Viridian Therapeutics Highlights 2025 Corporate Priorities Following Positive Phase 3 Topline Data in Thyroid Eye Disease

Rhea-AI Summary

Viridian Therapeutics (NASDAQ: VRDN) announces its 2025 priorities following positive Phase 3 results for veligrotug in thyroid eye disease (TED). The company plans to submit a Biologics License Application (BLA) for veligrotug in H2 2025, following successful THRIVE and THRIVE-2 trials showing significant improvements in proptosis, clinical activity score, and diplopia.

Two Phase 3 trials (REVEAL-1 and REVEAL-2) for subcutaneous VRDN-003 are ongoing, with topline data expected in H1 2026. The company submitted an IND for VRDN-006 (FcRn inhibitor) in December 2024, with clinical data expected in Q3 2025. VRDN-008, a bispecific FcRn inhibitor, is advancing with preclinical studies.

The company reports a strong cash position of $753 million as of September 2024, providing runway into H2 2027.

Positive

• Positive Phase 3 topline data achieved for veligrotug in both active and chronic TED trials
• Strong cash position of $753M providing runway through H2 2027
• 54% of treated patients achieved complete resolution of diplopia in THRIVE trial
• Rapid onset of treatment effect with majority showing response after one infusion
• Low adverse event rates with 5.5-9.6% placebo-adjusted hearing impairment

Negative

• BLA submission timeline extends to second half of 2025
• VRDN-003 topline data not expected until first half of 2026

Insights

Biotech Research Analyst

The topline results from THRIVE and THRIVE-2 trials for veligrotug represent a major clinical breakthrough in Thyroid Eye Disease treatment. The drug achieved 54% complete diplopia resolution and demonstrated rapid efficacy after just one infusion. The low 5.5-9.6% hearing impairment rates suggest a superior safety profile compared to existing treatments.

The subcutaneous VRDN-003 program, sharing the same binding domain as veligrotug but with a 4-5x longer half-life, could revolutionize treatment convenience with Q4W or Q8W dosing. The robust <money>753 million</money> cash position provides runway through H2 2027, covering multiple key milestones including commercial launch.

Investment Portfolio Manager

The company's dual-pronged approach with both IV and subcutaneous formulations positions it strongly in the TED market. The successful phase 3 results significantly de-risk the investment thesis, while the FcRn inhibitor portfolio adds valuable pipeline diversification. The cash runway into H2 2027 eliminates near-term financing concerns, enabling focus on execution.

The potential best-in-class profile of both veligrotug and VRDN-003, combined with the expanding autoimmune disease portfolio, suggests substantial market opportunity. Key catalysts in 2025-2026, including the BLA submission and REVEAL trial results, provide multiple value inflection points.

Medical Device Specialist

The planned autoinjector pen delivery system for VRDN-003 represents a significant competitive advantage in the TED market. Self-administration capability could drive higher patient compliance and market penetration. The comprehensive clinical program, studying both active and chronic TED in the largest patient population to date, provides robust evidence for regulatory approval and market adoption.

The development of VRDN-008 with 3x longer half-life than efgartigimod and 20% deeper IgG reduction demonstrates superior engineering capabilities in biologic drug development.

- Veligrotug, with recent positive phase 3 topline data in both active and chronic thyroid eye disease (TED), has the potential to transform the standard of care in TED with a differentiated clinical profile achieved with less drug and fewer infusions; Biologics License Application (BLA) submission on track for second half of 2025 -

- REVEAL-1 and REVEAL-2, phase 3 clinical trials for subcutaneous (SC) VRDN-003 in active and chronic TED respectively, are currently dosing patients; topline data for both trials, which are evaluating VRDN-003 dosed Q4W or Q8W, is on track for the first half of 2026 -

- Investigational New Drug (IND) submitted in December 2024 for VRDN-006, an Fc fragment inhibitor of the neonatal Fc receptor (FcRn); proof-of-concept IgG reduction clinical data in healthy volunteers anticipated in the third quarter of 2025 -

- VRDN-008, a bispecific FcRn inhibitor with an extended half-life, progressing with preclinical studies as planned; additional preclinical data expected in 2025 with an IND submission planned for year-end 2025 -

- Strong cash position of $753 million as of September 30, 2024; provides cash runway into the second half of 2027 through the anticipated commercial launch of veligrotug, topline results and BLA submission for VRDN-003, and multiple FcRn inhibitor portfolio clinical catalysts -

WALTHAM, Mass.--(BUSINESS WIRE)-- Viridian Therapeutics, Inc. (NASDAQ: VRDN), a biotechnology company focused on discovering and developing potential best-in-class medicines for serious and rare diseases, today announces the company’s key priorities and catalysts for 2025.

