Arrowhead Pharmaceuticals (NasdaqGS:ARWR) Q4 2025 Earnings Call November 25, 2025 04:30 PM ET Company ParticipantsVince Anzalone - VP of Investor RelationsAndy Davis - SVP and Head of Global Cardiometabolic FranchiseChris Anzalone - President and CEOBruce Given - Chief Medical OfficerAndrea Newkirk - Vice President of Biotechnology Equity ResearchDan Apel - CFOMike Ulz - Executive Director of Biotechnology Equity ResearchJames Hamilton - Chief Medical Officer and Head of R&DConference Call ParticipantsPrakh ...

Financial Data and Key Metrics Changes - The net loss for fiscal year 2025 was $2 million, a significant improvement from a net loss of approximately $599 million in fiscal year 2024, resulting in a loss of $0.01 per share compared to a loss of $5 per share [32] - Revenue for fiscal year 2025 totaled $829 million, driven primarily by licensing and collaboration agreements, with $697 million from Sarepta Therapeutics [32][33] - Total operating expenses for fiscal year 2025 were approximately $731 million, an increase from $605 million in fiscal 2024, primarily due to higher R&D and SG&A costs [33][34] - Cash and investments totaled $919 million as of September 30, 2025, up from $681 million a year earlier, reflecting strong cash flow from licensing agreements [35][36] Business Line Data and Key Metrics Changes - The FDA approved Redemplo, Arrowhead's first FDA-approved medicine, marking a major milestone as it transitions into the commercial stage [6][7] - Redemplo is indicated for reducing triglycerides in adults with familial chylomicronemia syndrome (FCS) and can be self-administered at home [19][20] - The One Redemplo pricing model is set at $60,000 annually, aimed at providing consistent pricing across indications [11][25] Market Data and Key Metrics Changes - There are an estimated 6,500 people in the U.S. living with genetic or clinical FCS, with a prescriber base comprising specialists such as lipidologists and endocrinologists [25] - The broader patient population at risk of acute pancreatitis includes approximately 750,000 patients with persistent chylomicronemia [10] Company Strategy and Development Direction - The company is focusing on the cardiometabolic space, with ongoing clinical trials for Zodasiran and ArrowDiamond PA, targeting conditions like homozygous familial hypercholesterolemia and atherosclerotic cardiovascular disease [12][13] - Arrowhead aims to have 20 individual drug candidates in clinical trials by the end of 2025, with a balanced pipeline of wholly owned and partnered programs [39] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the commercial launch of Redemplo and the encouraging feedback from the FCS community [39] - The company does not anticipate substantial financial impact from Redemplo sales in fiscal year 2026 but believes its cash runway is sufficient to support operations into fiscal year 2028 [38] Other Important Information - Arrowhead received a $200 million upfront payment from Novartis for a licensing agreement and is eligible for up to $2 billion in future milestone payments [36][37] - The company has made significant progress in business development, including a $200 million milestone payment from Sarepta for ARO-DM1 [17][38] Q&A Session Summary Question: What is the plan to show benefit in terms of acute pancreatitis for Plozasiran? - Management indicated that while shots at three and four were powered for triglyceride reduction, there is reasonable power to see a difference in acute pancreatitis, with a specific design for SHASTA-5 to demonstrate benefit [42][44][45] Question: Can you provide details on the upcoming data for obesity programs? - Management confirmed that initial data from the ARO-INHBE program will be shared in early January, with a focus on safety and biomarker data [48][50] Question: What are the estimates on acute pancreatitis events accrual based on patient baseline characteristics? - Management noted that it is challenging to predict due to the adoption of modified Atlanta criteria for adjudication, but they expect a sufficient number of events based on patient history [53][54] Question: Can you discuss the differentiation of ArrowMapT from J&J's failed anti-tau antibody? - Management highlighted that ArrowMapT targets intracellular tau, potentially reducing both intracellular and extracellular tau levels, unlike J&J's approach [57][59] Question: What is the visibility on launching a CBOT study? - Management stated that they expect to have a clearer idea by summer 2026 based on data from ongoing studies, with plans to move into pivotal studies if results are favorable [62][63]

Arrowhead Pharmaceuticals (NasdaqGS:ARWR) 2025 Conference November 19, 2025 06:00 AM ET Company ParticipantsChristopher Anzalone - CEOConference Call ParticipantsMaury Raycroft - AnalystMaury RaycroftHi, everyone. My name is Maury Raycroft. I'm one of the biotech analysts at Jefferies. I'd like to welcome Chris Anzalone, the CEO of Arrowhead. Thanks so much for joining us today, Chris, and congrats on the FCS approval yesterday.Christopher AnzaloneThanks very much. It's a pleasure to be here, as always, and ...

