Fulcrum Therapeutics(FULC) - 2022 Q1 - Earnings Call Transcript

Financial Data and Key Metrics Changes - The company ended Q1 2022 with $195.1 million in cash, cash equivalents, and marketable securities, which is expected to support operations into 2024 [32] - Collaboration revenue for Q1 2022 was $2.6 million, down from $4.8 million in Q1 2021, primarily due to reduced research services [33] - Research and development expenses increased to $17.8 million in Q1 2022 from $16.3 million in Q1 2021, while general and administrative expenses rose to $10.8 million from $5.5 million in the same period [34] - The net loss for Q1 2022 was $28.9 million, compared to a net loss of $17 million in Q1 2021 [35] Business Line Data and Key Metrics Changes - The company is advancing two clinical-stage assets: FTX-6058 for sickle cell disease and losmapimod for FSHD, with significant progress reported in Q1 2022 [9][10] - FTX-6058 is positioned as the only oral HbF inducer in development, with initial data expected to be shared at the European Hematology Association Congress [10][11] - Losmapimod is set to begin dosing patients in the Phase 3 REACH trial, which is the first-ever Phase 3 trial for FSHD [16] Market Data and Key Metrics Changes - Sickle cell disease affects an estimated 100,000 people in the U.S. and millions worldwide, highlighting a significant unmet medical need [26] - The company aims to address the unmet medical need for better treatment options for sickle cell disease through FTX-6058, which has shown potential for broad clinical benefits [27] Company Strategy and Development Direction - The company is focused on treating the root cause of rare genetic diseases and aims to establish itself as a leading rare disease company [23] - The strategic focus includes advancing clinical programs while leveraging the FulcrumSeek product engine to build out the pipeline [22] - The anticipated timing for the initiation of trials in non-sickle cell hemoglobinopathies has been shifted to the second half of the year to prioritize sickle cell disease [15] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential of FTX-6058 and losmapimod to provide transformative therapies for patients with significant unmet needs [30] - The company is optimistic about the upcoming data disclosures and the potential for these therapies to become standard of care [50] Other Important Information - The REACH trial for losmapimod is designed to evaluate its efficacy over a 48-week treatment period, with a primary endpoint of reachable workspace [16] - The company plans to nominate its next development candidate by the end of 2022 and submit its fourth IND by the end of Q1 2023 [22] Q&A Session Summary Question: Enrollment update for the first cohort of the 6058 Phase 1b trial - Management indicated that initial data from the first cohort would be shared at the upcoming EHA, with a focus on the primary analysis at one month [44] Question: Expectations for data at EHA - Management emphasized the goal of achieving a 5% to 10% absolute increase in HbF, which would be transformative for patients [50] Question: Enrollment expectations for the REACH trial - Management expressed confidence in a reasonable enrollment timeline for the REACH trial, leveraging previous experience from the ReDUX4 trial [61] Question: Data expectations for 6058 at EHA - Initial data from the 6 mg cohort will be presented, with treatment duration up to three months, aiming to show meaningful increases in HbF [66] Question: Confidence in starting pivotal trials for 6058 - Management stated that robust HbF increases would support transitioning into a registrational trial as early as possible in 2023 [80]