Protoporphyria, Erythropoietic, X-Linked Dominant

Patient Special Access Program continues during Health Canada reviewMELBOURNE, Australia, Oct. 01, 2024 (GLOBE NEWSWIRE) -- CLINUVEL has filed a New Drug Submission (NDS) to Health Canada, seeking approval for its novel photoprotective therapy SCENESSE® (afamelanotide) for the prevention of phototoxicity in adult patients with erythropoietic protoporphyria (EPP). If approved, SCENESSE® would be the first treatment for Canadian EPP patients. Health Canada review process Health Canada’s Health Products and Food Branch (HPFB) reviews and approves medications for use in Canada, assessing the safety and efficacy of the products in the proposed indication, as well as the drug’s quality. Following a formal dossier validation period, the HPFB may complete the review of a new drug candidate within 300 days. Canadian Special Access Program ongoing In 2023 CLINUVEL announced that the first Canadian EPP patient had received treatment with SCENESSE® under Canada’s Special Access Program (SAP). The SAP allows individual physicians to facilitate access to treatment for patients who have serious or life-threatening conditions and lack therapeutic alternatives. Patient treatment under the SAP continued without interruption prior to Health Canada’s review of the NDS. All Canadian patients treated under the SAP have received insurance coverage to support their treatment access. Two Canadian Specialty Centers have been trained and accredited to treat EPP patients with SCENESSE®. Further potential Canadian treatment centres have been identified to enable prompt treatment access pending regulatory and pricing approvals. To date, CLINUVEL has trained and accredited 85 Specialty Centers across North America. EPP affects approximately 1:140,000 individuals, with an estimated 280 EPP patients in Canada. Commentary “The SAP has provided an important bridge for Canadian patients to access treatment and helped us understand the Canadian therapeutic landscape,” CLINUVEL’s Chief Scientific Officer, Dr Dennis Wright said. “A formal authorisation will enable more Canadian patients to receive SCENESSE® and is a logical next step. “The dossier submitted contains both data which led to the FDA’s approval, as well as long-term data collected during the follow up of EPP patients worldwide,” Dr Wright concluded. SCENESSE® in EPP: systemic photoprotection EPP is a rare genetic disorder which causes phototoxicity, debilitating reactions and burns following light exposure. CLINUVEL has spent nearly two decades developing SCENESSE® as the first treatment for EPP. The drug, administered as a controlled-release injectable implant every 60 days, stimulates the production of melanin in skin, protecting skin cells from visible and ultraviolet light (photoprotection) and acting as a strong antioxidant. Clinical and long-term post-marketing studies of SCENESSE® have shown that it can prevent and reduce the severity of phototoxic reactions, as well as improving patients’ quality of life. The drug has been approved for adults by the European Medicines Agency, US Food and Drug Administration (FDA), and regulatory authorities in Australia and Israel. To date, over 16,000 doses of SCENESSE® have been administered to EPP patients worldwide. References Ceresnie, M. S., et al. (2022). Association of quality of life measures with afamelanotide treatment in patients with erythropoietic protoporphyria and x-linked protoporphyria: A retrospective cohort study. Journal of the American Academy of Dermatology, S0190962222028729. Elder, G., et al. (2013). The incidence of inherited porphyrias in Europe. Journal of Inherited Metabolic Disease, 36(5), 849–857. Langendonk, J., et al. (2015). Afamelanotide for Erythropoietic Protoporphyria. The New England Journal of Medicine, 373(1), 48–59. Wensink, D., Wagenmakers, M. A. E. M., & Langendonk, J. G. (2021). Afamelanotide for prevention of phototoxicity in erythropoietic protoporphyria. Expert Review of Clinical Pharmacology, 14(2), 151–160. About CLINUVEL PHARMACEUTICALS LIMITED CLINUVEL (ASX: CUV; ADR LEVEL 1: CLVLY; Börse Frankfurt: UR9) is a global specialty pharmaceutical group focused on developing and commercialising treatments for patients with genetic, metabolic, systemic, and life-threatening, acute disorders, as well as healthcare solutions for specialised populations. As