ArticleCPT: pharmacometrics & systems pharmacology2026
Utilization of Model-Informed Drug Development in Early Clinical Development: Case Study of a Hb Modulator for Treatment of Sickle Cell Disease.
Article in CPT: pharmacometrics & systems pharmacology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
Model-informed drug development (MIDD) has an important role to play at various stages of drug development. In this case study, the application of MIDD in the early clinical development of a small-molecule inhibitor of sickle hemoglobin (Hb) polymerization is highlighted. Starting from the setting of quantitative criteria, to using a combination of PKPD and semi-mechanistic RBC modeling, and key biomarker analysis, based on data from a healthy participant first-in-human (FIH) study and historical data, we chart the strategic approaches planned and taken to inform decisions for the program, and the role of cross-functional collaboration. Based on the projections from the PKPD and semi-mechanistic RBC models, and key biomarker results from healthy participants, an informed decision was made to not proceed to a Phase 1b study in sickle cell disease (SCD) patients, underscoring the value of MIDD in early drug development decision making.
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