Release Date: March 24, 2025
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Q: What efficacy is required to pick one Annamycin dose at 45 patients rather than waiting until 90 if it doesn't happen at 45? A: Walter Klemp, CEO: If Annamycin performs as well in Part A as it did in the Phase 2 trial, and HiDAC performs as expected, we might hit the statistical significance required to accelerate approval. However, HiDAC might underperform due to being limited to one cycle. Paul Waymack, Senior Chief Medical Officer, added that it's a multivariable equation, and while early termination is possible, it's not highly likely.
Q: Towards the end of the trial, what was the thinking that went into cutting off about 10% of patients from Part D? A: Walter Klemp, CEO: The FDA suggested a different biostatistical scheme during protocol review, which allowed us to reduce the number of patients.
Q: What is the STS lung met efficacy bogey you need to hit to proceed to a pivotal trial? A: Walter Klemp, CEO: We've already hit that. The STS patients treated were more challenged than expected, yet we achieved OS numbers typical of first-line patients. This has attracted interest from sarcoma experts, and a pivotal trial is being considered.
Q: What are the overall costs of the trial? A: Jonathan Foster, CFO: The full patient load for the Phase 3 trial could cost $60-$70 million. Our cash burn for 2025 is $5 million per quarter, increasing to $7-$8 million in 2026 as we prepare for an NDA.
Q: What are your thoughts on moving Annamycin to frontline AML treatment? A: Paul Waymack, Senior Chief Medical Officer: Once we document efficacy and file our NDA, we plan to explore multiple areas, including first-line therapy. Annamycin's lack of cardiotoxicity and resistance issues makes it suitable for first-line therapy, especially for unfit patients.
Q: What was the rationale for choosing the 190 mg/m dose for the MIRACLE trial? A: Paul Waymack, Senior Chief Medical Officer: The 230 mg/m dose showed great results in the 106 study. The FDA's Project Optimus led us to include a lower 190 mg/m dose, which also showed efficacy. Both doses are expected to perform similarly.
Q: Regarding the 190 mg/m dose, do you need to be superior to the 17%-18% CR rate observed in other trials to apply for early approval? A: Paul Waymack, Senior Chief Medical Officer: If one dose is clearly superior, the comparison would be against placebo. For early approval after Part A, the P-value would need to be less than 0.01, considering the totality of data.
Q: Will you preserve the 190 mg/m dose in case of safety signals with the 230 mg/m dose? A: Walter Klemp, CEO: We have enough experience with Annamycin to be confident in its safety profile. However, if 230 mg/m becomes problematic, we could revert to 190 mg/m. By the end of Part A, we expect to have enough data to make an informed choice.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
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