Repeat-dose GLP toxicology study showed no ERNA-101-related adverse findings across all dose levels tested, including no clinical pathology or histopathology findings; single-dose assessment showed no evidence of acute cytokine release
No teratoma formation observed in ERNA-101-treated animals through the six-month tumorigenicity in-life phase, addressing a fundamental safety consideration for an iPSC-derived cell therapy
Biodistribution study showed no significant ERNA-101 signal in tumor-free animals or non-tumor tissues, supporting ERNA-101’s tumor-homing delivery strategy
Encouraging safety results build on previously reported complete tumor clearance and durable survival in ovarian cancer models, strengthening the nonclinical foundation for ERNA-101
Core IND-enabling safety studies complete; GMP manufacturing underway; Company remains on track for planned Q4 2026 IND submission
CAMBRIDGE, Mass., Sept. 30, 2026 (GLOBE NEWSWIRE) — Ernexa Therapeutics (Nasdaq: ERNA), an industry innovator developing novel cell therapies for the treatment of advanced cancers, today announced highly encouraging results from three core Investigational New Drug (IND)-enabling safety studies of ERNA-101, its lead synthetic, allogeneic induced mesenchymal stromal cell (iMSC) therapy engineered to express an IL-7/IL-15 fusion cytokine.
Across repeat-dose GLP toxicology, tumorigenicity and biodistribution studies, ERNA-101 demonstrated a favorable nonclinical safety profile, with results addressing three critical questions as the Company prepares to advance the program toward first-in-human development:
- Repeat-dose safety: No ERNA-101-related adverse findings were observed at any dose level tested in a 50-day GLP toxicology study in tumor-bearing mice with an intact immune system.
- Tumorigenicity: No teratoma formation was observed in ERNA-101-treated animals through the six-month tumorigenicity in-life phase, addressing an important safety consideration for an iPSC-derived therapy.
- Biodistribution: No significant ERNA-101 signal was detected in tumor-free animals or non-tumor tissues, supporting ERNA-101’s design to home to tumors without taking up residence elsewhere in the body.
Importantly, the results add a critical safety dimension to previously reported ERNA-101 efficacy data demonstrating complete tumor clearance and durable long-term survival in preclinical ovarian cancer models when combined with PD-1 blockade. Together, Ernexa believes the efficacy, safety and biodistribution findings provide an increasingly comprehensive nonclinical foundation for advancing ERNA-101 toward the clinic.
“We believe these results represent one of the most important milestones in Ernexa’s history because the ERNA-101 story is now coming together across efficacy, safety and tumor-targeted delivery,” said Sanjeev Luther, President and Chief Executive Officer of Ernexa Therapeutics. “We have previously demonstrated compelling anti-tumor activity, including complete tumor clearance and durable survival in preclinical ovarian cancer models. Now, across three critical IND-enabling safety studies, we observed no ERNA-101-related adverse findings in our repeat-dose GLP study, no teratoma formation through the six-month tumorigenicity in-life phase and no significant ERNA-101 signal in tumor-free animals or non-tumor tissues.”
“From a scientific perspective, these findings provide important support for the fundamental design of ERNA-101,” said Robert H. Pierce, M.D., Chief Scientific Officer of Ernexa Therapeutics. “Across repeat-dose toxicology, tumorigenicity and biodistribution, we are seeing encouraging results that strengthen the nonclinical foundation for advancing ERNA-101 toward the clinic.”
Luther continued, “In straightforward terms, these results support what we designed ERNA-101 to do: find the tumor, deliver powerful immune-enhancing cytokines where they are intended to work and limit unnecessary exposure elsewhere in the body. With our core safety studies complete, GMP manufacturing underway and our FDA Pre-IND meeting behind us, we remain on track for our planned IND submission in the fourth quarter of 2026. Our focus now is execution and moving ERNA-101 toward the milestone that matters most: treating our first patient.”
Repeat-Dose GLP Toxicology: No ERNA-101-Related Adverse Findings
In Ernexa’s pivotal 50-day GLP toxicology study, immunocompetent C57BL/6 mice with established ID8 ovarian tumors received ERNA-101 intraperitoneally once weekly for four doses at 0.33 × 10⁶, 1 × 10⁶ or 1 × 10⁷ cells per animal, compared with vehicle-treated controls.
