After 3 years of follow-up in AMPECT trial,
confirmed clinically meaningful overall response rate, including
multiple patients with complete responses
Demonstrated 40 months median duration of
response and median survival >53 months
Historically, median survival has ranged from
16 to 29 months in the setting of metastatic/unresectable malignant
PEComa1,2
LOS
ANGELES, March 1, 2024 /PRNewswire/ -- Aadi
Bioscience, Inc. (NASDAQ: AADI), a biopharmaceutical company
focused on developing and commercializing precision therapies for
patients with mTOR-driven cancers, announced today that long-term
efficacy and safety results from its completed Phase 2
registrational AMPECT study of nab-sirolimus in malignant
PEComa were published in the Journal of Clinical
Oncology (JCO). The manuscript, "A Phase 2 Trial of
nab-Sirolimus in Patients with Advanced Malignant
Perivascular Epithelioid Cell Tumors (AMPECT): Long-term Efficacy
and Safety Update," authored by Andrew J.
Wagner, MD, PhD and colleagues can be accessed online ahead
of print here.
"We are excited to report the final outcomes of this
registrational trial after an additional 3 years of follow-up.
Responses to nab-sirolimus in patients with advanced PEComa
lasted a median of 39.7 months," commented Dr. Wagner, Senior
Physician at the Dana-Farber Cancer Institute and Associate
Professor of Medicine at Harvard Medical
School. "The median survival outcomes were consistent with
the primary analysis, and the confirmed overall response rate
remained at 38.7% and included an additional patient with a
complete response. The long-term, durable responses to
nab-sirolimus with tolerable safety are great news for
patients with this rare disease."
"The data published in JCO highlight the
potential for nab-sirolimus to be an effective and highly
differentiated treatment that may help patients suffering from this
aggressive cancer achieve longer duration of responses and survival
rates," said Loretta Itri, MD, Chief Medical Officer of Aadi.
"We want to thank the principal investigators, study coordinators,
and the patients who participated in AMPECT. We believe these
results provide a significant contribution to sarcoma research and
we look forward to continuing to
advance nab-sirolimus."
The AMPECT trial (NCT02494570) evaluated the efficacy and safety
of nab-sirolimus in patients with advanced malignant
PEComa and was the first prospective registrational study in this
setting. Data from this Phase 2, open-label, single-arm,
multi-center study served as the basis for the FDA approval of
FYARRO® in advanced malignant PEComa regardless of
mutational status.
Key updates reported:
- Efficacy: At study completion, the confirmed ORR on the
basis of independent radiographic review using RECIST v1.1 remained
at 38.7% (95% CI, 21.8%-57.8%), with an additional converted
confirmed complete response (CR) since the previously published
analysis. The disease control rate (DCR) remained at 71% (95% CI,
52.0%–85.8%).
- Durability: At the time of the primary analysis, the
median duration of response (mDOR) had not been reached. At study
completion, the mDOR was reached at 39.7 months (95% CI, 6.5 months
to NR), median PFS remained the same at 10.6 months (95% CI,
5.5-41.2 months), and median OS was 53.1 months (95% CI, 22.2
months to NR).
- Safety: The most common TRAEs were stomatitis (82.4%)
and fatigue and rash (each 61.8%). No new or unexpected adverse
events occurred, and no grade 4 or 5 TRAEs were reported.
- Bleeker JS, et al. Sarcoma. 2012;541626.
- Benson C, et al. Anticancer Res.
2014;34(7):3663-3668.
About Aadi Bioscience, Inc.
Aadi is a commercial-stage biopharmaceutical company focused on
precision therapies for cancers to bring transformational therapies
to cancer patients with alterations in the mTOR pathway, a key
regulator of cell growth and cancer progression. 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 clinical trials of nab-sirolimus
as a single agent or in combination for the treatment of other
mTOR-driven cancers. PRECISION 1 (NCT05103358), a Phase 2
tumor-agnostic registration-intended trial in mTOR inhibitor-naïve
malignant solid tumors
harboring TSC1 or TSC2 inactivating
alterations is currently enrolling in the US and globally. A
multicenter, open-label, single-arm Phase 2 study of
nab-sirolimus in combination with letrozole is currently
enrolling patients with advanced or recurrent endometrioid
endometrial cancer (NCT05997017); nab-sirolimus is also
being investigated as a single agent in an open-label, single-arm,
Phase 2 trial for the treatment of patients with neuroendocrine
tumors of the GI tract, pancreas, or lung (NCT05997056). More
information on the Company's development pipeline is available on
the Aadi website at www.aadibio.com and connect with us
on X (formerly Twitter) and LinkedIn.
Forward-Looking Statements
This press release contains certain forward-looking statements
regarding the business of Aadi Bioscience that are not a
description of historical facts within the meaning of the Private
Securities Litigation Reform Act of 1995. Forward-looking
statements are based on the Company's current beliefs and
expectations and may include, but are not limited to, statements
relating to: the Company's plans and potential for success relating
to commercializing FYARRO; expectations regarding the beneficial
characteristics, safety, efficacy and therapeutic effects of
FYARRO; plans related to further development and manufacturing of
FYARRO; and the sufficiency of the Company's existing capital
resources and the expected timeframe to fund the Company's future
operating expenses and capital expenditure requirements. Actual
results could differ materially from those anticipated in such
forward-looking statements as a result of these risks and
uncertainties, which include, without limitation, those associated
with the ability to successfully commercialize FYARRO;
uncertainties associated with the clinical development and
regulatory approval of FYARRO in additional indications; the risk
that unforeseen adverse reactions or side effects may occur in the
course of commercializing, developing and testing FYARRO; and risks
related to Aadi's estimates regarding future expenses, capital
requirements and need for additional financing.
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 December
31, 2022, including under the caption "Item 1A. Risk Factors," and
in Aadi's subsequent Quarterly Reports on Form 10-Q, and elsewhere
in Aadi's reports and other documents that Aadi has filed, or will
file, with the SEC from time to time and available
at www.sec.gov.
All forward-looking statements in this press release are current
only as of the date hereof and, except as required by applicable
law, Aadi undertakes no obligation to revise or update any
forward-looking statement, or to make any other forward-looking
statements, whether as a result of new information, future events
or otherwise. All forward-looking statements are qualified in their
entirety by this cautionary statement. This cautionary statement is
made under the safe harbor provisions of the Private Securities
Litigation Reform Act of 1995.
Contact:
IR@aadibio.com
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