Discovery
Labs Provides Business Update and Reports Second Quarter 2010 Financial
Results
Warrington, PA — August 4, 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
reports financial results for the second quarter ended June 30, 2010. The
Company will host a conference call this morning at 8:45 AM
EDT. The call-in
number is 866-332-5218.
Highlights
and upcoming milestones, discussed further below, include:
·
|
Surfaxin® –
Discovery Labs continues to advance its program to gain regulatory
approval for Surfaxin for the prevention of respiratory distress syndrome
(RDS) in premature infants. Discovery has taken into account
recently-received guidance from the U.S. FDA in the conduct of its ongoing
comprehensive preclinical program, continues to interact with the FDA, and
believes it remains on track for the potential filing of a complete
response in the first quarter of
2011.
|
·
|
Surfaxin
LS™ (next-generation, lyophilized formulation of Surfaxin) – Discovery
Labs is preparing the Surfaxin LS program for planned clinical
activities. Fourth quarter 2010 milestones include establishing
a commercial-scale Surfaxin LS manufacturing capability at a
cGMP-compliant contract manufacturer with expertise in lyophilized
formulations; producing necessary process validation
batches. Additionally, Discovery Labs is intending to seek
regulatory and scientific guidance with respect to its planned Surfaxin LS
development program, first with the FDA in the fourth quarter of 2010 and
then with the European Medicines Agency (EMA) in the first quarter of
2011.
|
·
|
Aerosurf®
(drug/device combination for noninvasive administration of aerosolized
KL4
surfactant to address neonatal RDS) – Discovery Labs and industry-leading
engineers are optimizing the design of the novel capillary aerosolization
device to potentially reduce development risk and satisfy the regulatory
and clinical requirements for Aerosurf. First half 2011
milestones include finalizing Aerosurf clinical device design, producing a
sufficient number of devices to conduct design verification testing, and
seeking regulatory guidance from the FDA and EMA for the planned Aerosurf
development program.
|
·
|
Phase
2a trial assessing safety and tolerability of aerosolized KL4
surfactant for cystic fibrosis – Top-line results anticipated to be
available in the third quarter of
2010.
|
·
|
Discovery
Labs ended the second quarter of 2010 with $23.3 million in cash and
marketable securities and a new Committed Equity Financing Facility which,
subject to certain conditions, could allow the Company to raise up to $35
million in additional capital. Second quarter 2010 cash burn
from operations (before financings and debt service) was $5.3
million. For the second half of 2010, Discovery Labs is
providing guidance of projected cash burn from operations (before
financings and debt service) of $12.0 million which includes investments
to advance the Surfaxin, Surfaxin LS and Aerosurf
programs.
|
W. Thomas
Amick, Chairman and interim Chief Executive Officer of Discovery Labs, commented
“Discovery’s ongoing
program to gain potential Surfaxin approval in 2011 continues to benefit from
the FDA’s direction and willingness to continue to interact with our
team. While remaining focused on Surfaxin, we are advancing our
Surfaxin LS and Aerosurf programs in a resource-effective way intended to reduce
development risk for these promising, high-value programs.
Discovery
Labs has made progress in securing additional capital while exploring potential
strategic alliances to adequately support our promising pipeline
programs. On the financial front, we have streamlined our operating
plans, significantly reduced debt and secured additional financial resources
intended to advance Surfaxin to approval and move Surfaxin LS and Aerosurf
towards clinical trials. Additionally, we are continuing discussions
with several potential strategic alliance partners that clearly recognize the
medical and commercial opportunity that Surfaxin, Surfaxin LS and Aerosurf may
represent as a valuable RDS franchise. We believe that success in
entering into meaningful strategic alliances will likely parallel success in
advancing Surfaxin towards a complete response and positioning Surfaxin LS and
Aerosurf for initiation of clinical trials.”
Although
a key priority for Discovery Labs is to secure strategic partners to support
ongoing research and development activities and future progress, there can be no
assurances that any strategic alliance will be successfully identified or
concluded.
Selected Updates on KL4
Surfactant Pipeline Development
Surfaxin® for neonatal
RDS: 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). Discovery Labs is currently
conducting a comprehensive preclinical program that is intended to satisfy the
FDA in this respect. If approved, Surfaxin would be the first
synthetic, peptide-containing surfactant for commercial use in neonatal
medicine.
The
ongoing comprehensive preclinical program has involved the optimization and
revalidation of the BAT which is now being employed in a series of
prospectively-designed, side-by-side preclinical studies with the
well-established preterm lamb model of RDS. In June 2010, Discovery
Labs received written guidance from the FDA regarding the comprehensive
preclinical program that is consistent with its ongoing program. Also
in June 2010, Discovery Labs submitted data and analysis from its program to
optimize and revalidate the BAT.
