Avanti Biosciences: From Neurodegeneration to a Breakthrough in Fibrosis

Avanti Biosciences, founded in 2009 in New York by Dr. Gian Luca Araldi, began its journey in the world of biotechnology as a nimble licensing-focused startup. For several years, the company forged partnerships and evaluated external opportunities on the rapidly changing frontiers of pharmaceutical innovation. In 2016, Avanti’s transformation accelerated when its scientists turned their attention inward, driven by a growing curiosity about catechins, a family of polyphenolic compounds with promising biological potential. This strategic pivot set Avanti on a path of active R&D and, in 2018, the company relocated to JLABS in San Diego, immersing itself in a world-class innovation ecosystem to advance its proprietary small molecule pipeline.

Initially inspired by the potential of epigallocatechin gallate (EGCG) analogs in the treatment of neurodegenerative disorders like Alzheimer’s and Down syndrome, the Avanti team set out to optimize molecules that could inhibit the kinase DYRK1A pivotal target in brain pathologies. This foray into medicinal chemistry yielded not only potent DYRK1A inhibitors, but also a surprising new direction: amid the data emerged ABI-01, a small molecule with an unexpected and highly promising dual mechanism of action. In addition to inhibiting DYRK1A, ABI-01 potently blocked PIM1 kinase, a master regulator implicated in fibrotic disease.

With ABI-01 in hand, the focus of Avanti Biosciences shifted dramatically. Preclinical experiments revealed ABI-01’s remarkable anti-fibrotic activity in validated models of idiopathic pulmonary fibrosis (IPF) and NASH-related liver fibrosis. The data was compelling: in rigorous head-to-head mouse studies, ABI-01 showed zero mortality and delivered striking reductions in fibrosis markers and lung function decline. Perhaps even more notably, ABI-01 outperformed all current standards of care—including nintedanib, pirfenidone, and even the promising next-generation agent nerandomilast—on critical readouts like forced vital capacity (FVC). These robust results supported once-daily oral dosing, addressing a major barrier for patient compliance.

Safety, a keystone of any breakthrough therapy, was not overlooked. IND-enabling GLP toxicology studies demonstrated no drug-related toxicities, and ABI-01 emerged with a high therapeutic index, paving the way for a confident transition to human studies.

The scientific rigor and potential of ABI-01's novel dual mechanism has garnered significant recognition from the federal government. The National Heart, Lung, and Blood Institute (NHLBI) has scored Avanti's Catalyze grant application as one of the top proposals, providing prestigious validation of the company's innovative approach to fibrotic disease treatment. This federal endorsement not only validates the scientific merit of ABI-01's dual DYRK1/PIM1 inhibition strategy but also positions Avanti for potential non-dilutive funding support to advance the program through

the IND application.

Today, Avanti Biosciences stands at the threshold of clinical development, led by a world-class team including Dr. Ganesh Raghu (CMO, and a pioneer in interstitial lung disease research) and Dr. Marianne Mann (regulatory strategist and former FDA leader). The company’s discoveries have resulted in a robust intellectual property estate, an FDA Orphan Drug Designation for IPF, NHLBI Catalyze program recognition, and $7 million in non-dilutive NIH grant funding. A $3M convertible note raise is now underway to propel ABI-01 through first-in-human trials and position it as a transformative therapy for both IPF and NASH.

Avanti’s story is one of scientific agility and relentless innovation—of following biological insight wherever it may lead. From a licensing-first startup to a fierce R&D engine, Avanti Biosciences exemplifies how focus, bold experimentation and commitment to unmet patient need can result in genuine breakthrough medicines.