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    Find Neurology Drugs in Phase I/II Clinical Development

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              Prevail Therapeutics

              • Development Update

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              UNITED STATES United States

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              WRIB

              Participation Not Confirmed

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              Prevail Therapeutics Provides Clinical Advancement Update on PR001 for the Treatment of Parkinson’s Disease with GBA1 mutations

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              Lead Product(s): PR001

              Therapeutic Area: Neurology

              Highest Development Status: Phase I/ Phase II Product Type: Large molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable March 03, 2020

              Details:

              Enrollment in the PR001 Phase 1/2 PROPEL clinical trial is progressing, patient dosing continues, and the Company is on track to report interim data in the second half of 2020.

              Herantis Pharma

              • Development Update

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              FINLAND Finland

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              WRIB

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              Herantis Pharma Plc Announces Topline Results of Phase 1-2 CDNF Trial

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              Lead Product(s): CDNF

              Therapeutic Area: Neurology

              Highest Development Status: Phase I/ Phase II Product Type: Large molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable February 25, 2020

              Details:

              Topline analysis confirms positive safety and tolerability of CDNF in advanced-stage Parkinson's disease patients, with encouraging biological responses.

              BioXcel Therapeutics

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              UNITED STATES United States

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              WRIB

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              BioXcel Therapeutics Announces Proposed Public Offering

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              Lead Product(s): Dexmedetomidine

              Therapeutic Area: Neurology

              Highest Development Status: Phase I/ Phase II Product Type: Small molecule

              Partner/Sponsor/Collaborator: BofA Securities

              Deal Size: $64.0 million Upfront Cash: Undisclosed

              Deal Type: Public Offering February 19, 2020

              Details:

              The Company intends to use the net proceeds of this offering to advance the development of product candidates, including the funding of clinical trials for BXCL501.

              resTORbio

              • Development Update

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              UNITED STATES United States

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              WRIB

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              resTORbio Announces Interim Results for Phase 1b/2a Trial of RTB101 in Patients with Parkinson’s Disease

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              Lead Product(s): RTB101

              Therapeutic Area: Neurology

              Highest Development Status: Phase I/ Phase II Product Type: Small molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable February 19, 2020

              Details:

              Interim data from three cohorts in the Phase 1b/2a study demonstrate that RTB101 is well tolerated, effective in preclinical models to inhibit the activity of TORC1 and induce autophagy.

              Alexion Pharmaceuticals

              • Development Update

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              UNITED STATES United States

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              WRIB

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              Alexion Announces Planned Initiation of Pivotal Phase 3 Study of ULTOMIRIS® (ravulizumab) in ALS

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              Lead Product(s): Ravulizumab

              Therapeutic Area: Neurology

              Highest Development Status: Phase I/ Phase II Product Type: Large molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable January 14, 2020

              Details:

              A 50-week global study to enroll approximately 350 patients across broad ALS population.

              Axovant Sciences

              • Development Update

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              UNITED STATES United States

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              WRIB

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              AXOVANT ANNOUNCES POSITIVE 12-MONTH DATA ON AXO-LENTI-PD AND PROVIDES UPDATES ACROSS GENE THERAPY PIPELINE PROGRAMS

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              Lead Product(s): AXO-Lenti-PD

              Therapeutic Area: Neurology

              Highest Development Status: Phase I/ Phase II Product Type: Large molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable January 13, 2020

              Details:

              AXO-Lenti-PD was observed to be well-tolerated with no serious adverse events attributable to the gene therapy.

              Kadimastem

              • Development Update

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              ISRAEL Israel

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              WRIB

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              Kadimastem reports positive ALS cell therapy results

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              Lead Product(s): AstroRx

              Therapeutic Area: Neurology

              Highest Development Status: Phase I/ Phase II Product Type: Large molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable January 02, 2020

              Details:

              A statistically significant decline in disease progression was observed during the first 3- 4 months post-treatment period, indicating a clinical benefit of the AstroRx® cell product.

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