WATERTOWN, Mass., Jan. 07, 2026 (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, announced today that John Quisel, J.D., Ph.D., President and Chief Executive Officer, will give a corporate presentation at the 44th Annual J.P. Morgan Healthcare Conference on Wednesday, January 14th at 3:00 pm PT / 6:00 pm ET. A live webcast ...

Core Viewpoint - The bullish outlook for Disc Medicine (IRON) is attributed to its strategic positioning that alleviates the common cash constraints faced by research-stage biotech companies, allowing it to focus on promising candidates [1] Group 1: Company Analysis - Disc Medicine has structured itself to overcome the typical financial challenges that hinder research-stage biotech firms, which often have innovative candidates but lack sufficient funding [1] - The company is positioned in a high-growth sector, indicating potential for exponential expansion and substantial returns through innovation [1] Group 2: Investment Strategy - The investment approach emphasizes a combination of fundamental analysis and future trend prediction, focusing on disruptive technologies and forward-thinking enterprises [1]

Core Insights - Disc Medicine Inc. is recognized as a promising biotech stock by Wall Street analysts, with price target increases from multiple firms following positive developments in its drug pipeline [1][2][3] Group 1: Price Target Increases - Leerink Partners raised the price target for Disc Medicine Inc. to $115 from $110, maintaining an Outperform rating [1][2] - Raymond James also increased its price target to $117 from $108, reiterating a Strong Buy rating [3] Group 2: Drug Development and Clinical Trials - The price target hikes are attributed to the acceptance of the New Drug Application for bitopertin, an oral GlyT-1 inhibitor for erythropoietic protoporphyria (EPP) by the FDA [2] - The company reported topline interim data from the Phase 2 RALLY-MF trial in patients with myelofibrosis anemia, which has bolstered confidence in the drug's prospects [2][3] - Revenue forecasts for the company have been raised significantly to approximately $748 million from $359 million, reflecting optimism about the success of its candidate drug DISC-0974 in myelofibrosis [2] Group 3: Company Overview - Disc Medicine, Inc. is a clinical-stage biopharmaceutical company focused on developing treatments for serious hematologic diseases by targeting red blood cell biology, specifically heme production and iron balance [4]

Core Points - The conference call is hosted by John Quisel, the CEO of the company, indicating a focus on management and strategic discussions [1] Group 1 - The call is part of a management conference, suggesting an emphasis on operational updates and future direction [1] - The recording of the conference indicates that the information shared may be significant for stakeholders and investors [1]

Summary of Disc Medicine Management Call - December 07, 2025 Company Overview - **Company**: Disc Medicine (NasdaqGM:IRON) - **Focus**: Development of therapies for hematologic diseases, particularly myelofibrosis (MF) and related anemias Key Updates on Products and Trials Bitopertin - **Status**: NDA accepted for review by the FDA for accelerated approval for erythropoietic protoporphyria (EPP) with a potential launch by the end of January 2026 [3][6][8] - **Review Process**: Engaged in a rigorous review process with the FDA, characterized by increased communication and collaboration [6][41] - **Commercialization**: Accelerated efforts for launch readiness, including drug supply expected by the end of January 2026 [7][8] DISC-0974 - **Indication**: Treatment for anemia of myelofibrosis (MF) - **Trial Updates**: Phase II RALLI-MF study shows significant decreases in hepcidin, leading to improved hemoglobin levels and reduced transfusion burden [4][10][12] - **Response Rates**: - Non-transfusion-dependent (NTD) group: 63% overall response, 50% major response - Transfusion-dependent (TD) low group: 71% overall response, with consistent results across different JAK inhibitors [12][14] - **Safety Profile**: Well tolerated with no treatment-related serious adverse events reported [14] DISC-3405 - **Mechanism**: Increases endogenous hepcidin production to limit iron availability, targeting conditions like polycythemia vera and sickle cell disease [21][22] - **Trial Initiation**: Phase II trials for polycythemia vera and sickle cell disease initiated, with initial data expected in 2026 [26][27] Market Opportunity - **Anemia of MF**: Affects approximately 22,000 patients in the U.S., representing a significant unmet need [17][18] - **Potential for Blockbuster Status**: DISC-0974 is positioned to address this unmet need effectively, with high response rates and the ability to work alongside existing therapies [19][18] Future Plans - **Regulatory Engagement**: Plans for an end-of-phase II meeting with the FDA for DISC-0974 to align on registrational trial design [19] - **Expansion of Trials**: Plans to initiate a phase II study in anemia of inflammatory bowel disease (IBD) and explore other anemias of inflammation [19][55] Financial and Operational Updates - **Funding**: Completed two follow-on offerings, providing a runway into 2029 without relying on revenue from bitopertin [29] - **Sales Force Preparation**: Hiring 24 sales representatives in anticipation of bitopertin's launch [90] Additional Insights - **Patient Experience**: Anecdotal reports indicate patients feel significantly better on DISC-0974, with improvements in fatigue and overall well-being [70] - **Combination Therapy Potential**: Ongoing discussions about potential combination studies with other agents to enhance efficacy [49][50] Conclusion Disc Medicine is making significant strides in the development of therapies for hematologic conditions, with promising data from ongoing trials and a clear path toward commercialization of its lead products. The company is well-positioned to address significant unmet needs in the market, particularly in the treatment of anemia associated with myelofibrosis.

