On October 4, 2018 ArQule, Inc. (Nasdaq: ARQL) reported that it will be presenting clinical data on the company’s lead AKT inhibitor, miransertib (ARQ 092), in three poster presentations at the American Society of Human Genetics (ASHG) 2018 Annual Meeting to be held from October 16 to 20, 2018 in San Diego, CA (Press release, ArQule, OCT 4, 2018, View Source [SID1234529813]).

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The data to be presented is from the Phase 1/2 company-sponsored trial in PROS and the single patient use program with select physicians.

Presentation Details

Title:

Personalized medicine in rare diseases and cancer: A case report of a lasting response in a young teenage patient with Proteus syndrome and secondary ovarian cancer

Program #:

1251

Session:

Complex Traits and Polygenic Disorders
Date:

Wednesday, October 17, 2018

Time:

2:00-3:00 PM PT
Location:

Exhibit Hall

Title:

An open-label, phase 1/2 study of miransertib (ARQ 092), an oral pan-AKT inhibitor, in patients (pts) with PIK3CA-related Overgrowth Spectrum (PROS): Preliminary results

Program #:

2538

Session:

Complex Traits and Polygenic Disorders
Date:

Wednesday, October 17, 2018
Time:

3:00-4:00 PM PT
Location:

Exhibit Hall

Title:

Severe PI3Kinase overgrowth syndrome treated with the AKT inhibitor miransertib

Program #:

1288

Session:

Mendelian Phenotypes
Date:

Thursday, October 18, 2018
Time:

3:00-4:00 PM PT
Location:

Exhibit Hall
About Miransertib
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 National Institutes of Health (NIH/NHGRI), and a Phase 1b study in combination with the hormonal therapy, anastrozole, in patients with advanced endometrial cancer with AKT and PI3K mutations. Miransertib has been granted Rare Pediatric Disease Designation and Fast Track Designation by the U.S. Food and Drug Administration (FDA), as well as Orphan Designation by the FDA and European Medicines Agency in the rare overgrowth disease, Proteus syndrome.

About PROS
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.