ADVANCING A BROAD AND DIVERSE PIPELINE

Rubius Therapeutics is advancing a broad pipeline of RCT™ product candidates. Our current programs are investigating applications across multiple therapeutic areas—starting with cancer and autoimmune diseases. We are currently enrolling patients in a Phase 1/2 clinical trial of RTX-240; the trial has three separate Phase 1 arms: an ongoing monotherapy dose escalation arm in adults with relapsed/refractory or locally advanced solid tumors, an ongoing monotherapy dose escalation arm in adults with relapsed/refractory acute myeloid leukemia, and a combination therapy dose escalation arm with pembrolizumab in adults with relapsed/refractory or locally advanced solid tumors. We are also enrolling patients in a Phase 1 clinical trial of RTX-321 for the treatment of human papillomavirus 16-positive cancers. Additionally, Rubius plans to file an IND for RTX-224 for the treatment of solid tumors by year-end 2021.

Candidate
Preclinical
IND Enabling
Phase 1
Phase 2
Phase 3

Cancer

R/R Solid Tumors
RTX-240

RTX-240 co-expresses 4-1BBL and IL-15TP, a fusion of IL-15 and IL-15 receptor alpha, with the goal of improving anti-tumor activity and overcoming resistance to immunotherapy in patients with solid tumors.

R/R Solid tumors
RTX-240

RTX-240 co-expresses 4-1BBL and IL-15TP, a fusion of IL-15 and IL-15 receptor alpha, with the goal of improving anti-tumor activity and overcoming resistance to immunotherapy in patients with solid tumors.

R/R Acute Myeloid Leukemia
RTX-240

RTX-240 is designed to induce NK and T cell activation and proliferation, leading to the killing of AML cells.

R/R Acute Myeloid Leukemia
PreClinical
RTX-240

RTX-240 is designed to induce NK and T cell activation and proliferation, leading to the killing of AML cells.

RTX-240
R/R-240 + PD-1 R/R Solid Tumors
RTX-240

RTX-240
R/R-240 + PD-1 R/R Solid Tumors
RTX-240

R/R HPV 16+ Solid Tumors
RTX-321 aAPC (HPV 16+)

RTX-321 (HPV+) is engineered to express on the cell surface an HPV peptide antigen on the major histocompatibility complex (MHC) I, a costimulatory signal and a cytokine to mimic the human immunobiology of T cell-APC interactions.

R/R HPV 16+ Solid Tumors
RTX-321 aAPC (HPV 16+)

RTX-321 (HPV+) is engineered to express on the cell surface an HPV peptide antigen on the major histocompatibility complex (MHC) I, a costimulatory signal and a cytokine to mimic the human immunobiology of T cell-APC interactions.

R/R Solid Tumors
RTX-224

RTX-224 co-expresses 4-1BBL and IL-12 on the cell surface to activate the innate and adaptive immune systems.

R/R Solid Tumors
PreClinical
RTX-224

RTX-224 co-expresses 4-1BBL and IL-12 on the cell surface to activate the innate and adaptive immune systems.

RTX-aAPC
Cancer
RTX-aAPC

RTX-aAPC
Cancer
RTX-aAPC

Candidate
Preclinical
IND Enabling
Phase 1
Phase 2
Phase 3

Autoimmune Diseases

RTX-T1D
Type 1 Diabetes
RTX-T1D

RTX-T1D
Type 1 Diabetes
PreClinical
RTX-T1D

THERAPEUTIC
AREAS

DESIGNED TO ACTIVATE BOTH ARMS OF THE IMMUNE SYSTEM TO STOP CANCER

Recent treatment advances have focused on activating the immune system against cancer. Despite this progress, existing immunotherapies are effective in a relatively small number of patients and types of cancer. Furthermore, even when therapies are initially effective, the disease eventually no longer responds to treatment and progresses.

RCTTM product candidates can be engineered to express combinations of co-stimulatory molecules and cytokines on their cell surface to directly engage both the adaptive immune system and the innate immune system. By stimulating both powerful arms of the immune system, RCTs may be better able to destroy cancer cells to improve anti-tumor activity and overcome resistance to immunotherapy.

We believe RCTs will have broad therapeutic application across a range of both solid tumors and hematological cancers.

AIMING TO RETRAIN THE IMMUNE SYSTEM
TO DEFEAT AUTOIMMUNE DISEASES

Autoimmune diseases are the result of a hyperactive immune response that mistakes healthy tissue as foreign. Over time, these attacks on healthy cells lead to disease. Current therapies are generally administered on a lifelong basis, initially work for only a subset of patients, tend to lose effectiveness over time and are often associated with serious side effects, including opportunistic infections, lymphoma and, in some cases, severe or fatal reactions.

RCTs can be engineered to express disease-causing antigens on their cell surface. When these RCTs are processed by the reticuloendothelial system, these antigens are expected to be presented to the immune system and thereby retrain the body to no longer see these self-antigens as foreign. The resulting restoration or induction of immune tolerance may lead to more effective treatments—potentially even cures—for certain autoimmune diseases, such as Type I diabetes.

Patient Access

At Rubius Therapeutics, we are focused on developing an entirely new class of medicine to treat cancer and autoimmune diseases. We are conducting several clinical trials to demonstrate the safety and efficacy of these potential treatments. These investigational treatments have not been approved by regulatory authorities, such as the United States Food and Drug Administration (FDA).

Patient access programs allow patients with immediately life-threatening conditions or serious diseases to gain access to an investigational therapy outside of clinical trials. As we carefully consider the health and well-being of patients and families, our first commitment is to avoid inadvertently doing harm while the safety of our treatments is still being evaluated.

At this time, we are not able to provide access to our investigational therapies outside of a clinical trial setting. We believe the best way to develop safe and effective treatments that have a positive benefit for the largest number of patients is to continue our rigorous clinical development program.

We encourage any person interested in learning more about participating in one of our clinical trials to discuss their eligibility with their physician or to visit www.rubiustrials.com.