SRPT Sarepta Therapeutics Inc.

80.24
-0.68  -1%
Previous Close 80.92
Open 80.66
52 Week Low 65.3
52 Week High 181.83
Market Cap $6,902,043,719
Shares 86,017,494
Float 74,256,760
Enterprise Value $6,391,530,614
Volume 713,490
Av. Daily Volume 977,220
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Drug Pipeline

Drug Stage Notes
SRP-9003-101
Limb-girdle muscular dystrophy type 2E (LGMD2E)
Phase 1/2
Phase 1/2
Phase 1/2a 3-year safety and functional data presented at WMS September 20, 2021 showed all four patients demonstrated a clinically meaningful improvement on NSAA, with a mean change from baseline to Year 3 of +7.5 (3.42 SD) and trial met primary endpoint of safety. Results from most recent study found that treated participants (n=4, ages 4 to 7 years) improved 8.6 points on the NSAA compared to a matched natural history cohort 3 years after treatment- noted October 11, 2021.
SRP-9001-301 (EMBARK)
Duchenne Muscular Dystrophy
Phase 3
Phase 3
Phase 3 pivotal trial to enroll 120 patients between ages 4 to 7 in the US, Europe and Asia, conducted in partnership with Roche.
SRP-9001-102
Duchenne muscular dystrophy
Phase 2
Phase 2
Phase 2 safety and efficacy data presented at WMS September 20, 2021 showed trial met primary endpoint of change in micro-dystrophin expression, the change in NSAA total score did not achieve statistical significance compared to placebo, and four treatment-related SAEs were reported in the SRP-9001-treated group. Results from study found that treated participants (n=12, ages 6 to 7) had a positive 2.9-point difference on NSAA compared to a matched natural history cohort one year after treatment - noted October 11, 2021.
SRP-9001-103 (ENDEAVOR)
Duchenne muscular dystrophy
Phase 1b
Phase 1b
Phase 1b open label interim results from the first 11 participants in Cohort 1 (ambulatory boys aged 4-7 years) provided evidence of robust expression of micro-dystrophin and no new safety signals were identified, September 24, 2021. Functional results from study cohort 1 (n=11, ages 4-7) found participants improved 3.0 points on NSAA six months after treatment - noted October 11, 2021.
SRP-5051 (MOMENTUM)
Duchenne muscular dystrophy
Phase 2
Phase 2
Phase 2 30 mg/kg arm data presented May 3, 2021 - mean exon skipping of 10.79% and mean dystrophin expression of 6.55%. Part B of Phase 2 trial to be initiated in 4Q 2021.
SRP-4045 + SRP-4053 (ESSENCE)
Duchenne muscular dystrophy
Phase 3
Phase 3
Phase 3 interim data presented at WMS September 20, 2021 showed significant increased exon skipping and dystrophin expression at 48 weeks relative to baseline and compared with placebo. Of the 27 patients, all displayed an increase in exon 45 skipping (P<0.001) over baseline, representing a 100% response rate. No patients discontinued due to adverse events (AEs).
AMONDYS 45 (casimersen)
Duchenne muscular dystrophy
Approved
Approved
FDA approval announced February 25, 2021.
EXONDYS 51 (eteplirsen)
Duchenne muscular dystrophy
Approved
Approved
Approved September 19, 2016.
LYS-SAF302
Mucopolysaccharidosis type IIIA (MPS IIIA)
Phase 2/3
Phase 2/3
Phase 2/3 ongoing.
Golodirsen - Exon 53
Duchenne muscular dystrophy
Approved
Approved
FDA Approval announced December 12, 2019.

