This section highlights FDA-related milestones and regulatory updates for drugs developed by IO Biotech (IOBT).
Over the past two years, IO Biotech has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
IO102-IO103, Cylembio, and IO112. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Select a button below to view the list of FDA events for that drug.
IO102-IO103 + KEYTRUDA (pembrolizumab) FDA Regulatory Timeline and Events
IO102-IO103 + KEYTRUDA (pembrolizumab) is a drug developed by IO Biotech for the following indication: First-Line Treatment in Advanced Melanoma.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- IO102-IO103 + KEYTRUDA (pembrolizumab)
- Announced Date:
- December 15, 2025
- Indication:
- First-Line Treatment in Advanced Melanoma
Announcement
IO Biotech announced the publication in Nature Communications of the long-term clinical and immunological outcomes from MM1636, the Phase 1/2 trial evaluating the investigational peptide vaccine IO102-IO103 targeting both tumor cells and immune-suppressive cells expressing IDO1 and/or PD-L1, in combination with PD-1 blockade in the treatment of first line metastatic melanoma.
AI Summary
IO Biotech announced the publication in Nature Communications of long-term clinical and immunological outcomes from MM1636, a Phase 1/2 trial testing the peptide vaccine IO102-IO103. The vaccine targets tumor cells and immune-suppressive cells that express IDO1 and PD-L1 and was given with PD-1 blockade as first-line treatment for metastatic melanoma.
The five-year follow-up showed durable clinical activity: median progression-free survival was 25.5 months, median duration of response exceeded 53 months, and median overall survival was 60 months. Researchers also identified vaccine-specific immune biomarkers linked to long-term benefit that were not seen in a matched anti-PD-1 monotherapy cohort, suggesting the vaccine induces distinct immune changes that boost anti-tumor activity.
MM1636 enrolled 30 patients at Copenhagen University Hospital, Herlev, who received IO102-IO103 plus nivolumab. The published findings support further development of immune-modulatory vaccination combined with PD-1 inhibitors.
Read Announcement- Drug:
- IO102-IO103 + KEYTRUDA (pembrolizumab)
- Announced Date:
- September 23, 2025
- Indication:
- First-Line Treatment in Advanced Melanoma
Announcement
IO Biotech announces a late-breaking abstract has been accepted and selected as a Proffered Paper oral presentation at the 2025 European Society for Medical Oncology (ESMO) Congress taking place in Berlin, Germany from October 17-21, 2025.
AI Summary
IO Biotech announced that a late-breaking abstract has been accepted as a Proffered Paper oral presentation at the 2025 European Society for Medical Oncology (ESMO) Congress, taking place in Berlin, Germany from October 17–21, 2025.
The oral session will feature primary Phase 3 results from the randomized IOB-013/KN-D18 trial. It compares the IO102-IO103 cancer vaccine plus pembrolizumab against pembrolizumab alone as first-line treatment for advanced melanoma. Dr. Jessica Hassel of the University Hospital Heidelberg will present the data on October 20.
In a poster session on October 19, final data from the Phase 2 IOB-022/KN-D38 basket trial will be shared. This includes efficacy, safety, progression-free survival, overall survival, and biomarker insights for first-line treatment of non-small cell lung cancer and head and neck cancer with the same vaccine–pembrolizumab combination.
IO Biotech’s off-the-shelf therapeutic vaccine targets IDO1 and PD-L1, aiming to boost T-cell responses against tumors and their suppressive microenvironment.
Read Announcement- Drug:
- IO102-IO103 + KEYTRUDA (pembrolizumab)
- Announced Date:
- April 25, 2025
- Indication:
- First-Line Treatment in Advanced Melanoma
Announcement
IO Biotech announced the presentation of new preclinical data for two vaccine candidates developed based on its T-win® platform at the American Association for Cancer Research (AACR) Annual Meeting 2025 in Chicago, Illinois.
AI Summary
IO Biotech presented new preclinical data for two vaccine candidates using its T-win® platform at the AACR Annual Meeting 2025 in Chicago, Illinois. The data focused on the company’s dual-antigen vaccine, IO102-IO103, and its TGF-β-directed vaccine, IO170. IO102-IO103 is designed to target cells expressing IDO1 and PD-L1, generating strong T-cell responses and modulating the tumor microenvironment in mouse models. The preclinical results showed that gene expression changes triggered by this vaccine were distinct from those observed with standard checkpoint inhibitors, suggesting a potentially synergistic mechanism for fighting tumors. Meanwhile, IO170 demonstrated significant tumor growth inhibition in breast and prostate cancer models by increasing the presence of vaccine-specific CD8+ T cells and reshaping the immune landscape within tumors. These findings offer promising insight into new strategies for enhancing anti-tumor responses.
