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
July
1, 2009
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:
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Written
communications pursuant to Rule 425 under the Securities Act (17 CFR
230.425)
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Soliciting
material pursuant to Rule 14a-12 under the Exchange Act (17 CFR
240.14a-12)
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Pre-commencement
communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR
240.14d-2(b))
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Pre-commencement communications
pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR
240.13e-4(c))
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Item
8.01.
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Other
Events.
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On July
1, 2009, Discovery Laboratories, Inc. (the “Company”) issued a press release and
on July 2, 2009 held a conference call reporting on the results of its June 2,
2009 end-of-review meeting with the U.S. Food and Drug Administration
(FDA). The meeting was convened to discuss certain issues identified
in an April 17, 2009 Complete Response Letter that the FDA had delivered to the
Company with respect to the Company’s new drug application for Surfaxin®
(lucinactant) for the prevention of Respiratory Distress Syndrome (RDS) in
premature infants. The meeting focused primarily on the Surfaxin
fetal rabbit biological activity test (BAT), a quality control stability and
release test, and whether (i) data that had been previously submitted to the FDA
and generated using both the BAT and a well-established preterm lamb model of
RDS adequately supports the comparability between Surfaxin drug product used in
Phase 3 clinical trials and the Surfaxin drug product intended to be
manufactured for commercial use (“Comparability”), and (ii) the BAT can
adequately distinguish change in Surfaxin biological activity over
time.
At the
meeting, the Company presented a compilation of previously-submitted data from
the preterm lamb model and BAT studies, together with a comprehensive
statistical evaluation of such data (in the form of a comparative regression
analysis) that was intended to establish Comparability to the satisfaction of
the FDA. The FDA stated, for the first time, that an agreement
that it had reached with the Company in 2006 and 2008 to allow for establishing
Comparability using the preterm lamb and BAT studies is unprecedented and the
determination of whether the Company has adequately established Comparability is
solely within the FDA’s discretion. The FDA, for the first time, also
stated that data generated from the preterm lamb model and BAT studies must
demonstrate, in a point-to-point analysis, the same relative changes in
respiratory compliance between both models over time. Based on this
newly-defined standard, the FDA indicated that, from the FDA’s perspective, the
data analysis provided by the Company did not meet that standard. The
FDA suggested that the comparability studies in the preterm lamb model and the
BAT would not be necessary if the BAT had been implemented to assess Surfaxin
drug product used in the Phase 3 clinical trials. As an alternative
to demonstrating Comparability using the preterm lamb model and BAT, the FDA
suggested that the Company could consider conducting a limited clinical trial
employing only the BAT as a path forward to Surfaxin approval.
Although
the Company could consider conducting a limited clinical trial to establish
Comparability and potentially gain approval of Surfaxin, the Company believes
that, given its strategic and financial priorities, it is more prudent to focus
resources on the ongoing lyophilized and aerosolized surfactant development
programs, including Surfaxin LS™ and Aerosurf®. The
Company believes that Surfaxin LS™ and Aerosurf® have the
potential to greatly advance the management of RDS and treat more patients
suffering from RDS, while creating a significant economic
opportunity.
In
addition, at the June 2 meeting, with respect to the BAT, (a) the FDA commented
that the data presented appears to confirm that the BAT can distinguish active
from inactive drug product (although it had previously questioned whether the
BAT, when compared to the preterm lamb model, could adequately monitor Surfaxin
biological activity over time), and (b) the Company advised the FDA of ongoing
efforts to further refine the BAT in accordance with the Company’ continuing
quality improvement initiatives. The Company believes that the BAT,
as an ICH validated method, represents an acceptable quality control test to
assess biological activity and is continuing to employ the BAT during the
conduct of ongoing clinical trials addressing Acute Respiratory Failure and
Cystic Fibrosis, consistent with guidance from the FDA, and plans to use the BAT
in its pending clinical programs, Surfaxin LS and Aerosurf for RDS.
2
With
respect to ongoing regulatory activities for Surfaxin, the Company has submitted
proposed corrections and additions to the formal FDA
minutes. Following finalization of the meeting minutes, the Company
will determine its next course of action with respect to the Surfaxin NDA, which
may entail further interaction with the FDA to assess whether Surfaxin approval
can be gained without additional clinical trials, or potentially exercising its
right of appeal through the FDA’s Formal Dispute Resolution
process.
