Ayala Pharmaceuticals, Inc. (NASDAQ: AYLA), a clinical-stage
oncology company focused on developing and commercializing small
molecule therapeutics for patients suffering from rare and
aggressive cancers, today announced the completion of its
end-of-Phase 1 meeting with the U.S. Food and Drug Administration
(FDA) on AL102, a potent, selective, oral gamma secretase inhibitor
(GSI), for the treatment of desmoid tumors. The FDA has agreed,
based on data from AL101 and AL102 studies including durable
responses observed in patients with Desmoid tumors, to proceed with
a Phase 2/3 pivotal study, which can potentially be used as a
registrational study. Ayala expects to initiate the pivotal Phase
2/3 RINGSIDE study in adult and adolescent patients with desmoid
tumors in the first half of 2021.
“This exciting news of entering into a potentially
registration-enabling pivotal trial, earlier than expected,
represents an important step for Ayala as we are able to accelerate
the development of AL102 for the treatment of desmoid tumors based
on positive and encouraging feedback from the FDA following our
end-of-Phase 1 meeting,” said Roni Mamluk, Ph.D., Chief Executive
Officer of Ayala. “Desmoid tumors are a rare, debilitating and
often disfiguring class of soft-tissue tumors for which there are
currently no approved therapies. We believe AL102 is well
positioned to potentially provide effective systemic treatment
based on the body of data conducted by BMS in patients with desmoid
tumors implicating the role of Notch pathway in activating aberrant
growth pathways contributing to desmoid tumor growth.”
The pivotal Phase 2/3 RINGSIDE trial is designed to evaluate the
efficacy, safety and tolerability of AL102 in adult and adolescent
patients with desmoid tumors. Part 1 of the study will be open
label and will enroll up to 36 patients with progressive desmoid
tumors in three study arms across three doses of AL102: 1.2 mg
daily (QD), 2 mg twice weekly (QIW), and 4mg twice weekly (QIW)
with initial follow up of safety, tolerability and tumor volume by
MRI after 16 weeks in order to determine the optimal dose. At the
end of part 1, all patients will be eligible to enroll into an open
label extension study at the selected dose where long-term efficacy
and safety will be monitored.
Part 2 of the study will start immediately after dose selection
from part 1 and will be a double-blind placebo-controlled study
enrolling up to 156 patients with progressive disease, randomized
2:1 between AL102 or placebo. The study’s primary endpoint will be
progression free survival (PFS) with secondary endpoints including,
objective response rate (ORR), duration of response (DOR) and
patient reported Quality of Life (QOL) measures.
The study is expected to commence in the first half of 2021 with
an initial interim data read-out from part 1 and dose selection
expected by mid-2022 with part 2 of the study to commence
immediately thereafter.
About AL102
AL102 is a potent, selective, oral gamma secretase inhibitor
(GSI). AL102 is currently being developed for the treatment of
desmoid tumors, as well as in combination with Novartis’ B-cell
maturation antigen (BCMA)-targeting agents for the treatment of
multiple myeloma (MM).
About Desmoid Tumors
Desmoid tumors, also called aggressive fibromatosis or
desmoid-type fibromatosis, are rare connective tissue tumors that
typically arise in the upper and lower extremities, abdominal wall,
head and neck area, mesenteric root and chest wall with the
potential to arise in additional parts of the body. Desmoid tumors
do not metastasize, but often aggressively infiltrate neurovascular
structures and vital organs. People living with desmoid tumors are
often limited in their daily life due to chronic pain, functional
deficits, general decrease in their quality of life and organ
dysfunction. Desmoid tumors have an annual incidence of
approximately 1,700 patients in the United States and typically
occur in patients between the ages of 15 and 60 years. They are
most commonly diagnosed in young adults between 30-40 years of age
and are more prevalent in females. Today, surgery is no longer
regarded as the cornerstone treatment of desmoid tumors due to high
rate of recurrence post-surgery and there are currently
no FDA-approved systemic therapies for the treatment of
unresectable, recurrent or progressive desmoid tumors.
About Ayala Pharmaceuticals
Ayala Pharmaceuticals, Inc. is a clinical-stage oncology company
focused on developing and commercializing small molecule
therapeutics for patients suffering from rare and aggressive
cancers, primarily in genetically defined patient populations.