The study was designed to evaluate ERNA-101 under conditions relevant to its planned clinical use, with tumor present, an intact immune system and repeat dosing.
Across all dose levels tested, there were:
- No ERNA-101-related changes in clinical pathology;
- No pathological evidence of treatment-related toxicity on microscopic evaluation by an independent board-certified veterinary pathologist; and
- No treatment-related differences in the incidence or timing of unscheduled deaths, all of which were attributed to progression of the underlying tumor.
What the data mean: ERNA-101 is engineered to deliver an IL-7/IL-15 fusion cytokine directly into the tumor microenvironment. Cytokines can powerfully activate the immune system, but systemic exposure can also cause significant toxicity. Ernexa designed ERNA-101 to use the natural tumor-homing properties of mesenchymal stromal cells to concentrate cytokine activity within tumors while limiting systemic exposure. The absence of ERNA-101-related toxicity in the repeat-dose study, together with the absence of acute cytokine release in the single-dose assessment, provides important support for that design strategy.
“A cytokine-armed cell therapy needs to be evaluated where its biology is active, in the presence of tumor and an intact immune system,” said Robert H. Pierce, M.D., Chief Scientific Officer of Ernexa Therapeutics. “That is what makes these results particularly encouraging. Across the repeat-dose study, we observed no ERNA-101-related adverse findings in clinical pathology or histopathology, and in the single-dose assessment, we observed no evidence of acute cytokine release. We believe the results support the fundamental concept behind ERNA-101: bring powerful immune activation to the tumor while limiting systemic exposure.”
Tumorigenicity: No Teratoma Formation Observed Through Six-Month In-Life Phase
Because ERNA-101 is derived from induced pluripotent stem cells, or iPSCs, tumorigenicity is an important component of its nonclinical safety assessment. Specifically, the studies evaluate whether the final differentiated ERNA-101 cell product could form unwanted tumors known as teratomas.
Ernexa has completed a three-month tumorigenicity study and the in-life phase of a six-month tumorigenicity study in immunocompromised NSG mice. No gross evidence of teratoma formation was observed in ERNA-101-treated groups.
Histopathologic assessment confirmed the absence of tumor formation in the three-month study. Histopathologic evaluation of the six-month study is ongoing and is expected to be completed prior to IND submission.
What the data mean: For an iPSC-derived cell therapy, demonstrating that the final cell product does not form teratomas is a fundamental safety consideration. The absence of teratoma formation through the six-month in-life phase provides an encouraging answer to this important question as Ernexa prepares its IND package.
Biodistribution: No Significant ERNA-101 Signal in Tumor-Free Animals or Non-Tumor Tissues
Ernexa also completed a five-week biodistribution and persistence study designed to answer another straightforward but critical question: Where do ERNA-101 cells go after they are administered?
ERNA-101 was evaluated in both tumor-bearing and tumor-free animals. Using highly sensitive digital droplet PCR (ddPCR) specific for ERNA-101, the Company found no significant ERNA-101 signal in non-tumor-bearing animals or non-tumor-containing tissues.
The findings are consistent with the intrinsic tumor-homing properties of mesenchymal stromal cells and prior studies in which ERNA-101 cells were detected within tumors.
What the data mean: Ernexa designed ERNA-101 to home to tumors, deliver its immune-enhancing payload and avoid taking up residence elsewhere in the body. The biodistribution results provide important support for that targeted-delivery strategy.
“Biodistribution goes directly to the heart of the ERNA-101 thesis,” Pierce said. “Put simply, we want the cells to go to the tumor, deliver their payload and not take up residence where they do not belong. Finding no significant ERNA-101 signal in tumor-free animals or non-tumor tissues is exactly the type of biodistribution profile we hoped to see and provides important support for the targeted delivery concept underlying our platform.”
Efficacy + Safety + Tumor-Homing: Building a More Complete ERNA-101 Profile
The newly reported safety results build on previously reported ERNA-101 preclinical efficacy findings.
In ovarian cancer models, ERNA-101 remodeled the tumor microenvironment from immunosuppressive to immune-activated and increased infiltration of CD4+ and CD8+ T cells. When combined with PD-1 blockade, ERNA-101 achieved a high rate of complete tumor clearance and durable long-term survival, findings that were validated in independent studies.
