BlossomHill Therapeutics Presents Initial Clinical Data from Dose Escalation in Ongoing Phase 1/1b Trial of Novel Macrocyclic CLK Inhibitor, BH-30236, in Patients with R/R AML or HR-MDS at EHA 2026

On June 11, 2026 BlossomHill Therapeutics, Inc., a privately-held, clinical-stage biopharmaceutical company applying an intentional, chemistry-based approach to design and develop innovative small molecule medicines for the treatment of cancer, reported initial clinical data from its ongoing first-in-human Phase 1/1b trial of BH-30236 in relapsed or refractory acute myeloid leukemia (R/R AML) and higher-risk myelodysplastic syndrome (HR-MDS) at the European Hematology Association (EHA) (Free EHA Whitepaper) 2026 Congress in Stockholm, Sweden. BH-30236 is an intentionally designed novel, orally bioavailable, macrocyclic CDC-like kinase (CLK) inhibitor that modulates aberrant alternative mRNA splicing, a defining feature implicated in cancer progression and therapeutic resistance across both hematologic malignancies and solid tumors.

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"The preliminary anti-leukemic activity observed with BH-30236 in R/R AML and HR-MDS, both as a monotherapy and in combination with venetoclax, is very encouraging," said Geoff Oxnard, M.D., Chief Medical Officer of BlossomHill Therapeutics. "In addition to the reassuring tolerability profile, we have seen provocative responses in patients after progression on prior venetoclax-based therapy, a population with few treatment options. In the ongoing study of BH-30236, we will further characterize the effect of this novel mechanism in myeloid malignancies, both as a monotherapy and in synergy with venetoclax to maximize the potential of this promising program."

"We intentionally designed BH-30236 to target CLK, which is upstream of multiple resistance pathways, not by addressing one resistance mechanism after it develops, but by suppressing the splicing machinery that enables resistance to begin," said Jean Cui, Ph.D., Founder and Chief Executive Officer of BlossomHill Therapeutics. "We believe these data further validate our approach of leveraging a deep understanding of disease and protein dynamics and applying our structure-based rational drug design expertise to identify specific structural liabilities that limit existing therapies or approaches and design novel chemical scaffolds to directly address these limitations."

Presentation Highlights:

As of the data cutoff date of April 10, 2026, 30 patients received BH-30236 monotherapy at doses ranging from 5 to 120 mg on a continuous daily administration schedule (QD), and 18 patients received BH-30236 at doses ranging from 20 to 90 mg QD in combination with venetoclax
BH-30236 was generally well tolerated as both a monotherapy and in combination with venetoclax, with most treatment-related adverse events being low-grade and manageable, with one dose limiting toxicity of Grade 3 diarrhea at the 120 mg QD monotherapy dose level
Dose escalation showed predictable pharmacokinetics without drug accumulation, and no significant drug-drug interactions were observed with venetoclax
With efficacy follow-up through May 20, 2026, preliminary anti-leukemic activity was observed in patients with R/R AML and HR-MDS when treated with BH-30236 as a monotherapy or in combination with venetoclax, including in patients previously relapsed or refractory to venetoclax-based therapy:
In the monotherapy cohort, 28.6% (n=6) of evaluable patients achieved at least a 50% reduction in bone marrow blast counts, including one HR-MDS patient treated at 60 mg with ongoing blast count reduction with treatment ongoing for more than a year
In the combination cohort, 87% (n=13) of patients had prior venetoclax exposure and 53% (n=8) of evaluable patients experienced at least a 50% blast reduction, including one patient refractory to all prior therapy including venetoclax, who achieved a minimal residual disease (MRD)-negative complete remission

About R/R AML and HR-MDS
AML is the most common acute leukemia in adults, with approximately 21,000 new diagnoses annually in the United States, with as many as 75% of those being patients resistant to or relapsing on first-line venetoclax therapy. Approximately 14,000 patients are diagnosed with HR-MDS annually in the United States. There is no currently approved targeted therapy for the venetoclax-resistant and refractory segment of AML patients without targetable genetic alterations. Patients with HR-MDS closely mirror AML in clinical course, frontline treatment, and unmet need in the relapsed and refractory setting.

About BH-30236
BH-30236 is an investigational orally bioavailable, macrocyclic inhibitor of the CDC-like kinase (CLK) family. BH-30236 is was intentionally designed to potently inhibit CLK, leading to modulation of aberrant alternative splicing in cancerous tissue, targeting the same aberrant splicing machinery that drives relapsed or refractory (R/R) acute myeloid leukemia (AML) and higher-risk myelodysplastic syndrome (HR-MDS) disease biology and that cancer cells exploit to develop resistance to venetoclax, FLT3 inhibitors and cytarabine.

