Disc Medicine Presents Positive Updated Results from Phase 1b Trial in Patients with Myelofibrosis (MF) and Anemia in an Oral Presentation at the 66th American Society of Hematology (ASH) Annual Meeting
Disc Medicine (NASDAQ:IRON) presented positive updated results from its Phase 1b trial of DISC-0974 in patients with myelofibrosis (MF) and anemia at the 2024 ASH annual meeting. The trial enrolled 35 adult patients across different transfusion dependency groups and demonstrated significant clinical benefits.
Key results include: 68% of non-transfusion dependent patients achieved hemoglobin increase ≥1.5 g/dL; 100% of low transfusion dependent patients achieved ≥50% reduction in transfusion needs, with 80% reaching transfusion independence; 60% of high transfusion dependent patients achieved ≥50% reduction in requirements, with 40% reaching independence. The treatment showed >75% reduction in hepcidin across patients.
DISC-0974 was generally well-tolerated, with diarrhea being the only related adverse event reported in multiple subjects. The company has initiated a Phase 2 study following these positive results.
Disc Medicine (NASDAQ:IRON) ha presentato risultati aggiornati positivi dal suo studio di Fase 1b di DISC-0974 in pazienti con mielofibrosi (MF) e anemia durante la riunione annuale ASH 2024. Lo studio ha arruolato 35 pazienti adulti provenienti da diversi gruppi con dipendenza da trasfusioni, dimostrando significativi benefici clinici.
I risultati principali includono: 68% dei pazienti non dipendenti da trasfusioni ha mostrato un aumento della emoglobina ≥1.5 g/dL; 100% dei pazienti con bassa dipendenza da trasfusioni ha ottenuto una riduzione ≥50% dei bisogni trasfusionali, con l'80% che ha raggiunto l'indipendenza trasfusionale; 60% dei pazienti con alta dipendenza da trasfusioni ha ottenuto una riduzione ≥50% delle richieste, con il 40% che ha raggiunto l'indipendenza. Il trattamento ha mostrato una riduzione >75% dell'epcidina tra i pazienti.
DISC-0974 è stato generalmente ben tollerato, con la diarrea come unica reazione avversa correlata riportata in più soggetti. L'azienda ha avviato uno studio di Fase 2 dopo questi risultati positivi.
Disc Medicine (NASDAQ:IRON) presentó resultados actualizados positivos de su ensayo de Fase 1b de DISC-0974 en pacientes con mielofibrosis (MF) y anemia en la reunión anual ASH 2024. El ensayo inscribió a 35 pacientes adultos de diferentes grupos de dependencia de transfusiones, demostrando beneficios clínicos significativos.
Los resultados clave incluyen: 68% de los pacientes no dependientes de transfusiones lograron un aumento de hemoglobina ≥1.5 g/dL; 100% de los pacientes con baja dependencia de transfusiones lograron una reducción ≥50% en sus necesidades transfusionales, con un 80% alcanzando independencia transfusional; 60% de los pacientes con alta dependencia de transfusiones lograron una reducción ≥50% en los requisitos, con un 40% alcanzando independencia. El tratamiento mostró una reducción >75% en hepcidina entre los pacientes.
DISC-0974 fue generalmente bien tolerado, siendo la diarrea el único evento adverso relacionado reportado en múltiples sujetos. La compañía ha iniciado un estudio de Fase 2 tras estos resultados positivos.
Disc Medicine (NASDAQ:IRON)은 2024 ASH 연례 회의에서 골수섬유증(MF) 및 빈혈 환자를 대상으로 한 DISC-0974의 1b 단계 시험에서 긍정적인 업데이트 결과를 발표했습니다. 이 시험은 다양한 수혈 의존 그룹에서 35명의 성인 환자를 등록하였고, 상당한 임상적 이점을 보여주었습니다.
주요 결과는 다음과 같습니다: 68%의 비수혈 의존 환자가 헤모글로빈 증가 ≥1.5 g/dL를 달성하였고; 100%의 낮은 수혈 의존 환자가 수혈 요구 사항에서 ≥50% 감소를 달성하였으며, 80%는 수혈 독립성을 달성하였습니다; 60%의 높은 수혈 의존 환자가 요구 사항에서 ≥50% 감소를 달성하였고, 40%는 독립성에 도달하였습니다. 치료는 환자들 사이에서 헤프시딘이 >75% 감소하는 것으로 나타났습니다.
