For Health Care Professionals

Red Cell Therapeutics™:
A New Era of Medicine for the Treatment of Cancer

Our innovations in creating human red blood cells and biological engineering have made it possible to explore using these cells as a foundation for an entirely new class of cellular therapies, which we call Red Cell Therapeutics™. Red Cell Therapeutics product candidates are manufactured from CD34+ hematopoietic progenitor cells from blood type O, Rh-negative, Kell-negative donors. The cells are expanded and transduced with a lentiviral vector to insert a gene or genes of interest, followed by further expansion and differentiation into enucleated red blood cells that express one or more therapeutic proteins inside or on the cell surface, resulting in cellular therapies for the potential treatment of cancer.

About RTX‑224

RTX-224 is an engineered red blood cell that is being evaluated in a Phase 1/2 clinical trial for the treatment of patients with relapsed/refractory or locally advanced solid tumors, including non-small cell lung cancer, cutaneous melanoma, urothelial cancer, head and neck squamous cell carcinoma and triple-negative breast cancer. RTX-224 is engineered to simultaneously present hundreds of thousands of copies of the costimulatory proteins 4-1BBL and IL-12 and is designed to stimulate innate and adaptive immunity by activating natural killer (NK) cells and T cells inside the patient’s body to generate an anti-tumor immune response.

The IND for RTX-224 has been cleared by the FDA and we are now enrolling patients into our ongoing Phase 1/2 clinical trial, which includes a dose escalation arm in adults with relapsed/refractory or locally advanced tumors in five cohorts: non-small cell lung cancer, cutaneous melanoma, urothelial cancer, head and neck squamous cell cancer and triple-negative breast cancer.

About the RTX‑224 Phase 1/2 Clinical Trial

This is a Phase 1/2 open label, multicenter, multidose, first-in-human dose-escalation and expansion study designed to determine the safety and tolerability, pharmacokinetics, maximum tolerated dose and a recommended Phase 2 dose and dosing regimen of RTX-224. The trial will also assess the pharmacodynamics of RTX-224 measured by changes in T and NK cell number and function relative to baseline and anti-tumor activity.

Inclusion/Exclusion Criteria

Adults who meet the following criteria may be able to participate in the trial.

  • Men and women aged 18 or older.
  • Participants must have a relapsed, refractory, or locally advanced, unresectable, histologically or cytologically confirmed non-small cell lung cancer, cutaneous melanoma, urothelial carcinoma, head and neck squamous cell carcinoma, or triple-negative breast cancer, for which no standard therapy exists, or for which the patient is ineligible or has declined standard therapy.
  • Participants must have completed prior therapy, including radiation, at least 28 days prior to study treatment.
  • Prior therapy in each disease setting must include the following:
  • Non-small cell lung cancer: Patients must have experienced disease progression following platinum-containing chemotherapy and a programmed cell death 1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor. Patients with EGFR, ALK, ROS-1, or other actionable mutations should have previously received or been ineligible for therapies targeting their respective mutation(s).
  • Cutaneous melanoma: Patients must have experienced disease progression following a PD-1 or PD-L1 inhibitor. Patients with V600E mutations should have previously received or been ineligible for approved BRAF inhibitor or MEK inhibitor therapy.
  • Head and neck squamous cell carcinoma: Patients must have experienced disease progression following platinum-based combination chemotherapy and a PD-1 or PD-L1 inhibitor.
  • Urothelial carcinoma: Patients must have experienced disease progression following platinum-based combination chemotherapy and a PD-1 or PD-L1 inhibitor.
  • Triple-negative breast cancer: Patients must have experienced disease progression following single-agent or combination chemotherapy. Patients with BRCA1/2 mutations should have previously received or been ineligible for an approved PARP inhibitor; patients who are PD-L1-positive should have received or been ineligible for an approved PD-1 or PD-L1 inhibitor.
  • For females of reproductive potential, agreement to use highly effective contraceptive throughout and for six months following last dose study treatment.

If you are interested in becoming a potential clinical trial site or would like more information about RTX‑224, please contact us at clinicaltrials@rubiustx.com.