The clinical trial results 2 October 2026 span hepatitis delta, wet macular degeneration, Niemann-Pick disease, COVID-19 and skin cancer.
Mirum Pharmaceuticals’ brelovitug met its primary endpoint in chronic hepatitis delta. Kodiak Sciences’ Zenkuda and tabirafusp-ted matched aflibercept in wet age-related macular degeneration. Rafael Holdings’ Trappsol Cyclo missed its primary endpoint in Niemann-Pick disease type C. Invivyd’s VYD2311 met tolerability endpoints against an mRNA COVID-19 vaccine. Highlight Therapeutics’ BO-112 met its primary endpoint in a single-arm basal cell carcinoma study. A new Wolverhampton clinical trials unit, announced on 22 September, completes the roundup. Here, Life Science Daily News brings you the week’s most significant readouts.
Brelovitug Meets Its Primary Endpoint in the Phase 2b/3 AZURE-1 Study in Chronic Hepatitis Delta
Mirum Pharmaceuticals announced on 28 September that brelovitug met the Week 24 primary endpoint in AZURE-1. The study enrolled treatment-naive adults with chronic hepatitis delta virus (HDV) infection. The primary endpoint combined virologic response with alanine aminotransferase (ALT) normalisation. It was reached by 56 per cent of patients on 300 mg weekly (n=59). The rate was 45 per cent on 900 mg every four weeks (n=65). The delayed-treatment control arm (n=29) recorded 0 per cent, with p<0.0001 for both comparisons. Virologic response alone was 86 per cent and 85 per cent in the two active arms. ALT normalisation was 63 per cent and 54 per cent.
AZURE-1 is a randomised Phase 2b/3 study that planned to enrol about 200 treatment-naive patients. Randomisation was 2:2:1, with a 24-week delayed-treatment control rather than an active comparator. Brelovitug is a fully human monoclonal antibody. It is designed to neutralise and remove hepatitis B and hepatitis D virions and to deplete HBsAg-containing subviral particles. Through Week 24, Mirum reported no new safety signals and no Grade 3 or higher adverse events. There were no discontinuations due to safety. Injection site reactions occurred in 10.2 per cent and 18.5 per cent of patients in the two active arms. Mirum expects topline results from the AZURE-4 study in the fourth quarter of 2026. It plans a Biologics License Application in the first half of 2027.
Zenkuda and Tabirafusp-Ted Meet Non-Inferiority Primary Endpoints Against Aflibercept in the Phase 3 DAYBREAK Study
Kodiak Sciences announced on 28 September results from DAYBREAK, a Phase 3 study in wet age-related macular degeneration. Zenkuda (tarcocimab tedromer) and tabirafusp-ted (KSI-501) both met the one-year primary endpoint. That endpoint was non-inferior vision gains versus aflibercept. It measured the change in best corrected visual acuity from baseline to the average of Weeks 40, 44 and 48. The p-values were 0.0007 for Zenkuda and 0.0036 for tabirafusp-ted. Kodiak reported that 54 per cent of Zenkuda patients reached 24-week dosing intervals at year one. The company described the Zenkuda profile as a potential new standard of care, a characterisation that is its own. Tabirafusp-ted also met an anatomical key secondary endpoint (p<0.0001).
DAYBREAK (NCT06556368) is a non-inferiority study. It used treat-to-dryness retreatment criteria intended to mirror real-world practice. Both investigational drugs began with four monthly loading doses. Zenkuda was then dosed every 4 to 24 weeks on an individualised basis. Tabirafusp-ted was given every 8 weeks, with up to monthly as-needed dosing. Aflibercept was dosed per its label. Intraocular inflammation was 0 per cent with Zenkuda and 0.4 per cent with tabirafusp-ted. Cataract adverse events were 0.5 per cent and 0 per cent respectively, against 0.9 per cent with aflibercept. Kodiak plans a multi-indication Biologics License Application for Zenkuda in the fourth quarter of 2026. It will draw on five Phase 3 studies in three indications. These are wet age-related macular degeneration, diabetic retinopathy and macular oedema after retinal vein occlusion. For tabirafusp-ted, post-hoc analyses will look for patient subgroups that benefit from IL-6 inhibition. The ALTO Phase 3 study in diabetic macular oedema is enrolling.
Trappsol Cyclo Misses Its Primary Endpoint in the Phase 3 TransportNPC Study in Niemann-Pick Disease Type C
Rafael Holdings announced on 30 September that intravenous Trappsol Cyclo did not reach statistical significance on its primary endpoint. That endpoint was the change in the 4-domain NPC Clinical Severity Scale at Week 96. The least-squares mean change was 0.46 points with Trappsol Cyclo and 1.28 points with placebo. The difference was -0.81 points (95 per cent CI -2.04 to 0.41; p=0.19). The company described this as a 64 per cent slowing of disease progression. A prespecified subgroup of 78 patients received background miglustat and/or leucine, about 83 per cent of the study population. In that subgroup, the figures were 0.46 points against 1.57 points. The difference was -1.11 points (95 per cent CI -2.19 to -0.02; p=0.046). The company described this as 71 per cent slowing. That subgroup result sits beneath a missed primary analysis and should be read as supportive at best.
