Clinical Trials Round-Up: July/August 2026

The summer months are often quieter for clinical trial reporting than the spring conference season, but while the volume of announcements hit a mid-year slump, July and August still produced an exciting array of updates across the drug development arena.

Unsurprisingly, GLP-1s and obesity research accounted for many pharmaphorum headlines in these months; most notably, the release of positive top-line data for Eli Lilly’s retatrutide, which boosted the company’s plan to file for US approval in early 2027, and the launch of a major trial to explore the benefits of GLP-1 medicines in treating dementia.

Outside of the GLP-1 space, other highlights included Sanofi’s decision to drop multiple R&D programmes, including anti-IL33 antibody itepekimab and TNF inhibitor balinatunfib, after disappointing trial readouts, and an historic phase 3 win for MSD and Moderna’s mRNA-based skin cancer vaccine.

But, while we aim to cover as many of these stories as possible, not every update that hits our inbox can become a standalone news piece. So, to highlight prominent developments, we have compiled this round-up of clinical trials developments to bring you all the updates that we missed in our daily reporting.

Oncology: Precision medicine keeps broadening its reach

As was to be expected in the wake of big-name conferences like ASCO, oncology dominated clinical trial news during July and August. The stories were diverse, highlighting how developers are trying to make treatment more selective by targeting a specific mutation, directing radiation to tumour deposits, exploiting tumour-associated proteins, or changing where chemotherapy is delivered.

One of the clearest examples came from ITM's Phase 3 COMPETE trial in early July, which directly compared the radiopharmaceutical Lu-edotreotide (ITM-11) with everolimus in patients with advanced, progressive, somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumours. Results published in The Lancet showed a substantial improvement in median progression-free survival – 23.9 months with Lu-edotreotide versus 14.1 months with everolimus – as well as objective response rates (22% compared with 4%).

Radiopharmaceutical development was also on the menu for Oncoinvent, which published Phase 1 dosimetry results in August for Radspherin, an intraperitoneal alpha-emitting therapy being developed for peritoneal metastases from ovarian and colorectal cancer. During the study, radiation exposure to normal organs, including the kidneys and red bone marrow, remained below levels associated with toxicity risk in the nine patients included in the dosimetry cohorts. The company is now continuing development in a randomised Phase 2 ovarian cancer study.

Elsewhere, Arcus Biosciences reported new research in Nature linking the activity of its investigational HIF-2α inhibitor casdatifan with suppression of serum erythropoietin and tumour biology in heavily pretreated clear cell renal cell carcinoma. Across four monotherapy cohorts involving 121 patients, median progression-free survival was 12.2 months and the confirmed response rate was 31%. The Phase 3 PEAK-1 study is now evaluating casdatifan in combination with cabozantinib in patients who have previously received immunotherapy.

Brain cancer offered another example of a targeted programme gaining momentum. In July, Nuvation Bio reported a 36-month progression-free survival rate of 79.1% from its Phase 2 J201 study of safusidenib in chemotherapy- and radiotherapy-naïve patients with grade 2 IDH1-mutant glioma. The results prompted an expansion of the programme into two further settings: a Phase 3 study in newly diagnosed grade 2 IDH1-mutant glioma outside the US and a Phase 2 study for patients whose disease has progressed following treatment with vorasidenib.

How to combat treatment resistance was also the driving question behind a new Phase 1/2a clinical trial from Cancer Research UK and KisoJi Biotechnology, which treated its first patient with KJ-103, a naked anti-TROP2 antibody being studied in selected TROP2-expressing solid tumours. Unlike TROP2 antibody-drug conjugates, KJ-103 carries no cytotoxic payload. Instead, researchers explained, the candidate is designed to recruit immune cells to target and kill tumour cells. The study will evaluate the antibody both as monotherapy and alongside pembrolizumab in head and neck cancer.

Also in August, RenovoRx completed enrolment in the Phase 3 TIGeR-PaC study of intra-arterial gemcitabine delivered through its RenovoCath device in locally advanced pancreatic cancer. The trial is designed to compare targeted delivery with systemic gemcitabine plus nab-paclitaxel, with overall survival as the primary endpoint. Of the 86 required events, 78 had occurred by 11 August, with topline results expected in the second half of 2027.

Cardiovascular and metabolic medicine

Perhaps the most impactful cardiovascular announcement came from Cytokinetics, which reported positive Phase 3 results from ACACIA-HCM in symptomatic non-obstructive hypertrophic cardiomyopathy in August.

Results showed that the trial met both of its dual primary endpoints, with aficamten producing statistically significant improvements in patient-reported health status and maximal exercise capacity compared with placebo at 36 weeks. The least-squares mean improvement in KCCQ Clinical Summary Score was 11.4 points with aficamten versus 8.4 with placebo, while pVO2 increased by 0.64 ml/kg/min compared with a 0.03 ml/kg/min decline in the placebo group.

There were safety considerations, however, particularly reductions in left ventricular ejection fraction (LVEF). LVEF below 50% occurred in 10.5% of patients receiving aficamten compared with 0.8% on placebo, although, most affected patients were able to complete treatment on the same or a lower dose.

The cardiovascular story was not limited to HCM. In kidney disease, R1 Therapeutics kicked off a global Phase 2b study of AP306, a first-in-class inhibitor of three active phosphate transporters, in patients receiving maintenance dialysis. The multi-centre, randomised, double-blind, placebo-controlled study will enrol approximately 168 patients, across six fixed-dose AP306 regimens and placebo, over an eight-week treatment period.

