Neuromyelitis Optica Spectrum Disorder Nmosd Pipeline Insight

DelveInsight’s, “Neuromyelitis Optica Spectrum Disorder (NMOSD) – Pipeline Insights, 2021” report provides comprehensive insights about 12+ companies and 12+ pipeline drugs in Neuromyelitis Optica Spectrum Disorder 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

Neuromyelitis Optica Spectrum Disorder Understanding

Neuromyelitis Optica Spectrum Disorder: Overview

Neuromyelitis optica spectrum disorder (NMOSD), also known as Devic disease, is a chronic disorder of the brain and spinal cord dominated by inflammation of the optic nerve (optic neuritis) and the spinal cord (myelitis). Classically, it was felt to be a monophasic illness, consisting of episodes of inflammation of one or both optic nerves and the spinal cord over a short period of time (days or weeks) but, after the initial episode, no recurrence. It is now recognized that most patients satisfying current criteria for NMOSD experience repeated attacks separated by periods of remission. The interval between attacks may be weeks, months or years. In its early stages, NMOSD may be confused with multiple sclerosis (MS).

Symptoms of Neuromyelitis Optica Spectrum Disorder

Most symptoms are related to optic nerve, spinal cord, and brainstem inflammation and include:

  • Loss or blurring of vision in one or both eyes
  • Loss of color vision
  • Paralysis (no motor function) of a limb or limbs
  • Paraparesis (weakness) of a limb or limbs
  • Loss of sensation
  • Loss of bladder or bowel control
  • Profound bladder retention
  • Intractable nausea and vomiting
  • Intractable hiccups

Causes of Neuromyelitis Optica Spectrum Disorder

Greater than 95% of patients with NMOSD report no relatives with the disease, but approximately 3% report having other relatives with the condition. There is a strong association with a personal or family history of autoimmunity, which are present in 50% of cases. NMOSD is regarded as an autoimmune disease though the exact cause for the autoimmunity is unknown.

Treatment of Neuromyelitis Optica Spectrum Disorder

For acute attacks, the standard treatment is high-dose intravenous corticosteroids, typically methylprednisolone. Plasma exchange may be effective in patients who experience acute severe attacks that do not response to intravenous corticosteroids.


For long-term suppression of the disease, a variety of immunosuppressive drugs are regarded by many clinicians as first-line therapy. Corticosteroids, azathioprine, mycophenolate mofetil and rituximab are the treatments most widely prescribed treatments.


Symptom treatment may also involve the use of low doses of carbamazepine to control paroxysmal (sudden) tonic spasms that often occur during attacks of NMOSD and antispasticity agents to treat long term complication of spasticity that frequently develops in those with permanent motor deficits.

Neuromyelitis Optica Spectrum Disorder Emerging Drugs Chapters

This segment of the Neuromyelitis Optica Spectrum Disorder report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, I and preclinical. 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 Emerging Drugs

  • Satralizumab: Hoffmann-La Roche

Satralizumab, created by Chugai, is an anti-IL-6 receptor recycling antibody. The drug is expected to suppress relapse of NMOSD by inhibiting IL-6 signal transduction which is deeply related to the pathology. In two global phase III clinical studies in neuromyelitis optica (NMO) and NMOSD patients, the primary endpoint was achieved with satralizumab either as an add-on therapy to baseline treatment or as monotherapy. It is designated as an orphan drug for the treatment of NMO and NMOSD in Japan, and for the treatment of the same disease group in Europe and the US. In addition, it has been granted Breakthrough Therapy Designation by the US Food and Drug Administration in December 2018. In October 2019, EMA and FDA Accept Marketing Applications for Chugai's Satralizumab in Neuromyelitis Optica Spectrum Disorder (NMOSD). The EMA’s Committee for Medicinal Products for Human Use (CHMP) recommendation and the FDA decision are expected in 2020.


