Drug intelligence / Profile preview

ono-1301

Development stage
Unknown
Lead developer
Ono Pharmaceutical
Modality
Small Molecules
Administration
Oral, Intravenous, Local (e.g., Epicardial Placement Or Targeted Delivery With Nanoparticle Systems)
01

Overview

ONO-1301 is a **novel, synthetic, long-acting prostacyclin (prostaglandin I2) receptor agonist** and **thromboxane A2 synthase inhibitor** developed to promote tissue repair and regeneration. It is chemically a nonprostanoid prostacyclin mimetic, which confers greater chemical and biological stability compared to natural prostanoids. ONO-1301 not only **stimulates the prostaglandin I2 (PGI2, IP) receptor**, leading to increased intracytoplasmic cAMP and downstream release of tissue-protective cytokines (such as hepatocyte growth factor [HGF], vascular endothelial growth factor [VEGF], and stromal cell-derived factor-1 [SDF-1]), but also inhibits thromboxane A2 synthase, thereby reducing vasoconstriction and platelet aggregation. Preclinical studies show ONO-1301 can **ameliorate pulmonary arterial hypertension, fibrosis, cardiac failure, myocardial ischemia, nephritis, and liver damage (and tumorigenesis) related to NASH**. It demonstrates anti-inflammatory effects by reducing cytokines such as IL-1β, IL-6, TGF-β, and PDGF-β. Modified sustained-release forms (e.g., ONO-1301SR) and nanoparticle/nanosphere formulations have also been described to enhance targeted delivery and reduce systemic side effects.

02

Targets

PTGIR (Prostanoid IP Receptor)TBXAS1 (Thromboxane A2 synthase)

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