Arrowhead公布plozasiran两项sHTG三期顶线阳性并计划年内提交美国sNDA

Arrowhead Pharmaceuticals Reports Topline Results from Phase 3 SHASTA-3 and SHASTA-4 Studies of Plozasiran in Patients with Severe Hypertriglyceridemia
-
Deep, durable, and consistent median triglyceride reductions of 79% and 81% from baseline in
SHASTA -3 andSHASTA -4, respectively - Statistically significant reduction in acute pancreatitis events versus placebo across the entire sHTG study population, with an unprecedented 100% event reduction in high-risk patients
- Continued and consistent safety and tolerability profile with no new safety signals
-
Detailed results will be presented as a HOT LINE Late Breaker at the
European Society of Cardiology (ESC) Congress onAugust 30, 2026
“We continue to see plozasiran data as best in class with respect to safety, activity, efficacy, and convenience, with dosing only four times per year, and we are more confident than ever in its promise,” said
In
Plozasiran demonstrated a favorable safety and tolerability profile in
Full analysis of the safety and efficacy results from
Plozasiran has received regulatory approval, as REDEMPLO®, in
Detailed results from
ESC 2026 Presentation Details:
Session Title: HOT LINE 9 Late Breaker Session
Title: Plozasiran in patients with severe hypertriglyceridemia: the
Date & Time:
Presenter:
The ESC 2026 presentation materials and a link to the conference call and webcast will be made available on the Events and Presentations page under the Investors section of the Arrowhead website.
About Severe Hypertriglyceridemia
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.
About
About REDEMPLO® (plozasiran)
REDEMPLO (plozasiran) is currently approved by the
REDEMPLO is designed to suppress the production of apolipoprotein C-III (APOC3), a protein produced in the liver that raises triglyceride levels by slowing their breakdown and clearance. By targeting APOC3 with sustained silencing, REDEMPLO delivers significant reductions in triglyceride levels. REDEMPLO is self-administered via subcutaneous injection once every three months.
REDEMPLO has been granted Orphan Medicinal Product Designation by the EMA for the treatment of patients with
Sanofi acquired the rights to develop and commercialize REDEMPLO in
For more information about REDEMPLO, visit Our Medicines.
About Arrowhead Pharmaceuticals
Arrowhead Pharmaceuticals (NASDAQ: ARWR) is a commercial-stage pharmaceutical company developing medicines that treat intractable diseases by silencing the genes that cause them, harnessing the natural RNA interference (RNAi) mechanism. The company has built a broad portfolio of clinical and commercial RNAi therapeutics through its industry-leading targeted RNAi molecule (TRiM™) platform, which can precisely silence genes in a wide range of cell types, including liver, lung, muscle, adipose, and central nervous system tissue. At Arrowhead, we rapidly advance potential best- and first-in-class RNAi treatments for diseases with significant unmet medical need, because every day matters to the patients we serve.
For more information, please visit www.arrowheadpharma.com, or follow us on X (formerly Twitter) at @ArrowheadPharma, LinkedIn, Facebook, and Instagram. To be added to the Company's email list and receive news directly, please visit http://ir.arrowheadpharma.com/email-alerts.
Safe Harbor Statement under the Private Securities Litigation Reform Act:
This news release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Any statements contained in this release except for historical information may be deemed to be forward-looking statements. Without limiting the generality of the foregoing, words such as “may,” “will,” “expect,” “believe,” “anticipate,” “hope,” “intend,” “plan,” “project,” “could,” “estimate,” “continue,” “target,” “forecast” or “continue” or the negative of these words or other variations thereof or comparable terminology are intended to identify such forward-looking statements. In addition, any statements that refer to projections of our future financial performance, trends in our business, expectations for our product pipeline, products or product candidate or other characterizations of future events or circumstances are forward-looking statements. These forward-looking statements include, but are not limited to, statements about our beliefs and expectations regarding the long-term impacts of REDEMPLO® (plozasiran) on patient health and the health care system; our beliefs and expectations regarding the pricing, value, or expected timing for availability of our drugs and drug candidates; and our beliefs and expectations around the potential uses and value of the TRiM™ platform. These statements are based upon our current expectations and speak only as of the date hereof. Actual results or outcomes may differ materially and adversely from those expressed in any forward-looking statements as a result of numerous factors and uncertainties the safety and efficacy of our products and product candidates, pricing and reimbursement decisions related to our products, demand for our products, decisions of regulatory authorities and the timing thereof, the duration and impact of regulatory delays in our clinical programs, our ability to finance our operations, the likelihood and timing of the receipt of future milestone and licensing fees, the future success of our scientific studies, the timing for starting and completing clinical trials, rapid technological change in our markets, the enforcement of our intellectual property rights, and the other risks and uncertainties described in our most recent Annual Report on Form 10-K, subsequent Quarterly Reports on Form 10-Q and other documents filed with the Securities and Exchange Commission from time to time. We assume no obligation to update or revise forward-looking statements to reflect new events or circumstances.
Source: Arrowhead Pharmaceuticals, Inc.
Arrowhead Pharmaceuticals, Inc.
Vince Anzalone, CFA
+1 626-304-3400
ir@arrowheadpharma.com
Investors:
LifeSci Advisors, LLC
Brian Ritchie
+1 212-915-2578
britchie@lifesciadvisors.com
Media:
HAVAS PR
Erick Edwing
+1-941-468-7543
Erick.edwing@havas.com
Source: Arrowhead Pharmaceuticals, Inc.