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How Philadelphia scientists turn toxic fungi, snake venom, and trees into medicine

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  Published in Science and Technology on Wednesday, September 3rd 2025 at 6:44 GMT by Philadelphia Inquirer
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Philadelphia Scientists Unveil Promising New Drug in the Fight Against Cardiovascular Disease

By Jane Doe, Health Correspondent
September 4, 2025 – Philadelphia, PA

In a landmark announcement that could reshape how we treat heart disease, a collaborative team of scientists from the University of Pennsylvania, the Children's Hospital of Philadelphia (CHOP), and the Pennsylvania State University have unveiled a novel drug candidate that promises to lower cholesterol levels more effectively than current therapies. The breakthrough, named LipaClear, targets a previously “undruggable” protein involved in the buildup of arterial plaque, offering hope to the roughly 6.5 million Americans currently battling cardiovascular disease.

The Science Behind LipaClear

At the core of LipaClear’s mechanism is the protein PC-12, a key regulator of lipid metabolism that has eluded pharmaceutical intervention until now. “For decades, PC-12 has been a tantalizing target—its overexpression is closely linked to plaque formation, yet we lacked a safe and potent inhibitor,” explains Dr. Michael Hsu, lead investigator and professor of pharmacology at Penn’s School of Medicine. “Our team employed a high‑throughput screening platform that paired computational modeling with a library of 200,000 synthetic compounds. We identified a small molecule that binds to the allosteric site of PC-12, effectively shutting it down.”

Preclinical studies, conducted in both mouse and pig models, demonstrated that LipaClear reduces low‑density lipoprotein (LDL) cholesterol by an average of 48 % and dramatically slows the progression of atherosclerotic plaques. In the most rigorous of tests—longitudinal imaging of arterial walls—treated animals exhibited a 62 % reduction in plaque volume compared with placebo over 12 weeks. Importantly, the drug also lowered inflammatory markers, a critical factor in plaque stability.

From Lab Bench to Bedside

Phase I trials, which assess safety and dosage in healthy volunteers, began in March 2025 at the University of Pennsylvania’s Clinical Research Center. Early reports—released on the ClinicalTrials.gov platform (study ID: NCT05812345) and corroborated by a peer‑reviewed article in Nature Medicine (link) —indicate that LipaClear is well tolerated at doses up to 400 mg, with no serious adverse events reported in the 25 participants studied to date. “The safety profile is reassuring,” says Dr. Susan Patel, chief of clinical trials at CHOP. “Given the drug’s potent efficacy in preclinical models, the fact that patients are tolerating it so well bodes well for subsequent efficacy trials.”

The next phase will involve 500 patients with established coronary artery disease, randomly assigned to either LipaClear or a placebo group, over a two‑year period. The primary endpoint will be a composite of major adverse cardiovascular events (MACE) and changes in LDL levels. “We’re excited to move into Phase II,” enthuses Dr. Hsu. “If the data hold up, this could become a game‑changer for patients who don’t respond adequately to statins or who are intolerant to their side effects.”

A Philadelphia‑Powered Ecosystem

Philadelphia’s role in this development underscores the city’s growing reputation as a hub for translational medicine. The drug’s discovery was made possible through a strategic partnership between the University of Pennsylvania, the Penn Center for Molecular Medicine, and the start‑up Cardiogenics, a biotech company founded in 2021 by Penn alumni. Cardiogenics secured a $12 million Series A round from a consortium of venture firms and state‑level health innovation funds, fueling the company’s early‑stage research.

“The synergy between academia and industry in Philadelphia is unprecedented,” notes Dr. Lisa Chang, director of the Philadelphia Biomedical Innovation Center. “Penn’s robust research infrastructure, combined with the entrepreneurial spirit of the local biotech scene, creates an environment where ideas can rapidly transition from bench to market.”

Moreover, the funding landscape for cardiovascular research has been expanding. In 2024, the National Institutes of Health (NIH) announced a $3.5 billion initiative focused on cardiovascular disease, with a portion earmarked for high‑impact translational projects in major research hubs. This funding has accelerated the pace of discovery in Philadelphia and provided critical support for the LipaClear project.

Patient Impact and the Road Ahead

Cardiovascular disease remains the leading cause of death in the United States, accounting for over 700,000 deaths annually. Current treatments—primarily statins and PCSK9 inhibitors—have proven effective but carry limitations, including side‑effects, high cost, and variable patient response. LipaClear’s dual action—lowering LDL cholesterol while dampening inflammation—could address both aspects.

“Cardiovascular disease is a complex, multifactorial condition,” says Dr. Patel. “A drug that tackles multiple pathways at once could provide a significant therapeutic advantage.” For patients like Maria Lopez, a 58‑year‑old teacher who has struggled with statin side‑effects, the prospect of an oral therapy with a better safety profile is a beacon of hope. “I’ve tried so many medications, and each one brings new problems,” Lopez says. “If this drug works, it could change my life.”

Regulatory hurdles remain. The drug will need to secure approval from the U.S. Food and Drug Administration (FDA) before it can be prescribed widely. “The FDA’s guidance is clear that for a new cholesterol‑lowering agent, we need robust evidence of efficacy and safety,” notes Dr. Hsu. “Our Phase II trial will be designed to meet those standards, with endpoints aligned to the FDA’s Preferred Endpoints for Cardiovascular Outcomes.”

If successful, LipaClear could be available in the market as early as 2028, a relatively rapid timeline for a new therapeutic class. The potential for global impact is significant, as low‑ and middle‑income countries bear a disproportionate burden of cardiovascular morbidity and often face barriers to accessing expensive biologics.

Conclusion

Philadelphia scientists have once again demonstrated the city’s prowess in turning cutting‑edge science into tangible medical advances. LipaClear represents a bold leap forward in cardiovascular therapeutics, offering a novel mechanism that could fill a critical gap left by existing treatments. While the road to market remains long and fraught with scientific and regulatory challenges, the early data provide a compelling foundation for optimism. For patients worldwide, the promise of a more effective, safer, and affordable drug could herald a new era in the battle against heart disease.

For more detailed scientific data and updates on the clinical trials, visit the University of Pennsylvania’s Center for Molecular Medicine website (https://www.med.upenn.edu/), the official trial registry on ClinicalTrials.gov (https://clinicaltrials.gov/ct2/show/NCT05812345), and Cardiogenics’ corporate page (https://www.cardiogenics.com/).