Silence Therapeutics (SLN) H.C. Wainwright 4th Annual BioConnect Investor Conference summary

Pipeline overview and value drivers

• mRNAi GOLD platform enables targeted, potent, and long-acting siRNA medicines for cardiometabolic and hematological diseases.

• Key clinical assets include divesiran (polycythemia vera), zerlasiran (Lp(a) lowering), and SLN312 (dyslipidemia), with additional preclinical programs in hypercholesterolemia and obesity.

• Phase II SANRECO data for divesiran and phase III readiness for zerlasiran are major milestones expected in the next year.

• Proprietary siRNA chemistry and openness to partnerships support differentiation and development.

RNAi technology advantages

• siRNA enables gene silencing of previously undruggable targets with high potency and long duration.

• Dosing can be infrequent (every three months or longer), improving patient convenience.

• RNAi medicines offer reversibility and precision, expanding therapeutic options.

Divesiran for polycythemia vera

• Targets TMPRSS6 to increase hepcidin, reducing iron availability and controlling hematocrit.

• Phase I data show durable hematocrit control below 45% and favorable safety up to 9 mg/kg.

• Phase II SANRECO trial is fully enrolled, with top-line data expected August 2026; primary endpoint is hematocrit control without phlebotomy.

• Both 6- and 12-week dosing arms are being evaluated, with six-week dosing already seen as commercially compelling.

• Positioned for first-line use in phlebotomy-dependent and cytoreductive-naive PV patients.