Sample Alerts - inThought Research

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…

Analysis

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. …

Analysis

BioMarin releases Roctavian 3-year data | FDA PDUFA date set for March 31st, 2023 with possible extension

HIGH IMPACT - BioMarin released 3-year data from its ongoing global phase 3 GENEr8-1 study for Roctavian in adults with severe hemophilia A. Mean/median FVIII activity (chromogenic) at year 3 was 18.8/8.4% (n=132). The 3 year mean FVIII…

Analysis

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…

Analysis

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…

Analysis

FDA Approved Hemgenix (Etranadez) - the first approved hemophilia B gene therapy

HIGH IMPACT - CSL/uniQure's Hemgenix (etranacogene dezaparvovec, Etranadez) was approved by the FDA for hemophilia B. Key Takeaways Hemgenix was approved for adults with hemophilia B who currently use FIX prophylaxis, have current or…

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