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SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C.
FORM 8-K
CURRENT REPORT PURSUANT
TO SECTION 13 OR 15 (D) OF THE
SECURITIES EXCHANGE ACT OF 1934
AUGUST 25, 1999
Date of report (Date of earliest event reported)
CELL PATHWAYS, INC.
(Exact Name of Registrant as Specified in Its Charter)
DELAWARE
(State or Other Jurisdiction of Incorporation)
0001066284 23-2969600
(Commission File Number) (IRS Employer Identification No.)
702 ELECTRONIC DRIVE
HORSHAM, PA 19044
(Address of Principal Executive Offices)
215-706-3800
(Registrant's Telephone Number, Including Area Code)
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ITEM 5. OTHER EVENTS
On August 25, 1999, Registrant submitted a New Drug Application (NDA)
to the U.S. Food and Drug Administration (FDA) for AptosynTM (exisulind),
Registrant's lead drug candidate. AptosynTM exisulind was formerly known as
Prevatac(TM) exisulind. The NDA seeks marketing approval of Aptosyn(TM)
exisulind for the treatment of adenomatous polyposis coli ("APC"), a rare
disease that puts those afflicted at high risk of developing colon cancer.
Aptosyn(TM) exisulind is an orally active drug that is designed to trigger cell
death by apoptosis in abnormal precancerous and cancerous cells without the
toxicities associated with conventional chemotherapeutic agents.
The NDA submission is based upon outcomes achieved in two pivotal
clinical trials in APC in which Aptosyn(TM) (exisulind) demonstrated the ability
to impede the progression of disease. In the patient group targeted by the
Company's Phase III trial which concluded in January 1999, Aptosyn demonstrated
a clinically and statistically significant reduction in new polyp formation when
compared to placebo. In a Phase I/II trial funded by the National Cancer
Institute, Aptosyn demonstrated a clinically and statistically significant dose
response at six months; and, over periods ranging from six months to thirty
months, this study and its extensions continued to demonstrate clinically and
statistically significant differences in the mean change in number of polyps
between dose groups. Regressing polyps showed substantial increases in the rate
of apoptosis, while the rate of apoptosis in nearby normal tissue was unchanged,
indicating the likelihood of selective induction of apoptosis in neoplastic
tissue without affecting normal cells.
In July 1998, the FDA designated Aptosyn(TM) (exisulind) a Fast Track
Product for which the FDA would take appropriate actions to expedite development
and review. The agency's determination of exisulind as a Fast Track product
signals its determination that APC is a serious and potentially life-threatening
condition for which there is currently an unmet medical need. Aptosyn also
received Orphan Drug Designation from the FDA in 1994 as a treatment for APC.
Acting pursuant to the Fast Track designation, Registrant submitted the chemical
section of the NDA to the FDA in November of 1998 and the
pharmacology/toxicology section in December of 1998. The clinical section
submitted on August 25, 1999 completes the NDA submission. Additional data
previously requested by the FDA will be submitted later this year. As is the
case with all NDA submissions, there can be no assurance that marketing approval
will be granted.
Further detail is set forth in Registrant's press releases dated August
26, 1999 and June 15, 1999, appended to this report as exhibits.
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ITEM 7. FINANCIAL STATEMENTS AND EXHIBITS
99.1 Press release of Registrant dated August 26, 1999.
99.2 Press release of Registrant dated June 15, 1999.
Certain statements made in this report, and oral statements made in respect
hereof, constitute "forward-looking statements" within the meaning of the
Private Securities Litigation Reform Act of 1995. Such statements are those
which express plan, anticipation, intent, contingency or future development
and/or otherwise are not statements of historical fact. These statements are
subject to risks and uncertainties, known and unknown, which could cause actual
results and developments to differ materially from those expressed or implied in
such statements. Such risks and uncertainties relate to, among other factors,
the absence of approved products; history of operating losses; early stage of
development; the costs, delays and uncertainties inherent in basic
pharmaceutical research, drug development, clinical trials and the regulatory
approval process, with respect to both the Registrant's current product
candidates and its future product candidates, if any; dependence on development
of exisulind; the limitations on, or absence of, the predictive value of data
obtained in laboratory tests, animal models and human clinical trials when
planning additional steps in product development; the uncertainty of obtaining
regulatory approval, including uncertainty as to FDA determinations in respect
of the evidence of safety and efficacy in the NDA submitted for exisulind for
APC; the timing and scope of any approval which might be received for any
compound for any indication in the future; acceptance by providers of healthcare
reimbursement; the validity, scope and enforceability of patents; the actions of
competitors; dependence upon third parties; product liability; the need for
further financing; and other risks detailed in Cell Pathways, Inc. reports filed
from time to time under the Securities Act of 1933 and/or the Securities
Exchange Act of 1934, including the sections entitled "Business" and "Risk
Factors" in the Registrant's report on Form 10-K for the year ended December 31,
1998. Given these uncertainties, current and prospective investors are cautioned
not to place undue reliance on any such forward-looking statements. Registrant
undertakes no obligation to update or revise the statements made herein or the
factors which may relate thereto.
