Silence Therapeutics (SLN) H.C. Wainwright 28th Annual Global Investment Conference summary

Platform overview and differentiation

• Proprietary siRNA technology uses unique chemistry modifications and algorithms for potency, specificity, and safety.

• Transitioned from research to clinical stage over the past 5–6 years, with three products entering the market.

• IP covers linkers and GalNAc technology for liver targeting.

Divesiran in polycythemia vera (PV)

• Targets TMPRSS6 to increase hepcidin, restricting iron and reducing red blood cell production.

• Phase II SANRECO trial showed 88% of treated patients maintained hematocrit below 45% without phlebotomy, versus 19% on placebo.

• Both Q6-week and Q12-week dosing intervals were robustly effective and well tolerated.

• Safety profile showed no major issues; injection site reactions were low and manageable.

Upcoming milestones and regulatory plans

• Full phase II data, including symptom and quality of life scores, to be presented at ASH in December.

• End of phase II meeting with FDA planned by year-end to confirm dose, interval, and trial size for phase III.

• Phase III trial design aims for efficiency, possibly fewer than 250 patients, focusing on both efficacy and symptom outcomes.