Hepc

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 levelMeaningful 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 levelEarly 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. Media Contact Peg RusconiDeerfield Grouppeg.rusconi@deerfieldgroup.com Investor Relations Contact Christina TartagliaPrecision AQchristina.tartaglia@precisionaq.com

WATERTOWN, Mass., Oct. 11, 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 upcoming clinical data to be presented at the 2024 American Society of Nephrology (ASN) Kidney Week, which will be held in San Diego, CA on October 24-27, 2024. The poster will report on additional SAD cohorts from the Phase 1b, randomized, placebo-controlled study of DISC-0974 in patients with non-dialysis dependent chronic kidney disease (NDD CKD) and anemia. This study was designed to assess safety as well as changes to hepcidin, iron, and key hematologic parameters. “We are excited to present an update from our ongoing Phase 1b SAD study of DISC-0974 in NDD-CKD patients with anemia at the upcoming Kidney Week,” said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc. “At the meeting, we’ll be sharing data from the 40mg and 60mg dose cohorts on safety, pharmacokinetics, and effects of DISC-0974 on hepcidin, iron and anemia.” The abstract is now available through the ASN Kidney Week conference website. Details of Presentation Abstract Number: 4126178 Title: A Phase 1b, Double-Blind, Placebo-Controlled Study of DISC-0974, an Anti-Hemojuvelin Antibody, in Patients with Non-Dialysis-Dependent Chronic Kidney Disease and Anemia Date / Time: Friday, October 25, 10:00am – 12:00pm PST Presenter: Pablo Pergola, M.D., Ph.D. 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 . 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, the results thereof, and anticipated timing of release of data; and projected timeline for the initiation of the MAD portion of this clinical study. 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. Media Contact Peg Rusconi Deerfield Group peg.rusconi@deerfieldgroup.com Investor Relations Contact Christina Tartaglia Precision AQ christina.tartaglia@precisionaq.com

