Positive results from Elevation Pharmaceuticals’ EP-101 Phase 2b study on COPD

Elevation Pharmaceuticals, Inc. today presented positive efficacy and safety results from the GOLDEN-1 study – a Phase 2b study evaluating EP-101 in patients with moderate-to-severe chronic obstructive pulmonary disease (COPD) – at the annual meeting of the European Respiratory Society in Vienna, Austria.  As previously announced, EP-101 demonstrated a rapid onset, dose-related, statistically significant improvement in lung function compared to placebo.  EP-101 is a proprietary inhalation solution formulation of glycopyrrolate, a long-acting muscarinic antagonist (LAMA), delivered by an investigational eFlow® nebulizer (PARI Pharma GmbH). 

The GOLDEN-1 study (Glycopyrrolate for Obstructive Lung Disease via Electronic Nebulizer), was a Phase 2b, randomized, placebo-controlled, double-blind, seven-arm, four-period cross-over, incomplete block design, dose-ranging study with seven days of once-daily dosing to assess the pharmacokinetics, safety and efficacy of EP-101 in 140 patients with moderate-to-severe COPD.  All four doses (25mcg, 50 mcg, 100 mcg and 200 mcg) of EP-101 administered as once daily for seven days using the investigational eFlow provided a rapid onset of action (five minutes), short treatment time (two minutes), and a robust magnitude of bronchodilation compared to placebo.  The bronchodilatory improvements on day seven as assessed by 24-hour forced expiratory volume in one second (FEV1)  and the FEV1 AUC (0-12 hours and 12-24 hours) were dose-dependent, statistically significant (p<0.001) at all doses compared to placebo, and clinically meaningful at several doses compared to placebo.  In addition, the improvements in lung function with EP-101 doses were comparable to those of once-daily tiotropium dry powder inhaler and three- times daily nebulized ipratropium administered via jet nebulizer.  EP-101 was well tolerated at all doses, and the incidence of adverse events was generally similar to placebo and to ipratropium but tended to be higher than treatment with tiotropium.  The most commonly reported adverse events were cough and headache with no dose relationship for the incidence of adverse events.  One death was reported in the study in a patient treated with EP-101, but this death was considered to be unrelated to study treatment.

"The positive outcome of this large Phase 2b study is an important milestone towards our goal of significantly elevating the standard of care for COPD patients who require nebulizer delivery of their medicines," said Dr. Ahmet Tutuncu, Senior Vice President and Chief Medical Officer of Elevation Pharmaceuticals. 

LAMAs are preferred for patients with more severe COPD, and EP-101 is believed to be the only nebulized LAMA in clinical development.  EP-101 is being developed in combination with an Investigational eFlow device – a portable nebulizer providing targeted lung delivery and short treatment times – to significantly improve the standard of care for the large underserved subpopulation of COPD patients not adequately treated by dry powder or metered dose inhalers.

A second Phase 2b study will be initiated in Q4 2012 to select the dose for Phase 3 studies, which are expected to start in the second half of 2013.