“After a great year of execution across the portfolio, we enter 2025 poised to deliver on a number of important catalysts,” said Steve Mahoney, Viridian’s President and CEO. “We believe that the positive and better-than-expected topline data for veligrotug in both THRIVE and THRIVE-2 confirms its potential to be the treatment-of-choice for all forms of active and chronic TED at its anticipated commercial launch. Our BLA and early commercial preparations are well underway, and we are on track to submit the BLA in the second half of this year. The results of THRIVE and THRIVE-2 give us even higher conviction that our subcutaneous VRDN-003 program will deliver positive topline data in the first half of 2026, which would enable a BLA submission for VRDN-003 in the second half of 2026.

“In addition to TED, we are equally excited by the potential of our FcRn inhibitor portfolio. In Q3 of this year, we anticipate showing proof-of-concept data, including IgG reduction, from our planned VRDN-006 phase 1 clinical trial in healthy volunteers. We also continue to advance our bispecific, half-life extended, potential best-in-class VRDN-008 program. We previously showed that, in non-human primates (NHPs), VRDN-008 had 3x the half-life of efgartigimod and a more sustained IgG reduction. We anticipate sharing additional data from ongoing VRDN-008 preclinical studies later this year. We have another exciting year ahead and we look forward to continued execution across our portfolio of derisked programs.”

TED Portfolio: Well-Positioned with Potential Best-in-Class Intravenous (IV) and Subcutaneous Programs

Veligrotug, Potential IV Treatment-of-Choice for Active & Chronic TED

Viridian’s lead product candidate, veligrotug, is a monoclonal antibody that acts as a full antagonist of TED’s clinically and commercially validated target of insulin-like growth factor-1 receptor (IGF-1R). In late 2024, Viridian reported positive topline data for veligrotug in two of the largest global pivotal clinical trials conducted to date in TED, THRIVE and THRIVE-2. Viridian plans to submit a BLA for veligrotug for the treatment of patients with active and chronic TED in the second half of this year.

• Phase 3 THRIVE Results in Active TED - Achieved All Primary and Secondary Endpoints: Viridian announced positive topline results for THRIVE in September 2024. Veligrotug met all the primary and secondary endpoints at 15 weeks, including measures of proptosis, clinical activity score (CAS), and diplopia, with a high degree of statistical significance. Highlights of clinical efficacy included 54% of treated patients achieving complete resolution of their diplopia, or double vision, and a consistently rapid onset of treatment effect across all disease endpoints, with a majority of patients achieving a proptosis response after just one infusion, or 3 weeks after the start of therapy. Veligrotug was generally well-tolerated with no treatment-related serious adverse events, and importantly for a key area of interest in the class, had a low 5.5% placebo-adjusted rate of hearing impairment adverse events (AEs).
• Phase 3 THRIVE-2 Results in Chronic TED - Achieved All Primary and Secondary Endpoints: Viridian announced positive topline results for THRIVE-2 in December 2024. THRIVE-2 studied the broadest population of chronic TED patients in a global phase 3 clinical trial to date and, consistent with THRIVE, veligrotug met all the primary and secondary endpoints at 15 weeks with a high degree of statistical significance. Additionally, veligrotug is the first product candidate to demonstrate a statistically significant and clinically meaningful reduction and resolution of diplopia in patients with chronic TED. Also consistent with THRIVE, veligrotug demonstrated a rapid onset of treatment effect, showing a statistically significant proptosis response as early as 3 weeks and diplopia reduction and resolution as early as 6 weeks. Similar to THRIVE, veligrotug was generally well-tolerated in THRIVE-2 and showed a low 9.6% placebo-adjusted rate of hearing impairment AEs.

Together, THRIVE and THRIVE-2 evaluated veligrotug in the largest and broadest population of active and chronic TED patients studied to date in global phase 3 clinical trials. Viridian believes that these robust and consistent clinical efficacy and safety results, after only five infusions of veligrotug, support a differentiated product profile and the potential for veligrotug to be the IV treatment-of-choice for all forms of active and chronic TED.

Subcutaneous VRDN-003, a Potential Best-In-Class Anti-IGF-1R for Active and Chronic TED

VRDN-003 is a monoclonal antibody targeting IGF-1R and shares the same binding domain as veligrotug. VRDN-003 is engineered to have an extended half-life, shown to be 40-50 days, or 4-5x that of veligrotug, enabling evaluation of convenient dosing, as infrequently as once every 8 weeks.

