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* For heart failure with preserved ejection fraction and certain other indications, we may be required to file an investigational new drug application, or IND, prior to initiating Phase 2 clinical trials. We expect to have the ability to initiate these Phase 2 clinical trials without the need to conduct additional Phase 1 trials.


About Respiratory Tract Infections in Older Adults

As part of the aging process, the immune system weakens and becomes less effective at detecting and fighting infections such as respiratory tract infections, or RTIs. As a result, RTIs are more likely to be of greater severity, prolonged duration, and are more likely to be associated with medical complications in people age 65 years and older as compared to younger adults. In the U.S., RTIs are the fourth leading cause of hospitalization and seventh leading cause of death in people aged 65 and older. Given that RTIs are caused by many different types of viruses, most of which lack effective therapies, there remains a significant unmet medical need for an immunotherapy that enhances the ability of the immune system to fight multiple viruses to reduce illness associated with RTIs in older adults.


About Parkinson’s Disease in Older Adults

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. Glucocerebrosidase, or GBA1, gene mutations are the most common of the currently known PD-related genetic mutations and up to 10 percent of people with PD in the United States carry it. Patients with PD develop shaking, rigidity, slowness of movement and difficulty walking. PD may be attributed in part to neuronal damage caused by the accumulation within neurons of abnormal aggregates containing the protein α-synuclein. Preclinical studies of PD have shown that mTOR inhibition can induce autophagy, reduce α-synuclein accumulation and decrease neuronal cell death. Therefore, induction of autophagy with RTB101 in combination with a rapalog, such as sirolimus, may have a therapeutic benefit for patients with PD. 


About RTB101

RTB101 is an oral, selective, and potent TORC1 inhibitor being investigated for the potential treatment of aging-related diseases, including illness associated with RTIs and Parkinson’s Disease. RTB101 inhibits TORC1 by binding to the active site of mTOR on the TORC1 complex, a mechanism known as catalytic inhibition. TORC1 inhibition has shown to be of therapeutic benefit in multiple aging-related diseases conditions in preclinical studies, including immunosenescence and several neurologic diseases.


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