UNITED STATES
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
November 25, 2003
Date of Report (Date of earliest event reported)
Discovery Laboratories, Inc.
(Exact name of Registrant as specified in its charter)
Delaware 000-26422 94-3171943
(State or other jurisdiction (Commission File Number) (IRS Employer
of incorporation) Identification Number)
350 Main Street, Suite 307
Doylestown, Pennsylvania 18901
(Address of principal executive offices)
(215) 340-4699
(Registrant's telephone number, including area code)
(Former name or former address, if changed since last report)
Item 5. Other Events and Regulation FD Disclosure
On November 25, 2003, the Company issued a press release to announce
positive primary endpoint results of its pivotal, multinational, landmark phase
3 superiority clinical trial of Surfaxin(R) for Respiratory Distress Syndrome
(RDS) in premature infants. This pivotal trial was designed as a controlled,
prophylaxis, event-driven, superiority trial to demonstrate the safety and
effectiveness of the Company's humanized lung surfactant, Surfaxin, over Exosurf
(R), an approved non-protein containing synthetic surfactant with a reference
arm to Survanta(R), the leading cow-derived surfactant used in the United
States. The trial's independent Data Safety Monitoring Board (DSMB) informed the
study's Steering Committee and the Company that the trial had achieved
statistical significance in its co-primary endpoints for Surfaxin versus
Exosurf. The results of this pivotal study, together with the successful results
of an earlier RDS Phase 3 supportive study for Surfaxin versus Curosurf, are
intended to form the basis for worldwide regulatory approval, including the
filing of a new drug application with the FDA.
Item 7. Financial Statements, Pro Forma Financial Statements and Exhibits
(c) Exhibits:
99.1 Press Release dated November 25, 2003
2
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.
By: /s/ Robert J. Capetola
--------------------------------
Name: Robert J. Capetola, Ph.D.
Title: President and Chief Executive
Officer
Date: November 25, 2003
3
Exhibit 99.1
Discovery Laboratories Announces Positive Phase 3 Top-Line Results
in Pivotal Trial of Surfaxin(R) for the Treatment of
Respiratory Distress Syndrome in Premature Infants
Significant advancement in promising respiratory pipeline of Surfactant
Replacement Therapies - Conference Call and Webcast Today at 11:00 a.m. EST
Doylestown, PA -- November 25, 2003 -- Discovery Laboratories, Inc. (Nasdaq:
DSCO), announced today positive primary endpoint results of its pivotal,
multinational, landmark phase 3 superiority clinical trial of Surfaxin(R) for
Respiratory Distress Syndrome (RDS) in premature infants. The trial's
independent Data Safety Monitoring Board (DSMB) informed the study's Steering
Committee and Discovery that the trial had achieved statistical significance in
its co-primary endpoints for Surfaxin versus Exosurf(R) (a non-protein
containing synthetic surfactant). Survanta(R), a cow-derived surfactant, served
as a reference arm in the trial. Discovery intends to use the results of this
pivotal study, together with the successful results of an earlier RDS Phase 3
supportive study, to form the basis for a new drug application (NDA) with the
United States Food and Drug Administration in the first quarter of 2004 and
regulatory applications for approval in the rest of the world.
The DSMB, comprised of leading neonatologists, statisticians and a bioethicist,
were responsible for monitoring the overall safety of the trial. The DSMB
informed Discovery that statistical significance was achieved in favor of
Surfaxin over Exosurf for the co-primary endpoints of RDS-related mortality
through 14 days of life and the incidence of RDS at 24 hours of life. The DSMB
also determined that the trial, originally estimated to enroll approximately
1500 patients, could be concluded at 1294 patients. The co-primary endpoint
outcomes are as follows:
- ----------------------------------------------------------------------------------------------------------------------
Co-Primary Endpoints Surfaxin Exosurf Survanta p-value
-------- ------- -------- Surfaxin vs. Exosurf
- ----------------------------------------------------------------------------------------------------------------------
RDS related mortality 25/527 (4.7%) 49/509 (9.6%) 27/258 (10.5%) 0.001
through day 14
- ----------------------------------------------------------------------------------------------------------------------
Infants with RDS at 24 207/527 (39%) 240/509 (47%) 86/258 (33%) 0.006
hours of life
- ----------------------------------------------------------------------------------------------------------------------
The DSMB noted no major safety issues during the enrollment phase of the trial.
