Full Press Release Details
Sarepta Investor and Media Contact:
Therapeutics Announces First Quarter
2014 Financial Results and Recent Corporate Developments
New Drug Application for eteplirsen planned for submission to FDA by year end;
Multiple eteplirsen clinical studies in broader population of DMD patients to begin dosing later this year;
Investigational New Drug applications for two additional DMD drug candidates targeting different genetic subpopulations to be submitted to
FDA in third quarter;
Well capitalized with $233.1 million in cash and other investments at quarter end, with an additional $94.5
million raised post-quarter end
CAMBRIDGE, MA, May 8, 2014 Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative
RNA-based therapeutics, today reported financial results for the three months ended March 31, 2014, and provided an update of recent corporate developments.
The path toward an NDA filing and a potential accelerated approval of eteplirsen has been laid out for us and we are busy preparing for the important
clinical and regulatory milestones toward achieving this goal, said Chris Garabedian, president and chief executive officer of Sarepta. We are also preparing to advance our broader DMD program beyond eteplirsen in the U.S., and will
begin the global clinical development program on our next exon-skipping drugs, as well as seek EMA guidance on requirements for a potential eteplirsen submission in the EU, later this year.
For the first quarter of 2014,
Sarepta reported a non-GAAP net loss of $20.7 million, or $0.55 per share, compared to a non-GAAP net loss of $13.0 million for the first quarter of 2013, or $0.41 per share. The incremental loss is primarily the result of an increase of $9.1
million in non-GAAP operating expenses due to corporate growth, offset by an increase of $1.6 million in contract revenue.
On a GAAP basis, the net loss
for the first quarter of 2014 was $28.3 million, or $0.75 per share (including $4.4 million of stock-based compensation and restructuring expenses), compared with a net loss of $42.1 million for the first quarter of 2013, or $1.32 per share
(including $2.1 million of stock-based compensation and restructuring
expenses). The decrease in net loss is primarily due to a decrease of $23.7 million in loss on change in warrant valuation and an increase of $1.6 million in contract revenue offset by an
increase of $11.3 million in operating expenses. The fluctuation in the fair value of the Company s outstanding warrant liability is primarily affected by the fluctuation of the Company s stock price during each financial reporting period.
Revenue for the first quarter of 2014 was $6.1 million, up from $4.5 million for the first quarter of 2013. The $1.6 million increase was primarily due
to the timing of activities in connection with the Marburg portion of the July 2010 U.S. government contract. Revenue from the Company s European Union SKIP-NMD agreement supporting development of an exon 53 skipping therapeutic and the
Company s Children s National Medical Center agreement also increased in the first quarter of 2014. These increases were partially offset by a decrease in revenue from the August 2012 intramuscular agreement with the U.S. government, which
was completed in the third quarter of 2013.
Non-GAAP research and development expenses were $19.0 million for the first quarter of 2014, compared to
$13.0 million for the first quarter of 2013, an increase of $6.0 million. GAAP research and development expenses were $20.9 million for the first quarter of 2014 (including $1.9 million of stock-based compensation and restructuring expenses),
compared to $13.8 million for the first quarter of 2013 (including $0.8 million of stock-based compensation and restructuring expenses), an increase of $7.1 million.
Non-GAAP general and administrative expenses were $7.8 million for the first quarter of 2014, compared to $4.8 million for the first quarter of 2013, an
increase of $3.0 million. GAAP general and administrative expenses were $10.3 million for the first quarter of 2014 (including $2.5 million of stock-based compensation expense), compared to $6.1 million for the first quarter of 2013 (including $1.3
million of stock-based compensation and restructuring expenses), an increase of $4.2 million.
The increased operating expenses were primarily caused by
corporate growth as the Company continues the development of its programs in Duchenne muscular dystrophy (DMD).
The Company had cash, cash equivalents,
short-term investments and restricted investments related to its letters of credit of $233.1 million as of March 31, 2014 compared to $264.9 million as of December 31, 2013, a decrease of $31.8 million. The decrease was primarily driven by
the use of cash to fund the Company s ongoing operations in the first quarter of 2014.
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 general and administrative expenses, non-GAAP operating
expenses, non-GAAP net loss, and non-GAAP basic and diluted net loss per share, which present operating results on a basis adjusted for certain items. The Company uses these non-GAAP measures as
key performance measures for the purpose of evaluating performance internally. The Company also believes these non-GAAP measures provide the Company s investors with useful information regarding the Company s historical operating results.
These non-GAAP measures are not intended 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 general and administrative expenses, non-GAAP
operating expenses, non-GAAP net loss, and non-GAAP basic and diluted net loss per share may differ from similar measures reported by other companies. 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
Duchenne Muscular Dystrophy Program
The conference call may be accessed by dialing 800-708-4540 for domestic callers and 847-619-6397 for international callers. The passcode for the call is
37204717. Please specify to the operator that you would like to join the Sarepta First Quarter 2014 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. An audio replay will be
available through May 22, 2014 by calling 888-843-7419 or 630-652-3042 and entering access code 37204717.
