Question-and-Answer SessionAwesome. John I think we have a lot going on for the Disc, which is all towards the very good direction. Maybe you give us 1, 2 minutes overview of the company? what are the key program you are focusing? And then what's the underlying philosophy for the company, and then we can go into each individual programs.John QuiselCEO, President & Director Yes, that's great. Thank you. Yes, it's been a great year Disc. So we're a company focused on controlling red blood cell biology by cont ...

Company Overview - Disc Medicine focuses on developing treatments for serious and debilitating hematologic diseases, with a name that reflects the shape of red blood cells [2] - The company's portfolio is constructed by targeting pathways fundamental to red blood cell biology, including programs that affect heme synthesis and regulate iron metabolism through the hormone Hepcidin [2] Lead Program - The lead program of the company is called Bitopertin, which is an oral treatment [3] Growth and Development - The company is currently experiencing significant growth, with ongoing developments in its portfolio that have been followed by Stifel for some time [1]

Group 1 - The article is authored by Terry Chrisomalis, who operates the Biotech Analysis Central pharmaceutical service on Seeking Alpha Marketplace, offering a two-week free trial for new subscribers [1] - Biotech Analysis Central provides a library of over 600 biotech investing articles, a model portfolio of more than 10 small and mid-cap stocks, and various analysis and news reports to assist healthcare investors [2] Group 2 - The service is priced at $49 per month, with a yearly plan available at a discounted rate of $399, reflecting a 33.50% discount [1] - The analyst has no current stock or derivative positions in any mentioned companies and does not plan to initiate any within the next 72 hours [3]

Core Insights - Successful investing requires understanding the fundamentals of owned stocks, as emphasized by Abigail Johnson, CEO of Fidelity Investments [1] Company Developments - Disc Medicine Inc. (IRON) has submitted a New Drug Application (NDA) for its lead drug candidate, Bitopertin, to the FDA for treating erythropoietic protoporphyria (EPP) [3][4] - The FDA is expected to decide on the NDA within 60 days of submission [3] - Disc Medicine received a Commissioner's National Priority Voucher (CNPV) for Bitopertin, expediting the review process to 1-2 months [5] - The stock price of IRON increased from $66.08 to a 52-week high of $95.95 [5] Clinical Trials and Data Readouts - Mind Medicine Inc. (MNMD) is preparing for pivotal data readouts in 2026 for its lead drug candidate, MM120 ODT, with results expected from multiple phase 3 trials [6][7] - MNMD's stock rose from $7.69 to a 52-week high of $13.86 [8] - Atai Life Sciences N.V. (ATAI) is progressing towards shareholder approval for its combination with Beckley Psytech Limited, aiming to lead in psychedelic-based mental health therapies [8] - ATAI's stock increased from $2.35 to a 52-week high of $6.55 [12] Ongoing Studies and Future Expectations - Assembly Biosciences Inc. (ASMB) is advancing its four key development programs, with ABI-5366 in a Phase 1b study for recurrent genital herpes [12][13] - ASMB's stock rose from $14.53 to a 52-week high of $30.20 [15] - Belite Bio Inc. (BLTE) is expected to report final topline data from the phase 3 DRAGON trial for its lead candidate, Tinlarebant, this quarter [16] - BLTE's stock increased from $69.60 to an all-time high of $91.92 [16] Earnings Expectations - Aveanna Healthcare Holdings Inc. (AVAH) is set to release its third-quarter results, with analysts expecting earnings of $0.08 per share on revenue of $577.01 million [17] - AVAH's stock rose from $5.35 to a 52-week high of $10.23 [18] Significant Trial Results - Nektar Therapeutics (NKTR) reported positive data from its phase 2b REZOLVE-AD trial for atopic dermatitis, with 52-week data expected in early 2026 [19][21] - NKTR's stock increased from $11.25 to a 52-week high of $63.92 [21]

