Fulcrum Therapeutics(FULC) - 2025 Q1 - Earnings Call Transcript

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 global development cost sharing reimbursement under the Sanofi collaboration [11] - 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 a workforce reduction [12] - Net loss improved to $17.7 million in Q1 2025 compared to a net loss of $26.9 million in Q1 2024 [12] - Cash, cash equivalents, and marketable securities decreased to $226.6 million as of March 31, 2025, from $241 million as of December 31, 2024, a decrease of $14.4 million mainly due to cash used for operating activities [12][13] 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 [5][7] - The data monitoring committee recommended continuing the study and initiating the 20 mg cohort, which is now underway [7] Market Data and Key Metrics Changes - Approximately 100,000 people in the US and 4.4 million people worldwide are afflicted by sickle cell disease, indicating a significant market opportunity for treatments targeting this condition [5] Company Strategy and Development Direction - The company aims to induce fetal hemoglobin as a strategy for treating sickle cell disease, supported by data showing that even modest increases in fetal hemoglobin correlate with reduced disease severity [8] - The company plans to submit an IND for Diamond Blackfan anemia in Q4 2025, indicating a focus on expanding its treatment portfolio for inherited hematological conditions [9][10] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the progress made in 2025 and looks forward to delivering important data releases from the PIONEER trial [14] - The company believes that the current operating plans will allow existing cash reserves to fund operations into at least 2027 [13] Other Important Information - The company has two abstracts accepted for presentation at the upcoming European Hematology Association meeting, highlighting ongoing research efforts [9] Q&A Session Summary Question: Data expectations from the twelve milligram cohort of PIONEER - Management confirmed that data will include hematological parameters and that all 16 patients will have data from the three-month treatment phase, with a subset having four-week follow-up data [16][17] Question: Adherence data and dosing timing - Management explained that adherence is monitored using an AI tool, with over 90% adherence reported, and that they can track the timing of doses taken by patients [19][20][22] Question: Representativeness of the patient sample - Management acknowledged that while the sample size is small, the baseline fetal hemoglobin levels were higher than expected, and they do not believe baseline levels will determine response to treatment [25][28] Question: Expectations for the twelve-week data - Management indicated that any increase in fetal hemoglobin is beneficial, with clinically meaningful reductions in vaso-occlusive crises expected from the data [34][36] Question: Enrollment dynamics and site selection - Management attributed faster enrollment to having the right sites on board and the loss of other treatment options, which increased patient interest in the trial [42][44] Question: FDA interactions and endpoint selection - Management plans to interact with the FDA at the end of the 20 mg cohort to discuss future study plans and endpoint selection [54][56]