pioneers in photomedicine and the family of melanocortin peptides, CLINUVEL’s research and development has led to innovative treatments for patient populations with a clinical need for systemic photoprotection, assisted DNA repair, repigmentation and acute or life-threatening conditions who lack alternatives. CLINUVEL’s lead therapy, SCENESSE® (afamelanotide 16mg), is approved for commercial distribution in Europe, the USA, Israel, and Australia as the world’s first systemic photoprotective drug for the prevention of phototoxicity (anaphylactoid reactions and burns) in adult patients with erythropoietic protoporphyria (EPP). Headquartered in Melbourne, Australia, CLINUVEL has operations in Europe, Singapore, and the USA. For more information, please go to https://www.clinuvel.com. Authorised for ASX release by the Board of Directors of CLINUVEL PHARMACEUTICALS LTD. Head of Investor RelationsMr Malcolm Bull, CLINUVEL PHARMACEUTICALS LTD Investor Enquirieshttps://www.clinuvel.com/investors/contact-us Forward-Looking Statements This release contains forward-looking statements, which reflect the current beliefs and expectations of CLINUVEL’s management. Please see CLINUVEL’s website for the full Forward-Looking Statements disclaimer. Contact:Tel: +61 3 9660 4900Fax: +61 3 9660 4909Email: mail@clinuvel.comAustralia (Head Office), Level 22, 535 Bourke Street, Melbourne, Victoria, 3000, Australia CLINUVEL on InstagramCLINUVEL on XCLINUVEL on LinkedInCLINUVEL on FacebookCLINUVEL on YouTube

Top-line results from AURORA, the placebo-controlled phase 2 study of bitopertin in erythropoietic porphyrias (EPP), expected March / April 2024On track to deliver multiple read-outs in 2024, including updated results from phase 1b/2 study of DISC-0974 in anemia of myelofibrosis (MF) 1H’24Strengthened leadership team with the appointments of Jean Franchi as Chief Financial Officer and Pamela Stephenson as Chief Commercial Officer, and the promotion of Jonathan Yu to Chief Operating OfficerWell-capitalized, ending 2023 with approximately $360M in cash and cash equivalents, which provides runway well into 2026 WATERTOWN, Mass., March 21, 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 fourth quarter and full year ended December 31, 2023, and provided an update on recent program and corporate developments. “The past year has been truly remarkable for Disc and marks another important chapter in our company’s story. During 2023, we provided the first evidence of the therapeutic potential of our programs, with positive read-outs in patients for both bitopertin and DISC-0974. In addition, we advanced a third program, DISC-3405, into the clinic and fortified our balance sheet, so we can execute our strategy with confidence,” said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc. “These achievements have positioned us well for 2024, which promises to be another transformational year, and I want to express my deep gratitude to our team for their determination and hard work. We’re looking forward to sharing AURORA data in the coming weeks, as well as updated data for DISC-0974 in MF and initial data for DISC-3405 in healthy volunteers in the first half of this year.” Recent Business Highlights and Upcoming Milestones: Bitopertin: GlyT1 Inhibitor (Heme Synthesis Modulator) Completed enrollment of its phase 2 program of bitopertin in erythropoietic porphyrias: BEACON, an open-label phase 2 clinical study of bitopertin in patients with EPP and X-linked protoporphyria (XLP), which was expanded to include adolescentsAURORA a phase 2 randomized, placebo-controlled clinical study of bitopertin in adults with EPP Presented positive results from the phase 2 BEACON trial at ASH 2023, showing: Significant, sustained reductions in whole blood protoporphyrin IX (PPIX) levels >40%Robust and consistent improvements across all measures of sunlight tolerance, including >3x improvement over historical control of precedented pivotal endpointBitopertin was generally well-tolerated with stable hemoglobin at both dose levels Topline AURORA data planned for press release and corporate presentation in March/April 2024 DISC-0974: Anti-Hemojuvelin Antibody (Hepcidin Suppression) Presented positive, initial data from ongoing phase 1b/2 clinical study in MF patients with severe anemia at ASH 2023: Treatment at initial dose levels (14 mg, 28 mg, 50 mg) resulted