Drug intelligence / Profile preview

VDA-1275

Development stage
Preclinical
Lead developer
Vidac Pharma Holding
Modality
Nucleic Acid-Directed Small Molecules → Small Molecules, Covalent Small Molecules → Small Molecules, Classical Binding Small Molecules → Small Molecules
Administration
Oral
01

Overview

VDA-1275 is a novel, orally administered small molecule anti-neoplastic agent developed by Vidac Pharma for the treatment of solid tumors. It acts as a metabolic checkpoint modulator targeting cancer cell metabolism through a triple mechanism of action: (1) reversal of the Warburg effect by disrupting excessive glycolysis in cancer cells and restoring normal cellular metabolism; (2) reactivation of apoptosis by reopening mitochondrial pores that are often disabled in tumors; and (3) modulation of the tumor microenvironment, reducing lactate production to create conditions more favorable for immune response and enhancing chemotherapy efficacy. Mechanistically, VDA-1275 disrupts the interaction between hexokinase 2 (HK2)—an enzyme overexpressed in many cancers—and voltage-dependent anion channels (VDACs) on mitochondria. This uncoupling restores programmed cell death pathways, inhibits tumor proliferation, and triggers anti-tumor immune responses including induction of M1 macrophages and memory T-cells while inhibiting tumor-promoting M2 macrophages. Preclinical studies have shown strong monotherapy efficacy as well as synergistic effects with standard-of-care chemotherapies such as sorafenib and cisplatin across multiple solid tumor models[1][2][4][5][7].

02

Targets

VDAC1 (Hexokinase 2–voltage-dependent anion channel 1 complex)

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