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Highlights notable upcoming milestones including data from Phase 1b trial in chronic neutropenia, expected in late September, and results from global, pivotal Phase 3 trial in WHIM syndrome, expected in 4Q 2022
Announces additional positive data from Phase 1b trial in Waldenström’s Macroglobulinemia supportive of a potential partnership to advance clinical development
Conference call to be hosted today at 8:30 a.m. EDT
BOSTON, Aug. 04, 2022 (GLOBE NEWSWIRE) -- 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, today reported financial results for the second quarter ended June 30, 2022. 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.
“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
Positive Data Update from Phase 1b Trial in Waldenström’s Macroglobulinemia (WM)
Second Quarter 2022 Financial Results
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.
About X4 PharmaceuticalsX4 Pharmaceuticals is a late-stage clinical biopharmaceutical company leading the discovery and development of novel therapies for people with diseases of the immune system. Our lead clinical candidate is mavorixafor, a first-in-class, small molecule antagonist of chemokine receptor CXCR4 that is being developed as a once-daily oral therapy. Due to mavorixafor’s ability to antagonize CXCR4 and improve the healthy maturation and mobilization of white blood cells, we believe that mavorixafor has the potential to provide therapeutic benefit across a wide variety of diseases, including a range of chronic neutropenic disorders and certain types of cancer. The efficacy and safety of mavorixafor are being evaluated in a global Phase 3 clinical trial in patients with WHIM syndrome, a rare, inherited, primary immunodeficiency disease caused by genetic mutations in the CXCR4 receptor gene. We are also studying mavorixafor in two Phase 1b clinical trials – one as a monotherapy in patients with chronic neutropenic disorders including congenital, idiopathic, and cyclic neutropenia, and one in combination with ibrutinib in patients with Waldenström’s macroglobulinemia (WM), a rare B-cell lymphoma. Further clinical development of mavorixafor in WM will now be subject to completing a strategic partnership as we focus our resources on advancing mavorixafor for the benefit of patients with chronic neutropenic disorders. We continue to leverage our insights into CXCR4 biology at our corporate headquarters in Boston, Massachusetts and at our research facility in Vienna, Austria. For more information, please visit our website at www.x4pharma.com.
Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by the words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target,” or other similar terms or expressions that concern X4's expectations, strategy, plans, or intentions. Forward-looking statements include, without limitation, statements regarding the clinical development and therapeutic potential of mavorixafor; the anticipated achievement of upcoming clinical milestones; the expected availability, content, and timing of clinical trial data; anticipated regulatory milestones; and the company’s cash runway. Any forward-looking statements in this press release are based on management's current expectations and beliefs. Actual events or results may differ materially from those expressed or implied by any forward-looking statements contained herein, including, without limitation, on account of uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development; the risk that trials and studies may be delayed, including, but not limited to, as a result of the effects of the ongoing COVID-19 pandemic, and may not have satisfactory outcomes; the risk that the outcomes of preclinical studies or earlier clinical trials will not be predictive of later clinical trial results; the risk that initial or interim results from a clinical trial may not be predictive of the final results of the trial or the results of future trials; the potential adverse effects arising from the testing or use of mavorixafor or other product candidates; the risks related to X4’s ability to raise additional capital, and other risks and uncertainties, including those described in the section entitled “Risk Factors” in X4’s Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on May 12, 2022, and in other filings X4 makes with the SEC from time to time.
