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Alpha1H was granted a fast track designation in November 2023.
Hamlet BioPharma recently announced the successful completion of an in-person meeting with the FDA, paving the way for initiation of the company’s pivotal phase 3 trial of Alpha1H in patients with non–muscle invasive bladder cancer (NMIBC).1
The phase 2 study of Alpha1H met all primary safety and efficacy end points.
The company said that they remain on track to initiate the study, subject to final protocol agreement from the FDA.
“We appreciate the FDA’s engagement and valuable feedback as we continue the late-stage development of Alpha1H and the treatment of bladder cancer,” said Catharina Svanborg, MD, CEO of Hamlet Biopharma, in a news release from the company.1 “This meeting brings us one step closer to delivering a new treatment option for patients facing bladder cancer.”
Alpha1H was granted a fast track designation by the FDA for the treatment of NMIBC in November 2023.2 With thisdesignation, the development process for Alpha1H can benefit from more frequent engagement with the FDA and eligibility for accelerated approval and priority review.
A phase 1/2 trial (NCT03560479)of Alpha1H was completed in December 2024, and data were presented at the 2025 American Association for Cancer Research Annual Meeting in Chicago, Illinois.3 Overall, the study met all primary safety and efficacy end points.
On the trial’s primary efficacy end point of cell shedding, the authors reported, “Intra-vesical instillations of Alpha1-oleate triggers massive shedding of tumor cells and the tumor size is reduced.” Further, no treatment-related adverse events (AEs) were reported.
The investigators also found that “Shed cells contain alpha1-oleate, treated tumors show evidence of apoptosis, and the expression of cancer-related genes is inhibited,” meeting the secondary end points in the study.
In total, the single-center, phase 1/2 trial planned to enroll 52 adult patients with NMIBC who are awaiting transurethral resection of bladder tumor. Patients were eligible for enrollment if they showed papillary disease on cystoscopy and had the ability to keep the content of their bladder for at least 1 hour. Patients were not eligible for the study if they had a previous history of muscle-invasive bladder cancer, an acute urinary tract infection, or previous intravesical chemotherapy or intravesical BCG within the 12 months prior to enrollment.
In the main part of the study, participants were randomly assigned 1:1 to receive 7.4 mg/mL intravesical Alpha1H or placebo. In part 2 of the study, 2 cohorts of patients received escalating doses (37 mg/mL and 74 mg/mL) of Alpha1H in a standard 3+3 design.
The primary safety end point was the AE profile, and the primary efficacy end point was cell shedding. The investigators also assessed the change in baseline in characteristics of papillary tumors.
Based on findings from the study, the authors concluded, “The results are especially encouraging for bladder cancer, where therapeutic failures and high recurrence rates create a great, unmet medical need.”
REFERENCES
1. Hamlet BioPharma holds successful in person meeting with US FDA. News release. Hamlet BioPharma. June 25, 2025. Accessed July 16, 2025. https://hamletbiopharma.com/hamlet-biopharma-holds-successful-in-person-meeting-with-us-fda/
2. Hamlet BioPharma receives U.S. FDA Fast Track designation for Alpha 1H for the treatment of non-muscle invasive bladder cancer. News release. Hamlet BioPharma. November 29, 2023. Accessed July 16, 2025. https://hamletbiopharma.com/hamlet-biopharma-receives-u-s-fda-fast-track-designation-for-alpha-1h-for-the-treatment-of-non-muscle-invasive-bladder-cancer/
3. Tran H, Brisuda A, Ambite I, et al. Bladder cancer therapy with Alpha1H: From preclinical development to successful Phase II trials. Cancer Res. 2025;85(8, suppl 2):CT174. doi:10.1158/1538-7445.AM2025-CT174
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