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Discovery Labs Announces Completion of Enrollment in AEROSURF® Phase 2a Dose Expansion Trial

Top-line Results Expected to be Released in Mid-November

Company Expects to Initiate AEROSURF Phase 2b Clinical Trial in Fourth Quarter of 2015

Oct 21, 2015

WARRINGTON, Pa., Oct. 21, 2015 /PRNewswire/ -- Discovery Laboratories, Inc. (NASDAQ: DSCO), a specialty biotechnology company focused on developing aerosolized KL4 surfactant therapies for respiratory diseases, today announced that it has completed enrollment in the AEROSURF® phase 2a dose expansion clinical trial to assess the administration of higher doses of AEROSURF in premature infants 29 to 34 week gestational age (GA) with respiratory distress syndrome (RDS). With the completion of enrollment in this trial, the Company expects to initiate its AEROSURF phase 2b trial in the fourth quarter of 2015 beginning with premature infants 29 to 32 weeks GA.

The AEROSURF phase 2a dose expansion trial is designed to expand upon the knowledge gained in the initial phase 2a clinical trial, which assessed the safety and tolerability of a single exposure of three escalating (15, 30 and 45 minutes) inhaled doses of aerosolized KL4 surfactant to 48 premature infants 29 to 34 weeks GA receiving nasal continuous positive airway pressure (nCPAP) for RDS, compared to infants receiving nCPAP alone. All key objectives in the initial trial were met, including establishment of proof of concept based on physiological data suggesting that aerosolized KL4 surfactant is being delivered into the lung of premature infants. The dose expansion trial is a multicenter, randomized, open-label, controlled study in 32 premature infants, which is designed to evaluate the safety and tolerability of aerosolized KL4 surfactant administered in higher (60 and 90 minutes) doses in premature infants 29 to 34 weeks. Additionally, the Company continues to evaluate the physiological effects of aerosolized KL4 surfactant. The Company anticipates releasing top-line results of the dose expansion study and holding an investor conference call in mid-November.

"We are pleased to have achieved another milestone in our AEROSURF development program with the completion of enrollment in the phase 2a expansion study and look forward to the start of our AEROSURF phase 2b program." commented Steve Simonson, M.D., Discovery Labs' Chief Development Officer.

In the fourth quarter of 2015, the Company expects to initiate an AEROSURF phase 2b clinical trial in up to 250 premature infants 26 to 32 week GA. The study is designed to evaluate two escalating doses (with potentially repeat doses) of aerosolized KL4 surfactant with a primary purpose of demonstrating evidence of efficacy. If successful, this study will inform the final design of a phase 3 clinical program. Enrollment in the phase 2b trial will begin with premature infants 29 to 32 weeks GA and will then be followed by premature infants 26 to 28 weeks GA following completion of the phase 2a program in this age group. The phase 2b clinical trial is expected to be completed in mid – 2016.

The Company is currently enrolling a 32-patient phase 2a clinical trial to assess the safety and tolerability of escalating (30 and 45 minutes) and potentially repeat inhaled doses of aerosolized KL4 surfactant administered to premature infants 26 to 28 week GA. The Company anticipates releasing top-line results of this phase 2a trial in the first quarter of 2016.
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Source: press release, 10/21/15. http://discoverylabs.investorroom.com/2015-10-21-Discovery-Labs-Announce...

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Discovery Labs Announces Enrollment of First Patient in AEROSURF® Phase 2a Dose Expansion Trial

Building on results of the recently concluded phase 2a clinical trial, this phase will assess higher and repeat doses of aerosolized KL4 surfactant

May 20, 2015

WARRINGTON, Pa., May 20, 2015 /PRNewswire/ -- Discovery Laboratories, Inc. (NASDAQ: DSCO), a specialty biotechnology company focused on developing aerosolized KL4 surfactant therapies for respiratory diseases, today reported that the first patient has been enrolled in the next phase of its phase 2a clinical evaluation of AEROSURF®, which is designed to evaluate the safety and tolerability of higher and repeat doses of aerosolized KL4 surfactant administered to premature infants 29 to 34 weeks gestational age (GA) who are receiving nasal continuous positive airway pressure (nCPAP) for respiratory distress syndrome (RDS), compared to infants receiving nCPAP alone. This phase is intended to expand upon the knowledge gained in the recently completed phase 2a clinical trial, the results of which were reported in mid-May 2015. The Company expects to complete this study in the fourth quarter of 2015.