The press
release is attached as Exhibit 99.1 hereto.
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Item
9.01.
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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 July 1, 2009
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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.
| Discovery Laboratories, Inc. | |||
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By:
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/s/ Robert J. Capetola | |
| Name: Robert J. Capetola, Ph.D. | |||
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Title: President
and Chief Executive Officer
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Date: July
2, 2009
4
Exhibit
99.1
Discovery
Labs Reports on Surfaxin End of Review Meeting with FDA
Conference
Call Thursday, July 2, 2009 at 8:30 AM
Warrington, PA – July 1, 2009 --
Discovery Laboratories, Inc. (Nasdaq:DSCO) announces today, after receipt
of written minutes from the United States Food and Drug Administration, the
results of its June 2, 2009 meeting with the FDA. This meeting followed the
FDA’s April 17 Complete Response Letter for Surfaxin®
(lucinactant) for the prevention of Respiratory Distress Syndrome (RDS) in
premature infants and was convened to discuss resolution of the remaining
primary issue necessary for marketing approval of Surfaxin. The meeting focused
on the Surfaxin fetal rabbit biological activity test (BAT, a quality control
stability and release test), specifically whether data that had been previously
submitted to the FDA and generated using both the BAT and a well-established
preterm lamb model of RDS adequately supports the comparability of Surfaxin
clinical drug product to commercial drug product, and whether the BAT can
adequately distinguish change in Surfaxin biological activity over
time.
At the
recent June 2 meeting, Discovery Labs learned that the FDA will now apply a
newly-defined standard to determine whether Discovery Labs has adequately
demonstrated comparability of Surfaxin clinical to commercial drug
product. This new standard represents a significant hurdle for
approval of Surfaxin. Discovery Labs believes that the information
provided to the FDA for the meeting demonstrates comparability and supports
Surfaxin approval. However, in light of the FDA’s newly-defined standard,
Discovery Labs now believes that it is unlikely to satisfy this requirement with
existing preclinical comparability data and gain Surfaxin approval in the near
term.
Robert J.
Capetola, Ph.D., President and Chief Executive Officer of Discovery Labs,
commented, “With agreement from the FDA, we have diligently pursued initiatives,
including multiple preclinical experiments, that were expected to lead to
Surfaxin approval for RDS. The totality of this work, along with the FDA’s
acknowledgment of the robustness of our Phase 3 clinical trial and the
tremendous strides made in manufacturing and quality operations, led us to
believe that we had comprehensively satisfied all of the FDA requirements. Per
the June 2 meeting, the central remaining issue relates to whether data
generated using the BAT and preterm lamb model of RDS supports the comparability
of Surfaxin clinical to commercial drug product to the FDA’s satisfaction. This
comparability issue is limited to this New Drug Application and does not alter
our existing clinical programs or development plans for our
pipeline.”
Discovery
Labs will now focus on maximizing the inherent value of its novel KL4 surfactant
and aerosolization platforms and will minimize development risk by leveraging
Surfaxin’s established proof-of-efficacy in RDS. The two highest priority
pipeline programs are Surfaxin LS™ and Aerosurf® -- drugs
that have the potential to greatly advance the management of RDS and treat more
patients suffering from RDS, while creating a significant economic
opportunity. The synthetic nature and formulation flexibility of
Discovery Labs’ KL4 surfactant
platform also supports expansion into a wide range of respiratory disease
conditions. Discovery Labs intends to pursue these opportunities
through strategic alliances, although there can be no assurance that such
alliances can be obtained.
Comparability of Surfaxin
Clinical Drug Product to Commercial Drug Product
During
Surfaxin’s Phase 3 clinical trials, a leading academic neonatologist assessed
the biological activity of the clinical batches by measuring respiratory
compliance in a well-established preterm lamb model of RDS. After
completing Surfaxin’s Phase 3 clinical trials, in accordance with discussions
with the FDA, Discovery Labs validated and implemented the BAT as a recurring
quality control test to confirm biological activity for Surfaxin release and
stability testing. Based on agreements reached in meetings with the
FDA in 2006 and 2008, Discovery Labs conducted a series of preclinical
experiments to establish comparability between Surfaxin drug product used in
Phase 3 clinical trials and the Surfaxin drug product intended to be
manufactured for commercial use. Accordingly, Discovery Labs
initiated a series of side-by-side studies employing both the preterm lamb model
of RDS and the BAT and believes that the correlated results demonstrate
comparability and support approval of Surfaxin.
