SECURITIES
AND EXCHANGE COMMISSION
Washington,
D.C. 20549
FORM
8-K
CURRENT
REPORT
Pursuant
to Section 13 or 15(d) of the
Securities
Exchange Act of 1934
February
1, 2011
Date of
Report (Date of earliest event reported)
Discovery
Laboratories, Inc.
(Exact
name of registrant as specified in its charter)
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Delaware
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000-26422
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94-3171943
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(State
or other jurisdiction
of
incorporation)
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(Commission
File Number)
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(IRS
Employer
Identification
Number)
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2600
Kelly Road, Suite 100
Warrington,
Pennsylvania 18976
(Address
of principal executive offices)
(215)
488-9300
(Registrant's
telephone number, including area code)
(Former
name or former address, if changed since last report)
Check the
appropriate box below if the Form 8-K filing is intended to simultaneously
satisfy the filing obligation of the registrant under any of the following
provisions:
¨ Written
communications pursuant to Rule 425 under the Securities Act (17 CFR
230.425)
¨ Soliciting
material pursuant to Rule 14a-12 under the Exchange Act (17 CFR
240.14a-12)
¨ Pre-commencement
communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR
240.14d-2(b))
¨ Pre-commencement communications
pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR
240.13e-4(c))
Item
8.01. Other
Events.
On
February 1, 2011, Discovery Laboratories, Inc. (the “Company”) issued a press
release providing an expanded update regarding its ongoing efforts to file a
Complete Response intended to gain U.S. Food and Drug Administration (“FDA”)
marketing authorization of Surfaxin® for the prevention of respiratory distress
syndrome (“RDS”) in premature infants. The Company previously
announced on January 10, 2011, that the FDA had recently provided detailed,
written direction in response to an earlier submitted proposal by the Company to
its ongoing comprehensive preclinical program intended to gain Surfaxin
approval.
The
Company believes that a key remaining step to potentially gain FDA marketing
approval for Surfaxin is to satisfy the FDA as to the final validation of the
fetal rabbit biological activity test (“BAT”). The BAT is an important
quality control release and stability test for Surfaxin. Final BAT
validation is intended to satisfy the FDA with respect to the ability of the BAT
to adequately reflect the biological activity of Surfaxin throughout its shelf
life and to discriminate biologically active from inactive Surfaxin drug
product. The Company has been conducting a comprehensive preclinical
program with regard to this key remaining step. The BAT has undergone a
number of method refinements intended to optimize its performance and reduce
assay variability. The optimized BAT has subsequently been used to generate
data to support BAT validation as well as to demonstrate concordance between the
BAT and the well-established preterm lamb model of RDS by performing a series of
prospectively-designed, side-by-side preclinical studies (i.e., concordance
studies). Data from the preterm lamb model and BAT concordance studies are
intended to support final BAT validation and to demonstrate comparability of
drug product used in the Phase 3 clinical program with Surfaxin drug product to
be manufactured for commercial use.
The
Company has been interacting with the FDA in an effort to ensure that the
comprehensive preclinical program (and the Complete Response) ultimately will
satisfy the FDA as to the final validation of the BAT. The FDA's most
recent communication clearly indicates that several aspects of the Company's
proposed approach to the BAT validation are reasonable and provides detailed,
written direction regarding certain components of the Company’s comprehensive
pre-clinical program. The Company believes that it can incorporate the
FDA's direction into its ongoing efforts to complete the comprehensive
preclinical program and be in a position to file a Complete Response for
Surfaxin by early third quarter 2011, which, after an anticipated six-month FDA
review cycle, could lead to potential Surfaxin approval early in the first
quarter 2012.
The press
release is attached as Exhibit 99.1 hereto and the text of the press release is
incorporated herein by reference to such exhibit.
