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IO Biotech Announces New Preclinical Data for Additional Pipeline Candidate Presented at the AACR-IO Conference

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IO Biotech (IOBT) presented new preclinical data for its novel peptide vaccine IO170 at the AACR-IO conference. The vaccine targets Transforming Growth Factor Beta (TGF-β), a key immune-suppressive factor in solid tumors. Unlike existing approaches that inhibit TGF-β systemically, IO170 selectively modulates TGF-β activity using IO Biotech's proprietary T-win® platform.

The preclinical studies demonstrated that IO170 significantly reduced tumor growth in pancreatic adenocarcinoma and prostate cancer models. The vaccine induced robust immune responses and reshaped the tumor microenvironment (TME) without causing systemic toxicity or adverse off-target effects. The company is preparing for an Investigational New Drug Application (IND) submission to FDA for IO170.

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Positive

  • Preclinical data showed significant tumor growth reduction in multiple cancer models
  • IO170 demonstrated effectiveness without systemic toxicity or adverse effects
  • Company advancing toward FDA IND submission for IO170

Negative

  • Product still in early preclinical stage
  • No human trial data available yet

Insights

The preclinical data for IO170 represents a potentially significant breakthrough in cancer immunotherapy, particularly addressing a critical challenge in treating solid tumors. The targeting of TGF-β is especially noteworthy as this protein plays a central role in tumor immune evasion, but previous attempts at systemic inhibition have been by toxicity concerns.

The key differentiator here is IO Biotech's selective modulation approach through their T-win® platform. Rather than completely blocking TGF-β systemically, which can lead to serious side effects, their peptide vaccine specifically targets TGF-β+ cells within the tumor microenvironment. This selective approach could potentially overcome the safety limitations that have plagued other TGF-β targeting therapies while maintaining therapeutic efficacy.

The demonstration of efficacy in both pancreatic adenocarcinoma and prostate cancer models is particularly significant. Pancreatic cancer, with its 5-year survival rate below 10%, represents one of the most challenging oncology indications with treatment options. Similarly, advanced prostate cancer often develops resistance to existing therapies, creating a substantial market opportunity.

The progression toward an IND submission suggests confidence in the preclinical package and indicates potential clinical trials could begin within 12-18 months, assuming standard regulatory timelines. This positions IO Biotech to potentially expand its pipeline beyond its current lead programs, strengthening its market position in the competitive immuno-oncology space.

However, investors should note that while preclinical data is promising, the path to clinical validation remains long and complex. The true differentiation of IO170 from other immunotherapy approaches will only become clear in human trials, where factors such as immune response durability, combination potential, and real-world safety profile will be critical determinants of success.

• Findings support the development of a novel peptide vaccine targeting the immunosuppressive effects of Transforming Growth Beta (TGF-β) in solid tumors

NEW YORK, Feb. 23, 2025 (GLOBE NEWSWIRE) -- IO Biotech (Nasdaq: IOBT), a clinical-stage biopharmaceutical company developing novel, immune-modulatory, off-the-shelf therapeutic cancer vaccines, today announced details of its poster presentation at the American Association for Cancer Research (AACR)-IO conference taking place February 23-26, 2025, in Los Angeles, California.

Poster Presentation Details
Title: A TGF-β-directed peptide vaccine induces T cell activation & drives anti-tumor activity by modulating the architecture of the tumor microenvironment
Poster number: B117
Presenter: Matteo Bocci, PhD, Senior Scientist
Date and time: Tuesday, February 25, 2025, 1:45-4:00 PM PST
Location: JW Marriott Los Angeles, Platinum Ballroom A-E

Transforming Growth Factor Beta (TGF-β) is a key immune-suppressive factor in the tumor microenvironment (TME) of most solid tumors, often limiting the efficacy of various immune-oncology therapies. While global TGF-β inhibition strategies have shown limited success due to systemic toxicity, a more targeted modulation approach may be key to achieving sustained anti-tumor activity. IO Biotech’s proprietary T-win® platform is designed to selectively modulate TGF-β activity rather than fully inhibit it, targeting both tumor cells and key immune-suppressive cells within the TME.

