X4 Pharmaceuticals Secures EMA Opinion for WHIM Syndrome, Extends $253M Cash Runway
X4 Pharmaceuticals posted a $79.2 million full-year net loss in 2025 and $23.9 million loss in Q4 on product sales of $2.3 million. EMA’s positive opinion for mavorixafor in WHIM syndrome and a $253M cash runway bolster Phase 3 4WARD trial, on track to finish enrollment in Q3 2026.
1. Q4 and Full-Year 2025 Financial Results
X4 Pharmaceuticals reported net product sales of $2.3 million in Q4 and $6.5 million for 2025, with license revenue of $28.6 million. Operating losses were $23.7 million in Q4 and $86.9 million for the year, resulting in net losses of $23.9 million (−$0.22/share) and $79.2 million (−$1.87/share). Cash, equivalents and short-term investments of $253.0 million as of December 31, 2025 provide runway through 2028.
2. EMA CHMP Positive Opinion for WHIM Syndrome
The EMA’s Committee for Medicinal Products for Human Use adopted a positive opinion recommending marketing authorization of mavorixafor for WHIM syndrome in the EU. The recommendation now awaits final approval from the European Commission, anticipated in Q2 2026, which would establish mavorixafor as the first and only WHIM therapy in Europe.
3. Phase 3 4WARD Trial Progress
Enrollment for the global 4WARD Phase 3 chronic neutropenia trial remains on track to complete by Q3 2026. X4 has expanded to over 100 active sites, enhanced Medical Affairs engagement, established patient referral pathways and invested in database mining to accelerate participant identification and enrollment.