At the
recent June 2 meeting, Discovery Labs presented a compilation of
previously-submitted data from the preterm lamb model and BAT studies, together
with a comprehensive statistical evaluation of such data, intended to establish
to the satisfaction of the FDA comparability of clinical drug product to
Surfaxin drug product to be manufactured for commercial use. The
comprehensive statistical evaluation was a comparative regression analysis using
an accepted FDA statistical method. Discovery Labs believes that the
data and related statistical evaluation that it submitted to the FDA are highly
supportive of the comparability of clinical drug product to commercial
Surfaxin.
The FDA
stated, for the first time, that the 2006 and 2008 agreement with Discovery Labs
to establish comparability through these studies is unprecedented and the
determination of whether Discovery Labs has adequately established comparability
is solely within the FDA’s discretion. The FDA now insists, for the first time,
that data generated from the preterm lamb model and BAT studies must
demonstrate, in a point-to-point analysis, the same relative changes in
respiratory compliance between both models over time. Based on this
newly-defined standard, the FDA indicated that to adequately establish
comparability in this manner would be an extremely high hurdle and that, from
the FDA’s perspective, the data analysis provided by Discovery Labs did not meet
that standard.
The FDA
suggested that the comparability studies in the preterm lamb model and the BAT
would not be necessary if the BAT had been implemented to assess Surfaxin drug
product used in the Phase 3 clinical trials. Additionally, the FDA
suggested that, to increase the likelihood of gaining Surfaxin approval and as
an alternative to demonstrating comparability using the preterm lamb model and
BAT, Discovery Labs could consider conducting a limited clinical trial employing
only the BAT as a path forward to Surfaxin approval.
The BAT as a Quality Control
Drug Product Release Assay
The BAT
has been validated as a quality control test in accordance with current Good
Manufacturing Practices and ICH guidelines and was implemented for Surfaxin
release and stability testing as well as for Discovery Labs’ other pipeline
programs. The BAT is only one of numerous methods that Discovery Labs
employs in an extensive quality surveillance program to assess quality and
stability. These highly sophisticated tests monitor drug product quality at
release and through shelf-life and represent very sensitive methods for
detecting changes in quality over time.
2
At the
June 2 meeting, the FDA commented that the data presented appears to confirm
that the BAT can distinguish active from inactive drug product (although it had
previously questioned whether the BAT, when compared to the preterm lamb model,
could adequately monitor Surfaxin biological activity over
time). Also at this meeting, Discovery Labs advised the FDA of
ongoing efforts to further refine the BAT in accordance with Discovery Labs’
continuing quality improvement initiatives.
Discovery
Labs believes that the BAT, as an ICH validated method, represents an acceptable
Quality Control test to assess biological activity. Accordingly,
Discovery Labs is continuing to employ the BAT during the conduct of ongoing
clinical trials addressing Acute Respiratory Failure and Cystic Fibrosis,
consistent with guidance from the FDA, and plans to use the BAT in its pending
clinical programs, Surfaxin LS and Aerosurf for RDS.
Discovery Labs Going
Forward
As
suggested by the FDA at the June 2 meeting, Discovery Labs could consider
conducting a limited clinical trial to support comparability of clinical to
commercial Surfaxin drug product and potentially gain approval. However,
Discovery Labs believes that, given its strategic and financial priorities, it
is more prudent to focus resources on the ongoing lyophilized and aerosolized
surfactant development programs. Discovery Labs believes that the best way to
address the global RDS patient population is to advance KL4 surfactant
programs that are intended to gain worldwide commercial approvals and target
both traditional endotracheal tube delivery and novel, less-invasive surfactant
administration through aerosolization. Surfaxin LS, Discovery Labs’
initial lyophilized product, and Aerosurf, its initial aerosolized surfactant
product, may provide benefits that will greatly advance the management of RDS
and represent a significant improvement from a medical and economic
perspective.
