Financial Performance - The company reported a net loss of $55.712 million for the three months ended June 30, 2023, compared to a net loss of $21.212 million for the same period in 2022, representing an increase in loss of $34.500 million[134]. - The company incurred a net loss of $79.7 million for the six months ended June 30, 2023, compared to a net loss of $43.2 million for the same period in 2022, representing a 84.5% increase in net loss year-over-year[149]. - Net cash used in operating activities for the six months ended June 30, 2023, was $47.9 million, an increase from $38.7 million in the same period of 2022, primarily due to increased research and development expenses[149]. Research and Development - Research and development expenses increased by $1.780 million and $9.730 million for the three and six months ended June 30, 2023, respectively, compared to the same periods in 2022, primarily due to higher costs associated with the pivotal Phase 3 clinical trial of mavorixafor[136]. - The pivotal Phase 3 clinical trial for mavorixafor met its primary endpoint, showing statistically significant improvements in absolute neutrophil counts and was well tolerated throughout the 52-week trial[127]. - The company expects to share additional data from the Phase 2 clinical trial of mavorixafor in chronic neutropenic disorders in Q4 2023 and initiate a pivotal Phase 3 trial in the first half of 2024[128]. Revenue Generation - The company has not generated revenue from product sales to date and does not expect to do so in the foreseeable future[132]. - The company has not yet commercialized any products and does not expect to generate revenue from sales for several years, if at all[145]. - The company expects to need additional capital to fund operations beyond 2025, as it does not anticipate generating revenue from product sales for several years[148]. Cash and Financing - As of June 30, 2023, the company had cash and cash equivalents of $136.4 million, restricted cash of $1.0 million, and investments in marketable securities of $4.9 million[145]. - The company amended its Hercules Loan Agreement on August 2, 2023, increasing the maximum borrowing capacity to $115.0 million, which includes $32.5 million outstanding and an additional $22.5 million drawn at closing[146]. - Net cash provided by financing activities for the six months ended June 30, 2023, was $67.2 million, significantly higher than $4.6 million in the same period of 2022, primarily due to a private placement[151]. - The company believes it has sufficient cash and marketable securities to fund operations into 2025, but may face a cash covenant violation in the first quarter of 2025[152]. Operational Challenges - Selling, general and administrative expenses increased by $3.455 million and $3.032 million for the three and six months ended June 30, 2023, respectively, due to increased headcount and third-party costs[137]. - Other expenses, net, increased significantly due to a rise in the fair value of Class C warrants, resulting in a total other expense of $29.892 million for the three months ended June 30, 2023[138]. - Macroeconomic conditions, including rising inflation and interest rates, may negatively impact the company's growth and results of operations[133]. Regulatory and Compliance - The company plans to submit a New Drug Application (NDA) for mavorixafor in the treatment of WHIM syndrome in early Q3 2023[127]. - The Amended Loan Agreement requires the company to maintain a minimum cash level of $20.0 million through January 31, 2025, subject to adjustments based on operational milestones[146]. - The company is classified as a smaller reporting company, allowing it to take advantage of scaled disclosures under certain conditions[156].

