Full Press Release Details
Sarepta Therapeutics Announces Fourth Quarter 2017 and Full-Year 2017 Financial Results and Recent Corporate
Fourth quarter 2017 EXONDYS 51 (eteplirsen) total net revenues of
Cash and investment balance of $1.1 billion as of December 31, 2017
CAMBRIDGE, Mass., March 1, 2018 (GLOBE NEWSWIRE) Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a commercial-stage biopharmaceutical company focused
on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases, today reported financial results for the three and twelve months ended December 31, 2017.
In 2017, Sarepta advanced our ambitious vision to make a profound difference in the lives of those with Duchenne muscular dystrophy (DMD) and furthered
our position as an important global genetic medicine company in rare disease. We accelerated our industry-leading DMD pipeline composed of some 16 programs across our RNA-targeted, gene therapy and
gene-editing platforms; raised substantial resources to invest in our programs; and delivered on our commitment to execute a successful launch for EXONDYS 51, our first genetic medicine therapy for DMD, said Douglas Ingram, Sarepta s
president and chief executive officer.
Mr. Ingram continued, The exceptional 2017 performance of EXONDYS 51 and the advancement of our
multi-platform pipeline reflects our leadership position in DMD and our commitment to turning our vision into reality. As we track into 2018, our commitment to the DMD community is unwavering, exemplified by our continued focus on the launch of
EXONDYS 51, multiple important data read outs over the year, and our progress in bringing new therapies to the DMD community with a sense of urgency.
For the fourth quarter of 2017, on a
GAAP basis, Sarepta reported a net loss of $24.0 million, or $0.37 per share, compared to a net loss of $88.5 million for the same period of 2016, or $1.62 per share. On a non-GAAP
basis, the net loss for the fourth quarter of 2017 was $18.0 million, or $0.28 per share, compared to a net loss of $38.6 million for the same period of 2016, or $0.71 per share.
For the year ended December 31, 2017, on a GAAP basis, Sarepta reported a net loss of
$50.7 million, or $0.86 per share, compared to a net loss of $267.3 million for the same period of 2016, or $5.49 per share. On a non-GAAP basis, the net loss for 2017 was
$88.7 million, or $1.51 per share, compared to a net loss of $191.9 million for the same period of 2016, or $3.94 per share.
For the three and twelve months
ended December 31, 2017, the Company recorded net product revenues of $57.3 million and $154.6 million, respectively, which reflects sales from EXONDYS 51 compared to net revenues of $5.4 million for fourth quarter of 2016. The
Company did not achieve any product revenues for the first three quarters of 2016. The increase primarily reflects increasing demand for EXONDYS 51 in the U.S.
Cost and Operating Expenses
(excluding amortization of in-licensed rights)
For the three and twelve months ended
December 31, 2017, cost of sales (excluding amortization of in-licensed rights) were $3.5 million and $7.4 million, respectively, compared to $0.1 million for the same periods
of 2016. The increase primarily reflects royalty payments to BioMarin Pharmaceuticals (BioMarin) as a result of the execution of the settlement and license agreements with BioMarin in July 2017 as well as higher inventory costs related to increasing
demand for EXONDYS 51 during 2017. Prior to the approval of EXONDYS 51, the Company expensed related manufacturing and material costs as research and development expenses.
Research and development
Research and development
expenses were $44.4 million for the fourth quarter of 2017, compared to $70.7 million for the same period of 2016, a decrease of $26.3 million. The decrease was primarily driven by an
up-front payment of $40.0 million to Summit (Oxford) Ltd. (Summit) in the fourth quarter of 2016 partially offset by increased patient enrollment in the Company s
on-going late stage clinical trials and a ramp up of preclinical studies for the Company s PPMO platform and other follow-on exons.
Non-GAAP research and development expenses were $41.8 million for the fourth quarter of 2017, compared to $27.8 million for the same period of 2016, an increase of $14.0 million. The increase
was primarily due to increased patient enrollment in the Company s on-going late-stage clinical trials and a ramp up of preclinical studies for the Company s PPMO platform and other follow-on exons.
Research and development expenses were $166.7 million for 2017, compared to
$188.3 million for the same period of 2016, a decrease of $21.6 million. The decrease was primarily driven by lower manufacturing expenses due to the capitalization of inventory following the approval of EXONDYS 51 and
$40.0 million and $7.0 million up-front payments, respectively, to Summit and University of Western Australia in 2016. The decreases were partially offset by a $22.0 million payment to
Summit in 2017 as a result of achieving the milestone of the last patient being dosed in the safety arm cohort to the PhaseOut DMD study, increased patient enrollment in the Company s ongoing late-stage clinical trials and a ramp up of
preclinical studies for the Company s PPMO platform and other follow-on exons and increases in professional fees and compensation and other personnel expenses.
Non-GAAP research and development expenses were $136.0 million for 2017, consistent with the same period of 2016.
Selling, general and administration
general and administrative expenses were $32.2 million for the fourth quarter of 2017, compared to $22.9 million for the same period of 2016, an increase of $9.3 million.
Non-GAAP selling, general and administrative expenses were $27.3 million for the fourth quarter of 2017, compared to $16.1 million for the same period of 2016, an increase of
$11.2 million. The year-over-year increases for both GAAP and non-GAAP selling, general and administrative expenses for the fourth quarter were primarily driven by increases in professional
services due to global expansion and compensation and other personnel expenses.
