21 June 2019 - - US-based ArQule, Inc. (NASDAQ: ARQL) has received preliminary results from the company's phase 1/2 study of its pan-AKT inhibitor, miransertib (ARQ 092), in patients with PIK3CA-related Overgrowth Spectrum and Proteus syndrome in an oral presentation at the European Society of Human Genetics Conference in Gothenburg, Sweden, the company said.
The reported interim data are from the phase 1/2 study, an international, multi-center, open-label 2-part study evaluating miransertib in patients with PS and PROS.
The first part of the study is evaluating the safety, tolerability, PK profile and preliminary evidence of clinical activity of miransertib at different dose levels. MOSAIC, the registrational part of the study, is expected to begin patient enrollment in 3Q19.
Key findings presented include that the recommended initial dose for the registrational study was defined as 15mg/m2 QD with subsequent maximum dose increase to 25mg/m2.
A manageable safety profile was observed in patients as young as 2 years old, with mostly Grade 1 or 2 AEs.
Improvement in disease related symptoms and performance status as measured by Karnofsky/Lansky scale was reported in the majority of patients.
The majority of patients demonstrated improvement or no disease progression extending beyond one year on treatment.
Miransertib (ARQ 092) is an orally available, selective, pan-AKT (protein kinase B) inhibitor that potently inhibits AKT 1, 2 and 3 isoforms. Additionally, it binds both the active and inactive forms of AKT which directly inhibits and prevents membrane localization, respectively. 
Dysregulation of AKT has been implicated in a variety of rare overgrowth diseases and cancers; however, there are currently no approved inhibitors of AKT. AKT inhibitors, either as single agent or combination therapy, show significant promise in molecularly defined patient populations.
Miransertib has been granted Rare Pediatric Disease Designation for Proteus syndrome by the US Food and Drug Administration as well as Orphan Drug Designation by both the FDA and European Medicines Agency. Fast Track Designation has been granted by the FDA for PROS.
ArQule is a biopharmaceutical company engaged in the research and development of targeted therapeutics to treat cancers and rare diseases.
ArQule's mission is to discover, develop and commercialize novel small molecule drugs in areas of high unmet need that will dramatically extend and improve the lives of our patients.