With
respect to ongoing regulatory activities for Surfaxin, Discovery Labs has
submitted proposed corrections and additions to the formal FDA
minutes. Following finalization of the meeting minutes, Discovery
Labs will determine its next course of action, which may entail further
interaction with the FDA to assess whether Surfaxin approval can be gained
without additional clinical trials, or potentially exercising its right of
appeal through the FDA’s Formal Dispute Resolution process.
“We have
worked diligently to satisfy the requirements of the FDA but will now limit
investment to gain regulatory approval of Surfaxin in the United States. Our top
priority at this time is to leverage the totality of our Surfaxin experience and
established pharmacology of our KL4 surfactant
to advance our robust pipeline. I firmly believe that the robust nature of our
clinical experience and the tremendous progress that we have made in
manufacturing and quality operations meaningfully reduces development risk in
advancing our promising pipeline programs.
3
We are
pioneering some of the most exciting science in the development of new
surfactant applications to improve respiratory critical care medicine. We
believe that our proprietary technology platform makes it possible, for the
first time, to develop a significant pipeline of therapeutic surfactant products
targeted to treat a wide range of respiratory conditions. Our pipeline programs,
including Surfaxin LS and Aerosurf, are focused on addressing unmet medical
needs and represent significant market opportunities. Investing in this
pipeline, we believe, is the best strategic decision for our patients, medical
practitioners and other stakeholders,” commented Dr. Capetola.
Discovery
Labs will now focus its efforts primarily on the following lead pipeline
programs:
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·
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Respiratory
Distress Syndrome (RDS) -- one of the most common, acute, potentially
life-threatening disorders with more than 500,000 low birth weight
premature infants at risk annually globally. Discovery Labs
believes that the RDS market represents a significant opportunity from
both a medical and business
perspective.
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o
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Surfaxin
LS is a lyophilized formulation of KL4
surfactant, which is a dry powder that is reconstituted to a liquid
surfactant prior to administration. This formulation has the
potential to improve product flexibility and ease of use for healthcare
practitioners, eliminate the need for cold-chain storage and may
demonstrate characteristics that further improve product clinical
performance. Discovery Labs is planning to meet with regulatory
authorities this year with a view towards initiating a late-stage clinical
development program in 2010.
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o
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Aerosurf
is KL4
surfactant in aerosolized form using Discovery Labs’ proprietary Capillary
Aerosolization Technology. The current RDS management approach,
while life-saving, often results in serious respiratory conditions and
complications due to the invasive method of administration. Aerosurf, if
approved, holds the promise to significantly expand the use of KL4
surfactant by providing neonatologists with a novel means of delivering
KL4
surfactant without invasive endotracheal intubation and mechanical
ventilation. Discovery Labs has met with and received guidance
from the FDA with respect to the design of its planned Phase 2 clinical
program, which is expected to be initiated in
2010.
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·
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In
addition, Discovery Labs has initiated exploratory development programs
targeting Acute Respiratory Failure (ARF), Acute Lung Injury (ALI), Cystic
Fibrosis (CF), and the feasibility of drug combination therapies utilizing
KL4
surfactant:
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o
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ARF
/ ALI - Discovery Labs is conducting a Phase 2 ARF clinical trial to
determine whether Surfaxin improves lung function and reduces duration of
mechanical ventilation in children diagnosed with ARF following exposure
to a pathogen infection such as influenza. Additionally, Discovery
Labs has initiated a preclinical collaboration assessing the potential
utility of aerosolized KL4
surfactant in the prevention and treatment of Acute Lung Injury, a severe
form of respiratory failure.
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4
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o
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CF
- Aerosolized KL4
surfactant is being evaluated in an investigator-initiated Phase 2a
clinical trial in CF patients. The trial has been designed to assess the
safety, tolerability and short-term effectiveness (via improvement in
mucociliary clearance) of aerosolized KL4
surfactant in CF patients.
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o
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Drug
Combination Therapies - Discovery Labs has initiated a preclinical program
to assess the potential utility of KL4
surfactant either alone or in combination with the Capillary
Aerosolization Technology as a novel approach for direct lung delivery of
small and large molecule
therapeutics.
