Full Press Release Details
Sarepta Therapeutics Announces Third Quarter 2017 Financial Results and Recent Corporate Developments
Third quarter 2017 EXONDYS 51 (eteplirsen) total net revenues of $46 million
Company raises annual 2017 revenue guidance to between $150 million and $155 million
CAMBRIDGE, Mass., October 25, 2017 (GLOBE NEWSWIRE) Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a commercial-stage biopharmaceutical company
focused on the discovery and development of precision genetic medicines to treat rare neuromuscular diseases, today reported financial results for the third quarter of 2017.
Driven by continued exceptional execution, physician adoption, and adherence, EXONDYS 51 achieved strong performance in the third quarter, said
Douglas Ingram, Sarepta s president and chief executive officer. For the third straight quarter, we have been in a position to raise our annual revenue guidance. We are proud that EXONDYS 51 is on track to be one of the most successful
ultra-rare disease launch in history, but we are prouder still that EXONDYS 51 performance and the advancement of our pipeline in the third quarter furthered our mission to improve the lives of children with Duchenne muscular dystrophy (DMD).
During the quarter, the Company announced results of the 4053-101 study for golodirsen, a phosphorodiamidate
morpholino oligomer (PMO) designed to treat exon 53 skipping amenable DMD patients. Golodirsen results were highly statistically significant on all three biological endpoints reported, RT-PCR, Western blot and
immunohistochemistry. Golodirsen exhibited a 100 percent response rate in all subjects as measured by RT-PCR, precisely excising exon 53 and creating in reading frame RNA transcript in all patients. The
full data set presented at the 2017 Annual Congress of the World Muscle Society by Dr. Francesco Muntoni, a lead investigator in the 4053-101 study, showed a 10.7 fold mean increase in dystrophin
production, along with the proper localization of the protein in the muscle.
Additionally, in the quarter the Company appointed Dr. Guriq Basi as chief scientific officer to facilitate
the rapid expansion of Sarepta s current and next-generation chemistry platforms, including the PMO and next-generation peptide-conjugated PPMO platforms.
We intend to move with a sense of urgency to translate our innovative science and impressive pipeline into potentially life-saving and life-enhancing
medicines, continued Mr. Ingram. As we look to the rest of the year and into 2018, we will focus on continued performance of EXONDYS 51 and our numerous near term milestones, including the anticipation of first patients dosed this
year in two of our gene therapy collaboration programs with Nationwide Children s Hospital, initiating our PPMO 51 trial in patients, obtaining the readout from our collaborative program for utrophin
up-regulation with Summit Therapeutics, preparing to meet with the FDA in early 2018 regarding the pathway for approval of golodirsen, and continuing preparation for a hearing with the European CHMP regarding
a European marketing authorization for eteplirsen.
For the third quarter of 2017, on a GAAP basis, Sarepta reported a net loss of $47.7 million, or $0.78 per share, compared to a net loss of
$56.7 million for the same period of 2016, or $1.18 per share. On a non-GAAP basis, the net loss for the third quarter of 2017 was $12.4 million, or $0.20 per share, compared to a net loss of
$45.9 million for the same period of 2016, or $0.95 per share. The decrease in net loss for the quarter was primarily driven by increased net revenues offset by increases in costs and expenses.
For the third quarter of 2017, the
Company recognized net revenues of $46.0 million which reflects sales from EXONDYS 51 in the U.S. No revenue was recognized for the same period of 2016.
Cost and Operating Expenses
$3.1 million in the third quarter of 2017, which relates to sales of EXONDYS 51 following its commercial launch in the U.S. There was no cost of sales recognized for the same period of 2016. Prior to the approval of EXONDYS 51, the Company
expensed such manufacturing and material costs as research and development expenses.
