Congenital Hyperinsulinism International’s Post

Recently published in JCO: Intracranial outcomes data from LIBRETTO-431, a phase 3 study investigating front-line use of a selective RET inhibitor in patients with RET fusion+ advanced #NSCLC. Read the results here: https://e.lilly/3XC9MwI #PrecisionMedicine #LungCancer #LCSM

Excellent news from ImmunoGen, which was recently acquired by AbbVie. ELAHERE is the first antibody-drug conjugate (ADC) to be approved for the treatment of ovarian cancer. Data from a Phase 3 trial showed that ELAHERE treatment reduces the risk of cancer progression by 35% and improves overall survival by 33%. Ovarian cancer is the leading cause of gynecologic cancer death in the U.S., and most patients present with late-stage disease. The current standard of care is treatment with conventional platinum-based chemotherapy but the majority of patients eventually develop resistance to these drugs. ELAHERE is a targeted therapy for patients with folate receptor-alpha (FRα) positive, platinum-resistant ovarian, fallopian tube, or primary peritoneal cancer, who have received one to three prior systemic treatment regimens. Over 80% of ovarian tumors express FRα on their cell surface, and increased expression is associated with more aggressive tumors. ELAHERE targets these receptors via an FRα-binding antibody linked to DM4, a drug designed to kill actively dividing tumor cells. These are exciting, game-changing times for cancer treatment! 🦀💊⚕️

Mirvetuximab soravtansine-gynx approved: first antibody/antigen-drug conjugate (ADC) in advanced or recurrent ovarian cancer

Paltusotine, an investigational, first-in-class, oral somatostatin receptor type 2 (SST2) agonist, is in Phase 3 clinical development for acromegaly and Phase 2 clinical development for carcinoid syndrome associated with neuroendocrine tumors. Crinetics has demonstrated pharmacologic proof-of-concept in a Phase 1 clinical study for CRN04894 a first-in-class, investigational, oral ACTH antagonist, that is currently in Phase 2 clinical studies for the treatment of congenital adrenal hyperplasia and Cushing’s disease. #Congenitaladrenalhyperplasia #Cushingdisease #ACTHantagonist #Drugdevelopment #Innovation #Acromegaly #Firstinclass

At #ESMO24, late-breaking primary results from the DESTINYBreast-12 trial revealed substantial and durable efficacy with the antibody–drug conjugate (#ADC) trastuzumab deruxtecan in patients with HER2+ metastatic #BreastCancer, including those with brain metastases. Encouraging data were also presented from the smaller ICARUS-BREAST01 study with the novel anti-HER3 ADC patritumab deruxtecan in patients with HR+/HER2- #AdvancedBreastCancer after failure of CDK-4/6 inhibitor therapy. 👉Read the full-data commentary by Philippe Aftimos MD on the #ESMODailyReporter https://ow.ly/gRCQ50TmTCX

TiNivo-2 Study of Nivolumab and Tivozanib Fails to Meet Primary End Point in #RCC

Late-Breaking primary results from the phase IIIb/IV, open-label DESTINYBreast-12 trial revealed substantial and durable efficacy in 504 patients with HER2-positive metastatic breast cancer, including those with brain metastases (LBA18). In the cohort with brain metastases (n=263), the 12-month progression-free survival (PFS) rate was 61.6%, while the 12-month central nervous system (CNS) PFS was 58.9%, with similar results in those with stable (PFS, 57.8%) and active (PFS, 60.1%) brain metastases. The 504 patients enrolled in the open-label trial had experienced disease progression on ≤2 prior lines of therapy

🚩 Despite high initial response rates to platinum-based chemotherapy, inoperable, locally recurrent or metastatic squamous cell carcinoma of the anal canal (SCAC) is still associated with poor PFS and OS. 💊 The POD1UM-303 study presented at #ESMO24 discovered that the addition of #retifanlimab to #carboplatin-paclitaxel can significantly improve PFS and ORR in patients with locally recurrent/metastatic SCAC:

In this systematic review and network meta-analysis of 27 randomized clinical trials (including a total of 13 413 individuals), the pCR rates achieved with total neoadjuvant therapy regimens outperformed standard long-course chemoradiotherapy, with long-course chemoradiotherapy plus consolidation chemotherapy showing the greatest effect estimate.

Valved PICC comparison study - Full Text... "In this study, to identify the most suitable PICC for continuous pumping of 5-fluorouracil (5-FU) in patients undergoing chemotherapy for gastrointestinal tract tumors, the clinical application of the double-lumen high-pressure-resistant PICC generally adopted in clinical practice was compared with the traditional anterior-valved single-lumen non-high-pressure-resistant PICC" Chen et al (2024).