The recently completed phase 2a clinical trial was designed to assess the safety and tolerability of three escalating single doses of aerosolized KL4 surfactant in premature infants 29 to 34 weeks gestational age who are receiving nCPAP for RDS, compared to infants receiving nCPAP alone. All key objectives were achieved, including the primary objective of demonstrating a favorable safety and tolerability profile of AEROSURF, establishing proof of concept for the Company's proprietary technology platform based on physiological data suggesting that aerosolized KL4 surfactant is being delivered into the lung of premature infants, and acceptable performance of the novel capillary aerosol generator (CAG) technology in the neonatal intensive care unit (NICU).

This next phase 2a dose expansion study is a multicenter, randomized, open-label, controlled study and is designed to evaluate the safety and tolerability of aerosolized KL4 surfactant administered in higher and repeat doses in premature infants 29 to 34 weeks GA who are receiving nCPAP for RDS, compared to infants receiving nCPAP alone. In addition to safety and tolerability, another objective of the expansion study is to continue to evaluate the physiological effects of higher and repeat dosing of aerosolized KL4 surfactant. The results of this expansion trial are expected to provide further insight and data to support the design of the planned AEROSURF phase 2b clinical program.

"The initiation of the 2a dose expansion phase is an important next step to build upon the promising results observed in our initial phase 2a clinical trial. We believe that, with higher and/or repeat doses, AEROSURF may have the potential to reduce the need for invasive intubation due to nCPAP failure in this fragile patient population. Our objective for this phase is to gain additional knowledge that can be incorporated into the design of our phase 2b clinical trial," commented Steve Simonson, M.D., Discovery Labs' Chief Development Officer.

In addition, the Company plans the following phase 2 clinical assessments going forward: (1) A phase 2a clinical trial assessing the safety and tolerability of AEROSURF of escalating doses administered to 26 to 28 week gestational age infants, with an ability to administer repeat doses. The Company anticipates completing this trial in the fourth quarter of 2015; and (2) A phase 2b clinical trial to determine the optimal dose and define the expected efficacy margin of AEROSURF treatment. The Company anticipates completing this trial in mid 2016.
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Source: press release, 5/20/15. http://discoverylabs.investorroom.com/2015-05-20-Discovery-Labs-Announce...

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Encouraging safety and physiological data suggest that aerosolized KL4 surfactant can be delivered to the lung of premature infants with respiratory distress syndrome

Key study objectives achieved; Company advancing AEROSURF phase 2 clinical program

May 14, 2015

WARRINGTON, Pa., May 14, 2015 /PRNewswire/ -- Discovery Laboratories, Inc. (NASDAQ: DSCO), a specialty biotechnology company focused on developing aerosolized KL4 surfactant therapies for respiratory diseases, today reported top line data from its recently completed AEROSURF® phase 2a clinical trial in premature infants with respiratory distress syndrome (RDS). Key objectives of the study were achieved, including (1) the primary objective of demonstrating the safety and tolerability of a single exposure of aerosolized KL4 surfactant administered in three escalating inhaled doses to premature infants 29 to 34 week gestational age and receiving nasal continuous positive airway pressure (nCPAP) for RDS, compared to infants receiving nCPAP alone, and (2) establishing proof of concept for the Company's proprietary technology platform based on physiological data suggesting that aerosolized KL4 surfactant is being delivered into the lung of premature infants, and acceptable performance of the novel capillary aerosol generator (CAG) technology in the neonatal intensive care unit (NICU).

