Tysabri

Another Tysabri patient contracts PML

Tracy Staton — Thu, 30 Oct 2008 11:08:47 -0400

Another Tysabri patient has taken ill with the deadly brain infection progressive multifocal leukoencephalopathy, or PML. It's the sixth such case overall, and the third since the multiple sclerosis drug was reintroduced to the market in 2006 under a strict risk management program. This sixth patient, a woman, is alive, as are three of the five previous PML cases linked to Tysabri. About 35,500 patients were taking the drug at the end of last month, according to Biogen Idec.

The Wall Street Journal said the new case would "[darken] the commercial prospects for the medicine." There's no doubt that growth in Tysabri usage already has slowed since the two previous post-reintroduction cases were announced in July. And the latest news sent shares of Biogen, listed on the Nasdaq Stock Market, down 11 percent to $37.40 in after-market trading yesterday. Depository receipts of Biogen's marketing partner, Ireland's Elan, fell 23 percent after the announcement.

The new case also calls into question the theory that strictly governing Tysabri's usage would prevent PML from arising. But even so, the number of cases remains small, compared with the number of patients using the drug.

- read the WSJ story

FDA lets dying lawyer have Tysabri

Tracy Staton — Fri, 17 Oct 2008 10:50:51 -0400

Biogen Idec said no, but the FDA said yes. We told you yesterday about the case of Fred Baron, a generous Democratic donor who's dying of multiple myeloma. The Clintons, Ted Kennedy and several other prominent politicians, as well as Lance Armstrong, were lobbying Biogen for Tysabri. Well, now Baron will get the drug, even though it's not yet approved for multiple myeloma treatment.

Biogen didn't cave, however; Baron's doctors at the Mayor Clinic worked with the FDA to find a "legal basis" for Tysabri use, the Associated Press reports. You'll recall that Tysabri is sold under a restrictive risk management program because of its links to a potentially fatal brain infection, PML. That program usually prohibits off-label use, and as you know, Tysabri is only approved to treat multiple sclerosis and Crohn's disease in the U.S. Clinical trials for multiple myeloma began in September.

You can be sure that the media will be following Baron's case, so we'll find out how he fares on Tysabri. One of the reasons his illness has attracted so much attention: Baron was the donor who acknowledged sending money to former presidential candidate John Edwards' ex-mistress. That adultery-and-payoff scandal hit the tabloids, of course. So it's no wonder that the money man is now making headlines.

- read the Washington Post story

Celebs plea for off-label Tysabri

Tracy Staton — Thu, 16 Oct 2008 10:54:08 -0400

No one can accuse Biogen Idec of playing favorites. The company has been under assault by luminaries from Bill and Hillary Clinton to Lance Armstrong, who are trying to get Biogen's Tysabri drug for Fred Baron, a legal bigwig who's dying of multiple myeloma. Tysabri is in Phase I testing in multiple myeloma patients. But Baron doesn't meet the trial criteria. And Biogen has an ironclad policy of refusing the med for so-called "compassionate use."

Biogen's reasoning is rooted in Tysabri's complex biography. As you all know, the drug was hailed as a major breakthrough for multiple sclerosis patients after its first launch, but then was pulled off the market in February 2005 because of links to a potentially fatal brain infection, PML. The drug was reintroduced in 2006 under a restrictive access program. Since then, Tysabri has won a broader market, with an approval to treat Crohn's disease. It's also seen a couple of new cases of PML in Europe; those cases were added to Tysabri's labeling.

Biogen CEO Jim Mullen (photo) has held up under the assault of publicity ginned up on Baron's behalf. Calls from the likes of Armstrong, Ted Kennedy, the Clintons, John Kerry and Henry Waxman haven't swayed him. The company doesn't want to set a precedent by allowing Baron to use Tysabri, given the PML risks--and the risk to Tysabri's future should Baron experience an adverse reaction. "We want to protect access for patients who are on the drug now and rely on it," a Biogen spokeswoman told Pharmalot. "[W]e feel like we can't make an exception in this case."

