Drug Development
Infigratinib topped “even the most optimistic expectations” for efficacy and safety in the late-stage PROPEL 3 study in achondroplasia, Truist Securities analysts said Thursday.
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Analysts, investors and scientists are eager for Biogen’s 2026 BIIB080 readout. Even if successful, executives warn that there are many more steps before the Alzheimer’s therapy could reach the market.
With a clutch of key data and planned regulatory applications this year from Avidity Biosciences, REGENXBIO and Capricor Therapeutics, CureDuchenne CSO Michael Kelly sees “momentum” in the Duchenne muscular dystrophy pipeline, as Sarepta’s Elevidys leaves the door open.
After advancing in lockstep through the pandemic, the fortunes of the biotechs have diverged as their use of COVID-19 windfalls has taken shape.
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The experimental drug, being developed to treat immunoglobulin A nephropathy, is IONIS-FB-L Rx, which hit the mark in a Phase II study showing a change in urinary protein after 29 weeks of treatment.
Sanofi announced positive data for its therapeutic, fitusiran, for treating patients with hemophilia A and B, as well as efanesoctocog alfa therapy for treating hemophilia A.
The study was assessing the investigational drug enoblituzumab as part of a combination therapy for head and neck cancer.
Scientists found that SARS-CoV-2 spike protein in the plasma could be a hallmark of long COVID, suggesting that the virus can linger in the body who suffer from prolonged symptoms.
Weeks after BridgeBio presented positive biomarker data in Canavan Disease, gene therapy company Myrtelle added its own data to the emerging research space.
Imago’s early failed experiments investigating the potential of LSD1 in sickle cell disease and thalassemia have turned out to be fortuitous for patients with myeloproliferative neoplasms.
Seagen recently presented positive pivotal Phase II data from its trial of Tukysa (tucatinib) in combination with trastuzumab in HER2-positive metastatic colorectal cancer.
Dynacure has ended its Unite-CNM Phase I/II trial on DYN101, an investigational therapy for the treatment of myotubular and centronuclear myopathies.
Ayala announced positive data for its desmoid tumor treatment, while Vertex and Dyne announced the FDA had lifted the hold on their clinical studies.
In a filing with the SEC, Abbvie’s decision to terminate came after the two firms jointly conducted a review of their next steps for the drug.