Moleculin's Groundbreaking Approval Marks Start of Phase 3 AML Clinical Trials in Europe
targeting patients with acute myeloid leukemia (AML) who have either relapsed after initial treatments or are refractory.
The regulatory clearance from Ukraine's Ministry of Health came just in time as the company gears up to expand its clinical trial activities not only across Europe but also in the U.S. and the Middle East, expected to ramp up throughout the first three quarters of 2025.
Walter Klemp, the Chairman and CEO of Moleculin, expressed optimism surrounding the company's progress. ''Achieving this important milestone of receiving our first country's approval puts us another step closer to getting this study well underway,'' he stated. The MIRACLE trial is poised to provide valuable insights into the effectiveness of Annamycin in mitigating the challenges presented by relapsed or refractory AML.
The study, designed with a pivotal adaptive approach, will randomly assign between 75 to 90 participants to receive either high-dose cytarabine (HiDAC) combined with one of two Annamycin dosages or a placebo. This targeted design, which was developed with FDA input during a prior meeting, allows for early unblinding of preliminary primary efficacy data and insights on safety for the participants. According to the amended protocol, preliminary results are anticipated in the second half of 2025.
Participation in the trial will require approximately 244 additional volunteers for Part B, who will follow similar randomization to either the HiDAC plus placebo or the optimum dosage of Annamycin, reflecting the FDA's new Project Optimus initiative implications. This adaptive design is expected to provide the basic statistical support aimed at demonstrating the novel treatment's effectiveness.
Annamycin has already been granted Fast Track Status and Orphan Drug Designation by the FDA for its efforts to treat AML. Furthermore, the European Medicines Agency has also recognized Annamycin for its potential in treating relapsed or refractory AML in the EU.
Moleculin's pipeline goes beyond Annamycin with several other innovative therapeutic candidates designed to tackle hard-to-treat tumors and viruses, showcasing the company’s commitment to addressing unmet medical needs. The announcement of this pivotal trial aligns with their promise to accelerate innovative treatment strategies in oncology while prioritizing patient safety and well-being.
As the company prepares to initiate the trial, it plans to generate additional preliminary data from the first cohort of participants to inform future study protocols and contribute to a potential new standard of care in AML treatment.
For those interested in following the MIRACLE trial's updates, further information is available on clinicaltrials.gov under the identifier NCT06788756.
This trial symbolizes a beacon of hope for AML patients and represents Moleculin's dedication to leading innovative clinical solutions that could redefine treatment pathways within oncology. Stay tuned for further developments as they unfold.
The regulatory clearance from Ukraine's Ministry of Health came just in time as the company gears up to expand its clinical trial activities not only across Europe but also in the U.S. and the Middle East, expected to ramp up throughout the first three quarters of 2025.
Walter Klemp, the Chairman and CEO of Moleculin, expressed optimism surrounding the company's progress. ''Achieving this important milestone of receiving our first country's approval puts us another step closer to getting this study well underway,'' he stated. The MIRACLE trial is poised to provide valuable insights into the effectiveness of Annamycin in mitigating the challenges presented by relapsed or refractory AML.
The study, designed with a pivotal adaptive approach, will randomly assign between 75 to 90 participants to receive either high-dose cytarabine (HiDAC) combined with one of two Annamycin dosages or a placebo. This targeted design, which was developed with FDA input during a prior meeting, allows for early unblinding of preliminary primary efficacy data and insights on safety for the participants. According to the amended protocol, preliminary results are anticipated in the second half of 2025.
Participation in the trial will require approximately 244 additional volunteers for Part B, who will follow similar randomization to either the HiDAC plus placebo or the optimum dosage of Annamycin, reflecting the FDA's new Project Optimus initiative implications. This adaptive design is expected to provide the basic statistical support aimed at demonstrating the novel treatment's effectiveness.
Annamycin has already been granted Fast Track Status and Orphan Drug Designation by the FDA for its efforts to treat AML. Furthermore, the European Medicines Agency has also recognized Annamycin for its potential in treating relapsed or refractory AML in the EU.
Moleculin's pipeline goes beyond Annamycin with several other innovative therapeutic candidates designed to tackle hard-to-treat tumors and viruses, showcasing the company’s commitment to addressing unmet medical needs. The announcement of this pivotal trial aligns with their promise to accelerate innovative treatment strategies in oncology while prioritizing patient safety and well-being.
As the company prepares to initiate the trial, it plans to generate additional preliminary data from the first cohort of participants to inform future study protocols and contribute to a potential new standard of care in AML treatment.
For those interested in following the MIRACLE trial's updates, further information is available on clinicaltrials.gov under the identifier NCT06788756.
This trial symbolizes a beacon of hope for AML patients and represents Moleculin's dedication to leading innovative clinical solutions that could redefine treatment pathways within oncology. Stay tuned for further developments as they unfold.
Topics Health）
【About Using Articles】
You can freely use the title and article content by linking to the page where the article is posted.
※ Images cannot be used.
【About Links】
Links are free to use.