SRPT Sarepta Therapeutics Inc.

90.32
+0.42  (+0%)
Previous Close 89.9
Open 89.95
52 Week Low 78.06
52 Week High 181.83
Market Cap $7,127,426,224
Shares 78,913,045
Float 66,749,649
Enterprise Value $6,080,021,224
Volume 3,386,192
Av. Daily Volume 1,887,561
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Upcoming Catalysts

Drug Stage Catalyst Date
Casimersen
Duchenne muscular dystrophy
PDUFA priority review
PDUFA priority review
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Drug Pipeline

Drug Stage Notes
SRP-9001 - Study 102
Duchenne muscular dystrophy
Phase 2
Phase 2
Phase 2 data released January 7, 2021. Primary biological endpoint met. Primary functional endpoint not met.
SRP-5051 (MOMENTUM)
Duchenne muscular dystrophy
Phase 1/2
Phase 1/2
Phase 2 interim data released December 7, 2020. At a total dose exposure approximately 10x lower than eteplirsen, SRP-5051 at 20 mgs/kg showed enhanced tissue exposure, greater exon skipping, and greater dystrophin production with no negative renal or other laboratory findings.
LYS-SAF302
Mucopolysaccharidosis type IIIA (MPS IIIA)
Phase 2/3
Phase 2/3
Phase 2/3 ongoing.
SRP-9003
Limb-girdle Muscular Dystrophy Type 2E (LGMD2E)
Phase 1/2
Phase 1/2
Phase 1/2 18-month functional results from Cohort 1 presented September 28, 2020.
rAAVrh74.MCK.GALGT2
Duchenne muscular dystrophy
Phase 1/2
Phase 1/2
Phase 1/2 ongoing.
MYO-102
Duchenne muscular dystrophy - LGMD2D
Phase 1/2
Phase 1/2
Phase 1/2 complete.
Golodirsen - Exon 53
Duchenne muscular dystrophy
Approved
Approved
FDA Approval announced December 12, 2019.
MYO-201
Duchenne muscular dystrophy - (LGMD2B)
Phase 1
Phase 1
Phase 1 trial ongoing.
Eteplirsen - Exon 51
Duchenne muscular dystrophy
Approved
Approved
Approved September 19, 2016.

Latest News

  1. -- Alliance will assess the use of Sarepta's proprietary gene editing technology and Genevant's proprietary LNP delivery platform for multiple neuromuscular targets --

    -- Sarepta to have options for an exclusive license to Genevant's LNP technology for four neuromuscular indications --

    -- Genevant may receive approximately $50 million in near-term payments and is also eligible for significant future milestones and royalties --

    CAMBRIDGE, Mass., VANCOUVER, British Columbia, and BASEL, Switzerland, Jan. 13, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, and Genevant Sciences, a leading nucleic acid delivery company with world-class platforms and the industry's…

    -- Alliance will assess the use of Sarepta's proprietary gene editing technology and Genevant's proprietary LNP delivery platform for multiple neuromuscular targets --

    -- Sarepta to have options for an exclusive license to Genevant's LNP technology for four neuromuscular indications --

    -- Genevant may receive approximately $50 million in near-term payments and is also eligible for significant future milestones and royalties --

    CAMBRIDGE, Mass., VANCOUVER, British Columbia, and BASEL, Switzerland, Jan. 13, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, and Genevant Sciences, a leading nucleic acid delivery company with world-class platforms and the industry's most robust and expansive lipid nanoparticle (LNP) patent estate, today announced a research collaboration and option agreement for the delivery of LNP-gene editing therapeutics in Sarepta's pipeline for neuromuscular diseases. LNPs offer the potential for a non-viral approach to gene editing and can provide both optimal uptake into desired cells and efficient release, resulting in functional delivery of gene editing cargo, such as CRISPR-Cas, to target tissues.

    Gene editing has the potential to revolutionize the treatment of diseases caused by genetic mutations - including rare neuromuscular diseases - by permanently altering genes that lead to disease. Sarepta is pursuing a variety of approaches to genetic medicine including exon skipping, gene therapies and gene editing in pursuit of cures for rare diseases.

