Core Insights - X4 Pharmaceuticals has activated approximately 90% of targeted sites for its Phase 3 4WARD trial in chronic neutropenia, with full enrollment expected in Q3 or Q4 2025 and top-line data anticipated in the second half of 2026 [1][8] - The company reported net revenues of $2.6 million for XOLREMDI® since its launch in May 2024, with expectations for increased sales in 2025 as physician outreach improves [1][3] - A strategic restructuring was announced in February 2025 to focus on mavorixafor in chronic neutropenia, aiming to reduce annual spending by $30-35 million [9][12] Company Developments - X4 Pharmaceuticals launched XOLREMDI® (mavorixafor) in May 2024 after receiving FDA approval for treating WHIM syndrome, marking a significant milestone for the company [2][3] - The company has entered into commercialization partnerships in the EU, Australia, New Zealand, and MENA regions to expand its reach for mavorixafor [2][4][5] - The Phase 2 trial of mavorixafor showed positive results, leading to the ongoing Phase 3 4WARD trial, which aims to enroll 150 participants with chronic neutropenia [11][18] Financial Performance - For the fourth quarter and full year ended December 31, 2024, X4 reported net product revenue of $1.4 million and $2.6 million, respectively, with a net loss of $39.8 million for Q4 and $37.5 million for the full year [17][23] - The company had a cash position of $102.8 million as of December 31, 2024, which is expected to support operations into the first half of 2026 [10][12] - X4 recognized a gain of $105 million from the sale of a priority review voucher in 2024, contributing to its financial results [17][23]

Core Insights - X4 Pharmaceuticals will report its financial results for Q4 and the full year ended December 31, 2024, on March 25, 2025 [1] - A conference call and webcast will be held on the same day at 8:30 a.m. ET for investors [2] - The company focuses on developing therapies for rare immune system diseases, with its lead product mavorixafor currently marketed in the U.S. as XOLREMDI [3] Company Overview - X4 Pharmaceuticals is dedicated to improving the lives of patients with rare immune system diseases and significant unmet medical needs [3] - The company has developed mavorixafor, an orally available CXCR4 antagonist, and is exploring additional indications for this drug [3] - X4 is conducting a global pivotal Phase 3 clinical trial (4WARD) for mavorixafor in patients with chronic neutropenic disorders [3]

Core Points - X4 Pharmaceuticals and taiba rare have entered into an exclusive agreement for the distribution and commercialization of XOLREMDI (mavorixafor) in select Middle Eastern countries [1][3] - XOLREMDI is an oral treatment approved by the FDA for WHIM syndrome, a rare primary immunodeficiency, aimed at increasing circulating mature neutrophils and lymphocytes [2][12] - The agreement allows taiba to lead the distribution and marketing efforts in the region, with local regulatory filings based on X4's FDA registration dossier [3] Company Overview - X4 Pharmaceuticals focuses on developing innovative therapies for rare diseases of the immune system, with mavorixafor being its first marketed product [13] - Taiba Healthcare specializes in marketing, sales, and distribution of orphan and rare disease treatments in the MENA region, established in 1980 [14] Product Information - XOLREMDI is the first and only treatment approved for WHIM syndrome, which is characterized by low blood levels of neutrophils and lymphocytes, leading to serious infections [11][12] - The drug has received Orphan Drug Designation in both the U.S. and the European Union, indicating its significance in addressing unmet medical needs [3]

Core Insights - X4 Pharmaceuticals is restructuring its workforce and capital spending to focus on advancing mavorixafor for chronic neutropenia and optimizing the promotion of XOLREMDI for WHIM syndrome [1][2] - The company expects to reduce annual spending by $30-35 million, extending its cash runway into the first half of 2026 [2][5] - Workforce reductions will involve a decrease of 43 employees, approximately 30% of the total workforce, and the discontinuation of research efforts in Vienna, Austria [5] Company Overview - X4 Pharmaceuticals develops therapies for rare immune system diseases, with mavorixafor being a key product marketed in the U.S. as XOLREMDI [3] - The company is conducting a global pivotal Phase 3 clinical trial (4WARD) for mavorixafor in chronic neutropenic disorders [3] Strategic Focus - The restructuring aims to enhance operational and capital efficiency while maximizing the global market opportunity for mavorixafor [2] - The company plans to streamline spending to support ongoing clinical development for chronic neutropenia [5]

