X4 Pharmaceuticals, Inc Q3 FY2023 earnings call

• NDA for mavorixafor to treat WHIM syndrome accepted by FDA for Priority Review with PDUFA date of April 30, 2024, with potential U.S. launch in second quarter 2024 if approved.
• Advanced mavorixafor chronic neutropenia Phase 2 study with over 15 participants enrolled, and finalized design of global pivotal Phase 3 trial for chronic neutropenic disorders.
• Added Dr. Christophe Arbet-Engels as Chief Medical Officer. Strengthened Board of Directors with Keith Woods.
• Completed expanded loan facility with Hercules Capital. Cash and equivalents over $140M at end of third quarter to fund operations into 2025.
• Commercialization efforts for WHIM syndrome: Partnered with WHIM community, initiated programs to drive earlier diagnosis, built launch readiness including fit-for-purpose commercial organization.
• Phase 2 CN study progressing; finalized Phase 3 trial design for chronic neutropenia with input from FDA.

No specific financial performance data for product segments provided in the transcript.

• Anticipates potential 2024 launch of mavorixafor in U.S. if approved for WHIM syndrome.
• Plans to initiate global pivotal Phase 3 trial for chronic neutropenia in first half of 2024.
• Confident in cash and equivalents balance to fund operations into 2025.

Forward-looking statements are subject to risks and uncertainties, details in X4’s most recent filings with the SEC, including this quarter’s Form 10-Q.

Q: Eva Privitera asked about reasons for inadequate response in CN patients on GCSF.

A: Christophe Arbet-Engels discussed the heterogeneous nature of the population and genetic mutations in congenital patients.

Q: Stephen Willey asked about sites in Phase 2 and Phase 3.

A: Paula Ragan and Christophe Arbet-Engels discussed lessons learned from Phase 2 and plans for Phase 3 sites.

Q: Edward Tenthoff asked about WHIM OLE and ASH poster.

A: Paula Ragan discussed WHIM OLE data and ASH poster plans for six-month data on three patients in the CN study.

Q: Andy Fleszar asked about timing of CN 15-patient update.

A: Christophe Arbet-Engels mentioned comprehensive data on 15 patients to be presented in first half of next year.

Q: Kristen Kluska asked about Phase 3 CN study design.

A: Christophe Arbet-Engels and Paula Ragan discussed study design confidence and enrollment factors.

Q: Swayampakula Ramakanth asked about CN trial label and subpopulations.

A: Adam Mostafa and Paula Ragan discussed label expectations and trial power.

Q: David Bautz asked about WHIM diagnosis doctors.

A: Mark Baldry discussed engagement with key physicians and digital marketing efforts to reach the correct physician population.