X4 Pharmaceuticals Announces Q1 2024 Financial Results and Corporate Updates

X4 Pharmaceuticals, based in Boston and listed on Nasdaq as XFOR, is committed to enhancing the lives of individuals with rare immune system diseases. The company has recently announced its financial results for the first quarter of 2024, ending March 31, and highlighted significant recent accomplishments and forthcoming objectives. The critical update is the U.S. approval and market launch of XOLREMDI™ (mavorixafor) for treating WHIM syndrome.

According to Paula Ragan, Ph.D., the President and CEO of X4 Pharmaceuticals, this approval marks a significant milestone. The company's specialized field force is now actively working to ensure that patients can access XOLREMDI as quickly as possible. Concurrently, X4 has received a Rare Pediatric Disease Priority Review Voucher, which they plan to monetize. Additionally, interim data from a Phase 2 clinical trial investigating mavorixafor for chronic neutropenic disorders will be shared in June 2024.

The FDA's approval of XOLREMDI in late April 2024 was based on positive results from the pivotal 4WHIM Phase 3 clinical trial. This trial assessed the drug's efficacy and safety in WHIM syndrome patients aged 12 and older, showing that XOLREMDI effectively increased circulating mature neutrophils and lymphocytes. Results from this trial were published in the journal Blood and presented at the Clinical Immunology Society's annual meeting.

In addition to planning a global, pivotal Phase 3 trial for chronic neutropenia, X4 aims to evaluate the safety, efficacy, and tolerability of mavorixafor, either as monotherapy or in combination with granulocyte colony-stimulating factor (G-CSF). The company also intends to seek approval from the European Medicines Agency (EMA) for using XOLREMDI in WHIM syndrome, targeting late 2024 or early 2025 for this application.

Financially, X4 Pharmaceuticals reported that as of March 31, 2024, it had $81.6 million in cash, cash equivalents, restricted cash, and short-term marketable securities. The company believes this is sufficient to fund operations into 2025, excluding potential gains from monetizing the Priority Review Voucher awarded alongside XOLREMDI’s U.S. approval.

The company’s expenditure on research and development (R&D) was $19.9 million for Q1 2024, compared to $22.1 million in the same period in 2023. R&D expenses included $0.8 million in non-cash expenses. Selling, General, and Administrative (SG&A) expenses rose sharply to $17.4 million in Q1 2024 from $7.2 million in Q1 2023, including $1.0 million in non-cash expenses. X4’s net loss for the quarter was $51.8 million, up from $24.0 million in the previous year, primarily due to a non-cash loss related to the Class C warrant liability.

X4’s first-quarter 2024 results reflect heightened costs linked to commercial and medical team expansions and preparations for the commercial launch. The company aims to continue its momentum through additional clinical trials and international regulatory submissions. The ongoing development of mavorixafor for other indications underlines X4 Pharmaceuticals' dedication to addressing unmet needs in rare immune system diseases.