in substantial, dose-dependent decreases in hepcidin and increases in serum ironHematologic activity was observed in MF patients, regardless of concomitant JAK inhibitor use. Four of seven (57%) evaluable non-transfusion-dependent (NTD) patients reached a >1.5g/dL hemoglobin increase and one of two transfusion-dependent (TD) patients achieved transfusion independence.DISC-0974 was generally well-toleratedEnrollment and dose escalation is ongoing Presented initial data from the 28 mg cohort of the ongoing phase 1b/2 clinical study of patients with anemia of chronic kidney disease who are not receiving dialysis (NDD-CKD) Data demonstrated meaningful reductions in serum hepcidin and corresponding increases in serum iron with a similar PK/PD profile as seen in Disc’s healthy volunteer studyEnrollment and dose escalation is ongoing Updated phase 1b/2 data from DISC-0974 in anemia in MF patients is expected to be presented in the first half of 2024Updated phase 1b/2 data from DISC-0974 in anemia in NDD-CKD is expected to be shared in the second half of 2024Received FDA fast track designation in February 2024 for DISC-0974 for the treatment of anemia in NDD-CKD DISC-3405: Anti-TMPRSS6 Antibody (Hepcidin Induction) Initiated a phase 1 SAD / MAD study in healthy volunteers in October 2023Initial SAD data expected to be presented in the first half of 2024Received FDA orphan drug designation in February 2024 for DISC-3405 for the treatment of PV Corporate: As the company enters late-stage development and begins planning for commercialization, Disc expanded its leadership team with the appointments of industry veterans Jean Franchi as Chief Financial Officer and Pamela Stephenson as Chief Commercial Officer, and the promotion of Jonathan Yu to Chief Operating Officer Full Year 2023 Financial Results: Cash Position: Cash and cash equivalents were $360.4 million as of December 31, 2023, which are expected to fund our operational plans well into 2026, compared to $194.6 million as of December 31, 2022. The increase was primarily due to the completion of a registered direct offering in February 2023 and a publicly marketed follow-on offering in June 2023.Research and Development Expenses: R&D expenses were $69.3 million for the full year ended December 31, 2023, as compared to $33.4 million for the full year ended December 31, 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, phase 1 study initiation for DISC-3405, and increased headcount.General and Administrative Expenses: G&A expenses were $21.9 million for the full year ended December 31, 2023, as compared to $14.0 million for the full year ended December 31, 2022. The increase in G&A expenses was primarily due to increased headcount and increased costs as a public company.Net Loss: Net loss was $76.4 million for the full year ended December 31, 2023, as compared to $46.8 million for the full year ended December 31, 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 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; Disc’s business plans, objectives and expected contributions of management; 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 and DISC-0974; Disc’s ability to retain and recognize the intended incentives conferred by Orphan Drug Designation for DISC-3405 and bitopertin; 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, 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) ​Year Ended December 31, ​ 2023 ​ 2022 Operating expenses:​ ​ Research and development​$69,264 ​$33,437 General and administrative​ 21,861 ​ 14,038 Total operating expenses​ 91,125 ​ 47,475 Loss from operations​ (91,125)​ (47,475)Other income (expense), net 14,795 ​ 648 Income tax expense​ (99) — Net loss​$(76,429)​$(46,827)Weighted-average common shares outstanding-basic and diluted​ 22,315,877 1,039,490 Net loss per share-basic and diluted​$(3.42)​$(45.05) DISC MEDICINE, INC. CONDENSED CONSOLIDATED BALANCE SHEETS (In thousands) ​December 31, December 31, ​ 2023 ​ 2022 Assets​ ​ Cash and cash equivalents​$360,382 ​$194,611 Other current assets​ 5,280 ​ 3,880 Total current assets​ 365,662 ​ 198,491 Non-current assets​ 2,334 1,714 Total assets​$367,996 ​$200,205 Liabilities and Stockholders’ Equity​ ​ Current liabilities​$21,439 ​$22,578 Non-current liabilities​ 1,436 ​ 1,027 Total liabilities​ 22,875 ​ 23,605 Total stockholders’ equity​ 345,121 ​ 176,600 Total liabilities and stockholders’ equity​$367,996 ​$200,205 Media Contact Peg RusconiVerge Scientific Communicationsprusconi@vergescientific.com Investor Relations Contact Christina TartagliaStern Investor Relationschristina.tartaglia@sternir.com

