Fulcrum Therapeutics (FULC) Investor Presentation - Slideshow

Fulcrum's Platform and Pipeline - Fulcrum's platform identifies targets with the potential to rebalance monogenic, polygenic, and heterogenous diseases[4] - The company's product engine has rapidly enabled two clinical programs: Losmapimod for FSHD and FTX-6058 for SCD and β-Thalassemia[7] - Fulcrum's pipeline includes multiple clinical programs advancing, with additional screens and FulcrumSeek planned for 2021[10] - Fulcrum is eligible to receive over $800 million in milestone payments plus upfront and R&D reimbursement from research and discovery collaborations[54] Losmapimod for FSHD - The estimated US FSHD population is 16,000-38,000, while the estimated global population is 300,000-780,000[13] - Interim analysis of the ReDUX4 trial showed a 38-fold reduction in DUX4 in the losmapimod arm compared to a 54-fold reduction in the placebo arm in highest expressing muscle biopsies[22] - The Phase 2b ReDUX4 trial (n=80) is a randomized, double-blind, placebo-controlled study with full data anticipated in Q2 2021[24] FTX-6058 for Sickle Cell Disease (SCD) and β-Thalassemia - Approximately 300,000 babies are born globally with a severe hemoglobin disorder per year[28] - An estimated 100,000 patients in the US and 50,000 patients in the EU are currently diagnosed with SCD[28] - FTX-6058 demonstrated a consistent 2-3 fold HbF induction across preclinical studies[40] - Phase 1 doses of FTX-6058 are projected to achieve maximal target engagement of approximately 30% of H3K27me3 mark[45]