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TranslateTop-line data from pivotal 4WHIM Phase 3 clinical trial in WHIM syndrome expected in 4Q22
Clinical and regulatory updates from ongoing chronic neutropenia Phase 1b trial anticipated during 2Q/3Q 2022
Additional Waldenström’s macroglobulinemia Phase 1b data expected in second half of 2022; supportive pre-clinical data to be presented at AACR 2022
Conference call today at 8:30 a.m. EDT
BOSTON, March 17, 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 fourth quarter and twelve months ended December 31, 2021, and provided a corporate update.
“We are very pleased with our many accomplishments in 2021, particularly completing enrollment in the pivotal 4WHIM Phase 3 clinical trial in WHIM syndrome, the first indication we are pursuing for our oral, first-in-class CXCR4 inhibitor, mavorixafor,” commented Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. “We also continued enrolling and treating patients in both our chronic neutropenia and Waldenström’s macroglobulinemia Phase 1b clinical trials, which are designed to expand the potential market opportunities for mavorixafor, and we look forward to reporting key clinical and regulatory updates for these programs throughout the year.”
Dr. Ragan continued, “Our clinical and research efforts into the potential of CXCR4 antagonism have also resulted in the discovery of several novel WHIM-causing CXCR4 mutations associated with expanded disease characteristics, which we believe further underscore WHIM syndrome as an underrecognized condition and strengthen our confidence in prevalence being potentially higher than the current estimates of WHIM patients in the U.S. We look ahead to the remainder of 2022 with great excitement, as we continue to anticipate top-line data from 4WHIM by the end of the year, and additional clinical and research data throughout the year, further supporting the broad clinical and commercial potential of mavorixafor across multiple indications.”
Fourth Quarter 2021 Highlights
Upcoming Presentations
Clinical Immunology Society (CIS)
X4 will present three posters at the CIS 2022 Annual Meeting: Immune Deficiency and Dysregulation North American Conference, March 31 - April 3, 2022:
American Association for Cancer Research (AACR)
X4 will present emerging preclinical data on mavorixafor’s ability to significantly enhance the tumor cell killing activity of the leading commercial and clinical Bruton Tyrosine Kinase Inhibitors (BTKi) including ibrutinib, zanubrutinib, pirtobrutinib (LOXO-305) and nemtabrutinib (ARQ-531), at the 2022 AACR Annual Meeting, taking place April 8 – 13, 2022.
Fourth Quarter and Full Year 2021 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 (866) 721-7655 from the United States or (409) 216-0009 internationally, followed by the conference ID: 9772687. 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. The company’s lead 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 trafficking of white blood cells, X4 believes that mavorixafor has the potential to provide therapeutic benefit across a wide variety of diseases, including primary immunodeficiencies (PIDs) and certain types of cancer. Mavorixafor has already demonstrated clinical potential in a Phase 2 trial in people with WHIM syndrome, a rare PID. Its efficacy and safety continue to be evaluated in a global Phase 3 clinical trial in WHIM (fully enrolled) and in two Phase 1b clinical trials – one, as monotherapy in people with chronic neutropenia, including Severe Congenital Neutropenia (SCN), and another in combination with ibrutinib in people with Waldenström’s macroglobulinemia. X4 is continuing to leverage its insights into CXCR4 biology at its corporate headquarters in Boston, Massachusetts and at its research facility in Vienna, Austria, to discover and develop additional product candidates. For more information, please visit 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 and X4’s other product candidates or programs; X4’s possible exploration of additional opportunities for mavorixafor; the anticipated achievement of upcoming clinical milestones; the expected availability, content, and timing of clinical trial data; anticipated updates on regulatory interactions and commercial plans; clinical trial design, 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, 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 or delayed patient enrollment, 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; 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 November 4, 2021, and in other filings X4 makes with the SEC from time to time.
