Silence Therapeutics plc (LON:SLN) remains confident that it will kick off its first human clinical trial by the end of the year.
The AIM-quoted company specialises in RNA interference – an experimental type of treatment which aims to treat diseases by suppressing, or ‘silencing’, genes that are thought to cause diseases.
Silence has recruited five senior managers to spearhead the clinical drive.
This is in anticipation of filing Silence's first GalNAc-siRNA CTA (clinical trial authorisation) for the treatment of liver-related iron overload disorders by the end of 2018.
Animal disease models have reinforced that its lead candidate, SLN124, can be effective in the treatment of the genetic conditions.
Data presented over the weekend seemed to back this up, with Silence’s drug discovery project leader Dr Ute Schaeper describing the drug as a “promising candidate”.
A second programme in alcohol use disorder is set for a CTA filing mid-2019, potentially through a partnership.
On lookout for new CEO
It will be doing this without its long-serving chief executive Ali Mortazavi who stepped down from his role earlier this month (June).
Silence’s chair, Annalisa Jenkins, is filling the position temporarily until a permanent successor is found. Her job, and that of the new boss, will be to build on Mortazavi’s work.
In particular, they will be tasked with defending the company’s intellectual property.
Silence believes that a number of companies, including US giant Alnylam Pharmaceuticals Inc (NASDAQ:ALNY), have been using technology covered by its patents in drugs that silence genes and may be fundamental in the war against cancer.
The firm has, over the past year, sought to shore up the patent protection around what it calls a “foundational innovation” that is core to the emerging RNAi industry.
“Silence believes that these newly granted claims are relevant to third-party RNAi medicines and that its foundational IP is essential for the clinical and commercial viability of the RNAi field.”
Silence revealed earlier this year that litigation with Alnylam is “progressing” and it expects a trial to start in the UK at the beginning of December.
Plenty of cash…
At the end of 2017, the company had £43mln of cash in the bank compared to £30mln this time last year.
One of the primary reasons for the jump in the net cash balance was Silence’s investment in Arrowhead Pharmaceuticals Inc (NASDAQ:ARWR).
At the end of 2016, Silence spent £8.3mln (US$11.3mln) to take a 10% stake in Arrowhead with the view to some form of strategic transaction.
By March 2017, Silence said it had become clear that such a transaction would not be possible, so it started to close its position in Arrowhead.
The AIM-quoted group has now completed the sale of its entire stake, generating net sale proceeds of US$24.7mln, a net profit of US$13.4mln.
Some of that cash will be used to fund the aforementioned clinical trials, while it also wants to invest in developing a “deep pre-clinical pipeline”.
“Well-positioned” for 2018
“Silence [has] demonstrated that it is now has a powerful, reproducible and modular GalNAc-siRNA platform that can rapidly and safely be used to specifically and effectively silence any disease-associated target gene in hepatocytes,” said now ex-CEO Ali Mortazavi.
“With £43mln of cash at the start of 2018, Silence is well positioned to further develop its existing pipeline, as well as adding significant new targets, some incorporating next generation technology.
“At the same time, we are actively looking to partner our platform and programmes, and to enter into both commercial and academic collaborations, strengthening our balance sheet and widening our portfolio.”