Disc Medicine Presents Positive Data from SAD Cohorts of a Phase 1b Trial in Patients with Chronic Kidney Disease (CKD) and Anemia at the 2024 American Society of Nephrology (ASN) Kidney Week
Disc Medicine (NASDAQ:IRON) presented positive Phase 1b trial data for DISC-0974 in patients with non-dialysis-dependent chronic kidney disease (NDD-CKD) and anemia. The single ascending dose (SAD) study included 40mg and 60mg cohorts, demonstrating that a single dose effectively suppressed hepcidin and increased iron mobilization, erythropoiesis, and hemoglobin levels. Key results showed >75% hepcidin reduction at the highest dose level, up to 3x increase in transferrin saturation, and mean hemoglobin increases of +0.54 g/dL at 40mg and +0.55 g/dL at 60mg compared to placebo. The treatment demonstrated acceptable safety with only Grade 1 or 2 related adverse events.
Disc Medicine (NASDAQ:IRON) ha presentato dati positivi della fase 1b per il DISC-0974 in pazienti con malattia renale cronica non dipendente dalla dialisi (NDD-CKD) e anemia. Lo studio con dose ascendente singola (SAD) ha incluso coorti da 40 mg e 60 mg, dimostrando che una singola dose ha soppressa efficacemente l'epingedina e ha aumentato la mobilizzazione del ferro, l'eritropoiesi e i livelli di emoglobina. I risultati chiave hanno mostrato una riduzione di oltre il 75% dell'epingedina al livello più alto di dose, fino a un aumento di 3 volte della saturazione della transferrina, e aumenti medi dell'emoglobina di +0,54 g/dL a 40 mg e +0,55 g/dL a 60 mg rispetto al placebo. Il trattamento ha dimostrato una sicurezza accettabile con solo eventi avversi correlati di grado 1 o 2.
Disc Medicine (NASDAQ:IRON) presentó datos positivos del ensayo de fase 1b para el DISC-0974 en pacientes con enfermedad renal crónica no dependiente de diálisis (NDD-CKD) y anemia. El estudio de dosis ascendente única (SAD) incluyó cohortes de 40 mg y 60 mg, demostrando que una sola dosis suprimió efectivamente la hepcidina y aumentó la movilización de hierro, la eritropoyesis y los niveles de hemoglobina. Los resultados clave mostraron una reducción de más del 75% en hepcidina al nivel de dosis más alto, hasta un aumento de 3 veces en la saturación de transferrina, y aumentos medios de hemoglobina de +0.54 g/dL a 40 mg y +0.55 g/dL a 60 mg en comparación con el placebo. El tratamiento demostró una seguridad aceptable con solo eventos adversos relacionados de grado 1 o 2.
Disc Medicine (NASDAQ:IRON)는 비투석성 만성 신장 질환(NDD-CKD) 및 빈혈 환자를 위한 DISC-0974의 1b상 긍정적인 시험 데이터를 발표했습니다. 단일 용량 상승(SAD) 연구는 40mg 및 60mg 집단을 포함하였으며, 단일 용량이 헤프시딘을 효과적으로 억제하고 철 이동, 적혈구 생성 및 헤모글로빈 수치를 증가시켰음을 입증했습니다. 주요 결과는 최고 용량에서 헤프시딘이 75% 이상 감소하고, 트랜스페린 포화도가 최대 3배 증가하며, 40mg에서 +0.54 g/dL, 60mg에서 +0.55 g/dL의 평균 헤모글로빈 증가를 보여주었습니다. 치료는 1도 또는 2도의 관련 부작용만으로 허용 가능한 안전성을 입증했습니다.
Disc Medicine (NASDAQ:IRON) a présenté des données positives d'essai de phase 1b pour le DISC-0974 chez des patients atteints de maladie rénale chronique non dépendante de la dialyse (NDD-CKD) et d'anémie. L'étude à dose unique ascendante (SAD) a inclus des cohortes de 40 mg et 60 mg, montrant qu'une seule dose a efficacement supprimé l'hepcidine et augmenté la mobilisation du fer, l'érythropoïèse et les niveaux d'hémoglobine. Les résultats clés ont montré une réduction de plus de 75 % de l'hepcidine au niveau de dose le plus élevé, jusqu'à trois fois l'augmentation de la saturation en transferrine, et des augmentations moyennes de l'hémoglobine de +0,54 g/dL à 40 mg et +0,55 g/dL à 60 mg par rapport au placebo. Le traitement a montré une sécurité acceptable avec seulement des événements indésirables liés de grade 1 ou 2.
Disc Medicine (NASDAQ:IRON) hat positive Phase-1b-Studienergebnisse für DISC-0974 bei Patienten mit nicht dialysepflichtiger chronischer Nierenerkrankung (NDD-CKD) und Anämie vorgestellt. Die Studie zur einmaligen Dosissteigerung (SAD) umfasste 40 mg und 60 mg Kohorten und zeigte, dass eine Einzeldosis die Hepcidinproduktion effektiv unterdrückte und die Eisenmobilisierung, Erythropoese und Hämoglobinspiegel erhöhte. Die wichtigsten Ergebnisse zeigten eine Reduktion von über 75 % des Hepcidinspiegels bei der höchsten Dosis, bis zu einem dreifachen Anstieg der Transferrinsättigung und durchschnittliche Erhöhungen des Hämoglobinspiegels um +0,54 g/dL bei 40 mg und +0,55 g/dL bei 60 mg im Vergleich zu Placebo. Die Behandlung zeigte eine akzeptable Sicherheit mit nur Grad 1 oder 2 bezogenen unerwünschten Ereignissen.