"The ability to administer surfactant without invasive endotracheal intubation in the management of premature infants with RDS would represent a significant medical advancement. We are encouraged by the results from this AEROSURF phase 2a clinical trial as it represents a positive indication that our technology has the potential to achieve this goal. While the trial was designed as a safety study and the number of patients was limited, the results suggest that the Company's combination drug/device product has the ability to deliver aerosolized KL4 surfactant to the lungs of premature infants with RDS," commented Steve Simonson, M.D., Discovery Labs' Chief Development Officer. "These results will inform and improve the further development of our AEROSURF clinical program."

AEROSURF is Discovery Labs' combination drug/device product that combines the Company's novel synthetic peptide-containing (KL4) surfactant and its proprietary aerosol delivery technologies, including the CAG, that are designed to enable efficient delivery of aerosolized KL4 surfactant. Currently, the available surfactants (animal-derived) can only be administered using endotracheal intubation with mechanical ventilation, invasive procedures that may each result in serious complications and other respiratory conditions. With AEROSURF, neonatologists potentially will be able to administer aerosolized KL4 surfactant to premature infants supported with nCPAP alone, without having to resort to invasive intubation and mechanical ventilation.

The AEROSURF phase 2a trial was a multi-center, open-label trial to evaluate safety and tolerability of a single dose of aerosolized KL4 surfactant in premature infants 29 to 34 weeks gestational age who are receiving nCPAP for RDS ("AEROSURF group"; n = 24), compared to infants receiving nCPAP alone ("control group"; n=24). The study evaluated three escalating doses of AEROSURF.

Discovery Labs' management will host a conference call and live webcast, with a slide presentation including data from the clinical trial, today at 8:00 a.m. Eastern time to review and discuss the results of the trial. See below for details of the call.

Safety and Tolerability

Overall, the safety and tolerability profile of the AEROSURF group in this initial trial was generally comparable to the control group. All reported adverse events and serious adverse events were those that are common and expected among this fragile patient population. The most common adverse events observed included neonatal jaundice, constipation and apnea. The most common serious adverse event observed was pneumothorax, the incidence of which was comparable in the AEROSURF and control groups. The incidence of adverse events and serious adverse events in the AEROSURF and control groups were generally comparable and there was no pattern observed of increasing adverse events or serious adverse events with increasing doses of AEROSURF.

Regarding tolerability of AEROSURF administration, the patient interface was well tolerated in that there were no observed upper airway obstructions. Peri-dosing events, which are common in the administration of surfactants currently, were infrequent in the AEROSURF group.

Based on the safety and tolerability profile observed in this clinical trial, the independent Safety Review Committee approved progressing to the next phases of the AEROSURF clinical development program.

Physiological Evaluation

In exploratory analyses of certain safety and tolerability measures to assess whether a single dose of aerosolized KL4 surfactant was being delivered to the lungs of premature infants and potentially having a physiological effect, measurements of gas exchange in the lungs and the timing of or need for endotracheal intubation and delayed (rescue) surfactant therapy due to nCPAP failure were evaluated in both the AEROSURF and control groups.

Gas Exchange

Gas exchange parameters were assessed as part of the safety and tolerability profile of AEROSURF. No safety signals were observed with respect to gas exchange. These parameters are physiological measurements that the Company also used to assess whether there is evidence of KL4 surfactant being delivered to the lungs of premature infants.

The fraction of inspired oxygen (FiO2) required by an infant is considered a key measurement of how well the lung is functioning to oxygenate the blood. Healthy lungs can achieve appropriate blood oxygen saturation breathing room air, which is 21% oxygen; however, to achieve the desired level of oxygen saturations, premature infants with RDS frequently require supplemental oxygen. Supplemental oxygen can be toxic, however, so the goal of clinicians is to decrease the amount of supplemental oxygen as quickly as possible.

