SLN-124

SLN-124

 

Update: 31 March 2020

  • In a corporate presentation, Silence Therapeutics has identified its 2019 accomplishments as:
  1. the initiation of a multi-centred, single blind, randomized Phase Ib clinical trial for assessing safety, tolerability, pharmacodynamics and pharmacokinetics of SLN-124 in patients with NTD β-thalassaemia or HbE/ β-thalassaemia
  2. receiving orphan drug designation by the EMA.
  • The first patient is expected to receive the treatment in Q1 of 2020 with interim results to be announced in the second half of the year.
  • The trial is separated into 2 parts – Part A: Single Ascending Dose; Part B: Multiple Dose
  • SLN-124 is administered subcutaneously

**Patients with β-thalassaemia are known to display hepcidin deficiency which in turn leads to iron overload**

  • SLN-124 aims to increase hepcidin thus improving anaemia, reducing ineffective erythropoiesis, reducing splenomegaly, reducing iron overload.
  • SLN-124 inhibits (stops) the expression of TMPRSS6 in the liver thus raising hepcidin levels.

Sources:

TIF Meeting with company representatives

 

Update: 31 January 2020

No update available

 

Update: 25 November 2019

No update available.

 

Update: 27 September 2019

No update available

 

Update: 30 July 2019

No update available.

 

Update: 30 May 2019

No update available.

 

Update: 29 March 2019

  • SLN124 has been submitted to the UK Medicines and Healthcare Products Regulatory Agency (MHRA).
  • Subject to approval from the MHRA, Silence intends to begin a Phase I/II First-in-Human study in ß -Thalassemia and MDS patients, with the first patient expected to enter the study in Q3 2019 (July – September).

Sources:

https://www.silence-therapeutics.com/media/1900/sln124-cta-announcement.pdf

 

Update: 01 February 2019

Silence Therapeutics is working on the development of therapeutic RNA molecules that use the body’s natural mechanism of RNAi to stop (or silence) targeted / specific genes in the genome from producing disease-causing proteins (i.e. gene silencing).

RNAi is the natural regulation of gene expression achieve by the body’s cells through a complex system of increasing or decreasing the production of specific gene products.

SLN124 has shown to reduce serum iron levels and balance tissue iron distribution in mice with β-thalassaemia.

Having been granted orphan drug designation by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA), Phase Ib trials are planned to begin in the second half of 2019. 

Sources:

https://www.silence-therapeutics.com/media/1874/190115-silence-sln124-orphan-drug-status-rns.pdf