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Pipeline

Ernexa Therapeutics is building a pipeline of off-the-shelf, synthetic cell therapies designed to modulate the immune system and restore balance in the face of disease. Our lead candidate targets high-need areas in oncology with a focus on delivering powerful, tumor-targeted treatments that can scale.

ERNA-101 (Ovarian Cancer)

Ovarian cancer remains the deadliest gynecologic cancer, with most cases diagnosed at an advanced stage. Additionally, an alarming 85% of patients experience recurrence after standard treatment. Despite advances in chemotherapy and targeted therapies, outcomes for patients with platinum-resistant disease remain poor, highlighting a major unmet need.

Immune checkpoint inhibitors have shown limited success in ovarian cancer. This is likely due to the immunosuppressive tumor microenvironment, which blocks immune cell function and limits therapy effectiveness.

Ernexa Therapeutics’ lead program, ERNA-101, is designed to overcome these challenges by remodeling the tumor microenvironment to support a stronger immune response. 
Our approach builds on the foundational work of world-renowned expert Michael Andreeff, M.D., Ph.D., from The University of Texas MD Anderson Cancer Center. Dr. Andreeff’s studies showed that engineered mesenchymal stromal cells (MSCs) can successfully home to tumor sites, deliver immunostimulatory molecules, and reduce tumor burden in preclinical ovarian cancer models.

We believe this strategy, using engineered iMSCs to deliver targeted immune activation, represents a promising and scalable new path forward for patients with ovarian cancer.

References

  1. Siegel, R.L., et al., Cancer statistics, 2022. CA Cancer J Clin, 2022. 72(1): p. 7-33.
  2. Goff, B., Symptoms associated with ovarian cancer. Clin Obstet Gynecol, 2012. 55(1): p. 36-42.
  3. Peres, L.C., et al., Invasive Epithelial Ovarian Cancer Survival by Histotype and Disease Stage. J Natl Cancer Inst, 2019. 111(1): p. 60-68.
  4. Torkildsen, C.F., et al., New immune phenotypes for treatment response in high-grade serous ovarian carcinoma patients. Front Immunol, 2024. 15: p. 1394497.
  5. Hao, J., et al., Prognostic impact of tumor-infiltrating lymphocytes in high grade serous ovarian cancer: a systematic review and meta-analysis. Ther Adv Med Oncol, 2020. 12: p. 1758835920967241.
  6. Ovarian Tumor Tissue Analysis, C., et al., Dose-Response Association of CD8+ Tumor-Infiltrating Lymphocytes and Survival Time in High-Grade Serous Ovarian Cancer. JAMA Oncol, 2017. 3(12): p. e173290.
  7. Sato, E., et al., Intraepithelial CD8+ tumor-infiltrating lymphocytes and a high CD8+/regulatory T cell ratio are associated with favorable prognosis in ovarian cancer. Proc Natl Acad Sci U S A, 2005. 102(51): p. 18538-43.
  8. Bobisse, S., et al., Sensitive and frequent identification of high avidity neo-epitope specific CD8 (+) T cells in immunotherapy-naive ovarian cancer. Nat Commun, 2018. 9(1): p. 1092.
  9. Nelson, B.H., et al., Immunological and molecular features of the tumor microenvironment of long-term survivors of ovarian cancer. J Clin Invest, 2024.
  10. Shalaby, A., et al., Correlation of PD-L1 expression with different clinico-pathological and immunohistochemical features of ovarian surface epithelial tumors. Clin Transl Oncol, 2024.
  11. Varga, A., et al., Pembrolizumab in patients with programmed death ligand 1-positive advanced ovarian cancer: Analysis of KEYNOTE-028. Gynecol Oncol, 2019. 152(2): p. 243-250.
  12. Hamanishi, J., et al., Nivolumab Versus Gemcitabine or Pegylated Liposomal Doxorubicin for Patients With Platinum-Resistant Ovarian Cancer: Open-Label, Randomized Trial in Japan (NINJA). J Clin Oncol, 2021. 39(33): p. 3671-3681.
  13. Hensler, M., et al., M2-like macrophages dictate clinically relevant immunosuppression in metastatic ovarian cancer. J Immunother Cancer, 2020. 8(2).
  14. Fu, W., Q. Feng, and R. Tao, Machine learning developed a fibroblast-related signature for predicting clinical outcome and drug sensitivity in ovarian cancer. Medicine (Baltimore), 2024. 103(16): p. e37783.
  15. Desbois, M., et al., Integrated digital pathology and transcriptome analysis identifies molecular mediators of T-cell exclusion in ovarian cancer. Nat Commun, 2020. 11(1): p. 5583.
  16. Dembinski, J.L., et al., Tumor stroma engraftment of gene-modified mesenchymal stem cells as anti-tumor therapy against ovarian cancer. Cytotherapy, 2013. 15(1): p. 20-32.
  17. Olson, A., et al., A Phase I Trial of Mesenchymal Stem Cells Transfected with a Plasmid Secreting Interferon Beta in Advanced Ovarian Cancer. Biology of Blood and Marrow Transplantation, 2018. 24(3): p. S473.
Therapeutic Candidates
Indications
Preclinical
Phase 1
Registrational Trial (Phase 2/3)

ERNA-101 (IL7_IL15)

± Pembrolizumab + Bevacizumab

Platinum-Resistant Ovarian Cancer

ERNA-101 (IL7_IL15)

+ TBD

Other Gynecological Cancer, Immunologically Cold Tumors

ERNA-101 (IL7_IL15)

+ Immunotherapies*

Immunologically ‘Cold’ Tumors

ERNA-102 (IL7_IL15 + IL15R); ERNA-103 (IL7_IL15 + CXCL9)

+ TBD

Solid Tumors

*Adoptive cell therapies with CAR-NK cells, CAR-Ts, TILs; T cell engagers (e.g. BiTEs); therapeutic vaccines and T cell checkpoints