Clinical Trials Round-Up – Jan to Feb 2026

Welcome to the first instalment of the pharmaphorum Clinical Trials Round-Up for 2026.

While we might have covered such data releases as GSK's antisense-based drug for chronic hepatitis B (CHB), bepirovirsen, hitting the mark in two phase 3 trials; or Hutchmed preparing to file for approval of a drug for the rare autoimmune disorder warm antibody autoimmune haemolytic anaemia (wAIHA) in China, after chalking up a win in a pivotal phase 2/3 trial; or even a hole being blown in Bausch Health's late-stage pipeline, after a follow-up to its top-selling drug product Xifaxan failed not just one but two phase 3 trials – it is the purpose of these bimonthly round-ups to report on earlier stage trials, in the main.

So, while Sanofi reported positive phase 3 results with venglustat in Gaucher disease, setting up regulatory filings; while Roche revealed the data behind a positive phase 3 trial for its oral BTK inhibitor fenebrutinib in primary progressive multiple sclerosis (PPMS); and while a study of Roche's Gazyva has also raised the prospect of a first approved therapy for rare autoimmune kidney disorder primary membranous nephropathy (pMN) – in this edition, read how the first two months of the year offered a revealing cross-section of how R&D is evolving across therapy areas, from rhythm control in atrial fibrillation to precision vaccines and AI‑enabled endpoints.

Cardiometabolic signals

Acesion Pharma opened the year by dosing the first patients in its Phase 2 trial of AP31969, an oral small‑molecule inhibitor of the SK ion channel for rhythm control in atrial fibrillation (AF). Two hundred patients across eight European countries will be followed with implantable loop recorders to quantify AF burden continuously and provide a rigorous efficacy readout – while directly surveilling for ventricular proarrhythmia, the class‑defining risk that has limited older antiarrhythmics.

The Phase 1 in 92 healthy volunteers was reassuring, with pharmacokinetics consistent with chronic dosing and clinically relevant QTc effects ruled out. If the signal seen preclinically for atrial selectivity holds in patients, AP31969 could re‑set expectations for safety in chronic rhythm control, a space with little drug innovation for two decades.

In metabolic disease, meanwhile, Arrowhead Therapeutics shared interim Phase 1/2a data for two RNAi agents that aim to change what weight is lost. ARO‑INHBE targets hepatically derived activin E and the study showed a reduction in visceral fat by ~10% after a single dose and ~16% after two doses, and, in a small cohort with type 2 diabetes on tirzepatide, roughly doubled weight loss by week 16 and tripled fat loss by week 12 versus tirzepatide alone.

ARO‑ALK7, aimed at adipocyte ALK7, produced a ~14% visceral fat reduction at eight weeks, and early safety was favourable. The concept is straightforward, but important: pairing incretin‑based appetite suppression with body‑composition-targeted RNAi to preserve lean mass and disproportionately deplete cardiometabolically harmful fat. Confirmation in larger cohorts will be needed, but the mechanistic complementarity to GLP‑1/GIP therapy is compelling.

Speaking of which, another mechanistic play – in cardiac energetics – arrived from Imbria’s Phase 2a IMPROVE‑DiCE trial. Ninerafaxstat improved myocardial phosphocreatine‑to‑ATP ratio, reduced myocardial triglyceride content by a third, and increased six‑minute walk distance in people with type 2 diabetes and obesity, with or without symptomatic HFpEF. Direct, in‑human evidence of energetic improvement in HFpEF is rare and, while the sample was small, the data supports the viability of metabolic re‑programming as an adjunct to haemodynamic and diuretic strategies.

Immunology and dermatology

For AstraZeneca, subcutaneous (SC) self‑administration of anifrolumab (Saphnelo) delivered full Phase 3 results in SLE that mirrored its IV performance: 56.2% achieved BICLA response at 52 weeks vs 37.1% on placebo. DORIS remission was 29.0% vs 14.7%, and attainment of LLDAS was 40.1% vs 26.0%, with a safety profile consistent with IV. With SC already approved in the EU, the practical upshot is broader access and reduced infusion burden at a time when guidelines increasingly emphasise earlier biologic use to drive remission and taper steroids.

