Silence Therapeutics (SLN) Piper Sandler 36th Annual Healthcare Conference summary

Company overview and strategy

• Operates globally with offices in the UK, US, and Germany, employing about 125 people.

• Focuses on siRNA therapeutics, leveraging decades of expertise and intellectual property.

• Utilizes a hybrid model combining proprietary programs and partnerships for non-dilutive funding.

• Maintains collaborations with major pharma companies, including AstraZeneca.

• Portfolio approach guides funding allocation and potential partnering decisions.

Zerlasiran (Lp(a) siRNA program)

• Targets Lp(a), a genetic cardiovascular risk factor affecting 20% of the global population.

• Achieves over 90% maximum knockdown of Lp(a) in phase 1 and 2 studies, with durable effects.

• Dosing intervals range from quarterly to potentially every 24 weeks, with a 300 mg single injection under consideration.

• Phase 2 ALPACAR-360 study showed 81–86% time-averaged Lp(a) reduction at longer intervals.

• Phase 3 trial will enroll 6,000–8,000 patients, focusing on secondary prevention and broader high-risk vascular populations.

Competitive landscape and trial design

• Zerlasiran offers longer duration and greater knockdown compared to antisense oligonucleotides.

• Differentiation in phase 3 design includes broader patient inclusion and endpoints beyond MACE.

• Trial design informed by payer and HTA input to support future reimbursement.

• Ongoing cardiovascular outcomes trials by competitors may inform but not alter the planned phase 3 design.