Full Press Release Details
Labs Provides a Third Quarter 2010 Business and
Warrington, PA - November 9, 2010 -
Discovery Laboratories, Inc. (Nasdaq: DSCO), a biotechnology company
developing its novel, synthetic, peptide-containing surfactant and related
aerosolization technologies as first in class therapies for severe respiratory
diseases, today provides an update on key pipeline and business initiatives and
a financial update for the third quarter ended September 30, 2010. The Company
will host a conference call this morning at 10:00 AM EST. The call-in number is
highlights, discussed in greater detail below, include:
Amick, Chairman of the Board and Chief Executive Officer of Discovery Labs
commented, "We are at an important transition point for our Company and the
third quarter has been very productive for Discovery Labs. We have
solidified our Company's leadership, significantly progressed the Surfaxin
complete response initiative and have begun to understand the potential role of
surfactant in two new disease targets - cystic fibrosis and acute respiratory
failure. As we move towards the end of 2010, Discovery Labs
will be focused on remaining activities to support the Surfaxin complete
response filing and ongoing strategic alliance initiatives while diligently
managing our financial resources."
KL4 Surfactant Pipeline
Surfaxin for Neonatal RDS: If
approved, Surfaxin would become the first synthetic, peptide-containing
surfactant for commercial use in neonatal medicine. The safety and efficacy of
Surfaxin for neonatal RDS has been previously demonstrated in a large,
multinational Phase 3 clinical program. Discovery Labs believes that the last
remaining step necessary to potentially gain FDA marketing approval for Surfaxin
for the prevention of RDS is to satisfy the FDA as to the final validation of an
important quality control release and stability test for Surfaxin, the fetal
rabbit biological activity test (BAT).
respect, Discovery Labs conducted a comprehensive preclinical program intended
to satisfy the FDA. The comprehensive preclinical program, as
proposed to the FDA, involved the optimization and subsequent revalidation of
the BAT, which was then employed in a series of prospectively-designed,
side-by-side preclinical studies with the well-established preterm lamb model of
RDS. Discovery Labs has taken into account the FDA's guidance in
conducting its comprehensive program. These proposed studies
were recently completed. The resulting dataset is undergoing final
review and compilation in preparation for submission to the FDA.
this process, Discovery Labs has had multiple interactions with the FDA intended
to ensure that the comprehensive preclinical program satisfies the FDA as to the
final validation of the BAT and its ultimate appropriateness as a release and
stability test for Surfaxin, upon potential approval. Previously,
Discovery Labs provided additional analysis to the FDA regarding the
revalidation of the BAT intended to aid the FDA in its final determination of
whether the BAT is appropriately validated for use as an ongoing quality control
release and stability test for Surfaxin, if approved. Discovery Labs
is awaiting FDA feedback regarding this analysis and anticipates further
interactions in advance of the pending complete response. Such
interactions with the FDA could affect the ultimate timing, conduct and outcomes
of remaining steps necessary to gain Surfaxin approval, including the potential
filing of the complete response. Discovery Labs believes it remains
on track to submit a complete response to the FDA in the first quarter of 2011,
potentially leading to Surfaxin approval later in the year.
Surfaxin LS and Aerosurf Neonatal RDS
Programs: Surfaxin LS (lyophilized lucinactant) is an
important life-cycle initiative, intended to further improve on the Surfaxin
product profile and provide access to international
markets. Discovery Labs has contracted with a leading pharmaceutical
contract manufacturing organization to establish a Surfaxin LS clinical supply
manufacturing capability that is compliant with current good manufacturing
practices (cGMP). Preparation for the manufacture of process
validation lots of lyophilized lucinactant has initiated in the fourth quarter
of 2010. Additionally, the Company intends to seek regulatory
guidance for its planned development program, first with the FDA in the fourth
quarter of 2010 and then with the EMA in early 2011.
is a novel drug/device combination therapy intended to allow early
administration of aerosolized surfactant to address neonatal RDS. Aerosurf holds
the promise to significantly expand the use of surfactant therapy by providing
neonatologists with a less invasive means of delivering KL4 surfactant
without the current requirement of invasive endotracheal intubation and
mechanical ventilation. The Company is preparing to engage the FDA
and EMA in the first half of 2011 for regulatory guidance with respect to the
planned Aerosurf development program. Discovery Labs, working with a
leading technology company with expertise in biomedical device development, is
optimizing the design of the capillary aerosolization device to potentially
reduce development risk and satisfy regulatory and development requirements for
Surfaxin Phase 2 trial for
ARF: Discovery Labs recently completed a comprehensive
analysis of its Phase
2 clinical trial of Surfaxin in children with ARF, a critical condition often
caused in children by severe respiratory infections. The study was a
multicenter, randomized, masked trial that enrolled 165 children under the age
of two and compared Surfaxin treatment to standard of care alone. The
objective was to evaluate the safety and tolerability of intratracheal
administration of Surfaxin and to assess whether Surfaxin treatment could
decrease the duration of mechanical ventilation in children with ARF. The trial
was designed as an estimation trial, intended to evaluate multiple, potentially
clinically-relevant endpoints, as this was the first ever exposure of Surfaxin
in a pediatric ARF patient population. The previously reported preliminary
analysis indicated that Surfaxin treatment was generally safe and well tolerated
in the ARF patient population in this trial. Additionally, Surfaxin
treatment reduced time on mechanical ventilation by approximately 10% compared
with standard of care alone, although this observation was not statistically
recently completed comprehensive analysis demonstrated that, based on patient
stratification by severity of lung injury, Surfaxin treatment significantly
reduced time on mechanical ventilation in the least severe patient segment
compared with standard of care alone. Furthermore, the data
demonstrated that Surfaxin intervention reduced the need for a second dose
(p<0.05), suggesting
a decrease in disease severity following surfactant treatment. The