Disc Medicine's rare disease drug succeeds in late-stage clinical trials

Reuters, October 19 - Disc Medicine (IRON.O) said on Monday that its experimental oral drug for a rare disease that causes severe pain upon sun exposure achieved all primary and secondary endpoints in a late-stage clinical trial, sending the company's stock soaring 65% in premarket trading.

Details are as follows:

• The company is testing bitopertin as a treatment for erythropoietic protoporphyria (EPP), a blood disorder that makes patients extremely sensitive to sunlight.

• The disease causes a substance called protoporphyrin IX (PPIX) to accumulate in the blood, triggering painful skin reactions when patients are exposed to sunlight.

• Previously, the US Food and Drug Administration (FDA) refused to approve the drug earlier this year, citing "the lack of clear correlation between lowered protoporphyrin levels in patients and their ability to tolerate sunlight" (link).

• The company said that among 183 patients aged 12 and older who took bitopertin, their PPIX levels in blood were reduced by 41.4% compared to patients on placebo.

• Disc Medicine stated that bitopertin was generally well-tolerated, with no new safety issues identified.

• Avoiding sun exposure by staying indoors and wearing protective clothing remains the main way to manage the disease.

• The only currently approved treatment for EPP in the US is a subcutaneous hormonal implant called Scenesse.

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