Announcement • Apr 26

Arrowhead Pharmaceuticals Receives Positive CHMP Opinion Recommending Approval Of REDEMPLO (Plozasiran) To Reduce Triglycerides In Adults With Familial Chylomicronemia Syndrome In Europe

Arrowhead Pharmaceuticals, Inc. announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (EMA CHMP) has adopted a positive opinion, recommending the approval of REDEMPLO (plozasiran), a small interfering RNA (siRNA) medicine, as an adjunct to diet to reduce triglyceride levels in adult patients with familial chylomicronemia syndrome (FCS). FCS remains widely underdiagnosed and affects an estimated 1 to 13 people per million globally. This rare disease is characterized by triglyceride levels that can be orders of magnitude higher than normal, leading to a substantially higher risk of developing acute, recurrent, and potentially fatal pancreatitis. The European Commission is expected to issue a decision on REDEMPLO’s Marketing Authorization in the second quarter of 2026. If approved, REDEMPLO will be the first and only siRNA medicine authorized in the European Union for both genetically confirmed and clinically diagnosed adult patients with FCS. This recommendation follows approvals in the United States, Canada, and China, as Arrowhead continues its efforts to increase global access to care for people living with FCS. In a press release announcing the positive opinion, EMA stated, "Although other authorised medicines can help people with FCS confirmed by genetic testing, REDEMPLO does not require genetic confirmation of the condition, thus providing a treatment option for more adults with FCS and addressing the unmet medical need in these patients." Harnessing Arrowhead’s proprietary Targeted RNAi Molecule (TRiM) platform, REDEMPLO is designed to silence the mRNA encoding apolipoprotein C-III (apoC-III). ApoC-III is a key regulator of triglyceride metabolism that inhibits triglyceride catabolism and clearance, resulting in elevated triglyceride levels. Individuals with genetic loss-of-function variants in APOC3 typically have markedly lower triglyceride levels and a reduced risk of atherosclerotic cardiovascular disease. The CHMP positive opinion was supported by clinical data from the Phase 3 PALISADE study, a randomized, double-blind, placebo-controlled trial in adults with clinically diagnosed or genetically confirmed FCS. The PALISADE study met its primary endpoint and all multiplicity-controlled key secondary endpoints, including demonstrating significant reductions in triglycerides and apoC-III and in the incidence of acute pancreatitis in the pooled dose groups. In PALISADE, 25 mg REDEMPLO reduced triglycerides by a median of 80% from baseline versus a 17% reduction with placebo and significantly fewer cases of acute pancreatitis were seen in patients using REDEMPLO compared with those using placebo. REDEMPLO is self-administered via subcutaneous injection once every three months. The most common adverse reactions are hyperglycaemia (12.8%), headache (6.8%), nausea (4.7%), and injection site reaction (4.7%). The efficacy and safety results from the PALISADE study were presented at the European Society of Cardiology Congress 2024 and the American Heart Association Scientific Sessions 2024 (AHA24) and simultaneously published in The New England Journal of Medicine and Circulation, respectively. REDEMPLO has been granted Orphan Designation by the EMA for the treatment of patients with FCS, and Breakthrough Therapy Designation, Fast Track Designation, and Orphan Drug Designation by the U.S. FDA for the treatment of patients with FCS. Familial chylomicronemia syndrome (FCS) is a severe and rare disease leading to extremely high triglyceride (TG) levels, typically over 10 mmol/L (880 mg/dL). Such severe elevations can lead to various serious signs and symptoms including acute and potentially fatal pancreatitis, chronic abdominal pain, diabetes, hepatic steatosis, and cognitive issues. Currently, there are limited therapeutic options to adequately treat FCS. The PALISADE study (NCT05089084) was a Phase 3 placebo-controlled study to evaluate the efficacy and safety of plozasiran in adults with genetically confirmed or clinically diagnosed FCS. The primary endpoint of the study was percent change from baseline in fasting TG versus placebo at Month 10. A total of 75 subjects distributed across 39 different sites in 18 countries were randomized to receive 25 mg plozasiran, 50 mg plozasiran, or matching placebo once every three months. Participants who completed the randomized period were eligible to continue in a 2-part extension period, where all participants receive plozasiran. REDEMPLO (plozasiran) is the first and only siRNA treatment approved in these countries that has been studied in both genetically confirmed and clinically diagnosed patients living with FCS. REDEMPLO is a first-in-class siRNA therapeutic designed to suppress the production of apoC-III, a protein produced in the liver that raises triglyceride levels by slowing their breakdown and clearance. By targeting apoC-III with sustained silencing, REDEMPLO delivers significant reductions in triglyceride levels. REDEMPLO is self-administered via subcutaneous injection once every three months. The EMA CHMP has adopted a positive opinion recommending the European Marketing Authorization of REDEMPLO (plozasiran), which is already approved by the U.S. Food and Drug Administration, Health Canada, and China’s National Medical Products Administration as an adjunct to diet to reduce triglycerides for adults with Familial Chylomicronemia Syndrome (FCS). In addition to approvals of REDEMPLO in the U.S., Canada, and China, plozasiran has been submitted to additional global regulatory authorities for review and marketing authorization. Plozasiran is also being investigated in the SHASTA-3 (NCT06347003), SHASTA-4 (NCT06347016), and SHASTA-5 (NCT06880770) Phase 3 studies in adults with severe hypertriglyceridemia and the MUIR-3 (NCT06347133) Phase 3 study in adults with hypertriglyceridemia. In December 2025, plozasiran was granted Breakthrough Therapy designation by the U.S. FDA in severe hypertriglyceridemia.

