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Arrowhead Pharmaceuticals Initiates Phase 3 PALISADE Study of ARO-APOC3 for Treatment of Familial Chylomicronemia Syndrome

Arrowhead Pharmaceuticals Initiates Phase 3 PALISADE Study of ARO-APOC3 for Treatment of Familial Chylomicronemia Syndrome

PASADENA, Calif., January 12, 2022–(Business WIRE)–Arrowhead Prescribed drugs Inc. (NASDAQ: ARWR) today announced that it has dosed the 1st individuals in the PALISADE examine, a Stage 3 medical study to assess the efficacy and security of ARO-APOC3 in grownups with familial chylomicronemia syndrome (FCS). ARO-APOC3 is the company’s investigational RNA interference (RNAi) therapeutic that is created to inhibit the output of apolipoprotein C3 (APOC3), a vital regulator of triglyceride fat burning capacity. ARO-APOC3 is at the moment currently being investigated in multiple clinical studies, like the Phase 3 PALISADE analyze in patients with FCS, the Section 2b SHASTA-2 research in individuals with intense hypertriglyceridemia (SHTG), and the Period 2b MUIR review in patients with blended dyslipidemia.

Javier San Martin, M.D., main healthcare officer at Arrowhead, mentioned: “Initiating the Phase 3 PALISADE review is an important milestone for the ARO-APOC3 application, for people with FCS, and for Arrowhead. Individuals with genetically described FCS, and these who are phenotypically equivalent, have a tendency to have exceptionally higher triglycerides, which can direct to an elevated chance of recurrent and unpleasant bouts of pancreatitis. These patients presently have quite restricted remedy selections. In the previously conducted Stage 1/2 clinical review of ARO-APOC3 in 4 genetically verified FCS clients, ARO-APOC3 remedy led to a maximal indicate reduction in APOC3 of up to 98%, a maximal median minimize in triglycerides of up to 91% and was typically properly-tolerated. These variations are similar to individuals noticed in 26 participants that experienced SHTG and equivalent baseline triglyceride concentrations but did not have biallelic mutations regular with FCS. These effects had been offered in November 2021 at the American Coronary heart Association Scientific Sessions conference1. We glance ahead to investigating ARO-APOC3 in the larger sized Phase 3 PALISADE examine to even further assess its efficacy and security.”

Arrowhead is partnering with Ambry Genetics to deliver no-expense genetic tests to patients who may be at risk of severe hypertriglyceridemia and FCS. To find out far more about this software, go to the Ambry plan web-site, in this article.

PALISADE (NCT05089084) is a world wide, placebo-controlled Stage 3 research to evaluate the efficacy and basic safety of ARO-APOC3 in adult participants with FCS. Around 60 members who have satisfied all protocol eligibility standards in the course of screening will be randomized in a double-blinded manner to receive ARO-APOC3, or matching placebo, administered subcutaneously after each individual 3 months. Participants will be randomly assigned 2:1:2:1 to the dose cohorts (ARO-APOC3 25 mg, volume-matched placebo, ARO-APOC3 50 mg, and quantity-matched placebo, respectively). The research will enroll members with fasting triglycerides greater than or equal to 10 mmol/L (higher than or equal to 880 mg/dL) that are refractory to normal lipid-decreasing therapy and a diagnosis of FCS as defined in the inclusion requirements. The length of the research is around 56 weeks from screening to the month 12 conclude-of-study evaluation. Following month 12, individuals will be eligible to carry on in an open-label extension research. All contributors in the placebo team who decide to proceed will switch to energetic drug throughout the extension review. The primary objective of the analyze is to assess the transform from baseline in triglycerides involving just about every ARO-APOC3 dose and pooled placebo at month 10.

About Arrowhead Prescribed drugs

Arrowhead Pharmaceuticals develops medications that handle intractable diseases by silencing the genes that trigger them. Making use of a wide portfolio of RNA chemistries and efficient modes of shipping and delivery, Arrowhead therapies result in the RNA interference system to induce immediate, deep, and strong knockdown of concentrate on genes. RNA interference, or RNAi, is a system existing in residing cells that inhibits the expression of a certain gene, thereby affecting the generation of a precise protein. Arrowhead’s RNAi-dependent therapeutics leverage this organic pathway of gene silencing.

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Supply: Arrowhead Pharmaceuticals, Inc.

1 Peter Clifton et al., Presented by Christie Ballantyne on behalf of Peter Clifton, AHA Scientific Sessions 2021

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