    Under the terms of the agreement, Genevant will design and collaborate with Sarepta in the development of muscle targeted LNPs to be applied to gene editing targets in early stage development.  Sarepta will have rights to an exclusive license to Genevant's LNP technology for up to four neuromuscular indications, including Duchenne muscular dystrophy. Genevant may receive approximately $50 million in near-term payments and is also eligible for significant future development, regulatory and commercial milestones and tiered royalties ranging from the mid-single to low-double digits on future product sales.

    "As Sarepta works to advance precision genetic medicine across multiple modalities, we've invested in partnering and research efforts focused on improving the utility and benefit of gene-based medicines and providing the greatest possible outcome to patients. This includes advancing our pre-clinical gene editing program, looking at both viral and non-viral methods to produce a functional gene in order to treat a broad range of neuromuscular diseases," said Doug Ingram, president and chief executive officer, Sarepta Therapeutics. "Genevant's established leadership and proven LNP technology offers the potential to deliver gene editing machinery to targeted tissue through a non-viral delivery approach. Applying this science to neuromuscular diseases fits squarely within our mission to translate scientific breakthroughs into meaningful advances for patients whose lives have been impacted by rare disease."

    "Genevant scientists have been at the forefront of LNP delivery of nucleic acids for over 20 years. Our platform is the most clinically validated in the space and is the delivery technology behind the first nucleic acid-LNP product to have achieved FDA approval," said Pete Lutwyche, Ph.D., president and chief executive officer, Genevant Sciences Corporation. "Efficient, optimized delivery is often the difference between successful and unsuccessful nucleic acid drug development, and we are excited to bring our experience to Sarepta's gene editing programs in neuromuscular disease where new options – and new approaches – are desperately needed."

    About Genevant Sciences

    Genevant Sciences is a leading nucleic acid delivery company with world-class platforms, the industry's most robust and expansive lipid nanoparticle (LNP) patent estate, and decades of experience and expertise in nucleic acid drug delivery and development.  The Company's scientists have pioneered LNP delivery of nucleic acids for over 20 years, and the Company's LNP platform, which has been studied across more than a dozen discrete product candidates and is the delivery technology behind the first and only approved RNAi-LNP (patisiran), enables a wide array of RNA-based applications, including vaccines, therapeutic protein production, and gene editing.  Genevant Sciences is committed to transforming the future of human health. For more information, please visit www.genevant.com.

    About Sarepta Therapeutics

    At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Company's programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visit www.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.

    Forward-Looking Statements

    This press release contains "forward-looking statements." 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 include statements regarding the parties' obligations and responsibilities under the agreement, potential payments and fees and Sarepta's right to an exclusive license to Genevant's LNP technology for up to four neuromuscular indications; the potential benefits of LNPs, including offering a non-viral approach to gene editing that can provide both optimal uptake into desired cells and efficient release, resulting in functional delivery of gene editing cargo, such as CRISPR-Cas, to target tissues; the potential for gene editing to revolutionize the treatment of diseases caused by genetic mutations – including rare neuromuscular diseases – by permanently altering genes that lead to disease; the goal of Genevant to design and collaborate with Sarepta in the development of muscle-targeted LNPs that can be applied to gene editing targets in early stage development;  and Sarepta's goal to advance its pre-clinical gene editing program, looking at both viral and non-viral methods to produce a functional gene in order to treat  a broad range of neuromuscular diseases.

    These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta's control. Known risk factors include, among others: the expected benefits and opportunities related to the collaboration between Sarepta and Genevant may not be realized or may take longer to realize than expected due to challenges and uncertainties inherent in product research and development. In particular, the collaboration may not result in the discovery of any new therapeutic compounds or any viable treatments suitable for commercialization due to a variety of reasons, including any inability of the parties to perform their commitments and obligations under the agreement; Sarepta may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, clinical development plans, and bringing its product candidates to market, due to a variety of reasons, many of which may be outside of Sarepta's control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover Sarepta's product candidates and the COVID-19 pandemic; and those risks identified under the heading "Risk Factors" in Sarepta's most recent Annual Report on Form 10-K for the year ended December 31, 2019, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) 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. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

    Internet Posting of Information by Sarepta

    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. 

