Exhibit 99.1
ARIAD Announces Global Clinical Development Plan
for Its Lead Oncology Product, AP23573;
Phase 1 and 2 Clinical Investigators to Participate in Analyst Call Today
CAMBRIDGE, Mass.--(BUSINESS WIRE)--June 15, 2004--ARIAD
Pharmaceuticals, Inc. (Nasdaq: ARIA) today announced, for the first
time, highlights of the comprehensive global clinical development plan
for its lead oncology product, AP23573, in patients with hematologic
malignancies (leukemias and lymphomas) and solid tumors (brain,
breast, endometrial and prostate cancers, as well as sarcomas).
Initial Phase 2 Clinical Trial: The Company expects to begin
enrollment of patients with relapsed or refractory hematologic cancers
in a multicenter Phase 2 clinical trial of AP23573 - as a single agent
- - by the end of this month at the University of Texas M. D. Anderson
Cancer Center in Houston, Texas. The principal investigator is Frank
J. Giles, M.D., Professor and Chief, Developmental Therapeutics. The
drug's efficacy will be assessed in well-defined disease-specific
categories of leukemias and lymphoid malignancies in which current
therapeutic options are limited or only palliative. Up to
approximately 200 patients will be entered into this study at
approximately four cancer centers.
Professor Giles commented, "There is a strong scientific rationale
for treating patients with hematologic cancers by inhibiting mTOR
activity. In our Phase 2 study of AP23573, we expect to determine the
most appropriate hematologic cancers to be treated with AP23573 and
pursued in subsequent clinical trials."
Phase 2 Trials in Patients with Solid Tumors: AP23573 will be
studied further as a single agent in patients with specific solid
tumors in a series of Phase 2 clinical trials conducted in the United
States and Europe - the first of which is expected to be initiated
during third quarter 2004. The initial focus will be patients with
advanced, relapsed and/or metastatic cancers in which therapeutic
options are limited and well-defined clinical end-points can be
reached quickly. Trials will incorporate use of multiple biomarkers of
mTOR pathway activity to guide selection of patients who are likely to
benefit most from AP23573 treatment. Pharmacodynamic markers will also
be used to monitor mTOR inhibition and the patients' responses to
treatment.
Phase 1b Clinical Trials: In parallel with Phase 2 trials, a
series of Phase 1b clinical trials is planned in the United States and
Europe in selected cancers aimed at further evaluating the anti-tumor
activity, safety, and pharmacokinetics of AP23573, as a single agent
and in combination with chemotherapeutic agents and other molecularly
targeted drugs. Evaluation of biomarkers and pharmacodynamic markers
will be integral to these trials.
The first such trial is planned to begin by the end of this month
with enrollment of patients with progressive or recurrent glioblastoma
multiforme in a study of AP23573 - as a single agent - at Duke
University Medical Center, Durham, North Carolina and at The Cleveland
Clinic, Cleveland, Ohio. The trial is designed to identify the optimal
dosing of AP23573 to achieve maximal activity within brain tumors. Up
to approximately 36 patients will be entered into this study at the
two brain tumor centers. The principal investigators are Henry S.
Friedman, M.D., James B. Powell, Jr. Professor of Neuro-Oncology, and
David A. Reardon, M.D., Associate Professor of Neuro-Oncology, both in
the Brain Tumor Center at Duke, and Michael A. Vogelbaum, M.D., Ph.D.,
Associate Professor of Neurosurgical Oncology in the Brain Tumor
Institute at Cleveland.
Overall Clinical Development Strategy: The indications and
clinical trials have been prioritized based on (1) unmet medical need,
(2) biological rationale for inhibiting mTOR and clinical results of
mTOR inhibition in particular cancers, (3) opportunities to obtain
fast-track designation by the U.S. Food and Drug Administration and
comparable expedited procedures established by European regulatory
authorities, (4) the scientific data supporting enriching patient
populations for study using novel biomarkers, and (5) potential for
competitive differential advantages.
Plans for Registration Trials: Based on the results of the initial
Phase 2 trials, the Company anticipates initiating well-controlled
registration trials aimed at providing the basis for expedited
regulatory review of AP23573 in specific clinical indications, which
will be determined over the coming year.
