Discovery Laboratories, Inc has created a lyophilized formulation of its synthetic, peptide-containing KL4 surfactant technology. Lyophilized KL4 surfactant is being developed to support Discovery Labs' pipeline of surfactant product candidates including Surfaxin (lucinactant as a liquid formulation) and Aerosurf (lucinactant as an aerosolized formulation).
Discovery Labs is developing a pipeline of surfactant-based therapies targeted to address several respiratory disorders associated with surfactant deficiency or dysfunction. Lyophilized KL4 surfactant is manufactured as a dry powder formulation that is reconstituted prior to use. Key next steps include planned discussions with global regulatory agencies intended to determine the development pathway leading to the initiation of a late-stage neonatal clinical trial programme.
The currently available surfactants are animal-derived liquid formulations that require constant refrigeration prior to use.
Surfaxin, which is Discovery Labs' lead product that is currently under review by the United States FDA for potential approval for prevention of Respiratory Distress Syndrome (RDS) in premature infants, also requires refrigeration prior to use. Lyophilized KL4 surfactant is intended to eliminate continuous cold chain storage and refrigeration, greatly improving product flexibility and ease of use for healthcare practitioners.
Robert J Capetola, president and chief executive officer of Discovery Labs, commented, "We have established that the key functional attributes of our liquid KL4 surfactant are preserved following lyophilization and are now completing development activities to support our regulatory and clinical development paths. A comprehensive assessment of development scale manufacturing lots is ongoing including full drug product characterization. These development lots are also actively being assessed under both real-time and accelerated stability protocols and are being evaluated in several proof-of-concept experiments in well established pre-clinical RDS models. Importantly, the results of this work may provide additional intellectual property to support our technology platform. We believe that our lyophilized KL4 surfactant technology could provide several technical advantages for our surfactant product candidates."
Discovery Labs is planning a worldwide, late-stage development programme for Surfaxin LS, the lyophilized formulation of Surfaxin, for the prevention of RDS in premature infants. To pursue regulatory approval for Surfaxin LS in the United States, Discovery Labs plans to request a pre-IND meeting with the FDA with a view to agreeing upon and potentially initiating a clinical trial with Surfaxin LS in 2010.
Similarly, to gain regulatory approval for Surfaxin LS in Europe, Discovery Labs plans to request a scientific advice meeting with the EMEA with a view to agreeing upon and potentially initiating a clinical trial in 2010. If a similar clinical trial design satisfies both regulatory agencies, Discovery Labs anticipates conducting a single clinical program to gain regulatory approval for Surfaxin LS in the United States and Europe.
"We believe there is an unprecedented opportunity to improve the management of RDS throughout the world," commented Dr Thomas F Miller, Discovery Labs' senior vice president of Commercialization and Corporate Development. "The neonatal market is quite underserved today. We remain committed to the advancement of our KL4 surfactant technology platform to improve the quality of RDS management in the neonatal intensive care unit. Our three lead programmes, Surfaxin, Surfaxin LS and Aerosurf, represent a completely new and comprehensive approach for RDS management in which the therapeutic solution is tailored to the unique need of a patient."
Discovery Labs is a biotechnology company developing Surfactant Replacement Therapies (SRT) for respiratory diseases.