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.

Stage

Discovery

Preclinical

Clinical

Bitopertin (GlyT1 Inhibitor) – Heme Synthesis Modulation

  • 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 initiated a phase 2 study in erythropoietic porphyria patients in 2022 and are planning additional studies in Diamond-Blackfan Anemia and other indications. 

DISC-0974 (anti-HJV mAb) – Hepcidin Suppression

  • 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, initially for anemia of myelofibrosis.
  • 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 healhty volunteeers in 2021, a phase 1b / 2 study in patents with anemia of myelofibrosis in 2022 and are planning additional studies in patients with anemia of chronic kidney disease (CKD) and other indications. 

Matriptase-2 Inhibitor Program – Hepcidin Induction

  • Our preclinical program to develop a first-in-class, orally administered inhibitor of matriptase-2 (TMPRSS6) is designed to increase endogenous expression of hepcidin. There is increasing evidence suggesting therapeutic hepcidin has applications in a wide range of diseases, including myeloproliferative neoplasms, beta-thalassemia, and myelodysplastic syndromes. Our team has identified potent inhibitors of matriptase-2 that increase hepcidin production and reduce serum iron.