Latest News

  1. CAMBRIDGE, Mass., Oct. 13, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it has priced an underwritten public offering of 6,172,840 shares of its common stock at a price to the public of $81.00 per share. In addition, Sarepta has granted the underwriters a 30-day option to purchase up to an additional 925,926 shares of its common stock on the same terms and conditions as the initial shares sold to the underwriters. Sarepta anticipates the gross proceeds from the offering, before deducting underwriting discounts and commissions and other offering expenses, to be approximately $500.0 million, excluding any exercise of the underwriters' option…

    CAMBRIDGE, Mass., Oct. 13, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it has priced an underwritten public offering of 6,172,840 shares of its common stock at a price to the public of $81.00 per share. In addition, Sarepta has granted the underwriters a 30-day option to purchase up to an additional 925,926 shares of its common stock on the same terms and conditions as the initial shares sold to the underwriters. Sarepta anticipates the gross proceeds from the offering, before deducting underwriting discounts and commissions and other offering expenses, to be approximately $500.0 million, excluding any exercise of the underwriters' option to purchase additional shares. The offering is expected to close on or about October 18, 2021, subject to customary closing conditions.

    Goldman Sachs & Co. LLC, J.P. Morgan Securities LLC, Morgan Stanley & Co. LLC and Credit Suisse Securities (USA) LLC are acting as joint book-running managers for the offering. RBC Capital Markets, LLC, Robert W. Baird & Co. Incorporated and Cantor Fitzgerald & Co. are acting as co-managers for the offering.

    Sarepta intends to use the net proceeds from the offering principally for the continuation of, and initiation of further, clinical trials, commercialization, manufacturing, business development activities, including the potential licensing or acquisition of complementary products, technologies and entities, and other general corporate purposes.

    The shares are being offered by Sarepta pursuant to an effective shelf registration statement that was previously filed with the Securities and Exchange Commission (SEC). The offering is being made only by means of a written prospectus and prospectus supplement. The final prospectus supplement relating to and describing the terms of the offering will be filed with the SEC and will be available on the SEC's website at www.sec.gov. When available, copies of the final prospectus supplement relating to these securities may also be obtained from the offices of Goldman Sachs & Co. LLC, at Prospectus Department, 200 West Street, New York, New York 10282, by telephone at 866-471-2526, by facsimile at 212-902-9316 or by e-mail at prospectusgroup-ny@ny.email.gs.com; J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, telephone: (866) 803-9204, or by emailing prospectus-eq_fi@jpmchase.com; Morgan Stanley & Co. LLC, Attention: Prospectus Department, 180 Varick Street, 2nd Floor, New York, New York 10014, or by email at prospectus@morganstanley.com; or Credit Suisse Securities (USA) LLC, Attn: Prospectus Department, 6933 Louis Stephens Drive, Raleigh, North Carolina 27560, Telephone: 1-800-221-1037, or by email at usa.prospectus@credit-suisse.com.

    This press release does not constitute an offer to sell or a solicitation of an offer to buy any securities of Sarepta, nor shall there be any sale of securities in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.  

    About Sarepta Therapeutics

    Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing.

    Forward-Looking Statements

    This press release contains statements that are forward-looking, including the statements about the completion and timing of the public offering of Sarepta's common stock and the intended use of net proceeds. These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta's control, including risk and uncertainties related to satisfaction of customary closing conditions related to the public offering. Applicable risks also include those that are included in the "Risk Factors" section of Sarepta's Quarterly Report on Form 10-Q for the quarter ended June 30, 2021, and any subsequent SEC filings, including the prospectus supplement related to the offering filed with the SEC. Any forward-looking statement in this press release represents Sarepta's views only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except as required by applicable law.

    Source: Sarepta Therapeutics, Inc.

    Investor Contact:

    Ian Estepan, 617-274-4052

    iestepan@sarepta.com

    Media Contact:

    Tracy Sorrentino, 617-301-8566

    tsorrentino@sarepta.com



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  2. CAMBRIDGE, Mass., Oct. 12, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced preliminary financial results for its third quarter ended September 30, 2021.

