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Find Drugs for Genetic Disease in Phase II Clinical Development

A collaboration with SCORR Marketing

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            Lead Product(s): Isotretinoin

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Timber Pharmaceuticals

            Deal Size: $20.0 million Upfront Cash: Undisclosed

            Deal Type: Merger January 28, 2020

            Details:

            Under the terms of the merger agreement, a wholly-owned subsidiary of BioPharmX will be merged with and into Timber, with Timber surviving the merger as a wholly-owned subsidiary of BioPharmX.

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            Lead Product(s): Sonlicromanol

            Therapeutic Area: Genetic Disease

            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 January 27, 2020

            Details:

            The study’s primary objective is to evaluate the dose-effect of Sonlicromanol on the attention domain score of cognitive functioning, Results are expected in the second half of 2020.

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            Lead Product(s): Trehalose API

            Therapeutic Area: Genetic Disease

            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 January 22, 2020

            Details:

            Seelos seeks scientific advice and protocol assistance for a European clinical study of SLS-005 in patients with Sanfillipo syndrome type A and B from EMA.

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            Lead Product(s): ELX-02

            Therapeutic Area: Genetic Disease

            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 January 14, 2020

            Details:

            Phase 2 cystic fibrosis program for ELX-02 expected to be fully enrolled in the first quarter of 2020 and topline results expected during the first half of 2020.

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            Lead Product(s): Setrusumab

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 14, 2020

            Details:

            Setrusumab demonstrated a dose dependent increase in both failure load and stiffness at the radius at 12 months, achieving statistical significance in the high dose cohort.

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            Lead Product(s): IPI-549,Nivolumab,Atezolizumab

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase II Product Type: Undisclosed

            Partner/Sponsor/Collaborator: BVF Partners L.P

            Deal Size: $71.5 million Upfront Cash: Undisclosed

            Deal Type: Financing January 09, 2020

            Details:

            The funds will be used for all ongoing IPI-549 clinical trials through key data readouts.

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            Lead Product(s): Tideglusib

            Therapeutic Area: Genetic Disease

            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 January 09, 2020

            Details:

            Trial to assess AMO-02 in treatment of congenital myotonic dystrophy to begin patient enrollment in early 2020.

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            Lead Product(s): MS1819,Pancreatic enzyme replacement therapy

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 09, 2020

            Details:

            Initial data from the first five patients indicate positive trends for all primary and secondary safety and efficacy endpoints in AzurRx’s Phase 2 Clinical Study for MS1819-SD with standard PERT.

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            Lead Product(s): AT-007

            Therapeutic Area: Genetic Disease

            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 January 08, 2020

            Details:

            AT-007 demonstrated a robust and sustained reduction in galactitol vs placebo; significant plasma galactitol reduction of ~50%.

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            Lead Product(s): PAT-1251

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: PharmAkea

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Partnership January 07, 2020

            Details:

            The two firms will combine & put in robust fibrosis and cancer services, including a recently inhaled galectin-3 modulator ph IIb for the potential treatment of idiopathic pulmonary fibrosis.

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