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Find Drugs for Genetic Disease in Phase II Clinical Development

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            Lead Product(s): ATL1102

            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 May 21, 2020

            Details:

            Primary endpoint met with confirmation of drug’s safety and tolerability. Strong effects on secondary endpoints on activity markers and disease progression along with improvement or stabilisation across different measures of motor function & strength were noted.

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            Lead Product(s): IMR-687

            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 May 21, 2020

            Details:

            Imara will present interim data from the ongoing Phase 2a study of IMR-687 in patients with sickle cell disease at the 25th Annual European Hematology Association (EHA) Congress. Imara completed enrollment in the IMR-687 Phase 2a clinical trial in sickle cell patients.

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            Lead Product(s): Lonafarnib

            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 May 19, 2020

            Details:

            The Zokinvy NDA is backed by the data from a study published in Journal of the American Medical Association (JAMA) which demonstrated a survival benefit with an 88% reduction in the risk of mortality in patients with Progeria treated with lonafarnib monotherapy.

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            Lead Product(s): Isotretinoin

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: BioPharmX

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Merger May 18, 2020

            Details:

            The combined company will have a robust pipeline including Timber's two Phase 2b programs and a preclinical program targeting multiple niche orphan indications with no approved treatments as well as BioPharmX's two Phase 3-ready topical minocycline programs.

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            Lead Product(s): Odiparcil

            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 May 18, 2020

            Details:

            The results show that odiparcil was associated with decreased glycosaminoglycan (GAG) accumulation as well as increased GAG excretion, and highlight its distribution in MPS VI disease-relevant tissues and organs.

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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 May 15, 2020

            Details:

            Seelos has been granted Rare Pediatric Disease Designation (RPDD) for SLS-005 in Sanfilippo syndrome from the U.S. Food and Drug Administration (FDA).

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            Lead Product(s): RZ358

            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 May 14, 2020

            Details:

            While enrollment in the recently initiated RZ358-606 Phase 2b trial in congenital hyperinsulinism patients has been paused due to COVID-19, the Company is pursuing a number of strategies across several countries to mitigate the disruption of the trial caused by COVID-19.

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            Lead Product(s): Mitapivat

            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 May 14, 2020

            Details:

            Mitapivat and Ivosidenib clinical data will be presented at the European Hematology Association (EHA) Annual Congress. Data Suppots ustility of both the drugs in treating Thalassemia.

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            Lead Product(s): CAP-1002

            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 May 13, 2020

            Details:

            The data showed improvements in upper limb, cardiac and respiratory function. The 12-month data from HOPE-2 showed statistically meaningful improvements in the PUL 2.0 in CAP-1002 treated patients with a mean change of 2.4 points over placebo patients.

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            Lead Product(s): Setmelanotide

            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 May 13, 2020

            Details:

            FDA has accepted the company's New Drug Application (NDA) for setmelanotide, an investigational, melanocortin-4 receptor (MC4R) agonist, for the treatment of pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity.

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