Epitopea's 'Dark Genome' Cancer Vaccine Enters Human Trials for Ovarian Cancer

CAMBRIDGE, UK – April 23, 2026 – A new front has opened in the war against ovarian cancer. Transatlantic biotech firm Epitopea announced today it has received regulatory green light in the United Kingdom to begin the first-in-human clinical trial for its novel RNA-based cancer immunotherapy, CryptiVax™-1001. The approval from the Medicines and Healthcare products Regulatory Agency (MHRA) and the Regional Ethics Committee (REC) paves the way for the OVACT trial, which will target patients with advanced high-grade serous ovarian cancer, a particularly aggressive and difficult-to-treat form of the disease.

This milestone not only marks Epitopea's pivotal transition from a preclinical research entity to a clinical-stage company but also brings a potential new weapon to a patient population with desperately few effective options. To lead the landmark study, the company has appointed Professor Susana Banerjee, a world-renowned oncologist from The Royal Marsden NHS Foundation Trust, as Chief Investigator.

Addressing a Critical Unmet Need

High-Grade Serous Ovarian Cancer (HGSOC) is the most common and lethal subtype of ovarian cancer, accounting for over 70% of cases. Its notoriety stems from its silent progression; nearly 80% of women are diagnosed only after the cancer has reached an advanced, metastatic stage. While most patients initially respond well to the standard of care—aggressive surgery followed by platinum-based chemotherapy—the respite is often temporary. The vast majority of patients eventually relapse, and their disease frequently develops resistance to the platinum chemotherapy that was once effective.

The OVACT trial is specifically designed for a subgroup of patients who face the bleakest prognosis: those with tumors that are homologous recombination proficient (HRP) and BRCA-wildtype. This group represents roughly half of all ovarian cancer patients. While the development of PARP inhibitors has been a game-changer for patients with BRCA mutations or other defects in DNA repair (known as homologous recombination deficiency), they offer limited durable benefit for the HRP population. This has created a major therapeutic gap, leaving these patients with few effective maintenance therapies to prolong remission and a high likelihood of progressing to platinum-resistant disease, which is notoriously difficult to treat.

Professor Banerjee, who also serves as Professor in Women’s Cancers at the Institute of Cancer Research, underscored the urgency of this challenge. “There is a substantial unmet need in homologous recombination proficient (HRP) ovarian cancer, where available maintenance therapies deliver limited durable benefit,” she stated. “Epitopea’s CryptiVax™-1001 vaccine, which targets a novel repertoire of tumor-specific antigens, has the potential to meaningfully extend remission for patients with few effective treatment options.”

Unlocking the 'Dark Genome' with Novel RNA Technology

At the heart of Epitopea's strategy is a groundbreaking technology that ventures into previously uncharted territory of the human genome. The company’s proprietary discovery engine, CryptoMap™, is designed to identify novel tumor-specific antigens that arise from what scientists call the 'dark genome'—vast regions of our DNA once dismissed as non-functional or inaccessible for therapeutic targeting.

These newly identified antigens, which the company has named Cryptigens™, are highly specific to tumors and, crucially, demonstrate high sharing between different patients. This breakthrough is a key differentiator from many other cancer vaccine approaches, such as personalized neoantigen vaccines, which must be custom-manufactured for each individual. By targeting shared Cryptigens™, Epitopea can develop a true 'off-the-shelf' mRNA immunotherapy that is ready to be administered, dramatically reducing cost and complexity.

CryptiVax™-1001 works by delivering mRNA that instructs the patient’s own cells to produce these Cryptigens™. The immune system then recognizes these antigens as foreign and mounts a targeted attack against the cancer cells that present them, creating a durable and specific anti-tumor response. This approach represents a significant evolution in RNA therapeutics, moving beyond infectious diseases and into the complex landscape of oncology with a highly innovative targeting mechanism.

The OVACT Trial: A New Frontier in Clinical Research

The OVACT trial is a Phase 1/1b dose escalation and expansion study. Its primary goals are to evaluate the safety and tolerability of CryptiVax™-1001 in HGSOC patients. The study will also measure the vaccine's ability to provoke an immune response (immunogenicity) and look for early signals of clinical activity against the cancer. Securing approval from both the MHRA, which governs the safety and efficacy of medicines, and a REC, which protects the rights and well-being of trial participants, represents the successful navigation of a stringent regulatory and ethical pathway in the UK.

Dr. Klaus Edvardsen, MD, Chief Medical Officer of Epitopea, celebrated the achievement as a validation of the company's scientific and clinical rigor. “We are excited by the achievement of this significant regulatory milestone, which is a credit to our dedicated clinical and wider Epitopea team,” he commented. “We also welcome Professor Banerjee to her role as Chief Investigator of our first clinical trial. Her world-class expertise in ovarian cancer and leadership in global clinical development will bring invaluable insights as we transition to a clinical-stage company.”

The leadership of Professor Banerjee is a significant endorsement of the trial's scientific premise. Her extensive experience at The Royal Marsden, one of the world's leading cancer centers, and her deep research background at the Institute of Cancer Research, place the trial at the forefront of gynecological cancer research. The initiation of the OVACT trial under her guidance represents a critical test for this pioneering science and a closely watched development for patients and clinicians awaiting a breakthrough in ovarian cancer treatment.