Doer Biologics' First-in-Class Triple Agonist DR10624 Granted Breakthrough Therapy Designation by CDE for Severe Hypertriglyceridemia

Severe Hypertriglyceridemia (sHTG) generally refers to a condition where triglycerides (TG) exceed 500 mg/dL (5.7 mmol/L), with a few patients even surpassing 880 mg/dL (10 mmol/L). Epidemiological data shows that the incidence of hypertriglyceridemia is increasing year by year, with the global prevalence of HTG in adults being approximately 10%, and the prevalence of sHTG in adults being about 1%. sHTG significantly increases the risk of developing atherosclerotic cardiovascular disease (ASCVD) and acute pancreatitis (AP), and may lead to recurrent episodes in patients, resulting in poor prognosis and potentially life-threatening conditions. Additionally, sHTG is associated with chronic diseases such as cardiovascular disease, chronic kidney disease, and fatty liver. Traditional lipid-lowering drugs, such as statins, fibrates, fish oil, and niacin, have limited efficacy in reducing TG levels and typically cannot lower TG concentration below 500 mg/dL. Moreover, long-term use of statins and fibrates carries various safety risks, such as elevated liver enzymes or renal impairment.

DR10624 is a globally pioneering long-acting trispecific agonist independently developed by Doer Biologics, targeting FGF21R, GCGR, and GLP-1R. It was approved by China's CDE in October 2023 and the U.S. FDA in October 2025 to initiate clinical research for sHTG. In September 2025, the results of DR10624’s Phase II clinical study (“DR10624-201 Study”) for treating severe hypertriglyceridemia were successfully selected for the Late-Breaking Science session at the AHA Scientific Sessions 2025 and listed as a Groundbreaking Trial in Cardiometabolic Therapeutics. In November 2025, the results of the DR10624-201 study were presented as the opening report at AHA 2025, with the following key data: The 12-week treatment group showed a 74.5% reduction in TG in the 12.5mg dose group, a 66.2% reduction in the 25mg dose group, and a 68.9% reduction in the 50mg titration dose group. Among the subjects in the trial drug treatment group, 78.5% experienced a TG reduction of over 50%, and 89.5% achieved TG levels below 500 mg/dL. Additionally, DR10624 demonstrated excellent efficacy in improving atherogenic lipid profiles. Compared to placebo, all dose groups of DR10624 significantly reduced total cholesterol, non-HDL cholesterol, triglyceride-rich lipoprotein cholesterol, very low-density lipoprotein cholesterol, and apolipoprotein C3. Simultaneously, DR10624 significantly increased high-density lipoprotein cholesterol, which is beneficial for cardiovascular health. DR10624 also exhibited strong efficacy in reducing liver fat: compared to the placebo group, liver fat content in all dose groups of DR10624 was significantly reduced, with a median percentage reduction reaching up to 67%. All dose groups of DR10624 demonstrated good safety and tolerability in this study, with adverse reactions primarily being mild to moderate in severity.

Clinical study data from the DR10624-201 study show that DR10624, with its innovative triple-target synergistic mechanism, demonstrates potent lipid-lowering effects and good safety. In addition to reducing TG, DR10624 also exhibits pharmacodynamic activity in lowering liver fat and comprehensively regulating metabolism, which are important clinical advantages and application values of DR10624. It is expected to provide a new and better treatment option for patients with sHTG in the future.

About Doer Biologics