ArQule, Inc. announced 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 (PROS) and Proteus syndrome (PS) in an oral presentation at the European Society of Human Genetics Conference in Gothenburg, Sweden.
“The results presented from our ongoing phase 1/2 study highlight the potential for miransertib to provide a molecularly targeted treatment for patients with rare PI3K/AKT driven overgrowth diseases,” said Dr. Brian Schwartz, CMO of ArQule. “We have been committed to rapidly advancing miransertib for patients with these devastating diseases since collaborating with The National Human Genome Research Institute in 2015 and are now poised to start the registrational study, MOSAIC, in the third quarter of this year. We are thrilled with the preliminary safety and clinical activity data and look forward to continued clinical progress for the program.”
Dr. Chiara Leoni, an investigator on the study from the Fondazione Policlinico Universitario Agostino Gemelli, said, “The precision medicine approach of the study has led to very encouraging preliminary results with the observed safety and tolerability profile, along with clinically meaningful improvement in disease related symptoms in patients. In addition, the majority of patients have had stable disease while on treatment, demonstrating the potential of miransertib to halt disease progression. The ability to attain durable treatment responses with a manageable safety profile is an important step forward for this patient population that currently has no medicinal treatment options, and I look forward to advancing the registrational MOSAIC study in the coming months.”
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 Q3 2019.
Key findings presented include:
- 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 1 year on treatment