Summary of Disc Medicine Conference Call Company Overview - **Company**: Disc Medicine - **Focus**: Hematology, specifically targeting pathways of red blood cell biology and iron metabolism - **Lead Programs**: Bidipertin for erythropoietin protoporphyria and DISCO-nine 74 for anemia of myelofibrosis [3][6][8] Industry Context - **Disease Focus**: Myelofibrosis, a type of blood cancer characterized by anemia, splenomegaly, and constitutional symptoms [16][22] - **Current Treatment Landscape**: - Four JAK inhibitors approved for myelofibrosis, primarily targeting spleen size reduction and symptom improvement [46][49] - No approved therapies specifically for anemia of myelofibrosis, leading to significant unmet medical needs [19][45] Key Points from the Call Myelofibrosis and Anemia - **Prevalence**: Approximately 25,000 patients in the U.S. diagnosed with myelofibrosis, with a five-year survival rate around 50% [26][27] - **Anemia Statistics**: - 40% of patients present with significant anemia at diagnosis, increasing to 60% within one year [36] - Nearly all patients will develop anemia over the disease course, with transfusion dependency rising from 25% at diagnosis to 45% within a year [36][37] - **Prognostic Importance**: Anemia is a significant prognostic factor, with lower hemoglobin levels correlating with worse survival outcomes [30][38] Current Treatment Limitations - **JAK Inhibitors**: While effective for managing symptoms, they often exacerbate anemia, leading to treatment discontinuation in many patients [60][61] - **Existing Treatments for Anemia**: Use of erythropoietin, steroids, and androgens, but these are not specifically approved for myelofibrosis-related anemia [49][50] Emerging Therapies - **DISCO-nine 74**: - A monoclonal antibody targeting hepcidin suppression to mobilize iron for red blood cell production [8][10] - Phase 1b data shows promise in treating anemia of myelofibrosis [14][19] - **Luspatercept and Elitracept**: - Both are in development as potential treatments for anemia, with luspatercept already approved for MDS [79][80] - Phase 3 trials for luspatercept are ongoing, focusing on patients requiring transfusions while on stable doses of JAK inhibitors [80] Market Opportunity - **Anemia of Myelofibrosis**: Significant market opportunity exists for effective treatments targeting anemia, given the high prevalence and unmet needs [19][45] - **Potential Impact of New Therapies**: New treatments could improve patient quality of life, reduce healthcare costs associated with transfusions, and address the underlying pathophysiology of anemia in myelofibrosis [40][41][44] Conclusion - The call emphasized the critical need for new therapies specifically targeting anemia in myelofibrosis, highlighting the potential of DISCO-nine 74 and other emerging treatments to fill this gap in the current treatment landscape [19][45][76]

About DISC-0974 DISC-0974 is an investigational monoclonal antibody (mAb) targeting a BMP-signaling co-receptor called hemojuvelin (HJV) and is designed to suppress hepcidin production and increase serum iron levels in patients suffering from anemia of inflammation. DISC-0974 was in-licensed by Disc from AbbVie in 2019. Anemia of inflammation arises from abnormally elevated hepcidin and is the second most common form of anemia, affecting millions of patients in the US across numerous diseases, such as chron ...