Avalyn Presents Clinical Data on AP01 for Pulmonary Fibrosis, Including Long-Term Efficacy and Imaging Biomarker Data, at ATS 2024
20 Mayo 2024 - 3:16PM
Avalyn Pharma Inc. (Avalyn), a clinical-stage biopharmaceutical
company focused on development of inhaled therapies for treatment
of life-threatening pulmonary diseases, today presented clinical
data for AP01, its novel formulation of inhaled pirfenidone, for
the treatment of pulmonary fibrosis, supporting the Company’s
planned Phase 2b trial in patients with progressive pulmonary
fibrosis (PPF). Analysis from Avalyn’s ongoing ATLAS open-label
extension study demonstrated that treatment with AP01 stabilized
forced vital capacity (FVC) in patients with both idiopathic
pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF),
with enhanced safety and tolerability compared to oral pirfenidone.
Furthermore, findings from post-hoc analyses examining disease
progression and Brainomix’s e-Lung biomarker scores support the
Company’s selected dosing of 100 mg twice-daily and 50 mg
twice-daily in the planned Phase 2b study.
The data were presented in multiple poster presentations and
featured events during the 2024 American Thoracic Society (ATS)
International Conference held May 17-22, 2024, in San Diego,
CA.
“Having dedicated decades to treating patients with respiratory
diseases, I’ve seen firsthand the pressing need for new treatments.
There is a significant need for therapies that not only slow
disease progression but are also tolerable for long-term use,” said
Howard M. Lazarus, M.D., FCCP, Chief Medical Officer at Avalyn.
“Pulmonary fibrosis is a devastating, and often fatal, disease
despite currently available oral therapies. Our excitement stems
from the long-term data with AP01 that shows treatment of patients
out to nearly four years, surpassing the average survival rates for
patients with progressive disease. We’ve generated a robust dataset
with AP01 that shows substantially better efficacy and safety
compared to today’s orally available therapies, which enables
sustained treatment and ultimately, the potential for improved
outcomes.”
Poster Title: Safety and Efficacy of
Inhaled Pirfenidone (AP01) in the ATLAS Open-label Extension
Study (Thematic Poster Session: B48, Poster #: 1242)
Avalyn’s open-label extension study enrolled a total of 100
patients across three cohorts: 41 patients with IPF who rolled over
from the company’s ATLAS Phase 1b clinical trial and had been
previously treated with AP01; 31 AP01-naïve patients with IPF; and
28 AP01-naïve patients with progressive pulmonary fibrosis (PPF).
All patients received 100 mg AP01 twice-daily in the open-label
extension. Data presented show that both IPF and PPF patients
treated with AP01 experienced a clinically meaningfully lower
annualized change in FVC than with oral pirfenidone as reported in
prior pivotal studies.
Data from the cohort of roll-over IPF patients who were
previously treated with AP01 were out to 180 weeks and showed an
annualized change in FVC of -107 ml/year. These findings were
substantially lower than the -235 ml/year previously reported with
oral pirfenidone across three pivotal trials. Further, data from
the cohorts of AP01- naïve IPF and PPF patients were out to 84
weeks and showed an annualized change in FVC of -134 ml/year and
-13 ml/year, respectively.
A total of 47 patients across all three cohorts remain in the
study and continue to receive AP01 treatment as of today, with some
patients on therapy out to 252 weeks.
AP01 has continued to be generally well-tolerated, enabling
long-term treatment. Patients treated with AP01 have reported
markedly lower rates of nausea, rash, diarrhea, fatigue, dyspepsia
and vomiting compared to the rate of side effects reported with
oral pirfenidone.
Poster Title: Inhaled Pirfenidone
Reduces the Risk of Disease Progression: A Post-hoc Analysis of the
ATLAS Study (Thematic Poster Session: B48, Poster #:
1244)
Avalyn conducted a post-hoc analysis of disease progression in
the ATLAS Phase 1b study in patients with IPF, which demonstrated
that AP01 reduced the risk of disease progression, as defined by
decline of greater than 10% FVC in patients with IPF. Fewer IPF
patients in the AP01 100 mg twice-daily dosing arm (21%)
experienced greater than 10% FVC decline by 48 weeks compared to
the 50 mg once-daily dosing arm (35%). A similar, favorable trend
of the AP01 100 mg twice-daily cohort was observed leading up to 48
weeks, at 12- and 24- weeks. This was consistent with the numerical
advantages that were observed when comparing AP01 100 mg
twice-daily to published results for placebo from pivotal studies.
These data support Avalyn’s selection of AP01 100 mg twice-daily
and 50 mg twice-daily for the Company’s planned Phase 2b clinical
trial.
Poster Title: Differences in
e-Lung Biomarker Scores Between Treatment Groups in Post-hoc
Analysis of the ATLAS Inhaled Pirfenidone Solution (AP01) for IPF
Clinical Trial (Thematic Poster Session: B48, Poster #:
1251)
Avalyn, together with Brainomix, presented promising data on
e-Lung imaging biomarkers in a post-hoc analysis of the ATLAS Phase
1b study in patients with IPF. The imaging analysis utilized
Brainomix’s proprietary weighted reticulovascular score (WRVS), an
AI-enabled computed tomography (CT) processing software. Findings
showed that WRVS strongly and substantially correlated with the
risk of future IPF progression. AP01’s 100 mg twice-daily dosing
arm was associated with stabilization of IPF with trends to a mean
relative decrease in the WRVS and mean relative increase in FVC.
These data suggest that e-Lung WRVS may be able to identify the
risk of disease progression and identify treatment effects on CT,
even when FVC is stable.
About Avalyn PharmaAvalyn is a
biopharmaceutical company developing inhaled therapies for the
treatment of rare respiratory diseases including pulmonary fibrosis
and other interstitial lung diseases (ILD). Pulmonary fibrosis is
characterized by scarring, decline in lung function, reduced
exercise capacity and quality of life, and is associated with
increased mortality. Currently approved therapeutic options slow
pulmonary fibrosis progression but are associated with significant
toxicities that restrict their use and dosing. Avalyn is developing
a pipeline of new inhaled formulations of approved medicines
designed to reduce systemic exposure and deliver medication to the
site of disease. Avalyn’s lead pipeline program, AP01, an optimized
inhaled formulation of pirfenidone, which has been assessed in 150
individuals with different forms of pulmonary fibrosis and
demonstrated clinical proof-of-concept with improved efficacy and
safety over existing therapies. For more information, please visit
avalynpharma.com, and follow us on social media: LinkedIn.
Investor Contact:Monique AllaireTHRUST
Strategic Communicationsmonique@thrustsc.com