DISC-0974는 일반적으로 잘 견디며, 설사가 여러 환자에서 보고된 유일한 관련 부작용이었습니다. 이 회사는 이러한 긍정적인 결과를 바탕으로 2단계 연구를 시작했습니다.
Disc Medicine (NASDAQ:IRON) a présenté des résultats positifs mis à jour de son essai de Phase 1b de DISC-0974 chez des patients souffrant de mielofibrose (MF) et d'anémie lors de la réunion annuelle de l'ASH 2024. L'essai a recruté 35 patients adultes de différents groupes de dépendance aux transfusions et a démontré des bénéfices cliniques significatifs.
Les résultats clés incluent: 68% des patients non dépendants des transfusions ont obtenu une augmentation de l'hémoglobine ≥1,5 g/dL; 100% des patients à faible dépendance transfusionnelle ont atteint une réduction ≥50% de leurs besoins transfusionnels, avec 80% atteignant l'indépendance transfusionnelle; 60% des patients à forte dépendance transfusionnelle ont obtenu une réduction ≥50% de leurs besoins, avec 40% atteignant l'indépendance. Le traitement a montré une réduction >75% de l'hepcidine chez les patients.
DISC-0974 a été généralement bien toléré, la diarrhée étant le seul événement indésirable associé rapporté chez plusieurs sujets. L'entreprise a lancé une étude de Phase 2 suite à ces résultats positifs.
Disc Medicine (NASDAQ:IRON) hat auf dem ASH-Jahrestreffen 2024 positive aktualisierte Ergebnisse aus seiner Phase-1b-Studie zu DISC-0974 bei Patienten mit Myelofibrose (MF) und Anämie präsentiert. Die Studie umfasste 35 erwachsene Patienten aus verschiedenen Transfusionsabhängigkeitsgruppen und zeigte signifikante klinische Vorteile.
Wichtige Ergebnisse umfassen: 68% der nicht transfusionsabhängigen Patienten erreichten einen Hämoglobin-Anstieg von ≥1,5 g/dL; 100% der Patienten mit niedriger Transfusionsabhängigkeit erreichten eine Reduktion ihrer Transfusionsbedürfnisse um ≥50%, wobei 80% die Transfusionsunabhängigkeit erreichten; 60% der hoch transfusionsabhängigen Patienten erreichten eine Reduktion ihrer Anforderungen um ≥50%, wobei 40% die Unabhängigkeit erreichten. Die Behandlung zeigte eine >75%ige Reduktion von Hepcidin bei den Patienten.
DISC-0974 wurde im Allgemeinen gut vertragen, wobei Durchfall das einzige berichtete relevante unerwünschte Ereignis war, das bei mehreren Patienten auftrat. Das Unternehmen hat nach diesen positiven Ergebnissen eine Phase-2-Studie initiiert.
- Strong efficacy with 68% of non-transfusion dependent patients achieving significant hemoglobin increase
- 100% of low transfusion dependent patients achieved ≥50% reduction in transfusion needs
- 60% of high transfusion dependent patients achieved ≥50% reduction in transfusion needs
- 54% of patients on JAK inhibitor therapy achieved major hematologic response
- Treatment well-tolerated with minimal adverse events
- Successfully advanced to Phase 2 trial
- None.
Insights
The Phase 1b trial results for DISC-0974 represent a significant breakthrough in treating myelofibrosis-associated anemia. The data shows remarkable efficacy with
The broad efficacy across different patient subgroups, including those on JAK inhibitors, suggests DISC-0974 could become a versatile treatment option. The favorable safety profile, with only diarrhea noted as a recurring treatment-related adverse event, strengthens its potential clinical utility. The initiation of Phase 2 trials indicates strong confidence in the drug's potential and accelerates its development timeline.
These positive trial results significantly strengthen Disc Medicine's market position in the hematology space. With a market cap of
The comprehensive efficacy data across patient subgroups expands the potential patient population and market opportunity. The drug's compatibility with JAK inhibitors is particularly valuable, as it could position DISC-0974 as a complementary rather than competitive therapy. This could facilitate faster market adoption and potential partnership opportunities with companies marketing JAK inhibitors.
