ReCode Therapeutics has initiated the third part of its Phase II clinical trial of the inhaled cystic fibrosis transmembrane conductance regulator (CFTR) messenger ribonucleic acid (mRNA) therapy RCT2100 combined with ivacaftor for cystic fibrosis (CF).
This stage of the study has received approval from the US Food and Drug Administration (FDA), with clinical sites in the country now operational.
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The ongoing open-label, multi-part Phase II trial will assess RCT2100’s biodistribution, safety, and tolerability via oral inhalation.
Part I evaluated single ascending doses in healthy participants, and Part II assessed multiple ascending doses in CF patients.
Part III will focus on the primary goals of tolerability and safety of RCT2100 co-administered with ivacaftor over six weeks in up to 12 CF patients.
Secondary outcomes include lung function and quality of life metrics. Data from this study will support the development and optimisation of the RCT2100 regimen.
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By GlobalDataReCode Therapeutics CEO Shehnaaz Suliman said: “Advancing the study of the combination of RCT2100 with ivacaftor represents an important milestone for the programme and for patients.
“This study will provide further insight into the potential additive benefits of RCT2100 when combined with ivacaftor on clinically meaningful measures such as lung function and patient-reported outcomes. Together with data generated from the other cohorts in this study, we are building a comprehensive dataset to inform our future development strategy.”
Furthermore, there are plans to initiate enrolment in the UK and the European Union in early 2026.
RCT2100 uses ReCode’s Selective Organ Targeting (SORT) lipid nanoparticle (LNP) platform to deliver CFTR mRNA to lung cells directly, aiming to restore function of the CFTR protein absent in some CF patients.
In January 2024, ReCode dosed the first subject in a Phase I clinical trial of its inhaled mRNA-based therapy, RCT1100, for primary ciliary dyskinesia (PCD) caused by dynein axonemal intermediate chain 1 (DNAI1) gene mutations.