BH-30236 is being evaluated in a Phase 1/1b multicenter, open-label, first-in-human dose escalation and expansion trial in adults with R/R AML and HR-MDS. The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to BH-30236 for the treatment of AML.

(Press release, BlossomHill Therapeutics, JUN 11, 2026, View Source [SID1234666595])

Curium Group, PeptiDream and PDRadiopharma Announce Completion of Patient Dosing in Clinical Trial of 64Cu-PSMA-I&T for Prostate Cancer in Japan

On June 11, 2026 Curium Group reported that together with PeptiDream Inc. and PDRadiopharma Inc., patient dosing has been completed in the registrational Phase 2 clinical trial for 64Cu-PSMA-I&T in Japan.

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64Cu-PSMA-I&T is a PET radiopharmaceutical targeting PSMA. 64Cu-PSMA-I&T is being assessed as a PET agent labeled with the radioisotope Copper-64. The trial is conducted under the strategic collaboration between PDRadiopharma, a wholly owned subsidiary of PeptiDream, and Curium, aiming at advancing innovative diagnostic radiopharmaceuticals for prostate cancer in Japan.

The open-label, single-arm Phase 2 study is designed to evaluate the sensitivity, specificity, and safety of 64Cu-PSMA-I&T in Japanese patients newly diagnosed with unfavorable intermediate, high or very high-risk prostate cancer and are scheduled for prostatectomy with pelvic lymph node dissection. The results from this study, together with data from Curium’s ongoing global clinical trials, are expected to support future regulatory submission in Japan.

In parallel, Curium announced in February, a registrational clinical trial of the therapeutic counterpart, 177Lu-PSMA-I&T, is being advanced to evaluate its efficacy and safety in patients with metastatic castration-resistant prostate cancer (mCRPC), as part of a theranostic approach.

Renaud Dehareng, CEO of Curium Group commented: "Conducting these trials, in partnership with PeptiDream and PDRadiopharma, marks a significant milestone in our mission to expand access to cutting-edge radiopharmaceuticals to patients with prostate cancer across Asia. By combining Curium’s global development expertise with PDRadiopharma’s deep local knowledge and infrastructure, we are well-positioned to deliver transformative solutions to patients with prostate cancer in Japan."

Patrick C. Reid, President & CEO of PeptiDream commented: "The completion of patient dosing marks an important milestone in the development of 64Cu-PSMA-I&T in Japan. This program represents a key component of our growing radiopharmaceutical pipeline and our broader theranostics strategy. We would like to thank the patients, investigators and clinical sites for their participation and support."

About Prostate Cancer

Prostate cancer continues to be widely prevalent in Japan. Annually, there are approximately 90,000 – 100,000 new cases (*1). *1: National Cancer Center Japan

Clinical trial progress

Phase 3 ECLIPSE trial – 177Lu-PSMA-I&T, a PSMA-targeting ligand conjugated with the radioisotope Lutetium-177, has been tested by Curium in a global pivotal Phase 3 ECLIPSE trial (ClinicalTrials.gov identifier; NCT05204927). It reported that the primary endpoint was met, demonstrating a statistically significant and clinically meaningful benefit for patients with mCRPC.

Phase 3 SOLAR RECUR and SOLAR STAGE trial – 64Cu-PSMA-I&T trials are being conducted to diagnose biochemical recurrence of prostate cancer (SOLAR RECUR trial, NCT06235099) and for men newly diagnosed with unfavorable intermediate to very high-risk prostate cancer, electing to undergo surgery (SOLAR STAGE trial, NCT06235151). The first in human Phase 1/2 SOLAR trial met the co-primary endpoints of region-level correct localization rate and patient-level correct detection rate in patients with histologically-proven prostate cancer.

Partnership Details

Under the terms of the partnership, Curium and PDRadiopharma will jointly collaborate on clinical development activities of 64Cu-PSMA-I&T and 177Lu-PSMA-I&T and in Japan, with PDRadiopharma leading regulatory filing, manufacturing, commercialization, and distribution activities in Japan. Curium will continue to lead global development of the two agents and support PDRadiopharma through technology transfer to support the set-up of manufacturing lines in Japan – including a high throughput Copper 64 manufacturing line based on Curium’s proprietary technology.

