SLN124 is being developed for the treatment of iron overload disorders like ß -Thalassemia, Myelodysplastic syndrome (MDS) and Hereditary Hemochromatosis (HH).
The investigational candidate was demonstrated to reduce serum iron levels, regulate tissue iron distribution and ameliorate anemia in preclinical models for both ß-Thalassemia and HH.
Silence Therapeutics believes that the preclinical results represent a high potential of the drug for the treatment of patients with iron overload disorders.
The company’s CTA is for what will be the first-in-human trial of the investigational drug. If approved, Silence Therapeutics is looking to enroll the first patient in the study in the third quarter of this year, which will evaluate it for the treatment of ß -Thalassemia and MDS.
Silence Therapeutics CEO David Horn Solomon said: “This is a very positive step forward for Silence Therapeutics as we continue preparations for our return to the clinic. With robust data generated in several preclinical disease models, favorable safety profile and patient-friendly administration, we believe that SLN124 is well positioned against current standard of care and other medicines in development for the treatment of iron overload disorders.
“The filing of this CTA with the MHRA is another positive step towards progressing this promising candidate into the clinic in Q3 2019.”
According to Silence Therapeutics, SLN360 has been designed to silence apolipoprotein, a component of lipoprotein, which is said to be a highly validated target based on extensive human genetic data. Elevated levels of apolipoprotein have been associated with increased risk of cardiovascular disease, irrespective of additional risk factors.
Earlier this year, SLN124 was granted orphan drug designation in the European Union for the treatment of β-Thalassemia. The designation was granted by the Committee for Orphan Medicinal Products (COMP), a committee of the European Medicines Agency (EMA).
The orphan designation gives SLN124 the benefit of expedited clinical development in addition to ten years of market exclusivity, should it get approved by the European drug regulator.