Drug Development

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The prevalence of serious inflammatory safety issues such as cytokine release syndrome and immune effector cell–associated neurotoxicity syndrome limits the reach of these transformative cancer therapies.
After years stuck in the “doldrums,” the biopharma sector is in a “very good place” heading into the new year, analysts told BioSpace, with both rare and chronic diseases headlining investor and R&D interest as JPM26 kicks off.
Recent breakthroughs and three decades of progress in treating Huntington’s disease
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Ocuphire Pharma’s oral diabetic retinopathy drug missed the primary endpoint of an improvement in patient scores on the diabetic retinopathy severity scale.
Phase I/II data showed AC Immune’s anti-amyloid-beta vaccine candidate could safely elicit an antibody response in patients with Alzheimer’s disease. Now, it will expand to Down Syndrome patients.
Following the death of a patient, Magenta Therapeutics placed a hold on Phase I/II dose-escalation trial of its acute myeloid leukemia therapy.
Small Pharma announced positive top-line results Wednesday from a Phase IIa study evaluating a short-duration psychedelic for major depressive disorder.
Three venture capitalists debuted Dimension I Wednesday, a new fund with $350 million to fuel modern biotech.
Merck posted a rare defeat Wednesday for its blockbuster Keytruda (pembrolizumab) in prostate cancer while simultaneously announcing a victory in biliary tract cancer.
Cassava Sciences reported Phase II data from its Alzheimer’s candidate simufilam Tuesday. The company’s stock dropped 17% following the reveal.
Results from a study published in PNAS demonstrated that delivering therapeutics intranasally was sufficient to enhance functional recovery in rat models of ischemic stroke.
Research hasn’t shown that removing amyloid plaques alone improves function in Alzheimer’s patients, said Sharon L. Rogers, CEO of AmyriAD.
Axcella Therapeutics’ Long COVID fatigue therapeutic is moving forward as the company received guidance from the MHRA and filed an IND with the FDA seeking a Phase IIb/III trial.