Arrowhead Pharmaceuticals (ARWR) Soars to Fresh Highs on Alzheimer’s Trial and FDA Breakthrough Wins

Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) is finishing 2025 with the kind of momentum small-molecule biotechs daydream about. On 8 December 2025, the RNA interference (RNAi) specialist climbed to a new 52‑week high near $69 per share after the company announced it had dosed the first subjects in its Alzheimer’s disease trial ARO‑MAPT and highlighted a string of recent FDA victories and deal-driven cash inflows. ^[1]

The rally caps a stunning year in which Arrowhead stock has delivered a year‑to‑date return of roughly 270% and a one‑year gain in the 160–200% range, depending on the data provider, vastly outpacing the broader market. ^[2]

Arrowhead Pharmaceuticals stock today: powerful breakout in December

As of intraday trading on 8 December 2025, Arrowhead shares were changing hands around $68–69, having traded between roughly $65.7 and $69.6 during the session. That move followed a prior close of $61.44 and pushed the stock to a new 52‑week high just under $69.6, versus a 52‑week low near $9.6. ^[3]

Key trading and performance metrics:

Intraday move: roughly +14–15% on 8 December after the Alzheimer’s trial update. ^[4]
Market capitalization: about $9–9.6 billion, depending on source and exact price snapshot. ^[5]
Liquidity: current and quick ratios both around 4.8–4.9, indicating a strong short‑term balance sheet. ^[6]
Volatility: beta in the 1.3–1.5 range, meaning Arrowhead tends to move more than the market in both directions. ^[7]

In short, Arrowhead has transitioned from a beaten‑down RNAi developer into one of 2025’s standout biotech momentum names.

Clinical catalysts: Alzheimer’s, FCS and severe hypertriglyceridemia

ARO‑MAPT: a new RNAi shot at Alzheimer’s disease

The immediate spark behind the 8 December surge was Arrowhead’s announcement that it has dosed the first subjects in a Phase 1/2a trial of ARO‑MAPT, an investigational RNAi therapy targeting tau protein in Alzheimer’s disease and other tauopathies. ^[8]

Highlights from the company’s Alzheimer’s update:

Mechanism: ARO‑MAPT uses Arrowhead’s TRiM™ RNAi platform and a new delivery system designed to cross the blood–brain barrier after a subcutaneous injection, allowing systemic dosing rather than invasive intrathecal infusions. ^[9]
Target: It aims to silence the MAPT gene, which encodes tau. Aggregation of abnormal tau is a key driver of several neurodegenerative diseases, including Alzheimer’s. ^[10]
Trial design: The Phase 1/2a study (AROMAPT‑SC‑1001) will enroll up to 64 healthy volunteers and 48 patients with early Alzheimer’s disease, testing multiple dose levels and dosing regimens over weekly and monthly injections. ^[11]
Timeline: Arrowhead currently expects initial data in the second half of 2026, underscoring that the Alzheimer’s program is a long‑dated, high‑risk, high‑reward asset. ^[12]

An AI‑aided note from Investing.com framed the market reaction succinctly: Arrowhead stock jumped roughly 14–15% after the company confirmed dosing of the first subjects in ARO‑MAPT, reflecting growing investor enthusiasm for CNS‑targeted RNAi platforms. ^[13]

Plozasiran / REDEMPLO: first commercial product and a broader cardiometabolic play

The ARO‑MAPT headline landed on top of a major November win: FDA approval of Redemplo (plozasiran), Arrowhead’s first commercialized drug. Redemplo is an siRNA medicine that targets apolipoprotein C‑III (apoC‑III) to cut triglyceride levels in familial chylomicronemia syndrome (FCS), a rare genetic disorder associated with extremely high triglycerides and recurrent, sometimes fatal, pancreatitis. ^[14]

Key points about plozasiran/Redemplo:

