On November 14, 2025 IO Biotech (Nasdaq: IOBT), a clinical-stage biopharmaceutical company developing novel, immune-modulatory, off-the-shelf therapeutic cancer vaccines, reported financial results for the third quarter of 2025 and recent business highlights.
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
"We remain keenly focused on our mission to develop novel, immune-modulatory, off-the-shelf cancer therapies for the treatment of multiple types of tumors including melanoma, lung, and head and neck cancer," said Mai-Britt Zocca, PhD, President and CEO of IO Biotech. "Although the IOB-013 study narrowly missed statistical significance on the primary PFS endpoint, the results of the study support the mechanism of action of our therapeutic cancer vaccines and, we believe, have significantly de-risked the program. We look forward to discussing the next Phase 3 study design for Cylembio with the FDA in December and remain committed to bringing Cylembio to cancer patients seeking alternative treatment options as quickly as possible."
Recent Business Highlights
•
The company presented topline results of its Phase 3 pivotal trial (IOB-013/KN-D18) evaluating the company’s lead investigational vaccine, Cylembio (imsapepimut and etimupepimut, adjuvanted), in combination with Merck’s anti-PD-1 therapy KEYTRUDA (pembrolizumab) for the treatment of advanced melanoma in an oral presentation at the 2025 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) congress. The trial evaluated Cylembio in combination with pembrolizumab vs. pembrolizumab alone as a first-line treatment in 407 patients with unresectable or metastatic (advanced) melanoma. In the study, Cylembio plus pembrolizumab demonstrated a clinically relevant improvement in progression free survival (PFS) compared to pembrolizumab alone, which was also observed in virtually all subgroups, but the trial narrowly missed statistical significance.
•
The company has a meeting with the United States (US) Food and Drug Administration (FDA) scheduled in December to align on the design of a potential new Phase 3 registrational trial for the treatment of advanced melanoma.
•
The company also presented a poster at ESMO (Free ESMO Whitepaper) of final data from the Phase 2 basket trial (IOB-022/KN-D38) of IO102-IO103 plus pembrolizumab for 1L treatment of advanced non-small cell lung cancer (NSCLC) and recurrent/metastatic squamous cell carcinoma of head and neck (SCCHN) highlighting encouraging landmark PFS and overall survival (OS) rates.
•
The company presented two posters at the SITC (Free SITC Whitepaper) annual meeting with pre-clinical data for two additional T-win vaccine candidates. Data presented in a poster for its next therapeutic vaccine candidate expected to enter clinical development, IO112 targeting arginase 1, demonstrated anti-tumor activity with dynamic changes in the tumor microenvironment (TME) driven by the vaccine-targeted modulation of immunosuppressive myeloid cells, including tumor-associated macrophages (TAMs). Data presented in the poster for an additional candidate, IO170 targeting Transforming Growth Factor (TGF)-ß, demonstrated induction of immune responses that could inhibit tumor growth and reduce lung metastasis.
Investor Conferences Participation
•
Jefferies Global Healthcare Conference in London: Mai-Britt Zocca, PhD, President and CEO, will give a company presentation beginning at 3:00 PM GMT on November 18, 2025.
•
Piper Sandler 37th Annual Piper Sandler Healthcare Conference: Mai-Britt Zocca, PhD, President and CEO, and Amy Sullivan, CFO, will participate in a fireside chat beginning at 3:00 PM ET on December 3, 2025.
The webcasts for these two upcoming conferences will be available from the Investors section of the company’s website at View Source
Third Quarter 2025 Financial Results
•
Total operating expenses for the three months ended September 30, 2025, were $19.4 million, compared to $26.5 million for the three months ended September 30, 2024.
•
Research and development expenses were $13.7 million for the three months ended September 30, 2025, compared to $20.2 million for the three months ended September 30, 2024. The company recognized $0.6 million in research and development equity-based compensation for the three months ended September 30, 2025 and 2024, respectively.
