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]

Core Insights - The U.S. FDA has approved REDEMPLO (plozasiran), a small interfering RNA (siRNA) medicine, for reducing triglycerides in adults with familial chylomicronemia syndrome (FCS) [1] - FCS is a severe and rare disease affecting an estimated 6,500 individuals in the U.S., characterized by extremely high triglyceride levels [1] Company Summary - Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) is the company behind the newly approved siRNA medicine [1] - The approval of REDEMPLO represents a significant advancement in treatment options for patients suffering from FCS [1] Industry Context - The approval of siRNA therapies like REDEMPLO highlights the growing importance of RNA-based treatments in addressing rare genetic disorders [1] - The market for treatments targeting rare diseases is expanding, driven by increasing awareness and advancements in biotechnology [1]

Core Insights - The U.S. Food and Drug Administration (FDA) has approved Arrowhead Pharmaceuticals' drug for a genetic disorder that leads to extremely high levels of fat in the bloodstream [1] Company Summary - Arrowhead Pharmaceuticals has received FDA approval for its drug targeting a specific genetic disorder [1] Industry Summary - The approval signifies a potential advancement in treatment options for genetic disorders related to lipid metabolism [1]

Core Insights - Arrowhead Pharmaceuticals (ARWR) has significant upcoming events, including the PDUFA date for plozasiran for treating familiar chylomicronemia syndrome (FCS) and the expected release of fiscal Q4 2025 results [2] Company Developments - The PDUFA date for plozasiran is scheduled for tomorrow, indicating a critical regulatory milestone for Arrowhead Pharmaceuticals [2] - Fiscal Q4 2025 results are anticipated next week, which may provide insights into the company's financial performance and future outlook [2] Investment Focus - The Growth Stock Forum, led by a former stockbroker, emphasizes identifying growth and biotech stocks with substantial growth potential, focusing on risk-adjusted returns [2]

Core Insights - Arrowhead Pharmaceuticals (ARWR) has significant upcoming events, including the PDUFA date for plozasiran for treating familiar chylomicronemia syndrome (FCS) and the release of fiscal Q4 2025 results [2] Company Developments - The PDUFA date for plozasiran is scheduled for tomorrow, indicating a critical regulatory milestone for Arrowhead Pharmaceuticals [2] - Fiscal Q4 2025 results are anticipated next week, which may provide insights into the company's financial performance and future outlook [2] Investment Focus - The Growth Stock Forum, led by a former stockbroker, emphasizes identifying growth and biotech stocks with substantial growth potential, focusing on risk-adjusted returns [2]

Regulatory Overview - The FDA has approved 34 novel drugs from January to October 2025, compared to 38 during the same period in 2024 [2] - Several biotech stocks are highlighted as they approach key regulatory decisions in November 2025 [2] Arrowhead Pharmaceuticals Inc. (ARWR) - ARWR's New Drug Application for Plozasiran is under FDA review, with a decision expected on November 18, 2025 [3] - Plozasiran targets familial chylomicronemia syndrome, a condition characterized by extremely high triglyceride levels, which can lead to acute pancreatitis [3] - Clinical trials showed significant reductions in triglycerides and the incidence of acute pancreatitis, potentially providing a new treatment option alongside Tryngolza, the only FDA-approved drug for this condition [4] Otsuka Holdings Co. Ltd. (OTSKF) - OTSKF is awaiting the FDA's decision on Sibeprenlimab, scheduled for November 28, 2025 [6] - Sibeprenlimab is an investigational monoclonal antibody for treating immunoglobulin A nephropathy (IgAN), a chronic kidney disease [6] - Clinical trials demonstrated a significant reduction in urine protein-to-creatinine ratio, indicating its potential as a blockbuster drug if approved [8] Bayer (BAYRY) - Bayer's investigational compound Sevabertinib is under priority review by the FDA, with a decision expected on November 28, 2025 [9] - Sevabertinib is proposed for treating advanced non-small cell lung cancer (NSCLC) with HER2 mutations, providing an additional option for previously treated patients [10] Ascendis Pharma A/S (ASND) - The FDA decision on Navepegritide, aimed at treating children with achondroplasia, is due on November 30, 2025 [11] - Achondroplasia is a rare genetic condition affecting over 250,000 people globally, leading to serious complications [12] - Navepegritide is designed for weekly subcutaneous administration, targeting the overactive FGFR3 pathway [13] Kura Oncology Inc. (KURA) - KURA is expecting FDA approval for Ziftomenib on November 30, 2025, for treating relapsed or refractory acute myeloid leukemia (AML) with NPM1 mutations [15] - Ziftomenib has shown significant efficacy in clinical trials, with a market opportunity estimated at $350 million to $400 million annually in the U.S. if approved [17]

