$AMD 50% rise was expected. The after-market spike on Thursday night spooked me away, but I should know that something up on a couple different good news topics + earnings + forming the handle of a cup is going to spike all day! Especially Friday.

Anyway...Lessons.

Update:: It has been brought to my attention that $SRPT will most likely be halted Monday during the FDA decision, which I am so bummed about! I think it would have been a great short.

$SRPT Sarepta spiked 35% mid-day on this news: http://www.reuters.com/article/us-sarepta-fda-idUSKCN0XJ29V

   Basically, the FDA staff reviewers gave a scathing review of their muscle drug up for a New Drug Application yesterday (Thursday) which crashed the stock. Quote: "“The data overall did not provide statistical evidence to support the efficacy in subjects who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping.” 
The FDA staff's opinion is really all that matters with approving the drug or not. However, the FDA has to do everything by the books, and the next step is for an INDEPENDENT PANEL of scientists to give feedback on the drug's TRIAL DATA. The FDA staffers specifically stated yesterday that they didn't want to hear the independent panel's recommendation opinions of the drug, only feedback on the trial's data (which the FDA hated with a fiery passion). 

   Mid-day today for whatever reason, they said hey, lets go ahead and ask the panel 5 questions after their review of the trial data, and these questions will get at their recommendation of whether we should approve this drug. This gave dumb people HOPE! Which spiked the stock into the close. 

Here are the questions from http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/PeripheralandCentralNervousSystemDrugsAdvisoryCommittee/UCM497241.pdf :

1. Has the Applicant provided substantial evidence from adequate and well controlled studies that eteplirsen induces production of dystrophin to a level that is reasonably likely to predict clinical benefit?

Answer: NO
Study 201, when analyzed according to the pre-specified intent-to-treat (ITT) methods, did not show an advantage of eteplirsen over placebo on the 6‐minute walk test (6MWT) after 24 weeks of treatment. 
When analyzed according to the prespecified ITT methods, Study 202 did not demonstrate an advantage of eteplirsen over placebo on the 6‐minute walk test.

The Applicant then continued open-label treatment with eteplirsen in Study 202, which is still ongoing, and is seeking approval primarily based on a post hoc comparison of 12 patients from Study 201 to 13 patients from an untreated external control group amenable to exon 51 skipping (from two DMD patient registries, the “Italian Telethon DMD Registry” database and the “Leuven Neuromuscular Reference Center” database). Because of difficulty of controlling bias in historical control studies, important issues to consider include: 1) whether there are identified or possible differences between the treatment and control groups, at baseline or during treatment, that may have had an impact on clinical course; 2) whether the endpoint(s) used to assess benefit was (were) objective and assessed in a sufficiently similar way in the treatment and control groups to allow a valid comparison; and 3) whether the reported effect size is large enough to conclude that the course of patients in Study 201/202 is clearly different from the usual course of patients with DMD.

3. DISCUSSION: Discuss the strengths and weaknesses of the clinical evidence of efficacy provided by Study 201/202, with particular consideration of the design of the study, sample size, statistical methods, general concerns regarding a comparison to a historical control group, specific concerns with respect to the comparability of these two groups (in particular, how motivational factors and differences in assessment of physical performance outcomes may have affected the 6-minute walk endpoint and other endpoints), and any other issues that you think may be important.

4. VOTE: Were decisions to administer the 6-minute walk test (vs. conclusions that the patient could no longer walk) sufficiently objective and free of bias and subjective decision-making by patients, their caregivers, and/or health care professionals to allow for a valid comparison between patients in Study 201/202 and an external control group?

5. VOTE: What is the impact of the North Star Ambulatory Assessment results on the persuasiveness of the findings in Study 201/202? a. Strengthen b. Weaken c. No effect

6. VOTE: What is the impact of the other tests of physical performance (e.g., rise time, 10-meter run/walk) on the persuasiveness of findings in Study 201/202? a. Strengthen b. Weaken c. No effect

7. VOTE: Do the clinical results of the single historically-controlled study (Study 201/202) provide substantial evidence (i.e., evidence from adequate and well-controlled studies or evidence from a single highly persuasive adequate and well-controlled study that is accompanied by independent findings that substantiate efficacy) that eteplirsen is effective for the treatment of DMD?

 

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I may not be a doctor, but I am a graduate student in the field of Wildlife Ecology and I understand study design. This study doesn't even have basic study design. Essentially, they only have 6 boys to take data from.
It sits at $14.95 from a low of around $11 this morning, and it's already up to 15.26 in afterhours! 

If only this could be shorted on Monday :((

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Quote from Forbes yesterday: The story of Sarepta and eteplirsen should also stand as a warning to those who seek to speed the approval of experimental drugs to market using smaller, faster studies. In some cases a lower regulatory bar is a good thing. But the worst thing is when it seems the regulatory bar has gone down when it hasn’t. That means companies don’t do the work they need to do, and patients don’t get the medicines they need. More patients will suffer because of regulatory mirages than regulatory walls.

Sarepta decided to try to get FDA approval based on this small study. The company made public comments that indicated that, in private meetings, the FDA was okay with this decision. The company also said that, in order to confirm the drug works as expected, it would not conduct a study comparing eteplirsen to placebo. Instead, it would compare eteplirsen patients to patients with another mutation who wouldn’t respond to eteplirsen. Sarepta indicated that the FDA had approved this approach, too.

In briefing documents first released in January and update today, the FDA says that it had always expressed qualms about this approach.

Here’s what the FDA says.

FDA strongly encouraged the sponsor to conduct an adequately powered, randomized, placebo‐controlled trial(s) to assess the clinical effect of eteplirsen. But in the context of an ongoing series of reports from the applicant and its academic associates describing marked effects on dystrophin production and stabilization of disease progression, many in the DMD community had strong reservations regarding the ethics and practicality of conducting another placebo‐controlled trial of eteplirsen. Given the apparent difficulty of doing such a trial, FDA expressed willingness to consider an externally controlled trial, although stating clearly that interpretation of the data could be difficult, and that the acceptability of the study would be a matter for NDA review.

In other words, the FDA acquiesced to a study it does not know if it will believe because of pressure created by public statements by Sarepta, by FDA officials own meetings with patient advocates, and by the fact that Sarepta’s statements on its meetings with the FDA went publicly uncorrected.