X-Linked Adrenoleukodystrophy Pipeline Insight 2025: A New Era of Gene Therapy and Novel Therapeutics to Halt Disease Progression | DelveInsight

X-Linked Adrenoleukodystrophy (X-ALD) is a rare, inherited metabolic disorder caused by mutations in the ABCD1 gene, leading to the accumulation of very long-chain fatty acids (VLCFAs) that damage the adrenal cortex and white matter of the brain. The disorder predominantly affects males and manifests in multiple phenotypes, including the severe childhood cerebral form, adrenomyeloneuropathy (AMN), and Addison-only disease. Without timely intervention, cerebral X-ALD can progress rapidly, leading to severe neurological decline and death.

Until recently, hematopoietic stem cell transplantation (HSCT) was the only curative option, primarily effective when performed at early disease stages. However, recent advances in gene therapy have reshaped the therapeutic landscape. Bluebird Bio's elivaldogene autotemcel (eli-cel), an ex vivo lentiviral gene therapy, received EMA approval under the brand Skysona and is under FDA review for the U.S. market. This one-time therapy shows promise in halting disease progression in early cerebral ALD by inserting a functional ABCD1 gene into the patient's hematopoietic stem cells.

Beyond gene therapy, pipeline programs include small molecules that modulate peroxisomal function and metabolic pathways to reduce VLCFA levels. Novel approaches like CRISPR-based gene editing and antisense oligonucleotides (ASOs) are being explored in preclinical stages, offering hope for less invasive or systemic treatments that address both cerebral and spinal forms of the disease.

By 2025, multiple trials across North America and Europe are anticipated to yield pivotal data, especially regarding long-term safety and durability of gene-modified cell therapies. In parallel, natural history studies and biomarker discovery efforts are helping refine patient stratification and earlier diagnosis, critical for timely therapeutic intervention. The expanding pipeline reflects a deepening commitment to address X-ALD at the molecular level, offering renewed hope to patients and families navigating this devastating disease.

Interested in learning more about the current treatment landscape and the key drivers shaping the X-Linked Adrenoleukodystrophy pipeline? Click here: https://www.delveinsight.com/report-store/x-linked-adrenoleukodystrophy-x-ald-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Key Takeaways from the X-Linked Adrenoleukodystrophy Pipeline Report
• DelveInsight's X-Linked Adrenoleukodystrophy pipeline analysis depicts a strong space with 3+ active players working to develop 3+ pipeline drugs for X-Linked Adrenoleukodystrophy treatment.
• The leading X-Linked Adrenoleukodystrophy companies include Minoryx Therapeutics, Viking Therapeutics, Poxel, Orpheris, Applied Genetic Technologies Corporation, NEURALGENE, and others are evaluating their lead assets to improve the X-Linked Adrenoleukodystrophy treatment landscape.
• Key X-Linked Adrenoleukodystrophy pipeline therapies in various stages of development include PXL770, PXL065, OP101, Leriglitazone, PRCN 323, and others.
• In November 2023, Minoryx Therapeutics enrolled the first patients in the U.S. Phase III CALYX trial investigating its lead candidate, leriglitazone, for cerebral adrenoleukodystrophy (cALD) in adult male X‐ALD patients. The trial aims to confirm the disease-modifying potential observed in earlier studies.
• In January 2023, Results from Minoryx's Phase II/III ADVANCE trial of leriglitazone in male X‐ALD patients were published in The Lancet Neurology, showing the drug was well tolerated and slowed progression of cerebral lesions and myelopathy. These findings support regulatory filings and highlight the potential for improving clinical outcomes in X‐ALD.

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X-Linked Adrenoleukodystrophy Overview
X-Linked Adrenoleukodystrophy (X-ALD) is a rare genetic disorder affecting the nervous system and adrenal glands. It is caused by mutations in the ABCD1 gene, leading to the accumulation of very long-chain fatty acids (VLCFAs) that damage myelin-the protective coating of nerve cells-and impair adrenal function. X-ALD primarily affects males and presents with a wide range of symptoms, including progressive neurological decline, adrenal insufficiency, and behavioral changes. The most severe form, cerebral ALD, leads to rapid brain degeneration in childhood. Currently, treatment options are limited and focus on managing symptoms, hormone replacement for adrenal insufficiency, and hematopoietic stem cell transplantation in early stages. Research is ongoing to develop targeted therapies to slow or halt disease progression.

