Arrowhead Pharmaceuticals, Inc. (ARWR.US), which has surged a staggering 253% year-to-date in 2025, ranks among the top 20 performing stocks in the U.S. market this year. Its gains have significantly outpaced those of the "AI chip titan" NVIDIA (NVDA.US) and even slightly exceeded the full-year performance of Micron (MU.US), the current leader in market share and valuation within the hottest global equity investment theme: memory chips. The prominent Wall Street investment firm Piper Sandler recently issued a research report, reiterating its "Overweight" rating on Arrowhead Pharmaceuticals, Inc. while simultaneously raising its price target on the stock from $70 to $100. With the stock closing at $64.560 last week, this implies a potential short-to-medium term upside of up to 55%. Piper Sandler's exceptionally bullish 12-month target of $100 for Arrowhead serves as a powerful booster shot for the global innovative drug sector, which is currently undergoing a corrective phase. Arrowhead's stock performance has been lackluster this year, declining nearly 3% year-to-date and substantially underperforming the S&P 500 index.
What exactly is Arrowhead Pharmaceuticals, the company focused on "gene silencing"? Arrowhead Pharmaceuticals, Inc. is a publicly traded biopharmaceutical/biotechnology company headquartered in Pasadena, California. Its core research and development focus is on creating novel therapies for a range of serious diseases, including cardiometabolic diseases, liver diseases, and viral diseases, based on RNA interference (RNAi) technology. For many years since its founding, this biotech firm has primarily dedicated itself to developing "gene silencing" therapeutics, which treat conditions by targeting specific genes. The company has concentrated on developing therapies that utilize the RNAi mechanism to "silence" disease-causing genes, building a pipeline of drugs targeting various difficult-to-treat illnesses, and possesses its own proprietary RNAi platform with multiple drug candidates in clinical stages.
For most of its history, Arrowhead has been a clinical-stage biotechnology company, with a pipeline featuring numerous candidates at various stages of clinical development (ranging from early to late phases), including drugs targeting liver diseases, hypertriglyceridemia, and dozens of other conditions. Undoubtedly, for a long period, the majority of Arrowhead's projects remained in the clinical R&D phase. However, with the regulatory approval and market launch of Redemplor (plozasiran), the company has transitioned from a purely clinical-stage enterprise into a commercial-stage entity, marking its evolution into a biopharmaceutical company that balances both R&D and commercial operations, rather than remaining a pure "clinical-stage company."
Redemplor is a highly novel innovative drug, representing one of the pioneering therapies based on RNAi technology and can be classified as "first-in-class" or near-first-in-class among similar siRNA therapies; it has received formal regulatory approval. Redemplor has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of familial chylomicronemia syndrome (FCS) in adults and has begun its market entry. This is Arrowhead's first drug to receive FDA approval, signaling its transition from the clinical phase to the commercialization phase. According to recent media reports, Health Canada has also approved Redemplor for FCS, indicating significant regulatory recognition for the drug in the North American market.
Redemplor is a small interfering RNA (siRNA) drug targeting the APOC3 gene, designed to significantly reduce triglyceride levels in the blood for the treatment of the rare inherited metabolic disorder, familial chylomicronemia syndrome (FCS). Its mechanism of action involves using RNA interference to inhibit the production of the apoC-III protein, thereby improving lipid metabolism.
Redemplor, Arrowhead Pharmaceuticals' "Future Revenue Engine." The formal approval of Redemplor signifies Arrowhead's transformation from an R&D-focused company into a commercial pharmaceutical enterprise. In the short term, it is expected to generate steadily expanding sales figures; in the medium to long term, with expansion into global markets and broader indications, it has the potential to become the core driver of the biotech firm's total revenue, or even a potentially massive "revenue engine," though clinical, competitive, and market risks remain throughout this process.
Redemplor (plozasiran) has received FDA approval for the treatment of familial chylomicronemia syndrome (FCS) in adults and is the first and only approved siRNA therapy for this rare disease. This market launch marks Arrowhead's successful transition from its long-standing market positioning as a "clinical-stage company" to a "commercial-stage biotechnology company," as noted by Piper Sandler analyst Edward Tenthoff. Piper projects U.S. FCS sales to be at least $625,000 in Q4 2025, growing substantially to approximately $12.3 million in 2026. The firm suggests these positive projections might be conservative, citing Redemplor's superior efficacy, a quarterly injection regimen that offers a more convenient treatment model, and a cost that is a full $60,000 cheaper per year than Ionis's Trygolza.
Piper Sandler also expresses considerable confidence in the biotech company's upcoming Phase 3 clinical trial data for Redemplor in severe hypertriglyceridemia (sHTG), expected in Q3 2026, believing it could enable an unprecedented breakthrough label expansion in 2027. Furthermore, on January 7, 2026, Wall Street financial giant Morgan Stanley maintained its "Equal-Weight" rating on Arrowhead Pharmaceuticals, Inc. but significantly raised its price target for the biotech firm from $48 to $81. The financial giant pointed out that early single-agent treatment data for ARO-INHBE and ARO-ALK7 suggest clinical results validate one of the fat-reduction targets and demonstrate promise in treating long-term obesity, which Morgan Stanley believes could become an additional, powerful driver of value expansion for the drug.
Research data from Morgan Stanley indicates that if Redemplor gains approval and is successfully commercialized for sHTG, or even broader dyslipidemia indications, its projected global peak sales could exceed $2 billion (or even higher). One biotech industry research institution anticipates sales potential by 2031 or beyond to be far greater than the revenue generated from FCS alone.
