Core Viewpoint - X4 Pharmaceuticals successfully closed an upsized private placement, raising $85 million to support the development of therapies for rare immune diseases, particularly focusing on mavorixafor for chronic neutropenia and WHIM commercialization [1][2]. Group 1: Financing Details - The private placement included 11,040,776 shares of common stock and pre-funded warrants for 48,852,772 shares, increasing from an initially announced $60 million offering to accommodate an additional investor [1]. - The offering was led by Coastlands Capital, with participation from notable investors such as Empery Asset Management, Bain Capital Life Sciences, and New Enterprise Associates [1]. Group 2: Use of Proceeds - The net proceeds from the financing will be utilized for the continued development of mavorixafor, aiming for additional approval in chronic neutropenia and supporting the commercialization of WHIM [2]. Group 3: Company Overview - X4 Pharmaceuticals focuses on developing innovative therapies for rare immune diseases, leveraging expertise in CXCR4 biology [4]. - The company has successfully marketed mavorixafor in the U.S. under the name XOLREMDI and is conducting a pivotal Phase 3 clinical trial (4WARD) for chronic neutropenic disorders [4].

Core Viewpoint - X4 Pharmaceuticals has announced a $60 million PIPE financing led by Coastlands Capital, with a new management team appointed to enhance the company's strategic direction and operational capabilities in the life sciences sector [1][2][3]. Financing Details - The PIPE financing is expected to yield gross proceeds of approximately $60 million, with the closing anticipated around August 13, 2025, subject to customary conditions [1]. - X4 will issue 11,040,776 shares of common stock at a purchase price of $1.42 per share, or pre-funded warrants for up to 31,234,731 shares at $1.419 each [5]. - The net proceeds will be utilized for the continued development of mavorixafor for chronic neutropenia and the commercialization of WHIM [6]. Management Changes - Paula Ragan, PhD, and Adam Mostafa have stepped down from their roles as President and CFO, respectively, with Dr. Adam Craig appointed as Executive Chairman, John Volpone as President, and David Kirske as CFO [2][7]. - The new management team brings extensive experience from CTI BioPharma, with a focus on operational turnarounds and strategic leadership in life sciences [2][7][8]. Strategic Focus - Dr. Adam Craig expressed enthusiasm for establishing a leading hematology company by unlocking the commercial potential of mavorixafor, with a clear pathway for potential approval in treating chronic neutropenia [3]. - The company is currently conducting a pivotal Phase 3 clinical trial (4WARD) for mavorixafor, which is crucial for its future growth and market positioning [3][10].

[PART I: FINANCIAL INFORMATION](index=7&type=section&id=PART%20I%3A%20FINANCIAL%20INFORMATION) [Item 1. Financial Statements](index=7&type=section&id=Item%201.%20FINANCIAL%20STATEMENTS) The company reports H1 2025 revenue of $30.8 million and a net loss of $25.5 million, citing substantial doubt about its going concern status [Condensed Consolidated Balance Sheets](index=7&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Key Balance Sheet Metrics | Financial Metric | June 30, 2025 (in thousands) | December 31, 2024 (in thousands) | | :--- | :--- | :--- | | Cash and cash equivalents | $39,220 | $55,699 | | Marketable securities | $23,731 | $46,361 | | Total current assets | $72,942 | $112,175 | | Total assets | $105,168 | $146,447 | | Total current liabilities | $22,896 | $32,877 | | Long-term debt, net | $75,841 | $75,425 | | Warrant liability | $283 | $13,755 | | Total liabilities | $101,197 | $124,298 | | Total stockholders' equity | $3,971 | $22,149 | [Condensed Consolidated Statements of Operations and Comprehensive (Loss) Income](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20(Loss)%20Income) Statements of Operations | Metric (in thousands) | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :--- | :--- | :--- | :--- | :--- | | Total revenue | $1,973 | $563 | $30,780 | $563 | | Research and development | $18,352 | $20,914 | $36,865 | $40,768 | | Selling, general and administrative | $9,527 | $13,278 | $24,548 | $30,713 | | Gain on sale of non-financial asset | $0 | $(105,000) | $0 | $(105,000) | | (Loss) income from operations | $(26,232) | $71,103 | $(35,675) | $33,814 | | Net (loss) income | $(25,741) | $90,833 | $(25,459) | $39,067 | | Net (loss) income per share: basic | $(3.47) | $13.59 | $(3.59) | $5.85 | [Condensed Consolidated Statements of Cash Flows](index=11&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Cash