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Find Drugs for Genetic Disease in Phase II/ Phase III Clinical Development

A collaboration with SCORR Marketing

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            Lead Product(s): Olipudase alfa

            Therapeutic Area: Genetic Disease

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 30, 2020

            Details:

            Olipudase alfa is the first and only investigational enzyme replacement therapy in late-stage development for the treatment of ASMD.

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            Lead Product(s): ZYN002

            Therapeutic Area: Genetic Disease

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 26, 2020

            Details:

            The trial is evaluating the efficacy and safety of Zygel™ CBD Gel as a treatment for behavioral symptoms of Fragile X syndrome (FXS) in 210 children ages three through 17 with full mutation FXS.

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            Lead Product(s): LYS-SAF302

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase II/ Phase III 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:

            The AAVance Phase 2/3 clinical study is designed as an open-label, single-arm, multicenter study of LYS-SAF302 for the treatment of MPS IIIA.

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            Lead Product(s): Elamipretide

            Therapeutic Area: Genetic Disease

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 18, 2020

            Details:

            Elamipretide achieves statistical significance on key secondary endpoints

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            Lead Product(s): JR-141

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase II/ Phase III Product Type: Undisclosed

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 13, 2020

            Details:

            The presentations highlight two candidates from JCR’s development pipeline for lysosomal storage disorders; JR-141 and JR-162.

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            Lead Product(s): Cannabidiol

            Therapeutic Area: Genetic Disease

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 03, 2020

            Details:

            Zynerba Pharma has successfully achieved its patient screening target in its 14-week pivotal CONNECT-FX trial assessing the efficacy and safety of Zygel™ CBD Gel in children and adolescents.

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            Lead Product(s): Cannabidiol

            Therapeutic Area: Genetic Disease

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 13, 2020

            Details:

            The trial has enrolled more severely affected population than that enrolled in the previously completed Phase 2 FAB-C trial as measured by baseline behavioral symptoms.

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            Lead Product(s): Arimoclomol

            Therapeutic Area: Genetic Disease

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 03, 2020

            Details:

            Orphazyme expects to receive approval for NPC in H1 2021 in the US through the fast track approval process, with the aim to bring the product to patients shortly thereafter.