2025 EHA Management Call Corporate Update June 16, 2025 Disclaimer and FLS This presentation includes "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These forward-looking statements include express or implied statements relating to Disc's management team's expectations, hopes, beliefs, intentions or strategies regarding Disc's expectations with respect to its preclinical studies, clinical trials and research and development programs, in p ...

Summary of DISC Medicine Corporate Call Company Overview - **Company**: DISC Medicine - **Focus**: Development of therapies for rare hematologic diseases, particularly Erythropoietic Protoporphyria (EPP) and Myelofibrosis (MF) anemia Key Points Industry and Product Pipeline - **Bidapertin**: - NDA submission planned for the second half of the year for EPP [11] - Positive data from HELIOS study showing long-term efficacy and safety, with significant PP9 reduction and quality of life improvement [7][20] - Evidence of liver function improvement indicated by sustained ALT level reduction [7][20] - **DISCO-974**: - Monoclonal antibody targeting anemia of inflammation, with ongoing Phase II RALEY MF trial [28][50] - Durable efficacy observed in Phase 1b study, with sustained activity on key biomarkers [29][50] - Initial data from the RALEY MF trial expected later this year [50] - **DISC-3405**: - Monoclonal antibody for polycythemia vera, Phase II trial initiated [44][48] - Demonstrated ability to suppress iron absorption, with significant reductions in dietary iron uptake [47][48] Clinical Data and Efficacy - **Bidapertin**: - 86 patients in HELIOS study showed approximately 40% reduction in PP9 levels [17][19] - Patients on the 60 mg dose exhibited the greatest benefits [8][19] - Positive patient-reported outcomes with nearly universal positive responses regarding disease severity and quality of life [20][21] - **DISCO-974**: - Strong responses across various anemia patient categories, with major responders maintaining hemoglobin increases [31][32] - Evidence of synergistic effects when combined with other anemia-targeted agents [33][34] - **DISC-3405**: - Phase I study showed significant reductions in iron levels and hematologic responses in healthy volunteers [46][48] Commercialization and Market Opportunity - **EPP Market**: - Approximately 6,000 engaged EPP patients in the U.S. with a total of 14,000 diagnosed patients [24][25] - Focused commercial model targeting key treatment centers and healthcare professionals [25][26] - Disease state education campaign launched to raise awareness among patients and physicians [27] - **MF Anemia Market**: - Estimated 22,000 addressable patients in the U.S. with a significant unmet need for anemia treatment [39][40] - DISCO-974 aims to address anemia across all levels of transfusion dependence [41] Regulatory and Future Outlook - **Regulatory Progress**: - Pre-NDA meeting with the FDA scheduled to align on NDA package [12][49] - Enrollment for the APOLLO trial expected to be completed by Q2 next year [83] - **Future Milestones**: - Initial data from the RALEY MF trial and multiple dose portion of the Phase 1b study in chronic kidney disease expected later this year [50] - Initial results from the Phase II study in polycythemia vera anticipated in 2026 [48] Additional Insights - **Physician Engagement**: - Physicians are optimistic about the potential liver health benefits of bidapertin, which may influence prescription decisions [63] - Market research indicates that many EPP patients have been disengaged from healthcare due to a lack of treatment options, but awareness campaigns may drive re-engagement [84] - **Combination Therapies**: - Potential for combination therapies with existing anemia treatments like luspatercept, which may enhance patient outcomes [75][78] This summary encapsulates the key points discussed during the DISC Medicine corporate call, highlighting the company's pipeline, clinical data, market opportunities, and future regulatory steps.

