CAMBRIDGE, Mass. - Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), a leader in RNAi therapeutics, reported positive results from the KARDIA-2 Phase 2 study of zilebesiran, an investigational RNAi therapeutic for hypertension. The study met its primary endpoint, demonstrating significant reductions in systolic blood pressure (SBP) when combined with standard antihypertensive medications.
Patients in the global, multicenter study, which enrolled 672 adults with mild-to-moderate hypertension, experienced clinically and statistically significant reductions in 24-hour mean SBP at month three. These results were consistent across three cohorts, each receiving a different standardized background therapy, including a diuretic, calcium channel blocker, or angiotensin receptor blocker.
Zilebesiran, which targets angiotensinogen in the liver, also exhibited a favorable safety and tolerability profile. The data suggests the potential for biannual dosing, which could offer a more convenient treatment regimen for patients.
Simon Fox, Ph.D., Vice President and Zilebesiran Program Lead at Alnylam, expressed confidence in the drug's differentiated profile and its ability to provide additional blood pressure control. The full study results are set to be presented at the American College of Cardiology Annual Scientific Session on April 7, 2024.
In parallel, Alnylam and Roche have initiated the KARDIA-3 Phase 2 study to evaluate zilebesiran in patients at high cardiovascular risk with uncontrolled hypertension, despite existing treatments. This study will further explore the efficacy and safety of zilebesiran as an add-on therapy.
Zilebesiran utilizes Alnylam's Enhanced Stabilization Chemistry Plus (ESC+) GalNAc-conjugate technology, designed for infrequent subcutaneous dosing. The investigational drug is part of a new class of RNAi therapeutics, which silence gene expression and have the potential to transform treatment for diseases with unmet medical needs. However, the safety and efficacy of zilebesiran have not yet been established by regulatory authorities.
These developments come as hypertension remains a significant global health challenge, affecting over one billion people worldwide. Despite available treatments, control rates are suboptimal, highlighting the need for innovative therapeutic options like zilebesiran.
The information reported is based on a press release statement from Alnylam Pharmaceuticals.
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