FULC
Published on 05/01/2025 at 07:25
FULC May 2025
Unlocking the Power of Small Molecules to Change the Course of Genetically Defined Rare Diseases
Strategic Focus
Developing oral small molecules designed to modify gene
expression in rare diseases with a focus on benign hematology
Pociredir
Potential best-in class oral small molecule HbF inducer for sickle cell disease (SCD)
Fast Track and Orphan Designations
Planned timing for Phase 1b PIONEER data disclosure
Cohort 3 (12 mg): early Q3 20251
Cohort 4 (20 mg): YE 2025
Discovery & Cash Position
Advancing discovery programs for pipeline sustainability
IND submission planned in Q4 2025
Cash position of $226.6M as of 3/31/2025 with runway into at least 2027
3 HbF: Fetal hemoglobin
1Enrollment compete with 16 patients enrolled in Cohort 3
F UL CRUM T HE RA P E UT I CS
Indication
Asset / MOA
Preclinical
Phase 1
Phase 2
Phase 3
Collaborator
Clinical Programs
Sickle Cell Disease
Pociredir (HbF Induction)
Discovery Programs
DBA & Inherited Aplastic Anemias
Novel HbF Inducers
Fibrotic Disorders
DBA: Diamond-Blackfan anemia
4
f o r S i c k l e C e l l D i s e a s e
Fast Track Designation Orphan Drug Designation
Disclaimer
Fulcrum Therapeutics Inc. published this content on May 01, 2025, and is solely responsible for the information contained herein. Distributed via Public Technologies (PUBT), unedited and unaltered, on May 01, 2025 at 11:24 UTC.