In July
2010, Discovery Labs took the opportunity to further interact with the FDA via a
conference call regarding several important aspects of the ongoing comprehensive
preclinical program. The FDA’s comments support the activities
undertaken by Discovery Labs to optimize and revalidate the BAT. Also
during the meeting, the FDA requested that Discovery Labs provide additional
data and analysis 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 also obtained further FDA guidance regarding
certain aspects of the ongoing side-by-side preclinical studies involving the
BAT and preterm lamb model of RDS.
Discovery
Labs has taken into account the FDA’s guidance into ongoing activities,
including the planned submission of the additional BAT-related data and analysis
requested by the FDA. During the July teleconference, the FDA
reiterated its willingness to provide continued guidance on plans to gain
Surfaxin approval. Since then, Discovery Labs has continued
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. However, future 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 can provide the data and
analysis requested by the FDA and remain on track to submit a complete response
to the FDA in the first quarter of 2011, potentially leading to Surfaxin
approval that year.
Surfaxin LS™ for neonatal
RDS: Discovery Labs’ development strategy for Surfaxin LS is to
build upon the Surfaxin clinical experience to create a best-in-class surfactant
therapy with improved preparation and administration flexibility and the
potential to further improve clinical performance. Data presented at
the May 2010 Pediatric
Academic Societies Meeting indicate that Surfaxin LS,
when compared to currently marketed, animal-derived surfactant
products, favorably improved lung function and oxygenation while attenuating
lung inflammation in an animal model of RDS.
To
support plans for finalizing the intended development pathway for Surfaxin LS,
the Company is currently conducting a series of key chemistry, manufacturing and
control (CMC) activities. The Company has determined that clinical
and commercial manufacturing requirements for Surfaxin LS would be most
efficiently met by collaborating with a world-class contract manufacturing
organization (CMO) with a successful, multi-national regulatory track record and
established expertise in current good manufacturing practices (cGMP) and
lyophilized formulations. In that regard, Discovery Labs has
contracted with a leading pharmaceutical CMO to establish a cGMP-compliant
clinical and commercial Surfaxin LS manufacturing capability. The
production of required process validation batches is anticipated to be initiated
in the fourth quarter of 2010.
Additionally,
the Company intends to seek regulatory and scientific guidance with respect to
its planned development program, first with the FDA in the fourth quarter of
2010 and then with the EMA in the first quarter of 2011.
Aerosurf® for neonatal
RDS: Aerosurf is a novel drug/device combination therapy
incorporating Discovery Labs’ synthetic surfactant and unique capillary
aerosolization technology to allow early administration of aerosolized
surfactant to prevent 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.
Through
the conduct of the Aerosurf program, the Company’s aerosol engineers and
scientists have meaningfully improved the design of the capillary aerosolization
device and patient interface for neonatal application, employing a flexible
design strategy to support potential downstream applications of aerosolized
KL4surfactant
in other respiratory disorders. The Company has recently engaged a
leading global technology company with expertise in biomedical devices to assist
in incorporating design improvements into the capillary aerosolization
device. The design of the capillary aerosolization device is being
optimized to potentially reduce development risk and satisfy regulatory and
clinical requirements for Aerosurf. The Company anticipates
finalizing the clinical Aerosurf device design and producing a sufficient number
of Aerosurf devices to complete required design verification testing in the
first half of 2011.
Additionally,
the Company is preparing to engage the FDA and EMA in the first half of 2011 for
regulatory and scientific guidance with respect to the planned Aerosurf clinical
development program. In preparation, the Company is currently
conducting important CMC activities for Aerosurf, including dose-ranging
experiments conducted in a well-established animal model of RDS and
comprehensive aerosol characterization studies.
Phase 2a trial for cystic
fibrosis: Aerosolized KL4 surfactant
is being evaluated in an investigator-initiated Phase 2a clinical trial as a
single center, pilot study to evaluate the effects of aerosolized KL4 surfactant
in patients with mild to moderate cystic fibrosis (CF). The study is
designed to assess the safety, tolerability and potential effect on mucociliary
clearance from short term administration of aerosolized KL4
surfactant. Enrollment has been completed and top-line results are
expected to be available in the third quarter of 2010.
Surfaxin,
Surfaxin LS and Aerosurf are investigational products and are not approved by
the FDA or any other world health regulatory authority for use in
humans.