- Single dose showed significant efficacy with >75% hepcidin reduction
- Demonstrated 3x increase in transferrin saturation at highest dose
- Achieved meaningful hemoglobin increases up to 1.8 g/dL at 60mg dose
- Favorable safety profile with only Grade 1-2 related adverse events
- Successful proof of concept in both myelofibrosis and CKD settings
- Drug still in early clinical phase (Phase 1b)
- Not yet approved for therapeutic use in any jurisdiction
Insights
The Phase 1b trial results for DISC-0974 demonstrate significant potential in treating anemia in CKD patients. The data shows impressive efficacy metrics:
- Hepcidin reduction >
75% at highest dose - Transferrin saturation increased up to 3x from baseline
- Hemoglobin increases of up to 1.8 g/dL at the 60mg dose level
The dose-dependent response and sustained effects from a single administration are particularly promising. The safety profile appears favorable with only Grade 1-2 related adverse events. This positions DISC-0974 as a potential breakthrough in CKD anemia treatment, addressing a significant market need. The company's expansion plans into other inflammatory and chronic disease-related anemias could substantially broaden the drug's commercial potential.
These clinical results represent a significant milestone for Disc Medicine's lead program. The CKD anemia market is substantial, with over 37 million Americans affected by CKD and approximately
WATERTOWN, Mass., Oct. 25, 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 additional data from an ongoing Phase 1b study of DISC-0974 in patients with non-dialysis-dependent chronic kidney disease (NDD-CKD) and anemia, including results from the 40 mg and 60 mg single ascending dose (SAD) cohorts. The data presented at the 2024 American Society of Nephrology (ASN) Kidney Week in San Diego, CA demonstrated that a single dose of DISC-0974 results in sustained suppression of hepcidin and mobilization of iron, and increased erythropoiesis and levels of hemoglobin in NDD-CKD patients with anemia.
“We are pleased to present these updated results, which provide the first clinical evidence in CKD anemia that reducing hepcidin through hemojuvelin translates into increased erythropoiesis and hemoglobin. Importantly, we were able to observe activity following only a single dose of DISC-0974, which will enable us to efficiently explore dose regimens in the multiple-dose portion of the Phase 1b study,” said John Quisel, JD, PhD, President and Chief Executive Officer of Disc Medicine. “We have now shown that DISC-0974 can improve anemia in the settings of both myelofibrosis and chronic kidney disease. This speaks to the much broader potential for DISC-0974 to address anemia caused by a range of inflammatory and chronic diseases, each of which are associated with elevated hepcidin, and we look forward to providing our plans to access these indications.”
In the SAD portion of this study, participants with Stage 2-5 NDD-CKD were given a single dose of placebo (n=7) or DISC-0974 subcutaneously (SC) at 28 mg (n=9), 40 mg (n=6), or 60 mg (n=6). Dose escalation is ongoing in the SAD. This interim data set demonstrated:
- Substantial, durable, dose-dependent reduction in hepcidin from baseline compared to placebo across dose levels, with median reduction greater than
75% from baseline at highest dose level - Meaningful and sustained increase in transferrin saturation from baseline compared to placebo across dose levels, with median increase up to 3x from baseline at highest dose level
- Early and sustained increase in mean reticulocyte hemoglobin from baseline across all dose groups through Day 22 and beyond
- Maximal mean values through Day 22 of +1.14 pg at 28 mg, +1.49 pg at 40 mg, and +1.53 pg at 60 mg, compared with +0.21 pg on placebo
- Increase in mean hemoglobin from baseline across dose groups over the study period
- Change greater than placebo: +0.35 g/dL at 28 mg, +0.54 g/dL at 40 mg, and +0.55 g/dL at 60 mg
- Mean maximal increase in hemoglobin of +0.8 g/dL at 40 mg and +0.7 g/dL at 60 mg compared with +0.2 g/dL on placebo
- Maximal observed individual increase in hemoglobin up to +0.95 g/dL at 28 mg, +1.5 g/dL at 40 mg, and +1.8 g/dL at 60 mg
- DISC-0974 demonstrated acceptable safety and tolerability at all evaluated dose levels
- The majority of adverse events were deemed not related to DISC-0974 and all adverse events assessed as treatment-related were Grade 1 or 2
DISC-0974 is an investigational agent and is not approved for therapeutic use in any jurisdiction worldwide.
About Anemia of Chronic Kidney Disease
Chronic kidney disease is a global, widespread disease characterized by progressive loss of kidney function and may lead to end-stage renal disease or kidney failure. CKD affects over 37 million patients in the United States and an estimated 700 million patients worldwide. Anemia is a serious and frequent complication of CKD, as patients are unable to produce sufficient red blood cells and hemoglobin. It affects approximately 5 to 6 million patients in the U.S. alone, may result in fatigue, shortness of breath, and reduced physical and cognitive function, and can be associated with a higher risk of mortality, hospitalization and other complications. Elevated hepcidin is a primary cause of anemia in CKD patients and prevents erythropoiesis by depriving developing red blood cells of iron. Hepcidin accumulates at high levels in CKD patients because its production is stimulated by inflammation and its clearance is reduced due to impaired renal function. Most CKD patients do not receive any treatment for their anemia due to the complexity of outpatient administration and potential safety concerns related to the current treatments. In severe cases, patients may receive blood transfusions, but use may lead to increased administrative burden, safety risks and the potential for immune sensitization which precludes eligibility for kidney transplantation.
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 NDD-CKD patients with anemia, including the multiple dose portion of this clinical study and the results thereof; and the potential for expansion opportunities in anemia associated with other inflammatory diseases. 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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