Viridian Therapeutics Announces Completion of THRIVE-2 Enrollment for VRDN-001 in Patients with Chronic Thyroid Eye Disease (TED)

Rhea-AI Summary

Viridian Therapeutics (NASDAQ: VRDN) has completed enrollment for its THRIVE-2 phase 3 clinical trial of VRDN-001 in patients with chronic Thyroid Eye Disease (TED). The trial exceeded its target, enrolling 188 patients globally, with about 40% from US sites. Topline data from THRIVE-2 is expected by year-end 2024.

The company's THRIVE trial for active TED also surpassed its enrollment goal in March 2024, with approximately 50% of patients from US sites. Topline results for THRIVE are anticipated in September 2024. Viridian plans to initiate two subcutaneous VRDN-003 phase 3 clinical trials, REVEAL-1 and REVEAL-2, in August as scheduled.

Positive

• Exceeded enrollment targets for both THRIVE and THRIVE-2 clinical trials
• Significant patient demand, particularly from US sites
• On track for timely topline data readouts in September and year-end 2024
• Planned initiation of two additional phase 3 trials (REVEAL-1 and REVEAL-2) in August

Negative

• None.

Insights

Medical Research Analyst

The completion of enrollment for THRIVE-2, Viridian Therapeutics' phase 3 clinical trial for VRDN-001 in chronic Thyroid Eye Disease (TED), marks a significant milestone in the company's drug development journey. The trial's oversubscription, with 188 patients enrolled against a target of 159, indicates strong patient interest and potentially robust market demand for new TED treatments.

Several key points stand out:

• The 40% enrollment from US sites suggests a balanced geographical representation, which could be important for regulatory approvals and market access strategies.
• The on-track status for both THRIVE (active TED) and THRIVE-2 (chronic TED) topline readouts in September and year-end 2024, respectively, positions Viridian for potentially market-moving data releases within the next 12 months.
• The planned initiation of two subcutaneous VRDN-003 phase 3 trials (REVEAL-1 and REVEAL-2) in August indicates a robust pipeline strategy, potentially offering multiple shots on goal in the TED market.

From a medical research perspective, the successful enrollment in both active and chronic TED trials could provide valuable comparative data on treatment efficacy across different disease stages. This comprehensive approach may give Viridian a competitive edge in the TED treatment landscape.

Financial Analyst

Viridian Therapeutics' announcement carries significant implications for its financial outlook and market position. The oversubscription of the THRIVE-2 trial suggests strong market interest, which could translate into faster patient adoption if VRDN-001 receives approval. This potential for rapid market penetration could positively impact future revenue projections.

Key financial considerations include:

• The company's ability to exceed enrollment targets may indicate efficient clinical trial management, potentially reducing time-to-market and associated costs.
• With both THRIVE and THRIVE-2 trials fully enrolled, Viridian can now more accurately forecast its cash burn rate through the end of 2024, providing clearer visibility for investors.
• The planned initiation of two additional phase 3 trials for VRDN-003 suggests continued R&D investment, which may impact short-term financials but could diversify long-term revenue potential.

Investors should monitor the upcoming data readouts closely, as positive results could significantly boost Viridian's valuation and potentially attract partnership or acquisition interest from larger pharmaceutical companies. Conversely, any setbacks in these trials could have a substantial negative impact on the stock price, given the company's focus on these TED programs.

Market Research Analyst

The successful enrollment of THRIVE-2 provides valuable insights into the Thyroid Eye Disease (TED) market dynamics. The oversubscription of the trial, particularly with 40% enrollment from US sites, indicates a significant unmet need in chronic TED treatment, especially in the lucrative US market.

Market implications to consider:

• The strong patient demand suggests a potentially larger addressable market for TED treatments than previously estimated, which could attract more competition but also validate Viridian's market strategy.
• The balanced enrollment between US and international sites positions VRDN-001 for potential global market penetration, which could be important for long-term revenue growth.
• The company's dual approach with trials for both active and chronic TED could allow Viridian to capture a broader segment of the TED patient population, potentially differentiating it from competitors who may focus on only one stage of the disease.

From a competitive standpoint, Viridian's progress puts pressure on other players in the TED space to accelerate their development programs. The market will be watching closely to see how VRDN-001's efficacy and safety profile compare to existing treatments and other candidates in development. A successful outcome could position Viridian as a leading player in the TED market, while also potentially expanding the overall market size for TED treatments.

- THRIVE-2 exceeded its enrollment target due to patient demand; 188 patients enrolled with approximately 40% from US sites -

- THRIVE topline readout in patients with active TED on track for September 2024 -

- THRIVE-2 topline readout on track for year-end 2024 -

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 announced that enrollment is complete in THRIVE-2, its phase 3 clinical trial for VRDN-001 in patients with chronic TED.