  • Ravulizumab: Alexion Pharmaceuticals

Alexion Pharmaceuticals is developing Ravulizumab in phase III stage of development for the treatment of Neuromyelitis Optica Spectrum Disorder. It is the first and only long-acting C5 inhibitor administered every eight weeks. Ultomiris works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. The terminal complement cascade, when activated in an uncontrolled manner, plays a role in severe ultra-rare disorders like PNH, including atypical hemolytic uremic syndrome (aHUS), anti-acetylcholine receptor (AchR) antibody-positive myasthenia gravis (MG) and anti-aquaporin-4 (AQP4) auto-antibody-positive Neuromyelitis Optica Spectrum Disorder (NMOSD).


  • Telitacicept: RemeGen

Investigational candidate RC18 is a fusion antibody created by RemeGen scientists to target signaling factors involved in the development and survival of B cells, the cell responsible for generating antibodies. RC18 is a fusion of a TACI (transmembrane activator and calcium modulator and cyclophilin ligand interactors) protein and the IgG protein. RC18 binds to BLyS (B lymphocyte stimulator) and APRIL (a proliferation-inducing ligand), preventing these cell-signaling molecules from binding to TACI proteins on the surface of the B cell. This inhibits the development and survival of mature B cells, preventing the formation of autoantibodies. The product is currently in phase III stage of development for Neuromyelitis Optica Spectrum Disorder.


  • HBM9161: Harbour BioMed

HBM9161 is a fully human anti-FcRn monoclonal antibody which is being developed by Harbour BioMed in clinical stage for the treatment of Neuromyelitis Optica Spectrum Disorder (NMOSD). FcRn plays a pivotal role in preventing the degradation of IgG antibodies. The physiologic function of FcRn is to modulate the catabolism of IgG antibodies, and inhibition of FcRn, such as through use of an FcRn targeting antibody, has been shown to reduce levels of pathogenic IgG antibodies. HBM9161 is a fully human monoclonal antibody targeting the FcRn receptor. Blocking the FcRn-IgG interaction may alleviate flare-ups in neuromyelitis optica spectrum disorder. HBM licensed HBM9161 from HanAll Biopharma and has the right to develop, manufacture and commercialize the product in Greater China (including Hong Kong, Macau and Taiwan).


  • Lu AG06466: Lundbeck

Lu AG06466 (formerly ABX-1431) is being developed by Abide Therapeutics which is currently in phase I stage of development for the treatment of Neuromyelitis Optica Spectrum Disorder. ABX-1431 is a first-in-class, investigational oral therapy designed to inhibit monoacylglycerol lipase (MGLL), an enzyme that regulates a key system that serves as a natural brake on excessive brain signaling. Inhibition of MGLL by ABX-1431 may potentiate putting the brakes on overactive neural circuits, which may serve to correct abnormal neurotransmission that is often dysregulated in neurological diseases. In May 2019, H. Lundbeck and Abide Therapeutics has signing of a definitive agreement in which Lundbeck has agreed to acquire Abide.


  • BAT 4406F: Bio-Thera Solutions

BAT4406F, developed by Bio-Thera Solutions is being evaluated in phase I of development for neuromyelitis optica spectrum disorders. This is a Phase 1, open-label, dose-escalation study in NMOSD patients in which subjects will receive BAT4406F injection via intravenous infusion. A 3 + 3 design will be utilized to define a maximum tolerated dose (MTD). The overall objective is to assess the safety, tolerability, and pharmacokinetics of BAT4406F injection in NMOSD patients.

Further product details are provided in the report……..

Neuromyelitis Optica Spectrum Disorder: Therapeutic Assessment

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

  • Major Players in Neuromyelitis Optica Spectrum Disorder

There are approx. 12+ key companies which are developing the therapies for Neuromyelitis Optica Spectrum Disorder. The companies which have their Neuromyelitis Optica Spectrum Disorder drug candidates in the most advanced stage, i.e. Pre-registration and Phase III phase include, Chugai/Roche, Alexion Pharmaceuticals and RemeGen.