(Press release, Curium Pharma, JUN 11, 2026, View Source [SID1234666564])

Anixa Biosciences Announces Presentations of Two Clinical-Stage Immunotherapy Programs at New York Academy of Sciences’ Frontiers in Cancer Immunotherapy Symposium

On June 11, 2026 Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, reported that two of its clinical-stage immunotherapy programs will be presented at the New York Academy of Sciences’ Frontiers in Cancer Immunotherapy symposium, being held June 22 – 23, 2026, at Memorial Sloan Kettering Cancer Center in New York City.

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The presentations will highlight Anixa’s ovarian cancer CAR-T therapy, liraltagene autoleucel, or lira-cel, which is being evaluated in an ongoing Phase 1 clinical trial, and Anixa’s breast cancer vaccine program, which completed a Phase 1 clinical trial in which all major primary endpoints were met and protocol-defined immune responses were generated in 74% of participants.

Frontiers in Cancer Immunotherapy brings together leading researchers, clinicians and industry innovators to explore next-generation therapies that are transforming cancer treatment. The symposium will showcase research and emerging approaches in areas including cancer vaccines, bispecifics, modulation of the tumor microenvironment, AI-driven discovery of immunotherapy and cell-based therapies for solid tumors. Additionally, Dr. Stephen Schoenberger, PhD, a world-renowned immunologist at the La Jolla Institute for Immunology and a member of Anixa’s Clinical Advisory Board, will be a featured speaker at the symposium.

"Presenting both of our clinical-stage immunotherapy programs at this symposium is an important opportunity to highlight the progress of our pipeline and the strength of the collaborations supporting these programs," said Dr. Amit Kumar, PhD, Chairman and CEO of Anixa Biosciences. "Our ovarian cancer CAR-T therapy and breast cancer vaccine program are being advanced with leading clinical and scientific institutions, and we believe these presentations underscore the potential of Anixa’s approach to treating and preventing cancer."

The Company’s lira-cel presentation, titled "A Phase I Clinical Trial of an Infusion of Autologous T cells Genetically Engineered with a Chimeric Receptor to Target the Follicle-Stimulating Hormone Receptor in Patients with Recurrent Ovarian Cancer," will be given by Dr. Pamela D. Garzone, PhD, Chief Development Officer of Anixa Biosciences. The presentation will report the clinical trial design and objectives, as well as the current status of Anixa’s ongoing Phase 1 clinical trial of lira-cel.

The lira-cel presentation reflects contributions from investigators and collaborators across Anixa, Moffitt Cancer Center, Roswell Park Cancer Center, the University of Chicago School of Medicine and Duke University Medical Center. Project team members cited on the poster include:

Dr. Marco L. Davila, MD, PhD (Department of Medicine, Roswell Park Cancer Center)
Dr. Daniel Abate-Daga, PhD (Department of Immunology, Moffitt Cancer Center)
Dr. Melissa McGettigan, MD (Department of Radiology, Moffitt Cancer Center)
Dr. Xuefeng Wang, PhD (Department of Biostatistics and Bioinformatics, Moffitt Cancer Center)
Dr. Theresa Boyle, MD, PhD (Department of Pathology, Moffitt Cancer Center)
Dr. Amit Kumar, PhD (Anixa Biosciences, Inc.)
Denise Dorman, RN (Department of Gynecologic Oncology, Moffitt Cancer Center)
Dr. Richard Koya, MD, PhD (University of Chicago School of Medicine)
Dr. Christopher Cubitt, PhD (Immune Monitoring Core, Moffitt Cancer Center)
Dr. Jose Conejo-Garcia, MD, PhD (Department of Immunology, Duke University Medical Center)
Dr. Robert M. Wenham, MD, MS (Principal Investigator, Department of Gynecologic Oncology, Moffitt Cancer Center)
The Company’s breast cancer vaccine presentation, titled "Phase I Trial of an Alpha-Lactalbumin (aLA) Vaccine for Breast Cancer," will be given by Dr. Emily Esakov Rhoades, PhD, FDA/IND Trial Program Manager, Cleveland Clinic Cancer Institute. The presentation will report final Phase 1 findings for the investigational vaccine, including that all major primary endpoints were met, the vaccine was safe and well tolerated at the maximum tolerated dose based on safety and tolerability, and protocol-defined immune responses were elicited in 74% of trial participants. Immunohistochemistry of tumor samples demonstrating alpha-lactalbumin expression will also be reported.

