ArQule Hits 12-Year High Following Favorable Midstage Study Results

Shares of the small-cap biotech stock ArQule, Inc. ARQL were seeing strength on brisk volume Monday.

What Happened

Close on the heels of a 30% rally Friday on the back of positive results from an early stage study of its leukemia drug candidate, ArQule announced preliminary results from a Phase 1/2 study of its pan-AKT inhibitor miransertib, or ARQ 092, in patients with PIK3CA-related Overgrowth Spectrum, or PROS, and Proteus syndrome, or PS. The company said the results were favorable. 

The data was presented at the European Society of Human Genetics Conference in Gothenburg, Sweden.

The recommended initial dose for the registrational study was defined as a 15mg/square meter once-daily dose, with a subsequent maximum dose increase to 25 mg/square meter.

The pipeline asset also showed a manageable safety profile, even in patients as young as 2 years old, and an improvement in disease-related symptoms and performance status as measured by the Karnofsky/Lansky scale in a majority of patients, ArQule said. 

Miransertib is an orally available selective pan-AKT inhibitor that potently inhibits AKT 1, 2 and 3 isoforms.

Why It's Important

P13K/AKT driven overgrowth diseases are rare diseases that have no current treatment options.

"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," Brian Schwartz, ArQule's chief medical officer, said in a statement.

What's Next

ArQule, which has collaborated with the National Human Genome Research Institute since 2015 in advancing miransertib, said it will start the registrational MOSAIC study in the third quarter of 2019.

ArQule shares were soaring by 16.10% to $9.52 at the time of publication Monday. 

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