Avrobio Inc., stung by variable outcomes in a phase II test of its investigational Fabry disease therapy, is quitting further enrollment in the program, one of the first from its gene therapy platform, Plato. The team's attention will shift instead to other clinical-stage lysosomal disorder programs amid "an increasingly challenging market and regulatory environment for Fabry disease," the company said.
Preliminary data from a phase IIIb study of Johnson & Johnson’s Ad26.COV2.S COVID-19 vaccine showed a homologous booster dose was 85% effective against hospitalization in participants from South Africa.
PARIS – Two months after successfully listing on Euronext Brussels and Euronext Amsterdam stock exchanges, raising nearly $100 million, Onward Medical NV reported completion of enrollment in its Up-LIFT study on its noninvasive electrical spinal cord stimulation, called Arc therapy.
Ikena Oncology Inc.’s plan to launch the first-in-human phase I study with IK-930, a transcriptional enhanced associate domain inhibitor targeting the Hippo pathway in cancer, brought new attention to the Hippo space. The FDA accepted Ikena’s IND application early last month. Now, work with other candidates is picking up steam at a handful of companies pursuing early stage prospects.
Schizophrenia remains one of the toughest mental health issues to treat, as well as an indication that has created substantial challenges for drug makers. The pipeline of potential new schizophrenia medicines is busy, with Cortellis showing that 10 new phase III schizophrenia trials that began recruiting patients in 2021 and 59 phase III trials ongoing at the end of the year.
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Bio-Thera, Cantargia, Hansa, Kolon, Lidds, Salarius, Saniona.
With the memory of Dec. 20’s stock-denting, top-line phase III fizzle by Aldeyra Therapeutics Inc. in dry eye disease (DED) still fresh, Palatin Technologies Inc. is launching a late-stage effort in the same indication. Aldeyra offered data from the Tranquility trial with reproxalap – a small-molecule, immune-modulating covalent inhibitor of reactive aldehyde species, known as RASP, formulated as an ophthalmic solution – that showed a miss on the primary endpoint of ocular redness.
PARIS – Bellaseno GmbH reported publication of first-in-human data for its 3D-printed biodegradable and highly porous Senella scaffold filled with autologous fat graft to correct a chest deformation in a 22-year-old patient with severe pectus excavatum. The article was published in the European Journal of Plastic Surgery.
Top-line results from Bridgebio Pharma Inc.’s ongoing phase III study of acoramidis are no holiday gift for the company. The clinical trial for treating symptomatic transthyretin amyloid cardiomyopathy missed its primary endpoint. The Palo Alto, Calif.-based company’s stock (NASDAQ:BBIO) plunged on the news as shares closed 72% lower Dec. 27 at $11.38 each.
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