FDA recommends against IO Biotech’s BLA for melanoma vaccine

Published 29/09/2025, 12:16
FDA recommends against IO Biotech’s BLA for melanoma vaccine

NEW YORK - IO Biotech (NASDAQ:IOBT), a clinical-stage biotech company with a market capitalization of $104 million and whose shares have gained over 70% year-to-date according to InvestingPro data, announced Monday that the U.S. Food and Drug Administration has recommended against submitting a Biologics License Application for its cancer vaccine Cylembio based on data from the IOB-013 clinical trial.

The company said the FDA’s recommendation follows trial results where Cylembio plus pembrolizumab improved progression-free survival in advanced melanoma patients but narrowly missed statistical significance. InvestingPro analysis indicates the company’s overall financial health score is weak, with rapid cash burn being a key concern.

IO Biotech plans to design a new registrational study for Cylembio in the treatment of first-line patients with advanced melanoma while continuing discussions with the FDA. The company also intends to engage with European regulators about a potential submission path in the EU.

"While this is not the outcome we had hoped for, we respect FDA’s feedback and remain confident in the therapeutic potential of Cylembio," said Mai-Britt Zocca, president and CEO of IO Biotech, in a press release statement.

The clinical-stage biopharmaceutical company is implementing a restructuring plan that includes reducing its workforce by approximately 50 percent. IO Biotech expects to incur a non-recurring charge between $1.0-$1.5 million in the third quarter of 2025 related to the restructuring.

The company reported it has sufficient capital to fund operations into the first quarter of 2026.

Cylembio is an investigational therapeutic cancer vaccine designed to kill both tumor cells and immune-suppressive cells in the tumor microenvironment. IO Biotech has completed enrollment in its Phase 3 trial and two Phase 2 trials investigating Cylembio in combination with Merck’s KEYTRUDA (pembrolizumab).

In other recent news, IO Biotech has announced the results of its Phase 3 trial for the cancer vaccine Cylembio, used in combination with pembrolizumab, for treating advanced melanoma. The trial demonstrated an improvement in progression-free survival, achieving a median of 19.4 months compared to 11.0 months for patients receiving pembrolizumab alone. However, the results narrowly missed reaching statistical significance. In light of these findings, Morgan Stanley has adjusted its price target for IO Biotech from $6.00 to $4.00, while maintaining an Overweight rating on the stock. The company plans to present detailed data at the European Society for Medical Oncology (ESMO) Congress in October 2025, highlighting the potential implications for clinical practice. This presentation has been selected as a late-breaking abstract, indicating the high quality of the research. IO Biotech also held a conference call to discuss the trial outcomes, emphasizing the combination therapy’s potential benefits for melanoma patients. These developments reflect ongoing efforts to assess the efficacy of Cylembio in improving patient outcomes.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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