Our Pipeline

Rubius Therapeutics is advancing a broad pipeline of potential RCT medicines. Our RED PLATFORM enables us to engineer and culture RCT product candidates with a wide array of biotherapeutic proteins and biological functions that enable their use in rare diseases, cancer and autoimmune diseases. We plan to file an Investigational New Drug application (IND) for RTX-134 for the treatment of phenylketonuria (PKU) in the first quarter of 2019, and INDs for additional RCTs during 2019, 2020 and thereafter.

Rare Diseases

We believe that Red Cell Therapeutics (RCTs) may be used to treat certain rare diseases caused by a single genetic defect that results in the inactivation of a critical metabolic enzyme or bioactive protein. We engineer RCTs that express potent enzymes within the cell for the treatment of patients with rare enzyme deficiency diseases. These enzymes are shielded from being neutralized by the immune system, thereby allowing the enzymes to degrade and clear the pathogenic metabolites that build up in such diseases. RCTs may have a longer and sustained treatment duration and could avoid the immune-driven reduction in efficacy and induction of adverse events associated with currently available therapies.

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Product Candidate

RTX-134

Phenylketonuria

Phase

IND Enabling
Estimated IND Filing Q1’19

RTX-134 is an RCT that expresses the enzyme phenylalanine ammonia lyase and coverts Phe to the metabolite transcinnamic acid in order to potentially treat phenylketonuria (PKU), a rare inherited disease that causes build-up of the neurotoxic amino acid phenylalanine.

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Product Candidate

RTX-Uricase/URAT1

Refractory Gout

Phase

Lead Optimization

RTX Uricase/URAT1 is an RCT that has been genetically engineered to express hundreds of thousands of copies of uricase and URAT1, a uric acid transporter that ensures optimal uptake of uric acid into the cell, in order to augment a patient’s ability to clear uric acid levels in the blood.

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Product Candidate

RTX-CBS

Homocystinuria

Phase

Lead Optimization

RTX-CBS is an RCT that has been genetically engineered to express hundreds of thousands of copies of cystathionine beta-synthase (CBS) in the cell in order to replace the patient’s missing or ineffective enzymes and rapidly drop total plasma homocysteine, or tHcy, levels to clinically meaningful targets through infrequent, low volume intravenous infusions.

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Product Candidate

RTX-OXOX

Hyperoxaluria

Phase

Lead Optimization

Product Candidate

RTX-ALA-D

AIP

Phase

Lead Optimization

Oncology

We believe that RCT medicines will have broad therapeutic applicability across a range of both solid and hematological cancers.

We are developing a pipeline of RCT product candidates to target, starve, penetrate and destroy cancer cells, by targeting T cells, NK cells, dendritic cells and tumor cells. Our initial RCTs are based on a 4-1BBL backbone, a ligand that binds to and activates the co-stimulatory receptor 4-1BB, an important regulator of the immune system.

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Product Candidate

RTX-212

R/R aPD1 Solid Tumor

Phase

Lead Optimization

RTX-212 is designed to co-express 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 whose disease has progressed following checkpoint inhibitor therapy.

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Product Candidate

RTX-212

R/R AML Post-HSCT

Phase

Lead Optimization

We plan to conduct a Phase 2 trial of RTX-212 administered to acute myeloid leukemia (AML) patients post-allogeneic hematopoietic stem cell transplantation (HSCT) using the single agent dose determined in our initial Phase 1 trial of RTX-212 in solid tumors.

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Product Candidate

RTX-Target-4-1BBL

Tumor Targeted

Phase

Lead Optimization

Product Candidate

RTX-4-1BBL

Solid Tumor

Phase

Lead Optimization

Product Candidate

RTX-aAPC

Cancer

Phase

Lead Optimization

Autoimmune Diseases

In preclinical studies, our RCT product candidates have shown potential for the treatment of autoimmune diseases. We believe RCTs can be designed to more specifically modulate complex counter regulatory immune responses and enable greater efficacy with lower toxicity, potentially providing treatments for a number of diseases with high unmet need

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Product Candidate

RTX-PV

Pemphigus Vulgaris

Phase

Lead Optimization

Product Candidate

RTX-T1D

Type 1 Diabetes

Phase

Discovery

Rare Diseases

We believe that Red Cell Therapeutics may be used to treat certain rare diseases caused by a single genetic defect that results in the inactivation of a critical metabolic enzyme or bioactive protein. We engineer RCTs that express potent enzymes within the cell for the treatment of patients with rare enzyme deficiency diseases. These enzymes are shielded from being neutralized by the immune system, thereby allowing the enzymes to degrade and clear the pathogenic metabolites that build up in such diseases. RCTs may have a longer and sustained treatment duration and could avoid the immune-driven reduction in efficacy and induction of adverse events associated with currently available therapies.

Our lead programs are targeting rare enzymatic diseases where no available treatment options exist or currently available treatment options may be dramatically improved. Our initial focus is to create RCTs for the potential treatment of phenylketonuria, chronic refractory gout and homocystinuria.

RTX-134

RTX-134 is an RCT that expresses the enzyme phenylalanine ammonia lyase and coverts Phe to the metabolite transcinnamic acid, which is naturally cleared by the body.

Phenylketonuria

We plan to file an IND application for RTX-134 during the first quarter of 2019, for the potential treatment of phenylketonuria, also called PKU, a rare inherited disease that causes build-up of the neurotoxic amino acid phenylalanine. Without the functional enzymes to process phenylalanine (Phe), a dangerous buildup can occur, causing impaired cognitive function.