SIGNATURE
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.
CELL PATHWAYS, INC.
Dated: September 28, 1999 By: /s/ Robert J. Towarnicki
-----------------------------------
Robert J. Towarnicki
President, Chief Executive Officer and
Director (Principal Executive Officer)
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Exhibit 99.1
CELL PATHWAYS SUBMITS NEW DRUG APPLICATION FOR APTOSYNTM(TM) (EXISULIND)
TO TREAT RARE PRE-CANCEROUS CONDITION
HORSHAM, PA (AUGUST 26, 1999): Cell Pathways, Inc. (Nasdaq: CLPA) today
announced the submission of a New Drug Application (NDA) to the U.S. Food and
Drug Administration (FDA) for Aptosyn(TM) (exisulind), the company's lead drug
candidate. (Aptosyn(TM) was formerly known as Prevatac(TM)). The company is
seeking marketing approval of exisulind for the treatment of adenomatous
polyposis coli ("APC"); a rare disease that puts those afflicted at high risk of
developing colon cancer. Exisulind is an orally active drug that is designed to
trigger cell death by apoptosis in abnormal precancerous and cancerous cells
without the toxicities associated with conventional chemotherapeutic agents.
"We are excited to announce the submission of our first NDA for
Aptosyn," said Robert J. Towarnicki, chief executive officer of Cell Pathways.
"This important milestone caps a significant five-year clinical effort on the
part of our employees, our clinical investigators and their patients."
CLINICAL TRIALS IN APC
Cell Pathways has been developing Aptosyn(TM) (exisulind) in human
clinical trials since the beginning of 1994. If approved by the FDA for
marketing, Aptosyn could provide the first non-surgical treatment option to
individuals with APC.
The NDA submission is based upon significant outcomes achieved in two
pivotal clinical trials in which Aptosyn(TM) (exisulind) impeded the progression
of the disease in APC patients. In the patient group targeted by the Company's
Phase III trial, Aptosyn demonstrated a clinically and statistically significant
reduction in new polyp formation when compared to placebo. In a Phase I/II trial
funded by the National Cancer Institute, Aptosyn demonstrated a clinically and
statistically significant dose response at six months; and, over periods ranging
from six months to thirty months, this study and its extensions continued to
demonstrate clinically and statistically significant differences in the mean
change in number of polyps between dose groups. Regressing polyps showed
substantial increases in the rate of apoptosis, while the rate of apoptosis in
nearby normal tissue was unchanged, confirming a selective induction of
apoptosis in neoplastic tissue without affecting normal cells. The Company plans
to submit additional data requested by the FDA later this year.
In July 1998, the FDA designated Aptosyn(TM) (exisulind) a Fast Track
Product for which the FDA would take appropriate actions to expedite development
and review. The agency awarded that designation because APC is a serious and
potentially life-threatening condition for which there is currently an unmet
medical need. Aptosyn also received Orphan Drug Designation from the FDA in 1994
as a treatment for APC.
APC is characterized by the development of hundreds to thousands of
adenomatous polyps in the colon that, if left untreated, will invariably
progress to colon cancer. Most APC patients have a substantial portion of their
large intestine removed by the age of 20, greatly altering their quality of
life. Those who retain colorectal tissue continue to develop adenomatous polyps,
are monitored by endoscopy two to four times each year, and have polyps removed
at many of these examinations.
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Cell Pathways decided to change the trademark to be used with the drug
to Aptosyn after meeting with the FDA, which suggested the change in name.
