Disc Medicine Shares Positive Clinical Data at EHA 2024 Congress

Disc Medicine, Inc., a clinical-stage biopharmaceutical company, has unveiled promising data from several ongoing studies at the European Hematology Association (EHA) 2024 Congress. These results span various investigational treatments aimed at addressing serious hematologic diseases, including bitopertin for erythropoietic protoporphyria (EPP), DISC-0974 for myelofibrosis (MF) anemia, and DISC-3405 in healthy volunteers.

Bitopertin in EPP

The AURORA study, a Phase 2 clinical trial, demonstrated significant clinical benefits of bitopertin in EPP. The double-blind, placebo-controlled trial involved 75 adult subjects, who were randomized to receive either 20 mg or 60 mg of bitopertin, or a placebo, daily for 17 weeks. Key findings include:

- Reduction in Protoporphyrin IX (PPIX): Subjects receiving 60 mg of bitopertin showed a 40% reduction in PPIX levels compared to placebo.
- Improved Light Tolerance: Both 20 mg and 60 mg dose groups exhibited significant improvements in light tolerance, with a twofold enhancement in light exposure without pain relative to baseline.
- Phototoxic Reactions: There was a statistically significant decrease in phototoxic reactions, particularly in the 60 mg dose group, which showed a 75.3% reduction.
- Patient Global Impression of Change (PGIC)**: Significant dose-dependent improvements were noted, especially for the 60 mg group.
- Overall Safety: Bitopertin was generally well-tolerated, with no serious adverse events reported.

The BEACON study's full adult data set corroborated AURORA's findings, showing consistent clinical activity and reinforcing bitopertin's potential as a treatment for EPP.

DISC-0974 in MF Anemia

Updated data from a Phase 1b trial of DISC-0974 in MF patients with severe anemia demonstrated promising results. Conducted as an open-label, ascending-dose clinical trial, the study enrolled both transfusion-dependent and non-transfusion dependent patients. Key outcomes include:

- Hematologic Response: Approximately 68.9% of non-transfusion dependent participants experienced a hemoglobin increase of ≥1.5 g/dL, sustained for at least 12 weeks in many cases.
- Transfusion Reduction: All evaluable participants with baseline transfusion requirements saw at least a 50% reduction in transfusions over an 8-week period.
- Durability: Substantial and sustained reductions in hepcidin levels and increases in iron were observed following each dose.
- Overall Safety: DISC-0974 was generally well-tolerated across all dose levels.

DISC-3405 in Healthy Volunteers

Initial data from a Phase 1 trial of DISC-3405 in healthy participants demonstrated the drug's potential as an effective iron restriction agent. Key data presented include:

- Hepcidin Induction: A dose-dependent increase in hepcidin was observed, along with corresponding reductions in serum iron.
- Iron Reduction: Doses of 150 mg and 300 mg achieved over 50% reduction in mean serum iron, sustained for at least 4 weeks in the highest dose group.
- Safety Profile: DISC-3405 was well-tolerated, with no serious adverse events reported.

Disc Medicine's President and CEO, John Quisel, emphasized the significance of these findings, which collectively underscore the potential of their therapeutic programs. "We are excited to see the robust clinical activity across our portfolio, which strengthens our confidence in advancing these treatments," he stated.

These investigational treatments are not yet approved for therapeutic use, and further studies are needed to confirm their efficacy and safety. Nonetheless, the positive data presented at the EHA 2024 Congress highlight Disc Medicine's ongoing commitment to addressing unmet needs in hematologic diseases.

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