PASADENA, Calif. - Arrowhead Pharmaceuticals, Inc. (NASDAQ: NASDAQ:ARWR) unveiled results from its Phase 2b MUIR study of plozasiran, a drug aimed at treating mixed hyperlipidemia, a condition characterized by abnormal levels of cholesterol and triglycerides in the blood. The findings, which were presented at the European Atherosclerosis Society's 92nd Congress and published in the New England Journal of Medicine, indicate that plozasiran significantly lowers triglyceride levels and other atherogenic lipoproteins.
The double-blind, randomized MUIR study assessed the efficacy of plozasiran in reducing triglyceride-rich lipoproteins, which are linked to an increased risk of atherosclerotic cardiovascular disease (ASCVD). According to Christie M. Ballantyne, M.D., Principal Investigator for the MUIR study, the data supports the progression of plozasiran into Phase 3 trials for patients at an elevated risk for ASCVD.
The study, which involved 353 participants with mixed hyperlipidemia, showed that plozasiran treatment resulted in placebo-adjusted reductions in triglycerides of 50% to 62% across various doses at week 24. Additionally, most patients achieved normalized fasting triglyceride levels. The treatment also led to reductions in non-HDL-C, apoB, and remnant cholesterol levels, with strong correlations to changes in triglyceride levels.
Bruce Given, M.D., interim chief medical scientist at Arrowhead, expressed optimism about the potential of plozasiran in addressing multiple diseases with substantial unmet needs, including familial chylomicronemia syndrome, severe hypertriglyceridemia, and mixed hyperlipidemia.
Safety and tolerability of plozasiran were also favorable, with the occurrence of treatment-emergent adverse events (TEAEs) and discontinuations comparable to placebo throughout the 48-week observation period. The adverse events observed were generally reflective of the comorbidities and underlying conditions of the study population.
Arrowhead Pharmaceuticals is known for developing RNA interference (RNAi) therapeutics, which leverage a natural gene-silencing mechanism to inhibit the expression of specific genes. Plozasiran, an investigational RNAi therapeutic, is designed to reduce the production of Apolipoprotein C-III (APOC3), a key regulator of triglyceride metabolism. The company is also conducting additional clinical studies, including the PALISADE Phase 3 clinical study in patients with familial chylomicronemia syndrome, and the Phase 3 SHASTA-3 and SHASTA-4 studies in patients with severe hypertriglyceridemia.
Plozasiran has received Orphan Drug Designation and Fast Track Designation from the U.S. Food and Drug Administration and Orphan Drug Designation by the European Medicines Agency. The information in this article is based on a press release statement from Arrowhead Pharmaceuticals.
InvestingPro Insights
Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) is at a critical juncture as it advances its RNAi therapeutics, with recent studies showing promising results. However, a closer look at the company's financial health and market performance through InvestingPro data reveals a more nuanced picture. Arrowhead's market capitalization stands at $3.09 billion, indicating a significant presence in the biotech sector. Despite the positive clinical outcomes, analysts have flagged concerns, as reflected by the company's negative P/E ratios, with a standard P/E ratio of -5.83 and an adjusted P/E ratio for the last twelve months as of Q2 2024 at -6.56. This suggests that investors are not expecting immediate profitability.
Moreover, the revenue for the same period was $35.47 million, which represents a steep decline of 87% from the previous year. This sharp decrease raises questions about the company's revenue trajectory. InvestingPro Tips indicate that analysts have revised earnings upwards for the upcoming period, offering a glimmer of hope for future financial performance. Additionally, while Arrowhead is not currently profitable and analysts do not expect profitability this year, the company's liquid assets do exceed short-term obligations, providing some financial stability in the near term.
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