Disc Medicine (IRON) FY Conference Transcript

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]