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 genetic 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‑240

RTX‑240 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, or relapsed/refractory AML. RTX‑240 is engineered to simultaneously present hundreds of thousands of copies of the costimulatory proteins 4-1BBL and IL‑15TP 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.

To date, the use of agonists of 4-1BB for cancer immunotherapy has been limited because of hepatotoxicity.

However, in pre-clinical studies Red Cell Therapeutics such as RTX‑240 are restricted to the vasculature which may help limit toxicities unlike systemic administration of recombinant cytokines and agonist antibodies.

In preclinical studies, RTX‑240 was shown to potently stimulate the immune system and generate anti-tumor activity, with no observed toxicity. The IND for RTX‑240 has been cleared by the FDA and we are now enrolling patients into our ongoing Phase 1/2 clinical trial, which has two arms – one in all solid tumors, and one in relapsed/refractory AML. We are primarily conducting the trial at small cancer centers, rather than large hospitals with ICUs and emergency rooms.

About the RTX‑240 Phase 1/2 Clinical Trial

This is a Phase 1/2 open label, multicenter, multidose, first-in-human dose-escalation and expansion study to determine the safety and tolerability, pharmacokinetics, maximum tolerated dose and a recommended Phase 2 dose and dosing regimen of RTX‑240 in adult patients with relapsed/refractory or locally advanced solid tumors, or with relapsed/refractory AML. The trial will also assess the pharmacodynamics of RTX‑240 measured by changes in T and NK cell number and function relative to baseline and anti-tumor activity. The study will include a monotherapy dose escalation phase followed by an expansion phase in specified tumor types during the Phase 2 portion of the trial.

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 solid tumor for which no standard therapy exists, or for which the patient is ineligible or has declined standard therapy.
  • Men and women aged 18 or older with acute myeloid leukemia that has not responded to treatment or returned following treatment (relapsed or refractory AML per protocol).
  • Participants must have completed prior therapy, including radiation, at least 28 days prior to study treatment.
  • 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‑240, please contact us at clinicaltrials@rubiustx.com.