Find out more about X-Linked Adrenoleukodystrophy medication at https://www.delveinsight.com/report-store/x-linked-adrenoleukodystrophy-x-ald-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

X-Linked Adrenoleukodystrophy Treatment Analysis: Drug Profile
Leriglitazone: Minoryx Therapeutics
Leriglitazone (MIN-102) is a novel, orally bioavailable, selective PPAR gamma agonist with potential best-in-class properties for CNS diseases. As a metabolite of pioglitazone, it demonstrates strong brain penetration and a favorable safety profile, enabling effective PPAR gamma activation in the CNS at levels beyond those achievable with pioglitazone and other glitazones. Preclinical studies show it modulates pathways involved in mitochondrial dysfunction, oxidative stress, neuroinflammation, demyelination, and axonal degeneration. Leriglitazone is currently in Phase 2/3 development for treating Adrenoleukodystrophy.

PXL770: Poxel
PXL770 is a novel activator of adenosine monophosphate-activated protein kinase (AMPK), a key regulator of metabolic pathways and inflammation. AMPK activation has been linked to potential benefits in ALD based on animal and human studies. In vitro and in vivo ALD models demonstrated that PXL770 reduces elevated very long-chain fatty acids (VLCFAs) in patient-derived cells and increases expression of the compensatory ABCD2 transporter. Treatment with PXL770 in ABCD1-null mice lowered VLCFA levels in the spinal cord, brain, and plasma.

PXL065: Poxel
PXL065 is the R-stereoisomer of pioglitazone, modified with deuterium, offering a new therapeutic approach for ALD. Pioglitazone, marketed since 1999 for type 2 diabetes, has shown efficacy in ALD animal models and neuroprotective effects in other conditions. PXL065 targets the mitochondrial pyruvate carrier (MPC), implicated in neurodegeneration. In February 2022, PXL065 received Fast Track Designation from the FDA for treating adrenomyeloneuropathy (AMN), the most common form of X-linked adrenoleukodystrophy.

Learn more about the novel and emerging X-Linked Adrenoleukodystrophy pipeline therapies at https://www.delveinsight.com/report-store/x-linked-adrenoleukodystrophy-x-ald-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

X-Linked Adrenoleukodystrophy Therapeutics Assessment
By Product Type
• Mono
• Combination
• Mono/Combination.

By Stage
• Late-stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

By Route of Administration
• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Ophthalmic
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal

By Molecule Type
• Oligonucleotide
• Peptide
• Small molecule

Scope of the X-Linked Adrenoleukodystrophy Pipeline Report
• Coverage: Global
• Key X-Linked Adrenoleukodystrophy Companies: Minoryx Therapeutics, Viking Therapeutics, Poxel, Orpheris, Applied Genetic Technologies Corporation, NEURALGENE, and others.
• Key X-Linked Adrenoleukodystrophy Pipeline Therapies: PXL770, PXL065, OP101, Leriglitazone, PRCN 323, and others.

To dive deep into rich insights for drugs used for X-Linked Adrenoleukodystrophy treatment, visit: https://www.delveinsight.com/report-store/x-linked-adrenoleukodystrophy-x-ald-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Table of Contents
1. Introduction
2. Executive Summary
3. X-Linked Adrenoleukodystrophy Pipeline: Overview
4. Analytical Perspective In-depth Commercial Assessment
5. X-Linked Adrenoleukodystrophy Pipeline Therapeutics
6. X-Linked Adrenoleukodystrophy Pipeline: Late-Stage Products (Phase III)
7. X-Linked Adrenoleukodystrophy Pipeline: Mid-Stage Products (Phase II)
8. X-Linked Adrenoleukodystrophy Pipeline: Early Stage Products (Phase I)
9. Therapeutic Assessment
10. Inactive Products
11. Company-University Collaborations (Licensing/Partnering) Analysis
12. Key Companies
13. Key Products
14. Unmet Needs
15. Market Drivers and Barriers
16. Future Perspectives and Conclusion
17. Analyst Views
18. Appendix

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This release was published on openPR.