Cohort 2 will evaluate high-dose LTI-03 (5 mg
BID)
Previously announced Cohort 1 data
demonstrated positive trends in seven of the eight biomarkers
evaluated, suggesting a potential therapeutic effect
AUSTIN,
Texas, Sept. 23, 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 completion of enrollment
in Cohort 2 of the ongoing Phase 1b
clinical trial of LTI-03 in IPF patients.
The last patient was enrolled in mid-September, with a total of
24 patients enrolled in the trial. Eligible patients 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.
"We are pleased to have completed enrollment in the high-dose
cohort of our Phase 1b clinical trial
and grateful to all participating patients and investigators," said
Brian Windsor, Ph.D., President and
Chief Executive Officer of Aileron. "Following the positive trend
observed in the low-dose cohort, we are excited to assess the
safety, tolerability and biomarkers of 5 mg twice daily of inhaled
LTI-03 and expect to report topline data in the near term."
In May 2024, the Company announced
positive data from Cohort 1 evaluating low-dose LTI-03 (2.5 mg
BID). Following inhaled administration of low-dose LTI-03 in 12
patients, 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 that was statistically significant, reinforcing
the potential of LTI-03 to improve lung function and reverse the
course of IPF.
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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SOURCE Aileron Therapeutics, Inc.
