Disc Medicine Key Trial Succeeds, Offering New Hope for Photosensitive Patients

Disc Medicine Inc. on Monday reported positive top-line results from its APOLLO Phase 3 trial evaluating bitopertin for the treatment of erythropoietic protoporphyria, including X-linked protoporphyria.

EPP and XLP are characterized by severe pain upon skin exposure to sunlight.

The clinical-stage biopharmaceutical company confirmed that the trial successfully met all primary and secondary endpoints.

The APOLLO trial evaluated the efficacy and safety of bitopertin versus placebo in 183 adolescent and adult patients aged 12 years and older with EPP or XLP.

Participants from the United States, Canada, Europe, and Australia were randomized to receive either a 60 mg daily oral dose of bitopertin or placebo over six months.

Primary and Secondary Endpoints Met

Bitopertin showed statistical significance on both co-primary endpoints compared with placebo.

The treatment reduced metal-free PPIX levels in whole blood by 41.4% from baseline.

Additionally, at month six, patients experienced a mean increase of 12.2 hours in total monthly time spent in pain-free light exposure.

The trial also met all secondary endpoints with statistical significance. Bitopertin reduced the incidence of phototoxic reactions over six months and increased the cumulative total time of pain-free light exposure.

For adult patients, the treatment extended the mean daily sunlight exposure time before the onset of prodromal symptoms.

Quality of Life and Safety Profile

Beyond primary and secondary measures, bitopertin also demonstrated significant quality-of-life improvements.

Patients reported better scores on the Patient Global Impression of Change and Patient Global Impression of Severity assessments, with p-values <0.0001 for both measures.

The treatment was generally well tolerated. Dizziness was the most common treatment-emergent adverse event in the bitopertin group, affecting 50% of patients.

These cases were consistent with prior studies, typically transient and mild to moderate in severity. No TEAE exceeded grade 3.

Based on these APOLLO study results, Disc Medicine plans to resubmit its new drug application to the U.S. FDA by year-end. An FDA decision is expected by mid-2027.

IRON Stock Movement: As of Monday's data, Disc Medicine shares rose 26.07% to $71.66.