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 | | | | | | | | | ...

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].

X4 Pharmaceuticals, Inc. (NASDAQ:XFOR) Q2 2023 Results Conference Call August 10, 2023 8:30 AM ET Company Participants Dan Ferry - LifeSci Advisors Paula Ragan - CEO Adam Mostafa - CFO Murray Stewart - Interim CMO Mark Baldry - CCO Conference Call Participants Stephen Willey - Stifel Kristen Kluska - Cantor Fitzgerald Ted Tenthoff - Piper Sandler Mark Brown - Cowen and Company Mayank Mamtani - B. Riley RK - H.C. Wainwright Trevor Allred - Oppenheimer Operator Greetings. Welcome to X4 Pharmaceuticals' Secon ...

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. ...