In medical dermatology, Almirall unveiled LumiNE, a Phase III programme taking lebrikizumab – an IL‑13 inhibitor already established in atopic dermatitis (AD) – into nummular eczema, an often misdiagnosed condition with limited options beyond topicals. The study will randomise at least 270 adults across Europe for up to 48 weeks, with IGA‑NE as the primary endpoint and pruritus and DLQI as key secondaries. Mechanistically, the bet is that the centrality of IL‑13 extends beyond AD into nummular eczema’s pathophysiology – an archetype of lifecycle expansion grounded in shared type‑2 biology.

PoolbegPharma, meanwhile, advanced another immunology theme from a different angle: prevention of cytokine release syndrome (CRS). A peer‑reviewed LPS human challenge study showed that oral POLB 001 (a p38 MAPK inhibitor) significantly suppressed circulating TNF, IL‑6, and IL‑8 and curtailed recruitment of key immune cell subsets, with favourable tolerability. With interim data from the TOPICAL trial due this summer, the clinical question is whether calibrated p38 inhibition can dial down CRS without blunting antitumour immunity – potentially shifting administration of immunotherapy from specialist centres to broader settings.

And Zenas BioPharma reported that obexelimab met the primary and all key secondary endpoints in INDIGO – the largest registrational trial to date in IgG4‑related disease – with plans to file a BLA in the US in Q2 2026 and an EMA submission to follow. Given the chronic, relapsing nature of IgG4‑RD and the need for steroid‑sparing agents, an at‑home SC therapy with a clean safety profile would be notable for long‑term management.

Oncology: Precision, persistence, and platform plays

Adjuvant vaccination and TME rewiring

A preprint analysis from Transgene’s Phase I portion of the TG4050 programme in HPV‑negative head and neck cancer reported 100% 2‑year disease‑free survival when the AI‑selected, MVA‑based personalised vaccine was used as adjuvant monotherapy, along with durable polyepitopic CD8+ T‑cell responses in most evaluable patients and good tolerability. The dataset is early, non‑comparative, and awaiting peer review, but it reinforces how individualised neoantigen vaccination may fit where tumour burden is minimal and immunosurveillance is decisive. Phase II readouts are expected in H2 2026.

Bicara’s bifunctional EGFR–TGF‑β antibody, ficerafusp alfa, progressed rapidly, with a 1,500 mg weekly dose selected – earlier than anticipated – for the Phase 3 FORTIFI‑HN01 trial in first‑line HPV‑negative R/M HNSCC in combination with pembrolizumab. Prior expansion‑cohort data suggested more than a doubling of median overall survival (OS) versus standard care in this biology‑defined subgroup. The company will push for substantial enrolment in 2026 to enable a mid‑2027 interim analysis and is preparing for commercial scale‑up, while probing signals in colorectal cancer – a classical example of a ‘pipeline‑in‑a‑product’ strategy tied to EGFR expression and TGF‑β‑driven immune exclusion.

Oncolytic, viral, and dsRNA approaches

Genelux reported encouraging interim data from systemically administered Olvi‑Vec combinations in relapsed small cell lung cancer (SCLC) (ORR 33%; two high‑dose partial responses with ~55% and ~85% tumour shrinkage) and signs of disease control in non-small cell lung cancer (NSCLC), with acceptable tolerability. The studies are dose‑finding and small, but they test whether Olvi‑Vec’s intraperitoneal ‘primer’ concept in ovarian cancer can translate to intravenous delivery and other solid tumours. Topline Phase 3 ovarian data is expected in H2 2026.

Meanwhile, Oncolytics Biotech, pursuing a dsRNA immunotherapy (pelareorep) with atezolizumab in metastatic anal cancer, reported a 29% ORR in third‑line disease – nearly triple historical benchmarks – with a median response duration of 17 months, and a 30% ORR in second‑line disease. A Type C FDA meeting is planned to discuss a potential accelerated pathway. In an ultra‑orphan setting with no approved third‑line standards, durability of response will be the fulcrum for regulatory dialogue.

KAHR Bio presented Phase 2a results for DSP107 – a bispecific that anchors to tumour CD47 while delivering 4‑1BB co-stimulation – in late‑line, chemo‑refractory MSS colorectal cancer, showing a 17.5‑month median OS, exceeding outcomes reported for current options in this setting, alongside favourable tolerability even in patients with liver metastases. A randomised Phase 2b against fruquintinib is underway, with interim data expected late 2026. The mechanism – leveraging chemotherapy‑upregulated CD47 in liver mets to focus T‑cell co‑stimulation where immune evasion is greatest – is elegant and controlled comparison will determine how much of the survival signal is drug‑related versus selection.

