Lepodisiran Achieves Sustained Lp(a) Reduction in Trial

A single dose of lepodisiran, an investigational RNA interference therapy, safely reduced average blood levels of lipoprotein(a) [Lp(a)] by 94% over 180 days in a Phase 2 study. Lp(a) is a key genetic risk factor for cardiovascular disease affecting over one billion people worldwide. The durable decrease observed signals potential for significant CV event risk reduction if confirmed in ongoing large-scale trials.

The findings were presented at the American College of Cardiology’s annual scientific session in Chicago. Lepodisiran’s biosafety, combined with its potent pharmacologic activity, make it a promising candidate to fill an unmet need in managing atherosclerotic cardiovascular disease.

Spinogenix Initiates Phase 2 Trial of SPG302 for Schizophrenia

Spinogenix, a clinical-stage biopharmaceutical company, announced open enrollment for its US Phase 2 clinical trial of SPG302, the first synaptic regenerative therapy for schizophrenia. SPG302 is designed to restore glutamatergic synapses in brain regions affected by schizophrenia, a novel therapeutic mechanism aiming to improve symptoms not addressed by existing treatments.

The trial will evaluate efficacy, safety, and tolerability of once-daily dosing, building on early Australian study results. With approximately 24 million people affected globally, Spinogenix’s approach offers hope for improved functional outcomes and quality of life.

Dual Advancements in Cardiovascular and Neuropsychiatric Therapies

These developments highlight the pace of innovation in RNAi and regenerative medicine technologies addressing chronic and disabling diseases. Lepodisiran targets a widespread but poorly managed cardiovascular risk, while Spinogenix moves toward potentially transformative schizophrenia therapies.

Outlook: Clinical Milestones and Broader Impact

As lepodisiran progresses through larger Phase 3 trials and Spinogenix advances with regulatory clearance, both companies are positioned to change treatment paradigms in their respective fields. Industry stakeholders focus on upcoming efficacy readouts and expanded patient access strategies.