ATX‑304
Peripherally restricted, orally active small-molecule pan-AMPK (AMP-activated protein kinase) and mitochondrial activator, a fat-loss / cardiometabolic research compound. In Phase 2 clinical development for obesity + cardiometabolic disease (Amplifier Therapeutics, Cambrian BioPharma). NOT an approved drug – INVESTIGATIONAL status.
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WHAT IS ATX-304?
Detailed overview
ATX-304 (formerly designated O-304) is a peripherally restricted, orally active small-molecule pan-AMPK (AMP-activated protein kinase) activator + dual mitochondrial uncoupler. It is a unique fat-loss compound originally developed in the context of obesity, cardiometabolic disease, kidney disease and cancer; it is currently in Phase 2 clinical development for obesity and cardiometabolic disease indications, with developers including Amplifier Therapeutics and Cambrian BioPharma. The 'pan-AMPK' label means ATX-304 activates AMPK signalling across multiple subunit combinations, alongside mitochondrial uncoupling. The net effects of this dual action are enhanced glucose uptake into muscle, enhanced insulin sensitivity and improved microvascular perfusion. ATX-304 is potent at AMPK activation: AMPK is normally switched on by a calorie deficit or by exercise, and ATX-304 activates it and delivers those benefits WITHOUT requiring either. A key mechanistic nuance: it does NOT force AMPK on by draining cellular ATP. Instead it raises phosphorylated AMPK (P-AMPK) levels WITHOUT reducing cellular ATP, and protects phosphorylated AMPK from dephosphorylation, so it switches AMPK on and KEEPS it on (it does not let it turn off). The mitochondrial uncoupling raises actual energy expenditure: it increases metabolic demand so cells BURN fuel rather than STORE it. Where many AMPK activators raise glycogen storage, ATX-304 instead promotes glucose USAGE while also telling cells to take MORE glucose in. The net result is more fuel taken in plus a cell that wants to burn more, accompanied by anti-aging / pro-longevity benefits. Documented effects (animal/preclinical + early clinical): it diminishes body fat mass, lowers blood cholesterol, reduces liver steatosis and fibrosis (MASLD / fatty liver), and remodels cholesterol and lipid transport. Per Holm 2025 (JCI Insight PMID 40197369), ATX-304 reduces oxidative stress and improves MASLD via metabolic switching. Per Katerelos 2024 (Biomed Pharmacother PMID 38749175), it protects against cisplatin-induced acute kidney injury via AMPK-dependent metabolic reprogramming. IMPORTANT, HONEST FRAMING: ATX-304 is an INVESTIGATIONAL compound in Phase 2, NOT an approved drug. There is no established consumer or athlete dosing. It is available on the research-chemical market with the usual label-claim / purity caveats.
Mechanism
Peripherally restricted pan-AMPK (AMP-activated protein kinase) + mitochondrial activator, orally active small molecule
Development phase
Phase 2 clinical development for obesity + cardiometabolic disease (Amplifier Therapeutics, Cambrian BioPharma). NOT an approved drug.
Indication (under study)
Obesity, cardiometabolic disease, MASLD / fatty liver – investigational, no established human consumer dosing
Alternate designation
O-304 (same compound)
Legal status
Investigational (Phase 2), NOT an approved drug. Available on the research-chemical market with the usual label-claim / purity caveats. WADA: NOT prohibited.
Data console
Lab data
Safety
Side effects, stop signs, contraindications
Side effects · 6
- Investigational (Phase 2) compound: no published human side-effect profile and no established safe dose; human consumer safety is fundamentally unknown.
- As a dual mitochondrial uncoupler it theoretically raises energy expenditure and heat production; a known class risk of uncouplers in overdose is hyperthermia (overheating), though no human dose threshold is established for ATX-304.
- Increased glucose uptake and improved insulin sensitivity could theoretically cause low blood sugar (hypoglycemia), especially in a calorie deficit or combined with antidiabetic drugs; no human data exists.
- User (non-clinical) reports mention mild skin tingling; these are anecdotal, not verified adverse effects.
- With research-chemical sourced products, label-claim discrepancy and contaminated batches are a real risk: adverse effects from unknown purity cannot be separated from the active compound itself.
- Long-term human hepatic and renal safety is unconfirmed: although it showed liver- and kidney-protective signals in preclinical models, this does not substitute for human long-term safety data.
Contraindications · 6
- Pregnancy and breastfeeding: absolute contraindication – no reproductive safety data for an investigational compound.
- Human consumer (non-trial) use is generally not advised: Phase 2 investigational status, no approved human protocol or dosing.
- Diabetes / use of antidiabetic drugs (insulin, sulfonylureas): theoretical additive hypoglycemia risk – avoid without medical supervision.
- Pre-existing liver or kidney disease: caution warranted – the human hepatic/renal profile is investigational, and the preclinical protective signal does not equal human clinical clearance.
- Heat stress / hyperthermia risk (training in extreme heat, sauna, febrile illness): caution warranted due to the mitochondrial uncoupling mechanism.
- Competitive athletes: although ATX-304 is currently non-listed, the WADA 'S0 non-approved substance' clause may potentially apply – anti-doping agency consultation advised.
Related Performance Compounds
Same therapeutic category
Studies
Related research and clinical findings
AMPK activator ATX-304 reduces oxidative stress and improves MASLD via metabolic switching
Holm E, Vermeulen I, Parween S, López-Pérez A et al.
The AMPK activator ATX-304 alters cellular metabolism to protect against cisplatin-induced acute kidney injury
Katerelos M, Gleich K, Harley G, Loh K et al.
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The information here is for educational and scientific purposes only. Performance-enhancing compounds (AAS, prohormones, stimulants, doping agents) are illegal without prescription in Hungary and most of the EU, and carry serious health and legal risks. WADA bans them in competitive sport. This is NOT a usage guide, and we do not encourage any illegal use. If you do use them, medical supervision and regular bloodwork are ESSENTIAL. Severe endocrine, cardiovascular, hepatic and psychiatric side effects are possible.