    • Sarepta expects its net product revenues for the quarter to be approximately $166.9 million, compared to net product revenues of $121.4 million for the same period of 2020.
    • The Company had approximately $1.6 billion in cash, cash equivalents and investments as of September 30, 2021, compared to $1.9 billion as of December 31, 2020.

    These preliminary financial results are the responsibility of management and have been prepared on a consistent basis with prior periods. The Company has not completed…

    CAMBRIDGE, Mass., Oct. 12, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced preliminary financial results for its third quarter ended September 30, 2021.

    • Sarepta expects its net product revenues for the quarter to be approximately $166.9 million, compared to net product revenues of $121.4 million for the same period of 2020.
    • The Company had approximately $1.6 billion in cash, cash equivalents and investments as of September 30, 2021, compared to $1.9 billion as of December 31, 2020.

    These preliminary financial results are the responsibility of management and have been prepared on a consistent basis with prior periods. The Company has not completed its financial closing procedures for the quarter ended September 30, 2021 and its actual results could be materially different from these preliminary financial results. In addition, the Company's independent registered public accounting firm has not reviewed, compiled, or performed any procedures with respect to these preliminary financial results.

    About Sarepta Therapeutics

    Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing.

    Forward-Looking Statements

    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," "may," "intends," "prepares," "looks," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements relating to our expected financial results.

    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: we have incurred operating losses since our inception and we may not achieve or sustain profitability; the estimates and judgments we make, or the assumptions on which we rely, in preparing our consolidated financial statements could prove inaccurate; Our revenues and operating results could fluctuate significantly, which may adversely affect our stock price; 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, 2020 and most recent Quarterly Report on Form 10-Q filed with the SEC as well as other SEC filings made by the Company which you are encouraged to review.

    Source: Sarepta Therapeutics, Inc.

    Investor Contact:

    Ian Estepan, 617-274-4052

    iestepan@sarepta.com

    Media Contact:

    Tracy Sorrentino, 617-301-8566

    tsorrentino@sarepta.com



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  3. CAMBRIDGE, Mass., Oct. 12, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it is offering to sell, subject to market and other conditions, $500,000,000 in shares of its common stock in an underwritten public offering. Sarepta also intends to grant the underwriters a 30-day option to purchase up to an additional $75,000,000 in shares of its common stock.

    Goldman Sachs & Co. LLC, J.P. Morgan Securities LLC, Morgan Stanley & Co. LLC and Credit Suisse Securities (USA) LLC are acting as the underwriters of the proposed offering.

    Sarepta intends to use the net proceeds from the offering principally for the continuation of, and initiation of further…

    CAMBRIDGE, Mass., Oct. 12, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it is offering to sell, subject to market and other conditions, $500,000,000 in shares of its common stock in an underwritten public offering. Sarepta also intends to grant the underwriters a 30-day option to purchase up to an additional $75,000,000 in shares of its common stock.

    Goldman Sachs & Co. LLC, J.P. Morgan Securities LLC, Morgan Stanley & Co. LLC and Credit Suisse Securities (USA) LLC are acting as the underwriters of the proposed offering.

    Sarepta intends to use the net proceeds from the offering principally for the continuation of, and initiation of further, clinical trials, commercialization, manufacturing, business development activities, including the potential licensing or acquisition of complementary products, technologies and entities, and other general corporate purposes.

    The shares are being offered by Sarepta pursuant to an effective shelf registration statement that was previously filed with the Securities and Exchange Commission (SEC). The offering is being made only by means of a written prospectus and prospectus supplement. A preliminary prospectus supplement relating to and describing the terms of the offering will be filed with the SEC and will be available on the SEC's website at www.sec.gov. When available, copies of the preliminary prospectus supplement relating to these securities may also be obtained from the offices of Goldman Sachs & Co. LLC, at Prospectus Department, 200 West Street, New York, New York 10282, by telephone at 866-471-2526, by facsimile at 212-902-9316 or by e-mail at prospectusgroup-ny@ny.email.gs.com; J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, telephone: (866) 803-9204, or by emailing prospectus-eq_fi@jpmchase.com; Morgan Stanley & Co. LLC, Attention: Prospectus Department, 180 Varick Street, 2nd Floor, New York, New York 10014; or Credit Suisse Securities (USA) LLC, Attn: Prospectus Department, 6933 Louis Stephens Drive, Raleigh, North Carolina 27560, Telephone: 1-800-221-1037, or by email at usa.prospectus@credit-suisse.com. The final terms of the offering will be disclosed in a final prospectus supplement to be filed with the SEC.

