Fulcrum Therapeutics(FULC) - 2023 Q2 - Earnings Call Transcript

Financial Data and Key Metrics Changes - The company ended June 30, 2023, with cash, cash equivalents, and marketable securities of $278.2 million, an increase from $202.9 million as of December 31, 2022, indicating a strong financial position [36] - Collaboration revenue for Q2 2023 was $0.9 million, down from $1.9 million in Q2 2022 [36] - Research and development expenses decreased to $17.8 million in Q2 2023 from $25 million in Q2 2022, primarily due to a milestone payment to GSK in the previous year and reduced costs from the clinical hold [37] - General and administrative expenses were $10.3 million in Q2 2023, down from $11.1 million in Q2 2022, attributed to lower professional services costs [38] - The net loss for Q2 2023 was $23.8 million, compared to $34.1 million in Q2 2022, reflecting improved financial performance [38] Business Line Data and Key Metrics Changes - The REACH study for losmapimod, aimed at treating FSHD, has closed screening and expects to complete enrollment later this quarter, with top-line data anticipated in Q4 2024 [28] - The Phase 1b study for FTX-6058, an oral HbF inducer for sickle cell disease, is currently on clinical hold, with plans to resume dosing once the FDA's requirements are met [29][30] Market Data and Key Metrics Changes - The company is focused on underserved communities, having obtained an exclusive global license from CAMP4 Therapeutics for research into Diamond-Blackfan Anemia, expanding its pipeline [33] Company Strategy and Development Direction - The company aims to deliver groundbreaking therapies for rare genetic diseases, with a focus on advancing its clinical programs and strengthening its leadership team [34][35] - The priority is to reinitiate the Phase 1b study for FTX-6058 and to continue the REACH study for losmapimod, with a commitment to patient populations [19][32] Management's Comments on Operating Environment and Future Outlook - Management expressed encouragement regarding progress in the first half of 2023 and highlighted a strong cash position expected to fund operations into mid-2025 [12] - The company is optimistic about resolving the clinical hold on FTX-6058 and is engaged in productive discussions with the FDA [31][81] Other Important Information - The company appointed Alan Musso as Chief Financial Officer effective August 7, bringing valuable experience to the leadership team [35] Q&A Session Summary Question: Can you discuss the confidence in the one-year timeline for the FSHD study? - Management explained that the REACH study includes an MRI-based endpoint to evaluate muscle heterogeneity, which supports the confidence in the timeline [6] Question: What is the status of the clinical hold and the path forward? - Management confirmed that the FDA has not requested additional preclinical data to lift the hold, focusing instead on defining the patient population for the Phase 1b study [43][44] Question: What are the next updates expected regarding patients and healthy volunteers? - Management clarified that the priority is to return to the patient population for the Phase 1b study, with healthy volunteer studies proceeding in parallel as necessary [76] Question: Any updates on the DUX4 biomarker discussions with the FDA? - Management indicated that there have been no additional discussions regarding DUX4 as a biomarker in the REACH study [79]