) -- 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
. 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.
"Be nice if $SRPT gave us some insight as to when they might share some 62-week data that was taken a couple weeks ago."
Agreed. I rank more data from the eteplirsen phase IIb study as the second-most significant catalyst looming for Sarepta.
Eteplirsen doubters probably rank this first, understandably.
On the call Wednesday night, Sarepta said additional assessments of DMD patients in the eteplirsen trial are being conducted at 62 weeks and 74 weeks, but the company didn't offer a timeline for when these data would be released. Just like what we saw with the 48-week data in October, stabilization of disease progression (or better yet) improvement would make the case for early approval even stronger -- if not a slam dunk.
"How come $SRPT tapping ATM before data is good, but for $ACAD is a red flag?"
I was rebuked by a bunch of readers for my
decision to sell stock through its At-the-Market (ATM) equity facility ahead of announcing results from the pimavanserin phase III study. It's true, Sarepta also sold stock through an ATM agreement prior to releasing eteplirsen data, and yes, I didn't raise a ruckus about it, so why is Acadia different?
Point taken and appreciated. In my defense, I'll say that among institutional investors there was ample discussion -- and some degree of consternation -- about Sarepta's ATM and whether management would use it before the eteplirsen data came out. I was also more confident in eteplirsen than I am with pimavanserin currently. I have my doubts about the upcoming Parkinson's psychosis trial based on the previous trial data but I'm also more than happy to be persuaded otherwise if the new results come back positive.
The opacity of ATMs is what I primarily object to. Shareholders have no way of knowing if or when management is selling stock until well after the fact. I prefer traditional stock offerings for their relative transparency, even though I understand that ATMs carry certain fiscal advantages.
While on the subject,
entered into a $50 million ATM facility on Wednesday. The disclosure was made in an SEC filing yet Vical said nothing about the ATM in its third-quarter earnings release or during the conference call held the same day.
No transparency! You see what I mean with these ATMs?
Timothy A. asks,
"What do you make of Vical delaying the Allovectin trial results again to the middle of 2013?"
It's ridiculous. The latest delay tells you the melanoma assumptions upon which Vical designed the phase III study are worthless. The study is a mess. If allovectin manages to demonstrate a survival benefit (miracles happen occasionally) it will be small and clinically meaningless. And while explaining to investors why they're waiting even longer for data, Vical management slips an ATM facility through the back door. Classy move, guys.
Gary B. emails,
"What are your predictions for hepatitis C stocks at the meeting this weekend?"
I predict chaos and confusion. I long ago stopped expecting closure or certainty from these AASLD meetings. Hepatitis C drug development is too volatile.
Here's what to look for this weekend:
The AASLD media embargo lifts on Saturday at 9 a.m. Eastern time.
have told me that press releases with up-to-date data on their respective all-oral regimens will be issued at that time.
Abbott vs. Gilead is the main event at AASLD this year.
Abbott has set the efficacy bar high already with
in treatment-naive genotype 1 patients treated in its "AVIATOR" phase II study.
The Abbott drugs studied in AVIATOR were ABT-450, a protease inhibitor that requires blood-boosting with ritonavir; ABT-267, a NS5A inhibitor; and ABT-333, a "non-nuc" polymerase inhibitor. Ribavirin, a current backbone in hepatitis C treatment, was also included in some but not all of the combination regimens.
On Saturday, Abbott will announce updated results from the AVIATOR study.
Gilead will counter with SVR4 cure-rate data from the ELECTRON study involving a combination regimen of the nucleoside inhibitor GS-7977 and the NS5A inhibitor GS-5885, plus or minus ribavirin. ELECTRON also enrolls treatment-naive, genotype 1 hepatitis C patients.
may shine on the undercard. Watch for SVR12 cure-rate data from the phase II combination of Bristol's NS5A inhibitor daclatasvir in combination with Gilead's GS-7997. While this combination isn't being moved into phase III, results will be viewed as a proxy for Gilead's '7977/'5885 combination.
On its own, Bristol will also be presenting data on a combination therapy involving daclatasvir, asunaprevir (a protease inhibitor) and the non-nuc BMS-791325.
I'm attending the AASLD meeting, which starts tomorrow and runs through Tuesday, so I'll have lots more to say about hepatitis C over the weekend and next week.
Catalyst Pharmaceutical Partners
announced the phase II failure of its cocaine addiction drug CPP-109 on Thursday. Treatment with CPP-109 for nine weeks failed to increase the number of addicts who were cocaine-free at the end of the study compared to placebo treatment. I wrote about
turned in another subpart quarterly performance. Revenue from the sale of its MelaFind skin cancer detection device totaled just $69,000 in the September quarter. Cost of goods sold was a whopping $569,000, leaving MELA with a gross "profit" of negative $500,000.
Throw in the rest of MELA's operating expenses and the net loss for the quarter was $5.4 million. MELA cash fell to $13 million.
MELA said it was still on track to meet guidance of 200 MelaFind system placements in the U.S. and another 75 in Germany by the end of March 2013. However, the definition of "system placements" has now been loosed to mean a signed user agreement only and not an actual installation.
MELA also acknowledged that dermatologists are using the MelaFind device more infrequently than initially projected.
All in, the Melafind launch is a bomb.
-- Reported by Adam Feuerstein in Boston.
Adam Feuerstein writes regularly for TheStreet. In keeping with company editorial policy, he doesn't own or short individual stocks, although he owns stock in TheStreet. He also doesn't invest in hedge funds or other private investment partnerships. Feuerstein appreciates your feedback;
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