Sarepta Therapeutics is a biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare, infectious, and other diseases.
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|Sarepta Therapeutics Announces Additional Long-Term Efficacy and Safety Data from Pivotal Phase IIb Program of Eteplirsen for Treatment of Duchenne Muscular Dystrophy|
-- Eteplirsen provided a statistically significant 6 minute walk test advantage of 151 meters at three years compared to an external control --
-- Fourth muscle biopsy results confirm increased dystrophin production in nearly all eteplirsen-treated patients and exon skipping in 100 percent of patients --
-- Eteplirsen safety profile remains consistent with prior results --
Eteplirsen, Sarepta’s lead drug candidate, is designed to target the underlying cause of DMD by enabling the production of a functional dystrophin protein in patients with mutations amenable to exon 51 skipping. Approximately 13 percent of people with DMD are estimated to have a mutation targeted by eteplirsen/exon 51 skipping.
“We are encouraged by the positive clinical outcomes, such as the
statistically significant difference in the 6MWT in eteplirsen-treated
patients compared to a control, especially since we see them accompanied
by data that continues to demonstrate exon skipping and dystrophin
production in most patients,” said
Results of Sarepta’s Phase IIb program were included in the New Drug
Application (NDA) that Sarepta submitted to the
New Long-Term Efficacy Data
New results from a fourth biopsy performed on 11 patients demonstrated that exon skipping occurred in 100 percent of patients after 180 weeks of treatment, confirming the mechanism of action of eteplirsen. In addition, biochemical evidence from three quantification methods, analysis of dystrophin positive fibers, dystrophin intensity and Western Blot testing, confirmed that dystrophin was present in most patients following eteplirsen treatment.
Fourth Biopsy Results
New Long-Term Safety Data
New results from Sarepta’s safety database, which includes approximately 100 patients exposed to eteplirsen, showed that the eteplirsen safety profile remained consistent with prior results. Common adverse drug reactions included flushing, erythema, and mild temperature elevation. No pulmonary embolisms, hospitalizations, injection site reactions or thrombocytopenia have been observed.
Webcast & Conference Call
Sarepta will provide a corporate update and report on recent data from
the Phase IIb study of eteplirsen for Duchenne muscular dystrophy via a
live webcast and conference call on October 1, 2015 at
The presentation will be webcast live under the investor relations section of Sarepta’s website at www.sarepta.com and will be archived there 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.
The conference call may be accessed by dialing 877-727-3245 for US domestic callers and 530-379-4673 for international callers. The passcode for the call is 48471076. Please specify to the operator that you would like to join the “Sarepta Corporate Update and Report on Recent Data.”
About the 6-Minute Walk Test (6MWT)
The 6MWT was developed as an integrated assessment of cardiac, respiratory, circulatory, and muscular capacity for use in clinical trials of various cardiac and pulmonary conditions. In recent years, the 6MWT has been adapted to evaluate functional capacity in neuromuscular diseases and has served as the basis for regulatory approval of a number of drugs for rare diseases, with mean changes in the 6MWT ranging from 28 to 44 meters. Additionally, published data from longitudinal natural history studies assessing dystrophinopathy, a disease continuum comprised of DMD and Becker muscular dystrophy, support the utility of the 6MWT as a clinically meaningful endpoint in DMD. These data show that boys with DMD experience a significant decline in walking ability compared to healthy boys over one year, suggesting that slowing the loss of walking ability is a major treatment goal.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is an X-linked rare degenerative neuromuscular disorder causing severe progressive muscle loss and premature death. One of the most common fatal genetic disorders, DMD affects approximately one in every 3,500 boys born worldwide. A devastating and incurable muscle-wasting disease, DMD is associated with specific errors in the gene that codes for dystrophin, a protein that plays a key structural role in muscle fiber function. Progressive muscle weakness in the lower limbs spreads to the arms, neck and other areas. Eventually, increasing difficulty in breathing due to respiratory muscle dysfunction requires ventilation support, and cardiac dysfunction can lead to heart failure. The condition is universally fatal, and death usually occurs before the age of 30.
Eteplirsen is designed to address the underlying cause of DMD by
enabling the production of a functional dystrophin protein. Eteplirsen
uses Sarepta's novel phosphorodiamidate morpholino oligomer (PMO)-based
chemistry and proprietary exon-skipping technology to skip exon 51 of
the dystrophin gene. This enables the repair of specific genetic
mutations that affect approximately 13 percent of people with DMD. By
skipping exon 51, eteplirsen may restore the gene's ability to make a
shorter, but still functional, form of dystrophin from messenger RNA
(mRNA). Promoting the synthesis of a truncated dystrophin protein is
intended to stabilize or significantly slow the disease process and
prolong and improve the quality of life for patients with DMD.
Eteplirsen has not been approved by the
Data from clinical studies of eteplirsen in DMD patients have demonstrated a broadly favorable safety and tolerability profile and restoration of dystrophin protein expression.
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. Known risk factors include,
among others: the results of our ongoing research and development
efforts and clinical trials for eteplirsen and our other product
candidates may not be positive or consistent with prior results or
demonstrate a safe treatment benefit there may be delays in Sarepta's
projected regulatory and development timelines relating to the
eteplirsen NDA and plans for commercializing eteplirsen and developing
Sarepta's other product candidates for various reasons including
possible limitations of Sarepta's financial and other resources; Sarepta
may not be able to successfully complete its planned commercialization
of eteplirsen or continue developing its product candidates as planned
for a variety of reasons including due to regulatory, court or agency
decisions, such as decisions by the USPTO with respect to patents that
cover Sarepta's product candidates, scale-up of manufacturing may not be
successful, and any or all of Sarepta's product candidates may
fail in development or may not receive required regulatory approvals for
commercialization (including potentially under an accelerated pathway);
and those risks identified under the heading “Risk Factors” in Sarepta's
2014 Annual Report on Form 10-K or and most recent Quarterly Report on
Form 10-Q for the quarter ended
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 website at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.
“Safe Harbor” Statement under the Private Securities Litigation Reform Act of 1995: The statements that are not historical facts contained in this release are forward–looking statements that involve risks and uncertainties, including, but not limited to, the results of research and development efforts, the results of preclinical and clinical testing, the effect of regulation by the FDA and other agencies, the impact of competitive products, product development, commercialization and technological difficulties, and other risks detailed in the company’s Securities and Exchange Commission filings.