Prader Willi Syndrome Pipeline Insight

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

 

Introduction

Executive Summary

Prader-Willi syndrome: Overview

• Causes

• Mechanism of Action

• Signs and Symptoms

• Diagnosis

• Disease Management

Pipeline Therapeutics

• Comparative Analysis

Therapeutic Assessment

• Assessment by Product Type

• Assessment by Stage and Product Type

• Assessment by Route of Administration

• Assessment by Stage and Route of Administration

• Assessment by Molecule Type

• Assessment by Stage and Molecule Type

Prader-Willi syndrome – DelveInsight’s Analytical Perspective

In-depth Commercial Assessment

• Prader-Willi syndrome companies’ collaborations, Licensing, Acquisition -Deal Value Trends

Prader-Willi syndrome Collaboration Deals

• Company-Company Collaborations (Licensing / Partnering) Analysis

• Company-University Collaborations (Licensing / Partnering) Analysis

Late Stage Products (Phase III)

• Comparative Analysis

BSN175: Biospherics.net

• Product Description

• Research and Development

• Product Development Activities

Mid Stage Products (Phase II)

• Comparative Analysis

Pitolisant: Harmony Biosciences

• Product Description

• Research and Development

• Product Development Activities

Drug profiles in the detailed report…..

Early Stage Products (Phase I)

• Comparative Analysis

OPN-300: OptiNose

• Product Description

• Research and Development

• Product Development Activities

Drug profiles in the detailed report…..

Pre-clinical and Discovery Stage Products

• Comparative Analysis

INV-101: Inversago Pharma

• Product Description

• Research and Development

• Product Development Activities

Inactive Products

• Comparative Analysis

Prader-Willi syndrome Key Companies

Prader-Willi syndrome Key Products

Prader-Willi syndrome- Unmet Needs

Prader-Willi syndrome- Market Drivers and Barriers

Prader-Willi syndrome- Future Perspectives and Conclusion

Prader-Willi syndrome Analyst Views

Prader-Willi syndrome Key Companies

Appendix

List of Tables

 Table 1 Total Products for Prader-Willi syndrome

Table 2 Late Stage Products

Table 3 Mid Stage Products

Table 4 Early Stage Products

Table 5 Pre-clinical & Discovery Stage Products

Table 6 Assessment by Product Type

Table 7 Assessment by Stage and Product Type

Table 8 Assessment by Route of Administration

Table 9 Assessment by Stage and Route of Administration

Table 10 Assessment by Molecule Type

Table 11 Assessment by Stage and Molecule Type

Table 12 Inactive Products

List of Figures

 Figure 1 Total Products for Prader-Willi syndrome

Figure 2 Late Stage Products

Figure 3 Mid Stage Products

Figure 4 Early Stage Products

Figure 5 Preclinical and Discovery Stage Products

Figure 6 Assessment by Product Type

Figure 7 Assessment by Stage and Product Type

Figure 8 Assessment by Route of Administration

Figure 9 Assessment by Stage and Route of Administration

Figure 10 Assessment by Molecule Type

Figure 11 Assessment by Stage and Molecule Type

Figure 12 Inactive Products

• 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

  • Tags:
  • Prader-Willi Syndrome Pipeline
  • Prader-Willi Syndrome clinical tria...
  • Prader-Willi Syndrome companies
  • Prader-Willi Syndrome drugs
  • Prader-Willi Syndrome therapies
  • Prader-Willi Syndrome treatment alg...
  • Prader-Willi Syndrome pipeline drug...

Forward to Friend

Need A Quote