Genetic Disease | Phase II/ Phase III Developments

Sanofi

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FRANCE France

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Positive topline results demonstrated by olipudase alfa, first and only investigational therapy in late-stage development for acid sphingomy...

Lead Product(s): Olipudase alfa

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: Phase II/ Phase III Product Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable January 30, 2020

Details:

Olipudase alfa is the first and only investigational enzyme replacement therapy in late-stage development for the treatment of ASMD.

Zynerba Pharmaceuticals

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United States

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Zynerba Pharmaceuticals Announces Top Line Results from Pivotal CONNECT-FX Trial of Zygel™ (CBD Gel) in Fragile X Syndrome

Lead Product(s): Cannabidiol

Therapeutic Area: Genetic Disease Product Name: Zygel

Highest Development Status: Phase II/ Phase III Product Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable June 30, 2020

Details:

Zygel did not achieve statistical significance versus placebo in the primary endpoint of improvement in the Social Avoidance subscale of the Aberrant Behavior Checklist – Community FXS (ABC-CFXS).

The Lundquist Institute

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Unique Clinical Trial Investigates a New Treatment Approach for Fatal ‘Childhood Alzheimer’s’ and Continues Despite COVID-19 Pandemic

Lead Product(s): Anakinra

Therapeutic Area: Genetic Disease Product Name: Kineret

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

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable June 24, 2020

Details:

The trial, which began in May, involves 20 children with Sanfilippo syndrome, who will be given anakinra, which blocks the activity of interleukin-1 (IL-1), a protein in the body that causes joint damage, thereby reducing inflammation.

ProQR Therapeutics

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Netherlands

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ProQR Announces Virtual Presentation of Phase 1/2 Sepofarsen Data Through the Association for Research in Vision and Ophthalmology (ARVO)

Lead Product(s): Sepofarsen

Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable June 08, 2020

Details:

The presentation will highlight results of a phase 1b/2 trial of intravitreal (IVT) sepofarsen (QR-110) antisense oligonucleotide in Leber congenital amaurosis 10 (LCA10) due to p.Cys998X mutation in the CEP290 gene.

Lysogene

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FRANCE France

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Lysogene Announces Important Updates on AAVance Phase 2/3 Clinical Trial with LYS-SAF302 in MPS IIIA

Lead Product(s): LYS-SAF302

Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable June 05, 2020

Details:

Following discussions with the U.S. FDA, a clinical hold was issued for the clinical trial AAVance, a global Phase 2/3 clinical trial of LYS-SAF302, a gene therapy for the treatment of Mucopolysaccharidosis Type IIIA (MPS IIIA, also known as Sanfilippo syndrome type A).

Orphazyme

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DENMARK Denmark

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Orphazyme Initiates Rolling Submission Of New Drug Application For Arimoclomol With FDA in Niemann-Pick Disease Type C

Lead Product(s): Arimoclomol

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: Phase II/ Phase III Product Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable June 01, 2020

Details:

Orphazyme has initiated the submission of its New Drug Application (NDA) for a rolling review by the US Food and Drug Administration (FDA) for arimoclomol, based on the promising results reported from clinical trials. Trial conducted in collaboration with CytRx Corporation.

Ligand Pharmaceuticals

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United States

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Ligand Earns $3 Million Milestone Payment from Palvella Therapeutics

Lead Product(s): Sirolimus

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: Phase II/ Phase III Product Type: Small molecule

Partner/Sponsor/Collaborator: Palvella Therapeutics

Deal Size: $3.0 milion Upfront Cash: $2.0 milion

Deal Type: Agreement May 28, 2020

Details:

The milestone payment comes as a result of a development funding and royalties agreement to advance their lead product candidate PTX-022 (QTORIN™ 3.9% rapamycin anhydrous gel) for the treatment of pachyonychia congenita.

Orchard Therapeutics

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United Kingdom

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Orchard Therapeutics Unveils New Strategic Plan

Lead Product(s): OTL-101

Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable May 07, 2020

Details:

Orchard Therapeutics to establish OTL-200, OTL-103, OTL-203 and OTL-201 programs as top near-term priorities and reduce investment in OTL-101and OTL-300.

Millendo Therapeutics

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United States

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Millendo Therapeutics Announces Topline Results for Pivotal Phase 2b Study of Livoletide

Lead Product(s): Livoletide

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: Phase II/ Phase III Product Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable April 06, 2020

Details:

Livoletide did not achieve statistically significant improvement in primary endpoint of change in hyperphagia and food-related behaviors relative to placebo.

Zynerba Pharmaceuticals

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United States

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Zynerba Pharmaceuticals Announces the Completion of Enrollment in the Pivotal CONNECT-FX Trial of Zygel™ in Fragile X Syndrome

Lead Product(s): ZYN002

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: Phase II/ Phase III Product Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable February 26, 2020

Details:

The trial is evaluating the efficacy and safety of Zygel™ CBD Gel as a treatment for behavioral symptoms of Fragile X syndrome (FXS) in 210 children ages three through 17 with full mutation FXS.

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Find Drugs for Genetic Disease in Phase II/ Phase III Clinical Development

Phase II/ Phase III

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Companies

Sanofi

Positive topline results demonstrated by olipudase alfa, first and only investigational therapy in late-stage development for acid sphingomy...

Zynerba Pharmaceuticals

Zynerba Pharmaceuticals Announces Top Line Results from Pivotal CONNECT-FX Trial of Zygel™ (CBD Gel) in Fragile X Syndrome

The Lundquist Institute

Unique Clinical Trial Investigates a New Treatment Approach for Fatal ‘Childhood Alzheimer’s’ and Continues Despite COVID-19 Pandemic

ProQR Therapeutics

ProQR Announces Virtual Presentation of Phase 1/2 Sepofarsen Data Through the Association for Research in Vision and Ophthalmology (ARVO)

Lysogene

Lysogene Announces Important Updates on AAVance Phase 2/3 Clinical Trial with LYS-SAF302 in MPS IIIA

Orphazyme

Orphazyme Initiates Rolling Submission Of New Drug Application For Arimoclomol With FDA in Niemann-Pick Disease Type C

Ligand Pharmaceuticals

Ligand Earns $3 Million Milestone Payment from Palvella Therapeutics

Orchard Therapeutics

Orchard Therapeutics Unveils New Strategic Plan

Millendo Therapeutics

Millendo Therapeutics Announces Topline Results for Pivotal Phase 2b Study of Livoletide

Zynerba Pharmaceuticals

Zynerba Pharmaceuticals Announces the Completion of Enrollment in the Pivotal CONNECT-FX Trial of Zygel™ in Fragile X Syndrome