- read the story at Pharmalot

Tysabri to get new Euro label

Tracy Staton — Fri, 26 Sep 2008 10:55:19 -0400

The European Medicines Agency called for an update to product information for Biogen Idec and Elan's multiple sclerosis drug Tysabri, highlighting the risk of a potentially deadly brain infection. But the EMEA also confirmed that it still believes the drug's benefits outweigh its risks. EMEA release

Tysabri may deliver 'sustained improvement'

Tracy Staton — Mon, 22 Sep 2008 11:18:35 -0400

Tysabri may have scored a big plus for drugmakers Biogen Idec and Elan. A trial of the multiple sclerosis treatment showed that it might actually improve the physical disability patients suffer, rather than simply slowing down or staving off decline. "Tysabri-treated patients are significantly more likely to experience a sustained improvement in disability compared to placebo patients," said Frederick Munschauer a neurologist from the State University of New York, adding that, "This [post hoc] analysis represents the first evidence supporting a sustained improvement in function" from an approved MS drug.

That's good news for Biogen and Elan, offering even greater benefit to outweight the risk of a very rare but potentially deadly brain infection that, when it first appeared in Tysabri patients, forced the medication off the U.S. market. Tysabri has since been reintroduced under a risk-management program and continues its comeback story, despite the recent announcement that the infection, primary multifocal leucoencephalopathy, had cropped up again in two patients in Europe.

- see the Biogen release
- read the story in the Irish Times
- get analyst reaction at FinFacts

Biogen, Elan test Tysabri against cancer

Tracy Staton — Fri, 05 Sep 2008 10:45:03 -0400

Could Tysabri fight cancer as well as it battles multiple sclerosis? Biogen Idec and Elan aim to find out. The companies have given a multiple myeloma patient the first dose in the first clinical trial designed to see whether it might be effective against cancer. Patients in the study have either relapsed, or their cancer is resisting treatment with other meds. In the first phase, researchers will check the drug's safety in 12 patients. Then, they'll assess the efficacy in 30 patients.

Of course, a new indication for Tysabri is years away. But the new trial shows the companies plugging away at their strategy of racking up new uses for the MS drug, which recently won U.S. approval for treating Crohn's disease. Biogen and Elan are obviously undeterred by recent reports of two new cases of the brain ailment that pushed Tysabri off the market in 2006. The FDA was undeterred too; it simply added the news to Tysabri's label. And for patients with debilitating diseases like MS and treatment-resistant cancer, the benefits could definitely be worth that risk.

- check out Elan's release
- read the story in the Boston Globe

FDA, Biogen mull Tysabri label changes

Tracy Staton — Tue, 26 Aug 2008 11:15:54 -0400

The FDA is huddling with Biogen Idec and Elan on amendments to Tysabri's label. The reason, of course, is the news of two cases of the brain disorder progressive multifocal leukoencephalopathy, or PML, that emerged in late July.

In a notice on the agency's website, the FDA reiterated its belief that Tysabri taken on its own may generate a smaller risk of PML than when it's taken in combo with other meds that alter the immune system.

Compared with the response the last time PML emerged in Tysabri patients, it's mild. As you know, Tysabri was pulled from the market when PML first cropped up, but was allowed back on the market on a strict risk-management program, under which patients using the drug are monitored for infection. Investors showed their relief by pushing Biogen shares up by about 2 percent to $53.48, and Elan shares by 5 percent, to $14.25.

- check out the release
- read the Wall Street Journal story
- see the item in the Wall Street Journal Health Blog

Icahn snaps up more of Biogen

Tracy Staton — Tue, 12 Aug 2008 10:17:49 -0400

The Biogen Idec faithful have been preaching "buying opportunity" ever since the company announced two new cases of the brain infection that can afflict patients taking its vaunted multiple sclerosis drug Tysabri. After all, CEO James Mullen had warned that the potentially fatal malady, PML, could and would crop up again, and the MS sufferers taking the drug have been made fully aware of the risk.

We can now count Carl Icahn among those faithful. The activist investor has taken this opportunity to boost his stake in Biogen to 6 percent from about 4.3 percent. That's a $259 million investment.

What's next, given that Icahn rarely puts his money where his mouth won't go? According to SEC documents, he may talk to the company about "concerns relating to shareholder value." Hmm. You'll recall that Icahn recently pressured Biogen into putting itself up for sale, and then accused management sandbagging the bidding process. Perhaps another round of bids is in Biogen's future.

