SRPT Sarepta Therapeutics Inc.

162.75
-2.93  -2%
Previous Close 165.68
Open 169.17
52 Week Low 72.05
52 Week High 172.7
Market Cap $12,692,668,086
Shares 77,988,744
Float 65,825,348
Enterprise Value $11,503,239,105
Volume 539,637
Av. Daily Volume 986,868
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Upcoming Catalysts

Drug Stage Catalyst Date
SRP-5051
Duchenne muscular dystrophy
Phase 1/2
Phase 1/2
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SRP-9001
Duchenne muscular dystrophy
Phase 2
Phase 2
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Drug Pipeline

Drug Stage Notes
Casimersen
Duchenne muscular dystrophy
NDA Filing
NDA Filing
Rolling NDA has been completed - June 26, 2020.
LYS-SAF302
Mucopolysaccharidosis type IIIA (MPS IIIA)
Phase 2/3
Phase 2/3
Phase 2/3 dosing to be completed mid-2020.
MYO-101 (SRP-9003)
Limb-girdle Muscular Dystrophy Type 2E (LGMD2E)
Phase 1/2
Phase 1/2
Phase 1/2 one-year functional results from the low dose cohort presented June 8, 2020. Three patients showed improvement in functional mobility of 4,6 and 7 points.
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. -- Exclusive worldwide license enables Sarepta to develop and promote imlifidase as a pre-treatment for gene therapy in Duchenne and Limb-girdle muscular dystrophy patients who have pre-existing antibodies to AAV --

    CAMBRIDGE, Mass., July 02, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced an agreement with Hansa Biopharma, the leader in immunomodulatory enzyme technology for rare Immunoglobulin G (IgG) mediated diseases, for imlifidase. Under the terms of the agreement, Sarepta obtains an exclusive, worldwide license to develop and promote imlifidase as a pre-treatment to enable Sarepta gene therapy administration in Duchenne muscular dystrophy…

    -- Exclusive worldwide license enables Sarepta to develop and promote imlifidase as a pre-treatment for gene therapy in Duchenne and Limb-girdle muscular dystrophy patients who have pre-existing antibodies to AAV --

    CAMBRIDGE, Mass., July 02, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced an agreement with Hansa Biopharma, the leader in immunomodulatory enzyme technology for rare Immunoglobulin G (IgG) mediated diseases, for imlifidase. Under the terms of the agreement, Sarepta obtains an exclusive, worldwide license to develop and promote imlifidase as a pre-treatment to enable Sarepta gene therapy administration in Duchenne muscular dystrophy (DMD) and Limb-girdle muscular dystrophy (LGMD), for patients who may otherwise not be eligible for treatment.

    Sarepta's investigational gene therapies use an adeno-associated virus (AAV) and patients with Duchenne and LGMD who have pre-existing IgG antibodies are not currently eligible for treatment with any AAV-based gene therapies. Imlifidase is an antibody-cleaving enzyme that specifically targets IgG and inhibits an IgG-mediated immune response. Imlifidase has a rapid onset of action, cleaving IgG-antibodies and inhibiting their reactivity within hours after administration, thus clearing the AAV-IgG antibodies that would typically preclude dosing or re-dosing with AAV.

    "As we expand our leadership position in genetic medicine and build out our gene therapy engine, one of Sarepta's central ambitions is to find scientific solutions that bring our potentially life-saving therapies to the greatest number of the rare disease patients we serve," said Doug Ingram, president and chief executive officer, Sarepta Therapeutics. "One of the current limitations of gene therapy is the inability to treat patients who have pre-existing neutralizing antibodies to the AAV vector. While our AAVrh74 vector has been associated with a low screen out rate for neutralizing antibodies, even that low rate is inconsistent with our mission.  In pre-clinical and clinical models, Hansa's technology has shown the ability to clear the IgG antibodies that prevent dosing AAV-based gene therapies. If successful, this could offer the potential of extending our gene therapy treatments to DMD and LGMD patients who would otherwise have been denied access due to pre-existing antibodies." 

    "We see significant potential for our enzyme technology in the gene therapy space overall, and we are excited to partner with Sarepta, a leading player in the field, to use the unique features of imlifidase to potentially enable gene therapy treatment in patients who today aren't eligible for these breakthrough therapies due to pre-existing neutralizing antibodies in two conditions with a very high unmet medical need," said Søren Tulstrup, president and chief executive officer, Hansa Biopharma.

    Under the terms of the agreement, Hansa will receive an upfront payment of $10 million and is eligible for additional development, regulatory and sales milestone payments potentially totaling up to $397.5 million. Hansa will book all sales of imlifidase and will earn tiered royalties up to the mid-teens on any incremental gene therapy sales that arise from treating antibody-positive patients enabled through imlifidase pre-treatment.