Company Updates - X4 Pharmaceuticals announced that its Marketing Authorization Application (MAA) for mavorixafor, a treatment for WHIM syndrome, has been validated for review by the European Medicines Agency (EMA) [1] - Mavorixafor received U S Food and Drug Administration (FDA) approval in April 2024 as XOLREMDI, an oral, once-daily treatment for patients aged 12 and older with WHIM syndrome [1] - The company expects a decision on the MAA in the first half of 2026 [1] - X4 Pharmaceuticals has an exclusive licensing and supply agreement with Norgine to commercialize mavorixafor in Europe, Australia, and New Zealand [3] Clinical Trial Results - The global, pivotal 4WHIM Phase 3 trial met its primary endpoint and a key secondary endpoint, with no treatment-related serious adverse events reported and no discontinuations for safety events [2] - Once-daily oral mavorixafor resulted in reductions in the rate, severity, and duration of infections in participants with WHIM syndrome [2] Market Opportunity - If approved by the EMA, mavorixafor would be the first drug indicated for patients with WHIM syndrome in Europe, where the population is estimated to be approximately 1,000 people [3] - In the U S, at least 1,000 people are currently diagnosed with WHIM syndrome, with another 1,000 estimated in Europe [11] Product Information - Mavorixafor is a small-molecule antagonist of the CXCR4 receptor, developed as a once-daily oral therapy for rare primary immunodeficiencies, including WHIM syndrome [2] - XOLREMDI (mavorixafor) is a selective CXCR4 receptor antagonist approved in the U S for increasing the number of circulating mature neutrophils and lymphocytes in patients with WHIM syndrome [12] Company Background - X4 Pharmaceuticals focuses on developing and commercializing innovative therapies for rare diseases of the immune system, leveraging expertise in CXCR4 and immune system biology [13] - The company is headquartered in Boston, Massachusetts, and operates a research center of excellence in Vienna, Austria [13]

Licensing and Financial Terms - X4 Pharmaceuticals will receive an upfront payment of €28.5 million and up to €226 million in potential regulatory and commercial milestone payments, in addition to tiered, double-digit royalties up to the mid-twenties [1] - The upfront non-dilutive funds will strengthen X4's balance sheet as enrollment ramps up in the company's global Phase 3 clinical trial in chronic neutropenia [1] - X4 and Norgine will collaborate closely on regulatory filings, with X4 continuing to be responsible for the ongoing global, pivotal Phase 3 4WARD clinical trial evaluating mavorixafor in chronic neutropenia [7] Strategic Partnership and Market Expansion - Norgine will commercialize mavorixafor in Europe, Australia, and New Zealand following regulatory approvals [1] - The agreement underscores Norgine's commitment to bring transformative therapies to patients in need in these key strategic territories [1] - Norgine will be responsible for all market access and commercialization activities and will eventually hold all marketing authorizations in the licensed territories [7] Product Development and Regulatory Progress - Mavorixafor is a selective CXCR4 receptor antagonist approved in the U.S. and marketed by X4 as XOLREMDI®, an oral, once-daily treatment for patients 12 years of age and older with WHIM syndrome [4] - X4 expects to announce shortly the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for mavorixafor in the treatment of WHIM syndrome [4] - X4 is also developing mavorixafor to treat chronic neutropenia (CN) and is currently conducting a global, pivotal Phase 3 clinical trial in certain CN disorders [4] Company Profiles - X4 Pharmaceuticals is focused on developing and commercializing innovative therapies for rare diseases of the immune system, leveraging expertise in CXCR4 and immune system biology [2] - Norgine is a specialty pharmaceutical and consumer healthcare company with more than €500 million of annual revenues and a 120-year track record of bringing life-changing products to patients and consumers across Western Europe, Australia, and New Zealand [5] Executive Commentary - Paula Ragan, Ph.D., President and CEO of X4 Pharmaceuticals, highlighted the strategic agreement as a significant milestone for maximizing the global potential of mavorixafor and funding the ongoing Phase 3 trial in chronic neutropenia [7] - Janneke van der Kamp, CEO of Norgine, emphasized the partnership's role in expanding access to mavorixafor for patients in Europe, Australia, and New Zealand, and Norgine's position as a partner of choice in these regions [7]

