ATI-2307

ATI-2307 is a **novel antifungal small molecule** (classified as an arylamidine and described as "pentamidine-like") developed for the treatment of invasive fungal infections, including those caused by *Cryptococcus neoformans*, *Candida* spp. (including *C. auris*), and *Aspergillus* spp[1][2][3][5][10]. It functions by ** selectively inhibiting the mitochondrial respiratory chain complexes III and IV** in fungi, leading to disruption of mitochondrial membrane potential and energy production, ultimately causing fungal cell death[1][3][5][7][9]. The selectivity arises from the drug's uptake by a fungal-specific transporter absent in mammalian cells, minimizing host toxicity[1][3][9]. ATI-2307 has shown **broad-spectrum antifungal activity**, including efficacy against fluconazole-resistant and echinocandin-resistant strains, and demonstrates additivity or synergy in preclinical models when combined with fluconazole[1][5][9]. Clinical development is led by Appili Therapeutics, with the compound originally discovered by FUJIFILM Toyama Chemical[1][2]. ATI-2307 is currently developed as an **intravenous formulation**, with three completed phase 1 clinical studies in healthy adults[2]. It is under investigation for several severe invasive fungal diseases, particularly cryptococcal meningitis (CM), for which it has demonstrated potent preclinical efficacy, including reduction in brain and CSF fungal burden in animal models[5][7][9].

Other names

ATI-2307ATI2307ATI 2307T-2307T2307T 2307