Investigational ALS Drug May Have Clinical Benefit, FDA Staff Says
Tofersen, an investigational antisense drug developed to treat amyotrophic lateral sclerosis (ALS) associated with a mutation in the SOD1 gene (SOD1-ALS), may have clinical benefit, FDA reviewers indicated in briefing documents ahead of an advisory committee meeting. On Wednesday, the Peripheral and Central Nervous System Drugs Advisory Committee will vote on whether evidence supporting a […]
Novel PET Imaging Agent Accurately IDs Clear Cell Renal Cancers
An investigational PET imaging agent — zirconium-89 (89Zr)-deferoxamine (DFO)-girentuximab — identified clear cell renal cell carcinoma (ccRCC) with high sensitivity and specificity, according to results from the phase III ZIRCON trial. Use of the agent achieved sensitivity and specificity rates of 85.5% (95% CI 79.8-89.8) and 87% (95% CI 78.8-92.3), respectively, reported Peter Mulders, MD, […]
Sotatercept, Fixed-Dose Combo Therapy on the Right Path for PAH
NEW ORLEANS — The limited clinical evidence thus far supports investigational compounds for addition to or simplification of existing frontline therapies for pulmonary arterial hypertension (PAH), according to two trials presented here at the American College of Cardiology (ACC) annual meeting. The activin signaling inhibitor biologic sotatercept showed promise as an add-on to usual treatment […]
Combo Falters in Papillary Renal Cancer, but Some Promise in MET-Driven Subset
Treatment with an investigational MET inhibitor plus a PD-L1 inhibitor induced responses in fewer than a third of patients with metastatic papillary renal cancer, but the combination showed promise for one subgroup, findings from the phase II CALYPSO trial showed. In the single-arm study, 12 of 41 patients (29%, 95% CI 16-46) had a confirmed […]
Second Trial Participant Dies in Alzheimer’s Drug Study
Days before the release of promising phase III trial data of lecanemab, an investigational anti-amyloid drug for early Alzheimer’s disease, a death possibly linked to the treatment was reported. An unpublished case report featured in Science suggested the death of a 65-year-old lecanemab trial participant who also had cerebral amyloid angiopathy — a condition in […]
Novel BTK Inhibitor Looks Good for Sjogren’s Syndrome
PHILADELPHIA — Remibrutinib, an investigational Bruton’s tyrosine kinase (BTK) inhibitor, appeared effective in reducing signs of Sjogren’s syndrome in a randomized, placebo-controlled trial. Scores on the EULAR Sjogren’s Syndrome Activity Index (ESSDAI), a comprehensive measure of Sjogren’s severity, fell by 2.86 points with the oral drug relative to placebo during the 24-week trial, according to […]
Oral Microbiome Therapy Tied to Low Recurrence of C. Diff
A potential first-in-class investigational microbiome therapeutic, SER-109, was safe and observed to sustain low Clostridioides difficile (C. diff) recurrence rates for adults, according to the phase III, single-arm ECOSPOR IV study. Recurrence of C. diff infections occurred in 8.7% of patients at week 8 after receiving a short course of capsules containing purified Firmicutes bacteria […]
A Once-Rejected Antibiotic Equals Standard for Complicated Staph Infections
WASHINGTON — Investigational ceftobiprole was just as effective as daptomycin for bacterial bloodstream infections caused by Staphylococcus aureus bacteremia, the phase III ERADICATE trial demonstrated. In the randomized head-to-head comparison, post-treatment success at 70 days was 69.8% with ceftobiprole and 68.7% with daptomycin, meeting prespecified criteria for non-inferiority, Thomas Holland, MD, of Duke University in […]
Investigational ALS Drug Betters Biomarkers, but Can It Improve Motor Functions?
Tofersen, an investigational antisense oligonucleotide developed to treat an inherited form of amyotrophic lateral sclerosis (ALS), changed biomarkers and curbed neurodegeneration but did not improve motor control and muscle strength at 6 months, the phase III VALOR trial showed. In a subgroup of patients with fast-progressing SOD1 ALS, the 28-week change in scores on the […]
‘Senolytic’ Therapy Shows Promise in Neovascular Eye Disease
NEW YORK CITY — An investigational drug targeting senescent cells achieved sustained improvement in visual acuity in patients with diabetic macular edema (DME) or neovascular age-related macular degeneration (AMD), a small pilot study showed. After single doses of UBX1325, four of eight evaluable patients with advanced DME had at least 10 letters of improvement in […]