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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:

The study will explore the safety, tolerability, and potential signs of efficacy of BI-1808 as a single agent and in combination with KEYTRUDA® in patients with ovarian cancer, non-small cell lung cancer and cutaneous T cell lymphoma.

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): Fitusiran

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.

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.

Ipsen

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Ipsen Demonstrates Continued Commitment to Rare Diseases with Eight Abstracts Accepted at ENDO 2020

Lead Product(s): Palovarotene

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 08, 2020

Details:

The data include a first-of-its kind study highlighting one-year data on the natural progression of FOP and the impact of heterotopic ossification (HO) on patients’ physical functioning over time.

Pharnext

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Pharnext Receives Promising Innovative Medicine Designation for PXT3003

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 March 18, 2020

Details:

All existing data indicate that PXT3003 is a safe and well tolerated drug combination.

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Companies

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

Pharnext

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

Ipsen

Ipsen Demonstrates Continued Commitment to Rare Diseases with Eight Abstracts Accepted at ENDO 2020

Pharnext

Pharnext Receives Promising Innovative Medicine Designation for PXT3003