HJV

WATERTOWN, Mass., Feb. 20, 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 announced that the United States Food and Drug Administration (FDA) has granted Fast Track Designation to DISC-0974 for the treatment of patients with non-dialysis dependent chronic kidney disease (NDD-CKD) and anemia. “Receiving Fast Track designation highlights the unmet need for the millions of NDD-CKD patients with anemia, as well as the potential of DISC-0974 to address this need,” said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc. “We believe DISC-0974 could be a transformative therapy for these patients and are excited to share additional results from our ongoing Phase 1b/2 study in NDD-CKD patients with anemia this year.” Fast Track is a process designed by the FDA to facilitate the development and expedite the review of investigational drugs intended to treat serious conditions and for which nonclinical or clinical data demonstrate the potential to address unmet medical need. A therapeutic candidate that receives Fast Track designation may be eligible for more frequent interactions with the FDA to discuss the candidate’s development plan. Therapeutic candidates with Fast Track designation may also be eligible for priority review and accelerated approval if supported by clinical data. 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 affects 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 and has ongoing clinical studies of DISC-0974 in patients with myelofibrosis and anemia and also in patients with NDD-CKD and anemia. DISC-0974 is an investigational agent and is not approved for use as a therapy in any jurisdiction worldwide. About Anemia of Chronic Kidney Disease (CKD) Chronic kidney disease (CKD) is a global, widespread disease characterized by progressive loss of kidney function and may lead to end-stage renal disease (ESRD) 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 5-6 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 as a consequence of impaired renal function. The majority of 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 transfusions require significant healthcare utilization, incur safety risks and increase the potential for immune sensitization which precludes eligibility for kidney transplantation. 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 . 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/2 clinical study of DISC-0974 in NDD-CKD patients with anemia, projected timelines for the initiation and completion of its clinical trials, anticipated timing of release of data, and other clinical activities; Disc’s business plans and objectives; and Disc’s beliefs about operating expenses and that it will have capital to fund Disc well into 2026. 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; the other risks and uncertainties described in the “Risk Factors” section of our Annual Report on Form 10-K for the year ended December 31, 2022, Quarterly Reports on Form 10-Q for the quarters ended March 31, 2023, June 30, 2023, and September 30, 2023, and other documents filed by Disc from time to time with the SEC, as well as discussions of potential risks, uncertainties, and other important factors in Disc’s subsequent filings with the SEC. 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. Media Contact Peg Rusconi Verge Scientific Communications prusconi@vergescientific.com Investor Relations Contact Christina Tartaglia Stern Investor Relations christina.tartaglia@sternir.com

Substantial, dose-dependent reductions in serum hepcidin and increases in serum iron Hematologic response demonstrated by increased hemoglobin levels and reduction in transfusion burdenDISC-0974 was generally well-tolerated at all evaluated doses WATERTOWN, Mass., Dec. 11, 2023 (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 announced data from initial dose cohorts of its ongoing phase 1b/2 study of DISC-0974, a monoclonal antibody designed to suppress hepcidin by inhibiting the hemojuvelin (HJV) co-receptor, in MF patients with anemia. The initial data demonstrated that treatment with DISC-0974 substantially decreased serum hepcidin, increased serum iron and resulted in improvements in hemoglobin or reduced transfusion burden across a broad range of MF patients. “We are thrilled to see this level of hematologic activity so early during dose escalation and in a range of patient types. Elevated hepcidin is an important driver of anemia in patients with MF and the initial data suggest that DISC-0974 has leading activity in suppressing hepcidin,” said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc. “This is the second program in the last six months where Disc has shown proof-of-concept data in patients, and we look forward to advancing DISC-0974 deeper into development and presenting updated data from both this MF and the ongoing CKD anemia study next year.” The phase 1b/2a multi-center, open-label, ascending-dose study (NCT05320198) is enrolling patients with MF and severe anemia, including both transfusion and non-transfusion dependent patients. The trial also includes patients who may or may not be receiving concomitant janus kinase (JAK) inhibitor therapy. Study assessments include safety and tolerability of DISC-0974, as well as markers of iron regulation, such as hepcidin and iron, and hematologic parameters. In the phase 1b dose-escalation phase, DISC-0974 is administered subcutaneously every 4 weeks for up to 6 treatments. Dose escalation is ongoing and the data presented reflect 11 evaluable subjects from the initial three dose levels (14 mg, 28 mg and 50 mg) as of the October 20, 2023 data cutoff. Key initial data presented: DISC-0974 dosing resulted in meaningful, dose-dependent decreases in hepcidin across all treated patientsThese reductions in hepcidin corresponded to dose-dependent increases in serum iron Patients dosed with 28 mg of DISC-0974 had a >75% reduction in serum hepcidin and >75% increase in serum iron Four of seven (57%) evaluable non-transfusion-dependent (NTD) patients at the 28 mg and 50 mg dose levels had a >1.5 g/dL hemoglobin increase from baseline after starting DISC-0974One of two transfusion-dependent (TD) patients achieved transfusion independence by the Gale CriteriaHematologic activity was observed in MF patients, regardless of concomitant JAK inhibitor useTo date, DISC-0974 has been generally well-tolerated. The adverse events (AEs) seen in two or more subjects were fatigue, anemia, diarrhea, and nausea. The majority of AEs were deemed not related to DISC-0974. These data were presented at the 65th American Society of Hematology (ASH) Annual Meeting in San Diego, California and the poster is available on the ASH platform. Later today, the company will be presenting updated results from the phase 2 BEACON study of bitopertin in EPP as an oral presentation, December 11th at 5:30 pm PT / 8:30 pm ET. Management will host a call to review the presented data on Monday, December 11th at 6:30 pm PT / 9:30 pm ET. 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 and initiated a Phase 1b/2a clinical trial of DISC-0974 in patients with myelofibrosis and 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 MyelofibrosisMyelofibrosis (MF) is a rare, chronic blood cancer that currently affects an estimated 16,000 to 18,500 patients in the United States alone. Severe, progressive, and treatment resistant anemia is the primary clinical manifestation of MF. At diagnosis, over 80% of MF patients have anemia, which progressively worsens and ultimately renders the majority of patients dependent on chronic red blood cell transfusions. Recent studies have shown hepcidin to be a key molecular driver of anemia in myelofibrosis. Hepcidin is elevated by approximately 12-fold in MF patients, and is correlated with disease severity, anemia, and the need for red blood cell transfusions. About Disc MedicineDisc 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 Medicine 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/2 clinical studies of DISC-0974 in patients with MF and NDD-CKD patients with anemia, projected timelines for the initiation and completion of its clinical trials, anticipated timing of release of data, and other clinical activities; and Disc’s business plans and objectives. 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 nature, strategy and focus of Disc; Disc’s plans to research, develop and commercialize its current and future product candidates; the timing of the availability of data from Disc’s clinical trials; the timing and anticipated results of Disc’s preclinical studies and clinical trials and the risk that the results of Disc’s 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; the other risks and uncertainties described in the “Risk Factors” section of our Annual Report on Form 10-K for the year ended December 31, 2022, Quarterly Reports on Form 10-Q for the quarters ended March 31, 2023, June 30, 2023, and September 30, 2023, and other documents filed by Disc from time to time with the SEC, as well as discussions of potential risks, uncertainties, and other important factors in Disc’s subsequent filings with the SEC. 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. Media Contact Peg RusconiVerge Scientific Communicationsprusconi@vergescientific.com Investor Relations Contact Christina TartagliaStern Investor Relationschristina.tartaglia@sternir.com

