Gerald Watts, MD, PhD 

Reflections on Plozasiran Trial Data and Future Potential

Video content above is prompted by the following:

What other approaches have been tested or are in development for treating familial chylomicronemia syndrome (FCS)?

How can clinicians integrate emerging therapy advancements into their clinical practice for the management of FCS? What strategies should be adopted to stay updated with ongoing research and treatment options?

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Familial chylomicronemia syndrome (FCS) treatment advancements include gene therapies, antisense oligonucleotides, and RNA interference technologies. These approaches aim to reduce triglyceride levels and manage symptoms more effectively. Clinicians can integrate these emerging therapies by staying informed through continuous education, attending relevant conferences, and collaborating with specialists. Developing a multidisciplinary approach and leveraging digital platforms for real-time updates are crucial strategies. Engaging in clinical trials and fostering patient-centered care will also enhance the management of FCS, ensuring that patients receive the most current and effective treatments.

Panelists discuss how various new and developing approaches for treating familial chylomicronemia syndrome are emerging, and emphasize the importance for clinicians to integrate these advancements into practice while staying current with ongoing research and evolving treatment strategies.

Novel Research Frontiers on the Management of FCS

Familial chylomicronemia syndrome (FCS) treatment advancements include gene therapies, antisense oligonucleotides, and RNA interference technologies. These approaches aim to reduce triglyceride levels and manage symptoms effectively. Clinicians can integrate these emerging therapies by staying informed through continuous education, attending relevant conferences, and collaborating with specialists. Developing a multidisciplinary approach and utilizing decision-support tools can enhance patient management. To remain updated, clinicians should engage with professional networks, subscribe to medical journals, and participate in clinical trials, ensuring they are aware of the latest research and treatment options.

SHASTA-2 Trial: Plozasiran’s Efficacy in Reducing Triglycerides and APOC3

Please provide a brief overview of the SHASTA-2 study.

The SHASTA-2 study is a clinical trial evaluating the efficacy and safety of a novel therapeutic agent in patients with advanced solid tumors. The study focuses on a specific patient population with limited treatment options, aiming to assess the drug's impact on progression-free survival and overall response rate. It employs a randomized, double-blind, placebo-controlled design to ensure robust data collection and analysis. The trial also investigates potential biomarkers for predicting treatment response, contributing to personalized medicine approaches in oncology.

Panelists discuss how the SHASTA-2 study evaluated the efficacy and safety of plozasiran in reducing triglyceride levels and APOC3 in familial chylomicronemia syndrome, comparing outcomes with the placebo group and assessing implications for treating severe hypertriglyceridemia and preventing pancreatitis.

PALISADE Study: Plozasiran’s Efficacy and Potential in Reducing Triglycerides and Pancreatitis

What role does a multidisciplinary approach play in the management of familial chylomicronemia syndrome (FCS)? How can clinicians collaborate to optimize patient outcomes and address unmet needs in treatment and care?

What does a typical care team look like for an FCS patient? Are there differences between countries/regions?


What are the gaps in existing treatments for FCS in terms of reducing triglyceride levels, and how do these gaps contribute to the risk of acute pancreatitis and the need for hospitalization?

Panelists discuss how a multidisciplinary approach enhances the management of familial chylomicronemia syndrome by fostering collaboration among clinicians to address unmet treatment needs and optimize patient outcomes, while also exploring existing treatment gaps that contribute to ongoing risks of acute pancreatitis and hospitalization.

Gerald Watts, MD, PhD

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