WHIM syndrome - Pipeline Insight, 2024

This “WHIM syndrome - Pipeline Insight, 2024,” report provides comprehensive insights about companies and pipeline drugs in WHIM 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

WHIM syndrome Understanding

WHIM syndrome: Overview

WHIM syndrome is a rare primary immunodeficiency disorder, which are disorders in which the body’s immune system does not function properly. WHIM is an acronym for some of the characteristic symptoms of the disorder - (w)arts, (h)ypogammaglobulinemia, (i)nfections, and (m)yelokathexis. Individuals with WHIM syndrome are more susceptible to potentially life-threatening bacterial infections. They are also predisposed to viral infections. Affected individuals are particularly susceptible to human papillomavirus (HPV), which can cause skin and genital warts and potentially lead to cancer. Affected individuals have extremely low levels of certain white blood cells (neutrophils) leading to a condition called neutropenia. In most patients, WHIM syndrome is caused by changes (mutations) in the CXCR4 gene. This mutation is inherited in an autosomal dominant pattern.

Symptoms
The symptoms of WHIM syndrome can vary greatly from one individual to another. Some individuals may have mild expression of the disorder and others may develop potentially life-threatening complications. Common infections in children with WHIM syndrome include recurrent middle ear infections (otitis media), infection of the skin and underlying tissue (cellulitis, impetigo, folliculitis, and abscess), bacterial pneumonia, sinusitis, painful infections of the joints (septic arthritis), dental cavities, and infection of the gums (periodontitis). Chronic infections can potentially cause additional symptoms.

Diagnosis
A diagnosis of WHIM syndrome is based upon identification of characteristic symptoms, a detailed patient history, a thorough clinical evaluation and a variety of specialized tests including genetic tests. A complete blood count will show low levels of neutrophils, or neutropenia, a variable degree of lymphopenia, and normal to low hemoglobin and platelet levels. Initial workups can also reveal hypogammaglobulinemia.

If WHIM syndrome is suspected based on initial tests, the surgical removal and microscopic examination of bone marrow tissue (bone marrow biopsy) may be performed. If this biopsy confirms myelokathexis, this is strongly suggestive of WHIM syndrome.

Treatment
Treatment of WHIM syndrome may include immunoglobulin replacement therapy, granulocyte colony stimulating factor (G-CSF), or granulocyte macrophage colony stimulating factor (GM-CSF), to bolster production and maturation of neutrophils and reduce the incidence of infection. Immunoglobulin therapy, whether administered intravenously (IVIG) or subcutaneously (SCIG), can treat hypogammaglobulinemia and help reduce the frequency of recurrent bacterial infections.

WHIM syndrome Emerging Drugs Chapters

This segment of the WHIM 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.

WHIM syndrome Emerging Drugs

Mavorixafor (X4P-001): X4 Pharmaceuticals X4 is developing Mavorixafor as a first-in-class, oral, allosteric inhibitor of CXCR4 for the treatment of WHIM syndrome. WHIM syndrome is a rare genetic primary immunodeficiency that results from a “gain of function” mutation in the single gene that encodes for the CXCR4 receptor. This genetic mutation causes premature truncations in the CXCR4 protein, causing the receptor to remain in an “on” state longer than normal, resulting in compromised immune cell trafficking and surveillance. X4 has achieved clinical proof of concept for Mavorixafor in WHIM syndrome where, in the completed Phase 2 trial, X4 observed clinically meaningful increases in neutrophil and lymphocyte counts and a favorable safety profile. Based on the positive Phase 2 results, X4 has initiated the Phase 3 pivotal trial of mavorixafor in WHIM syndrome.

WHIM syndrome: Therapeutic Assessment

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

Major Players in WHIM syndrome

There are approx. 1+ key companies which are developing the therapies for WHIM syndrome. The companies which have their WHIM syndrome drug candidates in the mid to advanced stage, i.e. phase III and Phase II include, X4 Pharmaceuticals and others.

Phases

This report covers around 1 +products under different phases of clinical development like

• Late-stage products (Phase III)
• 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

WHIM 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

• Subcutaneous
• Intravenous
• Oral
• Intramuscular

Molecule Type

Products have been categorized under various Molecule types such as

• Small molecules
• Natural metabolites
• Monoclonal antibodies

Product Type

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

WHIM syndrome: Pipeline Development Activities

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

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence WHIM syndrome R&D. The therapies under development are focused on novel approaches to treat/improve WHIM syndrome.

WHIM syndrome Report Insights

• WHIM syndrome Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• X4 Pharmaceuticals

Key Products

• X4P-001

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