Porphyria, Erythropoietic

MALIBU, Calif., March 04, 2024 (GLOBE NEWSWIRE) -- Leading biotech company CLINUVEL last week joined an intimate event bringing together California’s most prolific investors and philanthropists at the Malibu home of Lady Gaga and Michael Polansky. The event, hosted by Lady Gaga and Mr. Polansky, and Sean Parker and Alexandra Parker, shone a light on the groundbreaking work of CLINUVEL, a global biopharmaceutical firm whose cutting-edge melanocortin therapies transform the lives of patients at the highest risk of photodamage and skin cancer. Mr. Parker and Mr. Polansky have supported CLINUVEL for more than 15 years, finding alignment between the Company’s pioneering approach to medicine and the boundary-pushing work of the Parker Institute for Cancer Immunotherapy which works to revolutionise healthcare. Over a family-style dinner by chef Evan Funke of Los Angeles hotspot Felix Trattoria, and a performance by Grammy-winning Attacca Quartet, the hosts shared how CLINUVEL’s treatments herald a new era for the treatment of light-related conditions. The Group’s CEO Dr. Philippe Wolgen also unveiled what is next for the most exciting biopharmaceutical firm in the field of photomedicine today. The future of photomedicine For 25 years, CLINUVEL introduced a proof of concept by treating groups of patients whose needs had been overlooked by the pharmaceutical industry. These include the genetic disorders erythropoietic protoporphyria (EPP), xeroderma pigmentosum (XP) and the skin disorder vitiligo, when people lose their pigmentation. In 2014 CLINUVEL developed the only approved treatment in the world for EPP, accelerating CLINUVEL’s evolution into a profitable, self-sustaining and dynamic company. Now the Company is exploring the vast potential of its technology: how can CLINUVEL’s unique knowledge of melanocortins be scaled up to treat widespread health conditions that affect the skin, brain and nervous system? Beyond the world of pharmaceuticals, CLINUVEL’s technology has the power to disrupt the skincare market. After 43 years of photomedicine research, the Company is launching PhotoCosmetics, a range of advanced skincare products based on a group of peptides known as melanocortins. Revolutionising healthcare for those in needPhilanthropy is central to the work of Lady Gaga and Michael Polansky, Sean Parker and Alexandra Parker. CLINUVEL shares their passion for creating long-lasting change and radically improving patients’ lives. At the event in Malibu, CLINUVEL announced the Photomedicine Foundation, which will count Mr Polansky as a trustee. The initiative will help those most affected by debilitating conditions related to the sun. Through the Foundation, CLINUVEL is taking its world-leading expertise directly to communities who cannot afford care or treatment. The foundation’s mission is to support vulnerable patients at extreme risk of photodamage and skin cancer in Africa, Middle East and Latin America. Michael Polansky said: “It’s critical that investors strive to find companies whose missions extend beyond profitability, by supporting the development of technologies that can drive meaningful and sustained improvement to human life. CLINUVEL is an example of a company that aims to improve human health and I am delighted to become a trustee of the Photomedicine Foundation, which will play a vital role in improving the lives of individuals with light-related conditions who cannot access life-saving medical care.” Sean Parker said: “CLINUVEL is one of the most innovative Life Sciences companies that exists today with a management that has the patience to see through long development. Both its groundbreaking melanocortin technology and long-term business plan will disrupt the world of biopharmaceuticals and luxury skincare. The Photomedicine Foundation is a welcome addition to a company on a path to growth unlike any other.” CLINUVEL’s CEO, Dr. Philippe Wolgen said: “Sean Parker and Michael Polansky are unafraid to rip up the rulebook in their mission to advance science and improve global health. CLINUVEL is grateful to have their support as we translate our pharmaceutical technology into PhotoCosmetics and give back to underserved