Fulcrum Therapeutics, Inc. · NASDAQ:FULC
Fulcrum Therapeutics — Corporate Update (August 2023)
12 slides from Fulcrum Therapeutics's corporate update, Aug 22, 2023
Slide 1: FTX-6058 for Sickle Cell Disease Program Update
Exhibit 99.1 FTX-6058 f o r S i c k l e C e l l D i s e a s e Program Update
Slide 2: Disclaimer and Notice
F U L C R U M T H E R A P E U T I C S Disclaimer and Notice This presentation contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties. All statements, other than statements of historical facts,
Slide 3: Phase 1b Clinical Trial in SCD Subjects
F U L C R U M T H E R A P E U T I C S Phase 1b Clinical Trial in SCD Subjects Study Population Study Design Males and females with Cohort 1 (6 mg) 12-Week Treatment Period SCD, between age 18 – 65 Completed Cohort 2 (2mg) 12-Week Treatment Period Approximately 10 subjects per cohort Cohort 3 (12 mg)
Slide 4: Initial FTX-6058 Data Demonstrates Dose-dependent Increases in HbF
F U L C R U M T H E R A P E U T I C S Initial FTX-6058 Data Demonstrates Dose-dependent Increases in HbF Absolute %HbF Change from Baseline Safety Follow-up 12 2mg FTX-6058 10 6mg FTX-6058 8 12mg FTX-6058 6 4 2 0 0 14 28 42 56 70 84 98 112 Day U.S. FDA issued a full clinical hold for FTX-6058 on Feb
Slide 5: Overview of Key Inclusion and Exclusion Criteria
F U L C R U M T H E R A P E U T I C S Overview of Key Inclusion and Exclusion Criteria Key Inclusion Criteria Key Exclusion Criteria Patient Severity Exclude subjects currently on Previous experience with /having received the following hydroxyurea therapies within 60 days prior to initiating FTX-605
Slide 6: Overview of Key Inclusion Criteria: Patient Severity
F U L C R U M T H E R A P E U T I C S Overview of Key Inclusion Criteria: Patient Severity Other Measures of Severity VOC ≥2 over 12 months Chronic kidney ≥2 other ≥4 over 12 disease or and at least one measures of months or ≥2 other measure of pulmonary arterial severity over 12 over 6 months hyper
Slide 7: Overview of Key Inclusion Criteria: Previous Use of Hydroxyurea AND One Other Approved Therapy
F U L C R U M T H E R A P E U T I C S Overview of Key Inclusion Criteria: Previous Use of Hydroxyurea AND One Other Approved Therapy • Continued VOC or episodes of acute chest syndrome • Unmanageable drug-drug interactions Hydroxyurea for at least 6 months at the maximum tolerated dose • Patient ref
Slide 8: FTX-6058 for Sickle Cell Disease Supplemental Detail
FTX-6058 f o r S i c k l e C e l l D i s e a s e Supplemental Detail
Slide 9: Modifications to Key Inclusion Criteria: Patient Severity
F U L C R U M T H E R A P E U T I C S Modifications to Key Inclusion Criteria: Patient Severity ▪ ≥4 episodes of SCD pain crisis over 12 months or ≥2 over 6 months prior to screening ▪ ≥2 episodes of SCD pain crisis plus at least one of the following over previous 12 months: • Acute chest syndrome (
Slide 10: Modifications to Key Inclusion Criteria: Previous Use of Hydroxyurea and One Other Approved Therapy
F U L C R U M T H E R A P E U T I C S Modifications to Key Inclusion Criteria: Previous Use of Hydroxyurea and One Other Approved Therapy Previous experience with hydroxyurea (HU) for at least 6 months at Previous experience with a stable dose of voxelotor, crizanlizumab, the maximum tolerated dose
Slide 11: Summary of Exclusion Criteria
F U L C R U M T H E R A P E U T I C S Summary of Exclusion Criteria ▪ Major surgery, serious illness, infection, fever, significant bleeding, cerebrovascular accident, or seizure within 14 days prior to starting study drug; elective surgery planned for the time period of the trial ▪ Sickle cell comp
Slide 12: Summary of Exclusion Criteria (Continued)
F U L C R U M T H E R A P E U T I C S Summary of Exclusion Criteria (Continued) ▪ Subjects receiving regularly scheduled transfusions or any subject who has been transfused within 60 days prior to initiating study drug ▪ Clinically diagnosed substance use disorder for alcohol or other illicit drugs
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