Summary of Disc Medicine FY Conference Call Company Overview - **Company**: Disc Medicine (NasdaqGM: IRON) - **Focus**: Red blood cell biology through manipulation of heme and iron metabolism - **Pipeline**: Three molecules in clinical stages, with lead program Bitopertin for erythropoietic protoporphyria (EPP) [2][6] Key Points Product Development and Pipeline - **Bitopertin**: NDA submission expected in October 2025, with potential launch in 2026 [2][6] - **Other Programs**: Two additional programs targeting myeloproliferative disorders, currently in phase two with data expected by year-end [2][6] - **Patient Population**: Estimated 14,000 patients in the U.S. with EPP, with 6,000 actively seeking therapy [9][10] Competitive Landscape - **Collaboration**: One program in-licensed from a Chinese biotech, indicating a positive collaboration experience [3] - **Market Position**: No significant competition identified in the U.S. or China for the unique biology being targeted [3] Regulatory Environment - **FDA Interactions**: Positive feedback from the FDA regarding rare disease development, aiding in the approval process [5] Commercial Strategy - **Launch Preparations**: Chief Commercial Officer hired, focusing on understanding the patient population and engaging with physicians [6][7] - **Sales Strategy**: Targeting 6,000 engaged patients initially, with plans to expand outreach through advocacy groups and social media [10][11] Clinical Data and Efficacy - **Dosing Strategy**: 60 mg dose of Bitopertin shows a 50% reduction in toxic metabolite PP-9, correlating with clinical improvement [14] - **Quality of Life**: Sustained PP-9 reductions lead to improved patient-reported outcomes [16] Upcoming Trials - **Apollo Trial**: Confirmatory trial for Bitopertin with 150 patients expected to enroll by May 2026 [19] - **RALI-MF Trial**: Focused on anemia in myelofibrosis, with data readout anticipated in Q4 2025 [34] Financial Position - **Cash Reserves**: $650 million as of Q2 2025, projected to fund operations into 2028, including commercial launch expenses for Bitopertin [61] Future Opportunities - **Potential Indications**: Exploring applications for iron overload conditions and sickle cell disease [59] Additional Insights - **AI Utilization**: Exploring AI for streamlining clinical and regulatory documentation [4] - **Patient Impact**: EPP significantly affects quality of life, with severe light sensitivity leading to lifestyle limitations [26][27] This summary encapsulates the critical aspects of Disc Medicine's conference call, highlighting the company's strategic direction, product pipeline, and market positioning.

Bitopertin in EPP - NDA preparation is on track with planned submission in H2 2025 under accelerated approval path[11] - HELIOS extension study data showed sustained PPIX reductions and improvements in QoL with favorable long-term efficacy and safety[8, 21, 25] - HELIOS data suggests potential for bitopertin to improve liver function, with continued treatment of 60 mg reducing ALT[8, 24] - The prevalence of EPP patients in the US is estimated to be around 20,000, with ~6,000 diagnosed and ~14,000 misdiagnosed or not seeking treatment[31] DISC-0974 - Phase 1b results in MF anemia showed durable anemia response through the continuation phase among major responders[8, 57] - Phase 1b data showed sustained activity on key biomarkers, supporting target engagement and iron mobilization[8, 53] - In MF Phase 1b study, major responders treated with DISC-0974 experienced a 43% and 29% reduction from baseline at Day 113 in erythropoietin and ferritin, respectively[53] - RALLY-MF Phase 2 enrollment is ongoing, with the momelotinib exploratory cohort fully enrolled[8, 62] DISC-3405 - Phase 1 data from healthy volunteers showed deep, sustained reductions in serum iron and meaningful changes in hematologic parameters[9, 78, 80] - Iron pulse study in healthy volunteers showed a 94% reduction in iron absorption at Day 2 and 68% at Day 15, confirming MOA and potential in diseases of iron overload[9, 86] - A Phase 2 study in polycythemia vera has been initiated[9, 87]