks, Q8W = every 8 weeks, TED = thyroid eye disease.
Elegrobart was generally well tolerated through week 24 Elegrobart Q4W Elegrobart Q8W Placebo N=70 N=68 N=66 n (%) n (%) n (%) Participants with any treatment-emergent 55 (79%) 56 (82%) 44 (67%) adverse event (TEAE) Participants with any serious AE (SAE) 3 (4%) 1 (1%) 0 Participants with any treatment-related TEAE 39 (56%) 39 (57%) 22 (33%) Participants with any treatment-related SAE 0 0 0 • Vast majority of TEAEs in both treatment arms were mild • No treatment-related SAEs • 91% of elegrobart-treated patients completed full course of treatment • 3 treatment-related TEAE discontinuations 1 2 ‒ 1 in elegrobart Q4W arm & 2 in elegrobart Q8W arm Source: Viridian REVEAL-2 week 24 topline data on file (interim topline database lock). 1 2 1 treatment-related TEAE discontinuation in Q4W arm: Gr2 hyperglycemia & Gr2 muscle spasms; 2 treatment-related TEAE discontinuations in Q8W arm: Gr1 tinnitus 40 (related; resolving) and Gr3 muscle spasms (foot cramps). AE = adverse event, MedDRA= medical dictionary for regulatory activities, SAE = serious adverse event, TEAE = treatment-emergent adverse event, Gr = grade.
AE categories for elegrobart in REVEAL-2 were consistent with those generally expected from the anti-IGF-1R class Elegrobart Q4W Elegrobart Q8W Placebo AEs occurring at ≥10% frequency in any arm N=70 N=68 N=66 n (%) n (%) n (%) Muscle spasms 15 (21%) 25 (37%) 7 (11%) 1,2 Injection site reactions (ISR) 16 (23%) 16 (24%) 18 (27%) 1 Hyperglycemia 12 (17%) 8 (12%) 1 (2%) Headache 7 (10%) 9 (13%) 3 (5%) 1,3 Hearing impairment 5 (7%) 8 (12%) 2 (3%) Diarrhea 3 (4%) 9 (13%) 1 (2%) Nasopharyngitis 3 (4%) 7 (10%) 4 (6%) Alopecia 2 (3%) 7 (10%) 5 (8%) 1,4 Menstrual disorders 11 / 30 (37%) 7 / 27 (26%) 2 / 24 (8%) Source: Viridian REVEAL-2 week 24 topline data on file (interim topline database lock). 1 2 Includes multiple terms aggregated using standard sets of MedDRA terms; All ISRs were mild (Grade 1) except for 3 moderate (Grade 2) (2 in Q8W arm & 1 in placebo arm); most 3 4 41 common ISR was erythema; Among participants that experienced hearing impairment AEs, the majority reported tinnitus; Reported as percentage of menstruating women. AE = adverse event, MedDRA = medical dictionary for regulatory activities, Q4W = every 4 weeks, Q8W = every 8 weeks.
FcRn Inhibitor Portfolio 42
Pathogenic autoantibodies drive disease pathophysiology in a number of autoimmune diseases FcRn-Mediated Recycling of IgGs, 1 Including Pathogenic Autoantibodies Pathogenic autoantibodies cause inflammation and damage to healthy 1 tissues and cells, driving the 1 pathology of autoimmune diseases 2 Serum levels of pathogenic autoantibodies are maintained, 1 3 4 in part, by FcRn-mediated recycling FcRn inhibition reduces pathogenic autoantibody IgGs, including pathogenic autoantibodies, enter the cell 1 1 levels , with demonstrated efficacy and safety 2 in patients with gMG, CIDP, and ITP IgGs and pathogenic autoantibodies bind to FcRns 2 Unbound antibodies are degraded by the lysosome 3 4 FcRn-bound IgGs, including pathogenic autoantibodies, are recycled Degraded 1 Source: Pyzik M et al. Nat Rev Immunol. 2023;23:415–432, IgG FcRn Endosome Lysosome 2 antibody Vyvgart Prescribing Information. CIDP = chronic inflammatory demyelinating polyneuropathy, FcRn = neonatal Fc receptor, 43 gMG = generalized myasthenia gravis, IgG = immunoglobulin G, ITP = primary immune thrombocytopenia.
