“This is our first Phase 2 clinical study, which is another important milestone for our lead product candidate,” Joachim Schupp, MD, Emerald Health’s chief medical officer, said in a press release.
“Regulatory approvals are in place and we have started site initiations. While the current COVID-19 pandemic presents some temporary challenges, we continue working remotely with the investigators and clinical site personnel and look forward to soon enrolling systemic sclerosis patients without risking their health or the health of study personnel,” Schupp added.
The Phase 2a study (NCT04166552) will assess EHP-101’s safety, tolerability, pharmacokinetics (uptake, distribution, and elimination in the body), and preliminary effectiveness in approximately 36 adults (age 18–70) with dcSSc. Information on enrollment, locations, and contacts will be available here.
Participants are expected to be recruited at about 30 sites across the U.S., Australia, and New Zealand. They will be randomly assigned to receive one of four dosages of EHP-101 or a placebo (administered orally once or twice daily) for 84 days, followed by a 28-day follow-up period.
Effectiveness will be assessed through several validated clinical tests and questionnaires. These include a composite measure of disease state and organ damage called the American College of Rheumatology composite response index in SSc, a global health assessment by both patients and physicians, a functional questionnaire known as the Scleroderma Health Assessment Questionnaire – Disability Index, and a quality of life assessment called the European Quality of Life 5 Domain Questionnaire.
Also, the team will assess changes in the levels of biomarkers associated with the multiple organs affected by scleroderma and with EHP-101’s mechanism of action.
The rationale for testing EHP-101 in people with scleroderma is based on its reported anti-inflammatory and anti-fibrotic (scarring) effects.
The active ingredient in EHP-101, known as VCE-004.8, is a new molecule derived from cannabidiol (CBD) — a non-psychoactive component of the cannabis plant that has gained increased scientific interest in recent years. Both the U.S. Drug Enforcement Administration and Canada’s Controlled Substances Directorate recently cleared the use of VCE-004.8, meaning that it is not considered a controlled substance.
EHP-101 was designed to be therapeutically superior to other CBD products due to its activation of cannabinoid receptor type 2 and peroxisome proliferator-activated receptor gamma, previously shown to help prevent inflammation.
In addition, EHP-101 targets the hypoxia-inducible factor pathway, which is involved in fibrotic diseases such as scleroderma.
In a mouse model of scleroderma, EHP-101 prevented blood vessel damage, and reduced skin and lung tissue fibrosis and inflammation. The data supported the initiation of a Phase 1 clinical trial (NCT03745001) evaluating the therapy’s safety, tolerability, and pharmacokinetics in 104 healthy volunteers in Australia.
Results showed that the therapy was well-tolerated, with only mild-to-moderate side effects reported at higher doses, which Emerald Health said were much higher than the doses anticipated to be used in patients.
“A safe and efficacious therapy for systemic sclerosis is clearly needed since there is currently no approved treatment,” said John Varga, MD, co-director of the Northwestern Scleroderma Program in Chicago and a clinical advisory board member of Emerald Health. “With its unique, targeted and multi-pronged mechanism of action, EHP-101 has the potential to be a significant advancement for patients with this life-threatening disease.”
EHP-101 has received fast track designation by the U.S. Food and Drug Administration as well as orphan drug designation for the treatment of systemic scleroderma in the U.S. and E.U. These designations are meant to provide support and financial benefits to accelerate the therapy’s development, as well as marketing exclusivity for a period of time upon regulatory approval (seven years in the U.S. and 10 years in the E.U.).
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