Neuromyelitis Optica Spectrum Disorder (NMOSD) - Pipeline Insight, 2025

This “Neuromyelitis Optica Spectrum Disorder (NMOSD) - Pipeline Insight, 2025” report provides comprehensive insights about 10+ companies and 12+ pipeline drugs in Neuromyelitis Optica Spectrum Disorder (NMOSD) 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.

Neuromyelitis Optica Spectrum Disorder (NMOSD): Understanding

Neuromyelitis Optica Spectrum Disorder (NMOSD): Overview

Neuromyelitis optica spectrum disorder (NMOSD), formerly known as neuromyelitis optica or Devic’s disease, was initially thought to be a variant of multiple sclerosis but is now recognized as a distinct autoimmune disorder characterized by inflammation of the optic nerves and spinal cord. Advances in immunology led to the discovery of aquaporin-4 immunoglobulin G antibodies (AQP4-IgG), which are present in many affected patients and have expanded the clinical definition of the disease. Today, NMOSD encompasses a broader spectrum of neurologic disorders beyond classical optic neuritis and transverse myelitis, reflecting its complex and evolving nature.

Neuromyelitis Optica Spectrum Disorder (NMOSD) is an autoimmune condition that targets the optic nerves and spinal cord, leading to a wide array of neurological symptoms. A hallmark feature is optic neuritis, which presents as eye pain, blurred vision, or vision loss. Transverse myelitis can cause limb weakness, numbness, and sensory disturbances due to spinal cord inflammation. Some patients may experience area postrema syndrome, characterized by persistent nausea, vomiting, and hiccups due to brainstem involvement. Additional symptoms include bladder or bowel dysfunction, muscle spasms, fatigue, and cognitive changes such as brain fog or mood disturbances.

Neuromyelitis Optica Spectrum Disorder (NMOSD) is an inflammatory autoimmune condition that primarily targets the optic nerves and spinal cord, with frequent involvement of the brainstem, particularly the area postrema. The disease is strongly associated with IgG antibodies against aquaporin-4 (AQP4), a water channel protein concentrated on astrocyte foot processes in key areas of the central nervous system. These antibodies are present in 60% to 90% of patients and are highly specific for NMOSD. The autoimmune response leads to perivascular lymphocytic infiltration, astrocyte damage, demyelination, and axonal loss, particularly in regions rich in AQP4 such as the optic nerves, spinal cord, and circumventricular zones like the periaqueductal gray matter.

Due to the rarity of NMOSD, standardized treatment guidelines are limited, but most approaches involve an initial short course of high-dose intravenous corticosteroids, such as methylprednisolone, to manage acute relapses. In refractory cases, plasmapheresis or intravenous immunoglobulin may be employed. Long-term management focuses on preventing relapses through chronic immunosuppressive therapy, with first-line agents including azathioprine and rituximab. Second-line treatments like mycophenolate mofetil and methotrexate are also used, often offering the advantage of less frequent dosing. Newer biologic therapies targeting IL-6, complement proteins, or AQP4-IgG are being explored for their specificity and effectiveness. Proper differentiation from multiple sclerosis is crucial, as certain MS therapies can worsen NMOSD.
"Neuromyelitis Optica Spectrum Disorder (NMOSD) - Pipeline Insight, 2025" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Neuromyelitis Optica Spectrum Disorder (NMOSD) pipeline landscape is provided which includes the disease overview and Neuromyelitis Optica Spectrum Disorder (NMOSD) treatment guidelines. The assessment part of the report embraces, in depth Neuromyelitis Optica Spectrum Disorder (NMOSD) 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, Neuromyelitis Optica Spectrum Disorder (NMOSD) 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 Neuromyelitis Optica Spectrum Disorder (NMOSD) R&D. The therapies under development are focused on novel approaches to treat/improve Neuromyelitis Optica Spectrum Disorder (NMOSD).

Neuromyelitis Optica Spectrum Disorder (NMOSD) Emerging Drugs Chapters

This segment of the Neuromyelitis Optica Spectrum Disorder (NMOSD) report encloses its detailed analysis of various drugs in different stages of clinical development, including Phase III, 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.

