Celldex Therapeutics (NASDAQ:CLDX)
On Monday March 7, 2016 shares of Celldex fell as much as 53% after the company announced that it had to discontinue a phase 3 trial. The reason for discontinuing the trial was because of insufficient efficacy needed to move on. This phase 3 trial recruited a total of 745 patients who had newly diagnosed glioblastoma — brain cancer. There is however a catch, and that is that these glioblastoma patients had the EFGRvIII mutation.
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When the patients entered the trial they were first put into a standard of care treatment. That means that patients had their tumor removed by surgery, and were then given a standard of care cancer drug. Once finished with that regimen, patients were randomized into two different dosing groups. Patients either received Rintega together with chemotherapy or an injection control drug together with chemotherapy.
The trial wasn’t halted on the merits of the company itself. Instead, it was stopped by the Data Safety and Monitoring Board or DSMB. It concluded that the trial didn’t reach its primary endpoint, and even if the trial was allowed to finish all the way to completion it still wouldn’t change that fact. Thus, Celldex chose to terminate the trial on the recommendation of the DSMB. While most patients will be booted from the trial, they will have the option to continue treatment based off of a compassionate use basis only.
The Rintega and chemotherapy regimen was only able to reduce the risk of death by 1%. The primary endpoint of the trial was to achieve overall survival improvement compared to the placebo regimen. The Rintega combo achieved an overall survival of 20.4 months which is good, but the placebo regimen in the trial got 21.1 months. The good news out of this is that the company will now save a lot of money by not having to run the phase 3 trial. The bad news is that the company has no other drug in phase 3 for the time being. That means it will possibly be a few years now before another phase 3 trial is initiated. Although, this will only happen with new successful results in another phase 3 trial, and that is the risk.
Sarepta Therapeutics (NASADAQ:SRPT)
On Thursday March 10, 2016 shares of Sarepta finished the day up 15% when the company announced that the FDA had set a new FDA panel review date for its drug Eteplirsen. This new date had to be set because the previous date was cancelled due to a potential weather storm. The new meeting date is set to take place in April 25 of this year, where the panel will review the drug and determine if they should recommend for approval.
One thing to keep in mind here is that this FDA panel is just a preliminary review, and they only give their recommendation to the FDA body itself. That means that the ultimate decision will be handed out by the FDA body itself on May 26 of this year. The FDA doesn’t have to accept or take the recommendation of the panel, but they typically do. Still, it is not a guarantee that Eteplirsen will be approved therefore investors should remain cautious.
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Before the meeting was cancelled, the FDA body itself had given a preliminary analysis of Eteplirsen stating that they are hesitant about the efficacy shown to date. This is especially true since a lot of the efficacy data only comes from a small 12 patient run trial. There are currently no FDA approved drugs for DMD so it is possible that the FDA might be more lenient than normal when deciding upon approval. Another factor is that a few other companies have already had their drugs for DMD rejected by the FDA. This means that it is possible the FDA may want to at least approve one. Again, to reiterate the point that it is not guaranteed that the FDA will approve. It is best to somehow hedge your position going into this review to avoid potential losses.
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