X linked Adrenoleukodystrophy - Pipeline Insight, 2024

This “X linked Adrenoleukodystrophy- Pipeline Insight, 2024,” 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.

Geography Covered

• Global coverage

X linked Adrenoleukodystrophy Understanding

X linked Adrenoleukodystrophy: Overview

X linked adrenoleukodystrophy (X-ALD) is a genetic disease that affects the nervous system and the adrenal glands (small glands located on top of each kidney). People with this disease often have progressive loss of the fatty covering (myelin) that surrounds the nerves in the brain and spinal cord. They may also have a shortage of certain hormones that is caused by damage to the outer layer of the adrenal glands (adrenal cortex). This is called adrenocortical insufficiency, or Addison disease. There are three forms of X-ALD: a childhood cerebral form, an adrenomyeloneuropathy (AMN) type, and an adrenal-insufficiency-only-type. The disease primarily affects males. X-ALD is caused by a variation (mutation) in the ABCD1 gene and it is inherited in an X-linked. manner. Diagnosis of the disease is based on testing the levels of a molecule called very long-chain fatty acids (VLCFA). The diagnosis can be confirmed with genetic testing. There is still no cure for X-ALD, but taking special oils such as Lorenzo’s oil can lower the blood levels of VLCFA. Bone marrow transplantation may be an option for boys who have evidence of brain involvement on MRI, but do not yet have obvious symptoms of the disease with a normal neurological exam. With an estimated birth incidence of 1 in 17,000 newborns (male and female), X-ALD is the most common peroxisomal disorder. It occurs in all regions of the world. Now that newborn screening has become technically feasible and may be implemented in some parts of the world. The treatment for X-linked adrenoleukodystrophy (X-ALD) depends on the signs and symptoms present in each person. It is important to remember that the exact signs and symptoms of a person with X-ALD cannot be predicted by the signs and symptoms of other members of the family. Therefore, the long-term outlook for individuals who have X-ALD within the same family can be very different. As in many X-linked diseases, it was assumed that female carriers remain asymptomatic. An increasing number of symptomatic heterozygous women are identified as the first member of their family to be affected by X-ALD. A diagnosis of X-ALD must be followed by extended family screening together with a geneticist.

X linked Adrenoleukodystrophy- Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the X linked Adrenoleukodystrophy pipeline landscape is provided which includes the disease overview and X linked Adrenoleukodystrophy treatment guidelines. The assessment part of the report embraces, in depth X linked Adrenoleukodystrophy commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, X linked Adrenoleukodystrophy collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The 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 andplasma.

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 cc ALD 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/kgIVQOD.

X linked Adrenoleukodystrophy: Therapeutic Assessment

This segment of the report provides insights about the different X linked Adrenoleukodystrophy drugs segregated based on 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.

Phases

This report covers around 3+ products under different phases of clinical development like

• 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

Route of Administration

X linked Adrenoleukodystrophy pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Ophthalmic
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal

Molecule Type

Products have been categorized under various Molecule types such as

• Oligonucleotide
• Peptide
• Small molecule

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

X linked Adrenoleukodystrophy: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses X linked Adrenoleukodystrophy therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging X linked Adrenoleukodystrophy drugs.

X linked Adrenoleukodystrophy Report Insights

• X linked Adrenoleukodystrophy Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

X linked Adrenoleukodystrophy Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing X linked Adrenoleukodystrophy drugs?
• How many X linked Adrenoleukodystrophy drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of X linked Adrenoleukodystrophy?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the X linked Adrenoleukodystrophy therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for X linked Adrenoleukodystrophy and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

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

Key Products

• PXL770
• PXL065
• OP101
• Leriglitazone
• PRCN 323

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