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Sarepta Surges on Rival Pfizer's DMD Gene Therapy Study Data
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Shares of Sarepta Therapeutics, Inc. (SRPT - Free Report) increased 11.2% on Friday, following the announcement of updated data from Pfizer’s (PFE - Free Report) early-stage gene therapy study, rivaling Sarepta’s gene therapy, as a treatment for Duchenne muscular dystrophy (“DMD”). Data from Pfizer’s study seems to fall short compared to Sarepta’s.
Please note that Sarepta is a leading company in the field of DMD with two FDA-approved drugs for the disease, which causes muscle degeneration leading to difficulty in walking. However, there are no approved gene therapies for DMD. Sarepta is developing SRP-9001, an AAV-mediated micro-dystrophin gene therapy candidate, in a phase I/II study for treating DMD. Pfizer’s gene therapy, PF-06939926, is being evaluated in a phase Ib study to treat DMD.
Data from Pfizer’s study showed that following treatment with PF-06939926, for a year, the patients in the low dose cohort achieved 24% of normal dystrophin at 12 months. In the high dose cohort, the gene therapy achieved 51.6% of normal dystrophin. Moreover, the gene therapy improved North Star Ambulatory Assessment (“NSAA”) score by 3.5 points from baseline compared to median 4 points decline in NSAA score for patients not receiving the gene therapy. The data for the comparator group was derived from external clinical studies on DMD patients not conducted by Pfizer.
Data showed statistically significant improvement in NSAA score as well as dystrophin levels. Pfizer is also planning to initiate a phase III study in the second half of 2020 based on these data, skipping the phase II stage. However, Sarepta’s SRP-9001 achieved improvement of 6.5 points in NSAA score from baseline, based on previously announced interim data from the early-stage study evaluating the candidate. Moreover, Pfizer reported three serious adverse events including dehydration, acute kidney injury and thrombocytopenia. Acute kidney injury and thrombocytopenia patients also had atypical hemolytic uremic syndrome (aHUS)-like complement activation, and required treatment with Alexion’s Soliris (eculizumab).
Although analysts believe it is difficult to compare data from Sarepta’s and Pfizer’s study on their DMD gene therapy candidates due to different study designs, Sarepta’s stock is likely to have rallied due to NSAA score superiority and safety issues related to Pfizer’s gene therapy. However, data from both companies are from early-stage studies and what differentiation is going to emerge is too early to predict.
Shares of Sarepta have increased 9.4% so far this year compared with the industry’s 7.9%.
Please note that Sarepta signed a licensing agreement with Roche (RHHBY - Free Report) granting exclusive rights to launch and commercialize SRP-9001 in ex-U.S. markets in exchange of an upfront payment of $1.15 billion last year. This deal boosted Sarepta’s cash resources and helped it to leverage Roche’s global reach, commercial presence and regulatory expertise to accelerate approval and commercialization process for SRP-9001.
Meanwhile, Sarepta is also developing several other gene therapies targeting Limb-girdle muscular dystrophy, Mucopolysaccharidosis type IIIA and Charcot-Marie-Tooth disease type 1A.
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Sarepta Surges on Rival Pfizer's DMD Gene Therapy Study Data
Shares of Sarepta Therapeutics, Inc. (SRPT - Free Report) increased 11.2% on Friday, following the announcement of updated data from Pfizer’s (PFE - Free Report) early-stage gene therapy study, rivaling Sarepta’s gene therapy, as a treatment for Duchenne muscular dystrophy (“DMD”). Data from Pfizer’s study seems to fall short compared to Sarepta’s.
Please note that Sarepta is a leading company in the field of DMD with two FDA-approved drugs for the disease, which causes muscle degeneration leading to difficulty in walking. However, there are no approved gene therapies for DMD. Sarepta is developing SRP-9001, an AAV-mediated micro-dystrophin gene therapy candidate, in a phase I/II study for treating DMD. Pfizer’s gene therapy, PF-06939926, is being evaluated in a phase Ib study to treat DMD.
Data from Pfizer’s study showed that following treatment with PF-06939926, for a year, the patients in the low dose cohort achieved 24% of normal dystrophin at 12 months. In the high dose cohort, the gene therapy achieved 51.6% of normal dystrophin. Moreover, the gene therapy improved North Star Ambulatory Assessment (“NSAA”) score by 3.5 points from baseline compared to median 4 points decline in NSAA score for patients not receiving the gene therapy. The data for the comparator group was derived from external clinical studies on DMD patients not conducted by Pfizer.
Data showed statistically significant improvement in NSAA score as well as dystrophin levels. Pfizer is also planning to initiate a phase III study in the second half of 2020 based on these data, skipping the phase II stage. However, Sarepta’s SRP-9001 achieved improvement of 6.5 points in NSAA score from baseline, based on previously announced interim data from the early-stage study evaluating the candidate. Moreover, Pfizer reported three serious adverse events including dehydration, acute kidney injury and thrombocytopenia. Acute kidney injury and thrombocytopenia patients also had atypical hemolytic uremic syndrome (aHUS)-like complement activation, and required treatment with Alexion’s Soliris (eculizumab).
Although analysts believe it is difficult to compare data from Sarepta’s and Pfizer’s study on their DMD gene therapy candidates due to different study designs, Sarepta’s stock is likely to have rallied due to NSAA score superiority and safety issues related to Pfizer’s gene therapy. However, data from both companies are from early-stage studies and what differentiation is going to emerge is too early to predict.
Shares of Sarepta have increased 9.4% so far this year compared with the industry’s 7.9%.
Please note that Sarepta signed a licensing agreement with Roche (RHHBY - Free Report) granting exclusive rights to launch and commercialize SRP-9001 in ex-U.S. markets in exchange of an upfront payment of $1.15 billion last year. This deal boosted Sarepta’s cash resources and helped it to leverage Roche’s global reach, commercial presence and regulatory expertise to accelerate approval and commercialization process for SRP-9001.
Meanwhile, Sarepta is also developing several other gene therapies targeting Limb-girdle muscular dystrophy, Mucopolysaccharidosis type IIIA and Charcot-Marie-Tooth disease type 1A.
Sarepta Therapeutics, Inc. Price
Sarepta Therapeutics, Inc. price | Sarepta Therapeutics, Inc. Quote
Zacks Rank
Sarepta currently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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This outperformance has not just been a recent phenomenon. From 2000 – 2019, while the S&P averaged +6.0% per year, our top strategies averaged up to +54.7% per year.
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