- Demonstrated durable hematologic response across all patient subgroups, regardless of baseline transfusion status and concomitant JAK inhibitor therapy
- Phase 2 study in MF anemia has been initiated, enrolling a broad range of patient types
WATERTOWN, Mass., Dec. 08, 2024 (GLOBE NEWSWIRE) -- 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 presented positive updated results from a Phase 1b trial of DISC-0974 in patients with myelofibrosis (MF) and anemia. The data, presented in an oral session at the 2024 American Society of Hematology (ASH) annual meeting in San Diego, CA, demonstrated that treatment with DISC-0974 results in substantial reductions in hepcidin and increases in iron levels translating to positive impact on clinically meaningful measures of anemia across a broad range of patient types.
“With the presentation of this expanded data set from our phase 1b study, we are encouraged by the continued demonstration of robust hematologic activity of DISC-0974, including durable hemoglobin increases in all patient subgroups and meaningful reductions in transfusion burden. Importantly, we observed strong responses regardless of patients’ baseline transfusion burden or concomitant use of JAK inhibitors,” said John Quisel, JD, PhD, President and Chief Executive Officer of Disc Medicine. “With this data in hand, I’m pleased to announce that we have now started a phase 2 trial in myelofibrosis patients with anemia.”
This Phase 1b multi-center, open-label study, enrolled 35 adult patients with MF and anemia, including patients who were: non-transfusion dependent receiving no transfusions (nTD, n=23), transfusion dependent with low transfusion burden (TD Low, n=5) and transfusion dependent with high transfusion burden (TD High, n=7). The trial was comprised of both patients receiving concomitant JAK inhibitor therapy (n=13) and not receiving JAK inhibitor therapy (n=22). DISC-0974 was administered subcutaneously at 14 mg (n=1), 28 mg (n=7), 50 mg (n=12), 75 mg (n=9), or 100 mg (n=6) every 4 weeks for up to 6 treatments. Results demonstrated:
- Consistent, substantial decreases in hepcidin reaching >
75% reduction from baseline and corresponding increases in serum iron across patients, which translated to increased levels of reticulocyte hemoglobin and hemoglobin 68% of baseline nTD patients achieved a hemoglobin increase of ≥1.5 g/dL during study period and50% had sustained increases for ≥12 weeks100% of TD patients with 1-2 transfusions within a 12-week period at baseline (TD Low) achieved a ≥50% reduction in transfusion requirement80% of TD Low patients achieved transfusion independence (TI) over a 16-week period
60% of TD patients with 3-12 transfusions within a 12-week period at baseline (TD High) achieved a ≥50% reduction in transfusion requirement40% of TD High patients achieved transfusion independence over a 12-week period
54% of patients receiving concomitant JAK inhibitor therapy achieved a major hematologic response- DISC-0974 was generally well-tolerated at all evaluated dose levels. Diarrhea was the only adverse event (AE) that was considered related to DISC-0974 and reported in two or more subjects. The majority of AEs were not considered related to DISC-0974.
Management will host a call today, Sunday, December 8 at 9:00pm EST / 6:00pm PST to review highlights of data presented throughout the meeting and plans for next steps in development. Please register for the event on the Events and Presentations page of Disc’s website (https://ir.discmedicine.com/).
About DISC-0974
DISC-0974 is an investigational monoclonal antibody (mAb) targeting a BMP-signaling co-receptor called hemojuvelin (HJV) and is designed to suppress hepcidin production and increase serum iron levels in patients suffering from anemia of inflammation. DISC-0974 was in-licensed by Disc from AbbVie in 2019. Anemia of inflammation arises from abnormally elevated hepcidin and is the second most common form of anemia, affecting millions of patients in the US across numerous diseases such as chronic kidney disease, myelofibrosis, cancer, autoimmune diseases, and other conditions with an inflammatory component. Disc has established clinical proof-of-mechanism of DISC-0974 in a Phase 1 trial of healthy volunteers, completed a Phase 1b clinical trial in patients with myelofibrosis and anemia, and initiated a Phase 2 clinical trial of DISC-0974 in patients with MF anemia, as well as a Phase 1b/2a clinical trial of DISC-0974 in patients with chronic kidney disease and anemia who are not receiving dialysis.
DISC-0974 is an investigational agent and is not approved for use as a therapy in any jurisdiction worldwide.
About Anemia of Myelofibrosis
Myelofibrosis (MF) is a rare, chronic blood cancer that currently affects an estimated 25,000 patients in the United States alone. Severe, progressive, and treatment resistant anemia is the primary clinical manifestation of MF. At diagnosis, over
About Disc Medicine
Disc Medicine 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 its phase 1b clinical study of DISC-0974 in patients with MF and anemia, including the results thereof. 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 our Annual Report on Form 10-K for the year ended December 31, 2023, 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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