Download Ability Pharmaceuticals Receives Positive Opinion from EMA

Ability Pharmaceuticals Receives Positive Opinion from
EMA for Orphan Drug Status of ABTL0812 in Pediatric
Cancer Neuroblastoma.
Barcelona (Catalonia, Spain), April 7, 2015. 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 lifethreatening 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.
Ability Pharmaceuticals, SL – Registre Mercantil de Barcelona, Volum 41611, Foli 122, Full B393915 Inscripció 1 - CIF B65217671 – VAT ESB65217671
www.abilitypharma.com - [email protected]
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.
Ability Pharmaceuticals, SL – Registre Mercantil de Barcelona, Volum 41611, Foli 122, Full B393915 Inscripció 1 - CIF B65217671 – VAT ESB65217671
www.abilitypharma.com - [email protected]
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
Carles Domènech
CEO
Email: [email protected]
Tel. +34 935 868 836
Ability Pharmaceuticals, SL – Registre Mercantil de Barcelona, Volum 41611, Foli 122, Full B393915 Inscripció 1 - CIF B65217671 – VAT ESB65217671
www.abilitypharma.com - [email protected]