    Source: Sarepta Therapeutics, Inc.

    Contacts:

    Sarepta Therapeutics, Inc.

    Investors: Ian Estepan, 617-274-4052,

    Media: Tracy Sorrentino, 617-301-8566,

    Genevant Sciences

    Pete Zorn,



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  2. CAMBRIDGE, Mass., Jan. 08, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that senior management will present virtually at the 39th Annual J.P. Morgan Healthcare Conference on Monday, Jan. 11, 2021 at 10:00  a.m. E.T. /7:00 a.m. P.T. Following the presentation there will be a Q&A session starting at 10:20 a.m. E.T. /7:20 a.m. P.T.

    The presentation and Q&A session will be webcast live under the Events & Presentations section of investor relations section of Sarepta's website at https://investorrelations.sarepta.com/events-presentations and will be archived there following the presentation for 90 days. Please connect to Sarepta's website several…

    CAMBRIDGE, Mass., Jan. 08, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that senior management will present virtually at the 39th Annual J.P. Morgan Healthcare Conference on Monday, Jan. 11, 2021 at 10:00  a.m. E.T. /7:00 a.m. P.T. Following the presentation there will be a Q&A session starting at 10:20 a.m. E.T. /7:20 a.m. P.T.

    The presentation and Q&A session will be webcast live under the Events & Presentations section of investor relations section of Sarepta's website at https://investorrelations.sarepta.com/events-presentations and will be archived there following the presentation 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.

    About Sarepta Therapeutics

    At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Company's programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visit www.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.

    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. 

    Source: Sarepta Therapeutics, Inc.

    Sarepta Therapeutics, Inc.

    Investors:

    Ian Estepan, 617-274-4052,

    Media:

    Tracy Sorrentino, 617-301-8566,



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  3. -- Study met the primary biological endpoint of micro-dystrophin protein expression at 12 weeks post-treatment, as measured by western blot, in SRP-9001-treated participants versus placebo --

    -- SRP-9001-treated participants showed an increase in NSAA total score compared to placebo at 48 weeks; however, the study did not achieve statistical significance on the primary functional endpoint of improvement in NSAA total score compared to placebo at 48 weeks post-treatment --

    -- In the pre-specified analysis by age-group, by which the randomization was stratified, participants aged 4-5 years at time of treatment with SRP-9001 demonstrated a statistically significant improvement in NSAA total score versus the age-matched placebo cohort, achieving

    -- Study met the primary biological endpoint of micro-dystrophin protein expression at 12 weeks post-treatment, as measured by western blot, in SRP-9001-treated participants versus placebo --

    -- SRP-9001-treated participants showed an increase in NSAA total score compared to placebo at 48 weeks; however, the study did not achieve statistical significance on the primary functional endpoint of improvement in NSAA total score compared to placebo at 48 weeks post-treatment --

    -- In the pre-specified analysis by age-group, by which the randomization was stratified, participants aged 4-5 years at time of treatment with SRP-9001 demonstrated a statistically significant improvement in NSAA total score versus the age-matched placebo cohort, achieving a 4.3-point improvement on NSAA at 48 weeks post-treatment from baseline --

    -- No new safety signals identified for SRP-9001, reinforcing the favorable safety profile observed to date --

    -- Sarepta to host conference call at 4:30 p.m. Eastern time --

    CAMBRIDGE, Mass., Jan. 07, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced top-line results from Part 1 of Study SRP-9001-102 (Study 102), an ongoing, randomized, double-blind, placebo-controlled clinical trial to evaluate the safety, efficacy and tolerability of a single dose of SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin) in 41 patients with Duchenne muscular dystrophy. SRP-9001 is an investigational gene transfer therapy intended to deliver its micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein.

