Today, we are pleased to announce that the results are in from a human trial that targets the aging immune system and that an immune system-boosting drug appears to be effective.

Targeting TORC1 to boost the immune system

As we age, the immune system becomes increasingly poor and unable to detect and fight infections. This can make the elderly vulnerable to respiratory tract infections (RTIs), thus impacting their health and quality of life and leaving them at risk of dying from such infections. There are currently no treatments for RTIs that target the aging immune system directly.

Recent research suggests that the aging processes cause the immune system to break down and are influenced by a number of specific signaling pathways, including the mTOR (mammalian target of rapamycin) pathway. mTOR is a protein kinase which signals via two multiprotein complexes: TORC1 and TORC2.

Inhibiting TORC1 has been shown to increase lifespan in mice and other species, boost immune function, ameliorate heart failure, enhance memory and mobility, decrease obesity, and delay the onset of age-related diseases. However, inhibiting TORC2 has been demonstrated to reduce lifespan in animals and cause hyperlipidemia and hyperglycemia in animals and humans.







Therefore, resTORbio developed RTB101, a drug that inhibits TORC1 but leaves TORC2 untouched and free to work as normal. The hope is that such a drug could help to reduce the incidence of RTIs in elderly people and improve their quality of life and health.

Positive results from phase 2b human clinical trial

Today, resTORbio, Inc. has published positive results from its Phase 2b dose-ranging clinical trial. The trial saw 652 aged people with increased risk of RTIs enroll in a test RTB101, a potent inhibitor of rapamycin complex 1 (TORC1).

Compared to the control group, there were significantly fewer patients treated with RTB101 who suffered from one or more RTIs during a 16-week trial period. This was achieved using a daily oral dose of 10 mg. Combining RTB101 with everolimus, which further inhibits TORC1, failed to achieve the endpoint of reducing RTI incidence.

This suggests that the optimal dosage is to only use 10 mg of RTB101 to provide a lower level of inhibition. These findings will provide the basis for future studies. The drug is also reported to have been well-tolerated in high-risk patients.







The full press release and trial details are available here.