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Opendata, web and dolomites

MIN4ALD SIGNED

Use of MIN-102 for the treatment of children with cerebral ALD

Total Cost €

EC-Contrib. €

Partnership

Views

MIN4ALD project word cloud

Explore the words cloud of the MIN4ALD project. It provides you a very rough idea of what is the project "MIN4ALD" about.

hematopoietic patients excellent 2015 adrenoleukodystrophy orphan suppose cells raised stem niche candidate us disease aggressive procedure linked 3 symptoms expand demonstration auditory stimulus occurring 24 always paediatric business 102 function progressive 9 phenotype fda diseases global rare company drug consolidation 576 reference treatment leaders investors cagr ald time min4ald disorder treat feasibility usually impressive opportunity minoryx valued huge indicated grow brain central 121 cell motor childhood cognitive onset pharma period pharmacological boy cerebral itself designation opinion nervous cald market nerve optimal demonstrated transplantation alternative amount therapy death billion min genetic pharmaceutical 2022 holds fatal visual devastating received internationally patent

Project "MIN4ALD" data sheet

The following table provides information about the project.

Coordinator	MINORYX THERAPEUTICS S.L. Organization address address: AVENIDA ERNEST LLUCH 32, TCM3, TECNOCAMPUS MATARO city: MATARO BARCELONA postcode: 8302 website: n.a. contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.
Coordinator Country	Spain [ES]
Total cost	4˙437˙500 €
EC max contribution	3˙106˙250 € (70%)
Programme	1. H2020-EU.3. (PRIORITY 'Societal challenges) 2. H2020-EU.2.3. (INDUSTRIAL LEADERSHIP - Innovation In SMEs) 3. H2020-EU.2.1. (INDUSTRIAL LEADERSHIP - Leadership in enabling and industrial technologies)
Code Call	H2020-SMEInst-2018-2020-2
Funding Scheme	SME-2
Starting year	2018
Duration (year-month-day)	from 2018-10-01 to 2021-03-31

Partnership

Take a look of project's partnership.

#	participants	country	role	EC contrib. [€]
1	MINORYX THERAPEUTICS S.L. MINORYX THERAPEUTICS S.L. Organization address address: AVENIDA ERNEST LLUCH 32, TCM3, TECNOCAMPUS MATARO city: MATARO BARCELONA postcode: 8302 website: n.a. contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	ES (MATARO BARCELONA)	coordinator	3˙106˙250.00

Map

Project 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.

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The information about "MIN4ALD" are provided by the European Opendata Portal: CORDIS opendata.