FDA grants veligrotug breakthrough status for TED treatment

Published 07/05/2025, 21:38
FDA grants veligrotug breakthrough status for TED treatment

WALTHAM, Mass. – Viridian Therapeutics, Inc. (NASDAQ: VRDN), specializing in serious and rare diseases, has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for its drug candidate veligrotug, aimed at treating thyroid eye disease (TED). This status is granted to drugs that may provide significant improvements over existing therapies for life-threatening conditions. According to InvestingPro data, the company maintains a strong liquidity position with a current ratio of 19.5x and holds more cash than debt on its balance sheet, providing financial flexibility to advance its drug development programs.

The FDA’s decision is based on veligrotug’s performance in the THRIVE and THRIVE-2 phase 3 clinical trials, where it met all primary and secondary endpoints, showing a rapid onset of proptosis response and a statistically significant diplopia improvement in patients with active and chronic TED. THRIVE-2 is notably the first global phase 3 trial to achieve such results in chronic TED. Despite these positive developments, InvestingPro analysis shows the stock has experienced significant volatility, declining over 51% in the past six months, with analysts maintaining a strong buy consensus and projecting substantial upside potential.

Veligrotug, an anti-insulin-like growth factor-1 receptor (IGF-1R) antibody administered intravenously, is the only therapy to date that has demonstrated a clinically meaningful resolution of diplopia in a phase 3 clinical trial for chronic TED. The Breakthrough Therapy Designation supports the drug’s eligibility for Priority Review, potentially expediting its approval process.

Viridian’s President and CEO, Steve Mahoney, expressed confidence in veligrotug’s potential as a differentiated treatment. The company anticipates submitting a Biologics License Application (BLA) in the second half of 2025 and, if approved, launching veligrotug in the U.S. in 2026.

In addition to veligrotug, Viridian is advancing VRDN-003, a subcutaneous IGF-1R antibody for TED, through two ongoing global phase 3 pivotal trials, REVEAL-1 and REVEAL-2. The company also has a portfolio of neonatal Fc receptor (FcRn) inhibitors in development for various autoimmune diseases.

This news is based on a press release statement from Viridian Therapeutics, Inc. and reflects the company’s current expectations regarding the development and potential commercialization of its product candidates. With a market capitalization of approximately $1 billion and an EBITDA of -$336.5 million in the last twelve months, investors seeking deeper insights into Viridian’s financial health and growth prospects can access comprehensive analysis through InvestingPro’s detailed research reports, which provide expert analysis on over 1,400 US stocks, including VRDN.

In other recent news, Viridian Therapeutics reported a narrower-than-expected loss for the first quarter, with a loss of $0.87 per share compared to analyst estimates of $0.98. The company also surpassed revenue expectations, bringing in $70,000 against the anticipated $52,090. Viridian’s lead candidate, veligrotug, remains on track for a Biologics License Application submission in the second half of 2025, with a potential U.S. launch in 2026 if approved. Stifel analysts maintained their Buy rating on Viridian with a price target of $41, noting the company’s progress towards becoming a commercial-stage entity. They highlighted the potential competitive edge of veligrotug in the thyroid eye disease market, which could benefit from Amgen’s efforts to expand the market. Viridian is also advancing its next-generation candidate, VRDN-003, with Phase 3 trial results expected in the first half of 2026. Additionally, the company is making strides in its FcRn portfolio, with first-in-human data for VRDN-006 expected in the third quarter of 2025. Viridian ended the quarter with $636.6 million in cash and investments, which it anticipates will fund operations into the second half of 2027.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

Latest comments

Risk Disclosure: Trading in financial instruments and/or cryptocurrencies involves high risks including the risk of losing some, or all, of your investment amount, and may not be suitable for all investors. Prices of cryptocurrencies are extremely volatile and may be affected by external factors such as financial, regulatory or political events. Trading on margin increases the financial risks.
Before deciding to trade in financial instrument or cryptocurrencies you should be fully informed of the risks and costs associated with trading the financial markets, carefully consider your investment objectives, level of experience, and risk appetite, and seek professional advice where needed.
Fusion Media would like to remind you that the data contained in this website is not necessarily real-time nor accurate. The data and prices on the website are not necessarily provided by any market or exchange, but may be provided by market makers, and so prices may not be accurate and may differ from the actual price at any given market, meaning prices are indicative and not appropriate for trading purposes. Fusion Media and any provider of the data contained in this website will not accept liability for any loss or damage as a result of your trading, or your reliance on the information contained within this website.
It is prohibited to use, store, reproduce, display, modify, transmit or distribute the data contained in this website without the explicit prior written permission of Fusion Media and/or the data provider. All intellectual property rights are reserved by the providers and/or the exchange providing the data contained in this website.
Fusion Media may be compensated by the advertisers that appear on the website, based on your interaction with the advertisements or advertisers
© 2007-2025 - Fusion Media Limited. All Rights Reserved.