#诺华

#诺华

Today 0 items

Oct 15Thu

Thursday

07:55
doi.orgScore4848
Open-label extension study of leniolisib in activated PI3Kδ syndrome: Long-term efficacy and safety
Long-term leniolisib was generally well tolerated in 37 patients with activated phosphoinositide 3-kinase delta syndrome treated for a median of 4.3 years. Annual infection rates (P = 0.0005), respiratory infection rates (P = 0.0017) and immunoglobulin replacement therapy use (P = 0.0095) all declined per year of treatment, with clinically meaningful gains in 36-Item Short Form Survey general health scores. This extends the efficacy and safety evidence for leniolisib in APDS well beyond the pivotal trials.
Clinical #Clinical readout #Small molecules & degraders #Rare disease

Sep 4Fri

Friday

21:00
LancetPickScore7474
[177Lu]Lu-PSMA-617 in patients with PSMA-positive metastatic androgen pathway modulator-naive/sensitive prostate cancer (PSMAddition): a phase 3 randomised, controlled trial.
Adding 177Lu-PSMA-617 to androgen deprivation therapy plus an androgen receptor pathway inhibitor reduced the relative risk of radiographic progression or death by 28% in PSMA-positive metastatic APMN/S prostate cancer, meeting the primary endpoint of prolonged radiographic progression-free survival. In the phase 3 PSMAddition trial, 572 patients per arm were randomised; radiographic progression or death occurred in 24% of the 177Lu-PSMA-617 arm versus 30% of the control arm. Grade 3 or worse adverse events occurred in 51% versus 43%, with dry mouth the most common event in the 177Lu-PSMA-617 arm.
Clinical #Clinical readout #Oncology #Novartis
Why it matters: It tests PSMA-directed radioligand therapy in first-line metastatic hormone-sensitive disease, earlier than current approved use.