Aadi Bioscience Reports Interim Results from PRECISION1 Trial of nab-Sirolimus Demonstrating Anti-Tumor Activity in Solid Tumors with TSC1 or TSC2 Inactivating Alterations
Interim results from investigator-assessed responses in first 40 patients from TSC1 and TSC2 arms demonstrate sustained tumor reductions in heavily pre-treated population
80 patients now enrolled in PRECISION1 supporting two-thirds interim analysis expected in 3Q 2024
Study on track for completion by end 2024; final data readout expected in early 2025
Company to host conference call today at 5:00 pm EST
"Our tumor agnostic PRECISION1 trial is designed to elucidate the impact of nab-sirolimus on cancers expressing inactivating alterations of TSC1 or TSC2, regardless of tumor type. We are encouraged by the preliminary data from this pre-planned analysis and by the responses and clinical benefit demonstrated in advanced cancer patients who have failed an average of three prior lines of therapy," said Loretta Itri, MD, CMO of
The interim analysis includes data from the first third of trial participants (n=40) with a minimum of 4.5 months of follow-up, including investigator-assessed response and safety analyzed separately in each of the TSC1 and TSC2 arms. Nine different tumor types were enrolled in the TSC1 arm and 13 tumor types were enrolled in the TSC2 arm.
Efficacy of nab-sirolimus in patients with tumors harboring pathogenic inactivating alteration in TSC1
Of the 22 patients enrolled, 19 patients received ≥ 1 post baseline scan and were evaluable for efficacy. Observations included:
- A 26% Overall Response Rate (ORR) including 5 partial responses (PR) with 4 confirmed responses and 1 unconfirmed response (uPR)
- All responses were ongoing at the time of data cutoff. The patient with uPR remains on treatment and is awaiting a confirmatory scan
- 9 patients had stable disease (SD), 3 of which were greater than or equal to six months in duration, resulting in a clinical benefit rate of 42% (5 PR + 3 SD ≥ 6 mos)
- Patients were heavily pre-treated with median of 3 prior lines of therapy
- Median time to response was 1.4 months
- Responses were seen across four different epithelial carcinomas
- 60% of responders experienced > 50% tumor reduction
Efficacy of nab-sirolimus in patients with tumors harboring pathogenic inactivating alteration in TSC2
Of the 18 patients enrolled, all 18 patients received ≥ 1 post baseline scan and were evaluable for efficacy. Observations included:
- An 11% ORR including 2 PRs with 1 confirmed and 1 uPR
- 12 patients had SD, 3 of which were greater than or equal to six months resulting in a clinical benefit rate of 28% (2 PR + 3 SD ≥ 6 mos)
- Patients were heavily pre-treated with median of 3.5 prior lines of therapy; 50% had ≥ 5 prior lines of therapy
- Responses were seen in one epithelial carcinoma and one sarcoma
No new safety signals were observed, and no grade four treatment-related events or deaths occurred. One patient discontinued the study due to grade two pneumonitis that completely resolved after discontinuation of therapy. Across both arms, the safety profile was consistent with the nab-sirolimus label and the mTOR inhibitor drug class.
80 patients are currently enrolled in the PRECISION1 trial, supporting the two-thirds interim analysis expected in the third quarter of 2024. The ORR analysis in this cohort will be based on independent radiological review with a minimum of six months of follow-up for all patients. The trial is expected to be completed by the end of 2024 with results anticipated in early 2025.
Conference Call Information
The Aadi management team is hosting a conference call and webcast today at
Participants may access a live webcast of the call and the associated slide presentation on these data through the "Investors & News" page of the
The PRECISION1 trial is a multi-center, open-label, tumor-agnostic prospective registration intended clinical trial of nab-sirolimus. This tumor agnostic study will evaluate approximately 60 mTOR inhibitor naïve patients in each of two independent study arms, or approximately 120 in total, comprised of patients with solid tumors harboring pathogenic inactivating alterations in either TSC1 or TSC2 genes. In
Nab-Sirolimus 100 mg/m2 is given weekly intravenously over 30 minutes on Days 1 and 8 of each 21-day cycle. The primary endpoint is overall response rate per independent radiographic review (IRR) using RECIST v1.1. Other endpoints include duration of response, time to response, progression-free survival by IRR, overall survival, patient-reported quality of life, and safety.
About Aadi Bioscience
Aadi is a commercial-stage biopharmaceutical company focused on precision therapies for genetically defined cancers to bring transformational therapies to cancer patients with mTOR pathway driver alterations. Aadi received FDA approval and has commercialized FYARRO® for the treatment of adult patients with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor (PEComa).
Aadi has also initiated PRECISION1, a Phase 2 tumor-agnostic registration-intended trial in mTOR inhibitor-naïve malignant solid tumors harboring TSC1 or TSC2 inactivating alterations. More information on the Company's development pipeline is available on the Aadi website at www.aadibio.com and connect with us on Twitter and LinkedIn.
This press release contains certain forward-looking statements regarding the business of
Additional risks and uncertainties that could cause actual outcomes and results to differ materially from those contemplated by the forward-looking statements are included in the Company's Annual Report on Form 10-K for the fiscal year ended
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