Anyone who follows the biotechnology industry surely is aware of the setback Cambridge, MA-based Sarepta Therapeutics (NASDAQ: [[ticker:SRPT]]) experienced this week after FDA changed their mind about the company’s ability to request early approval for its Duchenne Muscular Dystrophy drug, eteplirsen, on the basis of an encouraging Phase IIb study of 12 patients. However, the news appears to be considerably worse than the headline because there is also new doubt about what FDA will require of Sarepta’s Phase III study, the one needed to truly confirm the signal seen in the prior study.
The agency appears to be asking for new additional endpoints, a longer study duration, and a larger study population than what most people were expecting going into this. In other words, the company unfortunately has in many ways been sent back to the drawing board. Sarepta and FDA expect to formally discuss those things in the coming weeks, and hopefully will come to a speedy agreement.
Before getting to the main point I would like to make here, it is first important to recognize that there are no words to express how rightfully disappointed the Duchenne Muscular Dystrophy patient community must feel about this news. They currently have no good treatment options, and these patients undergo a steady decline before they typically die in their 20s. They deserve nothing less than urgency and clarity, yet seem to have come up on the short end of both of those things. That is particularly disappointing in this case because I have seen how they worked with all parties on this issue with dignity, class, and professionalism. Their contribution to this debate has truly been meaningful. I have the utmost confidence that all parties do place the highest importance on patients’ interests, and can only hope that will somehow bear itself out over the coming weeks and months in their favor.
With that being said, what I wanted to write about today is the job Sarepta CEO Chris Garabedian has done getting us to this point. No matter what the outcome, big news events like this bring out the “I told you so” crowd in full force, so it is only natural that some of those types of people will point the finger at him for this setback. The issue here is that some have said he should not have pushed for accelerated approval of eteplirsen as hard or openly as he did. While I don’t normally think those types of things are worth responding to, in this case I have also seen a handful of journalists, investors, and other commentators who I have the highest regard for say similar things, so I felt compelled to write something in his defense.
First of all, I think it is important to remember how we got here in the first place. Luke Timmerman has already done a great job here reporting about how Mr. Garabedian brought this company back from the brink a few years ago. Without his leadership, there arguably would have never been a phase IIb study to begin with. Therefore, I think it is important to put into context any debate we are having today about FDA decisions or early approvals, because they likely are a moot point without him. He and his team deserve a lot of credit and appreciation for even getting us here.
I do recognize that all of those previous accomplishments don’t change the fact that today we are left with a company that has suffered a significant setback, a DMD community that is sorely disappointed, and investors who have experienced a substantial decline of over 65 percent in one day (two quick disclosures: 1. I’m one of them and 2. investment losses PALE in comparison to what families of these kids are feeling right now). Add the fact that some critics are crowing loudly and you can see why a company’s leader will naturally be one of the first people to take some flack when news like this happens. However, take it from an investor who is usually one of the first people to loudly dish it in a case like this…I strongly believe any criticism is misplaced here. I’d like to offer three reasons why:
First, while many people have argued