(Tables Follow)
X4 PHARMACEUTICALS, INC. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS(in thousands, except per share amounts)(unaudited)
| Three Months Ended | Twelve Months Ended | ||||||||||||||
| December 31, | December 31, | ||||||||||||||
| 2021 | 2020 | 2021 | 2020 | ||||||||||||
| License revenue | $ | — | $ | — | $ | 3,000 | |||||||||
| Operating expenses: | |||||||||||||||
| Research and development | 12,162 | 12,298 | 50,647 | 41,932 | |||||||||||
| General and administrative | 7,135 | 5,357 | 24,702 | 20,942 | |||||||||||
| Goodwill impairment | 9,758 | — | 9,758 | — | |||||||||||
| Total operating expenses | 29,055 | 17,655 | 85,107 | 62,874 | |||||||||||
| Loss from operations | (29,055 | ) | (17,655 | ) | (85,107 | ) | (59,874 | ) | |||||||
| Other expense, net | (1,149 | ) | (745 | ) | (3,572 | ) | (2,109 | ) | |||||||
| Loss before provision for income taxes | (30,204 | ) | (18,400 | ) | (88,679 | ) | (61,983 | ) | |||||||
| Provision for income taxes | 3 | — | 17 | 148 | |||||||||||
| Net loss | (30,207 | ) | (18,400 | ) | (88,696 | ) | (62,131 | ) | |||||||
| Deemed dividend due to Class B warrant price reset | (5,704 | ) | — | (13,943 | ) | — | |||||||||
| Net loss attributable to common stockholders | $ | (35,911 | ) | $ | (18,400 | ) | $ | (102,639 | ) | $ | (62,131 | ) | |||
| Net loss per share attributable to common stockholders- basic and diluted | $ | (1.24 | ) | $ | (0.91 | ) | $ | (3.99 | ) | $ | (3.09 | ) | |||
| Weighted average common shares outstanding-basic and diluted | 29,011 | 20,174 | 25,749 | 20,077 | |||||||||||
X4 PHARMACEUTICALS, INC. CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS(in thousands)(unaudited)
| Twelve months ended December 31, | |||||||
| 2021 | 2020 | ||||||
| Net loss | $ | (88,696 | ) | $ | (62,131 | ) | |
| Adjustments to reconcile net loss to net cash used in operating activities | 19,289 | 7,376 | |||||
| Changes in operating assets and liabilities | (1,498 | ) | (4,063 | ) | |||
| Net cash used in operating activities | (70,905 | ) | (58,818 | ) | |||
| Net cash used in investing activities | (615 | ) | (1,362 | ) | |||
| Net cash provided by financing activities | 74,245 | 12,394 | |||||
| Impact of foreign exchange on cash, cash equivalents and restricted cash | (319 | ) | 402 | ||||
| Net increase (decrease) in cash, cash equivalents and restricted cash | 2,406 | (47,384 | ) | ||||
| Cash, cash equivalents and restricted cash at beginning of period | 80,702 | 128,086 | |||||
| Cash, cash equivalents and restricted cash at end of period | $ | 83,108 | $ | 80,702 | |||
X4 PHARMACEUTICALS, INC. CONSOLIDATED BALANCE SHEETS(in thousands)(unaudited)
| December 31, 2021 | December 31, 2020 | ||||
| Current assets: | |||||
| Cash and cash equivalents | $ | 81,787 | $ | 78,708 | |
| Research and development incentive receivable | 747 | 917 | |||
| Prepaid expenses and other current assets | 5,344 | 3,682 | |||
| Total current assets | 87,878 | 83,307 | |||
| Property and equipment, net | 1,514 | 1,237 | |||
| Goodwill | 17,351 | 27,109 | |||
| Right-of-use assets | 8,710 | 7,960 | |||
| Other assets | 1,723 | 3,258 | |||
| Total assets | $ | 117,176 | $ | 122,871 | |
| Current liabilities: | |||||
| Accounts payable | $ | 4,283 | $ | 3,144 | |
| Accrued expenses | 7,870 | 8,018 | |||
| Current portion of lease liability | 1,075 | 786 | |||
| Current portion of long-term debt | 795 | — | |||
| Total current liabilities | 14,023 | 11,948 | |||
| Long-term debt, including accretion, net of discount | 33,139 | 33,178 | |||
| Lease liabilities | 4,776 | 4,484 | |||
| Other liabilities | 826 | 462 | |||
| Total liabilities | 52,764 | 50,072 | |||
| Total stockholders' equity | 64,412 | 72,799 | |||
| Total liabilities and stockholders' equity | $ | 117,176 | $ | 122,871 | |
Contacts:Glenn Schulman, PharmD, MPH (Corporate) VP, Investor Relations & Corporate Communicationsglenn.schulman@x4pharma.com (203) 494-7411
Daniel Ferry (Investors)Managing Director, LifeSci Advisorsdaniel@lifesciadvisors.com (617) 430-7576
Mónica Rouco Molina (Investors)Senior Account Executive, LifeSci Communicationsmroucomolina@lifescicomms.com
