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Sarepta's DMD Portfolio Solid, Sole Drug Dependence Concerns
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On Oct 16, we issued an updated research report on Sarepta Therapeutics, Inc. (SRPT - Free Report) . This biopharmaceutical company, based in Cambridge, MA, is focused on the development of RNA-based therapeutics targeting rare and infectious diseases. The company’s sole marketed drug, Exondys 51, is indicated for the treatment of Duchenne muscular dystrophy (“DMD”).
Exondys 51 Performing Well
Sarepta received accelerated approval for Exondys 51 in 2016 for treating patients who have a confirmed mutation of the DMD gene susceptible to exon 51 skipping in the United States. The drug continued its strong growth in 2018 with sales soaring almost 170% in the first half of 2018 compared with the year-ago period. It increased 13.8% sequentially to $73.5 million in the second quarter of 2018. The increase was fueled by strong demand trends. The company expects the drug’s sales to reach $300 million this year.
The company is carrying out activities to further boost the drug’s sales. Sarepta has launched a Managed Access Program in Europe and Americas to increase accessibility of Exondys 51. The drug is under review in Europe. The settlement of a patent litigation with BioMarin Pharmaceutical Inc. (BMRN - Free Report) in 2017 also helped Sarepta as it now owns global exclusive rights for Exondys 51.
However, the company’s dependence on this drug for growth is a concern as a downfall may severely impact the stock. The majority of Sarepta’s pipeline candidates are in early to mid-stage development. Last month, the company suffered a small setback when the Committee for Medicinal Products for Human Use of the European Medicines Agency upheld its earlier negative opinion for marketing approval of Exondys 51.
Exon-Skipping Pipeline Progress
The company is building its DMD pipeline, which will enhance its approved drug portfolio. The pipeline candidates, upon approval, will be eligible for larger patient population than Exondys 51. The company has about eight exon-skipping pipeline candidates, which can treat 75-80% of DMD patients.
Golodirsen, the lead DMD candidate, showed better improvement in patients in a mid-stage clinical study compared to results achieved by Exondys 51. In fact, golodirsen increased mean dystrophin protein production by 10.7 times from baseline against nearly three times for Exondys 51. A phase III study is currently enrolling patients. Sarepta is on track to file a new drug application seeking approval for golodirsen in the United States by the end of 2018.
Gene Therapies
Apart from exon-skipping candidates, Sarepta is also focusing on developing gene therapies for treating DMD. The gene therapies have shown promising results so far in treating DMD patients by restoring dystrophin production in muscles throughout the body by delivering microdystrophin via adeno-associated virus. A successful development will create a treatment option eligible for all DMD patients. Sarepta is collaborating with other companies to boost its pipeline or manufacturing capacity for gene therapy.
Sarepta is evaluating AAVrh74.MHCK7.micro-Dystrophin in a phase I/II study in DMD patients. Preliminary data announced in June showed that the gene therapy dramatically reduced creatine kinase levels, a measure of abnormal muscle damage, and created significant levels of dystrophin.
However, these are very early results for the candidate and there is a long way to go before it becomes a potential treatment option. In July, a clinical hold was placed on the study due to the presence of trace amounts of DNA fragment in research-grade third-party supplied plasmid. The hold was lifted last month. Moreover, there are other companies developing gene therapies for treating DMD, which includes Pfizer (PFE - Free Report) , Solid Biosciences (SLDB - Free Report) and Scholar Rock.
Conclusion
Sarepta’s top line is expected to grow on the back of strong demand for Exondys 51. However, approval to any new drug for DMD will impact sales. The company is progressing well with development of golodirsen, which has shown better efficacy than Exondys 51. A successful commercialization upon approval will boost prospects of the company. It is also developing gene therapy for treating DMD. But these are in early stage of development and therefore there is still some time before any of these are approved and commercialized.