Summary of DISC Medicine Management Conference Call Company Overview - **Company**: DISC Medicine (NasdaqGM:IRON) - **Focus**: Development of therapies for anemia and related conditions, particularly in myelofibrosis (MF) and other hematological disorders Key Industry and Company Updates Bitopertin - **NDA Status**: The New Drug Application (NDA) for bitopertin has been accepted for review by the FDA, with a potential approval decision expected by the end of January 2026 [6][12][73] - **Commercialization Readiness**: The company is accelerating its commercialization efforts, with plans to hire 24 sales representatives across the U.S. [7][158] - **Regulatory Engagement**: The FDA is providing increased engagement and communication during the review process, which is part of the Commissioner's National Priority Voucher Program [11][73] DISC-0974 - **Rally MF Trial**: Initial data from the phase 2 Rally MF study shows significant decreases in hepcidin, leading to improved iron mobilization and clinically meaningful benefits in anemia measures such as hemoglobin levels and fatigue [8][22][27] - **Response Rates**: The overall response rate in the non-transfusion dependent (NTD) group is 63%, while the transfusion dependent low (TDLO) group shows a 50% major response rate [22][23] - **Market Opportunity**: Anemia of MF affects approximately 22,000 patients in the U.S., presenting a significant market opportunity for DISC-0974, which has shown high response rates and durability [35][37] DISC-3405 - **Mechanism of Action**: DISC-3405 aims to increase endogenous hepcidin production to limit iron availability, targeting conditions like polycythemia vera and sickle cell disease [43][45] - **Trial Updates**: The phase 2 trial for polycythemia vera has been expanded due to rapid enrollment, and initial data is expected in 2026 [9][48] Core Insights and Arguments - **Efficacy Across Background Therapies**: DISC-0974 shows consistent efficacy regardless of concomitant JAK inhibitors, indicating its potential as a versatile treatment option [26][70] - **Safety Profile**: The safety profile of DISC-0974 is consistent with background adverse events typical for the MF population, with no treatment-related serious adverse events reported [27] - **Future Development Plans**: The company plans to initiate a phase 2 study in anemia of inflammatory bowel disease (IBD) and is exploring the potential of a long-acting version of DISC-0974 [41][90] Additional Important Points - **Patient Experience**: Anecdotal reports suggest patients are experiencing significant improvements in quality of life and reduced fatigue while on DISC-0974 [118] - **Regulatory Landscape**: The acceptance of bitopertin's NDA under the accelerated approval pathway is a significant milestone, potentially paving the way for future therapies [73] - **Market Dynamics**: The company is positioning itself to address a serious unmet need in the anemia market, particularly for MF patients who currently have limited treatment options [40][41] This summary encapsulates the critical updates and insights from the DISC Medicine management conference call, highlighting the company's strategic direction and the potential impact of its therapies in the hematology space.

