Fulcrum Therapeutics (FULC) Investor Presentation - Slideshow

Company Overview - Fulcrum is a clinical-stage biopharmaceutical company focused on rebalancing gene expression to treat genetically defined diseases[4] - The company utilizes a systematic approach and its FulcrumSeek platform to identify small molecules that can modulate gene expression[4, 5] Pipeline Highlights - COVID-19 (Losmapimod): A Phase 3 trial (LOSVID) was initiated in Q3 2020[7, 8, 9] with topline data expected in Q1 2021[8, 17] The trial involves approximately 400 subjects randomized 1:1 to receive either 15mg of losmapimod or a placebo twice daily for 14 days[17] - FSHD (Losmapimod): Phase 2b ReDUX4 trial data is expected, with topline data in Q1 2021 and full data in Q2 2021[7, 8, 47] An interim analysis (IA) of the first 29 randomized subjects was conducted in Q3 2020[29] - Sickle Cell Disease (FTX-6058) & β-Thalassemia: An IND filing is planned for Q3 2020, with Phase 1 initiation in SCD expected in Q4 2020[7, 8] FTX-6058 has shown potent activity with less than 1 nM concentration and a clean off-target profile[50] Losmapimod for FSHD - The estimated US FSHD population is between 16,000 and 38,000, while the global population is estimated between 300,000 and 780,000[20] - Interim analysis of the ReDUX4 trial showed a 38-fold reduction in DUX4-driven gene expression in the losmapimod arm compared to a 5.4-fold reduction in the placebo arm in highest expressing muscle biopsies[35, 37] FTX-6058 for Hemoglobinopathies - FTX-6058 has demonstrated an absolute 8-18% increase in fetal hemoglobin (HbF) upon treatment, which could reduce mortality risk and recurring events in SCD patients[52] - The company's data indicates that small increases in HbF (1-5%) have the potential to provide clinical benefits to all SCD patients[52] Research and Discovery Collaborations - Fulcrum has research and discovery collaborations with Acceleron and MyoKardia, with potential milestone payments exceeding $800 million, plus upfront payments and R&D reimbursement[55, 56] - Fulcrum received a $10 million upfront payment from Acceleron and a $12.5 million payment at the close of the transaction with MyoKardia, along with reimbursement for relevant research expenses[55]