    About 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 Limb-Girdle Muscular Dystrophy

    Limb-girdle muscular dystrophies are genetic diseases that cause progressive, debilitating weakness and wasting that begin in muscles around the hips and shoulders before progressing to muscles in the arms and legs.

    Patients with limb-girdle muscular dystrophy Type 2E (LGMD2E) begin showing neuromuscular symptoms such as difficulty running, jumping and climbing stairs before age 10. The disease, which is an autosomal recessive subtype of LGMD, progresses to loss of ambulation in the teen years and often leads to early mortality. There is currently no treatment or cure for LGMD2E.

    Sarepta has five LGMD gene therapy programs in development, including subtypes for LGMD2E, LGMD2D, LGMD2C, LGMD2B and LGMD2L, and holds an option for a sixth program for LGMD2A.

    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 parties' obligations and responsibilities under the agreement and potential payments and fees; the potential of imlifidase to clear the IgG antibodies that prevent dosing AAV-based gene therapies and to enable Sarepta gene therapy administration in DMD and LGMD patients who would otherwise have been denied access due to pre-existing antibodies; and Sarepta's goal of finding scientific solutions that bring its potentially life-saving therapies to the greatest number of the rare disease 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: the expected benefits and opportunities related to the collaboration between Sarepta and Hansa Biopharma 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 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; success in preclinical and early clinical trials does not ensure that later clinical trials will be successful; 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 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, 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.

    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

    Primary Logo

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  2. CAMBRIDGE, Mass., June 30, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, granted equity awards on June 30, 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 26 individuals hired by Sarepta in June 2020. The equity awards were approved in accordance with Nasdaq Listing Rule 5635(c)(4).

    The employees received, in the aggregate, options to purchase 27,315 shares of Sarepta's common stock, and in the aggregate, 13,680 restricted stock units ("RSUs"). The options have an exercise price of $160.34 per share, which is equal…

    CAMBRIDGE, Mass., June 30, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, granted equity awards on June 30, 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 26 individuals hired by Sarepta in June 2020. The equity awards were approved in accordance with Nasdaq Listing Rule 5635(c)(4).

    The employees received, in the aggregate, options to purchase 27,315 shares of Sarepta's common stock, and in the aggregate, 13,680 restricted stock units ("RSUs"). The options have an exercise price of $160.34 per share, which is equal to the closing price of Sarepta's common stock on June 30, 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

    Primary Logo

    View Full Article Hide Full Article
  3. CAMBRIDGE, Mass., June 30, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced the retirement of Sandy Mahatme, Sarepta's executive vice president, chief financial officer and chief business officer, from the company effective July 10, 2020. The company has commenced a search process to identify the future chief financial officer. During the interim period, the finance and accounting functions will report directly to Sarepta's Chief Executive Officer, Doug Ingram, and other departments reporting to Mr. Mahatme will be overseen by members of Sarepta's executive committee.

    "The Sarepta from which Sandy retires is a very different one from the organization…

    CAMBRIDGE, Mass., June 30, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced the retirement of Sandy Mahatme, Sarepta's executive vice president, chief financial officer and chief business officer, from the company effective July 10, 2020. The company has commenced a search process to identify the future chief financial officer. During the interim period, the finance and accounting functions will report directly to Sarepta's Chief Executive Officer, Doug Ingram, and other departments reporting to Mr. Mahatme will be overseen by members of Sarepta's executive committee.

    "The Sarepta from which Sandy retires is a very different one from the organization he joined as our chief financial officer some eight years ago. And the Sarepta of today – a financially solid biotechnology organization with perhaps the industry's deepest and most valuable pipeline of genetic medicine candidates with the potential to extend and improve lives – would not have been possible without Sandy's business acumen and dedication," said Doug Ingram, president and chief executive officer, Sarepta Therapeutics. "On behalf of our board of directors and the entire organization, I want to wish Sandy all the best in his next journey and thank him for his invaluable and numerous contributions to our success and for having built a strong team of finance leaders who will continue to perform as he departs."

    Said Mr. Mahatme, "It has been a privilege to serve as Sarepta's CFO and CBO for almost eight years and to have participated in its remarkable transformation and extraordinary growth. Working with this leadership team and our talented colleagues, we have built a strong foundation for Sarepta's ongoing success in achieving its goal of changing the lives of patients with rare diseases around the world. Having built a strong team of finance, IT, facilities, manufacturing and business development professionals, I feel confident that this is a good time to transition to other opportunities, knowing that Sarepta is well-positioned to continue to lead the industry."

    Sandy will continue to serve on the Board of Directors for Flexion Therapeutics, Inc., Aeglea BioTherapeutics, Inc., and Idorsia Pharmaceuticals Ltd. 

