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Rayaldee™ Meets Primary Endpoints in Both Pivotal Phase 3 Trials;
NDA Submission Planned for Q4 2014
Rayaldee Results to be Presented at American Society of
Nephrologists Meeting
Clinical Trial Start for Rayaldee as Adjunctive Cancer Therapy to
Begin in Q4 2014
European Marketing Commenced for 4Kscore™ Blood Test to Identify
Risk of Aggressive Prostate Cancer; Latin America Rollout Planned for
Fourth Quarter 2014
Rolapitant™ NDA Submitted by OPKO Licensee Tesaro and Accepted for
Review by FDA
Cash and Cash Equivalents of $118.3 Million Providing Adequate
Liquidity To Fund Development Programs
hGH-CTP Development Program Continues to Progress Rapidly
Long Acting Factor VII for Hemophilia and Long Acting Oxyntomodulin
for Obesity and Diabetes Clinical Trials to Begin During 2015
MIAMI--(BUSINESS WIRE)--
OPKO Health, Inc. (NYSE:OPK) , a multi-national biopharmaceutical
and diagnostics company, today reported operating and financial results
for its third quarter ended September 30, 2014.
Business Highlights
NDA Submission planned for Q4 2014: OPKO announced successful
top-line results from both of its pivotal Phase 3 trials with
Rayaldee. These trials were identical randomized, double-blind,
placebo-controlled, multi-site studies intended to establish the
safety and efficacy of Rayaldee as a new treatment for secondary
hyperparathyroidism (SHPT) in patients with stage 3 or 4 chronic
kidney disease (CKD) and vitamin D insufficiency. OPKO plans to submit
a New Drug Application (NDA) with the United States (U.S.) Food and
Drug Administration (FDA) in the fourth quarter of 2014.
Rayaldee Results to be Presented at Upcoming American Society of
Nephrologists Meeting: Rayaldee Phase 3 trial data will be
unveiled in a late-breaking clinical presentation entitled "Safety and
Efficacy of Modified-release Calcifediol for Secondary
Hyperparathyroidism in Patients with Stage 3 or 4 CKD and Vitamin D
Insufficiency " on November 15, 2014 during the American Society of
Nephrology meeting in Philadelphia, PA.
Clinical Trial Start for Rayaldee as Adjunctive Cancer Therapy
Anticipated For Q4 2014: OPKO intends to initiate a clinical trial
to evaluate Rayaldee as an adjunctive therapy for the prevention of
skeletal-related events (SREs) in breast and prostate cancer patients
with bone metastases undergoing anti-resorptive therapy during the
fourth quarter of 2014.
hGH-CTP Development Program Continues to Progress Rapidly: OPKO’s
hGH-CTP development programs for long acting growth hormone continue
to make significant progress during the first nine months of 2014.
Enrollment of our ongoing Phase 3 clinical trial in adults continues
to advance and interim data for our pediatric Phase 2 clinical trials
were presented at the European Society of Pediatric Endocrinology
Conference in September.
for Obesity and Diabetes Clinical Trials to Begin During 2015: OPKO’s
development programs for long acting Factor VII and long acting
oxyntomodulin continue to progress toward entering clinical trials
during 2015.
European Launch for the 4Kscore Test Commenced; Adoption of 4Kscore
Test Continues to Grow: During September, OPKO launched the
4Kscore Test in Europe through its wholly owned subsidiary, OPKO
Health Spain. Earlier this year, OPKO completed a validation study and
launched the 4Kscore Test in the U.S. through its CLIA accredited OPKO
Lab. OPKO also expects to launch the 4Kscore Test in Latin America
through its Chilean and Mexican subsidiaries in late 2014 and
elsewhere shortly thereafter. OPKO is working to obtain reimbursement
for the 4Kscore Test by payers in the U.S. and abroad and expects
adoption to rapidly increase once reimbursement is received. The
presentation, “The 4Kscore Test as a Predictor of High-Grade Prostate
Cancer on Biopsy,” was presented at meetings of the New England
Section and Western Section of the American Urological Association in
October 2014.
Rolapitant NDA Filing Submitted in September and Accepted for
Review by FDA in November: OPKO’s partner, TESARO, submitted a NDA
to the FDA for approval of oral rolapitant, an investigational
neurokinin-1 (NK-1) receptor antagonist in development for the
prevention of chemotherapy-induced nausea and vomiting (CINV). The NDA
is supported by data from four controlled studies covering a spectrum
of patients receiving chemotherapy that commonly causes nausea and
vomiting. The top-line results of three of the Phase 3 studies were
previously announced by TESARO and were presented in detail at the
American Society for Clinical Oncology (ASCO) annual meeting in June
2014. On November 5, 2014, Tesaro announced the FDA accepted its NDA
filing for rolapitant which triggered a milestone payment to OPKO
under its license agreement with TESARO.