FCS approval: The FDA cleared Redemplo as an adjunct to diet for adults with FCS after a Phase 3 trial showed about an 80% reduction in triglycerides and a large reduction in pancreatitis risk versus placebo. ^[15]
Breakthrough Therapy designation: On 2 December, the FDA granted Breakthrough Therapy status to plozasiran to reduce triglycerides in adults with severe hypertriglyceridemia (SHTG, ≥500 mg/dL), intended to accelerate development and review in a broader population. ^[16]
Ongoing Phase 3 program: Arrowhead is running the SHASTA‑3 and SHASTA‑4 Phase 3 trials in SHTG and the MUIR‑3 study in mixed hyperlipidemia, with completion targeted around mid‑2026 and plans to file a supplemental NDA by the end of 2026. ^[17]
Commercial upside: Analysts cited by Reuters see potential > $1.4 billion in annual Redemplo sales by 2031, though that forecast is far from guaranteed and must contend with competition from Ionis Pharmaceuticals’ rival drug and ongoing patent litigation. ^[18]

In Arrowhead’s own words, Redemplo is the first medicine to bring its TRiM™ platform into full commercial use, effectively shifting the company from pure development‑stage biotech into a commercial‑stage RNAi company. ^[19]

Fiscal 2025 results: revenue explosion and deal‑driven growth

Arrowhead’s financial profile changed dramatically in fiscal 2025 (year ended 30 September 2025). According to the company’s year‑end results:

Revenue surged to about $829.4 million, compared with just $3.6 million in fiscal 2024. ^[20]
Operating income swung from a $601 million loss to a $98 million profit, helped by milestone and upfront payments. ^[21]
After non‑controlling interests, Arrowhead reported a near‑breakeven net loss of about $1.6 million, versus a loss of nearly $600 million the prior year. ^[22]
Cash and marketable securities totaled roughly $782 million, providing a sizable runway to fund its pipeline and commercial launch. ^[23]

What drove the revenue explosion?

Redemplo approval and launch for FCS, along with associated collaboration economics. ^[24]
A $200 million milestone payment from Sarepta Therapeutics linked to progress with ARO‑DM1, an RNAi therapeutic for myotonic dystrophy type 1, on top of an earlier $100 million milestone for enrollment and dose escalation. ^[25]
A global collaboration with Novartis for ARO‑SNCA, including a $200 million upfront payment and potential up to $2 billion in future milestones plus royalties. ^[26]
A Sanofi/Visirna transaction under which a majority‑owned Arrowhead subsidiary received a $130 million upfront payment and could earn additional milestones for plozasiran in Greater China, while Sanofi gained exclusive rights in that territory. ^[27]

A trading‑oriented summary from StocksToTrade noted that Arrowhead reported around $829 million in revenue and roughly $30 million in net income at the consolidated level, underscoring how milestones temporarily pushed the business into the black. ^[28]

However, analyst models suggest this revenue spike is not yet sustainable. Consensus forecasts compiled by StockAnalysis.com see revenue falling to about $398 million in fiscal 2026 and $292 million in 2027 as one‑time deal payments roll off, with EPS expected to remain negative over the next two years. ^[29]

Pipeline snapshot: beyond Redemplo

Arrowhead’s investment case rests on more than a single FCS drug. Its RNAi pipeline spans multiple organ systems, with a particular strength in cardiometabolic and pulmonary disease:

Plozasiran (REDEMPLO): In‑market for FCS, in Phase 3 for severe hypertriglyceridemia and mixed hyperlipidemia (SHASTA‑3/4 and MUIR‑3/MUIR‑5). ^[30]
Zodasiran: An RNAi therapeutic for severe dyslipidemia and homozygous familial hypercholesterolemia (HoFH), now in the YOSEMITE Phase 3 trial. ^[31]
ARO‑DM1 (with Sarepta): Targeting myotonic dystrophy type 1; progress in a Phase 1/2 study has already triggered $300 million in milestones for Arrowhead. ^[32]
ARO‑SNCA (with Novartis): A preclinical siRNA program against alpha‑synuclein for synucleinopathies such as Parkinson’s disease. ^[33]
ARO‑DIMER‑PA: A first‑in‑class “dual target” RNAi candidate for atherosclerotic cardiovascular disease, simultaneously silencing PCSK9 and APOC3; a Phase 1/2a filing is in place. ^[34]
Pulmonary programs (ARO‑RAGE, ARO‑MUC5AC): Early‑stage trials in inflammatory and muco‑obstructive lung diseases. ^[35]

The Los Angeles Business Journal recently highlighted that Arrowhead is now conducting clinical studies on around 20 different RNAi‑based candidates, supported by multi‑billion‑dollar partnerships with Janssen, Takeda, Novartis and Sarepta. ^[36]

Wall Street’s view: bullish on the science, cautious on the valuation

Ratings and price targets

Analysts have responded to Arrowhead’s clinical and regulatory momentum with a wave of target price upgrades, but the stock’s explosive move has already overtaken many of those targets.