2
The
information in this Form 8-K includes certain “forward-looking” statements
relating, among other things, to the Company’s understanding of the
recently-received written guidance from the FDA and the remaining questions
identified in the FDA’s April 2009 Complete Response Letter that must be
addressed to gain FDA approval of Surfaxin. Although the Company currently
believes that it may still succeed in submitting a Complete Response and gaining
approval of its New Drug Application for Surfaxin for the prevention of RDS in
premature infants, anticipated activities will require that the Company raise
significant amounts of additional capital. The Company has
initiated activities relating to this most recent FDA communication, and is
presently assessing whether to request further clarification from the FDA on
certain technical aspects of the comprehensive preclinical program, and also
anticipates potential further interactions with the FDA in advance of filing a
Complete Response. Such potential 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. In
addition to uncertainties related to the FDA's review of the Complete Response,
the Company presently anticipates that the FDA will likely inspect and otherwise
assess the manufacturing and quality assurance/quality control facilities for
Surfaxin including those of third-party raw material suppliers and testing
laboratories. The outcomes of such FDA activities could also affect the
ultimate timing and remaining steps necessary to gain Surfaxin approval. In
addition, the foregoing activities and the ultimate outcomes remain subject to a
variety of risks and uncertainties that could cause actual results to be
materially different. These risks and uncertainties include, but are not limited
to, risks that (i) the FDA may not accept the additional data or may interpret
the data in a different manner such that, ultimately, the FDA may not approve
Surfaxin or that the FDA may subject the marketing of Surfaxin to onerous
requirements that significantly impair marketing activities; (ii) the Company
may be unable to complete the manufacture of additional Surfaxin batches to
address the FDA’s request for additional data in a timely manner, (iii) the
Company may identify unforeseen problems that have not yet been discovered or
the FDA could in the future impose additional requirements to gain approval of
Surfaxin; (iv) the Company may be unable to raise sufficient additional capital,
through financings, strategic collaborations, or otherwise; and (v) other risks
included in the Company’s most recent reports on Forms 10-K, 10-Q and 8-K, and
any amendments thereto. Any failure to satisfy the issues raised by the FDA, in
the Complete Response letter or in related discussions, could significantly
delay, or preclude outright, gaining approval of Surfaxin, which could
potentially delay or prevent the approval of the Company’s other products and
would have a material adverse effect on the Company’s business.
Item
9.01. Financial Statements and
Exhibits.
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(d)
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Exhibits
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99.1
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Press
release dated February 1, 2011
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Cautionary
Note Regarding Forward-looking Statements:
To the
extent that statements in this Current Report on Form 8-K are not strictly
historical, including statements as to business strategy, outlook, objectives,
future milestones, plans, intentions, goals, future financial conditions, future
collaboration agreements, the success of the Company’s product development or
otherwise as to future events, such statements are forward-looking, and are made
pursuant to the safe harbor provisions of the Private Securities Litigation
Reform Act of 1995. The forward-looking statements contained in this Current
Report are subject to certain risks and uncertainties that could cause actual
results to differ materially from the statements made. Such risks and others are
further described in the Company's filings with the Securities and Exchange
Commission including the most recent reports on Forms 10-K, 10-Q and 8-K, and
any amendments thereto.
3
SIGNATURES
Pursuant
to the requirements of the Securities Exchange Act of 1934, the registrant has
duly caused this report to be signed on its behalf by the undersigned hereunto
duly authorized.
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Discovery
Laboratories, Inc.
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By:
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/s/ W. Thomas Amick
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Name:
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W.
Thomas Amick
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Title:
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Chairman
of the Board and Chief
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Executive
Officer
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Date: February
2, 2011
4
Exhibit
99.1
Discovery
Labs Provides Expanded Update Regarding its Program for Surfaxin® U.S.
Marketing Authorization
Company
to Hold Conference Call to Detail Progress Towards Surfaxin Complete
Response
Warrington,
PA — February 1, 2011 — Discovery Laboratories, Inc. (Nasdaq: DSCO) is providing an
expanded update regarding ongoing efforts to file a Complete Response intended
to gain U.S. Food and Drug Administration (FDA) marketing authorization for
Surfaxin®
for the prevention of respiratory distress syndrome (RDS) in premature
infants. On January 10, 2011, Discovery Labs issued a press release
reporting that the FDA had recently provided detailed, written direction in
response to an earlier submitted proposal by Discovery Labs relating to its
ongoing comprehensive preclinical program intended to gain Surfaxin
approval. Throughout the conduct of the program, Discovery Labs has
been interacting with the FDA and incorporating the FDA’s guidance into its
efforts to complete the program and file the Surfaxin Complete
Response. The filing of the Complete Response is currently targeted
for early third quarter 2011 and anticipated to lead to a potential approval
early in the first quarter 2012.
The
Company will host a conference call this morning at 10:00 AM EST to provide an
update regarding certain activities relating to the Surfaxin Complete Response.
The call-in number
is 866-332-5218 (additional call-in information below).
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 previously been demonstrated in a
large, multinational Phase 3 clinical program.
