First scientific presentation of previously
announced data from Cohort 1 of the ongoing Phase 1b clinical trial evaluating low-dose LTI-03 (2.5
mg BID) in IPF, affirms positive trends in seven of the eight
biomarkers evaluated, suggesting potential therapeutic
effect
Recently completed enrollment of Cohort 2
evaluating high-dose LTI-03 (5 mg BID) in mid-September; topline
data expected in the near-term
AUSTIN,
Texas, Oct. 12, 2024 /PRNewswire/ -- Aileron
Therapeutics, Inc. ("Aileron") (NASDAQ: ALRN), a biopharmaceutical
company advancing a novel pipeline of first-in-class medicines to
address significant unmet medical needs in orphan pulmonary and
fibrosis indications, today announced the presentation of two
abstracts detailing LTI-03's pre-clinical and Phase 1b (NCT05954988) results in Idiopathic Pulmonary
Fibrosis (IPF) at the 22nd International Colloquium on
Lung and Airway Fibrosis (ICLAF).
The Company previously announced positive
data from Cohort 1 of the ongoing Phase 1b clinical trial evaluating low-dose LTI-03 (2.5
mg BID) in patients with IPF. Following inhaled administration of
low-dose LTI-03 in 12 patients over the course of 14 days, a
positive trend was observed in seven out of eight biomarkers with
evidence of reduced expression among multiple profibrotic proteins
produced by basal-like cells and fibroblasts that contribute to the
progression of IPF, including data from three biomarkers (collagen
synthesis, inflammation, and fibrogenesis) that was statistically
significant, reinforcing the potential of LTI-03 to improve lung
function and reverse the course of IPF. The [poster][abstracts]
being presented at ICLAF will summarize the previously disclosed
data from Cohort 1.
Pre-clinical data presented at ICLAF further supports the
potential therapeutic effectiveness of LTI-03 for IPF through
precision cut lung slices (PCLS) performed ex-vivo.
Pre-clinical studies demonstrated molecular activity in IPF PCLS
explants indicative of fibrosis during five days in culture and
LTI-03 broadly attenuated pro-fibrotic proteins and pathways.
Additionally, the Company recently announced completion of
enrollment in Cohort 2 of the ongoing Phase 1b clinical trial evaluating high-dose LTI-03 (5
mg BID) in 12 patients with IPF. In the trial, eligible
patients (n=24) are randomly assigned (3:1) to receive either
inhaled LTI-03 or placebo. The primary objective of the trial is to
evaluate the safety and tolerability of LTI-03 in patients with IPF
after treatment for 14 consecutive days, with measurement of
multiple protein biomarkers as exploratory endpoints. The Company
expects to report topline data for this cohort in the
near-term.
Details of the [poster] presentations are as follows:
Presentation Title: Anti-Fibrotic Activity of Caveolin-1
scaffolding domain Peptide LTI-03 in Ex Vivo Precision Cut
Lung Slices from Patients with Idiopathic Pulmonary Fibrosis
Abstract #: 0186
Presenter: Professor Cory M.
Hogaboam, Cedars Sinai Medical Center, Los Angeles, CA
Date & Time: Tuesday, October 15,
2024 at 3:30 pm EEST/
8:30 am ET
Presentation Title: Inhalation of LTI-03 Modulates
Multiple Targets in a Phase 1B
Placebo Controlled Clinical Trial for IPF
Abstract #: 0183
Presenter: Professor Cory M.
Hogaboam, Cedars Sinai Medical Center, Los Angeles, CA
Date & Time: Tuesday, October 15,
2024 at 3:30 pm EEST/
8:30 am ET
About the Phase 1 Clinical Trial of LTI-03
The Phase 1b clinical trial of
LTI-03 is a randomized, double-blind, placebo controlled,
multi-center, dose escalation trial in patients recently diagnosed
with IPF that have not received prior treatment with anti-fibrotic
agents for at least two months (NCT05954988). Eligible
patients are randomly assigned (3:1) to receive one of two doses of
inhaled LTI-03 or placebo. The primary objective of the trial is to
investigate the safety and tolerability of LTI-03 in patients with
IPF after treatment for 14 consecutive days, with measurement of
multiple protein biomarkers as exploratory endpoints.
About IPF
IPF is a chronic lung disease characterized by progressive
tissue scarring that prevents proper lung function. It is a
progressive, fatal, age-associated lung disease affecting
approximately 100,000 people in the United States1. IPF typically
presents in adults 65 or older and is usually fatal within two to
five years after diagnosis2.
