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Find Drugs for Genetic Disease in Phase III Clinical Development in UNITED STATES

A collaboration with SCORR Marketing

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            Lead Product(s): Setmelanotide

            Therapeutic Area: Genetic Disease

            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 March 18, 2020

            Details:

            Rhythm is currently evaluating setmelanotide’s ability to reduce hunger and affect weight loss in an ongoing pivotal Phase 3 trial in patients living with Alström and Bardet-Biedl syndromes.

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            Lead Product(s): Berotralstat

            Therapeutic Area: Genetic Disease

            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 March 16, 2020

            Details:

            150 mg of oral, once-daily berotralstat for prophylaxis of hereditary angioedema attacks reduced patients’ monthly use of standard of care on-demand medicine by 53.6 percent compared to placebo.

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            Lead Product(s): Elamipretide

            Therapeutic Area: Genetic Disease

            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 March 03, 2020

            Details:

            Stealth BioTherapeutics is eligible for a voucher that can be used to obtain priority review for a subsequent human drug application.

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            Lead Product(s): Ganaxolone

            Therapeutic Area: Genetic Disease

            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 25, 2020

            Details:

            The Marigold Study is a pivotal Phase 3 study evaluating oral ganaxolone in children and young adults with CDKL5 Deficiency Disorder (CDD).

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            Lead Product(s): Valoctocogene Roxaparvovec

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 20, 2020

            Details:

            The acceptance by the FDA marks the first marketing application accepted for a gene therapy product for any type of hemophilia in the United States.

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            Lead Product(s): Edasalonexent

            Therapeutic Area: Genetic Disease

            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 20, 2020

            Details:

            Edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy, is the company's lead program.

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            Lead Product(s): Vutrisiran

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 18, 2020

            Details:

            The study was designed to enroll 160 patients with hereditary ATTR (hATTR) amyloidosis with polyneuropathy across 68 sites in 22 countries.

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            Lead Product(s): Berotralstat

            Therapeutic Area: Genetic Disease

            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 18, 2020

            Details:

            In the NDA filing acceptance letter, the FDA stated that they are not currently planning to hold an advisory committee meeting to discuss the NDA.

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            Lead Product(s): Berotralstat

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Torii Pharmaceutical

            Deal Size: $42.0 million Upfront Cash: $22.0 million

            Deal Type: Agreement February 03, 2020

            Details:

            Torii Pharmaceutical will pay tiered royalties of up to 40 percent of Japanese net sales of berotralstat to BioCryst upon receipt of a reimbursement price approval NHI Japan.

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            Lead Product(s): Copper histidinate

            Therapeutic Area: Genetic Disease

            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 January 16, 2020

            Details:

            If Cyprium’s new drug application is approved, the company may be eligible to receive a priority review voucher.

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