Emerald Health Pharmaceuticals Receives Safety Review Committee Approval to Proceed to Final Cohorts of EHP-101 Phase 2a Systemic Sclerosis Trial

Emerald Health Pharmaceuticals Inc. recently received a positive safety review following a pre-specified interim analysis by the Safety Review Committee (SRC) of its Phase 2a study of EHP-101 for systemic sclerosis (SSc). The SRC has recommended that the trial continue without modification. EHP expects to report interim results from the study in early 2023.

The SRC is comprised of two physicians, one independent and the other the principal investigator in the trial. The SRC has the ability to recommend that the trial continues at the same dose or at a lower dose, that it escalates to a higher dose, or that the study be terminated altogether due to safety concerns. The SRC evaluated the blinded safety data from all patients enrolled in cohorts 1 and 2 at least up to one month post first dose, at 25 mg daily and 25 mg twice daily (50 mg), respectively.  They determined that EHP-101 was well-tolerated, with no drug-related serious adverse events and only mild related adverse events reported.  The pharmacokinetic analysis of plasma samples over the course of the 3-month treatment did not show apparent drug accumulation in patients’ blood.

“We are pleased that the safety analysis of the first two cohorts of this Phase 2a study of EHP-101 in systemic sclerosis has confirmed a positive safety profile of the drug to date, consistent with the safety profile we observed in our large Phase 1 study,” said Alain Rolland, PharmD, PhD, COO of EHP. “EHP-101’s mechanism of action offers the prospect of important therapeutic effects for patients. We look forward to reporting the full interim analysis of this study in early 2023. We are working to secure the funds necessary to continue the next phase of this Phase 2a study.”

The Company’s lead product candidate, EHP-101, is an oral formulation of a patented new molecule. This SSc Phase 2a trial is a double-blind, placebo-controlled study to evaluate the safety, tolerability, pharmacokinetics and preliminary efficacy of EHP-101 in patients with SSc. SSc is a disease characterized by rapid fibrosis of the skin and internal organs. EHP has completed the first two cohorts, each with nine SSc patients, administering two doses either once daily or twice daily. The treatment lasted for 12 weeks, with a 4-week follow-up. Further details about the study design can be found on ClinicalTrials.gov: NCT04166552.

EHP has obtained clearance from the FDA to amend the study protocol to increase the size of the higher-dose cohorts 3 and 4 from a total of 18 to a total of 48 subjects and increase the treatment duration from 12 weeks to 24 weeks. The Company applied for this amendment to create a pathway to possibly advance directly into a Phase 3 clinical trial of EHP-101 in patients suffering from SSc once the Phase 2a trial is completed, rather than first conducting a Phase 2b before continuing to a Phase 3 trial.

EHP-101 is an oral formulation of VCE-004.8, a first-in-class activator of the subunit B55alpha of the protein phosphatase PP2A that is implicated in the hypoxia inducible factor pathway.  It also provides dual peroxisome proliferator-activated receptor gamma modulation and cannabinoid receptor type 2 agonist activity. Those receptors are important therapeutic targets for SSc, a disorder with various etiologies. EHP has received Orphan Drug Designation for EHP-101 in SSc in both the US and EU and Fast Track Designation for systemic sclerosis in the US.

SSc, a severe form of scleroderma, is a rare and chronic autoimmune disease, causing fibrosis of the skin and internal organs, including small blood vessel damage in the skin and multiple other organs in the body such as lung, heart, kidneys, musculoskeletal system and the gastrointestinal tract. The tissues of these organs become hard and fibrous, causing them to function less efficiently. While the symptoms of SSc vary for each person, it can be life-threatening, depending on which parts of the body are affected and the extent of the disease.

SSc is subclassified into diffuse cutaneous SSc (dcSSc) or limited cutaneous SSc (lcSSc) based on the extent of skin involvement. Patients with dcSSc have more areas involved, and measurements of the effects of treatment have been validated by international clinical trial experts for this subset of SSc patients. The disease is more common in adults, with approximately 80,000-100,000 people affected in the US.

There are no approved treatments specific to SSc. Current therapies for this disease include mainly drugs that suppress the immune system, are limited in efficacy, and sometimes present toxicities. New treatments will be critical to help reduce the symptoms of SSc and prevent further damage to the body.

EHP is developing novel product candidates for the treatment of CNS, autoimmune, and other diseases with unmet medical needs. The company has two families of patented new chemical entities that it has created through rational drug design to affect validated receptors and pathways in the body which are pertinent to targeted diseases. Its first drug product candidate, EHP-101, is in a Phase 2a study for the treatment of SSc, a severe form of scleroderma, and has met regulatory requirements to start a Phase 2 study for multiple sclerosis. Its second product candidate, EHP-102, is in preclinical development and is focused on treating Parkinson’s disease and Huntington’s disease. EHP-101 has received Orphan Drug Designation in the US and EU, as well as Fast-Track status by the US FDA for systemic sclerosis. EHP-102 has received Orphan Drug Designation in the US and EU for Huntington’s disease. For more information, visit http://www.emeraldpharma.com.