Silence Therapeutics Announces FDA Fast Track Designation for SLN124, a Novel Investigational siRNA Therapy for the Treatment of Polycythemia Vera

On September 8, 2022 Silence Therapeutics plc, Nasdaq: SLN ("Silence" or "the Company"), a leader in the discovery, development and delivery of novel short interfering ribonucleic acid (siRNA) therapeutics for the treatment of diseases with significant unmet medical need, reported that the U.S. Food and Drug Administration ("FDA") has granted Fast Track Designation to SLN124, a novel siRNA targeting the TMPRSS6 gene, for the treatment of polycythemia vera ("PV") (Press release, Silence Therapeutics, SEP 8, 2022, View Source [SID1234619247]). The Company plans to start a phase 1/2 study in PV patients this year.

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SLN124 is designed to address a range of hematological diseases by targeting TMPRSS6 to increase endogenous hepcidin, the body’s master regulator of iron balance and distribution. SLN124 demonstrated proof of mechanism and was well tolerated in a healthy volunteer study completed last year.

"The granting of Fast Track Designation represents an important recognition by the FDA of SLN124’s potential to address a significant unmet need in the treatment of PV," said Craig Tooman, President and Chief Executive Officer of Silence. "We plan to leverage Fast Track to expedite our development path as we seek to provide an important new treatment option to those suffering from this very serious and chronic genetic disease."

Fast Track designation aims to facilitate the development and accelerate the review of new therapeutics that are intended to treat serious or life-threatening conditions and that potentially address an unmet medical need. Drugs that are granted this designation are given the opportunity for more frequent interactions with the FDA, as well as potential pathways for expedited approval.

In addition to PV, SLN124 is being evaluated in a phase 1 study in patients with thalassemia. SLN124 has orphan disease designations for PV, thalassemia, and myelodysplastic syndrome as well as a rare pediatric disease designation for thalassemia.