The Catalan biopharmaceutical company Ability Pharmaceuticals, SL announced today the initiation of its first Phase 2 Clinical Trial with its novel targeted anticancer agent ABTL0812 to evaluate its efficacy and safety in combination with paclitaxel and carboplatin in 80 patients with advanced or recurrent endometrial cancer or squamous lung cancer as first-line therapy.  The study will be conducted in Spain and France with Dr. Ana Oaknin at Vall d’Hebron Institute of Oncology (VHIO, Barcelona) as principal investigator for endometrial cancer and Dr. Ernest Nadal at Institut Català d’Oncologia (ICO, Hospitalet-Barcelona) as principal investigator for lung cancer. Dr. Jordi Rodon (also from VHIO) is the coordinating investigator.
 
Dr. Carles Domènech, Chief Executive Officer and co-founder of AbilityPharma, said: “We are very happy to have reached this important development milestone. We face with enthusiasm taking to clinical proof of concept a new important product to treat cancer patients, especially as first line choice in squamous lung cancer and endometrial cancer, clear unmet medical needs”.
 
“ABTL0812 has shown to be an extremely safe compound in patients during the Phase 1b with initial clinical efficacy and high activity on biomarkers”, said Dr. José Alfón, Vice-President of Research and Development at AbilityPharma. “Additionally, the high efficacy and safety observed in combination in preclinical models make us expect good results in the phase 2 trial”.
 
About ABTL0812
 
ABTL0812 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 preclinical cancer models ABTL0812 is efficacious as single agent with an excellent safety profile in a broad spectrum of cancer types: lung, endometrial, pancreatic cancer and neuroblastoma. In these models the compound has also synergistic effect with chemotherapy (taxanes, platinum compounds and gemcitabine) without increasing its toxicity. ABTL0812 is also active on cells resistant to chemotherapy or other targeted therapies, on tumor stem cells and inhibits metastasis formation. Preliminary results show promising immunomodulatory effects.
 
In the phase 1/1b clinical trial (29 patients with advanced solid tumors), ABTL0812 showed the best safety and tolerability compared to other inhibitors of the pathway; no dose-limiting toxicities were identified. The efficacy in patients was comparable to the best PI3K/Akt/mTOR inhibitors in similar clinical trials. Remarkably 2 patients had extremely long disease stabilizations over one year (14 and 18 months). Additionally, ABTL0812 showed high efficacy on biomarkers of the pathway with dose-response effect. Due to its extremely high safety the recommended phase 2 dose was determined by pharmacokinetic-pharmadynamic modeling. 
 
ABTL0812 has Orphan Drug Status from US FDA and from Europe EMA for the pediatric cancer neuroblastoma.
 
About Ability Pharmaceuticals
 
 
Ability Pharmaceuticals (www.abilitypharma.com) is a clinical-stage biopharmaceutical company focused on creating the future of oncology by developing innovative therapies that address unmet medical needs. Established in 2009 and with two drug candidates in development:  ABTL0812, a targeted therapy already in phase 2 clinical trials and and ABTL0815 in preclinical development, AbilityPharma is positioning itself as one of the fast growers in the Catalan and Spanish biotech sector.
 
In April 2016, AbilityPharma signed a territorial license agreement for ABTL0812 with the NASDAQ US company SciClone Pharmaceuticals, Inc. to develop and market the product in Greater China.
 
Ability Pharmaceuticals has headquarters in Cerdanyola del Vallès (Barcelona, Catalonia, Spain) at Parc Tecnològic del Vallès and Parc de Recerca UAB. In addition to ABTL0812, the company has another candidate, ABTL0815 in preclinical development. Current shareholders include the biotech venture firms Inveready and SODENA, its founders and private investors, and has the financial support from ACCIO (Government of Catalonia), CDTI, ENISA and MINECO (Government of Spain).