Section
Mechanisms, platforms, and the research that matters.
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.
Two-year ORIGIN 3 data show weekly atacicept flattened eGFR decline to 0.6 mL/min per year and cut kidney-progression events by 76 percent, with no dialysis or transplants on drug.
The FDA has granted accelerated approval to Bayer's Hyrnuo as a frontline therapy in HER2-mutant lung cancer, matching rival zongertinib's response rates while carrying twice-daily meal requirements and steeper gastrointestinal toxicity.
FDA approval of Isembyld gives spinal muscular atrophy its first muscle-targeted therapy, moving motor function in patients plateaued on Spinraza or Evrysdi while introducing a 9% fracture warning and a double-biologic payer burden.
Pharvaris reported Phase 3 CHAPTER-3 data for deucrictibant extended-release 40 mg, delivering an 83% attack reduction in hereditary angioedema and matching the efficacy of injectable biologics.
KRAS sat on the undruggable list for decades. Silexion's SIL204 is built to silence the mutation earlier in the cell, and the Phase 2/3 program is now moving into patients.
Novartis said pelacarsen lowered Lp(a) in patients already on guideline-directed care, but Lp(a)HORIZON missed its cardiovascular composite versus placebo. The first large outcomes test of the inherited risk factor now hangs over later trials from Amgen and Lilly.
AbbVie's etentamig beat older salvage on response and progression-free survival in Phase 3 CERVINO. The harder question is whether a single step-up and monthly intravenous schedule can loosen the REMS-and-hospitalization architecture that still gates approved BCMA T-cell engagers.
A pre-specified VESALIUS-CV analysis found evolocumab cut all-cause death by 20% in high-risk primary prevention, with the benefit appearing only after 1.5 years and a 1.8-point absolute gap at five years.