Disc Medicine's DISC-0974 heads to Q4 topline after ASCO data on 61 patients
RALLY-MF's transfusion-independence and hemoglobin-response readout will test whether early anemia gains in myelofibrosis hold up as the full Phase 2 cohort matures.
Executive Summary
- Disc Medicine is heading toward a topline data readout for its anemia-of-myelofibrosis antibody program, building on an interim look already shown at a major oncology meeting.
- The trial tests whether suppressing a liver-derived iron-regulating pathway can make anemic myelofibrosis patients transfusion-independent or meaningfully raise their hemoglobin, a question with no approved anemia-specific answer today.
- Enrollment reached its target and the completion timeline has held steady since a 2024 revision, so the readout's timing rests on disclosure choice rather than execution risk.
- No other program in clinical testing shares this trial's mechanism, so the data will stand largely alone as the field's first read on this specific approach to myelofibrosis-associated anemia.
The catalyst and its evidence base
Disc Medicine, Inc. said it will share updated Phase 2 data from RALLY-MF, its trial of DISC-0974 in anemia of myelofibrosis, in an oral presentation at the ASCO Annual Meeting covering 61 patients with data through April 27, 2026. That presentation is now the most current disclosed evidence on the program; the company separately guided that full topline results from the same trial, registered as NCT05320198, will follow in the fourth quarter of 2026, alongside an end-of-Phase-2 meeting with the FDA expected by year end. The ASCO data represent an interim cut, not the trial's primary analysis, so the Q4 topline readout is the event that will determine what the program's actual endpoint results show. DiscDisc Medicine Reports First Quarter 2026 Financial Results and Provides Business UpdateMay 5, 2026
Probability of SuccessBased on the AppliedXL Probability of Success model. For more information about the methodology, read the research here.
What the trial measures
RALLY-MF is designed as three separate response definitions layered by transfusion burden. In the high-transfusion-burden cohort, the endpoint is transfusion independence, meaning no packed red blood cell transfusions over any rolling 12-week period while maintaining a hemoglobin floor of 7 g/dL. The low-burden cohort uses the same transfusion-independence definition over a 16-week window. Patients who are not transfusion-dependent are assessed on a composite anemia response: no transfusions over 12 weeks combined with a mean hemoglobin increase of at least 1.5 g/dL from baseline. DISC-0974 is dosed subcutaneously and works by suppressing hepcidin production through blockade of hemojuvelin, a mechanism aimed at freeing up iron for red blood cell production in patients whose anemia is driven by inflammation-linked iron restriction rather than iron deficiency. NCT05320198Study of DISC-0974 (RALLY-MF) in Participants With Myelofibrosis or Myelodysplastic Syndrome and AnemiaNCT05320198
Enrollment and timeline
The trial's target enrollment was raised from 56 to 150 patients in December 2024, the same date its primary completion date moved from October 2024 to September 2026. Enrollment has since held at the 150-patient target with no further change, which the trial's own operational-risk framework treats as a routine, in-band metric rather than a signal of distress. The primary completion date has likewise stayed fixed at September 1, 2026 since that 2024 revision, meaning the trial has held its most recent completion guidance for over 18 months heading into the reported Q4 2026 topline window. NCT05320198Study of DISC-0974 (RALLY-MF) in Participants With Myelofibrosis or Myelodysplastic Syndrome and AnemiaNCT05320198
The competitive frame
No other clinical-stage program targets hemojuvelin or hepcidin suppression in myelofibrosis, leaving DISC-0974 without a direct mechanistic comparator in this indication. The broader myelofibrosis field is active on other axes: sponsors are running Phase 3 trials of a BET inhibitor measuring symptom score, a telomerase inhibitor measuring overall survival, and an activin-pathway agent measuring red-cell transfusion independence, none of which shares DISC-0974's target. With no validated disease-modifying anemia therapy specific to myelofibrosis on the market, a topline result showing transfusion independence or a sustained hemoglobin rise that extends beyond the interim ASCO snapshot would be the finding that distinguishes this mechanism within a field otherwise pursuing symptom score, survival, or transfusion endpoints through unrelated biology.
This analysis was produced using AI-assisted reporting systems, AppliedXL data, and official public records. These systems undergo editorial review, quality checks, and regular audits by human experts. Errors may still occur, as with any automated system. Always consult the linked primary sources. Read our AI Editorial Policy.