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Highlights
- DISC-0974 demonstrates substantial reductions in hepcidin and increases in iron levels, leading to significant improvements in anemia across multiple patient types.
- 68% of non-transfusion dependent patients experienced a ≥1.5 g/dL increase in hemoglobin, with 50% sustaining this for ≥12 weeks.
- DISC-0974 was well-tolerated, with the only notable adverse event being mild diarrhea, and 54% of JAK inhibitor-treated patients achieved a major hematologic response.
Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage biopharmaceutical company, presented promising updated results from a Phase 1b trial of DISC-0974 at the 2024 American Society of Hematology (ASH) annual meeting in San Diego, CA. The trial evaluated the efficacy and safety of DISC-0974 in patients with myelofibrosis (MF) and anemia, showcasing substantial benefits for patients with varying transfusion needs.
The Phase 1b study, a multi-center, open-label trial, included 35 adult patients with MF and anemia. Patients were classified into three groups: non-transfusion dependent (nTD, n=23), transfusion dependent with low transfusion burden (TD Low, n=5), and transfusion dependent with high transfusion burden (TD High, n=7). Some patients were receiving concomitant JAK inhibitor therapy (n=13), while others were not (n=22). DISC-0974 was administered subcutaneously every 4 weeks in doses ranging from 14 mg to 100 mg for up to 6 treatments.
Key Findings:
- Reduction in Hepcidin and Iron Increases: DISC-0974 led to consistent reductions in hepcidin by over 75%, resulting in corresponding increases in serum iron levels. This, in turn, contributed to improved hemoglobin and reticulocyte hemoglobin levels.
- Hemoglobin Increases in nTD Patients: 68% of nTD patients achieved a ≥1.5 g/dL increase in hemoglobin, with 50% maintaining these increases for at least 12 weeks.
- Transfusion Independence in TD Patients: 100% of TD Low patients experienced a ≥50% reduction in transfusion requirements, with 80% achieving transfusion independence over a 16-week period. Similarly, 60% of TD High patients had a ≥50% reduction in transfusion need, and 40% achieved transfusion independence.
- JAK Inhibitor Therapy Impact: Among patients receiving concomitant JAK inhibitor therapy, 54% achieved a major hematologic response, further suggesting the potential for DISC-0974 in combination treatments.
Safety and Tolerability: DISC-0974 was well-tolerated across all dose levels. The most common adverse event, diarrhea, was observed in two or more patients but was not considered severe. Most adverse events were unrelated to the drug, demonstrating a favorable safety profile for DISC-0974.
