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Find Novel Neurology Drugs in Preclinical Development in UNITED STATES

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            Lead Product(s): Golimumab

            Therapeutic Area: Neurology Product Name: ION373

            Highest Development Status: Preclinical Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 30, 2020

            Details:

            ION373 is an Ionis-owned investigational antisense medicine designed to stop the mutated gene from producing excess GFAP. Earlier this year, the European Medicines Agency (EMA) granted orphan drug designation to ION373.

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            Lead Product(s): M102

            Therapeutic Area: Neurology Product Name: M102

            Highest Development Status: Preclinical Product Type: Undisclosed

            Partner/Sponsor/Collaborator: FightMND

            Deal Size: $0.7 million Upfront Cash: Undisclosed

            Deal Type: Funding September 29, 2020

            Details:

            The grant will support the translational development of M102, a drug candidate for the treatment of amyotrophic lateral sclerosis (ALS), also known as motor neuron disease (MND) or Lou Gehrig's disease.

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            Lead Product(s): PBFT02

            Therapeutic Area: Neurology Product Name: PBFT02

            Highest Development Status: Preclinical Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 17, 2020

            Details:

            In the study, a single administration of an optimized adeno-associated virus containing the GRN gene resulted in elevated levels of progranulin in the brain and cerebral spinal fluid, normalized lysosomal enzyme expression and corrected microgliosis in a mouse models.

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            Lead Product(s): RNA editing therapies

            Therapeutic Area: Neurology Product Name: Undisclosed

            Highest Development Status: Preclinical Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Wu Capital

            Deal Size: $91.5 million Upfront Cash: Undisclosed

            Deal Type: Series A Financing September 10, 2020

            Details:

            This financing enables Korro to advance its lead program to IND filing and establish a broad portfolio of innovative RNA editing therapies. Korro’s OPERA platform, harnesses the body’s natural base editing system, specifically ADAR to make targeted edits to a single RNA base.

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            Lead Product(s): 4-Chlorokynurenine,Probenecid

            Therapeutic Area: Neurology Product Name: AV-101

            Highest Development Status: Preclinical Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 03, 2020

            Details:

            The results of this new study complement previous preclinical data demonstrating the combination's potential to substantially increase the brain concentration of AV-101's active metabolite, 7-Cl-KYN, thereby reducing, rather than blocking, NMDA receptor signaling.

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            Lead Product(s): KLS-13019

            Therapeutic Area: Neurology Product Name: KLS-13019

            Highest Development Status: Preclinical Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 25, 2020

            Details:

            With the PCT Patent, along with Kannalife’s novel lead drug candidate, KLS-13019, and its other novel therapeutic agents like Atopidine™, Kannalife now has near global coverage in the top pharmaceutical markets in the world.

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            Lead Product(s): Dantrolene Sodium

            Therapeutic Area: Neurology Product Name: Ryanodex

            Highest Development Status: Preclinical Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 19, 2020

            Details:

            The article reported results from an academic-based study that demonstrated dantrolene sodium administered intranasally improved both memory and cognition in a mouse model of Alzheimer’s disease. Eagle markets a formulation of dantrolene sodium but did not sponsor the study.

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            Lead Product(s): Tau and Alpha-synuclein vectorized antibody

            Therapeutic Area: Neurology Product Name: Alpha-synuclein vectorized antibody

            Highest Development Status: Preclinical Product Type: Large molecule

            Partner/Sponsor/Collaborator: AbbVie Inc

            Deal Size: $250.9 million Upfront Cash: Undisclosed

            Deal Type: Termination August 03, 2020

            Details:

            Voyager retains full rights to the vectorization technology and certain novel vectorized antibodies developed as part of the collaborations.

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            Lead Product(s): AAV based gene regulation therapy

            Therapeutic Area: Neurology Product Name: Undisclosed

            Highest Development Status: Preclinical Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Novartis Pharmaceuticals Corporation

            Deal Size: $795.0 million Upfront Cash: $75.0 million

            Deal Type: Collaboration July 30, 2020

            Details:

            Sangamo’s propriety genome regulation technology, zinc finger protein transcription factors (ZFP-TFs), aim to develop gene regulation therapies to address three neurodevelopmental targets, including autism spectrum disorder (ASD) and other neurodevelopmental disorders.

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            Lead Product(s): Pterostilbene,Epigallocatechin Gallate,Sulforaphane

            Therapeutic Area: Neurology Product Name: QuadraMune

            Highest Development Status: Preclinical Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 28, 2020

            Details:

            Pterostilbene is one of four ingredients which comprise QuadraMune™, a nutraceutical being developed by the Company which is currently in a clinical trial for prevention of COVID-19.

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