When most people think of carbon monoxide, they envision odorless gas that can poison the body. For San Diego-based Hillhurst Biopharmaceuticals, Inc., carbon monoxide conjures a different vision – that of a healing therapeutic.
Since the company was founded a decade ago, Hillhurst has been developing a way to safely utilize carbon monoxide and other gases as drug therapies for a variety of indications – most notably, sickle cell anemia.
“It’s really a terrible disease,” said Hillhurst CEO Andrew Gomperts of the “debilitating” condition that mostly affects people of African descent that leads to premature organ damage and early death in many afflicted with the disease.
“For sickle cell, there are dugs available, they just don’t work very well,” he added.
Hillhurst hopes its novel GLASS platform (Gas in Liquid Advanced Stability System) can change than by delivering carbon monoxide to patients in a simple beverage.
Data Drives Founding
While the ability to store therapeutic gasses in a liquid beverage is novel, research into using carbon monoxide as a treatment for sickle cell is not.
In ‘60s and ‘70s researchers were looking at low doses of carbon monoxide as a treatment “and had some success, actually, with early clinical work,” Gomperts said. “But it was very difficult to deliver it on a regular basis because no one wants a cylinder of carbon monoxide in their home. Detectors in our homes are there for a reason.”
Because of the promising data on carbon monoxide therapy for sickle cell, different delivery methods for the gas were tried over the years, such as inhaling the gas from tanks and using hemoglobin as a carrier molecule, but both methods proved “difficult and problematic,” Gomperts said.
While researching carbon monoxide hemoglobin carriers at San Diego-based Sangart Inc., Andrew Gomperts mentioned the research to his father Edward Gomperts, MD, a hematologist at Children’s Hospital USC, who then thought up and developed the formulation for a drinkable carbon monoxide – the basis for the founding of Hillhurst. Andrew joined the company several years after as father was nearing retirement. Edward still serves as chairman and a key advisor for the company.
Hillhurst’s research has been mostly supported by the National Institutes of Health (NIH) and private foundations who have granted the company a total of around $15 million. Last year, the FDA cleared the company to begin its Phase 1 trial for HBI-002, an oral liquid carbon monoxide therapeutic for the treatment of sickle cell.
“We are very pleased that Hillhurst’s first IND has been favorably reviewed,” said Edward Gomperts, at the time. “This represents a major milestone as our lead product candidate, HBI-002, advances into the clinic. We look forward to embarking on our next chapter as a clinical-stage company, harnessing our platform to evaluate the therapeutic potential of low dose carbon monoxide to prevent vaso-occlusive crises and transform the lives of patients with sickle cell disease.”
That Phase 1 study on healthy volunteers is still ongoing, with the initial single dose phase complete and the final multiple dose phase expected to begin shortly, Andrew said, and Hillhurst is planning a Phase 2a study on the drug’s safety and efficacy in patients.
Hillhurst’s preclinical data on efficacy is “very, very strong,” Andrew said. “If a mouse has sickle cell disease, we really do almost fully prevent mice sickle cell crises, which we now want to see in humans.”
Other Uses for GLASS Platform
In addition to sickle cell, Hillhurst is also pursuing other indications for carbon monoxide, including a possible therapy for Parkinson’s disease.
“It turns out – totally counterintuitively – that smoking is protective of Parkinson’s. If patients with Parkinson’s smoked, they are protected and the more they smoked the more protected they are and current smokers are more protected than former smokers,” Andrew said, adding that “several studies” have shown this and additional studies found that nicotine was not the cause of the protection.
“So our collaborators think it might be the carbon monoxide,” he continued. “The Michael J. Fox Foundation is very supportive of this. We have great data in animals at this point. And we’re moving that program forward quite nicely. It’s exciting to have data supporting this.”
Another area Hillhurst is researching is using the GLASS platform to deliver nitrous oxide as an alternative pain medication.
“Right now, you have nsaids or opioids and nothing in between really,” Andrew said. “Given the opioid crisis, opioids are not ideal. So we’re looking to develop nitrous as a pain drug for home use.”
Hillhurst’s nitrous program is funded by the NIH Helping to End Addition Long-term (HEAL) Initiative that funds programs that combat the opioid crisis in the U.S.
Hillhurst Biopharmaceuticals, Inc.
CEO: Andrew Gomperts
Headquarters: San Diego
Business: Developer of liquified therapeutic medical gasses
Funding: $15 million
Notable: Hillhurst’s research into carbon monoxide treatment for Parkinson’s is supported by the Michael J. Fox Foundation.