communities. A bright future looms for CLINUVEL.” About The CLINUVEL Group: CLINUVEL is a public biopharmaceutical company whose pioneering melanocortin technology has revolutionised the field of photomedicine. Over the last 25 years, CLINUVEL’s work has furthered scientific knowledge, delivered breakthroughs in medical technology and transformed patients’ lives. The Company has developed and commercialised SCENESSE® (afamelanotide), the only approved treatment in the world for the prevention of phototoxicity in adult patients with EPP. The treatment was approved for use by the US FDA in 2019 and is also approved for commercial distribution in Europe, Israel and Australia. CLINUVEL is conducting clinical trials to assess the efficacy and safety of its melanocortin technology in treating severe disorders including xeroderma pigmentosum (XP), vitiligo and arterial ischemic stroke. In 2023 it announced its expansion into skincare with the introduction of its first PhotoCosmetic product CYACÊLLE, a polychromatic solar screen. For more information visit www.clinuvel.comInstagram:@clinuvel_pharmaceuticals @clinuvelDNATwitter:@ClinuvelNewsFacebook:@Clinuvel

Completed enrollment of BEACON and AURORA trials of bitopertin in erythropoietic protoporphyria (EPP); BEACON expanded to include adolescentsData from all adult patients in BEACON to be presented in an oral presentation at the 65th American Society of Hematology Annual Meeting and Exposition (ASH 2023), including the preliminary analysis of the precedented pivotal endpoint, cumulative time in sunlight over 6 months on days without painPreliminary pharmacodynamic data from the ongoing phase 1b/2 study of DISC-0974 in patients with myelofibrosis (MF) and anemia to be presented in a poster at ASH 2023, including changes in hemoglobin levelsData from the first 28 mg cohort of the phase 1b/2 study of DISC-0974 in patients with anemia of chronic kidney disease who are not receiving dialysis (NDD-CKD) to be presented as a part of a management call during ASH 2023 Initiated a Phase 1 trial of DISC-3405, an anti-TMPRSS6 antibody, in healthy volunteers and received fast track designation by the U.S. Food and Drug Administration (FDA) for the treatment of polycythemia vera (PV) WATERTOWN, Mass., Nov. 09, 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 reported financial results for the third quarter ended September 30, 2023, and provided an update on recent program and corporate developments. “We made tremendous progress this quarter in advancing our portfolio, including a major milestone of completing enrollment of both BEACON and AURORA studies within a year, as well as advancing our third development program into the clinic,” said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc. “We’re looking forward to the ASH 2023 meeting in December, when we will be presenting updated interim data from BEACON, as well as initial data for DISC-0974 in MF and CKD.” Recent Business Highlights and Upcoming Milestones: Bitopertin: GlyT1 Inhibitor (Heme Synthesis Modulator) Bitopertin is an investigational, clinical-stage, orally-administered inhibitor of glycine transporter 1 (GlyT1) that is designed to modulate heme biosynthesis. GlyT1 is a membrane transporter expressed on developing red blood cells and is required to supply sufficient glycine for heme biosynthesis and support erythropoiesis. Disc is planning to develop bitopertin as a potential treatment for a range of hematologic diseases including erythropoietic porphyrias, where it has potential to be the first disease-modifying therapy. Completed enrollment for both BEACON, an open-label Phase 2 clinical study of bitopertin in patients with EPP and X-linked protoporphyria (XLP), and AURORA a Phase 2 randomized, placebo-controlled clinical study of bitopertin in adults with EPPExpanded enrollment of BEACON to include adolescents (ages 12-18)Updated BEACON data from all patients and with longer duration of therapy will be presented at ASH 2023, including measures of protoporphyrin IX (PPIX), photosensitivity, quality of life, safety, and tolerability, as well as preliminary analysis of the precedented pivotal endpoint, cumulative time in light over 6 months on