Consistent and dose-dependent reductions of protoporphyrin IX (PPIX), the disease-causing metabolite in EPP, were observed in patients treated with bitopertin Patients reported significant improvements in sunlight tolerance and measures of quality-of-life Bitopertin was well-tolerated, with no meaningful changes in hemoglobin observed Disc Medicine to host an investor conference call today at 7:30 AM ET WATERTOWN, Mass., June 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 presented preliminary findings from its ongoing, Phase 2 open-label BEACON trial evaluating bitopertin, an orally administered glycine transporter 1 (GlyT1) inhibitor, in patients with erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) at the European Hematology Association (EHA) 2023 Congress in Frankfurt, Germany. The initial trial data demonstrated consistent decreases in PPIX, significant increases in reported sunlight tolerance and improvements in measures of patient quality of life. “We’re delighted to share these initial, positive data from BEACON, which provide the first clinical evidence supporting our therapeutic hypothesis of bitopertin in EPP. Over the next 12 months, we plan to build on this momentum with a series of additional clinical read-outs across our portfolio,” said John Quisel, J.D., Ph.D., Chief Executive Officer and President of Disc Medicine. “This is an important moment for Disc as a company, and I want to extend my gratitude to our team, collaborators, and most importantly, the patients and families participating in BEACON.” “We are excited to share these initial data from the BEACON trial, where we observed consistent and sustained suppression of PPIX, the disease-causing metabolite in EPP, in patients treated with bitopertin,” said Will Savage, M.D., Ph.D., Chief Medical Officer at Disc Medicine. “Importantly, this reduction translated into significant improvements in the time that patients can spend in sunlight without reporting pain or symptoms related to their disease. We’re encouraged by the data and plan to present additional data at the end of the year.” The BEACON trial is a randomized, open-label, parallel-arm trial enrolling up to 22 patients with EPP or XLP at trial sites in Australia. This trial was designed to assess changes in levels of PPIX, as well as measures of photosensitivity, quality of life, and safety and tolerability. Subjects are randomized to receive either 20 mg or 60 mg of bitopertin once-daily for 24 weeks, after which patients have the option of continuing in an open-label extension of the trial for up to an additional 24 weeks. The trial is ongoing and these data reflect initial data from 15 subjects enrolled as of the data cutoff of May 8, 2023, with a range of treatment durations from 18 days to 6 months. Due to batch processing of samples, the data cutoff for PPIX data was April 7, 2023. Highlights of the initial data presented: Protoporphyrin IX (PPIX) levels: Significant, consistent, dose-dependent, and sustained reductions of whole-blood, metal-free PPIX; mean reduction of >40% when compared to baseline Measures of light tolerance (individual) from two participants with the longest follow-up demonstrated substantial increases in sunlight tolerance as measured by time in sunlight without experiencing a prodrome (initial symptoms that signal a pain attack), or “sunlight challenge”: A participant on 20 mg bitopertin reported a >80-fold increase in sunlight tolerance on day 88 of treatment, increasing from 4.5 minutes at baseline to over 6 hours; the participant did not report a prodrome during any sunlight challenge after Day 20 A participant on 60 mg bitopertin reported a >200-fold increase in sunlight tolerance on day 74 of treatment, increasing from 1.25 minutes at baseline to over 4 hours, and did not report a prodrome during any sunlight challenge after Day 120 Measures of light tolerance (aggregated across participants from whom data was available in the trial): Average weekly total time spent in sunlight: increased from 344 minutes (approximately 49 minutes per day) to 1,200 minutes at Week 24 Time to prodrome during sunlight