A Phase 2a clinical trial will evaluate an oral low-dose liquid carbon monoxide (CO) therapeutic candidate for the treatment of sickle cell disease.
RT’s Three Key Takeaways:
- First Patient Dosed: Hillhurst Biopharmaceuticals has initiated a Phase 2a clinical trial by dosing the first patient with HBI-002, an oral low-dose carbon monoxide therapy for sickle cell disease.
- Trial Focus and Goals: The open-label study will evaluate the safety, tolerability, biomarkers, and pharmacokinetics of HBI-002, with preliminary results expected by the end of 2025 to inform a larger Phase 2b trial.
- Urgent Treatment Need: HBI-002 aims to address the significant unmet need in managing painful vaso-occlusive crises in sickle cell patients, who face serious health risks and reduced life expectancy.
The first subject has been dosed in a Phase 2a clinical trial evaluating an oral low-dose liquid carbon monoxide (CO) therapeutic candidate, HBI-002 for the treatment of sickle cell disease, according to Hillhurst Biopharmaceuticals Inc.
“Dosing the first subject marks a significant milestone,” said co-founder and Chief Executive Officer Andrew Gomperts. “There remains a substantial unmet medical need in preventing the painful vaso-occlusive crises experienced by sickle cell patients. We are hopeful that our novel drug product can provide meaningful relief for patients and their families. We look forward to advancing with this trial and expect the first readout by the end of 2025.”
The Phase 2a clinical trial for HBI-002, an oral low-dose carbon monoxide (CO) therapeutic candidate, is an open-label study designed to assess the safety and tolerability of HBI-002 in patients with sickle cell disease. The trial will also collect biomarker and pharmacokinetic data, providing preliminary insights into the investigational therapy’s potential efficacy. Data from this study will inform the design of the larger Phase 2b clinical trial, planned to begin in 2026.
Sickle cell disease (SCD) is a genetic disorder that causes severe vaso-occlusive pain crises and is associated with serious clinical consequences, including stroke, heart disease, kidney disease, and premature death. The average life expectancy of SCD patients in the U.S. is only 45 years, underscoring the urgent need for more effective treatments.
HBI-002 is Hillhurst’s lead product candidate, an oral low-dose carbon monoxide (CO) investigational drug designed for chronic use in a home setting by patients with sickle cell disease. Other potential indications for HBI-002 include conditions associated with inflammation and cell death, such as Parkinson’s disease. A Phase 1 clinical study in healthy subjects under an Investigational New Drug (IND) application has been successfully completed.