“We made significant progress during the third quarter, successfully
completing our two Phase 3 trials for Rayaldee with positive efficacy
and safety results, and successfully launching our 4Kscore Test in
Europe,” said Phillip Frost, M.D., Chairman and CEO. “We continue to see
the 4Kscore Test being increasingly adopted by Urologists in the U.S.
and abroad and expect to launch it in Mexico and Chile in the coming
months,” Dr. Frost continued. “We also aggressively pursued our Factor
VII product for hemophilia and oxyntomodulin for overweight and obesity
and hope to begin clinical trials in mid-2015.”
Financial Highlights
At September 30, 2014, OPKO had cash and cash equivalents of $118.3
million providing OPKO with adequate liquidity to continue the
development of its product candidates. During the nine months ended
September 30, 2014, OPKO continued to increase its investment in
research and development programs, utilizing $65.6 million of cash from
operations. The principal use of cash from operations include
investments in clinical trials for Rayaldee and hGH-CTP, a clinical
validation study for the 4Kscore Test which was completed in March 2014,
and the continued development of OPKO’s Claros 1™ Analyzer point of care
platform system, as well as OPKO’s earlier stage development programs.
Pharmaceutical product revenue for the three months ended September 30,
2014 increased to $17.3 million compared to $16.6 million for the 2013
period. This increase was principally the result of increased revenue
from OPKO’s active pharmaceutical ingredient business at FineTech. Total
revenue for the three months ended September 30, 2014 was $19.8 million
compared to $20.6 million for the 2013 period. The decrease in total
revenue was the result of the 2013 period including non-recurring
revenue of $1.3 million related to OPKO’s transaction with Pharmsynthez,
partially offset by increased pharmaceutical product revenue.
Net loss for the three months ended September 30, 2014 was $48.7
million, compared to $60.0 million in the comparable period of 2013. As
a result of the successful achievement of the primary efficacy and
safety endpoints for the Rayaldee Phase 3 clinical trials, the valuation
for contingent consideration payable to the sellers of Cytochroma
increased significantly during the three months ended September 30, 2014
resulting in $17.7 million of increased contingent consideration
expense. Further, OPKO continued to increase its investment in research
and development activities during the three months ended September 30,
2014 related to its ongoing Phase 3 programs for Rayaldee and hGH-CTP.
As a result, OPKO’s spending on research and development increased $9.4
million to $20.5 million for the three months ended September 30, 2014
from $11.1 million for the three months ended September 30, 2013. These
increases were partially offset during the three months ended September
30, 2014 by a decrease in non-cash expense of $40.8 million from the
change in fair value of our derivative instruments, principally related
to our Senior 2033 Notes.
For the nine months ended September 30, 2014, pharmaceutical product
revenue increased approximately 15% to $58.5 million compared to $50.7
million for the 2013 period. The increase in pharmaceutical product
revenue was principally the result of increased revenue from FineTech,
OPKO Health Europe and OPKO Mexico. Total revenue for the nine months
ended September 30, 2014 was $65.6 million compared to $75.8 million for
the 2013 period. Total revenue for the nine months ended September 30,
2013 included non-cash, non-recurring revenue of $12.5 million and $3.2
million of non-recurring revenue related to OPKO’s transactions with RXi
and Pharmsynthez, respectively, which was partially offset by increased
product revenue.
Net loss for the nine months ended September 30, 2014 was $118.7 million
compared to $98.0 million for the first nine months of 2013. OPKO
continued to increase its investment in research and development
activities related to its Phase 3 programs for Rayaldee and hGH-CTP, as
well as incurred costs associated with the clinical validation study for
the 4Kscore, the Claros 1 Analyzer point of care diagnostic platform and
earlier stage development programs. As a result, OPKO’s investment in
research and development increased $27.2 million to $57.7 million for
the nine months ended September 30, 2014 from $30.6 million for the nine
months ended September 30, 2013. In addition, net loss for the nine
months ended September 30, 2014 included a non-recurring in-process
research and development expense of $10.1 million due to a write-off of
in-process research and development expense in connection with the
acquisition of Inspiro. Further, as a result of the successful
achievement of the primary efficacy and safety endpoints for Rayaldee,
the valuation of the contingent consideration payable to the sellers of
Cytochroma increased significantly during the period resulting in $19.0
million of increased contingent consideration expense. The nine month
period ended September 30, 2013 included $12.5 million of non-cash
income related to the RXi transaction and a $10.8 million gain realized
from the successful exit of a strategic investment. These reductions
were partially offset during the nine months ended September 30, 2014 by
a decrease in non-cash expense of $52.1 million from the change in fair
value of our derivative instruments, principally related to our Senior
2033 Notes of which we retired $70 million during June 2014.
About OPKO Health, Inc.
