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. ...

X4 Pharmaceuticals, Inc. (NASDAQ:XFOR) Q4 2023 Earnings Conference Call March 21, 2024 8:30 AM ET Company Participants Dan Ferry - LifeSci Advisors, IR Dr. Paula Ragan - Chief Executive Officer Adam Mostafa - Chief Financial Officer Mark Baldry - Chief Commercial Officer Dr. Christophe Arbet-Engels - Chief Medical Officer Mary DiBiase - Chief Operating Officer Art Taveras - Chief Scientific Officer Conference Call Participants Kristen Kluska - Cantor Fitzgerald Ed Tenthoff - Piper Sandler Sean Lee - H.C. Wa ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 _____________________________________________________________________________________ FORM 10-Q _____________________________________________________________________________________ (Mark One) (Exact name of registrant as specified in its charter) _____________________________________________________________________________________ ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterl ...

Financial Data and Key Metrics Changes - The company reported a cash and equivalents balance of just over $140 million at the end of Q3 2023, which is expected to fund operations into 2025 [12] Business Line Data and Key Metrics Changes - The NDA for mavorixafor for WHIM syndrome was accepted by the FDA for Priority Review, with a PDUFA action date set for April 30, 2024, potentially leading to a launch in Q2 2024 if approved [6][15] - The chronic neutropenia program has enrolled more than 15 participants in the Phase 2 study, with positive initial responses observed [8][35] Market Data and Key Metrics Changes - The company estimates a prevalence of at least 1,000 patients living with WHIM syndrome in the U.S., indicating a significant market opportunity [25] Company Strategy and Development Direction - The company aims to build a fit-for-purpose commercial organization in anticipation of the mavorixafor launch, focusing on educating patients and physicians about WHIM syndrome [6][27] - There is a strategic focus on leveraging relationships and insights gained from the WHIM syndrome commercialization efforts to support the chronic neutropenia program [28] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the future, highlighting the robust data set from the chronic neutropenia Phase 2 study and the anticipated launch of mavorixafor for WHIM syndrome [47] - The management team emphasized the importance of early diagnosis and treatment for WHIM syndrome, aiming to mitigate the damage from infections [15][20] Other Important Information - The company completed an expanded loan facility with Hercules Capital, providing financial flexibility and non-dilutive financing options [11] - The addition of Mr. Keith Woods to the Board of Directors is expected to enhance the company's global commercial strategy and operations [10] Q&A Session Summary Question: What are the reasons for inadequate response in CN patients on GCSF? - The population is heterogeneous, with genetic mutations affecting responses to GCSF, leading to varied treatment outcomes [51] Question: What is the expected launch trajectory for WHIM? - The company is confident in a bolus of patients at launch due to strong engagement with physicians and patient groups [53] Question: How many sites are planned for the Phase 3 trial? - The company plans to have approximately three times as many sites for the Phase 3 trial compared to the Phase 2 trial [59] Question: Is there any open-label extension work being done in WHIM? - An open-label extension is ongoing, with data expected to be presented in the first half of next year [64] Question: What type of doctor typically diagnoses WHIM? - Patients may be diagnosed by either immunologists or hematologists, depending on their symptoms [86]

November 9, 2023 | --- | --- | --- | --- | --- | --- | |--------------------------------------------------|----------|-------|-------|-------|-------| | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | PROGRESS | PATIENTS | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | Developing the first oral treatments for chronic | | | | | | | | | | | | | | neutropenic disorders | | | | | | | | | ...