Sofi-cel gets FDA breakthrough status for aggressive blood cancers
Treatment aimed at relapsed, refractory T-ALL/LBL
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The U.S. Food and Drug Administration (FDA) has granted breakthrough designation to soficabtagene geleucel (sofi-cel), a cell therapy designed to treat relapsed or refractory T-cell acute lymphoblastic leukemia and T-cell lymphoblastic lymphoma (T-ALL/LBL), blood cancers caused by the uncontrolled growth of immune cells called T-cells.
The FDA grants breakthrough designation to therapies that have the potential to improve care for serious conditions. The designation aims to speed the development of potentially important new medicines, and it gives sofi-cel’s developer, Wugen, access to perks like more guidance from the FDA during the drug development process.
“The FDA’s Breakthrough Therapy designation underscores the promising clinical data we have generated and the potential for Sofi-cel to make a meaningful difference for patients with Relapsed or Refractory T-ALL/LBL,” Cherry Thomas, MD, Wugen’s chief medical officer, said in a company press release. “This recognition enables close collaboration with the FDA to accelerate development and, ultimately, help bring this innovative therapy to patients as quickly as possible.”
Sofi-cel, also known as WU-CART-007, is a CAR T-cell therapy, a type of cancer treatment designed to arm immune cells with a human-made receptor that allows them to better identify and kill cancer cells. Sofi-cel targets CD7, a protein expressed by cancer cells in T-ALL/LBL.
Most CAR T-cell therapies are autologous, meaning they use immune cells collected from a patient, modified in a lab, and then infused back into the patient. Sofi-cel is intended instead to be an allogeneic, or off-the-shelf, therapy that uses donor cells infused directly into the patient after a round of lymphodepletion (treatments like chemotherapy that kill a patient’s existing immune cells and make room for the therapeutic cells).
Trial underway in U.S., Australia
Wugen is running a Phase 2 study, T-RRex (NCT06514794), to test sofi-cel in people with T-ALL/LBL that is relapsed or refractory, meaning the cancer has either failed to respond to prior treatments or has come back after initially responding. The main goal is to see how many patients achieve remission (when the cancer goes away) or a response (when treatment results in a reduced cancer burden). If the results are positive, Wugen aims to use the study as a basis to apply for regulatory approvals of sofi-cel.
The T-RRex study is open to patients aged 1 and older, and it is currently recruiting participants at sites in the U.S. and Australia.
The new FDA designation was based in part on data from a previous Phase 1/2 study (NCT04984356) that tested sofi-cel in people with relapsed or refractory T-ALL/LBL. Data from 11 patients treated at the dose that’s now being tested in the T-RRex study showed more than 90% responded to the treatment, with more than 70% achieving remission.
The most common side effect reported in the study was an inflammatory reaction known as cytokine release syndrome, which affected almost all patients and was severe in about one-fifth of participants.
“Our goal is to bring this investigational off-the-shelf allogeneic CAR-T treatment to patients as soon as possible,” said Kumar Srinivasan, PhD, Wugen’s president and CEO. “Receiving Breakthrough Therapy Designation from the FDA is a significant milestone for our company and a testament to the potential of our therapy to address a critical unmet medical need.”
