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Fortress Biotech and Cyprium Therapeutics Report the US FDA’s NDA Acceptance with Priority Review of CUTX-101 to Treat Menkes Disease
Shots: Fortress and its subsidiary, Cyprium, have reported the US FDA’s NDA acceptance & priority review of CUTX-101 (copper histidinate) to treat Menkes disease, with the decision expected on Jun 30, 2025 Submission was based on a trial of CUTX-101 vs untreated historical control that depicted significantly improved OS & an 80% reduction in…
January 7, 2025
Stryker to Acquire Inari Medical for ~$4.9B
Shots: Stryker has signed a definitive agreement to initiate a tender offer for acquiring all the issued & outstanding shares of Inari Medical for $80 per share in cash, making it a total value of ~$4.9B Upon completion, Stryker will acquire remaining shares in a second-step merger at the same price. The deal is…
January 7, 2025
Ultragenyx Reports the EC’s Approval of Evkeeza (evinacumab) to Treat 6-Months Old Children with Homozygous Familial Hypercholesterolemia (HoFH)
Shots: The EC has approved Evkeeza (an angiopoietin-like 3 inhibitor), as an adjunct to diet & other lipid-lowering therapies, to treat children (6mos. to 5yrs.) with HoFH Approval was based on model-based analysis that predicted similar or greater LDL-C reductions with Evkeeza (15mg/kg, Q4W) in children (6mos. to 5yrs.) at 24wks. Data from compassionate…
January 6, 2025
NMD Pharma’s NMD670 Secures the US FDA’s Orphan Drug Designation to Treat Charcot-Marie-Tooth Disease
Shots: The US FDA has granted ODD to NMD670 for treating Charcot-Marie-Tooth disease (CMT). It is small molecule that inhibits CIC-1 to enhances muscle responsiveness, improving neuromuscular transmission & restoring function NMD670 (PO, BID) is under P-IIa (SYNAPSE-CMT) evaluation for its efficacy, safety & tolerability in adults (n=80) with CMT1 or CMT2 subtype for over…
January 6, 2025
JCR Pharmaceuticals and Modalis Progress into the Next Phase of Joint Research Agreement to Develop Gene Therapy
Shots: JCR Pharmaceuticals & Modalis have reported successful validation of initial PoC to develop gene therapy targeting CNS disease and will advance to the next phase under a new joint agreement The new agreement aims at conducting preclinical studies for gene therapy using JCR's BBB-crossing J-Brain Cargo tech and for gene therapy payload using Modalis' CRISPR-GNDM…
January 6, 2025
Pharmanovia Collaborates with Radius Health to Commercialize Abaloparatide for Osteoporosis Treatment
Shots: Pharmanovia has signed an exclusive in-licensing agreement with Radius Health for abaloparatide (US brand name: Tymlos) to treat osteoporosis, expanding its osteoporosis franchise Pharmanovia will handle clinical, regulatory & commercial responsibilities across APAC region (China, Singapore, Thailand, Indonesia, Vietnam, Taiwan, Hong Kong, Malaysia, Macau & Philippines), while Radius will get an upfront, regulatory…
January 3, 2025
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