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.

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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.

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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, 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.

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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.

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.

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