    This press release does not constitute an offer to sell or a solicitation of an offer to buy any securities of Sarepta, nor shall there be any sale of securities in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.  

    About Sarepta Therapeutics

    Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing.

    Forward-Looking Statements

    This press release contains statements that are forward-looking, including the statements about the completion, timing and size of the proposed public offering of Sarepta's common stock and the intended use of net proceeds. These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta's control, including risk and uncertainties related to market conditions and satisfaction of customary closing conditions related to the proposed public offering. Sarepta may be unable to complete the public offering on the anticipated terms or at all. Applicable risks also include those that are included in the "Risk Factors" section of Sarepta's Quarterly Report on Form 10-Q for the quarter ended June 30, 2021, and any subsequent SEC filings, including the prospectus supplement related to the proposed offering to be filed with the SEC. Any forward-looking statement in this press release represents Sarepta's views only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except as required by applicable law.

    Source: Sarepta Therapeutics, Inc.

    Investor Contact:

    Ian Estepan, 617-274-4052

    iestepan@sarepta.com

    Media Contact:

    Tracy Sorrentino, 617-301-8566

    tsorrentino@sarepta.com 

     



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    • Results presented at ‘Micro-dystrophin Day' highlight breadth, depth and strength of the clinical evidence to date for SRP-9001 in treating Duchenne muscular dystrophy; tolerability profile remains consistent across treated patients
    • Results from Study SRP-9001-101 found that SRP-9001-treated participants (n=4, ages 4 to 7 years) improved 8.6 points on the North Star Ambulatory Assessment (NSAA) compared to a matched natural history cohort three years after treatment
    • Results from Study SRP-9001-102 found that SRP-9001-treated participants (n=12, ages 6 to 7) had a positive 2.9-point difference on NSAA compared to a matched natural history cohort one year after treatment
    • Functional results from Study SRP-9001-103 Cohort 1 (n=11, ages 4-7) found
    • Results presented at ‘Micro-dystrophin Day' highlight breadth, depth and strength of the clinical evidence to date for SRP-9001 in treating Duchenne muscular dystrophy; tolerability profile remains consistent across treated patients
    • Results from Study SRP-9001-101 found that SRP-9001-treated participants (n=4, ages 4 to 7 years) improved 8.6 points on the North Star Ambulatory Assessment (NSAA) compared to a matched natural history cohort three years after treatment
    • Results from Study SRP-9001-102 found that SRP-9001-treated participants (n=12, ages 6 to 7) had a positive 2.9-point difference on NSAA compared to a matched natural history cohort one year after treatment
    • Functional results from Study SRP-9001-103 Cohort 1 (n=11, ages 4-7) found participants improved 3.0 points on NSAA six months after treatment
    • EMBARK, the first global Phase 3 pivotal double-blind gene therapy trial in Duchenne to enroll 120 patients in the US, Europe and Asia, conducted in partnership with Roche

    CAMBRIDGE, Mass., Oct. 11, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today shared new analyses and functional data from its SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin) development program and details of Study SRP-9001-301, known as EMBARK, its global pivotal Phase 3 trial of SRP-9001 for the treatment of Duchenne muscular dystrophy. SRP-9001, being developed in partnership with Roche, 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.

