- 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 Insights - Vir Biotechnology has announced significant partnerships and advancements in its pipeline, including a global collaboration with Astellas and promising Phase 1 data for its oncology program VIR-5500, which targets hard-to-treat cancers [2][4]. Pipeline Programs - The company is advancing several investigational therapies, including VIR-5500 for metastatic castration-resistant prostate cancer (mCRPC), with positive Phase 1 data indicating dose-dependent anti-tumor activity [4][7]. - The Phase 1 study of VIR-5818, targeting HER2, is ongoing, with response data expected in the second half of 2026 [4]. - The Phase 1 study of VIR-5525, targeting EGFR, continues to enroll participants as planned [5]. Chronic Hepatitis Delta (CHD) Program - Vir Biotechnology has licensed to Norgine the combination of tobevibart and elebsiran for treating CHD, aiming to reach patients globally [2][7]. - Phase 2 SOLSTICE data showed that the combination therapy achieved undetectable hepatitis delta virus RNA in 88% of participants at Week 96 [7]. - The ECLIPSE registrational program is designed to evaluate the safety and efficacy of tobevibart and elebsiran, with topline data from ECLIPSE 1 expected in Q4 2026 and from ECLIPSE 2 and 3 in Q1 2027 [21]. Financial Results - As of December 31, 2025, the company reported approximately $781.6 million in cash, cash equivalents, and investments, a decline of $29.1 million in Q4 2025 [9]. - Revenue for Q4 2025 was $64.1 million, a significant increase from $12.4 million in Q4 2024, primarily due to a $64.3 million license revenue from the Norgine agreement [10]. - The net loss for Q4 2025 was $(42.9) million, an improvement from a net loss of $(104.6) million in Q4 2024 [17]. Future Outlook - The company expects its cash reserves to fund operations into the second quarter of 2028, bolstered by the Astellas collaboration and equity investment [18]. - The company plans to initiate pivotal Phase 3 trials for its oncology programs in 2027, following the expansion of dose cohorts for VIR-5500 [7][8].

Summary of Vir Biotechnology FY Conference Call Company Overview - **Company**: Vir Biotechnology (NasdaqGS:VIR) - **Industry**: Biotechnology, focusing on infectious diseases and oncology - **Mission**: Harnessing the human immune system to combat diseases, including infectious diseases and cancer [2][3] Core Strategies and Pillars 1. **Hepatitis Delta Program**: - Aiming to deliver a transformational therapy for chronic hepatitis Delta, with a significant commercial opportunity in the U.S. and other regions [3][4] - Registration program for Hepatitis Delta is underway, with potential regulatory review expected in 2027 [4][16] - Estimated 174,000 viremic patients in key markets, with a global prevalence of about 7 million [6][9] 2. **T-cell Engagers for Oncology**: - Developing a clinical-stage lineup of dual-masked T-cell engagers targeting solid tumors, addressing high unmet needs [4][18] - Upcoming data on VIR-5500, a PSMA-targeting T-cell engager, expected in Q1 2026 [4][22] 3. **Discovery Engine**: - Focused on developing a pipeline of best-in-class preclinical T-cell engagers and cancer immunotherapies [5][30] - Utilizes a unique ProXTEN masking technology to enhance safety and efficacy of T-cell engagers [18][20] Key Data and Results - **Hepatitis Delta**: - Combination therapy of Tobevibart (monoclonal antibody) and elebsiran (siRNA) shows promising results, with 88% of patients achieving undetectable HDV RNA at 96 weeks [12][14] - Combination therapy demonstrated significant reductions in HBV surface antigen levels, critical for controlling the delta virus [13][15] - **Oncology**: - VIR-5500 has shown favorable efficacy and safety profiles in early trials, with low rates of cytokine release syndrome [22][25] - VIR-5818 (HER2-targeted) and VIR-5525 (EGFR-targeted) are also in development, with promising early efficacy signals [23][24] Financial and Strategic Partnerships - Entered a commercial license agreement with Norgine, including an upfront payment of EUR 550 million and milestone payments, which will help fund the Eclipse program [16][17] - Retained commercialization rights in the U.S. and other markets outside Greater China, indicating a strategic focus on growth in these regions [17] Future Milestones - Anticipated top-line data for the Eclipse studies in Q4 2026 and Q1 2027, with plans for rapid marketing authorization submission [31][32] - Continued focus on advancing the T-cell engager pipeline, with updates expected in early 2026 [32][33] Challenges and Considerations - Manufacturing scaling remains a focus, with expertise inherited from previous assets aiding in this process [39][40] - The company is not currently seeking additional opportunities in hepatitis B, focusing instead on existing programs [38] Conclusion Vir Biotechnology is positioned to make significant advancements in the treatment of hepatitis Delta and various cancers through its innovative therapies and strategic partnerships, with a strong pipeline and upcoming data expected to drive future growth and value creation [33]