Surfaxin®
for Neonatal RDS – Status of
Comprehensive Preclinical Program for Filing Complete Response to Gain
FDA Marketing Authorization
Discovery
Labs believes that a key remaining step to potentially gain FDA marketing
approval for Surfaxin is to satisfy the FDA as to the final validation of the
fetal rabbit biological activity test (BAT). The BAT is an important
quality control release and stability test for Surfaxin. Final BAT
validation is intended to satisfy the FDA with respect to the ability of the BAT
to adequately reflect the biological activity of Surfaxin throughout its shelf
life and to discriminate biologically active from inactive Surfaxin drug
product. Discovery Labs has been conducting a comprehensive
preclinical program with regard to this key remaining step. The BAT
has undergone a number of method refinements intended to optimize its
performance and reduce assay variability. The optimized BAT has
subsequently been used to generate data to support BAT validation as well as to
demonstrate concordance between the BAT and the well-established preterm lamb
model of RDS by performing a series of prospectively-designed, side-by-side
preclinical studies (i.e., concordance studies). Data from the
preterm lamb model and BAT concordance studies are intended to support final BAT
validation and to demonstrate comparability of drug product used in the Phase 3
clinical program with Surfaxin drug product to be manufactured for commercial
use.
Discovery
Labs has been interacting with the FDA in an effort to ensure that the
comprehensive preclinical program (and the Surfaxin Complete Response) will
ultimately satisfy the FDA as to the final validation of the BAT. The
FDA’s most recent communication clearly indicates that several aspects of
Discovery Labs’ proposed approach to the BAT validation are reasonable and
provides detailed, written direction regarding certain components of Discovery
Labs’ comprehensive pre-clinical program. Discovery Labs believes
that it can incorporate the FDA’s direction into its ongoing efforts to complete
the comprehensive preclinical program and be in a position to file a Surfaxin
Complete Response by early third quarter 2011, which, after an anticipated
six-month FDA review cycle, could lead to potential Surfaxin approval early in
the first quarter 2012.
BAT Optimization and Final
Validation: Before undertaking steps to optimize the BAT,
Discovery Labs submitted a proposal to FDA outlining several specific method
refinements intended to improve BAT performance and reduce assay
variability. After taking into account the FDA’s response, Discovery
Labs incorporated the proposed method refinements into the BAT and conducted
testing that demonstrates that all pre-specified acceptance criteria were met
and that BAT optimization has resulted in a greater-than-40% reduction in assay
variability relative to the BAT methodology that was employed prior to
2010.
Following
BAT optimization, at the FDA’s suggestion, Discovery Labs submitted an
additional proposal regarding specific and detailed aspects of final BAT
validation. With respect to certain technical criteria relating to
final BAT validation, the recent communication directs Discovery Labs to
increase the sample size of specified data sets by testing additional Surfaxin
batches. Discovery Labs currently has data from several batches that
have previously been manufactured and analyzed. To be responsive to
FDA’s direction, Discovery Labs has initiated and anticipates completing
manufacture of additional Surfaxin batches in the first quarter of this
year. The additional batches will be tested using the optimized BAT
and the additional data will be incorporated into the Complete
Response.
Concordance Studies –
Throughout the conduct of the comprehensive preclinical program, Discovery Labs
has interacted with the FDA on certain aspects of the concordance
studies. Multiple Surfaxin batches have been used to assess the
biological activity of Surfaxin via measurement of respiratory compliance at
various time points across the proposed shelf-life in both the preterm lamb
model and the optimized BAT. The FDA has previously indicated that,
to gain Surfaxin approval, data generated in the concordance studies must
demonstrate the same relative changes in biological activity over time in both
models using both regression analysis as well as a point-to-point
comparison. Discovery Labs has evaluated the concordance data
generated to date by these models using both regression analysis and
point-to-point comparisons. Discovery Labs believes that these data
demonstrate the same relative changes in biological activity over time in both
the optimized BAT and the preterm lamb models and support the comparability of
drug product used in the phase 3 clinical program and Surfaxin drug product to
be manufactured for commercial use.
2
The most
recent proposal submitted by Discovery Labs to the FDA also requested
clarification with respect to certain limited technical aspects of the
concordance study. The FDA’s response includes detailed direction
with respect to generating limited additional confirmatory data through further
concordance testing. Discovery Labs plans to generate these
additional data for inclusion in the Complete Response, which data must be
consistent with the concordance data generated to date.
