Ability Pharmaceuticals Announces FDA-Orphan Drug Designation for ABTL0812 in Biliary Tract Cancer

Ability Pharmaceuticals, a drug development biopharmaceutical company specialized in oncology, announced today that it has received orphan-drug designation (ODD) for ABTL0812 from the US Food and Drug administration (FDA) for the treatment of biliary tract cancer.

The ODD program of the FDA grants special status to medicines and biological products intended to treat, diagnose or prevent rare diseases, which affect fewer than 200,000 people in the USA. 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.

The biliary tract refers to the organs and ducts that make, store and release the bile into the small intestine, and includes the liver, gall bladder and bile ducts located inside and outside the liver. Biliary tract cancer is a rare, highly fatal and heterogenous malignancy that arises from different locations within the biliary tree and comprises less than 1% of all human cancers and around 10-15% of all primary liver cancers. It is a life-threatening disease with a significant unmet medical need and treatment options are very limited due to its poor response to chemotherapy, radiation therapy and surgery conventionally used.

In preclinical studies, ABTL0812 has shown efficacy in biliary tract cancer as single agent and synergistic effect 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 biliary tract cancer includes these compounds, and administered in combination with ABTL0812 could greatly improve the treatment outcome. Besides, in the phase 1 study evaluating ABTL8012 as monotherapy with advanced cancer patients, one patient with biliary tract cancer remained stable for 18 months

"We are particularly excited about the opportunity to provide benefit to patients with biliary tract cancer following the outstanding response of one patient that remained stable during 18 months in the phase 1 study with ABTL0812." said Carles Domenech, PhD, CEO of Ability Pharmaceuticals. "Receiving another orphan designation represents an important milestone as we evaluate to start the clinical program in biliary tract cancer and other cancer types."

About ABTL0812

ABTL0812 is a first-in-class fully differentiated oral targeted anticancer compound causing cell death by autophagy through binding to the nuclear receptors PPARα/γ. It induces TRIB3 overexpression which blocks Akt activation, blocking the Akt/mTOR axis of the PI3K/Akt/mTOR pathway, and induces PPAR-dependent Endoplasmic Reticular Stress (ER-stress). The combination of both effects results in a robust autophagy-mediated cancer cell death. Its unique mechanism of action was published at Clinical Cancer Research in May 2016.

In preclinical cancer models ABTL0812 is efficacious as single agent with an excellent safety profile in a broad spectrum of cancer types: lung, endometrial and pancreatic cancers, neuroblastoma and glioma. In these models the compound has also synergistic effect with chemotherapy (taxanes, platinum compounds and gemcitabine) without increasing its toxicity.

In Europe, 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.

The Investigational New Drug (IND) for endometrial cancer or squamous non-small cell lung cancer was approved by the US FDA in December 2017, followed by the approval of a phase 1/2 pancreatic cancer study one month later. Also, ABTL0812 has been granted the Orphan Drug Designation (ODD) for neuroblastoma and pancreatic cancer by the FDA in the US and by the EMA in Europe.

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. The company was established in 2009 with two drug candidates in development: ABTL0812, a targeted therapy already in phase 2 clinical trials and ABTL0815 in preclinical development.

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. 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).

AbilityPharma has launched a financial round of 3-5M euros which includes a 1M euros crowdfunding campaign with Capital Cell. https://capitalcell.es/campaign/ability-pharmaceuticals/