J.P. Morgan 44th Annual Healthcare Conference Corporate Overview January 2026 Disclaimer and FLS This presentation contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, express or implied statements regarding Disc's expectations with respect to its preclinical studies, clinical trials and research and development programs, in particular with respect to bitopertin, DISC-0974 and DISC-3405, and any developments or resul ...

Core Insights - The development of the biopharmaceutical industry in China and the U.S. appears to have a "time difference," with China experiencing a surge in activity while the U.S. market faces macroeconomic challenges [1][74]. Group 1: Market Trends - In the first three quarters of this year, particularly in the first half, the Chinese biopharmaceutical sector rapidly restored valuations due to a surge in business development (BD) activities, leading to a wave of IPOs in Hong Kong [2][74]. - The Chinese biopharmaceutical sector has seen a year-to-date increase of over 90%, with significant IPOs, including the successful listing of Heng Rui Medicine on the Hong Kong Stock Exchange [33][74]. - In contrast, the U.S. biopharmaceutical sector faced a downturn due to macroeconomic fluctuations, with a rebound occurring only in the fourth quarter as macro risks dissipated and strong earnings from pharmaceutical companies emerged [2][74]. Group 2: Key Issues in the Industry - The core issues affecting the global biopharmaceutical industry include the rise of China as a key player, the obesity epidemic, advancements in AI, the clustering of drug targets, and the emergence of new therapies [36][41][46]. - China has become a significant contributor to global clinical trials, with the proportion of trials initiated in China rising from 5% a decade ago to 30% today, matching the U.S. [37][74]. - The cost advantages in talent acquisition in China are notable, with CEO salaries in Chinese biopharmaceutical companies being significantly lower than their U.S. counterparts, leading to substantial cost savings for companies [39][74]. Group 3: Regulatory and Manufacturing Challenges - The U.S. faces challenges in drug pricing and manufacturing, with a historical trade deficit in pharmaceuticals reaching $140 billion this year and a commitment from the industry to invest $360 billion in domestic manufacturing [4][77]. - The FDA has experienced significant staff turnover, leading to instability and uncertainty in the regulatory environment, although recent statements from the new FDA director indicate a commitment to maintaining high standards for efficacy and safety [4][77]. - Concerns about the NIH budget cuts could adversely affect long-term industry development, particularly in terms of pipeline, talent, and foundational scientific progress [5][78]. Group 4: Financial Performance and Investment Trends - The biopharmaceutical sector's earnings remain robust, with a potential expansion in price-to-earnings ratios as the sector is currently at a 30-year low in valuation multiples [9][82]. - The industry has seen a significant milestone with 3.6 trillion defined daily doses (DDD) administered globally in 2024, indicating unprecedented coverage [12][74]. - The trend of increasing collaboration and partnerships in the industry is evident, with 63% of revenue coming from externally sourced assets, highlighting the importance of business development [16][58].

Summary of DISC Medicine Management Conference Call Company Overview - **Company**: DISC Medicine (NasdaqGM:IRON) - **Focus**: Development of therapies for anemia and related conditions, particularly in myelofibrosis (MF) and other hematological disorders Key Industry and Company Updates Bitopertin - **NDA Status**: The New Drug Application (NDA) for bitopertin has been accepted for review by the FDA, with a potential approval decision expected by the end of January 2026 [6][12][73] - **Commercialization Readiness**: The company is accelerating its commercialization efforts, with plans to hire 24 sales representatives across the U.S. [7][158] - **Regulatory Engagement**: The FDA is providing increased engagement and communication during the review process, which is part of the Commissioner's National Priority Voucher Program [11][73] DISC-0974 - **Rally MF Trial**: Initial data from the phase 2 Rally MF study shows significant decreases in hepcidin, leading to improved iron mobilization and clinically meaningful benefits in anemia measures such as hemoglobin levels and fatigue [8][22][27] - **Response Rates**: The overall response rate in the non-transfusion dependent (NTD) group is 63%, while the transfusion dependent low (TDLO) group shows a 50% major response rate [22][23] - **Market Opportunity**: Anemia of MF affects approximately 22,000 patients in the U.S., presenting a significant market opportunity for DISC-0974, which has shown high response rates and durability [35][37] DISC-3405 - **Mechanism of Action**: DISC-3405 aims to increase endogenous hepcidin production to limit iron availability, targeting conditions like polycythemia vera and sickle cell disease [43][45] - **Trial Updates**: The phase 2 trial for polycythemia vera has been expanded due to rapid enrollment, and initial data is expected in 2026 [9][48] Core Insights and Arguments - **Efficacy Across Background Therapies**: DISC-0974 shows consistent efficacy regardless of concomitant JAK inhibitors, indicating its potential as a versatile treatment option [26][70] - **Safety Profile**: The safety profile of DISC-0974 is consistent with background adverse events typical for the MF population, with no treatment-related serious adverse events reported [27] - **Future Development Plans**: The company plans to initiate a phase 2 study in anemia of inflammatory bowel disease (IBD) and is exploring the potential of a long-acting version of DISC-0974 [41][90] Additional Important Points - **Patient Experience**: Anecdotal reports suggest patients are experiencing significant improvements in quality of life and reduced fatigue while on DISC-0974 [118] - **Regulatory Landscape**: The acceptance of bitopertin's NDA under the accelerated approval pathway is a significant milestone, potentially paving the way for future therapies [73] - **Market Dynamics**: The company is positioning itself to address a serious unmet need in the anemia market, particularly for MF patients who currently have limited treatment options [40][41] This summary encapsulates the critical updates and insights from the DISC Medicine management conference call, highlighting the company's strategic direction and the potential impact of its therapies in the hematology space.

Company Overview - The company, Disc, focuses on controlling red blood cell biology by managing the metabolism of iron and heme, which are essential for red blood cell formation [2] - The lead program, Bitopertin, is in-licensed from Roche and aims to control heme biosynthesis in red blood cells [2] Key Programs - Bitopertin is being studied for a rare disease called erythropoietic protoporphyria (EPP), with approximately 14,000 diagnosed patients in the U.S. [2] - The company has completed a Phase II program for Bitopertin and is currently in an accelerated approval process with the FDA [2] - The approval process was further expedited by receiving a Commissioner's National Priority Voucher [2]

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]