Fulcrum Therapeutics : Corporate Presentation May 2025

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.