Financial Data and Key Metrics Changes - The company ended 2024 with just under $103 million in cash and cash equivalents, which includes the EUR 28.5 million received from Neurogene and the expected financial impact of the strategic restructuring [17] - Net revenues for Zolremdi were $1.4 million for Q4 and $2.6 million for the full year 2024 [17] - R&D expenditures totaled $21.7 million for Q4 and $81.6 million for the full year, with SG&A expenses at $15.1 million for Q4 and $61.5 million for the full year [17] - The net loss was $39.8 million for Q4 and $37.5 million for the full year, reflecting a one-time sale of a priority review voucher for $105 million [17] Business Line Data and Key Metrics Changes - Zolremdi sales topped $2.5 million, covering the 7.5 months since its launch in mid-May [8] - The company has engaged with top-tier immunologists and hematologists, expecting continued patient finding and shorter time to prescription [8] Market Data and Key Metrics Changes - The company has made significant progress in expanding the global reach of MAVERICKS-four, with an MAA submitted to the EMA and accepted for review [9] - Two international partnerships were established, one with Neurogene for commercialization in Europe, Australia, and New Zealand, and another with TABA Rare for the MENA region [10][11] Company Strategy and Development Direction - The company aims to maximize the opportunity for MAVERICKS-four in chronic neutropenia, believing it represents a larger market opportunity [16] - A strategic restructuring was announced to decrease spending by approximately $30 million to $35 million annually [16] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the engagement with regulatory agencies, indicating a supportive environment for trial design and success [20][24] - The company is focused on building awareness and education around WIM syndrome, with plans to pivot efforts towards the patient community [62] Other Important Information - The FORWARD trial for MAVERICKS-four has been activated at about 90% of targeted sites worldwide, with expectations for full enrollment in Q3 or Q4 of this year [15] - A slight price increase of 7% for Zolremdi was implemented at the start of 2025 [37] Q&A Session Summary Question: Regulatory conversation regarding ANC threshold - Management indicated that the FDA is supportive of the trial design focusing on moderate to severe patients, and the change in ANC threshold was aligned with this [20][21] Question: Impact of tightening eligibility criteria on enrollment pace - Management does not expect the tightening of eligibility criteria to slow down enrollment, as the targeted patient population remains consistent [25][26] Question: Current inventory status and discounting trends - The company noted that inventory metrics reflect stocking up in Q4, and there is no discounting occurring at this time [28][33] Question: Patient numbers and price increase for Zolremdi - Management refrained from disclosing specific patient numbers but confirmed a 7% price increase for the new year [36][38] Question: Screening progress for Phase III trial - Management reported that site activations are at about 90%, and screening is ramping up as expected [43][44] Question: Commercialization experiences since launch - The company has focused on building awareness and engaging physicians, successfully enrolling patients from the clinical trial onto the product [50][51] Question: Sales guidance for 2025 - Management is striving for continued demand increase but is not providing formal sales guidance [60][62] Question: Impact of primary outcome change on labeling for chronic neutropenia - Management does not believe the change in primary outcome will affect the labeling for chronic neutropenia [66]