Recent moves include: ^[37]

HC Wainwright: Strong Buy; price target raised from $80 to $85 (2 December 2025).
Bank of America: Strong Buy; target raised from $42 to $62 (1 December 2025).
Chardan Capital: Strong Buy; $60 target (1 December 2025).
Goldman Sachs: Hold/Neutral; target raised from $27 to $48 (20 November 2025).
Morgan Stanley: Hold/Equal‑weight; target lifted from $45 to $48 (26 November 2025).

Across different aggregators:

MarketBeat reports 2 Strong Buys, 5 Buys, 3 Holds and 1 Sell, for an overall “Moderate Buy” and a consensus target around $53–54. ^[38]
StockAnalysis shows a Buy / Strong Buy consensus with an average target in the low‑to‑mid $50s, based on roughly 9–16 covering analysts. ^[39]
GuruFocus quotes a recommendation score around 2.1 (on a scale where lower is more bullish) and a target price near $58, but also flags elevated valuation ratios. ^[40]

With the shares trading close to $69, the current price already sits above many published 12‑month targets, leaving only the most optimistic calls (like the $85 HC Wainwright target) comfortably ahead of the market. ^[41]

Valuation metrics

Quant screens now paint Arrowhead as a classic high‑growth, high‑valuation biotech:

Price‑to‑sales: roughly 8–10× trailing revenue, depending on whether you include non‑controlling interests and which price snapshot you use. ^[42]
Price‑to‑book: mid‑teens to high‑teens, significantly above large‑cap pharma averages. ^[43]
Profitability: trailing EPS is close to break‑even (around ‑$0.01 to ‑$0.08 per share), but consensus forecasts call for renewed losses as milestone revenue normalizes. ^[44]
Technical picture: GuruFocus notes a 14‑day RSI above 70, signaling an overbought condition, and an elevated valuation on price‑to‑sales and price‑to‑book screens. ^[45]

Several analytic pieces, including from Simply Wall St and others, point out that while the share price has catapulted more than 30% in recent weeks and well over 150% in a year, Arrowhead’s underlying earnings and cash flows still lag the broader biotech industry, leaving little room for execution missteps at current levels. ^[46]

Who’s buying? Institutional flows, insiders and ownership

Institutional money has piled into Arrowhead in 2025:

Marshall Wace LLP increased its ARWR position by an eye‑catching 3,508% in Q2, now holding about 2.16 million shares (roughly 1.56% of the company) worth around $34 million, according to a recent MarketBeat summary. ^[47]
Norges Bank, Norway’s sovereign wealth fund, disclosed a new stake of about 1.93 million shares, valued at roughly $30.4 million, amounting to about 1.4% of Arrowhead. ^[48]
Other large investors, including Vanguard and various state pension funds, have also added modestly to their holdings. ^[49]

On the other hand, insiders have been net sellers over the last quarter:

A senior executive sold 20,000 shares at around $35, while a director sold 8,750 shares at about $56.39, leaving insiders with roughly 4–6% of the float depending on the data source. ^[50]

GuruFocus estimates institutional ownership above 80%, underscoring how tightly Arrowhead is now held by professional investors. ^[51]

Arrowhead Pharmaceuticals stock forecast: key things to watch in 2026

Given the combination of clinical momentum, a new commercial product and aggressive share‑price appreciation, Arrowhead has moved squarely into the “story stock” bracket. For investors and traders trying to frame a forward‑looking view, several milestones stand out.

