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    Find Drugs for Rare Diseases and Disorders in Phase I Clinical Development in UNITED STATES

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              Inovio Pharmaceuticals

              • Development Update

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              UNITED STATES United States

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              AAPS 2020 PHARMSCI 360

              Participation Not Confirmed

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              Inovio Publishes Patient Benefit Results From A Pilot Clinical Study

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              Lead Product(s): INO-3106

              Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

              Highest Development Status: Phase I Product Type: Vaccine

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable February 03, 2020

              Details:

              Study results demonstrated that INO-3106 generated immunogenicity and engagement and expansion of an HPV 6-specific cellular response, including cytotoxic T cells.

              Epygenix Therapeutics

              • Development Update

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              UNITED STATES United States

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              AAPS 2020 PHARMSCI 360

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              Epygenix Therapeutics Successfully Completes EPX-100 Phase I, Placebo-Controlled, Double-Blind, 2-Period Study and Ready to Phase 2 Studies

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              Lead Product(s): Clemizole hydrochloride

              Therapeutic Area: Rare Diseases and Disorders 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 January 27, 2020

              Details:

              This Phase 1 study successfully confirmed that EPX-100 is safe, well-tolerated, and does not show any clinically significant abnormalities among equal numbers of male and female subjects.

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