We are a multi-national biopharmaceutical and diagnostics company that
seeks to establish industry-leading positions in large and rapidly
growing medical markets by leveraging our discovery, development and
commercialization expertise and our novel and proprietary technologies.
This press release contains "forward-looking statements," as that
term is defined under the Private Securities Litigation Reform Act of
1995 (PSLRA), which statements may be identified by words such as
"expects," "plans," "projects," "will," "may," "anticipates,"
"believes," "should," "intends," "estimates," and other words of similar
meaning, including statements regarding expected financial performance,
continued revenue growth and our ability to build a profitable business,
whether we have sufficient liquidity to fund development of our product
candidates and operations, our product development effort and the
expected benefits of our products, including whether our ongoing and
future Phase 3 clinical trials will be completed on a timely basis or at
all and whether the data from any of our trials will support approval,
validation and/or reimbursement for our products, the expected timing
for launch of our products in development, including Rayaldee and
hGH-CTP, the expected timing of our clinical trials, enrollment in
clinical trials, and disclosure of results for the trials, our ability
to market and sell any of our products in development, including
Rayaldee, the 4Kscore, and hGH-CTP, our ability to launch sales of the
4Kscore Test in Latin America and through our other subsidiaries,
increased adoption rates for the 4Kscore by Urologists in the U.S. and
abroad, the timing for submission of a NDA by us for Rayaldee, whether
the 4Kscore will provide substantial benefits to patients and doctors by
informing them of the risk of a patient having a high-grade cancer and
clarify the decision making process, whether the 4Kscore will reduce
unnecessary biopsies, as well as other non-historical statements about
our expectations, beliefs or intentions regarding our business,
technologies and products, financial condition, strategies or prospects.
Many factors could cause our actual activities or results to differ
materially from the activities and results anticipated in
forward-looking statements. These factors include those described in our
Annual Reports on Form 10-K filed and to be filed with the Securities
and Exchange Commission and in our other filings with the Securities and
Exchange Commission, as well as the risks inherent in funding,
developing and obtaining regulatory approvals of new,
commercially-viable and competitive products and treatments, that
earlier clinical results of effectiveness and safety may not be
reproducible or indicative of future results, that the 4Kscore,
Rayaldee, Rolapitant, hGH-CTP, and/or any of our compounds or diagnostic
products under development may fail, may not achieve the expected
results or effectiveness and may not generate data that would support
the approval or marketing of products for the indications being studied
or for other indications, that currently available over-the-counter and
prescription products, as well as products under development by others,
may prove to be as or more effective than our products for the
indications being studied. In addition, forward-looking statements may
also be adversely affected by general market factors, competitive
product development, product availability, federal and state regulations
and legislation, the regulatory process for new products and
indications, manufacturing issues that may arise, patent positions and
litigation, among other factors. The forward-looking statements
contained in this press release speak only as of the date the statements
were made, and we do not undertake any obligation to update
forward-looking statements. We intend that all forward-looking
statements be subject to the safe-harbor provisions of the PSLRA.
OPKO Health, Inc. and Subsidiaries
Condensed Consolidated Balance Sheets
(unaudited)
(in millions)
As of
September 30,
December 31,
Cash and cash equivalents
$
118.3
$
185.8
Other current assets
44.8
56.9
Total Current Assets
163.1
242.7
In-process Research and Development and Goodwill
1,018.0
1,019.7
Other assets
114.1
129.1
Total Assets
$
1,295.2
$
1,391.5
Current liabilities
$
76.8
$
91.8
2033 Senior Notes, net
114.9
211.9
Other long-term liabilities
216.2
214.8
Total Liabilities
407.9
518.5
Equity
887.3
873.0
Total Liabilities and Equity
Condensed Consolidated Statements of Operations
(unaudited)
(in millions, except per share data)
For the three months ended
September 30,
Revenues
$
19.8
$
20.6
Costs and expenses
68.0
39.7
Operating loss
(48.2
)
(19.0
)
Other income and (expense), net
(1.5
)
(38.9
)
Loss before income taxes and investment losses
(49.7
)
(57.9
)
Benefit from (provision for) income taxes
(0.3
)
(1.3
)
Loss before investment losses
(50.0
)
(59.2
)
Loss from investments in investees
(0.0
)
(1.6
)
Net loss
(60.8
)
Less: Net loss attributable to non-controlling interests
(0.8
)
Preferred stock dividend
Net loss attributable to common shareholders
$
(48.7
(60.0
)
Basic and diluted loss per share
$
(0.11
(0.17
)
For the nine months ended
September 30,
65.6
$
75.8
Costs and expenses
179.0
119.6
Operating loss
(113.4
)
(43.8
(4.3
)
(46.0
(117.7
)
(89.8
(1.0
)
(2.3
(118.7
)
(92.1
(2.5
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