X-linked Adrenoleukodystrophy Pipeline Market Research Report 2022 – ResearchAndMarkets.com

DUBLIN–(BUSINESS WIRE)–The “X linked Adrenoleukodystrophy – Pipeline Insight, 2022” drug pipelines has been added to ResearchAndMarkets.com’s offering.

The “X linked Adrenoleukodystrophy – Pipeline Insight, 2022” report provides comprehensive insights about 3+ companies and 3+ pipeline drugs in X linked Adrenoleukodystrophy pipeline landscape.

It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Report Highlights

Companies and academics are working to assess challenges and seek opportunities that could influence X linked Adrenoleukodystrophy R&D. The therapies under development are focused on novel approaches to treat/improve X linked Adrenoleukodystrophy.

X linked Adrenoleukodystrophy Emerging Drugs Chapters

This segment of the X linked Adrenoleukodystrophy report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

X linked Adrenoleukodystrophy Emerging Drugs

Leriglitazone: Minoryx Therapeutics

Leriglitazone (MIN-102) is a novel, orally bioavailable and selective PPAR gamma agonist with a potential best-in-class profile indicated for CNS diseases. It is one of the several metabolites of pioglitazone and has a demonstrated sufficient brain penetration and favorable safety profile in humans, allowing PPAR gamma engagement in the CNS above the level that can be safely achieved with pioglitazone and other glitazones.

It showed robust preclinical proof-of-concept in animal models of multiple diseases by modulating pathways leading to mitochondrial dysfunction, oxidative stress, neuroinflammation, demyelination and axonal degeneration. The drug is currently in Phase2/3 stage of development for the treatment of Adrenoleukodystrophy.

PXL770: Poxel

PXL770 is a novel drug candidate that directly activates adenosine monophosphate-activated protein kinase (AMPK). AMPK is a central regulator of multiple metabolic pathways leading to the control of multiple metabolic pathways and reduced inflammation. The rationale for considering AMPK activators in ALD is based on several published findings that show links between AMPK and disease in both animals and humans.

PXL770 was evaluated in both the in vitro and in vivo ALD models . PXL770 suppressed elevated VLCFA levels in patient derived cells with an associated increase in expression of the compensatory ABCD2 transporter. PXL770 treatment of ABCD1 null mice also suppressed elevated VLCFA in spinal cord – and in brain and plasma.

PLX065: Poxel

PXL065 offers a new approach for the treatment of ALD. PXL065 is the R-stereoisomer (deuterium-modified single R-isomer) of pioglitazone and its parent molecule has been marketed since 1999 for the treatment of type 2 diabetes. Published literature provides a potential rationale for considering D-TZDs in ALD. Firstly, pioglitazone has demonstrated strong efficacy in the classical ALD model, the ABCD1 null mouse; in addition, pioglitazone has been shown to confer neuroprotection in other contexts.

Also, a key non-genomic mechanisms modulated by pioglitazone and PXL065 (and other D-TZDs) – the mitochondrial pyruvate carrier – MPC – is also implicated as a target for neurodegeneration. In February 2022, PXL065 was granted Fast Track Designation (FTD) by the U.S. Food and Drug Administration (FDA) for the treatment of patients with adrenomyeloneuropathy (AMN), the most common form of X-linked adrenoleukodystrophy (ALD).

OP101: Orpheris

OP-101 is a new chemical entity consisting of N-acetyl cysteine (NAC) covalently coupled to a metabolically – stable inactive dendrimer. Studies in several small and large pre-clinical models have demonstrated the selective endocytosis uptake of OP-101 by activated microglia/ and astrocytes upon intravenous administration, localizing only in brain regions where there is with neuroinflammation-induced BBB impairment of the blood brain barrier.

OP-101 releases NAC intracellularly in activated microglia and astrocytes, which then acts to reduce the attenuating oxidative stress and inflammation, in these cells and leads producing to significant improvements in neurobehavioral outcomes in the preclinical models, unlike the free drug. Macrophages isolated from ccALD patients and stimulated with VLCFA have significantly reduced cytokine expression and glutamate secretion with increased glutathione levels, when treated ex vivo with OP-101. Pilot toxicity studies in juvenile rats show that no toxicity of OP-101 is safe at doses as high as even at 1000 mg/kg IV QOD.

X linked Adrenoleukodystrophy: Therapeutic Assessment

This segment of the report provides insights about the different X linked Adrenoleukodystrophy drugs segregated based on the following parameters that define the scope of the report, such as:

Major Players in X linked Adrenoleukodystrophy

There are approx. 3+ key companies which are developing the therapies for X linked Adrenoleukodystrophy. The companies which have their X linked Adrenoleukodystrophy drug candidates in the most advanced stage, i.e. phase II/ III include, Minoryx Therapeutics.

Key Players

• Minoryx Therapeutics
• Viking Therapeutics
• Poxel
• Orpheris
• Applied Genetic Technologies Corporation
• NEURALGENE

Key Products

• PXL770
• PXL065
• OP101
• Leriglitazone
• PRCN 323

For more information about this drug pipelines report visit https://www.researchandmarkets.com/r/m9895a

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