DISCLOSURE NOTICE:
The
information in this press release includes certain “forward-looking” statements
relating, among other things, to Discovery Labs’ understanding of the
recently-received written guidance from the FDA and the remaining questions
identified in the FDA’s April 2009 Complete Response Letter that must be
addressed to gain FDA approval of Surfaxin. Discovery Labs has
interacted with the FDA in an effort to ensure that the comprehensive
preclinical program (and the Surfaxin Complete Response) will ultimately satisfy
the FDA. In addition, Discovery Labs is presently assessing whether
to request further clarification from the FDA on certain technical aspects of
the comprehensive preclinical program. There can be no assurances,
however, that the FDA will be satisfied with all aspects of the comprehensive
preclinical program or that, should Discovery Labs request further
clarification, that the FDA will provide further direction prior to the planned
filing of the Complete Response. In any event, such potential
interactions with the FDA or lack thereof could affect the ultimate timing,
conduct and outcomes of remaining steps necessary to gain Surfaxin approval,
including the potential filing of the Complete Response. In addition
to uncertainties related to the FDA’s review of the Complete Response, Discovery
Labs presently anticipates that the FDA will likely inspect and otherwise assess
the manufacturing and quality assurance/quality control facilities for Surfaxin
including those of third-party raw material suppliers and testing
laboratories. The outcomes of such FDA activities could also affect
the ultimate timing and remaining steps necessary to gain Surfaxin
approval.
The
completion of the aforementioned activities will require Discovery Labs to raise
significant amounts of additional capital and, notwithstanding, the ultimate
outcomes remain subject to a variety of risks and uncertainties that could cause
actual results to be materially different. These risks and
uncertainties include, but are not limited to, risks that (i) the FDA may
not approve Surfaxin or may subject the marketing of Surfaxin to onerous
requirements that significantly impair marketing activities; (ii) Discovery
Labs may be unable to complete the manufacture of additional Surfaxin batches
within the time frame set forth above, (iii) Discovery Labs may identify
unforeseen problems that have not yet been discovered or the FDA could in the
future impose additional requirements to gain approval of Surfaxin; and (iv)
Discovery Labs may be unable to raise sufficient additional capital, through
financings, strategic collaborations, or otherwise. Any failure to
satisfy the issues raised by the FDA, in the Complete Response letter or in
related discussions, could significantly delay, or preclude outright, gaining
approval of Surfaxin, which could potentially delay or prevent the approval of
Discovery Labs’ other products.
Surfaxin
is an investigational drug product and is not approved by the FDA or any other
world health regulatory authority for use in humans.
3
About
Discovery Labs
Discovery
Laboratories, Inc. is a biotechnology company developing surfactant therapies
for respiratory diseases. Surfactants are produced naturally in the lungs
and are essential for breathing. Discovery Labs’ novel proprietary KL4 surfactant
technology produces a synthetic, peptide-containing surfactant that is
structurally similar to pulmonary surfactant and is being developed in liquid,
aerosol and lyophilized formulations. In addition, Discovery Labs’
proprietary capillary aerosolization technology produces a dense aerosol, with a
defined particle size that is capable of potentially delivering aerosolized
KL4
surfactant to the lung without the complications currently associated with
liquid surfactant administration. Discovery Labs believes that its
proprietary technology platform makes it possible, for the first time, to
develop a significant pipeline of surfactant products to address a variety of
respiratory diseases for which there frequently are few or no approved
therapies. For more information, please visit our website at www.Discoverylabs.com.
Conference Call Details
Discovery
Labs will hold a conference call today, February 1, 2011, at 10:00 AM EST to
further discuss the foregoing. The call in number is 866-332-5218.
The international call in number is 706-679-3237. An audio webcast will be
available through a live broadcast on the Internet at http://us.meeting-stream.com/discoverylaboratories_020111 and
www.discoverylabs.com.
A replay of the conference call will be available two hours after the call’s
completion and remain available through February 8, 2011. The replay
number to hear the conference call is 800-642-1687 or 706-645-9291. The
passcode is 40660551.
Forward-Looking
Statements
To
the extent that statements in this press release are not strictly historical,
all such statements are forward-looking, and are made pursuant to the safe
harbor provisions of the Private Securities Litigation Reform Act of 1995.
These forward-looking statements are subject to certain risks and uncertainties
that could cause actual results to differ materially from the statements
made. Examples of such risks and uncertainties, including those related to
the Company’s comprehensive preclinical program, development and manufacturing
activities and related regulatory efforts, are described in Discovery Labs’
filings with the Securities and Exchange Commission including the most recent
reports on Forms 10-K, 10-Q and 8-K, and any amendments thereto.
Contact
Information:
John G.
Cooper, President and Chief Financial Officer
215-488-9490
4