Read Announcement- Drug:
- IO102-IO103 + KEYTRUDA (pembrolizumab)
- Announced Date:
- March 25, 2025
- Indication:
- First-Line Treatment in Advanced Melanoma
Announcement
IO Biotech announced that two abstracts have been accepted for presentation at the American Association for Cancer Research (AACR) Annual Meeting 2025 taking place April 25-30 in Chicago, Illinois.
AI Summary
IO Biotech has announced that two abstracts have been accepted for presentation at the American Association for Cancer Research (AACR) Annual Meeting 2025 in Chicago, Illinois, scheduled for April 25-30. One abstract presents new non-clinical data on IO102-IO103, the company’s lead candidate, which shows its ability to control tumor growth by activating target-specific T cells. This candidate is currently being evaluated in a pivotal Phase 3 trial in advanced melanoma. The second abstract details new preclinical findings for IO170, which targets transforming growth factor beta (TGF-beta) to drive antitumor activity. In addition, the meeting will feature an education session on cancer vaccines chaired by Dr. Mads Hald Andersen, highlighting the latest advances in vaccine research and their potential to reshape cancer immunotherapy.
Read Announcement- Drug:
- IO102-IO103 + KEYTRUDA (pembrolizumab)
- Announced Date:
- January 9, 2025
- Indication:
- First-Line Treatment in Advanced Melanoma
Announcement
IO Biotech announced that it completed enrollment in its neoadjuvant/adjuvant Phase 2 basket trial (IOB-032/PN-E40).
AI Summary
IO Biotech recently announced that it has completed enrollment ahead of schedule in its neoadjuvant/adjuvant Phase 2 basket trial, IOB-032/PN-E40, for its cancer vaccine candidate IO102-IO103. This trial combines IO102-IO103 with Merck’s KEYTRUDA® (pembrolizumab) to treat patients with resectable melanoma and squamous cell carcinoma of the head and neck. The study enrolled 93 patients across sites in the United States, Europe, and Australia.
The trial will evaluate the combination’s anti-tumor activity and safety profile, with a primary focus on achieving a major pathologic response—either a complete or near-complete reduction of viable tumor cells. Initial data from the trial are expected in 2025, marking an important step in exploring the potential benefits of this immunotherapy approach in early-stage solid tumors.
Read Announcement- Drug:
- IO102-IO103 + KEYTRUDA (pembrolizumab)
- Announced Date:
- January 9, 2025
- Indication:
- First-Line Treatment in Advanced Melanoma
Announcement
IO Biotech announced that Initial Data Expected in 2025
AI Summary
IO Biotech has completed enrollment ahead of schedule in its Phase 2 basket trial evaluating its lead cancer vaccine candidate, IO102-IO103, in combination with Merck’s KEYTRUDA. The trial targets patients with resectable melanoma and head and neck cancer, using the vaccine both before and after surgery. IO Biotech announced that initial data from this trial are expected in 2025. This early look at the vaccine’s performance aims to determine its effectiveness at reducing tumor cells and enhancing patient outcomes through a major pathological response.
The study, conducted across sites in the United States, Europe, and Australia, will evaluate important clinical outcomes including complete response rates and survival metrics. With these results and additional Phase 3 trial data for advanced melanoma also anticipated in 2025, the company is optimistic about expanding new therapeutic options for patients with difficult-to-treat cancers.
Read Announcement- Drug:
- IO102-IO103 + KEYTRUDA (pembrolizumab)
- Announced Date:
- September 14, 2024
- Indication:
- First-Line Treatment in Advanced Melanoma
Announcement
IO Biotech announced promising data from the Phase 2 basket trial of IO102-IO103, the company's lead investigational therapeutic cancer vaccine candidate, in combination with Merck's (known as MSD outside of the United States and Canada) anti-PD-1 therapy KEYTRUDA® (pembrolizumab) (IOB-022/KN-D38) at the 2024 ESMO Congress in Barcelona from September 13-17.
AI Summary
At the 2024 ESMO Congress in Barcelona, IO Biotech shared promising Phase 2 trial data for its lead therapeutic cancer vaccine candidate, IO102-IO103, used in combination with Merck’s KEYTRUDA® (pembrolizumab). The trial focused on patients with advanced squamous cell carcinoma of the head and neck (SCCHN) who had high PD-L1 expression. In a cohort of 18 efficacy-evaluable patients, the combination achieved a 44.4% overall response rate and a median progression-free survival of 6.6 months. Additionally, a 66.7% disease control rate was reported, with T-cell responses observed against both IO102 and IO103 targets. These encouraging results suggest that the IO102-IO103 and pembrolizumab combination may provide an effective, safe first-line treatment option for both HPV-positive and HPV-negative patients with recurrent or metastatic SCCHN.
