Cryopyrin-Associated Periodic Syndrome - Pipeline Insight, 2024

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

Cryopyrin-Associated Periodic Syndrome Understanding

Cryopyrin-Associated Periodic Syndrome: Overview

Cryopyrin-associated periodic syndrome or syndromes (CAPS), also known as cryopyrinopathies, are genetic autoinflammatory syndromes defined by ‘gain-of-function' mutations affecting the cryopyrin protein. Cryopyrin-associated periodic syndromes are all autosomal dominant genetic conditions, meaning only one copy of the abnormal gene is required to develop the clinical syndrome. CAPS comprises 3 disorders on a continuum of severity: severe CINCA syndrome, intermediate Muckle-Wells syndrome (MWS) and milder familial cold urticaria (FCAS). Cryopyrin-Associated Autoinflammatory Syndromes (CAPS) are a group of illnesses related to defects in the protein cryopyrin (also called NLRP3). These diseases differ in the systems involved and in the severity of the disease. CAPS is rare, found in about one in 360,000 to 1,000,000 people. CAPS is suspected by the patient’s history, as well as by symptoms and signs found by the physician on physical examination during a fever episode. Bloodwork may reveal high levels of inflammation during flares. The diagnosis may be confirmed by a genetic test, although occasionally the genetic test may be normal. Other tests to support the diagnosis may include skin biopsy (examining a small sample of the skin), an eye examination, hearing tests, lumbar puncture (obtaining fluid from the spine) and imaging of the brain and inner ears by magnetic resonance imaging (MRI). Medications that target interleukin-1 are very effective in treating CAPS. Treatment must continue throughout life, as there is no known cure. It is important to diagnose early and start treatment quickly since there is usually no repair to damage that has occurred to brain, eyes, bones, and ears. Higher doses and more frequent injections are often needed to treat patients with brain involvement and severe headaches. Other treatments include physical therapy, splints and other aids to treat joint deformities if they occur. Surgery is occasionally needed. Hearing aids are needed for children with deafness. Untreated patients suffer from frequent episodes, decreasing the quality of life.

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

Cryopyrin-Associated Periodic Syndrome Emerging Drugs Chapters

This segment of the Cryopyrin-Associated Periodic 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.

Cryopyrin-Associated Periodic Syndrome Emerging Drugs

ZYIL 1 : Zydus CadilaZYIL1 is a novel oral small molecule NLRP3 inhibitor. It was found to be safe and well-tolerated in Phase I trials. Currently, it is in Phase II stage of development to treat Cryopyrin-associated periodic syndromes.

DFV890: Novartis PharmaceuticalsDFV 890 is a NLRP3 protein inhibitor being developed by Novartis. Currently, it is in Phase II stage of development to treat Cryopyrin-associated periodic syndromes.

Zunsemetinib : Aclaris TherapeuticsZunsemetinib is an investigational oral mitogen-activated protein kinase-activated protein kinase 2 (MK2) inhibitor. This mechanism potentially leads to the inhibition of multiple cytokines, chemokines, matrix metalloproteases and other inflammatory signals. Key inflammatory cytokines driven by this mechanism include tumor necrosis factor a (TNFa) and interleukin-1a, -1ß, -6 and -8 (IL1a, IL1ß, IL6 and IL8). Aclaris is developing zunsemetinib as a potential treatment for RA and other immuno-inflammatory diseases. Currently the drug is in phase 2 stage of development for the treatment of Cryopyrin-associated periodic syndromes.

IZD334: InflazomeIZD334 is a NLRP3 protein inhibitors. Activated NLRP3 acts as a ‘danger sensor’ in the body to release the pro-inflammatory cytokines IL-1ß, IL-18 and induce uncontrolled, lytic cell death (pyroptosis). These processes lead to chronic inflammation, and as such, NLRP3 has been implicated in a large number of diseases. Acquisition of Inflazome had given Roche full rights to the Inflazome portfolio.

Cryopyrin-Associated Periodic Syndrome: Therapeutic Assessment

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

Major Players in Cryopyrin-Associated Periodic Syndrome

There are approx. 4+ key companies which are developing the therapies for Cryopyrin-Associated Periodic Syndrome. The companies which have their Cryopyrin-Associated Periodic Syndrome drug candidates in the most advanced stage, i.e. phase II include, Zydus Cadila.

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

Cryopyrin-Associated Periodic 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

• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Ophthalmic
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal

Molecule Type

Products have been categorized under various Molecule types such as

• Oligonucleotide
• Peptide
• Small molecule

Product Type

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

Cryopyrin-Associated Periodic Syndrome: Pipeline Development Activities

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

Cryopyrin-Associated Periodic Syndrome Report Insights

• Cryopyrin-Associated Periodic Syndrome Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Zydus Cadila
• Novartis
• Inflazome
• Roche
• Aclaris Therapeutics
• Mabpharm

Key Products

• ZYIL1
• IZD334
• DFV890
• Zunsemetinib
• CMAB 816