days without painTopline AURORA data is expected to be presented in early 2024 DISC-0974: Anti-Hemojuvelin Antibody (Hepcidin Suppression) DISC-0974 is an investigational anti-hemojuvelin monoclonal antibody and is designed to suppress hepcidin production and increase serum iron levels in patients suffering from anemia of inflammation. Ongoing enrollment and dose escalation in a Phase 1b/2 clinical study in MF patients with severe anemia on stable background therapy; initial data from 10-20 patients in the dose-escalation phase of the study, including safety and changes in hepcidin, iron, and hemoglobin levels, will be presented at ASH 2023Ongoing enrollment and dose escalation in a Phase 1b/2 clinical study of patients with anemia of chronic kidney disease who are not receiving dialysis (NDD-CKD); initial data from the 28 mg cohort will be presented as part of a management call in December DISC-3405: Anti-TMPRSS6 Antibody (Hepcidin Induction) DISC-3405 is an investigational, anti-TMPRSS6 (Transmembrane Serine Protease 6, also known as Matriptase-2) monoclonal antibody designed to increase hepcidin production and restrict serum iron. Initiated a Phase 1 study in healthy volunteers in October 2023Received FDA fast track designation in September 2023 for DISC-3405 for the treatment of PV Third Quarter 2023 Financial Results: Cash Position: Cash and cash equivalents were $370.5 million as of September 30, 2023, which are expected to fund our operational plans well into 2026.Research and Development Expenses: R&D expenses were $14.4 million for the quarter ended September 30, 2023, as compared to $7.9 million for the quarter ended September 30, 2022. The increase in R&D expenses were primarily driven by the progression of Disc’s portfolio, including bitopertin’s ongoing two Phase 2 clinical studies and drug manufacturing, study initiation for DISC-3405, and increased headcount.General and Administrative Expenses: G&A expenses were $4.5 million for the quarter ended September 30, 2023, as compared to $2.6 million for the same period in 2022. The increase in G&A expenses was primarily due to increased headcount.Net Loss: Net loss was $14.1 million for the quarter ended September 30, 2023, as compared to $16.2 million for the third quarter of 2022. Although operating expenses increased in the current period relative to the prior period due to increased product development activities, these increases were offset by increased interest income and the absence of the change in fair value of the derivative liability related to a one-time share issuance obligation to F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc. in connection with the bitopertin license agreement which was settled in late 2022. 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 studies of bitopertin and the results thereof, and its Phase 1b/2 study of bitopertin in Diamond-Blackfan Anemia, its Phase 1b/2 clinical studies of DISC-0974 in patients with MF and NDD-CKD patients with anemia, its Phase 1 clinical study of DISC-3405 in healthy volunteers; 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; 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; that enrollment timelines of both the BEACON and AURORA studies may not necessarily be predictive of future enrollment timelines; the timing of initiation of Disc’s planned clinical trials; Disc’s ability to retain and recognize the intended incentives conferred by Fast Track Designation for its product candidates including DISC-3405; 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 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 and June 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. DISC MEDICINE, INC. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS(In thousands, except share and per share amounts)(Unaudited) ​Three Months Ended September 30, Nine Months Ended September 30, ​ 2023 ​ 2022 ​ 2023 ​ 2022 Operating expenses:​ ​ ​ ​ Research and development​$14,419 ​$7,886 ​$46,699 ​$23,421 General and administrative​ 4,539 ​ 2,593 ​ 14,712 ​ 9,033 Total operating expenses​ 18,958 ​ 10,479 ​ 61,411 ​ 32,454 Loss from operations​ (18,958) ​ (10,479) ​ (61,411) ​ (32,454) Other income (expense), net 4,856 ​ (5,671) ​ 8,628 ​ (3,129) Income tax expense​ (20) — (67) — Net loss​$(14,122) ​$(16,150) ​$(52,850) ​$(35,583) Weighted-average common shares outstanding-basic and diluted​ 24,316,817 960,317 21,605,202 943,058 Net loss per share-basic and diluted​$(0.58) ​$(16.82) ​$(2.45) ​$(37.73) DISC MEDICINE, INC. CONDENSED CONSOLIDATED BALANCE SHEETS (In thousands)(Unaudited) ​September 30, December 31, ​ 2023 ​ 2022 Assets​ ​ Cash and cash equivalents​$370,541 ​$194,611 Other current assets​ 3,722 ​ 3,880 Total current assets​ 374,263 ​ 198,491 Non-current assets​ 2,234 1,714 Total assets​$376,497 ​$200,205 Liabilities and Stockholders’ Equity​ ​ Current liabilities​$14,416 ​$22,578 Non-current liabilities​ 3,107 ​ 1,027 Total liabilities​ 17,523 ​ 23,605 Total stockholders’ equity​ 358,974 ​ 176,600 Total liabilities and stockholders’ equity​$376,497 ​$200,205 Media Contact Peg RusconiVerge Scientific Communicationsprusconi@vergescientific.com Investor Relations Contact Christina TartagliaStern Investor Relationschristina.tartaglia@sternir.com

Presented positive initial safety and efficacy data from the BEACON trial at the European Hematology Association (EHA) Congress in June 2023; data from all patients in BEACON to be presented year-end 2023 Initiated a phase 1/2 study of bitopertin in patients with Diamond-Blackfan Anemia who have failed corticosteroid treatment Enrolling patients in two separate Phase 1b/2 studies for DISC-0974, one in patients with anemia of chronic kidney disease who are not receiving dialysis (NDD-CKD) and one in patients with myelofibrosis and anemia; initial data from both trials expected by year-end 2023 Strengthened financial position through a $158 million upsized public offering; ended Q2 with approximately $378 million in cash that is expected to fund operations well into 2026 WATERTOWN, Mass., Aug. 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 reported financial results for the second quarter ending June 30, 2023, and provided an update on recent program and corporate developments. “This was a landmark quarter for our company, as we presented positive initial data from the BEACON study at EHA, which showed the promise of bitopertin as a potential treatment for EPP. The BEACON data catalyzed the successful completion of an upsized public offering, which provides runway well beyond key read-outs across our portfolio and positions us to advance bitopertin into late-stage development,” said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc. “We continue to make excellent progress across our portfolio and look forward to providing updated results from BEACON and our other studies at the end of the year.” Recent Business Highlights and Upcoming Milestones: Bitopertin: GlyT1 Inhibitor (Heme Synthesis Modulator) Bitopertin is an investigational, clinical-stage, orally-administered inhibitor of glycine transporter 1 (GlyT1) that is designed to modulate heme biosynthesis. GlyT1 is a membrane transporter expressed on developing red blood cells and is required to supply sufficient glycine for heme biosynthesis and support erythropoiesis. Disc is planning to develop bitopertin as a potential treatment for a range of hematologic diseases including erythropoietic porphyrias, where it has potential to be the first disease-modifying therapy. Presented positive initial data from BEACON, an open-label Phase 2 clinical study of bitopertin in patients with EPP and X-linked protoporphyria (XLP), demonstrating: Consistent and dose-dependent reductions of protoporphyrin IX (PPIX), the disease-causing metabolite in EPP Marked improvements in sunlight tolerance and quality of life Bitopertin was well-tolerated, with no meaningful changes in hemoglobin Continued enrollment for AURORA, a Phase 2 randomized, placebo-controlled clinical study of bitopertin in adults with EPP. Disc expects to have data by year-end 2023, to be presented in early 2024. Initiated and enrolled first patient in an NIH-sponsored Phase 1/2 study of bitopertin in patients with Diamond-Blackfan Anemia in July 2023 DISC-0974: Anti-Hemojuvelin Antibody (Hepcidin Suppression) DISC-0974 is an investigational anti-hemojuvelin monoclonal antibody (mAb) and is designed to suppress hepcidin