X4 Pharmaceuticals(XFOR) - 2022 Q3 - Earnings Call Transcript

Financial Data and Key Metrics Changes - The company is preparing for a potential NDA submission in early second half of 2023, with a commercial launch of mavericksphore anticipated in the first half of 2024 [24][32] - The average pretreatment absolute neutrophil count (ANC) for patients enrolled in the phase three trial was 220 cells per microliter, indicating severe neutropenia [12][13] - The company expects to announce phase three top line data in the fourth quarter of 2022 [14][20] Business Line Data and Key Metrics Changes - The phase three trial for mavericksphore in WHIM syndrome has completed enrollment with 31 patients from 12 countries, exceeding initial enrollment targets [12][14] - Over 90% of eligible participants from the placebo-controlled portion of the study opted to continue into the open-label extension study [14] Market Data and Key Metrics Changes - The company estimates that there are approximately 50,000 chronic neutropenia patients in the U.S., with mavericksphore potentially addressing a significant unmet need [7][41] - The company is focusing on the top five to seven European markets for the initial launch of mavericksphore following U.S. approval [24] Company Strategy and Development Direction - The company aims to establish mavericksphore as the first treatment specifically developed for WHIM syndrome, with plans to expand its use to other chronic neutropenic disorders [22][32] - The company is investing in activities to support education and awareness of WHIM syndrome, including collaborations with experts and patient organizations [23] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming phase three trial results and the potential for mavericksphore to transform the treatment landscape for chronic neutropenia [33] - The company is actively engaging with the FDA to ensure alignment on study design and regulatory pathways for both WHIM and chronic neutropenia indications [20][56] Other Important Information - The company has secured conditional approval of a brand name with the FDA for mavericksphore, marking a significant step in the commercialization process [29] - The company has completed registration batches for manufacturing, ensuring a stable supply chain for the anticipated launch [29] Q&A Session Summary Question: What is the average ALC for patients pretreatment? - Management indicated that the average ALC is not readily available but will follow up with the information [36] Question: Have you seen the blinded infection rate in the trial? - Management confirmed that infection events are being monitored and are consistent with expectations [38] Question: How do you reconcile the 50,000 patient estimate with the previously mentioned 2,500 patients on chronic GCSF? - Management explained that the previous number was based on registry data and acknowledged the challenges in defining chronic use of GCSF [40][41] Question: What are the powering assumptions for the WHIM trial? - Management stated that the trial was powered based on phase two results, with a current power greater than 95% for the primary endpoint [45] Question: What is the latest estimate on the prevalence of WHIM? - Management remains confident in guiding to a prevalence of 1,000 or higher for WHIM syndrome [46] Question: What are the differences in patient populations between phase two and phase three? - Management noted that both populations required a clinical diagnosis of WHIM syndrome, with the main difference being the inclusion of pediatric patients in phase three [50] Question: What activities will occur between the top line data and NDA filing? - Management outlined that the phase three data will be analyzed and prepared for NDA submission, with ongoing regulatory discussions for chronic neutropenia [56]