Familial adenomatous polyposis - Pipeline Insight, 2024

This “Familial adenomatous polyposis - Pipeline Insight, 2024” report provides comprehensive insights about 4+ companies and 4+ pipeline drugs in Familial adenomatous polyposis 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

Familial adenomatous polyposis: Understanding

Familial adenomatous polyposis: Overview

Familial adenomatous polyposis (FAP) is a hereditary disorder characterized by the development of numerous (sometimes hundreds or thousands) adenomatous polyps in the large intestine, typically beginning in the second decade of life. This condition is caused by a genetic mutation in the APC (adenomatous polyposis coli) tumor suppressor gene, which is inherited in an autosomal dominant pattern.

The APC gene normally acts as a "gatekeeper" to regulate cell growth and division in the intestinal lining. However, when the gene is mutated, it loses its ability to suppress tumor formation, resulting in the rapid and uncontrolled growth of polyps throughout the colon and rectum.

FAP is caused by a germline mutation of the adenomatous polyposis coli (APC) gene. This provides instructions for making the APC protein, which plays a critical role in several cellular processes that determine whether a cell will develop into a tumor. The inheritance of FAP is autosomal dominant so all individuals carrying the gene mutation have a 50% chance of passing it on to the next generation. Approximately 70% of subjects have inherited the mutated gene.

The main sign of familial adenomatous polyposis (FAP) is the development of hundreds or even thousands of polyps in the colon and rectum, typically beginning in the mid-teens. These polyps are almost certain to progress to colorectal cancer by the time the patient reaches their 40s if left untreated. In addition to the polyps in the large intestine, patients with FAP may also develop polyps in the upper gastrointestinal tract, particularly in the duodenum. Other potential symptoms include rectal bleeding, diarrhea, chronic abdominal pain, and dental abnormalities. Patients may also develop benign skin lesions, bone growths, and pigment changes in the retina. However, many patients are initially asymptomatic, with the condition being detected through routine screening.

The primary treatment for familial adenomatous polyposis (FAP) is prophylactic surgery to remove the colon and rectum, as polyps are nearly certain to progress to colorectal cancer if left untreated. Surgical options include total proctocolectomy with ileostomy, colectomy with ileorectostomy, or restorative proctocolectomy with ileal pouch-anal anastomosis. The choice depends on factors like the presence of rectal polyps, anal sphincter function, and patient preference. Endoscopic surveillance is crucial to determine the appropriate timing of surgery, with upper endoscopy every 1-4 years and flexible sigmoidoscopy annually.

"Familial adenomatous polyposis - Pipeline Insight, 2024" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Familial adenomatous polyposis pipeline landscape is provided which includes the disease overview and Familial adenomatous polyposis treatment guidelines.

The assessment part of the report embraces, in depth Familial adenomatous polyposis 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, Familial adenomatous polyposis 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 Familial adenomatous polyposis R&D. The therapies under development are focused on novel approaches to treat/improve Familial adenomatous polyposis.

Familial adenomatous polyposis Emerging Drugs Chapters

This segment of the Familial adenomatous polyposis 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.

Familial adenomatous polyposis Emerging Drugs

ALFA: SLA Pharma

ALFA is a unique, novel oral formulation of highly purified eicosapentaenoic acid free fatty acid (EPA-FFA) in gastro-resistant capsules that is delivered to the gut at optimal pH allowing maximal absorption. The drug is currently being evaluated under Phase III clinical trial for the treatment of patients with Familial adenomatous polyposis.

REC-4881: Recursion Pharmaceuticals

REC-4881 is an orally bioavailable, non-ATP-competitive allosteric small molecule inhibitor of MEK1 and MEK2 being developed to reduce polyp burden and progression to adenocarcinoma in people living with FAP. The drug is currently being evaluated under Phase II clinical trial for the treatment of patients with Familial adenomatous polyposis.

Familial adenomatous polyposis: Therapeutic Assessment

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

Major Players in Familial adenomatous polyposis

There are approx. 4+ key companies which are developing the therapies for Familial adenomatous polyposis. The companies which have their Familial adenomatous polyposis drug candidates in the most advanced stage, i.e. Phase III include, SLA Pharma.

Phases

The report covers around 4+ 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

Familial adenomatous polyposis 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.

Familial adenomatous polyposis: Pipeline Development Activities

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

Familial adenomatous polyposis Report Insights

• Familial adenomatous polyposis Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• SLA Pharma
• Recursion Pharmaceuticals
• TherapyX
• Eloxx Pharmaceuticals
• Emtora Biosciences
• Janssen Biotech

Key Products

• ALFA
• REC-4881
• FAPXIL
• ZKN-013
• eRapa
• Guselkumab

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