Release Date: May 01, 2025

For the complete transcript of the earnings call, please refer to the full earnings call transcript.

Positive Points

• X4 Pharmaceuticals Inc (NASDAQ:XFOR) has made significant progress in the development of Maverick 4 for chronic neutropenia, with over 90% of global trial sites activated.
• The company has received a notice of allowance from the US Patent Office for Maverick 4, with the patent expected to expire in March 2041.
• X4 Pharmaceuticals Inc (NASDAQ:XFOR) reported cumulative sales of $3.5 million for Zoremby since its launch, indicating growing demand.
• The company has secured international partnerships to expand the reach of Maverick 4 in Europe, Australia, New Zealand, and the MENA region.
• X4 Pharmaceuticals Inc (NASDAQ:XFOR) ended the first quarter of 2025 with nearly $90 million in cash, providing financial stability into the first half of 2026.

Negative Points

• Sales for the first quarter were slightly lower than the previous quarter due to fluctuations in inventory resupply.
• The company is still in the early stages of commercialization for Zoremby, with some sales volatility expected.
• X4 Pharmaceuticals Inc (NASDAQ:XFOR) had significant R&D and SG&A expenses, totaling $18.5 million and $15 million respectively for the first quarter.
• The company had to implement a 1 for 30 reverse stock split to address NASDAQ listing rule deficiencies.
• There is a high unmet need in the chronic neutropenia market, with approximately 15,000 individuals in the US still experiencing health challenges despite available treatments.

Q & A Highlights

• Warning! GuruFocus has detected 4 Warning Signs with XFOR.

Q: Can you provide insights into the types of patients being enrolled in the Forward trial and how this aligns with the commercial market? A: (Chief Medical Officer, Dr. Christoph Arbut Engels) The study has specific criteria, and the patient profile is promising. Many principal investigators (PIs) are eager to include more patients, indicating a high demand. The patient profile aligns well with the commercial market, showing a clear interest in enrolling patients with chronic neutropenia.

Q: Are the 50,000 diagnosed chronic neutropenia (CN) patients and the 15,000 with high unmet needs figures specific to the US or worldwide? A: (CEO, Dr. Paula Reagan) These figures are specific to the US, based on ICD-10 code analysis and US claims data.

Q: What is the target population for the label of Maverick 4, and how is it defined? A: (Chief Medical Officer, Dr. Christoph Arbut Engels) Although the Phase 3 study includes moderate to severe patients, we aim for a broad label covering the entire CN population. We have supporting data from previous studies and anticipate discussions with the FDA to finalize the label.

Q: Does the agreement with Norjee for commercialization in Europe and Australia cover specific indications or Maverick 4 in general? A: (CFO, Adam Mustafa) The agreement covers both WIM syndrome and CN indications for Maverick 4, with WIM being the first to be commercially launched, followed by CN.

Q: Can you comment on patient persistency and compliance with Zoremdi for WIM syndrome? A: (Chief Commercial Officer, Mark Baldry) Compliance and adherence rates are higher than expected for a daily oral medication, reflecting the unmet need and understanding of the disease by patients and physicians. We are enhancing patient education efforts, including launching a new website to support WIM patients.

For the complete transcript of the earnings call, please refer to the full earnings call transcript.