- read the post at the Wall Street Journal Health Blog
- see the item at StreetInsider

Judge strikes down Biogen preemption case

Maureen Martino — Thu, 07 Aug 2008 14:01:22 -0400

Biogen and Elan--along with proponents of preemption--were dealt a setback today as a federal judge declined to transfer a case from Massachusetts to a federal court in Iowa. The lawsuit stems from the death of a patient who contracted PML after taking Tysabri; the drug was pulled from the market 11 days later. The patient's wife sued the drugmakers in Massachusetts, where Biogen is based. But drugmakers are waiting for the Supreme Court to rule on the issue of preemption, which asserts that the FDA's approval of a drug shields companies from lawsuits. They're expected to hear a precedent-setting case--Wyeth v. Levine--this fall. Biogen and Elan argued that the Massachusetts case should be moved to federal court because the fundamental question is whether the FDA's word protects drugmakers.

However, Judge Douglas Woodcock ruled against the developers, saying that preemption is not part of the law at this time and as such, the suit should stay in state court where it was filed. "The Defendants are essentially proposing that I exercise federal jurisdiction given the unsettled nature of the law in this area. I decline their offer," he said in his opinion. "Present posture of case law does not justify removal from state court to this [federal] court.

- here's the Wall Street Journal article
- check out the Pharmalot post for more

Analysts: Tysabri scrips will suffer

Tracy Staton — Thu, 07 Aug 2008 10:46:02 -0400

When Biogen Idec announced last week that a potentially fatal brain infection had cropped up again in Tysabri patients, markets reacted swiftly--and negatively. But investors were only guessing at how that new information might affect new scrips for the drug, which has been hailed as a miracle treatment by many multiple sclerosis patients. Now, financial analysts have done some number-crunching to quantify those long-term effects. And the news isn't good.

Deutsche Bank analyst Mark Schoenbaum told investors that his best-case scenario pegged Tysabri at 75,000 users by 2013--not nearly the 100,000 patients Biogen had been shooting for by 2010. Under more conservative estimates, Schoenbaum predicted 56,000 Tysabri patients, again by 2013. And worst case? If more safety problems emerge, the drug could get pulled from the market--again. (For what it's worth, though, other analysts have said they don't expect Tysabri to be withdrawn.)

Meanwhile, a JP Morgan analyst estimated that the number of Tysabri patients by 2010 would be only 50,000--even before the two cases of progressive multifocal leukoencephalopathy (PML) were reported. The analyst said he doesn't expect that estimate to drop much further.

- read the story in the Boston Globe

Biogen: Tysabri patients have PML

Tracy Staton — Fri, 01 Aug 2008 10:26:41 -0400

Biogen Idec CEO James Mullen predicted it, and now it's happened: Two Tysabri patients have come down with the potentially fatal brain infection, progressive multifocal leukoencephalopathy, or PML, that caused Biogen to withdraw the multiple-sclerosis treatment back in 2005. This time around, though, the company doesn't plan to pull the med or further restrict it.

As you know, Tysabri was allowed back onto the market in July 2006 under a strict risk-management program. Since then, 31,600 patients have started on the drug, and Biogen (and its partner Elan) won an additional indication for Crohn's disease. Mullen had been predicting that 100,000 patients would be using Tysabri by 2010, with quarterly sales of $250 million or better by the end of this year.

It's unclear whether this news will decimate those sales. Investors seem to think so; both Biogen and Elan have seen their shares drop precipitously this morning. And the fact that the sickest PML patient--who's now hospitalized--was only taking Tysabri for 14 months, because the duration of treatment seems to be a factor in the disease. (Previous PML patients had used Tysabri for some two years before discovering they had it.) This patient had used other MS drugs before taking Tysabri, which also appears to be a risk factor. The other patient, who's at home and ambulatory, had been on Tysabri for 18 months, and hadn't used any other MS treatments before.

But patients know about the PML risks before they go on Tysabri; the risk-management program makes sure of that. And so far, the number of PML cases appears to be in line with the statistical risk listed on the drug's label. So we'll have to watch the scrip numbers to see whether verification of that risk deters patients.

