- Phase 1 results in healthy volunteers characterize the clinical profile of PROT-001, an investigational first-in-class lambda light chain kinetic stabilizer
- Preclinical presentations to evaluate PROT-001's potential to reduce light chain-induced cardiac toxicity in AL amyloidosis and its compatibility with bortezomib-based treatment
- AmyLiteDx™ Assay and presentations that highlight advances in pharmacodynamic and clinical activity measures for PROT-001 development
SAN DIEGO, Nov. 2, 2026 -- Protego Biopharma, Inc., a clinical-stage biotechnology company developing small-molecule therapies for protein misfolding diseases, today announced five presentations at the XX International Symposium on Amyloidosis (ISA 2026), taking place November 15–18, 2026.
The presentations follow the company's recently announced FDA clearance to proceed with its Phase 2/3 SHIELD-AL study, which will evaluate PROT-001 added to standard of care in patients with AL amyloidosis.
"PROT-001 was designed to stabilize amyloidogenic light chains and neutralize their toxicity, providing a distinct approach that could complement therapies directed at the plasma cells that produce them," said Brent J. Warner, MBA, Chief Executive Officer of Protego. "As we initiate SHIELD-AL, we believe our approach could help patients achieve faster and deeper organ responses beyond standard of care alone. Deeper cardiac responses are associated with improved survival, underscoring the potential of PROT-001 to make a meaningful difference for patients."
Protego's presentations at ISA 2026 will include four Discussed E-poster presentations and one traditional poster presentation:
Title: PROT-001, a first in class lambda light chain kinetic stabilizer for AL amyloidosis: Results from the Phase I PRO-1A-101 Study
- Abstract Number: 32458
- Session: E-poster Session 1 - Treatments of AL
- Presentation Date and Time: Monday, November 16, 2026; 10:35 AM - 11:00 AM
Title: PROT-001 Demonstrates Compatibility with Bortezomib-Based Regimens While Preserving Plasma Cell Cytotoxicity
- Abstract Number: 32147
- Session: E-poster Session 10 - Basic Science AL + Other More Rare Amyloidoses
- Presentation Date and Time: Tuesday, November 17, 2026; 11:00 AM - 12:00 PM
Title: PROT-001 Suppresses Amyloidogenic Light Chain Induced BNP Signaling and Cardiotoxicity in Human Cardiomyocytes
- Abstract Number: 32266
- Session: E-poster Session 10 - Basic Science AL + Other More Rare Amyloidoses
- Presentation Date and Time: Tuesday, November 17, 2026; 11:00 AM - 12:00 PM
Title: AmyLiteDx: A Mass Spectrometry-Based Platform for Sensitive and In-Depth Analysis of Amyloidogenic Immunoglobulin Light Chains in AL Amyloidosis
- Abstract Number: 32645
- Session: E-poster Session 10 - Basic Science AL + Other More Rare Amyloidoses
- Presentation Date and Time: Tuesday, November 17, 2026; 11:00 AM - 12:00 PM
Title: Retrospective Study Evaluating Cardiac Biomarkers in Patients with AL Cardiac Amyloidosis
- Abstract Number: 32646
- Session: General Poster Session
- Presentation Date and Time: Tuesday, November 17, 2026
About Protego Biopharma
Protego Biopharma is a clinical-stage biotechnology company developing first-in-class small molecule therapies for AL amyloidosis and other protein misfolding diseases. Protego aims to translate advances in protein stabilization into medicines that address the misfolding and toxicity responsible for progressive organ damage. Its therapies are designed to intervene in this disease process, with the goal of preserving organ function and improving patients' lives. For more information, visit www.protegobio.com.
About PROT-001
PROT-001 is Protego Biopharma's lead investigational small molecule pharmacological chaperone, being developed for the treatment of immunoglobulin light chain (AL) amyloidosis. PROT-001 is designed to stabilize amyloidogenic lambda light chain proteins and prevent their misfolding and the formation of proteotoxic species. The goal of this therapy is limiting the toxic effects that contribute to organ injury, including direct damage to cardiomyocytes in AL patients. PROT-001 represents a complementary approach to plasma cell-directed therapies, which reduce light chain production. By targeting the amyloidogenic light chain protein itself before it can adopt a pathogenic conformation, PROT-001 has the potential to complement existing treatments and address an additional mechanism of disease.
About the Phase 1 PRO-1A-101 Study
PRO-1A-101 was a first-in-human, randomized, double-blind, placebo-controlled four-part Phase 1 study that evaluated the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), food effect (FE), and drug-drug interaction (DDI) potential of PROT-001 in healthy adult participants. Part 1 was a single ascending dose (SAD) study. Part 2 was a multiple ascending dose (MAD) study. Part 3 was a non-randomized FE cross-over study. Part 4 was a non-randomized, open-label study that evaluated potential precipitant DDIs of PROT-001. Additional information on this study can be found on ClinicalTrials.gov under the identifier NCT06981299.
About AL Amyloidosis
Immunoglobulin light chain (AL) amyloidosis is a rare, life-threatening disease affecting over 5,100 newly diagnosed patients annually and 28,000 US adults in which abnormal plasma cells produce light chain proteins that can misfold and aggregate. The direct toxicity of disease-related light chains and their accumulation as amyloid deposits in tissues both result in injury to organs, including the heart and kidneys. Cardiac injury is particularly serious, as toxic light chains can directly impair cardiomyocyte function, while amyloid deposits disrupt the structure and function of the heart. Current treatments focus on reducing light chain production with plasma cell-directed therapies and managing organ complications, leaving patients with a substantial need for complementary approaches that directly address light chain toxicity and improve organ outcomes.
