Hillhurst Biopharmaceuticals Inc, a clinical-stage biopharmaceutical company focused on developing novel oral liquid drug products based on known inhaled therapeutics, announced on Wednesday that the first subject has been dosed in its Phase 2a clinical trial evaluating HBI-002 for the treatment of sickle cell disease.

The open-label study is designed to assess the safety and tolerability of HBI-002, an oral low-dose carbon monoxide (CO) therapeutic candidate, 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.

"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."

HBI-002 is Hillhurst's lead product candidate, an oral low-dose 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.