X4 Pharmaceuticals Q2 2024 Financial Results and Corporate Updates

X4 Pharmaceuticals, a company dedicated to improving the lives of people with rare immune system diseases, announced its financial results for the second quarter ending June 30, 2024. Significant achievements and upcoming milestones highlighted the report.

Dr. Paula Ragan, President and CEO of X4 Pharmaceuticals, reflected on the company's decade-long journey in advancing mavorixafor (now branded as XOLREMDI™) to help those with limited treatment options. In April 2024, the U.S. Food and Drug Administration (FDA) approved XOLREMDI™ for treating WHIM syndrome, a rare primary immunodeficiency. The commercial launch in the U.S. is currently underway, focusing on treating patients aged 12 and older, with the goal of addressing the root cause of WHIM syndrome.

Progress has also been made in using mavorixafor to treat chronic neutropenia (CN). The company reported positive interim data from its Phase 2 trial, showing that mavorixafor can significantly and sustainably increase neutrophil counts in CN patients. Furthermore, a pivotal Phase 3 trial for mavorixafor in CN has been initiated globally.

Key milestones for XOLREMDI™ in WHIM syndrome include receiving FDA approval and executing the U.S. commercial launch. Efforts include engaging with targeted physicians and patient advocacy groups, running disease awareness campaigns, and implementing the X4Connect™ and nurse educator programs to support patients. Additionally, data from the Phase 3 4WHIM clinical trial were published in the journal Blood and presented at the Clinical Immunological Society's annual meeting.

X4 Pharmaceuticals plans to seek regulatory approval for mavorixafor in WHIM syndrome from the European Medicines Agency (EMA) by early 2025. The company is also exploring additional opportunities in other regions based on the FDA approval.

For chronic neutropenia, X4 presented positive interim Phase 2 data in June 2024, demonstrating that mavorixafor is generally well tolerated and effectively increases neutrophil counts. Full Phase 2 data is expected in November 2024. A global, pivotal Phase 3 clinical trial, named 4WARD (NCT06056297), has been initiated. The 52-week study aims to enroll 150 participants and will evaluate the efficacy, safety, and tolerability of mavorixafor in patients with various forms of chronic neutropenia experiencing recurrent or serious infections.

Financially, X4 recognized a gain of $105.0 million from selling a priority review voucher awarded by the FDA. This contributed to the company's reported net income of $90.8 million for the second quarter of 2024, compared to a net loss of $55.7 million in the same period in 2023. The company’s cash, cash equivalents, restricted cash, and short-term marketable securities totaled $169.5 million as of June 30, 2024. X4 believes these funds will support operations into late 2025, excluding potential future revenues from XOLREMDI.

For the second quarter of 2024, X4 reported net product revenue of $0.6 million and cost of revenue of $0.3 million linked to XOLREMDI sales. Research and development (R&D) expenses were $20.9 million, up from $15.6 million in the previous year. Selling, general, and administrative (SG&A) expenses increased to $13.3 million from $10.2 million in 2023.

X4 Pharmaceuticals will host a conference call and webcast to discuss these financial results and business highlights. The call can be accessed by dialing the provided numbers with conference ID: X4PHARMA, or via a live webcast on the company's website. A replay will be available post-call.

X4 Pharmaceuticals continues to develop and commercialize innovative therapies for rare immune system diseases, leveraging its expertise in CXCR4 and immune system biology. The company’s headquarters are in Boston, Massachusetts, with a research center of excellence in Vienna, Austria.