Company Participation - X4 Pharmaceuticals will participate in the Stifel 2024 Healthcare Conference on November 18-19, 2024 [1] - The company will engage in a fireside chat on November 18, 2024, at 4:10 PM ET [2] - One-on-one meetings will be available for interested investors during the conference [3] Company Overview - X4 Pharmaceuticals focuses on developing and commercializing innovative therapies for rare diseases of the immune system [4] - The company has successfully developed mavorixafor, which is approved in the U.S. as XOLREMDI® (mavorixafor) capsules for its first indication [4] - X4 is also exploring additional potential indications for mavorixafor [4]

3Q 2024 Business Update & Phase 2 Chronic Neutropenia Study Results November 13, 2024 Forward-Looking Statements This presentation including any printed or electronic copy of these slides, the talks given by the presenters, the information communicated during any delivery of the presentation and any question and answer sessions and any documents or materials distributed at or in connection with the presentation, contains forward-looking statements within the meaning of applicable securities laws, including ...

Financial Data and Key Metrics Changes - The company ended Q3 2024 with cash and equivalents of almost $136 million, providing a runway into late 2025, not including the expected ramp-up of sales of XOLREMDI throughout next year [54] Business Line Data and Key Metrics Changes - Sales of XOLREMDI appear to be flat quarter-over-quarter, attributed to initial product stocking with specialty pharma during the launch [11] - The company engaged with all 3,400 targeted immunologists and hematologists for the launch, with over 75% of these prescribers now knowledgeable about WHIM syndrome [12][13] Market Data and Key Metrics Changes - More than 150 million lives are now covered under favorable reimbursement policies [14] - Approximately 50,000 people are diagnosed with chronic neutropenia (CN) in the U.S., with about 15,000 having high unmet needs [25] Company Strategy and Development Direction - The company aims to leverage its commercial infrastructure and relationships within the WHIM community to support a potential launch into chronic neutropenia if mavorixafor is approved [16] - The ongoing Phase III trial, named 4WARD, is expected to enroll 150 participants across 90 to 110 sites, with a majority of sites initiated by early 2025 [17] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential of mavorixafor to transform chronic care for neutropenic patients, highlighting the urgent need for innovation in this area [24][50] - The company is optimistic about the ongoing Phase III trial and the potential to reduce infection rates in the chronic neutropenia population [46][50] Other Important Information - The Phase II study of mavorixafor demonstrated that it durably and meaningfully increased mean absolute neutrophil count (ANC) in both monotherapy and combination therapy settings [49] - The treatment was generally well tolerated, with the most frequent treatment-related adverse events being mild to moderate gastrointestinal issues [47] Q&A Session Summary Question: What were the biggest criteria in the trial that made physicians comfortable to reduce G-CSF usage? - The comfort to adjust G-CSF dosing was primarily due to the high and robust ANC responses from mavorixafor, reflecting confidence in the data [57] Question: Were there any early anecdotes regarding reduced pain or better convenience benefits for patients? - It was considered too early to assess anecdotal evidence regarding pain reduction or convenience benefits [59] Question: How is the Phase III trial designed to capture benefits from G-CSF reduction? - The Phase III trial is designed to follow up on aspects of tolerability and safety as per FDA requirements [64] Question: Can you provide more detail on the new patient ambassador efforts for XOLREMDI? - The company has launched a new patient-directed campaign to raise disease awareness and encourage discussions between patients and physicians regarding WHIM syndrome [66] Question: How many patients are currently on XOLREMDI and what is the commercialization setup? - The focus is on raising disease awareness and encouraging screening for WHIM patients, with further details expected as the company ramps up into 2025 [68] Question: Was there any data collected on infections in the Phase II study? - The Phase II study was not designed to assess infection rates, but the correlation between ANC and infection risk is well established [77]

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].