Drug intelligence / Profile preview

SPC4955

Development stage
Phase 1
Lead developer
Roche
Modality
Small Molecules, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Administration
Subcutaneous
01

Overview

SPC4955 is an investigational antisense oligonucleotide drug developed using the Locked Nucleic Acid (LNA) platform. It is designed to inhibit the synthesis of apolipoprotein B (apoB) mRNA, thereby reducing the production and export of low-density lipoprotein cholesterol (LDL-C) in the liver. By targeting apoB, SPC4955 aims to lower plasma levels of LDL-C and triglycerides, which are key risk factors for cardiovascular disease. The drug was advanced into Phase 1 clinical trials for subcutaneous administration in healthy volunteers to assess its safety, tolerability, pharmacokinetics, and pharmacodynamics. The primary indication under investigation was hypercholesterolemia.

02

Targets

APOB (Apolipoprotein B)

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