• ABTL0812 is administered in combination with FOLFIRINOX in a randomized double-blind, placebo-controlled phase 2b trial
• University of Cincinnati Medical Center (Cincinnati, OH, USA) and Vall d’Hebron Institute of Oncology (VHIO, Barcelona) lead the study, which includes other centers in USA, Spain, France and Israel

BARCELONA, Catalonia, Spain - May 11, 2021. The biopharmaceutical company Ability Pharmaceuticals, SL today announced the inclusion of the first patients in a phase 2b clinical trial with its novel autophagy-inducer anticancer compound ABTL0812.

The trial will evaluate the efficacy and safety of ABTL0812 in combination with FOLFIRINOX in 150 patients with advanced pancreatic cancer as first-line therapy.  The study has started simultaneously at University of Cincinnati Medical Center (Cincinnati, OH, USA) and at Vall d’Hebron Institute of Oncology, VHIO (Barcelona, Catalonia, Spain) with Dr. Davendra Sohal and Dr. Teresa Macarulla as principal investigators respectively. In total, the international study will include sixteen sites, four in the USA, six in Spain, three in France and three in Israel.

The trial is partially funded by 5M € from the highly competitive EIC Accelerator Program of the European Commission Horizon 2020, and 2M $ of the NIH-R01 grant managed by the FDA in its Orphan Disease Program, which were granted during 2020.

"ABTL0812 has shown good efficacy in preclinical studies in pancreatic cancer models, providing a solid basis to start human studies, likewise the product has shown high safety in patients in clinical studies carried out previously", said Dr. Davendra Sohal, Oncologist, Director of Experimental Therapies and Clinic Medical Director at the University of Cincinnati Medical Center, and Principal Investigator in the United States. Dr. Sohal added, "The phase 2b study of ABTL0812 combined with FOLFIRINOX is a very important step in the development of therapies for patients with pancreatic cancer and opens new therapeutic perspectives for this devastating disease."

Dra. Teresa Macarulla, medical oncologist at Vall d'Hebron University Hospital and principal investigator of the Group of Gastrointestinal and Endocrine Tumors of the Vall d'Hebron Institute of Oncology (VHIO), and Principal Investigator of the study in Spain has stated that "Pancreatic cancer is one of the tumor types with more unfortunate prognosis and less therapeutic alternatives. In this sense it is very good news the start of this study with the molecule ABTL0812 in combination with FOLFIRINOX which could improve the results that chemotherapy treatment has given us so far."

Dr. Carles Domènech, Executive Chairman and CSO of AbilityPharma, said: “We are very happy to have reached this important development milestone. Following the good results obtained in endometrial and lung cancer patients, now we start the development in pancreatic cancer patients which could lead to a faster market approval due to the clear unmet medical need in this indication”.

“We have designed this protocol with top Key Opinion Leaders in the pancreatic cancer space, which validates the clinical strategy followed by the development of ABTL0812 in this indication”, said Dr. José Alfón, Vice-President of Research and Development at AbilityPharma.

“The inclusion of the first patients in the phase 2b clinical trial in pancreatic cancer is the result of the hard work performed by AbilityPharma employees and our advisors in the last two years” said Gemma Fierro, Vice-President of Regulatory and Clinical Affairs at AbilityPharma.

About ABTL0812

ABTL0812 is a first-in-class fully differentiated oral targeted anticancer compound causing cell death by autophagy (self-digestion).

ABTL0812 has demonstrated clinical efficacy in an open-label phase 2 clinical study in patients with endometrial cancer or squamous cell lung cancer, as a first-line treatment in combination with chemotherapy (CT) and as a maintenance treatment after CT cycles. The results show a significant increase in efficacy over first-line standard of care (carboplatin + paclitaxel), and even higher response rates in specific subpopulations. ABTL0812 increases by 40% CT efficacy in endometrial cancer patients, and by 50% in sqNSCLC patients, without increasing CT toxicity. The study was led by Vall d’Hebron Institute of Oncology, VHIO (Barcelona), Institut Gustave-Roussy (Paris) and Centre Léon Bérard (Lyons), and included other sites in Spain and France.

ABTL0812 has also received Orphan Drug Designations (ODD) for pancreatic cancer, biliary cancer and the pediatric cancer neuroblastoma by the FDA in the USA and by the EMA in Europe.

The mechanism of action of ABTL0812 is unique and novel. ABTL0812 is a first-in-class small molecule, orally administered that binds to the nuclear receptors PPARα and PPARγ inducing PPAR-dependent Endoplasmic Reticular Stress (ER-stress) and blocking Akt activation, the central kinase of the PI3K/Akt/mTOR pathway. The combination of inhibition of the PI3K/Akt/mTOR pathway and the ER-Stress induction results in an autophagy-mediated cancer cell death. The mechanism of action was published in Clinical Cancer Research in May 2016 and in Autophagy in May 2020.

In preclinical cancer models, ABTL0812 is effective as a single agent with an excellent safety profile in a broad spectrum of cancer types, including lung, endometrial and pancreatic cancer, neuroblastoma and glioma. Likewise, it also enhances chemotherapy (taxanes, platinum compounds and gemcitabine) and immunotherapy with a synergistic effect without increasing its toxicity. Translational results have been published in Gynecologic Oncology, International Journal of Cancer and Cell Death and Disease in 2019 and 2020.

About Ability Pharmaceuticals

Ability Pharmaceuticals (www.abilitypharma.com) is a biopharmaceutical company with a product, ABTL0812 in phase 2b clinical trials, focused on improving the future of oncology by developing innovative therapies that address unmet medical needs. In April 2016, AbilityPharma signed a territorial license agreement for ABTL0812 with SciClone Pharmaceuticals (Hong Kong) Development Co. Limited to develop and market the product in China.

Ability Pharmaceuticals is headquartered in Parc Tecnològic del Vallès and Parc de Recerca UAB (Cerdanyola del Vallès, Barcelona, Catalonia, Spain). Current shareholders include its founders, private investors, CDTI, the biotech venture firms Inveready, Fitalent and SODENA, and SciClone Pharmaceuticals, and has the financial support from ACCIO (Government of Catalonia), CDTI, ENISA and MINECO (Government of Spain).