TKT Provides Update on Product Pipeline at JPMorgan Healthcare Conference
January 11 2005 - 4:01PM
PR Newswire (US)
TKT Provides Update on Product Pipeline at JPMorgan Healthcare
Conference -- Replagal 2004 Sales Expected to Slightly Exceed
Guidance Range -- SAN FRANCISCO, Jan. 11 /PRNewswire-FirstCall/ --
Transkaryotic Therapies, Inc. (NASDAQ:TKTX) will present today an
overview of its product pipeline and the outlook for 2005 at the
JPMorgan Health Care Conference in San Francisco, California.
Specifically, TKT will provide an update on expected Replagal(TM)
2004 sales, provide an update on the Dynepo(TM) program and
describe a new research program for the treatment of familial
hypercholesterolemia. "We had a solid year in 2004 across the
organization. Replagal sales growth, the start-up of Dynepo
manufacturing, and clinical progress in our I2S and GA-GCB programs
were significant accomplishments that give us a strong platform for
growth in 2005," said Michael J. Astrue, President and Chief
Executive Officer of TKT. "With several near-term commercial
opportunities and the progress of our research pipeline, we believe
we are building a sustainable business that will offer a steady
flow of important new products for rare diseases." TKT announced
today that based on a preliminary review of vial shipments, it
expects Replagal sales in Europe during the fourth quarter 2004 to
be higher than expected and that the company expects 2004 Replagal
sales to slightly exceed its 2004 guidance of $67 to $77 million.
TKT also announced preliminary guidance on 2005 Replagal sales of
$90 to $100 million and greater than $100 million in 2006. TKT will
release its consolidated 2004 financial results and provide 2005
guidance in February 2005. To prepare for the commercial
introduction of Dynepo in Europe, TKT has contracted with Lonza
Biologics to manufacture the bulk drug substance at Lonza's cGMP
production facility in Slough, England. Production of the
validation batches to support regulatory approval of the
manufacturing site in Europe began last month. TKT now intends to
file variations to the existing product license for Dynepo for its
manufacturing facilities in the fourth quarter of 2005, and expects
that this will allow for a market introduction in the first half of
2006. In addition, TKT will describe today a new research program
outside the protein replacement area. The company is conducting
studies in animals to demonstrate LDL-cholesterol (LDLc) lowering
effect of its recombinant LDL receptor-transferrin fusion protein
(LDLR/TF) as a potential treatment of familial hypercholesterolemia
to reduce elevated plasma LDLc levels in patients. These
proof-of-concept studies are expected to be completed next year.
Familial hypercholesterolemia is an inherited genetic disorder
caused by mutations in the low-density lipoprotein receptor (LDLR)
gene where a significant unmet medical need exists. Absent or
malfunctioning LDLR causes severe elevations in LDL cholesterol
levels. Familial hypercholesterolemia leads to premature thickening
of fatty material along artery walls and may eventually block the
arteries resulting in heart attacks and premature death. The
heterozygous variant of familial hypercholesterolemia affects
approximately 1 per 500 persons and current treatments fail for a
significant minority of these patients. In addition, TKT will
provide the following overview of anticipated milestones for 2005:
Introduce Replagal Into Additional Countries TKT expects to obtain
Japanese approval of Replagal in late 2005 and is working to secure
government reimbursement in Taiwan and Canada. Report Clinical
Findings Top-line results from the I2S pivotal study for Hunter
syndrome are expected to be reported in June 2005. The company also
expects to report during the first half of 2005 top-line results
from a Phase III clinical trial previously conducted by TKT's U.S.
partner, Sanofi Aventis, evaluating Dynepo as a treatment for
anemia associated with cancer chemotherapy. Sanofi-Aventis is
conducting the clinical data analyses. In addition, TKT expects to
report top-line results from the Phase I/II study evaluating GA-GCB
for the treatment of Gaucher disease in the second half of 2005.
File Regulatory Applications TKT expects that if the pivotal I2S
clinical trial results are positive, it will file for regulatory
approval in the U.S. and Europe in the second half of 2005. TKT
also intends to file variations to the existing product license for
Dynepo for its manufacturing facilities in the fourth quarter of
2005. In addition, if the clinical trial results of Dynepo as a
treatment for cancer- related anemia are positive, TKT intends to
file an application seeking to expand the Dynepo label in Europe.