Financial Data and Key Metrics Changes - X4 Pharmaceuticals raised approximately $65 million in gross proceeds through a PIPE financing during the quarter, coinciding with positive Phase III results for WHIM [11] - The company reported cash, cash equivalents, restricted cash, and marketable securities totaling $142.3 million as of June 30, 2023, which, along with a $22.5 million drawdown from a debt facility, extends the cash runway into 2025 [13] - X4 was added to the Russell 3000 Index during its annual reconstitution in late June [12] Business Line Data and Key Metrics Changes - The company is focused on the chronic neutropenia program, particularly the development of Mavorixafor, which is positioned to be the first oral treatment option for chronic neutropenia and WHIM syndrome [14][15] - Emerging data from the Phase 2 trial of Mavorixafor showed robust increases in absolute neutrophil counts (ANC) and the potential for reducing G-CSF therapy [24][30] Market Data and Key Metrics Changes - Market research indicates that approximately 50,000 people in the U.S. are diagnosed with chronic neutropenia, with a significant unmet need identified among those experiencing severe symptoms [16][18] - The initial target population for Mavorixafor is estimated to be around 15,000 individuals with high unmet needs, which could expand with further market research [20][32] Company Strategy and Development Direction - X4 Pharmaceuticals is preparing for a Phase III clinical trial in chronic neutropenia, expected to initiate in the first half of 2024, following positive feedback from the FDA [31][36] - The company aims to deliver a meaningful oral treatment option first for WHIM syndrome and subsequently for chronic neutropenia, with plans for potential label expansion to other immunodeficiencies [69] Management's Comments on Operating Environment and Future Outlook - Management expressed excitement about the progress made in the chronic neutropenia program and the potential for Mavorixafor to address significant unmet needs in the patient population [35][36] - The company is optimistic about the upcoming NDA submission for Mavorixafor and the anticipated positive reception from the FDA based on the clinical efficacy demonstrated [54][65] Other Important Information - X4 received an additional patent for Mavorixafor, extending protection through December 2038 [9] - The company announced the appointment of Dr. Christophe Arbet-Engels as the new Chief Medical Officer, expected to bring valuable experience to the team [9] Q&A Session Summary Question: Can you remind us within the Phase 2 CN protocol, if there's a threshold ANC at which the investigator makes the GCSF tapering decision? - The protocol allows for tapering if ANC exceeds 10,000, but physicians can decide on tapering at other levels [40] Question: What's your level of confidence that the ANC will remain comfortably above the normal range? - There is optimism based on initial data, but long-term follow-up is necessary to confirm sustained results [42] Question: How many patients have you enrolled to date? - More than three patients have been enrolled, with ongoing data collection expected to provide a comprehensive picture later in the year [45] Question: How do the findings from the real-world analysis help in terms of Phase 3 protocol design? - Insights from the analysis have informed the understanding of unmet needs and will guide the Phase III trial preparations [49] Question: What remains to be done for the NDA submission? - The NDA is in the final stages of quality control and publishing, with readiness for potential FDA inspections [54] Question: Will the NDA package include recent infection data? - All relevant data, including recent findings, will be included in the NDA submission [64]

X4 Pharmaceuticals, Inc. (NASDAQ:XFOR) Q1 2023 Results Conference Call May 4, 2023 8:30 AM ET Company Participants Dan Ferry - IR, LifeSci Advisors Dr. Paula Ragan - President and CEO Adam Mostafa - CFO Mark Baldry - Chief Commercial Officer Conference Call Participants William Wood - B. Riley Securities Eva Privitera - TD Cowen Swayampakula Ramakanth - Wainwright Rick Miller - Cantor Fitzgerald Operator Greetings, and welcome to X4 Pharmaceuticals' First Quarter 2023 Earnings Conference Call. [Operator Ins ...

Financial Performance - The company reported a net loss of $24.02 million for the three months ended March 31, 2023, compared to a net loss of $21.97 million for the same period in 2022, reflecting an increase in loss of $2.05 million [112]. - For the three months ended March 31, 2023, the net loss was $24.0 million, compared to a net loss of $22.0 million for the same period in 2022, representing an increase in net loss of approximately 9.1% [126]. - Net cash used in operating activities for the three months ended March 31, 2023, was $26.5 million, an increase of 31.4% from $20.2 million in the same period of 2022 [127]. - Cash and cash equivalents at the end of the period were $94.4 million, down from $123.0 million at the beginning of the period, reflecting a net decrease of $28.6 million [126]. - Net cash used in financing activities was $2.1 million for the three months ended March 31, 2023, compared to net cash provided of $5.0 million in the same period of 2022 [129]. Research and Development - Research and development expenses increased by $8.0 million to $22.06 million for the three months ended March 31, 2023, compared to $14.11 million in the prior year, primarily due to higher accrued in-license fees and third-party costs associated with clinical trials [112][114]. - The company expects ongoing increases in expenses related to clinical trials for its product candidates, which may significantly impact future funding requirements [130]. - The company is preparing a U.S. New Drug Application (NDA) for mavorixafor, expected to be submitted in early Q3 2023, following positive Phase 3 trial results for WHIM syndrome [104]. Liquidity and Capital Resources - As of March 31, 2023, the company had cash and cash equivalents of $93.4 million, with a restricted cash balance of $1.0 million, but faces potential liquidity issues beginning Q1 2024 [124][125]. - The Hercules Loan Agreement provides for a term loan of $32.5 million, with an interest-only payment period through October 1, 2024, but the company has fully borrowed this amount [123]. - The company anticipates that its cash and cash equivalents will fund operations into the second quarter of 2024, but may require additional capital to meet the minimum cash covenant of the Hercules Loan Agreement [130]. - The company acknowledges substantial doubt about its ability to continue as a going concern without raising additional capital [125]. Other Income and Expenses - Other income increased to $5.29 million for the three months ended March 31, 2023, compared to a net expense of $674,000 in the prior year, primarily due to changes in fair value of Class C warrants and increased interest income [117]. - Non-cash expenses for the three months ended March 31, 2023, included adjustments for the change in fair value of Class C warrant liability and stock-based compensation, totaling $3.1 million [127]. - The company expects selling, general and administrative expenses to grow as it continues to build out its functions in these areas [116]. Company Classification and Accounting Policies - The company is classified as a smaller reporting company (SRC), allowing it to take advantage of scaled disclosures under certain conditions [135]. - The company has not reported any material changes to its critical accounting policies during the three months ended March 31, 2023 [134].