Arrowhead Pharmaceuticals Conference Call Summary Company Overview - **Company**: Arrowhead Pharmaceuticals - **Product**: Redemplo (FDA approved for reducing triglycerides in adults with Familial Chylomicronemia Syndrome, FCS) Key Points FDA Approval and Product Launch - Redemplo is the first RNAi-based medicine approved by the FDA for reducing triglycerides in adults with FCS [4][5] - The product demonstrated unprecedented median triglyceride reductions of 80% from baseline in clinical trials [4][20] - Redemplo is administered as a subcutaneous injection once every three months, making it convenient for patients [5][20] Clinical Data and Efficacy - The Palisade phase III study included both genetically confirmed and clinically defined FCS patients, showing similar responses [4][25] - The drug has a strong safety profile with no contraindications or warnings, and common adverse reactions include hyperglycemia, headache, and nausea [5][20][28] - The median triglyceride levels were reduced to below 500 mg/dL, a critical threshold for FCS patients [20][26] Market Opportunity and Pricing Strategy - Approximately 6,500 adults in the U.S. have either genetically confirmed or clinical FCS, representing a significant market opportunity [30] - The annual wholesale acquisition cost (WAC) for Redemplo is set at $60,000, reflecting its value in the high-risk severe hypertriglyceridemia (SHTG) population [12][37] - Arrowhead aims to establish a consistent pricing model across current and future indications, emphasizing sustainable innovation [12][37] Future Development and Pipeline - Arrowhead has ongoing phase III studies for SHTG patients, with results expected in Q3 2026, and plans to file for an SNDA in Q4 2026 [8][40] - The company is also developing Zodasiran for treating homozygous familial hypercholesterolemia (HoFH) and Arrow Dimer PA for mixed hyperlipidemia [15][41] - Arrowhead's goal is to create 2 to 4 new drug candidates annually, leveraging its TRIM platform to address various diseases [38][39] Healthcare Burden of FCS - FCS patients face significant healthcare challenges, including a high incidence of acute pancreatitis, which can exceed $60,000 in direct medical costs per event [9][10] - The condition often requires multiple specialist consultations before diagnosis, leading to delays in treatment [9][10] Commercial Strategy - Arrowhead's commercial strategy targets approximately 5,000 healthcare professionals specializing in lipid disorders, with a focus on personal engagement [30][31] - The "Redemplo Go Low" campaign aims to promote the importance of lowering triglyceride levels for FCS patients [31][32] Competitive Landscape - Arrowhead acknowledges competition from other companies but emphasizes the unique value proposition of Redemplo, particularly its efficacy and safety profile [55][56] - The company is preparing for a European launch and is open to partnerships for commercialization outside the U.S. [49][50] Conclusion - Arrowhead Pharmaceuticals is poised to make a significant impact in the treatment of FCS with Redemplo, backed by strong clinical data and a comprehensive commercial strategy aimed at addressing the needs of patients and healthcare providers [36][42]

Summary of Arrowhead Pharmaceuticals FY Conference Call Company Overview - **Company**: Arrowhead Pharmaceuticals (NasdaqGS:ARWR) - **Event**: FY Conference Call on September 10, 2025 - **Key Speakers**: Chris Anzalone (CEO), James Hamilton (CMO and Head of R&D) Core Points and Arguments Transition to Commercialization - Arrowhead Pharmaceuticals is transitioning into a commercial company with a PDUFA date for Plozasiran on November 18, 2025, indicating a significant milestone in their development pipeline [2][4] Plozasiran Overview - Plozasiran is designed to reduce ApoC3 expression, lowering triglycerides in patients with Familial Chylomicronemia Syndrome (FCS) [4] - Phase 3 study results showed an approximately 80% reduction in triglycerides from baseline and a significant decrease in the risk of acute pancreatitis [4][12] Market Opportunity in FCS - FCS