On September 8, 2020 Silence Therapeutics plc, AIM:SLN and NASDAQ: SLN ("Silence" or "the Company") a leader in the discovery, development and delivery of novel short interfering ribonucleic acid (siRNA) therapeutics for the treatment of diseases with significant unmet medical need, reported that provides a research and development update (Press release, Silence Therapeutics, SEP 8, 2020, View Source [SID1234568596]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Pipeline progress

SLN360

·Silence’s wholly owned lead product candidate SLN360 for the treatment of cardiovascular disease associated with high Lipoprotein(a), or Lp(a), levels, has received approval from the Food and Drug Administration (FDA) for an Investigational New Drug (IND) application to start dose escalation studies in healthy volunteers and secondary prevention patients with elevated Lp(a). SLN360 is designed to address increased cardiovascular risk associated with raised levels of Lp(a), which is considered to affect up to 10% of the world’s population. Whilst we are aiming to start dosing healthy volunteers in the Phase 1 trial by the end of the year, the Covid-19 pandemic may put this timing at risk.

SLN124

Silence has initiated dosing in its randomised, double-blind, placebo-controlled Phase 1 single-ascending dose study of SLN124, the Company’s wholly owned product candidate for beta-thalassaemia and myelodysplastic syndrome (MDS), in up to 24 healthy volunteers. This marks the first dose of a Silence siRNA therapeutic delivered to humans using the Company’s proprietary GalNAc-siRNA platform. GalNAc is a naturally occurring sugar that binds specifically to a receptor which is highly expressed on liver cells, or hepatocytes. Coupling GalNAc sugars to stabilized siRNA molecules allows them to specifically target liver cells and carry out their function.
The FDA recently granted SLN124 orphan drug designation (ODD) for the treatment of adult beta-thalassemia. The FDA previously granted SLN124 ODD for MDS and rare paediatric designation for beta-thalassaemia. SLN124 also has ODD for the treatment of beta-thalassaemia from the European Medical Agency.
Complement Collaboration

·Silence is pleased to announce work commenced on the second target being explored under its complement pathway RNAi collaboration with Mallinckrodt, which triggered a $2.0 million research milestone payment to the Company. Silence would be entitled to receive an additional $2.0 million research milestone payment if work commences on a third target in the collaboration.

Iain Ross, Executive Chairman of Silence Therapeutics, said: "In what has been a busy and successful year for Silence, we have continued to make significant progress with our R&D pipeline. We are delighted to announce today that we have received FDA approval of the IND application for our wholly owned lead product candidate, SLN360, which is designed to address a significant unmet medical need in cardiovascular disease for people born with high Lp(a) levels. We have also made strong progress with our wholly owned product candidate, SLN124, having now dosed the first healthy volunteer. This marks the first dose of a Silence GalNAc-siRNA given to humans and is the culmination of several years’ work in developing this platform. This is truly an exciting time for Silence and we look forward to providing further updates as we move ahead."