The breast cancer vaccine presentation reflects contributions from Cleveland Clinic clinicians, translational researchers, patient advocates, and collaborators involved in advancing the program. The Cleveland Clinic team includes:

Dr. Justin M. Johnson, PhD (Department of Inflammation & Immunity, Cleveland Clinic)
Holly B. Levengood (Department of Inflammation & Immunity, Cleveland Clinic Cancer Institute)
Dr. Azka Ali, MD (Cleveland Clinic Cancer Institute)
Dr. Hannah Gilmore, MD (Robert J. Tomsich Pathology and Laboratory Medicine Institute)
Dr. Megan L. Kruse, MD (Cleveland Clinic Cancer Institute)
Dr. Erin E. Roesch, MD (Cleveland Clinic Cancer Institute)
Dr. Tiffany Onger, MD (Cleveland Clinic Cancer Institute)
Brenna Elliott (Cleveland Clinic Cancer Institute)
Elena Haury (Cleveland Clinic Cancer Institute)
Carolyn Porvasnik (Cleveland Clinic Cancer Institute)
Tobey Young (Previvorsandsurvivors.com, Inc.)
Terri Coutee (DiepCJourney Foundation)
Judith A. Fitzgerald, BCPA (Sisters4Prevention)
Dr. Thaddeus S. Stappenbeck, MD, PhD (Co-Principal Investigator, Chair – Department of Inflammation & Immunity, Cleveland Clinic)
Dr. G. Thomas Budd, MD (Principal Investigator, Cleveland Clinic Cancer Institute)
About Lira-cel, Anixa’s CAR-T Therapy for Recurrent Ovarian Cancer
Liraltagene autoleucel, or lira-cel, uniquely targets the follicle-stimulating hormone receptor (FSHR), which is selectively expressed on ovarian and testis cells, tumor vasculature, and certain cancer cells, but not in other healthy tissue. The ongoing Phase 1 trial (ClinicalTrials.gov NCT05316129) is enrolling adult women with recurrent ovarian cancer who are platinum resistant and have progressed after at least two prior therapies.

(Press release, Anixa Biosciences, JUN 11, 2026, View Source [SID1234666580])

C4 Therapeutics Presents Phase 1 Data at European Hematology Association (EHA) 2026 Congress Highlighting Cemsidomide as a Potential Best-in-Class IKZF1/3 Degrader for Multiple Myeloma in Heavily Pretreated Relapsed/Refractory Population

On June 11, 2026 C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a clinical-stage biopharmaceutical company dedicated to advancing targeted protein degradation (TPD) science, reported it will present further analysis from its fully enrolled Phase 1 trial of cemsidomide, a next-generation oral IKZF1/3 degrader, in combination with dexamethasone for the treatment of relapsed/refractory multiple myeloma (RRMM) in a poster presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) 2026 Congress on Friday, June 12, 2026 at 6:45 pm CEST / 12:45 pm ET.

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The analysis is consistent with previous data disclosed from the Phase 1 clinical trial and highlights cemsidomide’s anti-myeloma activity and differentiated safety profile, further supporting its development as a potential best-in-class IKF1/3 degrader. The poster will be presented by Sagar Lonial, M.D., FACP, FASCO, chief medical officer at the Winship Cancer Institute at Emory University, and an investigator in the cemsidomide clinical trials.

"Despite advances in multiple myeloma therapies, IKZF1/3 degradation remains a foundational treatment strategy across lines of therapy because it is the only approach that addresses the underlying biology of the disease and has the built-in ability to stimulate the immune function, becoming a natural partner for immune therapies. Next-generation IKZF1/3 degraders are expected to help advance treatment regimens for this persistent disease, given data demonstrating their efficacy and tolerability," said Dr. Lonial. "The clinical activity and safety profile of cemsidomide are highly encouraging for patients with relapsed/refractory multiple myeloma as they continue to seek disease-altering treatment options. The data from the ongoing Phase 1 study support the continued development of cemsidomide for patients with relapsed/refractory multiple myeloma who may benefit from IKZF1/3 degradation."

"The totality of cemsidomide data, particularly data showing that patients have experienced a deepening response over time, continue to demonstrate its potential to deliver a tolerable therapy that can provide sustained benefit for patients who have progressed through other treatment options," said Len Reyno, M.D., chief medical officer of C4 Therapeutics. "We remain focused on advancing our clinical development strategy to capitalize upon cemsidomide’s differentiated profile in hopes patients at various stages of their treatment journey may be able to benefit from this important investigational therapeutic regimen."

