Disc Medicine Shares Positive Phase 1b Results in CKD and Anemia at 2024 ASN Kidney Week

WATERTOWN, Mass., October 25, 2024 – Disc Medicine, Inc. (NASDAQ:IRON), a biopharmaceutical company in the clinical stage, has showcased promising new data from an ongoing Phase 1b clinical trial of DISC-0974. This study involves patients with non-dialysis-dependent chronic kidney disease (NDD-CKD) who also suffer from anemia, focusing particularly on the 40 mg and 60 mg single ascending dose (SAD) cohorts. The results, presented at the 2024 American Society of Nephrology (ASN) Kidney Week in San Diego, CA, indicate that a single dose of DISC-0974 can produce lasting suppression of hepcidin, enhance iron mobilization, and lead to increased erythropoiesis and hemoglobin levels in NDD-CKD patients with anemia.

John Quisel, JD, PhD, President and CEO of Disc Medicine, expressed satisfaction with these findings, noting that they provide the first clinical evidence showing that reducing hepcidin in CKD anemia can lead to increased erythropoiesis and hemoglobin. He emphasized the significance of observing these effects after just one dose, which will facilitate the exploration of dosing regimens in the multiple-dose stage of the Phase 1b study. Quisel also highlighted the broader potential of DISC-0974 in treating anemia across various inflammatory and chronic diseases.

In this SAD study, patients with Stage 2-5 NDD-CKD were administered either a placebo (n=7) or DISC-0974 subcutaneously at doses of 28 mg (n=9), 40 mg (n=6), or 60 mg (n=6). The dose escalation is still ongoing. The interim data revealed notable findings including:

- A significant, dose-dependent reduction in hepcidin levels from baseline compared to placebo across all dose levels, with a median reduction exceeding 75% at the highest dose.
- A substantial and sustained increase in transferrin saturation from baseline compared to placebo across all dose levels, with a median increase up to three times the baseline at the highest dose.
- An early and sustained increase in mean reticulocyte hemoglobin from baseline across all dose groups through Day 22 and beyond, with maximal mean values of +1.14 pg at 28 mg, +1.49 pg at 40 mg, and +1.53 pg at 60 mg, compared to +0.21 pg on placebo.
- An increase in mean hemoglobin from baseline across dose groups during the study period, exceeding placebo effects: +0.35 g/dL at 28 mg, +0.54 g/dL at 40 mg, and +0.55 g/dL at 60 mg. The maximal mean increase in hemoglobin was +0.8 g/dL at 40 mg and +0.7 g/dL at 60 mg, compared to +0.2 g/dL on placebo.
- The highest observed individual increase in hemoglobin levels was up to +0.95 g/dL at 28 mg, +1.5 g/dL at 40 mg, and +1.8 g/dL at 60 mg.

The safety and tolerability profile of DISC-0974 was acceptable at all evaluated dose levels. Most adverse events were deemed unrelated to DISC-0974, and all treatment-related adverse events were assessed as Grade 1 or 2.

DISC-0974 remains an investigational agent and has not been approved for therapeutic use in any jurisdiction.

Anemia of chronic kidney disease (CKD) is a widespread and serious condition that affects millions globally, particularly in the U.S. where it impacts approximately 5 to 6 million patients. CKD leads to the progressive loss of kidney function, potentially resulting in end-stage renal disease or kidney failure. Anemia in CKD patients is typically caused by their inability to produce sufficient red blood cells and hemoglobin, leading to symptoms like fatigue, shortness of breath, and reduced physical and cognitive function. Elevated levels of hepcidin in CKD patients hinder erythropoiesis by restricting the availability of iron to developing red blood cells. Due to the complexity and safety concerns of current anemia treatments, many CKD patients remain untreated, resorting to blood transfusions in severe cases which carry risks and complicate eligibility for kidney transplantation.

Disc Medicine, a clinical-stage biopharmaceutical company, is dedicated to developing and commercializing innovative treatments for serious hematologic diseases. Their portfolio includes potentially first-in-class therapeutic candidates targeting fundamental biological pathways of red blood cell biology, specifically focusing on heme biosynthesis and iron homeostasis.