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

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            Lead Product(s): Elamipretide

            Therapeutic Area: Genetic Disease Product Name: MTP-131

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 19, 2020

            Details:

            The poster titled "TAZPOWER Analysis: Elamipretide Significantly Improves Disease Symptomatology versus Natural History Controls in Barth Syndrome," shows the importance of longitudinal natural history data in understanding and validating clinical trial outcomes for Barth.

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            Lead Product(s): Cannabidiol

            Therapeutic Area: Genetic Disease Product Name: Zygel

            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 October 15, 2020

            Details:

            New data demonstrate that in patients diagnosed with FXS with a fully methylated FMR1 gene significantly more patients who received Zygel achieved a clinically meaningful improvement in their behavioral symptoms compared to patients who received placebo.

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            Lead Product(s): ARO-AAT

            Therapeutic Area: Genetic Disease Product Name: ARO-AAT

            Highest Development Status: Phase II/ Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Takeda Pharmaceutical

            Deal Size: $1,040.0 million Upfront Cash: $300.0 million

            Deal Type: Licensing Agreement October 08, 2020

            Details:

            Takeda and Arrowhead will co-develop ARO-AAT which, if approved, will be co-commercialized in the U.S under a 50/50 profit-sharing structure. Outside the U.S., Takeda will lead the global marketing strategy and receive an exclusive license to commercialize ARO-AAT.

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            Lead Product(s): Risdiplam

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Royalty Pharma

            Deal Size: $650.0 million Upfront Cash: $650.0 million

            Deal Type: Agreement July 20, 2020

            Details:

            Risdiplam, to be marketed by Roche, is an investigational, orally administered survival motor neuron-2 (SMN2) splicing modifier for the treatment of spinal muscular atrophy (SMA).

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            Lead Product(s): Cannabidiol

            Therapeutic Area: Genetic Disease Product Name: Zygel

            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 June 30, 2020

            Details:

            Zygel did not achieve statistical significance versus placebo in the primary endpoint of improvement in the Social Avoidance subscale of the Aberrant Behavior Checklist – Community FXS (ABC-CFXS).

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            Lead Product(s): Sirolimus

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Palvella Therapeutics

            Deal Size: $3.0 milion Upfront Cash: $2.0 milion

            Deal Type: Agreement May 28, 2020

            Details:

            The milestone payment comes as a result of a development funding and royalties agreement to advance their lead product candidate PTX-022 (QTORIN™ 3.9% rapamycin anhydrous gel) for the treatment of pachyonychia congenita.

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            Lead Product(s): Livoletide

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 06, 2020

            Details:

            Livoletide did not achieve statistically significant improvement in primary endpoint of change in hyperphagia and food-related behaviors relative to placebo.

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            Lead Product(s): ZYN002

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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): Elamipretide

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            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): Cannabidiol

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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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