Read Announcement- Drug:
- IO102-IO103 + KEYTRUDA (pembrolizumab)
- Announced Date:
- August 30, 2024
- Target Action Date:
- H1 2025
- Estimated Target Date Range:
- January 1, 2025 - June 30, 2025
- Indication:
- First-Line Treatment in Advanced Melanoma
Announcement
IO Biotech announced that The primary endpoint of progression-free survival (PFS) is projected to be reached in the first half of 2025.
AI Summary
IO Biotech announced that its ongoing Phase 3 trial evaluating the IO102-IO103 cancer vaccine in combination with pembrolizumab showed promising progress. The trial, which focuses on advanced melanoma, has been guided by an Independent Data Monitoring Committee that recommended continuing the study without modifications after reviewing safety and efficacy data. No new safety concerns were reported during the interim analysis.
A significant milestone for the trial is the projection that the primary endpoint of progression-free survival (PFS) will be reached in the first half of 2025, based on the current study data. This milestone is crucial as achieving the PFS endpoint would support the vaccine’s potential to improve treatment outcomes for patients with unresectable or metastatic melanoma. The trial continues to build on earlier findings, including a median PFS of 25.5 months observed in previous studies.
Read Announcement- Drug:
- IO102-IO103 + KEYTRUDA (pembrolizumab)
- Announced Date:
- August 30, 2024
- Indication:
- First-Line Treatment in Advanced Melanoma
Announcement
IO Biotech announced that an Independent Data Monitoring Committee (IDMC) recommended continuation of the company's pivotal Phase 3 trial (IOB-013/KN-D18; NCT05155254) of its lead investigational therapeutic vaccine, IO102-IO103, following a per-protocol interim analysis.
AI Summary
IO Biotech announced that an Independent Data Monitoring Committee (IDMC) recommended that its pivotal Phase 3 trial continue without changes. This recommendation followed a per-protocol interim analysis of safety and efficacy data from the study evaluating IO102-IO103, the company’s lead therapeutic vaccine, used in combination with KEYTRUDA for patients with advanced melanoma. The analysis, performed one year after 225 patients were randomized, showed no new safety signals. Although the interim results did not exceed a high statistical threshold needed to demonstrate superiority in overall response rate, the data supports the trial’s continuation with a primary endpoint of progression-free survival. This key endpoint is projected to be achieved in the first half of 2025, providing a promising outlook for this novel treatment option in advanced melanoma.
Read Announcement
Cylembio FDA Regulatory Timeline and Events
Cylembio is a drug developed by IO Biotech for the following indication: For the Treatment of First-line Advanced Melanoma.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Cylembio
- Announced Date:
- October 20, 2025
- Indication:
- For the Treatment of First-line Advanced Melanoma
Announcement
IO Biotech today presented detailed results from its global Phase 3 trial (IOB-013/KN-D18) of Cylembio® (imsapepimut and etimupepimut, adjuvanted), in combination with Merck's (known as MSD outside the United States and Canada) anti-PD-1 therapy KEYTRUDA® (pembrolizumab), for the first-line treatment of patients with unresectable or metastatic (advanced) melanoma.
AI Summary
At the 2025 ESMO Congress, IO Biotech presented results from its Phase 3 trial (IOB-013/KN-D18) testing Cylembio® plus pembrolizumab against pembrolizumab alone in unresectable or metastatic melanoma. In 407 patients, the combo achieved a median progression-free survival (mPFS) of 19.4 months versus 11.0 months with pembrolizumab alone (HR 0.77; 95% CI, 0.58–1.00; p=0.0558), narrowly missing statistical significance.
Benefits favored the combo across most subgroups, especially patients with PD-L1-negative tumors (16.6 vs 3.0 months; HR 0.54), BRAF V600-mutated tumors (HR 0.60), and elevated LDH (HR 0.60). Safety was similar between arms, with no increase in grade ≥3 treatment-related or immune-mediated adverse events. Mild, transient injection-site reactions were more frequent with Cylembio. Final data from a Phase 2 basket trial in lung and head & neck cancers also showed encouraging progression-free survival and a consistent safety profile.
Read Announcement- Drug:
- Cylembio
- Announced Date:
- September 29, 2025
- Indication:
- For the Treatment of First-line Advanced Melanoma
Announcement
IO Biotech today provided an update on the regulatory pathway for Cylembio® (imsapepimut and etimupepimut, adjuvanted) following a pre-BLA meeting with the U.S. Food and Drug Administration (FDA).
