Fetal hemoglobin induction

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Fulcrum Therapeutics Announces Results from the 12 mg Dose Cohort of the Phase 1b PIONEER Trial of Pociredir in Sickle Cell Disease
Globenewswireยท 2025-07-29 10:45
Core Insights - Fulcrum Therapeutics reported positive results from the 12 mg dose cohort of the Phase 1b PIONEER trial for pociredir in sickle cell disease (SCD) [2][3] Efficacy Data - The mean absolute fetal hemoglobin (HbF) increased by 8.6% from a baseline of 7.6% to 16.2% after 12 weeks of treatment, with 7 out of 16 patients achieving HbF levels greater than 20% [4][10] - The proportion of F-cells increased from 34% at baseline to 67% at 12 weeks, indicating pan-cellular induction of HbF [4][10] - Key markers of hemolysis showed meaningful improvements, including a mean increase in total hemoglobin of 0.9 g/dL, from 7.8 g/dL to 8.7 g/dL [10] - A trend of reduced vaso-occlusive crisis (VOC) rates was observed, with 50% of patients reporting no VOCs during the treatment period [10] Safety Profile - Pociredir was generally well-tolerated, with no treatment-related serious adverse events (SAEs) reported, and all treatment-related adverse events (AEs) were Grade 1 [3][10] - The safety profile observed in the 12 mg dose cohort was consistent with previously reported data [10] Company Overview - Fulcrum Therapeutics is focused on developing small molecules for genetically defined rare diseases, with pociredir as its lead clinical program aimed at increasing HbF expression for SCD treatment [8][9]
Fulcrum Therapeutics(FULC) - 2025 Q1 - Earnings Call Transcript
2025-05-01 12:00
Financial Data and Key Metrics Changes - Research and development expenses decreased to $13.4 million in Q1 2025 from $19.8 million in Q1 2024, a reduction of $6.4 million attributed to the discontinuation of the losmapimod program and reimbursement under the Sanofi collaboration [10] - General and administrative expenses fell to $7 million in Q1 2025 from $10.1 million in Q1 2024, a decrease of $3.1 million primarily due to reduced employee compensation costs following workforce reductions [11] - Net loss improved to $17.7 million in Q1 2025 compared to a net loss of $26.9 million in Q1 2024 [11] - Cash, cash equivalents, and marketable securities decreased to $226.6 million as of March 31, 2025, from $241 million at the end of 2024, a decrease of $14.4 million mainly due to cash used for operating activities [11][12] Business Line Data and Key Metrics Changes - The lead program, coceridere, is currently enrolling in a phase 1b trial called PIONEER for sickle cell disease, with 16 patients enrolled in the 12 mg cohort [4][5] - The data monitoring committee recommended continuing the study and initiating the 20 mg cohort, which is now underway [5] - The company plans to report data from the 20 mg cohort by the end of 2025 [5] Market Data and Key Metrics Changes - Approximately 100,000 people in the US and 4.4 million people worldwide are affected by sickle cell disease [4] - Evidence suggests that even modest increases in fetal hemoglobin correlate with reduced disease severity, with a 1% increase in fetal hemoglobin leading to a 4% to 8% reduction in vaso-occlusive crises [7] Company Strategy and Development Direction - The company believes inducing fetal hemoglobin is the optimal strategy for treating sickle cell disease, supported by recent data analysis and approved gene therapies [6] - Plans to submit an IND for Diamond Blackfan anemia in Q4 2025, indicating a focus on expanding treatment options for inherited hematological conditions [8] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the progress made in 2025 and the upcoming data releases from the PIONEER trial [13] - The company expects existing cash reserves to fund operations into at least 2027, indicating a stable financial outlook [12] Other Important Information - The company has two abstracts accepted for presentation at the upcoming European Hematology Association meeting, highlighting ongoing research efforts [8] Q&A Session Summary Question: Data expectations from the twelve milligram cohort of PIONEER - Management confirmed that they will provide hematological parameters, including markers of hemolysis, and data for all 16 patients after a three-month treatment phase [15][16] Question: Observed dosing data and adherence - The company utilizes an AI tool to track adherence, reporting over 90% adherence to the once-daily oral regimen [19][21] Question: Baseline fetal hemoglobin levels and patient demographics - Management acknowledged that the baseline fetal hemoglobin levels were higher than expected, indicating a representative sample of the broader demographic [25][26] Question: Win criteria from the twelve-week data - Any increase in fetal hemoglobin is considered beneficial, with a clinically meaningful reduction in vaso-occlusive crises expected from increases in fetal hemoglobin [34][35] Question: Impact of FDA shifts on development - The company plans to interact with the FDA at the end of the phase 1b study to gauge regulatory perspectives on endpoint selection and development timelines [53][55]