Flow Summary | Cash Flow Activity (in thousands) | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | $(42,269) | $(63,874) | | Net cash provided by investing activities | $19,974 | $91,540 | | Net cash provided by financing activities | $5,631 | $20,159 | | Net decrease in cash, cash equivalents and restricted cash | $(16,453) | $47,748 | | Cash, cash equivalents and restricted cash at end of period | $40,022 | $147,996 | [Notes to Condensed Consolidated Financial Statements](index=12&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) - Management has concluded there is **substantial doubt about the Company's ability to continue as a going concern** due to significant operating losses and potential debt covenant violations[38](index=38&type=chunk)[39](index=39&type=chunk) - The company executed a **1-for-30 reverse stock split** on April 28, 2025, with all share and per-share amounts retroactively adjusted[9](index=9&type=chunk)[45](index=45&type=chunk)[125](index=125&type=chunk) - A license agreement with Norgine resulted in the recognition of **$27.6 million in license revenue** and **$0.5 million for R&D services** in H1 2025[72](index=72&type=chunk)[81](index=81&type=chunk)[86](index=86&type=chunk) - The company anticipates it will not meet the **$15.0 million minimum cash covenant** required by its loan agreement, risking loan acceleration[39](index=39&type=chunk)[107](index=107&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=34&type=section&id=Item%202.%20MANAGEMENT'S%20DISCUSSION%20AND%20ANALYSIS%20OF%20FINANCIAL%20CONDITION%20AND%20RESULTS%20OF%20OPERATIONS) The company discusses the commercial launch of XOLREMDI, a strategic restructuring, and a going concern risk due to insufficient cash reserves [Overview](index=34&type=section&id=Overview) - The U.S. FDA approved **XOLREMDI (mavorixafor)** on April 29, 2024, for patients aged 12 and older with WHIM syndrome[166](index=166&type=chunk) - A strategic restructuring in February 2025 involved a **30% workforce reduction** to decrease annual spending by **$30-35 million**[173](index=173&type=chunk) - The company is progressing a global, pivotal **Phase 3 clinical trial (4WARD study)** for mavorixafor in people with chronic neutropenia[172](index=172&type=chunk) [Results of Operations](index=36&type=section&id=Results%20of%20Operations) Comparison of Six Months Ended June 30, 2025 and 2024 | Metric (in millions) | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | Change | | :--- | :--- | :--- | :--- | | Revenue | $30.8 | $0.6 | $30.2 | | Research and development | $36.9 | $40.7 | $(3.8) | | Selling, general and administrative | $24.6 | $30.7 | $(6.1) | | Gain on sale of non-financial asset | $0.0 | $(105.0) | $105.0 | | Net (loss) income | $(25.5) | $39.1 | $(64.6) | - The significant increase in revenue for H1 2025 was primarily due to recognizing **$27.6 million** from a license delivery under the Norgine Agreement[179](index=179&type=chunk)[180](index=180&type=chunk) - R&D expenses decreased by **$3.8 million** in H1 2025, mainly due to the strategic restructuring and lower spending on non-clinical programs[185](index=185&type=chunk) - SG&A expenses decreased by **$6.1 million** in H1 2025, driven by lower stock appreciation rights compensation, reduced launch costs, and lower headcount[187](index=187&type=chunk) [Liquidity and Capital Resources](index=38&type=section&id=Liquidity%20and%20Capital%20Resources) - The company has concluded there is **substantial doubt about its ability to continue as a going concern**, as its **$63.0 million** in cash is insufficient for the next 12 months[196](index=196&type=chunk)[203](index=203&type=chunk) - The company is at risk of violating the **Minimum Cash Covenant and Performance Covenant** of its Hercules Loan Agreement, which could accelerate its outstanding loans[197](index=197&type=chunk)[198](index=198&type=chunk) - Net cash used in operating activities was **$42.3 million** for H1 2025, a decrease from **$63.9 million** for the same period in 2024[199](index=199&type=chunk)[200](index=200&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=41&type=section&id=Item%203.%20QUANTITATIVE%20AND%20QUALITATIVE%20DISCLOSURES%20ABOUT%20MARKET%20RISK) As a smaller reporting company, it is not required to provide information on market risk - As a smaller reporting company, the company is **not required to provide** quantitative and qualitative disclosures about market risk[210](index=210&type=chunk) [Item 4. Controls and Procedures](index=41&type=section&id=Item%204.