Silence Therapeutics PLC (LON:SLN, NASDAQ:SLN) has reported positive safety data from a phase I trial of its lead asset.
SLN124, which is being developed to treat iron-loading anaemia conditions such as thalassemia and myelodysplastic syndrome (MDS), was also shown to be effective in reducing plasma iron levels and had a long duration of action.
The data will support an ongoing phase I study of the drug’s effects on people with thalassemia and MDS, Silence said.
The GEMINI phase I evaluation assessed 24 healthy volunteers, with 18 receiving the drug.
The readout, which will be gone through in greater depth at the “appropriate scientific meeting later this year, is the first for a drug developed using the company’s mRNAi GOLD platform. Data from two more clinical assessments are expected later this year.
Chief executive Mark Rothera said the top-line findings underscored “the promising potential for our technology to deliver precision medicines”.
While the aim of a phase I trial is to check for safety and assess how the drug interacts with the body, the Silence team will have been buoyed by the potential therapeutic impact of SLN124.
Researchers observed an approximately four-fold increase in average hepcidin, a key regulator of iron in the body, and a 50% reduction in plasma iron levels. This was after a single dose.
The effects on hepcidin and iron appeared to be dose-dependent and were still observed at the end of the eight-week study at all dose levels, “indicating a sustained and long duration of action”, Silence said.
“We are encouraged by these data in healthy volunteers and the opportunity for SLN124 to potentially address iron-loading anaemia conditions such as thalassemia and MDS,” said Giles Campion, Silence’s head of research and development.
SLN124 has orphan drug designation for both conditions and a rare paediatric disease designation for beta-thalassemia, which could shorten the development timeline.