Neurofibromatosis Type 1 - Pipeline Insight, 2024

This “Neurofibromatosis Type 1 - Pipeline Insight, 2024,” report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in Neurofibromatosis Type 1 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 Type 1 Understanding

Neurofibromatosis Type 1: Overview

Neurofibromatosis type 1 is a condition characterized by changes in skin coloring (pigmentation) and the growth of tumors along nerves in the skin, brain, and other parts of the body. The signs and symptoms of this condition vary widely among affected people. Beginning in early childhood, almost all people with neurofibromatosis type 1 have multiple café-au-lait spots, which are flat patches on the skin that are darker than the surrounding area. These spots increase in size and number as the individual grows older. Freckles in the underarms and groin typically develop later in childhood. Mutations in the NF1 gene cause neurofibromatosis type 1. The NF1 gene provides instructions for making a protein called neurofibromin. This protein is produced in many cells, including nerve cells and specialized cells surrounding nerves. Neurofibromatosis type 1 is considered to have an autosomal dominant pattern of inheritance. People with this condition are born with one mutated copy of the NF1 gene in each cell. In about half of cases, the altered gene is inherited from an affected parent. The remaining cases result from new mutations in the NF1 gene and occur in people with no history of the disorder in their family.

Neurofibromatosis Type 1 - Pipeline Insight, 2021 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Neurofibromatosis Type 1 pipeline landscape is provided which includes the disease overview and Neurofibromatosis Type 1 treatment guidelines. The assessment part of the report embraces, in depth Neurofibromatosis Type 1 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 Type 1 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 Type 1 R&D. The therapies under development are focused on novel approaches to treat/improve Neurofibromatosis Type 1.

Neurofibromatosis Type 1 Emerging Drugs Chapters

This segment of the Neurofibromatosis Type 1 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 Type 1 Emerging Drugs

Mirdametinib: Spring Works Therapeutics Mirdametinib 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 significantmorbidity.

NFX-179: NFlection Therapeutics NFX-179 is formulated in a cosmetically elegant gel that is applied to the skin, enabling local suppression of the Ras/Raf/MEK/ERK pathway that drives the pathology of rare cutaneous RASopathies. NFX-179 is a metabolically labile “soft drug” that is rapidly broken down in the bloodstream so it no longer has the ability to inhibit MEK systemically.

Neurofibromatosis Type 1: Therapeutic Assessment

This segment of the report provides insights about the different Neurofibromatosis Type 1 drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Neurofibromatosis Type 1

There are approx. 10+ key companies which are developing the therapies for Neurofibromatosis Type 1. The companies which have their Neurofibromatosis Type 1 drug candidates in the most advanced stage, i.e. phase II include, Spring WorksTherapeutics.

Phases

This report covers around 10+ 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 Type 1 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 Type 1: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Neurofibromatosis Type 1 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 Type 1 drugs.

Neurofibromatosis Type 1 Report Insights

• Neurofibromatosis Type 1 Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Neurofibromatosis Type 1 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 Type 1 drugs?
• How many Neurofibromatosis Type 1 drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Neurofibromatosis Type 1?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Neurofibromatosis Type 1 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 Type 1 and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• NFlection Therapeutics
• Spring WorksTherapeutics
• Shanghai Fosun Pharmaceutical Development Co, Ltd.
• Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
• Novartis
• Array BioPharma

Key Products

• NFX 179
• Mirdametinib
• FCN-159
• AL2846
• Trametinib
• Binimetinib

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