Core Insights - Disc Medicine, Inc. is advancing its clinical programs for bitopertin, DISC-0974, and DISC-3405, with positive data presented at the EHA 2025 annual meeting, indicating favorable long-term efficacy and safety profiles for these investigational therapies [1][5][6] Bitopertin - The HELIOS study demonstrated sustained reductions in protoporphyrin IX (PPIX) and improvements in quality of life for patients with erythropoietic protoporphyria (EPP) [1][6] - Continuous treatment with 60 mg of bitopertin resulted in significant reductions in ALT and other hepatobiliary biomarkers, with nearly all participants reporting substantial quality of life improvements [6][4] - The company plans to submit a New Drug Application (NDA) for bitopertin in the second half of 2025 and has initiated a confirmatory clinical trial, APOLLO, for EPP [1][3] DISC-0974 - Data from the Phase 1b trial in myelofibrosis (MF) anemia showed sustained activity on key biomarkers and durable anemia responses among major responders [2][7] - The trial included various patient cohorts, with long-term results indicating that 50% of non-transfusion dependent patients achieved a major response with a sustained mean hemoglobin increase of ≥1.5 g/dL [10][17] - Enrollment for the Phase 2 RALLY-MF trial is ongoing, with initial data expected in the second half of 2025 [2][5] DISC-3405 - The Phase 1 trial in healthy volunteers demonstrated deep, sustained reductions in serum iron and significant changes in hematologic parameters, supporting its mechanism of action [2][9] - An iron pulse study showed that DISC-3405 inhibited dietary iron uptake, resulting in an average 94% reduction in iron absorption at Day 2 and 68% at Day 15 [12][13] - A Phase 2 trial of DISC-3405 in polycythemia vera (PV) has been initiated, with initial results anticipated in 2026 [11][17] Overall Company Strategy - The data presented at EHA supports the advancement of all three clinical candidates and highlights potential expansion opportunities into new indications [3][5] - Management will host a corporate update call on June 16, 2025, to discuss these developments further [5][3]

Summary of Disc Medicine (IRON) FY Conference Call Company Overview - **Company**: Disc Medicine - **Focus**: Non-malignant hematology, specifically manipulating red blood cell biology - **Lead Program**: Vitapertin targeting erythropoietic protoporphyria (EPP) with a projected NDA filing in the second half of 2025 [4][12][26] Core Points and Arguments Erythropoietic Protoporphyria (EPP) - **Disease Description**: Genetic condition caused by mutations in the heme biosynthetic pathway leading to excessive buildup of protoporphyrin IX (PP9), which causes severe pain and potential liver failure [6][8] - **Patient Impact**: Patients experience excruciating pain from light exposure, leading to significant lifestyle restrictions [7][8] - **Current Treatment Landscape**: Many patients resort to ineffective opioid therapy [7] Clinical Trials and Data - **Phase II Studies**: Completed two studies (AURORA and BEACON) with approximately 100 patients showing a significant reduction in PP9 levels (50-60% at the 60 mg dose) [10][11] - **Clinical Significance**: Reduction in PP9 correlates with improved quality of life and reduced phototoxic reactions [10][11] - **FDA Interaction**: Positive discussions with the FDA regarding the use of PP9 as a surrogate endpoint for accelerated approval [13][16] Upcoming Trials - **APOLLO Trial**: Designed as a confirmatory trial to support full approval, incorporating learnings from Phase II studies [18][21] - **Endpoints**: Co-primary endpoints include PP9 reduction and clinically meaningful measures of light exposure [21][22] Market Opportunity - **Patient Population**: Approximately 14,000 diagnosed EPP patients in the U.S., with 6,000 engaged patients based on claims analysis [27][28] - **Commercial Potential**: High motivation among patients to improve quality of life, leading to expected rapid uptake of the drug [28][29] Payer and Reimbursement Landscape - **Initial Interactions with Payers**: Payers recognize the severe nature of EPP and the potential value of effective treatments [31] - **Pricing Context**: Existing treatments for related conditions priced at $300,000 to $575,000 annually, indicating a willingness to pay for effective therapies [32] Second Asset: Nine Seven Four - **Indication**: Treatment for anemia associated with myelofibrosis, addressing a significant unmet need as no approved therapies exist for this condition [37][38] - **Phase II Study**: Ongoing with initial data expected in the second half of 2025 [41] Third Asset: Nine Thousand Nine Hundred Fourteen - **Indication**: Anemia in non-dialysis dependent chronic kidney disease (CKD) patients, with a significant unmet need in this population [48][49] Future Outlook - **Pipeline Development**: Disc Medicine aims to remain capital efficient while exploring new opportunities for pipeline expansion [59][60] - **Exciting Year Ahead**: Anticipation of NDA filing for Vitapertin and further data releases from ongoing studies [62] Important but Overlooked Content - **Patient Engagement**: High patient motivation and engagement in clinical trials, which may facilitate faster enrollment and data collection [26][27] - **Regulatory Environment**: Positive momentum from the FDA regarding accelerated approval pathways for rare diseases, which may benefit Disc Medicine's programs [16][46]

Disc Medicine (IRON) 2025 Conference June 05, 2025 10:30 AM ET Speaker0 Excellent. All right. Good morning, everyone. Welcome to twenty twenty five Jefferies Global Healthcare Conference. My name is Roger Song, one of the seniors covers MEKAP at Jefferies. It's my pleasure to have the next fireside chat with DISCMADISON and Theo Zhang. Welcome, Zhang. Speaker1 Yeah, great to be here. Speaker0 Awesome. All right. I think, you know, we had an interview with McCurry yesterday at our conference, and then pretty ...