The poster presentation includes data on 73 patients with a data cutoff of February 27, 2026. Patients were heavily pretreated, receiving a median of seven prior lines of therapy. Fifty-five patients (75%) received prior BCMA therapy, and 55 patients (75%) received prior CAR-T or T-cell engager therapy (TCE).

At the RP2D and maximum tolerated dose (100 µg,) cemsidomide achieved a 53% overall response rate (ORR). At the 75 µg dose level, cemsidomide achieved a 40% ORR. Across all doses evaluated, cemsidomide achieved a 36% ORR.

Key new data include:

Responses deepened over time across the cemsidomide 75 µg and 100 µg dose levels:
At 75 µg, one patient whose best response was previously a partial response (PR) deepened to a very good partial response (VGPR).
At 100 µg, several patients achieved a deeper response:
One patient whose best response was previously a PR deepened to a stringent complete response (sCR)
One patient whose best response was previously a PR deepened to a VGPR
Minimal residual disease (MRD) negativity was achieved in two patients who achieved a sCR and complete response (CR) at 100 µg.
ORR was consistent across key subgroups:

ORR % (95% confidence interval (CI))
All Doses
Prior CAR-T or TCE 37% (24, 51)
Prior BCMA 33% (21, 48)
Prior Lines of Therapy > 5 Lines 33% (20, 48)
100 µg (RP2D)
Prior CAR-T or TCE 53% (28, 77)
Prior BCMA 47% (21, 73)
Prior Lines of Therapy > 5 Lines 47% (21, 73)

Durable responses were observed across all dose levels:
Patients experienced a median duration of response of 7.9 months (95% CI, 3.0 – non-evaluable).
Seven patients remain on treatment currently.

Cemsidomide in combination with dexamethasone was generally well tolerated. Incidences of on-target neutropenia remained manageable; 42 patients (58%) experienced Grade 3/4 neutropenia. All treatment emergent adverse events were manageable with no discontinuations deemed related to cemsidomide and minimal dose reductions (five patients; 7%).

UPCOMING INVESTOR EVENTS

June 18, 2026 at 9 am ET: C4T will host an educational webinar with Nisha Joseph, M.D., associate professor at the Winship Cancer Institute at Emory University and investigator in the cemsidomide clinical trials to discuss the evolving multiple myeloma landscape, the role of IKZF1/3 degradation in treating multiple myeloma, and cemsidomide’s profile.

About Cemsidomide
Cemsidomide is an investigational, next-generation orally bioavailable MonoDAC degrader (molecular glue) of IKZF1/3, transcription factors foundational to multiple myeloma biology. Data from the fully enrolled Phase 1 trial show cemsidomide’s differentiated safety and tolerability profile and potentially class-leading anti-myeloma activity that support the potential for durable outcomes.

About Multiple Myeloma
Multiple myeloma is a blood cancer that affects plasma cells in the bone marrow. It is the second most common blood cancer, with approximately 36,000 people in the United States diagnosed each year. Multiple myeloma is characterized by cycles of remission and relapse, which leads to patients needing multiple lines of therapy to manage this persistent disease. More than 175,000 patients in the United States are estimated to be living with or in remission from myeloma. However, despite treatment advances, approximately 40% of patients do not survive beyond five years.

About IKZF1/3 Degradation
Targeted degradation of IKZF1 (Ikaros) and IKF3 (Aiolos) is a foundational therapeutic strategy to treat multiple myeloma, a blood cancer affecting plasma cells. IKZF1/3 degradation leads to downregulation of IRF4, which promotes myeloma cell death. IKZF1/3 degradation also activates T-cells, which contributes to broader anti-myeloma response. For decades, IKZF1/3 degradation has been used in approved therapies for multiple myeloma. Next-generation IKZF1/3 degraders are being developed to leverage advances in targeted protein degradation research while continuing to address the biology foundational to multiple myeloma.

About the MOMENTUM Trial
MOMENTUM (Multi-center trial Of cemsidoMidE iN relapsed/refracTory mUltiple Myeloma) is a Phase 2, open-label, single-arm study to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of cemsidomide in combination with dexamethasone in patients with relapsed/refractory multiple myeloma. Data from the Phase 1 trial identified 100 µg as the recommended Phase 2 dose. The primary endpoint is overall response rate per International Myeloma Working Group response criteria, as assessed by an independent review committee. Approximately 100 patients who have received at least three prior anti-myeloma regimens that must have included an IKZF1/3 degrader, a proteasome inhibitor, an anti-CD38 antibody, and a T-cell engager or CAR-T therapy will be enrolled in the trial. More information is available at clinicaltrials.gov (NCT07284758).

