Business Overview and History
Viridian Therapeutics is a biopharmaceutical company dedicated to discovering and developing potential best-in-class medicines for patients suffering from serious and rare diseases. With a focus on addressing unmet medical needs, the company has built a robust pipeline of therapeutic candidates targeting clinically validated molecular pathways.
Viridian Therapeutics was founded in 2008 with the vision of discovering and developing novel treatments for patients suffering from serious diseases. The company was established with the goal of advancing new therapies that could address unmet medical needs. In 2020, Viridian entered into a license agreement with Zenas BioPharma, granting the company exclusive rights to develop and commercialize certain IGF-1R directed antibody products in the greater China region. This partnership represented an important milestone for Viridian as it expanded the reach of its pipeline globally.
To bolster its pipeline, in 2021 and 2022 Viridian entered into several additional strategic collaborations and licensing agreements, including deals with Paragon Therapeutics and Enable Injections. These transactions broadened Viridian's portfolio of novel therapeutic candidates, particularly in the area of autoimmune diseases through the development of FcRn inhibitors. However, the company faced challenges in 2021 and 2022 as it navigated the complexities of developing combination products that included both a biologic drug and a delivery device. Navigating the regulatory landscape for these complex programs has presented some hurdles for the company.
Despite these challenges, Viridian has made important progress in advancing its lead candidate, veligrotug, for the treatment of thyroid eye disease. The company reported positive topline data from the phase 3 THRIVE clinical trial in 2024, which represented a significant milestone. However, the company has continued to face competition from other companies developing treatments for this disease area.
Viridian's pipeline now includes several programs at various stages of clinical development. The company's most advanced candidate, veligrotug, has demonstrated promising results in clinical trials for TED, a serious and debilitating autoimmune condition. Additionally, Viridian is developing a portfolio of novel neonatal Fc receptor (FcRn) inhibitors, including VRDN-006 and VRDN-008, which have the potential to address a broad array of autoimmune diseases.
Financial Overview
As of September 30, 2024, Viridian reported $753.20 million in cash, cash equivalents, and short-term investments, providing the company with a strong financial foundation to advance its research and development efforts. The company does not currently generate significant revenue from product sales, as its pipeline is still in the clinical development stage.
For the nine months ended September 30, 2024, Viridian reported a net loss of $190.22 million, compared to a net loss of $170.87 million for the same period in the prior year. The increase in net loss was primarily driven by higher research and development expenses associated with the advancement of the company's clinical programs, particularly the ongoing phase 3 trials for veligrotug.
Viridian's cash position has been bolstered by several successful financing activities, including a $150 million public offering in January 2024 and a $258.80 million public offering in September 2024. These capital raises have positioned the company to fund its operations and clinical development plan into the second half of 2027.
For the most recent fiscal year 2023, Viridian reported revenue of $314,000, a net loss of $237,734,000, operating cash flow of -$184,170,000, and free cash flow of -$185,068,000. In the most recent quarter (Q3 2024), the company reported revenue of $86,000, a net loss of $76,689,000, operating cash flow of -$67,706,000, and free cash flow of -$67,999,000. Year-over-year revenue growth was 19.44%, primarily attributable to a $14,000 increase in collaboration revenue from the company's agreement with Zenas BioPharma.
For the nine months ended September 30, 2024, Viridian reported collaboration revenue of $230 million related to its agreement with Zenas BioPharma. The company's research and development expenses were $166.29 million, and its general and administrative expenses were $45.50 million for the same period.
Liquidity
Viridian's strong cash position of $753.20 million as of September 30, 2024, provides the company with significant liquidity to fund its ongoing operations and clinical development programs. The successful completion of two public offerings in 2024 has further strengthened the company's financial position, extending its cash runway into the second half of 2027. This robust liquidity allows Viridian to continue advancing its pipeline without the immediate need for additional financing, providing stability and flexibility in its development efforts.
As of September 30, 2024, Viridian reported a debt-to-equity ratio of 0.029, with $200,320,000 in cash and cash equivalents. The company has access to additional undrawn funds under the Hercules Amended Term Loan. Viridian's current ratio and quick ratio both stand at 18.55, indicating a strong ability to meet short-term obligations.
Thyroid Eye Disease: Veligrotug's Potential
Viridian's lead candidate, veligrotug, has emerged as a promising treatment option for patients suffering from TED, a severe and debilitating autoimmune disorder. In September 2024, the company reported positive topline results from its pivotal phase 3 THRIVE trial, which evaluated the efficacy and safety of veligrotug in patients with active TED.
The THRIVE trial demonstrated that veligrotug achieved all primary and secondary endpoints with a high degree of statistical significance. Notably, the study showed a 70% proptosis responder rate (PRR) at week 15, with a placebo-adjusted PRR of 64% (p < 0.0001). Additionally, veligrotug treatment led to clinically meaningful patient outcomes, including a 54% complete resolution of diplopia (placebo-adjusted rate of 43%) and a 64% reduction in Clinical Activity Score (CAS) to 0 or 1 (placebo-adjusted reduction of 46%).