ON-GOING CLINICAL RESEARCH
Cell Pathways currently has two selective apoptotic antineoplastic
drugs ("SAANDs") in clinical trials. These compounds inhibit a cyclic GMP
phosphodiesterase and induce apoptosis (programmed cell death), but do not
produce the side effects normally associated with traditional chemotherapeutic
agents. SAANDs compounds also do not inhibit cyclooxygenase (COX) or produce the
gastric and renal toxicities reported to be associated with the COX inhibitors
and other NSAIDs. Aptosyn(TM) (exisulind) continues in trials involving
prostate, breast and lung cancers, Barrett's Esophagus and sporadic colonic
polyps. CP-461, a compound with higher apoptotic potency which will be targeted
for cancer indications, recently achieved high blood levels and good
tolerability in a Phase Ia trial. For additional information on Cell Pathways,
Inc., visit the Company's website at http://www.cellpathways.com.
Certain statements made herein, and oral statements made in respect hereof,
constitute "forward-looking statements" within the meaning of the Private
Securities Litigation Reform Act of 1995. Such statements are those which
express plan, anticipation, intent, contingency or future development and/or
otherwise are not statements of historical fact. These statements are subject to
risks and uncertainties, known and unknown, which could cause actual results and
developments to differ materially from those expressed or implied in such
statements. Such risks and uncertainties relate to, among other factors, the
absence of approved products; history of operating losses; early stage of
development; the costs, delays and uncertainties inherent in basic
pharmaceutical research, drug development, clinical trials and the regulatory
approval process, with respect to both the Company's current product candidates
and its future product candidates, if any; dependence on development of
exisulind; the limitations on, or absence of, the predictive value of data
obtained in laboratory tests, animal models and human clinical trials when
planning additional steps in product development; the uncertainty of obtaining
regulatory approval, including uncertainty of approval of the NDA submitted for
exisulind for APC; the timing and scope of any approval which might be received
for any compound for any indication in the future; acceptance by providers of
healthcare reimbursement; the validity, scope and enforceability of patents; the
actions of competitors; dependence upon third parties; product liability; the
need for further financing; and other risks detailed in Cell Pathways, Inc.
reports filed from time to time under the Securities Act of 1933 and/or the
Securities Exchange Act of 1934, including the sections entitled "Business" and
"Risk Factors" in the Company's report on Form 10-K for the year ended December
31, 1998. Given these uncertainties, current and prospective investors are
cautioned not to place undue reliance on any such forward-looking statements.
The Company undertakes no obligation to update or revise the statements made
herein or the factors which may relate thereto.
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Exhibit 99.2
CELL PATHWAYS COMPLETES ANALYSIS OF PHASE III APC TRIAL;
NDA FILING PLANNED FOR SUMMER
HORSHAM, PA (JUNE 15, 1999): Cell Pathways, Inc. (Nasdaq: CLPA) today
announced that it had concluded the evaluation of the Phase III trial of its
lead investigational drug, exisulind, in familial polyposis ("APC"). In the
patient group targeted by the study, i.e., those who form between approximately
10 and 40 polyps per year, exisulind demonstrated statistically significant
reduction in new polyp formation when compared to placebo. The Company plans to
file a New Drug Application ("NDA") with the Food and Drug Administration
("FDA") this summer and to amend that filing in the fourth quarter with
additional data that have been requested by the FDA. There can be no assurance
that the data submitted will be sufficient to warrant approval.
The recently concluded Phase III trial was a randomized, double-blind,
multi-center, placebo-controlled study conducted at seven centers in the United
States and at additional centers in Sweden, Israel and the United Kingdom.
Whereas the Phase I/II trial of exisulind in APC was an open-label regression
study which demonstrated the overall regression/prevention effect of exisulind,
the Phase III trial was a prevention trial designed to compare the cumulative
number of new polyps formed over twelve months by the drug and placebo groups.
Eligible patients were those who form approximately 10 to 40 polyps per year.
Patients were fully ablated (had all polyps removed) at the start of the study,
at the end of 6 months and at the end of 12 months. The study enrolled 73
patients and ended in January of 1999.
The Phase III study data revealed a higher degree of variability in
polyp formation by APC patients than previously thought by experts in the
disease. Reduction in new polyp formation was not statistically significant in
the sixty-five patients who completed the 12-month study. Detailed examination
indicated that only thirty-four of these patients met the study inclusion
criterion of forming approximately 10 to 40 polyps per year. In this target
group of thirty-four, the fifteen patients on exisulind showed a reduction in
new polyp formation of greater than 50% as compared with the nineteen patients
on placebo, with a `p' value of less than 0.05. A report of the study is planned
to be submitted to a peer review journal.