- read the Wall Street Journal article
- see the story in the Boston Herald

Biogen CEO: Tysabri to reach $1B in 2008

Tracy Staton — Mon, 21 Jul 2008 11:10:50 -0400

Two years ago this week, Biogen Idec relaunched Tysabri, the multiple sclerosis and Crohn's disease treatment, after a safety scare. Now, the drug is shaping up to be a blockbuster, the Wall Street Journal reports.

Tysabri was recalled in 2005 after three patients developed a rare brain infection and two died. FDA let the drug back on the market the next year under a monitoring program. Just two years later, Tysabri is expected to surpass $1 billion in annual sales, according to Biogen CEO James Mullen. First-quarter sales were $160 million, and second-quarter numbers will be announced tomorrow.

- read the WSJ article

AZ, Amgen earnings flat; Shire sales leap

Fri, 25 Apr 2008 06:59:54 -0400

It's a mixed bag of drugmaker earnings reports today, with two biggies posting flat results and two smaller firms chalking up respectable gains.

AstraZeneca's profits suffered under restructuring costs and falling Nexium sales, reporting a $2.1 billion gain, 5 percent less than the same quarter last year. Sales were 10 percent higher at $7.7 billion, but much of that increase came from the weak dollar. Analysts and shareholders were unimpressed; UBS wrote that the "ramifications of [the first quarter results] could be far reaching."
Amgen's earnings were mostly flat: Net income of $1.14 billion compared with $1.11 billion a year ago, with accounting charges eating into earnings. Revenues, however, slid by 2 percent, to $3.61 billion. No big surprise, considering that sales of its biggest drug, Aranesp, free-fell by 25 percent; it and its sister med Epogen have been hamstrung by safety concerns for months now.
U.K. specialty pharma Shire posted a 33 percent hike in sales to $702 million, slightly ahead of analyst expectations. EPS was up to 22.7 cents from 21.3 cents during last year's first quarter. One sour note: The company estimated that its Vyvanse ADHD drug--which the company hopes will replace its soon-to-go-off-patent Adderall--would generate sales on the "lower end" of its predicted range of $350 million to $400 million.
Ireland's Elan also posted a big increase in sales--22 percent, to $214.7 million--but still reported a loss of $85.5 million. At least the loss is smaller than last year's $93 million. The revenue increase came from surging sales of its lead drug, the MS treatment Tysabri it co-markets with U.S.-based Biogen Idec.

- see the AstraZeneca piece in the Financial Times
- check out MarketWatch's story on Amgen
- read the Shire article at Orange
- find Elan's numbers in Forbes

Biogen CEO: Tysabri patients will get PML

Wed, 19 Mar 2008 07:59:54 -0400

Expect the unexpected. That's essentially what Biogen Idec CEO James Mullen (photo) told investors yesterday when he predicted that more cases of a rare, fatal brain disorder will arise in Tysabri patients. "[W]e anticipate there will be some additional cases of PML," Mullen said, adding that he doesn't believe they would cause regulatory problems for the drug.

You'll recall that Biogen pulled Tysabri in 2005 when two patients acquired PML, or progressive multifocal leukoencephalopathy. It since has returned to the market under a patient-monitoring program. Despite the risks, many MS patients use Tysabri because it works. So many, in fact, that Mullen has been predicting blockbuster status for Tysabri by 2010. The drug's recent FDA approval for Crohn's disease could only help.

Recently, though, Biogen and marketing partner Elan warned doctors of the potential for liver damage in Tysabri patients. Injury could happen within six days of starting treatment, the letter warned.

- see the Triangle Business Journal story

Related Articles:
No new PML cases linked to Tysabri. Report
Tysabri: The comeback kid. Report
After reintroduction, Tysabri builds patient base. Report
FDA allows Tysabri back on market. Report

'Cloud of risk' swells around Tysabri

Thu, 28 Feb 2008 06:59:56 -0500

The multiple sclerosis drug Tysabri has hit an obstacle on its comeback trail. Drug makers Biogen Idec and Elan warned doctors yesterday that serious liver injury could arise in patients using the med. In some patients, signs of liver problems showed up just six days after their first dose.

The new warning was included in prescribing information for Tysabri use in patients with Crohn's disease, a new indication approved by the FDA last month. Now it's being added to inserts for patients using the product for MS. The rate of liver injuries is less than one in 1,000 patients.