Viridian’s portfolio of FcRn inhibitors aims to reduce circulating levels of pathogenic autoantibodies by blocking FcRn Inhibition of FcRn Reduces IgGs, VRDN-006 1 Including Pathogenic Autoantibodies Fc fragment that blocks IgG from binding to FcRn 1 Fc fragment 2 VRDN-008 4 3 Binds to albumin and FcRn for a more sustained reduction of pathogenic autoantibodies FcRn inhibitor and IgGs, including pathogenic autoantibodies, enter the cell 1 FcRn inhibitor blocks IgGs from binding to FcRn 2 Fc fragment Unbound IgGs, including pathogenic autoantibodies, are degraded by the 3 lysosome, reducing serum levels Albumin binding domain The bound FcRn inhibitor and IgG are recycled and released 4 FcRn Degraded IgG FcRn Endosome Lysosome Inhibitor antibody 1 44 Source: Pyzik M et al. Nat Rev Immunol. 2023;23:415–432. Fc = fragment crystallizable, FcRn = neonatal Fc receptor, IgG = immunoglobulin G.
FcRn inhibitors are a large market opportunity; market size of MG and CIDP alone are projected to be over $11B by 2030 1 Projected WW MG and CIDP FcRn Market ...with Potential in Additional Autoimmune Indications $11.1B Graves’ Myositis Disease $4.6B $2.5B Lupus Sjogren’s Nephritis Syndrome 2024 2025 2030 1 Source: 2024 and 2025 revenues calculated from argenx (Vyvgart + Vyvgart Hytrulo), Zai Labs (Vyvgart), and UCB (Rystiggo) annual reported earnings; 45 2030 estimates based on Evaluate Pharma data for Vyvgart, Vyvgart Hytrulo, Rystiggo, Imaavy, batoclimab, and IMVT-1402, accessed February 2026. CIDP = chronic inflammatory demyelinating polyneuropathy, FcRn = neonatal Fc receptor, MG = myasthenia gravis, WW = worldwide.
Viridian’s FcRn portfolio has the potential to capture significant market share in autoimmune indications v v VRDN-006 VRDN-008 Highly Selective Fc Fragment and FcRn Inhibitor Half-life Extended Bispecific FcRn Inhibitor IgG • IgG reduction data consistent with the FcRn • Deeper and more sustained reduction of Suppression inhibitor class IgG vs. efgartigimod in NHPs • Targeting patient self-administration in a • Targeting a less frequent, self-administered, Dosing convenient subcutaneous injection subcutaneous injection • Spared albumin and LDL in healthy • Expect to maintain the Fc fragment safety Safety volunteers, generally well-tolerated profile 46 Fc = fragment crystallizable, FcRn = neonatal Fc receptor, FIH = first in human, IgG = immunoglobulin G, IND = Investigational New Drug, LDL = low-density lipoprotein, NHP = non-human primate, YE = year-end.
Appendix 1) THRIVE in Active TED Pivotal Data 2) THRIVE-2 in Chronic TED Pivotal Data 3) FcRn Non-Human Primate Data 47
THRIVE in Active TED Global phase 3 clinical trial pivotal data 48
THRIVE is a phase 3 randomized, controlled, double-masked trial of veligrotug in active TED Treatment Phase (12-week treatment period with primary endpoint at 15 weeks) D1 W3 W6 W9 W12 W15 Through W52 Treatment Arms (2:1 randomization) Primary Endpoint Analysis Veligrotug Additional n = 75 Primary efficacy endpoint: Key Inclusion efficacy Proptosis responder rate Criteria & safety follow-up at: • CAS ≥3 Key secondary endpoints: • Week 24 • Onset of TED • Proptosis mean change symptoms within • Week 36 from baseline 15 months • Week 52 Placebo • Diplopia (double vision) • Proptosis of ≥3 mm n = 38 • Clinical Activity Score (CAS) Veligrotug Final THRIVE Key: Placebo 10 mg/kg readout at Week 52 49 D = day, mm = millimeter, TED = thyroid eye disease, W = week.