Announcement • Mar 28

Arrowhead Pharmaceuticals Presents New Long-Term Efficacy And Safety Data For Plozasiran Across A Spectrum Of Hypertriglyceridemia

Arrowhead Pharmaceuticals, Inc. announced long-term efficacy and safety data from a two-year long open-label extension (OLE) study of investigational plozasiran supporting its potential as therapeutic solution for a diverse spectrum of patients with hypertriglyceridemia (HTG). Patients with severe hypertriglyceridemia (sHTG) achieved an 83% median reduction in triglycerides (TG), with 96% of patients achieving TG levels below 500 mg/dL, a threshold associated with increased risk of acute pancreatitis. No adjudicated acute pancreatitis events occurred in any patient receiving plozasiran during the 2-year Phase 2b Open-Label Expansion (OLE) Study. Favorable and durable improvements in atherogenic lipoproteins, including remnant cholesterol, non-HDL cholesterol, and ApoB, were observed, with a safety profile consistent with earlier trials. Plozasiran is designed to reduce the hepatic production of apolipoprotein C-III (APOC3) through targeted RNA interference. APOC3 is a key regulator of triglyceride metabolism that inhibits both lipoprotein lipase (LPL)-dependent and LPL-independent pathways involved in triglyceride catabolism and clearance, leading to elevated triglyceride levels. Individuals with genetic loss-of-function variants in APOC3 typically have markedly lower triglyceride levels and a reduced risk of atherosclerotic cardiovascular disease. The ACC.26 presentation builds on positive findings from two Phase 2b double-blind, placebo-controlled studies of plozasiran: SHASTA-2, conducted in adults with severe hypertriglyceridemia (sHTG), and MUIR, which enrolled patients with hypertriglyceridemia, both of which demonstrated short-term efficacy and safety. With the same patient populations, the company initiated an extension phase in which both groups received plozasiran 25 mg quarterly via subcutaneous injections. During the two-year OLE, patients saw median reductions in their triglycerides by -83% in sHTG patients from SHASTA-2 and -67% in HTG patients from MUIR with favorable reductions in remnant cholesterol and non-HDL-cholesterol. In both studies, the majority of patients achieved TG levels below thresholds for acute pancreatitis (AP) risk or below normal thresholds. 96% of sHTG patients achieved TGs below 500 mg/dL and 63% achieved TGs below 150 mg/dL, a threshold associated with increased risk of ASCVD. 93% of HTG patients achieved TGs below 150 mg/dL. These findings support the potential of plozasiran as a promising approach for managing patients with moderate to severe HTG phenotypes who are at risk of AP and potentially other cardiometabolic comorbidities. Plozasiran demonstrated a consistent long-term safety and tolerability profile across both sHTG and HTG patient populations, with stable glycemic parameters, no clinically meaningful differences in routine clinical laboratory measurements, and no new safety signals. Common treatment-emergent adverse events included diabetes, COVID-19, upper respiratory tract infection, and back pain, consistent with prior studies; HbA1c levels remained stable. In SHASTA-2, mean placebo-adjusted liver fat content, as assessed by MRI-PDFF, did not change significantly over time with 25 mg plozasiran treatment. Arrowhead is on schedule to complete the SHASTA-3, SHASTA-4, and MUIR-3 Phase 3 clinical studies, the company’s global Phase 3 clinical studies designed to support regulatory submissions for marketing approval of plozasiran for the treatment of severe hypertriglyceridemia, in mid-2026 and intends to