Nikhil Prasad Fact checked by:Thailand Medical News Team Dec 06, 2025 1 hour, 24 minutes ago
Pharma News: Biotech Breakthrough Targets Amyloidosis at its Roots
In a major development for amyloidosis research, San Diego-based Protego Biopharma has announced a successful $130 million Series B funding round to advance a potentially game-changing drug targeting light-chain amyloidosis (AL amyloidosis). The company’s lead candidate, known as PROT-001, takes a unique approach—stabilizing misfolded proteins before they aggregate and damage vital organs, a contrast to current therapies that mainly focus on clearing the toxic buildup after it occurs.
A new protein stabilizing drug could transform treatment for AL amyloidosis patients by targeting the disease
at its earliest stage
This
Pharma News report highlights a critical step forward in the treatment of protein misfolding disorders, a category of diseases notoriously difficult to manage and often fatal if left unchecked. With financial backing from notable investors including Novartis Venture Fund and Forbion, Protego is setting its sights on late-stage clinical trials, with early trial results expected next year and a Phase 2/3 trial anticipated in the second half of 2026.
https://clinicaltrials.gov/study/NCT06981299
Why AL Amyloidosis Is So Difficult to Treat
AL amyloidosis occurs when plasma cells produce faulty antibody fragments known as “light chains.” These unstable proteins misfold, clump together, and accumulate in tissues, ultimately damaging organs like the heart, kidneys, liver, and nervous system. Existing treatment options include stem cell transplants or chemotherapy combinations like Darzalex, but these therapies often come with significant limitations, including high relapse rates.
Other pharmaceutical giants have tried and failed to conquer this disease. Biotech firm Prothena abandoned its long-developed candidate after disappointing study results. Similarly, AstraZeneca’s acquired compound failed in late-stage testing, despite high expectations. Both of these treatments were designed to remove amyloid plaques already present in the body.
Protego’s Unique Approach
Protego is betting on a preemptive strike. Rather than cleaning up toxic protein clumps, PROT-001 aims to prevent them from forming in the first place by binding to and stabilizing the light chains. CEO Brent Warner believes this approach could make all the difference, noting that “toxic light chain is really what’s driving a lot of the mortality in this disease.”
This innovative strategy is built on the pioneering research of Dr. Jeffery Kelly, a Scripps Research professor and co-founder of Protego. Dr. Kelly was instrumental in developing Vyndamax, the first FDA-approved drug for transthyretin amyloidosis, a related protein misfolding disease. That drug, now sold by Pfizer, generates billions in annual revenue and has changed lives for patients suffering from cardiac amyloidosis.
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A Potential Leap Forward in Treatment
By combining PROT-001 with existing treatments like Darzalex, Protego hopes to create a more robust and lasting response in patients. Instead of aggressively wiping out plasma cells with heavy chemotherapy, the addition of a stabilizer could lead to faster, deeper results. “In a disease like this,” Warner explained, “you may not need a sledgehammer, just a sharper knife.”
If successful, PROT-001 could not only provide new hope for AL amyloidosis patients but also pave the way for treating a broader class of protein misfolding diseases that currently have limited therapeutic options.
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