EHP-101, an oral cannabidiol treatment candidate for people with scleroderma (SSc) and multiple sclerosis (MS), was found to be safe and well-tolerated in a Phase 1 clinical trial in healthy volunteers.
These results clear the way for Phase 2 trials, now in planning stages, to test the effectiveness and safety of EHP-101 in SSc and MS patients. The first Phase 2 study is expected to start this year, Emerald Health Pharmaceuticals — EHP-101’s developer — announced in a press release.
EHP-101’s active compound — VCE-004.8 — is a synthetic derivative of cannabidiol (CBD) extracted from the cannabis plant, which activates two receptors called PPARgamma and CB2 thought to be involved in cellular mechanisms that include inflammation.
The randomized, double-blind Phase 1 trial (ACTRN12618001390279p) assessed the safety and pharmacological profile of increasing doses of the candidate therapy, as well as exploratory biomarkers in 104 healthy volunteers in Australia. (Pharmacokinetics refers to how a compound behaves inside the body.)
In the single ascending dose part of the trial, participants were divided into eight groups and given either a placebo or EHP-101 in doses ranging from 0.9 mg to 185 mg. In it multiple ascending dose part, they were assigned to four groups and given placebo or EHP-101 once or twice each day for seven days, with daily doses ranging from 20 mg (low-dose group) to 100 mg (high-dose group).
All doses of EHP-101 were well-tolerated, with only mild to moderate side effects observed with increasing doses. According to Emerald, the highest doses used in this trial are well above that expected to be used to treat scleroderma and multiple sclerosis.
“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 II studies,” Joachim Schupp, MD, Emerald’s chief medical officer, said in the release.
Topline Phase 1 results are expected to be released in the coming months, following complete data analysis. The company is planning to present them at international conferences.
“On behalf of our entire EHP team,” Schupp added, “I thank the investigators, volunteers and all study team members for contributing to the understanding of EHP-101 in humans as we work to develop treatments for diseases with significant unmet medical need.”
Preclinical work in mice given EHP-101 revealed anti-inflammatory and anti-fibrotic (scarring) responses, as well as reduced skin thickness and recovery of damaged blood vessels.
Jim DeMesa, MD, Emerald’s CEO, said that these results, along with preclinical findings and other work, had Emerald “well-positioned to advance our lead product candidate through clinical development.”
The U.S. Drug Enforcement Administration recently cleared the use of VCE-004.8, this treatment’s active ingredient, determining that it is not classified as a controlled substance under the Controlled Substances Act. As such, VCE-004.8 is not defined as a substance that could be linked with abuse, as are hallucinogens, narcotics, depressants, and stimulants.