Clinical Trial News

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.

New data in *The American Journal of Cardiology* shows TAVR is safe and effective for nonagenarians, with a 30-day mortality rate of 5.5% and 1-year mortality of 11.6%, suggesting fast recovery post-TAVR.

Rashid Sayyid and Zachary Klaassen discuss 2024 NCCN prostate cancer guideline updates for systemic therapy in metastatic hormone-sensitive prostate cancer (mHSPC), emphasizing treatment stratification by disease volume and timing. They cover clinical trials supporting triplet therapy for high-volume synchronous disease, like ARASENS and PEACE-1, and different treatment approaches for various patient subgroups. The role of prostate radiotherapy in low-volume synchronous mHSPC is reviewed, along with the expanding use of stereotactic body radiation therapy (SBRT) for oligometastatic disease. The discussion concludes with a personalized treatment paradigm considering both disease volume and timing in mHSPC management.

Wall Street is bullish on Viking Therapeutics (VKTX) due to successful clinical trials of its weight loss drug VK2735, which showed significant weight loss in patients. Billionaires Jeff Yass and Israel Englander have increased their stakes in the company. VK2735, a dual GLP-1 and GIP receptor agonist, outperformed competitors like Novo Nordisk's semaglutide and Eli Lilly's tirzepatide. Viking Therapeutics also has a promising drug, VK2809, for metabolic dysfunction-associated steatohepatitis (MASH). Upcoming catalysts include phase 3 trials for VK2735 and a phase 2 study of oral VK2735. Despite potential risks, Viking Therapeutics is considered a high-risk, high-reward investment.

Pediatric JAKi patients report more lymphatic and blood disorders, while adults report more nervous and musculoskeletal system disorders. Research by Talasila et al. analyzed FDA databases (2011-2023) for 5 approved JAK inhibitors in pediatric patients, finding common AEs in both age groups but with notable differences in distribution.