Silence Therapeutics submits clinical trial application to MHRA

Silence Therapeutics announced on Wednesday that a clinical trial application for its lead candidate SLN124 has been submitted to the UK Medicines and Healthcare products Regulatory Agency (MHRA).

The AIM-traded firm said SLN124, which had been shown to lower serum iron levels, modulate tissue iron distribution and ameliorate anemia in preclinical models for ß-Thalassemia and hereditary hemochromatosis (HH), represented a “highly promising” therapeutic candidate medicine for patients with iron overload disorders, such as ß -Thalassemia, myelodysplastic syndrome (MDS) and HH.

Subject to approval from the MHRA, Silence said it intended to begin a phase 1b first-in-human study in ß -Thalassemia and MDS patients, with the first patient expected to enter the study in the third quarter of 2019.

In January, SLN124 was granted orphan drug designation by the Committee for Orphan Medicinal Products (COMP) - the European Medicines Agency (EMA) committee responsible for recommending orphan designations of medicines for rare diseases.

Receipt of orphan drug designation meant that SLN124 was eligible for expedited clinical development, and 10 years of market exclusivity, subject to approval.

“This is a very positive step forward for Silence Therapeutics as we continue preparations for our return to the clinic,” said Silence Therapeutics chief executive officer Dr David Horn Solomon.

“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 the third quarter of 2019.”