    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 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 search process to identify the future chief financial officer, the reporting structure during the interim period and the performance of the finance team; Sarepta's potential to extend and improve lives; Sarepta's goal of changing the lives of patients with rare diseases around the world; and Sarepta being well-positioned to continue to lead the industry.

    These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta's control. Known risk factors include, among others: 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

    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

    Primary Logo

    View Full Article Hide Full Article
  4. -- Casimersen is designed for the treatment of exon 45 amenable patients, approximately eight percent of patients with Duchenne --

    -- Casimersen is the third exon-skipping medicine using the Company's proprietary PMO RNA-based platform -- 

    CAMBRIDGE, Mass., June 26, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced the Company has completed the submission of a rolling New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking accelerated approval for casimersen (SRP-4045). Casimersen, a phosphorodiamidate morpholino oligomer (PMO), is engineered to treat patients with Duchenne muscular dystrophy (DMD) who have genetic…

    -- Casimersen is designed for the treatment of exon 45 amenable patients, approximately eight percent of patients with Duchenne --

    -- Casimersen is the third exon-skipping medicine using the Company's proprietary PMO RNA-based platform -- 

    CAMBRIDGE, Mass., June 26, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced the Company has completed the submission of a rolling New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking accelerated approval for casimersen (SRP-4045). Casimersen, a phosphorodiamidate morpholino oligomer (PMO), is engineered to treat patients with Duchenne muscular dystrophy (DMD) who have genetic mutations that are amenable to skipping exon 45 of the Duchenne gene.

    The completion of the rolling submission includes data from the casimersen arm of the ESSENCE study (also known as study 4045-301), a global, randomized, double-blind, placebo-controlled Phase 3 study evaluating efficacy and safety in patients amenable to skipping exons 45 and 53. An interim analysis from ESSENCE demonstrated a statistically significant increase in dystrophin production as measured by western blot in patients who received casimersen compared to baseline and placebo. The study is ongoing, and remains blinded to collect additional efficacy and safety data. If the casimersen NDA is accepted and granted accelerated approval, the completed ESSENCE study will serve as a post-marketing confirmatory study.

    "The completion of our casimersen submission is an important milestone in our journey to advance treatments for the greatest possible number of people living with Duchenne muscular dystrophy," said Doug Ingram, president and chief executive officer, Sarepta Therapeutics. "If approved, casimersen will be our third approved therapy for sub-populations of Duchenne. Together with our other approved therapies, we have the potential to treat nearly 30% of Duchenne patients in the United States. Our proprietary PMO platform is an important focus of our pipeline, and we owe our clinical progress to the patients and families participating in our studies."

    About Casimersen

    Casimersen uses Sarepta's proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 45 of the DMD gene. Casimersen is designed to bind to exon 45 of dystrophin pre-mRNA, resulting in exclusion, or "skipping," of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 45 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein.

    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.

    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 casimersen and the PMO technology; the potential of casimersen, if approved, to treat approximately 8% of patients with DMD; Sarepta's potential to treat nearly 30% of DMD patients in the United States; the expectation that the completed ESSENCE study will serve as a post-marketing confirmatory study if the casimersen NDA is accepted and granted accelerated approval; and Sarepta's goal to advance treatments for the greatest possible number of people living with DMD.

    These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta's control. Known risk factors include, among others: Sarepta may not be able to complete clinical trials required by the FDA or other regulatory authorities for approval of casimersen; casimersen may not result in a viable treatment suitable for commercialization due to a variety of reasons including the results of future research may not be consistent with past positive results or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; and even if casimersen results in a commercialized product, Sarepta may not achieve any significant revenues from the sale of such product; if the actual number of patients suffering from DMD is smaller than estimated, our revenue and ability to achieve profitability may be adversely affected; 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

    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



    Primary Logo

    View Full Article Hide Full Article
  5. - Alliance to explore the utility of engineered exosomes developed with Codiak's engEx™ Platform to deliver gene therapy, gene editing and RNA technologies -

    - Two-year, global research and option agreement covers up to five neuromuscular targets -

    - Codiak is eligible to receive up to $72.5 million in upfront and near-term license payments plus research funding -

    CAMBRIDGE, Mass., June 22, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, and Codiak BioSciences, Inc., a company at the forefront of advancing engineered exosomes as a new class of biologic medicines, today announced a global research and option agreement to design and develop engineered exosome therapeutics…

    - Alliance to explore the utility of engineered exosomes developed with Codiak's engEx™ Platform to deliver gene therapy, gene editing and RNA technologies -

    - Two-year, global research and option agreement covers up to five neuromuscular targets -

    - Codiak is eligible to receive up to $72.5 million in upfront and near-term license payments plus research funding -

    CAMBRIDGE, Mass., June 22, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, and Codiak BioSciences, Inc., a company at the forefront of advancing engineered exosomes as a new class of biologic medicines, today announced a global research and option agreement to design and develop engineered exosome therapeutics to deliver gene therapy, gene editing and RNA technologies for neuromuscular diseases. The engineered exosome approach offers the potential to effectively deliver genetic therapeutics without triggering the adaptive immune response.

