Q3 Clinical trials round-up: July to September 2025
As the third quarter of 2025 draws to a close, the clinical research landscape has been marked by a surge of promising data, regulatory milestones, and strategic collaborations.
Across therapeutic areas – from neurodegeneration and oncology to rare diseases and regenerative medicine – biopharmaceutical companies have advanced novel mechanisms, precision approaches, and patient-centric delivery systems. This round-up captures the most compelling clinical trial developments shaping the future of medicine.
Alzheimer’s disease: Sustained progress with Leqembi
Eisai and Biogen’s lecanemab (Leqembi) continues to lead the charge in Alzheimer’s disease innovation. Four-year data from the Clarity AD Phase 3 open-label extension trial demonstrated sustained cognitive benefits, with a 1.75-point reduction in CDR-SB scores compared to natural disease progression. Notably, 56% of patients with low tau levels showed improvement, reinforcing the value of early intervention. Safety remained consistent, with declining ARIA rates over time.
Complementing this, Eisai unveiled data on a subcutaneous autoinjector formulation, offering comparable efficacy to IV dosing and enhanced convenience. With over 95% of participants successfully self-administering the device, the SC-AI option could redefine long-term care for early-stage Alzheimer’s patients. Regulatory momentum continues, with approvals in 46 countries.
Cardiometabolic and renal innovation: New frontiers
Corteria Pharmaceuticals advanced two first-in-class CRF2 agonists – COR-1167 for worsening heart failure and COR-1389 for obesity-related heart failure. Both programmes showed encouraging safety and efficacy signals, with COR-1389 demonstrating additive metabolic benefits when combined with GLP-1 agonists.
Meanwhile, Eleva initiated a Phase 1 trial for CPV-104, a Factor H-based therapy for C3 glomerulopathy. With orphan drug designation and promising preclinical data, Eleva’s moss-based biologics platform is gaining traction in rare renal and ocular diseases.
Ophthalmology and pain: Topical therapies gain ground
Exonate’s EXN407, a topical SRPK1 inhibitor, is poised to become the first eye-drop therapy for diabetic retinopathy. Following strong Phase Ib/IIa results, a Phase IIb trial is set to begin in early 2026.
Similarly, OKYO Pharma’s urcosimod showed remarkable efficacy in neuropathic corneal pain, with 75% of patients achieving over 80% pain reduction. With fast-track designation and expanded access plans, OKYO is pioneering treatment in an underserved indication.
Autoimmune and inflammatory disorders: Expanding options
Takeda’s zasocitinib (TAK-279) demonstrated robust efficacy in psoriatic arthritis, while mezagitamab (TAK-079) showed rapid and durable platelet responses in immune thrombocytopenia. Both programmes are progressing toward regulatory engagement, underscoring Takeda’s leadership in immunology.
UCB’s Bimzelx (bimekizumab) continued to impress in hidradenitis suppurativa, with three-year data showing sustained disease control and quality-of-life improvements. The company also reported promising Phase 1/2a results for galvokimig in atopic dermatitis, a multi-specific antibody targeting IL-13, IL-17A, and IL-17F.
Precision psychiatry and regenerative medicine: A paradigm shift
HMNC Brain Health’s OLIVE trial marked a breakthrough in precision psychiatry. BH-200 (nelivaptan), a vasopressin V1b antagonist, showed rapid and significant antidepressant effects in genetically defined subgroups. This trial validates biomarker-guided treatment in major depressive disorder and sets the stage for Phase 3 development.
Meanwhile, Resolution Therapeutics dosed its first patient in a regenerative macrophage therapy trial for end-stage liver disease. RTX001, engineered with IL-10 and MMP-9 mRNA, represents a novel approach to reversing fibrosis and restoring liver function.
Oncology: Targeted therapies and immunomodulation
Genmab and AbbVie’s epcoritamab combination therapy met dual primary endpoints in follicular lymphoma, with FDA priority review underway. Elsewhere, Transgene’s individualised neoantigen vaccine TG4050 showed 100% disease-free survival at two years in head and neck cancer, while Regeneron’s linvoseltamab achieved 100% MRD negativity in high-risk smoldering multiple myeloma.
In solid tumours, Nxera Pharma and Cancer Research UK initiated a Phase 2a trial of HTL0039732, an EP4 antagonist, showing early efficacy in combination with atezolizumab. And Citryll launched a Phase IIa trial for CIT-013 in rheumatoid arthritis, targeting neutrophil extracellular traps – a novel inflammatory pathway.
Rare diseases and paediatric innovation
CONNECTA Therapeutics received EIC Accelerator funding to advance CTH120, a neuroplasticity modulator, into paediatric trials for Fragile X Syndrome. With strong Phase I safety data and a scalable platform, CONNECTA is positioned to address multiple neurodevelopmental disorders.
Meanwhile, Bioxodes reported positive Phase 2a data for BIOX-101 in intracerebral haemorrhage, showing reduced haematoma volume and improved recovery trends. With orphan drug designation and plans for a Phase 2b trial, BIOX-101 could become a first-in-class therapy for stroke.
A quarter of clinical momentum
Overall, Q3 2025 has been defined by clinical progress, strategic funding, and regulatory engagement across a diverse array of therapeutic areas. From Alzheimer’s and depression to cancer and rare diseases, the biopharma sector continues to push boundaries with precision, innovation, and patient-centricity. As these programmes advance into pivotal trials and regulatory review, the promise of transformative therapies is closer than ever.
pharmaphorum invites companies to share their clinical trial press releases and updates for future round-ups. Please send submissions to editorial@pharmaphorum.com
