Drug intelligence / Profile preview

RX-0201 + gemcitabine

Development stage
Unknown
Lead developer
Rexahn Pharmaceuticals
Modality
Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Molecules, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous
01

Overview

RX-0201 (also known as Archexin) is an antisense oligonucleotide designed to target and inhibit the expression of AKT1 by binding to its mRNA, thereby reducing downstream AKT signaling. It is being developed primarily for oncology indications, including metastatic renal cell carcinoma and pancreatic cancer. Gemcitabine is a nucleoside analog chemotherapeutic agent that acts as a prodrug; it is metabolized intracellularly into active diphosphate and triphosphate forms that inhibit ribonucleotide reductase and are incorporated into DNA, leading to inhibition of DNA synthesis and induction of apoptosis. The combination of RX-0201 with gemcitabine has been studied in clinical trials for metastatic pancreatic cancer[2][4][6].

Brand names
Gemzar
Other names
Archexingemcitabine
02

Targets

DNAAKT1 (Proto-oncogene serine/threonine-protein kinase Akt1)RRM1

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