was a big winner this week and kicks off the Biotech Stock Mailbag.
Via Twitter, @hifromfc5 asks,
"Hi Adam. Do you think MARINE alone is sufficient for NDA filing? What would happen if the other Phase III trial ANCHOR does not give significant triglyceride reduction?"
Doesn't it feel good when a company under-promises and over-delivers? That's what
from the phase III MARINE study. Excellent results: 33% trigylceride (TG) reduction overall, 45% TG reduction for the subset of "Lovaza-like" patients with super high (>750mg) TG baseline levels. And best of all, AMR101 didn't cause an increase in LDL (bad) cholesterol levels, which makes the drug potentially superior to Lovaza -- a $1 billion drug for
All in, the MARINE data were a home run, which is why Amarin said it would accelerate the timing of the U.S. approval filing to next year. That filing will be based on data from MARINE and will target TG lowering in patients with "very high" TG baseline levels of 500 mg/dl or higher. If approved, these data would put AMR101 on par with Lovaza. Importantly, Amarin doesn't need data from the ANCHOR study to get AMR101 approved.
This week's strong MARINE data do boost confidence in similarly positive data from the ANCHOR study next year. This study enrolls patients with "mixed" TG baseline levels (200-500 mg/dl), which is a patient population about 10 times larger than the patient population targeted in the ANCHOR study. (Glaxo can't market Lovaza to this "mixed" patient group because the drug causes LDL elevations.)
If Amarin is successful in getting AMR101 approved and convinces FDA to include the LDL neutral or lowering data from MARINE in the drug's label, doctors will more than likely prescribe AMR101 to their "mixed" patients anyway, even without final data from the ANCHOR study.
AMR101 could be a very, very big drug, which is why my preference would be for Amarin's management to sell the entire company to the highest bidder. Amarin management talks about a partnership but I think (hope) that's just more under-promising with the intent to once again over-deliver in a big way.
Lonnie M. emails about
"Will the pixantrone data being presented this weekend convince FDA to change its mind and approve the drug? I think so, after all, why would the meeting want to see new pixantrone data if it weren't great showing patients surviving because of this badly needed drug."
No. The pixantrone data being presented at the American Society of Hematology (ASH) annual meeting are not new or significant, nor will the data convince the FDA to rethink or reverse the decision to reject the drug.
The data at ASH Sunday are just a rehash of results from the old PIX301 "EXTEND" study which formed the basis of Cell Therapeutics' FDA approval filing, which an
, and which the
If you're new to the pixantrone story or forgotten how inadequate the data were from PIX301 study, you can
re-read my column which outlined all the FDA's criticisms and concerns
. To sum up the FDA's conclusions about the PIX301 data, pixantrone doesn't help lymphoma patients live longer but it does put them at greater risk for cardiac side effects.
More recently, Cell Therapeutics filed an appeal with FDA, arguing that the agency's reviewers got the pixantrone analysis wrong. Cell Therapeutics is also using the same PIX301 data to seek European approval. The FDA appeal is a desperate Hail Mary that is highly unlikely to succeed. I also fail to see a scenario by which European regulators reach a more positive conclusion about pixantrone compared to their U.S. counterparts at FDA.
If Cell Therapeutics wants to advance the pixantrone story with new data, the company should release final results from the PIX203 "RAPID" study. Preliminary results from the PIX203 study were presented at the ASH meeting in 2007 but since then, Cell Therapeutics has not released final study data despite repeated promises to do so.
Cell Therapeutics did not respond to an email requesting an update on the PIX203 study.
The PIX203 RAPID study compares pixantrone against doxorubicin, an approved and commonly used chemo drug, in patients with aggressive lymphoma. Patients in the study were treated with a chemo regimen including pixantrone plus Roche's Rituxan (CPOP-R) or a doxorubicin-containing chemo regimen plus Rituxan (CHOP-R).
Cell Therapeutics announced what it called positive, interim results in 2007 showing equivalent response rates in both arms and better cardiac safety in the pixantrone arm. Final survival and safety results were to be reported later when the study concluded.
ClinicalTrials.gov shows the PIX203 study to be
any longer. Cell Therapeutics stopped enrolling patients earlier than expected in 2008 because the company claimed it had all the data required to demonstrate pixantrone's superior safety over doxorubicin.
These final PIX203 data were going to be announced in the fourth quarter of 2009, according to Cell Therapeutics' quarterly SEC filing in June 2009.
In subsequent quarterly and annual SEC filings, Cell Therapeutics promised the final PIX203 data "within next six months," in "mid-2010," in the "second half of 2010," and most recently in "the fourth quarter of 2010."
This weekend's ASH meeting would have been the timely place for presenting new data on pixantrone from the PIX203 study. Instead, Cell Therapeutics went with a rehash of old data from the PIX301 study.
