Results from the phase II clinical trial of dersimelagon have been published in the New England Journal of Medicine.

The trial was conducted by Mitsubishi Tanabe Pharma and was a randomized, placebo-controlled, phase 2 trial to study how effective and safe dersimelagon is in patients with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP).

The study measured the amount of time before symptoms started with sunlight exposure and how severe the symptoms were.

Patients 18-75 years of age were randomly assigned to either placebo or study drug (at dose of 100mg or 300 mg once-a-day for 16 weeks). The study measured the change from baseline to week 16 in the time to the first signs (prodrome) of symptoms associated with sunlight exposure. Patients recorded
daily sunlight exposure and symptom data in an eDiary. Quality of life and safety were also assessed. 

Highlights 

• 102 patients (93 EPP & 9 XLP)  
• 90% completed the treatment period 
• Time to first symptoms from sunlight exposure increased significantly with study drug 
• Results also suggest that quality of life improved in patients receiving study as compared with placebo. 
• Most common adverse events that occurred or worsened during treatment were nausea, freckles, headache, and skin hyperpigmentation. 

Conclusions
At both doses, the study drug (dersimelagon) significantly increased the length of symptom-free sunlight exposure in patients with EPP or XLP. 

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