Clinical Trial News

OncLive’s weekly roundup highlights FDA approvals, including afamitresgene autoleucel for synovial sarcoma, vorasidenib for astrocytoma/oligodendroglioma, CB-012 for AML, and companion diagnostics for olaparib/abiraterone in BRCA+ mCRPC. Interviews discuss targeted therapies in mCRC and ASCO’s health equity recommendations.

Elevidys, an AAV-based gene therapy for Duchenne muscular dystrophy, received FDA approval in 2023 for ambulatory patients aged 4-5 with specific DMD gene mutations, and expanded approval in 2024 for broader patient groups. While not curative, it offers a functional gene version, but faces challenges like insurance coverage and long-term data needs. Experts highlight the importance of patient education and choice amidst expanding treatment options.

Two biotechs, Bicara Therapeutics and Zenas BioPharma, plan to raise $200 million each in IPOs. Bicara's ficerafusp alfa targets EGFR and TGF-b for potent anti-tumor activity, while Zenas' obexelimab targets CD19 and FcγRIIb to treat autoimmune diseases. Both companies expect pivotal trial data in 2025.

A new bowel cancer trial using immunotherapy (pembrolizumab) before surgery showed 59% of patients cancer-free post-treatment, with all patients remaining cancer-free months later, potentially reducing the need for post-operative chemotherapy.

Reid Merryman, MD, discussed the evolution of minimal residual disease (MRD) testing in lymphoma, focusing on the use of circulating tumor DNA (ctDNA) assays at the SOHO 2024 Annual Meeting. He highlighted the increased sensitivity of next-generation sequencing and the potential for MRD-guided therapy in clinical trials, emphasizing the need for standardized assays to integrate MRD into treatment decisions.

The FDA granted breakthrough therapy designation to edaravone and dexborneol sublingual tablets (Sanbexin; Simcere Pharmaceuticals) for acute ischemic stroke. The phase 3 trial showed significant neurological recovery and independent living ability improvement compared to placebo. The sublingual formulation allows rapid absorption, with 64.4% achieving mRS score of 1 or less on day 90 in the study drug group versus 54.7% in the placebo group. Adverse events were similar between groups.

BVI to launch SERENITY and SERENITY Toric IOLs in 2025; modeling life after blue zones for longevity; hyperopia linked to depression risk; saline-powered ultra-thin battery for smart contact lenses; Ocuphire Pharma starts phase 3 VEGA-3 trial for presbyopia treatment.

Huntington Potter's clinical trial tests Leukine for reversing Alzheimer's memory loss. Alzheimer's affects 10% of those over 65 and 30-40% of those over 85. Leukine, an FDA-approved drug, showed improvement in a 3-week trial and is now in a 24-week trial. Alzheimer's and Down syndrome share a connection through chromosome 21, influencing Alzheimer's research. Current Alzheimer's drugs slow decline, not reverse it, with side effects like brain bleeding. Behavioral changes like exercise and diet can reduce Alzheimer's risk.

The study involves a 45-month trial with 8 centers, recruiting patients over 24 months for ziconotide or placebo treatment phases, each lasting 6 months, with a 1-month wash-out period. Patients undergo LP or continuous infusion tests to determine response, with responders implanted with continuous infusion pumps. The study includes detailed visit schedules, randomization procedures, and statistical analysis plans to evaluate pain reduction and patient satisfaction.

A study published in Science Translational Medicine identifies 34 protein modules in cerebrospinal fluid (CSF) linked to Alzheimer’s disease (AD), including those involved in autophagy, ubiquitination, and glycolysis. Proteins related to oxidative stress, mitochondrial function, and neddylation were tied to ApoE4. In serum, these modules predicted AD up to two decades in advance. A clinical trial showed atomoxetine, a norepinephrine reuptake inhibitor, could mitigate some AD-associated pathways, particularly glycolysis.