You'll recall that Tysabri was taken off the market in 2005 after a few patients developed a rare neurological disorder. In 2006, the FDA allowed it to be sold under a restricted distribution program. Since then it's been slowly gaining market share; it brought in $343 million for Biogen last year.

After nabbing the new indication last month, Biogen said it hoped to attract 100,000 new patients to the drug by 2010. We'll see whether the new warnings have any effect on those numbers. Some analysts are wary; one called Tysabri "a drug with a cloud of risk hanging over it."

- read the story in the Wall Street Journal
- see the item at Pharmalot
- check out the coverage at CNBC

Related Articles:
Docs raise new Tysabri fears in NEJM. Report
After reintroduction, Tysabri builds patient base. Report
FDA allows Tysabri back on market. Report

Docs raise new Tysabri fears in NEJM

Fri, 08 Feb 2008 06:59:55 -0500

There are two new exhibits to be entered into evidence that drug safety is complicated. First, the back-from-exile MS drug Tysabri may have unexpected effects on patients with a family history of melanoma. A letter published in the New England Journal of Medicine details the experience of two women who, soon after starting Tysabri therapy, developed fast-spreading melanoma. Already at least one Wall Street analyst has picked up on the letter, writing to investors that Biogen Idec, which makes Tysabri, may have forecast unrealistic growth for the drug. The company says its data shows no significant link between Tysabri and melanoma.

Second, there's Trasylol, which was suspended from the market last fall after research linked it to renal failure. A new study suggests that the link may not be so direct, or so clear. Patients given Trasylol during so-called "on-pump" heart surgery--when a machine takes over circulating a patient's blood--showed no significant kidney problems afterward. Patients given the drug along with an ACE inhibitor during "off-pump" surgery showed a twofold increase in the risk of kidney dysfunction. But it wasn't just the addition of the ACE inhibitor; "on-pump" patients given the combo showed no significant increase in renal problems.

Will this new research help usher Trasylol back onto the market? Maybe, maybe not--the drug has also been linked to other serious side effects, such as heart failure. But this new study also shows that--as in the tragic case of actor Heath Ledger--it's not just the drug, but a complex interaction of one drug with another, plus other possibly unknown factors. Of course, when people's lives are at stake, erring on the side of caution seems warranted. Red flags, such as the two melanoma cases in Tysabri patients, should be raised. Deciding what to do with that information, however, requires a lot more thought.

- check out an excerpt from the letter in the NEJM
- read CNN Money's Tysabri coverage
- see this item at Pharmalot
- read the Trasylol report in the Washington Post

Related Articles:
After reintroduction, Tysabri builds patient base. Report
FDA allows Tysabri back on market. Report
Bayer pulls Trasylol at FDA request. Report
FDA to revisit Trasylol death risk. Report

FDA clears Tysabri for Crohn's

Tue, 15 Jan 2008 06:59:56 -0500

Call it the comeback kid. Tysabri nabbed a new indication from the FDA yesterday, as a second-line treatment for the intestinal disorder Crohn's disease. Made by Biogen Idec and Elan, Tysabri is already marketed for use in multiple sclerosis.

You'll recall that Tysabri was pulled from the market in 2005 after three patients developed multifocal leukoencephalopathy, a rare nervous system disorder. It was only allowed back on the market in 2006 under a restricted distribution program. Since then, the drug has built up a following of 12,000 MS patients in the U.S., Biogen says, with no new reports of the deadly disorder.

Tysabri will be distributed under a similar program for Crohn's patients, who'll have to take classes on its risks and receive injections only from selected doctors. An incurable intestinal disorder, Crohn's affects about 500,000 people in the U.S.--a big new market for Tysabri. Indeed, Biogen says it hopes to attract a total of 100,00 patients to the drug by 2010. That would give Tysabri annual sales of $670 million. Not quite a blockbuster, but pretty good for a product that once faced extinction.