Ayala’s approach is focused on predicating, identifying and
addressing tumorigenic drivers of cancer through a combination of
its bioinformatics platform and next-generation sequencing to
deliver targeted therapies to underserved patient populations. The
company has two product candidates under development, AL101 and
AL102, targeting the aberrant activation of the Notch pathway with
gamma secretase inhibitors to treat a variety of tumors including
Adenoid Cystic Carcinoma, Triple Negative Breast Cancer (TNBC),
T-cell Acute Lymphoblastic Leukemia (T-ALL), Desmoid Tumors and
Multiple Myeloma (MM) (in collaboration with Novartis). AL101, has
received Fast Track Designation and Orphan Drug Designation from
the U.S. FDA and is currently in a Phase 2 clinical trial for
patients with ACC (ACCURACY) bearing Notch activating mutations and
in a Phase 2 clinical trial for patients with TNBC (TENACITY)
bearing Notch activating mutations and other gene rearrangements.
Ayala expects to initiate the pivotal Phase 2/3 RINGSIDE study of
AL102 for the treatment of desmoid tumors in the first half of
2021. For more information, visit www.ayalapharma.com.
Contacts:
Investors:Julie SeidelStern Investor Relations,
Inc.+1-212-362-1200Julie.seidel@sternir.com
Ayala
Pharmaceuticals:+1-857-444-0553info@ayalapharma.com
Forward-Looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995. All statements contained in this press release that do not
relate to matters of historical fact should be considered
forward-looking statements, including statements relating to our
development of AL101 and AL102, the timing, initiation, design,
timing and potential of the Phase 2/3 RINGSIDE study, the promise
and potential impact of our preclinical or clinical trial data, the
timing of and plans to initiate additional clinical trials of
AL101, AL102, upcoming milestones, including without limitation the
timing and results of any clinical trials or readouts, and the
sufficiency of cash to fund operations. These forward-looking
statements are based on management’s current expectations. The
words “may,” “will,” “should,” “expect,” “plan,” “anticipate,”
“could,” “intend,” “target,” “project,” “estimate,” “believe,”
“predict,” “potential” or “continue” or the negative of these terms
or other similar expressions are intended to identify
forward-looking statements, although not all forward-looking
statements contain these identifying words. These statements are
neither promises nor guarantees, but involve known and unknown
risks, uncertainties and other important factors that may cause our
actual results, performance or achievements to be materially
different from any future results, performance or achievements
expressed or implied by the forward-looking statements, including,
but not limited to, the following: the impact of the COVID-19
pandemic on our operations, including our preclinical studies and
clinical trials, and the continuity of our business; we have
incurred significant losses, are not currently profitable and may
never become profitable; our need for additional funding; our cash
runway; our limited operating history and the prospects for our
future viability; the lengthy, expensive, and uncertain process of
clinical drug development, including potential delays in regulatory
approval; our requirement to pay significant payments under product
candidate licenses; the approach we are taking to discover and
develop product candidates and whether it will lead to marketable
products; the expense, time-consuming nature and uncertainty of
clinical trials; enrollment and retention of patients; potential
side effects of our product candidates; our ability to develop or
to collaborate with others to develop appropriate diagnostic tests;
protection of our proprietary technology and the confidentiality of
our trade secrets; potential lawsuits for, or claims of,
infringement of third-party intellectual property or challenges to
the ownership of our intellectual property; risks associated with
international operations; our ability to retain key personnel and
to manage our growth; the potential volatility of our common stock;
costs and resources of operating as a public company; unfavorable
or no analyst research or reports; and securities class action
litigation against us. These and other important factors discussed
under the caption “Risk Factors” in Quarterly Report on Form 10-Q
for the three months ended September 30, 2020 filed with the U.S.
Securities and Exchange Commission (SEC) on November 13, 2020 and
our other filings with the SEC could cause actual results to differ
materially from those indicated by the forward-looking statements
made in this press release. Any such forward-looking statements
represent management’s estimates as of the date of this press
release. New risk factors and uncertainties may emerge from time to
time, and it is not possible to predict all risk factors and
uncertainties. While we may elect to update such forward-looking
statements at some point in the future, except as required by law,
we disclaim any obligation to do so, even if subsequent events
cause our views to change. Although we believe the expectations
reflected in such forward-looking statements are reasonable, we can
give no assurance that such expectations will prove to be correct.
These forward-looking statements should not be relied upon as
representing our views as of any date subsequent to the date of
this press release.
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