Sanofi's experimental oral drug venglustat demonstrated significant neurological benefits for patients with Type 3 Gaucher disease in a late-stage trial, but failed to meet its primary endpoint in a separate study for Fabry disease, casting uncertainty on its regulatory path.
New long-term results from the pivotal EMBARK trial indicate ELEVIDYS, Sarepta's gene therapy, continues to alter the disease trajectory for boys with Duchenne muscular dystrophy, with no new safety concerns emerging over three years.
After the COVID-19 windfall subsided, Pfizer CEO Albert Bourla is steering the pharmaceutical giant into a high-stakes, $23 billion pivot to cancer research. Facing a looming patent cliff and plummeting vaccine revenue, Bourla is betting on scientific innovation to secure the company's future—and redefine cancer treatment.
Eli Lilly commits $85 million upfront and over $1.8 billion in potential milestones to partner with Repertoire Immune Medicines, aiming to develop a new class of 'tolerizing' therapies that reset the immune system without broad suppression.
