Neutropenia - Pipeline Insight, 2024

This “Neutropenia- Pipeline Insight, 2024” report provides comprehensive insights about 15+ companies and 15+ pipeline drugs in Neutropenia 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 admin istration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered

• Global coverage

Neutropenia: Understanding

Neutropenia: Overview

Neutropenia is defined as a lower than normal number of neutrophils (a type of white blood cells). Neutropenia can be classified in asymptomatic (mild), moderate, and severe, and thus, the progression to infection concerning the number.

Neutropenia, with decreased production with marrow hypoplasia, can be primary and due to chronic benign neutropenia, cyclical neutropenia, and other congenital and familial neutropenias. It can be secondary to cytotoxic drugs, aplastic anemia, leukemia, drug reactions, and infections. Neutropenia, with increased destruction with marrow hyperplasia, is due to hypersplenism and immune neutropenia. Secondary causes are the commonest. For example, neutropenia caused as a side effect of chemotherapy for malignancies. Congenital forms are rare and vary in severity; some of them are life-threatening conditions including leukocyte adhesion deficiency, Chediak-Higashi syndrome, hyper-IgE, recurrent infection syndrome, and chronic granulomatous disease.

Pathophysiology
Neutrophils play a role in the immune defense against extracellular bacteria, including Staphylococci, Streptococci, and Escherichia coli, among others. They also protect against fungal infections, including those produced by Candida albicans. Once their count is below 1 x 10/L recurrent infections start. As compensation, the monocyte count may increase. In primary neutropenia disorders such as chronic granulomatous disease presents with recurrent infections affecting many organs since childhood. It is caused from a failure to produce toxic reactive oxygen species so that the neutrophils can ingest the microorganisms, but they are unable to kill them, as a significant consequence granuloma can obstruct organs such as the stomach, esophagus, or bladder. Patients with this disease are very susceptible to opportunistic infections by certain bacteria and fungi, especially with Serratia and Burkholderia.

Symptoms

• Fever
• Chills
• Mouth sores
• Cough
• Difficulty breathing
• Abdominal or rectal pain.

Treatment
Treatment may include one or a combination of the following:
Myeloid growth factors: These are proteins that stimulate the bone marrow to produce more white blood cells to help the body fight infections. They are also known as growth factors or colony-stimulating factors (CSFs). Examples of growth factors include filgrastim (Neupogen, other names), tbo-filgrastim (Granix), pegfilgrastim (Neulasta, other names), and eflapegrastim (Rolvedon).- Antibiotics may be given in certain instances to help prevent infection.

• Cancer treatment may be delayed to give body enough time to make more white blood cells and recover your ANC.

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

Neutropenia Emerging Drugs Chapters

This segment of the Neutropenia 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.

Neutropenia Emerging Drugs

Mavorixafor: X4 Pharmaceuticals
Mavorixafor is an investigational, orally available CXCR4 antagonist designed to facilitate the mobilization of white blood cells from the bone marrow into the blood, to increase levels of circulating neutrophils, lymphocytes, and monocytes, and to improve immune system function. In September 2022, company announced the successful completion of a proof-of-concept, Phase 1b clinical trial, designed to assess the safety and tolerability of a single daily dose of oral mavorixafor, with or without G-CSF, in participants with chronic neutropenic disorders including idiopathic, cyclic, and congenital neutropenia. Results showed that 100% of study participants (n=25) achieved robust responses to mavorixafor and that 100% of neutropenic participants (n=14) achieved normalized neutrophil counts.

It is currently investigated in Phase II clinical trial (NCT04154488) that aims to evaluate the safety and efficacy of chronic daily oral mavorixafor with or without G-CSF in people with chronic neutropenic disorders.

AKT-011: AKTHELIA
Akthelia's unique, innovative approach is to reduce this bacterial translocation by enhancing the barrier resistance of the GI tract to bacteria through upregulation of endogenous defence mechanisms. This holds the promise of a new standard of care treatment with potential to replace prophylactic use of fluoroquinolones and other antibiotics currently administered to patients at risk for febrile neutropenia, with reduced risk of SAEs, AMR, and microbiome dysbiosis. Its lead candidate AKT-011 is aimed at the gastro-intestinal system and has low bio-availability. The compound acts locally with less systemic risk of toxicity outside treatment area.

AKT-011 has high permeability into epithelial cells which is important as epithelial cells are the targets that express host defense peptides. With promising data it shows that its lead small molecule drug candidate effectively knocks down translocation in animal models and it is currently completing pre-clinical studies to demonstrate translatable survival benefit.

Neutropenia: Therapeutic Assessment

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

Major Players in Neutropenia

There are approx. 15+ key companies which are developing the therapies for Neutropenia. The companies which have their Neutropenia drug candidates in the most advanced stage, i.e. phase III.

Phases

This report covers around 15+ 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

Neutropenia 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.

Neutropenia: Pipeline Development Activities

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

Neutropenia Report Insights

• Neutropenia Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• X4 Pharmaceuticals
• AKTHELIA
• Spectrum Pharmaceuticals
• CSPC ZhongQi Pharmaceutical Technology Co., Ltd.
• Mabwell (Shanghai) Bioscience Co., Ltd.
• EmendoBio
• Phyto HealthCorporation
• Xiamen Amoytop Biotech Co., Ltd.

Key Products

• Mavorixafor
• AKT-011
• Eflapegrastim
• Amphotericin B liposomes
• MW05
• EMD-101
• Astragalus polysaccharides
• YPEG-rhG-CS

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