The study analysed that the Amyotrophic lateral sclerosis pipeline comprises of 69 drug candidates, of which one drug candidate is in Phase III stage, 16 drug candidates are in Phase II stage, nine drug candidates are in Phase I stage, 32 drug candidates are in Pre-Clinical stage, five candidates are inactive and six drug candidates are in discontinued stage.

1. GLOBAL INDUSTRY INSIGHT:
Amyotrophic Lateral Sclerosis Pipeline Analysis, 2017 - Clinical Trials &
Results, Patent, Designation, Collaboration, and Other Developments

2. Amyotrophic Lateral Sclerosis Exhibits Emerging Pipeline with
60+ Drug Candidates
According to a new research report “Amyotrophic Lateral Sclerosis Pipeline Analysis, 2017 -
Clinical Trials & Results, Patent, Designation, Collaboration, and Other Developments”
published by P&S Market Research, Amyotrophic lateral sclerosis currently exhibits a pipeline with
69 drug candidates.
Explore Report Overview at: https://www.psmarketresearch.com/market-analysis/amyotrophic-
lateral-sclerosis-pipeline-analysis
Amyotrophic Lateral Sclerosis pipeline in 2017
The study analysed that the Amyotrophic lateral sclerosis pipeline comprises of 69 drug candidates,
of which one drug candidate is in Phase III stage, 16 drug candidates are in Phase II stage, nine
drug candidates are in Phase I stage, 32 drug candidates are in Pre-Clinical stage, five candidates
are inactive and six drug candidates are in discontinued stage. Amyotrophic lateral sclerosis is a
neurodegenerative disorder in brain and spinal cord, the progression of the disease can further lead
to muscles getting enough nourishments, which can further cause atrophy of the muscles.
Amyotrophic Lateral Sclerosis occurs mostly in the lateral region of the body, in which spinal cord
reflexes are too intense. This can also lead to scarring and hardening of the muscles into that
region. Various new therapies and advanced technologies are driving the growth of Amyotrophic
lateral sclerosis pipeline.
Insights on pipeline segments
As per the findings of the research, around 31.0% of drug candidates for the treatment of
amyotrophic lateral sclerosis pipeline are being developed to be administered by oral route, 4.0%
by intravenous route, 48.0% are not disclosed and 17.0% are to be administered by other routes
which includes subcutaneous, intraperitoneal, sublingual, inhalation and intramuscular.
Explore Report Sample at: https://www.psmarketresearch.com/market-analysis/amyotrophic-
lateral-sclerosis-pipeline-analysis/report-sample
New innovating technologies offer development of promising drugs
Many technologies are being developed based on various innovative treatments that can control
the progression of Amyotrophic lateral sclerosis such as, NurOwn technology and Q-Cells
technology. The development of stem cells with the help of NurOwn technology, which is based on
novel differentiation protocol that differentiates the bone marrow-derived mesenchymal stem cells
into neuron-supporting cells, MSC-NTF cells, are capable of releasing several neurotrophic factors,
© P&S Market Research. All rights reserved 2

