Musculoskeletal | Phase III Developments

Pfizer Inc

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United States

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Pfizer to Host Free Webinar on Pfizer’s Advancement to Phase III Gene Therapy Clinical Trial

Lead Product(s): PF-06939926

Therapeutic Area: Musculoskeletal

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 March 10, 2020

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First ever Phase III gene therapy clinical trial for Duchenne muscular dystrophy.

Can-Fite BioPharma

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ISRAEL Israel

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Can-Fite Updates on Clinical Milestone for its Phase III Rheumatoid Arthritis Study with Piclidenoson

Lead Product(s): Piclidenoson

Therapeutic Area: Musculoskeletal

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 05, 2020

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Can Fite is exploring the possibility to collaborate with leading virology labs to explore the anti-viral effect of its drugs against the Coronavirus based on the known anti-viral effects.

Ra Pharmaceuticals

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United States

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Ra Pharmaceuticals Announces Publication of Zilucoplan Phase 2 gMG Trial Results in JAMA Neurology

Lead Product(s): Zilucoplan

Therapeutic Area: Musculoskeletal

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 February 18, 2020

Details:

the study demonstrated that zilucoplan resulted in rapid, clinically meaningful, statistically significant, and sustained improvements in the primary and key secondary endpoints.

Catabasis Pharmaceuticals

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United States

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Catabasis Pharmaceuticals Announces Pricing of $23 Million Underwritten Public Offering

Lead Product(s): Edasalonexent

Therapeutic Area: Musculoskeletal

Highest Development Status: Phase III Product Type: Small molecule

Partner/Sponsor/Collaborator: Oppenheimer & Co.

Deal Size: $23.0 million Upfront Cash: Undisclosed

Deal Type: IPO January 30, 2020

Details:

Catabasis intends to use the net proceeds from the offering for clinical trial and other research and development activities; initial commercialization preparations; and for working capital.

Ipsen

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FRANCE France

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Ipsen’s palovarotene clinical program in fibrodysplasia ossificans progressiva reaches prespecified interim analysis futility criteria

Lead Product(s): Palovarotene

Therapeutic Area: Musculoskeletal

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 January 24, 2020

Details:

Ipsen to conduct further assessment of the complete data set and work with the regulatory authorities, based on encouraging therapeutic activity signals observed in preliminary post-hoc analyses.

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Pfizer Inc

Pfizer to Host Free Webinar on Pfizer’s Advancement to Phase III Gene Therapy Clinical Trial

Can-Fite BioPharma

Can-Fite Updates on Clinical Milestone for its Phase III Rheumatoid Arthritis Study with Piclidenoson

Ra Pharmaceuticals

Ra Pharmaceuticals Announces Publication of Zilucoplan Phase 2 gMG Trial Results in JAMA Neurology

Catabasis Pharmaceuticals

Catabasis Pharmaceuticals Announces Pricing of $23 Million Underwritten Public Offering

Ipsen

Ipsen’s palovarotene clinical program in fibrodysplasia ossificans progressiva reaches prespecified interim analysis futility criteria