PRECISION 1 is the first prospective, tumor-agnostic trial of nab-sirolimus for patients who are mTOR inhibitor naive and with malignant solid tumors harboring inactivating alterations in TSC1 or TSC2 genes1
mTOR=mechanistic target of rapamycin; nab=nanoparticle albumin-bound; TSC1=tuberous sclerosis-1; TSC2=tuberous sclerosis-2.
PRECISION 1 is a prospective, phase 2, open-label, tumor-agnostic, multicenter basket trial of nab-sirolimus for patients aged 12 years or older with malignant solid tumors harboring pathogenic inactivating alterations in TSC1 or TSC2 genes, which are tumor suppressor genes upstream in the mTOR pathway.1-3
Enrolled patients (N=~120) will be allocated into 2 treatment arms1,4:
Arm A (N=~60)includes patients with pathogenic inactivating TSC1 alterations
Arm B (N=~60)includes patients with pathogenic inactivating TSC2 alterations
The primary objective of the study is to determine the overall response rate (ORR) in patients harboring pathogenic inactivating alterations in TSC1 or TSC2 genes until disease progression.1
mTOR=mechanistic target of rapamycin; nab=nanoparticle albumin-bound; TSC1=tuberous sclerosis-1; TSC2=tuberous sclerosis-2.
Since TSC1 and TSC2 inactivating alterations can be detected through next-generation sequencing (NGS) of a tumor tissue biopsy, patients who have already received an NGS report of their tumor profile do not require an additional biopsy to be considered for PRECISION 1.1
To enroll in PRECISION 1, patients must meet the following requirements1,4:
ECOG PS=Eastern Cooperative Oncology Group performance status; KPS=Karnofsky performance status; mTOR=mechanistic target of rapamycin; TSC1=tuberous sclerosis-1; TSC2=tuberous sclerosis-2.
TSC1 and TSC2 are tumor suppressor genes upstream in the mTOR pathway. Alterations or deletions in these genes may lead to mTOR overactivation, which prompts uncontrolled cell growth, metabolism, and survival.2,3,5
Alterations or deletions in TSC1 or TSC2 genes have been identified in multiple tumor types throughout the body, including the6-8*:
*Based on number of cases in AACR Project Genomics Evidence Neoplasia Information Exchange (GENIE), an international pan-cancer registry of real-world data assembled through data sharing between 19 leading international cancer centers with the goal of improving clinical decision-making.8
Currently, there are no drugs specifically approved to treat patients with malignant tumors harboring TSC1 or TSC2 inactivating alterations.9-13
AACR=American Association for Cancer Research; mTOR=mechanistic target of rapamycin; TSC1=tuberous sclerosis-1; TSC2=tuberous sclerosis-2.
nab-sirolimus is an mTOR inhibitor that uses nanoparticle albumin-bound (nab) technology engineered to preferentially deliver high levels of drug directly to proliferating tumors for patients aged 12 years or older with pathogenic TSC1 or TSC2 inactivating alterations, regardless of tumor type.14
Based on preclinical data, nab technology enhances tumor penetration and drug accumulation due to the preferential accumulation of albumin in proliferating tumors.14
PRECISION 1 is the first prospective trial designed specifically for patients with solid tumors harboring pathogenic inactivating alterations in TSC1 or TSC2 genes.1
mTOR=mechanistic target of rapamycin; TSC1=tuberous sclerosis-1; TSC2=tuberous sclerosis-2.
Aadi Bioscience, Inc. is a biopharmaceutical company developing precision therapies for genetically-defined cancers. Aadi’s primary goal is to bring transformational therapies to cancer patients with mTOR pathway driver alterations such as inactivating alterations in TSC1 or TSC2 genes, where other mTOR inhibitors have not been or cannot be effectively exploited due to problems of pharmacology, effective drug delivery, safety, or effective targeting to the disease site.
Aadi’s initial focus is the treatment of ultra-rare cancers, including tumor-agnostic TSC1 and TSC2 inactivating alterations in patients aged 12 years or older.
mTOR=mechanistic target of rapamycin; TSC1=tuberous sclerosis-1; TSC2=tuberous sclerosis-2.
Please contact Aadi Bioscience for more information on PRECISION 1, including how to enroll a patient.
Please contact Aadi Bioscience for more information on PRECISION 1, including how to enroll a patient.
Contact Aadi Bioscience
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