Silence Therapeutics PLC (LON:SLN) expects to kick off its first-in-human clinical trial in the second half of next year.
The AIM-quoted company specialises in RNA interference – a type of treatment which works by suppressing, or ‘silencing’, genes that are thought to cause diseases.
Until recently, this type of treatment had been experimental, but it has come on leaps and bounds of late, with US regulators approving the first RNAi drug last month.
SLN124 is the lead candidate
Silence is working on a clinical trial authorisation filing for SLN124 – a “highly promising” treatment for genetic conditions such as beta thalassemia, a rare blood disorder – which it has previously said it hoped to have concluded by the end of the year.
Once that is in hand, the plan is to apply for orphan drug designation, which would expedite progress towards a phase Ib trial, slated to begin in the second half of next year.
The next drug in Silence’s pipeline is SLN226 which has shown potential to aid abstinence in people with alcohol dependency.
SLN226 is in preclinical development at the moment, with plans in place for it to enter in-human clinical trials in 2020.
Reflecting the costs associated with progressing its two lead programmes towards the clinic, the company reported a loss before tax of £8.7mln (H1 17: £5.5mln) in the six months ended June 30.
Silence has also out-licensed its AtuRNAi technology to Quark Pharmaceuticals, which has two drugs in late-stage clinical trials using this technology.
In one of the phase III studies, Quark does its first patient in July, while results from the other are due later this year.
All of this will be overseen by a new management team, headed up by industry veteran David Horn Solomon, who replaced long-time chief executive Ali Mortazavi earlier this summer.
Chair Annalisa Jenkins stepped down last month with Andy Richards filling in for her temporarily while a permanent successor is found.
with the group’s move towards in-human studies, Richard Jenkins was brought in as head of clinical development.
Silence taking US drugs giant to court over IP infringement
Aside from developing Silence’s pipeline, Horn Solomon and co will be tasked with continuing to defend the company’s intellectual property.
Silence believes that a number of companies have been using technology covered by its patents on drugs that silence genes and may be fundamental in the war against cancer.
The company 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.”
US giant Alnylam – the company behind patisiran, the drug approved by the US Food and Drug Administration last month – is one of those Silence has accused and a trial is expected to start in the UK in December.
At the end of June, Silence had £34.3mln in the bank, leaving it with plenty of cash to fund the aforementioned clinical trials, invest in its pipeline and defend its IP where it deems necessary.
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.
“The RNAi space is maturing with the first marketing approval of patisiran from Alnylam,” said new chief executive David Horn Solomon.
“I am excited for the opportunity to build Silence into a leading drug development company in this cutting-edge field of technology”
He added: “In the coming months, we will apply for Orphan Drug designation for our clinically validated lead medicine candidate, SLN124, for the treatment of Beta-Thalassemia, in order to expedite progress towards a Phase Ib trial planned to begin in H2 2019.
“Whilst also advancing the development of our other pipeline of product candidates, we are targeting validating Business Development deals and continue to explore a range of financing options.”
Interim chair Andy Richards said it has been a “transformational period” for the company.