is classified as an ultra-orphan disease with an estimated 1,000 genetically defined patients in the U.S. [6] - Arrowhead also targets clinically defined FCS patients, potentially expanding the market beyond the genetically defined group [7] Severe Hypertriglyceridemia (SHTG) Market - The SHTG market is significantly larger, targeting patients with triglyceride levels above 500 mg/dL [9] - Arrowhead is conducting several Phase 3 studies (Shasta-3, Shasta-4, Shasta-5, NEER-3) to evaluate Plozasiran's efficacy in this population [10][19] Competitive Landscape - Arrowhead acknowledges competition in the market but believes that having multiple companies addressing the same medical need is beneficial for patient education and treatment options [16][17] - Plozasiran is expected to show greater efficacy in triglyceride reduction compared to competitors' products [25] Zodasiran Development - Zodasiran is focused on the Homozygous Familial Hypercholesterolemia (HoFH) population, with a Phase 3 study (Yosemite) underway [28] - The drug has shown promising results in earlier studies, with expectations of similar LDL-C reductions as existing therapies [29] Obesity Programs - Arrowhead is developing two obesity treatments targeting the INHBE and ALK7 pathways, with data expected by the end of the year [32][33] - The company aims to demonstrate high-quality weight loss and muscle sparing in clinical trials [35] Bispecific PCSK9/ApoC3 Dimer - A new bispecific dimer targeting both PCSK9 and ApoC3 is set to enter clinical trials, aiming to lower both LDL cholesterol and triglycerides [41][42] CNS Platform - Arrowhead has a promising CNS platform with three near-term clinical programs targeting tau, Huntington's disease, and alpha-synuclein [44][45] - The company has partnered with Novartis for the alpha-synuclein program, while retaining control over the MAPT program [49] Financial Position and Future Outlook - Arrowhead's current cash position and partnerships are expected to sustain operations into 2028, allowing continued development of their pipeline [60] - Upcoming catalysts include the PDUFA update for Plozasiran, obesity data, and Phase 3 readouts for severe hypertriglyceridemia [63][64] Additional Important Points - The partnership with Sarepta Therapeutics has been financially beneficial, with nearly $1 billion received in the past year [55] - Arrowhead emphasizes the importance of maintaining a productive R&D organization while transitioning to commercialization [61] This summary encapsulates the key points discussed during the conference call, highlighting Arrowhead Pharmaceuticals' strategic direction, product pipeline, market opportunities, and financial outlook.

Investment Rating - The report maintains an "Outperform" rating for the pharmaceutical and biotechnology sector [5][41]. Core Views - The pharmaceutical sector has shown resilience, outperforming the overall market with a 1.40% increase, while the total A-share market declined by 1.17% [1][32]. - The ANGPTL3 target has gained significant interest from multinational pharmaceutical companies (MNCs) due to its unique mechanism of action, which is non-LDLR dependent, making it effective in patients with LDLR deficiencies [3][30]. - The report highlights the potential for ANGPTL3 therapies to significantly lower LDL-C levels, especially in high-risk populations such as HoFH patients, and suggests a diversified drug development landscape with various forms of ANGPTL3 inhibitors [3][16]. Market Performance - The overall A-share market saw a decline of 1.17%, with the Shanghai Composite Index down 0.81% and the ChiNext Index up 2.35%. The biotechnology sector's performance was notably strong, with chemical pharmaceuticals rising by 3.92% [1][32]. - The TTM price-to-earnings (P/E) ratio for the pharmaceutical and biotechnology sector stands at 40.75x, compared to the overall A-share market's 19.80x [37][41]. Key Companies and Investment Recommendations - **Mindray Medical (300760.SZ)**: Rated "Outperform" with a projected net profit of 116.7 billion CNY for 2024 [4]. - **WuXi AppTec (603259.SH)**: Rated "Outperform" with a projected net profit of 93.5 billion CNY for 2024 [4]. - **Aier Eye Hospital (300015.SZ)**: Rated "Outperform" with a projected net profit of 35.6 billion CNY for 2024 [4]. - **New Industries (300832.SZ)**: Rated "Outperform" with a projected net profit of 18.3 billion CNY for 2024 [4]. - **Huitai Medical (688617.SH)**: Rated "Outperform" with a projected net profit of 6.7 billion CNY for 2024 [4]. - **Kaili Medical (300633.SZ)**: Rated "Outperform" with a projected net profit of 1.4 billion CNY for 2024 [4]. - **Aohua Endoscopy (688212.SH)**: Rated "Outperform" with a projected net profit of 0.2 billion CNY for 2024 [4]. - **Edding Biological (300685.SZ)**: Rated "Outperform" with a projected net profit of 2.5 billion CNY for 2024 [4]. - **Aibo Medical (688050.SH)**: Rated "Outperform" with a projected net profit of 3.9 billion CNY for 2024 [4]. - **Kingdom Medical (603882.SH)**: Rated "Outperform" with a projected net profit of -3.8 billion CNY for 2024 [4]. ANGPTL3 Development Landscape - The report emphasizes the growing interest in ANGPTL3 as a therapeutic target, with MNCs like Novartis, Lilly, Amgen, and AstraZeneca actively pursuing drug development in this area [3][16]. - ANGPTL3 therapies are expected to provide significant benefits in managing lipid levels, particularly in patients with mixed dyslipidemia and high triglycerides [30][31].

Investment Rating - The report maintains an "Outperform" rating for the pharmaceutical and biotechnology sector [5][41]. Core Views - The pharmaceutical sector has shown resilience, outperforming the overall market with a 1.40% increase, while the total A-share market declined by 1.17% [1][32]. - ANGPTL3 has gained significant interest from multinational pharmaceutical companies (MNCs) due to its unique mechanism of action, which is non-LDLR dependent, making it effective in patients with LDLR deficiencies [3][30]. - The report highlights the diverse drug forms targeting ANGPTL3, with MNCs like Novartis, Lilly, Amgen, and AstraZeneca actively investing in this area, indicating a potential for multi-target and multi-drug combinations in the future [3][16]. Summary by Sections Market Performance - The overall A-share market fell by 1.17%, with the Shanghai and Shenzhen 300 index down by 0.81%. The biotechnology sector, however, rose by 1.40%, indicating strong performance relative to the broader market [1][32]. - Specific segments within the pharmaceutical sector showed varied performance, with chemical pharmaceuticals up by 3.92% and medical services up by 1.69%, while medical devices and traditional Chinese medicine saw declines [1][32]. ANGPTL3 Developments - A collaboration between Argo Biopharma and Novartis was announced, focusing on RNA drugs for cardiovascular diseases, with a total potential payment of $5.2 billion, including a $160 million upfront payment [2][11]. - ANGPTL3's mechanism allows for significant reductions in LDL-C levels in patients with HoFH, outperforming traditional treatments like PCSK9 inhibitors [3][31]. Company Earnings Forecasts and Ratings - Key companies in the sector, such as Mindray Medical, WuXi AppTec, and Aier Eye Hospital, have been rated as "Outperform" with projected net profits for 2024 ranging from 1.4 billion to 116.7 billion yuan [4][41]. - The report provides detailed earnings forecasts for various companies, indicating a positive outlook for the sector as a whole [4][41]. Valuation Metrics - The TTM price-to-earnings ratio for the pharmaceutical and biotechnology sector stands at 40.75x, significantly higher than the overall A-share market's 19.80x [37][38]. - Sub-sectors such as chemical pharmaceuticals and biological products have even higher valuations, indicating strong investor interest and growth potential [37][38]. Recommended Stocks - The report recommends several companies, including Mindray Medical for its strong market position and international expansion, WuXi AppTec for its comprehensive drug development services, and Aier Eye Hospital for its leading position in the eye care sector [41][42].

