Amir SohailWATERTOWN, Mass., (ZEEST MEDIA) — Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, today announces updated data from multiple clinical programs to be presented at the EHA Annual Meeting in Stockholm, Sweden. Data from the RALLY-MF trial of DISC-0974 in patients with MF and anemia, to be presented in an oral session today, demonstrate meaningful, durable overall anemia responses across all patient subgroups, regardless of baseline transfusion status or concomitant JAK inhibitor use. Updated data from the HELIOS open-label extension trial of bitopertin in EPP, to be presented in a poster session tomorrow, June 13, show sustained reductions in PPIX, significant improvement in light tolerance measures, and favorable longer-term safety in patients treated with bitopertin.
“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, J.D., Ph.D., President and Chief Executive Officer of Disc Medicine. “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. For bitopertin, continued durability of PPIX reduction and light tolerance improvement in HELIOS is encouraging leading up to the APOLLO Phase 3 readout in Q4, which, as confirmed in a recent Type A meeting with FDA, can serve as the basis for a response to the CRL and could potentially support a traditional approval if successful. We also look forward to sharing initial data from the RESTORE-PV Phase 2 trial of DISC-3405 in polycythemia vera in Q4 this year, setting up the potential for a third program in pivotal-stage development in 2027.”
Management will host a call following the EHA meeting to review highlights of the presented data and next steps for the company on Monday, June 15 at 8:00am EDT. Please register for the event on the Events and Presentations page of Disc’s website (https://ir.discmedicine.com/).
Bitopertin, DISC-0974, and DISC-3405 are investigational agents and are not approved for use as therapies in any jurisdiction worldwide.
Details of Presentations and Abstracts:
DISC-0974: RALLY-MF Oral Presentation
RALLY-MF, an ongoing Phase 2 open-label study, had enrolled 61 adult patients with MF and anemia as of the data cutoff date of April 27, including 50 patients with sufficient follow up to be included in the responder analysis (non-transfusion dependent receiving no transfusions (nTD, n=31), transfusion dependent with low transfusion burden (TD Low, n=11) and transfusion dependent with high transfusion burden (TD High, n=8)). The trial was comprised of both patients receiving concomitant JAK inhibitor therapy (n=25) and not receiving JAK inhibitor therapy (n=25). DISC-0974 was administered subcutaneously at 50 mg every 4 weeks for up to 6 treatments. The updated results demonstrated:
- Consistent, substantial decreases in hepcidin reaching >75% reduction from baseline and corresponding increases in serum iron
- 55% (N=17 of 31) of baseline nTD patients achieved a hemoglobin increase of ≥1.5 g/dL for ≥12 weeks (major response) and 68% had an increase of ≥1 g/dL for ≥12 weeks (overall response)
- 64% (N=7 of 11) of TD Low patients achieved transfusion independence (TI, major response) over a 16-week period and 73% achieved a ≥50% reduction in transfusions (overall response)
- 50% (N=4 of 8) of TD High patients achieved transfusion independence (TI, major response) over a 12-week period and 88% achieved a ≥50% reduction in transfusion requirement (overall response)
- 56% of patients receiving concomitant JAK inhibitor therapy achieved a major hematologic response across transfusion groups and 72% achieved an overall response, with similar response rates regardless of which specific JAK inhibitor the patient received
- Dosing with DISC-0974 was associated with improvements in patient-reported outcomes:
- Clinically significant improvements in FACIT-Fatigue scores in nTD and TD Low participants that were correlated with hemoglobin change
- MPN-SAF TSS50 at EOS was achieved by 50% of nTD and TD low major responders
- DISC-0974 was generally well-tolerated. Diarrhea, not considered serious, was the only adverse event (AE) that was reported as related to DISC-0974 and reported in two or more subjects. The majority of AEs were not considered related to DISC-0974.
Bitopertin: HELIOS update poster
HELIOS is an ongoing Phase 2, open-label, long-term extension trial that enrolled 86 adult and adolescent patients with EPP from the BEACON and AURORA trials. Patients were randomized to receive 20 mg or 60 mg bitopertin in BEACON and 20 mg or 60 mg bitopertin or placebo in AURORA, with all patients transitioning to a 60 mg daily dose of bitopertin in HELIOS.
- Longer term treatment with bitopertin was associated with sustained reductions in the disease-causing toxin PPIX, with additional benefit for patients receiving the 60 mg dose continuously
- Treatment with bitopertin was associated with sustained, significant improvement in average light tolerance and time to prodrome measures
- Bitopertin exhibited a favorable longer-term safety profile with up to 2.5+ years of exposure and similar safety across adults and adolescents with EPP and XLP
DISC-3405: RESTORE-PV Trial-in-Progress poster
RESTORE-PV is a Phase 2 open-label study of the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of DISC-3405 in patients with polycythemia vera. Initial data from the trial is expected in Q4 2026.
About Disc Medicine
Disc Medicine (NASDAQ:IRON) is a clinical-stage biopharmaceutical company committed to discovering, developing, and commercializing novel treatments for patients who suffer from serious hematologic diseases. We are building a portfolio of innovative, potentially first-in-class therapeutic candidates that aim to address a wide spectrum of hematologic diseases by targeting fundamental biological pathways of red blood cell biology, specifically heme biosynthesis and iron homeostasis. For more information, please visit www.discmedicine.com.
Disc Cautionary Statement Regarding Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, express or implied statements regarding Disc’s expectations with respect to the next stages of its development programs for bitopertin, DISC-0974 and DISC-3405, including projected timelines for the initiation and completion of its clinical trials, anticipated timing of release of data, and other clinical activities; the registrational pathway for bitopertin, including the potential for traditional approval, the potential for the APOLLO clinical trial to serve as the basis for any such approval, and the timing of any such approval, if granted; and anticipated discussions with regulatory agencies. The use of words such as, but not limited to, “believe,” “expect,” “estimate,” “project,” “intend,” “future,” “potential,” “continue,” “may,” “might,” “plan,” “will,” “should,” “seek,” “anticipate,” or “could” or the negative of these terms and other similar words or expressions that are intended to identify forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on Disc’s current beliefs, expectations and assumptions regarding the future of Disc’s business, future plans and strategies, clinical results and other future conditions. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements.
Disc may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and investors should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements as a result of a number of material risks and uncertainties including but not limited to: the adequacy of Disc’s capital to support its future operations and its ability to successfully initiate and complete clinical trials; the nature, strategy and focus of Disc; the difficulty in predicting the time and cost of development of Disc’s product candidates; Disc’s plans to research, develop and commercialize its current and future product candidates; the timing of initiation of Disc’s planned preclinical studies and clinical trials; the timing of the availability of data from Disc’s clinical trials; Disc’s ability to identify additional product candidates with significant commercial potential and to expand its pipeline in hematological diseases; the timing and anticipated results of Disc’s preclinical studies and clinical trials and the risk that the results of Disc’s preclinical studies and clinical trials may not be predictive of future results in connection with future studies or clinical trials and may not support further development and marketing approval; and the other risks and uncertainties described in Disc’s filings with the Securities and Exchange Commission, including in the “Risk Factors” section of Disc’s Annual Report on Form 10-K for the year ended December 31, 2025, and in subsequent Quarterly Reports on Form 10-Q. Any forward-looking statement speaks only as of the date on which it was made. None of Disc, nor its affiliates, advisors or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as result of new information, future events or otherwise, except as required by law.
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