Disc Medicine(IRON) - 2025 FY - Earnings Call Transcript

Financial Data and Key Metrics Changes - The company is on an accelerated approval path for bitapertin, with strong scientific rationale supporting the use of protoporphyrin nine (PP9) as a surrogate endpoint for approval [4][5] - The data package provided to the FDA showed clear PP9 reduction and outperformance of the placebo group across multiple endpoints, which was persuasive in obtaining approval [5] Business Line Data and Key Metrics Changes - The target population for bitapertin is estimated to be around 20,000 individuals in the US with the genotype for EPP, with approximately 14,000 patients having received therapy under the EPP code [14][15] - The company aims to launch bitapertin targeting the 6,000 more recently treated patients while also reaching out to the full 14,000 patient population through advocacy campaigns [15] Market Data and Key Metrics Changes - The company acknowledges that many patients do not utilize the existing therapy, Senesse, due to access issues and the requirement for surgical procedures [7][8] - The competitive landscape includes the need for an oral therapy like bitapertin, which addresses the unmet needs of EPP patients [8] Company Strategy and Development Direction - The company is focusing on the EPP regulatory process and launch preparation, with plans to file an NDA in the second half of the year [34] - The company is also developing DISC 974, a monoclonal antibody aimed at treating anemia in myelofibrosis, with the goal of becoming the drug of choice for anemic patients [35][37] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the likelihood of success in the upcoming Apollo confirmatory trial, emphasizing the importance of both PP9 reduction and time in light as key endpoints [24] - The company is navigating the challenges of an accelerated launch timeline, having already built a commercial infrastructure and engaged with key opinion leaders [32][34] Other Important Information - The company is also exploring treatment options for anemia in chronic kidney disease (CKD) patients, with early data showing promising responses [45][47] - Management highlighted the importance of addressing anemia in myelofibrosis, noting that nearly 90% of patients are anemic and that there is currently no approved therapy for this condition [37][38] Q&A Session Summary Question: How did the FDA respond to the accelerated approval pathway for bitapertin? - The FDA required only one meeting to agree that PP9 could serve as a surrogate endpoint for accelerated approval, indicating a strong scientific rationale [5] Question: What is the estimated patient population for EPP? - The estimated patient population is around 20,000 individuals in the US, with 14,000 having received therapy under the EPP code [14][15] Question: How does the company plan to address the anemia in myelofibrosis? - The company aims to provide a drug that can be used from day one of diagnosis to manage anemia, which is a significant issue for myelofibrosis patients [37][38]