X4 Pharmaceuticals(XFOR) - 2023 Q2 - Earnings Call Transcript

Financial Data and Key Metrics Changes - X4 Pharmaceuticals raised approximately $65 million in gross proceeds through a PIPE financing during the quarter, coinciding with positive Phase III results for WHIM [11] - The company reported cash, cash equivalents, restricted cash, and marketable securities totaling $142.3 million as of June 30, 2023, which, along with a $22.5 million drawdown from a debt facility, extends the cash runway into 2025 [13] - X4 was added to the Russell 3000 Index during its annual reconstitution in late June [12] Business Line Data and Key Metrics Changes - The company is focused on the chronic neutropenia program, particularly the development of Mavorixafor, which is positioned to be the first oral treatment option for chronic neutropenia and WHIM syndrome [14][15] - Emerging data from the Phase 2 trial of Mavorixafor showed robust increases in absolute neutrophil counts (ANC) and the potential for reducing G-CSF therapy [24][30] Market Data and Key Metrics Changes - Market research indicates that approximately 50,000 people in the U.S. are diagnosed with chronic neutropenia, with a significant unmet need identified among those experiencing severe symptoms [16][18] - The initial target population for Mavorixafor is estimated to be around 15,000 individuals with high unmet needs, which could expand with further market research [20][32] Company Strategy and Development Direction - X4 Pharmaceuticals is preparing for a Phase III clinical trial in chronic neutropenia, expected to initiate in the first half of 2024, following positive feedback from the FDA [31][36] - The company aims to deliver a meaningful oral treatment option first for WHIM syndrome and subsequently for chronic neutropenia, with plans for potential label expansion to other immunodeficiencies [69] Management's Comments on Operating Environment and Future Outlook - Management expressed excitement about the progress made in the chronic neutropenia program and the potential for Mavorixafor to address significant unmet needs in the patient population [35][36] - The company is optimistic about the upcoming NDA submission for Mavorixafor and the anticipated positive reception from the FDA based on the clinical efficacy demonstrated [54][65] Other Important Information - X4 received an additional patent for Mavorixafor, extending protection through December 2038 [9] - The company announced the appointment of Dr. Christophe Arbet-Engels as the new Chief Medical Officer, expected to bring valuable experience to the team [9] Q&A Session Summary Question: Can you remind us within the Phase 2 CN protocol, if there's a threshold ANC at which the investigator makes the GCSF tapering decision? - The protocol allows for tapering if ANC exceeds 10,000, but physicians can decide on tapering at other levels [40] Question: What's your level of confidence that the ANC will remain comfortably above the normal range? - There is optimism based on initial data, but long-term follow-up is necessary to confirm sustained results [42] Question: How many patients have you enrolled to date? - More than three patients have been enrolled, with ongoing data collection expected to provide a comprehensive picture later in the year [45] Question: How do the findings from the real-world analysis help in terms of Phase 3 protocol design? - Insights from the analysis have informed the understanding of unmet needs and will guide the Phase III trial preparations [49] Question: What remains to be done for the NDA submission? - The NDA is in the final stages of quality control and publishing, with readiness for potential FDA inspections [54] Question: Will the NDA package include recent infection data? - All relevant data, including recent findings, will be included in the NDA submission [64]