Cholangiocarcinoma - Pipeline Insight, 2024

This “Cholangiocarcinoma- Pipeline Insight, 2024” report provides comprehensive insights about 55+ companies and 60+ pipeline drugs in Cholangiocarcinoma 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

Cholangiocarcinoma: Understanding

Cholangiocarcinoma: Overview

Cholangiocarcinoma is an epithelial cell malignancy arising from varying locations within the biliary tree and showing markers of cholangiocyte differentiation. The most contemporary classification based on anatomical location includes intrahepatic, perihilar, and distal cholangiocarcinoma. Many cases of cholangiocarcinoma arise de novo and do not have a specific risk factor, but there are a number of risk factors that have been identified, including primary hepatobiliary disease, genetic disorders, toxic exposures, and infections.

Similar to many malignancies, cholangiocarcinoma arises from precursor lesions such as the more common biliary intraepithelial neoplasia and the less common intraductal papillary mucinous neoplasm. Normal epithelium becomes one of these premalignant lesions through mutations in a variety of oncogenes and tumor suppressor genes. While the specific molecular pathway has not been identified, cholangiocarcinomas harbor mutations in genes such as RAS, BRAF, p52, SMAD4, and more.

In most cases of cholangiocarcinoma, these genetic changes are acquired during a person's lifetime and are present only in the bile duct cells that give rise to the tumor. The genetic changes, which are called somatic mutations, are not inherited. Somatic mutations in many different genes have been found in cholangiocarcinoma. Some of these genes act as tumor suppressors, which means they help keep the growth and division of cells tightly regulated. Mutations in or deletions of tumor suppressor genes can allow cells to grow and divide without control or order, which is a hallmark of cancer. Other genes associated with cholangiocarcinoma are oncogenes; when they are turned on (activated) abnormally, these genes have the potential to cause normal cells to become cancerous. Identifying somatic mutations in cholangiocarcinoma may provide clues to how quickly the cancer will grow and spread and which treatments might be most effective.

Several non-genetic risk factors for cholangiocarcinoma have been identified. These include a bile duct disease called primary sclerosing cholangitis, bile duct stones or cysts, and exposure to certain chemical toxins used in manufacturing. Other risk factors that have been studied include long-term infection with viral hepatitis B or C, scarring of the liver (cirrhosis), and chronic diseases such as inflammatory bowel disease and diabetes. Researchers’ suspect that certain lifestyle factors, including smoking, alcohol use, and obesity, may also contribute to the risk of developing cholangiocarcinoma.

Basic laboratory testing should consist of liver biochemical tests, including aminotransferases, alkaline phosphatase, and total, indirect, and direct bilirubin. There are medical and surgical treatment options for cholangiocarcinoma, but surgery is the only curative therapy.

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

Cholangiocarcinoma Emerging Drugs Chapters

This segment of the Cholangiocarcinoma 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.

Cholangiocarcinoma Emerging Drugs

Pembrolizumab: Merck Sharp & dohme Pembrolizumab is a highly selective humanized monoclonal IgG4 antibody directed against the PD-1 receptor on the cell surface. The drug blocks the PD-1 receptor, preventing binding and activation of PD-L1 and PD-L2. This mechanism causes the activation of T-cell mediated immune responses against tumor cells. It is currently being evaluated in Phase III stage of development to treat biliary tractcancer.

E7090: Eisai Discovered in-house by Eisai’s Tsukuba Research Laboratories, E7090 is an orally available novel tyrosine kinase inhibitor that demonstrates selective inhibitory activity against fibroblast growth factor receptors (FGFR) FGFR1, FGFR2, and FGFR3. Distinct from prior known FGFR inhibitors, E7090 has a basic structure that lacks the dimethoxyphenyl moiety, and in a kinetic interaction analysis study, it was observed that E7090 demonstrates antitumor effects due to inhibition of kinase activity with a binding mode (Type V) that exhibits rapid and potent binding as well as high selectivity to FGFR1. A Phase II clinical trial of E7090 is underway to evaluate the efficacy and safety in patients with cholangiocarcinoma with FGFR2 gene fusion. E7090 received orphan drug designation for a prospective indication for unresectable biliary tract cancer with FGFR2 gene fusion by the Ministry of Health, Labour and Welfare,Japan.