Core Insights - The biotech sector is experiencing significant momentum, with many clinical-stage and commercial biopharma stocks reaching new 52-week highs due to breakthrough trial data, strategic deals, and earnings surprises [1] Company Summaries - **Metsera Inc. (MTSR)**: A clinical-stage biopharmaceutical company focused on developing hormone analog peptides for obesity and related metabolic diseases. Recently, Novo Nordisk made a $9 billion unsolicited acquisition proposal, which Metsera's board considers superior to its existing agreement with Pfizer [2][3]. Metsera announced positive Phase 2b results for its GLP-1 receptor agonist MET-097i, showing up to 14.1% weight loss after 28 weeks, supporting Phase 3 initiation in late 2025 [4]. The stock rose from $32.35 to a 52-week high of $66.10, a gain of 104.3% [5]. - **Indivior Plc (INDV)**: Develops buprenorphine-based therapies for opioid dependence. The company reported Q3 net income of $42 million, up from $22 million a year ago, with adjusted earnings of $93 million, exceeding Wall Street's expectations [6][7]. The stock increased from $20.86 to a 52-week high of $30.55, reflecting a 46.5% gain [7]. - **Insmed Inc. (INSM)**: Focused on therapies for serious and rare diseases, Insmed reported a Q3 net loss of $370 million but saw net product revenue rise to $142.3 million from $93.4 million last year [8][9]. The company raised its full-year 2025 revenue guidance for ARIKAYCE to $420 million - $430 million, indicating 15% - 18% growth year-over-year [10]. The stock surged from $76.54 to a 52-week high of $194.70, marking a 154.4% gain [10]. - **Ventyx Biosciences Inc. (VTYX)**: A clinical-stage biotech company developing therapies for autoimmune and neurodegenerative diseases. Ventyx reported positive Phase 2 results for its NLRP3 inhibitor VTX3232, showing strong safety and tolerability [11][13]. The stock rose from $3.01 to a 52-week high of $8.52, a gain of over 183% [14]. - **Inhibrx Biosciences Inc. (INBX)**: Focuses on oncology and rare diseases, announcing positive topline results from its ChonDRAgon study for ozekibart in chondrosarcoma [15]. The stock increased from $18.35 to a 52-week high of $83.78, representing a gain of 356.6% [16]. - **ABIVAX Société Anonyme (ABVX)**: Developing therapies for chronic inflammatory diseases, ABIVAX presented positive Phase 3 data for obefazimod in ulcerative colitis [17]. The stock rose from $7.83 to an all-time high of $106.73, marking a significant gain of 1263% [18]. - **Arrowhead Pharmaceuticals Inc. (ARWR)**: Developing RNAi-based therapies, Arrowhead finalized a licensing agreement with Novartis for ARO-SNCA, with financial terms including a $200 million upfront payment [19][20][21]. The stock increased from $29.70 to a 52-week high of $43.33, representing a gain of over 45% [21]. - **Kodiak Sciences Inc. (KOD)**: Focused on retinal diseases, Kodiak announced positive Phase 1b data for KSI-101, showing significant vision improvements [22][23][24]. The stock climbed from $8.98 to a 52-week high of $21.17, delivering a 135.7% gain [22]. - **Arcutis Biotherapeutics Inc. (ARQT)**: Focused on dermatological treatments, Arcutis reported Q3 net income of $7.4 million, a turnaround from a net loss last year, with revenue up 122% year-over-year [25][26]. The stock rose from $14.99 to a 52-week high of $27.08, representing a gain of over 80% [27]. - **Maze Therapeutics Inc. (MAZE)**: Developing precision therapies for various diseases, Maze announced positive Phase 1 results for MZE782 and secured $150 million in a private placement [28][29]. The stock rose from $11.21 to a new 52-week high of $34.29, returning a gain of 206% [29]. - **Supernus Pharmaceuticals Inc. (SUPN)**: Focused on CNS disorders, Supernus is expected to report Q3 earnings of $0.82 per share and revenue of $180.22 million [30][31]. The stock increased from $38.21 to a 52-week high of $57.65, representing a gain of over 50% [31].