Core Insights - Disc Medicine, Inc. presented positive initial data from the RALLY-MF Phase 2 trial of DISC-0974 for treating anemia in myelofibrosis (MF) at the ASH Annual Meeting, showing significant reductions in hepcidin and increases in iron levels, leading to clinically meaningful improvements in anemia across various patient types [1][2]. Group 1: Trial Results - The ongoing Phase 2 trial enrolled 47 adult patients with MF and anemia, with 34 patients included in the responder analysis [2]. - DISC-0974 treatment resulted in over 75% reduction in hepcidin and significant increases in serum iron [6]. - 63% of non-transfusion dependent (nTD) patients achieved a hemoglobin increase of ≥1 g/dL for ≥12 weeks, while 50% had an increase of ≥1.5 g/dL for ≥12 weeks [6]. - 71% of low transfusion burden patients achieved transfusion independence over a 16-week period [6]. - 67% of high transfusion burden patients achieved a ≥50% reduction in transfusion requirement [6]. - 50% of patients receiving concomitant JAK inhibitor therapy achieved a major hematologic response [6]. Group 2: Treatment Administration and Tolerability - DISC-0974 was administered subcutaneously at a dose of 50 mg every 4 weeks for up to 6 treatments [2]. - The treatment was generally well-tolerated, with diarrhea and urinary tract infections being the only related adverse events reported in two or more subjects [6]. Group 3: Future Plans and Developments - The company plans to advance the DISC-0974 program to address the unmet need for anemia treatment in MF, as there are currently no approved therapies for this condition [2]. - Additional data from the trial is expected to be shared in the second half of 2026 [6].

Company Overview - The company, Disc, focuses on controlling red blood cell biology by managing the metabolism of iron and heme, which are essential for red blood cell formation [2] - The lead program, Bitopertin, is in-licensed from Roche and aims to control heme biosynthesis in red blood cells [2] Key Programs - Bitopertin is being studied for a rare disease called erythropoietic protoporphyria (EPP), with approximately 14,000 diagnosed patients in the U.S. [2] - The company has completed a Phase II program for Bitopertin and is currently in an accelerated approval process with the FDA [2] - The approval process was further expedited by receiving a Commissioner's National Priority Voucher [2]

Summary of Disc Medicine Conference Call Company Overview - **Company**: Disc Medicine (NasdaqGM:IRON) - **Focus**: Controlling red blood cell biology through iron and heme metabolism - **Lead Program**: Bitopertin, licensed from Roche, targeting erythropoietic protoporphyria (EPP) with approximately 14,000 diagnosed patients in the US [4][15] Key Programs and Developments 1. **Bitopertin for EPP** - **Status**: Submitted NDA at the end of September 2025, in accelerated approval process with FDA [5][11] - **Timeline**: Potential approval and launch expected late 2025 to early 2026, accelerated by a commissioner’s national priority voucher [5][12] - **Sales Force**: Team of 24 prepared to engage with physicians treating the 6,000 highly engaged patients identified [17] 2. **DISC-0974 for Myelofibrosis** - **Phase**: Ongoing phase 2 trial (RALI-MF) with data expected at ASH 2025 [5][26] - **Data**: Previous data showed unprecedented positive response rates in treating anemia in myelofibrosis patients [5][27] - **Market Size**: Approximately 22,000 anemic myelofibrosis patients in the US could benefit from this therapy [34] 3. **Polycythemia Vera Program** - **Status**: Phase 2 trial initiated, focusing on restricting iron to manage excess red blood cell production [44] Financial Position - **Balance Sheet**: Approximately $825 million available, sufficient to fund operations until 2029 without revenue [46] Competitive Landscape - **Current Competitors**: JAK inhibitors like Jakafi exacerbate anemia, creating a market opportunity for anemia-specific therapies [27][42] - **Emerging Competitors**: Incyte's early clinical programs targeting calreticulin mutations, but efficacy in managing anemia remains uncertain [41][43] Important Considerations - **Patient Engagement**: Identified patient population is engaged, with a significant number already receiving treatment, facilitating quicker access to therapy post-approval [15][18] - **Reimbursement Pathways**: Initial reimbursement pathways may be less established at launch, requiring strategies like free drug programs [13][20] - **Regulatory Strategy**: Plans for a pivotal trial with broad enrollment criteria to support rapid patient recruitment [39][40] Upcoming Milestones - **ASH Presentation**: Expected data from the RALI-MF trial to be presented, with insights into response rates across different patient categories [28][29] - **Pivotal Trial Design**: Anticipated discussions with FDA post-phase 2 trial completion to outline pivotal trial strategy [39][40] This summary encapsulates the key points discussed during the conference call, highlighting the strategic direction, clinical programs, financial health, and competitive landscape of Disc Medicine.

Company Overview - Disc Medicine focuses on developing treatments for serious and debilitating hematologic diseases, with a name that reflects the shape of red blood cells [2] - The company's portfolio is constructed by targeting pathways fundamental to red blood cell biology, including programs that affect heme synthesis and regulate iron metabolism through the hormone Hepcidin [2] Lead Program - The lead program of the company is called Bitopertin, which is an oral treatment [3] Growth and Development - The company is currently experiencing significant growth, with ongoing developments in its portfolio that have been followed by Stifel for some time [1]