X4 PHARMACEUTICALS, INC. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS(in thousands, except per share amounts)(unaudited)
| Three Months Ended | Six Months Ended | ||||||||||||||
| June 30, | June 30, | ||||||||||||||
| 2022 | 2021 | 2022 | 2021 | ||||||||||||
| Operating expenses: | |||||||||||||||
| Research and development | $ | 13,821 | $ | 13,193 | 27,934 | 25,297 | |||||||||
| Selling, general and administrative | 6,749 | 5,804 | 14,413 | 11,636 | |||||||||||
| Gain on sale of non-financial asset | — | — | (509 | ) | — | ||||||||||
| Total operating expenses | 20,570 | 18,997 | 41,838 | 36,933 | |||||||||||
| Loss from operations | (20,570 | ) | (18,997 | ) | (41,838 | ) | (36,933 | ) | |||||||
| Other expense, net | (638 | ) | (635 | ) | (1,312 | ) | (1,369 | ) | |||||||
| Loss before provision for income taxes | (21,208 | ) | (19,632 | ) | (43,150 | ) | (38,302 | ) | |||||||
| Provision for income taxes | 4 | 6 | 27 | 12 | |||||||||||
| Net loss | (21,212 | ) | (19,638 | ) | (43,177 | ) | (38,314 | ) | |||||||
| Deemed dividend due to Class B warrant price reset | — | — | (2,259 | ) | (8,239 | ) | |||||||||
| Net loss attributable to common stockholders | $ | (21,212 | ) | $ | (19,638 | ) | $ | (45,436 | ) | $ | (46,553 | ) | |||
| Net loss per share attributable to common stockholders- basic and diluted | $ | (0.60 | ) | $ | (0.74 | ) | $ | (1.31 | ) | $ | (1.97 | ) | |||
| Weighted average common shares outstanding-basic and diluted | 35,437 | 26,527 | 34,592 | 23,655 | |||||||||||
X4 PHARMACEUTICALS, INC. CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS(in thousands)(unaudited)
| Six months ended June 30, | |||||||
| 2022 | 2021 | ||||||
| Net loss | $ | (43,177 | ) | $ | (38,314 | ) | |
| Adjustments to reconcile net loss to net cash used in operating activities | 4,083 | 4,345 | |||||
| Changes in operating assets and liabilities | 391 | (3,672 | ) | ||||
| Net cash used in operating activities | (38,703 | ) | (37,641 | ) | |||
| Net cash used in investing activities | (60 | ) | (582 | ) | |||
| Net cash provided by financing activities | 4,609 | 54,117 | |||||
| Impact of foreign exchange on cash, cash equivalents and restricted cash | (271 | ) | (103 | ) | |||
| Net (decrease) increase in cash, cash equivalents and restricted cash | (34,425 | ) | 15,791 | ||||
| Cash, cash equivalents and restricted cash at beginning of period | 83,108 | 80,702 | |||||
| Cash, cash equivalents and restricted cash at end of period | $ | 48,683 | $ | 96,493 | |||
X4 PHARMACEUTICALS, INC. CONSOLIDATED BALANCE SHEETS(in thousands)(unaudited)
| June 30, 2022 | December 31, 2021 | ||||
| Current assets: | |||||
| Cash and cash equivalents | $ | 47,378 | $ | 81,787 | |
| Research and development incentive receivable | 843 | 747 | |||
| Prepaid expenses and other current assets | 4,364 | 5,344 | |||
| Total current assets | 52,585 | 87,878 | |||
| Property and equipment, net | 1,309 | 1,514 | |||
| Goodwill | 17,351 | 17,351 | |||
| Right-of-use assets | 7,976 | 8,710 | |||
| Other assets | 1,798 | 1,723 | |||
| Total assets | $ | 81,019 | $ | 117,176 | |
| Current liabilities: | |||||
| Accounts payable | $ | 3,677 | $ | 4,283 | |
| Accrued expenses | 8,582 | 7,870 | |||
| Current portion of lease liability | 1,136 | 1,075 | |||
| Current portion of long-term debt | 8,607 | 795 | |||
| Total current liabilities | 22,002 | 14,023 | |||
| Long-term debt, including accretion, net of discount | 24,490 | 33,139 | |||
| Lease liabilities | 4,140 | 4,776 | |||
| Other liabilities | 314 | 826 | |||
| Total liabilities | 50,946 | 52,764 | |||
| Total stockholders' equity | 30,073 | 64,412 | |||
| Total liabilities and stockholders' equity | $ | 81,019 | $ | 117,176 | |
Contacts:Daniel Ferry (investors)Managing Director, LifeSci Advisorsdaniel@lifesciadvisors.com (617) 430-7576
Mónica Rouco Molina, Ph.D. (media)Account Supervisor, LifeSci Communicationsmroucomolina@lifescicomms.com
