Dersimelagon

Tanabe Pharma is preparing to submit a US marketing application for its oral drug dersimelagon (MT-7117) after recent success in a Phase III study for people living with erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP), detailed results from which were presented over the weekend at the American Academy of Dermatology (AAD) annual meeting."Dersimelagon is the first oral therapy to demonstrate Phase III clinical functional benefit in EPP and XLP, and, if approved, it has the potential to become the first oral treatment option," said CEO Akihisa Harada.The INSPIRE study randomised 165 adults and adolescents with EPP or XLP to receive either placebo or the selective melanocortin-1 receptor (MC1R) agonist dersimelagon once daily for 16 weeks. Tanabe announced in January that the trial achieved its primary endpoint of time-to-first prodromal symptoms, such as burning, tingling, itching or stinging, associated with sunlight exposure. Results presented at AAD showed that dersimelagon was associated with a statistically significant prolongation of average daily sunlight exposure time-to-first prodromal symptom during weeks 12 to 16, with a placebo-adjusted least-squares mean difference of 23.19 minutes in the primary analysis, increasing to 29.64 minutes at week 16.Tanabe said that key secondary goals were also met, including statistically significant differences in Patient Global Impression of Change (PGIC) and a 39% reduction of total pain events compared to placebo.The company noted that dersimelagon was generally well tolerated in the INSPIRE trial, with the most common adverse events, occurring more frequently than placebo, being melanocytic nevi, headache, nausea, diarrhoea and skin hyperpigmentation."With the positive data from…we look forward to advancing dersimelagon toward a planned NDA submission," Harada said.Tanabe was taken private last year by private equity firm Bain Capital, in the process rebranding from its previous name of Mitsubishi Tanabe Pharma under parent company Mitsubishi Chemical Group.

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.