2021 expected to be a year of key value-driving catalysts as company advances multiple clinical programs with lead candidate mavorixafor
Company anticipates completing full enrollment in WHIM Phase 3 trial in 2021 and presenting clinical data from the ongoing Phase 1b Waldenström’s trial throughout the year
Conference call today at 8:30 a.m. ET
BOSTON, March 04, 2021 (GLOBE NEWSWIRE) — X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel therapies targeting diseases resulting from dysfunction of the CXCR4 pathway, today reported financial results for the fourth quarter and full year ended December 31, 2020. The company also provided an update on its lead product candidate, mavorixafor, a novel small molecule currently being evaluated in a Phase 3 clinical trial (4WHIM) for patients with WHIM (warts, hypogammaglobulinemia, infections, and myelokathexis) syndrome and in two Phase 1b trials for patients with Waldenström’s macroglobulinemia (Waldenström’s) and Severe Congenital Neutropenia (SCN), respectively.
“As many companies did, we encountered a number of unexpected challenges in 2020, most notably the COVID-19 pandemic. However, I could not be prouder of our accomplishments, as we remained steadfast in our commitment to advance our lead candidate, mavorixafor, in a number of rare disease indications,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. “We believe that we have much to look forward to this year, as we have overcome these obstacles, have made significant progress in advancing our clinical and research programs, and assembled an exceptional team to execute on our 2021 goals and beyond.
“On the clinical front, we continue to expect top-line data from our Phase 3 WHIM trial in 2022 and, as our Phase 1b Waldenström’s trial is progressing well, we continue to expect initial data from that study in the first half of 2021, with a more fulsome dataset expected towards the end of 2021. We also anticipate initial data from our SCN Phase 1b trial in 2021.”
Recent Highlights
Fourth Quarter 2020 Financial Results
Conference Call and Webcast
The Company will host a conference call and webcast today at 8:30 a.m. ET to discuss these 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: 1053189. 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 Pharmaceuticals
X4 Pharmaceuticals is a late-stage clinical biopharmaceutical company and a leader in the discovery and development of novel therapies for the treatment of diseases resulting from dysfunction of the CXCR4 pathway, with a focus on rare diseases and those with limited treatment options. The company’s lead candidate, mavorixafor, is a first-in-class, small molecule antagonist of chemokine receptor CXCR4 being developed as a once-daily oral therapy. X4 believes that inhibition of the CXCR4 receptor creates the potential for mavorixafor to provide therapeutic benefit across a wide variety of diseases, including primary immunodeficiencies and certain types of cancer. The efficacy and safety of mavorixafor, dosed once daily, is currently being evaluated in a global Phase 3 clinical trial in patients with WHIM syndrome, and in two Phase 1b clinical trials – in combination with ibrutinib in patients with Waldenström’s macroglobulinemia, and as monotherapy in patients with Severe Congenital Neutropenia. 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, and is discovering and developing additional product candidates. For more information, please visit www.x4pharma.com.
Forward-Looking Statements
This 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 of mavorixafor and X4’s other product candidates or programs, the potential benefits resulting from Fast Track and Rare Pediatric Disease designations, 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, the risks and uncertainties 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 5, 2020, and in other filings X4 makes with the SEC from time to time. X4 undertakes no obligation to update the information contained in this press release to reflect new events or circumstances, except as required by law.
