FDA Paves Way for Phase 2 Testing of EHP-101 for Scleroderma

The U.S. Food and Drug Administration (FDA) has cleared an application from Emerald Health Pharmaceuticals that paves the way to begin a Phase 2 clinical trial testing EHP-101 in people with scleroderma.

EHP-101 is an oral therapy based on cannabidiol (CBD), one of the main components of cannabis and one that does not get users “high.” The main ingredient in EHP-101, VCE-004.8, is a synthetic derivative of CBD. It works by activating two protein receptors — peroxisome proliferator-activated receptor gamma (PPARɣ) and cannabinoid receptor type 2 (CB2) — that may help limit inflammation in scleroderma.

After showing potential to prevent skin and lung tissue scarring in a mouse model of scleroderma (or systemic sclerosis), EHP-101 was evaluated recently in a Phase 1 trial (NCT03745001) in healthy volunteers. The therapy was well-tolerated, with only mild-to-moderate side effects at higher doses. According to Emerald, the highest doses are much greater than what is anticipated to be used in scleroderma patients.

EHP-101 was granted orphan drug designation to treat scleroderma by the FDA in 2017, and by the European Medicines Agency the following year.

The U.S. Drug Enforcement Administration cleared the use of VCE-004.8 in 2019, determining that it is not a controlled substance as classified under the Controlled Substances Act. This means that EHP-101, also being developed for people with multiple sclerosis, is not defined as a substance that could be associated with drug abuse.

With the FDA’s clearance of Emerald’s investigational new drug (IND) application, a multi-center Phase 2 trial (NCT04166552) will test EHP-101 in Australia and the U.S. Emerald expects to enroll 36 adults (age 18–70) with diffuse cutaneous scleroderma.

The primary goal will be the frequency and severity of adverse events (side effects). Effectiveness will be assessed through questionnaires and standardized measurements, such as the American College of Rheumatology composite response index in diffuse cutaneous Systemic Sclerosis.

“We are excited to be able to start the expansion of our Phase 2 clinical trial of EHP-101 for the treatment of systemic sclerosis [scleroderma],” Jim DeMesa, MD, Emerald’s president and CEO, said in a press release.

“The novel multi-pronged mechanism of action and the results of our preclinical and Phase 1 studies give us great confidence in the ability for EHP-101 to succeed in Phase 2. With the initiation of our Phase 2 clinical study in systemic sclerosis currently underway in Australia, and now with the addition of the U.S. with our cleared IND, we are on track to start getting data by the end of 2020,” DeMesa said.