CAMBRIDGE, Mass., July 24, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that theU.S. Food and Drug Administration(FDA) has granted Fast Track designation to SRP-9001 (AAVrh74.MHCK7.micro-dystrophin). 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. Safety and tolerability data at one year from four clinical trial participants who received SRP-9001 in Study 101 were recently published in JAMA Neurology, and Study 102, a randomized, double-blind, placebo-controlled study of SRP-9001, is ongoing with results expected in early 2021.
The Fast Track designation is a process designed to facilitate the development and expedited review of drugs that treat serious conditions and fill unmet medical needs.In addition to Fast Track, SRP-9001 has also been granted Rare Pediatric Disease (RPD) designation. SRP-9001 was previously granted Orphan Drug status in the United States, the European Union and Japan.
About SRP-9001
SRP-9001 is an investigational gene transfer therapy designed 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. 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 Childrens Hospital.
AboutSarepta TherapeuticsAt 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 Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visitwww.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.
Forward-Looking StatementsThis 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 SRP-9001s intention to deliver the micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein; Sareptas expectation to have results from Study 102 in early 2021; and Sareptas plan to commercialize SRP-9001 in the United States.
These forward-looking statements involve risks and uncertainties, many of which are beyond Sareptas control. Known risk factors include, among others: fast track designation by the FDA may not lead to faster development or regulatory review or approval process, and does not increase the likelihood that SRP-9001 will receive marketing approval; Sarepta may not be able to complete clinical trials required by the FDA or other regulatory authorities for approval of SRP-9001; SRP-9001 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; 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 Sareptas 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 Sareptas product candidates and the COVID-19 pandemic; and those risks identified under the heading Risk Factors in Sareptas 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 Sareptas business, results of operations and the trading price of Sareptas 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 atwww.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, 617-274-4052iestepan@sarepta.com
Media:Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com
Original post:
- IOM not webcast today. Why Not? - November 8th, 2009 [November 8th, 2009]
- National Academies skeptical at Best. - November 8th, 2009 [November 8th, 2009]
- Some Confusion Exists - November 8th, 2009 [November 8th, 2009]
- Why DTC Genomics IS Medicine. - November 8th, 2009 [November 8th, 2009]
- First Mari, Now Linda. Who's next? - November 8th, 2009 [November 8th, 2009]
- Is it true? - November 8th, 2009 [November 8th, 2009]
- Re-Reviewing the National Academies - November 8th, 2009 [November 8th, 2009]
- The problem with nonclinicians....... - November 8th, 2009 [November 8th, 2009]
- Crazy Night of Emails to Government - November 8th, 2009 [November 8th, 2009]
- Adrienne Carlson's Personalized Medicine. - November 8th, 2009 [November 8th, 2009]
- Tell Me, How do you feel now? Sherpa's RX - November 8th, 2009 [November 8th, 2009]
- This Just In. 23andMe to go to GPs. I love my readers!! - November 8th, 2009 [November 8th, 2009]
- Sorry so long away - November 8th, 2009 [November 8th, 2009]
- 2D6 Rears its ugly head..... - November 8th, 2009 [November 8th, 2009]
- Ok, Fine, Back to Plavix - November 8th, 2009 [November 8th, 2009]
- Kaiser a protoype for Collins' Aim - November 8th, 2009 [November 8th, 2009]
- A few months late to the party.... - November 8th, 2009 [November 8th, 2009]
- Stated Another Way....... - November 8th, 2009 [November 8th, 2009]
- Excuse Me? Harvard and Navigenics? WTF? - November 8th, 2009 [November 8th, 2009]
- Follow up to Yesterday's WTF? Harvard, Navi? and Pfizer??? - November 8th, 2009 [November 8th, 2009]
- Did you get your kit? Thanks Dr. Rob from MedCo - November 8th, 2009 [November 8th, 2009]
- Gluco...Wha? Parkinson's Disease and Glucocerebrosidase mutations. - November 8th, 2009 [November 8th, 2009]
- Away and now back, What did I miss???? 23andme layoffs? Selling Genomes for cheap up next! - November 8th, 2009 [November 8th, 2009]
- Change IS Needed. I agree with William, sometimes. - November 8th, 2009 [November 8th, 2009]