To evaluate whether AEROSURF was positively impacting oxygenation as assessed by the FiO2 requirement, two measurements were assessed: (i) the need for supplemental oxygen at one hour after either starting the AEROSURF treatment or randomization to the control group (the "one-hour measurement") and (ii) the FiO2 change from baseline over three hours (the "three-hour measurement"). Results indicate that: (i) at the one-hour measurement, 36% of the AEROSURF group (8/22) were at room air as compared to 14% in the control group (3/22); and (ii) at the three-hour measurement, the AEROSURF group (n=21) had an absolute reduction in FiO2 of approximately 6.5% from the baseline average FiO2 of 32%, compared to an absolute reduction in FiO2 of approximately 1.5% from the baseline average FiO2 of 28% for the control group (n=21).

Carbon dioxide levels in the blood (PCO2) are considered a measure of respiratory function and how efficiently the lungs eliminate carbon dioxide (CO2) from the bloodstream. A known benefit of surfactant replacement therapy for premature infants with RDS is a reduction of CO2 levels in the bloodstream. To evaluate whether AEROSURF may be having a positive impact on CO2 levels, PCO2 was also assessed over three hours after the start of AEROSURF treatment or randomization to control group. In those patients in whom PCO2 was measured (the two higher dose groups and corresponding control patients), a 9 mm of mercury (Hg) decrease in CO2 from the baseline average of 48 mm Hg was observed in the AEROSURF group (n=12), compared to a 1.5 mm Hg decrease from a baseline average of 46 mm Hg for control patients (n=10). This effect is consistent with the action of a surfactant exerting its effect in the lung.

The data related to gas exchange appear to suggest that with AEROSURF, KL4 surfactant is being delivered to, the lungs of premature infants with RDS and potentially improving gas exchange.

Requirement for Invasive Rescue Therapy due to nCPAP failure

Premature infants with severe RDS currently are treated with surfactants that can only be administered by endotracheal intubation supported with mechanical ventilation, invasive procedures that may each result in serious respiratory conditions and other complications. To avoid such complications, many neonatologists treat infants with less severe RDS by less invasive means, typically nCPAP. Unfortunately, a significant number of premature infants on nCPAP will respond poorly (an outcome referred to as nCPAP failure) and may require delayed surfactant therapy (invasive rescue therapy). Since neonatologists currently cannot predict which infants will experience nCPAP failure, neonatologists are faced with difficult choices in treating infants with less severe RDS. This is because the medical outcomes for those infants who experience nCPAP failure and receive delayed surfactant therapy may be less favorable than the outcomes for infants who received surfactant therapy in the first hours of life.

Parameters associated with invasive rescue therapy were assessed as part of the safety and tolerability profile of AEROSURF. These parameters were also used to assess whether there is evidence of KL4 surfactant being delivered to the lungs of premature infants.

To evaluate whether a single dose of AEROSURF had an impact on the need for invasive rescue therapy (due to nCPAP failure), the time to nCPAP failure was assessed for both the AEROSURF (n=24) and control group (n=24). The results indicate that: (i) at three hours after the start of treatment or randomization to the control group, no AEROSURF patients required rescue therapy compared to 17% of control patients; (ii) at six hours after the start of treatment or randomization to the control group, 13% of patients in the AEROSURF group required rescue therapy compared to 21% in the control group; and, (iii) at 12 hours after the start of treatment or randomization to the control group, nCPAP failure rates in the AEROSURF and control groups were the same at 33%.

Overall, the nCPAP failure rates through 72 hours after the start of treatment or randomization to the control group were comparable between the AEROSURF and control groups. In assessing nCPAP failure rates, the results suggest that inclusion criteria of the phase 2a trial (particularly the FiO2 requirement of at least 25%) resulted in a population at higher risk for nCPAP failure. The overall nCPAP failure rate of the AEROSURF and control groups combined was approximately 58% and was higher than what might be expected in the 29 to 34 weeks gestational age patient population. However, in the AEROSURF groups generally, as the dose increased, the time to nCPAP failure appeared to be prolonged. In addition, the nCPAP failure rate observed in the third (highest dose) AEROSURF group was 38% (3/8), as compared to a 58% (14/24) failure rate in the control group as a whole.