Selected Financial Results
for the Quarter Ended June 30, 2010
For the
quarter ended June 30, 2010, the Company reported a net loss of $6.3 million (or
$0.04 per share) on 160.4 million weighted average common shares outstanding
compared to a net loss of $7.9 million (or $0.07 per share) on 112.7 million
weighted average common shares outstanding for the same period in
2009. As of June 30, 2010, the Company had 194.1 million common
shares outstanding.
As of
June 30, 2010, the Company had cash and marketable securities of $23.3
million. Additionally in June, the Company secured a new Committed
Equity Financing Facility (CEFF) with Kingsbridge Capital Limited, a private
investment group, in which Kingsbridge has committed to provide up to $35
million of capital over a three-year period through the purchase of up to
approximately 31.6 million newly-issued shares of Discovery Labs’ common
stock. Under the terms of the CEFF agreement, Discovery Labs will be
able determine the exact timing and amount of any financings, subject to certain
conditions and limitations (including price and share
limitations). In connection with the CEFF, Discovery Labs issued a
five-year warrant to Kingsbridge to purchase up to 1,250,000 shares of common
stock at an exercise price of $0.4459 per share.
On June
22, 2010, the Company completed a public offering of common stock and warrants
resulting in gross proceeds of $10.0 million from the issuance of 35.7 million
shares of common stock, 17.9 million five-year warrants and 17.9 million
short-term (9-month) warrants. The shares and warrants were priced at
$0.28 per unit. The five-year warrants have an exercise price of
$0.40 per share and the short-term warrants have an exercise price of $0.28 per
share. Net proceeds from the offering, after underwriting discounts
and commissions and other fees and expenses, were $9.1 million. The
Company could realize up to an additional $5.0 million in proceeds by March 22,
2011, from the potential exercise of the short-term warrants.
Net cash
burn from ongoing operating activities (before financing and debt service
activities) for the second quarter of 2010 was $5.3 million. The
Company anticipates that its net cash burn for operating activities (before any
financing and debt service activities) for the second half of 2010 will be
approximately $12.0 million.
In April
2010, the Company restructured its $10.6 million loan with PharmaBio Development
Inc. (PharmaBio), the former strategic investment subsidiary of Quintiles
Transnational Corp. (Quintiles). The Company satisfied $6.6 million
of the loan in cash. As of June 30, 2010, $4.0 million remained as
the short-term outstanding balance of the loan. On July 30, 2010,
$2.0 of the loan was paid and the remaining balance of $2.0 million is due on
September 30, 2010. Additionally, contemporaneously with the
restructuring, PharmaBio purchased Discovery Labs common stock and warrants
resulting in net proceeds of $2.1 million to Discovery
Labs. Quintiles, PharmaBio and the Company have also agreed to
explore a long-term strategic collaboration for the development of Surfaxin LS
and Aerosurf, however, there can be no assurance that any such arrangements will
be achieved.
The
descriptions of the transactions referred to herein with respect to Kingsbridge,
the recently completed public offering and the PharmaBio loan restructuring and
the PharmaBio investment in Discovery Labs’ common stock are entirely qualified
by reference to the transaction documents, which are attached as exhibits to
each related Form 8-K filed by the Company with the Securities and Exchange
Commission (“SEC”). Readers are referred to, and encouraged to read
in their entirety, the Forms 8-K, including the exhibits attached
thereto, and the Company’s Quarterly Report on Form 10-Q for the
quarter ended June 30, 2010 to be filed with the SEC, which includes further
detail on the Company’s business plans and operations, financial condition and
results of operations.
Conference
Call Details
Discovery
Labs will hold a conference call on Wednesday, August 4, 2010 at 8:45 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://intercallus.stream57.com/DiscoveryLaboratories_080410 and
www.discoverylabs.com. The
replay number to hear the conference call is 800-642-1687 or
706-645-9291. The passcode is 89526041.
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 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.
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: (a) Discovery Labs and the U.S. Food and Drug Administration (FDA) or
other regulatory authorities will not be able to agree on the matters raised
during regulatory reviews, or Discovery Labs may be required to conduct
significant additional activities to potentially gain approval of its product
candidates, if ever, (b) 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 (c) 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 (i) 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 (ii)
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;
the risk that Discovery Labs may be unable to identify potential strategic
partners or collaborators to develop and commercialize its products, if
approved, in a timely manner, if at all; 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 (CEFFs), or that the minimum share
price at which Discovery Labs may access the CEFFs from time to time will
prevent Discovery Labs from accessing the full dollar amount potentially
available under the CEFFs; the risk that Discovery Labs or its strategic
partners or collaborators will not be able to retain, or attract, qualified
personnel; the risk that Discovery Labs will be unable to regain compliance with
The Nasdaq Capital Market listing requirements prior to the expiration of the
additional grace period currently in effect, which could cause 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 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:
John G.
