Sirius Therapeutics announces U.S. FDA Clearance of IND to Begin Clinical Trial of Novel siRNA Therapy for Hyperlipoproteinemia(a)

San Diego and Shanghai, April.9th, 2025 – Sirius Therapeutics today announced that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application to begin clinical trial of SRSD216, a novel small interfering RNA (siRNA) therapeutic for hyperlipoproteinemia(a), or elevated levels of lipoprotein(a). This milestone marks a significant step towards addressing the unmet medical needs of patients suffering from atherosclerotic cardiovascular disease (ASCVD).

Numerous genetic and epidemiologic studies have demonstrated an association between elevated levels of lipoprotein(a) and the occurrence of ASCVD. The exceptional durability of SRSD216's pharmacodynamic effects and benign safety profile in preclinical studies suggest that this novel siRNA could represent a substantial step forward in the prevention and management of ASCVD.

Dr. Curt Bradshaw, Sirius’ Chief Scientific Officer stated, “SRSD216 emerged from our platform for the discovery of siRNA therapeutics, and has the potential for a dosing interval of six months or greater to treat ASCVD. Underscoring our platform’s potential, SRSD216 is the third Sirius-discovered siRNA therapeutic to enter clinical development since the company was founded in 2021.”

Dr. Qunsheng Ji, Sirius’ Chief Executive Officer commented, "We are pursuing development of SRSD216 globally and the upcoming U.S Phase 1 clinical trial based on this IND is an important part of that effort. Our team has worked diligently to achieve this milestone as part of Sirius’ broader strategy to develop innovative treatments for cardiometabolic diseases.”

About ASCVD and hyperlipoproteinemia(a)

Atherosclerotic cardiovascular disease (ASCVD) is the most common cause of death worldwide. Dyslipidemia is regarded as a key contributor to the development of ASCVD. While low-density lipoprotein cholesterol (LDL-c) has been considered the primary lipid indicator and target for intervention for decades, numerous studies have identified lipoprotein(a) [Lp(a)] as an independent risk factor for ASCVD. Moreover, Lp(a) is not modified by age, diet, or exercise and currently available pharmacotherapies for dyslipidemia have only modest effects on Lp(a), highlighting a significant clinical unmet need for agents that directly target Lp(a).

About SRSD216

SRSD216 is a novel double-stranded small interfering ribonucleic acid (siRNA) that specifically modulates the LPA gene, decreasing hepatic Apo(a) production and reducing circulating Lp(a) levels. Preclinical in vivo studies have demonstrated a near 100% reduction in Lp(a) levels over 6 months, with no meaningful safety findings reported after a single subcutaneous dose.

About Sirius Therapeutics

Sirius is a clinical stage biotech company developing innovative siRNA therapies for global markets. We are dedicated to discovering and developing new treatment options for cardiovascular and cerebrovascular disease and translating siRNA technology into transformative medicine for chronic disease patients. Our most advanced products are SRSD107 for the treatment of thromboembolic disorders, and SRSD101 for the treatment of dyslipidemia.

Founded in 2021 by a world-class leadership team and investors, Sirius has established an innovation center in the United States and translational medicine center in China. Sirius has raised nearly US$100 million funding to date from OrbiMed, Creacion Ventures, Hankang Capital, and Delos Capital. Learn more at www.siriusrna.com