MEMPHIS, Tenn. (
CEO Mitch Steiner blasted the Food and Drug Administration on a conference call Monday morning after regulators refused to approve the company's bone drug acapodene.
"They moved the goalposts on us," said Steiner, referring to the FDA's complete response letter, which he says asks GTx to conduct one, perhaps two, new clinical studies in order to get acapodene approved.
Steiner said that the FDA previously had agreed to base its approval review on a single study showing acapodene could lower the risk of bone fracture in men undergoing hormonal treatment for prostate cancer.
Monday, however, the FDA rejected acapodene, asking GTx for additional clinical data on the drug's efficacy and safety as well as proof that treatment with the drug does not cause prostate tumors to grow or shorten a patient's overall survival.
Steiner called the first FDA request a "surprise" and the second request -- the one seeking data disproving acapodene's link to tumor progression -- "an even greater surprise."
He said that these FDA requirements were added on at the last minute of acapodene's review and never came up in conversations between the company and the agency.
Acapodene has been linked to
and risk to patients, including blood clots and prolongation of the Qt interval, a measure of the heart's electrical cycle. Yet, Steiner, on his conference call, says neither safety issue was brought up in the FDA's complete response letter.
GTx plans to meet with the FDA to figure out a path forward for acapodene, Steiner said. His hope is that the FDA's requests can be met with acapodene data already collected. If a new trial is needed, GTx may be forced to abandon acapodene as a bone-fracture reducer.
"Another study will cost us $30-35 million and take five years," said Steiner.
GTx had about $69 million in cash at the end of the second quarter. The company reports third-quarter results later this week.
GTx shares were down 37% to $5.64 in early Monday trading.
Dendreon Resubmits Provenge to FDA
(At 8:56 AM ET)
has re-submitted its approval package for Provenge to the Food and Drug Administration, the company said Monday.
Provenge is an immunotherapy, or cancer "vaccine" for the treatment of metastatic hormone-resistant prostate cancer. Data from a phase III study presented this spring demonstrated that Provenge could
of prostate cancer patients.
Dendreon's Provenge resubmission comes a bit earlier than the company's guidance. If the FDA accepts the Provenge application and grants a priority, six-month review, an approval decision will come on May 2, 2010.
Vertex Hep C Drug Emerges From Weekend Looking Stronger
(At 7:26 AM ET)
I hesitate before predicting a stock's movement in the hours before trading opens for the day, but by almost all measures,
did yeoman's work this weekend with its experimental hepatitis C drug telaprevir, warranting a higher stock price Monday.
As I reported Saturday, a twice-daily dose of telaprevir led to
, according to data from a new phase II study. The study demonstrated quite convincingly that a more convenient twice-daily dose of telaprevir is just as effective and safe as the current thrice-daily dose.
The hepatitis C cure rates of greater than 80% across all four patient groups of the study are also the highest ever recorded in any telaprevir study to date and exceed the cure rates reported by any of telaprevir's competitors.
The strong data from the so-called C-208 study made for a lot of happy faces at an investor event put on by Vertex Sunday night at a Boston hotel. Analysts, investors and hepatitis C researchers (of course) are all in town this week for the annual meeting of the
American Association for the Study of Liver Disease
During prepared remarks at the Vertex shindig, chief financial officer Ian Smith, referring to both the C-208 trial data as well as Wednesday's new data in non-responding patients, said, "The bar we are setting with telaprevir is a high one."
Smith has a reputation for sometimes being overly confident, but in this case, he's right. Vertex is surely not making it easy for competitors to knock telaprevir off the top the Hep C mountain.
( SGP) and
Johnson & Johnson
, among others, will present data on competing drugs this week that could raise more concerns that telaprevir's reign will be short lived. But with twice daily dosing a very viable option and cure rates over 80%, telaprevir isn't leaving much room for competing drugs to operate.
Two other data presentation of note from Sunday: Schering-Plough presented a new analysis of an old phase II study demonstrating that its Hep C drug boceprevir can produce a 55% cure rate tough-to-treat "null responder" patients.
These data look strong compared to a 57% cure rate for null responders treated with Vertex's telaprevir in its own phase II study. But it should be noted that Schering-Plough uses a more liberal clinical definition of null response, which allows the company to include patients who aren't as difficult to treat.
Phase II data from an experimental Merck drug MK-7009 were also presented Sunday night. Efficacy was impressive but there were precious few details provided about the vomiting side effect that has raised concerns in the past.
Amgen's Aranesp Might Make for Morose Monday
Monday may not be as joyous given the two nasty headlines from Friday. First, the attorney general of New York, among others, filed a lawsuit against Amgen accusing the company of an illegal kickback scheme aimed at boosting sales of its anemia drug Aranesp.
Hours later, a study presented at a medical meeting and published online in the
New England Journal of Medicine
showed that patients with kidney disease treated with Aranesp to correct anemia were almost twice as likely to suffer from strokes than those treated with a placebo. The study failed to demonstrate that treatment with Aranesp reduced the number of deaths or heart attacks in chronic kidney disease patients compared to placebo treatment. And if that's not enough bad news, Aranesp use was also tied to slightly higher deaths from cancer compared to placebo.
Aranesp sales have declined significantly over the past several years due to safety concerns that has reigned overuse of the drug by doctors in both cancer and kidney disease.
Analysts say chronic kidney disease (non-dialysis) accounted for about $600 million of Aranesp's $3.1 billion in 2008 sales. If there's a silver lining to Friday's bad news, it's that the data from the Aranesp "TREAT" study were largely pre-announced and in-line with investor expectations. Declines in Aranesp sales have been built into many Wall Street models already and most Amgen investors are paying more attention to the company's new bone drug, Prolia.
"While we continue to expect these findings to negatively impact Aranesp use, the study results will remain controversial, as some physicians we spoke with do not anticipate changing prescribing patterns based on these findings. We have already reduced our Aranesp estimates by 9% and 15% in 2010 and 2011, which we think adequately reflects expected sales declines. We believe the spillover in dialysis patients will be limited, as the anemia benefits are greater in this setting," writes Bank of American biotech analyst Rachel McMinn on Monday.
Barclays analyst Jim Birchenough writes, "With heightened scrutiny of excess stroke risk and cancer risk in pre-dialysis patients, Aranesp trends could suffer although overall impact will likely be small. We estimate $600M of $2.6B Aranesp revenues at risk with EPS impact of $0.20, although actual downside risk on potential Aranesp restriction to rescue use would be
less than $300M and
less than $0.10 EPS and is already reflected in pre-ASN weakness."
Deutsche Bank analyst Mark Schoenebaum writes, "Net-net, while our US Aranesp erosion is accelerated, risk to 2014 sales number is negligible compared to our previous forecast and equals $290M to consensus forecast. ROW Aranesp erosion was already well-captured by us and consensus; The changes have a minimal impact on our DCF and we are comfortable maintaining our BUY rating at $64 PT."
-- Reported by Adam Feuerstein in Boston
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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