- Good Enough Science? Apparently so at 23andme - November 8th, 2009 [November 8th, 2009]
- Long QT Syndrome, location matters - December 13th, 2009 [December 13th, 2009]
- Congratulations Generation Health. Nice pick up! - December 13th, 2009 [December 13th, 2009]
- An argument 23andSerge can't win...23andme but not medicine - December 13th, 2009 [December 13th, 2009]
- Stop. Breathe. Repeat. An analysis of the direction of DTC Genomics Field. - December 13th, 2009 [December 13th, 2009]
- Hey DTC genomics, Stay Private, Stay Alive, Go Public and Die - December 13th, 2009 [December 13th, 2009]
- You can't have it both way. Either scared your genome is sold off or not. - December 13th, 2009 [December 13th, 2009]
- 15 Days Away Gives Time for Perspective. - December 13th, 2009 [December 13th, 2009]
- What about the SACGHS registry? Another missed opportunity? - December 13th, 2009 [December 13th, 2009]
- AJHG is in and my Favorite Muin is in it! But He Is NOT the Father! - December 13th, 2009 [December 13th, 2009]
- Navigenics for 23andMe prices? - December 18th, 2009 [December 18th, 2009]
- Lp(a) Maybe there's something there that wasn't there before? - December 24th, 2009 [December 24th, 2009]
- Another Year, Another Bankruptcy - December 31st, 2009 [December 31st, 2009]
- 5 Technologies going bye bye in this decade? - January 6th, 2010 [January 6th, 2010]
- Hackers, HITECH and HIPAA in DTC Genomics, Oh My! - January 7th, 2010 [January 7th, 2010]
- Personal Genomics Flop.....big Belly Flop! - January 8th, 2010 [January 8th, 2010]
- Gotta Love It. Even the daycare....... - January 11th, 2010 [January 11th, 2010]
- Congratulations Navigenics. You ARE a clinical lab! Uh-Oh... - January 12th, 2010 [January 12th, 2010]
- CETP, Jewish Centenarians and Alzheimers - January 14th, 2010 [January 14th, 2010]
- Enter the "Not" DTC Genomics Rep - January 17th, 2010 [January 17th, 2010]
- Why Dr. Vanier's Navigenics appointment is good for PM - January 22nd, 2010 [January 22nd, 2010]
- Holy Crap! MedCo Follows in CVS footsteps - February 3rd, 2010 [February 3rd, 2010]
- FDA, Warfarin, still not as sexy to me. - February 5th, 2010 [February 5th, 2010]
- Hype, Hype, Hype from a single study. - February 11th, 2010 [February 11th, 2010]
- I love my readers, even Renata M! - February 17th, 2010 [February 17th, 2010]
- How can insurers use DTC genomics to profile? - February 17th, 2010 [February 17th, 2010]
- 9p21.....ahem. Paynter et.al. Smackdown. Again. - February 18th, 2010 [February 18th, 2010]
- Hey! It's Pete Hulick! Are you Going to GET? - February 19th, 2010 [February 19th, 2010]
- I was wrong......AHEM - February 28th, 2010 [February 28th, 2010]
- G2C2, finally a tool for genomic education! - March 2nd, 2010 [March 2nd, 2010]
- Just 4 million? What 23andMe is worth. - March 5th, 2010 [March 5th, 2010]
- What a difference a year makes - March 9th, 2010 [March 9th, 2010]
- ........DTC Genomic Medicine? - March 12th, 2010 [March 12th, 2010]
- The FDA, 2c19 and the ACC - March 13th, 2010 [March 13th, 2010]
- The problem with Comparative Whole Genomics...... - March 13th, 2010 [March 13th, 2010]
- BRCA testing by 23andME is the same as Myriad Genetics. - March 15th, 2010 [March 15th, 2010]
- The Argument Against DTC Genomics Marketing and such - March 16th, 2010 [March 16th, 2010]
- A moment of Clarity. Some DTCG is not bad. - March 18th, 2010 [March 18th, 2010]
- SNPs for breast cancer risk? It Depends. - March 18th, 2010 [March 18th, 2010]
- How can MDVIP use Navigenics Test for Medicine? - March 18th, 2010 [March 18th, 2010]
- Why did P&G invest in Navigenics? - March 23rd, 2010 [March 23rd, 2010]
- PGx in DTCG? Doesn't stand up to Useful testing. - March 25th, 2010 [March 25th, 2010]
- End of Gene Patents? - March 29th, 2010 [March 29th, 2010]
- Sherpa Accepting Chief Medical Officership - April 3rd, 2010 [April 3rd, 2010]
- The Rumors of My Death........ - April 20th, 2010 [April 20th, 2010]
- Happy DNA Day! - April 25th, 2010 [April 25th, 2010]
- 99 USD, DNA day and patient letters - April 25th, 2010 [April 25th, 2010]
- 2C19, Navigenics and Clinical Reality. - May 1st, 2010 [May 1st, 2010]
- Coriell Personalized Medicine Collaborative rising - May 7th, 2010 [May 7th, 2010]
- Personal Genomes in Clinical Care. Quake paper is a waste! - May 11th, 2010 [May 11th, 2010]
- Personal Genomes in Clinical Care. Quake paper Falls Short! - May 13th, 2010 [May 13th, 2010]
- Last post edited by Drew - May 13th, 2010 [May 13th, 2010]
- GateKeeper? FCUK U! - May 13th, 2010 [May 13th, 2010]
- GateKeeper? F! U! - May 15th, 2010 [May 15th, 2010]
- Potential of genomic medicine, LOST - May 19th, 2010 [May 19th, 2010]
- How Bad Can a House Investigation be for DTC Genomics? - May 20th, 2010 [May 20th, 2010]