Trial Watch; April Edition 7
U.S. Clinical Trial Announcements
This period shows growing interest in oral, once‑daily therapies for hypoparathyroidism, cardiac‑surgery‑associated acute kidney injury and urticaria. A rare gene therapy trial for heart failure completed enrollment, the IMPROVE‑PAH Phase 3 trial enrolled its first patient, and DA‑1726 advanced into higher‑dose Phase 1 cohorts for obesity. Collectively, these developments show continued innovation across gene therapy, small‑molecule and biologic modalities.
Therapeutic Areas Covered
Cardiometabolic & Cardiopulmonary
3 trialsGenePHIT; AB‑1002 Gene Therapy for Heart Failure
One-time intracoronary AAV gene therapy delivering modified inhibitor‑1 (I‑1c) to boost cardiac contractility
Timeline / Sites
Reported 1 April 2026. AskBio announced that the last participant was randomized in GenePHIT, an adaptive, double‑blind, placebo‑controlled study with 173 adults with heart failure. Initial results are expected in H1 2027.
What's New
AB‑1002 is a one-time intracoronary AAV gene therapy delivering a modified inhibitor‑1 (I‑1c) to boost cardiac contractility. Completing enrollment brings the study closer to assessing gene therapy for heart‑failure patients whose mortality remains high.
Why It Matters
This is a significant milestone in gene therapy for heart failure. Completion of enrollment in a Phase 2 study positions this program among the most advanced cardiac gene therapy efforts globally.
IMPROVE‑PAH; IKT‑001 for Pulmonary Arterial Hypertension
Oral prodrug of imatinib engineered to reduce gastrointestinal side‑effects with adaptive dose‑titration
Timeline / Sites
Reported 7 April 2026. Inhibikase enrolled the first patient in its pivotal adaptive Phase 3 trial designed to enroll up to ~180 sites globally. Part A will randomize ~140 patients with a primary endpoint of change in pulmonary vascular resistance at week 24; Part B will measure 6‑minute walk distance.
What's New
IKT‑001 is an oral prodrug of imatinib engineered to reduce gastrointestinal side‑effects. Imatinib has shown improvements in PAH parameters, and this study uses adaptive dose‑titration to optimize efficacy while minimizing toxicity.
Why It Matters
This pivotal Phase 3 study represents a major step forward for a reformulated tyrosine kinase inhibitor approach to PAH, a disease area in need of better-tolerated therapies.
DA‑1726 Higher-Dose Cohorts for Obesity
Dual GLP‑1/glucagon receptor agonist (oxyntomodulin analogue) exploring higher doses for weight loss
Timeline / Sites
Reported 10 April 2026. MetaVia dosed the first patient in Part 3 of its Phase 1 study evaluating higher doses of DA‑1726. Part 3 comprises two 16‑week titration cohorts (one‑step to 48 mg and two‑step to 64 mg). Up to 40 obese but otherwise healthy adults will be enrolled.
What's New
DA‑1726 is a dual oxyntomodulin analogue activating GLP‑1 and glucagon receptors, aiming to deliver greater weight loss, improved glycemic control and liver benefit compared to current GLP‑1 agonists.
Why It Matters
Higher‑dose cohorts may optimise efficacy and tolerability for a dual agonist approach that could offer advantages over single-mechanism GLP-1 therapies in obesity treatment.
Oncology
2 trialsNexTGen / SABRE / Dana‑Farber CAR T‑Cell Trials for Pediatric Solid Tumors
First-of-its-kind international trial testing dual-target CAR T‑cell therapies in children and young adults with relapsed/refractory solid tumors
Timeline / Sites
Reported 8 April 2026. The first two patients have been enrolled in a first‑of‑its‑kind international trial. Up to 60 participants will be enrolled across U.S. and UK sites.
What's New
The trial combines two CAR‑T platforms targeting two tumor markers, aiming to overcome the limited efficacy of CAR T in solid tumors. It seeks to improve survival and reduce long‑term side‑effects for young patients.
Why It Matters
This represents a paradigm shift in pediatric oncology, testing CAR T‑cell therapy against solid tumors for the first time in an international, multi-platform trial design.
IM‑1021; ROR1‑Targeted ADC for Solid Tumors & Lymphomas
First-in-human dose-escalation and expansion study of a ROR1-targeted antibody-drug conjugate with proprietary topoisomerase‑I inhibitor payload
Timeline / Sites
Reported April 2026. Immunome initiated a Phase 1, first‑in‑human dose‑escalation and expansion study of IM‑1021 for B‑cell lymphomas and solid tumors. The trial evaluates safety, PK and preliminary efficacy.
What's New
IM‑1021 incorporates Immunome's proprietary topoisomerase‑I inhibitor payload and targets ROR1, a receptor expressed on many tumors. This early‑phase study explores a novel ADC platform with potential across lymphomas and solid tumors.