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, 2022 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 2022 Earnings Conference Call March 21, 2023 9:00 AM ET Company Participants Dan Ferry - IR, LifeSci Advisors Paula Ragan - CEO Adam Mostafa - CFO Murray Stewart - Interim Chief Medical Officer Mark Baldry - Chief Commercial Officer Conference Call Participants Divya Rao - TD Cowen Edward Tenthoff - Piper Sandler Kristen Kluska - Cantor Fitzgerald Swayampakula Ramakanth - H.C. Wainwright Operator Thank you for standing by. This is the conference operator. Welcome to ...

Corporate Overview November 2022 Forward-Looking Statements 2 This presentation 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 inten ...

Financial Data and Key Metrics Changes - The company is preparing for a potential NDA submission in early second half of 2023, with a commercial launch of mavericksphore anticipated in the first half of 2024 [24][32] - The average pretreatment absolute neutrophil count (ANC) for patients enrolled in the phase three trial was 220 cells per microliter, indicating severe neutropenia [12][13] - The company expects to announce phase three top line data in the fourth quarter of 2022 [14][20] Business Line Data and Key Metrics Changes - The phase three trial for mavericksphore in WHIM syndrome has completed enrollment with 31 patients from 12 countries, exceeding initial enrollment targets [12][14] - Over 90% of eligible participants from the placebo-controlled portion of the study opted to continue into the open-label extension study [14] Market Data and Key Metrics Changes - The company estimates that there are approximately 50,000 chronic neutropenia patients in the U.S., with mavericksphore potentially addressing a significant unmet need [7][41] - The company is focusing on the top five to seven European markets for the initial launch of mavericksphore following U.S. approval [24] Company Strategy and Development Direction - The company aims to establish mavericksphore as the first treatment specifically developed for WHIM syndrome, with plans to expand its use to other chronic neutropenic disorders [22][32] - The company is investing in activities to support education and awareness of WHIM syndrome, including collaborations with experts and patient organizations [23] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming phase three trial results and the potential for mavericksphore to transform the treatment landscape for chronic neutropenia [33] - The company is actively engaging with the FDA to ensure alignment on study design and regulatory pathways for both WHIM and chronic neutropenia indications [20][56] Other Important Information - The company has secured conditional approval of a brand name with the FDA for mavericksphore, marking a significant step in the commercialization process [29] - The company has completed registration batches for manufacturing, ensuring a stable supply chain for the anticipated launch [29] Q&A Session Summary Question: What is the average ALC for patients pretreatment? - Management indicated that the average ALC is not readily available but will follow up with the information [36] Question: Have you seen the blinded infection rate in the trial? - Management confirmed that infection events are being monitored and are consistent with expectations [38] Question: How do you reconcile the 50,000 patient estimate with the previously mentioned 2,500 patients on chronic GCSF? - Management explained that the previous number was based on registry data and acknowledged the challenges in defining chronic use of GCSF [40][41] Question: What are the powering assumptions for the WHIM trial? - Management stated that the trial was powered based on phase two results, with a current power greater than 95% for the primary endpoint [45] Question: What is the latest estimate on the prevalence of WHIM? - Management remains confident in guiding to a prevalence of 1,000 or higher for WHIM syndrome [46] Question: What are the differences in patient populations between phase two and phase three? - Management noted that both populations required a clinical diagnosis of WHIM syndrome, with the main difference being the inclusion of pediatric patients in phase three [50] Question: What activities will occur between the top line data and NDA filing? - Management outlined that the phase three data will be analyzed and prepared for NDA submission, with ongoing regulatory discussions for chronic neutropenia [56]

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 September 30, 2022 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the trans ...

X4 Pharmaceuticals, Inc. (NASDAQ:XFOR) Q2 2022 Earnings Conference Call August 4, 2022 8:30 AM ET Company Participants Dan Ferry - LifeSci Advisors, IR Dr. Paula Ragan - Chief Executive Officer Adam Mostafa - Chief Financial Officer Art Taveras - Chief Scientific Officer Diego Cadavid - Chief Medical Officer Mary DiBiase - Chief Operating Officer Conference Call Participants Stephen Willey - Stifel Eva Privitera - Cowen Mayank Mamtani - B. Riley Securities Arthur He - H.C. Wainwright Operator Greetings. And ...