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. Manufacturing is underway and we expect to initiate a phase 2 study in erythropoietic porphyria patients in 2022.

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 and expect to initiate a first-in-human clinical study mid-2021.

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.