On Thursday, Amicus Therapeutics CEO John Crowley will join the long procession of biotech executives making presentations at one of the industry’s most important gatherings, the JP Morgan Healthcare Conference in San Francisco. But Crowley does have one claim to fame that makes him stand out from the crowd: He’s the only biotech CEO whose early career was portrayed in a glitzy Hollywood movie. And that film, Extraordinary Measures starring Harrison Ford, focused on one of the illnesses Amicus is tackling—Pompe disease, an enzyme-related muscle disorder that two of Crowley’s three children have. “That’s what got me into the biotech field,” Crowley (pictured at right) told Xconomy a few weeks before the JP Morgan event.

Like the others speaking at the event, Crowley will have a short 25 minutes to make his pitch to investors, Wall Street analysts, and fellow executives. His goal, in brief, is to build confidence in the company’s pipeline, which includes a drug that Amicus is testing in combination with alglucosidase alfa (Myozyme)—the Genzyme treatment for Pompe that Crowley famously helped develop and that his own children take.

Amicus’ investors will surely be looking for signs of hope. Shares of Amicus (NASDAQ: FOLD), which is based in Cranbury, NJ, dropped 43 percent to $3.44 in the nine months ended December 30. That was a time filled with “some measure of instability,” Crowley says, primarily because it took longer than expected for Amicus to enroll patients in a key clinical trial of a drug it’s developing for a second rare disease, called Fabry.

Before we delve into Amicus, though, let’s briefly review the Hollywood-worthy events leading up to Crowley’s tenure there. In 2000, Crowley, a Harvard-trained MBA, left a management position at New York-based Bristol-Myers Squibb to help start Novazyme, an Oklahoma City-based company founded by a scientist working on a Pompe treatment. They sold Novazyme to Cambridge, MA-based Genzyme in 2001 for $137.5 million and Crowley became chief of Genzyme’s Pompe program. (If you’re not impressed yet, consider that Crowley is also a graduate of Notre Dame law school and a commissioned officer in the U.S. Navy Reserve.)

Genzyme won approval for alglucosidase alfa in 2006. Crowley’s children, Meghan and Patrick, who were put on the drug in 2003 as part of a clinical trial, are still on it and doing well in the 9th and 8th grades, respectively, Crowley says. His family’s experience inspired a Wall Street Journal story, and later, a book called The Cure. Then Harrison Ford optioned the rights to the story, which led to the movie, starring Brendan Fraser as Crowley and Ford as a prickly (and fictional) scientist who worked on an enzyme treatment for Pompe.

What attracted Crowley to Amicus was the opportunity to make Genzyme’s treatment for Pompe—and drugs for related disorders—even more effective. Pompe and Fabry are among about 50 inherited, often fatal disorders that occur when the enzyme-making machinery in the body’s cells malfunction, causing them to make too little of a particular enzyme, or to make a “misfolded,” or unstable, version of it. Genzyme’s treatment is one of a number of enzyme replacement therapies designed to fix those deficiencies.

Amicus was founded in 2002 on a technology developed at Mount Sinai Medical School in New York. The technology yields small molecules that attach themselves to the defective enzymes, stabilize them, then transport them to the part of the cell where they need to go in order to … Next Page »

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On May 11, Shire (NASDAQ: SHPGY) CEO Angus Russell told a panel of journalists at the Reuters Health Summit in New York that the British drug giant is interested in pursuing gene therapy and stem cells. Those are two areas of science that have yet to prove they can produce effective, innovative new therapies. Still, over lunch later in the day in midtown Manhattan, Russell made a strong case to me that Shire is finding tremendous opportunities on the fringes of pharmaceutical development. “You can’t tar us with making the tenth me-too drug and trying to charge a bazillion dollars a year for that,” he says. “We’re not in that game. I don’t condone that.”

As proof, Russell pointed to Shire’s burgeoning Boston unit, which makes drugs to treat rare diseases. Shire plunged into the rare-disease field in 2005 when it bought Cambridge-based Transkaryotic Therapies (TKT) for $1.6 billion. The unit, which recently moved to a large campus in Lexington, specializes in drugs to treat enzyme disorders, and it is responsible for some of Shire’s biggest hits, including idursulfase (Elaprase) for Hunter syndrome, velaglucerase alfa (Vpriv) for Gaucher disease, and agalsidase alfa (Replagal) to treat Fabry disease.

Shire got a huge head start in Fabry disease and Gaucher disease back in 2009, when competitor Genzyme ran into manufacturing issues. The FDA had not yet approved Shire’s products, but the agency gave the company permission to market them anyway, to help address the shortage resulting from Genzyme’s troubles. “It accelerated the growth of that business,” Russell says. All told, products emerging from the TKT acquisition now account for 30 percent of Shire’s sales—up from 4 percent when the companies first merged.

