Trial Watch; Mar 2026 Edition 5
U.S. Clinical Trial Announcements
Early March 2026 saw a mixture of new trial initiations, enrollment milestones, and regulatory developments across therapeutic areas. Phase 2 and Phase 3 trials moved forward in oncology, neurology, rare pulmonary diseases, infectious disease, and advanced gene‑editing modalities; highlighting continued momentum toward mechanism‑driven and disease‑modifying therapies.
Therapeutic Areas Covered
Oncology
2 trialsINSIGhT Adaptive Trial Launches New Arm: INO‑5412 + Cadonilimab for Glioblastoma
Dual checkpoint blockade plus active DNA immunization for newly diagnosed glioblastoma within the INSIGhT adaptive platform
Timeline / Sites
4 March 2026: INOVIO and Akeso announced a clinical collaboration to evaluate the combination of INO‑5412 and cadonilimab in newly diagnosed glioblastoma as part of the Phase 2 INSIGhT adaptive platform trial sponsored by Dana‑Farber Cancer Institute. The study arm will enroll patients across U.S. sites participating in the INSIGhT platform. Dosing of patients in this combination arm is expected to begin in the second half of 2026.
What's New
INO‑5412 combines DNA immunotherapy plasmids encoding tumor antigens hTERT, WT1, and PSMA. Cadonilimab is a bispecific antibody targeting PD‑1 and CTLA‑4, designed to enhance immune checkpoint blockade while stimulating anti‑tumor immune responses.
Why It Matters
Glioblastoma remains one of the most difficult cancers to treat. Combining tumor‑targeting DNA immunotherapy with dual checkpoint blockade could strengthen immune responses and potentially improve outcomes in a disease with limited treatment options.
AMPLIFY; 64Cu‑SAR‑bisPSMA PET/CT (Target Participants Achieved)
Phase 3 registrational diagnostic imaging trial for copper‑64 labelled PSMA‑targeted PET tracer
Timeline / Sites
AMPLIFY commenced in May 2025. On 10 March 2026, Clarity Pharmaceuticals announced that the Phase 3 AMPLIFY trial evaluating the 64Cu‑SAR‑bisPSMA PET imaging agent had consented in excess of the planned number of participants. Consenting of new patients has stopped pending completion of screening of already consented participants. The trial enrolled patients across sites in the United States and Australia.
What's New
64Cu‑SAR‑bisPSMA is a copper‑64 labeled PSMA‑targeted radiopharmaceutical imaging agent used to detect prostate cancer recurrence with PET imaging.
Why It Matters
Copper‑64 provides a longer half life than gallium‑68, allowing more flexible imaging schedules and potentially improving diagnostic accuracy in patients with biochemical recurrence of prostate cancer.
Neurology / Psychiatry
3 trialsVIM0423 for Isolated Dystonia
Small‑molecule modulator targeting dopamine and acetylcholine signalling to restore basal ganglia circuitry
Timeline / Sites
11 March 2026: Vima Therapeutics announced dosing of the first patient in a Phase 2 clinical trial evaluating VIM0423 for isolated dystonia in the United States. Following encouraging safety results from Phase 1 testing, the company plans to initiate a Phase 2 study in Parkinson's disease in mid‑2026. Topline data from both Phase 2 trials are anticipated in the first half of 2027.
What's New
VIM0423 is a small molecule designed to modulate dopamine and acetylcholine signaling to restore balance within basal ganglia circuits responsible for motor control.
Why It Matters
Current dystonia treatments primarily address symptoms. A therapy targeting the underlying neural circuitry could offer disease‑modifying potential.
Tazbentetol (SPG302) for Schizophrenia
First‑in‑class synaptic‑regenerative compound enhancing dendritic spine formation and synaptic connectivity
Timeline / Sites
11 March 2026: Spinogenix announced completion of enrollment in its randomized double‑blind placebo‑controlled Phase 2 trial of tazbentetol for schizophrenia. The study enrolled 32 patients and is designed to evaluate improvements in cognitive and negative symptoms of schizophrenia. Interim results are expected to be shared at SIRS in late March 2026.
What's New
Tazbentetol is a synaptic regenerative therapy designed to promote dendritic spine formation and restore synaptic connectivity in the brain.
Why It Matters
Schizophrenia treatments currently focus mainly on controlling psychotic symptoms. A therapy targeting synaptic repair could address cognitive deficits and negative symptoms that remain difficult to treat.
Z‑basivarsen (DYNE‑101) for Myotonic Dystrophy Type 1; HARMONIA
Antisense oligonucleotide designed to reduce toxic DMPK RNA transcripts responsible for myotonic dystrophy
Timeline / Sites
8 March 2026: Dyne Therapeutics announced initiation of the Phase 3 HARMONIA trial evaluating z‑basivarsen for myotonic dystrophy type 1. The randomized double‑blind placebo‑controlled study is expected to enroll approximately 150 adults across a 48‑week blinded treatment period. The primary endpoint will measure change in the five times sit to stand test at Week 84. First sites are activated and open to enrollment.
What's New
Z‑basivarsen is an antisense oligonucleotide designed to reduce toxic DMPK RNA transcripts responsible for myotonic dystrophy.
Why It Matters
This program represents one of the first late‑stage attempts to modify disease progression in myotonic dystrophy type 1.
Rare & Pulmonary Diseases
2 trialsLTI‑03 for Idiopathic Pulmonary Fibrosis
Inhaled therapy mimicking Caveolin‑1 to modulate multiple fibrotic pathways
Timeline / Sites
3 March 2026: Rein Therapeutics announced dosing of the first patient in a Phase 2 randomized placebo‑controlled study evaluating LTI‑03 for idiopathic pulmonary fibrosis. Approximately 120 patients are expected to be enrolled across placebo, low‑dose, and high‑dose treatment arms. Enrollment is expected to continue through mid‑2027 with interim data anticipated in the second half of 2026.
