On August 4, 2022 X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel CXCR4-targeted small-molecule therapeutics to benefit people with diseases of the immune system, reported financial results for the second quarter ended June 30, 2022 (Press release, X4 Pharmaceuticals, AUG 4, 2022, View Source [SID1234617472]). In addition, the company highlighted important upcoming milestones related to its lead clinical candidate, mavorixafor, including the presentation of data from its ongoing Phase 1b clinical trial in chronic neutropenic disorders and from the global, pivotal Phase 3 clinical trial of mavorixafor in WHIM (Warts, Hypogammaglobulinemia, Infections, & Myelokathexis) syndrome. The company also provided an update on its clinical oncology program following its recent strategic announcement prioritizing its use of resources to advance mavorixafor solely in chronic neutropenic disorders.

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"With the completion of our recent financing and the sharpening of our strategic focus on chronic neutropenic disorders, we believe that we are well positioned to deliver significant future value to both patients and shareholders as we approach key milestones in the second half of this year," said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. "While the data from our cancer programs continue to show promise, we are now pivoting our efforts towards unlocking the full value of our oncology portfolio through potential strategic partnerships, enabling us to focus on making the largest possible impact on the treatment of chronic neutropenic disorders, including WHIM syndrome. In late September, we expect to deliver on our next milestone – data from our fully enrolled proof-of-concept study in chronic neutropenic disorders; we expect these results to inform the regulatory path forward for mavorixafor across a broad range of chronic neutropenic patient populations. Finally, and notably, the unveiling of data from our pivotal Phase 3 4WHIM trial is on track for the fourth quarter; these results are expected to support our first regulatory filing in the U.S. early in the second half of 2023 and to bring us one large step closer to improving the lives of patients with chronic neutropenic disorders."

Recent Highlights & Anticipated Upcoming Milestones

Completed a private investment in public equity (PIPE) financing, raising gross proceeds of approximately $56 million; the financing included participation from new and existing investors.
Entered into an amendment to the company’s loan and security agreement with Hercules Capital to extend the interest-only period of its loan facility by up to twelve months (into 2024), subject to achieving certain financial and business milestones.
Announced a strategic re-prioritization of resources towards advancing mavorixafor in chronic neutropenic disorder indications, including WHIM syndrome, while progressing oncology programs only upon completion of strategic partnership(s); the announced strategic update was also inclusive of cost-cutting initiatives estimated to support extension of X4’s cash runway into the third quarter of 2023.
Presented research data at the June 2022 meeting of the European Hematology Association (EHA) (Free EHA Whitepaper) further supporting mechanism and market potential of mavorixafor across of range of clinical indications.
Planned late September 2022 presentation of data from the fully enrolled Phase 1b trial evaluating mavorixafor for the treatment of chronic neutropenic disorders, including congenital, idiopathic, and cyclic neutropenia.
Planned fourth quarter 2022 presentation of results from the global, pivotal Phase 3 clinical trial of mavorixafor for the treatment of patients with WHIM syndrome; data anticipated to support a submission for U.S. regulatory approval early in the second half of 2023.
Positive Data Update from Phase 1b Trial in Waldenström’s Macroglobulinemia (WM)

The Phase 1b, open-label, multicenter, single-arm study is evaluating the safety and efficacy of mavorixafor in combination with the BTK inhibitor ibrutinib in adult patients (either treatment naïve or relapsed/refractory) with the rare B-cell lymphoma Waldenström’s macroglobulinemia (WM) and confirmed MYD88 and CXCR4 genetic mutations.
A total of 16 patients were enrolled in the study; all were dosed with oral, once-daily doses of ibrutinib (420 mg) and escalating doses (200 mg, 400 mg, 600 mg) of oral mavorixafor, also once daily.
As of June 2022, 10 of 12 evaluable patients (83%) achieved a major response (MR) to therapy, or a greater than 50% reduction in serum IgM from baseline:
In relapsed/refractory patients, 8 of 9 (89%) achieved a MR
In treatment-naïve patients, MR was seen in all patients escalated to >200 mg of mavorixafor
Adding mavorixafor to ibrutinib was associated with a higher MR rate at 9, 12, and 24 months compared to previously reported MR rates achieved with ibrutinib monotherapy.
In addition, patients achieved elevations in absolute neutrophil count (ANC), with no neutropenic events reported; patients also experienced fewer infections over time with chronic dosing.
No major safety signals had been identified in the trial as of the data cut-off; mavorixafor in combination with ibrutinib showed a safety profile similar to ibrutinib monotherapy (N=16, including 8 patients escalated to the 600 mg dose of mavorixafor).
In June, mavorixafor was granted Orphan Drug Designation by the U.S. Food & Drug Administration for the treatment of patients with WM, regardless of CXCR4 mutation status.
The Phase 1b clinical trial is expected to be completed in the fourth quarter of 2022; further clinical studies in WM will now be subject to completing a strategic partnership.
Second Quarter 2022 Financial Results

Cash, Cash Equivalents & Restricted Cash: X4 had $48.7 million in cash, cash equivalents, and restricted cash as of June 30, 2022. On June 30, 2022, the company announced a private equity financing (PIPE) of approximately $56 million before deducting offering costs. The financing closed on July 6, 2022, and proceeds are not reflected in the cash on hand as of June 30, 2022. Including funds raised in this offering, the company’s recently announced cost-cutting measures, and recent amendments to its loan and security agreement with Hercules, X4 believes that it has sufficient funds to support company operations into the third quarter of 2023.
Research and Development (R&D) Expenses were $13.8 million for the second quarter of 2022 as compared to $13.2 million for the comparable period in 2021. R&D expenses include $0.7 million and $0.8 million of certain non-cash expenses for the second quarter of 2022 and 2021, respectively.
Selling, General, and Administrative Expenses (SG&A) were $6.7 million for the second quarter of 2022 as compared to $5.8 million for the comparable period in 2021. SG&A expenses include $0.8 million and $1.0 million of certain non-cash expenses for the second quarter of 2022 and 2021, respectively.
Net Loss: X4 reported a net loss of $21.2 million for the second quarter of 2022, as compared to $19.6 million for the comparable period in 2021. Net losses include $1.5 million and $1.8 million of certain non-cash expenses for the second quarter of 2022 and 2021, respectively.
Conference Call and Webcast
X4 will host a conference call and webcast today at 8:30 am EDT to discuss financial results and business highlights. The conference call can be accessed by dialing (855) 327-6837 from the United States or (631) 891-4304 internationally, followed by the conference ID: 10019589. The live webcast can be accessed on the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com. Following the completion of the call, a webcast replay of the conference call will be available on the website.