Radiosensitisation returns

KORTUC’s intratumoural hydrogen peroxide-hyaluronate gel resurfaced as a practical way to counter hypoxia‑driven radioresistance. A Phase 1 in breast cancer found the approach feasible and well tolerated with external‑beam RT and a randomised Phase 2 is ongoing. While small and early, the idea that the biology around the beam – not just the physics of delivery – can be controlled could have unusually broad impact, given radiotherapy’s ubiquity.

Pancreatic optimism, carefully tempered

Immuneering’s MEK inhibitor, atebimetinib, paired with mGnP reported 64% 12‑month OS in first‑line pancreatic cancer – nearly double a ~35% historical benchmark – with a tolerability profile emphasising durability over maximum shrinkage. A global Phase 3 is planned for mid‑2026. Cross‑study comparisons are fraught and selection effects possible, but the combination’s quality‑of‑life‑first philosophy is resonant in a disease where treatment persistence matters.

Radiopharmaceuticals and surgical oncology

Oncoinvent expanded recruitment in its randomised Phase 2 trial of Radspherin – intraperitoneal radium‑224 microparticles administered after cytoreductive surgery for ovarian cancer peritoneal metastases – opening four new sites and enrolling the first patient at a newly activated centre. Safety from earlier cohorts at the recommended dose (7 MBq) was favourable. If the strategy of single‑dose local alpha emission can dent recurrence where microscopic disease remains, it would avoid the complexities of targeted biologics and broaden access to effective post‑operative control.

Rare disease and ophthalmology

AAVantgarde Bio completed enrolment of LUCE‑1, a Phase 1/2 study of AAVB‑081 for Usher 1B syndrome, administering a single subretinal dose to 15 participants to assess safety and preliminary efficacy. Across inherited retinal diseases, 2026 is shaping up as a year of careful dose‑refinement and functional endpoint negotiation and LUCE‑1 adds to that mosaic.

Atsena Therapeutics finished dosing across adult and paediatric cohorts in Part B of its Lighthouse trial of ATSN‑201 for X‑linked retinoschisis and plans to initiate the pivotal Part C in Q1 2026. The programme will be watched for its handling of structural and functional measures in heterogenous presentations – and for its surgical learnings as subretinal delivery broadens.

Respiratory: AI‑defined endpoints in familial pulmonary fibrosis risk

Boehringer Ingelheim’s DROP‑FPF study – a Phase 3b prevention‑minded trial of nerandomilast (Jascayd) in people with interstitial lung abnormalities and a family history of pulmonary fibrosis – broke methodological ground by adopting Brainomix’s automated, quantitative HRCT biomarkers as a co‑primary endpoint. In interstitial lung disease, where early functional changes are subtle and noisy, sensitive imaging metrics may accelerate signal detection and shrink sample sizes. The study will test a clinically important idea – intervene before irreversible fibrosis accrues – with tools purpose‑built to see it.

Digital diagnostics and precision psychiatry

Two technology‑enabled stories emphasised measurement as much as treatment. Orlucent’s pivotal trial of a handheld skin fluorescence imaging system for on‑skin assessment of suspicious moles – published in JAAD International – validated a non‑invasive method that improved classification of difficult lesions. The clinical implication is fewer unnecessary biopsies while catching biologically aggressive melanomas earlier.

In neuropsychiatry, Alto Neuroscience presented multiple datasets supporting EEG‑based biomarkers as objective endpoints and stratifiers. Theta inter‑trial coherence replicated as a robust marker of cognitive impairment associated with schizophrenia, and a biomarker predicting placebo and treatment response in major depression improved effect‑size detection across trials. If these markers continue to perform prospectively, they could reduce the signal risk that has dogged psychiatry development and help align mechanism to patient biology.

Vaccines and anti‑infectives

Finally, Virometix reported positive Phase 1 topline data for V‑212, a serotype‑independent pneumococcal vaccine candidate that produced robust responses across three target antigens with an excellent safety profile in healthy volunteers. While yet early, a serotype‑agnostic approach could future‑proof against capsular drift and expand coverage beyond current conjugates.

Overall, the first two months of the year did not deliver a single ‘moon‑shot’ approval; rather, they surfaced a pattern of disciplined innovation: better measurement, earlier intervention, biology‑matched combinations, and practical, scalable modalities.

Editor’s note: This round‑up is based on press releases, peer‑reviewed publications, and company communications received in January and February 2026. All results are preliminary unless stated, and many programmes are early‑phase or subject to further validation in controlled studies.