About LTI-03 and Caveolin-1 (Cav1)
LTI-03 is a seven amino acid peptide, the sequence of which is
derived from the caveolin scaffolding domain (CSD), an important
binding region of the Cav1 protein. Cav1 normally serves a critical
function in the prevention of fibrosis by maintaining a balance
between pathways that both initiate and arrest lung repair and cell
movement. Through the CSD, caveolin interacts with a large number
of signaling molecules, all of which possess a caveolin binding
domain region. Cav1 expression is decreased in IPF lung tissues and
has been demonstrated to decrease during the fibrotic phase of
bleomycin lung injury in mice. Restoring the balance of important
biological signals in the lung may not only slow lung function
decline but could also restore healthy lung function through the
protection of healthy epithelial cells.
About Aileron Therapeutics
Aileron Therapeutics is a biopharmaceutical company
advancing a novel pipeline of first-in-class medicines to address
significant unmet medical needs in orphan pulmonary and fibrosis
indications. Aileron's lead product candidate, LTI-03, is a novel,
synthetic peptide with a dual mechanism targeting alveolar
epithelial cell survival as well as inhibition of profibrotic
signaling. Currently, LTI-03 is being evaluated in a Phase
1b clinical trial for the treatment
of idiopathic pulmonary fibrosis. Aileron's second product
candidate, LTI-01, is a proenzyme that has completed Phase
1b and Phase 2a clinical trials for
the treatment of loculated pleural effusions. LTI-01 has received
Orphan Drug Designation in the US and EU and Fast Track Designation
in the US.
References
1 Pergolizzi, Jr., J., LeQuang, J., Varrassi,
M., Breve, F., Magnusson, P., Varrassi, G., (2023). What Do We Need
to Know About Rising Rates of Idiopathic Pulmonary Fibrosis? A
Narrative Review and Update. Springer Nature, Published online 2023
Jan 24. Doi: 10.1007/s12325-022-02395-9.
2 Nathan et al. "Long-term Course and Prognosis of
Idiopathic Pulmonary Fibrosis in the New Millennium". Chest
Journal Volume 140, ISSUE 1, P221-229, July 2011.
Forward-Looking Statements
This press release may contain forward-looking statements
of Aileron Therapeutics, Inc. ("Aileron", the "Company",
"we", "our" or "us") within the meaning of the Private Securities
Litigation Reform Act of 1995, including statements with respect
to: the timing and expectation of the topline results of Cohort 2
of the Phase 1b clinical trial of
LTI-03; future expectations, plans and prospects for the Company,
the sufficiency of the Company's cash resources; the status and
plans for clinical trials, including the timing of data; future
product development; and the potential commercial opportunity of
LTI-03 and LTI-01. We use words such as "anticipate," "believe,"
"estimate," "expect," "hope," "intend," "may," "plan," "predict,"
"project," "target," "potential," "would," "can," "could,"
"should," "continue," and other words and terms of similar meaning
to help identify forward-looking statements, although not all
forward-looking statements contain these identifying words. Actual
results may differ materially from those indicated by such
forward-looking statements as a result of various important
factors, including risks and uncertainties related to, changes in
applicable laws or regulations, the possibility that the Company
may be adversely affected by other economic, business, and/or
competitive factors, including risks inherent in pharmaceutical
research and development, such as: adverse results in the Company's
drug discovery, preclinical and clinical development activities,
the risk that the results of preclinical studies and early clinical
trials may not be replicated in later clinical trials or that
partial results of a trial such as the Cohort 1 results from the
Company's ongoing Phase 1b clinical
trial will be indicative of the full results of the trial, the
Company's ability to enroll patients in its clinical trials, and
the risk that any of its clinical trials may not commence, continue
or be completed on time, or at all; decisions made by the U.S.
Food and Drug Administration and other regulatory authorities,
investigational review boards at clinical trial sites and
publication review bodies with respect to our development
candidates; our ability to obtain, maintain and enforce
intellectual property rights for our platform and development
candidates; competition; uncertainties as to the sufficiency of the
Company's cash resources to fund its planned activities for the
periods anticipated and the Company's ability to manage unplanned
cash requirements; and general economic and market conditions; as
well as the risks and uncertainties discussed in the "Risk Factors"
section of the Company's Annual Report on Form 10-K for the year
ended December 31, 2023 and Quarterly Report on Form 10-Q
for the quarter ended June 30, 2024, which are on file with
the United States Securities and Exchange Commission (the
"SEC"), and in subsequent filings that the Company makes with
the SEC. These forward-looking statements should not be relied
upon as representing the Company's view as of any date subsequent
to the date of this press release, and we expressly disclaim any
obligation to update any forward-looking statements, whether as a
result of new information, future events or otherwise, except as
required by law.
Investor Relations & Media Contact:
Argot
Partners
aileron@argotpartners.com
212-600-1902
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