Viridian Therapeutics(US:VRDN)2022-11-14 19:08Financial Data and Key Metrics Changes - As of September 30, 2022, cash, cash equivalents, and short-term investments were $431 million, compared to $197 million as of December 31, 2021, indicating a significant increase in liquidity [58] - The company believes that its current cash position will be sufficient to fund operations into the second half of 2025 [58] Business Line Data and Key Metrics Changes - The IV VRDN-001 program showed promising results, with a 75% proptosis responder rate compared to 56% for TEPEZZA, and a 4.0-point improvement in Clinical Activity Score (CAS) [22][35] - The 20 mg per kg cohort demonstrated a mean change in proptosis of 2.04 millimeters, which is higher than the mean changes reported for TEPEZZA [34] Market Data and Key Metrics Changes - The Thyroid Eye Disease (TED) market is currently valued at $2 billion in the U.S. and is expected to grow to over $4 billion globally [8] - The company aims to capture a significant share of this market with its differentiated product offerings [55] Company Strategy and Development Direction - The company is focused on delivering a complete portfolio of products for the treatment of TED, including both intravenous and subcutaneous options [7][55] - The Phase 3 program consists of two pivotal efficacy studies, THRIVE and THRIVE-2, expected to read out in mid and late 2024, respectively [45] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the efficacy of VRDN-001, highlighting its potential to offer a shorter treatment course and faster symptom relief compared to existing therapies [44] - The company is well-funded and positioned to advance its portfolio rapidly, with a strong foundation for future growth in the TED market [56] Other Important Information - The company reported that the 3 mg per kg cohorts for VRDN-001 are fully enrolled, with top-line data expected in early January 2023 [11] - VRDN-002 has shown a half-life of up to 43 days, significantly better than TEPEZZA, positioning it well for upcoming trials [15] Q&A Session Summary Question: Can you provide more color on the discrepancy in the percent of patients who achieve a CAS of zero or one between the 10 and 20 mg per kg cohorts? - Management explained that both cohorts had a higher percentage of patients achieving CAS of zero or one compared to TEPEZZA, with baseline characteristics influencing the observed differences [62] Question: Is there any scenario where you would consider adding a weekly dosing cohort for the subcutaneous TED proof-of-concept trial? - Management indicated confidence in the every-other-week dosing paradigm and is exploring the potential for even less frequent dosing based on upcoming data [74] Question: Can you clarify what the mean proptosis reduction was in the placebo patients with this updated data set? - The mean proptosis change in the placebo group was an improvement of 0.5 millimeters, consistent with TEPEZZA studies [80] Question: What can you say about the ongoing 3 mg/kg cohort at this point? Any color on safety or tolerability? - No serious adverse events have been reported in the 3 mg/kg cohort so far [84] Question: Could you offer any additional color on the hyperglycemia event in the 20 mg/kg cohort? - Management clarified that variability in glucose measures was consistent with the underlying diabetes condition of the patient and not drug-related [90] Question: Will you require a comparison to active drug in the Phase 3 study? - Management confirmed that the study design includes two active arms and one placebo arm, with no requirement for comparison to an active drug [92] Question: What gives you the confidence for selecting either VRDN-002 or VRDN-003 for Phase III development by early 2024? - Management stated that both candidates are expected to perform similarly in terms of efficacy, allowing for a robust choice based on pharmacokinetic data [100]