TransportNPC (CTD-TCNPC-301, NCT04860960) randomised 94 patients aged 3 to 70 years over 96 weeks. Of these, 63 received Trappsol Cyclo at 2000 mg/kg every two weeks and 31 received placebo. Trappsol Cyclo is intravenous hydroxypropyl-beta-cyclodextrin. Niemann-Pick disease type C is a rare, progressive and fatal genetic disorder. Serious treatment-emergent adverse events occurred in 35.9 per cent of treated patients and 16.7 per cent of placebo patients. Treatment-related serious events were similar, at 3.1 per cent and 3.3 per cent. Hearing-related events were 15.6 per cent against 13.3 per cent. Rafael said it remains on track to submit a New Drug Application in the fourth quarter of 2026. It also cited a separate survival analysis in infantile-onset disease against external registry and historical controls. That analysis reported 85 per cent and 94 per cent reductions in mortality risk. It is not a randomised comparison. The release does not report how the FDA views the package.
VYD2311 Meets Tolerability Endpoints Against an mRNA COVID-19 Vaccine in the Phase 3 LIBERTY Study
Invivyd announced on 29 September positive topline results from LIBERTY. The study compared the safety and tolerability of the COVID-19 monoclonal antibody VYD2311 with the mRNA vaccine COMIRNATY. Over six days, 56.5 per cent of participants given VYD2311 alone reported adverse events, injection site reactions or hypersensitivity. The figure was 91.4 per cent with the vaccine (p<0.0001). Systemic adverse events were 44.1 per cent against 68.1 per cent (p=0.008). In the 56-day secondary analysis, the rates were 60.9 per cent and 91.4 per cent (p<0.0001). VYD2311 given together with the vaccine raised neutralising titres about 2.5-fold compared with the vaccine alone.
LIBERTY is a randomised, double-blind, active-controlled Phase 3 study in 210 healthy adults aged 18 to 49. Participants were allocated 1:1:1 to 250 mg VYD2311, the vaccine, or both. The company reported no VYD2311-related adverse events above Grade 2, and no hypersensitivity or anaphylaxis. An antibody provides passive protection while a vaccine prompts an immune response, so the comparison is not like-for-like. The study measured safety and immune response rather than protection against infection. Invivyd plans a Biologics License Application through the FDA Accelerated Approval Program. That plan depends on supportive data from the separate DECLARATION study. DECLARATION safety and immunogenicity topline data are expected in October 2026. Efficacy data are planned to be unblinded after Accelerated Approval, if granted, subject to clinical event accrual. Whether the agency will accept the package is not yet known.
BO-112 Meets Its Primary Endpoint in the Single-Arm Phase 2b SPOTLIGHT-204 Study in Basal Cell Carcinoma
Highlight Therapeutics announced on 30 September results from SPOTLIGHT-204. The data were presented as a late-breaker at the European Academy of Dermatology and Venereology Congress in Vienna. The primary endpoint was visual and pathological response at Week 24, assessed by a central review committee. It was achieved by 61 per cent of patients (28 of 46). Among high-risk head and neck patients, 56 per cent (18 of 32) had a complete clinical and pathological response. The figure was 62 per cent (16 of 26) in patients with facial lesions.
SPOTLIGHT-204 (NCT06422936) is a multicentre, single-arm study in patients with localised primary basal cell carcinoma. It enrolled 50 patients, 40 high-risk and 10 low-risk, with a median age of 75 years. The modified intention-to-treat population comprised 46 patients with 54 evaluable lesions. Patients received three once-weekly intralesional BO-112 injections, followed by surgical excision at Week 24. BO-112 is a double-stranded RNA nanoparticle thought to act by activating innate antiviral sensing pathways. Highlight reported no serious adverse events, and 91 per cent of treatment-related adverse events were Grade 1. With no control arm, the study cannot show benefit over surgery alone. Highlight says the results support late-stage development.
University of Wolverhampton and The Royal Wolverhampton NHS Trust Launch a Joint Clinical Trials Unit
The University of Wolverhampton and The Royal Wolverhampton NHS Trust announced on 22 September a joint Clinical Trials Unit. It followed a launch event held on 16 September. The joint venture is intended to provide the infrastructure to design and deliver trials. It also aims to expand regional research capacity and improve trial access for underserved populations. No funding figure was given.
The unit will serve a catchment of about 1.5 million people across four acute trusts. The partners are targeting UKCRC registration by 2030. The announcement names Professor Prashant Pillai, Pro Vice-Chancellor for Research and Knowledge Exchange at the University of Wolverhampton. It also names Professor Tonny Veenith, Consultant in Critical Care Medicine at The Royal Wolverhampton NHS Trust. He is also a Professor at the university. This is infrastructure news rather than a trial readout. UKCRC registration is a stated target for the end of the decade, not an achieved status.
Looking Ahead
This week’s clinical trial results 2 October 2026 are uneven in what they establish. The hepatitis delta result is the firmest, a controlled readout with a clear primary endpoint. Its comparator was delayed treatment, however, and longer-term durability is still to come. The retinal data show non-inferiority to an established drug at one year. The durability claims rest on the company’s own treat-to-dryness framework.
The Niemann-Pick study did not meet its primary endpoint. The prespecified subgroup and external-control survival analyses cannot substitute for it. The COVID-19 antibody study speaks to tolerability rather than protection, and its regulatory path depends on DECLARATION. The basal cell carcinoma result comes from a single-arm study that supports further testing, not a conclusion on efficacy. The Wolverhampton unit adds capacity rather than data. Life Science Daily News will continue to cover the readouts that matter across the global life sciences pipeline. Read the 25 September 2026 clinical trials roundup.