About Sarepta Therapeutics
Sarepta Therapeutics is focused on developing first-in-class RNA-based therapeutics to improve and save the lives of people affected by serious and
life-threatening rare and infectious diseases. Sarepta s diverse pipeline includes its lead program eteplirsen, for Duchenne muscular dystrophy, as well as potential treatments for some of the world s most lethal infectious diseases.
Sarepta aims to build a leading, independent biotech company dedicated to translating its RNA-based science into transformational therapeutics for patients who face significant unmet medical needs. For more information, please visit us at
Forward-Looking Statements and Information
In order to provide Sarepta s investors with an understanding of its current results and future prospects, this press release contains statements that
are forward-looking. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as believes, anticipates, plans,
expects, will, intends, potential, possible and similar expressions are intended to identify forward-looking statements. These forward-looking statements may include statements relating to
the company s future operations, financial performance, business plans and development of product candidates including; the timing and acceptance of an NDA submission for eteplirsen in the treatment of DMD; the timing of and the Company s
ability to conduct additional studies and submit additional data,
analysis and other information to the FDA necessary for the FDA to make regulatory determinations for eteplirsen and other follow-on exons; and the potential regulatory approval of eteplirsen on
an accelerated pathway; and our plans to advance our DMD program beyond the US. These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta s control. Actual results could materially differ from these
forward-looking statements as a result of such risks and uncertainties. Known risk factors include, among others: clinical trials may not demonstrate safety and efficacy of any of Sarepta s drug candidates and/or Sarepta s antisense-based
technology platform; scale-up of manufacturing may not be successful and any of Sarepta s drug candidates may fail in development or may not receive required regulatory approvals (including potentially under an accelerated pathway); Sarepta may
need additional funds to conduct research and development efforts; risks specific to obtaining FDA approval for eteplirsen including: we may not be able to comply with all FDA requests; the FDA may determine that our NDA submission for eteplirsen
does not qualify for filing, even with additional information; the results of our ongoing and new clinical trials may not be positive; there may be delays in our projected timelines relating to an NDA submission, initiating clinical trials, or
making a product commercially available; and those risks identified under the heading Risk Factors in Sarepta s Annual Report on Form 10-K for the fiscal year ended December 31, 2013 filed with the Securities and Exchange
Commission as well as other SEC filings made by Sarepta, which you are encouraged to review. Any of the foregoing risks could materially and adversely affect Sarepta s business, results of operations and the trading price of Sarepta s
common stock. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.
Sarepta Therapeutics, Inc.
(A Development-Stage Company)
Condensed Consolidated Statements of Operations and Comprehensive Loss
(in thousands, except per share amounts)
| Three Months Ended March 31, | ||||||||
| 2014 | 2013 | |||||||
| Revenues from grants and research contracts | $ | 6,088 | $ | 4,474 | ||||
| Operating expenses: | ||||||||
| Research and development | 20,906 | 13,762 | ||||||
| General and administrative | 10,303 | 6,127 | ||||||
| Operating loss | (25,121 | ) | (15,415 | ) | ||||
| Other non-operating income (loss): | ||||||||
| Interest income and other, net | 99 | 237 | ||||||
| Loss on change in warrant valuation | (3,251 | ) | (26,906 | ) | ||||
| Net loss | $ | (28,273 | ) | $ | (42,084 | ) | ||
| Net loss per share basic and diluted | $ | (0.75 | ) | $ | (1.32 | ) | ||
| Shares used in per share calculations basic and diluted | 37,821 | 31,813 |
Sarepta Therapeutics, Inc.
(A Development-Stage Company)
Reconciliation of GAAP to non-GAAP Net Loss
(in thousands, except per share amounts)
| Three Months Ended March 31, | ||||||||
| 2014 | 2013 | |||||||
| Net loss GAAP | $ | (28,273 | ) | $ | (42,084 | ) | ||
| Research and development: | ||||||||
| Stock-based compensation expense | 1,873 | 530 | ||||||
| Restructuring expense | 9 | 264 | ||||||
| Total research and development non-GAAP adjustments 1 | 1,882 | 794 | ||||||
| General and administrative: | ||||||||
| Stock-based compensation expense | 2,469 | 1,141 | ||||||
| Restructuring expense | 198 | |||||||
| Total general and administrative non-GAAP adjustments 1 | 2,469 | 1,339 | ||||||
| Other non-operating loss: | ||||||||
| Loss on change in warrant valuation non-GAAP adjustment | 3,251 | 26,906 | ||||||
| Net loss non-GAAP | $ | (20,671 | ) | $ | (13,045 | ) | ||
| Non-GAAP net loss per share basic and diluted | $ | (0.55 | ) | $ | (0.41 | ) | ||
| Shares used in per share calculations basic and diluted | 37,821 | 31,813 |
Sarepta Therapeutics, Inc.
(A Development-Stage Company)
Balance Sheet Highlights
| March 31, 2014 | December 31, 2013 | |||||||
| Cash, cash equivalents and short-term investments | $ | 225,192 | $ | 256,965 | ||||
| Restricted investments | 7,897 | 7,897 | ||||||
| Total assets | 275,389 | 291,569 | ||||||
| Total liabilities | 46,427 | 44,377 | ||||||
| Total stockholders equity | $ | 228,962 | $ | 247,192 |