Neuromyelitis Optica Spectrum Disorder (NMOSD) Emerging Drugs

BAT4406F: Bio-Thera Solutions

BAT4406 is an investigational ADCC-enhanced anti-CD20 mAb candidate in clinical development for the treatment of autoimmune diseases. BAT4406F is currently being evaluated in NMOSD, an orphan indication with an estimated prevalence of 0.5 to 10 per 100,000. BAT4406 is a type I glyco-engineered mAb that binds specifically to CD20 on B-cells, kills the B-cells by CDC, and enhances ADCC effect. B cells have been implicated in the pathogenesis of a number of autoimmune diseases, including the CNS disorders, multiple sclerosis (MS) and NMOSD. Depletion of B-cells could provide meaningful relief for these autoimmune diseases. NMOSD is an autoimmune inflammatory disorder of the central nervous system (CNS) with preferential localization to the optic nerve, spinal cord and brain stem. Patients typically experience bouts of vision loss or blindness, attacks of myelitis with often severe motor impairment including loss of ambulation, sensory disturbances, bowel/bladder dysfunction, and brainstem attacks with characteristic episodes of intractable nausea, vomiting and hiccups. Currently, the drug is in Phase III stage of its development for the treatment of Neuromyelitis Optica Spectrum Disorder (NMOSD).

B001: Shanghai Jiaolian Drug Research and Development Co., Ltd

B001 is a recombinant humanized anti-CD20 monoclonal antibody developed by Shanghai Jiaolian Drug Research & Development (originating from Shanghai Pharmaceuticals Holding). It works by targeting CD20 on B cells and triggering antibody-dependent cellular cytotoxicity (ADCC) to deplete autoreactive B cells, a mechanism aligned with current NMOSD treatment goals. Designed for intravenous administration, B001 aims to reduce disease relapses in AQP4-IgG positive NMOSD through sustained B-cell suppression. It also shows potential across other autoimmune neurologic conditions and B-cell malignancies, illustrating a broad therapeutic strategy rooted in targeted immunotherapy. Currently, the drug is in Phase II/III stage of its development for the treatment of Neuromyelitis Optica Spectrum Disorder (NMOSD).

CT103A cells: Nanjing IASO Biotherapeutics

Equecabtagene Autoleucel (Eque-cel) is an innovative fully human anti-BCMA CAR-T cell therapy which uses lentivirus as a gene vector to transfect autologous T cells. The CAR contains a fully human scFv, CD8a hinge and transmembrane, and 4-1BB co-stimulatory and CD3ζactivation domains. Based on rigorous molecular structure screening and comprehensive in vitro and in vivo functional evaluations, Eque-cel demonstrates rapid and potent efficacy, accompanied by exceptional long-term persistence in vivo, enabling patients to achieve higher and deeper responses. Currently, the drug is in Phase I stage of its development for the treatment of Neuromyelitis Optica Spectrum Disorder (NMOSD).

Neuromyelitis Optica Spectrum Disorder (NMOSD): Therapeutic Assessment

This segment of the report provides insights about the different Neuromyelitis Optica Spectrum Disorder (NMOSD) drugs segregated based on following parameters that define the scope of the report.

Major Players in Neuromyelitis Optica Spectrum Disorder (NMOSD)

• There are approx. 10+ key companies which are developing the therapies for Neuromyelitis Optica Spectrum Disorder (NMOSD). The companies which have their Neuromyelitis Optica Spectrum Disorder (NMOSD) drug candidates in the most advanced stage, i.e. Phase III include, Bio-Thera Solutions.

Phases
The report covers around 12+ 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
Neuromyelitis Optica Spectrum Disorder (NMOSD) pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs, such as:

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

Molecule Type
Products have been categorized under various Molecule types, such as:

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

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

Neuromyelitis Optica Spectrum Disorder (NMOSD): Pipeline Activities

The report provides insights into different therapeutic candidates in Phase III, II, I, preclinical and discovery stage. It also analyses Neuromyelitis Optica Spectrum Disorder (NMOSD) therapeutic drugs key players involved in developing key drugs.

Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Neuromyelitis Optica Spectrum Disorder (NMOSD) drugs.

Neuromyelitis Optica Spectrum Disorder (NMOSD) Report Insights

• Neuromyelitis Optica Spectrum Disorder (NMOSD) Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Bio-Thera Solutions
• Bioray Laboratories
• Nanjing Bioheng Biotech Co., Ltd.
• Nanjing IASO Biotherapeutics
• Shanghai Jiaolian Drug Research and Development Co., Ltd
• Shanghai Xiniao Biotech Co., Ltd.

Key Products

• BAT4406F
• Universal BCMA-CD19 CART
• RD06-04 cell infusion
• CT103A cells
• B001
• UCAR T-cell

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