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Viridian Therapeutics(VRDN) - 2024 Q1 - Earnings Call Transcript
VRDNViridian Therapeutics(VRDN)2024-05-08 18:27
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Financial Data and Key Metrics - The company ended the quarter with 613millionincash,cashequivalents,andshort−terminvestments,maintainingacashrunwayintothesecondhalfof2026[11][41]BusinessLineDataandKeyMetrics−TheTHRIVEPhase3trialforVRDN−001inactivethyroideyedisease(TED)completedenrollmentinMarch2024with113patients,exceedingthetargetof90duetostrongpatientdemand[9][24]−ThecompanyanticipatesfilingaBiologicsLicenseApplication(BLA)forVRDN−001inthesecondhalfof2025[10]−ThesubcutaneousVRDN−003programcompletedapositiveTypeCmeetingwiththeFDAandisontracktoinitiatepivotalclinicaltrialsmid−year2024[10][35]−TheFcRnportfolioisprogressingasplanned,withanINDsubmissionforVRDN−006expectedbytheendof2024andnon−humanprimatedataforVRDN−008expectedinthesecondhalfof2024[11][39]MarketDataandKeyMetrics−TheU.S.marketformoderatetosevereTEDisestimatedat190,000patients,withthecurrentstandardofcaregeneratingapproximately613 million in cash, cash equivalents, and short-term investments, maintaining a cash runway into the second half of 2026 [11][41] Business Line Data and Key Metrics - The THRIVE Phase 3 trial for VRDN-001 in active thyroid eye disease (TED) completed enrollment in March 2024 with 113 patients, exceeding the target of 90 due to strong patient demand [9][24] - The company anticipates filing a Biologics License Application (BLA) for VRDN-001 in the second half of 2025 [10] - The subcutaneous VRDN-003 program completed a positive Type C meeting with the FDA and is on track to initiate pivotal clinical trials mid-year 2024 [10][35] - The FcRn portfolio is progressing as planned, with an IND submission for VRDN-006 expected by the end of 2024 and non-human primate data for VRDN-008 expected in the second half of 2024 [11][39] Market Data and Key Metrics - The U.S. market for moderate to severe TED is estimated at 190,000 patients, with the current standard of care generating approximately 1.8 billion in U.S. sales in 2023 [13][14] - The TED market is considered a "new start market," meaning patients can choose from available treatments during each flare-up without displacing chronic therapies [15][16] Company Strategy and Industry Competition - The company aims to develop differentiated therapies for unmet medical needs, focusing on both IV and subcutaneous delivery options for TED and FcRn inhibitors for autoimmune diseases [7][36] - VRDN-001 is positioned as a potential best-in-class IV therapy with fewer doses and shorter infusion times compared to the current standard of care [27][28] - VRDN-003 is designed to offer a best-in-class subcutaneous dosing profile with the potential for at-home self-administration, which could significantly expand the market [29][31] Management Commentary on Operating Environment and Future Outlook - Management highlighted strong execution across the pipeline, with key catalysts including THRIVE top-line results in September 2024 and THRIVE-2 top-line results at the end of 2024 [40][41] - The company remains well-capitalized and is focused on delivering on upcoming milestones [41] Other Important Information - The company is conducting the STRIVE study to complete the safety database for the BLA submission of VRDN-001, with a data cut expected once the requisite number of patients is reached [26][72] - VRDN-003 demonstrated an extended half-life of 40-50 days in healthy volunteers, supporting its potential as a best-in-class subcutaneous therapy [32][34] Q&A Session Summary Question: What defines success for the THRIVE trial? - Success for THRIVE would be a safety and efficacy profile similar to TEPEZZA, with potential improvements in hearing impairment due to lower drug exposure [44][45] Question: How is the company addressing safety concerns in THRIVE? - The company is using standard methodologies for adverse event reporting and audiometry to monitor hearing, consistent with FDA guidance and current clinical practice [51][58] Question: What are the plans for VRDN-003 pivotal trials? - The company had a positive Type C meeting with the FDA and is on track to initiate pivotal trials mid-2024, with more details to be provided after receiving meeting minutes [56][62] Question: How does the company view the competitive landscape in TED? - The company sees strong patient demand for IGF-1R therapies, as evidenced by the over-enrollment in THRIVE, and believes its subcutaneous VRDN-003 could be a game-changer in the market [79][90] Question: What is the timeline for FcRn data? - Non-human primate data for VRDN-008 is expected in the second half of 2024, with healthy volunteer data for VRDN-006 anticipated in the second half of 2025 [91][92] Question: How does the company plan to expand internationally? - The company is considering ex-U.S. markets, particularly Europe, where the epidemiology of TED is similar to the U.S., but specific plans will be shared at a later time [66]