WATERTOWN, Mass., Feb. 16, 2023 (GLOBE NEWSWIRE) -- Disc Medicine, a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel treatments for patients suffering from serious hematologic diseases, announced today the initiation of a Phase 1b/2 clinical study of DISC-0974 in non-dialysis dependent chronic kidney disease (NDD-CKD) patients with anemia. DISC-0974 is a monoclonal antibody designed to suppress hepcidin by inhibiting the hemojuvelin (HJV) co-receptor, and thereby address anemia by enhancing the availability of iron for erythropoiesis. DISC-0974 is also currently being studied in a Phase 1b/2 clinical study in patients with myelofibrosis and anemia. “We are excited to initiate this clinical trial of DISC-0974 in chronic kidney disease, where hepcidin plays a key role in the pathophysiology of anemia. There is a tremendous need for innovative therapies that work through mechanisms outside of the erythropoietin pathway,” said John Quisel, JD, PhD, Chief Executive Officer at Disc Medicine. “We believe DISC-0974 has potential across a broad range of chronic and inflammatory diseases. With the start of this study, we now have ongoing clinical trials for DISC-0974 in both chronic kidney disease and myelofibrosis, and plan to explore its use in other indications as well.” The study will be a multi-center, Phase 1b/2 trial and will evaluate the safety, tolerability, and efficacy of DISC-0974 in NDD-CKD patients with anemia. The study endpoints will include hepcidin levels, serum iron and markers of iron mobilization and changes in hemoglobin. The study enrollment criteria will include patients with Stage II-V CKD not receiving dialysis, baseline Hb < 11.0 g/dL for males and Hb < 10.5 g/dL for females, and those not receiving concurrent treatment with erythropoietin-stimulating agents (ESAs). The study will be conducted in two parts: Phase 1b (Dose-Escalation): Randomized, double-blind, placebo-controlled study design; single, ascending doses of DISC-0974 will be administered subcutaneously at the following planned dose levels: 28 mg, 40 mg, 60 mg, 90 mg; safety, PK and hematologic effects will be assessed at each dose level; a dose for the expansion phase will be selected based on optimal increases in hemoglobin; Phase 2 (Expansion Stage): Open-label, single-arm study design; multiple doses of DISC-0974 administered subcutaneously, once-monthly at the dose level selected from the phase 1b portion of the study for three months. 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 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 and initiated a Phase 1b/2a clinical trial of DISC-0974 in patients with myelofibrosis and 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 Chronic Kidney Disease (CKD) Chronic kidney disease (CKD) is a global, widespread disease characterized by progressive loss of kidney function and may lead to end-stage renal disease (ESRD) 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 5-6 million patients in the U.S. alone, may results 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 as a consequence of impaired renal function. The majority of 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 (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, 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 . 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/2 clinical study of DISC-0974 in NDD-CKD patients with anemia and projected timelines for the initiation and completion of its clinical trials and other activities. 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; the other risks and uncertainties described in the “Risk Factors” section of the Current Report on Form 8-K filed with the SEC on December 29, 2022 and other documents filed by Disc from time to time with the SEC, as well as discussions of potential risks, uncertainties, and other important factors in Disc’s subsequent filings with the Securities and Exchange Commission. 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. Media Contact Peg Rusconi Verge Scientific Communications prusconi@vergescientific.com Investor Relations Contact Suzanne Messere Stern Investor Relations suzanne.messere@sternir.com