Strong pipeline of pre-clinical drug candidates
Moving into the clinical trial phase later this year
Patent dispute resolution provides small royalty on rival's drug sales
What does Silence do?
The name provides a hint. It is a specialist in the field of gene silencing. Its technology uses RNA interference, a biological process in which RNA molecules inhibit gene expression or translation, by neutralising targeted molecules.
The company is developing drugs for blood disorders, metabolic diseases and curbing alcohol use. The pipeline as it stands is pre-clinical. It expects to kick off its first-in-human trial in the second half of this year on SLN124. Silence describes it as a “highly promising” treatment for genetic conditions such as β-Thalassemia, a rare blood disorder.
In January it was granted orphan drug designation for SLN124. The European Medicines Agency has concluded it will be of “significant benefit” to people affected by β-Thalassemia.
Orphan drug status gives drug makers – in this case, Silence – certain benefits in order to encourage them to develop treatments for rare diseases which might otherwise lack sufficient profit motive.
For example, it can be easier to gain marketing approval for that drug or extend the exclusivity periods, while there are often also financial advantages such as tax breaks and R&D grants.
Without these incentives, companies might not pursue a drug’s development because the R&D costs would be too expensive.
The next drug in Silence’s pipeline is SLN226, which has shown potential to aid abstinence in people with alcohol dependency. It is in preclinical development at the moment, with plans in place for it to enter clinical trials in 2020.
After a long-running wrangle with an American company Alnylam Pharmaceuticals, which was claimed have ‘borrowed’ some of Silence’s intellectual property, a settlement and licence agreement was brokered. Silence will receive a small tiered royalty on net sales of the US biotech group’s ONPATTRO drug in the EU.
What the broker says
“We see today’s [Alnylam] news as further validation of the competence that Silence has in RNAi therapeutics,” said Peel Hunt recently.