    At 12 weeks post-treatment compared to baseline, the study met its primary biological endpoint of micro-dystrophin protein expression (P<0.0001). Participants who received SRP-9001 (n=20) had mean micro-dystrophin expression of 28.1%, as measured by western blot. Accompanying secondary biological endpoints including vector genome copies per nucleus, percent positive fibers, intensity, and reduction in creatine kinase (exploratory) were also met.

    In the primary functional endpoint, SRP-9001-treated participants showed an increase in NSAA total score compared to placebo at 48 weeks; however, the difference was not statistically significant (P=0.37). At every time point measured, the cohort of SRP-9001 treated participants outperformed the placebo group, and, at 48 weeks, participants in the treatment group demonstrated a statistically significant increase of 1.7 points in NSAA total score compared to baseline (P=0.009), while participants in the placebo group saw an increase of 0.9 points on the NSAA total score compared to baseline, which was not statistically significant (n=21, P=0.1411).

    Study randomization was stratified by age group and, in the pre-specified analysis of participants aged 4-5 (n=16) at the time of treatment, the treatment group demonstrated a statistically significant 4.3-point improvement on NSAA total score at 48 weeks post treatment compared to a 1.9-point improvement in the age-matched placebo group (P=0.0172). The functional status at baseline for participants in the 4-5 age group was balanced across the placebo and treatment cohorts. A statistically significant imbalance (P=0.0046) in baseline NSAA total score was present in the cohort of 6-7-year-old participants (n=25), resulting in milder participants in the placebo arm (n=13) than in the treated arm (n=12). The significantly different baseline characteristics between treatment and control groups in the 6-7 age group may have contributed to the inability to observe a treatment effect in the 6-7 age group at the week 48 timepoint in Part 1.

    The results from Study 102 reinforce the favorable safety and tolerability profile of SRP-9001 with no new safety signals identified. In line with previously reported clinical data, no clinical complement activation was observed. 85% of participants in the treatment group experienced at least one treatment-related adverse event compared to 43% in the placebo group. Among participants with treatment-related adverse events, 82% were mild or moderate in severity, and 4 participants experienced serious treatment-related adverse events including 3 participants in the treatment group (2 cases of rhabdomyolysis, 2 transaminase elevations) and 1 participant in the placebo (rhabdomyolysis).

    Study 102 is ongoing and remains blinded to participants, investigators, site staff and sponsor staff with direct site interaction. All 41 participants have completed their Part 1, 48-week assessment and have entered the Part 2 crossover phase. Participants continue to be monitored for safety and will undergo another biopsy at week 12 in Part 2 to assess expression and biological markers, in addition to longer-term assessments of functional outcomes.

    "Study 102 reinforces our confidence in the potentially transformative benefits of SRP-9001, including among other things, the fact that in the Study's pre-specified analysis, the participants in the 4-5 age group robustly achieved a statistically significant and clinically meaningful improvement in NSAA over placebo, as predicted by our prior Study 101. For the entire population, while we saw separation at every time point between the active and placebo cohorts, Study 102 did not achieve statistical significance on the primary functional endpoint. In this regard, we are very disappointed that the randomization process resulted in a significant imbalance in baseline NSAA scores between the active and placebo cohorts of the participants ages 6-7, making the 6-7 age groups non-comparable and likely substantially contributing to the inability to achieve statistical significance," said Doug Ingram, president and chief executive officer, Sarepta. "Study 102 remains blinded and we will analyze the functional results for all patients, including cross-over participants, once they have achieved the 48-week timepoint in Part 2. We have already enrolled and dosed 11 participants in Study 103, using our commercial process material, and we will have biomarker and safety results from that cohort in the second quarter. And very importantly, Study 102 has provided us with a wealth of information and insight which we will use to refine and complete the protocol for our upcoming trial using commercial process material. We intend to continue to move forward with diligence and urgency to generate the evidence necessary to bring SRP-9001 to waiting Duchenne patients around the world."

