New platform for Catalan companies and organizations accelerates drug R&D for minority diseases

There are between 7,000 and 8,000 minority diseases that affect more than 300,000 people in Catalonia (especially children) and, in most cases, there is no treatment on the market. Discovering new drugs for some of these diseases and accelerating development is the aim of AMMIC, the Acceleradora en Malalties Minoritàries de Catalunya led by Institut Universitari de Ciència i Tecnologia (IUCT) with members Vall d’Hebron Research Institute (VHIR), pharmaceutical corporation Esteve and biotechnology firms Aromics, BCN Peptides, Minoryx, Aniling and Qgenomics.

AMMIC is one of the projects of NEXTHEALTH, the RIS3CAT community for innovation in healthcare coordinated by Biocat and promoted by the Government of Catalonia through ACCIÓ. This community brings together universities, industry and government under a joint action plan that expects to have a direct, tangible impact: creating 5 R&D platforms (including AMMIC), dozens of new jobs and new start-ups; launching new drugs and therapies to market, and creating new training and entrepreneurship initiatives. The project is co-funded by the European Union's European Regional Development Fund (ERDF) under the framework of its Catalonia 2014-2020 Operational Program.

Acceleradora en Malalties Minoritàries de Catalunya (AMMIC) is working to accelerate development of therapeutic solutions from basic research through the clinical phases (gene therapy and small molecules). AMMIC is also working to develop genetic and epigenetic analysis tools and new screening platforms to identify biomarkers with diagnostic, prognostic and treatment value. So, AMMIC is searching for new therapies and their clinical applications to benefit patients.

In addition to working to develop their own drugs, AMMIC aims to act as a global benchmark for other companies working in the field of minority diseases, facilitating protocols and good practices to improve their drug development processes. AMMIC also aims to raise awareness of the Comunitat Catalana de Recerca en Malalties Minoritàries and forge national and international synergies with other agents involved in R&D into treatment for this type of diseases.

First results

Within the broad spectrum of conditions classified as minority diseases, AMMIC is focusing on a group of pediatric and adult tumors, metabolic diseases and neuromuscular diseases: Pediatric Rhabdomyosarcoma, a type of cancerous tumor that develops in soft tissue in the body, normally muscle; Malignant pleural mesothelioma, a cancerous tumor that affects the layer of tissue around the lungs and the chest cavity; Sanfilippo A syndrome, a disease caused by a deficiency in an enzyme that leads to progressive deterioration of neurological and motor skills in children; Adrenoleukodystrophy, a neurodegenerative disease that can affect children between 3 and 5 years old and young adults; and Myotonic dystrophy type 1, which can begin at any age and affects the muscular and cardiovascular systems, the central nervous system and ocular system, the digestive system and reproductive glands.

After its first year of work, the AMMIC project has seen its first scientific results, including the launch of a phase II/III study in Adrenoleukodystrophy, the preclinical assessment with animal models of the efficacy of three new candidate molecules to treat Malignant pleural mesothelioma, Pediatric Rhabdomyosarcoma and Myotonic dystrophy type 1.