Russell said he’s amazed by how many drug companies are now embracing rare, “orphan” diseases, which they used to shun as lacking in blockbuster potential. New York-based Pfizer (NYSE: PFE) is among the ranks of Big Pharma companies pursuing new therapies for orphan diseases. “Things have changed,” Russell says. “The classic pharmaceutical model was built around economies of scale, reach, global presence, and sheer size. Everything was focused on getting that physician to write that script.”

Russell believes the new economics of healthcare are driving the drug industry’s flight to the orphan-drug market. “The world is much more holistic now,” he says “We have to factor in payers, we have to factor in the patients being much better informed because of the Internet, and then we’ve got policymakers that are aggressively addressing issues of cost.” As a result, he says, “We’re seeing the shrinking of that mass-marketing model.”

Certainly the economics of rare diseases have been positive for Shire. On April 28, the company announced that revenues in the first quarter jumped 19 percent year-over-year to $972 million. Replagal sales skyrocketed … Next Page »

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Edimer Pharmaceuticals wants to break new ground in the treatment of patients with an ultra-rare genetic disorder called X-linked hypohidrotic ectodermal dysplasia, or XLHED. And the small venture-backed operation based in Cambridge, MA, is tapping the talents of some of the area’s rare disease veterans to try to pull it off.

The firm, with primary financial backing from Third Rock Ventures in Boston, has been laying the foundation for its hoped-for clinical trials for more than a year. Edimer has been doing various studies in hopes of asking regulators for permission to begin initial human trials of a protein-based drug for XLHED in the second half of this year, company CEO Neil Kirby says. The treatment has the potential to be the first drug specifically for the disorder.

A severe disease, it can cause patients to have few and pointed teeth, sparse hair, and thin skin. Most prevalent in boys, the disease can rob them of their ability to sweat when they need to prevent their bodies from overheating, among other symptoms. (Charles Darwin, having seen people with the disease in the late-1800s during a trip to what is now Pakistan, described them as “the toothless men of Sind.”) It can also lead to frequent pulmonary infections because patients lack the ability to produce enough mucus to clear infectious invaders from their lungs. The infections can be fatal.

There are only about 1,000 new cases of the disease diagnosed in the U.S. and Europe per year, according to Kirby. He certainly knows what it’s like to pursue a market like that, given his prior executive experience at Lexington, MA-based Shire Human Genetic Therapies (formerly Transkaryotic Therapies) from 2002 to 2007. The scant incidence of XLHED places it in a category of ailments that some call “ultra-orphan” diseases. One advantage: Kirby says that he knows of no other group that is developing a treatment for the disorder. Lack of competition also means there’s likely to be strong demand for any effective new therapy for this condition.

Cambridge, MA-based Genzyme (NASDAQ:GENZ), for example, has made billions of dollars selling rare disease drugs that cost hundreds of thousands of dollars annually for each patient who takes them. Shire HGT, Kirby’s former employer, is a fast-growing unit of Dublin-based Shire and brought in more than $900 million in revenue last year from sales of a handful of rare disease drugs. Both Shire and Genzyme have developed drugs for the rare genetic disorders known as Fabry and Gaucher diseases.

While patients with those rare diseases have multiple treatments that target their disorders, those with X-linked hypohidrotic ectodermal dysplasia haven’t been so fortunate. To control their body temperatures, and to compensate for the fact that their disease inhibits the development of sweat glands, people with the illness wear cooling vests and have to restrict their exposure to heat. (Imagine having to tell your child that he or she has to stay indoors on a beautiful, yet hot, summer day.) They might also opt to get dental implants, which often require reconstructive surgery to their jaws.

“It’s heartbreaking to go to these meetings and see a two or three-year-old wearing … Next Page »

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Six patients with the rare genetic disorder Fabry disease have filed a lawsuit against Cambridge, MA-based biotech company Genzyme (NASDAQ:GENZ) and Mt. Sinai Medical School over the handling of a rationing system for Genzyme’s Fabry treatment, agalsidase beta (Fabrazyme), the online publication Pharmalot reported yesterday. Genzyme, which is being bought by the French drug giant Sanofi-Aventis (NYSE:SNY) for $20.1 billion, started rationing its Fabry therapy after a temporary closure in 2009 of its Allston, MA, plant where the treatment is made. Mt. Sinai, of New York, is named in the lawsuit because it licensed the treatment to Genzyme and “went along with the rationing plan,” Pharmalot wrote.

