Neurofibromatosis - Pipeline Insight, 2024

This “Neurofibromatosis- Pipeline Insight, 2024” report provides comprehensive insights about 12+ companies and 14+ 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 is a neurocutaneous disorder characterized by tumors in the nervous system and skin. Neurofibromatosis types 1 and 2 are the most common and are distinct entities. Neurofibromatosis type 1, or von Recklinghausen disease, is an autosomal dominant disease. Neurofibromatosis type 1 presents with neurofibromas, freckling, and optic gliomas. It is a clinical diagnosis. Neurofibromatosis type 2 (NF2) is a disease characterized by bilateral vestibular schwannomas (VS) and meningiomas. It has an autosomal dominant inheritance pattern.

Neurofibromatosis type 1 is caused by a loss of function mutation, either de novo or inherited, on the neurofibromin 1 (NF1) gene. It is located on band 17q11.2 and codes for neurofibromin. Neurofibromin is a tumor suppressor that functions in the RAS/MAPK and mTOR pathways. Mosaicism can occur, resulting in the segmental, generalized, or gonadal NF1 gene. Segmental NF1 gene has pigment changes, tumors, or both that are limited to one or more body segments. Generalized NF1 gene appears similar to classic NF1 but does not have the NF1 gene mutation. Gonadal NF1 gene occurs when the mutation only affects the ova or sperm. NF2 gene is caused by a loss of function mutation of the NF2 gene. It is located on band 22q12 and codes for merlin. Merlin is a cell membrane protein that is also a tumor suppressor that functions in the PI3kinase/Akt, Raf/MEK/ERK, and mTOR pathways.

Neurofibromatosis type 1 makes up about 96% of all neurofibromatosis cases. Prevalence is 1 in 3000 births. It occurs equally between gender and races. Fifty percent of patients have a spontaneous mutation, and the other half have an inherited mutation. There is a 100% penetrance with variable expressivity. Neurofibromatosis type 2 makes up about 3% of all cases and has a prevalence between 1 in 33,000 births and 1 in 87,410. There is no gender or race predilection. Neurofibromatosis type 2 has variable presentations amongst different families. A more severe clinical presentation is associated with a frameshift or a nonsense mutation that results in a truncated protein.

Cafe-au-lait spots and neurofibromas are benign and do not require treatment. Lesions exhibiting symptoms may be surgically excised, however recurrence is possible. Plexiform neurofibromas may develop into cancer. Plexiform neurofibromas have an 8% to 13% chance of turning into malignant peripheral nerve sheath tumours.

"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 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.

Neurofibromatosis Emerging Drugs

• FCN-159: Shanghai Fosun Pharmaceutical

FCN-159 is an orally available and highly potent selective MEK1/2 inhibitor, and a candidate targeted therapy for NF1-related PN tumors. In vitro study showed that FCN-159 is metabolized primarily by CYP3A4 and eliminated in a first-order elimination. Preclinical studies using human colon cancer cells confirmed FCN-159 blocks the downstream MAPK pathway, preventing phosphorylation of MEK kinase and intracellular ERK. Anti-tumor activity was observed with FCN-159, inhibiting cell proliferation in selected RAS-mutant tumor cell lines, and tumor growth in nude mouse xenograft models (including human melanoma A375). The drug is currently being evaluated under Phase III clinical studies for the treatment of patients suffering from neurofibromatosis.

• NFX-179: NFlection Therapeutics

NFX-179 is an investigational mitogen-activated protein kinase (MEK) inhibitor. NFX-179 is a "soft" (metabolically labile) drug, which, when formulated as NFX-179 Gel for topical application, is designed to concentrate at the dermal site of action but degrade in systemic circulation, thereby significantly reducing side effects routinely seen with systemically available MEK inhibitors. NFlection is developing NFX-179 Gel for the treatment of cutaneous neurofibromas in people with neurofibromatosis type 1 and received orphan designation in the US and EU for this indication. The drug is currently being evaluated under Phase II clinical trials for the treatment of neurofibromatosis.

• PAS-004: Pasithea Therapeutics

PAS-004 is a small molecule allosteric inhibitor of MEK 1/2, which are dual-specificity protein kinases, in the MAPK signaling pathway. The MAPK pathway has been implicated in a variety of diseases, as it functions to drive cell proliferation, differentiation, survival and a variety of other cellular functions that, when abnormally activated, are critical for the formation and progression of tumors, fibrosis and other diseases. MEK inhibitors block phosphorylation (activation) of extracellular signal-regulated kinases (ERK). Blocking the phosphorylation of ERK can lead to cell death and inhibition of tumor growth. The drug received orphan-drug designation from the US FDA for the treatment of NF1. The drug is currently being evaluated under Phase I clinical trial for the treatment of intrahepatic cholangiocarcinoma.

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. 12+ 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 III include, Shanghai Fosun Pharmaceutical.

Phases

This report covers around 14+ 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

• 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.

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

• Recursion Pharmaceuticals
• Daiichi Sankyo
• Pasithea Therapeutics
• Bristol-Myers Squibb
• NFlection Therapeutics
• Shanghai Fosun Pharmaceutical
• Deciphera Pharmaceuticals

Key Products

• REC-2282
• PLX3397
• PAS-004
• Nivolumab
• NFX-179
• FCN-159
• DCC-3084

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