On June 4, 2021 Aadi Bioscience, Inc. ("Aadi"), a privately-held clinical-stage biopharmaceutical company focusing on precision therapies for genetically-defined cancers with alterations in mTOR pathway genes, reported preliminary data from its lead investigational product candidate, FYARRO (sirolimus albumin-bound nanoparticles for injectable suspension; nab-sirolimus; ABI-009), during the 2021 ASCO (Free ASCO Whitepaper) Annual Meeting being held virtually from June 4-8, 2021 (Press release, Aadi Bioscience, JUN 4, 2021, View Source [SID1234583540]). The poster is entitled "Institutional experience with nab-sirolimus in patients with malignancies harboring TSC1 or TSC2 mutations".1
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Dr. Mark Dickson, Principal Investigator of the study at Memorial Sloan Kettering Cancer Center stated, "I am encouraged by the activity of nab-sirolimus in multiple solid tumor histologies with TSC1 or TSC2 inactivating alterations. These new data provide strong rationale for conducting a broader investigation of nab-sirolimus in a tumor-agnostic setting. If the observed activity were reproduced, this could represent a meaningful advance in treatment for these patients."
Analysis from Expanded Access Patient Subset
Eight patients with malignancies and neoplasms bearing TSC1 or TSC2 inactivating alterations and representing histologies other than malignant PEComa were treated in a multi-institution expanded access program (NCT03817515) with FYARRO at 100 mg/m2 on day one and day eight of a 21-day cycle. RECIST v1.1 criteria were used for response analysis. Data cutoff occurred in March 2021.
Patients had a median of 3.5 lines of prior therapy and 6 of 8 patients were mTOR inhibitor-naïve. Treatment duration for all patients ranged from 0.7 to 12.0+ months. Five of 8 patients continued on treatment as of the data cutoff and 3 of 8 patients discontinued. Reasons for discontinuation were progressive disease (2 patients) and an adverse event (1 patient with acute kidney injury possibly secondary to administration of contrast).
Of the 8 patients treated, 7 patients were evaluable for response analysis and 1 patient progressed before the first scan. Five of 8 patients (63%, 95% CI: 25%-92%) achieved a confirmed partial response (PR). Amongst the patients who were mTOR inhibitor-naïve, 5 of 6 (83%, 95% CI: 36%-99+%) achieved a confirmed PR. Duration of response at data cutoff ranged from 3.1 to 9.7+ months and 3 of 5 responders continue on treatment.
Treatment-emergent adverse events that were ≥30% included edema, infections, mucositis, and pain (71% each), nail changes and vomiting (57% each), and hypertension and nausea (43% each). The majority of events were grade one or grade two. Treatment-related serious adverse events were reported in 2 patients and included hyperglycemia and infection; and acute kidney injury possibly secondary to administration of contrast. Three of 8 patients had a dose reduction from 100 mg/m2 to 75 mg/m2.
Dr. Neil Desai, founder and chief executive officer of Aadi, stated, "We are pleased to have provided FYARRO to patients through our expanded access program. Based on the data in this initial group of patients, and patients in the AMPECT trial with PEComa, we are planning to move forward with a tumor-agnostic registrational trial to confirm FYARRO’s activity in solid tumors with TSC1 or TSC2 inactivating alterations with planned initiation by the end of 2021."