submit a supplemental New Drug Application (sNDA) to the U.S. FDA by year-end 2026. Arrowhead also plans to seek regulatory approval with additional global regulatory authorities thereafter. Severe hypertriglyceridemia (SHTG) is characterized by triglyceride (TG) levels greater than 500 mg/dL, with the most severe form being familial chylomicronemia syndrome (FCS) where TGs typically exceed 880 mg/dL. SHTG significantly increases the risk of acute pancreatitis (AP), which can often include recurrent attacks requiring repeat hospital admissions and worsening outcomes. AP risk is proportional to the number, characteristics, and concentration of triglyceride rich lipoproteins (TRLs), particularly chylomicrons, and increases as TGs rise. Elevated TGs can also increase the risk of atherosclerotic cardiovascular disease (ASCVD). Limited treatment options exist to sustainably reduce TGs below guideline directed risk thresholds. Familial chylomicronemia syndrome (FCS) is a severe and rare disease leading to extremely high triglyceride (TG) levels, typically over 880 mg/dL. Such severe elevations can lead to various serious signs and symptoms including acute and potentially fatal pancreatitis, chronic abdominal pain, diabetes, hepatic steatosis, and cognitive issues. Currently, there are limited therapeutic options to adequately treat FCS. Plozasiran is a first-in-class investigational RNA interference (RNAi) therapeutic designed to reduce production of apolipoprotein C-III (apoC-III) which is a component of triglyceride rich lipoproteins (TRLs) and a key regulator of triglyceride metabolism. ApoC-III increases triglyceride levels in the blood by inhibiting breakdown of TRLs by lipoprotein lipase and uptake of TRL remnants by hepatic receptors in the liver. The goal of treatment with plozasiran is to reduce the level of apoC-III, thereby reducing triglycerides and restoring lipids to more normal levels. Plozasiran is being investigated in the SHASTA-3, SHASTA-4, and SHASTA-5 Phase 3 studies in patients with severe hypertriglyceridemia and the MUIR Phase 3 study in patients with mixed hyperlipidemia. REDEMPLO (plozasiran) is approved by the U.S. Food and Drug Administration, Health Canada, and the Chinese National Medical Products Administration as an adjunct to diet to reduce triglycerides for adults with Familial Chylomicronemia Syndrome (FCS). REDEMPLO is an siRNA therapeutic designed to suppress the production of apoC-III, a protein produced in the liver that raises triglyceride levels by slowing their breakdown and clearance. By targeting apoC-III with sustained silencing, REDEMPLO delivers significant reductions in triglyceride levels. REDEMPLO is the first and only siRNA FDA-approved treatment studied in both genetically confirmed and clinically diagnosed patients living with FCS.

Interest coverage ratio	n/a
Cash	US$1.78b
Equity	US$598.92m
Total liabilities	US$1.67b
Total assets	US$2.27b

Data	Last Updated (UTC time)
Company Analysis	2026/07/23 07:10
End of Day Share Price	2026/07/23 00:00
Earnings	2026/03/31
Annual Earnings	2025/09/30

Package	Data	Timeframe	Example US Source ^*
Company Financials	10 years	Income statement Cash flow statement Balance sheet	SEC Form 10-K SEC Form 10-Q
Analyst Consensus Estimates	+3 years	Forecast financials Analyst price targets	Analyst Research Reports Blue Matrix
Market Prices	30 years	Stock prices Dividends, Splits and Actions	ICE Market Data SEC Form S-1
Ownership	10 years	Top shareholders Insider trading	SEC Form 4 SEC Form 13D
Management	10 years	Leadership team Board of directors	SEC Form 10-K SEC Form DEF 14A
Key Developments	10 years	Company announcements	SEC Form 8-K

