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NeuroVia Initiates Phase 1/2 Trial of NV1205 for Treating Childhood Cerebral Adrenoleukodystrophy (CCALD)

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NeuroVia, Inc., a biopharmaceutical company focused on developing
innovative therapies for rare, genetic neurological diseases, today
announced the initiation of a Phase 1/2 clinical study of their lead
drug candidate, NV1205, in patients with childhood cerebral
adrenoleukodystrophy (CCALD). NV1205 is being developed to target the
underlying metabolic defect in all phenotypes of X-linked
adrenoleukodystrophy (X-ALD) patients by facilitating the metabolism of
very long chain fatty acids (VLCFA) in the brain and adrenal tissue,
which are the two main sites of the disease's pathology. X-ALD is a
severe and often fatal neurodegenerative disease with limited treatment
options and a significant need for innovation. NV1205 has the potential
to significantly improve the current therapeutic landscape.

"Advancing our lead candidate NV1205 into clinical trials represents a
key milestone in the company's development. The study initiation in
Australia, Chile and the United Kingdom is just the first step in our
global clinical development strategy," said John Henderson, Chief
Development Officer of NeuroVia. "We believe NV1205 has real potential
to better serve the X-ALD community as a novel, pharmaceutical treatment
for all X-ALD patients."

The Phase 1/2 study (ClinicalTrials.gov Identifier: NCT03196765) is
designed to assess the safety, tolerability and pharmacokinetics of
increasing doses of NV1205 in patients diagnosed with CCALD and will be
conducted in eight different countries – Argentina, Australia, Chile,
Colombia, France, the Russian Federation, Ukraine and the United
Kingdom. The trial will include an open label dose escalation study of
multiple dose levels of NV1205 with a long-term treatment phase.

For more information and updates on the trial, please consult www.clinicaltrials.gov
or http://www.ccald.com.

About NV1205

NeuroVia's lead compound, NV1205, is being developed as a novel
therapeutic option for X-ALD patients of all phenotypes. NV1205 is a
synthetic small molecule drug candidate that aims to restore metabolic
activity within the cells by enabling them to process the detrimental,
accumulating very long chain fatty acids (VLCFA) and avoid the resulting
damage to neuronal and mitochondrial membranes altogether.

About X-ALD

X-linked adrenoleukodystrophy (X-ALD) is a rare, genetic disorder
affecting 1 in 17,000 people worldwide that causes adrenal gland
dysfunction and loss of the protective sheath that surrounds nerve
fibers in the central nervous system. The childhood version progresses
quickly and results in a severe neurodegenerative phenotype, which is
often fatal within three to five years after onset of initial symptoms.
The adult-onset version is characterized by numbness in the legs leading
to difficulty or inability to walk and urinary and bowel incontinence.
With no treatment options currently available for symptomatic X-ALD
patients, there exists an unmet medical need for a therapeutic benefit
for X-ALD patients.

About NeuroVia

NeuroVia, Inc. is committed to addressing unmet medical needs in
neurological diseases with the primary goal to arrest the onset of
devastating neurological deficits associated with X-ALD. For more
information, please visit: http://www.neurovia-inc.com/.

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