On August 14, 2025 IO Biotech (Nasdaq: IOBT), a clinical-stage biopharmaceutical company developing novel, immune-modulatory, off-the-shelf therapeutic cancer vaccines, reported financial results and business highlights for the second quarter of 2025 (Press release, IO Biotech, AUG 14, 2025, View Source [SID1234655304]).
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"In the second quarter, we achieved a significant milestone advancing our pipeline of novel cancer therapies with the readout of the topline results of the Phase 3 trial for Cylembio, our potentially first-in-class, immune-modulatory, off-the-shelf therapeutic cancer vaccine, being investigated in advanced melanoma," said Mai-Britt Zocca, PhD, President and CEO of IO Biotech. "With the notable safety profile and clinical improvement observed in this trial, we are focused on discussing the results with the FDA and determining the next steps for a potential submission of a Biologics License Application (BLA) for the treatment of advanced melanoma."
Recent Business Highlights
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The company announced 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 (known as MSD outside of the United States and Canada) anti-PD-1 therapy KEYTRUDA (pembrolizumab) for the treatment of advanced melanoma. 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 clinical improvement in progression free survival (PFS) compared to pembrolizumab alone, but statistical significance was narrowly missed on the PFS primary endpoint. Based on these results, IO Biotech plans to meet with the United States (US) Food and Drug Administration (FDA) this fall to discuss the totality of data and determine next steps for a potential submission of a Biologics License Application (BLA) for the treatment of advanced melanoma.
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The company continues to expect initial data from the perioperative Phase 2 solid tumor basket trial (IOB-032/PN-E40), studying treatment with Cylembio in combination with pembrolizumab in patients with resectable squamous cell carcinoma of the head and neck (SCCHN) and melanoma that completed enrollment in January, as well as longer-term data from the company’s other ongoing Phase 2 basket trial, IOB-022/KN-D38, in patients with advanced SCCHN or non-small cell lung cancer (NSCLC), to be available in the second half of 2025 and presented at a medical meeting in 2026.
Upcoming Investor Conferences Participation
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Morgan Stanley 23rd Annual Global Healthcare Conference: Mai-Britt Zocca, PhD, President and CEO, Amy Sullivan, CFO, and Qasim Ahmad, MD, CMO, will participate in a fireside chat beginning at 7:45 AM ET on September 9, 2025.
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H.C. Wainwright 27th Annual Global Investment Conference: Mai-Britt Zocca, PhD, President and CEO, will give a company presentation beginning at 8:00 AM ET on September 10, 2025.
The webcasts for these two upcoming conferences will be available from the Investors section of the company’s website at View Source
Second Quarter 2025 Financial Results
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Net loss for the three months ended June 30, 2025, was $26.2 million, compared to $20.7 million for the three months ended June 30, 2024.
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Research and development expenses were $16.7 million for the three months ended June 30, 2025, compared to $15.8 million for the three months ended June 30, 2024. The company recognized $0.6 million in research and development equity-based compensation for the three months ended June 30, 2025, compared to $0.7 million for the three months ended June 30, 2024.
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General and administrative expenses were $6.5 million for the three months ended June 30, 2025, compared to $5.7 million for the three months ended June 30, 2024. The company recognized $1.0 million in general and administrative equity-based compensation for the three months ended June 30, 2025 and 2024, respectively.
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Cash and cash equivalents as of June 30, 2025 were $28.1 million, compared to $60.0 million at December 31, 2024. During the three months ended June 30, 2025, the company used cash, cash equivalents and restricted cash of $9.0 million. The company has drawn down on the Tranche A and Tranche B loan facility in principal amount of €10.0 million and €12.5 million, respectively, on May 6, 2025 and July 4, 2025, each before payment of certain fees and transaction related expense. With the funds received from the second tranche of the EIB loan facility on July 4th, the company expects that it will have sufficient cash to run the company into 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 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.
About IOB-032/PN-E40 Phase 2 Solid Tumor Basket Trial
IOB-032/PN-E40 (NCT05280314) is a multicenter Phase 2 basket trial investigating Cylembio in combination with Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab) as neo-adjuvant/adjuvant treatment of patients with solid tumors at sites in Australia, the United States, France, Germany, Spain, and Denmark. The study completed enrollment in all cohorts: 18 patients with melanoma in cohort A and 16 patients with SCCHN in cohort B, both as single arm cohorts receiving combination of Cylembio with pembrolizumab. In cohort C, 61 melanoma patients were randomized 1:1 to either the combination of Cylembio with pembrolizumab or pembrolizumab alone. In the neo-adjuvant period, for all cohorts, treatment is every 3 weeks (Q3W) for 3 cycles (melanoma) or 2-3 cycles (SCCHN). Patients entering the study will be scheduled for surgery and begin neoadjuvant treatment 4-9 weeks prior. Surgery will be followed by adjuvant treatment with the same regimen for 15 cycles. Cohort C patients with poor pathological response to pembrolizumab alone in the neo-adjuvant phase (>10% residual viable tumor) may cross over to combination treatment post-surgery. The primary endpoint is major pathological response at surgery (≤10% residual viable tumor; central assessment). IO Biotech is sponsoring the Phase 2 trial and Merck is supplying pembrolizumab.