Cerebral Adrenoleukodystrophy - Pipeline Insight, 2024

This “Cerebral Adrenoleukodystrophy - Pipeline Insight, 2024,” report provides comprehensive insights about 3+ companies and 3+ pipeline drugs in Cerebral 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

Cerebral Adrenoleukodystrophy Understanding

Cerebral Adrenoleukodystrophy: Overview

Cerebral adrenoleukodystrophy - or CALD - is a rare and devastating neurologic disease that robs young patients of the chance to live a full life. The disease results in rapid loss of neurological function after the initial onset of symptoms and sadly, nearly half of patients who do not receive treatment die within five years of symptom onset. Adrenoleukodystrophy (ALD) is a rare, X-linked, metabolic disorder caused by a mutation in the ABCD1 gene which results in the toxic buildup of very long-chain fatty acids (VLCFA) in the brain and spinal cord. CALD is the most severe form of this neurodegenerative condition. The accumulation of VLCFA in the adrenal cortex and white matter of the brain and spinal cord leads to the progressive destruction of myelin, the protective sheath of the nerve cells in the brain that are responsible for thinking and muscle control. Without myelin, these nerves can no longer relay information to and from the brain. For many boys, adrenal insufficiency or behavioral problems are the first detected symptoms, preceding the onset of neurologic symptoms. As the disease progresses, boys develop vision and hearing problems, seizures, poor coordination, and difficulty swallowing. ALD is estimated to affect 1 in 5,000 to 1 in 17,000 newborns (both male and female), and approximately 1 in 20,000 to 1 in 30,000 newborn males. CALD develops in approximately 40% of affected boys and in a smaller number of adult men.

Cerebral Adrenoleukodystrophy - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Cerebral Adrenoleukodystrophy pipeline landscape is provided which includes the disease overview and Cerebral Adrenoleukodystrophy treatment guidelines. The assessment part of the report embraces, in depth Cerebral 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, Cerebral Adrenoleukodystrophy collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

• A better understanding of disease pathogenesis contributing to the development of novel therapeutics for Cerebral Adrenoleukodystrophy.
• In the coming years, the Cerebral Adrenoleukodystrophy market is set to change due to the rising awareness of the disease, and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market.

The companies and academics that are working to assess challenges and seek opportunities that could influence Cerebral Adrenoleukodystrophy R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.

• A detailed portfolio of major pharma players who are involved in fueling the Cerebral Adrenoleukodystrophy treatment market. Several potential therapies for Cerebral Adrenoleukodystrophy are under investigation. With the expected launch of these emerging therapies, it is expected that there will be a significant impact on the Cerebral Adrenoleukodystrophy market size in the coming years.
• This in-depth analysis of the pipeline assets (in early-stage, mid-stage and late stage of development for the treatment of Cerebral Adrenoleukodystrophy) includes therapeutic assessment and comparative analysis. This will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities.

Cerebral Adrenoleukodystrophy Emerging Drugs Chapters

This segment of the Cerebral 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.

Cerebral Adrenoleukodystrophy Emerging Drugs

MIN-102: 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.

PXL770: POXEL SAPXL770 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 (slide below). Based on these observations, PXL770 was evaluated in both the in vitro and in vivo ALD models (charts below). 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 (not shown). Additional experimental results included an observed improvement in neural histology and neuro-behavior in ABCD1 null mice following chronic PXL770 treatment.

Cerebral Adrenoleukodystrophy: Therapeutic Assessment

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

Major Players in Cerebral Adrenoleukodystrophy

There are approx. 3+ key companies which are developing the therapies for Cerebral Adrenoleukodystrophy. The companies which have their Cerebral 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

Cerebral 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

• Inhalation
• Inhalation/Intravenous/Oral
• Intranasal
• Intravenous
• Intravenous/ Subcutaneous
• NA
• Oral
• Oral/intranasal/subcutaneous
• Parenteral
• Subcutaneous

Molecule Type

Products have been categorized under various Molecule types such as

• Antibody
• Antisense oligonucleotides
• Immunotherapy
• Monoclonal antibody
• Peptides
• Protein
• Recombinant protein
• Small molecule
• Stem Cell
• Vaccine

Product Type

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

Cerebral Adrenoleukodystrophy: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Cerebral 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 Cerebral Adrenoleukodystrophy drugs.

Cerebral Adrenoleukodystrophy Report Insights

• Cerebral Adrenoleukodystrophy Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Cerebral 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 Cerebral Adrenoleukodystrophy drugs?
• How many Cerebral Adrenoleukodystrophy drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Cerebral Adrenoleukodystrophy?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Cerebral 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 Cerebral Adrenoleukodystrophy and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Minoryx Therapeutics
• POXEL SA
• Orpheris, Inc.
• bluebird bio, Inc.

Key Products

• MIN-102
• PXL770
• OP-101
• elivaldogene autotemcel (eli-cel)