    In new analyses presented at "SRP-9001 Micro-dystrophin Day," results from participants treated with SRP-9001 in Study SRP-9001-101 (n=4, ages 4 to 7) found that participants in Study 101 improved 8.6 points on the North Star Ambulatory Assessment (NSAA)* compared to a matched natural history cohort three years following a single administration of SRP-9001 (p<0.0001). In Study SRP-9001-102, SRP-9001-treated participants ages 6 to 7 (n=12) had a positive 2.9-point difference on NSAA change from baseline compared to a matched natural history control (p=0.0129).

    In addition, the first functional results were presented from Study SRP-9001-103 (ENDEAVOR), which uses commercially representative SRP-9001 material. Results from the first 11 participants in Cohort 1, ages 4 to 7, demonstrated a 3.0-point improvement from baseline on NSAA six months after treatment.

    "With 77 patients treated to date, the multi-study development program for SRP-9001 represents the most comprehensive and long-term dataset for a Duchenne muscular dystrophy gene therapy in existence. The totality of evidence shows that SRP-9001 is a significantly differentiated gene therapy product candidate with one-time dosing and a stable tolerability profile, results in robust expression and evidence of sustained functional benefits across our various studies," said Doug Ingram, president and chief executive officer, Sarepta. "We commence our EMBARK pivotal trial – currently the only truly global Phase 3 trial with a Duchenne gene therapy – with great conviction in the transformative potential of SRP-9001. But while our expression, tolerability, functional evidence and CMC achievements may place SRP-9001 alone among potential therapies, we never forget that we remain in a daily race against a life-ending degenerative disease. To that end, Sarepta, along with our partner Roche, will continue working with tenacity and urgency to bring this potentially transformative treatment to individuals with Duchenne around the world."

    "When compared to a matched natural history cohort, individuals with Duchenne who received SRP-9001 are performing better on the NSAA or showing stabilization of NSAA scores where we would expect to see a decline. The functional results from Study 103, as early as 6 months following treatment, provide additional confidence in our ability to confirm a treatment effect with SRP-9001 as we advance our pivotal Phase 3 trial," said Louise Rodino-Klapac, Ph.D., executive vice president and chief scientific officer, Sarepta. "Given our experience with the AAVrh74 vector in more than 80 individuals with Duchenne and limb-girdle muscular dystrophy, we are very encouraged by the sustained and meaningful clinical results and consistency of the safety profile of SRP-9001."

    The safety and tolerability profile of SRP-9001 is similar to past reports. Across all three studies, treatment-related adverse events (TRAEs) generally occurred within 90 days of treatment and subsequently resolved. No clinically relevant complement activation was observed​ in any of the studies. The most common treatment-related adverse event was vomiting, generally within the first week post-infusion. Increases in liver enzymes were transient and responsive to steroids​. In Study 9001-103, safety data were consistent with data from previous studies of SRP-9001 (101 and 102). There was one immune-mediated myositis serious adverse event in Cohort 2​ specific to the participant's mutation; participant received treatment including plasmapheresis and has since returned to pre-event function.

    About the SRP-9001 Clinical Development Program

    The SRP-9001 clinical development program currently consists of four studies:

    • EMBARK, Study SRP-9001-301: a global, randomized, double-blind, placebo-controlled clinical trial of commercially representative SRP-9001 material in 120 participants with Duchenne muscular dystrophy between the ages of 4 to 7. The primary endpoint will assess the change in NSAA total score from baseline to week 52 compared to placebo.



      Key features of EMBARK include stratification of participants by age and baseline NSAA, with a minimum of 50 percent of patients ages 4 to 5 enrolled. Inclusion criteria include a stable daily dose of oral corticosteroids for at least 12 weeks before screening and rAAVrh74 antibody titers of less than 1:400. Participants with mutations between or including exons 1-17 or mutations fully contained within exon 45 (inclusive) are not eligible.