%20CONTROLS%20AND%20PROCEDURES) Management concluded disclosure controls and procedures were effective as of June 30, 2025, with no material changes in internal controls - Management concluded that the company's **disclosure controls and procedures were effective** as of June 30, 2025[212](index=212&type=chunk) - There were **no material changes** in internal control over financial reporting during the three months ended June 30, 2025[213](index=213&type=chunk) [PART II: OTHER INFORMATION](index=42&type=section&id=PART%20II%3A%20OTHER%20INFORMATION) [Item 1. Legal Proceedings](index=42&type=section&id=Item%201.%20LEGAL%20PROCEEDINGS) The company is not currently a party to any material legal proceedings - The company is **not currently a party** to any material legal proceedings[215](index=215&type=chunk) [Item 1A. Risk Factors](index=42&type=section&id=Item%201A.%20RISK%20FACTORS) Key risks include going concern uncertainty, reliance on a single product, and various operational, regulatory, and financial challenges [Risks Related to Our Financial Position and Need for Additional Capital](index=42&type=section&id=Risks%20Related%20to%20Our%20Financial%20Position%20and%20Need%20for%20Additional%20Capital) - The company's history of recurring losses raises **substantial doubt about its ability to continue as a going concern** and requires substantial additional funding[218](index=218&type=chunk) - The company anticipates it will **not be able to maintain the minimum cash required** to satisfy its debt covenant with Hercules for at least the next 12 months[223](index=223&type=chunk) - The company has an accumulated deficit of **$540.8 million** as of June 30, 2025, and expects to continue incurring losses[225](index=225&type=chunk) [Risks Related to Development of Our Product Candidates](index=46&type=section&id=Risks%20Related%20to%20Development%20of%20Our%20Product%20Candidates) - The company's business depends almost entirely on the successful development and commercialization of its lead product, **mavorixafor (XOLREMDI)**[241](index=241&type=chunk) - The commercial opportunity for mavorixafor in WHIM syndrome may be **smaller than anticipated**, which could adversely affect future revenue[239](index=239&type=chunk) - **Disruptions at the FDA** and other agencies could hinder the ability to develop or commercialize new products in a timely manner[246](index=246&type=chunk)[250](index=250&type=chunk) [Risks Related to the Marketing and Commercialization of Our Product Candidates](index=54&type=section&id=Risks%20Related%20to%20the%20Marketing%20and%20Commercialization%20of%20Our%20Product%20Candidates) - Approved products like XOLREMDI are subject to **extensive post-approval regulatory requirements** and could face marketing restrictions or withdrawal[277](index=277&type=chunk) - Commercial success is dependent on achieving **significant market acceptance** and securing adequate reimbursement from payors[285](index=285&type=chunk)[305](index=305&type=chunk) - The company faces **substantial competition** from major pharmaceutical and biotech companies with greater financial resources[292](index=292&type=chunk)[296](index=296&type=chunk) [Risks Related to Our Dependence on Third Parties](index=63&type=section&id=Risks%20Related%20to%20Our%20Dependence%20on%20Third%20Parties) - The company is dependent on a **single third-party manufacturer** for both the API and finished drug product of mavorixafor, posing a significant supply chain risk[324](index=324&type=chunk) - Reliance on third-party CROs for clinical trials means that **if these CROs fail to perform**, regulatory approval and commercialization could be harmed[328](index=328&type=chunk) [Risks Related to Our Intellectual Property](index=66&type=section&id=Risks%20Related%20to%20Our%20Intellectual%20Property) - Recent changes in U.S. patent laws create uncertainty and may **weaken the company's ability to obtain and enforce patents**[337](index=337&type=chunk)[338](index=338&type=chunk) - The company's competitive position could be harmed if it is **unable to maintain patent protection** for its technology and products[342](index=342&type=chunk)[345](index=345&type=chunk) - The company may become involved in **expensive and time-consuming lawsuits** to protect its intellectual property or defend against infringement claims[351](index=351&type=chunk)[359](index=359&type=chunk) [Risks Related to Our Business Operations, Employee Matters and Managing Growth](index=71&type=section&id=Risks%20Related%20to%20Our%20Business%20Operations%2C%20Employee%20Matters%20and%20Managing%20Growth) - The company's success depends on its ability to **retain key executives and personnel**, which may be challenging following the recent restructuring[373](index=373&type=chunk)[376](index=376&type=chunk) - The February 2025 