University of Milan

Agios announces positive Phase 3 ENERGIZE-T results for mitapivat in transfusion-dependent alpha- or beta-thalassemia, with 30.4% response rate vs 12.6% placebo, and files regulatory applications in U.S., EU, Saudi Arabia, and UAE.

ENERGIZE-T Phase 3 study showed mitapivat, an oral PK activator, significantly reduced transfusion burden in adults with transfusion-dependent alpha- or beta-thalassemia, meeting primary and key secondary endpoints. Agios filed for regulatory approval of mitapivat for both transfusion-dependent and non-transfusion-dependent thalassemia in the U.S., EU, Saudi Arabia, and UAE.

Agios Pharmaceuticals presented positive Phase 3 ENERGIZE-T study results for mitapivat, an oral PK activator, in transfusion-dependent alpha- or beta-thalassemia patients, achieving primary and key secondary endpoints. The company filed for regulatory approval of mitapivat in the U.S., EU, Saudi Arabia, and UAE. A live and webcast investor event will be hosted on December 9.

The EU's current GMO regulations hinder biotechnological innovation, particularly with New Genomic Techniques (NGT) like CRISPR. Experts argue for updated regulations to support agricultural progress and environmental sustainability, but legislative efforts have been stalled by ideological differences among member states.

Despite failed trials, hope is renewed in Huntington's disease treatment with a gene therapy showing 80% slowing of disease progression and another treatment seeking FDA accelerated approval.

Authors from various departments contributed to the study, with support from the Italian Ministry of Health and other projects. Supplemental digital content is available on the journal's website. Some authors received personal fees from medical companies outside the submitted work.

Dr. Silvia Formenti and Dr. Massimo Loda, both chairs at Weill Cornell Medicine, were elected to the National Academy of Medicine for their significant contributions to advancing medical sciences. Formenti pioneered the combination of radiation therapy with immunotherapy, while Loda's work illuminated prostate cancer's metabolic mechanisms, leading to targeted treatments.

Dr. Silvia Formenti and Dr. Massimo Loda, both at Weill Cornell Medicine, were elected to the National Academy of Medicine for their significant contributions to advancing medical sciences, health care, and public health. Formenti, a leader in radiation oncology and breast cancer research, pioneered combining radiation and immunotherapy, while Loda's work in molecular pathology highlighted prostate cancer metabolism, leading to targeted therapies.

Certain behaviors, such as little sleep, lack of exercise, feeling alone, eating wrong fats, overusing antibiotics, having a partner with weak immunity, excessive alcohol consumption, and being overweight, can weaken the immune system, making the body more vulnerable to disease.