Importantly, veligrotug was generally well-tolerated, with no treatment-related serious adverse events (SAEs) reported. The study did, however, observe a 5.5% placebo-adjusted rate of hearing impairment adverse events, an important safety signal that the company will continue to monitor.
The positive THRIVE results position veligrotug as a potential best-in-class treatment for active TED, a market currently dominated by Tepezza (teprotumumab), which was approved by the FDA in 2020. Viridian is now preparing to report topline data from its THRIVE-2 trial, which is evaluating veligrotug in patients with chronic TED, by the end of 2024.
Viridian plans to file a Biologics License Application (BLA) for veligrotug in the second half of 2025, marking a significant milestone in the company's regulatory pathway for this promising candidate.
FcRn Inhibitor Portfolio: Addressing Autoimmune Diseases
In addition to its lead TED program, Viridian is developing a portfolio of novel FcRn inhibitors, which have the potential to address a broad array of autoimmune diseases. The company's most advanced FcRn programs are VRDN-006 and VRDN-008.
VRDN-006 is a FcRn-targeting Fc fragment that has demonstrated specificity for blocking FcRn-IgG interactions in non-human primate studies, without the decreases in albumin or increases in LDL levels that have been associated with certain full-length anti-FcRn monoclonal antibodies. In head-to-head studies, VRDN-006 has shown comparable potency and IgG reductions to efgartigimod, the current standard of care in FcRn inhibition, while maintaining a similar safety profile.
Viridian's next-generation FcRn inhibitor, VRDN-008, aims to pair IgG suppression with extended half-life technology, potentially enabling deeper and more durable suppression of IgG than existing anti-FcRn therapies. The company plans to submit an Investigational New Drug (IND) application for VRDN-008 by the end of 2025 and anticipates proof-of-concept IgG reduction data in healthy volunteers in the second half of 2026.
Viridian plans to file an IND application for VRDN-006 by the end of 2024 and expects healthy volunteer data in the second half of 2025, further advancing its FcRn inhibitor portfolio.
Subcutaneous TED Program: VRDN-3
In addition to veligrotug, Viridian is advancing VRDN-3, a subcutaneous anti-IGF-1R product candidate for TED. In December 2023, the company selected VRDN-3 as its lead subcutaneous product candidate following positive data from a phase 1 clinical trial in healthy volunteers.
Viridian initiated two global phase 3 trials, REVEAL-1 and REVEAL-2, to evaluate VRDN-3 in active and chronic TED in August 2024. The company anticipates topline data from these trials in the first half of 2026 and plans to submit a BLA for VRDN-3 by the end of 2026. This subcutaneous formulation could potentially offer patients a more convenient administration option compared to intravenous treatments.
Risks and Challenges
While Viridian's pipeline holds significant promise, the company faces several risks and challenges common to the biopharmaceutical industry. The successful development and commercialization of its product candidates, including veligrotug and the FcRn inhibitor programs, are subject to the inherent uncertainties of the clinical trial process, regulatory approvals, and market acceptance.
Furthermore, Viridian's reliance on third-party contract research organizations (CROs) and contract manufacturing organizations (CMOs) for the conduct of its clinical trials and the production of its product candidates introduces operational and supply chain risks. Disruptions in these critical relationships or the company's manufacturing processes could delay or impede the progress of Viridian's development programs.
The competitive landscape in the TED and autoimmune disease spaces also poses a significant challenge, as Viridian's products may face competition from both approved therapies and other investigational agents in development by larger pharmaceutical and biotechnology companies.
Conclusion
Viridian Therapeutics is a biopharmaceutical company with a promising pipeline of innovative therapies targeting serious and rare diseases. The company's lead candidate, veligrotug, has demonstrated impressive results in clinical trials for the treatment of thyroid eye disease, a debilitating autoimmune condition. Additionally, Viridian's portfolio of FcRn inhibitors, including VRDN-006 and VRDN-008, hold the potential to address a broad range of autoimmune disorders.
Despite the challenges inherent to the industry, Viridian's strong financial position, with over $750 million in cash and investments, and its commitment to advancing its pipeline of differentiated product candidates, position the company well to continue its pursuit of developing transformative therapies for patients in need. The company's focus on both intravenous and subcutaneous formulations for TED treatment, along with its expanding FcRn inhibitor portfolio, demonstrates a strategic approach to addressing unmet medical needs in the autoimmune disease space.
As Viridian progresses towards potential regulatory submissions for veligrotug and VRDN-3, and advances its FcRn inhibitor programs, the company remains well-positioned to capitalize on the growing market for targeted therapies in rare and serious diseases. With a strong cash runway extending into the second half of 2027, Viridian has the financial resources to support its ambitious clinical development plans and potentially bring innovative treatments to patients in need.