- see the release from Biogen Idec
- read the article in the New York Times

Related Articles:
Tysabri: The comeback kid. Report
FDA committee backs Tysabri for Crohn's. Report
After reintroduction, Tysabri builds patient base. Report
FDA allows Tysabri back on market. Report

ALSO NOTED: Analysis: Marketing brings in money for R&D; Osmetech submits app for warfarin test;

Mon, 07 Jan 2008 06:59:50 -0500

> Last week, we covered a study of drug companies' marketing expenditures. Here's an R&D insider's analysis of that research, in which marketing is cast as a very necessary evil. Report

> Osmetech has submitted a test of patients' sensitivity to the blood thinner warfarin to the FDA for clearance. The agency recently suggested that patients be screened for genetic variances before they're given the drug. Report

> Biogen Idec and Elan released more safety data on Tysabri, saying patient outcomes continue to support the drug's safety since its relaunch in July 2006. Report

> GlaxoSmithKline won kudos from The Globe and Mail as one of the best 50 employers in Canada. Report

> WuXi PharmaTech, a provider of R&D outsourcing services, has agreed to pay $151 million for Minnesota-based medical research group Apptec. Report

> As America's obesity epidemic continues, biotech and pharmaceutical companies are racing to create a pill that delivers significant weight loss without the unpleasant side effect of current obesity treatments. Report

And Finally... A Chinese scientist has developed a grape variety with six times the amount of super-healthy resveratrol, a compound associated with increased longevity and decreased heart disease. Report

Is Big Pharma souring on Biogen deal?

Wed, 14 Nov 2007 06:59:57 -0500

Remember last week's rumors that Big Pharma was poised to jump at the chance to bid for Biogen Idec? This week, analysts aren't so sure. Investors are suddenly shying away from the Cambridge, MA-based biotech because word on the street is that a deal may be too rich even for a major drug maker to swallow.

Yesterday, Biogen stock dropped to just over $69 during the day, its lowest point since the company put itself on the block a month ago. Compare that to $84.75 on October 15. What's the problem? Some market observers say potential bidders sell drugs that compete with Biogen's. Others point to Biogen's partnerships with Genentech (for Rituxan) and Elan (for Tysabri) as complicating factors. And news on the Street says some potential bidders--namely Roche and Sanofi-Aventis--have dropped out.

One analyst told the Boston Globe that it all hinges on Tysabri, Biogen's MS drug. If the buyer thinks Tysabri will go platinum, then the company doesn't look overpriced. If not, well, then Biogen's the goose that might not lay a golden egg.

- check out this Boston Globe report

Related Articles:
Tysabri: The comeback kid. Report
Sources: Biogen gets bidders, with Pfizer in lead. Report
What's Biogen Idec really worth to Big Pharma? Report
Will Big Pharma buy Biogen Idec? Report
Biogen Idec in play after Icahn makes his move. Report

Pfizer among Biogen front-runners

Thu, 08 Nov 2007 06:59:58 -0500

Merger-watch fans, listen up. The Financial Times is reporting some tantalizing details about the bidding for Biogen Idec, the biotech based in Cambridge, MA, that sent stock analysts into an uproar when it put itself up for sale a few weeks ago. Unnamed sources told the London newspaper that the sequence of events leading to that surprise announcement actually began with Pfizer offering $80 per share for the company. Investor Carl Icahn (photo) then made a counter-offer, and the company retained financial advisors to conduct an orderly sale. Even now, Pfizer remains one of the front-runners for Biogen, the sources said, along with Merck and Johnson & Johnson.

Some other buyers have already dropped out of the race, spooked by the "nosebleed prices" expected. Icahn offered $23 billion, so the winning bid is likely to be higher. Are Biogen's assets worth that much? They include the multiple sclerosis drug Tysabri, in partnership with Elan; the lymphoma and arthritis remedy Rituxan, in collaboration with Genentech; and the MS drug Avonex, which is wholly owns. It also has a pipeline of drugs in development, including two promising MS products.

Meanwhile, headhunters are getting calls from Biogen types expecting a sale to lead to the layoff ax. The non-executive staff is anxious, recruiters said, even though the company continues to hire new workers.

- check out the buyout report from the Financial Times
- read about Biogen staff seeking shelter at Inside Recruiting

Related Articles:
Biotech stocks surge as investors anticipate buyouts. Report
Speculation soars over Pfizer's interest in biotech. Report
What's Biogen Idec really worth to Big Pharma? Report
Will Big Pharma buy Biogen Idec? Report
Biogen Idec in play after Icahn makes his move. Report
Who should Pfizer buy? Report