DelveInsight’s, “Prader-Willi syndrome – Pipeline Insight, 2020,” report provides comprehensive insights about 20+ companies and 20+ pipeline drugs in Prader-Willi 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
Prader-Willi syndrome Understanding
Prader-Willi syndrome: Overview
Prader-Willi syndrome (PWS) is a genetic multisystem disorder characterized during infancy by lethargy, diminished muscle tone (hypotonia), a week suck and feeding difficulties with poor weight gain and growth and other hormone deficiency. In childhood, features of this disorder include short stature, small genitals and an excessive appetite. Affected individuals do not feel satisfied after completing a meal (satiety).
Symptoms
Typical symptoms of Prader-Willi syndrome include:
• an excessive appetite and overeating, which can easily lead to dangerous weight gain
• restricted growth (children are much shorter than average)
• floppiness caused by weak muscles (hypotonia)
• learning difficulties
• lack of sexual development
• behavioural problems, such as temper tantrums or stubbornness
Diagnosis
A diagnosis of PWS is based upon a detailed patient history, a thorough clinical evaluation and identification of characteristic symptoms. Consensus diagnostic criteria for PWS have been established and are effective for identifying potential cases of PWS but genetic testing is required to confirm the diagnosis and to identify the specific genetic subtype (15q11-q13 deletion, maternal disomy 15, imprinting defect). Hence, all infants and newborns with unexplained hypotonia and poor suck should be tested for PWS. To confirm a diagnosis of PWS, certain specialized tests are required including DNA methylation tests and fluorescent in situ hybridization (FISH). More recently, high resolution chromosomal microarray studies with several hundred thousand DNA probes from throughout the genome representing all chromosomes can be utilized to identify small deletion or duplications of the chromosomes that cannot be seen with routine chromosome studies. Microarrays are most useful in identifying the typical chromosome 15q11q13 deletion, smaller rearrangements of this chromosome region, imprinting defects and specific maternal disomy 15 subclasses seen in PWS.
Treatment
The treatment of PWS is directed toward the specific symptoms that are apparent in each individual. Specific therapeutic procedures and interventions may vary, depending upon numerous factors, such as disease severity; the presence or absence of certain symptoms; an individual’s age and general health; and/or other elements. In infants, special nipples or gavage feeding may be used to ensure adequate nutrition. Gavage feeding is a procedure in which a small, thin tube is passed through the nose and mouth to the stomach to directly feed a newborn who has feeding difficulties. In males, the treatment of hypogonadism with either testosterone or human chorionic gonadotropin may be beneficial during infancy, potentially increasing the size of the genitalia or prompting testicular descent into the scrotum when cryptorchidism is present. Individuals with PWS also benefit from growth hormone (GH) therapy, which can help to increase height, improve lean body mass, mobility and respiratory function, decrease body fat and ultimately improve the quality of life. Children with PWS require early intervention to assess and treat issues with motor skills, intellectual disability, and speech and language development. Early intervention may include physical and occupational therapy, special education, and speech therapy.
Prader-Willi syndrome Emerging Drugs Chapters
This segment of the Prader-Willi 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.
Prader-Willi syndrome Emerging Drugs
• BSN175: Biospherics.net
BSN175 is being developed by Biospherics.net for the treatment of Prader-Willi syndrome which results from an abnormality on chromosome 15. BSN175 is an FDA-designated orphan compound for the treatment of Prader Willi syndrome. BSN175 is in phase III clinical trials.
• Pitolisant: Harmony Biosciences
Harmony Biosciences is currently evaluating the novel mechanism of action of pitolisant for broader applications in neurological disorders that may be mediated by the H3 receptor. This mechanism-based approach for pitolisant offers the potential for future clinical development in a variety of conditions beyond disorders of wakefulness. Its phase II trial is currently in not yet recruiting stage.
• OPN-300: OptiNose
OPN-300 has been developed to target Prader-Willi syndrome. The company conducted a phase I clinical trial in late 2013 using OPN-300 in healthy volunteers. In that trial, a low dose of oxytocin delivered using an Optinose EDS produced a statistically significantly greater social-cognitive effect, as measured with functional magnetic resonance imaging, performance on cognitive tests, and physiological markers, than intravenous administration of the same active ingredient that produced blood levels that were not statistically different. It is believed that this clinical trial supports the possibility of direct nose-to-brain activity of medication delivered using an Optinose EDS.

• INV-101: Inversago Pharma
INV-101, our lead program focusing on PWS and NASH, is a peripherally-acting CB1 inverse agonist in late-stage preclinical development. It is currently in preclinical stage of development.
Further product details are provided in the report……..
Prader-Willi syndrome: Therapeutic Assessment
This segment of the report provides insights about the different Prader-Willi syndrome drugs segregated based on following parameters that define the scope of the report, such as:
• Major Players in Prader-Willi syndrome
There are approx. 20+ key companies which are developing the therapies for Prader-Willi syndrome. The companies which have their Prader-Willi syndrome drug candidates in the most advanced stage, i.e. phase III include Biospherics.net and others
• Phases
DelveInsight’s report covers around 20+ products under different phases of clinical development like
• Late-stage products (Phase II and Phase II/III)
• Mid-stage products (Phase II and Phase II/III)
• Early-stage products (Phase I/II and Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

• Route of Administration
Prader-Willi 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
• Infusion
• Intradermal
• Intramuscular
• Intranasal
• Oral
• Parenteral
• Subcutaneous
• Topical.
• Molecule Type
Products have been categorized under various Molecule types such as
• Gene therapies
• Small molecule
• Vaccines
• Polymers
• Peptides
• Monoclonal antibodies

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

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

Report Highlights
• The companies and academics are working to assess challenges and seek opportunities that could influence Prader-Willi syndrome R&D. The therapies under development are focused on novel approaches to treat/improve Prader-Willi syndrome.
• The US FDA has granted orphan drug designation to the BSN175 for the treatment of Prader Willi syndrome
• Pitolisant was designed and developed by Bioprojet, which has marketed the product in Europe since its approval by the European Medicines Agency in 2016. Harmony has an exclusive license from Bioprojet to develop, manufacture and commercialize pitolisant in the United States.
• The US Food and Drug Administration (FDA) has granted a fast track designation to Soleno Therapeutics’s diazoxide choline controlled-release (DCCR) for the treatment of Prader-Willi syndrome (PWS).
Prader-Willi syndrome Report Insights
• Prader-Willi syndrome Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs
Prader-Willi 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 Prader-Willi syndrome drugs?
• How many Prader-Willi syndrome drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Prader-Willi syndrome?
• What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Prader-Willi 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 Prader-Willi syndrome and their status?
• What are the key designations that have been granted to the emerging drugs?
Key Players
• Soleno Therapeutics
• Levo Therapeutics
• Rhythm Pharmaceuticals
• Harmony Biosciences, LLC
• INSYS Therapeutics Inc
• LG Life Sciences
• Millendo Therapeutics
• Ferring Pharmaceuticals
• Erasmus Medical Center
• Biospherics.net
• OptiNose
• Inversago Pharma









Key Products
• DCCR
• BSN175
• OPN-300
• INV-101
• intranasal carbetocin Dose 1
• RM-493
• Pitolisant oral tablets
• Cannabidiol Oral Solution
• Eutropin
• Livoletide
• Somatropin