    Sarepta will host an investor webcast and conference call on Thursday, Jan. 7, 2021 at 4:30 pm Eastern Time, to discuss these results. The presentation will be webcast live under the investor relations section of Sarepta's website at https://investorrelations.sarepta.com/events-presentations and slides will be archived there following the call for one year. 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 (844) 534-7313 for domestic callers and (574) 990-1451 for international callers. The passcode for the call is 2538387. Please specify to the operator that you would like to join the "Micro-dystrophin SRP-9001 Study 102 Top-line Results Call."

    *The NSAA is a 17-item rating scale that is used to measure functional motor abilities in ambulant children with Duchenne. It is used to monitor the progression of the disease and treatment effects which makes it suitable as an endpoint in clinical trials for Duchenne.

    About SRP-9001-102

    Study SRP-9001-102 (Study 102) is a double-blind, 1:1 randomized, placebo-controlled clinical trial of SRP-9001 in 41 participants with Duchenne muscular dystrophy between the ages of 4-7. Study 102 uses clinical process SRP-9001 and has two primary endpoints: micro-dystrophin expression at 12 weeks and change in NSAA total score at 48 weeks compared to placebo. Secondary endpoints include certain timed functional tests; micro-dystrophin expression measured by immunofluorescence (IF) fiber intensity; and micro-dystrophin expression measured by IF percent dystrophin positive fibers. In Part 1, results from the treatment and placebo groups are compared through 48 weeks following treatment. In Part 2, the study remains blinded while all participants in the placebo group cross over to active treatment and all participants are followed for another 48 weeks while safety and efficacy continue to be evaluated.

    About SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin)

    SRP-9001 is an investigational gene transfer therapy intended to deliver the micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. Sarepta is responsible for global development and manufacturing for SRP-9001 and plans to commercialize SRP-9001 in the United States upon receiving FDA approval. In December 2019, the Company announced a licensing agreement granting Roche the exclusive right to launch and commercialize SRP-9001 outside the United States. Sarepta has exclusive rights to the micro-dystrophin gene therapy program initially developed at the Abigail Wexner Research Institute at Nationwide Children's Hospital.

    About Duchenne Muscular Dystrophy

    Duchenne muscular dystrophy (DMD) is a rare, fatal neuromuscular genetic disease that occurs in approximately one in every 3,500-5,000 males worldwide. DMD is caused by a change or mutation in the gene that encodes instructions for dystrophin. Symptoms of DMD usually appear in infants and toddlers. Affected children may experience developmental delays such as difficulty in walking, climbing stairs or standing from a sitting position. As the disease progresses, muscle weakness in the lower limbs spreads to the arms, neck and other areas. Most patients require full-time use of a wheelchair in their early teens, and then progressively lose the ability to independently perform activities of daily living such as using the restroom, bathing and feeding. 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 patients usually succumb to the disease in their twenties.

    About Sarepta Therapeutics

    At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Company's programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visit www.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.

    Sarepta Forward-Looking Statement

    This press release contains "forward-looking statements." 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 include statements regarding the potentially transformative benefits of SRP-9001; our plan to analyze the functional results for all the patients in Study 102 once they have achieved the 48-week timepoint; the expectation to have biomarker and safety results from Study 103 in the second quarter of 2021; our plan to use the information and insight from Study 102 to refine and complete the protocol for our upcoming trial using commercial process material; and our intention to continue to move forward with diligence and urgency to generate the evidence necessary to bring SRP-9001 to waiting Duchenne patients around the world.

    These forward-looking statements involve risks and uncertainties that may cause actual results to differ materially from those expressed or implied in the forward-looking statements. Many of these risks and uncertainties are beyond our control. Known risk factors include, among others: success in preclinical and clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful; the data presented in this release may not be consistent with the final data set and analysis or result in an assessment that SRP-9001 provides a safe or effective treatment benefit; different methodologies or assumptions than we utilize to assess particular safety or efficacy parameters may yield different statistical results, and, even if we believe the data collected from clinical trials are positive, the data may not be sufficient to support approval by the FDA or foreign regulatory authorities; we may not be able to execute on our business plans and goals, including meeting our expected or planned regulatory milestones and timelines, clinical development plans, and bringing our product candidates to market, due to a variety of reasons, many of which are outside of our control, including possible limitations on company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates; the impact of the COVID-19 pandemic; and those risks identified under the heading "Risk Factors" in our most recent Annual Report on Form 10-K for the year ended December 31, 2019, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings we make, which you are encouraged to review.

