Hillhurst Biopharmaceuticals, Inc. is gassing up for clinical trials of its liquid drug products containing therapeutic gases.
The company completed an “oversubscribed” financing round last month of “several mission dollars,” said CEO Andrew Gomperts. “In combination with the substantial non-dilutive funding we’ve received, it should fund us through two Phase 2a studies.”
In addition to the funding round, led by Friedman Bioventure Fund with participation from new and existing investors, Hillhurst has received funding from NIH programs and foundations.
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 disease and Parkinson’s Disease. The company’s novel GLASS platform (Gas in Liquid Advanced Stability System) delivers carbon monoxide to patients in a simple beverage.
Hillhurst recently completed Phase 1 studies in healthy adults of its lead product HBI-002, an oral drug product candidate of low dose carbon monoxide, with an administration route designed to enable chronic use in a home setting for patients suffering from sickle cell disease.
Gomperts said Hillhurst’s Phase 1 study demonstrated not only the safety of HBI-002 in healthy volunteers, but also the dosing level it achieved in preclinical studies on mice.
“In sickle cell mice, there’s a certain level you can achieve in the blood, and we achieved that level and beyond it,” he said.
Hillhurst’s preclinical data on HBI-002’s efficacy is “very strong,” Gomperts previously told the San Diego Business Journal. “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 potential targets for HBI-002 are conditions associated with inflammation and cell death such as Parkinson’s disease.
With the new funding, Hillhrust will be able to move both its sickle cell and Parkinson’s programs into Phase 2a studies, starting with an ascending multiple dose study in subjects with sickle cell disease with 14 days of daily dosing to assess the safety, tolerability, pharmacokinetics, and proof of concept efficacy of HBI-002. Hillhust expects readouts from the Phase 2a study this year.
Alternative Pain Drug
Gomperts said the clinical studies are a “great proof of concept” for the GLASS platform – demonstrating that an oral liquid with the gas/drug goes through the stomach or GI and into the body, “which is very helpful for other drugs, including our pain drug.”
In addition to exploring carbon monoxide, Hillhurst is researching 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,” Gomperts 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.
‘Major Medical Need’
Hillhurst advancing its sickle cell drug into the clinic comes on the heels of recent FDA approvals of two new therapies for the disease: bluebird bio’s stem cell therapy Lyfgenia and Vertex Pharmaceuticals’ and CRISPER Therapeutics’ Casgevy – the first authorized CRISPER-based gene-editing therapy in the world.
Gomperts said that Hillhurst, its investors and sickle cell experts are “encouraged” by the approvals of the new treatments.
“Historically, sickle cell research has been underfunded, so we’re excited about the approval of these drugs. But they are gene therapy drugs,” he said, adding that experts in the sickle cell community are concerned about the “availability and suitability” of these new treatments. Lyfgenia and Casgevy are priced at $3.1 million and $2.2 million respectively and are difficult to manufacture.
“Investors and we also believe a major medical need still exists, despite these therapies and also despite the therapies available, the existing standard of care therapies,” Gomperts said.
Hillhurst Biopharmaceuticals, Inc.
CEO: Andrew Gomperts
Headquarters: San Diego
Business: Developer of liquified therapeutic medical gasses
Funding: $24 million
Notable: Hillhurst’s research into carbon monoxide treatment for Parkinson’s is supported by the Michael J. Fox Foundation.