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
LOS
ANGELES, Dec. 14, 2023 /PRNewswire/ -- Aadi
Bioscience, Inc. (NASDAQ: AADI), a biopharmaceutical company
focused on developing and commercializing precision therapies for
genetically defined cancers with alterations in mTOR pathway genes,
today reported results from a planned interim analysis on the first
third of participants in the ongoing tumor-agnostic PRECISION1
trial evaluating nab-sirolimus in patients with TSC1
or TSC2 inactivating alterations.
"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 Aadi Bioscience. "Full enrollment in the trial is
expected by the spring of 2024 and we believe we are on track to
generate compelling clinical evidence for advancing
nab-sirolimus toward potential expansion of the current
registration, bringing this innovative therapeutic agent to more
cancer patients."
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 5:00 pm ET
(2:00 pm PT) to discuss the interim
results from the PRECISION1 trial.
Participants may access a live webcast of the
call and the associated slide presentation on these data through
the "Investors & News" page of the Aadi Bioscience website at
aadibio.com. To participate via telephone, please register in
advance at this link. Upon registration, all telephone participants
will receive a confirmation email detailing how to join the
conference call, including the dial-in number along with a unique
passcode and registrant ID that can be used to access the call. A
replay of the conference call and webcast will be archived on the
Company's website for at least 30 days.
About PRECISION1
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
September 2021, the FDA designated
the investigation of nab-sirolimus for the treatment of
adults and adolescents with solid tumors that have a pathogenic
inactivating alteration of the TSC1 or TSC2 gene as a Fast Track
development program.
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.
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 anticipated timing of commencement,
enrollment, data releases and completion of the Company's clinical
trials, including the expected full enrollment of the PRECISION 1
trial by spring of 2024, the expected PRECISION 1 two-thirds
interim analysis in 3Q 2024, the anticipated completion of the
PRECISION 1 study by the end of 2024, and the final PRECISION 1
data readout anticipated in early 2025; management's belief that
the Company is on track to generate additional clinical evidence in
the PRECISION 1 study and for advancing nab-sirolimus toward
registration; the timing and likelihood of regulatory filings and
approvals of FYARRO for new indications; the anticipated timing for
potential catalysts based on data for the Company's clinical
trials; 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, risks related to
the release of interim, topline and preliminary data from clinical
trials; uncertainties associated with the clinical development and
regulatory approval of FYARRO, including potential delays in the
commencement, enrollment and completion of clinical trials; the
risk that unforeseen adverse reactions or side effects may occur in
the course of commercializing, developing and testing FYARRO; risks
associated with the failure to realize further value from FYARRO in
light of inherent risks and difficulties involved in successfully
bringing FYARRO to market in additional indications, including in
patients harboring TSC1 and TSC2 inactivating
alterations; and risks related to the Company'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:
Marcy
Graham
IR@aadibio.com
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SOURCE Aadi Bioscience