      Secondary endpoints include the number of skills gained or improved at week 52 as measured by NSAA, the quantity of micro-dystrophin protein expression at week 12 (Part 1) as measured by western blot, timed function tests and safety. In Part 1, results from the treatment and placebo groups are compared through 52 weeks following treatment. In Part 2, the study remains blinded to investigators and participants while all participants in the placebo group cross over to active treatment and all participants are followed for another 52 weeks while safety and efficacy continue to be evaluated.
    • ENDEAVOR, Study SRP-9001-103 (Study 103): an open-label clinical trial of SRP-9001 that has enrolled 32 participants with Duchenne muscular dystrophy, with 20 participants ages 4 to 7 and expanded cohorts that include older ambulant and non-ambulant individuals. Study 103 uses commercially representative SRP-9001 material and the primary endpoint is the change from baseline in the quantity of micro-dystrophin protein expression measured by western blot at 12 weeks. Secondary outcome measures include change from baseline in micro-dystrophin expression fiber intensity as measured by immuno-fluorescence and micro-dystrophin expression measured by IF percent dystrophin positive fibers at 12 weeks. Exploratory endpoints include the change in vector genome copies per nucleus, NSAA and certain timed functional tests. Including the initial 12-week period, patients will be followed for a total of five years.



    • Study SRP-9001-102 (Study 102): 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 to 7. Study 102 uses clinical process SRP-9001 material 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 immuno-fluorescence fiber intensity; and micro-dystrophin expression measured by immuno-fluorescence 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 and for five years total after infusion.



    • SRP-9001-101 (Study 101): a single-center, open-label clinical trial of SRP-9001 to evaluate the safety, tolerability and proof of concept of a single dose of clinical process SRP-9001 material. The trial enrolled 4 ambulatory participants with Duchenne muscular dystrophy between the ages of 4 to 7. The primary endpoint was safety, and secondary endpoint included the change in micro-dystrophin expression pre- and post-treatment, decreases in creatine kinase, NSAA total score and timed function test. Participants are being followed for five years after treatment, while safety and efficacy continue to be evaluated.

    *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 (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, Roche partnered with Sarepta to combine Roche's global reach, commercial presence and regulatory expertise with Sarepta's gene therapy candidate for Duchenne to accelerate access to SRP-9001 for patients 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 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

    Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA 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.

    Forward-Looking Statements

    This press release contains "forward-looking statements." Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believe," "anticipate," "plan," "expect," "will," "may," "intend," "prepare," "look," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements relating to the potential benefits of SRP-9001; the potential of our collaborations and partnerships; and expected plans and milestones, including initiating SRP-9001-301 in the U.S., Europe and Asia.

    Known risk factors include, among others: success in pre-clinical trials and clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful; different methodologies, assumptions and applications 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 of our product candidates are positive, these data may not be sufficient to support approval by 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, some of which may be outside of our 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 our product candidates, and the COVID-19 pandemic; even if Sarepta's programs result in new commercialized products, Sarepta may not achieve the expected revenues from the sale of such products; 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, 2020, 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 the Company 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 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, except as required by law.

    Source: Sarepta Therapeutics, Inc.

    Investor Contact:

    Ian Estepan, 617-274-4052

    iestepan@sarepta.com

    Media Contact:

    Tracy Sorrentino, 617-301-8566

    tsorrentino@sarepta.com



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    • Center dedicated to research and development activities to advance Sarepta's industry-leading, multi-platform pipeline
    • The Center encompasses 85,000 square feet of space, tripling Sarepta's footprint in Ohio

    CAMBRIDGE, Mass., Oct. 04, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today celebrated the grand opening of the Genetic Therapies Center of Excellence (GTCOE), its new research facility in Columbus, Ohio.

