Disease

CervoMed will present 12-week plasma biomarker data from its Phase 2a nfvPPA trial at ISFTD in October, covering at least 22 participants.

The Munich biotech will use the HERA Invest-backed funds to progress its interferon-lambda mRNA candidate into Phase 2b trials in asthma and COPD.

MannKind will supply its Technosphere platform and manufacturing support to develop ROSE-010, an inhaled rapid-acting GLP-1, through Phase 1b.

Medicus Pharma published its Phase 2 CSR for SkinJect, selecting the 200 µg patch dose and pivoting development toward rare Gorlin syndrome.

The Vancouver-based manufacturer will run a 200-participant, Health Canada-authorised study of naturally derived psilocybin in major depressive disorder.

The Stamford biotech reported highly significant SRI-4 response rates versus placebo at Week 48, supporting pivotal development in systemic and cutaneous lupus.

Phase 2 results show the anti-TMPRSS6 antibody reduced mean phlebotomy events from 4.0 to 0.6 over 26 weeks, with 61.5% of

Week 52 sentinel cohort data show navepegritide stabilised foramen magnum stenosis and improved linear growth in infants with achondroplasia.

ImPact Biotech has treated all 91 patients in its Phase 3 LG-UTUC trial and expects to file an NDA with the FDA in 2027.

Kura Oncology's newly spun-out subsidiary will use menin inhibition to try to restore pancreatic beta-cell function in Type 1 and Type 2

All five Cohort 1 participants showed clinically meaningful visual improvement, and the FDA has aligned on a potential pivotal endpoint for the rare retinal disease

Exploratory analyses from the Phase 3 PROPEL 3 trial show oral infigratinib reduced otitis media rates by 38% and stabilised sleep apnea measures versus placebo.

The designation accelerates review of the dual IRAK1/JAK2 inhibitor in a rare autoinflammatory disease with no approved treatments; topline Phase 2 data expected in

The FDA has accepted Roche's sBLA for satralizumab in MOGAD, a CNS autoimmune disease with no approved treatments, with a decision due January 2027.

Dual PDE3/4 inhibitor AN01 improved lung function versus placebo in COPD patients already on bronchodilator therapy, with data presented at ERS 2026.

The Cambridge, Massachusetts biotech secured FDA acceptance of its Oxnimbi NDA with a PDUFA date of 28 February 2027, as it raised $221m in a

Assembly Biosciences will take a 40% share of US costs and profits for the GS-1179 helicase-primase inhibitor as a Phase 2 trial approaches.

The Seattle-based biotech will use proceeds to run its ILLUMINATE Phase 2 study of BCB-276 in diffuse intrinsic pontine glioma, a universally fatal

Dyne will present pooled Phase 1/2 data for z-basivarsen against a matched natural history cohort, with registrational topline results due Q1 2027.

The Waltham-based neurodegenerative disease biotech completed its merger with Cyclerion and a $380m private placement, beginning Nasdaq trading under "KRSA" on 9