• The study shows that the triple combination of ABTL0812, an innovative antitumor compound, with carboplatin and paclitaxel chemotherapy, is active and could become a new first-line treatment for patients with endometrial cancer
• The Disease Control Rate (DCR) of 100% and the Progression Free Survival (PFS) of 10.2 months are very promising compared to historical controls
• The results will be the starting point for conducting a phase 2b/3 clinical trial in patients with endometrial cancer

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Barcelona (Catalonia, Spain), September 22, 2020. The biopharmaceutical company, AbilityPharma (Ability Pharmaceuticals, SL) dedicated to the development of oncological drugs has presented, within the framework of the ESMO2020 Virtual Congress, the results of a phase 1/2a clinical trial to evaluate the efficacy of ABTL0812, an antitumor compound, in combination with carboplatin and paclitaxel (chemotherapy) for the treatment of metastatic/recurrent endometrial cancer.

The results have been presented by Dr. Alexandra Leary, Department of Medical Oncology, Institut Gustave Roussy, Villejuif, France, at the 2020 ESMO Virtual Congress 2020, during the Science Weekend, in 19-21 September 2020, with the poster #2727; Phase 2 of ABTL0812, a pro-autophagic drug, in combination with paclitaxel and carboplatin (P/C) as first-line treatment in advanced/recurrent endometrial cancer).

Study results have confirmed that patients who received the combination of ABTL0812 with carboplatin and paclitaxel, followed by ABTL0812 as maintenance after chemotherapy, had an Overall Response Rate (ORR) of 66% and a 100% Control Rate Disease (DCR). Furthermore, the progression-free survival (PFS) of the patients who took the triple combination was 10.2 months. These excellent results are superior to historical controls of chemotherapy alone and support further research for this product in patients with endometrial cancer.

Participation of Spanish and French centers

This study has been performed in leading cancer hospitals in Spain and France, including Vall d’Hebron Institute of Oncology (VHIO, Barcelona), Institut Català d’Oncologia (ICO, L'Hospitalet, Badalona and Girona), Institut Gustave Roussy (Paris), Centre Léon-Bérard (Lyon), Institut Paoli-Calmettes (Marseille), the INCLIVA (Valencia), and Hospital Universitario Virgen del Rocío (Sevilla).

Dr. Ana Oakinin, Medical Oncology at Vall d'Hebron University Hospital and investigator at VHIO, claimed that “The combination of ABTL0812 + carboplatin and paclitaxel showed promising results with an acceptable safety and tolerability profile. These outcomes look promising and further studies are warranted to demonstrate the potential benefit of the triple combination in metastatic/recurrent endometrial cancer”.

Dr Alejandro Pérez-Fidalgo, oncologist at INCLIVA-Hospital Clínico Universitario, Valencia, Spain added: “The efficacy of the triple combination of oral ABTL0812 + carboplatin and paclitaxel shows high response rates and suggests a potential benefit of the addition of ABTL0812 to chemotherapy, so a phase 2b/3 clinical trial in patients with endometrial cancer could confirm the role of this new drug”.

The Executive Chairman and CSO of AbilityPharma, Dr. Carles Domenech, declared that “The publication of these good results is a very encouraging milestone for AbilityPharma, which validates the project and encourages us to keep developing ABTL0812 to treat endometrial and other types of cancer”

About ABTL0812

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

ABTL0812 is currently in phase 2a open-label clinical trials in Europe in patients with endometrial cancer or squamous cell lung cancer, as a first-line treatment in combination with chemotherapy and as a maintenance treatment after the chemotherapy cycles. The study is being conducted in leading cancer hospitals in Spain and France.
This same phase 2 study was also approved by the US FDA in December 2017. In addition, the FDA approved the protocol for a phase 2 study in pancreatic cancer in January 2018.

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.

A phase 1/1b clinical trial was previously conducted (29 patients with advanced solid tumors), in which ABTL0812 showed great safety and tolerability and no dose-limiting toxicities were identified. There were signs of efficacy, and notably two patients had long-term disease stabilizations, for more than one year (14 and 18 months). In addition, ABTL0812 showed activity on biomarkers with dose-response.

The mechanism of action of ABTL0812 is unique and novel. ABTL0812 is a targeted therapy drug that kills cancer cells through the induction of robust cytotoxic autophagy. ABTL0812 binds and activates the transcriptional activity of the nuclear receptors PPARα and PPARγ, leading to the induction of endoplasmic reticular stress (ER-Stress), and to the blockade of Akt activation, the central kinase of the PI3K/Akt/mTOR pathway. This dual action of ER-Stress activation and Akt/mTOR axis blockade converge to strongly induce autophagy, which results in cancer cell death. The mechanism of action was published partially in Clinical Cancer Research in May 2016, and fully described in Autophagy journal 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) with a synergistic effect without increasing its toxicity.
 
About Ability Pharmaceuticals

Ability Pharmaceuticals (www.abilitypharma.com) is a biopharmaceutical company with a product, ABTL0812 in phase 2 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 the biotech venture firms Inveready, Fitalent and SODENA, its founders and private investors, and has the financial support from ACCIO (Government of Catalonia), and CDTI, ENISA and MINECO (Government of Spain).