Down syndrome - Pipeline Insight, 2024

This “Down syndrome - Pipeline Insight, 2024,” report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in Down 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

Down syndrome Understanding

Down syndrome: Overview

Down syndrome is a chromosome disorder associated with intellectual disability, a characteristic facial appearance, with small nose and an upward slant to the eyes, and low muscle tone in infancy. The degree of intellectual disability varies from mild to moderate. People with Down syndrome may also be born with various health concerns such as heart defects or digestive abnormalities, as well as short stature and a single deep crease across the center of the palm. They also have an increased risk to develop gastroesophageal reflux, celiac disease, hypothyroidism, hearing and vision problems, leukemia, and Alzheimer disease. Down syndrome is caused by having three copies of chromosome 21 (called trisomy 21) instead of the usual two copies and is typically not inherited. Treatment focuses on the specific symptoms in each person. There is ongoing research about the specific genes causing the disease aiming to find more effective treatments.

Down syndrome Emerging Drugs Chapters

This segment of the Down 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.

Down syndrome Emerging Drugs

ACI-24: AC Immune SAACI-24 is an active vaccine stimulating the patient's immune system to produce betasheet conformation-specific antibodies that prevent plaque deposition or enhance clearance of plaques. During preclinical development, the ACI-24 has shown high efficacy in vivo by memory restoration and plaque reduction. The vaccine is also characterized by a very high specificity due to generating a conformation-specific antibody response. The favourable safety profile of ACI-24 is underlined through the absence of local inflammation in relevant models as well as its T-cell independent mechanism shown in preclinical development.

AEF0217: Aelis Farma AEF0217 is the second drug candidate developed by Aelis Farma. It is specifically designed for the treatment of cognitivedeficits.

AEF0217's major characteristics:

• High potency in reversing cognitive deficits in validated translational models of Down syndrome, Fragile X syndrome and aging related cognitive impairments.
• No identifiable behavioral side effects even in fragile trisomic and aged mice.
• Very favorable ADMET profile, with good oral absorption, brain access, long half-life and a good tolerability (therapeutic index = 640).

Down Syndrome: Therapeutic Assessment

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

Major Players in Down Syndrome

There are approx. 10+ key companies which are developing the therapies for Down Syndrome. The companies which have their Down syndrome drug candidates in the mid to advanced stage, i.e. Phase II include, AC Immune SA.

Phases

This report covers around 10+ products under different phases of clinical development like

• Late-stage products (Phase III)
• Mid-Stage products (Phase II)
• Early-stage products (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

Down 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

• Intravenous
• Oral
• Molecule Type

Products have been categorized under various Molecule types such as

• Small molecules
• Peptide
• Antibiotics
• Product Type

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

Down Syndrome: Pipeline Development Activities

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

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Down Syndrome R&D. The therapies under development are focused on novel approaches to treat/improve Down Syndrome.

Down syndrome Report Insights

• Down Syndrome Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Eisai Inc.
• AC Immune
• Elixirgen Therapeutics
• Aphios Therapeutics
• Avanti Biosciences
• Annovis Bio
• AelisFarma
• Kinopharma
• Neuro Nascent,Inc.
• Pharmasum Therapeutics

Key Products

• E2020
• APH-1104
• ACI-24
• ABI102
• Buntanetap
• AEF0217
• NNI351