Clinical Trial News

A study led by UCL researchers found that vutrisiran, a gene silencer drug, reduced hospitalisation and death risk by 28% over 3.5 years in ATTR cardiac amyloidosis patients, with a 33% reduction in non-tafamidis users. Vutrisiran, one of four promising treatments, binds to mRNA to reduce TTR protein production, potentially becoming the new standard of care.

D-VRd followed by ASCT, D-VRd consolidation, and DR maintenance improved MRD negativity rates in transplant-eligible myeloma patients compared to VRd induction, ASCT, VRd consolidation, and lenalidomide maintenance, according to PERSEUS trial data. D-VRd showed higher MRD-negativity rates at 10^-5 and 10^-6 thresholds, sustained MRD negativity, and improved PFS, suggesting it as a new standard of care.

Moshe Ornstein discusses IO/TKI doublet regimens in non-ccRCC, highlighting KEYNOTE-B61 trial (ORR 51%, median PFS 17.9 months) and cabozantinib/nivolumab trial (ORR 48%, median PFS 13 months). Both regimens supported by NCCN guidelines, emphasizing IO/TKI as standard care for non-ccRCC patients.

Sandra Christensen discusses the challenges of accessing obesity medications, noting insurance barriers and the need for comprehensive obesity management, including lifestyle changes and anticipatory guidance for side effects. She predicts increased insurance coverage due to clinical trial data and future FDA approvals of new medications.

Justin Walsh from JonesTrading reiterated a Buy rating on Vor Biopharma (VOR) with a $18.00 price target, citing promising data from the VBP101 trial treating AML with trem-cel and MYLOTARG, and strategic discussions with the FDA around year-end 2024. Vor Biopharma also progresses with the VBP301 trial and introduces a new preclinical asset, VADC45.

Denileukin diftitox-cxdl (Lymphir) added to NCCN guidelines as a category 2A recommendation for CTCL, based on phase 3 Study 302 showing 36.2% ORR and 8.7% CR rate. The therapy targets IL-2 receptor and received FDA approval a month prior.

A new FDA-approved implantable spinal cord stimulator, Inceptiv™, may offer relief for chronic pain by sensing and adjusting stimulation to block pain signals before they reach the brain.

OrsoBio raises $67M to advance weight loss drugs; Vor Bio's CRISPR-edited stem cells show success in AML therapy; Boehringer Ingelheim to test eye drug for geographic atrophy; Novartis licenses capsid from Voyager for gene therapy; Vaxcyte raises $1.3B on positive pneumococcal vaccine data.

Eligibility criteria for OAG/OHT study eyes include C/D ratio ≤ 0.8, visual field mean deviation ≥ −12 dB, BCVA 20/80 or better, and specific IOP requirements. Exclusions involve prior glaucoma surgeries, ocular conditions affecting measurements, and systemic diseases. Procedures include informed consent, ophthalmic assessments, and randomization to FE/SE implants or sham. Follow-up visits assess IOP, visual acuity, and adverse events. Statistical analysis aims to confirm non-inferiority of implant effects on IOP reduction.

Advisory committee meeting on September 9, 2024, to discuss the NDA for oral sulopenem tablets, developed by Iterum Therapeutics to treat UTIs in women. The REASSURE trial demonstrated non-inferiority of sulopenem compared to Augmentin, with a similar safety profile and no new safety signals except those linked to β-lactams. The FDA accepted the resubmitted NDA with a PDUFA action date of October 25, 2024.