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Sarepta: Revenue Keeps Rising While We Wait For Functional Data

Nov. 01, 2021 8:29 AM ETSarepta Therapeutics, Inc. (SRPT)4 Comments


  • SRPT has continued growing its revenue line despite not producing placebo compared data.
  • The stock dropped hugely in January after a trial failure and hasn't recovered since.
  • Some have asked if they even need to produce placebo-controlled trial data.
  • Looking for more investing ideas like this one? Get them exclusively at The Total Pharma Tracker. Learn More »

House Oversight And Reform Committee Hears Testimony From Patients On The Impact Of Rising Drug Prices

Chip Somodevilla/Getty Images News

Like I said about Sarepta (NASDAQ:SRPT) in my previous coverage quoting Evaluate, "A remarkable fact about Sarepta, until yesterday a $13bn company, is that it had never demonstrated the efficacy of any of its projects in

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Comments (4)

One absolutely needs double blinded trials. Just because there is anecdotal evidence that something works does not mean it does. Whether it be hanging garlic around your neck to ward off the plague or many of the fad treatments that come and go there is always anecdotal evidence of efficacy.(remember St John's Wort? - was supposed to have replaced prescribed antidepressants and I recall a Yale Professor in Psychiatry declare " the pharmaceutical companies are shaking in their boots" !!) Of course there is anecdotal evidence of Elvis sighting, JFK making an appearance, bleach and horse deworming pills for Covid-19. Anyone presenting just anecdotal evidence and refusing a double blind trial is a snake oil salesperson
The placebo controlled Essence trial will probably release functional and other data in 2024. Top line data was already presented in the approval process for 45. Meanwhile, boys on commercial 51 and 53 are apparently doing better than history because they continue to get weekly infusions. Amazingly, they don’t seem to care what the share price is at any given time. It is what it is, be patient.
The Covid vaccines have shown that the FDA has a bias toward Pfizer. Is it corruption? I don’t know, but it sure seems like it. And right now Pfizer is the primary competitor of SRPT. The science of SRPT seems to be very good. But given the history of the FDA, I would be much more likely to be long SRPT if it were competing against a company other than Pfizer.
The original model for SRPT has always been to mimick the natural mutation seen in rare DMD patients that had the gene but also the exon skipping mutation. These patients had low dystrophin levels but we're otherwise asymptomatic. If it worked for nature it should work as a therapy provided enough cells could be reached in vivo. The percentages look promising but durable functionality will take time to demonstrate.
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