Analyst	Institution
Ying Huang	Barclays
William Pickering	Bernstein
Jason Matthew Gerberry	BofA Global Research

Arrowhead Pharmaceuticals Balance Sheet Health

Financial Health criteria checks 4/6

Key information

Recent financial health updates

Recent updates

Arrowhead Pharmaceuticals Reports Strong Phase 3 Data for Plozasiran in Hypertriglyceridemia

Price target increased by 8.4% to US$98.27

Arrowhead Pharmaceuticals Reports Topline Results from Phase 3 SHASTA-3 And SHASTA-4 Studies of Plozasiran in Patients with Severe Hypertriglyceridemia

Arrowhead Pharmaceuticals: Redemplo Meets High Expectations In SHTG Trials

Arrowhead Pharmaceuticals, Inc. to Report Q3, 2026 Results on Aug 04, 2026

ARWR: Upcoming SHASTA-3 And 4 Results Will Drive Bullish Repricing

Price target increased by 8.9% to US$96.00

Arrowhead Wins EU Approval for Redemplo Expanding Access in Rare FCS Treatment

ARWR: Cardiometabolic Progress And EU Approval Will Support But Not Eliminate SHTG Uncertainty

Arrowhead Pharmaceuticals, Inc.(NasdaqGS:ARWR) dropped from Russell 2000 Index

ARWR: Mixed Wave Data And Execution Risks Will Challenge Recent Rebound

Arrowhead Pharmaceuticals Receives Marketing Authorization In The European Union For REDEMPLO (Plozasiran) To Reduce Triglycerides In Adults With Familial Chylomicronemia Syndrome

Arrowhead Pharmaceuticals (ARWR) Stock Could Be 25.6% Undervalued After R And D Webinar Update

Arrowhead Pharmaceuticals Stock Gains Focus as Clinical Pipeline and EU Decision Drive Anticipation

Arrowhead Pharmaceuticals, Inc. Announces Appointment of Dr. Matt Cohen as Director, Effective June 12, 2026

ARWR: Cardiometabolic RNAi Readouts And Licensing Will Drive Future Repricing

Arrowhead Pharmaceuticals Presents New Clinical Data on RNAi-based Obesity and MASH Candidate ARO-INHBE at EASL 2026

Arrowhead Pharmaceuticals Presents New Positive Clinical Data for Plozasiran at the 94th European Atherosclerosis Society Congress

ARWR: Cardiometabolic RNAi Data And Licensing Will Drive Future Repricing

Second quarter 2026 earnings: EPS and revenues exceed analyst expectations

New minor risk - Profitability

ARWR: Cardiometabolic RNAi Data And Licensing Progress Will Drive Future Repricing

Arrowhead Pharmaceuticals Readies SHASTA Trial Results With REDEMPLO Launch Gaining Early Traction

Arrowhead Pharmaceuticals Inc Receives Tga Approval of Redemplo (Plozasiran) in Australia Expanding Global Access for Patients with Familial Chylomicronemia Syndrome

Arrowhead Pharmaceuticals Receives Positive CHMP Opinion Recommending Approval Of REDEMPLO (Plozasiran) To Reduce Triglycerides In Adults With Familial Chylomicronemia Syndrome In Europe

ARWR: Wave Data Concerns Will Test Sustainability Of Recent Share Price Rebound

ARWR: RNAi Obesity And Cardiometabolic Data Will Drive Future Repricing

Arrowhead Pharmaceuticals Presents New Long-Term Efficacy And Safety Data For Plozasiran Across A Spectrum Of Hypertriglyceridemia

ARWR: Obesity RNAi Data And Cardiometabolic Franchise Will Drive Future Repricing

