Summary of Arrowhead Pharmaceuticals FY Conference Call (December 04, 2025) Company Overview - **Company**: Arrowhead Pharmaceuticals (NasdaqGS:ARWR) - **Key Event**: Discussion on the company's accomplishments and future plans during the FY Conference Call Key Accomplishments - **Drug Launch**: The launch of Redemplo (Plozasiran) is highlighted as the most significant achievement, marking the company's first independent drug launch after nearly two decades of development [3][4] - **Market Position**: Arrowhead is positioned to become a large-cap biotech company, with a pipeline of nearly 20 drugs and multiple candidates in phase three studies [3] Product Details - **Redemplo (Plozasiran)**: Approved for treating familial chylomicronemia syndrome (FCS) and reducing triglycerides in patients with genetic or clinical FCS. It is the only drug studied in both populations, expanding commercial opportunities [4] - **Ongoing Studies**: Phase three studies (SHASTA 3, SHASTA 4, SHASTA 5, and MUIR 3) are ongoing, with results expected in Q3 2026. An SNDA filing is anticipated by the end of 2026 for SHTG launch in 2027 [5][4] Competitive Landscape - **Market Dynamics**: The company acknowledges competition from Ionis Pharmaceuticals but emphasizes a collaborative approach to grow the market for triglyceride-lowering drugs, particularly in underserved areas [7][8] - **Market Potential**: There is a significant unmet need in the high triglycerides (TG) market, especially for patients at risk of acute pancreatitis [7] Clinical Study Insights - **SHASTA Studies**: SHASTA 3 and SHASTA 4 are placebo-controlled studies with 750 patients, focusing on triglyceride lowering and safety in high-risk populations [10][11] - **Patient Demographics**: Approximately 37% of patients had triglycerides over 880 mg/dL, indicating a severe risk for acute pancreatitis [11] Future Pipeline and Innovations - **Dual PCSK9, ApoC3 Candidate**: A new program aimed at addressing mixed hyperlipidemia is set to begin phase 1/2 studies in early 2026, with initial data expected in Q3 2026 [13][14] - **Inhibin E and ALK7**: Phase 1/2 studies are ongoing, focusing on weight loss and fat reduction mechanisms. The regulatory pathway remains unclear due to evolving FDA guidelines [20][25] Upcoming Milestones - **Key Data Releases**: - Initial obesity data for Inhibin E and ALK7 in January 2026 - Readout for SHASTA 3 and 4 studies in Q3 2026 - Dimer data and MAPT program updates for Alzheimer's expected around the same time [28][29] Conclusion - **Strategic Focus**: Arrowhead Pharmaceuticals is poised for significant growth with multiple product launches and a robust pipeline. The company aims to address unmet medical needs in cardiometabolic diseases and expand its market presence in the biotech sector [27][30]

Investment Rating - The report maintains an "Outperform" rating for the pharmaceutical sector [6]. Core Insights - The small nucleic acid drug industry is entering a significant commercialization phase in 2025, with major advancements in product launches and new platform validations expected [16]. - Alnylam's AMVUTTRA is projected to exceed $2 billion in sales, marking a significant milestone for small nucleic acid drugs [3][17]. - Ionis is anticipated to have multiple catalysts in 2026, with key products like Olezarsen showing promising results [25]. - Arrowhead has launched its first commercial product, Plozasiran, and is focusing on its innovative Trim platform for weight loss and CNS projects [35]. Summary by Sections 1. Weekly Performance of Recommended Pharmaceutical Combinations - The recommended combinations outperformed the pharmaceutical index by 2.4 percentage points but underperformed the broader market by 0.6 percentage points [12]. 2. Small Nucleic Acid Industry Update - The small nucleic acid sector is witnessing rapid commercialization and platform breakthroughs, with significant investment opportunities emerging [16]. - Alnylam's AMVUTTRA sales are expected to reach $2.475 to $2.525 billion for the year, reflecting strong market demand [23]. - Ionis's Olezarsen has shown a 72% reduction in triglycerides in patients, indicating a strong commercial outlook [17][25]. - Arrowhead's Plozasiran is now approved for treating familial chylomicronemia syndrome, marking a significant achievement for the company [17]. 3. Weekly Market Review of the Pharmaceutical Sector - The CITIC Pharmaceutical Index fell by 6.8% during the week of November 17-21, 2025, underperforming the CSI 300 Index by 3 percentage points [47]. - The pharmaceutical sector has seen a year-to-date increase of 14.7%, outperforming the CSI 300 Index by 1.5 percentage points [47]. - The top-performing stocks in the sector included Hainan Haiyao (+23.8%) and Changyao Holdings (+19.7%) [59]. 4. Market Trends and Future Outlook - The report suggests a continued focus on innovative drugs, CXO, and the pharmaceutical supply chain, especially with upcoming ASH conference data releases [4]. - The overall valuation of the pharmaceutical sector is currently at 28.5, indicating a relative premium over the broader market [54].

