Fulcrum Therapeutics(FULC) - 2020 Q1 - Earnings Call Transcript

Financial Data and Key Metrics Changes - The company entered Q1 2020 with $81.2 million in cash, cash equivalents, and marketable securities, which is expected to support operations into Q3 2021 [29] - Research and development expenses for Q1 2020 were $14.5 million, a decrease from $34.6 million in Q1 2019, primarily due to a one-time cost in the previous year [30] - General and administrative expenses increased to $5.1 million in Q1 2020 from $2.6 million in Q1 2019, attributed to increased personnel costs and public company operating expenses [31] Business Line Data and Key Metrics Changes - The FSHD program received orphan drug designation from both the U.S. FDA and the European Commission, supporting the advancement of losmapimod [9] - The ReDUX4 trial was amended to extend the treatment period from 24 to 48 weeks, allowing for a more robust data set [16][20] - The sickle-cell program, FTX-6058, is progressing towards IND filing, with plans to submit in the second half of 2020 and initiate a Phase 1 trial by year-end [28] Market Data and Key Metrics Changes - The estimated patient population for FSHD in the U.S. is between 16,000 to 38,000, with no approved drugs currently available [12] - The company continues to advance its proprietary product engine, FulcrumSeek, to identify drug targets in genetically defined diseases [23] Company Strategy and Development Direction - The company aims to discover and develop therapeutics for genetically defined diseases, focusing on addressing root causes [6] - The strategy includes increasing fetal hemoglobin levels to develop therapies for sickle-cell disease and beta thalassemia [26] - The company is committed to maintaining high standards in patient safety and data integrity during clinical trials, especially in light of COVID-19 [14][20] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ability to adapt to challenges posed by COVID-19, emphasizing the commitment to patient safety and data collection [15][20] - The company anticipates reporting interim analysis data from the ReDUX4 trial in Q3 2020 and expects to report top-line data in Q1 2021 [18][32] Other Important Information - The company has made significant progress in its clinical candidate FTX-6058, with robust preclinical data supporting its efficacy [28] - The management highlighted the importance of flexibility in trial protocols to accommodate potential disruptions due to the pandemic [43][54] Q&A Session Summary Question: Concerns about power loss with interim analysis - Management believes the sample size of 80 subjects provides appropriate power for the trial, and the interim analysis will offer initial insights for Phase III planning [35][36] Question: Recruitment status for subjects - Recruitment has been completed, and management is confident they have all necessary subjects [37] Question: Biopsy completion amid pandemic concerns - Management has built flexibility into the protocol to ensure data collection at either 16 or 36 weeks, mitigating risks associated with potential patient dropouts [39] Question: Doubling duration of ReDUX protocol - The extension was made to provide flexibility for data collection over a longer period, accommodating the ongoing pandemic [42][43] Question: Insights from the open-label trial - The open-label trial has been impacted more significantly due to being a single-site study, but valuable learnings have been gained [46] Question: Powering assumptions and study length - Management clarified that the amendment does not impact power assumptions, and patient compliance has been high [50][52] Question: Integrity of biopsy readings at different time points - Studies indicate stability in the DUX4 gene signature, supporting the integrity of readings at both 16 and 36 weeks [53][54]