The Company has reviewed a summary of the results of the Phase III APC
trial with the FDA in a pre-NDA meeting. At the request of the FDA, in light of
the relatively low percentage of Phase III trial participants meeting inclusion
criteria, the Company will be assembling additional clinical data on exisulind
from other ongoing APC trials. The Company anticipates completing an NDA filing
during the summer and amending that filing with the requested additional
efficacy data in the fourth quarter of 1999. The Company cautions that neither
the filing of an NDA nor the submission of additional data provides assurance
that exisulind will be found to be of sufficient safety and efficacy to warrant
marketing approval for APC. Marketing approval, if ultimately granted, would
come only after thorough, detailed review by the FDA of all data, including the
data not yet submitted.
"We believe that the data from this polyp prevention trial complement
the data from the polyp regression trial," said Rifat Pamukcu, M.D., chief
scientific officer of the Company. "As we noted in our May 6 announcement with
respect to the interim results of the prostate study, we have now demonstrated
activity of our selective apoptotic antineoplastic drug (SAAND) technology in
two quite different human indications. We find these results encouraging as we
continue the clinical development of exisulind for both indications and the
broader program in other precancerous and cancerous indications to treat
neoplasia without adversely affecting normal tissue."
APC is a relatively rare inherited disease characterized by the
development of hundreds to thousands of adenomatous polyps in the colon and the
progression to colon cancer if left untreated. Most APC patients have a
substantial portion of their large intestine removed by age 20. Those who retain
rectal tissue continue to develop adenomatous polyps, are monitored by endoscopy
two to four times each year, and have polyps removed at many of these
examinations. In order to address this orphan drug population, Cell Pathways
commenced Phase I clinical trials of exisulind in February of 1994 and a Phase
I/II trial in 18 APC patients in August of 1995.
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Cell Pathways, Inc. is a development stage pharmaceutical company
focused on the development and commercialization of products to prevent and
treat cancer. Exisulind is the Company's lead compound. Exisulind and other Cell
Pathways SAAND compounds inhibit a cyclic GMP phosphodiesterase and induce
apoptosis (programmed cell death), but do not inhibit cyclooxygenase (COX) or
produce the gastric and renal toxicities reported to be associated with the COX
inhibitors and other NSAIDs. The Company has identified over 500 compounds, in
multiple chemical classes, many of which have shown in vitro significantly
greater apoptotic activity than exisulind. The selectivity of these compounds
for neoplastic cells is expected to yield potential agents to treat precancerous
and cancerous cells without the toxicities associated with conventional
chemotherapeutic agents. Cell Pathways recently commenced Phase I trials of
CP461, a compound with higher apoptotic potency, which will be targeted for use
in cancer indications. For additional information on Cell Pathways, Inc., visit
the Company's website at http://www.cellpathways.com.
Certain statements made herein, and oral statements made in respect hereof,
constitute "forward-looking statements" within the meaning of the Private
Securities Litigation Reform Act of 1995. Such statements are those which
express plan, anticipation, intent, contingency or future development and/or
otherwise are not statements of historical fact. These statements are subject to
risks and uncertainties, known and unknown, which could cause actual results and
developments to differ materially from those expressed or implied in such
statements. Such risks and uncertainties relate to, among other factors, the
absence of approved products; history of operating losses; early stage of
development; the costs, delays and uncertainties inherent in basic
pharmaceutical research, drug development and clinical trials, with respect to
both the Company's current product candidates and its future product candidates,
if any; dependence on development of exisulind; the limitations on, or absence
of, the predictive value of data obtained in laboratory tests, animal models and
human clinical trials when planning additional steps in product development; the
uncertainty of obtaining regulatory approval, including uncertainty in
connection with the adequacy of the data generated in the clinical trials of
exisulind for APC, and the timing and scope of any approval received; acceptance
by providers of healthcare reimbursement; the validity, scope and enforceability
of patents; the actions of competitors; dependence upon third parties; product
liability; the need for further financing; and other risks detailed in Cell
Pathways, Inc. reports filed from time to time under the Securities Act of 1933
and/or the Securities Exchange Act of 1934, including the sections entitled
"Business" and "Risk Factors" in the Company's report on Form 10-K for the year
ended December 31, 1998. Given these uncertainties, current and prospective
investors are cautioned not to place undue reliance on any such forward-looking
statements. The Company undertakes no obligation to update or revise the
statements made herein or the factors which may relate thereto.
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