THRIVE baseline characteristics were well-balanced between active and placebo arms 7.8 Veligrotug Placebo (n = 75) (n = 38) Age in years, mean (SD) 48.9 (12.4) 49.1 (12.5) Participant Female sex, n (%) 56 (75%) 31 (82%) Demographics White race, n (%) 51 (68%) 19 (50%) Months since TED onset, mean (SD) 7.9 (3.7) 7.2 (3.8) Baseline proptosis by exophthalmometry (mm), mean (SD) 23.2 (3.1) 23.2 (3.3) Disease Baseline CAS, mean (SD) 4.5 (1.0) 4.8 (1.1) Characteristics Participants with diplopia, n (%) 50 (67%) 26 (68%) 1 Diplopia (Gorman Score), mean (SD) 2.0 (0.8) 2.0 (0.7) Source: Viridian THRIVE week 15 topline data on file (interim topline database lock). Note: all proptosis & CAS reported values and endpoints in the data analysis are based on study eye (defined as eye with greater proptosis at baseline). 1 50 Of patients with diplopia at baseline. CAS = clinical activity score, mm = millimeter, SD = standard deviation, TED = thyroid eye disease.
THRIVE achieved high level of statistical significance across all primary and secondary endpoints at 15 weeks Veligrotug Placebo p-value (n=75) (n=38) Primary Endpoint: 70% 5% p < 0.0001 1 Proptosis responder rate (exophthalmometry) Proptosis Proptosis mean change from baseline (exophthalmometry) -2.89 mm -0.48 mm p < 0.0001 2 Diplopia complete resolution 54% 12% p < 0.0001 Diplopia 3 Diplopia responder rate 63% 20% p < 0.0001 Clinical activity score (CAS) 0 or 1 64% 18% p < 0.0001 CAS CAS mean change from baseline -3.4 -1.7 p < 0.0001 Overall 4 Overall responder rate (ORR) 67% 5% p < 0.0001 Response Source: Viridian THRIVE week 15 topline data on file (interim topline database lock). 1 2 Percentage of participants with ≥2 mm reduction in proptosis from baseline in the study eye, without deterioration in the fel low eye (≥2 mm increase), Percentage 3 of participants with baseline diplopia (Gorman Score >0) and a score of 0 at Week 15, Percentage of participants achieving a reduction of at least 1 on the Gorman 51 4 subjective diplopia scale at week 15, among patients with diplopia at baseline, Percentage of participants with ≥2 mm reduction in proptosis AND ≥2poi - nt reduction in CAS from baseline in the study eye, without corresponding deterioration [≥2 mm/point increase] in proptosis or CAS in the fellow eye. CAS = clinical activity score.
Primary endpoint of proptosis responder rate met at 15 weeks: 70% for patients receiving veligrotug compared with 5% on PBO Proptosis Responder Rate Proptosis Mean Change from Baseline 100 0 -0.48 -0.51 -0.67 -0.71 -0.72 72% 80 70% -1 64% 64% -1.82 53% 60 -2 -2.39 -2.66 -2.76 40 -2.89 -3 13% 13% 20 11% 11% 5% 0 -4 Baseline Week 3 Week 6 Week 9 Week 12 Week 15 Baseline Week 3 Week 6 Week 9 Week 12 Week 15 Analysis Visits Analysis Visits Placebo Veligrotug Placebo Veligrotug 53% of patients receiving veligrotug achieved a proptosis response at 3 weeks, after just 1 infusion of veligrotug Source: Viridian THRIVE week 15 topline data on file (interim topline database lock). 52 Results at time points before week 15 are from post-hoc analyses and are for descriptive purposes only. mm = millimeter, PBO = placebo. Proptosis Responder Rate (%) Proptosis Mean Reduction (mm)
Majority of patients receiving veligrotug had complete resolution of diplopia and minimal disease activity (CAS) at week 15 Diplopia Complete Resolution CAS Reduction to 0 or 1 100 100 80 80 64% 64% 63% Diplopia 53% 54% 60 60 Responder 46% 44% Rate at Week 15 35% 32% 40 40 30% 26% 25% 24% 20% 20% 18% 12% 12% Diplopia 20 20 12% 12% 7% Responder 4% Rate at Week 15 0 0 Baseline Week 3 Week 6 Week 9 Week 12 Week 15 Baseline Week 3 Week 6 Week 9 Week 12 Week 15 Analysis Visits Analysis Visits Placebo Veligrotug Placebo Veligrotug Source: Viridian THRIVE week 15 topline data on file (interim topline database lock). 53 Results at time points before week 15 are from post-hoc analyses and are for descriptive purposes only. CAS = clinical activity score. Diplopia Resolution Rate (%) CAS 0 or 1 Rate (%)
THRIVE demonstrated consistency between Hertel and MRI / CT and validates both as reliable tools for measurements of proptosis Hertel Exophthalmometry MRI / CT Veligrotug Placebo Veligrotug Placebo (n=75) (n=38) (n=75) (n=38) Proptosis responder rate Proptosis responder rate 70% 5% 69% 9% at week 15 at week 15 Proptosis mean change Proptosis mean change -2.89 mm -0.48 mm -2.91 mm -0.58 mm from baseline at week 15 from baseline at week 15 54 Source: Viridian THRIVE week 15 topline data on file (interim topline database lock). CT = computed tomography, IGF-1R = insulin-like growth factor-1 receptor, mm = millimeter, MRI = magnetic resonance imaging.