"These data suggest that a single dose of AEROSURF may delay the time to invasive rescue therapy," continued Dr. Simonson. "Patients receiving endotracheal surfactant therapy often require multiple doses; similarly, multiple or increased doses of AEROSURF may have the potential to reduce the need for invasive rescue therapy. This observation merits further study. Accordingly, our upcoming clinical program will incorporate the ability to administer repeat dosing."

To summarize the physiological data assessed, the data related to gas exchange and the requirement for rescue therapy due to nCPAP failure appear to suggest that with AEROSURF, KL4 surfactant is being delivered into the lungs of premature infants with RDS.

Performance of the Novel CAG Technology

The capillary aerosol generator performed as designed by delivering a high-output, dense aerosol stream that met all output specifications. During the course of the phase 2a trial there were no device failures during treatment or device related adverse events. Overall, the device was well accepted by NICU personnel at all study sites.

Next Steps in AEROSURF Phase 2 Program

The Company believes that the achievement of objectives in the initial phase 2a clinical trial will inform and improve the further development of its AEROSURF clinical program for premature infants with RDS. The Company plans the following phase 2 clinical assessments going forward:

A phase 2a dose expansion in 29 to 34 weeks gestational age infants to evaluate increased doses and repeat dosing. The Company anticipates starting this trial in the second quarter of 2015 with completion in the fourth quarter of 2015.
A phase 2a clinical trial assessing the safety and tolerability of escalating doses of AEROSURF administered to 26 to 28 weeks gestational age infants, with an ability to administer repeat doses. The Company anticipates initiating this trial in July 2015, with completion in the fourth quarter of 2015.
A phase 2b clinical trial to determine the optimal dose and define the expected efficacy margin of AEROSURF treatment. The Company anticipates initiating this clinical trial in the fourth quarter of 2015, with completion in mid 2016.
This clinical trial was supported, in part, by a $1.9 million Phase II award of a $2.4 million Fast Track Small Business Innovation Research (SBIR) grant from the National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health (NIH) under award number 4R44HL107000-02. The content of this press release is solely the responsibility of the Company and does not necessarily represent the official views of the National Institutes of Health.
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Source: press release, 5/14/15. http://discoverylabs.investorroom.com/2015-05-14-Discovery-Labs-Announce...

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A Multicenter, Randomized, Open-Label, Controlled Trial to Assess the Safety and Tolerability of Lucinactant for Inhalation in Preterm Neonates
Enrollment: 80
Study Start Date: February 2014
Study Completion Date: November 2015
Primary Completion Date: October 2015 (Final data collection date for primary outcome measure)
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Source: clinical trials.gov. http://clinicaltrials.gov/ct2/show/NCT02074059?term=aerosurf&rank=3

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Compound/DeviceSpecialtyIndicationCompound ClassTarget
AerosurfRespiratoryRespiratory Distress Syndrome (RDS) (prevention in premature infants)Surfactant Replacement Therapy (SRT)Alveoli

Mechanism of action: Aerosurf is an aerosolized form of Surfaxin, a peptide, closely mimic the essential properties of human lung surfactant protein B (SP-B) allowing for the premature lung to absorb oxygen and to maintain proper airflow through the respiratory system.

