Business Overview and History

Viridian Therapeutics is a biopharmaceutical company laser-focused on discovering, developing, and commercializing potential best-in-class medicines for serious and rare diseases. With a proven track record of innovation and a commitment to addressing unmet medical needs, Viridian has emerged as a rising force in the dynamic pharmaceutical landscape.

Viridian Therapeutics has a rich history that dates back to its founding as miRagen Therapeutics, Inc. in January 2010. Initially established as a Delaware limited liability company, miRagen was subsequently incorporated as a Delaware corporation in June 2014. The company's focus at that time was on the development of microRNA-based therapeutics.

A significant turning point came in January 2021 when miRagen Therapeutics acquired Viridian Therapeutics, Inc. through a merger agreement. This transformative event led to the company changing its name from Miragen Therapeutics, Inc. to Viridian Therapeutics, Inc., marking a new chapter in its corporate journey.

The merger process in 2020 presented substantial challenges, requiring significant time and resources to integrate the two companies effectively. Additionally, Viridian has had to navigate the complex regulatory landscape for biopharmaceutical products, working diligently to obtain necessary approvals from the FDA and other regulatory bodies.

Despite these hurdles, Viridian has achieved several important milestones. In October 2020, the company entered into a license agreement with Zenas BioPharma, securing an exclusive license to develop, manufacture, and commercialize certain IGF-1R directed antibody products for non-oncology indications in the greater area of China. This strategic move expanded Viridian's geographical reach and potential market opportunities.

Further enhancing its capabilities, in January 2022, Viridian entered into an antibody and discovery option agreement with Paragon Therapeutics, Inc. This collaboration aims to develop one or more therapeutic proteins or antibodies, with Viridian holding the option to obtain exclusive licenses for development, manufacturing, and commercialization of certain antibodies, proteins, and associated products.

Under the leadership of CEO Stephen Mahoney, Viridian has leveraged its expertise in protein and antibody engineering to advance a diverse pipeline of promising product candidates. The company's most advanced program, veligrotug, is a differentiated monoclonal antibody targeting the insulin-like growth factor-1 receptor (IGF-1R), a clinically and commercially validated target for the treatment of thyroid eye disease (TED).

Thyroid Eye Disease A Debilitating Rare Autoimmune Condition

TED is a serious and rare autoimmune disease that causes inflammation within the orbit of the eye, leading to a range of debilitating symptoms such as bulging eyes, redness, swelling, double vision, pain, and potential blindness. Prior to the approval of Tepezza (teprotumumab) by the FDA in 2020, TED patients had limited treatment options, often resorting to systemic steroids or invasive surgical interventions, both of which carried significant risks and limitations.

Viridian's Innovative Approach Veligrotug and VRDN-003

Viridian's most advanced product candidate, veligrotug, is a differentiated monoclonal antibody that acts as a full antagonist of IGF-1R, a mechanism of action that is clinically and commercially validated by Tepezza. In the company's phase 3 THRIVE and THRIVE-2 trials, veligrotug demonstrated impressive results, achieving statistically significant improvements in key TED symptoms, including proptosis, diplopia, and Clinical Activity Score (CAS), with a favorable safety profile.

The THRIVE study enrolled 113 patients with active TED and met all primary and secondary endpoints with statistical significance. Similarly, the THRIVE-2 trial, which enrolled 188 patients with chronic TED, also achieved its endpoints. Both studies demonstrated that veligrotug was generally well-tolerated.

Notably, the veligrotug regimen features fewer infusions and a shorter time per infusion compared to Tepezza, potentially offering patients a more convenient treatment option. To meet the 300-patient safety database requirement for the Biologics License Application (BLA), Viridian is conducting the STRIVE study, which had enrolled 231 patients as of January 2025, exceeding the target enrollment. The company plans to submit a BLA for veligrotug in the second half of 2025 and a Marketing Authorization Application (MAA) to the European Medicines Agency in the first half of 2026.

In addition to veligrotug, Viridian is developing VRDN-003 (previously referred to as VRDN-3.00), an extended half-life monoclonal antibody with the same binding domains as veligrotug, designed for administration as a convenient, low-volume, subcutaneous injection. A phase 1 study in healthy volunteers showed that VRDN-003 had a prolonged half-life of 40-50 days, four to five times longer than veligrotug.

The company's ongoing phase 3 REVEAL-1 and REVEAL-2 trials are evaluating the efficacy and safety of VRDN-003 dosed every four or eight weeks in active and chronic TED patients, respectively. Viridian anticipates reporting topline data from these trials in the first half of 2026 and plans to submit a BLA for VRDN-003 by the end of 2026. If successful, VRDN-003 could provide patients with an even more patient-friendly treatment option for TED.