Veligrotug was generally well-tolerated at week 15, with no treatment-related SAEs, and 96% of veligrotug-treated patients completed all doses Veligrotug Placebo N=75 N=38 n (%) n (%) Participants with any treatment-emergent • Vast majority of TEAEs in 66 (88%) 24 (63%) adverse event (TEAE) both arms were mild 1 Participants with any serious AE (SAE) 4 (5%) 0 • Low treatment discontinuation rate ‒ 4% in veligrotug arm Participants with any treatment-related TEAE 53 (71%) 9 (24%) • No treatment-related SAEs Participants with any treatment-related SAE 0 0 Source: Viridian THRIVE week 15 topline data on file (interim topline database lock). 1 6 unrelated SAEs in 4 participants: cellulitis, appendicitis, dyspnoea, hyperthyroidism, aortic dissection (planned surgery for known Type B aortic dissection), st 55 depression (diagnosed prior to 1 dose); Includes multiple terms aggregated using standard sets of MedDRA terms. AE = adverse event, MedDRA= medical dictionary for regulatory activities, SAE = serious adverse event, TEAE = treatment-emergent adverse event.
Veligrotug was generally well-tolerated at week 15, with a 5.5% placebo-adjusted rate of hearing impairment AEs Veligrotug Placebo AEs occurring at ≥10% frequency in either arm N=75 N=38 n (%) n (%) Muscle spasms 32 (43%) 2 (5%) Headache 16 (21%) 5 (13%) Infusion related reaction (IRR) 13 (17%) 1 (3%) 1 Hearing impairment 12 (16%) 4 (11%) 1 Hyperglycemia 11 (15%) 2 (5%) 1 Fatigue 10 (13%) 6 (16%) Nausea 10 (13%) 3 (8%) Ear discomfort 9 (12%) 1 (3%) Diarrhea 8 (11%) 1 (3%) Alopecia 6 (8%) 4 (11%) 1,2 Menstrual disorders 8 / 34 (24%) 1 / 12 (8%) Source: Viridian THRIVE week 15 topline data on file (interim topline database lock). 1 2 56 Includes multiple terms aggregated using standard sets of MedDRA terms, Reported as percentage of menstruating women. AE = adverse event, MedDRA = medical dictionary for regulatory activities.
70% of proptosis responders in THRIVE maintained response at Week 52 in long-term follow up Proptosis Durability Safety Resolution of Week 15 proptosis • No changes to veligrotug’s safety profile responders maintained during the follow-up period 70% a proptosis response at • Vast majority of adverse events reported at (21/30 participants) 1 topline resolved by Week 52 Week 52 Source: Viridian THRIVE week 52 data on file (final database lock). 1 57 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. Definition of durability is the same as that used for teprotumumab durability as reported in its U.S. Prescribing Information.
THRIVE-2 in Chronic TED Global phase 3 clinical trial pivotal data 58
THRIVE-2 is a phase 3 randomized, controlled, double-masked trial of veligrotug in chronic TED Treatment Phase (12-week treatment period with primary endpoint at 15 weeks) D1 W3 W6 W9 W12 W15 Through W52 Treatment Arms (2:1 randomization) Primary Endpoint Analysis Veligrotug n = 125 Additional Primary efficacy endpoint: Key Inclusion efficacy Proptosis responder rate Criteria & safety follow-up at: • Any CAS (0–7) Key secondary endpoints: • Week 24 • Onset of TED • Proptosis mean change symptoms >15 months • Week 36 from baseline • Proptosis of ≥3 mm • Week 52 Placebo • Diplopia (double vision) n = 63 • Clinical Activity Score (CAS) Veligrotug Final THRIVE-2 Key: Placebo 10 mg/kg readout at Week 52 59 D = day, mm = millimeter, TED = thyroid eye disease, W = week.
