- Primary Study Completion Anticipated in the Second Quarter of 2024
PASADENA, Calif., May 16, 2023--(BUSINESS WIRE)--Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it has completed enrollment of its global Phase 3 clinical trial (PALISADE) evaluating ARO-APOC3 for the treatment of familial chylomicronemia syndrome. The company anticipates that the primary portion of the study will be complete in the second quarter of 2024 with a data readout shortly thereafter and subsequent submissions for regulatory review and approval.
Javier San Martin, M.D., chief medical officer at Arrowhead, said: "Completing enrollment of the Phase 3 PALISADE study represents a significant milestone for Arrowhead and brings our potentially important RNAi-based medicines closer to the patients that may benefit from them. I am grateful to the FCS patient community and principal investigators for their trust and participation in the PALISADE study."
About the Familial Chylomicronemia Syndrome
Familial chylomicronemia syndrome (FCS) is a severe and ultrarare genetic disease often caused by various monogenic mutations. FCS leads to extremely high triglyceride (TG) levels, typically over 900 mg/dL. Such severe elevations can lead to various serious signs and symptoms including acute and potentially fatal pancreatitis, chronic abdominal pain, type 2 diabetes mellitus, hepatic steatosis, and cognitive issues. Currently, the therapeutic options that can adequately treat FCS are very limited.
About the PALISADE Phase 3 Study
The AROAPOC3-3001 PALISADE study (NCT05089084) is a Phase 3 placebo controlled study to evaluate the efficacy and safety of ARO-APOC3 in adults with FCS. The primary endpoint of the study is percent change from baseline in fasting TG at Month 10. A total of 75 subjects distributed across 39 different sites in 18 countries were randomized to receive 25 mg ARO-APOC3, 50 mg ARO-APOC3, or matching placebo once every three months. Participants who complete the randomized period are eligible to continue in a 2-part extension period, where all participants will receive ARO-APOC3.
ARO-APOC3 is an investigational RNAi therapeutic being developed as a treatment for patients with mixed dyslipidemia, severe hypertriglyceridemia, and FCS. ARO-APOC3 is designed to reduce production of Apolipoprotein C-III (apoC-III), a component of triglyceride rich lipoproteins (TRLs) including very-low-density lipoprotein (VLDL) and chylomicrons and is a key regulator of triglyceride metabolism. Knocking down the hepatic production of apoC-III by RNAi may result in reduced VLDL synthesis and assembly, enhanced breakdown of TRLs, and better clearance of VLDL and chylomicron remnants.
About Arrowhead Pharmaceuticals
Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing.
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Source: Arrowhead Pharmaceuticals, Inc.
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Arrowhead Pharmaceuticals, Inc.
Vince Anzalone, CFA