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
Date of Report (Date of earliest event reported): September 16, 2004
ENZON PHARMACEUTICALS, INC.
(Exact name of registrant as specified in its charter)
Delaware 0-12957 22-2372868
(State or other jurisdiction of (Commission (IRS Employer
incorporation) File Number) Identification)
685 Route 202/206, Bridgewater, New Jersey 08807
(Address of principal executive offices) (Zip Code)
(Registrant's telephone number, including area code: (908) 541-8600
----------------------------------------------------------------------
(Former name or former address, if changed since last report)
Item 8.01 Other Events
Enzon Pharmaceuticals, Inc. (NASDAQ:ENZN) and Pharmagene plc (LSE: PGN)
announced today they have signed an agreement for the development of a
long-acting version of Pharmagene's drug candidate, PGN0052. PGN0052 is a
peptide hormone that is initially being investigated as a treatment for cystic
fibrosis and may have additional applications for other respiratory disorders.
PGN0052, a synthetic version of human secretin, has been successfully advanced
through three Phase 1 studies, and is currently being evaluated in a Phase 2a
proof of concept trial. Clinical trial results to date have demonstrated a
favorable safety and tolerability profile and importantly, PGN0052 has proven
effective in improving airway function following intravenous administration.
These results, together with Pharmagene's preclinical findings using human
tissue, demonstrate the potential of this drug candidate to treat cystic
fibrosis. Company researchers believe that extending the duration of action of
PGN0052 may provide greater overall effectiveness and improve its potential as a
therapeutic.
Under the agreement, Enzon will apply its proprietary PEGylation technology to
engineer a molecule with optimized pharmacokinetic properties and an extended
duration of action. Enzon will receive an initial fee upon signing and a
milestone payment upon the molecule meeting certain pre-determined criteria.
Subject to achievement of such criteria, the companies have the option to enter
into a worldwide joint development and commercialization agreement for the
product. Should the companies exercise this option, they would share equally in
development costs and profits and Pharmagene would be eligible to receive future
licensing and milestone payments, which could reach approximately (pound)26.5
million. Should the companies not elect to enter into a joint development and
commercialization agreement, Pharmagene will be entitled to use the PEGylated
molecule for development and commercialization purposes and Enzon will receive
certain co-marketing rights and royalties on worldwide sales for all
indications. While cystic fibrosis has been chosen as the lead indication for
PGN0052, the companies also believe there is potential for the treatment of
chronic obstructive pulmonary disease (COPD) and asthma.
In May 2004 at the American Thoracic Society conference, Pharmagene presented
data from Phase 1 studies in which PGN0052 was administered by either the
inhaled or intravenous route. The results demonstrated that in the intravenous
form, the drug was well tolerated and effective in producing measurable effects
on airway function. There is also supporting in vitro evidence of a relaxant
effect on bronchial smooth muscle. Based in part on these positive results,
Pharmagene has initiated a Phase 2a proof of concept study in cystic fibrosis
patients designed to assess the impact of the drug on mucociliary clearance (the
ability of the lining of the airways lungs to clear the protective mucus
secreted by the epithelial cells), using specific objective indicators. If the
study confirms the therapeutic principle, the development program will continue
with a version of PGN0052 with an optimized pharmacokinetic profile. Results
from the Phase 2a study are anticipated in the fourth quarter of calendar 2004.
About Cystic Fibrosis
Cystic fibrosis is the most common terminal genetic condition among Caucasians.
About 30,000 people in the United States and about 70,000 people worldwide have
cystic fibrosis. Cystic fibrosis patients' epithelial cells produce a defective
form of a protein called CFTR or Cystic Fibrosis Transmembrane Conductance
Regulator. The CFTR forms a channel that controls the movement of salt and water
into and out of cells. In cystic fibrosis, the protein is altered due to a
genetic mutation and, as a consequence, not functional in the cell membrane of
cells lining mucosal epithelial. Cystic fibrosis is characterized by a
disruption in the essential balance of salt and water that is needed to maintain
a normal thin coating of fluid and mucus inside the lungs, pancreas, and
passageways in other organs, causing the organs to function improperly. In the
lungs, the accumulation of thick mucus results in chronic lung complications,
such as wheezing, coughing, pneumonia or infections. In the pancreas, the thick
mucus can block the channels that would normally carry pancreatic enzymes to the
intestines to digest foods, resulting in the improper processing and digestion
of foods.
Death from cystic fibrosis is mainly due to pulmonary complications. The
condition is commonly treated with inhaled medications that aim to minimize
infection of the lung.
