The Targeted Pulse: Unlock Recent FDA Highlights in Leukemia and Prostate Cancer and An Interesting Vaccine Gaining Traction in Triple-Negative Breast Cancer

Imatinib, Now in Liquid Form, Receives Approval in Leukemia and Other Cancers

The first liquid form of imatinib (Imkeldi) oral solution has received FDA approval for certain forms of leukemia and other cancers. This liquid formulation of imatinib assists in dosing accuracy, accessibility, and treatment adherence and helps slow or prevent growth of chronic myeloid leukemia, acute lymphoblastic leukemia, myelodysplastic syndrome/myeloproliferative disease, and gastrointestinal stromal tumors.

“We are thrilled to offer an oral solution option for patients with leukemia and other cancers, [which is] a meaningful advancement for thousands in need,” said Sharon Cunningham, chief executive officer of Shorla, in a press release. “Oral solutions may ensure more precise and consistent dosing, offering a convenient alternative to compounding for patients who have difficulty swallowing or require dosing tailored to body surface area.”

A New Vaccine for Triple-Negative Breast Cancer Shows Interesting Potential

A phase 1 trial (NCT04674306) showed positive protocol-defined immune responses of an investigational breast cancer vaccine targeting triple-negative breast cancer. Data were presented at the 39th Annual Society for Immunotherapy of Cancer Meeting, stating that the α-lactalbumin vaccine demonstrated safety, tolerability, and immune activity across all its 3 patient cohorts. For the phase 2 trial, investigators plan to evaluate patients with newly diagnosed breast cancer in the neoadjuvant setting, with this phase set to begin in 2025.

“Triple-negative breast cancer is the form of the disease for which we have the least effective treatments," said G. Thomas Budd, MD, of Cleveland Clinic’s Cancer Institute and principal investigator of the phase 1 study, in a press release. “Long term, we hope this can become a true preventive vaccine that would be administered to individuals who are cancer-free to prevent them from developing this highly aggressive disease.”

Brexu-cel Appears to Stand theCentral Nervous System Test in R/RB-ALL

Brexucabtagene autoleucel (brexu-cel; Tecartus) demonstrated high rates of central nervous system (CNS) remission in patients with relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL), according to data from the Real-World Outcomes Collaborative for Chimeric Antigen Receptor T in ALL (ROCCA) consortium. Ibrahim N. Muhsen, MD, a hematology and medical oncology fellow at Baylor College of Medicine, discussed his findings regarding the efficacy of brexu-cel in achieving CNS and systemic responses in adult patients with CNS involvement in relapsed/refractory B-ALL.

“When it comes to the safety of brexu-cel, [for] our patient population that has a very low rate of high-grade CRS, with only 1 patient experiencing grade 3 to 4 CRS. On the other hand, when it comes to immune effector cell-associate neurotoxicity syndrome [ICANS], we had a higher number of patients who had grade 3 to 4 ICANS. However, when we compared these numbers with those in the ROCCA consortium database—specifically patients without CNS involvement—the rates were similar,” Muhsen told Targeted Oncology^TM in the interview.

Darolutamide Starts its FDA Journey With a sNDA in mHSPC

The FDA has accepted the supplemental new drug application for darolutamide (Nubeqa) in combination with androgen deprivation therapy (ADT) for patients with metastatic hormone-sensitive prostate cancer (mHSPC). This acceptance of the application is based on data from the phase 3 ARANOTE trial (NCT04736199), which were presented at the 2024 ESMO Congress. In the trial, darolutamide plus ADT was compared with placebo plus ADT, showing a reduced risk of radiographic progression or death with the experimental combination.

“If approved, this would expand the indication for NUBEQA in patients with mHSPC to include both combinations with and without chemotherapy, providing physicians and their patients with an additional treatment option in this setting. We are working closely with the FDA to bring this additional NUBEQA treatment option to patients as soon as possible,” said Christine Roth, executive vice president, global product strategy and commercialization and member of the Pharmaceuticals Leadership Team at Bayer, in a press release.

Ribociclib Shows Significant Benefits in High-Risk Node-Negative Breast Cancer

Ribociclib (Kisqali) in combination with a nonsteroidal aromatase inhibitor (NSAI) showed significant improvement for patients with high-risk, node-negative, early-stage breast cancer, according to an analysis of data from the phase 3 NATALEE trial (NCT03701334). Denise Yardley, MD, director of breast cancer research at Sarah Cannon Research Institute in Nashville, Tennessee, discussed the positive data and outlined the next steps, which include identifying patients who could benefit from ribociclib and other therapies sparing chemotherapy.

“By focusing on a select high-risk group of node-negative patients, we were able to demonstrate that they also benefited from…the addition of ribociclib to their anastrozole or letrozole therapy. This broadens the group of early-stage breast cancer patients who stand to benefit from an agent like ribociclib added to their standard endocrine therapy,” Yardley told Targeted Oncology^TM in the interview. “The trial did show those that received only the current standard of care, which is endocrine therapy by itself, fared poorly compared with the group that had the NSAI and ribociclib combination.

Thank you for joining us for this week’s Targeted Pulse. Look out for more recaps to come.

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