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            Lead Product(s): Pridopidine

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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 October 27, 2020

            Details:

            Pridopidine Outcome On Function in Huntington’s Disease (PROOF-HD) is a randomized, double-blind, placebo-controlled, phase 3 study evaluating the efficacy and safety of pridopidine 45mg bid in patients with early stage HD.

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            Lead Product(s): Pegunigalsidase alfa

            Therapeutic Area: Genetic Disease Product Name: PRX-102

            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 October 02, 2020

            Details:

            Patients participating in the EAP will receive infusions of pegunigalsidase alfa every two weeks at 1mg/kg body weight. Information related to adverse events and other limited data will be collected from participants.

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            Lead Product(s): Pegunigalsidase alfa

            Therapeutic Area: Genetic Disease Product Name: PRX-102

            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 August 24, 2020

            Details:

            The BRIGHT study is a phase III, open label, switch over study to assess the safety, efficacy and pharmacokinetics of pegunigalsidase alfa 2 mg/kg administered by intravenous infusion in patients with Fabry disease currently treated with enzyme replacement therapy (ERT).

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            Lead Product(s): Pegunigalsidase alfa

            Therapeutic Area: Genetic Disease Product Name: PRX-102

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Chiesi Group

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 11, 2020

            Details:

            U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) and granted Priority Review designation for pegunigalsidase alfa for the proposed treatment of adult patients with Fabry disease.

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            Lead Product(s): Pridopidine

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Morningside Venture

            Deal Size: $62.5 million Upfront Cash: Undisclosed

            Deal Type: Series A Financing June 03, 2020

            Details:

            The proceeds will fund two late-stage trials, which could lead to the registration of pridopidine for the treatment of HD and ALS. Pridopidine is a highly selective sigma-1 receptor (S1R) agonist.

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            Lead Product(s): Pegunigalsidase alfa

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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 28, 2020

            Details:

            The BLA has been filed for pegunigalsidase alfa for the proposed treatment of adult patients with Fabry disease via the FDA's Accelerated Approval pathway. Pegunigalsidase alfa a long-acting recombinant, PEGylated, cross-linked α-galactosidase-A product.

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            Lead Product(s): Pegunigalsidase alfa

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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): Pegunigalsidase alfa

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Psagot Investment House

            Deal Size: $43.7 million Upfront Cash: Undisclosed

            Deal Type: Financing March 12, 2020

            Details:

            Financing will advance the Company's clinical programs and commercialization of PRX-102 for the treatment of Fabry disease, as well as to further develop its early stage pipeline of therapeutics.

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            Lead Product(s): Pegunigalsidase alfa

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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:

            Phase I/II dose-ranging studies show high correlation between two Fabry disease biomarkers, supporting potential effectiveness of pegunigalsidase alfa in treating Fabry disease.