As of Thursday morning,
( AMLN) CEO Dan Bradbury had a small lead over
CEO Jack Lief and
Steven Engle in the voting for the
Bradbury had 341 votes, or 33% of the total, just ahead of Lief and Engle, with 296 votes (29%) and 290 votes (28%), respectively.
Brian Pereira was far off the pace, with just 10% of the votes cast.
I'll keep the
for another few days before declaring the winner of the Nance Trophy. Make sure you vote, if you haven't done so. And if you have voted, vote again!
Judging by email feedback received so far, I should have nominated
CEO Kelly Martin for the Worst Bio CEO Award. Kelly was the inaugural winner of the prize in 2008 but he's apparently still very unpopular and is garnering a lot of write-in support for a second trophy!
Josh G., who describes himself as a long-time Elan sufferer, emails,
"Can we vote for Kelly Martin again for worst CEO? Lots more embarrassing screw-ups in 2010!"
John E. seconds that vote.
"Adam, You are slipping. I maintain that Kelly Martin (un-nominated) continues to reign supreme as the Worst Biotech CEO of 2010. Where else can you find that deft balance between fraud and spin, gold-plated incompetence, and 'mind in neutral' mentality?? All in my honest opinion of course."
Sammy E. asks,
Vertex will be a big story in 2011 with the approval and commercial launch of the groundbreaking hepatitis C drug telaprevir. Yes, telaprevir's approval is a certainty, or as close to a sure thing as you'll ever see. That's both a tribute to the scientists and drug developers at Vertex but also a significant challenge for the company's business executives.
Wall Street is already expecting big things from telaprevir, which is reflected and baked in to Vertex's nearly $7 billion market valuation to a large extent. That puts added pressure on Vertex to make sure telaprevir launches strong and garners the quick and bountiful sales that investors who own the stock now are demanding.
Based on all available data, including the comparative data from
rival Hep C drug boceprevir, telaprevir should have no problem dominating market share and generate $2-3 billion in U.S. sales plus a significant royalty stream from sales in Europe and elsewhere. What's that worth? Vertex in the mid- to high-$40s sounds about right.
As I write this, a shudder goes through me because the direction most biotech stocks are taking these days
drug approval is down, down, down. Here's hoping that Vertex is more like
and less like
Vertex shares move much higher if the company can develop a successful and effective all-oral Hep C treatment regimen. Most importantly, this would eliminate the need for patients to take weekly injections of interferon. Failures or setbacks/delays hampering the development of competing Hep C drugs, most notably
Johnson & Johnson's
TMC '435, would work in Vertex's favor, too.
And speaking of J&J, how Vertex manages the relationship with its European telaprevir marketing partner while J&J also develops its own rival Hep C drugs will be tricky but very important.
Patrimus B. emails,
"Hey Adam, you will be delighted to know I have added your page to my favorites. After trawling through your past articles it soon became apparent you know your stuff -- your recent articles on Amarin were what cemented it for me. I have my own opinions on the following two companies but was just wondering what your views are -- Elan and BioSante Pharmaceuticals (BPAX) ."
Elan: I see no reason to own the stock. Growth in Tysabri, the company's multiple sclerosis drug, is still stunted and is now under added pressure from the first MS pill recently launched by
. Elan still owns a minority stake in experimental Alzheimer's drug bapineuzumab but the drug isn't likely to work based on the data presented to date.
And let's not forget that Elan CEO Martin isn't exactly the most confidence-inspiring leader, as witnessed by the reader comments above.
BioSante: The challenge/risk is not upcoming results from the phase III study of Libigel in hypoactive sexual desire disorder (HSDD). I'm fairly confident the LibiGel studies will yield positive results. Libigel is just testosterone and we've already seen studies from
Procter & Gamble
that testosterone therapy can produce positive results in FSD.
The risks are getting 1) getting LibiGel approved by FDA in the face of what will be substantial opposition from people who believe HSDD is nothing more than a marketing gimmick masquerading as a real disease; and 2) convincing doctors that the improvement in sexual desire stemming from LibiGel are clinically meaningful; and 3) raising the huge sums of money it will take to market LibiGel, if approved.
P&G bailed on its testosterone patch for HSDD;
never got anywhere moving Viagra into the female population; and most recently an FDA advisory panel rejected
flibanserin, forcing the company to also give up.
With that backdrop of failure, I'm not surprised to see investors taking a wait-and-see attitude towards BioSante. To me, BioSante is a high-risk trade, not much more.
--Written by Adam Feuerstein in Boston.
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Adam Feuerstein writes regularly for TheStreet.com. In keeping with TSC's editorial policy, he doesn't own or short individual stocks, although he owns stock in TheStreet.com. He also doesn't invest in hedge funds or other private investment partnerships. Feuerstein appreciates your feedback;
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