FcRn Inhibitors Expanding the Therapeutic Horizon

Alongside its efforts in TED, Viridian is also developing a portfolio of engineered neonatal Fc receptor (FcRn) inhibitors, including VRDN-006 and VRDN-008. FcRn inhibitors have the potential to treat a broad array of autoimmune diseases, representing a significant commercial opportunity.

VRDN-006 (previously referred to as VRDN-6.00), a highly selective Fc fragment inhibitor of FcRn, is designed to be a convenient subcutaneous and self-administered option for patients. In non-human primate studies, VRDN-006 demonstrated comparable potency and IgG reductions to the current standard of care, efgartigimod, as well as a similar safety profile. Viridian submitted an Investigational New Drug (IND) application for VRDN-006 in December 2024, which was cleared in January 2025. The company expects to report proof-of-concept IgG reduction data in healthy volunteers in the third quarter of 2025.

VRDN-008 (previously referred to as VRDN-8.00), a half-life extended bispecific FcRn inhibitor, is designed to provide a potentially best-in-class subcutaneous option for patients. Impressive data from Viridian's non-human primate studies have shown VRDN-008 to have a three-fold increase in half-life and a deeper, more sustained IgG reduction compared to efgartigimod. In a head-to-head study, VRDN-008 demonstrated three times the half-life of efgartigimod and a deeper and more sustained IgG reduction. The company plans to submit an IND for VRDN-008 by the end of 2025.

Financial Performance and Liquidity

Viridian Therapeutics has a strong financial position to support its ambitious development pipeline. As of December 31, 2024, the company had $717.6 million in cash, cash equivalents, and short-term investments, providing ample runway to fund operations into the second half of 2027.

For the full year 2024, Viridian reported total revenue of $302,000, primarily reflecting revenue from its collaboration agreement with Zenas BioPharma. The company's net loss for the year was $269.95 million, driven by increased research and development expenses as it progressed its clinical trials. Research and development expenses were $238.25 million in 2024, while general and administrative expenses were $61.08 million.

Viridian's operating cash flow for 2024 was ($232.32) million, and its free cash flow was ($232.83) million. The company's debt-to-equity ratio stands at 0.031, indicating a low level of leverage. Viridian's current ratio and quick ratio are both 15.4, suggesting strong short-term liquidity.

In addition to its cash reserves, Viridian has access to additional undrawn funds under the Hercules Loan and Security Agreement Amended Term Loan, further bolstering its financial flexibility.

Key Risks and Challenges

As with any biopharmaceutical company, Viridian Therapeutics faces several risks and challenges that investors should be aware of:

1. Clinical Development Risks: The success of Viridian's product candidates is dependent on positive outcomes from its clinical trials. Delays, failures, or safety concerns in these trials could significantly impact the company's prospects.

2. Regulatory Approval Uncertainty: Even with positive clinical data, there is no guarantee that Viridian's product candidates will receive regulatory approval from the FDA or other governing bodies. Failure to obtain approval would severely limit the company's commercial potential.

3. Competition and Pricing Pressure: Viridian's product candidates, if approved, will face competition from existing and potentially emerging therapies in the TED and autoimmune disease spaces. Pricing and reimbursement challenges could also impact the commercial viability of Viridian's products.

4. Reliance on Third-Party Manufacturers: Viridian does not have in-house manufacturing capabilities and relies on third-party contract manufacturers. Any disruptions or quality issues in the supply chain could delay the development and commercialization of its product candidates.

5. Potential for Additional Funding Requirements: As Viridian continues to advance its pipeline, the company may require additional capital to support its operations. Failure to secure adequate funding could hinder the company's ability to execute on its strategic objectives.

Conclusion

Viridian Therapeutics has emerged as a promising player in the rare disease pharmaceutical landscape, with a robust pipeline of innovative product candidates and a steadfast commitment to addressing unmet medical needs. The company's lead program, veligrotug, has demonstrated compelling results in clinical trials for the treatment of TED, a debilitating condition with limited treatment options. Additionally, Viridian's FcRn inhibitor portfolio holds significant potential to expand the company's therapeutic reach into the broader autoimmune disease space.

While Viridian faces the inherent risks associated with biopharmaceutical development, the company's strong financial position, experienced management team, and innovative approach position it well to navigate the challenges and potentially deliver transformative treatments to patients in need. As Viridian continues to execute on its strategic initiatives, investors will undoubtedly keep a close eye on the company's progress and its ability to capitalize on the significant opportunities within the rare disease market.