On September 15, 2026 Ernexa Therapeutics (Nasdaq: ERNA), an industry innovator developing novel cell therapies for the treatment of advanced cancers, reported positive preliminary preclinical results for ERNA-201, its synthetic, allogeneic induced mesenchymal stem cell (iMSC) therapy engineered to express anti-inflammatory factors, while outlining a sharpened oncology strategy designed to accelerate the Company’s transition into the clinic and expand the long-term potential of its proprietary synthetic iMSC platform.

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In preliminary studies using the collagen-induced arthritis (CIA) model, an industry-standard preclinical model for rheumatoid arthritis and inflammatory joint disease, several ERNA-201 lead candidates demonstrated reductions in disease severity relative to untreated controls, including improvements in clinical arthritis scores consistent with the therapy’s intended anti-inflammatory mechanism of action.

Ernexa believes these results represent an important scientific milestone: ERNA-201 demonstrated meaningful preclinical proof-of-concept in inflammatory disease, providing additional evidence that the Company’s engineered iMSC platform can be programmed to deliver therapeutically relevant factors across distinct disease settings.

With this platform validation in hand, Ernexa is concentrating its near-term capital and operational resources on what management believes represents the Company’s most immediate opportunity to create value for patients and shareholders: advancing ERNA-101 into a first-in-human clinical study in platinum-resistant ovarian cancer (PROC).

Importantly, the decision to prioritize ERNA-101 does not reflect a change in Ernexa’s conviction in ERNA-201 or its underlying biology. Rather, following the positive ERNA-201 proof-of-concept results, the Company believes it has reached an appropriate point to preserve the program while directing near-term resources toward achieving clinical validation of its platform in oncology.

"We believe the ERNA-201 results are an important validation of both the program and the broader potential of our synthetic iMSC platform," said Sanjeev Luther, President and Chief Executive Officer of Ernexa Therapeutics. "ERNA-201 did what we wanted to see at this stage: it generated an encouraging proof-of-concept signal in an established inflammatory disease model and demonstrated the potential to engineer our iMSCs for therapeutic activity beyond cancer. That success gives us the confidence to preserve the significant future opportunity represented by ERNA-201 while concentrating our resources on the milestone directly ahead of us, bringing ERNA-101 into the clinic."

Mr. Luther continued, "Our objective is bigger than advancing a single candidate. We are building a differentiated oncology company around a scalable, off-the-shelf cell therapy platform. With ERNA-101 on track for FDA IND submission before year end, we believe Ernexa is approaching a potentially defining inflection point. We intend to execute against that milestone with urgency while broadening our oncology pipeline and identifying additional cancers where our platform could make a meaningful difference."

ERNA-201: Positive Data Strengthen Validation of Ernexa’s iMSC Platform

ERNA-201 is engineered to express anti-inflammatory factors designed to target inflammation and treat autoimmune disease. In completed preliminary studies, ERNA-201 was evaluated in the CIA model, which is widely used to assess potential therapies for rheumatoid arthritis and other inflammatory joint diseases.

Mice treated with several ERNA-201 lead candidates demonstrated reductions in disease severity relative to untreated controls, including improvements in clinical arthritis scores. Ernexa believes the findings are consistent with the candidate’s intended anti-inflammatory mechanism and provide meaningful proof-of-concept support for the use of synthetic, cytokine-engineered iMSCs in inflammatory and autoimmune disease.

"Seeing disease-modifying activity from ERNA-201 in the collagen-induced arthritis model is an encouraging scientific result," said Robert H. Pierce, M.D., Chief Scientific Officer of Ernexa Therapeutics. "These findings support the underlying thesis that engineered iMSCs can serve as a versatile therapeutic delivery platform. We designed ERNA-201 to deliver anti-inflammatory factors in disease, and the preliminary activity we observed is consistent with that objective. From a scientific perspective, these data increase our confidence in the program and provide another important validation point for the broader platform."

The ERNA-201 results are particularly important to Ernexa because the program employs the same fundamental synthetic iMSC platform architecture that underpins the Company’s broader pipeline. While ERNA-201 is engineered for anti-inflammatory activity and ERNA-101 is engineered to stimulate an immune response against cancer, both programs demonstrate the potential versatility of an allogeneic, scalable cell platform that can be engineered for distinct therapeutic purposes.

