Silence Therapeutics (SLN) Chardan Genetic Medicines Conference summary

Platform overview and accomplishments

• Proprietary siRNA technology platform (GOLD) targets hepatocytes using GalNAc ligands, enabling specific delivery and minimal off-target effects.

• Computational algorithms and a chemistry toolbox optimize sequence stability, potency, and durability.

• Transitioned from research to clinical stage, advancing multiple compounds into development in 2024.

Lead program: Zerlasiran for Lp(a)

• Zerlasiran targets Lp(a), a genetic, independent cardiovascular risk factor affecting 20-25% of the population.

• Phase I and II studies showed 96-99% Lp(a) reduction, with effects persisting up to 201 days.

• Phase III trial planned for the first half of next year, aiming to differentiate by targeting broader high-risk vascular populations and lower Lp(a) thresholds.

• Market opportunity estimated at $16 billion+, with room for multiple entrants.

Clinical data and competitive landscape

• Phase II data confirmed robust, durable Lp(a) suppression at 36 and 48 weeks; 60-week data and safety results to be presented at AHA.

• Outcome trials (CVOTs) are key for demonstrating clinical benefit; competitors (Novartis, Amgen) are at similar or slightly advanced stages.

• Differentiation strategies include broader patient inclusion and payer-focused trial design.