Hillhurst Biopharmaceuticals Begins Phase 2a Trial for HBI-002 in Sickle Cell Disease

Hillhurst Biopharmaceuticals Inc., a clinical-stage biopharmaceutical company, has announced the dosing of the first subject in its Phase 2a clinical trial of HBI-002, a novel therapy aimed at treating sickle cell disease (SCD). The trial, which commenced on July 23, 2025, is designed to evaluate the safety and tolerability of HBI-002, an oral low-dose carbon monoxide (CO) therapeutic candidate, in patients suffering from this debilitating genetic disorder.

Andrew Gomperts, co-founder and CEO of Hillhurst, emphasized the importance of this milestone, stating, "Dosing the first subject marks a significant milestone. 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."

Sickle cell disease affects approximately 100,000 individuals in the United States, with patients experiencing severe vaso-occlusive pain crises that can lead to serious complications such as stroke, heart disease, and even premature death. According to the Centers for Disease Control and Prevention (CDC), the average life expectancy of individuals with SCD in the U.S. is only 45 years, highlighting the urgent need for effective treatments (CDC, 2022).

The Phase 2a trial is an open-label study that will not only assess the safety and tolerability of HBI-002 but will also gather biomarker and pharmacokinetic data, offering preliminary insights into the therapy's potential efficacy. The data collected during this trial will inform the design of a larger Phase 2b clinical trial, which is anticipated to begin in 2026.

HBI-002 is designed for chronic use in a home setting, providing a convenient option for patients with SCD. The drug is also being explored for other indications related to inflammation and cell death, including Parkinson’s disease. Having successfully completed a Phase 1 clinical study in healthy subjects, HBI-002 represents Hillhurst’s lead product candidate developed using its proprietary GLASS™ platform, which aims to overcome the limitations associated with traditional inhaled delivery methods (Hillhurst Biopharmaceuticals, 2023).

The development of HBI-002 is supported by the National Heart, Lung, and Blood Institute (NHLBI) under grant number 2R44HL131065-05. This funding underscores the potential significance of the research, although the content and findings are solely the responsibility of the authors and do not necessarily reflect the official views of the National Institutes of Health (NIH).

As the trial progresses, the medical community and patients alike will be watching closely for results that could pave the way for a new treatment paradigm in the management of sickle cell disease, a condition that has long been marked by inadequate therapeutic options. The implications of successful trial outcomes could not only alter the landscape of SCD treatment but also provide hope for those affected by this chronic illness.

In conclusion, Hillhurst Biopharmaceuticals’ Phase 2a clinical trial for HBI-002 highlights a promising advancement in the search for effective therapies for sickle cell disease. As researchers gather data on the efficacy and safety of this novel treatment, the potential to improve the quality of life for patients with SCD remains a motivating force in the ongoing battle against this severe genetic disorder.