Viridian Therapeutics Highlights Recent Progress and Reports First Quarter 2025 Financial Results

- Biologics License Application $(BLA.AU)$ submission for veligrotug on track for second half 2025 with potential for U.S. launch in 2026; preparatory commercial activities underway -

- REVEAL-1 and REVEAL-2, phase 3 clinical trials assessing VRDN-003 in active and chronic thyroid eye disease (TED), are on track for topline data in the first half of 2026 -

- VRDN-006 clinical data in healthy volunteers on track for third quarter 2025 -

- VRDN-008, a bispecific neonatal Fc receptor (FcRn) inhibitor with an extended half-life, on track for an Investigational New Drug $(IND.AU)$ submission for year-end 2025 -

- Appointed Jeff Ajer, long-time Chief Commercial Officer of BioMarin, to Viridian's Board of Directors -

- Strong cash position of $636.6 million as of March 31, 2025, which supports cash runway into the second half of 2027 -

WALTHAM, Mass.--(BUSINESS WIRE)--May 06, 2025-- 

Viridian Therapeutics, Inc. (Nasdaq: VRDN), a biotechnology company focused on discovering, developing and commercializing potential best-in-class medicines for serious and rare diseases, today reported recent business highlights and financial results for the first quarter ended March 31, 2025.

"We continue to execute across the portfolio as we work towards submitting the veligrotug BLA in the second half of 2025, advancing our VRDN-003 subcutaneous clinical trials with anticipated topline data from both trials in the first half of 2026, and delivering healthy volunteer data for our first FcRn program in the third quarter of 2025," said Steve Mahoney, Viridian's President and CEO. "As we prepare for our transition to become a commercial organization, including the anticipated U.S. launch of veligrotug in 2026, we are excited to have added Jeff Ajer, an experienced commercial leader, to our Board of Directors. We believe the data from our two pivotal clinical trials support veligrotug having a differentiated clinical profile and believe it will be well-positioned to be the IV treatment-of-choice in TED. From a portfolio perspective, we are in many ways just getting started and we look forward to the potential of bringing new treatment options to TED patients as well as other autoimmune patients who may benefit from our anti-FcRn approaches."

TED Portfolio Progress

Veligrotug is an intravenously (IV) delivered, anti-insulin-like growth factor-1 receptor (IGF-1R) antibody in phase 3 development for thyroid eye disease, with the potential to be the IV treatment-of-choice for active and chronic TED patients.

   -- BLA On Track for 2H 2025; European Medicines Agency (EMA) Marketing 
      Authorization Application $(MAA)$ Submission Expected 1H 2026: Following 
      achievement of all primary and secondary endpoints in two pivotal phase 3 
      clinical trials for patients with active and chronic TED, Viridian is on 
      track to submit the veligrotug BLA to the U.S. Food and Drug 
      Administration (FDA) in the second half of 2025 and a MAA to the EMA in 
      the first half of 2026. Preparatory commercial activities are underway to 
      support an anticipated commercial launch in 2026, if approved. 
 
   -- Robust Clinical Profile Based on Phase 3 Pivotal Data: In its pivotal 
      phase 3 clinical trials, THRIVE and THRIVE-2, veligrotug demonstrated a 
      rapid onset of treatment effect and statistically significant and 
      clinically meaningful reduction and resolution of diplopia. This is the 
      first data set from a global phase 3 clinical trial in chronic TED 
      patients to demonstrate statistically significant diplopia response and 
      resolution. Veligrotug was generally well tolerated and had a low rate of 
      hearing impairment, a key adverse event of interest, in both clinical 
      trials. 

VRDN-003 is a potential best-in-class, subcutaneous, half-life extended anti-IGF-1R antibody with the same binding domain as veligrotug.

   -- Topline Data from Phase 3 Clinical Trials On Track for 1H 2026: Viridian 
      anticipates topline data from both REVEAL-1 and REVEAL-2 in the first 
      half of 2026, with a BLA submission planned by year-end 2026. Patient 
      enrollment and dosing continues in both phase 3 clinical trials. Viridian 
      plans to launch VRDN-003, if approved, with a commercially available, 
      low-volume autoinjector for patients to self-administer at home. 

