A Study of IMR-687 in Adult Participants With Sickle Cell Anemia (Homozygous HbSS or Sickle-β0 Thalassemia)

A Phase 2a, Randomised, Double-Blind, Placebo-Controlled Study of IMR-687 in Adult Patients With Sickle Cell Anaemia (Homozygous HbSS or Sickle-β0 Thalassemia)

Study of IMR-687 in adult participants with sickle cell anemia (SCA) (homozygous HbSS or sickle-β0 thalassemia).

This is a proof-of-concept study in adult SCA participants, ages 18 to 55 years old, to examine the safety, tolerability, and pharmacokinetic (PK), as well as the potential pharmacodynamic (PD) effects and clinical efficacy, of IMR-687 across a range of doses.

IMR-687 was administered in 2 populations of participants with SCA: those who were not receiving hydroxyurea (HU) and those who were receiving a stable dose of HU according to standard of care.

Key Inclusion Criteria:

• Male or female participants with confirmed SCA
• Age 18 to 55 years, inclusive
• For participants on HU, must have been on a stable dose for at least 60 days prior to screening

Key Exclusion Criteria:

• Total hemoglobin >12.5 or <6 grams/deciliter
• Red blood cell transfusion within 60 days of baseline
• >7 hospitalizations for vaso-occlusive crises (VOCs) within the last year
• Estimated glomerular filtration rate <50 milliliter/minute
• Aspartate aminotransferase/alanine aminotransferase >3x the upper limit of normal