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Conference
Call Details
Discovery
Labs will hold a conference call on Thursday, July 2, 2009 at 8:30 AM EDT to
further discuss the foregoing. The call in number is
866-332-5218. The international call in number is
706-679-3237. This audio webcast will be available through a live
broadcast on the Internet at http://investor.shareholder.com/media/eventdetail.cfm?mediaid=37583&c=DSCO&mediakey=A42F707AF3652FE376D31BE1F11CF04A&e=0 and
www.discoverylabs.com. The
replay number to hear the conference call is 800-642-1687 or
706-645-9291. The passcode is 17638259.
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 or 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 deep 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. Discovery Labs is focused initially on developing its
KL4
surfactant pipeline to build a pediatric franchise that will potentially address
several respiratory conditions affecting neonates and young children. For more
information, please visit our website at www.Discoverylabs.com.
5
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 are: risks relating to
the rigorous regulatory requirements required for approval of any drug or
drug-device combination products that Discovery Labs may develop, including
that: (i) Discovery Labs and the U.S. Food and Drug Administration (FDA) will
not be able to agree on the matters raised by the FDA during its regulatory
review, or the FDA may require Discovery Labs to conduct significant additional
activities to potentially gain approval of its product candidates, if ever,
(ii) the FDA or other regulatory authorities may not accept or may withhold
or delay consideration of any of Discovery Labs’ applications, or may not
approve or may limit approval of Discovery Labs’ products to particular
indications or impose unanticipated label limitations, and (iii) changes in the national or
international political and regulatory environment may make it more difficult to
gain FDA or other regulatory approval; risks relating to Discovery Labs’
research and development activities, including (a) time-consuming and expensive
pre-clinical studies, clinical trials and other efforts, which may be subject to
potentially significant delays or regulatory holds, or fail, and (b) the need
for sophisticated and extensive analytical methodologies, including an
acceptable biological activity test, if required, as well as other quality
control release and stability tests to satisfy the requirements of the
regulatory authorities; risks relating to Discovery Labs’ ability to develop and
manufacture drug products and capillary aerosolization systems for clinical
studies, and, if approved, for commercialization of drug and combination
drug-device products, including risks of technology transfers to contract
manufacturers and problems or delays encountered by Discovery Labs, its contract
manufacturers or suppliers in manufacturing drug products, drug substances and
capillary aerosolization systems on a timely basis or in an amount sufficient to
support Discovery Labs’ development efforts and, if approved, commercialization;
risks that (a) market conditions, the competitive landscape or otherwise, may
make it difficult to launch and profitably sell products, (b) Discovery Labs may
be unable to identify potential strategic partners or collaborators to market
its products, if approved, in a timely manner, if at all, and (c) Discovery
Labs’ products will not gain market acceptance by physicians, patients,
healthcare payers and others in the medical community; the risk that Discovery
Labs or its strategic partners or collaborators will not be able to attract or
maintain qualified personnel; the risk that Discovery Labs will not be able in a
changing financial market to raise additional capital or enter into strategic
alliances or collaboration agreements, or that the ongoing credit crisis will
adversely affect the ability of Discovery Labs to fund its activities, or that
additional financings could result in substantial equity dilution; the risk that
Discovery Labs will not be able to access credit from its committed equity
financing facilities, or that the share price at which Discovery Labs may access
the facilities from time to time will not enable Discovery Labs to access the
full dollar amount potentially available under the facilities; the risk that
Discovery Labs will be unable to maintain The Nasdaq Global Market listing
requirements, causing the price of Discovery Labs’ common stock to decline; the
risk that recurring losses, negative cash flows and the inability to raise
additional capital could threaten Discovery Labs’ ability to continue as a going
concern; the risks that Discovery Labs may be unable to maintain and
protect the patents and licenses related to its products, or other companies may
develop competing therapies and/or technologies, or health care reform may
adversely affect Discovery Labs; risks of legal proceedings, including
securities actions and product liability claims; risks relating to reimbursement
and health care reform; and other risks and uncertainties 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:
Lisa
Caperelli, Investor Relations
215-488-9413
6