Core Viewpoint - Arrowhead Pharmaceuticals reported significant misses in both GAAP revenue and earnings for Q3 FY2025, highlighting ongoing financial volatility despite progress in its clinical pipeline [1][5][6] Financial Performance - Q3 FY2025 GAAP revenue was $27.8 million, missing analyst estimates of $46.8 million by nearly 41% [5] - GAAP earnings per share for Q3 FY2025 were $(1.26), falling short of the $(0.95) forecast by $0.31 [1][5] - Operating loss for Q3 FY2025 was $(165.6 million), an improvement of 6% compared to $(176.1 million) in Q3 FY2024 [2][6] - Research and development expenses increased by 6.6% year-over-year to $162.4 million [2][6] - Total cash resources rose to $900.4 million, a 32.1% increase from $681.0 million in Q3 FY2024 [2][7] Company Overview and Strategy - Arrowhead Pharmaceuticals focuses on RNA interference (RNAi) therapeutics, targeting diseases with significant unmet needs using its TRiMTM platform [3][4] - The company is advancing its drug pipeline with an emphasis on late-stage development and regulatory approvals, while building strategic partnerships with larger pharmaceutical companies [4] Product Pipeline Progress - Plozasiran, Arrowhead's leading RNAi candidate, received NDA acceptance from the FDA for familial chylomicronemia syndrome, with a PDUFA target date of November 18, 2025 [8][11] - Phase 3 studies for severe hypertriglyceridemia are ongoing, with data expected in mid-2026 [8][11] - Zodasiran, targeting rare cholesterol disorders, has commenced its Phase 3 trial [12] - The obesity portfolio includes ARO-INHBE and ARO-ALK7, which are in Phase 1/2 studies [13] Strategic Partnerships and Milestones - Visirna, a subsidiary, entered a deal with Sanofi for rights to four cardiometabolic programs, resulting in immediate payments of $130 million and potential milestones of up to $265 million [9] - The company anticipates additional milestone payments from Sarepta Therapeutics, totaling up to $200 million by the end of 2025 [9] Commercial Readiness - Arrowhead is preparing for the potential launch of plozasiran by expanding its U.S. sales force and engaging with payers [10] - The company is focused on rare disease awareness and patient identification, crucial for the target population of plozasiran [10] Outlook and Guidance - Management indicated that Arrowhead is funded through 2028, focusing on regulatory approval for plozasiran and commercialization efforts [15] - Key near-term catalysts include pivotal SHTG data readout in mid-2026 and potential partnership milestones within the next twelve months [15]

Summary of Arrowhead Pharmaceuticals (ARWR) Conference Call Company Overview - **Company**: Arrowhead Pharmaceuticals (ARWR) - **Event**: BofA Annual Healthcare Conference - **Date**: May 14, 2025 Key Points Strategic Developments - The recent deal with Sarepta has significantly changed Arrowhead's strategic outlook, allowing the company to focus on core assets and reducing dependence on capital markets for a period of time [2][4][5] - The core assets from the Sarepta deal include two muscle assets, ARO DM1 and ARO DUX4, which Arrowhead is excited about due to Sarepta's expertise in drug approval [3][4] Pipeline and Research Focus - Arrowhead has a strong presence in cardiometabolic diseases, with ongoing projects targeting APOC3 and obesity [6][7] - The company is expanding its capabilities to address various tissue types, including adipocytes, and plans to introduce new metabolic targets in the clinic next year [8][9][10] FCS and SHTG Opportunities - The company views the FCS (Familial Chylomicronemia Syndrome) market as a gateway to the larger SHTG (Severe Hypertriglyceridemia) opportunity, with an estimated 3-4 million patients in the U.S. having triglyceride levels above 500 [12][14] - Arrowhead's Phase 3 data showed an 80% reduction in triglycerides from baseline, which is significantly higher than competitors [13][19] Competitive Landscape - Arrowhead believes it has a competitive edge over Ionis in the FCS market due to its superior triglyceride reduction capabilities and less frequent dosing [20][19] - The company is preparing for potential payer restrictions based on patient profiles but expects to address a broad patient population [23][24] Clinical Trials and Timelines - Arrowhead is currently enrolling patients for multiple Phase 3 studies, with expectations to launch in the SHTG market by 2027 [33][35] - The company has sufficient cash to sustain operations until 2028, allowing for multiple product launches around that time [35] Obesity Research - Arrowhead is exploring the ALK7 and Inhibin E pathways for obesity treatment, with promising animal data indicating high-quality weight loss without caloric restriction [42][43] - The company is considering combination therapies with existing GLP-1 drugs to enhance weight loss outcomes [51][52] Neuromuscular Programs - Arrowhead is advancing its neuromuscular programs, particularly ARO DM1 and ARO DUX4, with data disclosures expected this year [59][61] - The company anticipates triggering $300 million in milestone payments based on dosing in the DM1 study [62] Safety and Efficacy Considerations - Arrowhead emphasizes the importance of safety in drug development, aiming to use lower doses compared to competitors while maintaining efficacy [63][64] - The potential for subcutaneous administration of their drugs could be a significant advancement in the field [65][66] Additional Insights - Arrowhead is optimistic about the educational aspect of the market, believing that both it and Ionis can benefit from increased awareness of triglyceride management [18] - The company is open to exploring various avenues for funding future trials, including business development and partnerships [38] This summary encapsulates the key discussions and insights from the Arrowhead Pharmaceuticals conference call, highlighting the company's strategic direction, pipeline developments, and competitive positioning in the biotech landscape.

