Less than a week after the new weight loss drug Zepbound was approved by the US Food and Drug Administration (FDA), Lilly, the world's largest pharmaceutical company by market value (NYSE: LLC, stock price of $597.71, market value of $567.5 billion), has also seen positive results in the development of important drugs.
A study released by Lilly at the American Heart Association meeting in Philadelphia on November 12th (Sunday) Eastern Time found that a single dose of the new experimental drug Lepodisiran reduced the risk factor of a heart disease by 94% in its first human trial, lasting for nearly a year. The research results showed that after receiving the highest dose of Lepodisiran, a lipoprotein related to arterial obstruction in the body of the subjects rapidly decreased, and the median level of lipoprotein (a) remained 94% lower than the "baseline" within 48 weeks.
Steve Nissen, a cardiologist at the Cleveland Clinic in Ohio and the lead investigator of the study, said that this study gives people a glimmer of hope that by injecting a vaccine like drug once a year, it can clear the lipoprotein (a), also known as Lp (a), of people who are genetically prone to heart disease.
Lipoprotein (a) is about to decrease to an undetectable level in 29 days
Steve Nissen said in a statement, "If further experiments show that Lepodisiran is safe and can reduce heart attacks and strokes, it will be good news for patients as it eliminates risk factors that we cannot treat
Bloomberg reported that over 1 billion people worldwide have lipoprotein (a) levels that put them at moderate to high risk of developing cardiovascular disease, which is the main cause of death worldwide. The cholesterol carrying lipoprotein (a) in the human blood will accumulate on the vascular wall, forming plaques and reducing the flow of blood to the heart, brain, and other parts of the body. According to the American Heart Association, high lipoprotein (a) levels are hereditary and a common independent risk factor for cardiovascular disease. Even worse, exercise and a healthy diet are almost unable to reduce lipoprotein (a) levels, and existing drugs, including Lipitor, have no effect.
In the above-mentioned trial sponsored by Lilly, 48 high lipoprotein (a) subjects from the United States and Singapore were randomly assigned to receive six different doses of placebo or Lepodisiran. Except for the highest dose of 608 milligrams, which is administered in two separate doses, all others are single subcutaneous injections administered to the abdomen. The research results have been published in the Journal of the American Medical Association (JAMA).
The study stated that the blood levels of the subjects receiving the highest dose rapidly increased and returned to the "baseline" level within two days, possibly due to Lepodisiran being rapidly transported from the blood to the subjects' liver. At the same time, the levels of lipoprotein (a) in the blood of the subjects rapidly decreased and could no longer be detected by the 29th day. This situation persisted until the 281st day, and within 48 weeks, their median lipoprotein (a) levels were still 94% lower than the "baseline".
It is reported that the purpose of this study is to test the safety and tolerability of Lepodisiran. Currently, a mid-term trial is underway to investigate the impact of Lepodisiran on populations with high levels of lipoprotein (a) and high risk of early heart attack or stroke. It should be pointed out that due to the lack of reliable treatment methods, lipoprotein (a) in the human body is not often measured.
Steve Nissen pointed out, "If someone has a family history of early heart disease, they should adhere to measuring lipoprotein (a). At the same time, measures can be taken to lower blood pressure, treat low-density lipoprotein (LDL) cholesterol, maintain good dietary habits, and take other preventive measures
Years of research needed before FDA approval
The Daily Economic News reporter noticed that Lepodisiran is only one of several therapeutic drugs currently being developed for lipoprotein (a). In addition to Lilly, American pharmaceutical company Amgen Inc. has also made breakthroughs in this field, and its Olpasiran has also shown a sustained reduction in disease risk in participants in a mid-term study.
At the American Heart Association meeting on the same day, Boston based Verve Therapeutics (NASDAQ: VERV, stock price $15.7, market value $1 billion) also released the results of its Phase 1b mid-term trial of another therapy.
This therapy uses a gene editing method called base editing, which involves intravenous injection of a drug targeting the PCSK9 gene, which plays an important role in the production of low-density lipoprotein (LDL) cholesterol.
When the drug targets the PCSK9 gene, it will undergo minor changes to that gene. Dr. Sekar Kathiresan, co-founder and CEO of Verve Therapeutics, said that this effect is similar to a permanent eraser, eliminating the ability of genes to increase cholesterol. It should be pointed out that in theory, the effect of gene editing therapy should last for a lifetime, but so far, researchers have only followed these subjects for six months.
The preliminary study released by Verve Therapeutics on Sunday aimed to test the safety of its drug, with 10 participants participating in the trial. The results showed that the majority of participants received relatively low doses and there was no significant change in low-density lipoprotein levels. Only three subjects received higher doses of treatment, and their low density lipoprotein cholesterol (LDL) levels decreased by more than half.
Verve Therapeutics stated that the company needs to conduct more research to ensure its treatment plan is safe, free from unexpected side effects, and effective. Furthermore, it is currently unclear whether the Verve Therapeutics method will significantly reduce the risk of heart attack and stroke.
According to NBC News, the above two therapies require several years of additional research before obtaining FDA approval. Nevertheless, industry experts are still impressed by the current research results. Dr. Hugh Cassiere, Director of Intensive Care Services at the Northwell Cardiovascular Institute at South Bank University Hospital in New York, pointed out that "apart from revolutionary research, there is no other term to describe current research progress