AEROSURF^® (Aerosolized Drug-Device Combination Product for RDS)

Currently marketed respiratory surfactants are available only as liquid instillate formulations and their therapeutic use is limited to infants who are intubated and undergoing mechanical ventilation for the treatment of RDS.

AEROSURF^® (aerosolized KL4 Surfactant Drug-Device product) represents the first potential opportunity to deliver a clinically-relevant dose of surfactant to RDS patients in aerosol form, without the need for either intubation or mechanical ventilation. By doing so, AEROSURF^® holds the promise to significantly expand the use of surfactant in pediatric respiratory medicine.

Practicing neonatologists uniformly recognize the perceived risks associated with intubation and subsequent mechanical ventilation, including the risk of trauma and increased risk of infection. As a result, many neonatologists will only intubate in cases of severe respiratory disease, where the benefits of invasive surfactant administration clearly outweigh the associated risks.

For all but very low birth weight infants with severe RDS, a common, less invasive ventilatory support treatment alternative to intubation and mechanical ventilation is nasal continuous positive airway pressure (nCPAP). Unfortunately, a significant number of infants suffering from RDS do not adequately respond to nCPAP alone and require subsequent surfactant administration via intubation and mechanical ventilation. As neonatologists cannot ascertain in advance which patients will fail nCPAP, they are faced with a dilemma because the outcome for those infants who fail nCPAP and receive delayed surfactant therapy is compromised as compared with children who receive surfactant therapy in the first hours of life.

AEROSURF^® potentially eliminates this dilemma by allowing for the administration of a clinically relevant dose of aerosolized KL4 Surfactant using a preferred ventilator support modality: nCPAP. We believe that AEROSURF^®, delivering aerosolized KL4 Surfactant via nCPAP, represents a significant market opportunity and has the potential to significantly expand the use of surfactants worldwide to eventually result in a global market opportunity that is greater than $750 million.

AEROSURF^® Clinical Experience and CAG Technology

Discovery Labs has completed a Phase 2 feasibility study of aerosolized KL4 Surfactant and has met with and received guidance from the FDA with respect to further development for RDS.

Our CPAP-01 trial was designed to evaluate the safety and tolerability of aerosolized KL4 Surfactant in premature infants at risk for RDS. Aerosolized KL4 Surfactant was administered to infants within 30 minutes of birth for three hours. Based on clinical response, three additional treatments were permitted within 48 hours of life. A vibrating membrane aerosol generator [Aeroneb^® Pro, Artemis Med. UK] was used to aerosolize the KL4 Surfactant. Key results from this program can be summarized as follows:

• Aerosolized KL4 Surfactant was generally safe and well tolerated. ⁷¹
• All infants treated with aerosolized KL4 Surfactant survived through the evaluation period. ⁷¹
• Additional complications of prematurity were within expected limits at the conclusion of the study. ⁷¹
• Aerosolized KL4 Surfactant retained pharmacological activity throughout the aerosolization process. ⁷⁰

Although this trial demonstrated technical feasibility, Discovery Labs is optimizing the aerosolized KL4 Surfactant delivery approach for RDS through our proprietary CAG Technology and patient interface system.

AEROSURF^® is a drug-device product that consists of the entire technology system for the delivery of aerosolized surfactant to premature infants for RDS: KL4 Surfactant, Capillary Aerosol Generator, and a proprietary nCPAP compatible patient interface.

AEROSURF^® is an investigational drug-device product not approved by the US Food & Drug Administration. Several published and presented pre-clinical and clinical studies are referenced throughout this website; however, approved and final prescribing information from the US Food & Drug Administration for Aerosurf is not available at this time.