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]

PASADENA, Calif.-(BUSINESS WIRE)---- Sarwr--Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that yesterday it filed a Complaint for Declaratory Judgment in the United States District Court for the District of Delaware against Ionis Pharmaceuticals, Inc., to declare that Ionis's United States Patent No. 9,593,333 ("the '333 patent† ) is invalid and not infringed by Arrowhead's planned commercialization of investigational plozasiran, which is under FDA review. Arrowhead intends to vigorously. ...

PresentationMichael UlzEquity Analyst All right. Good afternoon, everyone. Thanks for joining us at the Morgan Stanley Global Healthcare Conference. I'm Mike Ulz, one of the biotech analysts here, and it's my pleasure to introduce the team from Arrowhead Pharmaceuticals. To my immediate left, Christopher Anzalone, CEO; and to my far left, James Hamilton, CMO and Head of R&D. Before we get started, I just need to read a quick disclosure. For important disclosures, please see the Morgan Stanley research discl ...

Company Overview - Arrowhead Pharmaceuticals is represented by its CEO Christopher Anzalone and CMO James Hamilton at the Morgan Stanley Global Healthcare Conference [1]. Conference Highlights - The conference serves as a platform for Arrowhead Pharmaceuticals to engage with investors and discuss their strategies and developments in the biotech sector [1][3].

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.

PASADENA, Calif.--(BUSINESS WIRE)---- $arwr--Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that it has filed a request for regulatory clearance to initiate a Phase 1/2a clinical trial of ARO-MAPT, the company's investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for tauopathies including Alzheimer's disease, a progressive neurodegenerative disease characterized by cognitive and functional decline. Alzheimer's disease is the most common cause of de. ...

Arrowhead Pharmaceuticals, Inc. (NASDAQ:ARWR) is one of the Best Stocks to Buy for the Next 3 Months According to Hedge Funds. On September 2, Patrick Trucchio from H.C. Wainwright reiterated a Buy rating on Arrowhead Pharmaceuticals, Inc. (NASDAQ:ARWR) with a price target of $80. The analyst noted the company’s recent deal with Novartis as a strong vote of confidence. The deal brings $200 million upfront, which has the potential to lead to a $2 billion milestone. Trucchio highlighted that this deal valid ...

Question-and-Answer SessionSo just to begin, if we could, start with an overview of the -- of Arrowhead today, the scope of the pipeline, therapeutic areas you're prioritizing and strategic pillars guiding execution.Dr. Christopher AnzaloneChairman, CEO & President Sure. Thanks very much, and thanks for having us. It's really a pleasure to be here. We are a broad-based RNAi company. We have leveraged this modality to get into a number of different cell types. And therefore, we have the ability to treat a nu ...

Question-and-Answer SessionSo just to begin, if we could, start with an overview of the -- of Arrowhead today, the scope of the pipeline, therapeutic areas you're prioritizing and strategic pillars guiding execution.Dr. Christopher AnzaloneChairman, CEO & President Sure. Thanks very much, and thanks for having us. It's really a pleasure to be here. We are a broad-based RNAi company. We have leveraged this modality to get into a number of different cell types. And therefore, we have the ability to treat a nu ...

Arrowhead Pharmaceuticals (NasdaqGS:ARWR) FY Conference September 09, 2025 10:30 AM ET Company ParticipantsChristopher Anzalone - President, CEO & Chairman of BoardJames Hamilton - Chief Medical Officer & Head of R&DConference Call ParticipantsPatrick Trucchio - MD & Senior Healthcare AnalystPatrick TrucchioHello, everyone. Good morning. Welcome back to HC Wainwright's twenty seventh annual global investment conference held on September 2025. I'm Patrick Trucchio, senior health care analyst at HC Wainwright ...