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Find Drugs for Genetic Disease in Phase II Clinical Development

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            Lead Product(s): Maralixibat

            Therapeutic Area: Genetic Disease Product Name: LUM001

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Mirum Pharmaceutical

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Partnership October 15, 2020

            Details:

            Mirum will utilize EVERSANA’s commercialization platform to provide integrated nationwide distribution, specialty pharmacy, patient services and Hub support for maralixibat, if approved.

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            Lead Product(s): RZ358

            Therapeutic Area: Genetic Disease Product Name: RZ358

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Rezolute

            Deal Size: $1.9 million Upfront Cash: Undisclosed

            Deal Type: Agreement October 14, 2020

            Details:

            Rezolute’s recent private placement triggers $1.4 million payment to XOMA in connection with its recent private placement to continue advancing RZ358. Another $0.5 million milestone payment triggered in an unrelated phase 2 development by an unnamed partner.

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            Lead Product(s): VX-814

            Therapeutic Area: Genetic Disease Product Name: VX-814

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 14, 2020

            Details:

            Phase 2 study of VX-814 in patients with alpha-1 antitrypsin deficiency discontinued based upon safety and pharmacokinetic data. Phase 2 study of VX-864 continues to enroll and dose patients.