PKU impacts approximately 15,000 people in the U.S. and 50,000 people worldwide.

RTX-Uricase/URAT1

RTX Uricase/URAT1 is an RCT that has been genetically engineered to express hundreds of thousands of copies of uricase and URAT1, a uric acid transporter that ensures optimal uptake of uric acid into the cell. We expect RTX Uricase/URAT1 to augment a patient’s ability to clear uric acid levels in the blood.

Chronic, Refractory Gout

Gout is a metabolic and inflammatory disease often affecting middle-aged to elderly men and postmenopausal women. After years of repetitive attacks, patients develop chronic refractory gout, which is characterized by the buildup of tophi, or deposits of uric acid crystals in the joints, kidney and heart. Tophi can lead to the development of chronic arthritis and an increased risk of developing kidney stones, chronic renal insufficiency and cardiovascular disease. Once patients reach this stage, they generally suffer multiple attacks every year.

Approximately 50,000 to 60,000 people with gout in the U.S. have a form of the disease that is considered chronic refractory and no longer responsive to available therapies.

RTX-CBS

RTX-CBS is an RCTTM that we have genetically engineered to express hundreds of thousands of copies of CBS in the cell. We expect RTX-CBS will replace the patient’s missing or ineffective enzymes and rapidly drop total plasma homocysteine, or tHcy, levels to clinically meaningful targets through infrequent, low volume intravenous infusions.

Homocystinuria

Homocystinuria refers to a group of enzyme deficiency disorders that result in elevated levels of circulating homocysteine, or Hcy, and its metabolites. The majority of homocystinuria patients have mutations of a gene that regulates the production of the enzyme known as cystathionine beta-synthase, or CBS, which is required for the conversion of Hcy to cystathionine.

Elevated levels of homocysteine result in a wide range of deforming and debilitating symptoms. Without treatment, children may suffer from progressive and severe neurodegeneration. In addition, many of these children will develop psychiatric disturbances and experience seizures. A failure to effectively treat patients over time can also result in aberrant musculoskeletal development, including Marfanoid features, characterized by abnormally long limbs and digits and scoliosis, or spinal curvature. Patients with homocystinuria may also experience extreme hypertension and are at an elevated risk for the development of thromboembolisms. If untreated, approximately 50% of patients will have a thromboembolic event and the overall mortality rate is approximately 20% by age 30.

Patient population estimates range widely, but the National Organization for Rare Disorders suggests a worldwide prevalence of 1:344,000, which when applied to the combined U.S. and E.U. population of 830 million, implies approximately 2,400 patients live with the disease in these regions. However, this is potentially an underrepresentation of the true population size as the scientific literature suggests that current newborn screening tests may not be adequately sensitive or specific.

Oncology

We believe that RCTTM medicines will have broad therapeutic applicability across a range of both solid and hematological cancers.

 

 

We are developing a pipeline of RCT product candidates to target, starve, penetrate and destroy cancer cells, by targeting T cells, NK cells, dendritic cells and tumor cells. Our initial RCTs are based on a 4-1BBL backbone, a ligand that binds to and activates the co-stimulatory receptor 4-1BB, an important regulator of the immune system.

RTX-212

RTX-212 is our first potential treatment for patients with solid tumors whose disease has progressed following checkpoint inhibitor therapy. RTX-212 has been engineered to stimulate both the adaptive and innate arms of the immune system to generate a synergistic response. RTX-212 is designed to co-express 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. We plan to evaluate RTX-212 both as a single agent and in combination with an anti-PD-1 inhibitor.

Solid Tumors

While extraordinary lifespan and quality of life gains have been made with the approval of checkpoint inhibitors, in many cases, the disease fails to respond to therapy or the disease progresses within 6-12 months of treatment due to cellular escape mechanisms that make specific tumors unresponsive to these treatments.

RTX-212

We plan to conduct a Phase 2 trial of RTX-212 administered to AML patients post-allogeneic hematopoietic stem cell transplantation (HSCT) using the single agent dose determined in our initial Phase 1 trial of RTX-212 in solid tumors.

Relapsed or Refractory Acute Myeloid Leukemia

Acute myeloid leukemia, or AML, is characterized by proliferation of myeloid blasts. They replace the bone marrow so that there is minimal production of platelets, red cells and neutrophils. It is primarily a disease of the elderly with a median age of diagnosis of 68.

In 2017, there were more than 20,000 new cases of AML and more than 10,000 deaths caused by AML in the U.S.

Autoimmune disease and beyond

In preclinical studies, our RCTs have shown potential for the treatment of autoimmune diseases. Available therapies are generally administered on a chronic, lifelong basis. Many treatments fail to provide benefit to patients with their disease eventually progressing despite continued therapy, requiring new treatment approaches. Furthermore, these existing treatments are associated with side effects that include opportunistic infections, lymphoma and in some cases severe and even fatal infusion reactions. We believe RCTs can be designed to more specifically modulate complex counter regulatory immune responses and enable greater efficacy with lower toxicity, potentially providing treatments for a number of diseases with high unmet need.

Our RCTs enable precise targeting of specific components of the immune system. We have generated RCTs that express antigens either within the cell or on the cell surface, which we believe could treat antigen specific autoimmune diseases. We expect to select our first clinical candidate in 2019.

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