Therapy Areas: Central Nervous System
ArQule Begins Enrollment in Trial of Miransertib in Overgrowth Diseases
2 November 2017 - - The US Food and Drug Administration has granted Rare Pediatric Disease Designation to Burlington, Massachusetts-based biopharmaceutical company ArQule, Inc. (NASDAQ: ARQL) for miransertib (ARQ 092) for the treatment of Proteus syndrome, a rare disease characterized by overgrowth of the skeleton, skin, adipose tissue and central nervous system, the company said.
Miransertib (ARQ 092) is an orally bioavailable, selective small molecule inhibitor of the AKT kinase. The AKT pathway, when abnormally activated, is implicated in multiple oncogenic processes.
The pathway has emerged as a target of for compounds that inhibit its activity, which has been linked to a variety of cancers as well as to select non-oncology indications. Dysregulation of AKT is also a driver of certain rare proliferative disorders such as Proteus syndrome.
ArQule is enrolling a phase 1/2 trial being conducted in the US and EU for Overgrowth Diseases, including Proteus syndrome and PIK3CA-Related Overgrowth Spectrum (PROS).
The E17K mutation of AKT1 causes Proteus syndrome, a rare non-cancerous segmental overgrowth disorder, and the analogous PROS is caused by genetic alterations in the PI3K pathway.
The phase 1 portion is enrolling six patients in a dose escalation cohort.
An additional 10 patients will be enrolled in an expansion cohort as part of the phase 2 portion of the trial. The company will also continue to provide miransertib on a named patient basis to those patients unable to travel to a clinical trial site due to severe disease.
ArQule is engaged in the research and development of targeted therapeutics to treat cancers and rare diseases.
The company's mission is to discover, develop and commercialize novel small molecule drugs in areas of high unmet need. Its clinical-stage pipeline consists of five drug candidates, all of which are in targeted, biomarker-defined patient populations.
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