Clinical Trial News

Iterum Therapeutics received a U.S. patent allowance for its sulopenem etzadroxil and probenecid combination, extending protection until 2039. This patent strengthens its portfolio for treating infections, including uncomplicated urinary tract infections, with ongoing Phase 3 trials. Iterum aims to combat multi-drug resistant pathogens globally.

Recent discussions highlight the ongoing challenges in Alzheimer's Disease clinical research, particularly in achieving diverse racial and ethnic representation in clinical trials. Additionally, the impact of hypertension on cognitive decline and dementia is underscored, alongside the exploration of IGC-AD1's efficacy in reducing agitation in Alzheimer's patients. Parkinson's disease psychosis, affecting over 20% of Parkinson's patients, is also a focal point, emphasizing the need for comprehensive patient care.

Agios Pharmaceuticals reported new Phase 2 study data on PYRUKYND® for α- and β-thalassemia, showing durable improvements in hemoglobin and hemolysis markers. The drug was well-tolerated, with a consistent safety profile. Agios is enrolling for Phase 3 trials and highlighted the need for new thalassemia therapies at the ASH meeting.

Psilocybin-assisted therapy significantly improved psycho-social-spiritual wellbeing in cancer patients with major depressive disorder, as measured by NIH-HEALS. Key factors—Connection, Reflection & Introspection, and Trust & Acceptance—showed notable improvements, sustained up to 8 weeks post-treatment.

Novartis' Phase III APPOINT-PNH study showed iptacopan, an oral monotherapy, significantly increased hemoglobin levels in complement-inhibitor-naïve PNH patients without blood transfusions. Iptacopan also demonstrated superiority over anti-C5 therapies in the APPLY-PNH study, offering potential as the first oral monotherapy for PNH.

HepaRegeniX announced positive Phase 1 trial results for HRX-0215, a MKK4 inhibitor, showing it's safe and well-tolerated in healthy volunteers. The trial, involving 48 participants, confirmed HRX-0215's potential in liver regeneration without adverse effects. HepaRegeniX plans Phase 2 trials to further explore its therapeutic benefits for liver diseases.

FDA approved olutasidenib for relapsed/refractory AML with IDH1 mutation, alongside Abbott RealTime IDH1 Assay for patient selection. Study 2102-HEM-101 showed 35% CR+CRh rate, median CR+CRh duration of 25.9 months, and significant transfusion independence. Common adverse effects included nausea, fatigue, and arthralgia.

Lecanemab, the first drug to slow Alzheimer's brain destruction, marks a historic breakthrough despite its modest effect and debate over real-world impact. Targeting amyloid, it offers hope for early-stage patients, yet challenges in early detection and treatment accessibility remain. Side effects and the need for further research highlight the complexity of Alzheimer's therapy.

CAR T cells, engineered immune cells, show early promise in treating lupus, an autoimmune disease, by targeting B cells. Pioneered by Carl June and team, this personalized therapy, initially developed for blood cancers, is being explored for broader applications, including autoimmune diseases, by researchers worldwide.

Lecanemab, an experimental Alzheimer's drug, shows potential in slowing cognitive decline by 27% in Phase 3 trials, despite safety concerns. It reduces amyloid levels and cognitive decline more than placebo at 18 months but is associated with adverse events like brain swelling and bleeding. Longer trials are needed to confirm its efficacy and safety.