Financial Performance - The company has not generated significant revenue from product sales to date and continues to face uncertainty regarding the commercialization of its product XOLREMDI [3]. - Product revenue for the three months ended September 30, 2024, was $560,000, compared to $0 for the same period in 2023 [28]. - The net loss for the three months ended September 30, 2024, was $36,696,000, compared to a net loss of $2,305,000 for the same period in 2023 [28]. - For the nine months ended September 30, 2024, the company reported a net income of $2.371 million, a significant improvement compared to a net loss of $82.037 million in the same period of 2023 [32]. - The comprehensive loss for the three months ended September 30, 2024, was $(36,651,000), compared to $(2,305,000) for the same period in 2023 [28]. - The basic net loss per share for the three months ended September 30, 2024, was $(0.18), compared to $(0.01) for the same period in 2023 [108]. Assets and Equity - As of September 30, 2024, total assets increased to $178.165 million from $147.258 million as of December 31, 2023, representing a growth of approximately 20.9% [26]. - Total stockholders' equity increased to $59.621 million from $51.099 million, a rise of approximately 16.6% [27]. - Total current assets increased to $142.972 million from $122.076 million, reflecting a growth of about 17% [26]. - The total stockholders' equity as of September 30, 2024, was $59,621,000, an increase from $51,099,000 at December 31, 2023 [29]. Cash and Liquidity - Cash and cash equivalents decreased slightly to $97.412 million from $99.216 million, a decline of about 1.8% [26]. - The liquidity position raises substantial doubt about the company's ability to continue as a going concern without additional funding [9]. - The company had $135.0 million in cash, cash equivalents, and short-term marketable securities as of September 30, 2024, with an accumulated deficit of $475.5 million [35]. - Total cash, cash equivalents, and restricted cash amounted to $98.2 million as of September 30, 2024, down from $100.2 million as of December 31, 2023 [50]. - The company is at risk of not meeting the Minimum Cash Covenant of $20 million required under its loan agreement, which could lead to accelerated loan repayments [36]. - The company must maintain at least $20.0 million in Qualified Cash until January 31, 2025, and thereafter at least 20% of the aggregate principal amount of loans outstanding under the Hercules Loan Agreement [81]. Expenses - Research and development expenses for the nine months ended September 30, 2024, totaled $59,941,000, up from $56,745,000 in the same period of 2023, representing a 3.85% increase [28]. - Selling, general and administrative expenses increased to $46,373,000 for the nine months ended September 30, 2024, compared to $25,578,000 in the same period of 2023, reflecting an increase of 81.36% [28]. - The net cash used in operating activities for the nine months ended September 30, 2024, was $97.906 million, compared to $68.765 million in the prior year [32]. - The company incurred a royalty on annual net sales at a rate of 6% up to $150 million, 10% on sales between $150 million and $300 million, and 12% thereafter [60]. Regulatory and Product Development - The FDA approved the company's New Drug Application for mavorixafor, marketed as XOLREMDI, for treating WHIM syndrome, marking the first approval for this drug [34]. - The company is currently engaged in the U.S. launch of XOLREMDI and plans to seek regulatory approvals for commercialization outside the U.S. [34]. - A global pivotal Phase 3 clinical trial (4WARD study) for mavorixafor has been initiated to evaluate its efficacy and safety in patients with chronic neutropenia [34]. - The company relies heavily on the success of its commercial product XOLREMDI and its development product candidate mavorixafor for future revenue generation [11]. Debt and Obligations - Long-term debt increased to $75,224,000 as of September 30, 2024, up from $54,570,000 as of December 31, 2023 [79]. - The Company has borrowed a total of $75,000,000 under the Hercules Loan Agreement, with an additional $20,000,000 borrowed during the nine months ended September 30, 2024, based on operational milestones [80]. - As of September 30, 2024, future principal and accrued end-of-term payments of $75.9 million under the Hercules Loan Agreement are due on July 1, 2027 [84]. Stock and Equity Incentives - The company granted 6,245,756 stock options during the nine months ended September 30, 2024, with a weighted average exercise price of $0.98 [101]. - As of September 30, 2024, the total unrecognized compensation expense related to unvested stock options and restricted stock units was $7.9 million, expected to be recognized over a weighted average period of 2.3 years [104]. - The company had 11,583,656 stock options outstanding as of September 30, 2024, with a weighted average exercise price of $1.96 [101]. - As of September 30, 2024, the company has approximately 3.9 million shares of common stock available for issuance under its equity incentive plans [98]. Miscellaneous - The company recorded a gain of $105 million from the sale of a Priority Review Voucher (PRV) during the nine months ended September 30, 2024 [110]. - The company recorded $0.5 million and $0.4 million of income related to the research and development incentive program during the nine months ended September 30, 2024 and 2023, respectively [56]. - The company maintains letters of credit secured by restricted cash totaling $788,000 as of September 30, 2024 [49]. - The company has determined that there were no triggering events necessitating an interim impairment test of goodwill during the nine months ended September 30, 2024 [51].

