Release Date: May 08, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- Viridian Therapeutics Inc (VRDN, Financial) successfully completed enrollment for the THRIVE Phase 3 trial in March, exceeding the target with 113 patients due to strong demand.
- The company anticipates filing a BLA for the O1 program in the second half of 2025, with ongoing preparations aligned with previous guidance.
- Viridian Therapeutics Inc (VRDN) reported a robust cash position with $613 million in cash, cash equivalents, and short-term investments, maintaining a cash runway into the second half of 2026.
- The company is developing a subcutaneous O3 program, which has the potential to be best-in-class with a positive Type C meeting with the FDA recently completed.
- Viridian Therapeutics Inc (VRDN) is also progressing with its FcRn portfolio, aiming to file an IND for O6 by the end of the year and planning to share O8 nonhuman primate data in the second half of the year.
Negative Points
- The current approved therapy for TED requires eight infusions every three weeks, posing a significant burden for patients, which VRDN aims to address but has not yet fully resolved.
- While the O1 and O3 programs show promise, they are still in clinical trials, and their success is not guaranteed until further data and regulatory approvals are obtained.
- The competitive landscape for TED treatments and FcRn inhibitors is evolving, with potential challenges from other companies developing similar or more advanced therapies.
- Details on the pivotal trial design for the O3 program and further specifics on regulatory submissions are pending, which could affect the company's timelines and market expectations.
- The need for extensive safety databases and the completion of additional studies like STRIVE to support BLA submissions could introduce delays or reveal issues not previously identified.
Q & A Highlights
Q: With respect to the THRIVE data coming in September, what does success look like on the efficacy side, and how might reduced drug exposure impact the rate of hearing impairment events seen in trials?
A: (Scott Myers - CEO, President) Success would look like achieving a safety and efficacy profile similar to existing therapies, with particular attention to maintaining a benign safety profile. The lower drug exposures could potentially improve hearing impairment outcomes due to lower peak drug concentrations.
Q: Can you elaborate on the inclusion of the active control in the STRIVE study?
A: (Scott Myers - CEO, President) The STRIVE study includes an active control arm to complete the safety database necessary for a BLA submission. This is a standard requirement, and the study is designed to be well-controlled, with a 3:1 randomization favoring the new dosage.
Q: Regarding the ongoing THRIVE study, how are you controlling for hearing impairment and loss, and what baseline measures are in place?
A: (Thomas Ciulla - Chief Medical Officer) Adverse events, including hearing changes, are recorded using standard methodologies. Audio Symmetry is monitored at baseline and pre-specified points during the study, consistent with current clinical practices and FDA guidance.
Q: For the subcutaneous O.3 program, what were the outcomes of the recent Type C meeting with the FDA, and how confident are you about moving directly into Phase 3 trials?
A: (Scott Myers - CEO, President) The Type C meeting was positive, and while the official minutes are pending, the company is reiterating its guidance to start pivotal trials mid-year. More details will be provided once the minutes are reviewed.
Q: Can you discuss the potential for filing for approval of O.1 in the US and Europe simultaneously, and what does the market opportunity look like in Europe?
A: (Scott Myers - CEO, President) The epidemiology in Europe is similar to the US, and the company is considering the best strategies for filing in various geographies. More details on the ex-US plans will be shared later.
Q: What factors are influencing the timeline for the BLA submission in the second half of 2025, particularly regarding the STRIVE study?
A: (Scott Myers - CEO, President) The timeline is primarily driven by the need to complete the THRIVE 2 study, including a follow-up period. The STRIVE study is designed to fit within this timeline and is not expected to delay the BLA submission.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