Immunology and blood disorders: Treatment is becoming more durable

In July, Takeda reported Phase 2 patient-reported outcomes for mezagitamab in primary immune thrombocytopenia. Patients receiving the 600 mg dose reported clinically meaningful improvements in symptoms and physical fatigue by Week 16, alongside improvements across measures of physical health, social activity, psychological health, and overall quality of life. Notably, improvements in symptoms and physical fatigue were maintained through Week 32, 24 weeks after the final dose.

In von Willebrand disease (VWD), Incyte presented multidose Phase 1/2 data for VGA039 (latarcibart), a monoclonal antibody targeting Protein S, a vital component needed to support blood clotting. Across 16 patients, the company reported substantial reductions in median annualised bleeding rate (81%) across different types of the inherited disorder and bleed categories, with 70% of patients with prior von Willebrand factor-treated bleeds not experiencing a VWF-treated breakthrough bleed while on treatment. The therapy is already in Phase 3 development and, if successful, could offer a once-monthly subcutaneous prophylactic alternative to the frequent intravenous infusions used with replacement factor concentrates.

Elsewhere, Oblenio began dosing patients in a Phase 1a trial of LBL-051, a trispecific T-cell engager targeting BCMA, CD19, and CD3. The aim is to deplete both B-cells and plasma cells and potentially produce a broader immune reset than approaches targeting a single B-cell population. The open-label study is enrolling patients across multiple refractory autoimmune diseases and will assess safety alongside depletion of B-cells and plasma cells and selected biomarkers.

Neuroscience

Neurology remains one of the more challenging areas of drug development, and the July/August reporting showed several attempts to approach difficult diseases through increasingly specific mechanisms.

In August, AC Immune reported preliminary Phase 1 data for ACI-19764, an oral NLRP3 inflammasome inhibitor designed to reduce chronic inflammation, which is commonly linked to disease progression across multiple metabolic and neurological conditions. Study findings showed that ACI-19764 was generally well tolerated among participants, with no serious adverse events or treatment withdrawals. Moreover, cerebrospinal fluid measurements confirmed brain penetration, while doses of 10 mg or less achieved concentrations above the reported IC90.

The programme is now moving beyond healthy volunteers, with a Phase 1b cohort enrolling people at cardiovascular risk associated with elevated hsCRP and type 2 diabetes and/or obesity.

NRG Therapeutics is pursuing a different route into neurodegeneration treatment. At the ALS Association’s ALS Nexus conference, the company presented mechanistic and preclinical findings for NRG5051, an orally bioavailable, CNS-penetrant inhibitor of the mitochondrial permeability transition pore being developed for ALS and Parkinson's disease. The work identified NLRX1 as a key regulator of mPTP opening and showed anti-inflammatory and neuroprotective effects in ALS mouse models, including reductions in plasma neurofilament light, a marker of neuronal damage.

Dermatology and paediatric medicine

Alongside progress in life-saving treatments, quality of life was also championed through developments in July/August. One such announcement was Takeda’s additional Phase 3 data for the oral TYK2 inhibitor zasocitinib in plaque psoriasis, which demonstrated high rates of skin clearance at difficult-to-treat sites.

Around 75% of patients with scalp psoriasis achieved clear or almost clear skin at Week 16, as did approximately 70% of patients with palmoplantar disease. Nail disease also improved, with statistically significant changes in Nail Psoriasis Severity Index compared with placebo. These results built on the broader Phase 3 programme, in which around 70% of patients achieved clear or almost clear skin at Week 16.

July also brought results that could have a much more immediate effect on clinical practice globally. The PediCAP trial, involving 1,101 children across 13 hospitals in South Africa, Uganda, Zambia, Zimbabwe, and Mozambique, found that children hospitalised with severe community-acquired pneumonia could safely switch from injectable to oral antibiotics once they had begun to recover.

Readmission or death within 28 days occurred in 6% of children receiving oral amoxicillin, 7% receiving oral amoxicillin-clavulanate, and 6% receiving injectable antibiotics. The trial also found that four to five days of total antibiotic treatment was as effective as seven or eight days, while children switched to oral treatment left hospital around a day earlier.

What stood out: 5 themes from July/August

1. Precision medicine is becoming more precise

The latest oncology programmes are increasingly being defined by molecular characteristics, biological pathways and even the location of disease, rather than simply by tumour type. HIF-2α inhibition in kidney cancer, IDH1 targeting in glioma, TROP2-directed immunotherapy, and SSTR-targeted radiopharmaceuticals all illustrate a move towards increasingly specific definitions of who should receive a treatment and why.

2. Delivery is becoming part of the therapeutic proposition

Several of the period's programmes were as much about where or how a therapy is delivered as the molecule itself. RenovoRx is testing regional chemotherapy delivery in pancreatic cancer, Radspherin is designed to deliver radiation directly into the abdominal cavity, while latarcibart and mezagitamab could reduce the frequency or burden of treatment for people with chronic bleeding disorders.

3. Durability is increasingly valuable

The safusidenib follow-up, persistent quality-of-life improvements after mezagitamab treatment, and long-term development strategies across several programmes all highlight the need for lasting benefit. As more therapies enter crowded markets, sustained responses and periods off treatment may become increasingly important differentiators.

4. Existing medicines are being challenged on how they are used

PediCAP is perhaps the clearest example, but the same principle appears elsewhere. Developers are questioning whether patients need prolonged intravenous therapy, frequent infusions, systemic exposure, or conventional treatment sequences.

5. Biology is moving closer to the centre of clinical decision-making

Casdatifan's relationship with serum EPO, ACI-19764's demonstrated CNS penetration, NRG5051's mitochondrial mechanism, and the biomarker work embedded in several early-stage programmes all show how clinical development is increasingly intertwined with translational biology. The strongest programmes are trying to establish measurable evidence that the intended biological pathway has actually been engaged.

This round-up is prepared by a human editor with assistance from AI.