  • Phases

DelveInsight’s report covers around 12+ products under different phases of clinical development like

  • Late stage products (Pre-registration and Phase III)
  • Mid-stage products (Phase II and Phase I/II) and
  • Early-stage products (Phase I) along with the details of
  • Pre-clinical and Discovery stage candidates
  • Discontinued & Inactive candidates
  • Route of Administration

Neuromyelitis Optica Spectrum Disorder pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

  • Intravenous
  • Oral
  • Subcutaneous etc.
  • Mechanism of Action

Products have been categorized under various MOAs such as

  • Amyloid beta-protein inhibitors
  • Fc receptor antagonists
  • Monoacylglycerol lipase inhibitors
  • Neuron modulators
  • T lymphocyte stimulants
  • Interleukin 6 receptor antagonists
  • CD20 antigen inhibitors
  • CD19-expressing B cells depletion
  • B cell activating factor inhibitors
  • RORg antagonist
  • Molecule Type
  • Amyloid beta-protein inhibitors
  • Fc receptor antagonists
  • Monoacylglycerol lipase inhibitors
  • Neuron modulators
  • T lymphocyte stimulants
  • Interleukin 6 receptor antagonists
  • CD20 antigen inhibitors
  • CD19-expressing B cells depletion
  • B cell activating factor inhibitors
  • RORg antagonist

Products have been categorized under various Molecule types such as

  • Product Type

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

Neuromyelitis Optica Spectrum Disorder: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase III, II, I and preclinical stage. It also analyses Neuromyelitis Optica Spectrum Disorder 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 Neuromyelitis Optica Spectrum Disorder drugs.

Report Highlights

  • The companies and academics are working to assess challenges and seek opportunities that could influence Neuromyelitis Optica Spectrum Disorder R&D. The therapies under development are focused on novel approaches to treat/improve Neuromyelitis Optica Spectrum Disorder.
  • In 2019, Soliris (eculizumab) was approved by the US Food and Drug Administration (FDA) for the treatment of NMOSD in adult patients who are anti-aquaporin-4 (AQP4) antibody positive. In 2020, Uplinza (inebilizumab-cdon) was approved for the treatment of NMOSD in adult patients with the same AQP4 antibody.
  • In February 2020, Harbour BioMed (HBM) announced completion of a Phase I study of HBM9161, a fully human anti-FcRn monoclonal antibody, to evaluate its safety, tolerability, pharmacokinetics and pharmacodynamics in healthy Chinese subjects. The results demonstrated an excellent safety profile and potent IgG reduction capability.
  • In May 2019, H. Lundbeck and Abide Therapeutics announced signing of a definitive agreement in which Lundbeck has agreed to acquire Abide. Under the terms of the agreement, Lundbeck may pay USD 250 million (approximately DKK 1.65 billion) upfront with a commitment to pay future development and sales milestones to the group of current owners of up to USD 150 million (approximately DKK 1 billion). This acquisition provides Lundbeck a novel discovery platform and a U.S.-based research hub. Further to the platform, Lundbeck also gets a number of development projects, the most advanced being ABX-1431.
  • In September 2019, Chugai Pharmaceutical announced that satralizumab received orphan drug designation by the Ministry of Health, Labour and Welfare (MHLW) for neuromyelitis optica (NMO) and neuromyelitis optica spectrum disorder (NMOSD). In December 2018, Chugai Pharmaceutical announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for Chugai’s anti-interleukin-6 (IL-6) receptor humanized recycling antibody satralizumab. In June 2016, orphan designation was granted by the European Commission to Chugai Pharma Europe Ltd, UK, for humanised anti-IL-6 receptor monoclonal antibody (also known as SA237) for the treatment of neuromyelitis optica spectrum disorders. Satralizumab is designated as an orphan drug in the US.
  • In August 2016, TG Therapeutics announced that the US Food and Drug Administration (FDA) has granted orphan drug designation for TG-1101 (ublituximab) the Company's novel, glycoengineered anti-CD20 monoclonal antibody, for the treatment of patients with neuromyelitis optica (NMO) and neuromyelitis optica spectrum disorder (NMOSD).
  • HanAll Biopharma has signed again a large-scale technology transfer agreement. This contract on HL 161, autoimmune antibody, is worth up to 500 million dollars. HanAll Biopharma announced that they have transferred technology for the treatment of autoimmune disease antibody to Roivant Sciences. Under the terms of agreement, Roivant Sciences will now have exclusive rights to clinical development, production, and product licensing in North America, Latin America, the EU, the UK, Switzerland, the Middle East and North Africa.

