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.
How’s it doing
Silence Therapeutics PLC (LON:SLN) has made a clinical trial application for its lead candidate, SLN124, which is being developed to treat iron overload disorders.
SLN124 already has orphan drug designation in Europe, which could shorten its timeline to market while providing the approved product with additional exclusivity.
Silence describes SLN124 as a “promising candidate medicine” for patients with iron overload disorders such as ß -Thalassemia, Myelodysplastic Syndrome and Hereditary Hemochromatosis.
A phase Ib, first-in-human trial is scheduled to start in the third quarter.
Another drug in Silence’s pipeline is SLN500, which will be used to treat rare diseases. Silence announced in July that it has agreed a partnership with US biopharmaceutical firm Mallinckrodt PLC (NYSE: MNK) for SLN500.
Silence will be responsible for taking SLN500 through pre-clinical studies and a phase I trial. After that, Mallinckrodt will take over clinical development and responsibility for global commercialisation.
Meanwhile, an investigational new drug/clinical trials application for SLN360, a treatment for a range of cardiovascular disease, is anticipated to be filed in the second half of 2020.
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What the boss says:
Chief executive David Horn Solomon said the partnership with Mallinckrodt is "charging ahead, with the milestone payment signalling that Silence and Mallinckrodt are advancing the programme rapidly".
"There'll be more milestones to come, but the space is hot."
Horn Solomon said Silence is "optimistic that Mallinckrodt will trigger that option" over its two more candidates in the deal, and that "brings in milestone payments and more assets to advance".
Elsewhere the clinical trial of SLN124 is now open, with first dosing of the first patient "now imminent", said Horn Solomon, with the first three cohorts to have reported results by mid-year 2020.
What the broker says:
Peel Hunt noted that big pharma money is flowing into RNAi therapies, with Roche agreeing in October to license Dicerna Pharmaceuticals hepatitis B therapy for $200mln upfront and $1.5bn in milestones, and Johnson & Johnson have invested in a rival treatment for the same disease.
“These RNAi deals signal a marked appetite for Pharma to partner early on RNAi assets, with sizeable upfront milestones,” said Peel Hunt.
The broker added that Mallinckrodt's upfront payment of $20mln, plus research and development funding of $10mln could spill over into potential milestones of $666mln.
With the deal including provision of rights to Mallinckrodt for two further assets in Silence’s pipeline, the broker said "Silence could net another $703 million in milestones for each asset”.