Discovery intends to announce further data relating to key safety and secondary
outcome measures from this trial once such data becomes available, which is
anticipated to be in the first quarter of 2004.
"More than 2.7 million premature infants are born worldwide each year at risk
for serious complications or death from RDS. To treat this disease, doctors and
parents are currently limited to using surfactants that are derived from pig and
cow lung sources. These animal-derived therapies are clinically effective, but
have inherent drawbacks. Tragically, there are hundreds of thousands of
premature babies born in the world each year who need, but do not receive,
effective surfactant replacement therapy," said Dr. Fernando Moya, Richard W.
Mithoff Professor of Pediatrics and Director, Division of Neonatal-Perinatal
Medicine at The University of Texas Medical School at Houston, a leading
authority in neonatal medicine and the head of the trial's steering committee.
"For the first time in neonatal medicine, we have available in clinical trials,
an engineered surfactant that has the most essential attributes of natural human
lung surfactant, Surfaxin. These results are extremely encouraging for the
pediatric community and represent a significant opportunity to improve the
standard of care and provide treatment for RDS in premature infants around the
world," continued Dr. Moya.
"These are extremely exciting results and we are grateful for the dedicated
efforts of our clinical investigators worldwide," said Robert J. Capetola,
Ph.D., President and Chief Executive Officer of Discovery. "We now have used
Surfaxin to treat infants with RDS at leading medical facilities throughout
North America, Central and South America, as well as in Western and Eastern
Europe. As a result, Surfaxin has gained broad exposure throughout the medical
community. This announcement, along with our earlier positive Phase 3 RDS
supportive data, and the data from Part A of our ongoing Phase 2 Acute
Respiratory Distress Syndrome trial, adds to the series of positive results
demonstrated by the clinical trials of our surfactant technology. We look
forward to continuing our efforts to build one of the most innovative
respiratory pipelines available."
Christopher J. Schaber, Ph.D., Executive Vice President and Chief Operating
Officer commented, "Our immediate priority is to complete the analysis of all of
the study endpoints, assemble a high quality data package to support the filing
of an NDA, and submit the results of the trial for publication in a prominent
peer-reviewed journal. We are pleased with the current progress of our
manufacturing initiatives and will now increasingly focus on commercialization
activities for the potential launch of Surfaxin."
"The neonatal community would welcome an alternative to the currently approved
animal-based surfactants. Although these products represented a major advance in
neonatal medicine, a number of us have always been concerned about the potential
risks associated with the use of these cow- and pig-derived products," said
Steven M. Donn, M.D., Professor of Pediatrics and Director of Neonatal-Perinatal
Medicine at C.S. Mott Children's Hospital of the University of Michigan Health
System. "These data represent an important advancement in treating RDS in
infants with an improved, humanized surfactant that does not have the potential
to cause adverse immunological responses or transmit animal-borne diseases,
based on what we have seen so far," continued Dr. Donn.
About the Pivotal Phase 3 Trial and Discovery's Supportive Phase 3 Trial
The landmark, pivotal trial was designed as a multinational, multicenter,
randomized, masked, controlled, prophylaxis, event-driven, superiority trial to
demonstrate the safety and efficacy of Discovery's Surfaxin, which is engineered
to closely mimic the most essential attributes of human lung surfactant, over
Exosurf, an approved, non-protein containing synthetic surfactant. Survanta, the
leading cow-derived surfactant used in the United States, served as a reference
arm in the trial. In addition to the co-primary endpoints, the study was
designed to evaluate key safety parameters and secondary endpoints which are
significant complications of prematurity. These secondary endpoints and certain
safety data will remain blinded in accordance with the trial protocol through 36
weeks post conceptual age and long-term follow-up.
The co-primary endpoints and certain secondary endpoints were assessed by an
independent adjudication committee comprised of leading neonatologists and
pediatric radiologists. This committee provided a consistent and standardized
method for assessing primary efficacy data in the trial. As part of the overall
evaluation of trial safety, the DSMB also reviewed these adjudicated endpoints.
