X4 Pharmaceuticals, Inc Q1 FY2023 earnings call

Dr. Paula Ragan highlighted that X4 is advancing Mavorixafor towards commercialization for WHIM syndrome. The Phase 3 clinical trial for Mavorixafor in WHIM syndrome met primary and key secondary endpoints. There are upcoming presentations at the Clinical Immunology Society (CIS) and an investor event on May 16th. Adam Mostafa reviewed financials: as of March 31, 2023, X4 had $94.4 million in cash, cash equivalents, and restricted cash. R&D expenses were $22.1 million (vs $14.1 million in Q1 2022) with a $5 million accrual for an in-licensing milestone. SG&A expenses were $7.2 million (vs $7.7 million in Q1 2022). Net loss was $24 million (vs $22 million in Q1 2022).

X4 is on track to file a US new drug application for Mavorixafor in WHIM syndrome in the second half of 2023. They expect to announce clinical data and provide clarity on the scope and timing of the Phase 3 clinical program for chronic neutropenia in Q2/Q3 2023.

Forward-looking statements regarding regulatory and product development plans are subject to risks and uncertainties that may cause actual results to differ from forecasts. Risks can be found in X4's filings with the SEC, including the 10-K for 2022 and the Q1 2023 10-Q.

Q: Could you give more detailed color on increased R&D expense and update on partnerships?

A: Adam Mostafa said the increase in R&D is due to a $5-million accrual for an in-licensing/regulatory milestone. On partnerships, they continue to look at beneficial financing ways like geographic-rights partnerships and will update when material.

Q: Can you provide extra info on infection data at CIS and KOL presentations, and if data sets are overlapping?

A: Dr. Paula Ragan said they'll share data on infection burden (frequency, severity, duration). Data sets are primarily the same, with one oriented to clinical communities and the other to investor community.

Q: Insight on FDA discussions and plans after Phase 2 data release for chronic neutropenia?

A: Dr. Paula Ragan said they'll provide additional data in Q2/Q3 and have completed interactions with the agency for clarity on Phase 3 registration program, focusing on durable neutrophil count and infection correlation.

Q: Will KOLs at May 16th event talk about additional neutropenic conditions for Mavorixafor?

A: Dr. Paula Ragan said KOLs will bring in experiences treating chronic neutropenia patients, and there will be Q&A around future indications.

Q: Talk about setting at CIS and audience, and what to expect from CN poster presentation?

A: Dr. Paula Ragan mentioned CIS is for immunologists, and Mark Baldry added they have meetings with key customers and a boost to raise WHIM disease awareness. The CN poster is a higher-level study connecting neutropenia degrees with infection morbidity/mortality, not focusing on genetics or G-CSF management specifically.