production and increase serum iron levels in patients suffering from anemia of inflammation. Enrollment ongoing for a Phase 1b/2 clinical study of patients with anemia of chronic kidney disease who are not receiving dialysis (NDD-CKD); initial data expected by year-end 2023 Continued enrollment in a Phase 1b/2 clinical study in MF patients with severe anemia on stable background therapy; initial data expected by year-end 2023 MWTX-003: Anti-TMPRSS6 Antibody (Hepcidin Induction) MWTX-003 is an investigational, anti-TMPRSS6 (Transmembrane Serine Protease 6, also known as Matriptase-2) monoclonal antibody designed to increase hepcidin production and suppress serum iron. Continued technology transfer and study preparation activities for MWTX-003. Disc plans to initiate a Phase 1 study in healthy volunteers during the second half of 2023. Disc plans to develop MWTX-003 initially as a treatment for polycythemia vera as well as other indications. Corporate: Completed an upsized public offering of common stock and pre-funded warrants in June 2023 for $157.8 million in gross proceeds. Second Quarter 2023 Financial Results: Cash Position: Cash and cash equivalents were $377.6 million as of June 30, 2023, which are expected to fund our operational plans well into 2026. Research and Development Expenses: R&D expenses were $12.1 million for the quarter ended June 30, 2023, as compared to $7.7 million for the quarter ended June 30, 2022. The increase in R&D expenses were primarily driven by the progression of Disc’s portfolio, including bitopertin’s ongoing two Phase 2 clinical studies, increased headcount, and costs related to technology transfer and study preparations for MWTX-003. General and Administrative Expenses: G&A expenses were $5.2 million for the quarter ended June 30, 2023, as compared to $4.3 million for the same period in 2022. The increase in G&A expenses was primarily due to increased headcount. Net Loss: Net loss was $15.9 million for the quarter ended June 30, 2023, as compared to $9.6 million for the second quarter of 2022. The increase was primarily due to higher operating costs in the current period to support the continued advancement of our pipeline. 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 . 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 studies of bitopertin, and its Phase 1b/2 study of bitopertin in Diamond-Blackfan Anemia, its Phase 1b/2 clinical study of DISC-0974 in NDD-CKD patients with anemia, its anticipated Phase 1 study of MWTX-003 and potential development of MWTX-003 as a treatment for polycythemia vera and other indications, 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 and June 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. 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, 2023 ​ 2022 ​ 2023 ​ 2022 Operating expenses: ​ ​ ​ Research and development $ 12,100 ​ $ 7,714 ​ $ 32,280 ​ $ 15,535 General and administrative 5,228 ​ 4,301 ​ 10,173 ​ 6,440 Total operating expenses 17,328 ​ 12,015 ​ 42,453 ​ 21,975 Loss from operations (17,328 ) ​ (12,015 ) ​ (42,453 ) ​ (21,975 ) Other income (expense), net 1,405 ​ 2,435 ​ 3,772 ​ 2,542 Income tax expense (24 ) — (47 ) — Net loss $ (15,947 ) ​ $ (9,580 ) ​ $ (38,728 ) ​ $ (19,433 ) Weighted-average common shares outstanding-basic and diluted 21,484,955 ​ 944,706 ​ 20,226,923 ​ 934,286 Net loss per share-basic and diluted $ (0.74 ) ​ $ (10.14 ) ​ $ (1.91 ) ​ $ (20.80 ) DISC MEDICINE, INC. CONDENSED CONSOLIDATED BALANCE SHEETS (In thousands) (Unaudited) ​ June 30, December 31, ​ 2023 ​ 2022 Assets ​ ​ Cash and cash equivalents ​ $ 377,602 ​ $ 194,611 Other current assets ​ 4,131 ​ 3,880 Total current assets ​ 381,733 ​ 198,491 Non-current assets ​ 2,441 1,714 Total assets ​ $ 384,174 ​ $ 200,205 Liabilities and Stockholders’ Equity ​ ​ Current liabilities ​ $ 9,683 ​ $ 22,578 Non-current liabilities ​ 3,272 ​ 1,027 Total liabilities ​ 12,955 ​ 23,605 Total stockholders’ equity ​ 371,219 ​ 176,600 Total liabilities and stockholders’ equity ​ $ 384,174 ​ $ 200,205 Media Contact Peg Rusconi Verge Scientific Communications prusconi@vergescientific.com Investor Relations Contact Christina Tartaglia Stern Investor Relations christina.tartaglia@sternir.com