Selling, general and administrative expenses were $122.7 million for
2017, compared to $83.7 million for the same period of 2016, an increase of $39.0 million. Non-GAAP selling, general and administrative expenses were $97.9 million for 2017, compared to $60.7 million for
the same period of 2016, an increase of $37.2 million. The year-over-year increases for both GAAP and non-GAAP selling, general and administration expenses for the full year were primarily driven by
increases in professional services due to global expansion, legal expenses and compensation and other personnel expenses.
EXONDYS 51 litigation and
license charges and amortization of in-licensed rights
As a result of the execution of the settlement and
license agreements with BioMarin in July 2017, the Company recorded $28.4 million in litigation and license charges. Additionally, the Company recognized an amortization of in-licensed rights of
$1.1 million during 2017, primarily due to the BioMarin transactions.
Gain from sale of Priority Review Voucher
connection with the completion of the sale of the Priority Review Voucher (PRV) in March 2017, the Company recorded a gain of $125.0 million from sale of PRV for 2017.
Interest expense and other, net
twelve months ended December 31, 2017, the Company recorded $2.7 million and $2.0 million, interest expense and other, net, respectively, compared to less than $0.1 million and $0.5 million,
respectively, for the same periods of 2016. The year-over-year increases for both periods were primarily driven by accrued interest expense related to the convertible note that the Company issued in November 2017.
Cash, Cash Equivalents, Restricted Cash and Investments
The Company had $1.1 billion in cash, cash equivalents, restricted cash and investments as of December 31, 2017 compared to $329.3 million as of
December 31, 2016, an increase of $760.5 million. The increase is primarily driven by the net proceeds from the Company s equity and debt offerings, proceeds from the sale of the Company s PRV and collection of accounts
receivable related to EXONDYS 51 sales offset by up-front payments of $35.0 million related to the Company s license and settlement agreements with BioMarin and a milestone payment of
$22.0 million to Summit Therapeutics, and the use of cash to fund the Company s ongoing operations.
In addition to the GAAP financial measures set forth in this press release, the Company
has included certain non-GAAP measurements: non-GAAP research and development expenses, non-GAAP selling, general and
administrative expenses, non-GAAP other income adjustments, non-GAAP income tax expense, non-GAAP net loss, and non-GAAP basic and diluted net loss per share, which present operating results on a basis adjusted for stock-based compensation, restructuring expenses, and other items.
1. Stock-based compensation expenses
Stock-based compensation
expenses represent non-cash charges related to equity awards granted by Sarepta. Although these are recurring charges to operations, management believes the measurement of these amounts can vary substantially
from period-to-period and depend significantly on factors that are not a direct consequence of operating performance that is within management s control. Therefore,
management believes that excluding these charges facilitates comparisons of the Company s operational performance in different periods.
2. Restructuring expenses
Restructuring expenses have been excluded as the Company believes that adjusting for these items more closely represents the Company s ongoing operating
performance and financial results.
Management evaluates other items of expense and income on an individual basis. It takes into consideration quantitative and qualitative characteristics of each
item, including (a) nature, (b) whether the items relates to the Company s ongoing business operations, and (c) whether the Company expects the items to continue on a regular basis. These other items include the aforementioned gain
from the sale of the Company s PRV and associated income taxes, upfront license and milestone payments to Summit, EXONDYS 51 litigation and license charges and amortization of in-licensed rights.
The Company uses these non-GAAP measures as key performance measures for the purpose of evaluating operational
performance and cash requirements internally. The Company also believes these non-GAAP measures increase comparability of
period-to-period results and are useful to investors as they provide a similar basis for evaluating the Company s performance as is applied by management. These non-GAAP measures are not intended to be considered in isolation or to replace the presentation of the Company s financial results in accordance with GAAP. Use of the terms
non-GAAP research and development expenses, non-GAAP selling, general and administrative expenses, non-GAAP other income
adjustments, non-GAAP income tax expense, non-GAAP net loss, and non-GAAP basic and diluted net loss per share may differ from
similar measures reported by other companies, which may limit comparability, and are not based on any comprehensive set of accounting rules or principles. All relevant non-GAAP measures are reconciled from
their respective GAAP measures in the attached table Reconciliation of GAAP to Non-GAAP Net Loss.
Recent Corporate Developments
Therapeutics Pre-Announces Fourth Quarter 2017 Revenue and Provides Full-Year 2018 Revenue Guidance for EXONDYS 51 (eteplirsen), Representing
Approximately 100 Percent Year-over-Year Growth
Sarepta Therapeutics Announces Publication of Long-Term Pulmonary Function of Eteplirsen-Treated Patients
Compared to Natural History of Duchenne Muscular Dystrophy in The Journal of Neuromuscular Diseases
Sarepta Therapeutics Elects Biopharmaceutical
Veteran, Michael W. Bonney, to its Board of Directors
Sarepta Therapeutics Announces Exercise of Initial Purchasers Option to Purchase
Additional Convertible Senior Notes Due 2024
Sarepta Therapeutics Prices $475 Million of Convertible Senior Notes Due 2024
Sarepta Therapeutics Announces Proposed Offering of $375 Million of Convertible Senior Notes Due 2024
Sarepta Therapeutics Announces FDA Clearance of IND for the Company s PPMO Exon 51 Candidate, SRP-5051
Sarepta Therapeutics and Nationwide Children s Hospital Announce FDA Clearance of IND for Micro-Dystrophin Gene Therapy Program for the
Treatment of Duchenne Muscular Dystrophy
Sarepta Therapeutics and Nationwide Children s Hospital Announce U.S. Food and Drug Administration