Discovery also recently announced positive results from its supportive RDS Phase
3 study that was designed as a non-inferiority trial comparing Surfaxin to
Curosurf(R), a pig-lung extract believed by many of the world's leading
neonatologists to be the best surfactant currently approved.
Surfaxin - an engineered surfactant with the potential to address RDS worldwide
Surfactants are substances that are produced naturally in the lungs and are
essential to the lungs' ability to absorb oxygen and to maintain proper airflow
through the respiratory system. Premature babies are born with a lack of natural
surfactant in their lungs. Without surfactant, the air sacs in the lungs
collapse and are unable to absorb sufficient oxygen resulting in RDS. The
current standard of care for treating these patients is Surfactant Replacement
Therapy using animal-derived surfactants. Animal-derived products are prepared
using a chemical extraction process from cow and pig lung washes or from the
mincing of these animal lungs. Because of the inherent limitations of
animal-derived products, the manufacture of large quantities of high quality
product can be problematic and their use is largely limited to North America and
Western Europe.
Discovery's Surfaxin is an engineered version of natural human lung surfactant
and contains a peptide, sinapultide, that is designed to closely mimic the
essential human lung surfactant protein B (SP-B). Surfaxin, unlike the animal
products, can be produced in virtually unlimited quantities, in consistent
pharmaceutical grade quality, and has no risk of potential transmission of
animal-associated diseases.
Note to Investors
Discovery will hold a conference call today at 11:00 AM EST. The call in number
is 800-665-0669. This audio webcast will be available to shareholders and
interested parties through a live broadcast on the Internet at
www.irconnect.com/primecast/dsco/387 and www.discoverylabs.com. It is
recommended that participants log onto one of these sites at least 15 minutes
prior to the call. The Internet broadcast will be available for up to 30 days
after the call at both website addresses.
About Discovery Laboratories
Discovery Laboratories, Inc. is a biopharmaceutical company developing its
proprietary surfactant technology as Surfactant Replacement Therapies for
respiratory diseases including Respiratory Distress Syndromes in infants and
adults, Acute Lung Injury, asthma, Chronic Obstructive Pulmonary Disease and
upper airway disorders. Surfaxin, Discovery's lead product, is in Phase 3 and
Phase 2 clinical trials for critical care patients with life-threatening
respiratory disorders where there are few or no approved therapies available.
Surfactants are compositions produced naturally in the lungs and essential for
breathing. Discovery's technology produces an engineered version of natural
human lung surfactant that is designed to closely mimic the essential properties
of human lung surfactant. Discovery believes that through its technology,
pulmonary surfactants have the potential, for the first time, to be developed
into a series of respiratory therapies for critical care and other hospitalized
patients.
More information about Discovery Laboratories is available on the Company's Web
site at www.discoverylabs.com.
The results of the study reported today have not been submitted to or reviewed
by any regulatory agency.
To the extent that statements in this press release 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,
events conditioned on stockholder or other approval, 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 release are subject to certain
risks and uncertainties that could cause actual results to differ materially
from the statements made. Among the factors which could affect the company's
actual results and could cause results to differ from those contained in the
forward-looking statements contained herein are the risk that financial
conditions may change, risks relating to the progress of the company's research
and development, the risk that the Company will not be able to raise additional
capital or enter into additional collaboration agreements (including strategic
alliances for our aerosol and Surfactant Replacement Therapies), risks relating
to the progress of the Company's research and development, risks relating to the
ability of the Company's third party contract manufacturers to provide the
Company with sufficient amounts of drug products for completion of any of the
Company's clinical studies, other risks relating to the lack of sufficient drug
product for completion of any of the Company's clinical studies, and risks
relating to the development of competing therapies and/or technologies by other
companies. Companies in the pharmaceutical and biotechnology industries have
suffered significant setbacks in advanced clinical trials, even after obtaining
promising earlier trial results. Data obtained from tests are susceptible to
varying interpretations which may delay, limit or prevent regulatory approval.
Those associated 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-KSB, 8-K, 10-Q and 10-QSB, and amendments thereto.
Company Contacts:
John G. Cooper, SVP, CFO
Kori Beer, IR & Communications
215-340-4699