Our Pipeline

We are a clinical-stage biopharmaceutical company developing innovative medicines that target the biology of aging to prevent or treat aging-related diseases.

Our lead program selectively inhibits the activity of a protein complex called target of rapamycin complex 1, or TORC1, an evolutionarily conserved pathway that contributes to the decline in function of multiple aging organ systems. Our lead product candidate is a TORC1 inhibitor called RTB101.

We are developing RTB101 alone and in combination with other TORC1 inhibitors for multiple aging-related diseases. In addition to developing our TORC1 product candidates, we are building a pipeline of products targeting multiple mechanisms underlying the biology of aging.

resTORbio Pipeline 12 2 2019 v2
* For Parkinson’s disease, we may be required to file an investigational new drug application, or IND, prior to initiating Phase 2 clinical trials
** For neurodegenerative diseases and diseases associated with TORC1 hyperactivation, subject to review by the U.S. Food and Drug Administration, we believe we may have the ability to initiate Phase 2 clinical trials without the need to conduct additional Phase 1 trials.

About RTB101

Our lead product candidate, RTB101, is an investigational, oral, selective, and potent TORC1 inhibitor.

RTB101 inhibits the phosphorylation of multiple targets downstream of TORC1. Inhibition of TORC1 has been observed to extend lifespan and healthspan in aging preclinical species and to improve the function of multiple organ systems, including neurologic function, suggesting potential benefits in several aging-related diseases.

We currently have a Phase 1b/2a program for RTB101 alone and in combination with sirolimus in Parkinson’s disease. We expect to develop RTB101 for additional indications in other aging-related diseases.

pic pipeline about rbt101

About Parkinson’s Disease

Parkinson’s disease, or PD, is a progressive neurodegenerative disease that affects approximately 7.5 million people worldwide.

The incidence of PD increases rapidly in people 60 years of age and older, with a mean age at diagnosis of 70.5 years. In PD, neurodegeneration is thought to be caused, at least in part, by the accumulation of aggregates of a protein called alpha-synuclein that are toxic to neurons in the brain. Patients with PD develop shaking, rigidity, slowness of movement and difficulty walking.

Selective and broad inhibition of TORC1 has been shown to ameliorate neurodegenerative disease in several preclinical studies, including models of PD. TORC1 inhibition with RTB101 in combination with sirolimus, another TORC1 inhibitor, may provide a therapeutic benefit to PD patients by potentially inducing autophagy to clear the toxic alpha-synuclein aggregates in neurons.

We initiated our Phase 1a/2b clinical trial for PD in the first quarter of 2019. The trial is evaluating the safety, tolerability and brain penetration of RTB101 alone or in combination with sirolimus in PD patients.
Data from this trial is expected in mid-2020.

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