Miransertib (ARQ 092) Granted Fast Track Designation for the Treatment of PIK3CA-Related Overgrowth Spectrum (PROS)
Miransertib has already been granted Rare Pediatric Disease Designation
“We are very excited by this important regulatory milestone for
miransertib and look forward to rapidly advancing this program for
patients suffering from this devastating condition with no approved
therapies,” said ArQule’s Chief Medical Officer, Dr.
Miransertib (ARQ 092) is an orally available, selective, pan-AKT (protein kinase B) inhibitor that potently inhibits AKT1, 2 and 3 isoforms. 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 is currently in a Phase 1/2 company-sponsored study for PIK3CA-Related Overgrowth Spectrum (PROS), a Phase 1 study for ultra-rare Proteus syndrome conducted by the
PROS is a term used to refer to a spectrum of rare diseases identified by somatic mutations in the PIK3CA gene, that result in excess growth in certain areas of the body. While the individual diseases that fall within the overgrowth spectrum have similar symptoms, each disease is defined by unique clinical characteristics. The implementation of genetic sequencing has led to the identification of the underlying genetic mutations that drive these overgrowth disorders, allowing for the development of medicines that target the specific causes of disease.
About Proteus Syndrome
Proteus syndrome is an ultra-rare condition characterized by the aberrant overgrowth of multiple tissues of the body. Patients with Proteus syndrome experience changes in the shapes of certain body structures over time, including abnormal, often asymmetric, massive growth (overgrowth) of the skeleton, skin, adipose tissue and central nervous system out of proportion to the rest of the body. Although patients may have minimal or no manifestations at birth, the disease develops and becomes apparent in early childhood (6-18 months) and rapidly progresses with intense growth in the first 10 years of life. The worldwide incidence is believed to be approximately one in a million. There are currently no approved medicinal treatments for Proteus syndrome, leaving patients with minimal treatment options to manage the disease and a mortality of 25% by age 22.
Forward Looking Statements
This press release contains forward-looking statements regarding the planned clinical development of miransertib, including a statement regarding its rapid advancement in PROS. These statements are based on the Company's current beliefs and expectations and are subject to risks and uncertainties that could cause actual results to differ materially. Positive information about early clinical results does not ensure that later-stage clinical trials will be successful. For example, miransertib may not demonstrate promising therapeutic effect in man; in addition, it may not exhibit an adequate safety profile in planned or later stage or larger scale clinical trials as a result of known or as yet unanticipated side effects. The results achieved in later stage trials may not be sufficient to meet applicable regulatory standards or to justify further development. Problems or delays may arise during clinical trials or in the course of developing, testing or manufacturing miransertib that could lead the Company to discontinue development. Even if later stage clinical trials are successful, unexpected concerns may arise from subsequent analysis of data or from additional data. Obstacles may arise or issues may be identified in connection with review of clinical data with regulatory authorities. Regulatory authorities may disagree with the Company's view of the data or require additional data or information or additional studies. In addition, we are utilizing diagnostic tests to identify patients in the Phase 1/2 trial with miransertib in PROS diseases and expect to utilize diagnostic tests in other clinical trials with miransertib. We or our collaborators may need to develop and register these or other diagnostic tests as companion diagnostics with the FDA. We or our collaborators may encounter difficulties in developing and obtaining regulatory approval for companion diagnostics, including issues relating to access to certain technologies, selectivity/specificity, analytical validation, reproducibility, or clinical validation. Any delay or failure by our collaborators or us to develop or obtain regulatory approval of companion diagnostics could delay or prevent approval of our product candidates. Drug development involves a high degree of risk. Only a small number of research and development programs result in the commercialization of a product. For more detailed information on the risks and uncertainties associated with the Company's drug development and other activities, see the Company's periodic reports filed with the
Marc Schegerin, M.D.
Senior Vice President, Head of Strategy, Finance and Communication
LifeSci Public Relations
Allison Blum, Ph.D., 646-627-8383