Financial Data and Key Metrics Changes - The company ended 2024 with just under $103 million in cash and cash equivalents, which includes the EUR 28.5 million received from Neurogene and the expected financial impact of the strategic restructuring [19] - Net revenues for Zolremdi were $1.4 million for Q4 and $2.6 million for the full year 2024 [19] - R&D expenditures totaled $21.7 million for Q4 and $81.6 million for the full year, including non-cash expenses [19] - SG&A expenses were $15.1 million for Q4 and $61.5 million for the full year, also including non-cash expenses [19] - The net loss was $39.8 million for Q4 and $37.5 million for the full year, with the annual figure reflecting a one-time sale of a priority review voucher for $105 million [19] Business Line Data and Key Metrics Changes - Zolremdi sales topped $2.5 million, covering 7.5 months since its launch in mid-May [9] - The company has engaged with top-tier immunologists and hematologists to increase patient finding and shorten the time to prescription [9] Market Data and Key Metrics Changes - The company has made significant progress in expanding the global reach of MAVERICKS-four, with an MAA submitted to the EMA for review [10] - The company entered into two international partnerships to commercialize MAVERICKS-four and Zolremdi in Europe, Australia, New Zealand, and the MENA region [11][12] Company Strategy and Development Direction - The company aims to maximize the opportunity for MAVERICKS-four in chronic neutropenia, which is viewed as a larger market opportunity [17] - A strategic restructuring is expected to decrease annual spending by $30 million to $35 million [17] - The commercialization strategy focuses on disease awareness and strengthening relationships with patient advocacy groups [6] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the engagement with regulatory agencies, indicating a supportive environment for trial success [23][26] - The company is focused on building awareness and education around WIM syndrome to drive demand for Zolremdi [52][64] - Management is confident in the enrollment trajectory for the FORWARD trial, expecting full enrollment by Q3 or Q4 of 2025 [45] Other Important Information - A slight price increase of 7% for Zolremdi was implemented at the start of 2025 [39][41] - The company is not currently engaging in discounting due to a tight distribution channel [35] Q&A Session Summary Question: Regulatory conversation regarding ANC threshold - Management indicated that the FDA is supportive of the trial design focusing on moderate to severe patients, and the change in ANC threshold does not impact the trial's case [21][24] Question: Impact of tightening eligibility criteria on enrollment pace - Management does not expect the tightening of eligibility criteria to slow down enrollment, as the targeted patient population has been consistent [28] Question: Current inventory status and discounting trends - The company noted that inventory reflects stocking up in Q4, and there are no current trends in discounting as they have a tight distribution channel [30][35] Question: Patient numbers and price increase for Zolremdi - Management is not disclosing patient numbers yet but confirmed a 7% price increase for Zolremdi [38][41] Question: Screening progress for Phase III trial - Management reported that site activations are at about 90% and screening is progressing well [45][46] Question: Commercialization learnings since launch - The company has focused on building awareness and engaging physicians, successfully enrolling patients from the clinical trial onto the product [52][53] Question: Sales goals for 2025 - Management is striving for continued demand increase but is not providing formal sales guidance [62]

Financial Performance - X4 Pharmaceuticals reported net revenues of $2.6 million for XOLREMDI® since its launch in May 2024, with expectations for increased sales in 2025 [3]. - X4 reported a net loss of $37.5 million for the full year 2024, compared to a net loss of $101.2 million in 2023 [20]. - Product revenue for Q4 2024 was $1.434 million, compared to $0 in Q4 2023, indicating a significant increase [28]. - The net loss for Q4 2024 was $39.821 million, compared to a net loss of $19.130 million in Q4 2023, representing a 108.5% increase in losses [28]. Expenses - Research and Development (R&D) expenses for the full year 2024 were $81.6 million, up from $72.0 million in 2023 [15]. - Selling, General, and Administrative (SG&A) expenses for the full year 2024 were $61.5 million, compared to $35.5 million in 2023 [15]. - Research and development expenses for Q4 2024 were $21.702 million, up from $15.272 million in Q4 2023, reflecting a 42.5% increase [28]. - Selling, general and administrative expenses rose to $15.145 million in Q4 2024 from $9.927 million in Q4 2023, a 52.5% increase [28]. - A strategic restructuring announced in February 2025 is expected to reduce spending by $30-35 million annually [12]. Cash and Assets - X4 had a cash position of $102.8 million as of December 31, 2024, and expects sufficient funds to support operations into the first half of 2026 [15]. - Total current assets decreased to $112.175 million as of December 31, 2024, down from $122.076 million as of December 31, 2023 [30]. - Cash and cash equivalents decreased to $55.699 million from $99.216 million year-over-year, a decline of 43.8% [30]. - The company reported a total stockholders' equity of $22.149 million as of December 31, 2024, down from $51.099 million a year earlier, a decrease of 56.7% [30]. - Total liabilities increased to $124.298 million as of December 31, 2024, compared to $96.159 million as of December 31, 2023, marking a 29.3% increase [30]. Clinical Trials and Regulatory Challenges - The company anticipates full enrollment in the Phase 3 4WARD trial for chronic neutropenia by Q3 or Q4 2025, with top-line data expected in the second half of 2026 [1]. - The 4WARD trial is now activated at approximately 90% of targeted sites worldwide [14]. - The ongoing Phase 3 clinical trial for mavorixafor may face delays or unsatisfactory outcomes, impacting future commercialization efforts [25]. - X4 Pharmaceuticals anticipates potential challenges in obtaining and maintaining regulatory approvals for mavorixafor, which could affect its market strategy [25]. Licensing and Partnerships - X4 entered into a licensing agreement with Norgine Pharma UK, receiving €28.5 million upfront and potential milestone payments of up to €226 million [6]. - The company recognized a gain of $105 million from the sale of a priority review voucher (PRV) in 2024 [20].

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]