Core Insights - Four biotechnology stocks have surged into the top 10th percentile for momentum ranking, indicating strong technical strength and price action [1][2] Group 1: Momentum Ranking - Arrowhead Pharmaceuticals Inc. (ARWR), Assembly Biosciences Inc. (ASMB), Atara Biotherapeutics Inc. (ATRA), and Corcept Therapeutics Inc. (CORT) have shown significant momentum percentile increases, making them stand out in the competitive biotech sector [2][5] - Joining the top decile for momentum ranking means these stocks are outperforming 90% of all tracked stocks in terms of price appreciation and volatility-adjusted returns [5] Group 2: Individual Stock Performance - ARWR's momentum percentile increased from 89.87 to 93.79, with a week-on-week gain of 3.92 percentage points; the stock has risen by 66.92% year-to-date and 69.33% over the year [7] - ASMB moved from 87.58 to 93.13, posting a 5.55 percentage point increase; it is up 43.85% YTD and 62.95% over the year [7] - ATRA climbed from 88.24 to 92.76, translating to a weekly gain of 4.52 percentage points; the stock has declined 2.97% YTD but advanced 69.00% over the year [8] - CORT advanced from 88.05 to 90.50, achieving a week-on-week improvement of 2.45 percentage points; it has risen by 67.84% YTD and 81.22% over the year [8]

Company Highlights - Aurinia Pharmaceuticals Inc. (AUPH) has seen solid growth for its product LUPKYNIS, with expected net product sales for 2025 ranging from $250 million to $260 million, up from $216.2 million last year [2][3] - Arrowhead Pharmaceuticals Inc. (ARWR) is preparing for its first commercial launch in 2025, with its lead drug candidate Plozasiran under FDA review, decision expected on November 18, 2025 [4][5] - Beta Bionics Inc. (BBNX) reported a 54% growth in second-quarter net sales, with expectations for full-year 2025 net sales to range between $88 million and $93 million, compared to $65.1 million last year [7][8] - Kiniksa Pharmaceuticals International plc (KNSA) anticipates net product revenue for its drug Arcalyst to be between $625 million and $640 million for 2025, up from $417 million in 2024 [10][11] - Ionis Pharmaceuticals Inc. (IONS) generated net product sales of $19 million in Q2 2025, with a supplemental new drug application expected by year-end [12][14] - Stoke Therapeutics Inc. (STOK) is developing Zorevunersen for Dravet syndrome, with a 52-week high of $24.60 reached recently, representing an 82% gain in less than two months [16][17] - Fortress Biotech Inc. (FBIO) is awaiting an FDA decision on its investigational drug CUTX-101 for Menkes disease, expected on September 30, 2025 [18][21] Stock Performance - AUPH stock increased by 168% over 1.5 years, reaching a 52-week high of $12.87 [3] - ARWR stock gained 65% in less than 9 months, hitting a 52-week high of $31.13 [5] - BBNX stock rose nearly 55% in less than 4 months, reaching a 52-week high of $26.66 [8] - KNSA stock saw a 46% increase in less than four months, touching a 52-week high of $37.34 [11] - IONS stock gained 52% in less than two months, reaching a 52-week high of $64.72 [15] - STOK stock increased by 82% in less than two months, achieving a 52-week high of $24.60 [17] - FBIO stock rose by 61% in less than a month, reaching a 52-week high of $3.97 [21]

Core Viewpoint - Arrowhead Pharmaceuticals, Inc. has filed a Complaint for Declaratory Judgment against Ionis Pharmaceuticals, Inc. to declare the invalidity of Ionis's U.S. Patent No. 9,593,333 and assert that Arrowhead's planned commercialization of investigational plozasiran does not infringe this patent [1] Company Summary - Arrowhead Pharmaceuticals is currently under FDA review for its investigational drug plozasiran [1] - The company intends to vigorously defend its position regarding the patent dispute with Ionis Pharmaceuticals [1] Industry Context - The legal action highlights ongoing patent disputes within the pharmaceutical industry, particularly concerning investigational drugs and their commercialization [1]