PASADENA, Calif. - Arrowhead Pharmaceuticals, Inc. (NASDAQ: NASDAQ:ARWR) announced that the FDA has granted Breakthrough Therapy designation to plozasiran for reducing triglycerides in adults with familial chylomicronemia syndrome (FCS). This rare genetic disorder, characterized by extremely high triglyceride levels, can lead to acute pancreatitis with potentially fatal outcomes. As of today, no FDA-approved treatments for FCS exist.
The Breakthrough Therapy designation is aimed at accelerating the development and review of drugs for serious conditions when preliminary clinical evidence suggests significant improvement over existing therapies. With this designation, plozasiran, which also holds Orphan Drug and Fast Track Designations by the FDA and Orphan Drug Designation by the European Medicines Agency, could reach patients sooner.
Clinical trials have shown plozasiran to reduce triglycerides by 80% from baseline and lower the risk of acute pancreatitis by 83% in patients with FCS. The company plans to submit a New Drug Application to the FDA by the end of 2024 and will seek regulatory approvals globally thereafter.
Plozasiran, an investigational RNA interference (RNAi) therapeutic, works by reducing the production of apolipoprotein C-III (APOC3), a key regulator of triglyceride metabolism. The PALISADE Phase 3 study, recently completed, along with other clinical studies, has demonstrated the drug's potential in reducing triglycerides and other atherogenic lipoproteins in patients with FCS and other lipid disorders.
Arrowhead has established an expanded access program (EAP) for certain individuals living with FCS to receive plozasiran while it remains under investigation. The company, known for developing RNAi-based therapeutics, emphasizes the importance of discussing all treatment options with a healthcare provider.
This news is based on a press release statement from Arrowhead Pharmaceuticals, Inc. and does not include any promotional content or endorsement of the drug's efficacy. The safety and effectiveness of plozasiran have not been established, as it has not yet been reviewed or approved for any disease by regulatory authorities.
In other recent news, Arrowhead Pharmaceuticals, following the release of comprehensive Phase 3 PALISADE trial data, received a reiterated Buy rating from TD Cowen and H.C. Wainwright analysts. The study, which focuses on patients with familial chylomicronemia syndrome (FCS), demonstrated an 83% reduction in the risk of acute pancreatitis. The robustness of the data suggests that the drug ploza could become a key standard of care for FCS treatment. Analysts from Citi and Piper Sandler also maintained their respective Neutral and Overweight ratings on Arrowhead.
The company anticipates submitting a New Drug Application by the end of 2024, with a potential product launch the following year. Arrowhead reported a net loss of $170.8 million for its fiscal 2024 third-quarter performance, with cash and investments totaling $436.7 million. To support its pipeline development, Arrowhead secured a $400 million loan from Sixth Street.
Arrowhead is also advancing two RNA interference candidates, ARO-INHBE and ARO-ALK7, into the final stages of preclinical development for obesity and metabolic disease treatment. These recent developments reflect the latest steps in Arrowhead's ongoing efforts to improve patient outcomes.
InvestingPro Insights
Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) has recently garnered significant attention due to the FDA's Breakthrough Therapy designation for plozasiran. As investors and industry analysts closely watch ARWR's progress, certain financial metrics and analyst insights provide a deeper understanding of the company's current market position. According to InvestingPro data, Arrowhead holds a market capitalization of $2.71 billion, reflecting its valuation within the biotechnology sector.
InvestingPro Tips indicate that Arrowhead is currently trading near its 52-week low, which could suggest a potential entry point for investors believing in the company's long-term prospects. However, it's important to note that analysts have revised their earnings downwards for the upcoming period, and the company is not expected to be profitable this year. These factors should be considered when evaluating the investment potential of ARWR.
Moreover, the company's revenue for the last twelve months as of Q3 2024 stands at $19.65 million, with a notable revenue decline of 92.33% compared to the previous period. This could be indicative of the challenges faced in the industry or specific to the company's pipeline development. Despite these financial figures, Arrowhead's liquid assets exceed its short-term obligations, which may provide some financial stability as it continues to invest in its drug pipeline.
For investors seeking a more comprehensive analysis, there are additional InvestingPro Tips available at InvestingPro's dedicated ARWR page. These insights could offer valuable context to Arrowhead's financial health and market potential, especially as the company navigates the pivotal phase of seeking regulatory approval for its promising drug candidate, plozasiran.
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