Core Viewpoint - X4 Pharmaceuticals has successfully closed a public offering of 52,844,000 shares, raising approximately $155.3 million to support its operations and development of therapies for rare hematology diseases [1][2]. Group 1: Offering Details - The public offering price was set at $2.90 per share, with an additional option for underwriters to purchase up to 6,984,000 shares [1]. - Pre-funded warrants were also offered at a price of $2.899, allowing the purchase of up to 700,000 shares at an exercise price of $0.001 per share [1]. - After the offering, the total shares outstanding increased to 79,214,708 [1]. Group 2: Underwriters and Registration - Leerink Partners, Stifel, and Guggenheim Securities served as joint bookrunning managers for the offering [2]. - A shelf registration statement for these securities was filed with the SEC on August 14, 2023, and became effective on August 24, 2023 [3]. Group 3: Company Overview - X4 Pharmaceuticals focuses on developing innovative therapies for patients with rare hematology diseases, particularly through its CXCR4 antagonist, mavorixafor [5]. - The company is currently marketing mavorixafor in the U.S. under the brand name XOLREMDI® and is conducting a pivotal Phase 3 clinical trial for chronic neutropenic disorders [5].

Core Viewpoint - X4 Pharmaceuticals has announced a public offering of 45,860,000 shares of common stock priced at $2.90 per share, aiming to raise approximately $135 million to fund the development of its drug mavorixafor and for general corporate purposes [1][2]. Group 1: Offering Details - The public offering includes pre-funded warrants for up to 700,000 shares at a price of $2.899 each, with an exercise price of $0.001 per share [1]. - The offering is expected to close on October 27, 2025, pending customary closing conditions [1]. - Underwriters have a 30-day option to purchase an additional 6,984,000 shares at the public offering price [1]. Group 2: Use of Proceeds - The net proceeds from the offering will be used to fund the pivotal Phase 3 development of mavorixafor for chronic neutropenic disorders, along with general administrative expenses, capital expenditures, and working capital [2]. Group 3: Company Background - X4 Pharmaceuticals focuses on developing therapies for rare hematology diseases, with mavorixafor being an orally available CXCR4 antagonist currently marketed in the U.S. as XOLREMDI® [5]. - The company is conducting a global Phase 3 clinical trial (4WARD) for mavorixafor in patients with chronic neutropenic disorders [5].

Core Viewpoint - X4 Pharmaceuticals has initiated an underwritten public offering of its common stock and pre-funded warrants, aiming to raise funds for the development of mavorixafor and other corporate purposes [1][2]. Group 1: Offering Details - The public offering includes an option for underwriters to purchase an additional 15% of the shares offered at the public offering price [1]. - The offering is subject to market conditions, and there is no guarantee regarding its completion or the terms [1]. Group 2: Use of Proceeds - The net proceeds from the offering will be allocated to the pivotal Phase 3 development of mavorixafor for chronic neutropenic disorders, as well as for general administrative expenses, capital expenditures, and working capital [2]. Group 3: Company Background - X4 Pharmaceuticals focuses on developing therapies for rare hematology diseases, with mavorixafor being a key product currently marketed in the U.S. as XOLREMDI® [5]. - The company is conducting a global pivotal Phase 3 clinical trial (4WARD) for mavorixafor in patients with chronic neutropenic disorders [5].

Core Viewpoint - X4 Pharmaceuticals (XFOR) has been upgraded to a Zacks Rank 1 (Strong Buy), indicating a positive outlook driven by rising earnings estimates, which significantly influence stock prices [1][3]. Earnings Estimates and Stock Price Movement - The Zacks rating system is based on changes in earnings estimates, which are closely correlated with near-term stock price movements, particularly due to institutional investors' reliance on these estimates for valuation [4][6]. - An increase in earnings estimates typically leads to higher fair value calculations by institutional investors, resulting in buying or selling actions that affect stock prices [4]. Recent Performance of X4 Pharmaceuticals - X4 Pharmaceuticals has seen a 61.3% increase in the Zacks Consensus Estimate over the past three months, reflecting a positive trend in earnings estimates [8]. - For the fiscal year ending December 2025, the expected earnings per share for X4 Pharmaceuticals is -$3.69, unchanged from the previous year [8]. Zacks Rank System - The Zacks Rank system classifies stocks into five groups based on earnings estimates, with Zacks Rank 1 stocks historically generating an average annual return of +25% since 1988 [7]. - The upgrade of X4 Pharmaceuticals to Zacks Rank 1 places it in the top 5% of Zacks-covered stocks, indicating strong potential for market-beating returns in the near term [10].

Core Insights - X4 Pharmaceuticals is undergoing a strategic restructuring that includes a 50% workforce reduction, expected to yield annualized cost savings of approximately $13 million [1][2][4] Corporate Updates - John Volpone has been appointed as Chief Operating Officer while retaining his role as President, overseeing daily operations and business execution [7] - Dr. Adam Craig, the Executive Chairman, will have oversight over clinical development [7] - The restructuring will lead to the exit of several key executives, including the Chief Legal & Compliance Officer, Chief Operating Officer, and Chief Commercial Officer [7] Clinical Development Focus - The restructuring aims to sharpen operational focus and align resources with the long-term strategy to successfully complete the 4WARD Phase 3 trial for chronic neutropenia [1][4] - X4 Pharmaceuticals is advancing mavorixafor, an orally available CXCR4 antagonist, in its clinical trials [5]

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)

Core Insights - X4 Pharmaceuticals presented positive data from its Phase 2 trial of mavorixafor for chronic neutropenia at the European Hematology Association Congress, indicating significant increases in circulating neutrophils and the potential to reduce G-CSF usage [1][2][3] Group 1: Clinical Trial Results - The Phase 2 trial was a six-month open-label study involving 23 participants with chronic neutropenia, assessing mavorixafor as a monotherapy and in combination with G-CSF [2][7] - Results showed that mavorixafor monotherapy durably increased mean absolute neutrophil count (ANC) from baseline, with severe chronic neutropenia patients achieving nearly three-fold increases in ANC levels [4][11] - 89% of participants and investigators opted to reduce or discontinue G-CSF while maintaining normal ANC levels, suggesting a potential transition to mavorixafor as an oral treatment option [4][11] Group 2: Ongoing and Future Trials - X4 is conducting a pivotal Phase 3 trial (4WARD) to evaluate mavorixafor's efficacy and safety in chronic neutropenia, aiming to enroll 150 participants and assess annualized infection rates and ANC response over 52 weeks [5][8] - The Phase 3 trial is expected to fully enroll in the third or fourth quarter of 2025, building on the encouraging Phase 2 results [2][5] Group 3: Mechanism and Background - Chronic neutropenia is characterized by low neutrophil levels, increasing infection risk and reducing quality of life; mavorixafor acts as a CXCR4 antagonist, mobilizing neutrophils from the bone marrow [6][9] - The Phase 1b/Phase 2 trial was designed to assess the safety and tolerability of mavorixafor, with the Phase 2 portion focusing on its impact on neutropenia over six months [7][8]

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].