restructuring, which **reduced headcount by 30%**, creates challenges in managing the scaled-down organization[377](index=377&type=chunk) - Internal IT systems are **vulnerable to cyber-attacks and data breaches**, which could lead to significant liabilities and operational disruption[380](index=380&type=chunk) [Risks Related to Ownership of Our Common Stock](index=74&type=section&id=Risks%20Related%20to%20Ownership%20of%20Our%20Common%20Stock) - Failure to maintain compliance with Nasdaq's minimum bid price requirement could lead to **delisting**[390](index=390&type=chunk)[393](index=393&type=chunk)[394](index=394&type=chunk) - The company's stock price is likely to **remain volatile**, and trading is subject to SEC "penny stock" rules which can limit liquidity[395](index=395&type=chunk)[398](index=398&type=chunk) - Provisions in the company's corporate charter and Delaware law could **make a potential acquisition more difficult**[412](index=412&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=79&type=section&id=Item%202.%20UNREGISTERED%20SALES%20OF%20EQUITY%20SECURITIES%20AND%20USE%20OF%20PROCEEDS) The company reports no unregistered sales of equity securities during the period - **None reported**[417](index=417&type=chunk) [Item 3. Defaults Upon Senior Securities](index=79&type=section&id=Item%203.%20DEFAULTS%20UPON%20SENIOR%20SECURITIES) The company reports no defaults upon senior securities - **None reported**[418](index=418&type=chunk) [Item 4. Mine Safety Disclosures](index=79&type=section&id=Item%204.%20MINE%20SAFETY%20DISCLOSURES) This item is not applicable to the company - **Not applicable**[419](index=419&type=chunk) [Item 5. Other Information](index=79&type=section&id=Item%205.%20OTHER%20INFORMATION) No directors or officers adopted, modified, or terminated any Rule 10b5-1 trading plans during the quarter - During the three months ended June 30, 2025, **no directors or officers adopted, materially modified, or terminated** any Rule 10b5-1 trading plans[420](index=420&type=chunk) [Item 6. Exhibits](index=80&type=section&id=Item%206.%20EXHIBITS) This section lists the exhibits filed with the Form 10-Q, including corporate documents and required officer certifications - The report includes a list of filed exhibits, such as the Certificate of Incorporation, By-laws, a Purchase Agreement, and required **CEO/CFO certifications**[421](index=421&type=chunk)

Poster #1654 highlights clinically meaningful and durable increases in circulating neutrophils observed with mavorixafor treatment +/- G-CSF over 6-month study Poster #1669 highlights findings that investigators were willing and able to reduce injectable G-CSF while maintaining ANC levels in the normal range Data continue to strengthen confidence in potential success of ongoing global, pivotal Phase 3 trial evaluating mavorixafor in primary chronic neutropenia BOSTON, June 16, 2025 (GLOBE NEWSWIRE) -- X4 Ph ...

Core Viewpoint - X4 Pharmaceuticals has received Fast Track designation from the FDA for mavorixafor, an oral CXCR4 antagonist, aimed at treating chronic neutropenia (CN), highlighting the company's commitment to addressing unmet medical needs in rare diseases of the immune system [1][2]. Company Overview - X4 Pharmaceuticals focuses on developing innovative therapies for rare immune system diseases, with mavorixafor being a key product currently marketed in the U.S. as XOLREMDI [6]. - The company is headquartered in Boston, Massachusetts, and is leveraging its expertise in CXCR4 and immune system biology to advance its therapeutic offerings [6]. Product Development - Mavorixafor is undergoing a global pivotal Phase 3 clinical trial (4WARD) to evaluate its efficacy, safety, and tolerability in patients with chronic neutropenia [5]. - The trial aims to enroll 150 participants and has a primary endpoint based on annualized infection rate and positive absolute neutrophil count (ANC) response [5]. Regulatory Milestones - The FDA's Fast Track designation facilitates more frequent communication with the agency and may lead to accelerated approval or priority review if certain criteria are met [2][3]. - Mavorixafor previously received Fast Track designation for the treatment of WHIM syndrome, which was approved by the FDA in April 2024 [1][2]. Market Context - Currently, the only FDA-approved treatment for chronic neutropenia is injectable human recombinant granulocyte-colony stimulating factor (G-CSF), which has significant side effects and risks associated with long-term use [2]. - Mavorixafor is positioned to offer a better balance of disease control and treatment burden compared to G-CSF, potentially benefiting patients with certain primary CN conditions [2].