With the positive results from THRIVE and THRIVE-2, Viridian views subcutaneous VRDN-003 as further derisked with the potential to be a best-in-class treatment for TED based on:

• Same Binding Domain as Veligrotug IV: VRDN-003 (SC) has the same binding domain as veligrotug (IV) and is therefore expected to inhibit IGF-1R as a full antagonist in the same manner;
• Half-life Extension: VRDN-003's confirmed extended half-life is expected to support infrequent Q4W or Q8W dosing;
• Sustained Pharmacodynamic (PD) Effects: VRDN-003 showed sustained serum increases in IGF-1 levels, a PD biomarker for inhibiting IGF-1R;
• Derisked Dosing Regimens: each REVEAL study is evaluating Q4W and Q8W dosing regimens of VRDN-003, which are each predicted to achieve drug exposure levels that have shown robust clinical activity with veligrotug in TED; and
• Autoinjector Delivery for Patient Convenience and Access: Viridian expects to launch VRDN-003 with a commercially validated autoinjector pen to enable at-home patient self-administration.

Viridian is currently dosing patients in two global phase 3 clinical trials for VRDN-003, REVEAL-1 and REVEAL-2, in active and chronic TED, respectively. Topline data for both REVEAL-1 and REVEAL-2 remains on track for the first half of 2026, which would enable a BLA submission by year-end 2026.

FcRn Inhibitor Portfolio:

In October 2023, Viridian first unveiled VRDN-006 and VRDN-008 in its portfolio of engineered FcRn inhibitors. FcRn inhibitors have the potential to treat a broad array of autoimmune diseases, representing significant commercial market opportunities.

VRDN-006, a Highly-Selective Fc Fragment that Inhibits FcRn

VRDN-006 is an Fc fragment that inhibits FcRn and is designed to be a convenient subcutaneous and self-administered option for patients. In NHPs, VRDN-006 showed specificity for blocking FcRn-IgG interactions while sparing albumin and low-density lipoprotein (LDL).

• IND Submitted in December 2024: As planned, Viridian submitted an IND for VRDN-006 in December 2024 and, upon clearance, anticipates initiating a phase 1 clinical trial in healthy volunteers in early 2025.
• Proof-of-Concept Phase 1 Clinical Data On-Track for Q3 2025: Viridian expects data from the phase 1 clinical trial in Q3 2025, including proof-of-concept IgG reductions in healthy volunteers. These data have historically been translatable to patient populations.

VRDN-008, a Half-life Extended Bispecific FcRn Inhibitor

VRDN-008 comprises an Fc fragment and an albumin-binding domain designed to prolong IgG suppression as a subcutaneous, self-administered, and potential best-in-class option for patients.

• Confirmed Half-Life Extension and Sustained PD Effects: Viridian announced in November 2024 that VRDN-008 demonstrated 3x the half-life of efgartigimod in a single, high-dose, head-to-head study in NHPs. Additionally, VRDN-008 showed a deeper and more sustained IgG reduction in the same study with peak IgG reductions that were 20% deeper than efgartigimod. VRDN-008 spared albumin and LDL, consistent with efgartigimod.
• Additional Preclinical Data Expected in 2025: Additional NHP studies are ongoing to generate data with lower doses and multiple doses of VRDN-008. Once completed, Viridian plans to use the totality of VRDN-008’s NHP data to build a robust pharmacokinetic and pharmacodynamic model to enable the prediction of potential human dosing regimens for VRDN-008.

Corporate Updates

Viridian announces the appointment and promotion of Seth Harmon to the role of Chief Financial Officer. Mr. Harmon joined Viridian in 2023 as the Senior Vice President of Finance and Accounting and has over 20 years of strategic finance, accounting, and operations experience. Prior to joining Viridian, Mr. Harmon served as the CFO of BioNTech US, where he oversaw the general and administrative functions for BioNTech SE’s US subsidiary. Prior to that, Mr. Harmon held multiple positions of increasing seniority in finance at Neon Therapeutics and Merrimack Pharmaceuticals, after starting his career at Ernst and Young, where he spent 10 years in the audit and assurance practices. Mr. Harmon holds an M.S. in Accounting and M.B.A. from Northeastern University and obtained his certified public accountant license while working at Ernst and Young, LLP. He received his B.A. in Economics and Mathematics from Bowdoin College.

Notice of Inducement Grants

Today Viridian announces that a majority of the independent directors serving on the Compensation Committee of the company’s Board of Directors approved the grant of non-qualified stock options to purchase an aggregate of 98,500 shares of the company’s common stock to four new employees (the “Inducement Grants”) on January 2, 2025 (the “Grant Date”). The Inducement Grants have been granted outside of the company’s Amended and Restated 2016 Equity Incentive Plan (the “Plan”) but remain subject to the terms and conditions of such Plan. The Inducement Grants were granted as an inducement material to these individuals entering into employment with Viridian in accordance with Nasdaq Listing Rule 5635(c)(4).

