Clinical Trial News

A study assessing drug regulatory agencies' decisions on extended-release methylphenidate for adult ADHD found that 7 out of 13 applications were missing entire trials, despite regulatory requirements suggesting all trials should be included.

Sanofi and GSK's Vidprevtyn, a protein subunit Covid-19 vaccine, shows promising phase 3 trial results, with 58% efficacy against symptoms, 75% against moderate/severe disease, and 100% against severe disease. It's effective as a booster, increasing antibodies 18-30 fold. Plans to seek US and EU authorization, with 100 million doses already produced.

The FDA approved PYRUKYND® (mitapivat) for treating hemolytic anemia in adults with pyruvate kinase (PK) deficiency, based on ACTIVATE and ACTIVATE-T Phase 3 studies. Agios offers patient access programs, including $0 copays and free medication for eligible patients. PYRUKYND® is the first disease-modifying therapy for PK deficiency.

Studies on PARPi in ovarian cancer show improved progression-free survival (PFS) but unclear overall survival (OS) benefits. Quality of life (QoL) data is heterogeneous, with some studies indicating maintained QoL during treatment. Serious adverse events are more common with PARPi, impacting QoL. Evidence is most robust for olaparib, with less data for other PARPis. Long-term follow-up is needed for clearer OS outcomes.

A review of the current landscape of glioblastoma clinical trials highlights the challenges in trial design and eligibility criteria, with recommendations from a Society for Neuro-Oncology Think Tank aimed at improving outcomes.

R-3750, R-2487, R-5280, and R-5780 are oral therapies targeting immune pathways for treating inflammatory bowel disease, rheumatoid arthritis, type 1 diabetes, and cancer, respectively. Each is in Phase 1 trials, focusing on safety, tolerability, and efficacy. Details for participation are available at NCT05666960, NCT05961592, and NCT06057454.

Vaccination for children under 5 delayed until April as FDA and Pfizer await more data on a third dose's safety and efficacy. The delay, aimed at ensuring vaccine safety, follows insufficient immune response from two doses in trials. FDA emphasizes adherence to rigorous standards amidst urgent public health needs.

Combination therapies targeting pancreatic cancer's extracellular stroma and membrane receptors show promise. Hyaluronidase and anti-PD-L1/PD-1 antibodies enhance tumor growth inhibition. IL-6 blockade with anti-PD-L1 improves survival. VDR ligands and TGF-β inhibitors combined with checkpoint inhibitors increase T cell infiltration. CD40 agonists and CXCR4 inhibitors with anti-PD-1 show potential in enhancing anti-tumor immunity. FAK and BTK inhibitors, alongside PARP inhibitors, are being explored for their roles in improving treatment efficacy.

FDA accepted Cytokinetics' NDA for omecamtiv mecarbil, targeting HFrEF, with a PDUFA action date of November 30, 2022. No advisory committee meeting is planned. GALACTIC-HF trial showed omecamtiv mecarbil reduced CV death or heart failure events risk. METEORIC-HF results expected early 2022.

Pembrolizumab, a cancer drug, shows potential in flushing HIV from its hiding spots in immune cells, offering a new direction for HIV cure research. Despite challenges with side effects, early studies indicate it could make latent HIV visible to the immune system, marking a significant, though not definitive, step towards a cure.