Financial Data and Key Metrics Changes - Research and development expenses for Q3 2025 were $14.3 million, a decrease from $14.6 million in Q3 2024, primarily due to reduced employee compensation costs and decreased costs associated with a discontinued program [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, including a dose-dependent increase in fetal hemoglobin and a reduction in vaso-occlusive crises [5][8] - Enrollment in the 20-milligram dose cohort was completed with 12 evaluable patients, and data will be presented at the upcoming ASH conference [6][7] - 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 [4] - The company estimates that about 20% of the U.S. sickle cell disease population meets the inclusion-exclusion criteria for the Phase 1B Pioneer trial [35] 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 [6][8] - 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] - The company is focused on expediting the drug's market entry to address the unmet needs of sickle cell disease patients globally [36] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the encouraging results from the 12-milligram cohort and the upcoming data readout at the ASH conference [12][13] - The management team highlighted the importance of generating additional safety data through the open-label extension study, which is expected to inform future discussions with the FDA [24][75] Other Important Information - The company has seen a high adherence rate of over 90% among patients in the study, indicating strong patient engagement with the treatment regimen [7] - The management team emphasized the need for a comprehensive approach to evaluating the drug's efficacy, considering multiple parameters such as fetal hemoglobin levels and reduction in vaso-occlusive crises [52] Q&A Session Summary Question: How does the company define a win in the context of the trial results? - Management believes they have already achieved a win with the 12-milligram cohort, showing robust increases in fetal hemoglobin and a favorable safety profile [16] Question: What are the expectations for the 20 mg dose cohort? - Management anticipates that the 20 mg cohort will outperform the 12 mg cohort based on previous healthy volunteer data [18] Question: What is the addressable market following the withdrawal of a competitor's product? - The company estimates that about 20% of the 100,000 U.S. patients with sickle cell disease currently meet the trial criteria, with plans to discuss potential market expansion with the FDA [35] Question: What metrics will be important for defining the activity of FTX-6058? - Management indicated that the totality of data, including fetal hemoglobin levels, pan-cellularity, and reduction in markers of hemolysis, will be crucial for evaluating the drug's efficacy [52] Question: What is the timeline for the final Pioneer data set? - The full data set is expected to be available in the first quarter of the following year [56]

Fulcrum Therapeutics (NasdaqGM:FULC) Q3 2025 Earnings Call October 29, 2025 08:00 AM ET Company ParticipantsAlan Musso - CFOIain Fraser - SVP of Clinical DevelopmentAlex Sapir - CEO and PresidentMatthew Biegler - Managing DirectorTazeen Ahmad - Managing DirectorConference Call ParticipantsCorinne Johnson - Equity Research AnalystAndres Maldonado - Senior Biotechology AnalystKristen Kluska - Equity Research AnalystEdward Tenthoff - Biotech Equity Research AnalystJoseph P. Schwartz - Senior Research AnalystLu ...

Fulcrum Therapeutics (NasdaqGM:FULC) Q3 2025 Earnings Call October 29, 2025 08:00 AM ET Speaker4Good morning and welcome to the Fulcrum Therapeutics third quarter 2025 financial results and business update conference call. Currently, all participants are in a listen-only mode. This call is being webcast live and can be accessed on the investor section of Fulcrum's website at www.fulcrumtx.com and is being recorded. Please be reminded that remarks during this call may contain forward-looking statements withi ...