Disc Medicine (IRON) FY 2025 Conference May 20, 2025 09:00 AM ET Speaker0 Okay. K. Good good morning, everybody. I'm Doug Tsao, senior analyst at H. C. Wainwright. We're thrilled to have us okay. K. Good good morning, everybody. I'm Doug Tsao. Okay. K. Good good morning, everybody. I'm Doug Tsao, senior analyst at H. C. Wainwright. We're thrilled to have us with us today, DISC Madison, represented by the company's CEO, John Quisel, and the CFO of CUNY French. And so thanks for being with us today, and it's ...

Poster presentations across all programs, including data from HELIOS long-term extension trial of bitopertin in erythropoietic protoporphyria (EPP), additional durability data from Phase 1b study of DISC-0974 in anemia of myelofibrosis (MF), and additional data from DISC-3405 studies in healthy volunteersManagement will host a corporate update conference call on Monday June 16 at 8:00 am ET / 2:00 pm CEST WATERTOWN, Mass., May 14, 2025 (GLOBE NEWSWIRE) -- Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage ...

Summary of Disc Medicine Conference Call Company Overview - **Company**: Disc Medicine - **Focus**: Hematology, specifically targeting pathways of red blood cell biology and iron metabolism - **Lead Programs**: Bidipertin for erythropoietin protoporphyria and DISCO-nine 74 for anemia of myelofibrosis [3][6][8] Industry Context - **Disease Focus**: Myelofibrosis, a type of blood cancer characterized by anemia, splenomegaly, and constitutional symptoms [16][22] - **Current Treatment Landscape**: - Four JAK inhibitors approved for myelofibrosis, primarily targeting spleen size reduction and symptom improvement [46][49] - No approved therapies specifically for anemia of myelofibrosis, leading to significant unmet medical needs [19][45] Key Points from the Call Myelofibrosis and Anemia - **Prevalence**: Approximately 25,000 patients in the U.S. diagnosed with myelofibrosis, with a five-year survival rate around 50% [26][27] - **Anemia Statistics**: - 40% of patients present with significant anemia at diagnosis, increasing to 60% within one year [36] - Nearly all patients will develop anemia over the disease course, with transfusion dependency rising from 25% at diagnosis to 45% within a year [36][37] - **Prognostic Importance**: Anemia is a significant prognostic factor, with lower hemoglobin levels correlating with worse survival outcomes [30][38] Current Treatment Limitations - **JAK Inhibitors**: While effective for managing symptoms, they often exacerbate anemia, leading to treatment discontinuation in many patients [60][61] - **Existing Treatments for Anemia**: Use of erythropoietin, steroids, and androgens, but these are not specifically approved for myelofibrosis-related anemia [49][50] Emerging Therapies - **DISCO-nine 74**: - A monoclonal antibody targeting hepcidin suppression to mobilize iron for red blood cell production [8][10] - Phase 1b data shows promise in treating anemia of myelofibrosis [14][19] - **Luspatercept and Elitracept**: - Both are in development as potential treatments for anemia, with luspatercept already approved for MDS [79][80] - Phase 3 trials for luspatercept are ongoing, focusing on patients requiring transfusions while on stable doses of JAK inhibitors [80] Market Opportunity - **Anemia of Myelofibrosis**: Significant market opportunity exists for effective treatments targeting anemia, given the high prevalence and unmet needs [19][45] - **Potential Impact of New Therapies**: New treatments could improve patient quality of life, reduce healthcare costs associated with transfusions, and address the underlying pathophysiology of anemia in myelofibrosis [40][41][44] Conclusion - The call emphasized the critical need for new therapies specifically targeting anemia in myelofibrosis, highlighting the potential of DISCO-nine 74 and other emerging treatments to fill this gap in the current treatment landscape [19][45][76]

WASHINGTON, D.C. 20549 FORM 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from __________________ to __________________ UNITED STATES SECURITIES AND EXCHANGE COMMISSION Commission File Number: 001-39438 Disc Medicine, Inc. (Exact name of registrant as specified in its charter) (State or other jur ...

Exhibit 99.1 Disc Medicine Reports First Quarter 2025 Financial Results and Provides Business Update WATERTOWN, Mass., May 7, 2025 – 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 first quarter ended March 31, 2025, and provided a recap of recent program and corporate developments. "2025 is off to a ...