About Secretin
Secretin is a 27-amino acid polypeptide hormone (about half the size of insulin)
that promotes the secretion of electrolytes and water from epithelial cells.
Secretin also stimulates the release of pancreatic juice by the pancreas and
bile by the liver, both of which contain bicarbonate (a salt) and change the pH
of the duodenum (the first or proximal portion of small intestine) from acid to
alkaline, thereby facilitating the action of intestinal digestive enzymes.
About Pharmagene
Founded in 1996, Pharmagene is a drug discovery company whose aim is to identify
and develop novel treatments for human disease. To achieve this objective,
Pharmagene focuses solely on the use of fully consented human tissue for which
it has established, and maintains, one of the most effective tissue supply
networks in the world. By the provision of products and services that add value
throughout the drug discovery process, Pharmagene has built a solid reputation
and a growing customer base, including many of the major pharmaceutical
companies. Pharmagene is also actively undertaking internal research programs to
generate substantial intellectual property. Its lead therapeutic programs are in
cystic fibrosis, irritable bowel syndrome and migraine, all with novel
mechanisms of action elucidated by the company.
About Enzon
Enzon Pharmaceuticals is a biopharmaceutical company dedicated to the discovery,
development and commercialization of therapeutics to treat life-threatening
diseases. The Company has developed or acquired a number of marketed products,
including PEG-INTRON(R), marketed by Schering-Plough, and ABELCET(R),
ONCASPAR(R), ADAGEN(R), and DEPOCYT(R), marketed in North America by Enzon's
specialized sales force. Enzon's science-focused strategy includes an
extensive drug development program that leverages the Company's macromolecular
engineering technology platforms, including PEG modification and single-chain
antibody (SCA(R)) technologies. Internal research and development efforts are
complemented by strategic transactions that provide access to additional
products and technologies. Enzon has several drug candidates in various stages
of development, independently and with partners, including Marqibo(R), for which
a U.S. marketing application is currently being reviewed by the FDA for the
treatment of relapsed aggressive non-Hodgkin's lymphoma. Further information
about Enzon and this press release can be found on the Company's web site at
www.enzon.com.
There are forward-looking statements contained herein that are not based on
historical fact, including without limitation statements containing the words
"believes," "may," "plans," "will," "estimates," "continue," "anticipates,"
"intends," "expects," and similar expressions. Examples include statements above
(i) regarding the potential benefits of extending the duration of action of
PGN0052, (ii) anticipating the optimization of the pharmacokinetic properties of
PGN0052 through the application of PEG technology, (iii) regarding the potential
receipt of milestone and royalty payments, (iv) regarding the potential
formation of a joint development and commercialization agreement, (v)
contemplating the potential usefulness of PGN0052 in cystic fibrosis and other
indications and (vi) predicting the timing of Phase 2 study results. Such
forward-looking statements involve known and unknown risks, uncertainties and
other factors that may cause actual results, events or developments to be
materially different from the future results, events or developments discussed
above. Such factors include the risks that the application of PEG technology may
not improve the pharmacokinetic properties of PGN0052, that the Phase 2 study
may be delayed or that PGN0052 may not emerge successfully from it, that PGN0052
may not receive regulatory approval from the FDA, and that Pharmagene and/or
Enzon may not be able to successfully commercialize PGN0052 if it does receive
regulatory approval from the FDA, as well as those described in Enzon's Form
10-K and Forms 10-Q on file with the SEC, such as, Enzon's ability to sustain
profitability, and positive cash flow; market acceptance of and continuing
demand for Enzon's products; and the impact of competitive products and pricing.
These factors should be considered carefully and readers are cautioned not to
place undue reliance on such forward-looking statements. All information in this
press release is as of September 16, 2004 and the Company undertakes no duty to
update this information.
Item 9.01 Financial Statements and Exhibits
Exhibit 99.1 Press Release dated September 16, 2004
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.
Dated: September 16, 2004
By: /s/ Kenneth J. Zuerblis
---------------------------
Kenneth J. Zuerblis
Vice President, Finance and
Chief Financial Officer
Exhibit 99.1
[LEETERHEAD OF ENZON PHARMACEUTICALS, INC.]
