Clinical Trial News

A phase 1b study of abemaciclib monotherapy in heavily pretreated patients with metastatic ccRCC showed no clinically meaningful activity, with no objective responses and rapid disease progression in most patients. Despite the negative results, these findings are crucial for understanding the potential of CDK4/6 inhibitors in combination therapies for RCC, guiding future trials.

At ESC 2024, Dr. Erin Michos discussed the success of obicetrapib trials, showing its ability to reduce LDL. Completion of Phase III TANDEM trial enrollment brings NewAmsterdam closer to launching obicetrapib for HeFH/ASCVD. If successful, it could offer a new treatment for high cholesterol, addressing the unmet need for Lp(a) reduction.

CGTLive®’s Weekly Rewind covers FDA actions, research, and interviews in gene and cell therapies. Highlights include no FVIII-specific responses in hemophilia A patients, mouse research findings at ASGCT’s 2024 conference, Galpagos expanding its ATALANTA-1 trial to the US, NMPA’s decision based on the CARTIFAN-1 trial, and Dr. Mark Walters discussing gene therapy’s impact on treatment.

NXP800, a novel GCN2 kinase activator, received orphan drug designation from the FDA for ARID1a-deficient ovarian, fallopian tube, and primary peritoneal cancers. It is an oral small molecule inhibitor targeting the HSF1 pathway, showing strong antitumor effects in ARID1a-mutated ovarian carcinoma. A phase 1b clinical trial (NCT05226507) is ongoing, evaluating NXP800 in platinum-resistant, ARID1a-mutated ovarian cancers.

A study at St Vincent's University Hospital challenges the belief that weight loss medications like Ozempic, Wegovy, or Monjaro work solely by promoting satiety. The randomized controlled trial, led by Professor Donal O'Shea, found a strong relationship between the increase in metabolic activity caused by GLP-1 treatment and weight loss, with low metabolic activity prior to treatment showing the most benefit.

The SELECT trial at ESC Congress 2024 evaluated semaglutide for obesity-related HFpEF, showing reduced risk of MACEs in females with overweight/obesity and ASCVD, without diabetes, and significant reductions in body weight, waist circumference, and hsCRP with semaglutide compared to placebo, though ARR was higher in males. Semaglutide's efficacy was consistent by sex across cardiovascular, metabolic, and kidney endpoints, with comparable safety profiles between sexes.

Phase I clinical trial of NH130, a selective serotonin 5-HT2A inverse agonist for Parkinson's disease psychosis, showed favorable pharmacokinetics and safety in healthy volunteers, with no serious adverse events reported. A physiologically based pharmacokinetic model accurately predicted NH130's behavior, supporting its potential as a therapeutic agent for Parkinson's disease psychosis.

Phase 3 trial initiated for oral cladribine in treating generalized myasthenia gravis, targeting B and T lymphocytes, potentially offering home administration and disease progression slowdown.

CellProthera's positive pre-IND meeting with FDA paves way for Phase III trial of ProtheraCytes, a cell therapy for myocardial infarction. The trial, with a two-year follow-up, aims to prevent subsequent heart diseases. The company plans to pursue RMAT designation for expedited development and review.

Orgenesis' ORG-101 CAR-T therapy showed efficacy and safety in 233 CD19+ B-cell ALL patients in China, with 82% CR rate in adults and 93% in children. Severe CRS was observed in 2% of adults and 6% of children. ORG-101 is produced via a decentralized approach and Orgenesis plans a multicenter phase 1/2 trial, starting in Greece.