Mirum's brelovitug hit its Phase 3 endpoint in hepatitis delta, but Gilead's Hepcludex already holds the US approval and Vir's program is in flight. A strong readout no longer guarantees the market follows.
Lilly's once-weekly insulin Onswik is the second FDA approval in a six-month window, after Novo's Awiqli. Both won on non-inferiority data, leaving the real question open: whether weekly dosing actually improves insulin initiation and adherence.
The FDA approved Atebrioz for FOP on a change in new bone volume, a measure the trial registry listed as secondary, after the lesion-count primary missed statistical significance at week 24.
Praxis Precision Medicines bypassed non-selective sodium channel toxicity in SCN2A and SCN8A DEEs by targeting persistent current. Now the FDA is examining its pivotal trial.
BigHat Biosciences raised $75 million as BHB810, a CDH17 VHH-Fc ADC, entered a Phase 1/2 gastric cancer trial. The round tests whether wet-lab data can fix AI drug discovery's translation problem.
FDA cleared lirafugratinib (Lyrfigtu) for FGFR2-altered cholangiocarcinoma after ReFocus posted a 46% response rate. The clinical argument is FGFR1 sparing. The cost is months of mandatory retinal monitoring.
Barzolvolimab cleared hives in about half of Phase 3 CSU patients by week 24, including many Xolair failures, but two anaphylaxis cases sent Celldex shares down 13% ahead of a 2027 BLA.
Iambic Therapeutics filed for a Nasdaq IPO the same morning it announced an AbbVie collaboration. Public markets will still judge the company on whether its brain-penetrant HER2 inhibitor holds up against Enhertu and Tukysa.
IntraBio’s Aqneursa cleared the FDA as the first treatment for ataxia-telangiectasia on a -1.88 SARA point crossover win, treating downstream neurodegeneration rather than an intractable 305 kDa kinase defect.
FDA approved Lilly's Inluriyo plus Verzenio for ESR1-mutated ER+/HER2- metastatic breast cancer after endocrine progression, doubling median PFS to 11.1 months in EMBER-3. The clinical gain comes with 86% diarrhea and neutropenia rates, challenging early liquid biopsy switching models.
Ultragenyx's AAV9 gene therapy is the first treatment for Sanfilippo syndrome Type A. Full approval on 17 treated children against a natural history control now collides with a diagnostic window most families still miss.
Curium’s Bexlutry is the first 505(b)(2) radioligand equivalent. The regulatory shortcut is done. Hospital clocks, isotope supply, and a pending $8 billion merger now decide whether Novartis’s NET franchise actually erodes.