Presented positive data for all three programs at the European Hematology Association (EHA) 2024 Congress; demonstrated potential efficacy for bitopertin in erythropoietic protoporphyria (EPP) and DISC-0974 in anemia of myelofibrosis (MF), as well as proof of mechanism in a Phase 1 study for DISC-3405Plan to provide an update on regulatory interactions for bitopertin in EPP and DISC-0974 in anemia of MF in the second half of 2024Strengthened financial position through an underwritten offering of common stock for $178 million in gross proceeds, ending Q2 with $501 million in cash, cash equivalents, and marketable securities that is expected to fund operations well into 2027 WATERTOWN, Mass., Aug. 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 reported financial results for the second quarter ended June 30, 2024. “The data we presented at EHA this past quarter were highly encouraging and supportive of the therapeutic potential of our entire portfolio,” said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc. “These data catalyzed the successful completion of a financing supported by a prestigious group of investors, which provides runway through key readouts across our programs over the next few years. We continue to make great progress and look forward to sharing updates on our regulatory interactions around bitopertin and DISC-0974 in the second half of the year.” Recent Business Highlights and Upcoming Milestones: Bitopertin: GlyT1 Inhibitor (Heme Synthesis Modulator) Presented updated results from AURORA showing that bitopertin had a meaningful impact on key aspects of EPP, and providing a range of potential efficacy endpointsEnd of Phase 2 meeting to discuss optimal registrational endpoints moving forward in EPP is expected to occur in the second half of 2024 DISC-0974: Anti-Hemojuvelin Antibody (Hepcidin Suppression) Presented updated Phase 1b data from DISC-0974 in anemia of MF patients demonstrating a durable hemoglobin response in the majority of patients and a positive impact on transfusion burden across a broad range of participantsPlan for interactions with regulators in the second half of 2024 to determine the optimal Phase 2 study design in anemia of MFUpdated Phase 1b data from DISC-0974 in anemia in non-dialysis-dependent chronic kidney disease (NDD-CKD) is expected to be shared in the second half of 2024 DISC-3405: Anti-TMPRSS6 Antibody (Hepcidin Induction) Presented initial data from the single-ascending dose (SAD) cohorts of the ongoing Phase 1 study in healthy volunteers, which demonstrated proof of mechanism with substantial increases in hepcidin levels and sustained reductions in serum iron supportive of monthly subcutaneous dosing, as well as positive impact on hematologic parameters at the highest doseData from the multiple ascending dose (MAD) cohorts of this study expected to be shared in the second half of 2024 Corporate Completed an underwritten public offering of common stock in June 2024 for $178.0 million in gross proceeds Second Quarter 2024 Financial Results: Cash Position: Cash, cash equivalents, and marketable securities were $500.9 million as of June 30, 2024, which are expected to fund our operational plans well into 2027.Research and Development Expenses: R&D expenses were $23.5 million for the quarter ended June 30, 2024, as compared to $12.1 million for the quarter ended June 30, 2023. The increase in R&D expenses were primarily driven by the progression of Disc’s portfolio, including bitopertin’s clinical studies and drug manufacturing, and advancing our DISC-0974 and DISC-3405 programs in the clinic, and increased headcount.General and Administrative Expenses: G&A expenses were $7.4 million for the quarter ended June 30, 2024, as compared to $5.2 million for the quarter ended June 30, 2023. The increase in G&A expenses was primarily due to increased headcount.Net Loss: Net loss was $26.4 million for the quarter ended June 30, 2024, as compared to $15.9 million for the quarter ended June 30, 2023. 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. Available Information Disc announces material information to the public about the Company, its products and services, and other matters through a variety of means, including filings with the U.S. Securities and Exchange Commission (SEC), press releases, public conference calls, webcasts and the investor relations section of the Company website at ir.discmedicine.com in order to achieve broad, non-exclusionary distribution of information to the public and for complying with its disclosure obligations under Regulation FD. 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 AURORA Phase 2 and BEACON Phase 2 clinical trials of bitopertin and the results thereof, its Phase 1b/2 clinical trial of DISC-0974 in patients with MF and NDD-CKD patients with anemia, its initial SAD data in its Phase 1 clinical trial of DISC-3405 in healthy volunteers; and projected timelines for the initiation and completion of its clinical trials, anticipated timing of release of data, and other clinical activities; and Disc’s belief about operating expenses and that it will have capital to fund Disc well into 2027. 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 2023, Quarterly Reports on Form 10-Q for the quarters ended March 31, 2024 and June 30, 2024, and other documents filed by Disc from time to time with the Securities and Exchange Commission (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. DISC MEDICINE, INC.CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS(In thousands, except share and per share amounts)(Unaudited) Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023Operating expenses: Research and development $23,485 $12,100 $47,189 $32,280 General and administrative 7,367 5,228 15,125 10,173 Total operating expenses 30,852 17,328 62,314 42,453 Loss from operations (30,852) (17,328) (62,314) (42,453)Other income (expense), net 4,560 1,405 9,078 3,772 Income tax expense (60) (24) (65) (47)Net loss $(26,352) $(15,947) $(53,301) $(38,728)Weighted-average common shares outstanding-basic and diluted​ 25,649,043 21,484,955 25,229,456 20,226,923 Net loss per share-basic and diluted $(1.03) $(0.74) $(2.11) $(1.91) DISC MEDICINE, INC.CONDENSED CONSOLIDATED BALANCE SHEETS(In thousands)(Unaudited) June 30, December 31, 2024 2023Assets Cash, cash equivalents, and marketable securities $500,945 $360,382 Other current assets 7,296 5,280 Total current assets 508,241 365,662 Non-current assets 1,976 2,334 Total assets $510,217 $367,996 Liabilities and Stockholders’ Equity Current liabilities $20,310 $21,439 Non-current liabilities 1,078 1,436 Total liabilities 21,388 22,875 Total stockholders’ equity 488,829 345,121 Total liabilities and stockholders’ equity $510,217 $367,996 Media Contact Peg RusconiVerge Scientific Communicationsprusconi@vergescientific.com Investor Relations Contact Christina TartagliaStern Investor Relationschristina.tartaglia@sternir.com