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Sarepta (SRPT) Rises on Robust DMD Gene Therapy Study Data
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Sarepta Therapeutics, Inc. (SRPT - Free Report) announced promising data from an open-label study — ENDEAVOR — evaluating a single administration of its lead gene therapy candidate, SRP-9001, in children with Duchenne muscular dystrophy (DMD), a progressive muscular weakness primarily affecting boys.
Data from the 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.
Please note that the ENDEAVOR study is the first clinical study to evaluate the commercially representative material for SRP-9001. The study is being conducted in partnership with Roche (RHHBY - Free Report) . Sarepta has previously reported data from clinical studies that evaluated clinical manufacturing process material for SRP-9001. A commercially representative material will likely help the company to build an at-scale gene therapy manufacturing process for SRP-9001.
Shares of Sarepta gained 8.4% on May 18, following the news release. However, the company’s shares are down 52.2% compared with the industry’s decline of 5.6%.
The ENDEAVOR study dosed 20 patients, aged four to seven years, so far with SRP-9001. Data from muscle biopsies of first 11 participants who completed 12 weeks of treatment showed robust transduction, with mean micro-dystrophin expression of 55.4% of normalas measured by western blot, in all 11 patients. Moreover, micro-dystrophin was properly localized to a specialized cell membrane known as sarcolemma. The treatment with SRP-9001 increased mean muscle fibers expressing micro-dystrophin to 70.5% from 12.8% at baseline. Intensity of micro-dystrophin expression was 116.9% of normal control, compared with 41% at baseline.
Moreover, the safety profile of commercial-grade SRP-9001 was consistent with prior studies and no new safety signals were identified.
The study will continue to follow the patient dosed in the study for five years, including the initial 12-week period.
However, we remind investors Sarepta received a major setback in January earlier this year when a study evaluating clinical manufacturing process material for SRP-9001 failed to achieve statistical significance for its primary functional endpoint.However, the study met its primary biological endpoint of micro-dystrophin protein expression, with a mean of 28.1%.
Meanwhile 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. Moreover, 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, 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. Successful development of gene therapies for DMD will increase competition for Sarepta in the long run.
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Most of the stocks in this report are flying under Wall Street radar, which provides a great opportunity to get in on the ground floor.
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Sarepta (SRPT) Rises on Robust DMD Gene Therapy Study Data
Sarepta Therapeutics, Inc. (SRPT - Free Report) announced promising data from an open-label study — ENDEAVOR — evaluating a single administration of its lead gene therapy candidate, SRP-9001, in children with Duchenne muscular dystrophy (DMD), a progressive muscular weakness primarily affecting boys.
Data from the 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.
Please note that the ENDEAVOR study is the first clinical study to evaluate the commercially representative material for SRP-9001. The study is being conducted in partnership with Roche (RHHBY - Free Report) . Sarepta has previously reported data from clinical studies that evaluated clinical manufacturing process material for SRP-9001. A commercially representative material will likely help the company to build an at-scale gene therapy manufacturing process for SRP-9001.
Shares of Sarepta gained 8.4% on May 18, following the news release. However, the company’s shares are down 52.2% compared with the industry’s decline of 5.6%.
The ENDEAVOR study dosed 20 patients, aged four to seven years, so far with SRP-9001. Data from muscle biopsies of first 11 participants who completed 12 weeks of treatment showed robust transduction, with mean micro-dystrophin expression of 55.4% of normalas measured by western blot, in all 11 patients. Moreover, micro-dystrophin was properly localized to a specialized cell membrane known as sarcolemma. The treatment with SRP-9001 increased mean muscle fibers expressing micro-dystrophin to 70.5% from 12.8% at baseline. Intensity of micro-dystrophin expression was 116.9% of normal control, compared with 41% at baseline.
Moreover, the safety profile of commercial-grade SRP-9001 was consistent with prior studies and no new safety signals were identified.
The study will continue to follow the patient dosed in the study for five years, including the initial 12-week period.
However, we remind investors Sarepta received a major setback in January earlier this year when a study evaluating clinical manufacturing process material for SRP-9001 failed to achieve statistical significance for its primary functional endpoint.However, the study met its primary biological endpoint of micro-dystrophin protein expression, with a mean of 28.1%.
Meanwhile 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. Moreover, 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, 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. 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.
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Most of the stocks in this report are flying under Wall Street radar, which provides a great opportunity to get in on the ground floor.
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