Disc Medicine Presents Positive Results from Phase 1 Clinical Study of DISC-0974 in Healthy Volunteers at the 2022 EHA Annual Congress

  • First-in-human study of DISC-0974 establishes inhibition of hemojuvelin (HJV) co-receptor as a novel approach to target hepcidin, the master regulator of iron homeostasis
  • Data demonstrate robust increases in serum iron and improvements in markers of erythropoiesis including statistically significant increases of hemoglobin at the highest dose 
  • Disc plans to initiate separate clinical studies in patients with myelofibrosis and in patients with non-dialysis dependent chronic kidney disease mid-2022

WATERTOWN, MA, USA I June 10, 2022 I Disc Medicine, a biotechnology company dedicated to the discovery and development of novel therapeutic candidates for serious and debilitating hematologic diseases, today presented the results from its phase 1 clinical study of DISC-0974, a first-in-class, monoclonal antibody designed to suppress hepcidin production by inhibiting hemojuvelin (HJV), at the European Hematology Association (EHA) Congress being held in Vienna, Austria. The study data demonstrated robust increases in serum iron and markers of erythropoiesis, decreases in hepcidin levels, and that DISC-0974 was well-tolerated in healthy volunteers, consistent with the mechanism of action.

"We are very excited by the robust activity and encouraging initial safety profile of DISC-0974 observed in this study and which provide strong validation for our approach to targeting the hepcidin axis," said John Quisel, J.D., Ph.D., Chief Executive Officer. "Having established clinical proof-of-mechanism, we intend to develop DISC-0974 across a wide range of anemias caused by elevated hepcidin, beginning with studies in patients with myelofibrosis and in patients with non-dialysis dependent chronic kidney disease, both of which we plan to initiate this year."

The phase 1 study was a double-blind, placebo-controlled, single-ascending trial in healthy volunteers to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of DISC-0974. In the study, 34 healthy, adult subjects received either a single dose of DISC-0974 at escalating dose levels (7 mg IV, 14 mg SC, 28 mg SC, or 56 mg SC) or placebo. The study included endpoints to assess pharmacodynamic activity including serum hepcidin, serum iron and transferring saturation, ferritin levels, and measures of erythropoiesis.

Key data presented:

  • Rapid, dose-dependent and sustained decrease in serum hepcidin and a corresponding, robust increase in measures of circulating iron, including more than a doubling of transferrin saturation from baseline at the highest dose level
  • Changes in serum iron also corresponded with markers of iron mobilization and erythropoiesis, including decreased ferritin levels, increased reticulocyte hemoglobin, and increased mean corpuscular hemoglobin
  • At the 56 mg SC dose level, a single administration of DISC-0974 resulted in a statistically significant improvement in hemoglobin compared to placebo (+1.1 g/dL, p=0.009) at Day 42 and a marked increase in red blood cell count
  • DISC-0974 was well-tolerated at all dose levels with no serious or severe adverse events, no adverse events leading to study withdrawal, and no adverse event greater than Grade 1
  • Plasma exposure was dose-related in the 14 to 56 mg SC range and effects were observed through 28 days post-dose, indicating a sustained and clinically meaningful duration of action

"Hepcidin has long been known as the master regulatory hormone of systemic iron homeostasis. However, despite its clear biological importance and role in driving anemia across a host of diseases, no agents that primarily target excess hepcidin have been successfully developed," said Elizabeta Nemeth, Ph.D., Professor of Medicine at the David Geffen School of Medicine at UCLA, and Director of the UCLA Center for Iron Disorders. "The dramatic effects of DISC-0974 on iron and erythropoiesis in this study of healthy volunteers are encouraging and I look forward to following its progress in future clinical trials."

These data were presented at the European Hematology Association Annual Congress in Vienna and the poster is available on the EHA Congress platform at www.ehaweb.org.

About DISC-0974

DISC-0974 is an investigational, first-in-class monoclonal antibody designed to suppress hepcidin production by inhibiting the hemojuvelin (HJV) co-receptor, a highly selective and critical target of the hepcidin pathway with biological activity that has been validated by human genetic evidence. Hepcidin is the primary regulatory hormone of iron homeostasis and plays a central role by restricting iron absorption and preventing deployment from internal iron stores. DISC-0974 is currently being studied in a phase 1 clinical study of healthy volunteers and is being developed as a potential treatment for anemia of inflammation by suppressing hepcidin and enhancing iron availability for erythropoiesis.

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

About Disc Medicine

Disc Medicine is a clinical-stage biopharmaceutical company that is dedicated to transforming the lives of patients with hematologic disorders. We are building a portfolio of innovative, first-in-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 hematologic disease. For more information, please visit www.discmedicine.com

SOURCE: Disc Medicine

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