- 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 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 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 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]