Experimental drug promising to bring down bad cholesterol by up to 69% completes early stage trial

The Print

26-05-2025

The potential breakthrough medicine developed by US-based biotechnology firm Verve Therapeutics is a novel, in vivo (delivered directly into the body), base editing medicine that permanently turns off the PCSK9 gene (a protein that plays a role in regulating cholesterol levels in the blood) in the liver, thereby reducing disease-causing bad cholesterol.
High levels of low-density lipoprotein cholesterol (LDL-C), commonly referred to as bad cholesterol, are among the leading causes of heart attacks and strokes, the two top reasons as per the World Health Organisation (WHO) for mortality worldwide as well as India.
New Delhi: Would you believe it if someone tells you that the risk of you suffering a heart attack or a stroke could be dramatically reduced by a single injection once in your lifetime? That is exactly the promise that results from an early stage trial of an experimental drug in the US has shown, bringing down bad cholesterol by up to 69 percent after just one shot.
The results from its stage 1b clinical trial show that intravenous infusion of the drug, named VERVE 102 currently, led to a mean LDL reduction of 53 percent among patients receiving the highest dose, with the maximum individual reduction reaching 69 percent, the company said in a statement.
The drug suppressed PCSK9 levels by an average of 60 percent at the highest dose.
Among the 14 participants across three dose levels, the drug was well-tolerated, with no treatment-related serious adverse events and no clinically significant laboratory abnormalities observed, the company said.
Specialists, however, insist results from a large group of patients would be crucial.
The drug has triggered a buzz in the global pharmaceutical industry and is now set to enter a larger trial across five countries to test the efficacy and safety.
'These initial results from the early trial of the drug are very interesting and promise, for the first time, a lifetime treatment of a condition that is main risk factor for several serious cardiovascular events,' Dr Ambuj Roy, a senior cardiologist at the All India Institute of Medical Sciences (AIIMS), New Delhi, told ThePrint.
The clinician-researcher also underlined that data from the drug's trial on a larger group of people would be pivotal before hopes are raised.
The company has maintained that the initial data are promising with respect to both safety and efficacy, and suggest the potential for a new era of cardiovascular disease treatment where a single dose 'might lead to lifelong control of LDL-C'.
Verve has tied up with pharma giant Eli Lilly and Company to co-commercialise the drug following successful trials and regulatory approvals.
WHO defines raised total cholesterol or dyslipidemia—which includes LDL-C—over 5 millimole or 190 mg/dL and it is estimated that nearly 39 percent of adults worldwide suffer from dyslipidemia.
A large survey carried out by the Indian Council of Medical Research (ICMR) and the Madras Diabetes Research Foundation, published in The Lancet in 2023, had shown 81.2 percent Indians have dyslipidemia and 20.9 percent—or 18.5 crore—have high levels of bad cholesterol.
Also Read: India gets 1st guidelines for managing lipid levels, the biggest risk factor for heart attack, stroke
What sets the drug apart
As of now, statins are the most commonly used pharmacotherapies to lower cholesterol levels and work by inhibiting an enzyme called HMG-CoA reductase that is involved in the synthesis of cholesterol in the liver.
But experts say that while statins and various lipid-lowering medications have greatly diminished cardiovascular risks in many patients, there is an urgent requirement for improved, more specific drugs to manage cholesterol.
Such need, explained Dr Amit Bhushan Sharma, the director and unit head of cardiology at Paras Health in Gurugram, is particularly evident in patients with statin intolerance, genetic dyslipidemias such as familial hypercholesterolemia (or high cholesterol), and in those who fail to reach target lipid levels despite receiving optimal treatment.
New therapies like PCSK9 inhibitors, inclisiran—a small interfering RNA (siRNA) therapy that offers sustained LDL-C reduction with just two doses a year launched in India last year—and bempedoic acid have created new possibilities, but they remain underused because of cost and access challenges, experts say. Amid this, there is an increasing interest in RNA-based and gene-editing treatments that may offer lasting cholesterol management with reduced side-effects.
'Improved cholesterol-lowering medications could help reduce the residual cardiovascular risk in high-risk populations, prevent recurrent cardiac events, and potentially offer better safety profiles for patients with comorbidities,' Sharma added.
The cardiologist also said that a more personalised approach to cholesterol management—driven by advanced pharmacological options—could be the next big step in preventive cardiology.
Verve has said the drug trial has been aimed at evaluating its result in patients with heterozygous familial hypercholesterolemia (HeFH) and/or premature coronary artery disease (CAD), two populations that require deep and durable reductions of bad cholesterol levels in the blood.
(Edited by Ajeet Tiwari)
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