Injection therapy eases fatigue for those with cancer-related anemia

Treatment with Disc Medicine’s investigational therapy DISC-0974 was tied to easing of fatigue, as well as a drop in overall symptoms, among certain myelofibrosis patients with cancer-associated anemia in a clinical trial called RALLY-MF.

Updated results from the trial’s Phase 2 portion show that half of the major responders who either did not require blood transfusions or had low transfusion needs achieved at least a 50% reduction in their total symptom score. Further, “meaningful, durable overall anemia responses across all patient subgroups” were seen in the study, conducted in the U.S. and Australia, Disc stated in a company press release.

These findings add to previously reported data showing that DISC-0974 led to lasting anemia responses for participants regardless of transfusion needs, and among patients receiving JAK inhibitors, a class of therapies commonly used to treat myelofibrosis.

The updated results were presented at the 2026 European Hematology Association (EHA) Annual Meeting, held last month in Stockholm. Disc now plans to meet with the U.S. Food and Drug Administration by the end of the year to discuss the Phase 2 findings and possible next steps in DISC-0974’s clinical development.

“The updates at this year’s EHA highlight continued progress across our portfolio heading into a catalyst-rich second half of the year,” said John Quisel, PhD, Disc’s president and CEO.

A rare blood cancer, myelofibrosis develops when scar tissue forms in the bone marrow — the soft, spongy tissue inside bones — and disrupts the production of healthy blood cells.

Many people with myelofibrosis develop anemia, meaning they have too few healthy red blood cells. Anemia is marked by fatigue, weakness, shortness of breath, and, for some patients, a need for regular blood transfusions.

Trial testing whether DISC-0974 can ease fatigue, other symptoms

DISC-0974 is an antibody therapy that’s designed to block hemojuvelin, a protein involved in producing the iron-regulating hormone hepcidin.

Hepcidin levels are often abnormally high in people with myelofibrosis. This traps iron in storage sites, limiting the amount available for producing red blood cells. By reducing hepcidin, DISC-0974 is intended to increase iron availability and improve red blood cell production.

The Phase 1/2 RALLY-MF (NCT05320198) study is testing DISC-0974 in adults with myelofibrosis and anemia. In the trial, the therapy is given as a 50 mg injection under the skin every 4 weeks for up to 6 treatments.

The Phase 2 portion of the study enrolled 61 participants, of whom 50 had sufficient follow-up data to be included in the response analysis. Previously reported results showed durable anemia responses regardless of transfusion status or JAK inhibitor use.

The data presented at EHA placed additional emphasis on patient-reported outcomes, or patients’ own assessments of how the disease and treatment affected their symptoms and daily lives.

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Fatigue was measured using the Functional Assessment of Chronic Illness Therapy–Fatigue (FACIT-Fatigue) scale. Clinically meaningful improvements were seen among participants who did not require transfusions and those with lower transfusion needs.

Lessening of fatigue also correlated with increases in hemoglobin, the protein in red blood cells that carries oxygen throughout the body. This means people whose hemoglobin improved tended to report greater reductions in fatigue.

The researchers also used the Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score, or MPN-SAF TSS, to assess common symptoms such as fatigue, abdominal discomfort, problems concentrating, night sweats, itching, bone pain, fever, and unintended weight loss.

At the end-of-study assessment, 50% of major responders who were not transfusion dependent or had low transfusion needs achieved MPN-SAF TSS50. This means their overall symptom score decreased by at least half from the start of the study.

Among participants included in the response analysis, 31 did not require transfusions, 11 had a low transfusion burden, and eight had a high transfusion burden. Half were receiving a JAK inhibitor while half were not.

Use of the experimental therapy reduced hepcidin levels by more than 75% from the study’s start and increased the amount of iron circulating in the blood. These changes eased anemia across patient groups, regardless of baseline transfusion needs.

“For DISC-0974 in MF anemia, our Phase 2 dataset continues to strengthen as we prepare for End of Phase 2 discussions with FDA by the end of this year,” Quisel said.

DISC-0974 was generally well tolerated. Diarrhea was the only side effect considered related to treatment. It was reported for at least two participants, but was not considered serious, according to the researchers. Most adverse events were considered unrelated to DISC-0974.