Disc Medicine(IRON) - 2025 Q3 - Quarterly Results

Drug Development and Regulatory Approvals - Bitopertin has been selected for the FDA's Commissioner's National Priority Voucher (CNPV) Pilot Program, which offers accelerated reviews for drug applications, with an anticipated review period of 1-2 months[7]. - The NDA for Bitopertin was submitted on September 29, 2025, with FDA acceptance expected on or before November 28, 2025[7]. - The company is advancing commercial readiness activities to support the potential launch of Bitopertin on an accelerated timeline, if approved[7]. - The ongoing APOLLO confirmatory trial aims to support potential US approval and launch of Bitopertin in late 2025 or early 2026[57]. - Positive data across two Phase 2 studies support the durability of efficacy, with potential approval anticipated late 2025 to early 2026[175]. - NDA submitted on September 29, 2025, with a designed review time of 1-2 months[175]. - Initial data from the RALLY-MF Phase 2 study in MF anemia is expected soon, alongside the initiation of a Phase 2 study in IBD anemia anticipated in Q1 2026[175]. Clinical Trials and Study Results - The APOLLO study for EPP is currently underway, with a confirmatory trial required for full approval in the US[44]. - The anticipated initiation of the Phase 2 study for Anemia of Inflammatory Bowel Disease (IBD) is expected in Q1 2026[20]. - The Phase 2 trial for Bitopertin in Diamond Blackfan Anemia is currently underway, sponsored by NIH[61]. - The study enrolled a total of 35 participants, with 32 (91%) completing the study[95]. - 54% of participants receiving concomitant JAK inhibitor therapy achieved a major hematologic response[105]. - A significant 66% of patients experienced an increase in hemoglobin of at least 1.5 g/dL during the treatment period[101]. - The overall response rate for any hemoglobin increase of ≥1.5 g/dL was 68%[101]. - The study demonstrated a 43% reduction in erythropoietin levels and a 29% reduction in ferritin levels from baseline among major responders[108]. - Safety profile indicated that 94.1% of participants experienced any treatment-emergent adverse events (TEAEs), with most being Grade 1 or 2[112]. - The ongoing RALLY-MF Phase 2 study is assessing options for the NDD-CKD anemia program[175]. - A Phase 1b study for Sickle Cell Disease is expected to be initiated by the end of the year[172]. - The study design includes dose escalation and safety follow-up for Sickle Cell Disease participants[173]. Patient Populations and Disease Impact - The Erythropoietic Protoporphyria (EPP) patient population is estimated at approximately 20,000 in the US, with around 6,000 diagnosed patients actively seeking treatment[32]. - Real-world data indicates that 56% of EPP patients experience anxiety and/or depression, highlighting the significant impact of the disease on quality of life[35]. - Anemia remains a high unmet need within the myelofibrosis patient population, with approximately 22,000 addressable patients in the US[120]. - Approximately 5 to 6 million anemic non-dialysis dependent chronic kidney disease (NDD-CKD) patients in the US, with hepcidin levels ~20 times higher than healthy subjects[122]. - The estimated number of myelofibrosis patients in the US is 25,000, with around 87% experiencing anemia[94]. Pipeline and Future Studies - The pipeline includes multiple programs targeting hematologic diseases, with significant potential for addressing severe and moderate prevalence conditions[18]. - The company plans to initiate a Phase 2 study in IBD patients with anemia in Q1 2026[90]. - The company plans to initiate a Phase 2 study in patients with inflammatory bowel disease (IBD) and anemia in 2026[134]. - Phase 1 studies for DISC-3405 in healthy volunteers began in October 2023, assessing safety, pharmacokinetics, and hematologic parameters[154]. - The company is exploring other indications for DISC-3405, including hereditary hemochromatosis and beta-thalassemia[154]. Treatment Efficacy and Safety - Bitopertin 60 mg demonstrated a significant 40% reduction in PPIX levels compared to baseline after 6 months of treatment[48]. - Patients experienced a 75% reduction in the rate of phototoxic reactions versus placebo, with 86% reporting that their condition was 'much better'[48]. - The average cumulative total time in sunlight without pain increased significantly, with patients in the highest tertile experiencing 262.1 hours[52]. - Nearly all participants in the HELIOS study reported substantial improvements in quality of life measures through week 24[56]. - DISC-0974, an anti-HJV mAb, is designed to enhance iron availability and address a wide range of hematologic disorders[75]. - Hepcidin levels in myelofibrosis patients are approximately 12 times higher than controls, correlating with anemia severity[93]. - DISC-0974 demonstrated a 1.7 g/dL increase in hemoglobin levels compared to the vehicle control in a rat model of CKD[127]. - DISC-0974 resulted in a substantial, durable, dose-dependent reduction in hepcidin and sustained increase in transferrin saturation (TSAT) from baseline[129]. - DISC-3405 achieved ~70% suppression of serum iron levels lasting for 3 weeks in non-human primates[145]. - DISC-3405 produced dose-related increases in serum hepcidin, with corresponding reductions in serum iron ranging from 50-80% from baseline[161]. - The study showed consistent decreases in hepcidin and increases in serum iron across patients[99]. - DISC-3405 demonstrated meaningful reductions in hematologic parameters, including reticulocyte hemoglobin and hematocrit, in healthy volunteers[162]. - DISC-3405 resulted in an average 94% reduction in iron absorption at Day 2 and 68% at Day 15, confirming its mechanism to block dietary iron absorption[171].