My take: None of these doubts about an accelerated approval are new, and as I've said many times before, this view is consensus and the stock still trades above $30 per share. If you don't day trade, that's pretty remarkable given where Sarepta came from last year.
I'm perfectly content being a contrarian and remain confident
FDA will allow Sarepta to seek accelerated approval
A related question emailed from Roger A.: "Thanks for taking my question. Could you tell me your long-term outlook for Sarepta?"
Sarepta with its exon-skipping therapeutic platform most definitely has the potential to drastically improve the lives of a majority of DMD patients (way more than just those eligible for treatment with eteplirsen) in the same way Vertex Pharmaceuticals has done already with Kalydeco and its other cystic fibrosis drugs.
As my friend and
Dan Rosenblum pointed out yesterday
, investors value Vertex's cystic fibrosis franchise at $11-12 billion. Sarepta's market value today is under $1 billion. That's a valuation gap that should -- and will -- narrow as Sarepta's exon-skipping platform churns out more drugs.
While I'm promoting the good work of my friend Dan, you should also go back and re-read his column explaining why
Sarepta is undervalued even without eteplirsen accelerated approval
Harry B. asks:
Cel-Sci (CVM - Get Report) issued a new statement where it claimed it had recruited 117 patients in its international phase III study and a new CRO [contract research organization] which will contribute $10 million to the study. I would like to know if you could publish your feelings as I highly consider your advices.
Cel-Sci's update this week on the Multikine head-and-neck cancer study resorted to some serious spin to deflect from the horrible reality that the company and its study are running off the rails.
Let's do some easy math. The
Multikine phase III study began in December 2010
, so in 28 months, the company has managed to enroll 117 patients, or 4.2 patients per month.
Cel-Sci designed the study to enroll 880 patients, so that means the study is still 763 patients short. If the current rate of patient recruitment continues, it will take Cel-Sci 182 months to reach full enrollment. That's 15 years.
But let's be optimistic and assume Cel-Sci, with two new CROs using that $10 million to pay off doctors and patients to try the Multikine study, triples the pace of enrollment. At this accelerated rate, Cel-Sci will reach full enrollment in five years.
Of course, Cel-Sci doesn't have enough cash to keep the lights on for 15 years or even five years, which explains this week's spin job. We've seen the movie play out before and it never ends well. There is no interest in Multikine as a treatment for head-and-neck cancer, forcing Cel-Sci to find (buy) patients in remote corners of the globe. At some point, Cel-Sci will give up because recruiting 880 patients is going to be impossible. But of course, Cel-Sci won't admit defeat, the company will simply conjure up some ridiculous excuse for why patient enrollment is being curtailed. No doubt, this excuse will include claims about Multikine demonstrating remarkable healing powers. These claims will be false but Cel-Sci will soldier on because bamboozlement is what the company does best.
Jay R. writes:
Adam, I enjoy your columns and I appreciate the informed opinion that you bring to the biotech space. You are amongst the best at what you do. That said, your Aveo Oncology (AVEO - Get Report) story today omitted several key points that I hope you will raise to your readers. Much of your argument about the challenges facing tivozanib approval rests on survival. It is important for readers to recognize two points. First, survival numbers are confounded by therapies given after the randomization. More therapy and/or better therapies given to one arm versus the other creates imbalances. Imbalances that are out of the control of the company/investigators should be recognized as such. Hence, progression-free survival becomes a more reliable endpoint in these instances. Second, statistical comparisons of survival rest heavily on balances between the two arms. If there are 158 patients in one arm and 18 patients in another, the analysis is universally viewed as poor. Yet, you leave this out. I am a big fan but I think your argument is full of many omissions that many investors do not fully understand.
You're correct in noting the imbalances in post-randomization treatment, but the blame lies with Aveo. You let them off the hook too easily. Aveo chose to run this study largely in countries that lack access to current kidney cancer therapies. FDA should ding them for doing that, not just shrug its collective shoulders.
I also disagree with your assertion about progression-free survival (PFS) being a more reliable endpoint. Nothing beats overall survival. Now, FDA has deemed PFS an acceptable and approvable endpoint for cancer drugs, so Aveo shouldn't be penalized for using PFS in the tivozanib study. However, you can't just ignore the survival data collected in the study. There are already eight or nine kidney cancer drugs approved and available to U.S. patients, so the regulatory bar should be higher now, not lower.
Mike P. has a complaint:
You really are a disgrace to the business. Your outright bashing of Amarin Corporation (AMRN) is only embarrassing you and the little bit of credibility you have left. Amarin has met every milestone and Vascepa's data is Evidence Based Proven Data. Several times you have stated they would launch late and file sNDA for Anchor late and Amarin has proven you wrong. GROW UP and get off it already. You have no background in the Pharma space, you have no financial licencing, which is why you have been and only will be a "columnist." You have no right telling the investment public what a company like Amarin or any other company should and should not release as informative PR. The way you attack companies using no factual basis behind your rants are crossing the line. Oh yeah by the way, great call on Sarepta. LOL. Just another assumption you were 100% wrong on.
I was wrong on Sarepta?
BSmith doesn't like me either:
I hope you understand that you need to be looking over your shoulder every day now. I think you should go to prison for your sins. You have constantly Bashed the work of good companies and have not had any remorse for your Sins because you think that you have done nothing wrong. Once the authorities Collar you and toss you in a cell then I think that you will somehow change. Some prison food, chains, and you getting slapped around in the bullpen should put some sense into you. You better get some good attorneys because you have cost many people out there a lot of misery (for you and your team's gain.) 15 years in the pen should straighten you out.
-- Reported by Adam Feuerstein in Boston.