ABC294640: Red Hill Biopharma Limited ABC294640 (Opaganib) is a first-in-class, proprietary sphingosine kinase-2 (SK2) selective inhibitor, administered orally, with anticancer, anti-inflammatory, and antiviral activities. Opaganib inhibits SK2, a lipid kinase that catalyzes the formation of the lipid signaling molecule sphingosine 1-phosphate (S1P). S1P promotes cancer growth, and proliferation and pathological inflammation, including TNFa signaling, and other inflammatory cytokine production. Specifically, by inhibiting the SK2 enzyme, opaganib blocks the synthesis of S1P, which regulates fundamental biological processes such as cell proliferation, migration, immune cell trafficking and angiogenesis, and is also involved in immune modulation and suppression of innate immune responses from T cells. Preliminary evidence suggests that because of its specificity for targeting SK2 rather than SK1, opaganib may have a better therapeutic ratio than nonspecific sphingosine kinase inhibitors or those targeting only SK1. Currently, the drug is being evaluated in the Phase II stage of its development for the treatmentofcholangiocarcinoma.

TT-00420: Trans Thera Science TT-00420 is a highly innovative, clinical-stage, spectrum-selective kinase inhibitor that exerts antitumor effects by targeting tumor cells and improving the tumor microenvironment. A large number of preclinical studies have found that TT-00420 has a promising inhibitory effect on triple-negative breast cancer, cholangiocarcinoma, and other malignant tumors. TT-00420 was granted Orphan Drug Designation and fast track designation by the FDA and is currently in the Phase II stage of its development for the treatmentofcholangiocarcinoma.

KIN-3248: Kinnate Biopharma KIN-3248 is a small molecule kinase inhibitor that targets cancer associated alterations in the FGFR2 and FGFR3 genes. KIN-3248 aims to address the primary driver alteration and clinically observed and predicted FGFR2/3 mutations. The company is currently evaluating the safety and tolerability of KIN-3248 in the Phase I stage of its development for the treatment ofcholangiocarcinoma.

Cholangiocarcinoma: Therapeutic Assessment

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

Major Players in Cholangiocarcinoma

• There are approx. 55+ key companies which are developing the therapies for Cholangiocarcinoma. The companies which have their Cholangiocarcinoma drug candidates in the most advanced stage, i.e. phase III include, Merck Sharp & dohme.

Phases

This report covers around 60+ 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

Cholangiocarcinoma 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
• Intravenous
• Subcutaneous
• Parenteral
• Topical

Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

Product Type

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

Cholangiocarcinoma: Pipeline Development Activities

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

Cholangiocarcinoma Report Insights

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

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

Key Players

• Merck Sharp & dohme
• Eisai
• Red Hill BiopharmaLimited
• Trans TheraScience
• Kinnate Biopharma
• Taiho Oncology
• Bristol-Myers Squibb
• AstraZeneca
• Jiangsu Hengrui Medicine Co. Ltd.
• GlaxoSmithKline
• Beijing Inno CarePharma
• Genoscience
• 3D Medicines
• Innovent Biologics (Suzhou) Co. Ltd.
• QED Therapeutics
• Hutchison MediPharma
• Tri Salus LifeSciences
• Relay Therapeutics
• Eli Lilly and Company
• Medivir
• Boehringer Ingelheim
• Compass Therapeutics
• Intensity Therapeutics
• Sirnaomics
• Wellmarker Bio

Key Products

• Pembrolizumab
• E7090
• ABC294640
• TT-00420
• KIN-3248
• TAS120
• Dasatinib
• Durvalumab
• Camrelizumab
• Niraparib
• ICP-192
• GNS561
• Pemigatinib
• 3D185
• Futibatinib
• BGJ398
• HMPL-453
• SD-101
• RLY-4008
• LY3410738
• MIV-818
• CTX-009
• BI 905711
• INT230-6
• STP705
• WM-S1-030

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