Tafamidis Meglumine

Protego Biopharma\'s oversubscribed series B was led by Novartis Venture Fund and Forbion. \n Protego Biopharma is gearing up to bring its amyloid light-chain (AL) amyloidosis prospect into pivotal testing using its new $130 million series B fundraise.The oversubscribed funding round, led by Novartis Venture Fund and Forbion, picked up new support from Omega Funds, Droia Ventures, YK Bioventures and Digitalis Ventures. Existing investors including Vida Ventures, MPM BioImpact, Lightspeed Venture Partners and Scripps Research also pitched in, underscoring “the promise of our science and the urgency of our mission,” Protego’s CEO Brent Warner said in a Monday press release. With the fresh capital, the biotech is positioned to advance lead candidate PROT-001 into pivotal trials, bringing the biotech one step closer to delivering “the first disease-modifying therapy for AL amyloidosis and offering new hope to patients who currently face devastating outcomes,” according to Warner.AL amyloidosis is a rare and potentially fatal condition triggered by abnormal plasma cells that produce excess light chain proteins that misfold, forming fibrils that deposit in various organs and tissues—including the heart. Protego’s strategy to address the condition is rooted in human genetics and “unique pharmacological chaperone mechanism,” it said in the release.By stabilizing immunoglobulin light chains and therefore preventing amyloid buildup, PROT-001 is designed to directly tackle the disease as opposed to managing symptoms, representing what Protego calls a “potential paradigm shift not only for AL amyloidosis, but also for a wide spectrum of protein misfolding disorders with profound unmet needs.”“At Forbion, we invest in bold teams turning breakthrough science into tangible medical and commercial impact, and Protego exemplifies that vision,” Forbion principal Tim Lohoff, Ph.D., commented.San Diego-based Protego was established in 2017 by Jeffery Kelly, Ph.D., Richard Labaudinière, Ph.D., and Xin Jiang, Ph.D. The company attributes its approach in protein misfolding diseases to the work done by Kelly and Labaudinière discovering and developing tafamidis, a transthyretin amyloid cardiomyopathy drug that Pfizer now brands as Vyndaqel after its 2010 acquisition of Kelly and Labaudinière’s FoldRx.Besides AL amyloidosis, Protego is focused on protein misfolding that causes myopathy, cardiomyopathy, stroke, renal disease, retinal diseases, channelopathies and various degenerative diseases, it says. In 2021, investors contributed $51 million in a series A financing round to support the company’s mission. Protego thinks PROT-001 has the promise to succeed in an area where Big Pharmas and biotechs alike have previously stumbled. AstraZeneca’s anti-fibril antibody anselamimab, for one, missed its primary endpoint of all-cause mortality or frequency of cardiovascular hospitalizations in a phase 3 trial over the summer. The results proved a big blow to the program the company considered (PDF) a potential rare disease blockbuster after splashing out $150 million for it in its 2021 Caelum Biosciences buy.  AZ’s flop came after similar failures for Prothena, which revived its AL amyloidosis candidate birtamimab after a 2018 miss that prompted mass layoffs. But, after a phase 3 study that read out in May and found Prothena’s antibody was no better in reducing time to all-cause mortality than placebo, the biotech finally gave up on the program and implemented more layoffs.