
Small Molecule Therapy Developer

December 1,San Diego, California, Protego Biopharma, a clinical-stage biotechnology company dedicated to developing first-in-class small molecule therapies that reprogram protein folding to address systemic amyloid diseases and other protein misfolding disorders, announced the completion of a $130 million oversubscribed Series B financing.
This round of financing was led by Novartis Venture Fund and Forbion, with new investors including Omega Funds, Droia Ventures, YK Bioventures, and Digitalis Ventures. Existing investors such as Vida Ventures, MPM BioImpact, Lightspeed Venture Partners, and Scripps Research also participated, highlighting their continued belief in Protego Biopharma's approach. This financing round also reflects the active involvement of Executive Chairman Ed Hurwitz and Dr. Chris Weyrer, Principal at Vida Ventures and interim Chief Business Officer of Protego Biopharma, as the company advances its leading project. November 2021,Protego Biopharma Completes$51 Million Series A Financing, with investors including Lightspeed Venture Partners, Vida Ventures, and MPM Capital.

The proceeds will advance Protego's lead candidate, PROT-001, into pivotal clinical trials for AL amyloidosis, a rare and often fatal disease caused by protein misfolding, leading to organ damage, particularly in the heart.

Brent Warner, CEO of Protego Biopharma, said:"We are deeply grateful for the steadfast support of our existing investors, who have shown confidence in us at every stage, and we are thrilled to welcome global leading investors to join us on this journey. Their trust underscores the promise of our science and the urgency of our mission. With the funds raised, we will advance PROT-001 into pivotal trials, moving closer to delivering the first disease-modifying therapy for AL amyloidosis and offering new hope to patients currently facing devastating outcomes."
Protego's therapeutic strategy is rooted in human genetics and a unique pharmacological chaperone mechanism. These small molecules act as "guides" for cells, ensuring proper protein folding. By stabilizing immunoglobulin light chains and preventing amyloid accumulation, PROT-001 addresses the disease at its root rather than merely managing symptoms. This approach represents a potential paradigm shift, applicable not only to AL amyloidosis but also to a wide range of protein misfolding diseases with unmet needs.

Protego Biopharma was co-founded in 2017 by Professor Jeffery W. Kelly, Ph.D., Xin Jiang, Ph.D., and Richard Labaudinière, Ph.D., of Scripps Research. The founders of Protego Biopharma possess unique expertise and a proven track record in the field of protein misfolding diseases. Dr. Kelly, a recipient of the 2022 Breakthrough Prize in Life Sciences, is a leader in the field of protein misfolding and aggregation (amyloidosis) and pioneered the understanding of the protein aggregation process in degenerative disease progression. Dr. Kelly’s research laid the foundation for FoldRx Pharmaceuticals, led by Dr. Labaudinière, which focused on first-in-class disease-modifying small molecule therapies for protein misfolding and aggregation diseases. Prior to its acquisition by Pfizer in 2010, FoldRx developed Vyndaqel® (tafamidis), a disease-modifying therapy for hereditary transthyretin amyloidosis, a fatal condition caused by protein aggregation in peripheral nerves, autonomic nerves, and the heart.
Correct protein folding and trafficking are crucial for biological function. Folding errors caused by genetic variations, reduced ability to maintain folding due to aging, or failure of proteins to be secreted or transported to the cell surface can lead to abnormal protein aggregation or loss of function. Many diseases, including Alzheimer's disease, Parkinson's disease, cystic fibrosis, emphysema, and many channelopathies, involve misfolded or mistrafficked proteins. Protego Biopharma’s platform technology of pharmacological chaperones and unfolded protein response (UPR) modulators enables the discovery of small molecule candidates that promote protein folding and trafficking, addressing the root causes of protein misfolding diseases.
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