Ayala Pharmaceuticals Announces Publication Highlighting Clinical Activity of its Gamma Secretase Inhibitor AL101 in Desmoid Tumors
23 September 2021 - 2:30PM
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 publication of two case
studies of adult patients with desmoid tumors treated with AL101 in
Current Oncology. This publication highlights the potential of a
gamma secretase inhibitor for the treatment of desmoid tumors.
The data included in the case study are based on earlier Phase 1
results and compassionate use of AL101 in desmoid tumors. Both
patients showcased in these case studies, Case One and Case Two,
presented with significant tumor burden and symptomatic and
life-threatening disease due to disease bulk and location. Both
patients achieved long-lasting partial responses (PR) with AL101
treatment with a maximal decrease in tumor size from baseline of
41% after approximately 1 year (55 weeks) of treatment in Case One,
and a maximal decrease in tumor size from baseline of 60% after
about 1.6 years (82 weeks) of treatment in Case Two. With continued
monitoring, one patient was able to discontinue AL101 after 4.6
years of treatment, while maintaining a PR, and the other patient
has maintained a PR at a reduced AL101 dose.
“Both of these patients’ case studies represent additional
evidence to support the development of our gamma secretase
inhibitor, AL102 for the treatment of desmoid tumors. The body of
data conducted by BMS in patients with desmoid tumors implicating
the role of gamma secretase inhibition furthers our hypothesis for
treating desmoid tumors through AL102’s mechanism of action,” said
Roni Mamluk, Ph.D., Chief Executive Officer of Ayala. “Desmoid
tumors continue to be an area of high unmet medical with a
significant impact to patients’ quality of life, and we are pleased
to have initiated our pivotal Phase 2/3 RINGISDE trial of AL102 to
potentially address this gap in the existing treatment
paradigm.”
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 is 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.
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.
AL102 is currently in a Pivotal Phase 2/3 clinical trials for
patients with Desmoid Tumors (RINGSIDE) and is being evaluated in a
Phase 1 clinical trial in combination with Novartis’ BMCA targeting
agent, WVT078, in patients with relapsed/refractory Multiple
Myeloma. For more information, visit www.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, including its treatment potential, the
promise and potential impact of our preclinical or clinical trial
data, and the timing of additional data from clinical trials of
AL101. 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 our Annual Report on Form 10-K for the
year ended December 31, 2020 filed with the U.S. Securities and
Exchange Commission (SEC) on March 24, 2021 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.
Investors: Julie Seidel Stern Investor
Relations, Inc. +1-212-362-1200 Julie.seidel@sternir.com
Ayala Pharmaceuticals: +1-857-444-0553
info@ayalapharma.com
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