Cerdanyola del Vallès (Barcelona, Catalonia, Spain), June 1, 2023. AbilityPharma (Ability Pharmaceuticals, SA), a Catalan biopharmaceutical company specializing in the development of oncology drugs, has announced today that the company will present the final results of the phase 2a clinical trial evaluating ABTL0812 in combination with carboplatin and paclitaxel in patients with squamous non-small cell lung cancer.

The results will be presented by the study investigators at the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago at the "Lung Cancer - Non-Small Cell Metastatic" session on Sunday, June 4. from 8:00 - 11:00 GMT-5, Chicago time (Poster Board: # 47, Abstract 9059). The title of the abstract is “Phase 2 clinical trial of the proautophagic drug ABTL0812 combined with paclitaxel and carboplatin in first-line patients with advanced squamous non-small cell lung carcinoma”.

The study has shown that the combination of ABTL0812 with carboplatin and paclitaxel, followed by ABTL0812 as maintenance after chemotherapy, has given an increase in treatment efficacy when compared to historical controls in patients treated with chemotherapy alone. The percentage of patients who responded to treatment was 52.0% (ORR) and 32.0% of patients had stable disease. If we compare with the historical controls ORR 32%, the combination represents an improvement. It also improves the progression free survival (PFS) which goes from the historical 4.2 months to 6.2 months. Where the increase is greatest is in the overall survival  (OS), which doubles and goes from the historical 11.3 months to 22.5 months.

This study was conducted in leading cancer hospitals in Spain and France: Institut Català d'Oncologia (ICO) in Hospitalet, Badalona and Girona, Hospital Clínic de Valencia (INCLIVA), Hospital Virgen del Rocío in Seville, Hospital Vall d’Hebron in Barcelona (VHIO ), Institut Gustave Roussy (Paris), Institut Paoli-Calmettes (Marseille) and Center Léon Bérard (Lyon).

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) both in overall response rate (ORR) and progression free survival (PFS). Patients in the endometrial cancer group had an increase of over 30% in ORR compared to standard of care treatment (66% ABTL0812 + Chemotherapy vs 51% Chemotherapy alone) and of 38% in PFS (7.1 months à 9.8 months). In the sqNSCLC group, ORR increased over 40% compared to standard of care (53% vs 32%) and PFS increased 44% (4.2 months à 6.2 months). Additionally, overall survival (OS) analysis shows an increase of 100% going from 11,3 months in the best historical control to 22.5 months in sqNSCLC patients treated with ABTL0812 + Chemotherapy. 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. Additional immunomodulatory antitumor effects show that ABTL0812 transforms “cold” tumors into “hot” immunogenic tumors. These effects were presented at AACR congress in April 2021. Publication is under way.

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 glioblastoma. Likewise, it also enhances chemotherapy and immunotherapy with a synergistic effect without increasing its toxicity. Translational results have been published in Gynecologic Oncology (2019), International Journal of Cancer and Cell Death and Disease (2020), Cancer Communications (2022) and Frontiers in Oncology (2022).

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 capital firms Inveready, Fitalent and SODENA, the EIC Fund and SciClone Pharmaceuticals, and has the financial support from ACCIO (Government of Catalonia), CDTI, ICO, ENISA, the Ministry of Science & Innovation (Government of Spain) and the European Commission (H2020 Program, European Innovation Council).

Contact information:

Carles Domènech, PhD

Executive Chairman and CEO

Ability Pharmaceuticals, SA

E-mail: media.relations@abilitypharma.com

www.abilitypharma.com