Hunter Syndrome Pipeline Insight
DelveInsight’s, “Hunter Syndrome – Pipeline Insights, 2021,” report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in Hunter 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.
Hunter Syndrome Understanding
Hunter Syndrome: Overview
Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, is a condition that affects many different parts of the body and occurs almost exclusively in males. It is a progressively debilitating disorder; however, the rate of progression varies among affected individuals. There are two types of MPS II, called the severe and mild types. While both types affect many different organs and tissues as described above, people with severe MPS II also experience a decline in intellectual function and a more rapid disease progression.
In the neuronopathic form of this disorder, physical and mental development reaches a peak at 2-4 years of age with subsequent deterioration. Recurrent upper respiratory infections, a chronic runny nose, hearing impairment, liver and spleen enlargement, inguinal and abdominal hernias, joint stiffness and multiplex dysplasia, compression of tendons in the wrist (carpal tunnel syndrome), and joint stiffness which can result in reduction of hand function, growth failure and valvular disease commonly occur with this form of MPS II. Manifestations of MPS II may include not inflammatory joint stiffness, with associated restriction of movements; and coarsening of facial features, including thickening of the lips, tongue (macroglossia), and nostrils.
The signs and symptoms of MPS II vary greatly, so the diagnosis cannot be made by physical exam alone. Diagnosis requires documentation of reduced or absent iduronate 2-sulfatase enzyme activity in blood or skin cells. A very simple determination of glycosaminoglycans in the urine may help in screening potential positive patients. Patients with MPSII disease accumulate heparin and dermatan sulfates in the urines. Molecular genetic testing for mutations in the IDS gene is available to confirm the diagnosis. It is always important to rule out a multiple sulfatase deficiency by testing other sulfatase enzymes.
- An enzyme replacement therapy, idursulfase (Elaprase), was approved in 2006 by the U.S. Food and Drug Administration (FDA) as a treatment for MPS II. This drug is sponsored by Shire.
- Other treatments of MPS II are symptomatic and supportive. These management interventions commonly include developmental, occupational and physical therapy. Hernias and joint contractures may be corrected by surgery.
- Tonsillectomy and adenoidectomy (surgical removal of both the tonsils and the adenoids) can be used to alleviate breathing and swallowing problems. Positive pressure ventilation (CPAP or tracheostomy) can be used to apply mild air pressure to keep the airway open to alleviate breathing issues. Carpal tunnel release can treat carpal tunnel syndrome.
- Surgical implantation of a ventricular shunt may be used to treat possible hydrocephalus. Hearing devices may be prescribed to treat hearing loss. Developmental, physical, and occupational therapy can be helpful.
Hunter Syndrome Emerging Drugs Chapters
This segment of the Hunter 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.
Hunter Syndrome Emerging Drugs
DNL310: Denali Therapeutics Inc.
DNL310, or ETV:IDS, is a recombinant form of the iduronate 2-sulfatase (“IDS”) enzyme engineered to cross the blood-brain barrier using Denali’s proprietary ETV technology. DNL310 is intravenously administered and intended to improve overall clinical manifestations of Hunter Syndrome, including neurological symptoms, which are not adequately addressed by currently approved therapies.
The TV technology enables improved uptake and distribution of therapeutic antibodies, enzymes and other proteins in the brain after i.v. administration by binding to natural transport receptors in the vasculature of the brain. Pre-clinical studies with the TV technology have shown up to 30-fold increased uptake and superior pharmacological activity compared to standard antibodies, enzymes and other proteins. Denali is developing a broad portfolio of biologic drug candidates that are enabled by its TV technology. Based on pre-clinical proof of concept with DNL310, Denali initiated two additional ERT programs that are enabled by the TV technology.
Further product details are provided in the report……..
Hunter Syndrome: Therapeutic Assessment
This segment of the report provides insights about the different Hunter Syndrome drugs segregated based on following parameters that define the scope of the report, such as:
- Major Players in Hunter Syndrome
There are approx. 10+ key companies which are developing the therapies for Hunter Syndrome. The companies which have their Hunter Syndrome drug candidates in the mid to advanced stage, i.e. phase III and Phase I/II include, JCR Pharmaceuticals, Shire, Denali Therapeutics Inc., Regenxbio Inc. etc.
DelveInsight’s report covers around 10+ products under different phases of clinical development like
- Mid-stage products (Phase II and Phase I/II)
- 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
Hunter 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
- Molecule Type
Products have been categorized under various Molecule types such as
- Recombinant fusion proteins
- Gene therapies
- Product Type
Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
Hunter Syndrome: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Hunter 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 Hunter Syndrome drugs.
- The companies and academics are working to assess challenges and seek opportunities that could influence Hunter Syndrome R&D. The therapies under development are focused on novel approaches to treat/improve Hunter Syndrome.
- May 2020: Denali therapeutics announces publication of two new papers describing its blood-brain barrier delivery technology in science translational medicine.
- The first Denali-authored paper published in STM today, by Kariolis et al. entitled “Brain delivery of therapeutic proteins using a novel Fc fragment blood-brain barrier transport vehicle in mice and monkeys,” describes the development and engineering of the TV platform, and demonstrates how it increases brain exposure and distribution of certain antibodies in preclinical models including non-human primates.
Hunter Syndrome Report Insights
- Hunter Syndrome Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
Hunter Syndrome Report Assessment
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Current Treatment Scenario and Emerging Therapies:
- How many companies are developing Hunter Syndrome drugs?
- How many Hunter Syndrome drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Hunter Syndrome?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Hunter 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 Hunter Syndrome and their status?
- What are the key designations that have been granted to the emerging drugs?