Disc Medicine's rare disease drug succeeds in late-stage trial
Disc Medicine's rare disease drug succeeds in late-stage trial Oct 19 (Reuters) - Disc Medicine IRON.O 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)
((Padmanabhan.Ananthan@thomsonreuters.com;))