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Arrowhead Pharmaceuticals Announces Promising Phase 3 Results for Plozasiran in Familial Chylomicronemia Syndrome
PASADENA, Calif.–Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) has shared encouraging outcomes from its Phase 3 PALISADE study, which investigated the efficacy of plozasiran in patients with familial chylomicronemia syndrome (FCS). FCS is a rare genetic disorder characterized by extremely elevated triglyceride levels and currently lacks FDA-approved treatments in the U.S.
The PALISADE trial successfully achieved its primary endpoint, demonstrating a statistically significant reduction in triglycerides, apolipoprotein C-III (APOC3), and lower incidences of acute pancreatitis. These findings were revealed in a late-breaking presentation at the European Society of Cardiology (ESC) Congress 2024 and published concurrently in The New England Journal of Medicine.
Based on these positive results, Arrowhead plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) by the end of 2024, aiming for subsequent approvals from other global regulatory agencies.
Individuals with severe hypertriglyceridemia face increased risks of acute pancreatitis and other serious health issues, heavily impacting their quality of life. Dr. Gerald F. Watts, a professor of cardio-metabolic medicine, emphasized the limited treatment options now available, which often involve stringent dietary restrictions and offer only minimal benefits. He noted that plozasiran showed exceptional effectiveness in reducing triglyceride levels, representing a potential breakthrough in therapy for patients suffering from FCS.
Bruce Given, M.D., chief medical scientist at Arrowhead, expressed optimism about the results from the SUMMIT program, which includes various studies targeting different patient populations. He highlighted that a significant number of patients in the PALISADE trial achieved triglyceride levels below clinically recognized thresholds for acute pancreatitis risk, a crucial objective in managing this condition. Notably, the trial included both genetically confirmed patients and those with clinically evident persistent chylomicronemia, suggesting that the efficacy of plozasiran may extend beyond those with known genetic mutations.
PALISADE Study Overview
In the PALISADE study, 75 patients with persistent chylomicronemia were randomized to receive either subcutaneous plozasiran at doses of 25 mg (26 patients), 50 mg (24 patients), or a placebo (25 patients) every three months over a year. Patients exhibited a baseline median triglyceride level of 2044 mg/dL. The trial featured a mix of participants, with 59% genetically confirmed to have FCS and the remainder diagnosed based on symptoms.
At the ten-month mark, patients receiving plozasiran exhibited an 80% reduction in triglyceride levels in the 25 mg group, compared to a 17% reduction in the placebo group.
Moreover, the medians for APOC3 saw notable decreases, with the 25 mg and 50 mg groups showing reductions of 93% and 96%, respectively, against only a 1% decline in the placebo cohort.
Importantly, the study also revealed an 83% reduction in the risk of developing acute pancreatitis. Among the evaluated subjects, only two cases of pancreatitis occurred in the plozasiran groups compared to seven cases in the placebo group, reinforcing the therapy’s potential as a safe alternative for managing this serious condition.
Safety Profile of Plozasiran
Plozasiran’s safety was affirmatively established in the PALISADE study, with adverse events aligning closely with those observed in the placebo group. Common side effects included abdominal pain, headaches, and upper respiratory infections, with serious adverse events reported more frequently among those receiving placebo. Instances of hyperglycemia were limited and primarily observed in patients with pre-existing diabetes or in pre-diabetic states.
Upcoming Events and Future Directions
The company will host a virtual analyst and investor event on September 3, 2024, at 8:00 am EDT, to offer deeper insights into the trial’s results, featuring discussions led by Arrowhead leadership and an encore presentation by Professor Watts.
About Familial Chylomicronemia Syndrome
Familial chylomicronemia syndrome is a rare, genetic disorder that can lead to dangerously high triglyceride levels and serious complications such as pancreatitis and diabetes. Currently, therapeutic interventions are limited, highlighting the need for innovative treatment solutions.
About Plozasiran
Plozasiran, previously known as ARO-APOC3, is a novel investigational therapy using RNA interference technology to lower APOC3 levels, thereby facilitating triglyceride metabolism. Through the SUMMIT program, plozasiran is also being evaluated in various patient populations for a range of related conditions.
About Arrowhead Pharmaceuticals
Arrowhead Pharmaceuticals focuses on developing RNAi-based therapies that silence the genes responsible for various diseases. The company employs advanced RNA chemistries to initiate the RNA interference process, potentially transforming treatment options for patients with challenging health conditions.
For additional information, visit www.arrowheadpharma.com.
Note: Plozasiran remains an investigational product and has not yet received FDA approval.
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