Disc Medicine Delivers Promise For Patients with Sunlight Sensitivity In Successful Pivotal Study

Disc Medicine Inc. (NASDAQ:IRON) on Monday reported positive topline results from its APOLLO Phase 3 trial evaluating bitopertin for erythropoietic protoporphyria (EPP), including X-linked protoporphyria (XLP).

EPP and XLP are characterized by severe pain in the skin with exposure to sunlight.

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

The APOLLO trial assessed the efficacy and safety of bitopertin against a placebo in 183 adults and adolescents aged 12 and older with EPP or XLP.

Participants across the U.S., Canada, Europe, and Australia were randomized to receive a daily 60 mg oral dose of bitopertin or a placebo over six months.

Primary And Secondary Endpoints Achieved

Bitopertin demonstrated statistical significance across both co-primary endpoints compared to the placebo.

The treatment led to a 41.4% reduction in whole-blood metal-free PPIX from baseline (p<0.0001).

Additionally, patients experienced an average monthly increase of 12.2 hours in total time spent in light without pain during the sixth month (p=0.0010).

The trial also met all secondary endpoints with statistical significance. Bitopertin reduced the incidence rate of phototoxic reactions over the six months (p<0.0001) and increased the cumulative total time in light without pain (p<0.0001).

For adult patients, the treatment improved the average daily sunlight exposure time before experiencing a prodrome (p=0.0077).

Quality Of Life and Safety Profile

Beyond the primary and secondary measures, bitopertin showed significant quality of life improvements.

Patients reported better scores on the Patient Global Impression of Change and Patient Global Impression of Severity assessments, both achieving a p-value of <0.0001.

The treatment was generally well-tolerated. Dizziness was the most frequent treatment-emergent adverse event, or TEAE, in the bitopertin arm, affecting 50% of patients.

These cases were consistent with previous studies, generally transient, and mild to moderate in severity. No TEAEs exceeded Grade 3.

Following these APOLLO study results, Disc Medicine intends to resubmit a new drug application to the U.S. FDA by the end of the year. An FDA decision is expected by mid-2027.

IRON Price Action: Disc Medicine shares were up 26.07% at $71.66 on Monday, according to Benzinga Pro data.

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