BARCELONA, Spain, December 14, 2016 /PRNewswire/ --
Ability Pharmaceuticals, a drug development biopharmaceutical company specialized in oncology, announced today that it has received from the US Food and Drug Administration (FDA), orphan-drug designation (ODD) for ABTL0812, for the treatment of pancreatic cancer. This regulatory milestone comes after the ODD in the pediatric cancer neuroblastoma granted by EMA and FDA in 2015.
In preclinical studies, ABTL0812 have shown efficacy in pancreatic cancer as single agent and synergistic effect (by 8 to 90 times) in combination with taxanes, platinum compounds and gemcitabine, with induction of tumor regression without increasing the toxicity associated with chemotherapy. First line therapy in patients with either locally advanced or metastatic pancreatic cancer includes these compounds, and administered in combination with ABTL0812 could greatly improve the treatment outcome.
ABTL0812 is currently in phase 2 as first line therapy in combination with chemotherapy in patients with endometrial or squamous lung cancer at Vall d'Hebron Institute of Oncology (VHIO) and Catalan Institute of Oncology (ICO) in Barcelona.
"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 Pharmaceuticals. "
ABTL0812, currently in phase 2 of clinical development, causes cell death by autophagy through the overexpression of TRIB3, an endogenous Akt regulator. It is a first in class fully differentiated oral targeted anticancer compound inhibiting the PI3K/Akt/mTOR pathway without being a direct kinase inhibitor. Its unique mechanism of action was published at Clinical Cancer Research in 2015.
In the phase 1/1b clinical trial (29 patients), ABTL0812 showed the best safety and tolerability compared to other inhibitors of the pathway; without dose-limiting toxicities. The efficacy was comparable to the best PI3K/Akt/mTOR inhibitors; remarkably 2 patients had extremely long disease stabilizations (14 and 18 months).
About Ability Pharmaceuticals
AbilityPharma (http://www.abilitypharma.com), a fast grower in the Catalan and Spanish biopharmaceutical sector, is focusing on creating the future of oncology by developing innovative therapies to address unmet medical needs. Current shareholders include its founders, private investor and the VCs Inveready and SODENA, and has financial support from ACCIO, CDTI, ENISA and MINECO
In April 2016, AbilityPharma signed a territorial license agreement with SciClone Pharmaceuticals, Inc to develop and market ABTL0812 in Greater China
Carles Domènech, PhD
SOURCE Ability Pharmaceuticals SL