• Cash and Cash Equivalents: Ended 2024 with just under $103 million.
• Net XOLREMDI Revenues: $1.4 million for Q4 2024; $2.6 million for the full year 2024.
• R&D Expenditures: $21.7 million for Q4 2024; $81.6 million for the full year 2024.
• SG&A Expenses: $15.1 million for Q4 2024; $61.5 million for the full year 2024.
• Net Loss: $39.8 million for Q4 2024; $37.5 million for the full year 2024, reflecting a one-time sale of a priority review voucher for $105 million.
• Strategic Restructuring Savings: Expected to decrease spending by $30 million to $35 million annually.

• Warning! GuruFocus has detected 5 Warning Signs with XFOR.

Release Date: March 25, 2025

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

Positive Points

• X4 Pharmaceuticals Inc (NASDAQ:XFOR) achieved US approval and launched its first product, XOLREMDI, for WHIM syndrome in 2024.
• The company has established strong partnerships with Norgine and Taiba Rare to expand the global reach of mavorixafor.
• X4 Pharmaceuticals Inc (NASDAQ:XFOR) completed a successful Phase 2 trial for mavorixafor in chronic neutropenia, showing promising results.
• The company has implemented a strategic restructuring to reduce annual spending by $30-35 million, extending operational runway.
• X4 Pharmaceuticals Inc (NASDAQ:XFOR) ended 2024 with $103 million in cash and cash equivalents, providing financial stability into 2026.

Negative Points

• X4 Pharmaceuticals Inc (NASDAQ:XFOR) reported a net loss of $37.5 million for the full year 2024.
• The company faces regulatory uncertainties and a lengthy approval process for mavorixafor in Europe, with potential approval not expected until 2026.
• X4 Pharmaceuticals Inc (NASDAQ:XFOR) is still in the early stages of building demand and awareness for XOLREMDI, with no specific patient numbers disclosed.
• The company has not provided formal sales guidance for 2025, creating uncertainty about future revenue growth.
• X4 Pharmaceuticals Inc (NASDAQ:XFOR) faces challenges in the competitive landscape of rare diseases, requiring significant investment in education and awareness campaigns.

Q & A Highlights

Q: Can you expand on the regulatory conversation regarding the ANC threshold change from below 1,500 to below 1,000? Was this discussed before the trial initiation, and is there a risk of needing to upsize the trial? A: Dr. Paula Ragan, CEO, explained that the FDA is interested in ensuring the trial's success by focusing on moderate to severe patients. This alignment with the FDA does not impact the trial's pace, as the requirement for at least two infections per year already narrowed the patient funnel. Dr. Christophe Arbet-Engels, CMO, added that both the FDA and EMA support the approach, focusing on infection benefits rather than ANC as a non-validated biomarker.

Q: With the tightening of eligibility criteria around baseline ANC, do you expect this to slow down enrollment? A: Dr. Christophe Arbet-Engels stated that they do not expect a slowdown in enrollment as mild patients were not anticipated to represent a substantial number. The history of infections already narrowed the patient pool, and the screening process is progressing as expected.

Q: Can you provide an update on inventory levels and any early discounting trends? A: Adam Mostafa, CFO, noted that the balance sheet reflects inventory levels, which are expected to be lumpy over time based on demand. Mark Baldry, CCO, added that there is no discounting with payers at this time, maintaining a tight distribution channel through their specialty pharmacy.

Q: How many patients are currently on XOLREMDI, and was there a price increase for 2025? A: Mark Baldry stated they are not disclosing patient numbers as demand is still building. They did implement a 7% price increase at the start of 2025, which is typical for the new year.

Q: What are the commercialization goals for XOLREMDI in 2025, and how is the patient engagement strategy evolving? A: Mark Baldry explained that the focus is on building awareness and education around WHIM syndrome. They are leveraging physician experience through a peer-to-peer speaker program and pivoting to engage the patient community. The goal is to increase demand by activating both physicians and patients, with payers quickly reimbursing the product.

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