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Find Clinical Drug Pipelines for Rare Diseases and Disorders

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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 Pharma

            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.

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            Lead Product(s): Melphalan Flufenamide,Dexamethasone

            Therapeutic Area: Rare Diseases and Disorders Product Name: Melflufen

            Highest Development Status: Phase I/ Phase II Product Type: Peptide

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 07, 2020

            Details:

            This open-label, phase 1/2 study of melflufen and dexamethasone for patients with AL amyloidosis, following at least one prior line of therapy, is the first study to explore the effect of melflufen outside of multiple myeloma.

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            Lead Product(s): CERC-006

            Therapeutic Area: Rare Diseases and Disorders Product Name: CERC-006

            Highest Development Status: Preclinical Product Type: Small molecule

            Partner/Sponsor/Collaborator: Calidi Biotherapeutics

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 03, 2020

            Details:

            U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to CERC-006, a dual inhibitor of mTOR complexes 1 and 2 for the treatment of lymphatic malformations (LM).

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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: Vaccine

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 29, 2020

            Details:

            The open-label, multicenter INO-3107 Phase 1/2 trial is currently open to enrollment to recruit 63 subjects in the U.S. and will evaluate the efficacy, safety, tolerability, and immunogenicity of INO-3107 in subjects with HPV 6 and/or 11-associated RRP.

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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 July 28, 2020

            Details:

            TARA-002 is an investigational cell-based therapy based on the broad immunopotentiator OK-432, which is approved in Japan and Taiwan for the treatment of LMs. LMs are rare, typically congenital, malformations of the lymphatic vasculature.

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            Lead Product(s): RBCEV-based gene therapy

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Discovery Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Carmine Therapeutics

            Deal Size: $900.0 million Upfront Cash: Undisclosed

            Deal Type: Collaboration June 30, 2020

            Details:

            The collaboration will leverage Carmine’s REGENT technology, which is based on red blood cell extracellular vesicles, allowing for repeat dosing, enhanced bio-distribution and larger transgene payload capacity compared to adeno-associated virus-based gene therapy.

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            Lead Product(s): SNF472

            Therapeutic Area: Rare Diseases and Disorders 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 February 13, 2020

            Details:

            CALCIPHYX, is designed to assess the effect of SNF472 when added to background care for the treatment of calciphylaxis patients with end stage kidney disease being treated with hemodialysis.

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