MIN4ALD
Financiado
Use of MIN 102 for the treatment of children with cerebral ALD
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 adrenoleuko...
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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Duración del proyecto: 45 meses
Fecha Inicio: 2018-09-05
Fecha Fin: 2022-06-30
Fecha Fin: 2022-06-30
Línea de financiación: concedida
El organismo H2020 notifico la concesión del proyecto el día 2022-06-30
Línea de financiación objetivo
El proyecto se financió a través de la siguiente ayuda:
EIC-SMEInst-2018-2020:
SME instrument
Cerrada
hace 6 años
Presupuesto
El presupuesto total del proyecto asciende a
5M€
Líder del proyecto
MINORYX THERAPEUTICS
La realizacion de actividades de investigacion, desarrollo e innovacion en el ambito tecnico sanitario y, en particular, de la biotecnologia...
Perfil tecnológico
TRL
4-5
5M
Perfil tecnológico
TRL
4-5
5M