(Tables Follow)
X4 PHARMACEUTICALS, INC.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
(in thousands, except per share amounts)
(unaudited)
Three Months Ended | Year Ended | ||||||||||||||
December 31, | December 31, | ||||||||||||||
2020 | 2019 | 2020 | 2019 | ||||||||||||
License revenue | $ | — | $ | — | $ | 3,000 | $ | — | |||||||
Operating expenses: | |||||||||||||||
Research and development | 12,298 | 7,065 | 41,932 | 30,163 | |||||||||||
General and administrative | 5,357 | 3,914 | 20,942 | 17,640 | |||||||||||
Loss on transfer of non-financial assets | — | (104 | ) | — | 3,900 | ||||||||||
Total operating expenses | 17,655 | 10,875 | 62,874 | 51,703 | |||||||||||
Loss from operations | (17,655 | ) | (10,875 | ) | (59,874 | ) | (51,703 | ) | |||||||
Other income (expense), net | (745 | ) | 38 | (2,109 | ) | (1,104 | ) | ||||||||
Loss before provision for income taxes | (18,400 | ) | (10,837 | ) | (61,983 | ) | (52,807 | ) | |||||||
Provision for income taxes | — | — | 148 | — | |||||||||||
Net loss | (18,400 | ) | (10,837 | ) | (62,131 | ) | (52,807 | ) | |||||||
Adjustments related to convertible preferred stock | — | — | — | (592 | ) | ||||||||||
Net loss attributable to common stockholders | $ | (18,400 | ) | $ | (10,837 | ) | $ | (62,131 | ) | $ | (53,399 | ) | |||
Net loss per share attributable to common stockholders- basic and diluted | $ | (0.91 | ) | $ | (0.66 | ) | $ | (3.09 | ) | $ | (4.63 | ) | |||
Weighted average common shares outstanding-basic and diluted | 20,174 | 16,466 | 20,077 | 11,530 |
X4 PHARMACEUTICALS, INC.
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS
(in thousands)
(unaudited)
Year ended December 31, | |||||||
2020 | 2019 | ||||||
Net loss | $ | (62,131 | ) | $ | (52,807 | ) | |
Adjustments to reconcile net loss to net cash used in operating activities | 7,376 | 7,988 | |||||
Changes in operating assets and liabilities | (4,063 | ) | (3,236 | ) | |||
Net cash used in operating activities | (58,818 | ) | (48,055 | ) | |||
Net cash (used in) provided by investing activities | (1,362 | ) | 27,232 | ||||
Net cash provided by financing activities | 12,394 | 140,661 | |||||
Impact of foreign exchange on cash, cash equivalents and restricted cash | 402 | (250 | ) | ||||
Net (decrease) increase in cash, cash equivalents and restricted cash | (47,384 | ) | 119,588 | ||||
Cash, cash equivalents and restricted cash at beginning of period | 128,086 | 8,498 | |||||
Cash, cash equivalents and restricted cash at end of period | $ | 80,702 | $ | 128,086 |
X4 PHARMACEUTICALS, INC.
CONSOLIDATED BALANCE SHEETS
(in thousands)
(unaudited)
December 31, 2020 | December 31, 2019 | ||||||
Current assets: | |||||||
Cash and cash equivalents | $ | 78,708 | $ | 126,184 | |||
Research and development incentive receivable | 917 | 1,998 | |||||
Prepaid expenses and other current assets | 3,682 | 1,096 | |||||
Total current assets | 83,307 | 129,278 | |||||
Property and equipment, net | 1,237 | 403 | |||||
Goodwill | 27,109 | 27,109 | |||||
Right-of-use assets | 7,960 | 1,959 | |||||
Other assets | 3,258 | 1,949 | |||||
Total assets | $ | 122,871 | $ | 160,698 | |||
Current liabilities: | |||||||
Accounts payable | $ | 3,144 | $ | 2,088 | |||
Accrued expenses | 8,018 | 6,461 | |||||
Current portion of lease liability | 786 | 898 | |||||
Total current liabilities | 11,948 | 9,447 | |||||
Long-term debt, including accretion, net of discount | 33,178 | 20,097 | |||||
Lease liabilities | 4,484 | 1,918 | |||||
Other liabilities | 462 | 16 | |||||
Total liabilities | 50,072 | 31,478 | |||||
Total stockholders’ equity | 72,799 | 129,220 | |||||
Total liabilities and stockholders’ equity | $ | 122,871 | $ | 160,698 |
Contacts:
Daniel Ferry (Investors)
Managing Director
LifeSci Advisors
daniel@lifesciadvisors.com
(617) 430-7576
Mónica Rouco Molina (Media)
Senior Account Executive
LifeSci Communications
mroucomolina@lifescicomms.com
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