Turner Syndrome - Pipeline Insight, 2024

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

Turner Syndrome: Understanding

Turner Syndrome: Overview

Turner syndrome, also referred to as congenital ovarian hypoplasia syndrome, was first described by Henri Turner, an Oklahoma physician in 1938. It is the most common sex chromosomal abnormality found in females. It results when one of the X chromosomes is missing, partially or completely.

Most instances of Turner syndrome are not inherited. When monosomy X is the cause, the chromosomal abnormality is a random event during the formation of reproductive cells in the person’s parent. An error in cell division is called nondisjunction and can result in reproductive cells with an abnormal number of chromosomes. For example, a sex chromosome can become lost from an egg or a sperm cell due to nondisjunction. If an atypical reproductive cell contributes to the genetic makeup of a child, each cell will possess a single X chromosome, and the other sex chromosome will be missing.

Mosaic Turner syndrome is likewise not an inherited condition. It occurs due to a random event during the cell division stage in the early fetal development of the affected individual. As a result, some of a person's cells have the usual two sex chromosomes, while other cells contain only one copy of the X chromosome. Other sex chromosome abnormalities are possible in females with X chromosome mosaicism. Rarely, Turner syndrome can result from a partial deletion of the X chromosome, and this can pass from one generation to the next.

Cardiac abnormalities are frequently associated with Turner syndrome. At the time of diagnosis, the patient should be evaluated by a cardiologist with an EKG to evaluate for prolonged QT interval. Blood pressure should be measured in the upper and lower extremities, and patients should get an echocardiogram or cardiac MRI to look for cardiac anomalies. Regular monitoring of hearing, including serial audiology evaluations, is recommended throughout life, with an audiology evaluation every 3 yrs. in children and every five years in adults.

Turner syndrome does not cause growth hormone deficiency. But, the patients respond well to growth hormone therapy and should be started on treatment with growth hormone once their height falls below 5% for age. If not treated appropriately, the estimates are that the adult height would be 20 cm below the average adult female height. Growth hormone therapy should continue until the patients reach their adult height and no longer have any growth potential. Once the patient is on the growth hormone therapy, it can sometimes expose underlying scoliosis. Hence, the patients should have their spines monitored closely during the treatment. Almost all girls with Turner syndrome need estrogen, even if they have spontaneous puberty, which may persist for some time but is usually followed by primary ovarian insufficiency. Later, cyclic progestins are added to the regimen to induce cyclic uterine bleeding and prevent endometrial hyperplasia. Estrogen therapy should be initiated at around 11 to 12 years of age if gonadotropins are elevated or AMH level is low. The treatment can begin at doses between 1/10 to 1/8 of adult replacement dose and gradually increased every six months to simulate normal pubertal progression until reaching adult dosing.

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

Turner Syndrome Emerging Drugs Chapters

This segment of the Turner 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.

Turner Syndrome Emerging Drugs

LUM201: Lumos Pharma Lumos Pharma’s drug candidate LUM 201, also known as Ibutamoren, is a small molecule which is to be administered orally, promotes the secretion (secretagogue) of Growth Hormone (GH) from the pituitary gland. LUM-201 acts as an agonist of the GH Secretagogue Receptor to stimulate GH release. LUM-201 has been observed to increase the amplitude of endogenous pulsatile GH secretion in humans, which mimics the natural pattern of GH secretion.It is currently in the Phase I of drug development for Turner Syndrome. The molecule has received the orphan drug status for somatotrophin deficiency. The company is planning to conduct a Phase II trial for Turnersyndrome.

Turner Syndrome: Therapeutic Assessment

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

Major Players in Turner Syndrome

• There are approx. 4+ key companies which are developing the therapies for Turner Syndrome. The companies which have their Turner Syndrome drug candidates in the most advanced stage, i.e. phase I include, Lumos Pharma.

Phases

This 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

Turner 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
• 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.

Turner Syndrome: Pipeline Development Activities

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

Turner Syndrome Report Insights

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

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

Key Players

• Lumos Pharma
• Ascendis Pharma

Key Products

• LUM 201
• TransCon hGH

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