Rare genetic diseases in the central nervous system may be less of a headache in future, as Barcelona-based biotech Minoryx has raised €21.3M in series B fundraising to test its lead drug candidate against other rare neurological conditions.

According to Philippe Monteyne, partner at investor Fund+, “it is the largest investment in biotech in Spain this year.” Fund+ is also investing in other Spanish companies, such as Aelix, which is developing an HIV vaccine.

In January this year, Minoryx began a Phase II/III trial of its lead compound, which has orphan designation, in patients with adrenomyeloneuropathy. This is the most common form of the neurodegenerative disorder X-linked adrenoleukodystrophy, which predominantly causes neurodegeneration in the spinal cord in adult patients.

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The rare X-linked disorder is caused by a mutation in the gene ABCD1, resulting in the buildup of fatty acids and inflammation in the central nervous system. Minoryx designed its lead compound, a metabolite of the antidiabetic drug pioglitazone, to counteract this by decreasing the inflammation and neuronal death associated with the condition.

Neurons CNS Minoryx

The company now plans to launch a new clinical trial of the same drug candidate in the second most common form of the X-linked condition, childhood cerebral adrenoleukodystrophy. Minoryx also hopes to target other new indications with the funding.

Minoryx Therapeutics is a very promising company…in the field of rare diseases of genetic origin with a high unmet medical need”, commented Monteyne, now a member of Minoryx’s Board.

It’s a small molecule, maybe not the most revolutionary approach but there’s a very high medical need where we can make a difference”, he added.


Images from Shutterstock

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