Pulmatrix Presents Top Line Clinical Data Showing Inhaled Drug Candidate, PUR118, Demonstrated Anti-Inflammatory Efficacy In Phase 1b Trial with COPD Patients
Clinical Results from Lead Product Candidate Created from Pulmatrix's Proprietary iSPERSE™ Inhaled Therapeutics Platform
Three Presentations at European Respiratory Society Highlight Data Related to PUR118
Lexington, MA – September 4, 2012 – Pulmatrix, Inc., a clinical stage biotechnology company creating
novel inhaled therapeutics, today announced top line results from a clinical study showing that the
novel inhaled therapeutic, PUR118, demonstrated anti-inflammatory efficacy and was well-tolerated in
two Phase 1b studies in patients with chronic obstructive pulmonary disease (COPD). These results,
that showed PUR118's beneficial impact on biomarkers of airway inflammation and mucus clearance,
are the first clinical efficacy results from PUR118 which is the company's lead iCALM™ inhaled
therapeutic candidate created from the iSPERSE™ dry powder delivery platform. Design aspects of
these clinical trials, along with supporting and related preclinical data, were presented at the European
Respiratory Society (ERS) Annual Congress on Monday, September 3, 2012, in Vienna, Austria.
"These clinical data clearly validate the potential for PUR118 to control COPD patients' symptoms and
significantly improve their lung function, while also showing PUR118 to be well tolerated," said John
Hanrahan, MD, MPH, Chief Medical Officer and Senior Vice President at Pulmatrix. "Based on the
strength of these initial efficacy signals, we intend to now move forward into a Phase 2 trial of PUR118
in COPD patients, accelerating this clinical program with a goal of more rapidly bringing this unique
treatment approach to patients with inflammatory airway conditions and the physicians who care for
them."
The presentations at ERS highlight PUR118's potential to offer a new inhaled approach for control of
airway inflammation in COPD and other respiratory conditions. PUR118 clinical and preclinical data
highlights include:
• A Phase 1b clinical trial that showed PUR118 was well tolerated in both healthy volunteers and
patients with COPD;
• Exploratory endpoints in the Phase 1b clinical study that assess biomarkers of inflammation and
mucus clearance support a favorable impact of PUR118 treatment on both airway inflammation
and mucociliary airway clearance velocity;
• Phase 1b clinical data demonstrated reduction in the levels of the same critical airway
inflammatory signaling molecules/biomarkers evident in preclinical models, and confirmed
preclinical data on the ability of PUR118 and iCALM therapies to reduce airway neutrophilic
inflammation, as reported in previous studies and those presented at ERS;• Preclinical data from PUR118 and related iCALM studies presented at ERS demonstrated robust
and consistent airway anti-inflammatory impact in animal models of a number of chronic
diseases. Highlights from these preclinical studies included:
• PUR118 reduced tobacco smoke induced chemokine and cytokine protein levels
associated with neutrophilic inflammation in mouse BAL samples, suggesting that its
anti-inflammatory efficacy is comparable to other clinical stage targets (p38 / PDE4)
with demonstrated clinical efficacy;
• PUR118 reduces TLR-induced chemokine and cytokine secretion and gene expression in
macrophages, suggesting that the in vivo activity of PUR118 and other iCALM
therapeutics is at least partially mediated through activity on macrophages.
• PUR118 was clinically evaluated in an innovative Phase 1 clinical study, and the design and
implementation of the Phase 1 study were jointly presented by Pulmatrix and Quotient Clinical
in a poster at ERS.
About the Phase 1 Trial Design for PUR118
The clinical development program for PUR118 comprises a Phase 1 trial structured into a four-part,
dose-ranging clinical study protocol, first in healthy normal volunteers and then patients with COPD.
Part 1 of this clinical study was a randomized, placebo controlled, single ascending dose, 4-way
crossover tolerability/safety study in 12 healthy male and female subjects aged 18 to 65. Part 2 was a
randomized, double-blind (2 active, 1 placebo), placebo-controlled, multiple-dose group, 14 day dose
escalation design in 24 healthy male and female subjects age 18 to 65. Part 3 was an open-label,
parallel group design in 36 male and female subjects aged 45 to 70 years, with mild (GOLD stage 1-2),
stable COPD. Part 4 was an open-label, single dose, 4 way crossover design in male and female exsmoking subjects age 45-70, with mild (GOLD stage 0-2), stable COPD. In each part of this four-part
Phase 1 trial, tolerability and safety of PUR118 was assessed at each dose level. Additionally, the effect
of PUR118 on mucociliary airway clearance velocity over the 2 hours after dosing was evaluated along
with exploratory and relevant biomarkers for efficacy.
About PUR118
PUR118 is the lead iCALM product candidate from Pulmatrix's proprietary iSPERSE dry powder delivery
platform. PUR118's novel mechanism of action as an iCALM therapeutic is designed to harness the
body's natural defenses to prevent and treat chronic respiratory diseases and respiratory infections in
diseases such as chronic obstructive pulmonary disease (COPD) and cystic fibrosis. Clinical and
preclinical data with PUR118 have demonstrated the ability to treat and prevent disease, in both viral
and bacterial diseases, as well as reduced inflammation in the lung. PUR118 is in human clinical
efficacy studies for COPD and cystic fibrosis.
About Pulmatrix
Pulmatrix, Inc. is a clinical stage biotechnology company developing and commercializing a novel
inhaled dry powder drug platform to create a new generation of inhaled therapeutics. The platform,
called iSPERSE™ (inhaled small particles easily respirable and emitted), enables drugs to be delivered in inhaled dry powders with unique properties for high drug loading and highly efficient dispersibility and
delivery to the airways. iSPERSE can create dry powder formulations with virtually any drug substance,
including small molecules, biologics and multi-drug combinations, as well as the company's proprietary
iCALM™ (inhaled Cationic Airway Lining Modulators) inhaled therapies. The Company is pursuing both
proprietary and partnered applications for iCALM and iSPERSE. For additional information about
Pulmatrix, please visit www.pulmatrix.com.
iCALM™ and iSPERSE™ are trademarks of Pulmatrix, Inc.
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For more information, please contact:
Kathryn Morris
The Yates Network
[email protected]
+1-845-635-9828