3. Amyotrophic Lateral Sclerosis Exhibits Emerging Pipeline with
60+ Drug Candidates
including Glial-derived neurotrophic factor (GDNF) and Brain-derived neurotrophic factor (BDNF),
both of which are critical for the growth, survival and differentiation of developing neurons.
Novel upcoming therapies for treatment of Amyotrophic lateral sclerosis
The combination of novel drugs for different organs as well as combination of drugs with biological
and biochemical agents may further enhance the treatment quality. Therefore, various novel
therapies are being used to improve quality of life of the patients, such as gene therapy, cell
therapy etc. Also, the inhibition of promising targets such as superoxide dismutase 1, interleukin 6,
Mesenchymal Bone Marrow Stromal Cells Secreting Neurotrophic Factors (MSC-NTF) are proven to
be effective for the treatment of amyotrophic lateral sclerosis.
Browse Other Related Report at: https://www.psmarketresearch.com/industry-
report/pharmaceuticals
More than ten drugs candidates with Orphan/Fast track drug designation
Amyotrophic lateral sclerosis pipeline has more than ten drugs, with either an orphan or a fast
track drug designation. In May 2016, European Commission approved and implemented the
decision related to the designation of "GM604" as an orphan medicinal product under Regulation
(EC) No 141/2000 of the European Parliament and of the Council. In December 2016, orphan
designation (EU/3/16/1801) was granted by the European Commission to MediciNova, Inc.
(Europe) Limited, for ibudilast for the treatment of Amyotrophic lateral sclerosis.
Major companies collaborate for the development of Amyotrophic Lateral Sclerosis
Pipeline
The research also found that various companies have collaborated for the development of
amyotrophic lateral sclerosis pipeline. In March 2013, Dainippon Sumitomo Pharma Co., Ltd. signed
a research, development and commercial licensing agreement with Edison Pharmaceuticals, Inc. for
EPI-743 and EPI-589 in Japan. Dainippon Sumitomo Pharma Co., Ltd. received exclusive research,
development and commercial rights in Japan for EPI-743 and EPI-589. Edison Pharmaceuticals,
Inc. received $35.0 million in an upfront payment and $15.0 million as research and development
support fee. In addition, Edison Pharmaceuticals, Inc. also received $10.0 to $35.0 million in
milestone payments per indication associated with successful development. After the launch,
Edison Pharmaceuticals, Inc. received royalties based on sales amounts and up to $460.0 million in
milestone payments in accordance with sales goals.
© P&S Market Research. All rights reserved 3

4. Amyotrophic Lateral Sclerosis Exhibits Emerging Pipeline with
60+ Drug Candidates
Many companies are developing Amyotrophic lateral sclerosis drug candidates in different phases.
Cytokinetics, Inc., AB Science, F. Hoffman La-Roche AG, FlexPharma, Inc., Orion Corporation,
Omeros Corporation, Genervon Biopharmaceuticals, LLC, Edison Pharmaceuticals, Inc., BrainStorm
Cell Therapeutics, Inc. and various other companies have their Amyotrophic lateral sclerosis drug
candidates in different stages of development. Cytokinetics, Inc. has drug candidate in Phase III
clinical stage.
Some of the key players developing drugs for Amyotrophic lateral sclerosis include FlexPharma,
Inc., Orion Corporation, Genervon Biopharmaceuticals, LLC, Orphazyme ApS, Edison
Pharmaceuticals, Inc., BrainStorm Cell Therapeutics, Inc. and Cytokinetics, Inc.
Chapter 1. Research Background
1.1 Research Objectives
1.2 Definition
1.3 Research Scope
1.3.1 Pipeline analysis by phase
1.3.2 Pipeline analysis by route of administration
1.3.3 Pipeline analysis by molecule type
1.4 Key Stakeholders
Chapter 2. Research Methodology
2.1 Secondary Research
2.2 Primary Research
2.2.1 Breakdown of primary research respondents
2.2.1.1 By industry participant
2.2.1.2 By company type
Chapter 3. Executive Summary
Chapter 4. Pipeline Outlook
4.1 Overview
4.2 Key Drivers
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5. Amyotrophic Lateral Sclerosis Exhibits Emerging Pipeline with
60+ Drug Candidates
4.2.1 Novel therapy for Amyotrophic lateral sclerosis pipeline
4.2.2 Use of various promising targets
4.3 Key Barriers
4.3.1 No early signs or symptoms
4.3.2 No specific treatment
4.4 Amyotrophic lateral sclerosis Pipeline Analysis
4.4.1 Pipeline analysis by phase
4.4.2 Pipeline analysis by molecule type
4.4.3 Pipeline analysis by route of administration
4.4.4 Pipeline analysis by company
4.5 Discontinued drugs
4.6 Inactive drugs
Chapter 5. Amyotrophic lateral sclerosis Pipeline Analysis by Phase (2017)
5.1 Phase III
5.1.1 Masitinib
5.1.1.1 Clinical trials
5.2 Phase II
5.2.1 FLX-787
5.2.1.1 Clinical trials
5.2.2 Olesoxime
5.2.2.1 Clinical trials
5.2.2.2 Strategic development
5.2.2.3 Clinical results
5.2.3 Tirasemtiv
5.2.3.1 Clinical trials
5.2.3.2 Strategic development
© P&S Market Research. All rights reserved 5