"Our Phase 1 clinical trials, recently reported at the ASCO
meeting, provide clear demonstration of the anti-tumor activity and
safety profile of AP23573, a well-defined dosage schedule and
pharmacokinetics, and the foundation for further clinical
development," said Harvey J. Berger, M.D., chairman and chief
executive officer of ARIAD. "Getting AP23573 to market expeditiously
in strategically selected indications is our highest priority."
Clinical Investigators Participating in Today's Analyst Conference
Call: Further details on the new clinical trials announced today and
the Phase 1 clinical data reported at the ASCO meeting will be
discussed during today's previously announced analyst conference call
at 11:00 am (ET). Professor Giles, principal investigator of the first
Phase 2 clinical trial, and Eric K. Rowinsky, M.D., principal
investigator of the Phase 1 daily dosing trial of AP23573 and Senior
Scientist and past Director, Clinical Research at the Institute for
Drug Development, Cancer Therapy and Research Center, San Antonio,
Texas, will join ARIAD senior management as participants in today's
conference call.
Update on Guidance Regarding Cash Used in Operations for 2004:
Based on anticipated patient accruals and the scope of the clinical
development plan for AP23573, the Company is projecting cash used in
operations for 2004 of $34 million, an increase of $5 million in
guidance. The Company ended first quarter 2004 with $102.3 million in
cash, cash equivalents and marketable securities.
Update on Partnering AP23573 for Use in Drug-delivery Stents: The
Company continues to negotiate with potential partners to develop and
commercialize drug-delivery stents incorporating AP23573. However, in
light of recent developments in the medical device arena, the Company
is reassessing the capabilities of potential partners to (1) develop
and optimize the metal stent platforms and polymer formulations
required for next-generation stents, (2) achieve substantial market
penetration due to growing domination of the drug-eluting stent market
by one company, and (3) provide meaningful economic value to ARIAD.
These considerations, among others, will determine the outcome of
these negotiations.
Background on AP23573: The small-molecule drug, AP23573, starves
cancer cells and shrinks tumors by inhibiting the critical
cell-signaling protein, mTOR, which regulates the response of tumor
cells to nutrients and growth factors, and controls tumor blood supply
and angiogenesis through effects on Vascular Endothelial Growth Factor
(VEGF).
ARIAD is engaged in the discovery and development of breakthrough
medicines to treat cancer by regulating cell signaling with small
molecules. The Company is developing a comprehensive approach to
patients with cancer that addresses the greatest medical need -
aggressive and advanced-stage cancers for which current treatments are
inadequate. ARIAD also has an exclusive license to pioneering
technology and patents related to certain NF-(kappa)B treatment
methods, and the discovery and development of drugs to regulate
NF-(kappa)B cell-signaling activity, which may be useful in treating
certain diseases.
Additional information about ARIAD can be found on the web at
http://www.ariad.com.
Some of the matters discussed herein are "forward-looking
statements" within the meaning of the Private Securities Litigation
Reform Act of 1995. Such statements are identified by the use of words
such as "anticipate," "estimate," "expect," "project," "intend,"
"plan," "believe," and other words and terms of similar meaning in
connection with any discussion of future operating or financial
performance. Such statements are based on management's current
expectations and are subject to certain factors, risks and
uncertainties that may cause actual results, outcome of events, timing
and performance to differ materially from those expressed or implied
by such forward-looking statements. These risks include, but are not
limited to, risks and uncertainties regarding the Company's ability to
conduct preclinical and clinical studies of its product candidates,
risks and uncertainties that clinical trial results at any phase of
development may be adverse or may not be predictive of future results
or lead to regulatory approval of any of the Company's product
candidates, and risks and uncertainties relating to regulatory
oversight, intellectual property claims, the timing, scope, cost and
outcome of legal proceedings, future capital needs, key employees,
dependence on the Company's collaborators and manufacturers, markets,
economic conditions, products, services, prices, reimbursement rates,
competition and other risks detailed in the Company's public filings
with the Securities and Exchange Commission, including ARIAD's Annual
Report on Form 10-K for the fiscal year ended December 31, 2003. The
information contained in this document is believed to be current as of
the date of original issue. The Company does not intend to update any
of the forward-looking statements after the date of this document to
conform these statements to actual results or to changes in the
Company's expectations, except as required by law.
CONTACT: ARIAD Pharmaceuticals, Inc.
Tom Pearson, 610-407-9260
or
Kathy Lawton, 617-621-2345