X4 Pharmaceuticals, Inc. Q1 FY2025 earnings call

Management Statement and Operational Highlights

• Chronic Neutropenia (CN): Launched the 4WARD Phase III trial focusing on high unmet need CN patients. Completed data analyses supporting trial success. Received patent allowance for mavorixafor in treating specific neutropenias.
• WHIM Syndrome and XOLREMDI: Cumulative XOLREMDI sales at $3.5M. Engaging with medical professionals, new patients make up ~40% of treated population. WHIM patient ambassador program launched. MAA for mavorixafor in WHIM accepted by EU regulators with potential Q1 2026 approval. Partnerships for global commercialization established. Abstracts on WHIM presented at CIS meeting.
• Financials: Ended Q1 2025 with ~$90M in cash. R&D spent $18.5M, SG&A $15M. Net income due to license revenue and warrant gain. Reverse stock split effective. Strategic restructuring mostly complete, expecting annual spending reduction of $30-$35M.

Segment Performance

• XOLREMDI for WHIM syndrome: Cumulative sales since May 2024 launch total $3.5 million. First quarter 2025 net revenues were just under $1 million.
• Mavorixafor for chronic neutropenia (CN): Ongoing 4WARD trial with enrollment in over 20 countries and over 90% of target sites activated. Received notice of allowance on a patent for mavorixafor in treating severe chronic idiopathic and autoimmune neutropenia without CXCR4 genetic variant. The value proposition for CN in the US could be a $1 billion to $2 billion opportunity.

Guidance

• CN Trial: Anticipates full enrollment in Q3/Q4 2025, with top-line data in H2 2026.
• WHIM Commercialization: Potential EMA approval for mavorixafor in WHIM by Q1 2026.
• Financial: Sufficient funds to support operations into H1 2026.

Risks

• Regulatory: Delays or issues in regulatory approvals for mavorixafor in CN or WHIM.
• Trial Outcomes: Unfavorable results in 4WARD trial impact CN market opportunity.
• Market Adoption: Slow adoption of XOLREMDI in WHIM due to small patient population or payer challenges.

Question and Answer

• Q: Can you give us a sense if you have any visibility into the types of patients that are being enrolled in the CN trial? A: Very sick patients being enrolled, enriching for success on the infection endpoint; higher demand locally in the trial; market research shows higher case flows in chronic neutropenia.
• Q: The numbers for CN diagnosis, are they US or worldwide? A: Just US, based on ICD-10 analysis and US claims data.
• Q: Does the agreement with Norgine go by indication or mavorixafor? A: Covers WHIM and CN; license to across both indications, with WHIM commercial first followed by CN.
• Q: Any comments on patient persistency and compliance with XOLREMDI? A: Compliance and adherence rates higher than expected for a daily oral medication; new patient education website launched.
• Q: Assumptions around patient dropout in the 4WARD trial? **A: Targeting 150 patients; enrolled subjects have good profile; event rates confirm assumptions; on track for Q3/Q4 enrollment.