AI Summary
IO Biotech announced that following a pre-BLA meeting with the FDA, the agency recommended against submitting a Biologics License Application for Cylembio based on the data from the IOB-013 Phase 3 trial. Although Cylembio plus pembrolizumab showed improved progression-free survival, the results narrowly missed statistical significance. The company respects the FDA’s feedback and remains confident in Cylembio’s potential against advanced melanoma.
IO Biotech plans to continue discussions with the FDA to agree on the design of a new registrational study in first-line advanced melanoma patients. It also intends to present the IOB-013 trial results to European regulators as part of its path toward submission in the EU.
To support these efforts, IO Biotech is conserving capital and restructuring its operations. The company will reduce its staff by about half, expects a one-time charge of $1.0–1.5 million, and has funding to operate into the first quarter of 2026.
Read Announcement- Drug:
- Cylembio
- Announced Date:
- August 11, 2025
- Indication:
- For the Treatment of First-line Advanced Melanoma
Announcement
IO Biotech announces topline results from the pivotal Phase 3 trial of its investigational, immune-modulatory, off-the-shelf therapeutic cancer vaccine, Cylembio® (imsapepimut and etimupepimut, adjuvanted).
AI Summary
IO Biotech announced topline results from its pivotal Phase 3 trial of the immune-modulatory, off-the-shelf cancer vaccine Cylembio® (imsapepimut and etimupepimut, adjuvanted) in combination with Merck’s anti-PD-1 therapy pembrolizumab as a first-line treatment in advanced melanoma.
Patients treated with Cylembio plus pembrolizumab had a 23% lower risk of disease progression than those on pembrolizumab alone (HR 0.77; 95% CI 0.58–1.00; p = 0.056), achieving a median progression-free survival (PFS) of 19.4 months versus 11.0 months with pembrolizumab by itself.
In patients without prior anti-PD-1 therapy, Cylembio plus pembrolizumab improved PFS (HR 0.74; nominal p = 0.037), with median PFS of 24.8 versus 11.0 months. Patients with PD-L1–negative tumors saw PFS rise from 3.0 to 16.6 months (HR 0.54; nominal p = 0.006). A trend toward better overall survival (HR 0.79) was also seen.
The combination was well tolerated, with no new safety signals. IO Biotech plans to meet with the FDA this fall to discuss these data and define next steps for a potential regulatory submission.
Read Announcement
IO112 FDA Regulatory Events
IO112 is a drug developed by IO Biotech for the following indication: Arginase 1-Targeting Therapeutic Cancer Vaccine Candidate.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- IO112
- Announced Date:
- February 4, 2025
- Indication:
- Arginase 1-Targeting Therapeutic Cancer Vaccine Candidate
Announcement
IO Biotech announced the publication of results from a preclinical study of its second immune-modulatory therapeutic cancer vaccine candidate, IO112, targeting arginase 1 (Arg1), in the Journal for ImmunoTherapy of Cancer.
AI Summary
IO Biotech announced that preclinical study results for its second immune-modulatory cancer vaccine candidate, IO112, have been published in the Journal for ImmunoTherapy of Cancer. IO112 targets arginase 1 (Arg1), an enzyme central to immune suppression in tumors. The study showed that IO112 enhances the activation of Arg1-specific T cells, which in turn reprogram tumor-associated macrophages (TAMs) to shift from an immune-suppressive state to a more proinflammatory profile. Researchers observed that these dynamic changes in the tumor microenvironment help control tumor growth. They emphasized that reprogramming TAMs could address key hurdles in current cancer immunotherapies. These promising findings support the further clinical development of Arg1-based vaccines and the potential of IO112 to improve treatment strategies for various cancers.
Read Announcement- Drug:
- IO112
- Announced Date:
- February 4, 2025
- Indication:
- Arginase 1-Targeting Therapeutic Cancer Vaccine Candidate
Announcement
IO Biotech announced that Submission of Investigational New Drug Application (IND) to US FDA expected in 2025 –
AI Summary
IO Biotech announced plans to submit an Investigational New Drug (IND) application for its cancer vaccine candidate, IO112, to the US FDA in 2025. This important move is part of the company’s ongoing efforts to develop new immuno-therapeutic approaches for cancer treatment. Recent preclinical study results, published in the Journal for ImmunoTherapy of Cancer, showed that IO112 helps expand Arg1-specific T cells. These T cells work to change tumor-associated macrophages in a way that strengthens the body’s anti-tumor response and promotes a proinflammatory tumor environment. The planned IND submission underscores IO Biotech’s commitment to advancing IO112 into clinical development and further exploring its potential benefits for patients with cancers that use arginase 1 to evade the immune system.
Read Announcement
