Silence Therapeutics (SLN) Leerink’s Global Healthcare Conference 2025 summary

Strategic positioning and technology

• Utilizes proprietary siRNA technology with GalNAc oligonucleotide delivery for gene silencing in disease treatment.

• Maintains a strong financial position with cash runway projected into 2027.

• Engaged in multiple clinical programs, including a phase III-ready cardiovascular asset and a first-in-class siRNA for polycythemia vera (PV).

• Active partnership with AstraZeneca, progressing from phase I with potential milestones ahead.

• Ongoing dialogues for additional partnerships to support late-stage trials.

Clinical pipeline and recent results

• Zerlasiran (SLN360) for Lp(a) is phase III-ready, targeting a large, underserved market.

• Divesiran (SLN124) for PV has shown strong efficacy, safety, and durability in phase I, with phase II enrollment underway and full enrollment expected by year-end.

• SLN548 targets complement-mediated diseases, with a phase I healthy volunteer study set to begin in the second half of the year.

• Multiple undisclosed preclinical programs are advancing toward clinical development.

• Positive regulatory feedback received globally for phase III CVOT design.

Polycythemia vera (PV) program insights

• PV is a rare blood cancer with significant unmet needs, affecting 150,000 in the US and 3.5 million globally.

• Divesiran aims to control hematocrit below 45%, reducing cardiovascular and thrombotic risks.

• Phase I showed a dramatic reduction in phlebotomies and strong hematocrit control, with no dose-limiting toxicities.

• Phase II focuses on well-controlled patients, aiming to sustain hematocrit control and minimize phlebotomy needs.

• Fast-track and orphan drug designations granted for divesiran in PV.