Musculoskeletal | Phase II Developments | UNITED STATES

Fulcrum Therapeutics, Inc

Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

United States

Post An Enquiry

Virtual Booth

Duphat

Participation Not Confirmed

Update your events

Fulcrum Therapeutics Receives Orphan Drug Designation for Losmapimod in Facioscapulohumeral Muscular Dystrophy (FSHD) and Announces Expanded...

Lead Product(s): Losmapimod

Therapeutic Area: Musculoskeletal

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 January 29, 2020

Details:

The Phase 1 clinical trial of losmapimod in patients with FSHD, indicated that losmapimod was generally well tolerated and achieved dose-dependent concentrations in plasma and muscle.

Ultragenyx Pharmaceutical

Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

United States

Post An Enquiry

Virtual Booth

Duphat

Participation Not Confirmed

Update your events

Ultragenyx and Kyowa Kirin Announce Submission of Supplemental Biologics License Application to U.S. FDA for CrysvitaÂ® (burosumab) for Tu...

Lead Product(s): Burosumab

Therapeutic Area: Musculoskeletal

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 January 14, 2020

Details:

The sBLA package includes data from two single-arm Phase 2 studies including 14 adult patients conducted by Ultragenyx in the U.S. and 13 adult patients conducted by Kyowa Kirin in Japan.