In simple terms, the preclinical development program has now begun to address three fundamental questions:
Can ERNA-101 mediate tumor clearance?
Preclinical efficacy studies demonstrated compelling anti-tumor activity, including complete tumor clearance and durable survival in ovarian cancer models.
Can ERNA-101 potentially do so with an acceptable safety profile?
The core IND-enabling studies showed no ERNA-101-related adverse findings in repeat-dose GLP toxicology and no observed teratoma formation through the six-month tumorigenicity in-life phase.
Does ERNA-101 go where it is designed to go?
Biodistribution studies showed no significant ERNA-101 signal in tumor-free animals or non-tumor tissues.
“We believe the significance is in the totality of the data,” Luther said. “Efficacy, safety and tumor-homing are not separate stories, they are interconnected parts of the same ERNA-101 thesis. The preclinical evidence generated to date is giving us encouraging answers across all three dimensions. That strengthens our conviction in the program as we prepare for the transition from preclinical development into the clinic.”
Advancing Toward a Planned Q4 2026 IND
Completion of the core IND-enabling safety studies represents another major step in Ernexa’s planned transition toward becoming a clinical-stage oncology company.
The Company has completed its FDA Pre-IND meeting, completed its core IND-enabling safety studies and is advancing clinical-grade GMP manufacturing of ERNA-101.
Ernexa remains on track to submit an IND for ERNA-101 in Q4 2026 for a first-in-human clinical study in platinum-resistant ovarian cancer. Subject to IND clearance, the Company intends to initiate the study and dose the first patient, with initial clinical data currently expected in Q4 2027.
“There is a point in the development of every potentially important new therapy when the question begins to change,” Luther concluded. “The question is no longer simply whether the science looks promising. It becomes whether you have built the foundation necessary to take that science into patients. With compelling preclinical efficacy, encouraging safety results, our core IND-enabling studies complete and manufacturing underway, we believe Ernexa is approaching that moment.”
Mr. Luther continued, “Our objective now is clear: execute the IND submission, advance ERNA-101 into the clinic and begin determining whether the promise we have observed in preclinical models can translate into meaningful benefit for patients. For women facing platinum-resistant ovarian cancer, better treatment options are urgently needed. That need gives purpose to every milestone we achieve and urgency to everything that comes next.”
For more information about ERNA-101 and the Company’s development plans, visit www.ernexatx.com
About Ernexa Therapeutics
Ernexa Therapeutics (NASDAQ: ERNA) is developing innovative cell therapies for the treatment of advanced cancers. Ernexa’s core technology focuses on engineering induced pluripotent stem cells (iPSCs) and transforming them into induced mesenchymal stromal cells (iMSCs). Ernexa’s allogeneic synthetic iMSCs provide a scalable, off-the-shelf treatment solution, without needing patient-specific cell harvesting.
ERNA-101 is the company’s lead cell therapy product, designed to activate and regulate the immune system’s response to recognize and attack cancer cells. The company’s initial focus is to develop ERNA-101 for the treatment of ovarian cancer, with the potential to address multiple difficult-to-treat solid tumors.
For more information, visit www.ernexatx.com.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, which are intended to be covered by the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements, in some cases, can be identified by terms such as “believe,” “may,” “will,” “estimate,” “continue,” “anticipate,” “design,” “intend,” “expect,” “could,” “plan,” “potential,” “predict,” “seek,” “should,” “would,” “contemplate,” “project,” “target,” “objective,” or the negative version of these words and similar expressions. Forward looking statements involve known and unknown risks, uncertainties and other factors that may cause Ernexa’s actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by the forward-looking statements in this press release, including, without limitation, risks and uncertainties related to: progress and possible outcomes of the Company’s lead research project, ERNA-101, and future research projects. Forward-looking statements are based upon Ernexa’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. For a detailed description of Ernexa’s risks and uncertainties, you are encouraged to review its documents filed with the SEC including its recent filings on Form 8-K, Form 10-K and Form 10-Q. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Ernexa does not undertake any obligation to update the forward-looking statements contained herein to reflect events that occur or circumstances that exist after the date hereof, except as required by applicable law.
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