    Any of the foregoing risks could materially and adversely affect the Company's business, results of operations and the trading price of Sarepta's common stock. For a detailed description of risks and uncertainties we face, we encourage you to review our SEC filings. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. We undertake no obligation to update forward-looking statements based on events or circumstances after the date of this press release.

    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.  

    Source: Sarepta Therapeutics, Inc.

    Sarepta Therapeutics, Inc.

    Investors:

    Ian Estepan, 617-274-4052





    Media:

    Tracy Sorrentino, 617-301-8566



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  4. CAMBRIDGE, Mass., Dec. 31, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, granted equity awards on December 31, 2020 that were previously approved by the Compensation Committee of its Board of Directors under Sarepta's 2014 Employment Commencement Incentive Plan, as a material inducement to employment to 11 individuals hired by Sarepta in December 2020. The equity awards were approved in accordance with Nasdaq Listing Rule 5635(c)(4).

    The employees received, in the aggregate, options to purchase 4,525 shares of Sarepta's common stock, and in the aggregate, 1,720 restricted stock units ("RSUs"). The options have an exercise price of $170.49 per share, which is…

    CAMBRIDGE, Mass., Dec. 31, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, granted equity awards on December 31, 2020 that were previously approved by the Compensation Committee of its Board of Directors under Sarepta's 2014 Employment Commencement Incentive Plan, as a material inducement to employment to 11 individuals hired by Sarepta in December 2020. The equity awards were approved in accordance with Nasdaq Listing Rule 5635(c)(4).

    The employees received, in the aggregate, options to purchase 4,525 shares of Sarepta's common stock, and in the aggregate, 1,720 restricted stock units ("RSUs"). The options have an exercise price of $170.49 per share, which is equal to the closing price of Sarepta's common stock on December 31, 2020 (the "Grant Date"). One-fourth of the shares underlying each employee's option will vest on the one-year anniversary of the Grant Date and thereafter 1/48th of the shares underlying each employee's option will vest monthly, such that the shares underlying the option granted to each employee will be fully vested on the fourth anniversary of the Grant Date, in each case, subject to each such employee's continued employment with Sarepta on such vesting dates.

    One-fourth of the RSUs will vest yearly on each anniversary of the Grant Date, such that the RSUs granted to each employee will be fully vested on the fourth anniversary of the Grant Date, in each case, subject to each such employee's continued employment with Sarepta on such vesting date.

    About Sarepta Therapeutics

    At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Company's programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visit www.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.

    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.  

    Source: Sarepta Therapeutics, Inc.

    Sarepta Therapeutics, Inc.

    Investors:

    Ian Estepan, 617-274-4052,

    Media:

    Tracy Sorrentino, 617-301-8566,



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  5. -- Ian Estepan named chief financial officer
    -- Dallan Murray named chief commercial officer
    -- Louise Rodino-Klapac, Ph.D., named chief scientific officer
    -- Ty Howton, general counsel, to retire from Sarepta 
    -- Ryan Brown named interim general counsel

    CAMBRIDGE, Mass., Dec. 14, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, announced today its board of directors approved the appointments of several executive leadership positions: Ian Estepan to executive vice president and chief financial officer, Dallan Murray to senior vice president and chief commercial officer, and Louise Rodino-Klapac, Ph.D., to executive vice president and chief scientific officer, effective…

    -- Ian Estepan named chief financial officer

    -- Dallan Murray named chief commercial officer

    -- Louise Rodino-Klapac, Ph.D., named chief scientific officer

    -- Ty Howton, general counsel, to retire from Sarepta 

    -- Ryan Brown named interim general counsel

    CAMBRIDGE, Mass., Dec. 14, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, announced today its board of directors approved the appointments of several executive leadership positions: Ian Estepan to executive vice president and chief financial officer, Dallan Murray to senior vice president and chief commercial officer, and Louise Rodino-Klapac, Ph.D., to executive vice president and chief scientific officer, effective today. Both Mr. Estepan and Dr. Rodino-Klapac will continue to serve as members of the company's executive committee (ECOM). Mr. Murray will become a member of the Company's ECOM.   Sarepta also announced the retirement of Ty Howton, executive vice president and general counsel. With Mr. Howton's retirement, Ryan Brown has been named interim general counsel, effective today, and will also become a member of the Company's ECOM.