THRIVE-2 enrolled 188 patients globally and exceeded the enrollment target of 159 patients due to patient demand. Approximately 40% of the enrolled patients were from US sites. Topline data from THRIVE-2 is on track for readout by year-end 2024. Previously, Viridian announced that THRIVE, its phase 3 clinical trial for VRDN-001 in patients with active TED, completed and exceeded enrollment in March 2024 with approximately 50% of patients enrolled from US sites. Topline data for THRIVE is on track for readout in September 2024.

“We are very pleased with our overall clinical trial enrollment for the VRDN-001 trials,” said Steve Mahoney, Viridian’s President and Chief Executive Officer. “THRIVE and THRIVE-2 have now each exceeded their enrollment targets and enrolled substantial patient numbers in the US due to patient demand. We look forward to our topline data readout for THRIVE in September and for THRIVE-2 at the end of this year. We are also on track to initiate two subcutaneous VRDN-003 phase 3 clinical trials, REVEAL-1 and REVEAL-2, in August as planned.”

About VRDN-001

Viridian’s lead product candidate, VRDN-001, is an intravenously (IV) delivered monoclonal antibody that acts as a full antagonist of the insulin-like growth factor-1 receptor (IGF-1R). IGF-1R is a clinically and commercially validated target for thyroid eye disease (TED) that had US revenues of approximately $1.8 billion in 2023. VRDN-001 has the potential to improve patient experience with a differentiated dosing regimen that features a shorter infusion time and fewer infusions compared to the currently approved and marketed IGF-1R inhibitor.

Viridian is evaluating VRDN-001 in two global phase 3 clinical trials, THRIVE and THRIVE-2, for the treatment of active and chronic TED, respectively. THRIVE and THRIVE-2 are each designed to compare a five-dose treatment arm of VRDN-001 to placebo, each dosed three weeks apart. In phase 2 clinical trials in active and chronic TED, VRDN-001 was shown to improve the signs and symptoms of TED at six weeks after two infusions in all dose cohorts and was generally well-tolerated.

Viridian believes that the differentiated VRDN-001 has the potential to establish a strong foothold in the multi-billion-dollar TED commercial market, if approved, and will help facilitate the introduction of VRDN-003, its potential best-in-class subcutaneous IGF-1R antibody for TED.

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 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. Viridian is also advancing VRDN-003 as a potential best-in-class subcutaneous therapy for the treatment of TED, including two planned global phase 3 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,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “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: clinical programs and clinical development of Viridian’s product candidates; anticipated start dates and designs of studies, including the REVEAL-1 and REVEAL-2 clinical trials; upcoming milestones and anticipated data readouts and results and timing of these readouts, including topline readouts and results; that VRDN-001 has the potential to improve patient experience with a differentiated dosing regimen that features a shorter infusion time and fewer infusions compared to the currently approved and marketed IGF-1R inhibitor; that that the differentiated VRDN-001 has the potential to establish a strong foothold in the multi-billion dollar TED commercial market, if approved, and will help facilitate the introduction of VRDN-003, its potential best-in-class subcutaneous IGF-1R antibody, for TED; anticipated dosing frequency; the potential utility, efficacy, potency, safety, clinical benefits, clinical response and convenience of VRDN-001, VRDN-003, VRDN-006 and VRDN-008; the potential for the portfolio of neonatal Fc receptor (FcRn) inhibitors to be developed in multiple autoimmune diseases; 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; the relationship between the results from the positive data from completed or ongoing clinical trials and 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; trial protocols for ongoing clinical trials; expectations regarding the timing for regulatory filings; regulatory interactions; expectations 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; Viridian’s intellectual property position; the timing of preclinical and clinical trial activities and reporting results from same; 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 May 8, 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/20240725241981/en/

Louisa Stone, 617-272-4604

Manager, Investor Relations

IR@viridiantherapeutics.com

Source: Viridian Therapeutics, Inc.

FAQ

What is the enrollment status of Viridian Therapeutics' THRIVE-2 clinical trial for VRDN-001?

Viridian Therapeutics has completed enrollment for the THRIVE-2 phase 3 clinical trial of VRDN-001 in chronic Thyroid Eye Disease patients, exceeding the target with 188 patients enrolled globally.

When is the expected topline data readout for Viridian's THRIVE-2 trial?

The topline data readout for the THRIVE-2 trial is expected by year-end 2024.

What is the status of Viridian's THRIVE trial for active Thyroid Eye Disease?

The THRIVE trial for active TED completed enrollment in March 2024, exceeding its target. Topline data is expected in September 2024.

What percentage of patients in THRIVE-2 were enrolled from US sites?

Approximately 40% of the patients enrolled in the THRIVE-2 trial were from US sites.

What are Viridian's plans for VRDN-003 clinical trials?

Viridian plans to initiate two subcutaneous VRDN-003 phase 3 clinical trials, REVEAL-1 and REVEAL-2, in August 2024.