New Drug Shows Promise in Reversing Heart Disease by Removing 'Artery Rust'

NOVATO, CA – May 14, 2026 – A potential new chapter in the fight against the world's leading killer, cardiovascular disease, may have just been written. At the American Heart Association's Vascular Discovery Scientific Sessions, clinical-stage biopharmaceutical company Cyclarity Therapeutics unveiled groundbreaking data from a human trial, suggesting a novel drug can safely flush a toxic form of cholesterol out of the body. The findings for their lead candidate, UDP-003, represent the first clinical evidence of its kind and point toward a paradigm shift in treatment: from merely slowing the progression of heart disease to potentially reversing the underlying damage.

For decades, the standard of care has focused on managing risk factors, primarily by lowering levels of LDL, or “bad,” cholesterol with drugs like statins. While highly effective, these treatments leave a significant residual risk, as they do not directly address the toxic, oxidized cholesterol already embedded within arterial walls. Cyclarity’s approach is fundamentally different. It aims to send a molecular cleanup crew directly into the arteries to remove the damage, a concept that could transform patient outcomes.

“Cardiovascular disease remains the world’s leading cause of death, yet most treatments focus on slowing its progression rather than removing the underlying damage that drives it,” said Dr. Stephen Nicholls, Director of the Monash Victorian Heart Institute and lead investigator of the trial. “Initial data from this clinical trial of UDP-003, offering the first evidence of safe excretion of oxidized cholesterol in humans, represents a fundamental shift in how we think about treating cardiovascular disease.”

The Toxic 'Rust' in Our Arteries

The villain at the center of this story is a molecule called 7-ketocholesterol, or 7KC. Unlike the cholesterol essential for building healthy cells, 7KC is a toxic byproduct formed when cholesterol is oxidized—a process analogous to metal rusting. This molecular “rust” is a key culprit in atherosclerosis, the gradual hardening and narrowing of arteries due to plaque buildup.

Scientific research has firmly established 7KC as a driver of the disease. It promotes inflammation, causes cell death within the arterial wall, and contributes to the formation of unstable plaques that can rupture and cause a heart attack or stroke. Elevated levels of 7KC are consistently found in the arterial plaques of patients with coronary artery disease. Despite its known toxicity, directly and safely removing 7KC from the body without disrupting essential biological functions has remained a major unsolved challenge in medicine—until now.

“Most people believe statins shrink plaque, but in reality, even the highest tolerated doses often result in less than 1% reduction,” explained Dr. Daniel M. Clemens, Vice President of Biology at Cyclarity. “Our approach is fundamentally different. We aren't just slowing down the fire; we’re sending in the 'firemen' to clear out the toxic fuel.”

A New Class of Molecular Sponges

UDP-003 is built on a unique chemical scaffold known as a cyclodextrin. These are ring-shaped molecules made of sugar units, which have a long history in pharmaceuticals as agents to improve drug solubility. Cyclarity has engineered a next-generation cyclodextrin, UDP-003, to act as an active therapeutic agent.

Its structure creates a tiny, hollow cone that is hydrophobic (water-fearing) on the inside and hydrophilic (water-loving) on the outside. This design makes it a perfect molecular trap for 7KC, which is also hydrophobic. UDP-003 circulates in the bloodstream, finds and encapsulates 7KC molecules—much like a sponge soaking up a spill—and renders them water-soluble. Once captured, the UDP-003-7KC complex is safely escorted to the kidneys and excreted from the body in urine.

This “capture and excrete” mechanism stands in stark contrast to existing therapies. Statins work systemically by reducing the liver's production of cholesterol, while PCSK9 inhibitors increase the liver's ability to clear LDL cholesterol from the blood. Neither is designed to extract the toxic oxidized cholesterol already causing damage inside the artery wall. By directly targeting and removing 7KC, UDP-003 aims to do just that, offering the potential for true plaque regression.

Promising Early Data and the Path Forward

The first-in-human, randomized, double-blind, placebo-controlled trial provided the critical first look at UDP-003's performance in people. The results, presented at the AHA conference, met all primary, secondary, and exploratory goals.

Most importantly, the drug was well-tolerated at all dose levels, with no serious adverse events reported. It demonstrated a short half-life of approximately three hours, meaning it clears from the body quickly and avoids bioaccumulation, which supports a feasible and safe dosing schedule. But the most exciting finding was the clear, dose-dependent evidence of 7KC being mobilized and excreted in the urine of participants who received the drug. This marked the first-ever clinical demonstration that this toxic molecule can be actively removed from the human body.

“By demonstrating it is possible to precisely bind to and safely excrete this toxic byproduct without disrupting the systems the body depends on, we look forward to furthering our work to bring forward treatments that save millions of lives,” said Cyclarity co-CEO and co-founder, Dr. Matthew O’Connor.

With this proof-of-concept secured, Cyclarity is already moving forward. The company is enrolling patients with acute coronary syndrome into an efficacy cohort of the ongoing trial, which will use advanced imaging (CCTA scans) to directly measure changes in plaque before and after treatment. A larger Phase 2 trial, designed to demonstrate plaque regression as its primary goal, is expected to begin later in 2026.

From AI Discovery to Broader Ambitions

The development of UDP-003 is also a story of modern drug discovery. The molecule is the first clinical-stage therapeutic to emerge from Cyclarity's proprietary AI drug discovery platform. This platform rapidly engineers and evaluates vast numbers of cyclodextrin variations to find candidates with the precise chemical properties needed to attract and encapsulate specific toxic molecules, dramatically accelerating a process that would be impossibly slow with traditional methods.

Furthermore, the implications of successfully targeting 7KC may extend far beyond the cardiovascular system. This same toxic “rust” is also implicated in a host of other age-related diseases, including Alzheimer's disease, where it contributes to neuroinflammation and cell death, and Non-Alcoholic Steatohepatitis (NASH), a severe form of fatty liver disease. A therapy that can safely remove 7KC could therefore represent a platform for treating a wide range of pathologies linked to aging.

For now, the focus remains squarely on atherosclerosis. The prospect of not just halting but reversing arterial plaque is a goal that has long animated cardiovascular medicine. The upcoming Phase 2 trial will be watched closely by clinicians and patients alike, as its results could determine whether this new class of 'molecular rust removers' can truly redefine the fight against one of humanity's oldest and most persistent killers.