Neurofibromatosis - Pipeline Insight, 2024

This “Neurofibromatosis - Pipeline Insight, 2024,” report provides comprehensive insights about 15+ companies and 17+ pipeline drugs in Neurofibromatosis 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

Neurofibromatosis Understanding

Neurofibromatosis: Overview

Neurofibromatosis (NF) is one of the most common genetic disorders. Inherited in an autosomal dominant fashion, this phacomatosis is classified into two genetically distinct subtypes characterized by multiple cutaneous lesions and tumors of the peripheral and central nervous system. Neurofibromatosis type 1 (NF1), also referred to as Recklinghausen's disease, affects about 1 in 3500 individuals and presents with a variety of characteristic abnormalities of the skin and the peripheral nervous system. Neurofibromatosis type 2 (NF2), previously termed central neurofibromatosis, is much more rare occurring in less than 1 in 25 000 individuals. Often first clinical signs of NF2 become apparent in the late teens with a sudden loss of hearing due to the development of bi-or unilateral vestibular schwannomas. In addition NF2 patients may suffer from further nervous tissue tumors such as meningiomas or gliomas.

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

Neurofibromatosis Emerging Drugs Chapters

This segment of the Neurofibromatosis 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.

Neurofibromatosis Emerging Drugs

NFX-179: NFlection TherapeuticsNFlection is developing a topical gel containing NFX-179, a proprietary “soft” (metabolically labile) MEK inhibitor for the reduction of tumor burden of persistently developing cutaneous neurofibromas (cNF) in neurofibromatosis type 1 (NF1). NFlection has developed an esthetically pleasing and well-tolerated topical formulation of the MEK inhibitor NFX-179 to treat cNFs. NFX-179 has been tested on human cNF explants and has demonstrated dose-dependent suppression of p-ERK, a biomarker of Ras/Raf/MEK/ERK pathway activation. The NFX-179 gel formulation can penetrate into the dermis of the skin to locally deliver the MEK inhibitor to the cNF and inhibit the overactive RAS pathway in the tumor. NFX-179 rapidly degrades upon reaching systemic circulation to minimize the side effects caused by systemic exposure to MEK inhibitors.

Mirdametinib: SpringWorks TherapeuticsMirdametinib is an oral, small molecule MEK inhibitor in development as a monotherapy treatment for neurofibromatosis type 1-associated plexiform neurofibromas, or NF1-PN, and as a combination therapy for the treatment biomarker defined metastatic cancers with mutations in the MAPK pathway, such as in RAS and RAF. Mirdametinib is designed to inhibit MEK1 and MEK2. MEK proteins occupy a pivotal position in the MAPK pathway, which is a key signaling network that regulates cell growth and survival, and that plays a central role in multiple oncology and rare disease indications. The U.S. Food and Drug Administration (FDA) and the European Commission granted Orphan Drug designation for mirdametinib for the treatment of NF1, and the FDA granted Fast Track designation for the treatment of patients = 2 years of age with NF1-PN that are progressing or causing significant morbidity.

Brigatinib: Takeda
Brigitanib acts as a tyrosine kinase inhibitor with activity against multiple kinases including ALK, ROS1, and insulin-like growth factor 1 receptor and against EGFR deletions and point mutations. It acts by inhibiting ALK phosphorylation and the activation of downstream signaling proteins. It presents selectivity against the mutant forms of EGFR compared to the wild-type. It also exhibits selectivity against 9 different Crizotinib-resistant mutants of the EML4-ALK fusion gene, which is a pivotal player in the transformation of susceptible lung parenchyma. Brigatinib was developed by Ariad Pharmaceuticals, a subsidiary of Takeda Pharmaceutical Company Limited, and FDA-approved on April 28, 2017. It is currently being evaluated in Phase II stage of development for the treatment of Neurofibromatosis Type II.

Neurofibromatosis: Therapeutic Assessment

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

Major Players in Neurofibromatosis

There are approx. 15+ key companies which are developing the therapies for Neurofibromatosis. The companies which have their Neurofibromatosis drug candidates in the most advanced stage, i.e. phase II include, NFlection Therapeutics.

Phases

This report covers around 17+ 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

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

• Oral
• Parenteral
• intravenous
• Subcutaneous
• Topical.

Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy

Product Type

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

Neurofibromatosis: Pipeline Development Activities

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

Neurofibromatosis Report Insights

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

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

Key Players

• NFlection Therapeutics
• SpringWorks Therapeutics
• Shanghai Fosun Pharmaceutical Development Co, Ltd.
• Array BioPharma
• Recursion Pharmaceuticals
• Zhejiang Betta Pharma
• Healx
• INTiDYN
• Takeda
• Chia Tai Tianqing Pharmaceutical Group
• Novartis

Key Products

• NFX-179
• Mirdametinib
• FCN-159
• Binimetinib
• REC 2282
• Icotinib
• AL2846
• RAD001

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