6. Amyotrophic Lateral Sclerosis Exhibits Emerging Pipeline with
60+ Drug Candidates
5.2.4 Levosimendan
5.2.4.1 Clinical trials
5.2.5 GM604
5.2.5.1 Clinical trials
5.2.5.2 Designation
5.2.6 Arimoclomol
5.2.6.1 Clinical trials
5.2.6.2 Strategic development
5.2.7 EPI-589
5.2.7.1 Clinical trials
5.2.7.2 Strategic development
5.2.8 Ibudilast
5.2.8.1 Clinical trials
5.2.8.2 Strategic development
5.2.9 Ezogabine
5.2.9.1 Clinical trials
5.2.9.2 Strategic development
5.2.10 Autologous MSC-NTF cells
5.2.10.1 Clinical trials
5.2.10.2 Strategic development
5.2.10.3 Technology
5.2.11 Deferiprone
5.2.11.1 Clinical trials
5.2.11.2 Strategic development
5.2.12 Seractide acetate
5.2.12.1 Clinical trials
© P&S Market Research. All rights reserved 6

7. Amyotrophic Lateral Sclerosis Exhibits Emerging Pipeline with
60+ Drug Candidates
5.2.12.2 Strategic development
5.2.13 RNS60
5.2.13.1 Pre-Clinical results
5.2.14 NP001
5.2.14.1 Clinical trials
5.2.14.2 Strategic development
5.2.15 TCH346
5.2.15.1 Clinical trials
5.2.16 ONO-2506PO
5.2.16.1 Clinical trials
5.3 Phase I
5.3.1 BHV-0223
5.3.1.1 Strategic development
5.3.1.2 Designation
5.3.2 Cutamesine
5.3.3 AAD-2004
5.3.3.1 Strategic development
5.3.4 Edaravone
5.3.4.1 Strategic development
5.3.5 Cu(II)ATSM
5.3.5.1 Clinical trials
5.3.6 Tocilizumab
5.3.6.1 Clinical trials
5.3.7 GDC-0134
5.3.7.1 Clinical trials
5.3.8 Q cells
© P&S Market Research. All rights reserved 7

8. Amyotrophic Lateral Sclerosis Exhibits Emerging Pipeline with
60+ Drug Candidates
5.3.8.1 Clinical trials
5.3.8.2 Pre-Clinical Studies
5.3.8.3 Strategic development
5.3.8.4 Technology
5.4 Pre-Clinical
Chapter 6. Competitive Landscape
6.1 Key Players Benchmarking for Amyotrophic Lateral Sclerosis Drug Candidates
6.2 SWOT Analysis of Amyotrophic Lateral Sclerosis Pipeline
Chapter 7. Company Profiles
7.1 Mallinckrodt plc
7.1.1 Business overview
7.1.2 Product and service offerings
7.2 Novartis AG
7.2.1 Business overview
7.2.2 Product and service offerings
7.3 Eisai & Co., Ltd.
7.3.1 Business overview
7.3.2 Product and service offerings
7.4 F. Hoffmann-La Roche Ltd.
7.4.1 Business overview
7.4.2 Product and service offerings
7.5 GlaxoSmithKline plc
7.5.1 Business overview
7.5.2 Product and service offerings
7.6 Bristol-Myers Squibb Company
7.6.1 Business overview
© P&S Market Research. All rights reserved 8

9. Amyotrophic Lateral Sclerosis Exhibits Emerging Pipeline with
60+ Drug Candidates
7.6.2 Product and service offerings
7.7 Mitsubishi Tanabe Pharma Corporation
7.7.1 Business overview
7.7.2 Product and service offerings
7.8 Bayer, AG
7.8.1 Business overview
7.8.2 Product and service offerings
7.9 Pfizer Inc.
7.9.1 Business overview
7.9.2 Product and service offerings
7.1 MediciNova, Inc.
7.10.1 Business overview
7.10.2 Product and service offerings
Chapter 8. Appendix
8.1 Abbreviations
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10. Amyotrophic Lateral Sclerosis Exhibits Emerging Pipeline with
60+ Drug Candidates
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