Argenx SE (NASDAQ:ARGX) on Monday announced it would discontinue the Phase 3 UplighTED studies evaluating efgartigimod subcutaneous (SC) (efgartigimod alfa and hyaluronidase-qvfc) in adults with moderate to severe thyroid eye disease (TED).The decision is based on the recommendation from an Independent Data Monitoring Committee (IDMC) to stop the trials for futility following its review of data from a pre-specified interim analysis.Efgartigimod showed a favorable safety and tolerability profile, and no new ...

Summary of Viridian Therapeutics Conference Call Company Overview - **Company**: Viridian Therapeutics - **Industry**: Biopharmaceuticals focusing on autoimmune diseases - **Key Participants**: Steve Mahoney (CEO), Tony Castianos (Chief Commercial Officer), Shan Wu (Chief Business Officer) [1][2] Core Points and Arguments Autoimmune Portfolio - Viridian is advancing an autoimmune portfolio with a focus on thyroid eye disease (TED), a market valued at approximately $2 billion in the U.S. [2] - The company has submitted a Biologics License Application (BLA) for its lead product, which has received breakthrough therapy designation from the FDA [4][2]. Market Dynamics - There is currently one competitor in the TED market, Tepezza, marketed by Amgen, which has shown a return to growth with sales of $560 million in the last quarter [10][11]. - The TED market consists of about 500,000 patients in the U.S., with 200,000 having moderate to severe forms of the disease [10][7]. Product Differentiation - Viridian's product, veligrotug, is expected to have a more favorable dosing regimen compared to Tepezza, requiring five infusions over three months versus eight infusions over five months for Tepezza [15][14]. - The onset of action for veligrotug is anticipated to be quicker, with significant proptosis response observed after just one infusion [15][16]. Clinical Data and Expectations - Phase three data from the THRIVE program indicates strong efficacy, with a proptosis response rate expected to meet or exceed the efficacy bar set by Tepezza [30][29]. - The company plans to launch with both active and chronic data in its label, which is a strategic advantage over Tepezza [17][16]. FCRN Portfolio - Viridian is also developing programs in the FCRN space, with two lead programs (006 and 008) targeting large markets projected to exceed $10 billion by 2030 [31][32]. - The company is focused on low-cost optionality in its FCRN portfolio, with ongoing studies to validate the efficacy and safety of its candidates [33][35]. Financial Position - Viridian recently announced a royalty deal with DRI, which is seen as a validation of its business model and does not encumber its lead products [37][38]. - The company expects to reach break-even and profitability, regardless of the FDA's review outcome [38][39]. Additional Insights - The TED market is characterized as a new start market, allowing for rapid adoption of new therapies without the need to switch patients from existing treatments [11][12]. - The concentration of prescribers in the TED market (approximately 2,000 core prescribers) allows for targeted commercial strategies [12][10]. - The company has engaged with over 500 key opinion leaders (KOLs) in October alone, indicating proactive market preparation [20][19]. This summary encapsulates the key points discussed during the conference call, highlighting the strategic positioning of Viridian Therapeutics within the biopharmaceutical industry, particularly in the treatment of autoimmune diseases.

Core Insights - Viridian Therapeutics, Inc. (NASDAQ:VRDN) is a biotechnology company focused on developing treatments for rare diseases, with RBC Capital setting a price target of $45, indicating a potential increase of about 63.64% from its current price of $27.50, reflecting strong confidence in the company's future performance [1][5] Financial Position - Viridian is positioned for profitability in 2026 due to successful financing transactions that have eliminated the need for further share dilution, which is a positive sign for investors [2][5] - The current stock price of VRDN is $27.50, showing a 12.61% increase or $3.08, with a market capitalization of approximately $2.23 billion and a trading volume of 4.86 million shares today [4] Competitive Landscape - The competitive landscape is shifting as Roche's Enspryng (satralizumab) has shown disappointing phase 3 results in treating thyroid eye disease, which reduces competitive pressure on Viridian's veligrotug and VRDN-003, potentially enhancing its market position and increasing market share in this therapeutic area [3][5]

Core Insights - Viridian Therapeutics, Inc. (NASDAQ:VRDN) has been upgraded to "Outperform" by RBC Capital, with a price target increase from $41 to $45, while the stock is currently priced at $27.45 [1][6] - The company is expected to have a promising year in 2026, having successfully completed financing transactions that will allow it to reach profitability without further share dilution [2][6] - Disappointing phase 3 results of Roche's Enspryng in treating thyroid eye disease have reduced competitive pressure on Viridian's treatments, veligrotug and VRDN-003, enhancing its market position [3][6] Financial Performance - VRDN's stock has seen a 12.41% increase, with a change of $3.03, and has fluctuated between $24.93 and $28.60 on the day of reporting [4] - The company's market capitalization is approximately $2.23 billion, with a trading volume of 3,957,014 shares, indicating strong investor interest [5]

Core Insights - Viridian Therapeutics, Inc. reported strong business performance with the successful submission of its Biologics License Application (BLA) for veligrotug [1] - The company completed enrollment in both pivotal clinical trials, indicating progress in its drug development pipeline [1] Financial Results - The financial results for the third quarter ended September 30, 2025, were highlighted, showcasing the company's ongoing commitment to developing treatments for serious and rare diseases [1]