Core Insights - X4 Pharmaceuticals announced the acceptance of two abstracts for presentation at the 30th Annual Congress of the European Hematology Association, highlighting positive results from a Phase 2 trial of mavorixafor for chronic neutropenia [1][2] - Mavorixafor is positioned as a potential first oral treatment for chronic neutropenia in over 30 years, with ongoing Phase 3 trials supporting its efficacy and safety [2][8] Company Overview - X4 Pharmaceuticals focuses on developing therapies for rare immune system diseases, with mavorixafor being a key product currently marketed in the U.S. as XOLREMDI® [9] - The company is headquartered in Boston, Massachusetts, and is actively pursuing additional indications for mavorixafor [9] Clinical Trial Details - The Phase 2 trial was a six-month open-label study involving 23 participants with various forms of chronic neutropenia, assessing the safety and efficacy of mavorixafor [3][7] - The ongoing pivotal Phase 3 trial, named 4WARD, aims to evaluate mavorixafor's efficacy and safety in a larger cohort of 150 participants with chronic neutropenia [8] Research Findings - The Phase 2 study demonstrated meaningful increases in functional neutrophils, with many participants able to reduce their G-CSF dosing when treated with mavorixafor [1][6] - Mavorixafor acts as a CXCR4 antagonist, mobilizing neutrophils from the bone marrow into the bloodstream, which is crucial for managing chronic neutropenia [5][6]

Mavorixafor Development and Commercialization - XOLREMDI® (mavorixafor) is approved and launched in the U S in May 2024 for WHIM syndrome, marking the first therapy for this condition[8, 32] - A Marketing Authorization Application (MAA) for WHIM syndrome has been submitted to and accepted by the EMA in January 2025, with potential EU approval and launch expected in the first half of 2026 via a partnership with Norgine[8] - The company anticipates full enrollment in the 4WARD trial for chronic neutropenia in the third or fourth quarter of 2025, with top-line data expected in the second half of 2026[8] Chronic Neutropenia (CN) Market and Unmet Needs - There are an estimated 50,000 chronic neutropenia patients in the U S , with approximately 15,000 representing a high unmet need and a minimum addressable market for mavorixafor[9, 49] - Currently, only injectable Granulocyte Colony-Stimulating Factor (G-CSF) is approved for severe chronic neutropenia, highlighting the need for innovation and alternative treatments[9, 10] - 67% of targeted WHIM hematologists in the U S would also be potential prescribers for chronic neutropenia if mavorixafor is approved for that indication[42] Clinical Trial Results and Analysis - Post-hoc analysis of the WHIM Phase 3 trial showed that 5 out of 13 mavorixafor-treated participants achieved the 4WARD "Responder" threshold, compared to 0 out of 16 in the placebo group[72] - In a Phase 2 study of mavorixafor in chronic neutropenia, mean Absolute Neutrophil Count (ANC) reached normal levels (≥ 1,500 cells/µL) at 3 and 6 months of treatment in the monotherapy group[59] - Physicians substantially reduced G-CSF dosage in 75% of eligible patients in the Phase 2 chronic neutropenia study, while maintaining normal mean ANC levels (>1,500 cells/µL)[68, 67] Financial Position - The company reported funds of $87 7 million as of March 31, 2025, which is expected to support operations into the first half of 2026[8]

Financial Data and Key Metrics Changes - The company ended Q1 2025 with just under $90 million in cash and cash equivalents, sufficient to support operations into the first half of 2026 [18] - Net revenues from ZOLREMD were just under $1 million for Q1 2025, bringing cumulative sales since the May 2024 launch to approximately $3.5 million [18] - R&D expenditures totaled $18.5 million, while SG&A expenses were $15 million for the first quarter [18] - The company reported a small net income due to $28 million in license and other revenue from the partnership with Norgene and a gain of $10.8 million on outstanding Class C warrants [19] Business Line Data and Key Metrics Changes - Cumulative sales of ZOLREMD reached $3.5 million since the mid-May launch, with Q1 sales slightly lower than Q4 due to inventory resupply timing [11][12] - The company is advancing the MAVERICK-four trial for chronic neutropenia, with over 90% of targeted global trial sites activated and enrollment ongoing in over 20 countries [7][10] Market Data and Key Metrics Changes - Approximately 50,000 people are diagnosed with chronic neutropenia in the U.S., with an estimated 15,000 individuals experiencing high unmet needs [6][29] - The company is focusing on a high unmet need patient population for the MAVERICK-four trial, targeting those with moderate to severe chronic neutropenia [7][10] Company Strategy and Development Direction - The company aims to expand the global potential of MAVERICK-four for chronic neutropenia and has received a notice of allowance from the U.S. Patent Office for its application [10][11] - Partnerships with Norgene and Taberare have been established to commercialize MAVERICK in Europe, Australia, New Zealand, and the MENA region [15] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in delivering upcoming milestones in chronic neutropenia and highlighted the potential market opportunity of $1 billion to $2 billion in the U.S. alone [11] - The company anticipates full enrollment in the FORWARD trial by Q3 or Q4 2025, with top-line data expected in the second half of 2026 [10] Other Important Information - The company completed a one-for-thirty reverse stock split to address NASDAQ listing rules compliance [19] - The FORWARD trial is designed to demonstrate statistically significant increases in ANC response and decreases in annualized infection rates [8][10] Q&A Session Summary Question: Visibility into patient types in the study - Management noted that there is a clear demand for more patients to be included in the study, with many principal investigators eager to enroll additional patients [21][22] Question: Patient demographics for chronic neutropenia - The 50,000 diagnosed patients and 15,000 with high unmet needs are U.S. figures based on ICD-10 code analysis [29] Question: Label definition for the target population - The company anticipates a broad label for the entire chronic neutropenia population, supported by data from previous studies [31][33] Question: Commercialization agreement with Norgene - The agreement covers both WHIM and chronic neutropenia indications [34] Question: Patient compliance and persistency - Management reported higher than expected compliance and adherence rates for ZOLREMD, indicating strong patient engagement [38] Question: Patient prescription sizes - Most patients are currently receiving a month's supply of medication [42] Question: Patient dropout assumptions in the FORWARD trial - Management confirmed that the enrolled subjects' profiles are on track, and they are seeing the expected event rates early in the study [44]

Financial Performance - The company reported revenue of $28.8 million for the three months ended March 31, 2025, compared to no revenue in the same period in 2024, marking a significant increase [164]. - The company reported a net income of $0.3 million for the three months ended March 31, 2025, a significant improvement from a net loss of $51.8 million in the same period in 2024 [164]. - Net income for the three months ended March 31, 2025 was $0.3 million, compared to a net loss of $51.8 million in the same period of 2024 [183]. - Other income for the three months ended March 31, 2025 increased by approximately $25 million compared to the same period in 2024, primarily due to a significant gain from the change in fair value of Class C warrants [174]. Expenses - The cost of revenue for the same period was $4.7 million, primarily due to amortization of an intangible asset and royalty payments [168]. - Research and development expenses decreased by $2 million to $18 million for the three months ended March 31, 2025, compared to $20 million in the prior year, mainly due to reduced pre-clinical drug candidate programs [171]. - Selling, general and administrative expenses also decreased by approximately $2 million to $15 million, attributed to lower compensation expenses and reduced commercialization launch costs [173]. - Net cash used in operating activities for the three months ended March 31, 2025 was $12.4 million, a decrease from $33.6 million in the same period of 2024 [184]. - Cash used in investing activities for the three months ended March 31, 2025 was $3.1 million, down from $5.3 million in the same period of 2024 [185]. Strategic Initiatives - The company completed a strategic restructuring in February 2025, resulting in a 30% reduction in workforce, which is expected to decrease annual spending by $30 to $35 million [159]. - The company is progressing with a pivotal Phase 3 clinical trial (the "4WARD" study") for mavorixafor, aiming to enroll 150 participants and complete enrollment by Q3 or Q4 2025 [157]. - The company launched XOLREMDI in the U.S. for WHIM syndrome and is actively engaging with physicians and patient advocacy organizations to promote awareness [152]. Financing and Capital Needs - The Hercules Loan Agreement allows for a term loan facility of up to $107.5 million, with $75.0 million borrowed to date [179]. - The company anticipates needing to raise additional capital to fund operations and satisfy covenants, which may include equity offerings and debt financings [182]. - The company has a Minimum Cash Covenant requiring a minimum cash level of $15.0 million, which is 20% of outstanding borrowings under the Hercules Loan Agreement [181]. - The company expects operating expenses in 2025 to be lower than those in 2024, excluding the sale of non-financial assets [188]. Shareholder Actions - A reverse stock split of 1-for-30 was approved by shareholders on April 17, 2025, to reduce the number of outstanding shares [162]. Cash Position - The company had cash, cash equivalents, and short-term marketable securities of $87.0 million as of March 31, 2025, with an accumulated deficit of $515.1 million [180]. - The company continues to maintain a valuation allowance against all remaining net deferred tax assets due to expected ongoing operating losses [176].

