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.

A San Diego biotech hoping to crack into a rare protein misfolding disease has drummed up $130 million in investor support to get into a potential pivotal trial next year. Protego Biopharma, from the creators of Pfizer’s amyloidosis medicine Vyndaqel, has secured a Series B from lead investors Novartis Venture Fund and Forbion. Omega Funds, Droia Ventures, Vida Ventures, MPM BioImpact and others also chipped into the round, the company said on Monday morning. The biotech, founded in 2017 , is developing small molecules to reprogram protein folding. Its lead drug is for amyloid light chain amyloidosis, or AL amyloidosis. The rare disease is marked by a buildup of misfolded proteins in the heart and other organs. The toxic proteins can damage organs and lead to heart failure, sometimes resulting in death. Protego’s funding arrives just a few weeks after the FDA stamped full approval onto Johnson & Johnson’s Darzalex Faspro for the disease. The treatment received accelerated approval in January 2021 . Meanwhile, two high-profile clinical failures occurred earlier this year at Prothena and for AstraZeneca’s anselamimab , dealing a blow to the UK pharma’s $3 billion projections for the antibody. Protego’s candidate, nicknamed PROT-001, works to help proteins fold correctly, the company said. Essentially, Protego doesn’t want to address symptoms, but rather the main cause of the disease. “The standard of care is shutting down the plasma cells or trying to eradicate most of them. Having a light chain stabilizer at its source that can capture the availability of this misfolded light chain should lead to significant improvements in morbidity and mortality,” Protego CEO Brent Warner said in an interview with Endpoints News . PROT-001 is in a Phase 1 healthy volunteer trial in Australia, Warner said. The biotech anticipates collecting the traditional single-ascending and multiple-ascending dose data in the first half of 2026 and then filing for a Phase 2/3 trial later in the year, the CEO said. “Even with standard of care, patients who’ve been on it, even post-treatment, still have some of those plasma cells available that is continuously pumping out toxic light chain, and even a small amount of that toxic misfolded light chain is detrimental to cells,” Warner said. “Having a stabilizer that would be available to neutralize that toxicity should lead to no further formation of aggregates and allow those patients to get back into more of a native, natural state.” The biotech was co-founded by Scripps Research professor Jeffery Kelly, Xin Jiang and Richard Labaudinière. Kelly’s research previously led to the creation of FoldRx Pharmaceuticals, which was led by Labaudinière and bought by Pfizer in 2010 . FoldRx developed the drug tafamidis, or Vyndaqel. That treatment is approved for patients with transthyretin amyloid cardiomyopathy. Protego still has to meet with regulators to determine whether it could also get an accelerated approval like J&J did four years ago. “The regulatory environment is ever changing, particularly in rare disease,” Warner said. “Further discussions need to occur, which we anticipate having next year.” The startup has under 20 employees today and aims to have about 30 or so full-time staffers over the next year, Warner said.