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 ...

Product Development - The company is developing mavorixafor, an oral therapy for WHIM syndrome, with a PDUFA target action date set for April 30, 2024, following FDA's Priority Review designation [119]. - The Phase 3 clinical trial (4WHIM) for mavorixafor met its primary endpoint, showing statistically significant improvements in absolute neutrophil counts and reductions in annualized infection rates compared to placebo [120]. - The company is advancing mavorixafor for chronic neutropenic disorders, with a Phase 2 trial currently underway and further data expected in the first half of 2024 [122]. Financial Performance - The company reported a net loss of $2.3 million for the three months ended September 30, 2023, compared to a net loss of $21.6 million in the same period of 2022, reflecting a significant improvement [127]. - For the nine months ended September 30, 2023, the company reported a net loss of $82.0 million, compared to a net loss of $64.8 million for the same period in 2022 [142]. - Total operating expenses for the three months ended September 30, 2023, were $27.2 million, up from $20.2 million in the same period of 2022, marking a $7.1 million increase [127]. - Other income for the three months ended September 30, 2023, was $24.9 million, a significant increase compared to a loss of $1.4 million in the same period of 2022, primarily due to changes in the fair value of Class C warrants [132]. Expenses - Research and development expenses increased by $5.0 million and $14.7 million for the three and nine months ended September 30, 2023, respectively, primarily due to higher regulatory and contract manufacturing costs [129]. - Selling, general and administrative expenses rose by $2.1 million and $5.1 million for the current three and nine month periods, driven by increased headcount and costs associated with preparing for the potential product launch [131]. Cash Flow and Financing - Net cash used in operating activities for the nine months ended September 30, 2023, was $68.8 million, an increase from $58.0 million in the prior year, primarily due to higher research and development expenses [142]. - Net cash provided by financing activities for the nine months ended September 30, 2023, was $88.4 million, primarily from a private placement that generated net proceeds of $60.4 million [144]. - The company had cash, cash equivalents, and restricted cash of $132.6 million as of September 30, 2023, compared to $81.1 million at the end of the same period in 2022 [142]. - The company has entered into an ATM Sales Agreement allowing for the sale of up to an additional $75 million in common stock, with approximately $14.3 million already sold [135]. - The Hercules Loan Agreement allows for maximum borrowings of up to $115.0 million, with $32.5 million already borrowed as of December 31, 2022 [138]. Future Outlook - The company expects to require additional capital to fund operations beyond 2025, as it does not anticipate generating revenue from product sales for several years [141]. - The company has assessed its ability to continue as a going concern and believes it has adequate financial resources to fund operations into 2025 [140]. - Macroeconomic conditions, including inflation and geopolitical events, may negatively impact the company's business and financial results [126]. - The company incurred significant operating losses and negative cash flows since inception, with no products commercialized to date [139].

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].