Tanabe Pharma America’s small molecule designed to boost melanin production has hit the main goal of a phase 3 trial for patients with rare hereditary disorders that cause extreme sensitivity to sunlight.
The top-line results come from dersimelagon, also known as MT-7117, an oral melanocortin-1 receptor agonist designed to treat erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP)—two rare conditions that cause severe and painful skin reactions when exposed to the sun and sometimes certain artificial light.
The global, double-blinded phase 3 study enrolled 165 patients aged between 12 and 75 years and measured the time to onset and severity of first early symptoms—such as burning, tingling, itching or stinging—tied to sun exposure. Participants were randomly assigned to receive either placebo or 200 mg dersimelagon daily for 16 weeks.
The study, coded Inspire, met the primary endpoint, though Tanabe Pharma America didn’t share any data behind the announcement. The experimental drug also showed a favorable safety profile, with most adverse events of mild or moderate severity, according to the U.S. subsidiary of Japan’s Tanabe Pharma.
The pharma is currently running an open-label extension of the trial.
“The limited treatment options for adults with EPP or XLP and lack of any approved treatments for adolescents have created a care deficit, forcing many to endure severe life disruptions as they rely only on sun protection or avoidance,” Bijan Nejadnik, M.D., Tanabe Pharma America’s head of global development and regulatory affairs, said in a Jan. 20 release.
“The positive topline results from the Inspire study represent key data that will guide our research for the porphyria community,” Nejadnik added.
The two inherited diseases typically present in childhood with extremely painful phototoxic reactions, with early symptoms possibly setting in after fewer than 10 minutes of sun exposure.
Tanabe’s dersimelagon has snared both fast track and orphan drug status from the FDA. The company has also studied the investigational drug in a phase 2 trial for systemic sclerosis.
While the U.S. subsidiary didn’t outline upcoming plans for dersimelagon in the Jan. 20 release, parent company Tanabe said it will “continue to advance the development of the oral agent MT-7117 as a new therapeutic option for patients with EPP and XLP,” according to a Jan. 15 announcement (PDF).
The parent company—formerly known as Mitsubishi Tanabe Pharma—is focused on developing precision medicines for conditions impacting the nervous system, immuno-inflammation, diabetes and the kidneys, along with cancer.