Project description
A drug for ALD
X-linked adrenoleukodystrophy (ALD) is a rare, fatal genetic condition caused by mutations in the ABCD1 gene, leading to accumulation of very long-chain fatty acids in all tissues. ALD affects mainly the nervous system, and patients present with a progressive loss of visual, auditory, motor and cognitive abilities. The scope of the EU-funded MIN4ALD project is to address the lack of pharmacological treatments for ALD currently on the market. The proposed formulation aims to prevent or delay the onset of inflammatory lesions in the brain, reduce disease progression and improve clinical outcome of patients.
Objective
The objective of the MIN4ALD project is the development of a new pharmacological therapy to treat paediatric patients with cerebral ALD (cALD) - the most rapidly progressive and devastating phenotype of X-ALD (X-linked adrenoleukodystrophy).
cALD is a rare, fatal genetic disorder that affects nerve cells in the brain. Symptoms of cALD usually occur in a boy’s early childhood, and are characterized by rapidly progressive loss of visual, auditory, motor and cognitive function, with death usually occurring after 2 to 4 years from disease onset.
Currently, there is no pharmacological treatment available on the market. The only existing alternative for cALD patients is hematopoietic stem cell transplantation, yet the procedure itself is very aggressive and results are not always optimal.
Minoryx has developed a candidate drug that has demonstrated in several studies an excellent potential to treat patients with X-ALD. MIN4ALD Phase 2 demonstration is key to bring this unique treatment to the market. The results obtained in the Feasibility Analysis carried out indicated that MIN4ALD could be a huge business opportunity within the orphan diseases market.
The Global orphan diseases market is a niche market valued at US$ 121.6 billion in 2015. It is expected to grow to US$ 576.9 billion by 2022, with an average CAGR of 24.9% during the period 2015-2022. This is an impressive growth compared to the Global pharma market, which projects a 6.3% CAGR through 2022.
Minoryx has raised an important funding amount for R&D development from leading investors and has the support of reference Key Opinion Leaders. Minoryx received Orphan Drug Designation from both EU and FDA and holds a patent application covering the use of MIN-102 for X-ALD and other diseases affecting the central nervous system.
MIN4ALD will suppose a crucial stimulus for Minoryx to expand internationally and improve time-to-market, leading to the consolidation of the company in the pharmaceutical market.
Fields of science
Programme(s)
Funding Scheme
SME - SME instrumentCoordinator
08302 MATARO BARCELONA
Spain
The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.