Phase of Development: IIa

Event Type: Data: Phase IIa trial results

Dates: 2015-11-16 - 2015-11-30

Results:

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Discovery Labs Announces Top Line Results of AEROSURF® Phase 2a Clinical Program in Premature Infants 29 to 34 Weeks Gestational Age

Encouraging phase 2a data suggest that aerosolized KL4 surfactant delivered to premature infants with RDS may decrease nCPAP failure and the need for intubation

Data provides foundation and direction for AEROSURF phase 2b clinical trial

Nov 12, 2015

WARRINGTON, Pa., Nov. 12, 2015 /PRNewswire/ -- Discovery Laboratories, Inc. (NASDAQ: DSCO), a specialty biotechnology company focused on developing aerosolized KL4 surfactant therapies for respiratory diseases, today reported top line data from its recently completed AEROSURF® phase 2a clinical program in premature infants 29 to 34 week gestational age (GA) receiving nasal continuous positive airway pressure (nCPAP) for respiratory distress syndrome (RDS). The two-part program enrolled a total of 80 premature infants including 40 infants in five AEROSURF dose groups and 40 control infants on nCPAP alone. The Company previously announced top line data from the initial trial in May 2015. The Company is now reporting data on the overall phase 2a program in premature infants 29 to 34 week GA including the recently completed phase 2a expansion study. The data are encouraging and suggest that aerosolized KL4 surfactant delivered to premature infants with RDS is generally safe and well tolerated and may decrease nCPAP failure and the need for intubation. The Company is now advancing AEROSURF to a phase 2b clinical trial beginning with enrollment of premature infants 29 to 32 week GA.

Discovery Labs' management will host a conference call and live webcast, with a slide presentation including data from the clinical trial, today at 8:00 a.m. Eastern time to review and discuss the results of the trial. See below for details of the call.

Key objectives of the program were achieved, including (1) the primary objective of evaluating the safety and tolerability of aerosolized KL4 surfactant, compared to nCPAP alone; (2) proof of concept for the Company's proprietary technology platform based on physiological data suggesting that aerosolized KL4 surfactant is being delivered into the lungs of premature infants; and, (3) acceptable performance of the novel aerosol delivery technology in the neonatal intensive care unit (NICU). Key observations from the data include:

Overall, the safety and tolerability profile of the AEROSURF group in this phase 2a program was generally comparable to the control group. All reported adverse events and serious adverse events were those that are common and expected among premature infants with RDS. Based on the safety and tolerability profile observed in the program, the Company is progressing to the AEROSURF phase 2b clinical trial.
Data suggest that AEROSURF may be reducing the incidence of nCPAP failure (the need for intubation and delayed surfactant therapy). Through 72 hours after the start of treatment, AEROSURF treated patients, predominantly receiving a single dose, had lower rates of nCPAP failure compared to control in each of the last three dose groups studied. nCPAP failure rates were 53% in the control group compared to 38%, 29% (excluding one patient who was inappropriately enrolled) and 38% in the 45, 60 and 90 minute AEROSURF dose groups, respectively.
The Company has focused its planning for the phase 2b clinical trial on the 45 and 60 minute dose groups. The combined data for these groups indicate that, through 72 hours after the start of treatment, AEROSURF treated patients had a 33% nCPAP failure rate compared to 53% in the control group. This represents a 20% absolute reduction or a 38% relative reduction in nCPAP failure compared to control.

The phase 2a program in premature infants 29 to 34 week GA has provided a foundation and direction for the AEROSURF phase 2b trial in premature infants 26 to 32 week GA. The Company expects to initiate the phase 2b trial in the fourth quarter of 2015. The phase 2b trial will include the ability to administer repeat doses, if necessary. Enrollment will begin with premature infants 29 to 32 week GA, followed by enrollment of premature infants 26 to 28 week GA.
"Premature infants with RDS are born with immature lungs and are frequently unable to produce their own endogenous surfactant for up to 48 to 72 hours of life. With AEROSURF, we potentially have an opportunity to administer aerosolized KL4 surfactant non-invasively to support premature infants until they are able to produce their own surfactant." commented Steve Simonson, M.D., Discovery Labs' Chief Development Officer. "The data from the phase 2a program to date are very encouraging. We are seeing signals that suggest our goal of reducing nCPAP failures and intubations with AEROSURF appears achievable. If we are successful, we believe AEROSURF will represent a transformational change in the management of RDS."