FcRn Inhibitor Portfolio Progress

FcRn inhibitors have the potential to treat a broad array of autoimmune diseases, representing multiple significant potential commercial market opportunities. The two currently marketed indications of myasthenia gravis $(MG)$ and chronic inflammatory demyelinating polyneuropathy (CIDP) alone are projected to have a market size close to $10 billion by 2030. An additional 17 indications are currently in clinical development with an FcRn inhibitor, with dozens more autoimmune diseases thought to be addressable by these inhibitors, highlighting the breadth of the therapeutic area and potential commercial opportunity.

VRDN-006 is a highly selective Fc fragment which inhibits FcRn and is designed to be a convenient subcutaneous and self-administered option for patients.

   -- Proof-of-Concept Phase 1 Clinical Data On Track for Q3 2025: Viridian 
      expects data from its phase 1 clinical trial in healthy volunteers in Q3 
      2025, including proof-of-concept IgG reduction. 

VRDN-008 is a half-life extended bispecific FcRn inhibitor comprising an Fc fragment and an albumin-binding domain designed to prolong IgG suppression and provide a potentially best-in-class subcutaneous option for patients.

   -- IND On Track for Year-End 2025: As previously disclosed, VRDN-008 showed 
      a longer half-life than efgartigimod and led to a more sustained IgG 
      reduction after a single, high dose in NHPs. An IND submission for 
      VRDN-008 is on track by year-end 2025. 

Corporate Updates -- Appointment of Jeff Ajer to Viridian's Board of Directors

On April 7, Viridian announced the appointment of Jeff Ajer to the Company's Board of Directors. Mr. Ajer was most recently the Executive Vice President and Chief Commercial Officer of BioMarin Pharmaceutical, Inc. and has more than 25 years of experience driving commercialization for rare diseases and specialty medicines. Mr. Ajer's extensive commercial experience includes leading commercial planning for late-stage pipeline programs, multiple product launches, and establishing BioMarin's commercial infrastructure and global footprint.

Upcoming Investor Conferences

Viridian will participate in the following upcoming investor conferences in May 2025. A live webcast of the presentation can be accessed under "Events and Presentations" on the Investors section of the Viridian website at viridiantherapeutics.com. A replay of the webcast will be available following the event.

   -- RBC Global Healthcare Conference: Presentation on Tuesday, May 20, 2025, 
      at 1:35 p.m. ET in New York, New York. 

Financial Results

   -- Cash Position: Cash, cash equivalents, and short-term investments were 
      $636.6 million as of March 31, 2025, compared with $717.6 million as of 
      December 31, 2024. The company believes that its current cash, cash 
      equivalents, and short-term investments will be sufficient to fund its 
      currently planned operations into the second half of 2027. 
 
   -- R&D Expenses: Research and development expenses were $76.8 million during 
      the three months ended March 31, 2025, compared to $40.9 million during 
      the three months ended March 31, 2024. The increase in research and 
      development expenses was driven by increased costs associated with 
      conducting more clinical trials than the same period last year, including 
      multiple ongoing phase 3 clinical trials for both veligrotug and VRDN-003 
      and a phase 1 clinical trial for VRDN-006, as well as increased 
      personnel-related costs as a result of increased headcount. 
 
   -- G&A Expenses: General and administrative expenses were $17.1 million 
      during the three months ended March 31, 2025, compared to $15.0 million 
      during the three months ended March 31, 2024. The increase in general and 
      administrative expenses was driven by increased costs associated with 
      preparatory commercial activities for veligrotug, as well as increased 
      legal, accounting, and other professional service costs to support the 
      growing organization. 
 
   -- Shares Outstanding: As of March 31, 2025, Viridian had 100,258,627 shares 
      of common stock outstanding on an as-converted basis, which included 
      81,589,427 shares of common stock and an aggregate 18,669,200 shares of 
      common stock issuable upon the conversion of 134,864 and 145,160 shares 
      of Series A and Series B preferred stock, respectively. 

About Viridian Therapeutics

Viridian is a biopharmaceutical company focused on discovering, developing and commercializing 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.

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