Anixa's Alpha-Lactalbumin Breast Cancer Vaccine Shows 74% Immune Response Rate in Phase 1 Trial
核心洞察
Anixa Biosciences' investigational alpha-lactalbumin breast cancer (搜索) vaccine met all major primary endpoints in Phase 1 trials, demonstrating safety and tolerability at maximum tolerated dose.
The vaccine elicited protocol-defined immune responses in 74% of participants across three cohorts, including women with triple-negative breast cancer (搜索) and BRCA mutation carriers.
Combination therapy with Keytruda showed no major additional side effects and generated antigen-specific T cell responses, supporting plans for Phase 2 neoadjuvant studies.
Anixa Biosciences announced positive final results from its Phase 1 clinical trial of an investigational breast cancer (搜索) vaccine targeting alpha-lactalbumin (aLA), with the therapy meeting all major primary endpoints and demonstrating immune responses in 74% of participants. The data was presented at the 2025 San Antonio Breast Cancer Symposium, marking a significant milestone for the company's cancer prevention strategy.
Trial Design and Patient Population
The Phase 1 study (NCT04674306) enrolled 35 participants across three distinct cohorts, conducted in collaboration with Cleveland Clinic and funded by a U.S. Department of Defense grant. Cohort Ia included 26 women who had completed standard-of-care treatment for early-stage triple-negative breast cancer (搜索) (TNBC) within three years and were tumor-free but at elevated risk of recurrence. Cohort Ib consisted of 4 cancer-free women with BRCA1 (搜索), BRCA2 (搜索), or PALB2 (搜索) mutations who elected preventive mastectomy and were vaccinated prior to surgery. Cohort Ic enrolled 5 women with TNBC receiving pembrolizumab (Keytruda) in the adjuvant setting to evaluate combination safety and immune responses.
Safety and Efficacy Results
The vaccine demonstrated a favorable safety profile at the maximum tolerated dose (MTD), with adverse events primarily limited to injection-site irritation. In Cohort Ia, participants experienced no flu-like symptoms, abnormal clinical laboratory tests, or other significant adverse effects. The vaccine successfully elicited aLA-specific T cell responses, including production of interferon gamma and interleukin-17.
"Triple-negative breast cancer (搜索) remains one of the most challenging subtypes to address, and Phase 1 trials are an important step in determining whether a new approach can be administered safely and activate the immune system as intended," said G. Thomas Budd, M.D., of Cleveland Clinic's Cancer Institute and principal investigator of the study. "In this trial, the investigational α-lactalbumin vaccine was safe and well tolerated at the maximum tolerated dose and generated protocol-defined immune responses in 74% of participants—results that support continued clinical evaluation."
Combination Therapy Potential
A key finding emerged from Cohort Ic, where the vaccine was administered alongside pembrolizumab. The combination generated antigen-specific T cell responses without major additional side effects, with only two participants experiencing Grade 3 adverse events consisting of greater irritation at injection sites. This safety profile supports plans for a Phase 2 neoadjuvant combination study in newly diagnosed breast cancer (搜索) patients.
Mechanism of Action and Target Selection
The investigational vaccine targets alpha-lactalbumin, a lactation protein typically expressed in breast tissue only during lactation but found to be expressed in many breast cancers. The vaccine is designed to activate the immune system to direct cytotoxic T cells toward tumor cells expressing alpha-lactalbumin, providing preemptive immune protection against emerging tumors that express this antigen.
Preliminary immunohistochemistry analysis of primary tumors showed aLA expression ranging from absent to strong, with ongoing analyses correlating expression levels to immune response and clinical outcomes. Participants will be followed for five years after completing the study to assess long-term outcomes.
Development History and Future Plans
The vaccine technology is based on preclinical research led by the late Vincent Tuohy, Ph.D., who served as the Mort and Iris November Distinguished Chair in Innovative Breast Cancer (搜索) Research at Cleveland Clinic. Justin Johnson, Ph.D., Program Manager at Cleveland Clinic and co-inventor of the technology, noted that the findings represent an encouraging step toward Dr. Tuohy's vision of immunization as a new approach to combat breast cancer.
"We are very encouraged that the final Phase 1 data met all major primary endpoints, with the vaccine demonstrating a favorable tolerability profile at the MTD and protocol-defined immune responses in the majority of participants," stated Dr. Amit Kumar, Chairman and CEO of Anixa Biosciences. "We look forward to engaging with regulators and advancing plans for a Phase 2 study."
The positive Phase 1 results will inform the design of planned Phase 2 studies, including potential combination trials with Keytruda in the neoadjuvant setting for newly diagnosed breast cancer (搜索) patients. The data supports the advancement of this novel immunotherapeutic approach into the next phase of clinical development.