Abu Dhabi to conduct clinical trials of thalassemia drug

Abu Dhabi: As an incubator and hub for life sciences research and innovation, Abu Dhabi will begin clinical trials with two global clinical trials to transform the treatment of thalassemia.

Under the supervision and support of the Abu Dhabi Department of Health (DoH), the phase 3 trials ENERGIZE and ENERGIZE-T in adults with thalassemia were designed to test the efficacy and safety of the new drug Mitapivat.

A clinical trial at Burjeel Medical City may bring new hope to thalassemia patients in the country. These trials will be conducted in collaboration with multiple centers and multiple sites in the United States, Europe and Asia, enabling access to state-of-the-art therapies from the very beginning of clinical development.

Mitapivat has already been approved in the US and EU for its ability to treat pyruvate kinase deficiency, however, recently, it showed potential to transform thalassemia treatment in early-stage trials.

In an upcoming phase, the Phase 3 program will evaluate the drug in two types of thalassemia with relatively different clinical needs. The ENERGIZE study was designed to evaluate whether Mitapivat can effectively and safely improve hemoglobin levels in patients with non-transfusion-dependent thalassemia who do not require regular blood transfusions.

Previous studies by Burjeel Medical City researchers have shown that low hemoglobin levels can lead to severe morbidity and possibly death. Additionally, ENERGIZE-T was designed to evaluate whether the same drug could reduce the need for blood transfusions in patients with transfusion-dependent thalassemia.

Dr Asma Al Mannaei, Executive Director, Research Centers and Innovation, DoH, said: “As the emirate’s healthcare regulator, we are working tirelessly to cement Abu Dhabi’s position as a leading global healthcare destination and a hub for research and innovation in the region’s living sciences. Abu Dhabi is able to play a key role in developing medicines and conducting clinical trials with multiple international partners. Today, we witnessed the launch of new clinical trials, so we will continue our efforts to further improve healthcare services in Abu Dhabi, And to help all members of our community and the globe.

“Last year, clinical trials in Abu Dhabi increased by 27%, reaching 71 clinical trials in 2022, compared to 56 in 2021. In addition, the UAE was one of the first countries to participate in clinical trials for COVID-19 vaccines, witnessing China’s The largest clinical trial of Sinopharm’s vaccine, covering more than 32,000 volunteers.”

Dr Khaled Musallam, Group Chief Research Officer, Burjeel Holdings, said: “With the full support and encouragement of the DoH, Abu Dhabi is now at the forefront of cutting-edge innovation. Projects from leading institutions to improve the health and well-being of patients. If these studies reach their endpoints, this could change the way diseases are treated today. A disease-modifying therapy could address a long-standing unmet need for patients in the UAE and around the world.”

According to Dr. Musallam, this milestone can only be achieved after obtaining stringent and thorough regulatory approvals to ensure that the facility meets the necessary global standards for clinical trials. The ENERGIZE trial will start with a typical screening period and last 24 weeks, while ENERGIZE-T will last 48 weeks with a primary outcome core treatment period followed by a five-year extension. The trials will include clinical, radiological and laboratory assessments which will be performed at Burjeel Medical City using state-of-the-art technology.