What's New
LTI‑03 is an inhaled therapy designed to mimic Caveolin‑1 and modulate multiple fibrotic signaling pathways involved in pulmonary fibrosis.
Why It Matters
Idiopathic pulmonary fibrosis has limited treatment options and poor prognosis. An inhaled disease‑modifying therapy could significantly improve patient outcomes.
TNX‑103 (Levosimendan) for Pulmonary Hypertension with HFpEF; LEVEL Program
Oral formulation enhancing cardiac contractility and reducing pulmonary vascular resistance
Timeline / Sites
10 March 2026: Tenax Therapeutics reported reaching the LEVEL randomization target of 230 patients and closing screening. Randomization is expected to finish by end of March 2026. Initial results from the LEVEL trial are expected in Q3 2026. In parallel, the company reported activation of the global Phase 3 LEVEL‑2 study, which is expected to complete enrollment by end of 2027.
What's New
TNX‑103 is an oral formulation of levosimendan that enhances cardiac contractility and reduces pulmonary vascular resistance.
Why It Matters
Patients with pulmonary hypertension associated with HFpEF currently have no approved targeted therapies. Successful results could introduce a novel mechanism‑based treatment for this population.
Infectious Diseases
2 trialsIbezapolstat for Recurrent Clostridioides difficile Infection
Narrow‑spectrum DNA polymerase III inhibitor targeting C. difficile while preserving beneficial gut microbiota
Timeline / Sites
9 March 2026: Acurx Pharmaceuticals announced initiation of a clinical development program evaluating ibezapolstat for multiply recurrent Clostridioides difficile infection. Start‑up activities began in March 2026. The open‑label pilot study will enroll up to 20 patients with two or more recurrences of infection. First patient dosing is expected in the fourth quarter of 2026.
What's New
Ibezapolstat is a narrow‑spectrum DNA polymerase III inhibitor targeting C. difficile while preserving beneficial gut microbiota.
Why It Matters
Recurrent C. difficile infection remains a major clinical challenge. A therapy capable of both treating infection and preventing recurrence could significantly improve patient outcomes.
PLG0206; RETAIN Trial for Prosthetic Joint Infection
Engineered antimicrobial peptide designed to disrupt bacterial biofilms and eliminate persistent bacteria on implants
Timeline / Sites
10 March 2026: Peptilogics announced enrollment of the first patient in the RETAIN registration trial evaluating PLG0206. The randomized double‑blind placebo‑controlled Phase 2/3 study will enroll up to 240 patients at approximately 50 global sites. The primary endpoint is treatment failure at 12 months. PLG0206 is administered during debridement, antibiotics and implant retention surgery for prosthetic joint infections.
What's New
PLG0206 is an engineered antimicrobial peptide designed to disrupt bacterial biofilms and eliminate persistent bacteria on implanted devices.
Why It Matters
Biofilm‑associated infections remain difficult to treat and often require removal of implants. A therapy capable of eliminating biofilms during surgery could significantly improve cure rates.
Advanced Modalities / Gene‑Editing & Precision Therapies
1 trialNexiguran Ziclumeran (nex‑z) for ATTR Cardiomyopathy; MAGNITUDE Trial
CRISPR/Cas9 in vivo gene‑editing therapy using lipid nanoparticles to reduce hepatic production of transthyretin protein
Timeline / Sites
2 March 2026: Intellia Therapeutics announced that the U.S. FDA removed the clinical hold on the Phase 3 MAGNITUDE trial evaluating nexiguran ziclumeran for transthyretin amyloidosis with cardiomyopathy. Enrollment activities in the randomized double‑blind placebo‑controlled trial of approximately 1,200 patients with ATTR‑CM will now resume.
What's New
Nexiguran ziclumeran uses CRISPR/Cas9 gene editing delivered through lipid nanoparticles to reduce hepatic production of transthyretin protein.
Why It Matters
This therapy represents one of the most advanced in vivo gene editing approaches currently in clinical development.
Other Notable Updates
Vima Therapeutics; Parkinson's Disease Expansion
Beyond dystonia, Vima Therapeutics plans a Phase 2 trial for Parkinson's disease using the same VIM0423 modulator in mid‑2026, reflecting cross‑disease application of dopaminergic/acetylcholine modulation. Topline data from both Phase 2 trials are expected in H1 2027.
Tenax Therapeutics; LEVEL‑2 Global Study
In parallel with reaching the LEVEL enrollment target, Tenax activated the global Phase 3 LEVEL‑2 study for TNX‑103, expected to complete enrollment by end of 2027.
Conclusion
Between 1 and 15 March 2026, the U.S. clinical‑trial landscape saw important progress across multiple therapeutic areas. In oncology, a new combination immunotherapy arm launched within the INSIGhT adaptive platform for glioblastoma, and the AMPLIFY diagnostic imaging trial achieved its target enrollment. Neurology and psychiatry saw first‑patient dosing in dystonia, completion of enrollment in schizophrenia, and initiation of a global Phase 3 myotonic dystrophy program. Rare and pulmonary disease trials moved forward with a Phase 2 IPF study and a key enrollment milestone for pulmonary hypertension. Infectious disease programs introduced novel approaches targeting recurrent C. difficile and prosthetic joint biofilm infections. In advanced modalities, the pivotal MAGNITUDE CRISPR gene‑editing trial resumed enrollment after FDA lifted a clinical hold. Across these areas, a shared theme is the continued shift toward mechanism‑driven, disease‑modifying therapies.
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