Research and development expenses were $34.2 million for the
third quarter of 2017, compared to $34.3 million for the same period of 2016, which is relatively consistent from period to period. The
change was primarily driven by lower manufacturing expenses due to the capitalization of inventory following the approval of EXONDYS 51, partially offset by increased patient enrollment in the
Company s ongoing clinical trials, a ramp up of preclinical studies for the Company s PPMO platform and other follow-on exons and slight increases in professional fees and compensation and other
personnel expenses. Non-GAAP research and development expenses were $32.4 million for the third quarter of 2017, compared to $30.9 million for the same period of 2016, an increase of
Selling, general and administrative expenses were $28.2 million for the third quarter of 2017, compared to $22.2 million for
the same period of 2016, an increase of $6.0 million, which was primarily driven by increases in professional services due to global commercial expansion and on-going litigation and compensation and other
personnel expenses. Non-GAAP selling, general and administrative expenses were $23.1 million for the third quarter of 2017, compared to $14.8 million for the same period of 2016, an increase of
As a result of the execution of the settlement and license agreements with BioMarin Pharmaceuticals (BioMarin) in July 2017, the
Company recorded $25.6 million in litigation and license charges. Additionally, the Company recognized an amortization of in-licensed rights of $0.8 million for the third quarter of 2017, primarily
due to the BioMarin transactions.
Cash, Cash Equivalents, Restricted Cash and Investments
The Company had $618.4 million in cash, cash equivalents, restricted cash and investments as of September 30, 2017 compared to $329.3 million as of
December 31, 2016, an increase of $289.1 million. The increase is primarily driven by the net proceeds from the Company s equity offering and term loan, proceeds from the sale of the Company s Priority Review Voucher (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.
Use of Non-GAAP Measures
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 to Present at the 22nd International Annual Congress of the World Muscle Society
Sarepta Therapeutics Appoints Guriqbal S. Basi, Ph.D. as Chief Scientific Officer
Sarepta Therapeutics to Ring Nasdaq Stock Market Closing Bell in
Recognition of World Duchenne Awareness Day
Sarepta Therapeutics Announces Positive Results in Its Study Evaluating Gene Expression, Dystrophin
Production, and Dystrophin Localization in Patients with Duchenne Muscular Dystrophy (DMD) Amenable to Skipping Exon 53 Treated with golodirsen (SRP-4053)
Sarepta Therapeutics Announces its Partner, Genethon, Published New Micro-Dystrophin Gene Therapy Data in Nature Communications
Sarepta Therapeutics Announces Pricing of $325 Million Public Offering of Common Stock
The Company will be hosting a conference
call at 4:30 p.m. Eastern Time, to discuss these financial results and provide a corporate update. The conference call may be accessed by dialing 844-534-7313 for
domestic callers and +1-574-990-1451 for international callers. The passcode for the call is 1598174. Please specify to the
operator that you would like to join the Sarepta Third Quarter 2017 Earnings Call . The conference call will be webcast live under the investor relations section of Sarepta s website at www.sarepta.com and will be archived there
following the call for 90 days. Please connect to Sarepta s website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary.
EXONDYS 51 uses Sarepta s
proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene. EXONDYS 51 is designed to bind to exon 51 of dystrophin pre-mRNA,
resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein. Data from clinical
studies of EXONDYS 51 in a
small number of DMD patients have demonstrated a consistent safety and tolerability profile. The pivotal trials were not designed to evaluate long-term safety and a clinical benefit of EXONDYS 51
has not been established.
Important Safety Information About EXONDYS 51
Adverse reactions in DMD patients (N=8) treated with EXONDYS 51 30 or 50 mg/kg/week by intravenous (IV) infusion with an incidence of at least 25% more
than placebo (N=4) (Study 1, 24 weeks) were (EXONDYS 51, placebo): balance disorder (38%, 0%), vomiting (38%, 0%) and contact dermatitis (25%, 0%). The most common adverse reactions were balance disorder and vomiting. Because of the small numbers of
patients, these represent crude frequencies that may not reflect the frequencies observed in practice. The 50 mg/kg once weekly dosing regimen of EXONDYS 51 is not recommended.
In the 88 patients who received 30 mg/kg/week of EXONDYS 51 for up to 208 weeks in clinical studies, the
following events were reported in 10% of patients and occurred more frequently than on the same dose in Study 1: vomiting, contusion, excoriation, arthralgia, rash, catheter site pain, and upper
respiratory tract infection.
There have been reports of transient erythema, facial flushing, and elevated temperature occurring on the day of EXONDYS 51
For further information, please see the full Prescribing Information.
About Sarepta Therapeutics
Sarepta Therapeutics is
a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicines to treat rare neuromuscular diseases. The Company is primarily focused on rapidly advancing the development of its potentially