challenge (averaged over a two-week period): increased >7-fold, from 25 minutes at baseline to 182 minutes at Week 24 Increased proportion of days without symptoms: 75% vs. 25% (baseline) Increased proportion of sunlight challenges without prodromes: 50% vs. 0% (baseline) Phototoxic reactions: 96% reduction in patient-reported phototoxic reactions while on treatment compared to baseline (n=15) Measures of patient quality of life Patient Global Impression of Change (PGIC): All 10 patients that had completed a day 43 visit reported their disease was much better (n=8) or a little better (n=2) in the last 7 days Patient Global Impression of Severity (PGIS): Nine out of 10 patients that had completed a day 43 visit reported their EPP was mild (n=3) or not at all severe (n=6) EPP Impact Questionnaire (EPIQ): For patients whose most recent data was Day 43, 4/8 patients reported an improvement in the impact of EPP on quality of life and 4/8 reported no change in the impact of EPP on quality of life. For patients whose most recent data was after Day 43, 2/2 reported marked improvement in the impact of EPP on quality of life, reporting no impact of EPP on quality of life. Bitopertin was well-tolerated at both dose levels with no reported serious adverse events, no reported discontinuations or dose reductions, no reported adverse events greater than Grade 1, and no meaningful changes observed in mean hemoglobin levels These data were presented at the European Hematology Association 2023 Congress in Frankfurt, Germany and the poster is available on the EHA Congress platform at . Management will host a call to review the presented data on Friday, June 9th at 7:30 am ET. Please register for the event on the Events and Presentations page of Disc’s website ( ). About Bitopertin 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. There are currently two ongoing Phase 2 clinical trials of bitopertin in patients with erythropoietic porphyria, including an open-label trial called BEACON and a randomized, double-blind placebo-controlled trial called AURORA. Bitopertin is an investigational agent and is not approved for use as a therapy in any jurisdiction worldwide. Disc obtained global rights to bitopertin under a license agreement from Roche in May 2021. About Erythropoietic Protoporphyria (EPP) and X-linked Protoporphyria (XLP) Erythropoietic protoporphyria (EPP) and X-linked Protoporphyria (XLP) are rare, debilitating and potentially life-threatening diseases caused by mutations that affect heme biosynthesis, resulting in the accumulation of a toxic, photoactive intermediate called protoporphyrin IX (PPIX). This causes severe reactions when patients are exposed to sunlight, characterized by excruciating pain, edema, burning sensations and potential blistering and disfigurement. PPIX also accumulates in the hepatobiliary system and can result in complications including gallstones, cholestasis, and liver damage in 20-30% of patients and in extreme cases liver failure. Current standard of care involves extreme measures to avoid sunlight, including restricting outdoor activities to nighttime, use of protective clothing and opaque shields, and pain management. This has a significant impact on the psychosocial development, quality of life, and daily activities of patients, particularly in young children and families. There is currently no cure for EPP and only one FDA-approved therapy, a surgically implanted synthetic hormone designed to stimulate melanin production called Scenesse® (afamelanotide). 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 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 BEACON Phase 2 clinical trial of bitopertin and projected timelines for the initiation and completion of its clinical trials, the timing of additional data and other activities. The use of words such as, but not limited to, “aim,” “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 data 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 data 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 data of Disc’s preclinical studies and clinical trials and the risk that the data of Disc’s preclinical studies and clinical trials may not be predictive of future data 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 Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on May 15, 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