HBI-002

SAN DIEGO, July 23, 2025 /PRNewswire/ -- Hillhurst Biopharmaceuticals Inc. ("Hillhurst"), a clinical-stage biopharmaceutical company focused on developing novel oral liquid drug products based on known inhaled therapeutics, today announced that the first subject has been dosed in its Phase 2a clinical trial evaluating HBI-002 for the treatment of sickle cell disease. "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. About Sickle Cell Disease: 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. About HBI-002: 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. About Hillhurst: Hillhurst is a clinical-stage company focused on its proprietary GLASS™ platform, which enables the development of novel drug products based on known inhaled therapeutics . The platform overcomes limitations of traditional inhaled delivery methods. This research is supported by the NHLBI under grant number 2R44HL131065-05. The content is solely the responsibility of the authors and does not necessarily represent the official views of the NIH. SOURCE Hillhurst Biopharmaceuticals, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

The intriguing finding that smokers did better than non-smokers in a clinical trial for an experimental cancer treatment led researchers to develop a drinkable, carbon monoxide-infused foam that boosted the effectiveness of the therapy, known as autophagy inhibition, in mice and human cells. The results suggest that therapeutic levels of carbon monoxide, delivered using gas-entrapping materials (GEMs), can increase the anti-cancer activity of autophagy inhibitors, which might have potential to improve therapies for many different cancers. Did smokers do better than non-smokers in a clinical trial for an experimental cancer treatment? That was the intriguing question that led University of Iowa researchers and their colleagues to develop a drinkable, carbon monoxide-infused foam that boosted the effectiveness of the therapy, known as autophagy inhibition, in mice and human cells. The findings were recently published in the journal Advanced Science. Looking for ways to exploit biological differences between cancer cells and healthy cells is a standard approach for devising new cancer treatments. But it is a painstaking process that requires a deep understanding of complex cancer biology and often a dose of unexpected insight. The potential of autophagy inhibitors Researchers have known for several decades that autophagy, which is the cell's natural recycling system, is increased in cancer cells relative to healthy cells, suggesting that inhibiting autophagy might be a way to target cancer cells. However, results from almost 20 clinical trials testing autophagy inhibitors have been inconclusive. "Within those clinical trials they found mixed results; there was some benefit, but for many patients there was no benefit, which really pushed researchers back to the drawing board," says James Byrne, MD, PhD, UI assistant professor of radiation oncology and biomedical engineering and senior author on the new study. Searching for insight into why autophagy inhibition only seems to work some of the time, the researchers made the surprising discovery that smokers in two of the previous trials of autophagy inhibitors seemed to do better than non-smokers. "When we looked at how the smokers did in those trials, we saw an increase in overall response in smokers that received the autophagy inhibitors, compared to (non-smoker) patients, and we also saw a pretty robust decrease in the target lesion size," Byrne says. This was an exciting finding for Byrne and his team because smoking is also associated with increased levels of carbon monoxide, a gas molecule that can increase autophagy in cells in a way that researchers think might enhance the anti-cancer effect of autophagy inhibitors. "We know also that smokers have higher carbon monoxide levels and while we definitely don't recommend smoking, this suggested that elevated carbon monoxide might improve the effectiveness of autophagy inhibitors. We want to be able to harness that benefit and take it into a therapeutic platform," says Byrne, who also is a member of University of Iowa Holden Comprehensive Cancer Center. Carbon monoxide boosts anti-cancer activity of autophagy inhibition The team already had just such a "platform" to test their ideas. Byrne specializes in crafting gas-entrapping materials (GEMs) -- foams, gels, and solids made from safe, edible substances that can be infused with different gas molecules. For this study, the researchers created a drinkable foam infused with carbon monoxide. When mice with pancreatic and prostate cancers were fed the carbon monoxide foam and simultaneously treated with an autophagy inhibitor, tumor growth and progression was significantly reduced in the animals. The team also showed that combining carbon monoxide with autophagy inhibitors had a significant anti-cancer effect in human prostate, lung, and pancreatic cancer cells in petri dishes. Ultimately, Byrne hopes to test this approach in human clinical trials. "The results from this study support the idea that safe, therapeutic levels of CO, which we can deliver using GEMs, can increase the anti-cancer activity of autophagy inhibitors, opening a promising new approach that might improve therapies for many different cancers," he says. In addition to Byrne, the research team included UI researchers Jianling Bi, Emily Witt, Megan McGovern, Arielle Cafi, Lauren Rosenstock, Lucas Absler, Srija Machkanti, Kellie Bodeker, Scott Shaw, Vitor Lira, and Michael Henry. The research team also included scientists from MIT, Harvard Medical School, University of Pennsylvania, Rutgers Cancer Institute of New Jersey, University of North Carolina Wilmington, and Oregon Health and Science University.