Disc Medicine Initiates Phase 1b/2 Clinical Study of DISC-0974 in Patients With Myelofibrosis and Severe Anemia

  • Open-label study will evaluate the potential of DISC-0974, a first-in-class anti-HJV (hemojuvelin) monoclonal antibody, to improve anemia in patients with myelofibrosis
  • DISC-0974 is administered as a subcutaneous injection once a month and is designed to suppress hepcidin, a key contributor to anemia in myelofibrosis and other inflammatory diseases
  • DISC-0974 has previously been shown to decrease hepcidin levels, increase serum iron, and improve markers of erythropoiesis, including hemoglobin, in a Phase 1 study in healthy volunteers

CAMBRIDGE, Mass., June 23, 2022 /PRNewswire/ — Disc Medicine, a clinical-stage biotechnology company dedicated to the discovery and development of novel therapeutic candidates for serious and debilitating hematologic diseases, today announced the initiation of a phase 1b/2 study to evaluate the efficacy and safety of DISC-0974 in myelofibrosis (MF) patients with anemia. DISC-0974 is a monoclonal antibody designed to suppress hepcidin by inhibiting the co-receptor for hemojuvelin (HJV), thereby treating anemia by improving the availability of iron for erythropoiesis.

“The initiation of this study in MF patients with anemia is an important step in the clinical development of DISC-0974 and builds on the encouraging results we observed in our study in healthy volunteers,” said declared Jean Quiselle, JD, PhD, CEO of Disc Medicine. “This also marks the first of several patient studies we plan to launch this year in our portfolio, and I am proud of our team for this achievement.”

The study will be an open label, multicenter, phase 1b/2 and will evaluate the safety, tolerability and efficacy of DISC-0974 in myelofibrosis patients with anemia. Endpoints for the study will include hepcidin levels, serum iron and markers of iron mobilization and measures of anemia benefits such as hemoglobin, reductions in transfusion burden and rate. of transfusion independence (TI). The study allows the recruitment of patients receiving stable disease-modifying therapy, including Janus Kinase (JAK) inhibitors. The study will take place in two parts:

  • Phase 1b (Dose escalation): Increasing Monthly Doses of DISC-0974 Administered for Six Months to MF Patients with Anemia (Hb < 10 g/dL), Where a Dose Level Will Be Selected Based on Optimal Increases in Hemoglobin and serum iron;
  • Phase 2 (expansion stage): Multiple doses of DISC-0974 given once monthly at the dose level selected in phase 1b portion of the study to MF patients with anemia who are transfusion dependent (TD) per the IWG-MRT criteria, defined as receiving >6 units of RBC over a 12 week period.

“There is a huge need for new therapies that specifically address the anemia in patients with MF, which is severe and progressively worsens over time such that almost all patients eventually require chronic red blood cell transfusions” , said Srdan Verstovsek, MD, PhD, professor of medicine at La University of Texas MD Anderson Cancer Center. “In addition, current treatments tend to exacerbate anemia, which limits optimal treatment or is a cause of treatment failure. We are excited to participate in this clinical trial, as there is growing evidence that hepcidin is a key contributor to anemia in myelofibrosis.

About DISC-0974

DISC-0974 is an investigational, first-in-class monoclonal antibody designed to suppress hepcidin production by inhibiting the hemojuvelin co-receptor (HJV), a highly selective and critical target of the hepcidin pathway with a biological activity that has been validated by human genetics. evidence. Hepcidin is the main regulatory hormone of iron homeostasis and plays a central role in limiting iron absorption and preventing the deployment of internal iron stores. DISC-0974 is designed to suppress hepcidin production and increase iron availability for erythropoiesis and is being developed as a potential treatment for multiple forms of inflammatory anemia. DISC-0974 is currently undergoing a phase 1 clinical study of DISC-0974 in healthy volunteers and a phase 1b/2 study in patients with myelofibrosis (MF) with severe anemia.

DISC-0974 is an investigational therapy that is not approved for use in any country. Disc obtained worldwide rights to DISC-0974 and related molecules under a licensing agreement from AbbVie in October 2019.

About Myelofibrosis Anemia

Myelofibrosis (MF) is a rare chronic blood cancer that currently affects approximately 16,000 to 18,500 patients in United States only. Severe, progressive and treatment-resistant anemia is the main clinical manifestation of MF and at the time of diagnosis over 80% of patients with MF have anemia, which progressively worsens and eventually renders the majority of patients dependent on chronic red blood cell transfusions. There are currently no approved treatments to treat MF anemia. Recent studies have shown hepcidin to be a key molecular driver of anemia in myelofibrosis. Hepcidin is approximately 12-fold elevated in patients with MF and correlates with disease severity, anemia, and the need for red blood cell transfusions.

About disc medicine

Disc Medicine is a clinical-stage biopharmaceutical company dedicated to transforming the lives of patients with blood disorders. We are building a portfolio of innovative, first-class therapeutic candidates that affect fundamental pathways of red blood cell biology. Disc Medicine is committed to developing treatments that empower and bring hope to the many patients who suffer from blood diseases. For more information, please visit www.discmedicine.com.

SOURCE Drug Disc

Leave a Comment