Alagille Syndrome - Pipeline Insight, 2020

DelveInsight’s, “Alagille Syndrome– Pipeline Insight, 2020,” report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in Alagille Syndrome 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
Alagille Syndrome Understanding
Alagille Syndrome: Overview
Alagille syndrome (ALGS) is a rare genetic disorder that can affect multiple organ systems of the body including the liver, heart, skeleton, eyes and kidneys. The specific symptoms and severity of Alagille syndrome can vary greatly from one person to another, even within the same family. Some individuals may have mild forms of the disorder while others may have more serious forms.

Symptoms
The symptoms and severity of Alagille syndrome can vary greatly from one person to another, even among members of the same family. Some individuals may have a mild form of the disorder that can virtually go unnoticed; other individuals may have a serious form of the disorder that can potentially cause life-threatening complications. Alagille syndrome can be associated with abnormalities of the liver, heart, eyes, skeleton, kidneys and other organ systems of the body. A main finding of Alagille syndrome is liver disease that often becomes apparent within the first three months of life. However, individuals with mild liver involvement may not be diagnosed until later in life. Liver disease in Alagille syndrome, if present, may range in severity from jaundice or mild cholestasis to severe, progressive liver disease that can potentially result in liver failure.

Diagnosis
A diagnosis of Alagille syndrome is made based upon identification of characteristic symptoms, a detailed patient history, a thorough clinical evaluation and a variety of specialized tests. Because the symptoms of Alagille syndrome are highly variable, obtaining a diagnosis can be difficult. Surgical removal and microscopic study of liver tissue (liver biopsy) can reveal bile duct paucity. Although bile duct paucity is considered a key characteristic of Alagille syndrome, this finding is not always present in infants with the disorder.

Treatment
The treatment of Alagille syndrome is directed toward the specific symptoms that are apparent in each individual. Treatment may require the coordinated efforts of a team of specialists. Pediatricians, gastroenterologists, cardiologists, ophthalmologists, and other healthcare professionals may need to systematically and comprehensively plan an affect child’s treatment. Individuals with Alagille syndrome should have a baseline echocardiogram (ultrasound of the heart) to screen for heart involvement, ultrasound of the abdomen to screen for liver and kidney anomalies, and a screening eye (ophthalmology) exam, In addition, if not previously obtained for specific symptoms, a screening imaging study of the blood vessels of the head (MRI/MRA) is recommended for children who are old enough to sit through the study without need for anesthesia or sedation.

Alagille Syndrome Emerging Drugs Chapters
This segment of the Alagille Syndrome 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.

Alagille Syndrome Emerging Drugs
• Maralixibat: Mirum Pharma
Maralixibat is a novel, minimally absorbed, orally administered investigational drug being evaluated in several rare cholestatic liver diseases. Maralixibat inhibits the apical sodium dependent bile acid transporter (ASBT), resulting in more bile acids being excreted in the feces, leading to lower levels of bile acids systemically, thereby potentially reducing bile acid mediated liver damage and related effects and complications. More than 1,600 individuals have received maralixibat, including more than 120 children who have received maralixibat as an investigational treatment for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC).

• Odevixibat: Albireo
Odevixibat is a highly potent, non-systemic ileal bile acid transport inhibitor (IBATi) that has has minimal systemic exposure and acts locally in the small intestine. Albireo is developing odevixibat to treat rare pediatric cholestatic liver diseases, including progressive familial intrahepatic cholestasis, biliary atresia and Alagille syndrome.

Further product details are provided in the report……..
Alagille Syndrome: Therapeutic Assessment
This segment of the report provides insights about the different Alagille Syndrome drugs segregated based on following parameters that define the scope of the report, such as:
• Major Players in Alagille Syndrome
There are approx. 10+ key companies which are developing the therapies for Alagille Syndrome. The companies which have their Alagille Syndrome drug candidates in the most advanced stage, i.e. phase II include, Mirum Pharmaceuticals.

• Phases
DelveInsight’s 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
Alagille Syndrome 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
• intravitreal
• Subretinal
• 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.

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

Report Highlights
• The companies and academics are working to assess challenges and seek opportunities that could influence Alagille Syndrome R&D. The therapies under development are focused on novel approaches to treat/improve Alagille Syndrome.
Alagille Syndrome Report Insights
• Alagille Syndrome Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs
Alagille Syndrome 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 Alagille Syndrome drugs?
• How many Alagille Syndrome drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Alagille Syndrome?
• What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Alagille Syndrome 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 Alagille Syndrome and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players
• Mirum Pharmaceuticals, Inc.
• Lumena Pharmaceuticals
• Albireo

Key Products
• LUM001
• A4250