Early clinical trial results offer a promising new avenue for treating high triglycerides, a major risk factor for cardiovascular disease.Developed by OrsoBio in partnership with EPFL Lausanne, the novel compound TLC-2716 has demonstrated notable triglyceride reduction in phase 1 studies and, notably, is administered orally. The findings, published in Nature Medicine, suggest a perhaps simpler and more accessible treatment option compared to existing therapies like statins and newer injectable medications.
LAUSANNE / LONDON – A new compound, TLC-2716, is showing promising results in early clinical trials for lowering triglyceride levels, potentially offering a simpler and safer oral treatment option for those with high cholesterol and related cardiovascular risks.
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Developed by OrsoBio in collaboration with the EPFL Lausanne, TLC-2716 demonstrated significant triglyceride reduction in a Phase 1 clinical study. High triglycerides are a major risk factor for heart disease, stroke, and other cardiovascular problems. The study, published in the journal Nature Medicine, highlights the safety and effectiveness of the orally administered medication.
The innovative approach of TLC-2716 centers on its function as a reverse agonist of the liver X receptor (LXR). This receptor plays a crucial role in regulating fat and cholesterol metabolism. Previous attempts to block the LXR have faced challenges due to the receptor’s protective functions. Researchers overcame this hurdle by focusing on a specific variant of the receptor, LXRβ, which is highly active in the liver and intestines and directly linked to elevated triglyceride levels.
By selectively suppressing the activity of LXRβ without disrupting essential protective pathways, TLC-2716 could address a significant gap in the treatment of lipid disorders. Current standard therapies, such as statins, fibrates, and omega-3 fatty acids, aren’t always effective for all patients. Newer injectable medications like olezarsen also present challenges with ease of use. The oral administration of TLC-2716 could potentially improve patient adherence to treatment plans.
While the Phase 1 study results are encouraging, TLC-2716 is still in the early stages of development. Larger Phase 2 and Phase 3 trials are needed to confirm its efficacy and safety in patients diagnosed with hypertriglyceridemia. Further research will also need to demonstrate that lowering triglyceride levels translates to a reduced risk of heart attack and stroke. If successful, TLC-2716 could become a valuable new tool in the fight against common conditions like high cholesterol and diabetes within the next few years.
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