THRIVE-2 baseline characteristics were well-balanced between active and placebo arms Veligrotug Placebo (n = 125) (n = 63) Age in years, mean (SD) 50.5 (13.5) 50.7 (12.0) Participant Female sex, n (%) 95 (76%) 46 (73%) Demographics White race, n (%) 94 (75%) 48 (76%) Months since TED onset, mean (SD) 69.8 (78.9) 81.7 (83.7) Baseline proptosis by exophthalmometry (mm), mean (SD) 24.3 (3.3) 23.8 (3.3) Baseline CAS, mean (SD) 2.7 (1.9) 2.5 (1.8) Disease Baseline CAS 0 or 1, n (%) 44 (35%) 22 (35%) Characteristics Baseline CAS ≥ 3, n (%) 71 (57%) 33 (52%) Participants with diplopia, n (%) 65 (52%) 37 (59%) 1 Diplopia (Gorman Score), mean (SD) 2.0 (0.8) 2.1 (0.9) Source: Viridian THRIVE-2 week 15 topline data on file (interim topline database lock). 60 Note: all proptosis & CAS reported values and endpoints in the data analysis are based on study eye (defined as eye with greater proptosis at baseline). 1 Of participants with diplopia at baseline. CAS = clinical activity score, mm = millimeter, SD = standard deviation, TED = thyroid eye disease.
THRIVE-2 met all primary and secondary endpoints at 15 weeks Veligrotug Placebo p-value (n=125) (n=63) Primary Endpoint: 56% 8% p < 0.0001 1 Proptosis responder rate (exophthalmometry) Proptosis Proptosis mean change from baseline (exophthalmometry) -2.34 mm -0.46 mm p < 0.0001 2 Diplopia responder rate 56% 25% p = 0.0006 Diplopia 3 Diplopia complete resolution 32% 14% p = 0.0152 Overall 4 Overall responder rate (ORR) 56% 7% p < 0.0001 Response 5 5 Clinical activity score (CAS) reduction to 0 or 1 54% 24% p = 0.0060 CAS (prespecified exploratory 5 endpoints) CAS mean change from baseline -2.9 -1.3 p < 0.0001 Source: Viridian THRIVE-2 week 15 topline data on file (interim topline database lock). 1 2 Percentage of participants with ≥2 mm reduction in proptosis from baseline in the study eye, without deterioration in the fel low eye (≥2 mm increase), Percentage of participants achieving a reduction of 3 at least 1 on the Gorman subjective diplopia scale, among patients with diplopia at baseline (n=102 participants), Percentage of participants with baseline diplopia (Gorman Score >0; n=102 participants) 4 and a score of 0 at the analysis timepoint, Percentage of participants with ≥2 mm reduction in proptosis AND no worsening in CAS from baseline in the study eye, without c orresponding deterioration (≥2 5 mm/point increase) in proptosis or CAS in the fellow eye, Of participants with CAS ≥3 at baseline (n=104 participants); CAS subpopulation analyses were prespecified, exploratory endpoints and 61 statistical p values are for descriptive purposes only. CAS = clinical activity score.
