- Potential First and Only Therapy in the EU for Ultra-Rare Immune Disorder - BOSTON, Feb. 27, 2026 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company focused on improving the lives of people with rare hematology diseases, today announced the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending the granting of marketing authorization, under exceptional circumstances, of mavorixafor for the treatment of WHIM syndrome ...

Core Perspective - The company aims to be a leader in developing new therapies for patients with rare blood disorders, specifically focusing on mavorixafor for chronic neutropenia [2]. Group 1: Company Strategy - The management team has been in place for about 6 months, concentrating efforts on achieving the company's goals [1]. - Significant cost reductions and headcount decreases have been implemented to streamline operations [3]. - The primary focus is on successfully completing the Phase III trial to bring the new indication to patients [3].

Summary of X4 Pharmaceuticals Conference Call Company Overview - **Company**: X4 Pharmaceuticals (NasdaqCM:XFOR) - **Focus**: Development of new therapies for patients with rare blood disorders, specifically targeting chronic neutropenia with the compound mavorixafor [4][5] Core Points and Arguments Vision and Strategy - X4 Pharmaceuticals aims to be a leading company in developing therapies for rare blood disorders, focusing on mavorixafor for chronic neutropenia [4] - The company has reduced costs and headcount to concentrate on successfully completing the phase 3 trial of mavorixafor [4] Product Details: Mavorixafor - Mavorixafor is an oral therapy targeting CXCR4, with advantages over G-CSF, which is subcutaneous and has limitations such as causing bone pain and potential long-term leukemia risk [5][6] - Approximately 40% of chronic neutropenia patients currently use G-CSF, but many do not adhere to the treatment due to its side effects [5][6] Clinical Data and Validation - Mavorixafor is FDA approved for WHIM syndrome, an ultra-rare condition, but the company is not actively commercializing it due to cost concerns [7][8] - WHIM data indicates that mavorixafor can improve ANC (absolute neutrophil count) and reduce infections, supporting its potential efficacy in chronic neutropenia [9] Market Opportunity - There are an estimated 15,000 patients with symptomatic chronic neutropenia, with a target to treat about 5,000 patients initially [17][18] - The unmet medical need is significant, as many patients experience frequent infections despite current treatments [20] Phase 3 Study: 4WARD Trial - The 4WARD trial includes 176 patients randomized between mavorixafor and placebo, with eligibility requiring two infections in the past year [28] - The primary endpoints are an increase in ANC and a reduction in infection rates [28] - Enrollment is expected to be completed by the end of Q3 2026, with data anticipated in 2027 [48][49] Competitive Landscape - There are currently no known competitors in the CXCR4 space, making mavorixafor a unique offering [51] - The company is exploring potential expansions into secondary neutropenias and other indications beyond chronic neutropenia [52] Pricing and Market Access - There is potential for premium pricing due to the drug's benefits in reducing hospital admissions for infections, although formal pricing research has not been conducted [50] - The current pricing is unsustainable, and adjustments are expected [50] Future Milestones - Upcoming presentations to clarify market opportunities and data from the 4WARD trial are planned for later this year [59] - A small phase 2 study to explore G-CSF titration in combination with mavorixafor is also being considered [36] Additional Important Information - The company is focusing on patient recruitment for the clinical trial, utilizing AI and fieldwork to identify potential participants [44][45] - Safety data from the trial has shown no new signals, with gastrointestinal toxicity being the primary concern [43][40] This summary encapsulates the key points discussed during the conference call, highlighting the strategic direction, clinical development, market potential, and future plans of X4 Pharmaceuticals.

Summary of X4 Pharmaceuticals FY Conference Call Company Overview - **Company**: X4 Pharmaceuticals (NasdaqCM:XFOR) - **Focus**: Specializes in rare hematology diseases, particularly chronic neutropenia and WHIM syndrome [4][5] Key Points Management Transition - New management team joined X4 Pharmaceuticals three months prior, focusing on restructuring and financing [2] - Raised nearly $240 million to fund the phase 3 trial for chronic neutropenia [2] Lead Compound: Mavorixafor - **Indications**: Approved for WHIM syndrome, now targeting chronic neutropenia [9] - **Mechanism of Action**: Antagonizes CXCR4, facilitating the release of neutrophils from bone marrow into the bloodstream [7] - **Administration**: Oral agent, providing a competitive advantage over G-CSF, which requires injections and can cause discomfort [8] Market Opportunity - Estimated patient population for chronic neutropenia is approximately 15,000, significantly larger than the WHIM indication [5][12] - Current management of chronic neutropenia is inconsistent, with 40-45% of patients receiving G-CSF and 60% managed through other means [10][11] Clinical Trials - **Phase 2 Study**: Demonstrated that mavorixafor can increase ANC levels and can be safely combined with G-CSF [13][14] - **Phase 3 Study**: Double-blinded, placebo-controlled trial with 176 patients, focusing on ANC increase and reduction in infection rates [17][20] - Expected to complete enrollment by Q3 2026, with top-line data anticipated in the second half of 2027 [25] Competitive Landscape - Main competitor is G-CSF; no direct oral competitors currently exist [28] - Focus on generating combination data with G-CSF in the ongoing study [28] Commercialization Strategy - Plans to target a similar prescriber base as previous experiences with CTI and Bendeka [27] - Anticipates a premium pricing environment typical for rare diseases, though specific pricing strategies are still under development [27] Future Milestones - Completion of patient enrollment in the phase 3 trial is a critical upcoming milestone [29] - Ongoing efforts to better communicate market opportunities to investors [29] Collaborations - Strong relationship with Norjin for European rights, while focusing on the US market for commercialization [31] Additional Insights - The WHIM data collected from previous trials is being used to inform assumptions for the larger chronic neutropenia trial [9] - The company is restructuring to enhance trial enrollment efforts, particularly in the US [23][24]

Core Insights - X4 Pharmaceuticals will participate in the Guggenheim Second Annual Healthcare Innovation Conference from November 10-12, 2025, in Boston [1] - The management team will hold a fireside chat on November 10th at 8:30 a.m. ET and will be available for one-on-one meetings with investors [1] - A webcast of the fireside chat will be available on the company's website, with a replay accessible for 30 days post-event [2] Company Overview - X4 Pharmaceuticals focuses on developing and commercializing innovative therapies for patients with rare hematology diseases and significant unmet needs [3] - The company has developed mavorixafor, an orally available CXCR4 antagonist, currently marketed in the U.S. as XOLREMDI for its first indication [3] - X4 is conducting a global pivotal Phase 3 clinical trial (4WARD) for mavorixafor in patients with certain chronic neutropenic disorders [3]

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 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].

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]