Ability Pharmaceuticals Announces EMA-Orphan Drug Designation for ABTL0812 in Pancreatic Cancer

Ability Pharmaceuticals, a drug development biopharmaceutical company specialized in oncology, announced today that has received orphan-drug designation (ODD) for ABTL0812 from the European Medicines Agency (EMA) for the treatment of pancreatic cancer. This regulatory milestone comes after the ODD for ABTL0812 in pancreatic cancer by the FDA in December 2016, and the ODD for pediatric cancer neuroblastoma granted by EMA and FDA in 2015.

The ODD program of the EMA grants special status to medicines and biological products intended to treat, diagnose or prevent rare diseases, which affect less than 5 people every 10 000 in the European Union (EU). This designation establishes a period of exclusivity for seven years of commercialization against competition, as well as certain incentives, such as federal grants, tax credits, a decrease in regulatory fees in the drug approval process, and clinical development advice.

Pancreatic cancer is a life-threatening disease with a significant unmet medical need. The incidence of this cancer has markedly increased over the past several decades. Moreover, treatment options of pancreatic cancer are very limited due to its poor response to chemotherapy, radiation therapy and surgery as conventionally used.

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, Barcelona) and Catalan Institute of Oncology (ICO, L’Hospitalet, Badalona and Girona), INCLIVA (Valencia) and Hospital Universitario Virgen del Rocio (Sevilla). The study will also include top leading centers in France.

"Receiving another Orphan Drug Designation for ABTL0812 is an important regulatory milestone achieved by AbilityPharma and an exciting step towards the clinical advancement of this first-in-class therapy," said Carles Domenech, PhD, CEO of Ability Pharmaceuticals. "Wewill continue to aggressively pursue our clinical development program with ABTL0812 for pancreatic cancer for the benefit of these patients, who currently have a short life expectancy once diagnosed”.

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 December 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.

ABTL0812 is currently in phase 2 as first line therapy in combination with chemotherapy in patients with endometrial or squamous lung cancer. The study includes centers in Spain and France.

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.

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 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 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. 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).