Financial Data and Key Metrics Changes - The net income for Q2 2025 was $370.4 million, or $2.75 per share, compared to a net loss of $125.3 million, or $1.02 per share, for Q2 2024 [49] - Revenue for Q2 2025 was $542.7 million, with no revenue recorded in Q2 2024, primarily due to the license and collaboration agreement with Sarepta [49][50] - Total operating expenses for Q2 2025 were $161.5 million, up from $126.2 million in Q2 2024, driven by increased costs associated with the clinical pipeline [53] Business Line Data and Key Metrics Changes - The company is preparing for the launch of Flodasiran, targeting severe hypertriglyceridemia (SHTG) with a PDUFA date set for November 18, 2025 [10][55] - Flodasiran has shown a reduction in triglycerides of about 80% from baseline in clinical studies, with 75% of patients achieving levels below 880 mg/dL [11][41] - The company is also advancing its obesity candidates ARO INHBE and ARO ALK7, with initial data expected by the end of 2025 [16][45] Market Data and Key Metrics Changes - The company has engaged with payers representing a significant number of US covered lives, delivering content on the clinical value of Flodasiran [39] - The market research indicates strong interest in Flodasiran's potential to reduce triglycerides and acute pancreatitis risk [40] Company Strategy and Development Direction - Arrowhead aims to transition from a development stage to a commercial stage with the planned launch of Flodasiran [8] - The company has a robust pipeline with multiple independent and partner launches expected over the next few years, supported by a strong balance sheet and access to non-dilutive capital [9][22] - The focus is on expanding its commercial presence while leveraging its RNAi technology platform for future drug development [21][55] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's financial strength and the potential for significant value creation despite current biotech market challenges [9][22] - The company anticipates that the approval of Flodasiran will mark a major advancement in treatment options for patients with familial chylomicronemia syndrome [26][43] Other Important Information - The company closed a significant collaboration agreement with Sarepta Therapeutics, bringing in $500 million upfront and additional potential milestone payments [22][23] - Arrowhead's cash and investments totaled $1.1 billion as of March 31, 2025, providing a strong financial foundation for future growth [54] Q&A Session Summary Question: What are the expectations for initial monotherapy and potential combo data for ARO INHBE and ARO ALK7? - Management indicated that they are not providing specific guidance on expectations but are looking forward to seeing data on weight loss and body composition [58][60] Question: How robust is the pancreatitis data for Flodasiran? - Management noted that they have not had labeling negotiations with the FDA yet and emphasized the importance of triglyceride reduction in relation to pancreatitis risk [66][70] Question: What are the expectations for acute pancreatitis rates in the SHTG population? - Management expects the baseline for acute pancreatitis to be around 2000 mg/dL, similar to the FCS population [80] Question: What is the potential for ARO C3 and ARO CFB in complement-mediated diseases? - Management is open to partnerships for these compounds but believes they could also build a commercial presence if necessary [84][86]