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Sarepta's DMD Portfolio Solid, Sole Drug Dependence Concerns
On Oct 16, we issued an updated research report on Sarepta Therapeutics, Inc. (SRPT - Free Report) . This biopharmaceutical company, based in Cambridge, MA, is focused on the development of RNA-based therapeutics targeting rare and infectious diseases. The company’s sole marketed drug, Exondys 51, is indicated for the treatment of Duchenne muscular dystrophy (“DMD”).
Exondys 51 Performing Well
Sarepta received accelerated approval for Exondys 51 in 2016 for treating patients who have a confirmed mutation of the DMD gene susceptible to exon 51 skipping in the United States. The drug continued its strong growth in 2018 with sales soaring almost 170% in the first half of 2018 compared with the year-ago period. It increased 13.8% sequentially to $73.5 million in the second quarter of 2018. The increase was fueled by strong demand trends. The company expects the drug’s sales to reach $300 million this year.
The company is carrying out activities to further boost the drug’s sales. Sarepta has launched a Managed Access Program in Europe and Americas to increase accessibility of Exondys 51. The drug is under review in Europe. The settlement of a patent litigation with BioMarin Pharmaceutical Inc. (BMRN - Free Report) in 2017 also helped Sarepta as it now owns global exclusive rights for Exondys 51.
However, the company’s dependence on this drug for growth is a concern as a downfall may severely impact the stock. The majority of Sarepta’s pipeline candidates are in early to mid-stage development. Last month, the company suffered a small setback when the Committee for Medicinal Products for Human Use of the European Medicines Agency upheld its earlier negative opinion for marketing approval of Exondys 51.
Exon-Skipping Pipeline Progress
The company is building its DMD pipeline, which will enhance its approved drug portfolio. The pipeline candidates, upon approval, will be eligible for larger patient population than Exondys 51. The company has about eight exon-skipping pipeline candidates, which can treat 75-80% of DMD patients.
Golodirsen, the lead DMD candidate, showed better improvement in patients in a mid-stage clinical study compared to results achieved by Exondys 51. In fact, golodirsen increased mean dystrophin protein production by 10.7 times from baseline against nearly three times for Exondys 51. A phase III study is currently enrolling patients. Sarepta is on track to file a new drug application seeking approval for golodirsen in the United States by the end of 2018.
Gene Therapies
Apart from exon-skipping candidates, Sarepta is also focusing on developing gene therapies for treating DMD. The gene therapies have shown promising results so far in treating DMD patients by restoring dystrophin production in muscles throughout the body by delivering microdystrophin via adeno-associated virus. A successful development will create a treatment option eligible for all DMD patients. Sarepta is collaborating with other companies to boost its pipeline or manufacturing capacity for gene therapy.
Sarepta is evaluating AAVrh74.MHCK7.micro-Dystrophin in a phase I/II study in DMD patients. Preliminary data announced in June showed that the gene therapy dramatically reduced creatine kinase levels, a measure of abnormal muscle damage, and created significant levels of dystrophin.
However, these are very early results for the candidate and there is a long way to go before it becomes a potential treatment option. In July, a clinical hold was placed on the study due to the presence of trace amounts of DNA fragment in research-grade third-party supplied plasmid. The hold was lifted last month. Moreover, there are other companies developing gene therapies for treating DMD, which includes Pfizer (PFE - Free Report) , Solid Biosciences (SLDB - Free Report) and Scholar Rock.
Conclusion
Sarepta’s top line is expected to grow on the back of strong demand for Exondys 51. However, approval to any new drug for DMD will impact sales. The company is progressing well with development of golodirsen, which has shown better efficacy than Exondys 51. A successful commercialization upon approval will boost prospects of the company. It is also developing gene therapy for treating DMD. But these are in early stage of development and therefore there is still some time before any of these are approved and commercialized.
Wall Street’s Next Amazon
Zacks EVP Kevin Matras believes this familiar stock has only just begun its climb to become one of the greatest investments of all time. It’s a once-in-a-generation opportunity to invest in pure genius.
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