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Find Clinical Drug Pipelines for Rare Diseases and Disorders

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            Lead Product(s): INO-3107

            Therapeutic Area: Rare Diseases and Disorders Product Name: INO-3107

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 23, 2020

            Details:

            The open-label, multicenter INO-3107 Phase 1/2 trial will evaluate the efficacy, safety, tolerability, and immunogenicity of INO-3107 in subjects who have required at least two surgical interventions per year for the past three years for the removal of associated papilloma.

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            Lead Product(s): Tesofensine,Metoprolol Tartrate

            Therapeutic Area: Rare Diseases and Disorders Product Name: Tesomet

            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 November 23, 2020

            Details:

            Tesomet was well-tolerated in hypothalamic obesity patients throughout the duration of the 48-week trial, with no clinically meaningful differences in heart rate or blood pressure observed. All patients who entered the OLE study completed it.

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            Lead Product(s): Exosome-based therapeutics

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Discovery Product Type: Undisclosed

            Partner/Sponsor/Collaborator: Evox Therapeutics

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration November 17, 2020

            Details:

            The collaboration will run over three years and will capitalise on the world-class exosome research environment in Oxford, leveraging exosome therapeutics expertise and resources from both Evox and the University to drive innovation within rare disease drug development.

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            Lead Product(s): Enzyme replacement therapy

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Undisclosed Product Type: Large molecule

            Partner/Sponsor/Collaborator: Chiesi Group

            Deal Size: $150.0 million Upfront Cash: $1.2 million

            Deal Type: Licensing Agreement November 03, 2020

            Details:

            XOMA gains rights to future milestone and royalty revenues associated with the advancement of the enzymes being investigated as potential treatments for four different lysosomal storage disorders (LSD) under Bioasis’ strategic alliance with Chiesi Group.

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            Lead Product(s): Mepolizumab

            Therapeutic Area: Rare Diseases and Disorders Product Name: Nucala

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 29, 2020

            Details:

            Submissions based on positive data from pivotal studies in hypereosinophilic syndrome, chronic rhinosinusitis with nasal polyps and eosinophilic granulomatosis with polyangiitis.

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            Lead Product(s): SNF472

            Therapeutic Area: Rare Diseases and Disorders Product Name: SNF472

            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 22, 2020

            Details:

            Phase 2b CaLIPSO study shows that SNF472 consistently slowed progression of coronary artery calcium (CAC) across key subgroups of patients on hemodialysis participating in the study.

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            Lead Product(s): TARA-002

            Therapeutic Area: Rare Diseases and Disorders Product Name: TARA-002

            Highest Development Status: Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Cowen

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Public Offering September 21, 2020

            Details:

            Protara intends to use the net proceeds from the Offerings primarily for development activities associated with TARA-002 in non-muscle invasive bladder cancer, lymphatic malformations and potential exploration of additional indications.

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            Lead Product(s): RZL 012

            Therapeutic Area: Rare Diseases and Disorders Product Name: RZL 012

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Fosun Pharmaceutical

            Deal Size: $74.0 million Upfront Cash: $27.0 million

            Deal Type: Licensing Agreement September 17, 2020

            Details:

            RZL-012 is an injectable drug product indicated for aesthetic treatment of adults having moderate to significant submental fullness. Under the terms of the agreement, Raziel Therapeutics will receive potential payments between now and marketing approval in the Territory.

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            Lead Product(s): Clemizole hydrochloride

            Therapeutic Area: Rare Diseases and Disorders Product Name: EPX-100

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: GreenLight Clinical

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 17, 2020

            Details:

            This Phase 2 study will confirm the efficacy and safety of EPX-100 with children suffering from Dravet Syndrome. The Company is now recruiting patients for this clinical study, collaborated with GreenLight Clinical.

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            Lead Product(s): TARA-002

            Therapeutic Area: Rare Diseases and Disorders Product Name: TARA-002

            Highest Development Status: Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 08, 2020

            Details:

            FDA Confirmed Initial Comparability Between TARA-002 and OK-432; Final GMP Comparability Expected to be Completed in Mid-2021.