The poster details the potential of IO170, a TGF-β peptide vaccine that showed significantly reduced tumor growth in pancreatic adenocarcinoma and prostate cancer models. The vaccine-induced robust immune responses reshaped the TME without causing systemic toxicity or adverse off-target effects. These findings support the continued development of an immune-modulatory vaccine targeting TGF-β as a novel approach for treating a wide range of cancers.

“Unlike other existing approaches that aim at inhibiting the TGFβ signaling systemically, our strategy uniquely targets TGFβ+ cells that are contributing to key immune resistance mechanisms in cancer,” said Ayako Wakatsuki Pedersen, PhD, Senior Vice President of Translational Research at IO Biotech. “These findings further validate our approach to immune-modulatory cancer vaccines that reshape the tumor microenvironment to enhance treatment efficacy. We look forward to advancing this research and sharing additional data as we explore its clinical applications while we prepare for an Investigational New Drug Application (IND) submission to FDA for IO170.”

About IO Biotech

IO Biotech is a clinical-stage biopharmaceutical company developing novel, immune-modulatory, off-the-shelf therapeutic cancer vaccines based on its T-win® platform. The T-win platform is based on a novel approach to cancer vaccines designed to activate T cells to target both tumor cells and the immune-suppressive cells in the tumor microenvironment. IO Biotech is advancing its lead investigational cancer vaccine candidate, Cylembio™ (imsapepimut and etimupepimut, adjuvanted) also known as IO102-IO103 in clinical trials, and additional pipeline candidates through preclinical development. Based on positive Phase 1/2 first line metastatic melanoma data, IO102-IO103, in combination with Merck’s anti-PD-1 therapy, KEYTRUDA® (pembrolizumab), has been granted a Breakthrough Therapy Designation for the treatment of advanced melanoma by the US Food and Drug Administration. IO Biotech is headquartered in Copenhagen, Denmark and has US headquarters in New York, New York.

For further information, please visit www.iobiotech.com. Follow us on our social media channels on LinkedIn and X (@IOBiotech).

Cylembio is a trademark of IO Biotech ApS.

Forward-Looking Statement

This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Forward-looking statements, including regarding the timing or outcome of primary analysis of the company’s Phase 3 trial, other current or future clinical trials, their progress, enrollment or results, or the company’s financial position or cash runway, are based on IO Biotech’s current assumptions and expectations of future events and trends, which affect or may affect its business, strategy, operations or financial performance, and actual results and other events may differ materially from those expressed or implied in such statements due to numerous risks and uncertainties. Forward-looking statements are inherently subject to risks and uncertainties, some of which cannot be predicted or quantified. Because forward-looking statements are inherently subject to risks and uncertainties, you should not rely on these forward-looking statements as predictions of future events. These forward-looking statements speak only as of the date hereof and should not be unduly relied upon. Except to the extent required by law, IO Biotech undertakes no obligation to update these statements, whether as a result of any new information, future developments or otherwise.

Contact:

Investors
Maryann Cimino, Director of Investor Relations
IO Biotech, Inc.
617-710-7305
mci@iobiotech.com

Media
Julie Funesti
Edelman
917-498-1967
julie.funesti@edelman.com


FAQ

What are the key findings from IO Biotech's (IOBT) preclinical study of IO170?

The preclinical study showed that IO170, a TGF-β peptide vaccine, significantly reduced tumor growth in pancreatic adenocarcinoma and prostate cancer models without causing systemic toxicity or adverse effects.

How does IO Biotech's IO170 differ from existing TGF-β treatments?

IO170 selectively modulates TGF-β activity rather than fully inhibiting it systemically, targeting specific TGF-β+ cells that contribute to immune resistance in cancer.

What is the next regulatory step for IO Biotech's IO170 vaccine?

IO Biotech is preparing to submit an Investigational New Drug Application (IND) to the FDA for IO170.

Which cancer types showed positive response to IO Biotech's IO170 in preclinical trials?

IO170 showed positive results in preclinical trials for pancreatic adenocarcinoma and prostate cancer models.
Io Biotech, Inc.

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