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            XENLETA (lefamulin) is first-in-class semi-synthetic pleuromutilin antibiotic for systemic administration in humans discovered and developed by the Nabriva Therapeutics team. It is designed to inhibit the synthesis of bacterial protein, which is required for bacteria to grow.

            Lead Product(s): Lefamulin Acetate

            Therapeutic Area: Genetic Disease Product Name: Xenleta

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 28, 2022

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            Xenleta (lefamulin) is a novel, first-in-class, IV and oral pleuromutilin antimicrobial agent that has been demonstrated to be highly potent against S. aureus, including MRSA and strains obtained from patients with CF.

            Lead Product(s): Lefamulin Acetate

            Therapeutic Area: Genetic Disease Product Name: Xenleta

            Highest Development Status: Phase I Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 11, 2022

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            XENLETA has the potential to provide a well-tolerated oral and intravenous anti-MRSA treatment option with a novel mechanism for this difficult to treat patient population. We look forward to sharing the results of this study with the medical community.

            Lead Product(s): Lefamulin Acetate

            Therapeutic Area: Genetic Disease Product Name: Xenleta

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 11, 2022

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            PRX004 is a phase 2 ready anti-amyloid immunotherapy designed to deplete the amyloid deposits that are associated with the disease pathology of ATTR amyloidosis.

            Lead Product(s): PRX004

            Therapeutic Area: Genetic Disease Product Name: PRX004

            Highest Development Status: Phase I Product Type: Large molecule

            Partner/Sponsor/Collaborator: Novo Nordisk

            Deal Size: $1,200.0 million Upfront Cash: $100.0 million

            Deal Type: Acquisition July 12, 2021

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            In this Phase 1 study, PRX004 was found to be generally safe and well tolerated across all dose levels. Positive clinical findings suggest PRX004’s depleter mechanism of action can result in benefits in both neuropathy and cardiac function.

            Lead Product(s): PRX004

            Therapeutic Area: Genetic Disease Product Name: PRX004

            Highest Development Status: Phase I Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 18, 2021

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            In the first report of clinical results with this depleter mechanism of action, PRX004 showed favorable results as demonstrated by slowing of neuropathy progression for all 7 evaluable patients at 9 months.

            Lead Product(s): PRX004

            Therapeutic Area: Genetic Disease Product Name: PRX004

            Highest Development Status: Phase I Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable December 09, 2020

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            Inzomelid is a potent, selective, orally-available, brain-penetrant NLRP3 inflammasome inhibitor intended for treatment of debilitating inflammatory diseases.

            Lead Product(s): Inzomelid

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 14, 2020

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            The Phase I study demonstrated Inzomelid has an excellent safety, tolerability and pharmacokinetic profile in healthy subjects, with dose-dependent target engagement.

            Lead Product(s): Inzomelid

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 26, 2020

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