    The two-year agreement includes up to five neuromuscular targets. Codiak is eligible to receive up to $72.5 million in upfront and near-term license payments plus research funding. Sarepta is granted an option to any of the candidates developed pursuant to the research alliance.

    Exosomes are natural nanoparticles that serve as the body's intercellular communication system, facilitating the transfer of a wide variety molecular payloads between cells. As they are derived from human cells, exosomes provide a unique advantage as a targeted delivery system for genetic medicines because they are inherently non-immunogenic. Through its proprietary, engEx Platform, Codiak can engineer exosomes with specific cargos and guide tropism to cell types of interest. The collaboration will leverage Codiak's exosome engineering capabilities with Sarepta's expertise in precision genetic medicine to develop next generation therapeutics for patients with neuromuscular diseases that have few or no treatment options.

    "As Sarepta expands its leadership position in precision genetic medicine, this alliance with Codiak furthers our goal to deliver the most advanced therapies to patients. Codiak's engEx technology could potentially address some of the limitations of current treatment approaches and offers broad utility across Sarepta's therapeutic modalities -- gene therapies, gene editing and RNA. Codiak's exosomes are engineered for precise tissue targeting and offer a non-viral delivery approach with non-immunogenic potential, thus opening up avenues for more efficient delivery and potential re-dosing," said Doug Ingram, president and chief executive officer, Sarepta Therapeutics.

    "The development of targeted delivery systems that enable repeat-dosing to select cell types has been a challenge for the emerging field of genetic medicine, especially in diseases of the muscle," said Douglas E. Williams, President and CEO of Codiak BioSciences. "Engineered exosomes may offer a solution through their potential to selectively target muscle cells. We are excited to work with the world-class team at Sarepta to further build the engEx Platform and evaluate the potential of exosomes as next generation constructs incorporating gene therapy, gene editing and other nucleic acid payload modalities."

    Under the terms of the agreement, Sarepta has the exclusive option to license Codiak's technology to develop and commercialize engineered exosome therapeutics for up to five neuromuscular targets. Sarepta and Codiak will collaborate to design exosomes that can deliver and functionally release select payloads, such as nucleic acids and gene therapy and gene editing constructs, in neuromuscular indications. If Sarepta elects to exercise its option on a target, Codiak will be responsible for research and preclinical development through IND preparation, and Sarepta will be responsible for clinical development and commercial activities. In addition to upfront, research funding and license payments, Codiak will be eligible to receive significant development and regulatory milestone payments and tiered royalties on future sales.

    About engEx™ Platform

    The engEx Platform is Codiak's proprietary exosome therapeutic engine for engineering and manufacturing novel exosome product candidates designed to target multiple pathways throughout the body. Using this platform, Codiak can design exosomes with precisely engineered properties, incorporate various types of biologically active molecules and direct them to specific cell types and tissues. These exosomes engage targets by cellular uptake, membrane-to-membrane interaction or a combination of both mechanisms and are designed to change the biological functioning of the recipient cells in order to produce the intended biological effect. Codiak is building a broad pipeline of engEx product candidates that may have a transformative impact on the treatment of many diseases.

    About Codiak BioSciences

    Codiak BioSciences is harnessing exosomes—natural intercellular messengers—to develop a new class of biologic medicines, exosome therapeutics. Utilizing our proprietary and versatile exosome engineering and manufacturing platform (engEx™ Platform), Codiak is developing exosomes with precisely engineered properties to engage pathways and deliver potent therapeutics to specific cell targets. We are building a broad pipeline of therapeutic candidates that may have a transformative impact on the treatment of many diseases, including in the areas of oncology, immune-based diseases, fibrotic disorders, neurological disorders and rare diseases.  For more information, visit http://www.codiakbio.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.

    Sarepta 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, potential neuromuscular targets and Sarepta's option to any of the candidates developed pursuant to the research alliance; the potential benefits of the engineered exosome approach, including effectively delivering genetic therapeutics without triggering the adaptive immune response and selectively targeting muscle cells; the potential of exosomes to provide a unique advantage as a targeted delivery system for genetic medicines; and the potential benefits of the collaboration between Sarepta and Codiak, including the development of next generation therapeutics for patients with neuromuscular diseases that have few or no treatment options. 

    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 Codiak BioSciences 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 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; success in preclinical trials does not ensure that later clinical trials will be successful; 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 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, 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.

    Contacts:

    Sarepta Therapeutics

    Investors: Ian Estepan, 617-274-4052,

    Media: Tracy Sorrentino, 617-301-8566,  

    Codiak BioSciences

    Kate Niazi-Sai, 617-949-5696,

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