Specialists at the Cleveland Clinic announced the results of the first phase of a clinical trial, which showed that a single dose of the experimental treatment Lepodisiran succeeded in reducing levels of lipoprotein (a) Lp(a), which is one of the main risk factors for heart disease. In the blood by more than 94% for about a year.
The results of the study were presented, entitled: “Efficacy and safety of lipodesiran: a long-term study of short-acting lipoprotein (A),” which
Efficacy and Safety of Lepodisiran: An Extended Duration Short-Interfering RNA Targeting Lipoprotein(a) Study), during a scientific session recently held within the framework of the activities of the 2023 Scientific Session of the American Heart Association, and it was also published simultaneously. The results of the study were published in the Journal of the American Medical Association.
Lipoprotein (A), known as Lp(a), is produced in the liver and has similar properties to low-density lipoprotein (LDL), known as “bad cholesterol.”
Heart attacks and strokes
Unlike other cholesterol particles, lipoprotein (A) levels are determined by genes by 80-90%, and the structure of lipoprotein (A) particles causes plaque to accumulate in the arteries, which increases the risk of heart attacks. And strokes significantly.
Although effective therapeutic methods are available to reduce the risk of heart attacks by lowering harmful cholesterol and other types of lipoproteins, there is not yet any approved drug treatment to reduce lipoprotein (A) levels, which, given that they are determined by the genes that the individual carries. Any change in lifestyle (diet or exercise) will not have any noticeable effect.
Trial participants who were injected with the highest dose of lipodesiran recorded a reduction in lipoprotein (A) levels of up to 96% within two weeks, and then maintained lower levels of more than 94% below baseline for a period of 48 weeks.
The drug is a short interfering RNA (siRNA) treatment that blocks the production of messenger RNA needed to manufacture one of the most important components of lipoprotein (A).
Promising treatment
The results of the study contribute to adding lipodesiran to the growing list of promising treatments for atherosclerotic cardiovascular diseases in people with high levels of lipoprotein (A), which is expected to affect about 1.4 billion people around the world.
Dr. Steven Nissen, chief academic officer of the Cardiovascular and Thoracic Institute at Cleveland Clinic, said: “The results indicate that the trial participants demonstrated good tolerance to the drug and recorded a long-term reduction in lipoprotein (A), which is one of the most important factors. “The risk of heart attacks, strokes, and aortic stenosis.”
The researchers included 48 patients from the United States and Singapore, with an average age of 47 years, in the trial. They studied the effects of 6 different doses and a placebo dose, which were given to all patients through injections. The patients were then monitored for 48 weeks.
The highest plasma lipoprotein (A) concentrations were reduced by 49%. No negative effects were recorded on the safety of the participants, and the only tolerability problem that was recorded was moderate-severe reaction symptoms at the injection site.
Dr. Nissen added: “Despite clear evidence of the role of lipoprotein (A) as a risk factor for heart disease, an effective treatment has been elusive. This new treatment approach gives hope to the approximately 20% of the world’s population who suffer from From high levels of lipoprotein (A).
The second phase of the clinical study of the drug “Lipodisiran” is currently being conducted.
