Our Pipeline
Program
Preclinical
Phase 1
Phase 2
- Bitopertin is an oral, selective inhibitor of glycine transporter 1 (GlyT1). We are developing bitopertin as the potentially first disease-modifying therapy to treat erythropoietic porphyrias – a family of rare, genetic disorders caused by dysregulated heme synthesis.
- We obtained an exclusive global license for bitopertin from Roche in 2021, which established the clinical safety profile of bitopertin in over 4,000 patients. We have initiated two phase 2 studies in patients with EPP and XLPP, an investigator-sponsored study in Diamond-Blackfan Anemia, and are planning additional studies other indications.
- DISC-0974 is a first-in-class, injectable mAb that inhibits signaling through hemojuvelin (HJV), a co-receptor required for hepcidin expression. We have demonstrated in numerous preclinical studies that DISC-0974 potently suppresses hepcidin production and are developing DISC-0974 to treat anemias of inflammation.
- We obtained an exclusive global license for DISC-0974 from Abbvie in 2019. We initiated a first-in-human clinical study of DISC-0974 in healthy volunteers in 2021, a phase 1b / 2 study in patients with anemia of myelofibrosis in 2022 and a phase 1b/2 study in patients with non-dialysis dependent chronic kidney disease (NDD-CKD) and anemia and are planning additional studies in other indications.
- DISC-3405 (formerly MWTX-003) is an injectable mAb that inhibits TMPRSS6 (Transmembrane Serine Protease 6, also known as Matriptase-2). Genetic studies show that TMPRSS6 affects red blood cell formation by controlling the level of iron that is available for erythropoiesis. DISC-3405 has demonstrated potent and durable suppression of serum iron and efficacy in animal models of beta-thalassemia and polycythemia vera (PV).
- We in-licensed DISC-3405 from Mabwell Therapeutics in 2022. The US FDA accepted the IND for DISC-3405 in November 2022 and granted it Fast Track Designation in September 2023. We initiated a phase 1 study in healthy volunteers in October 2023.
Preclinical
Phase 1
Phase 2
- Bitopertin is an oral, selective inhibitor of glycine transporter 1 (GlyT1). We are developing bitopertin as the potentially first disease-modifying therapy to treat erythropoietic porphyrias – a family of rare, genetic disorders caused by dysregulated heme synthesis.
- We obtained an exclusive global license for bitopertin from Roche in 2021, which established the clinical safety profile of bitopertin in over 4,000 patients. We have initiated two phase 2 studies in patients with EPP and XLPP, an investigator-sponsored study in Diamond-Blackfan Anemia, and are planning additional studies other indications.
Preclinical
Phase 1
Phase 2
- DISC-0974 is a first-in-class, injectable mAb that inhibits signaling through hemojuvelin (HJV), a co-receptor required for hepcidin expression. We have demonstrated in numerous preclinical studies that DISC-0974 potently suppresses hepcidin production and are developing DISC-0974 to treat anemias of inflammation.
- We obtained an exclusive global license for DISC-0974 from Abbvie in 2019. We initiated a first-in-human clinical study of DISC-0974 in healthy volunteers in 2021, a phase 1b / 2 study in patients with anemia of myelofibrosis in 2022 and a phase 1b/2 study in patients with non-dialysis dependent chronic kidney disease (NDD-CKD) and anemia and are planning additional studies in other indications.
Preclinical
Phase 1
Phase 2
- DISC-3405 (formerly MWTX-003) is an injectable mAb that inhibits TMPRSS6 (Transmembrane Serine Protease 6, also known as Matriptase-2). Genetic studies show that TMPRSS6 affects red blood cell formation by controlling the level of iron that is available for erythropoiesis. DISC-3405 has demonstrated potent and durable suppression of serum iron and efficacy in animal models of beta-thalassemia and polycythemia vera (PV).
- We in-licensed DISC-3405 from Mabwell Therapeutics in 2022. The US FDA accepted the IND for DISC-3405 in November 2022 and granted it Fast Track Designation in September 2023. We initiated a phase 1 study in healthy volunteers in October 2023.