We use cookies to understand how you use our site and to improve your experience. This includes personalizing content and advertising. To learn more, click here. By continuing to use our site, you accept our use of cookies, revised Privacy Policy and Terms of Service.
You are being directed to ZacksTrade, a division of LBMZ Securities and licensed broker-dealer. ZacksTrade and Zacks.com are separate companies. The web link between the two companies is not a solicitation or offer to invest in a particular security or type of security. ZacksTrade does not endorse or adopt any particular investment strategy, any analyst opinion/rating/report or any approach to evaluating individual securities.
If you wish to go to ZacksTrade, click OK. If you do not, click Cancel.
Sarepta (SRPT) Begins Pivotal Study on DMD Gene Therapy
Read MoreHide Full Article
Sarepta Therapeutics, Inc. (SRPT - Free Report) announced that it has initiated a pivotal study — EMBARK — on its lead gene therapy candidate, SRP-9001, for the treatment of Duchenne muscular dystrophy (“DMD”). The company is developing the micro-dystrophin-encoding gene therapy candidate in collaboration with Roche (RHHBY - Free Report) .
SRP-9001 is one of the leading investigational gene therapies that is being developed for treating DMD and the EMBARK study is the first pivotal study to evaluate any gene therapy for the indication. A successful development of SRP-9001 will provide a one-time treatment option for the debilitating disease affecting mainly children. The study will be conducted in the United States, Europe and Asia.
The company completed a successful end-of-phase-II meeting with the FDA’s Office of Tissues and Advanced Therapies in July. It discussed its proposed design for the pivotal study in the meeting.
Shares of Sarepta are down 44.5% so far this year compared with the industry’s decline of 9%.
Image Source: Zacks Investment Research
Sarepta’s mid-stage study evaluating SRP-9001 evaluating it in DMD patients had mixed results. While the study met its primary biological endpoint of micro-dystrophin protein expression, it failed to achieve the statistical significance for the primary functional endpoint. This led to a major drop in Sarepta’s shares in January 2021. However, data from the study had demonstrated a favorable trend for the primary functional endpoint, as measured by the 17-item North Star Ambulatory Assessment (NSAA) total score.
In May, the company announced data from an open-label study — ENDEAVOR — that evaluated commercially representative material for SRP-9001. Data from this study demonstrated robust expression of micro-dystrophin, a protein that helps to strengthen muscle fibers in patients after 12 weeks of a single infusion of SRP-9001 at a dose of 1.33x1014 vg/kg. The study will continue to follow the patient dosed in the study for five years, including the initial 12-week period.
We note that Sarepta has a strong commercial DMD portfolio with three marketed drugs — Exondys 51, Vyondys 53 and Amondys 45 — developed using its exon-skipping technology. The company is also developing a next-generation exon-skipping DMD candidate, SRP-5051. The candidate has demonstrated tremendous potential as a treatment for DMD. Moreover, the successful development of SRP-9001 will further boost Sarepta’s DMD dominance. However, other companies like Solid Biosciences (SLDB - Free Report) and pharma bigwig Pfizer (PFE - Free Report) are also developing gene therapies for treating DMD. The successful development of gene therapies for DMD will increase competition for Sarepta in the long run.
Image: Bigstock
Sarepta (SRPT) Begins Pivotal Study on DMD Gene Therapy
Sarepta Therapeutics, Inc. (SRPT - Free Report) announced that it has initiated a pivotal study — EMBARK — on its lead gene therapy candidate, SRP-9001, for the treatment of Duchenne muscular dystrophy (“DMD”). The company is developing the micro-dystrophin-encoding gene therapy candidate in collaboration with Roche (RHHBY - Free Report) .
SRP-9001 is one of the leading investigational gene therapies that is being developed for treating DMD and the EMBARK study is the first pivotal study to evaluate any gene therapy for the indication. A successful development of SRP-9001 will provide a one-time treatment option for the debilitating disease affecting mainly children. The study will be conducted in the United States, Europe and Asia.
The company completed a successful end-of-phase-II meeting with the FDA’s Office of Tissues and Advanced Therapies in July. It discussed its proposed design for the pivotal study in the meeting.
Shares of Sarepta are down 44.5% so far this year compared with the industry’s decline of 9%.
Sarepta’s mid-stage study evaluating SRP-9001 evaluating it in DMD patients had mixed results. While the study met its primary biological endpoint of micro-dystrophin protein expression, it failed to achieve the statistical significance for the primary functional endpoint. This led to a major drop in Sarepta’s shares in January 2021. However, data from the study had demonstrated a favorable trend for the primary functional endpoint, as measured by the 17-item North Star Ambulatory Assessment (NSAA) total score.
In May, the company announced data from an open-label study — ENDEAVOR — that evaluated commercially representative material for SRP-9001. Data from this study demonstrated robust expression of micro-dystrophin, a protein that helps to strengthen muscle fibers in patients after 12 weeks of a single infusion of SRP-9001 at a dose of 1.33x1014 vg/kg. The study will continue to follow the patient dosed in the study for five years, including the initial 12-week period.
We note that Sarepta has a strong commercial DMD portfolio with three marketed drugs — Exondys 51, Vyondys 53 and Amondys 45 — developed using its exon-skipping technology. The company is also developing a next-generation exon-skipping DMD candidate, SRP-5051. The candidate has demonstrated tremendous potential as a treatment for DMD. Moreover, the successful development of SRP-9001 will further boost Sarepta’s DMD dominance. However, other companies like Solid Biosciences (SLDB - Free Report) and pharma bigwig Pfizer (PFE - Free Report) are also developing gene therapies for treating DMD. The successful development of gene therapies for DMD will increase competition for Sarepta in the long run.
Sarepta Therapeutics, Inc. Price
Sarepta Therapeutics, Inc. price | Sarepta Therapeutics, Inc. Quote
Zacks Rank
Sarepta currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.