US-based biopharmaceutical firm ArQule has begun dosing patients with ARQ 092 in a Phase I/II clinical trial to treat patients with rare overgrowth diseases.
ARQ 092 is an orally bioavailable, selective small molecule AKT kinase inhibitor, which is said to drive certain proliferative disorders when dysregulated.
The two-part Phase I/II trial will enrol patients aged six and above with overgrowth diseases such as PIK3CA-related overgrowth spectrum (PROS), including Congenital Lipomatous Overgrowth, Vascular malformations, Epidermal nevi, spinal / skeletal anomalies and / or scoliosis (CLOVES), Fibroadipose hyperplasia and Proteus syndrome.
The dose escalation part of the trial will enrol six subjects for the Phase I portion, while the Phase II portion will include an additional ten patients in the trial’s expansion part.
The trial is currently being conducted at the US and EU sites and its primary objective is to establish a clinically meaningful endpoint for advancing into a registrational trial.
ArQule chief medical officer and research and development head Dr Brian Schwartz said: “There are a spectrum of overgrowth diseases that are rare, are known to be driven by PI3K or AKT1 mutations, and have no approved therapeutic options.
“These diseases begin showing symptoms in infancy and generally result in patients undergoing multiple surgeries in their childhood and sometimes into adulthood.
“With this trial, we are increasing our efforts to determine an appropriate dose, as well as a clinical endpoint for a registrational clinical trial with the hope of providing a therapeutic option for these patients.”
The firm intends to provide the product on a named patient basis to subjects who cannot travel to a clinical trial site due to severe disease.
ARQ 092 is also being investigated by the National Institutes of Health (NIH) in a Phase I trial for Proteus syndrome and in a Phase Ib expansion trial for patients with endometrial cancer, lymphomas and tumours harbouring AKT or PI3K mutations.