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Introduction & Market Context
X4 Pharmaceuticals (NASDAQ:XFOR) delivered a strong financial performance in Q1 2025, with significant revenue and earnings beats, despite facing ongoing stock price challenges. The company’s May 2025 investor presentation highlighted its dual focus on commercializing XOLREMDI for WHIM syndrome while advancing its pipeline for the larger chronic neutropenia market.
The rare disease-focused biotech reported Q1 earnings of $0.04 per share, substantially outperforming the forecasted loss of $0.12. Revenue reached $28.81 million, more than doubling analyst expectations of $12.03 million, primarily driven by the Norgine partnership. Despite these positive results, XFOR shares have declined 87% over the past year and are currently trading at $4.31, well below the 52-week high of $36.00.
As shown in the following strategic overview, X4 is pursuing a multi-pronged approach to maximize the potential of its lead compound mavorixafor:
Strategic Initiatives
X4’s primary near-term focus is maximizing the commercial potential of XOLREMDI (mavorixafor), which received FDA approval and launched in the U.S. in May 2024 for WHIM syndrome, a rare primary immunodeficiency. The company has also secured strategic partnerships with Norgine for Europe and Australia/New Zealand, and with Taiba Access Rare for the Middle East and North Africa region.
The company’s European expansion is progressing with the Marketing Authorization Application (MAA) for WHIM syndrome accepted by the European Medicines Agency in January 2025. Approval and launch in the EU market are expected in the first half of 2026.
X4 is addressing a significant unmet need in the treatment landscape, as illustrated in this overview of the chronic neutropenia market:
The presentation emphasized that there has been no innovation in chronic neutropenia treatment for over 30 years, with only one approved therapy - injectable Granulocyte Colony-Stimulating Factor (G-CSF), which was approved in 1995. G-CSF is associated with frequent treatment-related adverse events and long-term risks of myelodysplastic syndrome and leukemia, creating a clear opportunity for new treatment options.
Pipeline Development
X4’s pipeline strategy centers on expanding mavorixafor’s indications from the ultra-rare WHIM syndrome (estimated 1,000+ U.S. patients) to the much larger chronic neutropenia market (estimated 50,000 U.S. patients, with approximately 15,000 having high unmet needs).
The following pipeline overview illustrates the company’s development strategy and timelines:
The pivotal Phase 3 4WARD trial for chronic neutropenia is a key focus, with full enrollment expected in Q3 or Q4 2025 and top-line data anticipated in the second half of 2026. The trial design builds on positive results from earlier studies, including the successful 4WHIM Phase 3 trial that led to XOLREMDI’s approval.
As shown in the following trial design, the 4WARD study is well-positioned to deliver potentially transformative results:
The trial aims to enroll approximately 150 participants with congenital, autoimmune, or idiopathic chronic neutropenia who have an Absolute Neutrophil Count (ANC) less than 1,000 cells/µL and a history of at least two infections requiring intervention within the last 12 months. The study features a two-component primary endpoint measuring both positive ANC response and annualized infection rate.
X4’s confidence in the 4WARD trial is supported by promising Phase 2 results, which demonstrated that mavorixafor durably and meaningfully increased mean ANC levels in patients:
Financial Performance
X4 reported a strong financial position with $87.7 million in funds as of March 31, 2025, which is expected to support operations into the first half of 2026. This runway aligns with key upcoming milestones, including the anticipated completion of 4WARD enrollment and potential EU approval for WHIM syndrome.
The Q1 2025 earnings report revealed significant financial outperformance, with revenue of $28.81 million compared to the forecasted $12.03 million. This revenue surge was primarily attributed to the Norgine partnership. The company’s strategic restructuring aims to reduce annual spending by $30-35 million, helping to extend its cash runway.
Despite these positive financial developments, X4’s stock has experienced significant pressure, declining 8.72% on May 1, 2025, following the earnings announcement. This disconnect between financial performance and stock price may reflect broader market concerns about the biotech sector or investor skepticism about the commercial potential of rare disease treatments.
Market Opportunity (SO:FTCE11B) & Future Outlook
X4 has outlined a clear roadmap for growth, focusing on both near-term commercial execution with XOLREMDI and longer-term expansion into the chronic neutropenia market. The company’s timeline for key milestones and market opportunity is illustrated below:
The presentation highlighted significant market opportunities in both WHIM syndrome (>1,000 U.S. patients) and chronic neutropenia (>15,000 U.S. patients with high unmet needs). The company’s strategy leverages the substantial overlap between WHIM and chronic neutropenia treating physicians, with 67% of WHIM-treating hematologists also treating chronic neutropenia patients.
X4 is particularly optimistic about the chronic neutropenia opportunity, where mavorixafor could potentially be used either as monotherapy or in combination with G-CSF:
For patients already on G-CSF, the combination approach could enable meaningful dose reductions, potentially lessening pain, discomfort, and long-term risk of malignancies associated with chronic G-CSF use. This strategy addresses a key unmet need while building on X4’s existing commercial infrastructure and relationships with treating physicians.
Conclusion
X4 Pharmaceuticals’ Q1 2025 presentation portrays a company at an inflection point, with an approved product generating revenue while advancing a potentially larger market opportunity through its pipeline. The strong financial performance in Q1 provides validation for the company’s strategy, though the disconnect with stock performance suggests ongoing investor skepticism.
The next 12-18 months will be critical for X4, with several key catalysts including potential EU approval for XOLREMDI in WHIM syndrome, completion of 4WARD enrollment, and eventually top-line data from the 4WARD trial. Success in these areas could potentially reverse the stock’s downward trajectory and position the company for sustainable growth in the rare disease space.
Full presentation:
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