Disc Medicine’s new US patent covers bitopertin therapy for EPPs

BEACON trial results to be presented at hematology conference in December

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by Steve Bryson, PhD |

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Disc Medicine has been granted a U.S. patent for treating erythropoietic protoporphyrias (EPPs) with glycine transport inhibitors, such as its investigational oral therapy bitopertin.

“We are pleased to have been issued this patent that further secures [intellectual property] protection for bitopertin for the treatment of erythropoietic protoporphyria as we work to deliver a potentially transformative therapy to patients,” John Quisel, PhD, president and CEO of Disc, said in a company press release.

“This patent further highlights the innovative approach we are taking to treat EPP, as demonstrated by the data we shared in June, which we will update at ASH [the American Society of Hematology Annual Meeting and Exposition] in December,” Quisel added.

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BEACON, AURORA trials of bitopertin complete enrollment

US patent covers bitopertin as potential treatment for EPP, XLP until 2041

The patent, No. 11,813,257, covers bitopertin for treating erythropoietic protoporphyria or X-linked protoporphyria (XLP) with an anticipated expiration date in 2041. Disc intends to pursue additional applications with the U.S. Patent and Trademark Office for bitopertin as a treatment for other blood disorders.

EPP and XLP are caused by mutations in genes coding for molecules needed to produce heme, a molecule that enables red blood cells to carry oxygen throughout the body. Such defects lead to the buildup of a heme precursor called protoporphyrin IX (PPIX).

This results in hypersensitivity to sunlight, which leads to painful skin redness, swelling, itching, burning, and blisters, and a greater likelihood of developing certain liver conditions.

Bitopertin inhibits, or blocks, the glycine transporter 1, a protein that supplies glycine to red blood cells and is essential for heme production. By reducing glycine in red blood cells, the experimental oral therapy is expected to help control heme production and reduce PPIX buildup.

Originally developed by Roche for schizophrenia and acquired by Disc in 2021, bitopertin was previously shown to be safe in more than 4,000 people in clinical trials.

In a June congress, Disc presented data from the ongoing Phase 2 BEACON trial (ACTRN12622000799752), which is testing bitopertin in adolescents and adults with EPP and XLP in Australia.

Participants are taking bitopertin at 20 mg or 60 mg once daily for 24 weeks, or about six months. All had the option to continue treatment at the high dose for an additional 24 weeks.

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Bitopertin seen to boost sunlight tolerance, life quality in patients

Bitopertin led to better PPIX levels, sunlight tolerance, quality of life

Preliminary results from 15 adults showed that up to six months of the oral therapy safely dropped PPIX levels by more than 40%, increased sunlight tolerance, and improved life quality. Toxic reactions to sunlight were reduced by 96% with bitopertin, according to patient reports.

Updated results from all BEACON participants will be shared at ASH, to be held Dec. 9-12 in San Diego and online.

Like in BEACON, patient recruitment is now complete in the Phase 2 AURORA trial (NCT05308472), which has enrolled 75 adults with EPP in the U.S. to evaluate bitopertin’s safety and efficacy against a placebo over four months.

The study’s main goal is to assess changes in blood PPIX levels, while secondary goals include changes in sunlight sensitivity and tolerability. Top-line results are anticipated by early 2024.