Lymphangioleiomyomatosis (LAM) - Pipeline Insight, 2020

Lymphangioleiomyomatosis (LAM) - Pipeline Insight, 2020

  • November 2020 •
  • 60 pages •
  • Report ID: 5989624 •
  • Format: PDF
“Lymphangioleiomyomatosis– Pipeline Insight, 2020,” report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in Lymphangioleiomyomatosis 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

Lymphangioleiomyomatosis Understanding

Lymphangioleiomyomatosis: Overview
Lymphangioleiomyomatosis (LAM) also known as lymphangiomyomatosis is a condition that affects the lungs, the kidneys, and the lymphatic system. The lymphatic system consists of a network of vessels that transport lymph fluid and immune cells throughout the body. Lymph fluid helps exchange immune cells, proteins, and other substances between the blood and tissues. LAM is found almost exclusively in women. It often occurs as a feature of an inherited syndrome called tuberous sclerosis complex. When LAM occurs alone it is called isolated or sporadic LAM.

Signs and symptoms of LAM most often appear during a woman’s thirties. Affected women have an overgrowth of abnormal smooth muscle-like cells (LAM cells) in the lungs, resulting in the formation of lung cysts and the destruction of normal lung tissue. They may also have an accumulation of fluid in the cavity around the lungs (chylothorax). The lung abnormalities resulting from LAM may cause difficulty breathing (dyspnea), chest pain, and coughing, which may bring up blood (hemoptysis). Many women with this disorder have recurrent episodes of collapsed lung (spontaneous pneumothorax).

Routine investigations can be supportive but not diagnostic in LAM. The chest radiograph often appears normal in early disease, although may show a pneumothorax or pleural effusion. The most common abnormalities are reticulonodular shadowing and cysts or bullae. The lung volumes are generally preserved and the combination of preserved lung volumes and interstitial changes occurs in a small number of conditions including LAM, Langerhans’ cell histiocytosis, sarcoidosis and chronic hypersensitivity pneumonitis.

Standard treatment of lymphangioleiomyomatosis is lung transplantation, but the disorder can recur in transplanted lungs. Rapamune (sirolimus) has been approved to treat lymphangioleiomyomatosis (LAM), a rare progressive lung disease that primarily affects women of childbearing age. Rapamune is manufactured by Wyeth Pharmaceuticals, Inc., a subsidiary of Pfizer, Inc. Treatment with sirolimus is recommended for patients with abnormal or declining lung function. Alternative treatments, such as hormonal manipulation with progestins, tamoxifen, and oophorectomy, are largely ineffective and not recommended.

Lymphangioleiomyomatosis Emerging Drugs Chapters
This segment of the Lymphangioleiomyomatosis report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, I and preclinical. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Lymphangioleiomyomatosis Emerging Drugs
• Rapamycin- AI Therapeutics
LAM-001 is the world’s first inhaled mTOR inhibitor and designed to treat the rare lung disease, lymphangioleiomyomatosis. LAM is a genetic-based disease found primarily in women and characterized by hyperactivation of mTOR signaling. LAM-001 has completed clinical trials in normal healthy volunteers and in patients with LAM disease.
• Saracatinib: AstraZeneca
Saracatinib is a small molecule, highly-potent and selective inhibitor of src tyrosine kinase. The drug is currently in phase II stage of development for the treatment of LAM.
Further product details are provided in the report……..

Lymphangioleiomyomatosis: Therapeutic Assessment
This segment of the report provides insights about the different Lymphangioleiomyomatosis drugs segregated based on following parameters that define the scope of the report, such as:
• Major Players in Lymphangioleiomyomatosis
There are approx. 10+ key companies which are developing the therapies for Lymphangioleiomyomatosis. The companies which have their Lymphangioleiomyomatosis drug candidates in the most advanced stage, i.e. phase II include, AI Therapeutics.
• Phases
report covers around 10+ products under different phases of clinical development like
• Late stage products (BLA Filed and Phase III)
• Mid-stage products (Phase II and
• Early-stage products (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

• Route of Administration
Lymphangioleiomyomatosis pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
• Infusion
• Intradermal
• Intravenous
• Intravesical
• Oral etc.
• Molecule Type
Products have been categorized under various Molecule types such as
• Antineoplastics
• Vaccine
• Gene therapies
• Immunotherapy
• Metal
• Monoclonal antibodies
• Nanoparticle
• Oncolytic viruses
• Peptide
• Plasmid
• Protein
• Small molecule
• Ligand
• Bacteria and others.

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

Lymphangioleiomyomatosis: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase III, II, I and preclinical stage. It also analyses Lymphangioleiomyomatosis 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 Lymphangioleiomyomatosis drugs.

Report Highlights
• The companies and academics are working to assess challenges and seek opportunities that could influence Lymphangioleiomyomatosis R&D. The therapies under development are focused on novel approaches to treat/improve Lymphangioleiomyomatosis.
Lymphangioleiomyomatosis Report Insights
• Lymphangioleiomyomatosis Pipeline Analysis

• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players
• AI Therapeutics
• AstraZeneca

Key Products
• Rapamycin
• Saracatinib