Pearl Therapeutics Inc. announced that their lead combination bronchodilator, PT003, met the primary efficacy endpoint in a recently completed Phase 2b clinical trial in patients with moderate to very severe chronic obstructive pulmonary disease (COPD). These top-line results show that PT003 provides superior bronchodilation compared to the current market leader, tiotropium bromide (Spiriva® Handihaler®), as well as to formoterol fumarate (Foradil® Aerolizer®), placebo and the individual components of PT003 (p less than or equal to 0.0002 for all comparisons). In addition PT003 was shown to be safe and well tolerated.

PT003 is an inhaled combination bronchodilator product comprised of glycopyrrolate, a long-acting muscarinic antagonist (LAMA), and formoterol, a long-acting beta-2-agonist (LABA), delivered via a hydrofluoroalkane metered dose inhaler (MDI). In this Phase 2b study, two doses of PT003 were compared to Spiriva, Foradil, placebo, glycopyrrolate MDI (PT001) and formoterol MDI (PT005). The primary assessment for this study was change in lung function following one week of dosing, as assessed by FEV1 AUC (0-12) (forced expiratory volume in one second)*, relative to baseline at the start of treatment. Both doses of PT003 were superior to Spiriva (p The Phase 2b dose-ranging study compared twice-daily (BID) dosing of PT003 against its monotherapy components, PT001 and PT005, as well as Spiriva, Foradil and placebo in 118 patients with moderate to very severe COPD. Placebo, PT003, PT001 and PT005 were administered BID for one week while Spiriva and Foradil were administered according to their approved label: 18 ug (via Handihaler® inhaler) once daily, and 12 ug (via Aerolizer® inhaler) BID, respectively, each for one week.

About PT003

PT003 is an inhaled combination bronchodilator product comprised of glycopyrrolate (PT001), a long-acting muscarinic antagonist (LAMA), and formoterol (PT005), an established, long-acting beta-2-agonist (LABA), delivered via a hydrofluoroalkane metered dose inhaler (HFA-MDI). Pearl's proprietary porous particle technology allows the formulation of both formoterol and glycopyrrolate in the MDI format, with highly stable, robust and aerodynamically efficient drug delivery. PT003 is the first and only dual long-acting rapid bronchodilator LAMA-LABA combination product in development in an HFA-MDI formulation, the most widely used inhalation drug delivery format.

About COPD

Chronic obstructive pulmonary disease (COPD) is a preventable and treatable disease with significant extrapulmonary effects that may contribute to the severity in individual patients. Its pulmonary component is characterized by airflow limitation that is not fully reversible and results in symptoms such as shortness of breath that worsens with exercise and chronic cough. The airflow limitation is usually progressive and associated with an abnormal inflammatory response of the lung to noxious particles or gases. While other major causes of death have been decreasing, COPD mortality has continued to rise and is now the fourth leading cause of death in the United States. Each year 12 million Americans are diagnosed with COPD, and research shows that many do not get optimal treatment. An additional 12 million Americans may have COPD and remain undiagnosed. Worldwide, cigarette smoking is the most common risk factor for COPD, and smoking cessation is the only intervention that has been shown to modify the course of the disease.

FEV1 (forced expiratory volumes in one second) is a common measurement of lung function in patients with asthma, cystic fibrosis, and COPD and is typically used to predict the severity of pulmonary disease. AUC (area under the curve) is a measure of therapeutic benefit over a period of time.

Spiriva® HandiHaler® (tiotropium bromide inhalation powder) is a registered trademark of Boehringer Ingelheim Pharmaceuticals; Foradil® is a registered trademark of Astellas Pharma; and Aerolizer® is a registered trademark of Novartis AG.