X4 Pharmaceuticals, Inc. Q4 FY2024 earnings call

• 2024 was transformative with the U.S. approval and launch of XOLREMDI for WHIM syndrome, with sales topping $2.5 million covering 7.5 months since mid-May launch.
• Advanced disease awareness through in-person and digital campaigns, speaker program, and patient services.
• Submitted MAA to EMA for mavorixafor in WHIM, expected approval by Q1 2026. Entered partnerships with Norgine (EUR 28.5 million upfront, potential up to EUR 226 million in milestones) and taiba rare for global commercialization.
• Completed Phase 2 trial for mavorixafor in chronic neutropenia, initiated Phase 3 trial (4WARD), refined protocol to focus on moderate to severe neutropenic participants.
• Implemented strategic restructuring to reduce annual spending by $30-$35 million.

For the fourth quarter of 2024, net XOLREMDI revenues were $1.4 million, and for the full year 2024, net revenues were $2.6 million. R&D expenditures were $21.7 million in the fourth quarter and $81.6 million for the full year, including noncash expenses of $1.2 million and $4.3 million respectively. SG&A expenses were $15.1 million in the fourth quarter and $61.5 million for the full year, including noncash expenses of $1 million and $3.9 million respectively. The company ended 2024 with just under $103 million in cash and cash equivalents.

• Expect potential EMA approval for mavorixafor in WHIM by Q1 2026.
• 4WARD trial expected to be fully enrolled in Q3/Q4 2025, with top-line data in H2 2026.
• Sufficient funds to support company operations into H1 2026.

• Uncertainties in the EMA review process for mavorixafor approval potentially delaying timelines.
• Risks associated with clinical trial outcomes for chronic neutropenia that could impact approval.
• Macro environment challenges affecting financial performance and commercialization efforts.

Q: Was just wondering if you can maybe just kind of expand a little bit around the regulatory conversation that was had regarding taking the ANC threshold to below 1,500 to below 1,000...

A: Paula Ragan and Christophe Arbet-Engels discuss regulatory engagement with FDA and EMA, alignment on focusing on moderate to severe patients, and positive interactions with agencies.

Q: With the tightening up of the eligibility criteria around baseline ANC, would you expect that to potentially slow down the pace of enrollment a little bit?

A: Christophe Arbet-Engels and Paula Ragan state that mild patients were not a substantial portion, and enrollment timelines are not impacted.

Q: Can you tell us, give or take, how many patients are on drug right now? And did you have any price increase to start 2025 for XOLREMDI?

A: Mark Baldry says patient numbers not disclosed, but there was a 7% price increase.

Q: For the Phase 3 trial, how are you doing the screening of these patients? What percent of the patients that go through the screening are you getting on the drug versus those who are not?

A: Paula Ragan and Christophe Arbet-Engels mention site activation at 90% of target, screening ramp up, and no concerns on enrollment timelines.

Q: With your partners in the agreement, are there any sort of like clauses regarding timing or milestones for registration in order to really engage with those commercial partners?

A: Paula Ragan says next key milestones are around WHIM approval in Europe, and CN approval's market potential is recognized by partners.

Q: With the change in primary outcome for the 4WARD trial, do you see the change possibly affecting the label again if approved in chronic neutropenia?

A: Christophe Arbet-Engels states no impact on label at this time.