Silence Therapeutics (SLN) 12th Annual Cantor Fitzgerald Global Healthcare Conference summary

Company overview and pipeline priorities

• Focused on siRNA technology with over 20 years of development, emphasizing liver-targeted therapies using the proprietary GOLD platform.

• Three programs have advanced to clinical development: divesiran (lead, just completed phase II), zerlasiran (phase III-ready for cardiovascular), and ANGPTL3 (recent phase I data).

• Recently raised $200 million to accelerate clinical advancement, especially for divesiran.

• Key priority is rapid progression of divesiran into phase III trials.

Technology differentiation and intellectual property

• Utilizes proprietary algorithms for potent and durable siRNA sequence selection.

• Employs a unique chemistry toolbox and proprietary linkers, enhancing IP protection.

• Distinctive GalNAc conjugation methods differentiate from competitors.

Divesiran mechanism and clinical data highlights

• Targets TMPRSS6 to increase hepcidin, restricting iron to bone marrow and reducing RBC production in polycythemia vera (PV).

• Phase II data showed about 90% response rate, with placebo-adjusted effect just under 70%.

• Both 6-week and 12-week dosing intervals demonstrated robust efficacy.

• Durable pharmacodynamic effect allows for infrequent dosing, potentially quarterly.