Frontotemporal Dementia - Pipeline Insight, 2024

This “Frontotemporal Dementia- Pipeline Insight, 2024” report provides comprehensive insights about 25+ companies and 25+ pipeline drugs in Frontotemporal Dementia 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

Frontotemporal Dementia: Understanding

Frontotemporal Dementia: Overview

Frontotemporal dementia (FTD) or frontotemporal degeneration refers to a group of disorders caused by progressive nerve cell loss in the brain's frontal lobes (the areas behind your forehead) or its temporal lobes (the regions behind your ears).

There are a number of different diseases that cause frontotemporal degeneration. The two most prominent are 1) a group of brain disorders involving the protein tau and 2) a group of brain disorders involving the protein called TDP43. For reasons that are not yet known, these two groups have a preference for the frontal and temporal lobes that cause dementia.

Types
There are three types of frontotemporal disorders (FTD): behavioral variant frontotemporal dementia (bv FTD), primary progressive aphasia (PPA), and movementdisorders.

Behavioral variant frontotemporal dementia (bv FTD), sometimes also called behavior variant FTD, is characterized by prominent changes in personality and behavior that often occur in people in their 50s and 60s, but can develop as early as their 20s or as late as their 80s. In behavioral variant frontotemporal dementia, the nerve cell loss is most prominent in areas that control conduct, judgment, empathy and foresight, among otherabilities.

Primary progressive aphasia (PPA) is the second major form of frontotemporal degeneration that affects language skills, speaking, writing and comprehension. PPA normally comes on in midlife, before age 65, but can occur in late life also. The two most distinctive forms of PPA have somewhat different symptoms:
? In semantic variant of PPA, individuals lose the ability to understand or formulate words in a spoken sentence.

? In nonfluent/agrammatic variant of PPA, a person’s speaking is very hesitant, labored or ungrammatical.

Causes and risks
Frontotemporal degeneration is inherited in about a third of all cases. Genetic counseling and testing are available now for individuals with family histories of frontotemporal degeneration. There are no known risk factors for any frontotemporal degeneration except for a family history or a similar disorder.

Diagnosis
The diagnosis of behavioral variant frontotemporal dementia and PPA are based on expert evaluation by a doctor who is familiar with these disorders. The type of problems experienced by the patient and the results of neurological exams are the core of the diagnosis. Brain scans such as magnetic resonance imaging (MRI) and glucose positron emission scans are very helpful additional tests, but they must be interpreted in the context of the patient’s history and neurological exam.

Treatment
There are no specific treatments for any of the frontotemporal subtypes. There are medications that can reduce agitation, irritability and/or depression. These treatments should be used to help improve quality of life.

Frontotemporal dementia inevitably gets worse over time and the speed of decline differs from person to person. For many years, individuals with frontotemporal dementia show muscle weakness and coordination problems, leaving them needing a wheelchair - or unable to leave the bed.

These muscle issues can cause problems swallowing, chewing, moving and controlling bladder and/or bowels. Eventually, people with frontotemporal degeneration die because of the physical changes that can cause skin, urinary tract and/or lung infection.

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

Frontotemporal Dementia Emerging Drugs Chapters

This segment of the Frontotemporal Dementia 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.

Frontotemporal Dementia Emerging Drugs

PR 006: Prevail Therapeutics
PR006 is being developed as a potentially disease-modifying, single-dose gene therapy for patients with frontotemporal dementia with GRN mutations (FTD-GRN). PR006 is designed to slow or stop disease progression in FTD-GRN patients by increasing progranulin levels via delivery of a healthy GRN gene into the central nervous system (CNS). PR006 is administered by injection into the cisterna magna, using the well-studied viral vector AAV9.The U.S. FDA has granted Orphan Drug designation for the treatment of FTD and Fast Track designation for the treatment of FTD-GRN. The European Commission has granted orphan designation for PR006 for the treatment of FTD.PR006 is currently being studied in our PROCLAIM trial, a Phase 1/2 clinical trial of PR006 for the treatment of patients with FTD-GRN.

Latozinemab: Alector
AL001 modulates progranulin (PGRN), a key regulator of immune activity in the brain with genetic links to multiple neurodegenerative disorders. We are initially developing AL001 for frontotemporal dementia (FTD) with progranulin mutation (FTD-GRN). The global INFRONT-3 Phase 3 clinical trial is currently enrolling both at-risk and symptomatic participants with FTD-GRN.AL001 is being developed in collaboration with GSK.

PBFT-02: Passage Bio
Passage Bio, gene therapy product candidate, PBFT02, an AAV1 viral vector to deliver a modified DNA encoding the granulin gene (GRN) to a patient’s cells. The vector will be delivered directly to the cerebrospinal fluid by a single injection to the cisterna magna (ICM injection). The goal of this vector and delivery approach is to provide higher-than-normal levels of the progranulin protein (PGRN) to the CNS to overcome the progranulin deficiency in GRN gene mutation carriers. Currently the product is in Phase I/II for the treatment of Frontotemporal Dementia.

WVE-004: WaVe life Sciences
WVE-004 is an investigational variant-selective silencing candidate designed to selectively target the transcript variants containing a hexanucleotide repeat expansion (G4C2) in the C9orf72 gene, while sparing the healthy C9orf72 protein. G4C2 expansions are one of the most common genetic causes of the sporadic and inherited forms of ALS and FTD.

EXO-050: Coya Therapeutics
EXO 050, is the lead drug candidate of Coya Therapeutics for the treatment of FTD. Currently, the drug is in Pre-Clinical stage of development for the treatment of FTD. .

Frontotemporal Dementia: Therapeutic Assessment

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

Major Players in Frontotemporal Dementia

There are approx. 25+ key companies which are developing the therapies for Frontotemporal Dementia. The companies which have their Frontotemporal Dementia drug candidates in the most advanced stage, i.e. phase III include, Deerland Probiotics & Enzymes.

Phases

This report covers around 25+ 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

Frontotemporal Dementia 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.

Frontotemporal Dementia: Pipeline Development Activities

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

Frontotemporal Dementia Report Insights

• Frontotemporal Dementia Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Prevail Therapeutics
• Coya Therapeutics
• Alector, Inc.
• Passage Bio
• WaVe life Sciences
• CAMP4 Therapeutics
• SOLA Biosciences
• Denali Therapeutics
• Neurimmune
• Autifony Therapeutics

Key Products

• PR 006
• EXO-050
• AL001
• PBFT02
• WVE-004
• Research program
• SOL-257
• Progranulin
• NI308
• Research program (Novel genetically linked ion channel)

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