Statistically significant proptosis responder rate at all time points, including at 3 weeks, after just one infusion of veligrotug Proptosis Responder Rate Proptosis Mean Change from Baseline 100 0 -0.46 -0.62 -0.69 -0.72 -0.75 80 -1 56% 60 54% 50% -1.06 -1.70 40% -2.04 40 -2.22 -2 25% -2.34 20 13% 11% 13% 8% 10% 0 -3 Baseline Week 3 Week 6 Week 9 Week 12 Week 15 Baseline Week 3 Week 6 Week 9 Week 12 Week 15 Analysis Visits Analysis Visits Placebo Veligrotug Placebo Veligrotug Rapid and statistically significant proptosis responder rate at 3 weeks, after just 1 infusion of veligrotug 62 Source: Viridian THRIVE-2 week 15 topline data on file (interim topline database lock). Results at time points before week 15 are from prespecified, exploratory endpoint analyses. Proptosis Responder Rate (%) Proptosis Mean Reduction (mm)
THRIVE-2 is the first phase 3 study in patients with chronic TED to demonstrate statistically significant diplopia response & resolution Diplopia Responder Rate Diplopia Complete Resolution 100 100 80 80 55% 56% 52% 60 60 47% 36% 33% 32% 31% 40 40 29% 25% 24% 19% 18% 14% 14% 11% 11% 20 20 5% 22% 14% 0 0 Baseline Week 3 Week 6 Week 9 Week 12 Week 15 Baseline Week 3 Week 6 Week 9 Week 12 Week 15 Analysis Visits Analysis Visits Placebo Veligrotug Placebo Veligrotug 63 Source: Viridian THRIVE-2 week 15 topline data on file (interim topline database lock). Results at time points before week 15 are from prespecified, exploratory endpoint analyses. TED = thyroid eye disease. Diplopia Responder Rate (%) Diplopia Resolution Rate (%)
THRIVE-2 demonstrated consistency between Hertel exophthalmometry and MRI / CT as measurements of proptosis Hertel exophthalmometry MRI / CT Veligrotug Placebo Veligrotug Placebo (n=125) (n=63) (n=125) (n=63) Proptosis responder rate Proptosis responder rate 56% 8% 48% 3% at week 15 at week 15 Proptosis mean change Proptosis mean change -2.34 mm -0.46 mm -2.07 mm -0.36 mm from baseline at week 15 from baseline at week 15 THRIVE-2 demonstrated both exophthalmometry and MRI / CT are reliable tools for measurement of proptosis, building on data from THRIVE Source: Viridian THRIVE-2 week 15 topline data on file (interim topline database lock). Study eye is defined as eye with greater proptosis at baseline, as measured by corresponding measurement modality (i.e., Hertel study eye for Hertel endpoints, 64 and MRI / CT study eye for MRI / CT endpoints). CT = computed tomography, mm = millimeter, MRI = magnetic resonance imaging.
Veligrotug was generally well-tolerated, and 94% of veligrotug-treated patients completed their treatment course Veligrotug Placebo N=125 N=63 n (%) n (%) Participants with any treatment-emergent • Vast majority of TEAEs in 106 (85%) 43 (68%) adverse event (TEAE) both arms were mild 1 2 Participants with any serious AE (SAE) 3 (2%) 2 (3%) • Low treatment discontinuation rate ‒ 6% in veligrotug arm Participants with any treatment-related TEAE 79 (63%) 14 (22%) 1 2 1 (2%) Participants with any treatment-related SAE 1 (1%) Source: Viridian THRIVE-2 week 15 topline data on file (interim topline database lock). 1 3 SAEs in 3 participants: Grade 3 vertigo (related), Grade 2 arthralgia (unrelated), Grade 2 metabolic encephalopathy (unrelated); 2 65 2 SAEs in 2 participants: Grade 3 urticaria (related), Grade 3 fatigue (unrelated). AE = adverse event, SAE = serious adverse event, TEAE = treatment-emergent adverse event.
Veligrotug was generally well-tolerated, with a 9.6% placebo-adjusted rate of hearing impairment AEs Veligrotug Placebo AEs occurring at ≥10% frequency in either arm N=125 N=63 n (%) n (%) Muscle spasms 45 (36%) 4 (6%) Headache 18 (14%) 8 (13%) 1 Hearing impairment 16 (13%) 2 (3%) 1 Fatigue 15 (12%) 5 (8%) Diarrhea 14 (11%) 6 (10%) 1 Hyperglycaemia 13 (10%) 3 (5%) 1,2 Menstrual Disorders 16 / 48 (33%) 2 / 20 (10%) Source: Viridian THRIVE-2 week 15 topline data on file (interim topline database lock). 1 2 66 Terms aggregated utilizing methodology used by FDA for approved products for treatment of thyroid eye disease, Reported as percentage of menstruating women. AE = adverse event.