Phase 2a Clinical Program in Premature Infants 29 to 34 Weeks GA

Study Design

The phase 2a clinical studies were multicenter, randomized, open-label, controlled studies in 80 premature infants 29 to 34 weeks GA receiving nCPAP for RDS, and designed to evaluate the safety and tolerability of aerosolized KL4 surfactant administered in five dose groups (15, 30, 45, 60 and 90 minute), compared to infants receiving nCPAP alone. In addition to safety and tolerability, another objective of the study was to establish proof of concept for the Company's proprietary technology platform based on physiological data suggesting that aerosolized KL4 surfactant is being delivered into the lung of premature infants, and acceptable performance of the aerosol delivery technology in the NICU.

Safety and Tolerability

Overall, the safety and tolerability profile of the AEROSURF group in the studies was generally comparable to the control group. All reported adverse events and serious adverse events were those that are common and expected among this fragile patient population. The most common adverse events observed included neonatal jaundice, constipation, apnea and anemia. The most common serious adverse events were air leaks (pneumothorax and pneumomediastinum). The incidence of adverse events and serious adverse events in the AEROSURF and control groups were generally comparable and there was no pattern observed of increasing adverse events or serious adverse events with increasing doses of AEROSURF.

Air leaks (including pneumothorax, pneumomediastinum and pulmonary interstitial emphysema) were the most common complication of prematurity in both the AEROSURF and control groups. There were a total of nine patients with air leaks in the AEROSURF group and seven patients with air leaks in the control group. One AEROSURF treated infant was found to have an air leak prior to study drug administration. One other infant in the AEROSURF group was inappropriately enrolled in the study, received only a brief exposure to study drug and has been excluded from the analysis of all of the physiological evaluations below. The incidence of air leaks was not unexpected and comparable to what has been reported in the literature (23-47%) for infants in this age group (Dargaville et al., 2013). All air leaks were resolved without complication and there was no pattern of increased incidence of air leaks with increasing AEROSURF dose.

Regarding tolerability of AEROSURF administration, the patient interface was well tolerated. Peri-dosing events, which are common in the endotracheal administration of surfactants currently, were infrequent in the AEROSURF group.

Physiological Evaluation

In exploratory analyses of certain safety and tolerability measures to assess whether aerosolized KL4 surfactant was being delivered to the lungs of premature infants and potentially having a physiological effect, measurements of gas exchange in the lungs and the timing of or need for endotracheal intubation and delayed (rescue) surfactant therapy due to nCPAP failure were evaluated in both the AEROSURF and control groups.

Requirement for Invasive Rescue Therapy due to nCPAP failure

Parameters associated with delayed surfactant therapy and nCPAP failure were assessed as part of the safety and tolerability profile of AEROSURF. These data were also used to evaluate whether AEROSURF had an impact on the need for intubation and delayed surfactant therapy due to nCPAP failure.

Overall, the nCPAP failure rates through 72 hours after the start of treatment or randomization to the control group were 63% (15 min), 75% (30 min), 38% (45 min), 29% (60 min) and 38% (90 min) for the five AEROSURF groups compared to 53% in the control group. AEROSURF treatment of 45 minutes and greater (primarily single doses) appears to be associated with lower rates of nCPAP failure representing a 28% (45 and 90 min) and 45% (60 min) relative reduction in nCPAP failure compared to control.

These data, combined with the favorable safety profile, have formed the basis for dose selection in the phase 2b program. The Company has focused on two dose groups (45 and 60 minutes) to define the appropriate upper dose to take into the phase 2b study. In doing so, the Company combined the data in the 45 min and 60 min AEROSURF dose groups (n=15) and assessed nCPAP failure compared to the control group (n=40). The results suggest that: (i) at six hours after the start of treatment or randomization to the control group, 18% of control patients required intubation and delayed surfactant therapy compared to no AEROSURF patients; and, (ii) at 72 hours after the start of treatment or randomization to the control group, 53% in the control group required intubation and delayed surfactant therapy compared to 33% of patients in the AEROSURF group. This represents a 20% absolute reduction or a 38% relative reduction in nCPAP failure compared to control.