1. Commercial execution for Redemplo

Uptake in FCS: The near‑term commercial test is how quickly Redemplo can penetrate the small but high‑need FCS population, where only a few thousand patients worldwide are eligible. ^[52]
Pricing and access: Arrowhead has announced a “One‑Redemplo” pricing model and a support program called Rely On Redemplo, designed to smooth reimbursement and adherence. Early feedback from prescribers and payers will be closely scrutinized. ^[53]
Label and indication expansion: Success in the SHASTA and MUIR Phase 3 programs could open up much larger SHTG and mixed‑hyperlipidemia markets, radically expanding Redemplo’s commercial footprint. ^[54]

2. ARO‑MAPT clinical readouts

The first human safety, biomarker and dose‑finding data for ARO‑MAPT will be a major binary event in the second half of 2026. Encouraging signals on tau knockdown, CSF biomarkers or cognitive measures in early patients could justify today’s Alzheimer’s premium; disappointing data could deflate it. ^[55]

3. Partnership‑driven milestones and royalties

Investors will be watching for additional milestone payments from Sarepta, Novartis and Sanofi, as well as the first signs of royalty income if partnered products move closer to market. ^[56]

4. Normalization of revenue and cash burn

With 2025 revenue heavily skewed by one‑time collaboration payments, analyst forecasts calling for a >50% revenue decline in 2026 will be tested against actual Redemplo sales and partnership economics. ^[57]
Arrowhead’s sizeable cash position gives it room to invest in its pipeline, but investors will pay close attention to R&D intensity, operating expenses and potential future capital raising if the pipeline expands faster than partnership cash flows. ^[58]

Risks to the Arrowhead bull case

Even ardent bulls acknowledge that Arrowhead remains a high‑risk biotech despite the recent string of wins.

Clinical and regulatory risk
- ARO‑MAPT is in early‑stage human testing, and many CNS drug candidates fail despite compelling preclinical data.
- Redemplo’s broader hypertriglyceridemia program still depends on successful Phase 3 outcomes and regulatory approvals beyond FCS. ^[59]
Revenue volatility and dependence on deal economics
- 2025’s revenue spike was driven heavily by milestones and upfronts, not recurring product sales. Consensus expects a pullback in revenue and a return to GAAP losses in the near term. ^[60]
Patent litigation and competition
- Redemplo directly competes with Ionis Pharmaceuticals’ FCS therapy, and the two companies are engaged in patent disputes over RNA‑targeting IP. Although analysts see room for Redemplo to win meaningful market share, court outcomes are uncertain. ^[61]
Valuation risk
- With the stock trading around or above the highest end of several published price targets, and valuation multiples well above sector averages, even minor disappointments on clinical, commercial or regulatory fronts could trigger sharp pullbacks. ^[62]
Market‑wide biotech sentiment
- RNAi and gene‑targeted therapies remain a risk‑on corner of the market. A shift in risk appetite, higher interest rates, or sector‑wide headline risk could weigh on ARWR regardless of company‑specific execution. ^[63]

Bottom line: a transformed RNAi player with a stretched stock

As of 8 December 2025, Arrowhead Pharmaceuticals looks very different from the loss‑making RNAi story that many investors wrote off two years ago. The company now:

Has its first FDA‑approved drug (Redemplo) on the market for FCS. ^[64]
Enjoys Breakthrough Therapy status for plozasiran in severe hypertriglyceridemia and a clear regulatory road map to expand into broader lipid disorders. ^[65]
Has launched a first‑in‑class Alzheimer’s RNAi trial (ARO‑MAPT) that, if successful, could push Arrowhead into one of the largest therapeutic markets in medicine. ^[66]
Is backed by deep‑pocketed partners (Novartis, Sanofi, Sarepta), meaningful milestone payments and a substantial late‑stage cardiometabolic pipeline. ^[67]

At the same time, the share price has sprinted ahead of most published valuation work, and future revenue is still heavily dependent on unproven indications, future approvals and partnership execution. For investors, Arrowhead has become a classic high‑beta biotech: the upside case is now visible in Redemplo’s expanding label and the ARO‑MAPT Alzheimer’s program, but the room for error at current prices has narrowed considerably.