Financial Data and Key Metrics Changes - The company is preparing to file its New Drug Application (NDA) for bitopertin in October, with a potential PDUFA date between June and October of the following year, marking a significant milestone for the company [4][5] - The company reported a P-value of less than 0.001 for the reduction in protoporphyrin-9 (PP9), indicating a clinically meaningful reduction in patients with erythropoietic protoporphyria [9][10] Business Line Data and Key Metrics Changes - The lead program, bitopertin, is in the accelerated approval pathway with the FDA, focusing on rare diseases related to red blood cell biology [4][5] - The second program is expected to present data on anemia of myelofibrosis and anemia of chronic kidney disease at upcoming conferences [5][6] Market Data and Key Metrics Changes - The company identified approximately 14,000 diagnosed patients in the U.S. for the target disease, with enrollment in trials not being a problem due to strong patient advocacy [18][23] - The company plans to leverage claims data to identify and validate physicians treating these patients, enhancing their market access strategy [23][24] Company Strategy and Development Direction - The company is focused on increasing disease awareness and ensuring that patients and physicians are informed about available therapies, as historically there have been no options for these patients [22][23] - The company aims to build a self-sustaining entity driven by revenues from bitopertin, with plans to explore additional indications within their iron modulation programs [60][61] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the regulatory pathway for bitopertin, citing positive interactions with the FDA regarding the use of PP9 as a surrogate endpoint [10][11] - The management highlighted the importance of continuous engagement with healthcare providers to ensure that patients receive appropriate care and treatment options [26][30] Other Important Information - The company has a strong financial position with $650 million on the balance sheet, which is expected to fund operations into 2028 [60] - The company has orphan protection for bitopertin, providing seven years of exclusivity in the U.S. plus potential pediatric extensions [36] Q&A Session Summary Question: Can you recap the interactions with the FDA regarding the accelerated approval path for bitopertin? - Management detailed a series of meetings with the FDA that led to the decision to pursue an accelerated approval pathway, emphasizing the strong rationale for using PP9 as a surrogate endpoint [10][11] Question: What are the key differences between the Apollo and Aurora trials? - Management explained that the Apollo trial will focus on measuring the time patients can spend in light, which is a clinically meaningful endpoint, alongside the reduction in PP9 levels [18][19] Question: How does the company plan to address the patient population for bitopertin? - Management discussed the importance of identifying engaged patients who regularly seek healthcare, estimating about 6,000 such patients, while also planning to reach out to the remaining diagnosed but less engaged patients [26][27] Question: What is the expected market opportunity for the company's anemia treatments? - Management indicated that there is a significant unmet need for anemia treatments in myelofibrosis, with an estimated market of about 22,000 patients, and potential pricing similar to existing therapies [42][44] Question: How does the company view its capital position and future financing needs? - Management expressed confidence in their capital position, indicating that they are well-funded to support ongoing trials and commercial preparations without immediate need for additional equity financing [60][61]

Financial Data and Key Metrics Changes - The company is preparing to file its New Drug Application (NDA) for bitopertin in October, with a potential PDUFA date between June and October of the following year, marking a significant milestone for the company [4][5] - The company reported a strong reduction in protoporphyrin-9 (PP9) levels with a P-value of less than 0.001, indicating clinically meaningful results from its phase 2 studies [9][10] Business Line Data and Key Metrics Changes - The lead program, bitopertin, is focused on treating erythropoietic protoporphyria, with the company ramping up efforts for its launch [4][5] - The second program is expected to present data on anemia of myelofibrosis and anemia of chronic kidney disease at upcoming conferences [5][6] Market Data and Key Metrics Changes - The company identified approximately 14,000 diagnosed patients in the U.S. for its lead indication, with enrollment in trials not being a problem due to strong patient advocacy [19][24] - The company plans to target the European and Japanese markets, with the genetic prevalence in Japan estimated to be four times higher than in the U.S. [39] Company Strategy and Development Direction - The company is focused on raising disease awareness and ensuring that patients and physicians are informed about available therapies, which is crucial given the historical lack of treatment options [23][24] - The company aims to build a self-sustaining entity driven by revenues from bitopertin to support its broader heme portfolio and explore larger indications [62] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the regulatory pathway for bitopertin, highlighting positive interactions with the FDA regarding the accelerated approval process [11][12] - The company is well-funded with $650 million on the balance sheet, providing a runway into 2028 to support ongoing trials and commercial preparations [62] Other Important Information - The company has orphan protection for bitopertin, which provides seven years of exclusivity in the U.S., along with method-of-use patents extending until 2042 [37] - The safety profile of bitopertin shows that dizziness occurs in over 50% of patients initially but resolves over time, indicating a manageable side effect [35] Q&A Session Summary Question: Can you recap the interactions with the FDA regarding the accelerated approval path for bitopertin? - The company had a series of meetings with the FDA, which were responsive to the rationale for using PP9 as a surrogate endpoint, leading to the decision to submit an NDA this fall [11][12] Question: What are the key differences between the Apollo and Aurora trials? - Apollo will focus on measuring the time patients can spend in light as a co-primary endpoint, while Aurora primarily assessed the reduction in PP9 levels [18][20] Question: How does the company plan to address the patient population for bitopertin? - The company segments the patient population based on engagement with healthcare providers, identifying about 6,000 engaged patients who regularly seek care [27][28] Question: What is the expected market opportunity for the anemia treatment in myelofibrosis? - The company estimates a market of about 22,000 patients for anemia in myelofibrosis, with potential pricing similar to other anemia treatments in related indications [45] Question: How does the company view its capital position and runway? - The company is well-funded and expects its current capital to support its operations and trials through 2028, aiming to become self-sustaining through revenues from bitopertin [62]

