Oncolytic Virus Shows High Response Rates in BCG-Unresponsive Bladder Cancer
WASHINGTON — Treatment with cretostimogene grenadenorepvec, an investigational intravesically delivered oncolytic immunotherapy, achieved high response rates in patients with high-risk non-muscle-invasive bladder cancer (NMIBC) unresponsive to Bacillus Calmette-Guérin (BCG), according to an interim analysis of the phase III BOND-003 trial. Among 66 patients evaluable for efficacy, 75.7% (95% CI 63-85) achieved a complete response (CR) […]
Noninvasive Therapy Shows Efficacy in Non-Muscle-Invasive Bladder Cancer
WASHINGTON — An investigational non-surgical treatment led to a “robust” complete response rate in patients with recurrent, low-grade non-muscle-invasive bladder cancer, the phase III ENVISION study showed. Patients treated with the UGN-102 (mitomycin) intravesical solution achieved a complete response rate of 79.2% (95% CI 73.5-84.1) at 3 months without having to undergo transurethral resection of […]
Novel Gout Drug Aces Mid-Stage Tests
SAN DIEGO — An investigational once-daily oral agent that inhibits urate transporter-1 (URAT1) as a means of forcing uric acid excretion showed good efficacy and safety in a pair of phase II trials reported here. In one of these studies, the new product, called AR882 for the time being, reduced serum urate by 40% to […]
Tyrosine Kinase Inhibitor Active in Heavily Treated Renal Cell Carcinoma
NASHVILLE, Tenn. — An investigational multitargeted drug produced objective responses in clear-cell renal cell carcinoma (ccRCC), irrespective of prior exposure to cabozantinib (Cabometyx), a small clinical trial showed. Overall, 12 of 32 patients responded to zanzalintinib, including four of 17 evaluable patients with prior exposure to cabozantinib and eight of 14 without. All but one […]
Novel Aldosterone Synthase Inhibitor Cuts Albuminuria in Phase II Trial
PHILADELPHIA — An oral investigational aldosterone synthase inhibitor (ASI) cut albuminuria by nearly 40% in people in chronic kidney disease (CKD) in a phase II trial. The 10-mg dose of ASI BI 690517 yielded an average 37.4% (95% CI -52.2 to -18.2%) greater reduction in urine albumin-creatinine ratio (UACR) from baseline to week 14 compared […]
Potential Cure for Sickle Cell Clears Hurdle for Approval
An FDA advisory committee meeting on Tuesday did nothing to dispel expectations that the investigational agent exagamglogene autotemcel (exa-cel) will become the first approved gene therapy for sickle cell disease (SCD). Members of the Cellular, Tissue, and Gene Therapies Advisory Committee were not asked to vote on the agent’s efficacy or safety, but instead directed […]
Subcutaneous Ocrelizumab Not Inferior to IV Infusion
MILAN — An investigational 10-minute subcutaneous injection of ocrelizumab (Ocrevus) was non-inferior to the established IV infusion formulation of the drug in multiple sclerosis (MS), the phase III OCARINA II trial showed. During the first 12 weeks of treatment, blood levels of ocrelizumab — measured as the area under the serum concentration time curve (AUC) […]
FDA Staff Voice Serious Concerns About Investigational ALS Cell Therapy
FDA reviewers voiced serious concerns about the investigational amyotrophic lateral sclerosis (ALS) treatment debamestrocel — mesenchymal stromal cells secreting neurotrophic factors, also known as MSC-NTF, or NurOwn — from BrainStorm Cell Therapeutics ahead of an advisory committee meeting this week. When the FDA first received the drug’s application for approval, it determined the submission was […]
Donanemab Slows Progression in Early Alzheimer’s
AMSTERDAM — Investigational donanemab significantly slowed clinical progression at 76 weeks in people with early symptomatic Alzheimer’s disease who had amyloid and tau pathology, the phase III TRAILBLAZER-ALZ 2 trial showed. The drug, which targets a modified form of beta amyloid known as N3pG, showed significant slowing versus placebo on measures of cognition and daily […]
Novel Turmeric Derivative Drug Shows Promise in Head and Neck Cancer
MONTREAL – An investigational drug derived from turmeric (Curcuma longa) appeared to have activity in patients being evaluated for head and neck cancer therapy, at least in an early phase trial. All nine oropharyngeal squamous cell carcinoma patients treated in the phase II trial had their disease under control after 4 to 6 weeks of […]