Entries by old admin

Roche’s crovalimab pivotal studies in PNH are positive; poised to be third C5 inhibitor to enter market

HIGH IMPACT – Roche announced positive results from its COMMODORE1 and 2 studies of crovalimab in PNH patients. COMMODORE2 (naïve study) met co-primary endpoint of transfusion avoidance and control of hemolysis (LDH). Crovalimab was non-inferior to eculizumab. COMMODORE1 (switch study) efficacy and safety data supported a favorable benefit-risk profile of crovalimab. Based on these results […]

NICE recommends reimbursement for Translarna

MODERATE IMPACT – The National Institute for Health and Care Excellence (NICE) has issued a Final Evaluation Document recommending Translarna (ataluren) for reimbursement and use across the National Health Service (NHS) in England and Wales. Translarna is licensed in multiple countries, including Great Britain, Northern Ireland, and the European Economic Area, for treating nonsense mutation […]

Biogen and Alcyone taking the next step in collaboration for SC delivery of ASO therapies

MODERATE IMPACT – Biogen and Alcyone entered into a license and collaboration agreement to develop Alcyone’s ThecaFlex DRx System, an implantable medical device intended for subcutaneous delivery of antisense oligonucleotide (ASO) therapies into the intrathecal space in patients with spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS). The ThecaFlex DRx System would allow routine […]

Madrigal’s resmetirom hits dual primary endpoints in phase 3 NASH study

HIGH IMPACT – Madrigal announced positive topline results from the pivotal Phase 3 MAESTRO-NASH trial of resmetirom (THR-β agonist) in non-cirrhotic NASH with liver fibrosis. MAESTRO-NASH achieved both liver histological endpoints and potentially clinically meaningful effects with 80 mg and 100 mg doses of the drug, relative to placebo. Study summary: 52-week phase 3 study in […]