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We asked our readers to chime in this week on the value of the venerable biotech Genzyme (NASDAQ:GENZ), which has reportedly become an acquisition target of the French drug giant Sanofi-Aventis (NYSE:SNY). Well, the results are in, but the question remains how our readers’ predicted price will match up with what Sanofi offers, and whether that offer will be enough to persuade the powers that be at Genzyme—especially company chief Henri Termeer—to sell.

Our readers sent in the Genzyme stock price that they felt best reflected the company’s value in a buyout, and the average price from our first 100 respondents was $76.29 per share. (See our graph below for a distribution of prices that people submitted.) Thus, our readers think that Genzyme, the world’s biggest maker of drugs for rare diseases, is worth about $20.4 billion. That’s a good deal more than the $70.24 price of Genzyme stock as of 10:31 am Eastern time today, and it might be more than Sanofi wants to part with to own the biotech company.

Sanofi’s board has authorized the company’s executives, led by CEO Chris Viehbacher, to formally offer up to $70 per share or $18.7 billion for Genzyme, Thomson Reuters reported this morning. If it were up to our readers, Genzyme’s Termeer will say “no thanks” to Sanofi’s offer. And according to a Bloomberg News report, Termeer already passed on an informal offer from Sanofi last week, and sources tell the news service that Genzyme is worth up to $22 billion or $80 per share in a takeover.

Genzyme, which had 2009 revenue of $4.5 billion, makes several high-margin drugs for rare illnesses like Gaucher’s and Fabry diseases that would make nice additions to almost any Big Pharma company’s menu of products. (Reports say that other potential Genzyme bidders include British drug behemoth GlaxoSmithKline (NYSE:GSK) and New Jersey-based Johnson & Johnson (NYSE:JNJ). On the other hand, Genzyme’s manufacturing woes in recent years, especially at its Allston Landing factory in Boston, present any prospective buyer with some unwanted baggage to inherit in a takeover. Genzyme already paid $175 million in fines to the FDA this year for messy drug-vial-filling operations in Allston, and the agency is threatening the firm with further expensive penalties if it doesn’t clean up its act.

After major shareholders complained about the company’s handling of the manufacturing problems, Genzyme cut deals with activist investors Ralph Whitworth and Carl Icahn this year. Whitworth and two Icahn associates have taken seats on Genzyme’s 13-member board of directors.

Very quickly, we will learn what the Genzyme board’s appetite is for selling the company. In most such cases, it all comes down to how much.

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With his leadership under attack and FDA regulators cracking down on his company’s manufacturing, Genzyme chairman and CEO Henri Termeer is traveling to meet with the firm’s top 30 shareholders over the next month.

The road trip, discussed yesterday in an interview with Genzyme spokesman Bo Piela, comes just over a month before the company’s annual meeting on June 16. At the meeting, the billionaire investor Carl Icahn aims to unseat Termeer and three others on the firm’s board of directors. And Icahn, who has a 4.9 percent stake in the firm and a record of ousting executives from troubled companies, holds plenty of ammo for his campaign to gain greater control of Genzyme.

A litany of mishaps has riddled Cambridge, MA-based Genzyme (NASDAQ:GENZ), the world’s largest maker of rare disease drugs, during the past year. Viral contamination caused a temporary shutdown at its Allston, MA, drug plant. Shortages of key products have slowed sales. The FDA has slapped fines on the firm for manufacturing violations. And some unhappy investors, Icahn the most vocal among them, want changes—including an end to Termeer’s nearly three-decade reign at the company.

Henri Termeer, CEO of Genzyme

David Meeker, Genzyme’s chief operating officer, candidly summarized the situation at his company when, during his speech at an industry meeting in Cambridge last month, he said: “We’re obviously in a tough spot.”

Many of Genzyme’s problems stem from its manufacturing problems. In June, the company revealed that it was temporarily closing the Allston Landing plant because a virus was found in a bioreactor needed to produce the firm’s top-selling product, imiglucerase (Cerezyme), which is used to treat a rare genetic illness called Gaucher’s disease. Sales of the drug fell from $1.23 billion in 2008 to … Next Page »

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Genzyme, the maker of drugs for rare genetic diseases, said today it has started enrolling patients in a study of patients with mild Fabry disease to see if a low dose of Fabrazyme will help. Data from the study could enable the company to apply for expanded approval of the drug for early treatment of the disease, the Cambridge, MA-based company (NASDAQ: GENZ) said in a statement. Fabry disease is a hereditary condition related to a defective X chromosome, and affects about 5,000 patients in the world, Genzyme says. Sales of the product grew 21 percent in the second quarter, to $126.6 million.

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