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Find Drugs for Genetic Disease in Phase III Clinical Development

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            Lead Product(s): Avalglucosidase alfa

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 10, 2020

            Details:

            In patients with late-onset Pompe disease (LOPD), 5.5 years of safety and exploratory efficacy results showed avalglucosidase alfa was well-tolerated and stabilized pulmonary and motor function.

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            Lead Product(s): Idebenone

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 20, 2020

            Details:

            The large Phase 3 SIDEROS study was designed to confirm the efficacy of idebenone in patients with respiratory function decline who are concurrently taking glucocorticoids.

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            Lead Product(s): Luspatercept

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 20, 2020

            Details:

            Data from multiple studies, including results in the outpatient administration setting and using machine learning technology, evaluating potential of CAR T liso-cel in relapsed and refractory large b-cell lymphoma.

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            Lead Product(s): Pegzilarginase

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 19, 2020

            Details:

            Aeglea BioTherapeutics will present 56 week analysis on patients who have been treated with pegzilarginase from the completed Phase 1/2 clinical trial and the ongoing Phase 2 open-label extension study at the 6th Congress of the European Academy of Neurology.

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            Lead Product(s): Tasimelteon

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 13, 2020

            Details:

            Following the completion of a Type A Meeting with the U.S. FDA on May 8, 2020, it has reached agreement with the FDA to resubmit its application for HETLIOZ® (tasimelteon) for the treatment of Smith-Magenis Syndrome (SMS).

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            Lead Product(s): Edasalonexent

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 12, 2020

            Details:

            The posters provide an overview of the baseline status of patients involved in PolarisDMD Ph 3 trial, findings from the MoveDMD Ph 2 trial and effect of oral edasalonexent in both the clinical trials.

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            Lead Product(s): Pegunigalsidase alfa

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 11, 2020

            Details:

            The BRIDGE study was a Phase III study evaluating the safety and efficacy of pegunigalsidase alfa, 1 mg/kg infused every two weeks, in up to 22 Fabry patients previously treated with agalsidase alfa.

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            Lead Product(s): Berotralstat

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 05, 2020

            Details:

            The allowed patent application covers crystalline salt forms of berotralstat active pharmaceutical ingredient (API).

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            Lead Product(s): Pegzilarginase

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: J.P. Morgan Securities

            Deal Size: $100.0 million Upfront Cash: Undisclosed

            Deal Type: Public Offering April 27, 2020

            Details:

            Aeglea intends to use the net proceeds to advance the clinical development of pegzilarginase through its Phase 3 PEACE trial and BLA submission and advance other clinical and Pre-clinical activities.

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            Lead Product(s): Givinostat

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 27, 2020

            Details:

            The Interim data from EPYDIS Phase 3 trial shows that the trial is NOT FUTILE and the Independent Data Monitoring Committee recommends trial continuation.

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