The Inducement Grants have an exercise price per share that is equal to the closing price of Viridian’s common stock on the Grant Date. The Inducement Grants will vest over a four-year period, with 25% of the shares vesting on the one-year anniversary of the employee’s start date, and thereafter the remainder of the shares vest in 36 equal monthly installments, subject to each employee’s continued employment with Viridian through the applicable vesting dates.

About Viridian Therapeutics

Viridian is a biopharmaceutical company focused on engineering and developing potential best-in-class medicines for patients with serious and rare diseases. Viridian’s expertise in antibody discovery and protein engineering enables the development of differentiated therapeutic candidates for previously validated drug targets in commercially established disease areas.

Viridian is advancing multiple candidates in the clinic for the treatment of patients with thyroid eye disease (TED). The company is conducting a pivotal program for veligrotug (VRDN-001), including two global phase 3 clinical trials (THRIVE and THRIVE-2), to evaluate its efficacy and safety in patients with active and chronic TED. Both THRIVE and THRIVE-2 reported positive topline data, meeting all the primary and secondary endpoints of each study. Viridian is also advancing VRDN-003 as a potential best-in-class subcutaneous therapy for the treatment of TED, including two ongoing global phase 3 pivotal clinical trials, REVEAL-1 and REVEAL-2, to evaluate the efficacy and safety of VRDN-003 in patients with active and chronic TED.

In addition to its TED portfolio, Viridian is advancing a novel portfolio of neonatal Fc receptor (FcRn) inhibitors, including VRDN-006 and VRDN-008, which has 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,” “estimate,” “expect,” “intend,” “may,” “might,” “on track,” “plan,” “potential,” “predict,” “project,” “design,” “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 (formerly VRDN-001), VRDN-003, VRDN-006 and VRDN-008, including Viridian's view that the THRIVE and THRIVE-2 data provides strong support for VRDN-003's clinical profile; anticipated start dates of studies, including the initiation of the phase 1 clinical trial for VRDN-006; anticipated data results and timing of their disclosure, including VRDN-003 topline data from the REVEAL-1 and REVEAL-2 trials in the first half of 2026, anticipated VRDN-006 proof-of-concept clinical data in the third quarter of 2025, and anticipated VRDN-008 preclinical data in 2025; regulatory interactions and anticipated timing of regulatory submissions, including the anticipated BLA submissions for veligrotug in the second half of 2025 and VRDN-003 in the second half of 2026, and the anticipated IND submission for VRDN-008 by year-end 2025, pending data; Viridian’s expectation that its data package will support a BLA for VRDN-003; the potential utility, efficacy, potency, safety, clinical benefits, clinical response, convenience and number of indications of veligrotug, VRDN-003, VRDN-006 and VRDN-008; veligrotug’s potential to transform the standard of care and the potential for veligrotug to be the IV treatment-of-choice for all forms of active and chronic TED; Viridian’s product candidates potentially being best-in-class; Viridian’s view that its portfolio programs are derisked, including VRDN-003; whether veligrotug will serve an unmet need; Viridian’s expectations regarding the potential commercialization of veligrotug and VRDN-003, if approved; and that Viridian’s cash, cash equivalents and short-term investments will be sufficient to fund its operations into the second half of 2027.

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 financial position and projected cash runway; our future operating results and financial performance; Viridian’s intellectual property position; the timing of preclinical and clinical trial activities and reporting results from same; that our product candidates may not be commercially successful, if approved and those risks set forth under the caption “Risk Factors” in our most recent quarterly report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on November 12, 2024 and other subsequent disclosure documents filed with the SEC. 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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20250108385710/en/

Anabel Chan, 617-458-8725

Vice President, Investor Relations & Communications

IR@viridiantherapeutics.com

Louisa Stone, 617-272-4604

Manager, Investor Relations

IR@viridiantherapeutics.com

Source: Viridian Therapeutics, Inc.

FAQ

What were the key results from VRDN's Phase 3 THRIVE trials for veligrotug?

The THRIVE trials met all primary and secondary endpoints, with 54% of treated patients achieving complete diplopia resolution. The drug showed rapid onset of treatment effect and was well-tolerated with low hearing impairment adverse event rates.

When will Viridian (VRDN) submit the BLA for veligrotug?

Viridian plans to submit the Biologics License Application (BLA) for veligrotug in the second half of 2025.

What is VRDN's current cash position and runway?

Viridian has $753 million in cash as of September 30, 2024, providing runway into the second half of 2027.

When will VRDN-003's Phase 3 REVEAL trial results be available?

Topline data for both REVEAL-1 and REVEAL-2 trials is expected in the first half of 2026.

What are the key differences between VRDN-003 and veligrotug?

VRDN-003 is a subcutaneous version with an extended 40-50 day half-life (4-5x longer than veligrotug), enabling less frequent dosing of Q4W or Q8W.