Positive results from completed Phase 2 study of mavorixafor in chronic neutropenia (CN) announced today Pivotal Phase 3 trial of mavorixafor in CN on track to fully enroll in mid-2025 U.S. launch of XOLREMDI® (mavorixafor) in WHIM underway; submission of Marketing Authorization Application (MAA) to European Medicines Agency (EMA) expected by early 2025 Conference call and webcast today at 8:00 a.m. ET BOSTON, Nov. 13, 2024 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve ...

BOSTON, Oct. 24, 2024 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today announced that it will report financial results for the third quarter ended September 30, 2024 and present results from its concluded Phase 2 clinical trial evaluating mavorixafor in people with chronic neutropenia on Wednesday, November 13, 2024. The company will host a conference call and webcast on the same day at 8:00 a.m. ET. T ...

BOSTON, Aug. 28, 2024 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today announced that management will be participating in three upcoming investor conferences in September 2024: Morgan Stanley 22nd Annual Global Healthcare Conference Date: Wednesday, September 4, 2024 Time: 2:35 PM EDT Format: Fireside Chat and 1x1 Meetings Webcast Link Wells Fargo Healthcare Conference Date: Thursday, September 5, 2024 Forma ...

Financial Performance - The company reported an accumulated deficit of $438.84 million as of June 30, 2024, compared to $477.91 million for the same period in 2023, indicating a reduction in losses [16]. - Total stockholders' equity increased to $94.45 million as of June 30, 2024, up from $51.09 million in the previous year [16]. - The net income for the three months ended June 30, 2024, was $90,833,000, compared to a net loss of $55,712,000 for the same period in 2023 [17]. - The basic net income per share for the three months ended June 30, 2024, was $0.45, while for the same period in 2023, it was a loss of $0.33 per share [91]. - The company generated a gain of $105 million from the sale of a Priority Review Voucher (PRV) during the quarter ended June 30, 2024 [93]. Revenue Generation - The company has not generated significant revenue from product sales to date and continues to face uncertainty regarding the market size and growth potential for its approved product, XOLREMDI [3]. - Product revenue for the three months ended June 30, 2024, was $563,000, compared to $0 for the same period in 2023 [17]. - The company recorded $0.7 million in gross revenue from product sales for the three and six months ended June 30, 2024, resulting in net revenue of $0.6 million after accounting for variable considerations [47]. Expenses and Cash Flow - Research and development expenses increased to $20,914,000 for the three months ended June 30, 2024, from $15,601,000 in the same period of 2023, representing a 34.8% increase [17]. - Selling, general, and administrative expenses rose to $13,278,000 for the three months ended June 30, 2024, compared to $10,204,000 in the same period of 2023, a 30.4% increase [17]. - Cash used in operating activities for the six months ended June 30, 2024, was $(63,874,000), compared to $(47,937,000) for the same period in 2023 [22]. - The company has incurred significant operating losses since inception and expects to continue generating negative cash flows in the foreseeable future [25]. Capital and Financing - The company faces significant risks related to its ability to raise additional capital, which may impact its product development and commercialization efforts [8]. - The company has borrowed an additional $20 million under the Hercules Loan Agreement during the quarter ended June 30, 2024, based on achieving an operational milestone [64]. - The annual effective interest rate of the Hercules Loan Agreement is 12.6% as of June 30, 2024 [67]. - The company must maintain a minimum cash level of $20 million under its Hercules Loan Agreement, with adjustments starting January 31, 2025 [25]. Product Development and Regulatory Approval - The company is dependent on the success of its commercial product, XOLREMDI, and its development product candidate, mavorixafor, for