Selective mTORC1 Inhibitors

(RTB101 and RTB101/RAD001)

Immune System

TRIAL PHASE
MECHANISMINDICATION(S)PRODUCT NAMEPreclinicalPhase 1Phase 2Phase 3
Selective mTORC1 Inhibitors
Immunosenescence and Aging-Related Disorders, Respiratory Tract Infections
resTORbio
(RTB101 and RTB101/RAD001)

Immune System

Selective mTORC1 Inhibitors
INDICATION(S):Immunosenescence and Aging-Related Disorders, Respiratory Tract Infections
NAME:resTORbio (RTB101 and RTB101/RAD001)
STAGE:Phase 2
Immunosenescence / immuno autophagy

Our Selective TORC1 Inhibitor program is a new approach selectively targeting TORC1 in the mTOR (mechanistic Target Of Rapamycin) pathway, which is linked to increased lifespan and the potential to ameliorate multiple aging-related diseases.

There are limited therapeutic options existing today to prevent and treat RTIs. Some vaccines protect against specific viruses; however, there is no available treatment for most RTIs, which are largely caused by unknown viruses.

Our Selective TORC1 Inhibitor program is an immunotherapy that works by enhancing the immune system to reduce the incidence of RTIs. This program also has potential in other age-related indications including other types of infections, organ failure and neurology indications.


  • Patient Need & Market Potential
    • Immunosenescence, the age-dependent decline in immune function, is associated with a decreased ability to fight infections, an increase in cancer incidence and a decline in organ function in the elderly. With a rapidly aging population, there is an urgent need to address these and other aging-related diseases.
  • Our Approach to Solving the Problem
    • Mechanistic target of rapamycin (mTOR) is a protein serine/threonine kinase that regulates multiple cell functions, including cell growth and metabolism, via two complexes: TORC1 and TORC2
    • TORC1 inhibition has been found to have many beneficial effects on aging, while TORC2 inhibition has been associated with adverse events including decreased lifespan, hyperglycemia and hypercholesterolemia¬†
    • Our product candidates selectively inhibit mTORC1 and may therefore have therapeutic potential to ameliorate multiple aging-related conditions with a favorable safety profile. Preclinical data suggests that TORC1 inhibitors may enhance immune response to vaccines improve tendon stiffening, cardiac dysfunction, cognitive dysfunction, aging-related decreased mobility and laminopathies.
  • Intellectual Property
    • The intellectual property portfolio covers the composition of matter, methods of use and manufacture of selective mTORC1 inhibitor molecules
    • Our mTORC1 program has broad worldwide intellectual property coverage, including exclusive rights to over 128 issued patents and patent applications
  • Team
    • The program is led by Chen Schor, Dr. Joan Mannick, and Sarb Shergill.
  • Expected Milestones and Timing
    • A Phase 2b study is currently in progress to determine if multiple doses of RTB101 alone or in combination with RAD001 decrease the incidence of respiratory tract infections (RTI) in elderly patients at increased risk of RTI-related morbidity and mortality. We plan to complete the study during the second half of 2018

Our Selective TORC1 Inhibitors target pathways that may revitalize immune homeostasis to help address aging-related diseases. We have initiated a Phase 2b trial for our lead product candidate, RTB101, which builds upon two Phase 2a clinical studies demonstrating promising safety and efficacy results in almost 500 elderly individuals.

We are taking an innovative and pragmatic clinical development path to regulatory approval in specific aging-related conditions and have a high sense of urgency to make our medicines available globally.