X4 Pharmaceuticals Reports Fourth Quarter and Full Year 2021 Financial Results and Provides Corporate Update
Top-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
“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
Fourth Quarter 2021 Highlights
- Completed enrollment in the ongoing pivotal 4WHIM Phase 3 clinical trial of mavorixafor in patients with WHIM (Warts, Hypogammaglobulinemia, Infections, Myelokathexis) syndrome. Thirty-one adult and pediatric patients have been enrolled in the 4WHIM trial, which was originally designed to enroll 18 - 28 patients. Top-line data from the 4WHIM trial are expected in the fourth quarter of 2022.
- Presented a wide range of clinical and research data at the 2021
American Society of Hematology(ASH) Annual Meeting supporting the potential use of mavorixafor across a number of immunodeficiencies and certain cancers:
- Ongoing studies across a wide variety of diseases, including Waldenström’s macroglobulinemia (WM), clear cell renal cell carcinoma, WHIM syndrome, and chronic idiopathic neutropenia, showed that oral administration of mavorixafor increased blood neutrophils, lymphocytes, and monocytes regardless of the presence or absence of CXCR4 mutations.
- Mavorixafor efficacy has been clinically observed with short-term and long-term treatment both alone and in combination with other therapies, including axitinib, ibrutinib, and granulocyte-colony stimulating factor (G-CSF).
- Additional data from the Phase 2 open-label extension study of mavorixafor in WHIM patients continued to show durable increases in neutrophils, lymphocytes, and monocytes; decreased frequency, severity, and duration of infections; fewer hospital/doctor visits; and sustained improvements in warts. Chronic daily administration of mavorixafor continued to be well tolerated (median treatment duration = 148.4 weeks).
- Clinical and laboratory research resulted in the identification of a novel missense CXCR4 mutation, D84H, that further supports the company’s WHIM prevalence estimate of at least 1,000 to 3,500 patients in the
- Initial results from the ongoing mavorixafor Phase 1b clinical trial in people with chronic neutropenia concurrently treated with granulocyte colony stimulating factor (g-CSF) and mavorixafor demonstrated elevations in white blood cells and absolute neutrophil, lymphocyte, and monocyte counts (n=4); enrollment continues, with additional data expected in the second or third quarter of 2022.
December 2021, interim results reported from the ongoing Phase 1b clinical trial in WM from the low- (200 mg) and mid-level (400 mg) dose groups ( October 2021data cut) showed a 100% overall response rate (n=10, median treatment duration of 272.5 days), sustained decreases in serum IgM (n=14), and trends towards normalization of hemoglobin levels (n=14 at baseline, n=3 at 12 months).
- As of
March 2022, cohort B evaluating 600 mg of mavorixafor in combination with ibrutinib met the safety requirements to allow for the dose escalation of patients enrolled in cohort A and previously treated at low- and mid-level doses. All eligible patients are being escalated to receive 600 mg of mavorixafor once daily in combination with ibrutinib. Additional data from this trial are expected in the second half of 2022.
- As of
X4 will present three posters at the CIS 2022 Annual Meeting:
- PATH4WARD: A Genetic Testing Program to Aid in Molecular Diagnosis of Congenital Neutropenia and Other Primary Immunodeficiencies Including WHIM Syndrome.
- Characterization of CXCR4(S341Y) Variant of Uncertain Significance in the Setting of Infections, Hypogammaglobulinemia, and Warts.
- 4WHIM: Evaluating the Oral CXCR4 Antagonist Mavorixafor in Patients With WHIM Syndrome via a Global Phase 3, Randomized, Placebo-Controlled Trial With Open-label Extension.
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
- e-Poster #6093: Mavorixafor Enhances Efficacy of Bruton Tyrosine Kinase Inhibitors by Overcoming the Protective Effect of Bone Marrow Stroma on Tumor Cells in Waldenström’s Macroglobulinemia.
Session: OPO.TB06.01 - Tumor Microenvironment
E-posters are expected to be released at
12:00 PM CTon Friday, April 8.
Fourth Quarter and Full Year 2021 Financial Results
- Cash, Cash Equivalents & Restricted Cash: X4 had
$83.1 millionin cash, cash equivalents, and restricted cash as of December 31, 2021. The company expects that its cash and cash equivalents will fund company operations into the fourth quarter of 2022.
- Research and Development (R&D) Expenses were
$12.2 millionand $50.6 millionfor the fourth quarter and full year ended December 31, 2021, as compared to $12.3 millionand $41.9 millionfor the comparable periods in 2020. R&D expenses include $0.7 millionand $2.7 millionof certain non-cash expenses for the quarter and full year ended December 31, 2021, respectively.
- General and Administrative Expenses (G&A) were
$7.1 millionand $24.7 millionfor the fourth quarter and full year ended December 31, 2021, as compared to $5.4 millionand $20.9 millionfor the comparable periods in 2020. G&A expenses include $0.9 millionand $3.5 millionof certain non-cash expenses for the quarter and full year ended December 31, 2021, respectively.
- Net Loss: X4 reported a net loss of
$30.2 millionand $88.7 millionfor the quarter and full year ended December 31, 2021, as compared to a net loss of $18.4 millionand $62.1 millionfor the comparable periods in 2020. Net loss for the current quarter and full year period includes a non-cash goodwill impairment charge of $9.8 million. There was no goodwill impairment charge in the prior year periods. Net losses include $1.6 millionand $6.2 millionof stock-based compensation expense for the quarter and full year ended December 31, 2021, respectively. Net losses included $1.4 millionand $5.4 millionof stock-based compensation expense for the quarter and full year ended December 31, 2020, respectively.
Conference Call and Webcast
X4 will host a conference call and webcast today at
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 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
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
(in thousands, except per share amounts)
|Three Months Ended||Twelve Months Ended|
|Research and development||12,162||12,298||50,647||41,932|
|General and administrative||7,135||5,357||24,702||20,942|
|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|
|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||)||$||(3.09||)|
|Weighted average common shares outstanding-basic and diluted||29,011||20,174||25,749||20,077|
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS
|Twelve months ended
|Adjustments to reconcile net loss to net cash used in operating activities||19,289||7,376|
|Changes in operating assets and liabilities||(1,498||)||(4,063||)|
|Net cash used in operating activities||(70,905||)||(58,818||)|
|Net cash used in investing activities||(615||)||(1,362||)|
|Net cash provided by financing activities||74,245||12,394|
|Impact of foreign exchange on cash, cash equivalents and restricted cash||(319||)||402|
|Net increase (decrease) in cash, cash equivalents and restricted cash||2,406||(47,384||)|
|Cash, cash equivalents and restricted cash at beginning of period||80,702||128,086|
|Cash, cash equivalents and restricted cash at end of period||$||83,108||$||80,702|
CONSOLIDATED BALANCE SHEETS
|Cash and cash equivalents||$||81,787||$||78,708|
|Research and development incentive receivable||747||917|
|Prepaid expenses and other current assets||5,344||3,682|
|Total current assets||87,878||83,307|
|Property and equipment, net||1,514||1,237|
|Current portion of lease liability||1,075||786|
|Current portion of long-term debt||795||—|
|Total current liabilities||14,023||11,948|
|Long-term debt, including accretion, net of discount||33,139||33,178|
|Total stockholders' equity||64,412||72,799|
|Total liabilities and stockholders' equity||$||117,176||$||122,871|
VP, Investor Relations & Corporate Communications
Senior Account Executive,
Source: X4 Pharmaceuticals