For Immediate Release
================================================================================
PRESS RELEASE
Contacts:
Enzon Pharmaceuticals, Inc: Pharmagene plc:
Susan M. Mesco Alastair J. Riddell, Chief Executive Officer
Director, Investor Relations Ronald Openshaw, Chief Financial Officer
(908)541-8777 +44 (0)1763 211600
Euro RSCG Life NRP Financial Dynamics
Mark R. Vincent, Media Relations David Yates / Sophie Pender-Cudlip
(212)845-4239 +44(0)207 831 3113
ENZON AND PHARMAGENE ANNOUNCE AGREEMENT TO DEVELOP
PEGYLATED SECRETIN FOR CYSTIC FIBROSIS
Bridgewater, NJ and Royston, UK - September 16, 2004 - Enzon Pharmaceuticals,
Inc. (NASDAQ:ENZN) and Pharmagene plc (LSE: PGN) announced today they have
signed an agreement for the development of a long-acting version of Pharmagene's
drug candidate, PGN0052. PGN0052 is a peptide hormone that is initially being
investigated as a treatment for cystic fibrosis and may have additional
applications for other respiratory disorders.
PGN0052, a synthetic version of human secretin, has been successfully advanced
through three Phase 1 studies, and is currently being evaluated in a Phase 2a
proof of concept trial. Clinical trial results to date have demonstrated a
favorable safety and tolerability profile and importantly, PGN0052 has proven
effective in improving airway function following intravenous administration.
These results, together with Pharmagene's preclinical findings using human
tissue, demonstrate the potential of this drug candidate to treat cystic
fibrosis. Company researchers believe that extending the duration of action of
PGN0052 may provide greater overall effectiveness and improve its potential as a
therapeutic.
Under the agreement, Enzon will apply its proprietary PEGylation technology to
engineer a molecule with optimized pharmacokinetic properties and an extended
duration of action. Enzon will receive an initial fee upon signing and a
milestone payment upon the molecule meeting certain pre-determined criteria.
Subject to achievement of such criteria, the companies have the option to enter
into a worldwide joint development and commercialization agreement for the
product. Should the companies exercise this option, they would share equally in
development costs and profits and Pharmagene would be eligible to receive future
licensing and milestone payments, which could reach approximately (pound)26.5
million. Should the companies not elect to enter into a joint development and
commercialization agreement, Pharmagene will be entitled to use the PEGylated
molecule for development and commercialization purposes and Enzon will
-more-
Pharmagene Agreement/ Page 2
receive certain co-marketing rights and royalties on worldwide sales for all
indications. While cystic fibrosis has been chosen as the lead indication for
PGN0052, the companies also believe there is potential for the treatment of
chronic obstructive pulmonary disease (COPD) and asthma.
In May 2004 at the American Thoracic Society conference, Pharmagene presented
data from Phase 1 studies in which PGN0052 was administered by either the
inhaled or intravenous route. The results demonstrated that in the intravenous
form, the drug was well tolerated and effective in producing measurable effects
on airway function. There is also supporting in vitro evidence of a relaxant
effect on bronchial smooth muscle. Based in part on these positive results,
Pharmagene has initiated a Phase 2a proof of concept study in cystic fibrosis
patients designed to assess the impact of the drug on mucociliary clearance (the
ability of the lining of the airways lungs to clear the protective mucus
secreted by the epithelial cells), using specific objective indicators. If the
study confirms the therapeutic principle, the development program will continue
with a version of PGN0052 with an optimized pharmacokinetic profile. Results
from the Phase 2a study are anticipated in the fourth quarter of calendar 2004.
Uli Grau, Enzon's Chief Scientific Officer, said: "We have been impressed with
the data we have seen on PGN0052 and its potential for treating respiratory
disorders. We look forward to working with the team at Pharmagene to apply
Enzon's PEG expertise to engineer an optimized version of secretin for clinical
development."
Dr Alastair Riddell, Chief Executive Officer of Pharmagene, added: "With their
expertise in PEGylated reformulation and specialized sales and marketing
capabilities in North America, Enzon is an excellent partner for this product.
The agreement announced today will enable the rapid progression of the product's
development whilst at the same time maximizing its value for our shareholders."
About Cystic Fibrosis
Cystic fibrosis is the most common terminal genetic condition among Caucasians.
About 30,000 people in the United States and about 70,000 people worldwide have
cystic fibrosis. Cystic fibrosis patients' epithelial cells produce a defective
form of a protein called CFTR or Cystic Fibrosis Transmembrane Conductance
Regulator. The CFTR forms a channel that controls the movement of salt and water
into and out of cells. In cystic fibrosis, the protein is altered due to a
genetic mutation and, as a consequence, not functional in the cell membrane of
cells lining mucosal epithelial. Cystic fibrosis is characterized by a
disruption in the essential balance of salt and water that is needed to maintain
a normal thin coating of fluid and mucus inside the lungs, pancreas, and
passageways in other organs, causing the organs to function improperly. In the
lungs, the accumulation of thick mucus results in chronic lung complications,
such as wheezing, coughing, pneumonia or infections. In the pancreas, the thick
mucus can block the channels that would normally carry pancreatic enzymes to the
intestines to digest foods, resulting in the improper processing and digestion
of foods.