About Cemsidomide in Combination With Elranatamab (ELREXFIO)
The Phase 1b trial is designed to evaluate the safety, tolerability and preliminary efficacy of cemsidomide and dexamethasone in combination with elranatamab, an FDA-approved B-cell maturation antigen CD3 targeted bispecific antibody. Data generated from the cemsidomide Phase 1 trial in relapsed/refractory multiple myeloma demonstrate robust T-cell activation and cytokine expression across multiple doses. By activating immune T-cells, cemsidomide, when combined with a BCMAxCD3 bispecific such as elranatamab, may amplify the anti-myeloma immune response and lead to deeper and more durable responses. The study will evaluate different cemsidomide dose levels (beginning with 75 µg, with the opportunity to simultaneously explore 50 µg and 100 µg) in patients who have received one to four prior lines of therapy, which must have consisted of at least one IKZF1/3 degrader. Exclusion criteria for patients include those who have received prior treatment with a BCMA-directed T-cell engager or BCMA-directed CAR-T therapy. More information is available at clinicaltrials.gov (NCT07280013).

(Press release, C4 Therapeutics, JUN 11, 2026, View Source [SID1234666596])

Evaxion presents new preclinical data for EVX-04, an off-the-shelf therapeutic vaccine for acute myeloid leukemia

On June 11, 2026 Evaxion A/S (NASDAQ: EVAX) ("Evaxion"), a clinical-stage TechBio company developing novel vaccines with its pioneering AI-Immunology platform, reported new preclinical data for EVX-04, an off-the-shelf therapeutic vaccine for acute myeloid leukemia (AML). Developed with AI-Immunology, EVX-04 targets multiple non-conventional endogenous retrovirus (ERV) tumor antigens from the dark genome.

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The new data demonstrates EVX-04’s complete expression in human cells, including correct transcription and translation. Further, EVX-04 is secreted in human cells, enabling immune recognition and activation.

The data also shows that all ERV antigens included in EVX-04 drive specific immune responses both in mice (in vivo) and human cells (in vitro) across different human immune profiles. These vaccine-induced immune cells mediate targeted cell-killing, highlighting EVX-04’s potential as a new effective therapeutic cancer vaccine.

Data will be presented at a poster presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) 2026 Congress taking place in Stockholm, Sweden, on June 13, 2026.

"We are excited to share the new data on EVX-04, which could potentially greatly improve treatment options for AML patients. We are successfully executing the preclinical activities and in parallel preparing the regulatory filing for clinical testing and are looking forward to discussing the data and the program at the EHA (Free EHA Whitepaper) congress," says Birgitte Rønø, CSO & COO of Evaxion.

About EVX-04
Developed with our AI-Immunology platform, EVX-04 targets non-conventional endogenous retrovirus (ERV) tumor antigens from the dark genome. These antigens are selectively expressed in specific tumors but absent in normal tissue, making them highly attractive therapeutic cancer targets.

Using sequencing data from AML patients, our AI-Immunology platform first identified ERV tumor antigens and then mined these to determine smaller fragments with the potential for immune recognition. From the five million ERV antigen fragments discovered, AI-Immunology combined and selected 16 optimal sets of ERV fragments based on their cross-patient relevance and immunogenic potential. All 16 ERV fragments included in EVX-04 elicit a specific immune response and EVX-04 prevents tumor growth in preclinical tumor models.

The data-driven target selection ensures that EVX-04 provides broad tumor coverage regardless of immune and tumor ERV antigen differences across patients. Thus, EVX-04 is developed as an off-the-shelf vaccine pre-produced and ready for immediate administration after diagnosis. The same concept is broadly applicable across cancers where immunotherapies remain inadequate and conserved immunogenic antigens can be identified.

About AML
AML is an aggressive hematologic malignancy characterized by clonal expansion of undifferentiated myeloid precursor cells (AML blasts) in the bone marrow. It has poor outcomes for patients ineligible for intensive chemotherapy or stem cell transplantation, highlighting the need for novel and less toxic treatment strategies.

AML is the most frequent leukemia, occurring across all age groups, however, predominantly observed in older adults (median age at diagnosis of 68 years).

Approximately 50% of patients, typically the elderly, are not fit for intensive treatment, so the standard of care is low-intensity chemotherapy. Remissions are, however, short lived with a 3‐year overall survival rate at only 25% reported (Kantarjian et al. 2025).

(Press release, Evaxion, JUN 11, 2026, View Source [SID1234666565])