France

Sanofi

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NEJM Publishes Positive Final Results from Phase 1/2a Study of BIVV001 in People with Severe Hemophilia A

Lead Product(s): rFVIIIFc-VWF-XTEN

Therapeutic Area: Genetic Disease Product Name: BIVV001

Highest Development Status: Phase III Product Type: Large molecule

Partner/Sponsor/Collaborator: Swedish Orphan Biovitrum AB

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable September 10, 2020

Details:

BIVV001 is the first investigational factor VIII therapy independent of von Willebrand Factor and has the potential to transform replacement therapy for people with hemophilia A.

Sanofi

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Data presented at ISTH Virtual Congress highlights Sanofi’s commitment to advancing treatments for people with rare blood disorders

Lead Product(s): rFVIIIFc-VWF-XTEN,Recombinant Antihemophilic Factor

Therapeutic Area: Genetic Disease Product Name: BIVV001

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 June 29, 2020

Details:

Presentations include findings on hemophilia and acquired thrombotic thrombocytopenic purpura portfolio therapies and pipeline candidates.

Sanofi

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Sanofi Announces Long-Term Efficacy and Safety Data for Fitusiran from Interim Analysis of Phase 2 Extension Study in Hemophilia A and B

Lead Product(s): Givosiran

Therapeutic Area: Genetic Disease Product Name: ALN-AT3SC

Highest Development Status: 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 19, 2020

Details:

Long-term interim results from the Phase 2 open-label extension (OLE) study reinforce fitusiran’s potential to restore hemostatic balance and to lower annualized bleed rates (ABRs) over a period up to 57 months.

Sanofi

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Sanofi’s Investigational Enzyme Replacement Therapy Improves Critical Manifestations of Late-Onset Pompe Disease

Lead Product(s): Avalglucosidase alfa

Therapeutic Area: Genetic Disease Product Name: Undisclosed

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 June 16, 2020

Details:

Avalglucosidase alfa showed a 2.4-point improvement in percent-predicted forced vital capacity, an important measure of respiratory function in Pompe disease, compared to alglucosidase alfa (standard of care).

Sanofi

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Sanofi To Present Phase 3 Results of Avalglucosidase Alfa in Patients with Late-Onset Pompe Disease

Lead Product(s): Avalglucosidase alfa

Therapeutic Area: Genetic Disease Product Name: Undisclosed

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 June 08, 2020

Details:

Sanofi will host a virtual scientific session to present data from the Phase 3 COMET trial of investigational enzyme replacement therapy (ERT) avalglucosidase alfa in patients with late-onset Pompe disease (LOPD).

Sanofi

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Avalglucosidase alfa showed positive exploratory efficacy

Lead Product(s): Avalglucosidase alfa

Therapeutic Area: Genetic Disease Product Name: Undisclosed

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 10, 2020

Details:

In patients with late-onset Pompe disease (LOPD), 5.5 years of safety and exploratory efficacy results showed avalglucosidase alfa was well-tolerated and stabilized pulmonary and motor function.

Ipsen

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Ipsen To Present New Insights at ASBMR For Potential Treatment of Ultra-Rare Disease Fibrodysplasia Ossificans Progressiva, Including Global...

Lead Product(s): Palovarotene

Therapeutic Area: Genetic Disease Product Name: IPNG 60120

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 September 10, 2020

Details:

The presentation will highlight data from Ipsen’s MOVE trial, the first and only multicenter Phase III study in fibrodysplasia ossificans progressiva.

Ipsen

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Ipsen to Present Results from Move, the First Global Phase III Trial in Fibrodysplasia Ossificans Progressiva (fop), at Asbmr 2020

Lead Product(s): Palovarotene

Therapeutic Area: Genetic Disease Product Name: IPNG 60120

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 August 25, 2020

Details:

Post hoc analyses showed substantial reduction (62%) in mean annualized new heterotopic ossification volume in patients with FOP who were treated with oral investigational therapy palovarotene.

GenSight Biologics

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GenSight Biologics Obtains a €7m Non-Dilutive Loan and Renegotiates the Bond Agreement With Kreos Capital Extending Runway to Mid-2021

Lead Product(s): Lenadogene nolparvovec

Therapeutic Area: Genetic Disease Product Name: Lumevoq

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

Partner/Sponsor/Collaborator: Kreos Capital

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Agreement July 09, 2020

Details:

Together with anticipated revenues from the Temporary Authorization for Use of LUMEVOQ® in France, The company expect to be financed until at least mid-2021.

Pharnext

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Pharnext Provides Regulatory and Clinical Update on PXT3003 Phase III Study for the Treatment of Charcot-Marie-Tooth Type 1A

Lead Product(s): Baclofen,Naltrexone Hydrochloride,Sorbitol API

Therapeutic Area: Genetic Disease 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 June 10, 2020

Details:

PXT3003 is a novel drug candidate for the treatment of CMT1A and has been granted both Orphan Drug Designation and Fast Track Designation by the US Food and Drug Administration.

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Companies

Sanofi

NEJM Publishes Positive Final Results from Phase 1/2a Study of BIVV001 in People with Severe Hemophilia A

Sanofi

Data presented at ISTH Virtual Congress highlights Sanofi’s commitment to advancing treatments for people with rare blood disorders

Sanofi

Sanofi Announces Long-Term Efficacy and Safety Data for Fitusiran from Interim Analysis of Phase 2 Extension Study in Hemophilia A and B

Sanofi

Sanofi’s Investigational Enzyme Replacement Therapy Improves Critical Manifestations of Late-Onset Pompe Disease

Sanofi

Sanofi To Present Phase 3 Results of Avalglucosidase Alfa in Patients with Late-Onset Pompe Disease

Sanofi

Avalglucosidase alfa showed positive exploratory efficacy

Ipsen

Ipsen To Present New Insights at ASBMR For Potential Treatment of Ultra-Rare Disease Fibrodysplasia Ossificans Progressiva, Including Global...

Ipsen

Ipsen to Present Results from Move, the First Global Phase III Trial in Fibrodysplasia Ossificans Progressiva (fop), at Asbmr 2020

GenSight Biologics

GenSight Biologics Obtains a €7m Non-Dilutive Loan and Renegotiates the Bond Agreement With Kreos Capital Extending Runway to Mid-2021

Pharnext

Pharnext Provides Regulatory and Clinical Update on PXT3003 Phase III Study for the Treatment of Charcot-Marie-Tooth Type 1A