future revenue generation [8]. - The FDA approved the Company's New Drug Application for mavorixafor, marketed as XOLREMDI, for WHIM syndrome, marking the first approval for this drug [23]. - The regulatory approval processes for the company's product candidates are lengthy and unpredictable, posing a risk to future commercialization efforts [9]. - The company is planning to seek regulatory approvals to commercialize mavorixafor outside of the U.S. [23]. Inventory and Assets - Total inventory as of June 30, 2024, is $831 thousand, with raw materials valued at $612 thousand and finished goods at $219 thousand [58]. - The company has capitalized inventory-related costs post-FDA approval, including bottling, labeling, and packaging of XOLREMDI [33]. - The company had cash, cash equivalents, and restricted cash of $147,996,000 at the end of the period, compared to $137,456,000 at the end of the previous period [22]. Stock and Compensation - The weighted average shares of common stock outstanding for basic earnings per share increased to 200,440,473 for the three months ended June 30, 2024, from 168,737,764 in the same period of 2023 [17]. - The company granted 6,242,459 restricted stock units during the six months ended June 30, 2024, increasing unvested shares to 8,217,471 [85]. - Total unrecognized compensation expense related to unvested stock options and restricted stock units was $8.5 million, expected to be recognized over a weighted average period of 2.2 years [86]. Risks and Uncertainties - The company expects to continue incurring losses for the foreseeable future and may never achieve profitability [7]. - The company relies on third-party manufacturers for the production of its product candidates, which could lead to delays if issues arise [12]. - The company continues to maintain a valuation allowance against all remaining net deferred tax assets, expecting to generate operating losses in the future [89].

Timing The Knife Dr_Microbe X4 Pharmaceuticals is an intriguing healthcare company that is tackling rare diseases. X4's flagship pipeline drug is mavorixafor, an oral CXCR4 antagonist that is taking on WHIM Syndrome (Warts, Hypogammaglobulinemia, Infections, Myelokathexis), and is expected to combat chronic neutropenia, as well as other immunodeficiency disorders. X4 is using mavorixafor to take on rare diseases, which has enoouraged the FDA to grant a few of their special designations for the regulatory pa ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 _____________________________________________________________________________________ FORM 10-Q _____________________________________________________________________________________ (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2024 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transitio ...

X4 Pharmaceuticals Inc (NASDAQ:XFOR) Q1 2024 Results Conference Call May 7, 2024 8:30 AM ET Company Participants Dan Ferry - IR Paula Ragan - CEO Adam Mostafa - CFO Christophe Arbet-Engels - COO Mark Baldry - CMO Conference Call Participants Stephen Willey - Stifel Edward Tenthoff - Piper Sandler Kristen Kluska - Cantor Fitzgerald Swayampakula Ramakanth - H.C. Wainwright David Bautz - Zacks Small Cap Research Operator [Indiscernible] X4 Pharmaceuticals First Quarter 2024 Earnings Conference Call. At this ti ...

Exhibit 99.1 15 participants treated with fixed doses of either mavorixafor monotherapy or mavorixafor in combination with injectable granulocyte colony-stimulating factor (G-CSF). X4 Pharmaceuticals Reports First-Quarter 2024 Financial Results and Provides Corporate Updates U.S. launch underway for XOLREMDI™ (mavorixafor) in WHIM syndrome XOLREMDI Phase 3 4WHIM clinical trial data published online in Blood, the journal of the American Society of Hematology Presentation of interim clinical data from the ong ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K (Mark One) x ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 or o ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-38295 _________________________________________________________________________________________________________ X4 PHARMACEUTICALS, INC. ...