US Biologic Lung Volume Reduction (BLVR) Phase 2 Emphysema Study

Phase 2 Study of the 20 mL Biologic Lung Volume Reduction System (BLVR) in Patients With Advanced Upper Lobe Predominant Emphysema

The purpose of this study it to evaluate the efficacy and safety of the 20 mL BLVR System in patients with advanced upper lobe predominant emphysema.

Background:

Patients with emphysema currently have limited treatment choices. Many patients are treated with steroids and inhaled medications, which often provide little or no benefit. In recent years, lung volume reduction surgery has become an accepted therapy for advanced emphysema. Lung volume reduction surgery involves the removal of diseased portions of the lung in order to enable the remaining, healthier portions of the lung to function better. This procedure, although effective for many patients, is complicated and is accompanied by substantial morbidity and mortality risk.

Aeris Therapeutics has developed the Biologic Lung Volume Reduction (BLVR) System which is intended to achieve lung volume reduction without surgery and its attendant risks. Patients are treated using a bronchoscope to direct treatment to the most damaged areas of the lung. The treatment delivers a precisely proportioned proprietary mixture of drugs and biologics which, when combined at the treatment site, form a biodegradable hydrogel. The hydrogel acts to reduce lung volume by permanently collapsing and sealing the diseased areas of the lung. This provides room within the chest to allow the remaining healthier portions of the lung to function better.

Aeris' BLVR development program has been granted Fast Track designation by the U.S. FDA, and is the subject of ongoing clinical trials designed to investigate the safety and efficacy of the BLVR System as a treatment for patients with advanced heterogeneous emphysema. Fast Track designation is reserved for drug and biologic development programs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.

Inclusion Criteria:

• clinical diagnosis of advanced upper lobe emphysema
• age >/= 40 years
• clinically significant dyspnea
• failure of standard medical therapy to relieve symptoms (inhaled beta agonist & inhaled anticholinergic)
• pulmonary function tests within protocol-specified ranges (post bronchodilator FEV1 < 45% predicted & experiencing < 30% or 300 mL improvement using bronchodilator; total lung capacity > 110% predicted; residual volume > 150% predicted)
• 6 Minute Walk Distance >/= 150 m

Exclusion Criteria:

• alpha-1 protease inhibitor deficiency
• homogeneous emphysema
• tobacco use within 4 months of initial visit
• body mass index < 15 kg/m2 or> 35 kg/m2
• clinically significant asthma, chronic bronchitis or bronchiectasis
• allergy or sensitivity to procedural components
• pregnant, lactating or unwilling to use birth control if required
• prior lung volume reduction surgery, lobectomy, pneumonectomy, lung transplant, endotracheal valve placement, airway stent placement or pleurodesis
• comorbid condition that could adversely influence outcomes
• inability to tolerate bronchoscopy under conscious sedation (or anesthesia)
• history of renal infarction or renal failure