    The 85,000 square foot state-of-the-art facility expands Sarepta's research and development capabilities and footprint, which includes sites in Cambridge, Andover and Burlington, Mass. With more than 70 employees today and plans to double the number…

    • Center dedicated to research and development activities to advance Sarepta's industry-leading, multi-platform pipeline
    • The Center encompasses 85,000 square feet of space, tripling Sarepta's footprint in Ohio

    CAMBRIDGE, Mass., Oct. 04, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today celebrated the grand opening of the Genetic Therapies Center of Excellence (GTCOE), its new research facility in Columbus, Ohio.

    The 85,000 square foot state-of-the-art facility expands Sarepta's research and development capabilities and footprint, which includes sites in Cambridge, Andover and Burlington, Mass. With more than 70 employees today and plans to double the number of employees by the end of 2022, the Center is focused on discovery, pre-clinical and clinical development supporting Sarepta's pipeline of genetic medicines which includes RNA, gene therapy and gene editing programs. The Center also supports process development and optimization work that enables the transition from clinical-scale to commercial-scale manufacturing, a critical task facing companies developing gene therapies.

    "Advances in the science of genetic medicine are creating incredible opportunities to develop medicines with the potential to transform the lives of people with rare diseases. Sarepta's Genetic Therapies Center of Excellence complements and enhances our existing research and development expertise and will play a central and strategic role in our future as the leader in precision genetic medicine," said Doug Ingram, president and chief executive officer, Sarepta.

    Among the guests joining the Sarepta team today for a dedication, ribbon-cutting ceremony and facility tours: The Honorable Jon Husted, Ohio's Lieutenant Governor; Pat Furlong, president and chief executive officer, Parent Project Muscular Dystrophy (PPMD); Jessica Evans, assistant director, The Speak Foundation; local officials; and luminaries from Columbus' growing biotechnology sector. At the event, Sarepta also announced a $20,000 donation to the Ronald McDonald House Charities of Central Ohio, with Dee Anders, chief executive officer and executive director, Ronald McDonald House Charities of Central Ohio, present to accept.

    "Sarepta has operated in Columbus since 2018 and we're proud to be at the forefront of Columbus' emergence as a leading hub for biotechnology – committed to the local community and the patients and families we serve," said Louise Rodino-Klapac, Ph.D., Sarepta's Columbus-based executive vice president and chief scientific officer. "Our growing presence in Ohio will help us strengthen our close working relationships with long-standing local partners such as Nationwide Children's Hospital, while we work with the greatest urgency to advance our pipeline, further the science of genetic medicine and create an environment where future generations of scientific talent will thrive."

    "Sarepta Therapeutics' decision to expand in Ohio is the latest example that Ohio is a great state to grow a business," said Lt. Governor Jon Husted. "When we created the Columbus Innovation District last year, we were focused on cultivating the right environment in central Ohio to attract new investments and jobs in gene and cell therapy. This new facility is a victory, as it builds on our strategy, creating jobs and producing some of the most advanced research and development of precision genetic medicine, further solidifying Ohio as a leader in gene therapy."

    About Sarepta Therapeutics

    Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA 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.

    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 potential opportunities in the rare disease space; the potential transformative benefits of medicines in the rare disease space; our plans to double the number of employees in Columbus, Ohio by the end of 2022; and the potential for our growing presence in Ohio to help strengthen our close working relationships with long-standing local partners while we work with the greatest urgency to advance our pipeline, further the science of genetic medicine and create an environment where future generations of scientific talent will thrive.

    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: 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, 2020, 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, except as required by law.

    Source: Sarepta Therapeutics, Inc.

    Investor Contact:

    Ian Estepan, 617-274-4052

    iestepan@sarepta.com

    Media Contact:

    Tracy Sorrentino, 617-301-8566

    tsorrentino@sarepta.com

    Multimedia

    Photos accompanying this announcement are available at

    https://www.globenewswire.com/NewsRoom/AttachmentNg/a2e78dd7-47fc-440c-bb7e-e8fb3abb992f

    https://www.globenewswire.com/NewsRoom/AttachmentNg/e902e92d-0591-4dfd-b6f5-c1a82a9591e9



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