Dersimelagon

Tanabe Pharma unveiled positive data from a Phase 3 trial of its oral drug for a pair of rare diseases, its first major clinical milestone after it was acquired by Bain Capital last year. The Japanese company tested its MC1R receptor agonist, dersimelagon, in 165 adults and teens with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP) enrolled in a trial called INSPIRE. Patients with the diseases suffer from extreme pain when they’re exposed to light, as a result of a buildup of protoporphyrin in the blood and erythrocytes in tissues. In January, Tanabe revealed that the trial met its primary endpoint, but didn’t present detailed results. On Saturday, it said that patients treated with dersimelagon for 12 to 16 weeks were exposed to sunlight for an average 23.19 minutes longer than placebo patients before they started experiencing early symptoms like stinging and burning. At week 16, the average time of exposure before symptoms presented increased to almost 30 minutes — more than double with a placebo. The results were presented at the American Academy of Dermatology meeting in Denver. There are currently no FDA-approved treatments for XLP, and Clinuvel Pharmaceuticals’ Scenesse is the only medicine available for adults with EPP. That drug also targets the MC1R receptor, but comes as a subcutaneous implant instead of being taken orally. In February, the FDA rejected Disc Medicine’s candidate for EPP, known as bitopertin. That drug targeted GlyT1. Tanabe said it has already started preparing to apply for approval with the FDA. It estimates there are around 20,000 patients in the US with the diseases. Dersimelagon was one of several internal programs Tanabe had at the time of the Bain takeover, along with the experimental drug ND0612 for Parkinson’s disease (which is being advanced by its subsidiary NeuroDerm) and MT-4561 for advanced solid tumors. At the time of the deal, Bain said Tanabe would partly focus on in-licensing assets for the Japan market to tackle the problem of “drug loss” in the country. The term describes the phenomenon whereby around half of medicines developed in Western markets don’t make it to Japan because of the country’s conservative regulations and the high cost of running trials there. While that strategy remains a focus, Saturday’s data also offer evidence that Tanabe “knows how to discover, develop, partner and commercialize innovative science,” the company’s Chief Business Officer Sean Ryan told Endpoints News in an interview.

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