PXL065 (DRX-065)

PXL065 (DRX-065) (deuterium-stabilized R-pioglitazone), is a mitochondrial pyruvate carrier (MPC) inhibitor that is currently in Phase 1 development. PXL065 (DRX-065) is the R-stereoisomer (single isomer) of pioglitazone. Pioglitazone, a drug approved for the treatment of type 2 diabetes, has demonstrated efficacy in NASH and is currently the only drug recommended in practice guidelines for biopsy-proven NASH patients1. However, pioglitazone’s use has been limited in NASH due to its side effect profile, which includes weight gain, bone fractures and fluid retention. PXL065 (DRX-065), a novel patent-protected drug candidate, offers a new approach for the treatment of NASH with the potential for similar efficacy and a reduction of side effects associated with the parent drug, pioglitazone.

1. J Hepatol. 2016, 64(6),1388-402; Hepatology 2018, 67, 328-357

Phase 1a

Phase 1a results demonstrated that PXL065 was shown to be safe and well-tolerated with no adverse events. Based on the pharmacokinetic (PK) results, modeling predicts that a 15 mg dose of PXL065, a deuterium-stabilized R-stereoisomer of pioglitazone, is expected to yield similar efficacy as 45 mg of the parent drug, pioglitazone (Actos®*) with an improved side effect profile, including reduced weight gain and fluid retention.

Our Development and Commercialization Strategy

PXL065, as a first-in-class novel mechanism, provides a unique opportunity for patients with chronic metabolic disorders, including NASH.