Cooper, EVP and Chief Financial Officer
215-488-9300
Condensed Consolidated Statement of
Operations
(in
thousands, except per share data)
|
|
Three
Months Ended
|
|
|
Six
Months Ended
|
|
|
|
June
30,
|
|
|
June
30,
|
|
|
|
(unaudited)
|
|
|
(unaudited)
|
|
|
|
2010
|
|
|
2009
|
|
|
2010
|
|
|
2009
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Revenue
|
|
$ |
-- |
|
|
$ |
-- |
|
|
$ |
-- |
|
|
$ |
-- |
|
Operating expenses: (1)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Research
and development
|
|
|
4,363 |
|
|
|
5,052 |
|
|
|
8,496 |
|
|
|
10,659 |
|
General
and administrative
|
|
|
1,865 |
|
|
|
2,592 |
|
|
|
4,797 |
|
|
|
5,688 |
|
Total
expenses
|
|
|
6,228 |
|
|
|
7,644 |
|
|
|
13,293 |
|
|
|
16,347 |
|
Operating
loss
|
|
|
(6,228 |
) |
|
|
(7,644 |
) |
|
|
(13,293 |
) |
|
|
(16,347 |
) |
Other
income / (expense)
|
|
|
(84 |
) |
|
|
(264 |
) |
|
|
(307 |
) |
|
|
(561 |
) |
Net
loss
|
|
$ |
(6,312 |
) |
|
$ |
(7,908 |
) |
|
$ |
(13,600 |
) |
|
$ |
(16,908 |
) |
Net
loss per common share
|
|
$ |
(0.04 |
) |
|
$ |
(0.07 |
) |
|
$ |
(0.09 |
) |
|
$ |
(0.16 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Weighted
average number of common shares outstanding
|
|
|
160,425 |
|
|
|
112,712 |
|
|
|
149,133 |
|
|
|
107,433 |
|
(1) Expenses include a charge for
stock-based compensation in accordance with ASC Topic 718. For
the three and six months ended June 30, 2010, the charges associated with
stock-based compensation were $0.4 million ($0.1 million in R&D and $0.3
million in G&A) and $0.8 million ($0.3 million in R&D and $0.5 million
in G&A), respectively. For the three and six months ended June
30, 2009, the charges associated with stock-based compensation were $1.0 million
($0.3 million in R&D and $0.7 million in G&A) and $1.8 million ($0.4
million in R&D and $1.4 million in G&A), respectively.
Condensed
Consolidated Balance Sheets
(in
thousands)
|
|
June
30,
|
|
|
December
31,
|
|
|
|
2010
|
|
|
2009
|
|
ASSETS
|
|
(unaudited)
|
|
|
|
|
Current
Assets:
|
|
|
|
|
|
|
Cash
and marketable securities
|
|
$ |
23,320 |
|
|
$ |
15,741 |
|
Receivables,
prepaid expenses and other current assets
|
|
|
383 |
|
|
|
233 |
|
Total
Current Assets
|
|
|
23,703 |
|
|
|
15,974 |
|
Property
and equipment, net
|
|
|
4,116 |
|
|
|
4,668 |
|
Restricted
Cash
|
|
|
400 |
|
|
|
400 |
|
Other
assets
|
|
|
184 |
|
|
|
361 |
|
Total
Assets
|
|
$ |
28,403 |
|
|
$ |
21,403 |
|
|
|
|
|
|
|
|
|
|
LIABILITIES AND STOCKHOLDERS'
EQUITY
|
|
|
|
|
|
|
|
|
Current
Liabilities:
|
|
|
|
|
|
|
|
|
Accounts
payable
|
|
$ |
1,235 |
|
|
$ |
1,294 |
|
Accrued
expenses
|
|
|
3,766 |
|
|
|
3,446 |
|
Loan
payable, including accrued interest
|
|
|
4,000 |
|
|
|
10,461 |
|
Equipment
loan and other liabilities
|
|
|
331 |
|
|
|
597 |
|
Total
Current Liabilities
|
|
|
9,332 |
|
|
|
15,798 |
|
Long-Term
Liabilities:
|
|
|
|
|
|
|
|
|
Equipment
loan and other liabilities
|
|
|
1,022 |
|
|
|
1,118 |
|
Total
Liabilities
|
|
|
10,354 |
|
|
|
16,916 |
|
Stockholders'
Equity
|
|
|
18,049 |
|
|
|
4,487 |
|
Total
Liabilities and Stockholders' Equity
|
|
$ |
28,403 |
|
|
$ |
21,403 |
|