•
General and administrative expenses were $5.6 million for the three months ended September 30, 2025, compared to $6.3 million for the three months ended September 30, 2024. The company recognized $0.9 million in general and administrative equity-based compensation for the three months ended September 30, 2025 and $1.0 million for the three months ended September 30, 2024.
•
Cash and cash equivalents as of September 30, 2025 were $30.7 million, compared to $60.0 million at December 31, 2024. During the three months ended September 30, 2025, the company increased cash, cash equivalents and restricted cash by $2.5 million primarily due to the draw down of €12.5 million in gross proceeds from our Tranche B loan with the European Investment Bank on July 4, 2025 and net proceeds of $6.6 million from the issuance of common stock in connection with our at-the-market program. The company now expects that it will have sufficient cash to run the company through the first quarter of 2026.
About Cylembio
Cylembio (imsapepimut and etimupepimut, adjuvanted) is an investigational, immune-modulatory, off-the-shelf therapeutic cancer vaccine candidate designed to kill both tumor cells and immune-suppressive cells in the tumor microenvironment (TME) by stimulating activation and expansion of T cells against indoleamine 2,3-dioxygenase 1 (IDO1) positive and/or programmed death-ligand 1 (PD-L1) positive cells. The company is currently conducting a pivotal Phase 3 trial (IOB-013/KN-D18; NCT05155254) investigating Cylembio in combination with Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab) versus pembrolizumab alone in patients with advanced melanoma, a Phase 2 basket trial (IOB-022/KN-D38; NCT05077709) investigating Cylembio in combination with pembrolizumab as first line treatment in patients with advanced solid tumors, and a Phase 2 basket trial (IOB-032/PN-E40; NCT05280314) investigating Cylembio in combination with pembrolizumab as neo- adjuvant/adjuvant treatment of patients with solid tumors. Enrollment in the Phase 3 trial was completed rapidly by December 2023 with topline results from this trial reported in the third quarter of 2025. Enrollment in the two ongoing company-sponsored Phase 2 clinical trials is now complete.
The clinical trials are sponsored by IO Biotech and conducted in collaboration with Merck, which is supplying pembrolizumab. IO Biotech maintains global commercial rights to Cylembio.
Cylembio is a registered trademark of IO Biotech ApS, a subsidiary of IO Biotech.
KEYTRUDA is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA (known as MSD outside of the US and Canada).
About the IOB-013/KN-D18 Pivotal Phase 3 Clinical Trial
IOB-013/KN-D18 (ClinicalTrials.gov: NCT05155254) is an open label, randomized Phase 3 pivotal clinical trial evaluating Cylembio in combination with Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab) versus pembrolizumab alone in patients with previously untreated, unresectable or metastatic (advanced) melanoma. Enrollment in the trial was completed by December 2023 with a total of 407 patients enrolled from more than 100 centers across the United States, Europe, Australia, Turkey, Israel and South Africa. The primary endpoint of the study was progression-free survival. Secondary endpoints include overall response rate, overall survival, durable objective response rate, complete response rate, duration of response, time to complete response, disease control rate, and incidence of adverse events and serious adverse events (safety and tolerability). Biomarkers in the blood and tumor tissue will also be assessed as exploratory endpoints. The company reported topline results from this trial in the third quarter of 2025. IO Biotech is sponsoring the Phase 3 trial and Merck is supplying pembrolizumab.
About IOB-022/KN-D38 Phase 2 Solid Tumor Basket Trial
IOB-022/KN-D38 (NCT05077709) is a non-comparative, open label trial to investigate the safety and efficacy of Cylembio in combination with Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab) in the first-line treatment of metastatic non-small cell lung cancer (NSCLC) or recurrent/metastatic squamous cell carcinoma of the head and neck (SCCHN) at sites in the United States, Spain, and the United Kingdom. IO Biotech is sponsoring the Phase 2 trial and Merck is supplying pembrolizumab.
(Press release, IO Biotech, NOV 14, 2025, View Source [SID1234659972])