    Mr. Estepan previously served as senior vice president with responsibility for Investor Relations, Corporate Strategy, Corporate Affairs and Chief of Staff. As the chief financial officer of Sarepta, Mr. Estepan will oversee Finance, Corporate Strategy, Business Development, Strategic Alliances, Investor Relations, and Corporate Affairs. Mr. Estepan joined Sarepta in 2015 and is intimately knowledgeable about all aspects of the company's business. He has been responsible for the development of Sarepta's strategy over the past 3 years and was a key architect in securing $2.5 billion in capital to support the Company's growth. During his tenure, he has assumed increasing responsibilities, establishing the Corporate Strategy and Program Management functions, with an intense focus on advancing the interest of patients. Mr. Estepan began his career in finance as a healthcare sector analyst at Salomon Smith Barney in June 1999. Before joining Sarepta, he worked for more than a decade as a portfolio manager for Spectra Financial Group, overseeing a portfolio of pharmaceutical, biotech and medtech equities. He holds a Bachelor of Arts degree in psychology with a concentration in pre-medicine from Columbia University, where he graduated magna cum laude.

    Immediately preceding his appointment to chief commercial officer, Mr. Murray served as Sarepta's interim chief commercial officer. Prior to this, Mr. Murray was vice president, commercial strategy and portfolio management, overseeing the commercial positioning and readiness for the Company's pipeline of more than 40 investigational therapies. With his permanent appointment as chief commercial officer of Sarepta, Mr. Murray will have oversight of U.S. and Global Commercial, Commercial Operations, Global Market Access, Patient Services, Payer & Distribution, Latin America and Japan. Mr. Murray joined Sarepta in 2013 and has been instrumental in the launch of Sarepta's two approved RNA therapies, including one of the top-five most successful rare disease product launches in history. Mr. Murray's scientific knowledge, his relationships with neuromuscular thought leaders and treating physicians around the world, his insight in access and reimbursement, and the depth of his understanding and experience in what will be required to launch a transformative gene therapy, make him uniquely suited for this role. Prior to joining Sarepta, Mr. Murray held commercial leadership roles at Vertex Pharmaceuticals, Gilead Sciences, Biogen, and Janssen Ortho. He holds a Bachelor of Commerce degree from University of Alberta, and a Master's in Business Administration from Queen's University.

    Dr. Rodino-Klapac has been named Sarepta's chief scientific officer. Dr. Rodino-Klapac previously served as Sarepta's senior vice president, gene therapy. Dr. Rodino-Klapac has led the design of most of Sarepta's late-stage gene therapy candidates, has built and led Sarepta's Gene Therapy Center of Excellence (GTCOE) in Columbus, Ohio, and has oversight for Sarepta's Gene Editing Innovation Center (GEIC) in Durham, N.C.   In her new role as chief scientific officer, she will add to her oversight and responsibilities RNA Discovery, Translational Medicine, and Genomics. Dr. Rodino-Klapac is renowned for her contributions to neuromuscular biology, which have profoundly advanced the field. She is author to a significant body of published, peer-reviewed work, the recipient of multiple awards recognizing her impact and accomplishments as a scientist and leader, is a National Institutes of Health (NIH) Fellow appointee, and is a current Board member of the Association for Regenerative Medicine, as well as a member of the American Society for Gene and Cell Therapy, and the American Academy of Neurology. She is the former head of the Laboratory for Gene Therapy Research at Nationwide Children's Hospital and co-founded and served as chief scientific officer of Myonexus Therapeutics before it was acquired by Sarepta in 2019. She received her bachelor's degree in biology from Kings College, and her Ph.D. in molecular genetics from The Ohio State University.

