Ability Pharmaceuticals receives positive opinion from EMA for Orphan Drug Status of ABTL0812

Ability Pharmaceuticals, a drug development biopharmaceutical company with headquarters in UAB Research Park, 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, researcher at UAB Institut de Neurociències and Coordinator of the Research Group on Mechanisms of Cell Signaling and its Alteration in Disease of Autonomous University of Barcelona and with Miquel F. Segura, PhD of the Laboratory of Translational Research in Pediatric Cancer of Vall d'Hebron Research Institute in Barcelona.