Protego Biopharma Advances AL Amyloidosis Treatment with $130M Series B Funding

Protego Biopharma, a San Diego-based biotech company, has successfully closed a $130 million Series B funding round to advance its innovative treatment for amyloid light-chain (AL) amyloidosis. The oversubscribed round, led by Novartis Venture Fund and Forbion, positions the company to push forward with pivotal clinical trials for its lead candidate, PROT-001.

Novel Approach to Protein Misfolding Diseases

Protego's PROT-001 represents a potentially groundbreaking approach to treating AL amyloidosis, a rare and often fatal condition caused by the misfolding and toxic accumulation of light chain proteins in various organs. Unlike current therapies that focus on managing symptoms or eliminating plasma cells, PROT-001 is designed to address the root cause of the disease by stabilizing light chain proteins and preventing amyloid buildup.

"We are positioned to deliver the first disease-modifying therapy for AL amyloidosis, offering new hope to patients who currently face devastating outcomes," said Brent Warner, CEO of Protego Biopharma. The company's approach is rooted in human genetics and utilizes a unique pharmacological chaperone mechanism to guide proper protein folding.

Pivotal Trial on the Horizon

With the new funding, Protego is set to launch a pivotal clinical trial for PROT-001 in the second half of 2026. The company has already initiated an early-stage study in the second quarter of 2025, with results expected next year. This accelerated timeline underscores the urgency of addressing the unmet needs in AL amyloidosis treatment.

The Series B round attracted new investors, including Omega Funds, Droia Ventures, YK Bioventures, and Digitalis Ventures, while also garnering continued support from existing backers such as Vida Ventures, MPM BioImpact, Lightspeed Venture Partners, and Scripps Research.

Building on a Legacy of Protein Science

Protego's foundation rests on the pioneering work of co-founder Jeffery Kelly, Ph.D., a protein folding expert at Scripps Research. Kelly's previous research was instrumental in developing tafamidis (marketed as Vyndaqel by Pfizer), a successful treatment for transthyretin amyloidosis that generates billions in annual sales.

Tim Lohoff, Ph.D., Principal at Forbion, emphasized the potential impact of Protego's work: "At Forbion, we invest in bold teams turning breakthrough science into tangible medical and commercial impact, and Protego exemplifies that vision."

As Protego advances its clinical program, the company aims to not only transform the treatment landscape for AL amyloidosis but also explore applications of its technology in other protein misfolding disorders, potentially opening new avenues for addressing a wide range of degenerative diseases.