Core Points - Viridian Therapeutics has successfully submitted a Biologics License Application (BLA) for its investigational therapy, veligrotug, to the U.S. FDA for the treatment of thyroid eye disease (TED) [1][2] - The BLA submission is supported by data from two pivotal phase 3 clinical trials, THRIVE and THRIVE-2, which met all primary and secondary endpoints [2][5] - Veligrotug has received Breakthrough Therapy Designation, which may support eligibility for Priority Review by the FDA [1][3] Company Overview - Viridian Therapeutics is a biotechnology company focused on developing potentially best-in-class medicines for serious and rare diseases [1][6] - The company is advancing multiple candidates for TED, including veligrotug and VRDN-003, with ongoing global phase 3 clinical trials [7] - Viridian's expertise in antibody discovery and protein engineering enables the development of differentiated therapeutic candidates [6][8] Clinical Development - Veligrotug is an intravenously delivered anti-insulin-like growth factor-1 receptor (IGF-1R) antibody in phase 3 development for TED [4] - The pivotal trials THRIVE and THRIVE-2 demonstrated a rapid onset of clinical benefit and statistically significant effects on multiple diplopia endpoints [5][7] - The BLA submission aims for a potential commercial launch of veligrotug in mid-2026, contingent on FDA approval [3]

Financial Performance - Viridian Therapeutics reported quarterly losses of $(1.00) per share, beating the analyst consensus estimate of $(1.02) per share [1] - The company reported quarterly sales of $75.000 million, exceeding the analyst consensus estimate of $43.643 million [1] Product Development and Market Position - Veligrotug received Breakthrough Therapy Designation, indicating strong momentum as the company approaches its planned Biologics License Application (BLA) filing and expected commercial launch [2] - The company is making significant progress in commercial preparation and aims to be launch-ready on a Priority Review designation timeline [2] - A recent license agreement with Kissei to develop and commercialize veligrotug and VRDN-003 in Japan further validates the value of the company's programs and potential global opportunities [2] Analyst Ratings and Price Targets - Oppenheimer analyst Leland Gershell maintained an Outperform rating and raised the price target from $28 to $32 [8] - Goldman Sachs analyst Richard Law maintained a Buy rating and raised the price target from $27 to $30 [8] - RBC Capital analyst Lisa Walter maintained an Outperform rating but lowered the price target from $45 to $41 [8] - Wells Fargo analyst Derek Archila maintained an Equal-Weight rating and lowered the price target from $27 to $26 [8] Stock Performance - Following the earnings announcement, Viridian Therapeutics shares fell 4.1% to $16.50 [2]

Thyroid Eye Disease (TED) Portfolio - Veligrotug's BLA submission is planned for the second half of 2025, with a potential U S launch in the second half of 2026, and the EU MAA submission is planned for the first half of 2026[13] - Topline data for VRDN-003's REVEAL-1 and REVEAL-2 trials are expected in the first half of 2026, with a BLA submission planned by year-end 2026[13] - In the THRIVE trial for active TED, the proptosis responder rate at week 15 was 70% for patients receiving veligrotug compared to 5% on placebo (p < 0 0001)[56, 58] - In the THRIVE-2 trial for chronic TED, the proptosis responder rate at week 15 was 56% for patients receiving veligrotug compared to 8% on placebo (p < 0 0001)[79] - 70% of proptosis responders in the THRIVE trial maintained their response at Week 52[69] FcRn Inhibitor Portfolio - VRDN-006 proof-of-concept Phase 1 clinical trial healthy volunteer data is expected in Q3 2025[13] - VRDN-008 is on track for IND submission by year-end 2025[13] - A single dose of VRDN-008 demonstrated a longer half-life and a ~20% deeper and more sustained reduction of IgG compared to efgartigimod in NHPs[149] - The market size of MG and CIDP alone are projected to be over $11 2 billion by 2030[133, 134] Corporate/Financial - Viridian had $563 million in cash as of June 30, 2025, providing a runway into the second half of 2027[13]

Thyroid Eye Disease (TED) Portfolio - Veligrotug (Intravenous) - Positive THRIVE and THRIVE-2 topline data in active and chronic TED showed a robust clinical profile[12] - In the THRIVE trial, 70% of patients receiving veligrotug achieved a proptosis response at 15 weeks, compared to 5% on placebo[57] - In the THRIVE trial, 53% of patients receiving veligrotug achieved a proptosis response at 3 weeks, after just 1 infusion[59] - In the THRIVE-2 trial (chronic TED), 56% of patients achieved a proptosis response compared to 8% in the placebo group[78] - Viridian plans to submit the BLA for veligrotug in the second half of 2025 and the EU MAA in the first half of 2026[3, 12] Thyroid Eye Disease (TED) Portfolio - VRDN-003 (Subcutaneous) - REVEAL-1 and REVEAL-2 trials are enrolling and dosing patients, with topline data expected in the first half of 2026[12] - The BLA submission for VRDN-003 is anticipated by year-end 2026[3, 12] - Phase 1 HV Study showed VRDN-003 has an extended half-life of 40–50 days and increases IGF-1 levels ~4-fold[102, 104] FcRn Inhibitor Portfolio - VRDN-006 proof-of-concept Phase 1 clinical trial data is expected in Q3 2025[3, 12] - VRDN-008 is on track for IND submission by year-end 2025[3, 12] - A single dose of VRDN-008 demonstrated a longer half-life and deeper IgG reduction compared to efgartigimod in NHPs[148, 149] Financial - Viridian had $637 million in cash as of March 31, 2025, providing a runway into the second half of 2027[12]