AN2 to Voluntarily Pause Enrollment in Phase 3 Part of Phase 2/3 Pivotal Clinical Trial Evaluating Epetraborole for Treatment-Refractory Mycobacterium Avium Complex (MAC) Lung Disease
February 12 2024 - 5:45AM
Business Wire
-Enrolled patients allowed to continue on
study-
AN2 Therapeutics, Inc. (Nasdaq: ANTX), a clinical-stage
biopharmaceutical company focused on developing treatments for
rare, chronic, and serious infectious diseases with high unmet
needs, today announced the company’s decision to voluntarily pause
Phase 3 enrollment in the seamless Phase 2/3 clinical trial
(EBO-301) evaluating epetraborole in treatment-refractory MAC lung
disease, pending further data review. The double-blind,
placebo-controlled trial has two arms comparing epetraborole plus
optimized background regimen (OBR) versus placebo plus OBR. The
voluntary pause was instituted following a blinded aggregate
analysis of the ongoing Phase 2 study, including pooled patients
from both treatment arms, which showed potentially lower than
expected efficacy. The decision was not due to safety concerns. The
Phase 2 part of the trial is intended to inform the Phase 3 part of
the trial. The company continues to expect to announce topline data
from the Phase 2 part of the trial in summer 2024.
The company’s decision to pause enrollment enables time to
further evaluate study data and for the independent Data Safety
Monitoring Board (DSMB) to conduct an unblinded assessment and
recommend next steps, which could include changes to the Phase 3
part of the study protocol. The Phase 2 part of the trial completed
enrollment in September 2023 with 80 patients. The Phase 3 part of
the trial has enrolled nearly 100 patients to date, a faster
enrollment rate than previously anticipated. Patients already
enrolled in the Phase 2/3 trial will be allowed to continue on
study.
“Developing new therapies for patients suffering with
treatment-refractory MAC lung disease, a disease with limited
treatment options and low response rates to existing therapies, is
a complex undertaking. In light of recently available blinded
aggregate data, we believe our voluntary decision to pause
enrollment in the Phase 3 part of the study will provide us the
opportunity to further evaluate study data, and determine the best
path forward,” said Eric Easom, Co-Founder, President and Chief
Executive Officer of AN2 Therapeutics. “We plan to provide further
details regarding the epetraborole Phase 2/3 clinical trial in the
second quarter of 2024 following completion of our analysis.”
About the Epetraborole Pivotal Phase 2/3 Trial (EBO-301)
This double-blind, placebo-controlled superiority trial uses an
adaptive design to evaluate the safety and efficacy of epetraborole
in patients with treatment-refractory MAC lung disease. The Phase 2
part of the trial (n=80) assesses clinical response using various
patient-reported outcome tools as well as the safety, efficacy and
pharmacokinetics of epetraborole plus an OBR, when compared to a
placebo plus an OBR. The Phase 2 findings will be utilized to
inform the clinical response measures evaluated in the Phase 3 part
of the trial and confirm the final sample size. The primary
objective in Phase 3 (n=at least 234) will be to determine if
epetraborole plus an OBR, consisting of two or more
standard-of-care drugs, is superior to placebo plus an OBR. For
more information, please visit: www.clinicaltrials.gov
(NCT05327803).
About AN2 Therapeutics, Inc.
AN2 Therapeutics, Inc. is a clinical-stage biopharmaceutical
company developing treatments for rare, chronic, and serious
infectious diseases with high unmet needs. Our initial candidate is
epetraborole, which we are studying as a once-daily, oral treatment
with a novel mechanism of action for patients with nontuberculous
mycobacteria (NTM) lung disease, a rare, chronic, and progressive
infectious disease caused by bacteria known as mycobacteria, that
leads to irreversible lung damage and can be fatal. For more
information, please visit our website at
www.an2therapeutics.com.
Forward-Looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995. Forward-looking statements expressed or implied in this press
release include, but are not limited to, statements regarding:
timing of topline data, further evaluation of study data and DSMB
review; potential changes to the Phase 3 study protocol;
continuation of enrolled patients in the Phase 2/3 trial; timing of
completion of AN2’s analysis of study data; and other statements
that are not historical fact. These statements are based on AN2’s
current estimates, expectations, plans, objectives, and intentions,
are not guarantees of future performance and inherently involve
significant risks and uncertainties. Actual results and the timing
of events could differ materially from those anticipated in such
forward-looking statements as a result of these risks and
uncertainties, which include, but are not limited to, risks and
uncertainties related to: the recommendations of the DSMB following
its review of blinded data; the number of patients who remain
enrolled in the Phase 2/3 trial; the ability of AN2 to resume
enrollment in the Phase 2/3 trial, in the event the determination
to resume enrollment is made; the ability of AN2 to effectively and
timely make amendments to the Phase 2/3 pivotal trial design based
on its analysis of the Phase 2 portion of the study and/or pursuant
to additional FDA feedback; possible changes to AN2’s plans or
priorities as it assesses study data; potential for protocol
modifications, redesign or study termination; timely enrollment of
patients in AN2’s existing and future clinical trials; AN2’s
ability to procure sufficient supply of its product candidate for
its existing and future clinical trials; the potential for results
from clinical trials to differ from preclinical, early clinical,
preliminary, or expected results; significant adverse events,
toxicities, or other undesirable side effects associated with AN2’s
product candidate; the significant uncertainty associated with
AN2’s product candidate ever receiving any regulatory approvals;
AN2’s ability to obtain, maintain, or protect intellectual property
rights related to its current and future product candidates;
implementation of AN2’s strategic plans for its business and
current and future product candidates; the sufficiency of AN2’s
capital resources and need for additional capital to achieve its
goals; global macroeconomic conditions and global conflicts; and
other risks, including those described under the heading “Risk
Factors” in AN2’s reports filed with the U.S. Securities and
Exchange Commission (SEC), including AN2’s Quarterly Report on Form
10-Q for the quarter ended September 30, 2023. These filings, when
made, are available on the investor relations section of AN2’s
website at investor.an2therapeutics.com and on the SEC’s website at
www.sec.gov. Forward-looking statements contained in this press
release are made as of this date, and AN2 undertakes no duty to
update such information except as required under applicable
law.
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version on businesswire.com: https://www.businesswire.com/news/home/20240212430072/en/
COMPANY CONTACT: Lucy O. Day Chief Financial Officer
l.day@an2therapeutics.com
INVESTOR AND MEDIA CONTACT: Anne Bowdidge
ir@an2therapeutics.com
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