Ability Pharmaceuticals has announced that the Food and Drug Administration has granted orphan drug designation to ABTL0812, an investigational candidate for pancreatic cancer.

ABTL0812 is an oral targeted anticancer compound which inhibits the PI3K/Akt/mTOR pathway without being a direct kinase inhibitor. The compound causes cell death by autophagy through the overexpression of TRIB3, an endogenous Akt regulator. 

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In a Phase 1/1b clinical trial (29 patients), ABTL0812 demonstrated comparable efficacy to other PI3K/Akt/mTOR inhibitors, with two patients showing disease stabilization for as long as 14 and 18 months.

ABTL0812 is currently being evaluated in a Phase 2 trial as a first-line therapy in combination with chemotherapy in patients with endometrial or squamous lung cancer.

“Receiving Orphan Drug Designation for ABTL0812 is an important regulatory milestone achieved and an exciting step towards clinical advancement of this first-in-class therapy,” said Carles Domenech, PhD, CEO of Ability.

For more information visit AbilityPharma.com.