Q2 clinical trials round-up: April to June 2025
The second quarter of 2025 has seen a surge of clinical trial activity across the pharmaceutical and biotech landscape, with companies advancing novel therapies in oncology, neurodegeneration, rare diseases, and metabolic disorders.
This Q2 round-up highlights key developments from early-phase breakthroughs to pivotal trial readouts, underscoring the sector’s commitment to precision medicine, immunotherapy, and gene-based innovation.
Neurodegeneration: Biomarker-driven progress
AC Immune reported promising Phase 2 data for ACI-7104.056 in early Parkinson’s disease. The active immunotherapy induced a >20-fold increase in anti-alpha-synuclein antibodies versus placebo, with strong boostability and no serious adverse events. Expansion to 150 patients is planned, with biomarker and symptom progression endpoints.
Lantheus’ MK-6240 tau PET imaging agent met co-primary endpoints in two pivotal Alzheimer’s studies. An NDA submission is expected in Q3, positioning MK-6240 as a next-generation diagnostic alongside NAV-4694 (beta-amyloid PET agent).
Oncology: Immunotherapy, ADCs, and radiopharmaceuticals
AstraZeneca and Daiichi Sankyo’s DESTINY-Breast09 trial showed ENHERTU plus pertuzumab significantly improved progression-free survival in HER2+ metastatic breast cancer versus THP. The combination is poised to redefine first-line treatment.
Nimbus Therapeutics initiated a Phase 1/2 trial of NDI-219216, a non-covalent WRN inhibitor for MSI-H tumours. Preclinical data demonstrated superior efficacy and resistance mutation coverage, supporting best-in-class potential.
Akiram Therapeutics dosed its first patient in a Phase 1 trial of 177Lu-AKIR001, a radiopharmaceutical targeting CD44v6-expressing tumours. The trial, conducted at Karolinska University Hospital, focuses on aggressive cancers including NSCLC and refractory thyroid cancer.
March Biosciences launched a Phase 2 trial of MB-105, a CD5-targeted CAR-T therapy for relapsed/refractory T-cell lymphoma. Early data showed durable remissions and a favourable safety profile, with orphan drug designation granted earlier this year.
Enterome presented Phase 1/2 data for EO4010 (OncoMimics) in MSS/pMMR colorectal cancer. The combination with nivolumab yielded a 40% 12-month survival rate and strong T-cell responses, with a survival plateau at 34%.
Cancer interception and immunoprevention
Nouscom’s NOUS-209 vaccine for Lynch syndrome demonstrated 100% neoantigen-specific T-cell response and durable immunity in a Phase Ib/II trial. The therapy is advancing to a potentially registration-enabling Phase 2/3 study.
Metabolic disorders and obesity: Weight loss and muscle preservation
Skye Bioscience reported >30% weight loss in preclinical models using nimacimab (CB1 antibody) combined with tirzepatide. Nimacimab alone matched GLP-1 agonist efficacy, with superior potency due to its allosteric mechanism. Phase 2a data are expected later this year.
Actimed Therapeutics began enrolling patients in its PROACT study, evaluating S-pindolol benzoate for preserving muscle mass during and after GLP-1 therapy. Results are anticipated in 2026.
Gene therapy and rare disease: FTD and Lowe syndrome
AviadoBio launched the ASPIRE-FTD trial of AVB-101, a one-time gene therapy for FTD-GRN, at UK sites including Cambridge and Cardiff. The therapy delivers a functional GRN gene directly to the brain via MRI-guided infusion.
Vesper Bio reached a key enrolment milestone in its Phase Ib/IIa trial of VES001, an oral sortilin inhibitor for asymptomatic GRN mutation carriers. The trial targets early intervention in frontotemporal dementia.
Fondazione Telethon and Can-Fite initiated a Phase II trial of piclidenoson in Lowe syndrome, repurposing a psoriasis drug to address kidney dysfunction in this rare genetic disorder.
Autoimmune and inflammatory diseases: Targeted immunomodulation
ILTOO Pharma published results from the MIROCALS trial inThe Lancet, showing that low-dose IL-2 significantly improved survival in ALS patients with low neurofilament levels. The company is pursuing regulatory approval for ILT-101.
Philikos began a Phase 1/2 trial of T-Guard in diffuse cutaneous systemic sclerosis. The therapy aims to reset the immune system by depleting activated T- and NK-cells, offering a less invasive alternative to stem cell transplantation.
Cardiovascular disease: Precision biomarkers in CAD
PlaqueTec’s BIOPATTERN trial revealed early evidence of protein gradients in blood samples taken upstream and downstream of coronary plaques. These findings support the development of CAD endotypes for precision medicine.
Pulmonary and haematologic innovation
Incyte presented late-breaking data on INCA033989, a first-in-class monoclonal antibody targeting mutant calreticulin (mutCALR) in essential thrombocythaemia. 86% of patients achieved haematologic response, with molecular response observed in 21%. A Phase 3 trial is planned for early 2026.
Everest Medicines unveiled Phase 1b/2a data for EVER001, a reversible BTK inhibitor in primary membranous nephropathy. The therapy showed >90% reduction in anti-PLA2R antibodies and strong proteinuria remission, with a favourable safety profile.
Ophthalmology and neuromuscular disorders
Breye Therapeutics completed a Phase 1b trial of danegaptide in diabetic retinopathy. The oral therapy was well tolerated and showed early signs of efficacy, with a Phase 2 trial planned.
Argenx announced plans to advance ARGX-119, a MuSK agonist antibody, to a registrational trial in congenital myasthenic syndromes following positive Phase 1b results.
Q2 2025 has demonstrated the pharmaceutical industry’s continued momentum in developing transformative therapies across diverse indications. From precision diagnostics and immunotherapies to gene editing and novel biologics, the clinical pipeline is rich with innovation.
pharmaphorum invites companies to share their clinical trial press releases and updates for future round-ups. Please send submissions toeditorial@pharmaphorum.com.
