Key Outcomes: Arrowhead (Plozasiran, sHTG) – P3 SHASTA-3/4 met primary endpoint; TG reductions 79%/81% vs ~27% PBO; AP events cut 78% overall, 100% in high-risk patients; sNDA planned by YE’26 Azafaros (Nizubaglustat, GM1/GM2 Gangliosidosis) – Completed enrollment (~75 pts) in P3 NAVIGATE; topline data expected early 2028; NPC study enrollment ongoing (72 pts) Novartis Fabhalta […]

Key Outcomes: Teva (TEV’408, vitiligo): P1b shows 75% facial improvement (42% F-VASI50), Q12M dosing feasible, advancing to P2b in Q4 2026 with Royalty Pharma funding Ipsen (Dysport, migraine): First botulinum toxin to hit P3 success in both episodic and chronic migraine prevention Novartis (Itvisma, SMA): EC approves first one-time gene therapy for 5q SMA in […]

Key Outcomes: Nerandomilast (Boehringer) – FIBRONEER studies met the primary endpoint (slowed FVC decline); pooled analysis showed 59% reduction in death risk vs. placebo, but the key secondary composite endpoint was missed Evategrel/CG-0255 (CureGene) – First patient dosed in US pivotal trial vs. clopidogrel; prior data show fast onset (<15 min IV/<30 min oral), high […]

Key Outcomes: Riliprubart (Sanofi) – P3 MOBILIZE, CIDP: Trial stopped after interim analysis showed insufficient efficacy likelihood; no safety concerns. No material impact on 2026 guidance TransCon PTH (Ascendis Pharma) – P3 PaTHway, Hypoparathyroidism: Wk 182 data show durable efficacy and favorable long-term safety, with normalized calcium, improved kidney function, and sustained QoL benefits Selinexor […]

Key Outcomes: LET003 (Harbour BioMed): AI-designed ACVR2A/2B mAb showed 76% fat mass reduction and 6.5% better muscle preservation vs semaglutide alone in DIO mice, advancing to clinical development Ziihera (Jazz): P3 HERIZON-GEA-01 published in NEJM demonstrating superior PFS & OS vs SOC in 1L HER2+ GEA, submitted to NCCN for guideline inclusion Arexvy (GSK): Japan’s […]

Key Outcomes: TREMFYA met primary endpoint in P3 FUZION study for perianal fistulizing Crohn’s disease – first RCT to show an approved IBD therapy works in this population Tovorafenib (OJEMDA) P3 FIREFLY-2 completed enrollment in frontline pLGG; preliminary results expected 2027 Ligufalimab triplet showed improved survival in P2 AML study with comparable safety to standard […]