Neuromyelitis Optica Spectrum Disorder Report Insights

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

Neuromyelitis Optica Spectrum Disorder 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 drugs?
  • How many Neuromyelitis Optica Spectrum Disorder 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?
  • What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Neuromyelitis Optica Spectrum Disorder 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 and their status?
  • What are the key designations that have been granted to the emerging drugs?


Introduction

Executive Summary

Multiple Myeloma: Overview

• Causes

• Mechanism of Action

• Signs and Symptoms

• Diagnosis

• Disease Management

Pipeline Therapeutics

• Comparative Analysis

Therapeutic Assessment

• Assessment by Product Type

• Assessment by Stage and Product Type

• Assessment by Route of Administration

• Assessment by Stage and Route of Administration

• Assessment by Molecule Type

• Assessment by Stage and Molecule Type

Multiple Myeloma – DelveInsight’s Analytical Perspective

In-depth Commercial Assessment

• Multiple Myeloma companies’ collaborations, Licensing, Acquisition -Deal Value Trends

Multiple Myeloma Collaboration Deals

• Company-Company Collaborations (Licensing / Partnering) Analysis

• Company-University Collaborations (Licensing / Partnering) Analysis

Late Stage Products

• Comparative Analysis

Idecabtagene vicleucel: Bristol-Myers Squibb/Bluebird bio

• Product Description

• Research and Development

• Product Development Activities

Belantamab mafodotin (GSK2857916): GlaxoSmithKline

• Product Description

• Research and Development

• Product Development Activities

Drug profiles in the detailed report…..

Mid Stage Products

• Comparative Analysis

LB-401: Leadiant Biosciences Ltd.

• Product Description

• Research and Development

• Product Development Activities

Durvalumab: Celgene Corporation (acquired by Bristol-Myers Squibb Company)

• Product Description

• Research and Development

• Product Development Activities

Drug profiles in the detailed report…..

Early Stage Products

• Comparative Analysis

ABBV-075: AbbVie

• Product Description

• Research and Development

• Product Development Activities

S65487: ADIR (Servier Group)

• Product Description

• Research and Development

• Product Development Activities

Drug profiles in the detailed report…..

Pre-clinical and Discovery Stage Products

• Comparative Analysis

HDP-101-ATAC: Heidelberg Pharma AG

• Product Description

• Research and Development

• Product Development Activities

Drug profiles in the detailed report…..

Inactive Products

• Comparative Analysis

Multiple Myeloma Key Companies

Multiple Myeloma Key Products

Multiple Myeloma - Unmet Needs

Multiple Myeloma - Market Drivers and Barriers

Multiple Myeloma - Future Perspectives and Conclusion

Multiple Myeloma Analyst Views

Multiple Myeloma Key Companies

Appendix

List of Tables

Table 1 Total Products for Multiple Myeloma

Table 2 Late Stage Products

Table 3 Mid Stage Products

Table 4 Early Stage Products

Table 5 Pre-clinical & Discovery Stage Products

Table 6 Assessment by Product Type

Table 7 Assessment by Stage and Product Type

Table 8 Assessment by Route of Administration

Table 9 Assessment by Stage and Route of Administration

Table 10 Assessment by Molecule Type

Table 11 Assessment by Stage and Molecule Type

Table 12 Inactive Products

List of Figures

Figure 1 Total Products for Multiple Myeloma

Figure 2 Late Stage Products

Figure 3 Mid Stage Products

Figure 4 Early Stage Products

Figure 5 Preclinical and Discovery Stage Products

Figure 6 Assessment by Product Type

Figure 7 Assessment by Stage and Product Type

Figure 8 Assessment by Route of Administration

Figure 9 Assessment by Stage and Route of Administration

Figure 10 Assessment by Molecule Type

Figure 11 Assessment by Stage and Molecule Type

Figure 12 Inactive Products

• Hoffmann-La Roche

• Alexion Pharmaceuticals

• RemeGen

• Nihon Pharmaceutical

• Harbour BioMed

• Lundbeck

• Bionure

• Opexa Therapeutics

• TG Therapeutics

• Bio-Thera Solutions

• Boston Pharmaceuticals

• Cour Pharmaceutical

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