Cellarity has dosed the first subject in a Phase I study of oral medication, CLY-124, to treat sickle cell disease (SCD).
This development follows the US Food and Drug Administration’s (FDA) clearance of the company’s investigational new drug application.
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The global trial will evaluate the tolerability, pharmacokinetic, and safety profile of the therapy in healthy volunteers and then in individuals with SCD.
It aims to provide crucial data on foetal haemoglobin (HbF) production, which has been associated with minimising disease burden in SCD patients.
SCD leads to pain crises, chronic inflammation, and organ damage. Reactivation of HbF has been linked to alleviating these symptoms, with a 1% rise in HbF correlating to a 4%-6% decrease in pain crisis rates.
Cellarity chief medical officer Cameron Trenor said: “Initiation of our first-in-human clinical study for CLY-124 represents the delivery of our first clinical asset from our platform, with the potential for transformative improvement in care for individuals suffering from SCD.
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By GlobalData“The novel mechanism of CLY-124, which naturally increases HbF through Globin-Switching, could address pain, anaemia, and other symptoms to improve organ function and quality of life.
“Our strong body of preclinical evidence suggests CLY-124 may offer best-in-class HbF production without the cytotoxicity associated with conventional therapies, and importantly, could be accessible to all patients through a once-daily oral pill.”
CLY-124 was developed using the company’s drug discovery platform.
The company stated that it carried out an extensive analysis of single-cell transcriptomics during the production of red blood cells.
Unlike other treatments that directly attach to DNA or target transcription factors, the therapy claims to inhibit post-translational modification of an upstream protein complex.
Preclinical studies using human cell models demonstrated that the therapy could increase HbF levels above 20%, without cytotoxic effects.
