Encephalopathy - Pipeline Insight, 2024

This “Encephalopathy- Pipeline Insight, 2024” report provides comprehensive insights about 70+ companies and 70+ pipeline drugs in Encephalopathy 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

Encephalopathy: Understanding

Encephalopathy: Overview

Encephalopathy is a broad term used to describe abnormal brain function or brain structure. (Encephalo= brain + pathy= disorder). The abnormality may be transient, recurrent, or permanent. The loss of brain function may be reversible, static and stable, or progressive with increasing loss of brain activity over time. There are two main types of encephalopathy: reversible and irreversible. Reversible causes include: Hepatic encephalopathy, Hashimoto’s encephalopathy, metabolic encephalopathy, infections of the brain, such as encephalitis or meningitis, or in another part of the body, such as a urinary tract infection. An extreme response to an infection, called sepsis, can also lead to encephalopathy. Types of encephalopathy that are irreversible include: Chronic traumatic encephalopathy, Hypoxic-ischemic encephalopathy and many others. The symptoms depend on the type and cause of encephalopathy, but some of the most common ones are: Confusion, memory loss, personality changes and trouble thinking clearly or focusing. Some people will also have: Trouble speaking, muscle weakness or twitches, eye movements which can’t be control, tremors and trouble swallowing. Any patient with unexplained confusion, altered mental function, coma, seizure, or weakness should access emergency care immediately. The diagnosis of encephalopathy begins with the history provided by the patient, family, or friends. The health care professional will have a better understanding about potential causes of the encephalopathy based upon information regarding symptoms, for example, how quickly they appeared, how long they have been present, whether they wax and wane, or whether there is a progressive downward course. Past history of medical illness, intentional or unintentional ingestions, prescribed medications, chemical exposure, and infections may provide clues as to why the patient has encephalopathy. Imaging studies such as X-rays, computerized tomography (CT), and magnetic resonance imaging (MRI) may be useful depending upon the clinical situation. Lumbar puncture (spinal tap) may be helpful to look for infection, bleeding, and inflammation. The cerebrospinal fluid can also be analyzed for abnormal proteins, chemicals, and cells. Patients who have known recurrent encephalopathy may have home treatment programs that may be attempted.

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

Encephalopathy Emerging Drugs Chapters

This segment of the Encephalopathy 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.

Encephalopathy Emerging Drugs

XEN496: Xenon Pharmaceuticals XEN496, a Kv7 potassium channel opener, is a proprietary pediatric formulation of the active ingredient ezogabine being developed for the treatment of KCNQ2 developmental and epileptic encephalopathy (KCNQ2-DEE).Xenon has received Fast Track designation and Orphan Drug Designation for XEN496 for the treatment of seizures associated with KCNQ2-DEE from the U.S. Food and Drug Administration (FDA), as well as orphan medicinal product designation from the European Commission.

AXA 1665: Axcella Health AXA1665 is an EMM composition of 8 amino acids and derivatives that targets three key nodes: ammonia toxicity, amino acid imbalance, and muscle wasting. Asp and Orn are intended to promote the function of the urea cycle. AXA1665 is designed to support pathways related to ammonia handling: Asp and Orn promote the function of the urea cycle and improved muscle mass and function promote ammonia handling within skeletal muscle. Currently, the drug is in Phase II stage of development for the treatment of HepaticEncephalopathy.

RBX 7455: Rebiotix Rebiotix Inc., a Ferring Company, has leveraged its clinical, manufacturing and regulatory experience with RBX2660 to develop the first of its kind non-frozen, room temperature stable oral microbiota-based formulation under the MRT™ drug platform, known as RBX7455.Currently, the drug is in Phase II stage of clinical trial evaluation for the treatment of HepaticEncephalopathy.

Encephalopathy: Therapeutic Assessment

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

Major Players in Encephalopathy

There are approx. 70+ key companies which are developing the therapies for Encephalopathy. The companies which have their Encephalopathy drug candidates in the most advanced stage, i.e. phase III include, Xenon Pharmaceuticals.

Phases

This report covers around 70+ 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

Encephalopathy 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.

Encephalopathy: Pipeline Development Activities

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

Encephalopathy Report Insights

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

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

Key Players

• Xenon Pharmaceuticals
• Rebiotix
• Axcella Health
• Neurocrine Biosciences
• Takeda
• Praxis Precision Medicines
• Stoke Therapeutics
• Longboard Pharmaceuticals
• Knoop Biosciences
• Praxis Precision Medicines
• Q-State Biosciences, Inc
• Ocera Therapeutics

Key Products

• NBI-921352
• XEN496
• TAK-935
• PRAX562
• STK001
• LP352
• KB-3061
• PRAX222
• SCNA2 LoF ASO
• Researchprogramme:DEE13-SCN8A(ASO)
• AXA 1665
• RBX 7455

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