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Sarepta Therapeutics Announces Third Quarter 2016 Financial Results and Recent Corporate Developments
10/27/16 7:01 AM EDT
"We are thrilled that the first patient has been infused with EXONDYS
51. We are pleased with the early stages of the launch and that multiple
insurance carriers are providing coverage for patients to have access to
EXONDYS 51. We plan on providing a corporate update at the 35th
Annual
"We believe our recent financing puts us in a strong financial position
to execute on both our internal and external clinical development
programs and global manufacturing development plans. We continue to
evaluate the sale of the priority review voucher as a potential source
of non-dilutive financing to help support these efforts and advance
potential therapies for patients with DMD,” said
Financial Results
For the third quarter of 2016, Sarepta reported a net loss of
Excluding
No revenue was recognized for the three months ended
Research and development expenses were
General and administrative expenses were
The Company had
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 general and administrative expenses, non-GAAP operating expense adjustments, 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 and restructuring 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 from non-GAAP research and development expenses, non-GAAP general and administrative expenses, non-GAAP net loss and non-GAAP net loss per share facilitates comparisons of the Company’s operational performance in different periods.
Restructuring related expenses have been excluded from non-GAAP research and development expenses, non-GAAP general and administrative expenses, non-GAAP net loss and non-GAAP net loss per share as the Company believes that the adjustments for these items represent more closely the sustainability of the Company’s operating performance and understanding of its financial results.
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 general and administrative expenses, non-GAAP operating expense adjustments, 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
Duchenne Muscular Dystrophy Program
--Sarepta Therapeutics Announces FDA Accelerated Approval of EXONDYS 51™ (eteplirsen) injection, an Exon Skipping Therapy to Treat Duchenne Muscular Dystrophy (DMD) Patients Amenable to Skipping Exon 51
--
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--Sarepta Therapeutics Announces First Patient Dosed in Phase III Clinical Trial of SRP-4045 and SRP-4053 for the Treatment of Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
--Sarepta Therapeutics Announces Favorable USPTO Decisions in Exon 51
and Exon 53 Composition of Matter Patent Interference Cases against
Corporate Updates
--Sarepta Therapeutics Announces Pricing of
Conference Call
The Company will be hosting a conference call at
About EXONDYS 51™
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
Adverse reactions observed in patients (N=8) treated with 30 or 50 mg/kg/wk of EXONDYS 51 with incidence ≥ 25% and higher than in the placebo group (N=4) (Study 1) were: balance disorder (38%), vomiting (38%) and contact dermatitis (25%). The most common adverse reactions were balance disorder and vomiting.
The following events were reported in ≥ 10% of patients treated with EXONDYS 51 for up to 208 weeks (N=88) and occurred more frequently than placebo in a controlled trial for 24 weeks (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 infusion.
For the full prescribing information please refer to U.S. Full Prescribing Information at www.EXONDYS51.com.
About Sarepta Therapeutics
Forward-Looking Statements
This press release contains "forward-looking statements" within the
meaning of the safe harbor provisions of the
These forward-looking statements involve risks and uncertainties, many
of which are beyond Sarepta’s control. Actual results could materially
differ from those stated or implied by these forward-looking statements
as a result of such risks and uncertainties. Known risk factors include
the following: the commercial launch in the US for EXONDYS 51 may not be
successful in part or at all for various reasons including the actual
market size and drug supply needed may not be consistent with the
company’s expectations, EXONDYS 51 may not be accepted by patients and
prescribed by physicians to the degree expected by the Company, there
may be a significant number of insurance carriers that delay coverage,
may not cover or significantly limit coverage of EXONDYS 51, our
manufacturing, sales, distribution and specialty pharmacy network may
not be efficient in getting EXONDYS 51 to the market and economic,
competitive, reimbursement and regulatory conditions could negatively
impact the commercial launch of EXONDYS 51; the confirmatory and other
studies for EXONDYS 51 may not yield data consistent with prior results
or demonstrate a benefit that supports continued or full regulatory
approval by the
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. For a detailed description of risks and
uncertainties Sarepta faces, you are encouraged to review the Company's
filings with the
Internet Posting of Information
We routinely post information that may be important to investors in the 'For Investors' section of our web site at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.
