Oct 19 (Reuters) – Disc Medicine said on Monday its experimental pill for a rare disease that causes extreme pain from sunlight met all of its main and secondary goals in a late-stage trial, sending its shares surging 65% in premarket trading.
Here are some details:
• The company was testing bitopertin as a treatment for erythropoietic protoporphyria (EPP), a blood disorder that makes patients extremely sensitive to sunlight
• The condition causes a substance called protoporphyrin IX (PPIX) to build up in the blood, which causes painful skin reactions when patients are exposed to sunlight
• The results come after the US FDA declined to approve the drug earlier this year, citing a lack of a clear link between the reduction in patients’ protoporphyrin levels and their tolerance to sunlight
• A total of 183 patients aged 12 and older who took bitopertin had 41.4% less PPIX in their blood compared with those on placebo, the company said
• Bitopertin was generally well tolerated with no new safety concerns, Disc Medicine said
• Avoiding sunlight, by staying indoors and wearing protective clothing, remains the main way to manage the disease
• The only US-approved treatment for EPP is an under-the-skin hormone implant known as Scenesse
(Reporting by Padmanabhan Ananthan in Bengaluru; Editing by Maju Samuel)