Disc Medicine (IRON) FY 2025 Conference September 04, 2025 04:30 PM ET Speaker0All right. I'm Chad Gufalchian, biotech analyst here at Wells Fargo. We have Disc Medicine for a fireside chat at our halfway conference. John, I appreciate you guys making the time.Speaker1It's great to be here. Thank you.Speaker0Again, usually I will just give a couple of minutes for the overall winter remarks, where you guys are at, next catalyst, and then we'll go into a more detailed discussion across the pipeline if that wo ...

Core Viewpoint - Disc Medicine, Inc. received positive feedback from the FDA regarding its planned New Drug Application (NDA) submission for bitopertin, aimed at treating Erythropoietic Protoporphyria (EPP) [1][2] Group 1: NDA Submission Details - The pre-NDA meeting confirmed alignment with the FDA on the timing and content of the NDA submission, which is scheduled for October 2025 [2] - The NDA will be submitted under the FDA's accelerated approval pathway based on existing data [2] Group 2: About Bitopertin - Bitopertin is an investigational, orally administered inhibitor of glycine transporter 1 (GlyT1), designed to modulate heme biosynthesis [3] - It has potential as a first disease-modifying therapy for various hematologic diseases, including EPP [3] - Bitopertin has undergone multiple clinical trials, including the Phase 2 BEACON trial and the Phase 2 AURORA trial [3] Group 3: About Erythropoietic Protoporphyria (EPP) - EPP is a rare and potentially life-threatening disease caused by mutations affecting heme biosynthesis, leading to the accumulation of protoporphyrin IX (PPIX) [5] - Symptoms include severe pain and complications such as gallstones and liver damage, with current treatment options being limited [5] - There is currently only one FDA-approved therapy for EPP, Scenesse® (afamelanotide) [5] Group 4: About Disc Medicine - Disc Medicine is focused on discovering and developing novel treatments for serious hematologic diseases, targeting heme biosynthesis and iron homeostasis [6] - The company aims to build a portfolio of innovative therapeutic candidates to address a wide spectrum of hematologic diseases [6]

About Disc Medicine WATERTOWN, Mass., July 14, 2025 (GLOBE NEWSWIRE) -- Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, today announced the appointment of Nadim Ahmed to its Board of Directors. Mr. Ahmed is currently the President and Chief Executive Officer of Cullinan Therapeutics. "I am excited to welcome Nadim to Disc's Board of Director ...

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]

Core Insights - Disc Medicine, Inc. is set to present data from its hematology portfolio at the European Hematology Association (EHA) 2025 Congress in Milan from June 12-15, 2025, highlighting progress in its clinical programs [1][2] Company Developments - The company will showcase data from the HELIOS long-term extension trial, supporting bitopertin as a potential first disease-modifying treatment for erythropoietic protoporphyria (EPP) [2] - Additional data will be presented from the Phase 1b study of DISC-0974 in patients with myelofibrosis (MF) anemia, along with new findings from DISC-3405 in healthy volunteers, which supports its progression into a Phase 2 study in polycythemia vera [2][5] Presentation Details - The HELIOS study results will be presented on June 14, 2025, by Melanie Chin, while multiple presentations on DISC-0974 and DISC-3405 will occur on June 13 and 14, 2025, featuring various authors [4][6] - Management will host a corporate update call on June 16, 2025, to review the presented data [5]

Core Insights - Disc Medicine, Inc. reported strong operational progress in Q1 2025, with a focus on advancing its lead program, bitopertin, for erythropoietic protoporphyria (EPP) [2][5] - The company has a solid financial foundation, with cash reserves expected to fund operations into 2028, following a public offering that raised $259 million [3][9] Financial Performance - Cash, cash equivalents, and marketable securities totaled $694.7 million as of March 31, 2025, compared to $489.9 million at the end of 2024 [20] - Research and Development (R&D) expenses increased to $27.8 million in Q1 2025 from $23.7 million in Q1 2024, driven by advancements in clinical studies and increased headcount [14] - Selling, General and Administrative (SG&A) expenses rose to $12.2 million in Q1 2025 from $7.8 million in Q1 2024, reflecting the establishment of infrastructure for potential commercialization [14] - The net loss for Q1 2025 was $34.1 million, compared to a net loss of $26.9 million in Q1 2024 [14] Clinical Development Updates - The APOLLO trial for bitopertin has been initiated, aimed at supporting the conversion of a potential accelerated approval to full approval [6] - The company plans to submit a New Drug Application (NDA) for bitopertin in EPP in the second half of 2025 [5][6] - Data readouts for the DISC-0974 program, including results from the Phase 2 study for myelofibrosis (MF) anemia and the Phase 1b study for non-dialysis dependent chronic kidney disease (NDD-CKD) anemia, are expected in the second half of 2025 [5][7] - A Phase 2 study of DISC-3405 in polycythemia vera (PV) is set to be initiated in the first half of 2025 [8]