Silence Therapeutics (SLN) H.C. Wainwright 26th Annual Global Investment Conference 2024 summary

Platform and pipeline overview

• Focused on siRNA therapeutics targeting genetic diseases, primarily in the liver over the past 7–8 years.

• Lead program zerlasiran targets Lp(a) for cardiovascular disease, recently completed phase 2.

• Divasiran program targets TMPRSS6 for polycythemia vera, completed phase 1.

• Significant productivity and program advancement over the past year.

Zerlasiran (Lp(a) program) clinical progress

• Lp(a) is a genetically determined, causal cardiovascular risk factor with no approved therapies; affects 1 in 5 globally.

• Phase 2 ALPACAR-360 trial showed highly significant, durable Lp(a) reduction (≥90% median knockdown) and strong safety profile through 48 weeks.

• Additional LDL-C and ApoB lowering observed, even in patients on standard of care.

• Next data readout at 60 weeks will include further durability, safety, and efficacy.

• Planning for phase 3 and cardiovascular outcomes trial, with regulatory discussions later this year.

Market and competitive landscape

• Lp(a) market seen as a multi-billion-dollar global opportunity with room for multiple players.

• Infrequent dosing and potent, long-acting siRNA profile seen as key differentiators.

• Upcoming ASO CVOT data expected to validate Lp(a) lowering as a clinical benefit, supporting broader market confidence.