Lenabasum (formerly known as anabasum) is an oral medication being developed by Corbus Pharmaceuticals to treat scleroderma and other diseases characterized by chronic inflammation and fibrosis (or scarring). It is considered a “first of its kind” treatment because it targets the signaling system that cells use to resolve or turn off inflammation without suppressing the immune system as a whole.

How lenabasum works

Scleroderma, or systemic sclerosis, is an autoimmune disease that involves chronic inflammation. The underlying cause of the disease is not known, but it is thought to develop when the body is unable to shut off the inflammatory response appropriately. Such chronic inflammation leads to fibrosis, or the formation of excessive connective tissue that is characteristic of scleroderma.

Lenabasum is a so-called synthetic endocannabinoid-mimetic medication that acts via the signaling system that cells use to turn off the inflammatory response when it is no longer necessary. During inflammation, activated immune cells start producing additional cannabinoid receptors on their surface. These receptors are activated by endocannabinoid, a type of neurotransmitter that works to signal to cells that it is time to turn off the inflammatory response. Cannabinoid receptors essentially serve as a cell’s antennae, waiting for the “all clear” signal.

When an endocannabinoid molecule binds to the cannabinoid receptor and activates it, a cascade of events is triggered and leads to the resolution (or shutting off) of the inflammatory response, which is overactive in scleroderma. Lenabasum is designed to mimic the action of endocannabinoids in the body, binding to the cannabinoid receptor and initiating the resolution of the inflammatory response.

The U.S. Food and Drug Administration (FDA) has not yet approved any medications specifically for the treatment of scleroderma. Those that suppress the immune system, such as corticosteroids and methotrexate, are often used to treat patients with severe scleroderma symptoms. But by suppressing the immune system as a whole, these medications may leave patients vulnerable to infections.

Lenabasum in clinical trials

Lenabasum is being tested in an ongoing Phase 2 clinical trial (NCT02465437) to evaluate the safety and efficacy of the treatment in patients with scleroderma. Results so far show that lenabasum reduced scleroderma symptoms (measured by CRISS scores) compared to placebo, and was acceptably well-tolerated and safe. The study is expected to conclude in October 2018.

A Phase 3 trial (NCT03398837) of lenabasum in scleroderma patients is currently recruiting participants in New York, and expects to open test sites across North America, Europe, Asia and Australia.

It plans to enroll around 350 patients, ages 18 or older and diagnosed with diffuse cutaneous scleroderma. Participants will be treated with one of two doses of lenabasum (5 mg or 20 mg twice a day) or a placebo for one year while their scleroderma symptoms are assessed. More information about the trial, which is expected to run through March 2020, can be found by clicking on the trial’s identification number.

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