Financial Data and Key Metrics Changes - The company ended Q1 2025 with just under $90 million in cash and cash equivalents, sufficient to support operations into the first half of 2026 [18] - Net revenues from ZOLREMD were just under $1 million for Q1 2025, bringing cumulative sales since the May 2024 launch to approximately $3.5 million [18] - R&D expenditures totaled $18.5 million, while SG&A expenses were $15 million for the first quarter [18] - The company reported a small net income due to $28 million in license and other revenue from the partnership with Norgene and a gain of $10.8 million on outstanding Class C warrants [18] Business Line Data and Key Metrics Changes - Cumulative sales of ZOLREMD reached $3.5 million since its launch, with Q1 sales slightly lower than Q4 due to inventory resupply timing [12][18] - The FORWARD trial for MAVERICK-four in chronic neutropenia is currently enrolling participants in over 20 countries, with more than 90% of targeted global trial sites activated [7] Market Data and Key Metrics Changes - Approximately 50,000 people are diagnosed with chronic neutropenia in the U.S., with an estimated 15,000 individuals experiencing high unmet needs [6] - The company anticipates a potential market opportunity of $1 billion to $2 billion in the U.S. for MAVERICK-four in chronic neutropenia [12] Company Strategy and Development Direction - The company is focused on advancing clinical trials for MAVERICK-four in chronic neutropenia and expanding the commercialization of ZOLREMD for WHIM Syndrome [5][12] - Partnerships with Norgene and Taberare aim to enhance the global reach of MAVERICK-four and ZOLREMD in Europe, Australia, New Zealand, and the MENA region [15] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving upcoming milestones in chronic neutropenia and emphasized the importance of long-term patent protection for MAVERICK-four [12] - The company is optimistic about the potential approval from the EMA for MAVERICK-four in WHIM Syndrome as early as Q1 2026 [14] Other Important Information - The company completed a one-for-thirty reverse stock split to address deficiencies with NASDAQ listing rules [19] - The FORWARD trial is designed to demonstrate statistically significant increases in ANC response and decreases in annualized infection rates [11] Q&A Session Summary Question: Visibility into patient types in the study - Management noted that there is a clear demand for including more patients in the study, with many principal investigators eager to enroll additional patients [21] Question: Patient diagnosis numbers - The 50,000 diagnosed patients and 15,000 with high unmet needs are specific to the U.S. based on ICD-10 code analysis [27] Question: Target population for the label - The company aims for a broad label that includes moderate and severe patients, supported by data from previous studies [29] Question: Commercialization agreement with Norgene - The agreement covers both WHIM and chronic neutropenia indications [32] Question: Patient compliance and persistency - Compliance rates are reportedly higher than expected for a daily oral medication, indicating a strong unmet need [36] Question: Patient prescription sizes - Most patients are currently receiving a month's supply of medication [39] Question: Patient dropout assumptions in the FORWARD trial - Management confirmed that the enrolled subjects' profiles are balanced and aligned with the trial's objectives, indicating confidence in the enrollment timeline [42]