Summary of Ionis Pharmaceuticals Conference Call Company Overview - **Company**: Ionis Pharmaceuticals - **Industry**: Biotechnology, specifically focusing on genetic medicines targeting RNA for therapeutics - **Recent Achievements**: Launched two FDA-approved medicines independently in the U.S. in 2025: Tryngolza for Familial Chylomicronemia Syndrome (FCS) and Donidalorsen for hereditary angioedema [2][3] Core Points and Arguments - **Successful Product Launches**: - Tryngolza, the first FDA-approved medicine for FCS, launched successfully in 2025 [2] - Donidalorsen launched as a prophylactic treatment for hereditary angioedema [2] - **Pipeline Progress**: - Announced positive phase three data for severe hypertriglyceridemia and a neurology drug for Alexander disease [3][4] - Anticipating five phase three readouts and two to three FDA approvals in the upcoming year [4] - **Market Opportunity**: - Severe hypertriglyceridemia affects millions in the U.S., with a focus on patients at high risk for acute pancreatitis [7][9] - Plans to target both severely at-risk patients and those with mildly elevated triglycerides [10][11] - **Combination Therapy Potential**: - Exploring combination therapies for managing triglycerides and other cardiovascular risk factors [12][13] - Collaboration with AstraZeneca to combine treatments for TTR cardiomyopathy [14][17] Additional Important Insights - **Self-Administration Preference**: - Patients prefer self-administered treatments, which could enhance market penetration, especially in community settings [20][21] - **Regulatory and Market Dynamics**: - Anticipated inclusion in treatment guidelines for severe hypertriglyceridemia, with a significant patient population of over 3 million in the U.S. [40] - **Spinraza Update**: - Ionis is developing a next-generation Spinraza with once-per-year dosing, expected to re-emerge as a leading treatment for spinal muscular atrophy (SMA) [32][33] - Economic terms for the next-gen product are more favorable, with royalties in the mid-20% range compared to mid-teens for the current compound [34] Conclusion - Ionis Pharmaceuticals is positioned for significant growth with its innovative therapies targeting severe hypertriglyceridemia and SMA. The company is leveraging its unique RNA-targeting platform to address unmet medical needs and expand its market presence through strategic partnerships and a focus on patient convenience.

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Q3:2025 Business Update and Financial Results October 29, 2025 Nasdaq: IONS 1 On Today's Earnings Call Eugene Schneider, M.D. Chief Clinical Development Officer Eric Swayze, Ph.D. Executive Vice President, Research 2 Richard Geary, Ph.D. Chief Development Officer Kyle Jenne Chief Global Product Strategy Officer Beth Hougen Chief Financial Officer Forward-Looking Statements This presentation includes forward-looking statements regarding our business, financial guidance and the therapeutic and commercial pote ...

2 Ionis Leadership Here Today Innovation Day 2025: Accelerating Growth through Life-Changing Medicines OCTOBER 7, 2025 | Nasdaq: IONS Forward-Looking Statements This presentation includes forward-looking statements regarding our business, financial guidance and the therapeutic and commercial potential of our commercial medicines, additional medicines in development and technologies and our expectations regarding development and regulatory milestones. Any statement describing Ionis' goals, expectations, fina ...

Group 1 - Arrowhead Pharmaceuticals, Inc. (NASDAQ:ARWR) is considered one of the best stocks to buy for the next three months according to hedge funds, with a Buy rating and a price target of $80 set by H.C. Wainwright analyst Patrick Trucchio [1] - The recent deal with Novartis is highlighted as a strong vote of confidence, providing $200 million upfront and the potential for a $2 billion milestone, which validates Arrowhead's approach to treating CNS diseases and adds funding without share dilution [2] - Positive results from Ionis Pharmaceuticals' Olezarsen support Arrowhead's Plozasiran drug, both targeting severe hypertriglyceridemia, with Arrowhead's offering of quarterly dosing potentially attracting more patients and doctors [3]

Q2:2025 Business Update and Financial Results July 30, 2025 Nasdaq: IONS 1 On Today's Earnings Call Eugene Schneider, M.D. Chief Clinical Development Officer Eric Swayze, Ph.D. Executive Vice President, Research 2 Brett Monia, Ph.D. Chief Executive Officer Kyle Jenne Chief Global Product Strategy Officer In this presentation, unless the context requires otherwise, "Ionis," "Company," "we," "our," and "us" refers to Ionis Pharmaceuticals and its subsidiaries. Ionis Pharmaceuticals® is a registered trademark ...