Core Viewpoint - Vir Biotechnology has entered into an exclusive licensing agreement with Norgine Pharma UK Limited for the commercial rights to the combination treatment of tobevibart and elebsiran for chronic hepatitis delta (CHD) in Europe, Australia, and New Zealand, which is expected to enhance the commercialization efforts and extend the company's financial runway into Q4 2027 [1][2][5]. Financial Terms - Vir Biotechnology will receive an initial reimbursement payment of EUR 55 million and is eligible for up to EUR 495 million in clinical, regulatory, and sales milestones, along with tiered royalties on net sales in the licensed territory [1]. - Norgine will contribute approximately 25% of the ongoing external clinical development costs for the ECLIPSE registrational program [1]. Product Development - The combination of tobevibart and elebsiran is being evaluated as a potentially first-of-its-kind therapy for CHD, utilizing a human monoclonal antibody and a small interfering RNA (siRNA) to disrupt the viral lifecycle [2]. - Enrollment for the ECLIPSE 3 trial has been completed, which is a Phase 2b head-to-head trial comparing the combination treatment with bulevirtide in bulevirtide-naïve patients [3]. Clinical Data - Data from the Phase 2 SOLSTICE trial indicated that 66% of participants receiving the combination treatment achieved hepatitis delta virus (HDV) RNA target not detected (TND) after 48 weeks, with a high proportion of TND observed in patients with cirrhosis [4]. Regulatory Designations - The combination treatment has received Breakthrough Therapy and Fast Track designations from the U.S. FDA, as well as Priority Medicines (PRIME) and orphan drug designations from the European Medicines Agency (EMA) [9]. Market Context - Chronic Hepatitis Delta (CHD) is recognized as the most severe form of chronic viral hepatitis, with no approved treatments currently available in the U.S. and limited options in the European Union and globally [10].

Financial Data and Key Metrics Changes - The company ended 2024 with just under $103 million in cash and cash equivalents, which is expected to support operations into the first half of 2026 [24] - Net revenues from XOLREMDI were reported at $1.4 million for the fourth quarter and $2.6 million for the full year 2024 [25] - R&D expenditures totaled $21.7 million for the fourth quarter and $81.6 million for the full year, while SG&A expenses were $15.1 million and $61.5 million respectively [25] - The net loss for the fourth quarter was $39.8 million, and for the full year, it was $37.5 million, reflecting a one-time sale of a priority review voucher for $105 million in May of the previous year [25][26] Business Line Data and Key Metrics Changes - XOLREMDI sales topped $2.5 million for the 7.5 months since its launch in mid-May 2024 [10] - The company has successfully engaged with top-tier immunologists and hematologists, which is expected to continue to increase patient finding and shorten the time to prescription [11] Market Data and Key Metrics Changes - The company has made significant progress in expanding the global reach of mavorixafor, with the EMA accepting the submitted MAA for review, potentially leading to approval as early as Q1 2026 [12] - The company entered into a partnership with Norgine for commercialization in Europe, Australia, and New Zealand, receiving EUR 28.5 million upfront and potential milestone payments of up to EUR 226 million [13] Company Strategy and Development Direction - The company aims to maximize the opportunity for mavorixafor in chronic neutropenia, which is viewed as a larger market opportunity [22] - A strategic restructuring is expected to decrease annual spending by about $30 million to $35 million, implemented to sharpen focus on chronic neutropenia [22] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the engagement with regulatory agencies, indicating a supportive environment for the trial design [32][34] - The company is focused on building awareness and education around WHIM syndrome and chronic neutropenia, with plans to pivot efforts towards the patient community [86] Other Important Information - The company has initiated a peer-to-peer speaker program to enhance physician education regarding WHIM syndrome [7] - The 4WARD trial for chronic neutropenia has been activated at about 90% of targeted sites worldwide, with expectations for full enrollment by Q3 or Q4 of this year [22] 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 is not expected to impact trial pace [32][33] Question: Inventory 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 [43][49] Question: Patient numbers and price increase for XOLREMDI - Management did not disclose patient numbers but confirmed a 7% price increase for the new year [54][56] Question: Commercialization experiences since launch - The company has successfully engaged physicians and identified additional patients, with ongoing efforts to build demand through conferences and education [70][71] Question: Expected patient numbers for WHIM and CN - Management reiterated the guidance of approximately 1,000 diagnosed WHIM patients in the U.S. and about 50,000 total chronic neutropenia patients, targeting the refractory severe end of that population [78]