Fulcrum Therapeutics(FULC) - 2025 Q3 - Earnings Call Transcript

Financial Data and Key Metrics Changes - Research and development expenses for Q3 2025 were $14.3 million, a slight decrease from $14.6 million in Q3 2024, primarily due to reduced employee compensation costs and discontinued programs [10] - General administrative expenses decreased to $7.6 million in Q3 2025 from $8.4 million in Q3 2024, mainly due to lower professional services costs [10] - The net loss for Q3 2025 was $19.6 million, compared to a net loss of $21.7 million in Q3 2024 [11] - Cash, cash equivalents, and marketable securities at the end of Q3 2025 totaled $200.6 million, down from $241 million at the end of 2024, primarily due to cash used for operating activities [11] - The company expects its existing cash resources to fund operations into 2028, allowing for substantial progress in clinical development [11] Business Line Data and Key Metrics Changes - The lead program, FTX-6058, for sickle cell disease showed promising results, with a dose-dependent increase in fetal hemoglobin and a reduction in vaso-occlusive crises [4][8] - Enrollment in the 20-milligram dose cohort was completed, with data to be presented at the upcoming ASH conference [5][6] - The company is also advancing programs for bone marrow failure syndromes and plans to submit an IND for these conditions in Q4 2025 [9] Market Data and Key Metrics Changes - Approximately 100,000 people in the U.S. and 7.7 million worldwide suffer from sickle cell disease, indicating a significant market need for effective treatments [3] - The company estimates that about 20% of the U.S. sickle cell population meets the inclusion-exclusion criteria for the Phase 1B Pioneer trial [30] Company Strategy and Development Direction - The company aims to position FTX-6058 as a best-in-class once-daily oral therapy for sickle cell disease, with plans for an open-label extension trial to evaluate long-term safety and efficacy [5][21] - The strategy includes engaging with the FDA for an end-of-Phase 1 meeting in Q1 2026 to discuss the next stages of clinical development [8] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the encouraging results from the FTX-6058 program and the potential to provide differentiated therapeutic options for sickle cell disease patients [8][12] - The company is focused on expediting the drug's market entry to address the unmet needs of patients, especially following the withdrawal of other treatment options [31] Other Important Information - The company presented preclinical data for FTX-6274, an oral EED inhibitor, showing efficacy in prostate cancer models, indicating potential expansion beyond hematology programs [9] Q&A Session Summary Question: Insights on baseline characteristics of the 20 mg cohort - Management noted that the baseline characteristics of the 20 mg cohort are similar to previous cohorts, and they are analyzing the data to determine the dose response [14][15] Question: Interest in the open-label extension study - The decision to initiate the open-label extension study was influenced by patient and investigator feedback, allowing continued treatment for patients post-trial [20][21] Question: Addressable market assumptions - The company estimates that about 20% of the 100,000 U.S. patients with sickle cell disease meet the trial criteria, and they aim to expand treatment access [30][31] Question: Safety follow-up requirements from the FDA - The FDA has not specified numerical criteria for safety follow-up, but ongoing favorable safety profiles will be crucial for future discussions [64] Question: Future clinical plans beyond Phase 1B - The next study design will depend on discussions with regulators and may include a registrational trial focused on VOC reduction as a primary endpoint [65][66]