One molecule, multiple therapeutic targets
EHP-101: Designed for targeted mechanism of action
EHP-101 is an oral formulation of a new chemical entity derived from synthetic cannabidiol, also known as CBD, one of the most abundant molecules found in the cannabis plant. The fully synthetic novel molecule formulated in EHP-101 has been rationally designed to enhance the therapeutic benefits of CBD by being a dual peroxisome proliferator-activated receptor gamma (PPARγ) and cannabinoid type 2 receptor (CB2) agonist that also activates the hypoxia inducible factor (HIF) pathway. PPARγ and CB2 are preclinically validated therapeutic targets, supported by scientific literature, for the development of novel drugs modulating inflammatory responses for the treatment of multiple sclerosis (MS) as well as for the treatment of systemic sclerosis, a form of scleroderma (SSc). In addition, EHP-101 is an activator of the hypoxia-inducible factor (HIF) pathway, which may have additional benefits to MS and SSc patients, as the HIF pathway modulates the immune response that favors both neuroprotection and myelin regeneration in MS as well as prevents collagen deposition around blood vessels and reduces vascular damage in SSc.
Furthermore, evidence shows these validated receptors may be beneficial in preventing neuroinflammation and demyelination in the CNS, and fibrogenesis in the periphery. Based on these activities, EHP is initially developing this proprietary new drug candidate for multiple sclerosis and systemic sclerosis (scleroderma).
To improve on CBD’s known positive effects by affecting validated targets in MS:
- PPARγ: Peroxisome proliferator-activated receptor gamma
- CB2: Cannabinoid receptor Type 2
- HIF: Hypoxia inducible factor
Systemic Sclerosis, A form of Scleroderma
TARGETS SYSTEMIC SCLEROSIS
- Systemic Sclerosis is a life-threatening disease with no current approved treatment
- PPARγ and CB2: extensively studied molecular targets for the treatment of systemic sclerosis*
- Combined effect on PPARγ, CB2
and HIF not described for other types of marketed drugs
- Preclinical proof-of-concept established in relevant animal models
*Minghua et al, Tavarares et al, Akhmetshina et al, Del Rio et al
In preclinical studies, EHP-101 demonstrated potential disease-modifying benefits relating to prevention of demyelination and stimulation of remyelination in multiple sclerosis. In SSc models, similar disease-modifying benefits were seen, both in reducing inflammation and fibrosis, as well as promoting the protection and regeneration of the vasculature, which is important in SSc.
Data from our large Phase 1 (104 healthy volunteers) randomized, double-blind, placebo-controlled study of EHP-101 demonstrated favorable safety and tolerability profiles. Mainly mild adverse events were observed with increasing doses, providing EHP with flexibility in setting the dosing of patients in future clinical studies.