Silence Therapeutics (NasdaqGM:SLN) FY Conference Transcript

Summary of Silence Therapeutics FY Conference Call Company Overview - Silence Therapeutics is focused on siRNA technology and has developed a strong intellectual property portfolio over two decades [2][3] - The company has a global presence with labs in Berlin, the UK, and a recently opened office in the US [2] - Silence Therapeutics has partnerships, notably with AstraZeneca, to explore various therapeutic areas [2][3] Core Technology and Advantages - The mRNAi GOLD™ platform offers advantages over traditional small molecules and antibodies, including efficacy, safety, durability, and reversibility [4][5] - The technology targets messenger RNA to silence disease-associated proteins, demonstrating robustness and specificity [6] Clinical Programs Polycythemia Vera (PV) - PV is a rare blood cancer characterized by the overproduction of red blood cells, leading to significant unmet medical needs [9] - Approximately 150,000 patients in the U.S. and 3.5 million worldwide are affected, with many being undertreated [10] - Current management involves phlebotomy and cytoreductive agents, with Silence Therapeutics being first in class in the siRNA arena for this condition [11] - Phase 1 trial results showed nearly 100% efficacy in well-controlled patients, with significant reductions in hematocrit levels [11][15] - The ongoing Phase 2 study aims to maintain control of hematocrit levels while reducing the need for phlebotomies, with enrollment expected to complete by year-end 2025 [18][19] Lp(a) Program - Silence Therapeutics is preparing for a Phase 3 trial for Lp(a), a condition affecting up to 20% of the global population, with no approved treatments currently available [38][39] - Phase 1 results demonstrated over 90% reduction in Lp(a) levels, with durable effects observed [41][42] - The company is seeking partners for the large-scale cardiovascular outcome trial (CVOT) due to the significant investment required [38] Competitive Landscape - The siRNA technology is positioned competitively against other treatments, such as antisense oligonucleotides and PCSK9 inhibitors, which have shown modest effects [52][55] - Silence Therapeutics emphasizes the potency and durability of its siRNA compounds, aiming for a less frequent dosing schedule compared to competitors [36][52] Collaboration with AstraZeneca - Silence Therapeutics has a multi-target collaboration with AstraZeneca, focusing on cardiovascular, metabolic, respiratory, and renal areas [56][59] - The collaboration allows for up to 10 targets to be explored, with AstraZeneca handling clinical development [56] Financial Position - The company reported $114 million in cash at the end of Q2 2025, providing a runway into 2028 [67] - This financial position supports ongoing clinical trials and operational needs [67] Macro Considerations - The rise of biotech innovation in China is acknowledged as a long-term competitive factor [68] - Silence Therapeutics is leveraging artificial intelligence and machine learning in its discovery and development processes to enhance efficiency and effectiveness [69][70] Future Focus Areas - The primary focus will be on the PV trial and Lp(a) program, with continued collaboration with AstraZeneca [83] - The company aims to manage costs while exploring additional pipeline opportunities [83]