CHICAGO, Dec. 02, 2025 (GLOBE NEWSWIRE) — Quetzal Therapeutics, a biopharmaceutical company specializing in developing transformative treatments for rare diseases, announced the initiation of a pivotal Phase III clinical trial of QTX-2101, an investigational oral arsenic trioxide capsule. The trial will evaluate the efficacy, safety, and pharmacokinetics of QTX-2101, as a potential new treatment for acute promyelocytic leukemia (APL), a rare and aggressive blood cancer.
This global, multicenter, randomized, controlled study will compare QTX-2101 to standard-of-care therapy in patients with newly diagnosed APL. The trial builds on encouraging pharmacokinetic and safety data from two prior Phase I studies in U.S. patients, which laid the groundwork for advancing QTX-2101 into late-stage development1,2.
QTX-2101 is an oral capsule developed with the goal of overcoming the limitations of intravenous arsenic trioxide. By replacing daily infusions with an oral capsule, QTX-2101 is being evaluated for its potential to offer precise dosing and stability, and, most importantly, meaningful benefits for patients and caregivers: less time in clinics, fewer disruptions to daily life, and reduced strain on healthcare resources. The goal is to make treatment more accessible and manageable, while avoiding the potential variability and handling challenges of oral liquid regimens.
“The launch of this Phase III trial is an important milestone in the development of QTX-2101,” said Dr. Shaad Abedin, Chief Medical Officer at Quetzal Therapeutics. “While survival rates in APL are favorable, treatment-related burden and tolerability remain significant challenges. Our oral capsule formulation is designed to address key limitations of intravenous arsenic trioxide, with the goal of making treatment more accessible and manageable for patients and providers.”
About Acute Promyelocytic Leukemia (APL)
APL is a rare and aggressive subtype of acute myeloid leukemia, accounting for approximately 10-15% of all AML cases. It is defined by the PML-RARA gene fusion and is associated with severe bleeding complications and rapid disease progression if untreated. While advances in therapy have transformed outcomes, current treatments often require patients to undergo a large number of lengthy intravenous infusions over the course of therapy. This relentless treatment schedule can disrupt daily life and place a significant burden on both patients and their support networks. There remains a clear need for innovative and more accessible therapies.
About Quetzal Therapeutics
Quetzal Therapeutics is a biopharmaceutical company dedicated to developing treatment for rare diseases, including hematologic malignancies. The company’s lead product is QTX-2101, a novel approach for treating patients with APL. Quetzal is also advancing QTX-2102, a pre-clinical, next-generation antifungal and antiparasitic therapy designed to enhance efficacy while minimizing toxicity.
QTX-2101 is an investigational, oral arsenic trioxide therapy that has not yet been approved by any regulatory authority. Its safety and efficacy are currently under evaluation. This press release contains forward-looking statements reflecting current expectations for ongoing development, which may evolve over time.
For more information, please visit: www.quetzaltx.com
Media Contact:
info@quetzaltx.com
References
- Ravandi, F., Koumenis, I., Johri, A., Tallman, M., Roboz, G. J., Strickland, S., … & Kantarjian, H. (2019). Oral arsenic trioxide ORH-2014 pharmacokinetic and safety profile in patients with advanced hematologic disorders. Haematologica, 105(6), 1567.
- Ravandi, F., Rangaraju, S., Kantarjian, H., Garcia-Manero, G., Yilmaz, M., Baker, K., … & Ghiaur, G. (2025). A pharmacokinetic and safety study of oral arsenic trioxide in patients with acute promyelocytic leukemia. Blood Advances, 9(9), 2136-2143.