Disease

New modelling data links antibiotic disc exposure to clinical benefit, strengthening the rationale for PP353 as a targeted, non-opioid therapy for vertebrogenic lumbar

Tarsus gains worldwide rights to Phase 3 oral candidate ALK-001 for Stargardt disease, a blinding inherited retinal condition with no approved therapy.

Palopegteriparatide gains a positive reimbursement recommendation in Canada for adults with chronic hypoparathyroidism inadequately controlled on standard therapy.

Phase 1 data show CLYM116 achieved near-complete APRIL suppression and a 29-day half-life, supporting quarterly dosing in IgA nephropathy.

Eledon Pharmaceuticals has submitted an IND for a registrational islet cell transplant study of tegoprubart, while reaffirming its Q4 2026 Phase 3 kidney trial start.

The Thousand Oaks biotech floated on Nasdaq in August 2026 and published NEJM data on its lead NaV 1.8 inhibitor onzotrigine in acute pain.

Newron Pharmaceuticals has finished screening for its 600-patient Phase 3 trial of evenamide in treatment-resistant schizophrenia, with topline results expected early

Four-year open-label extension data showed durable seizure reductions and cognitive gains in Dravet syndrome; Phase 3 readout is expected in Q3 2027.

The London RNAi biotech said its siRNA candidate cleared a safety review in healthy volunteers and now enters multiple ascending dose testing in MASH patients.

The Milan biotech signed a convertible loan with the EIB, covering half of its €40m plan to reach CE Mark and EU launch for REGENERA

The San Carlos biotech reported preliminary proof of concept for ATTO-1310 in chronic pruritus and atopic dermatitis, with two Phase 2 studies planned for

Topline results for lanifibranor's pivotal 1,009-patient trial are expected in Q4 2026, with regulatory submission targeted for H1 2027 if data are

China's NMPA has approved Eisai and Biogen's once-weekly lecanemab SC formulation for early Alzheimer's, making China the second market globally to

Nyxoah's Genio device achieved a 77.2% AHI responder rate with zero serious device-related adverse events, clearing the path for a US PMA

The North Carolina gene therapy company will share Cohort 1 topline results for its BEST1 retinal disease programme at an investor webcast and three ophthalmology

The Alameda-based CRISPR company began a Phase 1 trial of its epigenetic LDL-C therapy and secured $25m in non-dilutive CIRM grants alongside

Teva's anti-IL-15 antibody TEV-408 significantly reduced gluten-induced intestinal damage versus placebo in a 50-patient Phase 2a study.

Ultragenyx's apazunersen missed both its primary and key secondary endpoints in the Phase 3 Aspire study, leaving Angelman syndrome without an approved treatment.

Alpha Tau Medical has finished enrolling 48 patients in its IMPACT pilot study of Alpha DaRT for unresectable pancreatic cancer, with data expected in early

BioVie says its ADDRESS-LC Phase 2 trial of bezisterim has finished dosing, with topline results expected before end of September 2026.