Form New Partnership TKT is seeking to enter into a commercial
partnership for Dynepo. There has been interest from a variety of
companies and TKT's target is to close a deal in the first half of
2005. Select New Protein Replacement Programs to Enter Preclinical
Development The company is currently evaluating three undisclosed
protein replacement programs outside the lysosomal storage disease
area and intends to make decisions on whether to begin preclinical
development for these proteins in the second half of 2005. Webcast
A webcast of TKT's corporate presentation at the JPMorgan
Healthcare Conference may be accessed in the Investor Information
section of TKT's website at http://www.tktx.com/ at 4:30 p.m.
Pacific Time under the Events category. A replay of the
presentation will be available for approximately 30 days. About TKT
Transkaryotic Therapies, Inc. is a biopharmaceutical company
primarily focused on researching, developing and commercializing
treatments for rare diseases caused by protein deficiencies. Within
this focus, the company markets Replagal(TM), an enzyme replacement
therapy for Fabry disease, and is developing treatments for Hunter
syndrome and Gaucher disease. In addition to its focus on rare
diseases, TKT intends to commercialize Dynepo(TM), its Gene-
Activated(R) erythropoietin product for anemia related to kidney
disease, in the European Union. TKT was founded in 1988 and is
headquartered in Cambridge, Massachusetts, with additional
operations in Europe, Canada and South America. Additional
information about TKT is available on the company's website at
http://www.tktx.com/. Forward-Looking Statements This press release
contains forward-looking statements within the meaning of the
Private Securities Litigation Reform Act of 1995, including
statements containing the words "believes," "anticipates," "plans,"
"expects," "intends," "should," "could," "will," "may," and similar
expressions. There are a number of important factors that could
cause the company's actual results to differ materially from those
indicated by such forward-looking statements, including: whether
any of the company's products will achieve the commercial success
anticipated by the company; whether competing products will reduce
the market opportunity for such products; whether I2S will be safe
and effective as a treatment for Hunter syndrome; whether GA-GCB
will be safe and effective as a treatment for Gaucher disease;
whether LDLR/TF will be safe and effective as a treatment for
familial hypercholesterolemia; whether TKT will successfully
identify and develop new products suitable for commercialization;
whether TKT is able to successfully complete clinical trials of its
products; enrollment rates for clinical trials; whether the results
of clinical trials, including the results of the I2S and GA-GCB
trials referred to above, will be consistent with results of prior
clinical trials or will warrant submission of applications for
regulatory approval; whether future clinical trials will be
conducted on a timely basis; the ability of TKT to successfully
find a partner for Dynepo; the ability of TKT and its partners to
successfully complete development of and to commercialize its
products; the ability of TKT to manufacture sufficient quantities
of its products to satisfy both clinical trial requirements and
commercial demand, if approved; the timing of submissions to and
decisions by regulatory authorities in the United States, Europe,
Japan and other countries regarding clinical trials and marketing
and other applications for TKT's products; whether the FDA and
equivalent regulatory authorities will grant marketing approval for
the company's products on a timeline consistent with TKT's
expectations or at all; the availability and extent of coverage
from third party payors and the receipt of reimbursement approvals
for the company's products; results of patent or other litigation;
and other factors set forth under the caption "Certain Factors That
May Affect Future Results" in the company's Quarterly Report on
Form 10-Q for the quarter ended September 30, 2004, which is on
file with the Securities and Exchange Commission and which factors
are incorporated herein by reference. While the company may elect
to update forward-looking statements at some point in the future,
the company specifically disclaims any obligation to do so, even if
its expectations change. Gene-Activated(R) is a registered
trademark and Replagal(TM) is a trademark of Transkaryotic
Therapies, Inc. Dynepo(TM) is a trademark of Sanofi-Aventis SA. For
More Information Contact: Justine E. Koenigsberg Senior Director,
Corporate Communications (617) 349-0271 Daniella M. Lutz Corporate
Communications Manager (617) 349-0205 DATASOURCE: Transkaryotic
Therapies, Inc. CONTACT: Justine E. Koenigsberg, Senior Director,
Corporate Communications, +1-617-349-0271, or Daniella M. Lutz,
Corporate Communications Manager, +1-617-349-0205 both of
Transkaryotic Therapies, Inc. Web site: http://www.tktx.com/
Company News On-Call: http://www.prnewswire.com/comp/120657.html
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