Adrenoleukodystrophy - Pipeline Insight, 2024

Adrenoleukodystrophy - Pipeline Insight, 2024

DelveInsight’s, “Adrenoleukodystrophy - Pipeline Insight, 2024” report provides comprehensive insights about 10+ companies and 11+ pipeline drugs in 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

Adrenoleukodystrophy: Understanding

Adrenoleukodystrophy: Overview

Adrenoleukodystrophy (ALD) is a rare, inherited disorder that primarily affects the nervous system and adrenal glands. It is caused by a genetic defect that prevents the breakdown of very long-chain fatty acids (VLCFAs), leading to their accumulation in the brain, spinal cord, and adrenal glands. This buildup of VLCFAs damages the protective myelin sheath that insulates nerve cells, impairing their ability to transmit signals effectively. ALD is an X-linked recessive disorder, meaning it is caused by a mutation on the X chromosome. As a result, males are more severely affected than females, who are typically carriers of the disease. There are several forms of ALD, including the childhood cerebral form, adrenomyeloneuropathy (AMN), adrenal insufficiency only, and asymptomatic forms. Early diagnosis of ALD is crucial, as it allows for prompt treatment and management of symptoms. Treatment options include medications to manage symptoms, physical therapy, and in some cases, stem cell transplants to halt disease progression. However, there is currently no cure for ALD, and the prognosis varies depending on the form and severity of the disease.

Adrenoleukodystrophy presents with varying symptoms depending on the type and progression, ranging from behavioral problems and learning disabilities in the childhood cerebral form to walking difficulties, bowel/bladder dysfunction, and adrenal insufficiency in the adult-onset forms. The diverse spectrum of symptoms, which can include muscle weakness, seizures, vision/hearing loss, and paralysis, highlights the complexity of ALD and the need for personalized management strategies.

Treatment and management of adrenoleukodystrophy focus on slowing the progression of the disease and improving symptoms. For children diagnosed with ALD, a stem cell transplant may be a promising treatment to halt the progression of the disease. Medications and physical therapy are also used to manage symptoms, such as seizures, muscle weakness, and adrenal insufficiency. Additionally, supportive treatments like speech therapy, occupational therapy, and psychological counseling may be necessary to address the cognitive and behavioral changes associated with ALD. Investigational therapies, including gene therapy, are being explored to treat X-ALD. Early intervention is crucial, as it offers the best chance for successful treatment and slowing the disease's progression. A multidisciplinary care team, including neurologists, endocrinologists, geneticists, and other specialists, is essential for managing ALD and providing comprehensive care to affected individuals.

""Adrenoleukodystrophy - Pipeline Insight, 2024"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Adrenoleukodystrophy pipeline landscape is provided which includes the disease overview and Adrenoleukodystrophy treatment guidelines. The assessment part of the report embraces, in depth 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, 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 Adrenoleukodystrophy R&D. The therapies under development are focused on novel approaches to treat/improve Adrenoleukodystrophy.

Adrenoleukodystrophy Emerging Drugs Chapters

This segment of the Adrenoleukodystrophy report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II/III, 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.

Adrenoleukodystrophy Emerging Drugs

• Leriglitazone: Minoryx Therapeutics

Minoryx’s lead program is a selective, differentiated PPAR gamma agonist (leriglitazone) under development for the treatment of X-linked Adrenoleukodystrophy (X-ALD); it has the potential to treat both the severe cerebral form of ALD (cALD) and the chronic form, adrenomyeloneuropathy (AMN), as well as other orphan CNS diseases, such as Friedreich's Ataxia (FRDA) and Rett's disease. The drug is currently under preregistration stage of development for the treatment of patients with Adrenoleukodystrophy.

• VK0214: Viking Therapeutics

Viking is developing VK0214, which is a novel, orally available small molecule thyroid hormone receptor agonist that possesses selectivity for the beta receptor subtype. VK0214 has been granted orphan drug designation by the US FDA for the treatment of X-ALD. The drug is currently being evaluated under Phase I clinical trial for the treatment of patients with Adrenoleukodystrophy.

Further product details are provided in the report……..

Adrenoleukodystrophy: Therapeutic Assessment

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

• Major Players in Adrenoleukodystrophy

There are approx. 10+ key companies which are developing the therapies for Adrenoleukodystrophy. The companies which have their Adrenoleukodystrophy drug candidates in the most advanced stage, i.e. Preregistration include, Minoryx Therapeutics.

• Phases

DelveInsight’s report covers around 11+ 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

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

• Intravenous
• Subcutaneous
• Oral
• Intramuscular
• Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal antibody
• Small molecule
• Peptide
• Product Type

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

Adrenoleukodystrophy: Pipeline Development Activities

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

Adrenoleukodystrophy Report Insights

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

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

Key Players

• Minoryx Therapeutics
• Viking Therapeutics
• Poxel
• MedDay Pharmaceuticals
• SwanBio Therapeutics

Key Products

• Leriglitazone
• VK0214
• PXL-770
• MD1003
• SBT101