Earnings

X4 Cuts 4WARD Enrollment Target After FDA Alignment

The Tip Desk · November 11, 2026 · 3 min read

The company expects to finish the pivotal neutropenia study with 126 patients by year-end after the FDA said the smaller cohort keeps adequate power.

X4 Pharmaceuticals (XFOR), a biopharmaceutical company developing therapeutics for rare hematology diseases, said it expects to complete enrollment in the 4WARD Phase 3 trial of mavorixafor for primary chronic neutropenia by the end of 2026 after the U.S. Food and Drug Administration agreed that a smaller cohort preserves statistical power.

In August 2026 the company received written FDA responses to a Type C meeting request on a revised sample size and safety database for a potential supplemental new drug application. The agency said that reducing the trial from 176 to 126 participants maintains adequate overall power for the co-primary endpoints of reduction in annualized infection rate and positive absolute neutrophil count response. The FDA indicated the proposed safety database appears acceptable to support a potential sNDA, although its adequacy would remain a review issue.

The company had previously targeted up to 176 patients, with full enrollment expected by the end of the third quarter of 2026. It has transitioned management of the study to a Clinical Research Organization as part of an overhaul of its clinical operating infrastructure.

In October 2026, X4 announced the 4STEPS Phase 2b trial, designed to evaluate whether granulocyte colony-stimulating factor dosage can be safely reduced after starting mavorixafor while maintaining stable neutrophil counts. The study is designed to enroll approximately 25 patients, with the first participant expected in the first quarter of 2027 and top-line data expected in the first quarter of 2028.

For the three months ended September 30, 2026, total revenue was $2.9 million, down from $8.8 million in the immediately preceding quarter, as a $5.5 million Norgine regulatory-milestone license recognized in the second quarter did not recur. Net product sales were $2.8 million, which the company attributed to an increase in the number of patients prescribed XOLREMDI, its accelerated-approval therapy for WHIM syndrome. License and other revenue was $0.1 million. Gross-to-net adjustments were approximately 9% of gross revenues.

Net loss was $20.2 million, or $0.16 a share, versus $16.2 million, or $0.13 a share, in the prior quarter. Loss from operations was $20.0 million. Research and development expenses were $13.3 million, down from $15.1 million; direct mavorixafor costs fell to $8.7 million from $10.4 million. General and administrative expenses were $9.1 million. The company said the R&D decline reflected lower compensation after prior-year restructuring, while G&A reflected lower legal and IT costs, partially offset by higher stock-based compensation and sales and marketing costs related to PCN market assessment.

Cash, cash equivalents and restricted cash were $186.5 million at September 30, 2026, down from $195.8 million at June 30. Common shares outstanding were 100,903,502.

In October 2026 the company refinanced and retired the Hercules Loan Agreement, on which $75.0 million was outstanding, with a new K2 Loan Agreement providing up to $150.0 million of term-loan capacity maturing in October 2030. The first $80.0 million tranche funded on October 7, 2026, and was used to pay off approximately $78.7 million of Hercules obligations. The K2 facility requires interest-only payments through October 1, 2029, with principal and interest thereafter for 13 months, and the interest-only period is extended if certain operational milestones are met.

Based on cash, cash equivalents and marketable securities on hand as of November 4, 2026 and its current operating plan, the company said those resources will allow it to fund operations and debt obligations for at least the next 12 months. It said it expects to continue to incur operating losses as it advances mavorixafor through 4WARD and will need additional capital beyond that period.