Corbus Pharmaceuticals Holdings, Inc. (CRBP) (“Corbus” or the “Company”), a Phase 3 clinical-stage pharmaceutical company focused on the development and commercialization of novel therapeutics to treat rare, chronic and serious inflammatory and fibrotic diseases, announced today that the European Commission has granted Orphan Designation in the European Union (“EU”) to lenabasum, its novel, synthetic oral endocannabinoid-mimetic drug, for the treatment of dermatomyositis (“DM”).
The Company recently announced that lenabasum was also granted Orphan Drug Designation (“ODD”) for the treatment of DM by the U.S. Food and Drug Administration (“FDA”).
Orphan Designation is granted by the European Commission to drugs that are intended for the treatment, prevention or diagnosis of life-threatening or chronically debilitating rare diseases where no satisfactory method of diagnosis, prevention or treatment of the condition concerned is authorized. If such a method exists, then the medicine must be of significant benefit to those affected by the condition. Rare diseases are those defined as having a prevalence of not more than five per 10,000 population in the EU. The Orphan Designation provides potential incentives for the sponsor to develop a medicine for a rare disease, such as protocol assistance, reduced fees, funding for clinical trials, and protection from competition in the EU once the medicine is placed on the market, including ten years of market exclusivity.
Dermatomyositis is a rare and serious systemic autoimmune condition characterized by skin and muscle involvement. Like other autoimmune diseases, it affects more women than men and morbidity is more severe in black, Asian and Native American populations. The disease is characterized by distinct skin lesions that can be accompanied by erosions, photosensitivity, itch, ulcers, calcinosis and hair loss as well as other abnormalities. Muscle inflammation and atrophy is a characteristic of the disease and can manifest as weakness. Dermatomyositis affects as many as 80,000 people in the US, EU and Japan. Mortality is high with 5-year survival of 70% and 10-year survival of 57%. Standard of care includes antimalarial drugs and potent immunosuppressive agents, which often lead to significant adverse effects.
Lenabasum (formerly known as anabasum) is a synthetic, oral, small-molecule, selective cannabinoid receptor type 2 (CB2) agonist that has been shown to preferentially bind to CB2 expressed on activated immune cells and fibroblasts in animal studies. CB2 activation triggers physiologic pathways that resolve inflammation, speed bacterial clearance and halt fibrosis. CB2 activation also induces the production of specialized pro-resolving lipid mediators that activate an endogenous cascade responsible for the resolution of inflammation and fibrosis, while reducing production of multiple inflammatory mediators. Through activation of CB2, lenabasum also is believed to have a direct effect on fibroblasts to halt tissue scarring. In preclinical and clinical studies conducted so far, lenabasum has been shown to induce resolution rather than immunosuppression by triggering biological pathways to turn “off” chronic inflammation and fibrotic processes. Lenabasum has demonstrated promising potency in preclinical models of inflammation and fibrosis. Preclinical data and clinical studies to date have shown lenabasum to have a favorable safety, tolerability and pharmacokinetic profile. Data to date suggest that the drug may have clinical benefit as well as a beneficial impact on inflammatory and immunological markers in Phase 2 studies in diffuse cutaneous systemic sclerosis, dermatomyositis and cystic fibrosis. Additional clinical studies are being conducted and/or planned to confirm these preliminary results and support applications for regulatory approval.
Corbus Pharmaceuticals Holdings, Inc. is a Phase 3 clinical-stage pharmaceutical company focused on the development and commercialization of novel therapeutics to treat rare, chronic, and serious inflammatory and fibrotic diseases. The Company’s lead product candidate, lenabasum, is a novel, synthetic oral endocannabinoid-mimetic drug designed to resolve chronic inflammation and fibrotic processes. Lenabasum is currently being evaluated in systemic sclerosis, cystic fibrosis, dermatomyositis, and systemic lupus erythematosus.