Ability Pharmaceuticals, a drug development biopharmaceutical company specialized in oncology, today announced that the Committee for Orphan Drug Products (COMP) of the European Medicines Agency (EMA) has issued a positive opinion on the application for orphan drug designation of the investigational compound ABTL0812 for the treatment of neuroblastoma. ABTL0812 is a first-in-class Akt/mTOR signaling pathway inhibitor with a novel mechanism of action. It is currently being evaluated for safety and efficacy in patients with advanced solid tumors in a phase I/Ib clinical trial at Hospital Clínic of Barcelona and Institut Català d’Oncologia, leading university hospitals in Barcelona. 
 
Neuroblastoma is a rare cancer of the nervous system that affects children and infants, mostly happening in the abdomen. It comprises 6-10% of all childhood cancers and causes about 15% of cancer-related deaths in children.
 
"The orphan drug designation is an important regulatory advancement for neuroblastoma, a life-threatening pediatric disease with highly deleterious long-term adverse effects due to current toxic treatment based on chemotherapy combinations, radiotherapy and aggressive immunotherapy" said Carles Domenech, PhD, CEO of Ability Pharmaceuticals. "We will continue developing ABTL0812 in Phase I/II trials in pediatric patients following the Phase I/Ib trial in adults currently being finalized and will develop a companion diagnostic tool to stratify patients and improve efficacy in selected populations”.
 
“Development of novel drugs or small molecules directed at specific pathways is necessary to change the outcome of neuroblastoma and improve current therapeutic options” said Jose Alfon, PhD, VP of Research and Development of Ability Pharmaceuticals. “A better understanding of the interplay between pharmacogenomics, tumor and its microenvironment, is critical to achieve the main goal: curing patients and improving their quality of life”.
 
The scientific data that allowed to identify ABTL0812 as a promising tool for the treatment of neuroblastoma was generated with the successful research collaborations Ability Pharmaceuticals has with Jose Miguel Lizcano, PhD, Coordinator of the Research Group on Mechanisms of Cell Signaling and its Alteration in Disease, UAB Universitat Autonoma de Barcelona and with Miquel F. Segura, PhD of the Laboratory of Translational Research in Pediatric Cancer, VHIR Vall d'Hebron Research Institute in Barcelona.
 
About Orphan Drug Designation
 
Orphan drug designation is a status assigned to a medicine intended for use in rare diseases. The Orphan Drug Designation program provides orphan status to medicines intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 5 in 10,000 people in Europe or that are not expected to recover the costs of developing and marketing a treatment. The criteria include that the medicine must be intended for the treatment, prevention or diagnosis of a disease that is life threatening or chronically debilitating and the intended medicine must aim to provide significant benefit to those affected by the condition. Orphan status provides sponsors with development and commercial incentives for designated compounds and medicines.
 
The approval of an orphan designation request does not alter the standard regulatory requirements and process for obtaining marketing approval. Sponsors must establish safety and efficacy of a compound in the treatment of a disease through adequate and well-controlled studies.
 
 
About ABTL0812
 
ABTL0812 is an investigational small molecule which inhibits the Akt/mTOR signaling pathway by a novel mechanism of action, fully differentiated from other PI3K/Akt/mTOR pathway inhibitors under development, and with cell death mediated by autophagy. In preclinical models ABTL0812 has shown activity similar or higher than reference compounds, but with higher safety. Additionally, ABTL0812 potentiates the antitumor effect of several standards of care chemotherapeutic agents, while still maintaining its safety profile. Moreover ABTL0812 keeps its potency and efficacy in tumor cells that have become resistant to standard chemotherapy or targeted therapies, with superiority over other PI3K/Akt/mTOR inhibitors.
 
ABTL0812 is currently finalizing a phase I/Ib clinical trial (first in humans) in advanced cancer patients with solid tumors by the oral route. The study is confirming ABTL0812 predicted high safety profile with signals of efficacy with several disease stabilizations and with inhibition of the Akt/mTOR pathway as monitored with biomarkers. A phase I/II clinical trial in neuroblastoma with patient stratification is being designed.
 
About Ability Pharmaceuticals
 
Ability Pharmaceuticals (www.abilitypharma.com) is research-based biopharmaceutical company formed in 2009 with headquarters in Bellaterra (Barcelona, Catalonia, Spain) at the Research Park of the Autonomous University of Barcelona. The company develops a highly differentiated new class of cancer drugs, with a novel mechanism of action. The company has two drug candidates in development: ABTL0812, which is finalizing its first-in-humans phase I/Ib clinical trial with 27 patients, and ABTL0815 in preclinical development.
 
Current shareholders include the biotech venture capital firm Inveready Seed Capital, its founders and private investors, and has financial support from ACCIO (Government of Catalonia), CDTI and MINECO (Government of Spain).
 
Ability Pharmaceutics is currently raising funds in a Series A financing round of 10 million Euros.
 
About Neuroblastoma
 
Neuroblastoma is a rare cancer that mostly affects young children. Neuroblastoma develops from neural crest-derived cells which, among other locations, are found in a chain running down the back of the chest and abdomen. In many cases, neuroblastoma first develops in the adrenal glands and can spread to other organs such as bone marrow, bones, liver and skin. Neuroblastoma comprises 6-10% of all childhood cancers and about 15% of cancer-related deaths in children. The annual mortality rate is 10 per million children in the 0- to 4-year-old age group, and 4 per million in the 4- to 9-year old age group. The highest incidence is in the first year of life. Initial symptoms most common are aches and pains, loss of energy and loss of appetite. Additionally, the symptoms depend on where the cancer is and whether it has spread. A relatively late sign is a lump or swelling in the abdomen, as this is where the cancer commonly starts.
 
Given the high toxicity of the treatments, based on chemotherapy combinations, radiotherapy and aggressive immunotherapy, it is critical to accurately stratify patients to administer the most deleterious treatments only to those patients who are more likely to progress on the disease. Low risk group represents approximately 35% of the new diagnosed patients and surgical resection is the treatment commonly used, however in the cases of organ dysfunction due to tumor mass, chemotherapy is used, as well as in the intermediate risk group, however the long-term deleterious adverse effects associated to the exposure to the genotoxic chemotherapy drugs should ideally be avoided. Standard therapy for high-risk tumors generally includes four consecutive phases: induction therapy, local control, consolidation therapy and biological treatments to treat minimal residual disease.
 
Ability Pharmaceuticals Contact
 
Email: media.relations@abilitypharma.com