Arrowhead Pharmaceuticals, Inc. Initiates Expanded Access Program for Plozasiran
March 25, 2024 at 08:00 am EDT
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Arrowhead Pharmaceuticals, Inc. announced that it has initiated an Expanded Access Program (EAP) to make investigational plozasiran available outside of a clinical trial for patients with familial chylomicronemia syndrome (FCS) who meet certain program eligibility criteria. Arrowhead will also present new final Phase 2 clinical data from the double-blind portion of the SHASTA-2 study of plozasiran in a late-breaking oral presentation at the upcoming American College of Cardiology 73rd Annual Scientific Session & Expo (ACC.24), being held in Atlanta on April 6-8, 2024. Arrowhead is committed to bringing new investigational medicines to patients with serious diseases as quickly and efficiently as possible. The company?s EAP is a potential pathway for patients with serious or immediately life-threatening diseases or conditions to gain access to an investigational medical product for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available. The plozasiran EAP is for individuals living with FCS. As with any investigational medicine that has not been approved by regulatory authorities, investigational plozasiran may or may not be effective in treating diagnosis or condition, and there may be risks associated with its use. Plozasiran, previously called ARO-APOC3, is a first-in-class investigational RNA interference (RNAi) therapeutic designed to reduce production of Apolipoprotein C-III (APOC3) which is a component of triglyceride rich lipoproteins (TRLs) and a key regulator of triglyceride metabolism. APOC3 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 APOC3, thereby reducing triglycerides and restoring lipids to more normal levels.
In multiple clinical studies, investigational plozasiran demonstrated reductions in triglycerides and multiple atherogenic lipoproteins in patients with familial chylomicronemia syndrome (FCS), severe hypertriglyceridemia (SHTG), and mixed dyslipidemia (MD). Plozasiran has demonstrated a favorable safety profile to date with treatment emergent adverse events reported that reflect the comorbidities and underlying conditions of the study populations. Plozasiran is currently being investigated in the Phase 3 PALISADE clinical study in patients with FCS, which is on schedule to be completed in the middle of 2024. Phase 2 studies in patients with SHTG and MD, SHASTA-2 and MUIR respectively, are complete and additional Phase 3 studies are planned to begin shortly.
Arrowhead Pharmaceuticals, Inc. develops medicines that treat intractable diseases by silencing the genes that cause them. The Company's therapies, using a broad portfolio of ribonucleic acid (RNA) chemistries and modes of delivery, trigger the RNA interference (RNAi) mechanism to induce rapid, deep and durable knockdown of target genes. The Company's Targeted RNAi Molecule (TRiMTM) platform utilizes ligand-mediated delivery and is designed to enable tissue-specific targeting while being structurally simple. It is focused on various therapeutic areas, such as cardiometabolic, pulmonary, liver, muscle and central nervous system. It has around 14 clinical-stage investigational medicines (nine wholly owned and five partnered), which range in development stage from Phase I to Phase III. The Company's pipeline products include Plozasiran, Zodasiran, Olpasiran, ARO-RAGE, ARO-MUC5AC, ARO-MMP7, GSK-4532990, Fazirsiran, JNJ-3989, HZN-457, ARO-C3, ARO-PNPLA3, ARO-DUX4 and ARO-SOD1.