FcRn Non-Human Primate Data 67
VRDN-006 in vitro, multi-dose NHP PK and IgG reduction data v compared to efgartigimod pH-Dependent Binding Multi-Dose NHP Pharmacokinetics 5 4×10 Dose VRDN-006 1000 5 3×10 VRDN-006 (20 mg/kg, IV) Efgartigimod pH 6 Efgartigimod (20 mg/kg, IV) WT Fc Control 5 100 2×10 5 1×10 10 pH 7.4 0 1 0 5 10 15 20 25 Concentration (nM) Time (Days) Internal Target Occupancy Multi-Dose NHP Pharmacodynamics 6 150 3×10 Dose 6 2×10 100 VRDN-006 Efgartigimod 6 1×10 50 VRDN-006 (20 mg/kg, IV) WT Fc Control Efgartigimod (20 mg/kg, IV) Vehicle 0 0 0 5 10 15 20 25 Time (Days) Concentration (nM) Non-human primates (NHPs) were dosed with IV bolus of 20 mg/kg VRDN-006, 20 mg/kg efgartigimod (internally generated benchmark), or buffer vehicle every 4 days for 4 doses. 68 Source: Viridian data on file. IgG = Immunoglobulin G, IV = intravenous, NHP = non-human primate, PK = pharmacokinetics, WT Fc = wild type neonatal fragment. 0.0001 0.001 0.01 0.1 1 10 100 1000 0.01 0.1 1 10 100 1000 10000 Mean Flourescence Intensity Mean Flourescence Intensity IgG Response (%) Serum Concentration [μg/mL]
v VRDN-006 spares albumin and LDL in multi-dose NHP study VRDN-006 Spares Albumin VRDN-006 Spares LDL 150 200 Dose Dose 150 100 100 VRDN-006 (20 mg/kg, IV) 50 Efgartigimod (20 mg/kg, IV) VRDN-006 (20 mg/kg, IV) Vehicle 50 Efgartigimod (20 mg/kg, IV) Vehicle 0 0 0 5 10 15 20 25 0 5 10 15 20 25 Time (Days) Time (Days) Non-human primates (NHPs) were dosed with IV bolus of 20 mg/kg VRDN-006, 20 mg/kg efgartigimod (internally generated benchmark), or buffer vehicle every 4 days for 4 doses. 69 Source: Viridian data on file. CFB = change from baseline, IV = intravenous, LDL = low-density lipoprotein, NHP = non-human primate. CFB Concentrations (%) CFB Concentrations (%)
A single dose of VRDN-008 demonstrated a longer half-life, v deeper and more sustained reduction of IgG vs. efgartigimod VRDN-008 Showed ~3x Longer Half-life VRDN-008 Showed ~20% Deeper and More Head-to-Head vs. Efgartigimod in NHPs Sustained IgG Reduction Head-to-Head vs. Efgartigimod in NHPs 10000 150 VRDN-008 (60 mg/kg, IV) Efgartigimod (48.5 mg/kg, IV) 125 100 100 75 1 50 VRDN-008 (60 mg/kg, IV) Efgartigimod levels were Efgartigimod (48.5 mg/kg, IV) 25 BLQ at Days 35 and 42 Vehicle 0 0.01 0 7 14 21 28 35 42 0 7 14 21 28 35 42 Time (Days) Time (Days) Non-human primates (NHPs) were given equimolar doses of 60 mg/kg VRDN-008, 48.5 mg/kg efgartigimod (internally generated benchmark), or buffer vehicle - all via IV bolus. 70 Source: Viridian data on file. BLQ = below limit of quantification, IgG = Immunoglobulin G, IV = intravenous, NHP = non-human primate. Concentration (μg/ml) IgG Concentration Mean % Change from Baseline
v A single dose of VRDN-008 spares albumin and LDL in NHPs VRDN-008 Spares Albumin VRDN-008 Spares LDL 125 150 100 100 VRDN-008 (60 mg/kg, IV) 75 VRDN-008 (60 mg/kg, IV) Efgartigimod (48.5 mg/kg, IV) Efgartigimod (48.5 mg/kg, IV) Vehicle Vehicle 50 50 0 7 14 21 28 35 42 0 7 14 21 28 35 42 Time (Days) Time (Days) Non-human primates (NHPs) were given equimolar doses of 60 mg/kg VRDN-008, 48.5 mg/kg efgartigimod (internally generated benchmark), or buffer vehicle - all via IV bolus. 71 Source: Viridian data on file. IV = intravenous, LDL = low-density lipoprotein, NHPs = non-human primates. Albumin Concentration Mean % Change from Baseline LDL Concentration Mean % Change from Baseline