CommercialProtego Biopharma Secures $130M Series B to Advance PROT-001...

Protego Biopharma Secures $130M Series B to Advance PROT-001 Into Pivotal Testing

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Protego Biopharma has completed an oversubscribed series B fundraise totaling $130 million to support the advancement of its lead program for amyloid light-chain (AL) amyloidosis. The round was co-led by Novartis Venture Fund and Forbion, with new participation from Omega Funds, Droia Ventures, YK Bioventures, and Digitalis Ventures. Existing backers, including Vida Ventures, MPM BioImpact, Lightspeed Venture Partners, and Scripps Research, also contributed.

The company stated that the new capital will enable the transition of PROT-001, its investigational small molecule, into pivotal clinical testing. Protego CEO Brent Warner said the support from investors reflects “the promise of our science and the urgency of our mission,” a remark included in Monday’s release. Warner also said the program brings the company closer to providing “the first disease-modifying therapy for AL amyloidosis and offering new hope to patients who currently face devastating outcomes.”

AL amyloidosis is a rare disorder in which abnormal plasma cells generate excess light chain proteins that misfold and form fibrils. These accumulations can affect multiple organs, including the heart. According to the company, its scientific approach is based on human genetics and a “unique pharmacological chaperone mechanism” intended to stabilize light chain proteins and prevent amyloid formation. Protego describes this strategy as a potential shift for AL amyloidosis and other protein misfolding conditions.

Forbion principal Tim Lohoff said his firm supports teams pursuing scientific advances aimed at significant medical and commercial impact and noted that Protego aligns with this focus. Lohoff also said PROT-001 offers a method to address the “root cause” of the condition by acting as a guide for correct protein folding.

Protego was founded in 2017 by Jeffery Kelly, Ph.D., Richard Labaudinière, Ph.D., and Xin Jiang, Ph.D. The company ties its development strategy to the work of Kelly and Labaudinière, whose earlier research led to the discovery and development of tafamidis, later acquired through Pfizer’s 2010 purchase of FoldRx and marketed under the Vyndaqel name.

In addition to its AL amyloidosis program, Protego is investigating treatments for several other disorders driven by protein misfolding, including myopathy, cardiomyopathy, stroke, renal disease, retinal diseases, channelopathies, and multiple degenerative diseases. Protego previously raised $51 million in a 2021 series A financing round.

AstraZeneca’s anselamimab did not achieve its primary endpoint of all-cause mortality or cardiovascular hospitalizations in a phase 3 study earlier this year. This outcome is notable in the context of Protego’s work because the AL amyloidosis field has seen multiple setbacks, with other companies also facing challenges in late-stage development. 

AstraZeneca had previously regarded anselamimab as a potential rare disease blockbuster after acquiring it in the 2021 Caelum Biosciences deal. Its failure followed earlier difficulties at Prothena, which revived its AL amyloidosis antibody birtamimab after a 2018 setback but ultimately discontinued the program when phase 3 findings released in May showed no improvement over placebo.

Protego plans to use the Series B funding not only for PROT-001 but also to expand research into other protein misfolding conditions. By focusing on disease-modifying strategies, Protego aims to differentiate its pipeline from competitors and offer more durable clinical benefits. The company also intends to strengthen clinical infrastructure and accelerate enrollment for pivotal trials, ensuring timely data generation and regulatory engagement.

Protego Biopharma’s successful $130 million Series B fundraising reinforces its commitment to advancing PROT-001 and its broader pipeline of protein misfolding therapies. With robust scientific backing and strategic investor support, Protego is poised to deliver potentially transformative treatments for patients with AL amyloidosis and other rare protein misfolding disorders.

Protego is also focused on building a robust clinical and scientific team to support the development of PROT-001 and future programs. By recruiting experts in protein misfolding, rare disease clinical trials, and translational medicine, the company aims to accelerate trial execution and ensure rigorous data collection.

Protego’s emphasis on combining cutting-edge science with operational excellence positions it to become a leader in the AL amyloidosis field and sets the stage for potential expansion into other protein misfolding disorders.

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