    Ty Howton, who has served as Sarepta's general counsel since 2012, has announced his plans to retire from his role as general counsel. He will remain a consultant to the company through the first quarter of 2021 to support the transition and remain a resource to the Company's ECOM. Ryan Brown, who currently serves as vice president, global chief compliance officer and regulatory counsel for Sarepta, will serve as interim general counsel and become a member of the company's ECOM. Mr. Brown joined Sarepta in 2018. Prior to Sarepta, Mr. Brown served as vice president at Acadia Pharmaceuticals and before that held various roles of increasing responsibility at Allergan, Inc., including senior counsel of regulatory and compliance; general counsel to SkinMedica, an Allergan company; special assistant to the president; and vice president and chief compliance officer. Mr. Brown holds a Bachelor of Arts degree in political science from Loyola Marymount University and his Juris Doctor degree from Harvard Law School.

    "As we continue to execute our vision of becoming the leader in multi-platform genetic medicine to rescue lives stolen by rare disease, the organizational changes we have announced today greatly strengthen our Executive Committee, streamline our operations under passionate, mission-driven leaders, and ensure that we will continue to hone our execution as we move to the next stage in our evolution. I have a long-held bias for building leaders from within the organization when possible and after evaluating impressive external candidates, it was readily clear that we will take our performance to the next level with the elevation of Mr. Estepan as our chief financial officer and Mr. Murray as chief commercial officer, proven Sarepta leaders with a detailed understanding of our strategy who know precisely what must be done to achieve our ambitions and rapidly bring our therapies to patients. Further, with Dr. Rodino-Klapac as Sarepta's chief scientific officer, we will organize under a brilliant scientist and proven leader our interdependent research and translational efforts across our RNA, gene transfer, and gene editing platforms. Finally, on behalf of employees, the executive leadership team and the Sarepta board of directors, we want to extend our thanks to Ty for his significant contributions in shaping Sarepta into what it is today. He has been a steady, guiding hand these past eight years and we are grateful for his leadership and intellect as our general counsel and for his role as thoughtful adviser to me and to the organization. I wish him all the best in his well-deserved retirement," said Doug Ingram, president and chief executive officer, Sarepta Therapeutics.

    Mr. Ingram continued, "Louise, Ian, Dallan and Ryan share a singular passion for our mission and a keen understanding of what must be done to achieve our ambitions. Across the Company, we are prepared for flawless execution to make 2021 our strongest – and most impactful – year yet for the patients we serve."

    "Being part of Sarepta's transformation into a leader in genetic medicine has been the opportunity of a lifetime. After nearly a decade as general counsel, I depart with confidence knowing that the organization is strong and ready to execute to support its fundamental mission of transforming the lives of patients," said Mr. Howton.

    About Sarepta Therapeutics

    At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Company's programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visit www.sarepta.com or follow us on TwitterLinkedInInstagram and Facebook.

    Forward-Looking Statement

    This press release contains "forward-looking statements." 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 include statements regarding the potential benefits of the organizational changes Sarepta has announced and Sarepta being prepared for flawless execution to make 2021 its strongest and most impactful year yet for the patients it serves.

    These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta's control. Known risk factors include, among others: leadership transitions can be inherently difficult to manage and may cause uncertainty or a disruption to Sarepta's business or may increase the likelihood of turnover in other key officers and employees; Sarepta may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, clinical development plans, and bringing its product candidates to market, due to a variety of reasons, many of which may be outside of Sarepta's control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover Sarepta's product candidates and the COVID-19 pandemic; and those risks identified under the heading "Risk Factors" in Sarepta's most recent Annual Report on Form 10-K for the year ended December 31, 2019, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) 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. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

    Source: Sarepta Therapeutics, Inc.

    Media and Investors:

    Sarepta Therapeutics, Inc.

    Investors:

    Ian Estepan, 617-274-4052,

    Media:

    Tracy Sorrentino, 617-301-8566,



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