Building Momentum Toward the Clinic: ERNA-101 Advancing Toward Planned IND Submission in Q4 2026

With ERNA-201 generating positive preliminary proof-of-concept data, Ernexa is prioritizing the next major milestone in the Company’s evolution: advancing ERNA-101 toward a planned IND submission for a first-in-human clinical study in platinum-resistant ovarian cancer.

ERNA-101 is Ernexa’s lead oncology cell therapy candidate, engineered to express an IL-7/IL-15 fusion cytokine designed to activate and regulate the immune system’s response to recognize and attack cancer cells.

The Company is focused on advancing ERNA-101 toward a planned IND submission in the fourth quarter of 2026. Ernexa believes achieving this milestone would represent an important step toward the Company’s transition from a preclinical development company to a clinical-stage oncology company and the generation of human clinical evidence necessary to evaluate the therapeutic potential of its platform.

Clinical data from the ERNA-101 PROC study are expected in Q4 2027, providing the Company with a series of potential clinical and operational milestones over the coming 12–18 months.

A Focused Oncology Strategy Built Around One Scalable Platform

Ernexa’s strategy extends beyond a single indication or product candidate. The Company’s proprietary platform begins with induced pluripotent stem cells (iPSCs), which are engineered and transformed into synthetic induced mesenchymal stem cells. The resulting allogeneic iMSCs are designed to provide a scalable, off-the-shelf therapeutic approach without the need for patient-specific cell harvesting.

Management believes this architecture creates the potential to engineer iMSCs with different therapeutic factors and apply the platform across multiple diseases. The positive preliminary ERNA-201 findings provide additional support for that platform thesis, while ERNA-101 now represents the Company’s most advanced opportunity to demonstrate its potential in patients.

As Ernexa moves toward the clinic, the Company intends to broaden the scope of its oncology program by evaluating additional high-need cancer indications and advancing next-generation iMSC candidates designed to build on the Company’s existing scientific and manufacturing capabilities.

The Company will prioritize opportunities based on scientific rationale, unmet patient need, commercial potential and capital efficiency, including opportunities that may be pursued internally or through strategic collaborations.

Disciplined Capital Allocation: Advancing the Nearest Value-Creating Milestones

Following the encouraging ERNA-201 results, Ernexa has elected to pause additional preclinical development expenditures on ERNA-201 at this time and preserve the program while concentrating near-term resources on ERNA-101 and the expansion of its oncology pipeline.

The decision is a capital allocation priority rather than a scientific decision to discontinue ERNA-201. The Company believes the positive preliminary results provide a foundation from which ERNA-201 could potentially be advanced in the future as resources, strategic priorities and development opportunities warrant.

"We have achieved an important objective with ERNA-201 by generating encouraging proof-of-concept data," Luther said. "The question for us now is not whether we believe in the program. We do. The question is where each incremental dollar and each hour of our team’s effort can have the greatest impact today. With ERNA-101 approaching the clinic, we believe concentrating resources behind first-patient dosing and expanding our oncology opportunity is the most disciplined path forward."

"We are preserving ERNA-201’s future potential while accelerating the program that is closest to human clinical validation. We believe that the combination of scientific validation, disciplined execution and a clear path into the clinic positions Ernexa for an important next chapter."

Multiple Potential Catalysts Ahead

Over the coming 12–18 months, Ernexa intends to:

Submit ERNA-101 IND in Q4 2026 for a first-in-human clinical study in platinum-resistant ovarian cancer;
Generate clinical data from the ERNA-101 PROC program, currently expected in Q4 2027;
Continue generating translational and preclinical evidence designed to strengthen validation of the synthetic iMSC platform;
Evaluate opportunities to expand the platform into additional high-need oncology indications;
Advance next-generation synthetic iMSC oncology candidates;
Explore strategic collaborations that could accelerate or broaden the application of Ernexa’s technology; and
Maintain disciplined capital allocation focused on milestones management believes can create the greatest value for patients and shareholders.
"We believe we are entering one of the most consequential periods in Ernexa’s development," Luther concluded. "ERNA-201 has provided encouraging evidence that our engineering approach can translate into therapeutic activity in a well-established disease model. ERNA-101 is approaching the clinic. And behind it is a platform we believe can be engineered and deployed against multiple high-need cancers. Our focus now is execution, reaching the clinic, generating human data and building a broader oncology pipeline capable of creating sustainable value."

(Press release, Ernexa Therapeutics, SEP 15, 2026, View Source [SID1234670884])