CRESCENT BIOPHARMA, INC. Q3 FY2022 earnings call

• Harout noted substantial progress in the pivotal Phase 3 trial of uproleselan in relapsed/refractory AML, and the FDA cleared an Interim Utility Analysis for the study. Enrollment of the pivotal trial was completed a year ago, and the population is similar to the Phase 1/2 study.
• Ed Rock discussed the Phase 3 trial's survival events, compared to historical AML trials, and mentioned investigator-sponsored trials with posters at the ASH Annual Meeting. The NCI trial in frontline newly diagnosed AML patients over 60 is ongoing.
• Brian Hahn reported cash and cash equivalents at $51.6M as of 9/30/2022, Q3 cash burn at $8.6M, R&D expenses decreased to $4.9M, and G&A expenses decreased to $3.8M due to lower professional fees.

No traditional product segment financial performance reported; focus is on uproleselan development

• Anticipate the Independent Data Monitoring Committee (IDMC) to meet by end of Q1 2023 to discuss trial continuation or un-blinding if compelling efficacy is observed. If positive, aim for rapid regulatory pathway with FDA.
• Current cash resources expected to fund operations to end of 2023, with resources prioritized for uproleselan development program, including regulatory and pre-commercial activities.

• Uncertainties surrounding clinical trial outcomes, including the timing and results of the Phase 3 trial and investigator-sponsored trials.
• Risks related to regulatory interactions and the ability to obtain necessary approvals.
• Uncertainties in market adoption of uproleselan even if approved, and risks associated with forward-looking statements involving assumptions and uncertainties.

Q: Regarding the potential IDMC meeting and determination in Q1 2023 and regulatory timeframes if data is positive?

A: Anticipate the IDMC to meet by end of Q1 2023. If compelling efficacy is seen, they may recommend un-blinding the trial, and the company's regulatory team is ready to rapidly move forward with the regulatory pathway.

Q: Potential opportunities outside the U.S. for uproleselan?

A: Engaging medical experts globally, planning registration in major markets outside the U.S., and exploring potential partnerships. The trial has significant enrollment outside the U.S. as well.

Q: Thoughts on investigator-sponsored trials (ISTs) and future indications?

A: The company is pursuing pivotal trials in relapsed/refractory AML and the NCI trial in frontline AML. ISTs supplement understanding of uproleselan in different AML subpopulations, with opportunities to explore adding uproleselan to other standard therapies due to its low toxicity profile and potential additive efficacy