Disc Medicine Buys Global Rights to Potential Oral Therapy for EPP

Disc Medicine has entered into an exclusive worldwide licensing agreement to develop and commercialize Roche’s bitopertin, an oral candidate therapy for blood disorders.

Disc will initially develop bitopertin for erythropoietic porphyrias, a group of porphyrias that mainly affect red blood cells and bone marrow. The company is planning to launch a Phase 2 trial in 2022 to test bitopertin in patients with erythropoietic protoporphyria (EPP), one of the three subtypes of erythropoietic porphyria.

EPP is caused by mutations in genes involved in the production of heme — a molecule essential for red blood cells’ oxygen transport and the breakdown of compounds in the liver. This leads to the accumulation of intermediate byproducts, called porphyrins, which react when a person is exposed to sunlight, causing pain and damage to the skin and liver.

Glycine, a key component in the production of heme, is supplied to red blood cells via a membrane transporter called glycine transporter 1 (GlyT1).

Bitopertin is a first-in-class, oral small molecule inhibitor of GlyT1. By limiting the amount of glycine imported by red blood cells, the candidate therapy was shown to limit heme production and, as a consequence, the buildup of porphyrins that underlie EPP and other forms of porphyria.

“This collaboration is a major milestone in establishing Disc Medicine as a leader in hematology [study of blood] and enables us to target heme synthesis, a fundamental biological pathway of red blood cells that is highly complementary to our existing programs in iron homeostasis [balance],” John Quisel, PhD, president and CEO of Disc Medicine, said in a press release.

Originally developed to ease symptoms of schizophrenia, the investigational therapy was deemed safe in clinical trials that enrolled more than 4,000 healthy volunteers and patients with psychiatric and rare blood cell disorders.

“Importantly, the well-established clinical safety profile of bitopertin will allow us to move rapidly into patient studies of several serious, hematologic diseases,” Quisel added.

Under the terms of the agreement, Disc Medicine holds the exclusive worldwide rights to develop, manufacture, and commercialize bitopertin and other related compounds.

In turn, Roche will receive an upfront payment and additional payments totaling more than $200 million upon the achievement of certain developmental, regulatory, and commercial milestones. It is also eligible to receive tiered royalties according to net revenues, as well as part of the proceeds from future transactions related to bitopertin.