Several late-stage programs are reaching key milestones in 2026, spanning Alzheimer’s disease, ALS and emerging gene-editing approaches in cardiovascular health.
In this Xtalks blog, we review five such clinical trials. The trials below were selected for their defined timelines, regulatory steps and potential to clarify whether new mechanisms can translate into meaningful clinical benefit.
POLARIS-AD (AriBio & SK chemicals)
POLARIS-AD is a global Phase III trial of AR1001 (mirodenafil), a once-daily oral therapy being studied for early Alzheimer’s disease. Developed by SK chemicals and licensed to AriBio, AR1001 is intended to act on several disease processes, including protecting neurons, supporting neurogenesis and improving cerebral blood flow. The study has enrolled 1,535 participants across 13 countries and is expected to be completed in early 2026, with topline results anticipated ahead of a potential FDA filing.
Buntanetap Phase III in Early Alzheimer’s Disease (Annovis)
Annovis is conducting a Phase III study of buntanetap in early Alzheimer’s disease, enrolling participants with biomarker-confirmed amyloid buildup. All 84 sites are active, and the first cohort has completed the six-month treatment period. The study includes two planned readouts: a six-month symptomatic analysis expected in the second half of 2026, followed by an 18-month assessment of whether the drug may slow disease progression. The FDA has agreed to the study design, potentially supporting New Drug Applications (NDAs) for both symptomatic and disease-modifying indications.
ADEPT-2: Cobenfy for Psychosis in Alzheimer’s Disease (Bristol Myers Squibb)
ADEPT-2 is a Phase III trial evaluating Cobenfy (xanomeline/trospium) for hallucinations and delusions associated with Alzheimer’s disease. The study uses a clinician-rated psychosis scale as the primary endpoint, with a global severity measure as a key secondary assessment. After irregularities were identified at a small number of sites, the affected data were removed, and an independent review recommended enrolling additional patients to maintain statistical power. Bristol Myers Squibb remains blinded, with results from ADEPT-2 and related studies expected by the end of 2026.
CTX310: One-Time Gene Editing for Severe Dyslipidemia (CRISPR Therapeutics)
CTX310 is an in vivo CRISPR/Cas9 therapy delivered as a single IV infusion to edit the ANGPTL3 gene, which helps regulate cholesterol and triglycerides. Phase I data presented at the American Heart Association (AHA) 2025 meeting and published in The New England Journal of Medicine (NEJM) showed deep, dose-dependent reductions in ANGPTL3 (up to 89%) with corresponding decreases in triglycerides and LDL cholesterol, including in patients already on standard lipid-lowering therapy. CRISPR Therapeutics plans to advance CTX310 into Phase Ib studies in 2026 to investigate whether one-time gene editing can address major lipid drivers of cardiovascular risk.
CNM-Au8: Biomarker-Driven Accelerated-Approval Bid in ALS (Clene Nanomedicine)
CNM-Au8 is an investigational therapy for ALS supported by FDA-recommended analyses showing significant reductions in two biomarkers of nerve injury and inflammation: neurofilament light (NfL) and glial fibrillary acidic protein (GFAP). Updated analyses from the HEALEY ALS Platform Trial suggest a notable reduction in the risk of death in reviewed populations, alongside a favorable safety profile across more than 1,000 patient-years of exposure. Clene has requested a Type C meeting in early 2026 and plans to file an accelerated approval NDA shortly after.
Setting the Stage for What Comes Next
Though not every program will read out in 2026, several are shaping the late-decade landscape as they progress toward pivotal development.
Gotistobart (PRESERVE-003) in Squamous NSCLC (BioNTech & OncoC4)
Gotistobart is a tumor-selective CTLA-4 antibody designed to deplete regulatory T cells in the tumor microenvironment while sparing healthy tissue. The non-pivotal stage of its Phase III program is complete, and the pivotal stage is now enrolling globally. In 2026, the focus will shift to recruitment progress and updates ahead of an eventual overall-survival readout.
Neflamapimod in Dementia with Lewy Bodies (CervoMed)
Neflamapimod is an oral p38 MAPK inhibitor in development for dementia with Lewy bodies, which currently lacks disease-modifying treatments. Recent Phase IIb RewinD-LB trial findings support further study, and a global Phase III trial is scheduled to begin in the second half of 2026. Its design is expected to guide future approaches to biomarker-informed therapy development in dementia with Lewy bodies.
ELEVATE-PD: Crexont in Parkinson’s Disease (Amneal Pharma)
ELEVATE-PD is a Phase IV study evaluating Crexont, an extended-release formulation of carbidopa/levodopa, in individuals with moderate to advanced Parkinson’s disease. Interim findings from the first cohort show improved motor control and more consistent “Good On” time after switching from several prior oral levodopa regimens. Full 13-month results are expected in 2026.
Alongside these programs, 2026 will bring the launch of new pivotal trials and the continuation of large cardiovascular and oncology studies, even as some late-stage efforts discontinued in 2025 remind us that not every program reaches its goals.
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