Sarepta Therapeutics, Inc. | |||||||||||||||||
Condensed Consolidated Statements of Operations | |||||||||||||||||
(in thousands, except per share amounts) | |||||||||||||||||
(unaudited) | |||||||||||||||||
Three months ended | Nine months ended | ||||||||||||||||
September 30, | September 30, | ||||||||||||||||
2016 | 2015 | 2016 | 2015 | ||||||||||||||
Revenues from grants and research contracts | $ | - | $ | - | $ | - | $ | - | |||||||||
Operating expenses: | |||||||||||||||||
Research and development | 34,349 | 36,673 | 117,523 | 105,018 | |||||||||||||
General and administrative | 22,184 | 15,090 | 60,812 | 50,714 | |||||||||||||
Operating loss | (56,533 | ) | (51,763 | ) | (178,335 | ) | (155,732 | ) | |||||||||
Other income (loss): | |||||||||||||||||
Interest (expense) income and other, net | (209 | ) | (176 | ) | (478 | ) | 383 | ||||||||||
Net loss | $ | (56,742 | ) | $ | (51,939 | ) | $ | (178,813 | ) | $ | (155,349 | ) | |||||
Net loss per share - basic and diluted | $ | (1.18 | ) | $ | (1.25 | ) | $ | (3.83 | ) | $ | (3.75 | ) | |||||
Shares used in per share calculation | |||||||||||||||||
basic and diluted | 48,254 | 41,565 | 46,709 | 41,416 | |||||||||||||
Sarepta Therapeutics, Inc. | |||||||||||||||||
Reconciliation of GAAP to Non-GAAP Net Loss | |||||||||||||||||
(in thousands, except per share amounts) | |||||||||||||||||
(unaudited) | |||||||||||||||||
Three months ended | Nine months ended | ||||||||||||||||
September 30, | September 30, | ||||||||||||||||
2016 | 2015 | 2016 | 2015 | ||||||||||||||
Net loss - GAAP | $ | (56,742 | ) | $ | (51,939 | ) | $ | (178,813 | ) | $ | (155,349 | ) | |||||
Research and development: | |||||||||||||||||
Stock-based compensation expense | 2,674 | 2,631 | 7,527 | 7,639 | |||||||||||||
Restructuring Expense | 770 | - | 1,783 | - | |||||||||||||
Total research and development non-GAAP adjustments 1 |
3,444 | 2,631 | 9,310 | 7,639 | |||||||||||||
General and administrative: | |||||||||||||||||
Stock-based compensation expense | 6,899 | 3,052 | 15,566 | 18,130 | |||||||||||||
Restructuring Expense | 494 | - | 640 | - | |||||||||||||
Total general and administrative non-GAAP adjustments 1 |
7,393 | 3,052 | 16,206 | 18,130 | |||||||||||||
Net loss - non-GAAP | $ | (45,905 | ) | $ | (46,256 | ) | $ | (153,297 | ) | $ | (129,580 | ) | |||||
Non-GAAP net loss per share - basic and diluted |
$ | (0.95 | ) | $ | (1.11 | ) | $ | (3.28 | ) | $ | (3.13 | ) | |||||
Shares used in per share calculations - basic and diluted |
48,254 | 41,565 | 46,709 | 41,416 | |||||||||||||
1 Non-GAAP operating expense adjustments are comprised of total general and administrative non-GAAP adjustments and total research and development non-GAAP adjustments. Total non-GAAP operating expense adjustments were $10,837 and $5,683 for the three months ended September 30, 2016 and 2015, respectively. Total non-GAAP operating expense adjustments were $25,516 and $25,769 for the nine months ended September 30, 2016 and 2015, respectively. |
Sarepta Therapeutics, Inc. | |||||||||
Balance Sheet Highlights | |||||||||
(in thousands) | |||||||||
(unaudited) | |||||||||
September 30, | December 31, | ||||||||
2016 | 2015 | ||||||||
Cash, cash equivalents and short-term investments | $ | 395,140 | $ | 192,491 | |||||
Restricted investments | 11,479 | 11,478 | |||||||
Total assets | 487,310 | 273,782 | |||||||
Total liabilities | 78,686 | 83,435 | |||||||
Total stockholders' equity | $ | 408,624 | $ | 190,347 | |||||
View source version on businesswire.com: http://www.businesswire.com/news/home/20161027005535/en/
Source:
Media and Investors:
Sarepta Therapeutics, Inc.
Ian Estepan,
617-274-4052
iestepan@sarepta.com
or
W2O
Group
Brian Reid, 212-257-6725
breid@w2ogroup.com
This section of our website may contain dated or archived information which should not be considered current and may no longer be accurate. For current information, you are encouraged to review our most recent official corporate documents on file with the U.S. Securities and Exchange Commission.