Silence Therapeutics (SLN) 2024 Cantor Fitzgerald Global Healthcare Conference summary

Strategic overview and technology platform

• Focus on precision-engineered siRNA medicines with proprietary technology, demonstrating strong efficacy, safety, and durability in clinical trials.

• Advancing both wholly owned and partnered clinical programs, with a global presence and operations in London, Berlin, and New Jersey.

• Platform has broad utility, initially focused on liver targets but expanding beyond, with over a dozen partnered programs and up to $5 billion in potential milestones.

• Financially stable with a cash runway into 2026 and recent analyst coverage upgrades.

• Partnerships with AstraZeneca and Hansoh provide significant non-dilutive capital and global reach.

Clinical program highlights

• Zerlasiran targets Lp(a), a major genetic risk factor for cardiovascular disease affecting up to 25% of the global population.

• Phase I data showed up to 99% reduction in Lp(a) with high durability and no safety issues; phase II met primary endpoint with >90% reduction, moving to phase III in the first half of next year.

• Divesiran, for polycythemia vera, demonstrated strong efficacy in phase I, with significant reduction in phlebotomy needs and hematocrit control.

• Divesiran is first-in-class siRNA for PV, with fast-track and orphan drug designations, and phase II initiation planned by year-end.

• Both programs are supported by robust clinical data and are positioned for further advancement.

Market and industry context

• Lp(a) is increasingly recognized as a key cardiovascular risk factor, with growing awareness and recommendations for lifetime testing by major societies.

• Zerlasiran addresses a large, underserved market with no current approved therapies to lower Lp(a).

• Polycythemia vera represents a significant rare disease market, with current treatments often leading to iron deficiency and high disease burden.

• Partnerships and collaborations are expanding, with increasing interest from industry leaders following positive clinical results.

• The siRNA platform is considered revolutionary by key opinion leaders, with ongoing innovation and pipeline expansion.