ARWR: Obesity RNAi Data Will Test Sustainability Of Recent Share Price Rebound

Chief Medical Officer and Head of R&D notifies of intention to sell stock

ARWR: Obesity And Cardiometabolic Pipeline Progress Will Drive Future Repricing

Arrowhead Pharmaceuticals' (NASDAQ:ARWR) Strong Earnings Are Of Good Quality

Consensus revenue estimates increase by 12%, EPS downgraded

ARWR: Obesity Pipeline Progress Will Drive Future Repricing In My View

First quarter 2026 earnings: EPS and revenues exceed analyst expectations

Arrowhead Pharmaceuticals, Inc. to Report Q1, 2026 Results on Feb 05, 2026

Arrowhead Pharmaceuticals, Inc., Annual General Meeting, Mar 19, 2026

Arrowhead Pharmaceuticals Initiates Phase 1/2a Study of ARO-DIMER-PA - the First Dual Functional RNAi Therapeutic for the Treatment of Mixed Hyperlipidemia

ARWR: Early Obesity RNAi Data Will Challenge Durability Of Recent Share Repricing

ARWR: Obesity RNAi Data Will Test Sustainability Of Recent Share Repricing

Arrowhead Pharmaceuticals, Inc. has completed a Follow-on Equity Offering in the amount of $199.998502 million.

Price target increased by 15% to US$75.91

Arrowhead Pharmaceuticals Announces Nmpa Approval of Redemplo (Plozasiran) for Familial Chylomicronemia Syndrome in China

Arrowhead Pharmaceuticals Announces Health Canada Approval of Redemplo to Reduce Triglycerides in Adults with Familial Chylomicronemia Syndrome

Chairman notifies of intention to sell stock

ARWR: Upcoming SHTG Readouts Will Temper Optimism Despite Recent Cardiometabolic Progress

Chairman recently sold US$12m worth of stock

Chairman notifies of intention to sell stock

Now 20% overvalued after recent price rise

Independent Director recently sold US$992k worth of stock

Price target increased by 7.4% to US$59.25

Arrowhead Pharmaceuticals Initiates Phase 1/2A Study of ARO-MAPT for the Treatment of Alzheimer's Disease and Other Tauopathies

ARWR: Upcoming SHTG Data Will Likely Undermine Optimistic Cardiometabolic Expectations

Independent Director recently sold US$493k worth of stock

Arrowhead Pharmaceuticals, Inc. Receives FDA Breakthrough Therapy Designation for Plozasiran in Severe Hypertriglyceridemia

Arrowhead Pharmaceuticals, Inc. (NASDAQ:ARWR) Stock Catapults 33% Though Its Price And Business Still Lag The Industry

Independent Director notifies of intention to sell stock

New minor risk - Share price stability

Consensus estimates of losses per share improve by 87%

ARWR: FDA Milestone Will Drive Cost Advantage In Cardiometabolic Markets

Price target increased by 16% to US$55.00

Arrowhead Pharmaceuticals, Inc Announces FDA Approval of Redemplo (plozasiran) to Reduce Triglycerides in Adults with Familial Chylomicronemia Syndrome

ARWR: Expect Cost Efficiency To Drive Competitive Advantage In Upcoming Metabolic Trials

Analysts Boost Arrowhead Pharmaceuticals Price Target Amid Cost Advantages and Strategic Partnerships

Consensus EPS estimates fall by 221%

Arrowhead Pharmaceuticals, Inc. Files CTA for Investigational ARO-DIMER-PA - the First Dual Functional RNAi Therapeutic for the Treatment of Mixed Hyperlipidemia

What Arrowhead Pharmaceuticals, Inc.'s (NASDAQ:ARWR) 29% Share Price Gain Is Not Telling You

RNAi Pipeline Will Unlock Emerging Markets Despite Regulatory Risks

Chief Medical Officer and Head of R&D notifies of intention to sell stock

Chief Medical Officer and Head of R&D notifies of intention to sell stock

Ionis Pharmaceuticals, Inc. Files Complaint for Patent Infringement Against Arrowhead Pharmaceuticals, Inc

Consensus EPS estimates have been downgraded.

Arrowhead Pharmaceuticals, Inc. Requests Regulatory Clearance to Initiate Phase 1/2A Study of Aro-Mapt for the Treatment of Alzheimer's Disease and Other Tauopathies

No longer forecast to breakeven