) -- The end of the year is upon us, which means I'm starting to consider nominations for the best and worst biotech CEOs of 2012. If you have suggestions, please send them my way.
Mitch Gold took worst CEO dishonors last year. Best CEO went to
Matt Emmens and
held its third-quarter conference call Wednesday night, prompting a good bit of discussion and comments on Twitter.
This week's Biotech Stock Mailbag opens with a tweet from @patrick_vr:
"Do you think there is a catalyst for the stock to go higher over the next several months?"
I'm glad you asked me about a three-month time horizon and not three minutes. Thank you.
Sarepta's most important upcoming catalyst is the "end of phase II" meeting with the FDA. This is the sit-down where Sarepta will discuss with FDA officials the collective preclinical and clinical data on eteplirsen (most importantly, the results of the phase IIb study). In return, FDA will offer advice and guidance on the next steps. Sarepta hopes to emerge from the meeting with FDA blessing to seek accelerated approval for eteplirsen, based on the clinical data in hand.
This meeting between Sarepta and FDA is expected to take place in the first quarter of 2013, said Sarepta CEO Chris Garabedian on last night's call, which is a reiteration of the timeline he's talked about previously.
Do not expect, on the day of this FDA meeting, Sarepta to immediately issue a press release announcing what the agency's regulators told the company. Sarepta is more likely to wait for FDA's formal written comments and minutes from the meeting before it lets us all know if the eteplirsen accelerated approval filing plan is a go -- or not.
My best guess is March 2013. That's a bit longer than three months from today but not by much.
My second-best guess is FDA agrees to an accelerated approval filing for eteplirsen. If I'm right, Sarepta shares will go higher.
Five reasons for my Sarepta bullishness: 1) The "exon-skipping" mechanism by which eteplirsen works is supported by the increase in functional dystrophin and the improved walking ability of DMD patients. The clinical data are strong and convincing. 2) Eteplirsen's safety profile is excellent, making this a relatively easy decision on a risk-benefit analysis. 3) DMD advocates and patients are
lining up to support Sarepta and etelirsen
. Their collective voices matter and will make a difference. 4) Sarepta's management team, led by Garabedian, is top-notch. I'm confident they'll make a convincing argument to FDA. 5) FDA has nothing to lose -- but so much to gain politically -- by approving eteplirsen early.