Our Pipeline

We are committed to bringing hope to patients who suffer 
from hematologic diseases through innovation. Our team 
has built a pipeline of first-in-class therapies that each 
have the potential to address a wide array of hematologic 
diseases, ranging from rare genetic disorders to widely 
prevalent anemias associated with chronic disease.

Program

Preclinical

Phase 1

Phase 2

Erythropoietic Porphyrias
(EPP and XLP)

Erythropoietic Porphyrias
(EPP and XLP)

Diamond-Blackfan Anemia
and other indications

Diamond-Blackfan Anemia
and other indications

  • 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.

Anemia of Myelofibrosis (MF)

Anemia of Myelofibrosis (MF)

Anemia of Chronic Kidney Disease (CKD)

Anemia of Chronic Kidney Disease (CKD)

Anemia Associated with Inflammatory Diseases

Anemia Associated with Inflammatory Diseases

  • 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.

Currently in Phase 1 in healthy volunteers
Potential to treat polycythemia vera and
diseases of iron overload

Currently in Phase 1 in healthy volunteers
Potential to treat polycythemia vera and
diseases of iron overload

  • 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

Erythropoietic Porphyrias
(EPP and XLP)

Diamond-Blackfan Anemia
and other indications

  • 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

Anemia of Myelofibrosis (MF)

Anemia of Chronic Kidney Disease (CKD)

Anemia Associated with Inflammatory Diseases

  • 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

Currently in Phase 1 in healthy volunteers
Potential to treat polycythemia vera and
diseases of iron overload

  • 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.
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