Drug Development
ITF, IntraBio and Orchard are among the companies that have won FDA nods in the past year for Duchenne muscular dystrophy, Niemann-Pick disease type C, metachromatic leukodystrophy and more.
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The past four years have brought disappointment for the Huntington’s community, but optimism is growing as companies including Prilenia and Wave Life Sciences eye paths to approval of therapies that could address the underlying cause of the disease.
A fatal, highly hereditary illness with no disease-modifying treatments, Huntington’s is long overdue for a therapeutic win. Here, BioSpace looks at five candidates that could change the trajectory for patients.
With Eisai and Biogen’s Leqembi and Eli Lilly’s Kisunla launching onto the market, the 2024 Clinical Trials of Alzheimer’s Disease conference focused on the role these drugs might play, as well as combination therapies and innovative new treatment options.
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Expanding volumes of data point to mechanisms beyond weight loss and blood sugar control that contribute to cardiovascular benefits in the world’s fastest-growing drug class.
With two earlier trials meeting their primary endpoints, Axsome claimed it has the data to support a filing for FDA approval in the second half of 2025.
Roche has once again returned to China to bolster its antibody-drug conjugate pipeline, this time striking a licensing deal with Innovent for $1 billion in biobucks.
Regulators squeezed in two final approvals before the calendar change with the UK approval of Merck’s Winrevair and the FDA’s greenlight for an injectable formulation of BMS’s cancer blockbuster.
In a highly anticipated readout for the kappa opioid receptor class in major depressive disorder, Neumora’s navacaprant failed to meet the primary and key secondary endpoint in the first of three identical Phase III studies.
Sangamo is on course to run out of money within months and has now lost access to up to $220 million in milestone payments from Pfizer.
The new combination, dubbed Alyftrek, is designed to improve on the Trikafta, a product that generated sales of $8.9 billion in 2023. It’s welcome good news for Vertex following last week’s subpar clinical results for its non-opioid analgesic.
The mega-blockbuster weight loss GLP-1 drug is now also approved to treat obstructive sleep apnea in adults with obesity, in combination with diet and exercise.
Following a 90% drop in share price due to an FDA rejection and a warning letter over Applied Therapeutics’ conduct during a clinical trial, the biotech’s CEO has stepped aside.
Already established as cornerstone therapies in diabetes and obesity, GLP-1 receptor agonists also show potential in several other indications, including cancer, addiction and neurodegenerative diseases.