Why It Matters
ROR1 is an emerging target across multiple tumor types, and this novel ADC platform could expand the therapeutic reach of antibody-drug conjugates into new indications.
Infectious Diseases & Vaccines
1 trialC104; rVSV‑Marburg Single-Dose Vaccine
Single-dose recombinant VSV-vectored Marburg virus vaccine using a live attenuated rVSV platform similar to the approved Ebola vaccine
Timeline / Sites
Reported 6 April 2026. IAVI began a randomized, observer‑blind, placebo‑controlled Phase 1 trial (C104) at U.S. sites, enrolling 112 healthy adults at four dose levels. The study will evaluate safety and immune responses.
What's New
The candidate uses a live attenuated rVSV platform, similar to the approved Ebola vaccine, to elicit rapid, durable immunity. The trial is part of a U.S. preparedness program for high‑consequence viral outbreaks.
Why It Matters
A single-dose Marburg vaccine using proven VSV-vector technology could provide rapid outbreak response capability, particularly important given recent Marburg outbreaks globally.
Neurology & Rare Diseases
3 trialsSEP‑479; Oral PTH1R Agonist for Hypoparathyroidism
Once-daily oral small-molecule agonist of the parathyroid hormone receptor designed to replace injectable PTH replacement
Timeline / Sites
Reported 13 April 2026. Septerna announced that its first participants have been dosed in a single‑ and multiple‑ascending‑dose study assessing SEP‑479 in healthy volunteers.
What's New
SEP‑479 is a once‑daily oral agonist designed to replace parathyroid hormone signalling; if successful, it could provide a convenient alternative to injectable PTH replacement for hypoparathyroidism.
Why It Matters
An oral replacement for injectable PTH therapy would be a major quality-of-life improvement for patients with hypoparathyroidism, representing a potential paradigm shift in treatment convenience.
AKB‑9090; HIF‑PH Inhibitor for Cardiac Surgery‑Associated AKI
Novel hypoxia-inducible factor prolyl-hydroxylase inhibitor designed to protect kidneys during cardiac surgery
Timeline / Sites
Reported 13 April 2026. Akebia began a randomized, double‑blind, placebo‑controlled single‑ and multiple‑ascending‑dose trial enrolling healthy volunteers to assess safety, tolerability and PK/PD.
What's New
AKB‑9090 is a novel hypoxia‑inducible factor prolyl‑hydroxylase (HIF‑PH) inhibitor designed to protect the kidneys during cardiac surgery by activating cellular hypoxia pathways.
Why It Matters
If successful, AKB‑9090 could become the first preventive pharmacotherapy for cardiac surgery‑associated acute kidney injury, addressing a major unmet need in perioperative care.
EDP‑978; Oral KIT Inhibitor for Urticaria
Once-daily oral KIT kinase inhibitor targeting mast-cell activation for chronic spontaneous urticaria and mast-cell driven diseases
Timeline / Sites
Reported 13 April 2026. Enanta initiated a double‑blind, placebo‑controlled Phase 1 trial of EDP‑978 in healthy volunteers. The trial includes single‑ and multiple‑ascending‑dose cohorts and will evaluate safety, PK, and biomarkers.
What's New
EDP‑978 is a once‑daily KIT kinase inhibitor targeting mast‑cell activation. A safe, oral KIT inhibitor could provide a disease‑modifying therapy for chronic spontaneous urticaria and other mast‑cell driven diseases.
Why It Matters
An oral, once-daily approach to targeting mast-cell activation represents a convenient and potentially disease-modifying alternative to current biologic therapies for urticaria.
Emerging Trends
Gene Therapy Advancing in Heart Failure
AskBio's completion of enrollment in GenePHIT marks one of the most advanced cardiac gene therapy programs, demonstrating growing confidence in AAV-based approaches for cardiovascular indications with high unmet need.
Oral, Once-Daily Therapies for Rare & Immune Conditions
Three programs this period; SEP‑479 for hypoparathyroidism, AKB‑9090 for cardiac surgery AKI, and EDP‑978 for urticaria; reflect a trend toward convenient oral dosing in areas historically dominated by injectable therapies.
CAR T Expanding Into Pediatric Solid Tumors
The NexTGen/SABRE trial represents a first-of-its-kind effort to bring CAR T‑cell therapy to children with solid tumors, potentially overcoming one of the biggest limitations of current cell therapy approaches.
Dual Agonism & Reformulated Kinase Inhibitors
MetaVia's dual GLP‑1/glucagon agonist and Inhibikase's reformulated imatinib for PAH both demonstrate the trend of refining proven mechanisms through novel formulations and combination strategies.
About Trial Watch
Trial Watch is Kitsa's bi-weekly overview of early-phase clinical trial activity, tracking new study initiations, first-patient dosing, and data that may shape future trial design and execution.
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