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 it is 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.
Another drug in Silence’s pipeline is SLN500, which will be used to treat rare diseases. Silence is in 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.
The firm launched in February a scientific advisory board to provide governance for its research and development activities, a month after unveiling a deal with a Japanese pharma giant worth “single-digit million dollars” in research funding. It is also planning to create a US subsidiary.
What the boss says: Iain Ross, executive chairman
"The company has made substantive progress in 2019, cementing its reputation as a leading participant in the burgeoning field of RNAi therapeutics, securing a major deal with Mallinckrodt Pharmaceuticals."
"In 2020, Silence will look to continue leveraging its expertise to advance its pipeline of new medicines through the clinic to show safety, tolerability and efficacy for patients. In addition, we will continue to explore multiple opportunities to create shareholder value through significant partnerships that leverage the company's proprietary GalNAc-siRNA platform."
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”.