Ultragenyx Doses First Patient in New Angelman Syndrome Study
Ultragenyx has fully randomized 129 patients aged 4–17 with full maternal UBE3A gene deletion in the global Phase 3...
FDA Grants Orphan Drug Status for Gastric Cancer Treatment
Dewpoint Therapeutics secured FDA Orphan Drug Designation for DPTX3186, an oral condensate modulator targeting the Wnt/β-catenin pathway in gastric...
gMG Biologics Expand in Early Treatment, Newcomers Challenge
Retention on first-line biologics in generalized myasthenia gravis sits at 90% in US practice, with Alexion’s Ultomiris and argenx’s...
IN8BIO Expands INB-100 Phase 1 Trial to Ohio State Site
Previously reported readouts from the Phase 1 INB-100 program have shown no severe graft-versus-host disease, persistence of allogeneic γδ...
Medicus & Gorlin Alliance Seek Compassionate Use for Skinject
An interim analysis from Medicus Pharma’s randomized, double-blind, placebo-controlled Phase 2 study (SKNJCT-003) reported more than 60% clinical clearance...
Avalo Completes Enrollment for Phase 2 LOTUS Trial in HS
Avalo Therapeutics has fully enrolled its Phase 2 LOTUS study of AVTX-009 in hidradenitis suppurativa, randomizing roughly 250 adults...
BlossomHill’s Promising Early Trial Data for NSCLC Drug
In the dose escalation of the first-in-human SOLARA study, BH-30643 was administered to 39 previously treated EGFR-mutant NSCLC patients...
Flare Therapeutics Reveals FX-909 Phase 1 Study Results
In Part A of a Phase 1 study in advanced urothelial carcinoma (UC), FX-909 monotherapy produced five objective responses...
Successful Phase 1 Data for REM-422 in Cancer Patients
REM-422, an oral small-molecule mRNA degrader targeting MYB, produced a 43% overall response rate by RECIST in the efficacy...
Halda’s New Prostate Cancer Drug Shows Encouraging Activity
In a first-in-human Phase 1/2 study of HLD-0915 in metastatic castration-resistant prostate cancer, 31% of all 31 treated patients...