Gas Exchange

Gas exchange parameters were assessed as part of the safety and tolerability profile of AEROSURF. No safety signals were observed with respect to gas exchange. The fraction of inspired oxygen (FiO2) required by an infant is considered a key measurement of how well the lung is functioning to oxygenate the blood. Healthy lungs can achieve appropriate blood oxygen saturation breathing room air; however, premature infants with RDS frequently require supplemental oxygen. Carbon dioxide levels in the blood (PCO2) are considered a measure of respiratory function and how efficiently the lungs eliminate carbon dioxide (CO2) from the bloodstream.

The data for all five dose groups suggest that AEROSURF is associated with a decrease in FiO2 shortly after initiation of treatment and numerically greater change in PCO2 levels compared to control. The changes in these parameters are consistent with the effects that one would expect to see with a surfactant delivered to the lung.

Performance of the Novel Aerosol Delivery Technology

The aerosol delivery technology performed as designed by delivering a high-output, dense aerosol stream that met all output specifications. During the course of the phase 2a trial, there were no device-related adverse events. Overall, the device was well accepted by NICU personnel at all study sites.

The clinical trials in 29 to 34 week GA premature infants was supported, in part, by a $1.9 million Phase II award of a $2.4 million Fast Track Small Business Innovation Research (SBIR) grant from the National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health (NIH) under award number 4R44HL107000-02. The content of this press release is solely the responsibility of the Company and does not necessarily represent the official views of the National Institutes of Health.

Conference Call and Webcast Details
Discovery Labs' management will host a conference call and live webcast, with a slide presentation including data from the clinical trial, today at 8:00 a.m. Eastern time to review and discuss the results of the trial. The live webcast and archive of the conference call can be accessed at http://discoverylabs.investorroom.com/events.

For "listen-only" participants and those who wish to take part in the question and answer portion of the call, dial (888) 346-0767 (domestic) or (412) 902-4251 (international). After placing the call, request to be joined into the Discovery Labs conference call. A replay of the conference call will be accessible through November 20, 2015 by dialing (877) 344-7529 (domestic) or (412) 317-0088 (international) and referencing conference ID number 10076116.
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Source: press release, 11/12/15. http://discoverylabs.investorroom.com/2015-11-12-Discovery-Labs-Announce...

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Discovery Labs to Announce Results from AEROSURF® Phase 2a Expansion Clinical Trial

Conference Call Scheduled for Thursday, November 12, 2015 at 8:00 a.m. Eastern Time

Nov 6, 2015

WARRINGTON, Pa., Nov. 6, 2015 /PRNewswire/ -- Discovery Laboratories, Inc. (Nasdaq: DSCO), a specialty biotechnology company focused on developing aerosolized KL4 surfactant therapies for respiratory diseases, will announce top-line results from its AEROSURF® Phase 2a expansion clinical trial in premature infants 29 – 34 gestational age with respiratory distress syndrome (RDS) on Thursday, November 12, 2015 at approximately 7:00 a.m. EST.

Discovery Labs management will host a conference call and live webcast, including a slide presentation, on Thursday, November 12, 2015 at 8:00 a.m. EST to review and discuss the results of the trial. The live webcast and archive of the conference call can be accessed at http://discoverylabs.investorroom.com/events.

For "listen-only" participants and those who wish to take part in the question and answer portion of the call, dial (888) 346-0767 (domestic) or (412) 902-4251 (international). After placing the call, request to be joined into the Discovery Labs conference call. A replay of the conference call will be accessible one hour after completion through November 20, 2015 by dialing (877) 344-7529 (domestic) or (412) 317-0088 (international) and referencing conference ID number 10076116.
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Source: press release, 11/05/15. http://discoverylabs.investorroom.com/2015-11-06-Discovery-Labs-to-Annou...

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