Financial Data and Key Metrics Changes - The company ended Q1 2025 with just under $90 million in cash and cash equivalents, sufficient to support operations into the first half of 2026 [18] - Net revenues from ZOLREMD were just under $1 million for Q1 2025, bringing cumulative sales since the May 2024 launch to approximately $3.5 million [18] - R&D expenditures totaled $18.5 million, while SG&A expenses were $15 million for the first quarter [18] - The company reported a small net income due to $28 million in license and other revenue from the partnership with Norgene and a gain of $10.8 million on outstanding Class C warrants [19] Business Line Data and Key Metrics Changes - Cumulative sales of ZOLREMD reached $3.5 million since the mid-May launch, with Q1 sales slightly lower than Q4 due to inventory resupply timing [11][12] - The company is advancing the MAVERICK-four trial for chronic neutropenia, with over 90% of targeted global trial sites activated and enrollment ongoing in over 20 countries [7][10] Market Data and Key Metrics Changes - Approximately 50,000 people are diagnosed with chronic neutropenia in the U.S., with an estimated 15,000 individuals experiencing high unmet needs [6][29] - The company is focusing on a high unmet need patient population for the MAVERICK-four trial, targeting those with moderate to severe chronic neutropenia [7][10] Company Strategy and Development Direction - The company aims to expand the global potential of MAVERICK-four for chronic neutropenia and has received a notice of allowance from the U.S. Patent Office for its application [10][11] - Partnerships with Norgene and Taberare have been established to commercialize MAVERICK in Europe, Australia, New Zealand, and the MENA region [15] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in delivering upcoming milestones in chronic neutropenia and highlighted the potential market opportunity of $1 billion to $2 billion in the U.S. alone [11] - The company anticipates full enrollment in the FORWARD trial by Q3 or Q4 2025, with top-line data expected in the second half of 2026 [10] Other Important Information - The company completed a one-for-thirty reverse stock split to address NASDAQ listing rules compliance [19] - The FORWARD trial is designed to demonstrate statistically significant increases in ANC response and decreases in annualized infection rates [8][10] Q&A Session Summary Question: Visibility into patient types in the study - Management noted that there is a clear demand for more patients to be included in the study, with many principal investigators eager to enroll additional patients [21][22] Question: Patient demographics for chronic neutropenia - The 50,000 diagnosed patients and 15,000 with high unmet needs are U.S. figures based on ICD-10 code analysis [29] Question: Label definition for the target population - The company anticipates a broad label for the entire chronic neutropenia population, supported by data from previous studies [31][33] Question: Commercialization agreement with Norgene - The agreement covers both WHIM and chronic neutropenia indications [34] Question: Patient compliance and persistency - Management reported higher than expected compliance and adherence rates for ZOLREMD, indicating strong patient engagement [38] Question: Patient prescription sizes - Most patients are currently receiving a month's supply of medication [42] Question: Patient dropout assumptions in the FORWARD trial - Management confirmed that the enrolled subjects' profiles are on track, and they are seeing the expected event rates early in the study [44]

Financial Data and Key Metrics Changes - The company ended Q1 2025 with just under $90 million in cash and cash equivalents, sufficient to support operations into the first half of 2026 [18] - Net revenues from ZOLREMD were just under $1 million for Q1 2025, bringing cumulative sales since the May 2024 launch to approximately $3.5 million [18] - R&D expenditures totaled $18.5 million, while SG&A expenses were $15 million for the first quarter [18] - The company reported a small net income due to $28 million in license and other revenue from the partnership with Norgene and a gain of $10.8 million on outstanding Class C warrants [18] Business Line Data and Key Metrics Changes - Cumulative sales of ZOLREMD reached $3.5 million since its launch, with Q1 sales slightly lower than Q4 due to inventory resupply timing [12][18] - The FORWARD trial for MAVERICK-four in chronic neutropenia is currently enrolling participants in over 20 countries, with more than 90% of targeted global trial sites activated [7] Market Data and Key Metrics Changes - Approximately 50,000 people are diagnosed with chronic neutropenia in the U.S., with an estimated 15,000 individuals experiencing high unmet needs [6] - The company anticipates a potential market opportunity of $1 billion to $2 billion in the U.S. for MAVERICK-four in chronic neutropenia [12] Company Strategy and Development Direction - The company is focused on advancing clinical trials for MAVERICK-four in chronic neutropenia and expanding the commercialization of ZOLREMD for WHIM Syndrome [5][12] - Partnerships with Norgene and Taberare aim to enhance the global reach of MAVERICK-four and ZOLREMD in Europe, Australia, New Zealand, and the MENA region [15] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving upcoming milestones in chronic neutropenia and emphasized the importance of long-term patent protection for MAVERICK-four [12] - The company is optimistic about the potential approval from the EMA for MAVERICK-four in WHIM Syndrome as early as Q1 2026 [14] Other Important Information - The company completed a one-for-thirty reverse stock split to address deficiencies with NASDAQ listing rules [19] - The FORWARD trial is designed to demonstrate statistically significant increases in ANC response and decreases in annualized infection rates [11] Q&A Session Summary Question: Visibility into patient types in the study - Management noted that there is a clear demand for including more patients in the study, with many principal investigators eager to enroll additional patients [21] Question: Patient diagnosis numbers - The 50,000 diagnosed patients and 15,000 with high unmet needs are specific to the U.S. based on ICD-10 code analysis [27] Question: Target population for the label - The company aims for a broad label that includes moderate and severe patients, supported by data from previous studies [29] Question: Commercialization agreement with Norgene - The agreement covers both WHIM and chronic neutropenia indications [32] Question: Patient compliance and persistency - Compliance rates are reportedly higher than expected for a daily oral medication, indicating a strong unmet need [36] Question: Patient prescription sizes - Most patients are currently receiving a month's supply of medication [39] Question: Patient dropout assumptions in the FORWARD trial - Management confirmed that the enrolled subjects' profiles are balanced and aligned with the trial's objectives, indicating confidence in the enrollment timeline [42]