Discovery Labs revalidates optimized BAT in program to gain FDA approval of Surfaxin for RDS

May 19 2010

Discovery Laboratories, Inc. (Nasdaq:DSCO) announces today that the revalidation of its optimized fetal rabbit Biological Activity Test (BAT, an important quality control release and stability test) has been completed, having met all pre-specified acceptance criteria. The optimization and successful revalidation of the BAT is a key milestone in Discovery Labs' plans to resolve the sole remaining Chemistry, Manufacturing & Control (CMC) issue necessary to potentially gain U.S. Food and Drug Administration (FDA) marketing approval for Surfaxin^® (lucinactant) for the prevention of  Respiratory Distress Syndrome (RDS) in premature infants.  The safety and efficacy of Surfaxin for neonatal RDS has been previously demonstrated in a comprehensive Phase 3 clinical program.  If approved, Surfaxin would be the first synthetic, peptide-containing surfactant for commercial use in neonatal medicine.

The successful optimization and revalidation of the BAT represents a key component of the comprehensive preclinical program that Discovery Labs is now conducting to potentially address the sole remaining issue to gain FDA approval of Surfaxin.  Prior to optimizing and revalidating the BAT, Discovery Labs had several interactions with the FDA and submitted a proposed revalidation protocol, which also included pre-specified acceptance criteria.  The BAT optimization and recently completed revalidation has taken into account FDA suggestions and comments. 

To complete the comprehensive preclinical program, Discovery Labs will now employ the optimized BAT in a series of prospectively-designed, side-by-side preclinical studies with the well-established preterm lamb model of RDS.  Multiple Surfaxin batches will be employed to assess the short-term physiologic response following Surfaxin administration (via measurement of respiratory compliance) in both the preterm lamb model and the optimized BAT at various time points.  The resulting data will be examined to evaluate the relative changes, over time, in biological activity upon Surfaxin administration to determine the degree of comparability between the optimized BAT and the preterm lamb model.  These studies are intended to satisfy the FDA as to the BAT's ability to adequately discriminate biologically active from inactive Surfaxin drug product and establish the Surfaxin drug product's final acceptance criteria (with respect to biological activity as assessed by the BAT) for release and ongoing stability. 

Jerry Orehostky, Senior Vice President, Quality Operations, commented, "We believe that optimizing and revalidating the BAT has improved its performance and increased the likelihood that the results of our preclinical program will demonstrate, to the FDA's satisfaction, a level of comparability between data generated using the BAT and the preterm lamb model."      

Discovery Labs has also been communicating with the FDA regarding other important aspects of the comprehensive preclinical program and submitted for FDA review and comment a protocol outlining a proposed study design and success criteria for the side-by-side preclinical studies.  Recent communications with the FDA indicate that its written feedback to the protocol should be anticipated in June 2010.  Subject to satisfactory FDA feedback, Discovery Labs believes it remains on track to complete the preclinical program and submit its Complete Response to the FDA in the first quarter of 2011.

W. Thomas Amick, Chairman and interim Chief Executive Officer, commented, "We have worked productively with the FDA to improve the performance of the BAT, an important milestone for Surfaxin and our KL4 surfactant pipeline.  We will continue to avail ourselves of the FDA's willingness to provide guidance towards potential Surfaxin approval and advancing our other KL4 surfactant pipeline programs."