-more-
Pharmagene Agreement/ Page 3
Death from cystic fibrosis is mainly due to pulmonary complications. The
condition is commonly treated with inhaled medications that aim to minimize
infection of the lung.
About Secretin
Secretin is a 27-amino acid polypeptide hormone (about half the size of insulin)
that promotes the secretion of electrolytes and water from epithelial cells.
Secretin also stimulates the release of pancreatic juice by the pancreas and
bile by the liver, both of which contain bicarbonate (a salt) and change the pH
of the duodenum (the first or proximal portion of small intestine) from acid to
alkaline, thereby facilitating the action of intestinal digestive enzymes.
About Pharmagene
Founded in 1996, Pharmagene is a drug discovery company whose aim is to identify
and develop novel treatments for human disease. To achieve this objective,
Pharmagene focuses solely on the use of fully consented human tissue for which
it has established, and maintains, one of the most effective tissue supply
networks in the world. By the provision of products and services that add value
throughout the drug discovery process, Pharmagene has built a solid reputation
and a growing customer base, including many of the major pharmaceutical
companies. Pharmagene is also actively undertaking internal research programs to
generate substantial intellectual property. Its lead therapeutic programs are in
cystic fibrosis, irritable bowel syndrome and migraine, all with novel
mechanisms of action elucidated by the company.
About Enzon
Enzon Pharmaceuticals is a biopharmaceutical company dedicated to the discovery,
development and commercialization of therapeutics to treat life-threatening
diseases. The Company has developed or acquired a number of marketed products,
including PEG-INTRON(R), marketed by Schering-Plough, and ABELCET(R),
ONCASPAR(R), ADAGEN(R), and DEPOCYT(R), marketed in North America by Enzon's
specialized sales force. Enzon's science-focused strategy includes an extensive
drug development program that leverages the Company's macromolecular engineering
technology platforms, including PEG modification and single-chain antibody
(SCA(R)) technologies. Internal research and development efforts are
complemented by strategic transactions that provide access to additional
products and technologies. Enzon has several drug candidates in various stages
of development, independently and with partners, including Marqibo(R), for which
a U.S. marketing application is currently being reviewed by the FDA for the
treatment of relapsed aggressive non-Hodgkin's lymphoma. Further information
about Enzon and this press release can be found on the Company's web site at
www.enzon.com.
There are forward-looking statements contained herein that are not based on
historical fact, including without limitation statements containing the words
"believes," "may," "plans," "will," "estimates," "continue," "anticipates,"
"intends," "expects," and similar expressions. Examples include statements above
(i) regarding the potential benefits of extending the duration
-more-
Pharmagene Agreement/ Page 4
of action of PGN0052, (ii) anticipating the optimization of the pharmacokinetic
properties of PGN0052 through the application of PEG technology, (iii) regarding
the potential receipt of milestone and royalty payments, (iv) regarding the
potential formation of a joint development and commercialization agreement, (v)
contemplating the potential usefulness of PGN0052 in cystic fibrosis and other
indications and (vi) predicting the timing of Phase 2 study results. Such
forward-looking statements involve known and unknown risks, uncertainties and
other factors that may cause actual results, events or developments to be
materially different from the future results, events or developments discussed
above. Such factors include the risks that the application of PEG technology may
not improve the pharmacokinetic properties of PGN0052, that the Phase 2 study
may be delayed or that PGN0052 may not emerge successfully from it, that PGN0052
may not receive regulatory approval from the FDA, and that Pharmagene and/or
Enzon may not be able to successfully commercialize PGN0052 if it does receive
regulatory approval from the FDA, as well as those described in Enzon's Form
10-K and Forms 10-Q on file with the SEC, such as, Enzon's ability to sustain
profitability, and positive cash flow; market acceptance of and continuing
demand for Enzon's products; and the impact of competitive products and pricing.
These factors should be considered carefully and readers are cautioned not to
place undue reliance on such forward-looking statements. All information in this
press release is as of September 16, 2004 and the Company undertakes no duty to
update this information.
# # #