Phase 1 Clinical Trial of EHP-101 Concludes with Positive Safety Results

The first in-human clinical trial of EHP-101, an investigational treatment for multiple sclerosis (MS) and systemic scleroderma (SSc), has been successfully completed, Emerald Health Pharmaceuticals (EHP) announced.

EHP-101 demonstrated it was safe and well-tolerated in healthy volunteers, supporting further clinical development of EHP-101 in future Phase 2 clinical trials.

The company is currently preparing the launch of Phase 2 studies to explore the safety and impact of EHP-101 in patients with MS and SSc. The first Phase 2 trial is expected to begin by the end of 2019.

“Based on the novel mechanism of action of our product candidates, our preclinical proof-of-concept, these positive first-in-human clinical results, and our experienced team, we are now well-positioned to advance our lead product candidate through clinical development,” Jim DeMesa, MD, CEO of EHP, said in a press release.

EHP-101 (previously known as VCE-004.8) is an oral formulation of a synthetic derivative compound of cannabidiol (CBD) — one of the non-psychoactive components of the cannabis plant that has garnered increased scientific interest in recent years. Its different biological activities are  believed to be beneficial for MS patients.

Studies in mice models of MS demonstrated that treatment with EHP-101 could effectively reduce the amount of several genes involved in MS, including inflammatory molecules and proteins implicated in cellular adhesion to other cells or the extracellular matrix, which provides structural and biochemical support to cells.

Data also revealed that the investigational agent could prevent microgliosis, which is a nonspecific and intense reaction by microglia — immune cells of the central nervous system — in response to damage.

The recently completed Phase 1 clinical trial (NCT03745001) enrolled 104 healthy volunteers (people without MS or SSc) who were divided into 12 groups to receive one of eight single ascending dose (SAD), or one of the four tested multiple ascending dose (MAD), of EHP-101. In the SAD groups, participants were given either a placebo or one dose of EHP-101, with doses ranging from 0.9 mg up to 185 mg. In the MAD groups, participants got EHP-101 or placebo once or twice a day for a week, with doses of EHP-101 ranging from 20 mg per day to 100 mg per day.

According to EHP’s press release, the investigational compound was well-tolerated, “with only mild to moderate adverse events [side effects] observed with increasing dose levels.” It should be noted that the highest doses tested in the trial are quite a bit higher than what the therapeutic dose of EHP-101 for MS or SSc was expected to be.

These positive results give a good flexibility to the dose to be used in future clinical studies of EHP-101.

“The safety and tolerability profile of EHP-101 in our large first-in-human study allows us to define an appropriate dosing regimen and proceed with our planned Phase 2 studies in multiple sclerosis and systemic scleroderma patients,” said Joachim Schupp, MD, EHP’s chief medical officer.

Detailed Phase 1 results are expected to be announced before the end of 2019, with accompanying presentations at scientific conferences.

EHP-101 has received orphan drug designation for SSc by both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA).