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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Shire (LSE:SHP)—an Ireland-based drug company that has its Human Genetic Therapies unit based in Cambridge, MA—reports that it has asked European regulators to approve its treatment for Gaucher disease, velaglucerase alfa. If approved by the European Medicines Agency, Shire’s enzyme-replacement treatment will compete with Cambridge-based biotech Genzyme’s (NASDAQ:GENZ) market-leading imiglucerase (Cerezyme), which has faced supply shortages. Shire said it is also seeking approval of its Gaucher drug in Canada and the U.S.

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When Cambridge, MA-based Genzyme (NASDAQ: GENZ) shut down its Allston, MA, bioreactor plant in June after discovering viral contamination, it was clear that there would be worldwide shortages of its best-selling enzyme replacement therapies, agalsidase beta (Fabrazyme) and imiglucerase (Cerezyme), and that there would be a hit to the company’s bottom line. The company got more specific about those matters today, saying in a press release that it’s taking longer than expected to restore full production of agalsidase beta, and that sales of both drugs this year will be below previously anticipated levels.

Revenue from agalsidase beta, a treatment for Fabry disease, is expected to be roughly $450 million in 2009, about $60 to $70 million less than the company had predicted in previous guidance. Revenue from the Gaucher disease treatment imiglucerase is expected to reach $800 million, which is at the low end of the previously predicted range of $750 million to $1 billion. Genzyme’s stock price was roughly flat this morning.

After sanitizing the Allston plant, Genzyme resumed production of both drugs in July, and it said today that it expects to begin shipping newly produced batches of the Gaucher drug in November, as planned.

But the news for Fabry disease patients is not as encouraging. New shipments of agalsidase beta won’t go out until mid-December, the company says, and there will be less of it to go around than previously thought. The problems: Genzyme took some extra time to do preventive maintenance before restarting the bioreactors that make agalsidase beta; the vessels are now producing the drug in lower quantities than in the past, for reasons the company isn’t disclosing; and the company has been making the drug in smaller batches, which is faster but less efficient.

As a result, “additional product conservation measures are now needed to help ensure that the limited remaining Fabrazyme inventory can be made available to as many patients as possible until new material is available,” the company said in today’s announcement. Specifically, the company said that from October 1 through the end of the year, it will only ship enough agalsidase beta to meet 30 percent of global demand; up to now, the company said it had enough of the drug on hand to cover about 80 percent of demand.

Genzyme says it’s working with doctors to set up clinical guidelines for conserving doses of the drug. The company expects to work its way back to full production—covering 100 percent of demand—sometime in the first quarter of 2010.

“We will continue to work with all stakeholders to prepare for the release of this material so that it can reach patients as quickly as possible,” Genzyme chairman and CEO Henri Termeer said in today’s announcement. “I want to express my deep appreciation to the Gaucher and Fabry communities for their support of the existing product conservation guidelines and for their anticipated collaboration in helping to manage the remaining limited product supply through the end of this year.”

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Genzyme (NASDAQ:GENZ) said this afternoon that its next-generation, oral drug for Gaucher disease passed a mid-stage clinical trial, providing a degree of assurance that the Cambridge, MA-based biotech powerhouse can sustain its franchise for treating the rare genetic disease.

The study, which included 26 patients, showed the oral drug was safe and effective. And the trial showed that the drug met its main goal in 91 percent of patients who took the drug over a year’s time. The results were presented today at the Lysosomal Disease Network World meeting in San Diego.

The trial represents an important test for the drug, although it will still have to clear another late-stage clinical trial expected to begin in mid-2009 before it can win FDA approval for sale in the U.S. This drug is strategically important to Genzyme. Imiglucerase (Cerezyme), the company’s current treatment for Gaucher, is its top-selling product that raked in $1.24 billion in 2008 sales, or more than a quarter of the firm’s total revenue of $4.6 billion. But two critical patents for the treatment expire in August 2010 and August 2013, according to regulatory filings.

“Given Cerezyme’s unique safety and efficacy profile, we set a high threshold for success, and the results were better than anticipated, indicating a potent, highly-specific and well-tolerated molecule,” said Geoff McDonough, a senior vice president with Genzyme, in a statement.

The oral Gaucher drug, tentatively called Genz-112638, would come with a longer patent life, and is also intended to be a more convenient option for people with Gaucher. About 10,000 patients worldwide have the disorder, and the current imiglucerase treatment is only available through intravenous injection.

The study of Genzymes’s oral Gaucher drug showed that the treatment decreased spleen and liver growth that is common among patients with the disease. The drug also proved effective in raising levels of hemoglobin, a protein that carries oxygen in the blood, and platelet cells, which help form clots, according to Genzyme. The study also showed lower levels of an antibody released in fatty tissues in patients who took the drug.

The company says that the there was a small number of patients who had mild adverse reactions to the drug early in the trial, but those adverse events did not require medical treatment. Of the 26 patients who enrolled in the trial, 22 completed the full year of treatment, and 20 chose to stick with the drug after the study ended, Genzyme says.

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Many biotech firms in the Boston area and elsewhere are starved for capital, and initial public offerings appear to be off the table due to the economic recession. These harsh realities are likely to flavor the Massachusetts Biotechnology Council’s (MBC) annual MassBio Investors Forum next week, when 17 biotech outfits (including a new regenerative medicine startup with a new approach to developing stem cell therapies) plan to make the case for betting on their firms.

Due to the constraints on raising cash from venture firms and the other traditional biotech backers who usually attend the forum, the MBC has expanded its guest list of potential investors to include some 10 disease foundations—including the Cystic Fibrosis Foundation and Michael J. Fox Foundation for Parkinson’s Research—as well as an official from the National Cancer Institute who is launching a new Small Business Innovation Research grant program, MBC president Bob Coughlin told me.

The weakened financial market also presented fresh challenges for the MBC to have the annual event, Coughlin says. “A lot of the companies that used to sponsor these events were financial services institutions, and that money’s dried up,” he says. “By the same token, it’s very easy to get our member companies to participate because capital formation is the number one issue facing [many firms] in our industry.”

For instance, Idera Pharmaceuticals (NASDAQ:IDRA), a Cambridge, MA-based developer of drugs that modulate immune-system receptors to treat infectious diseases and other illnesses, is presenting at this year’s MassBio event to attract new equity investors and partners, CEO Sudhir Agrawal says. “I think our [target] audience is always the long-term shareholders, who can see the investment grow in time,” he adds.

Here’s an early glimpse of all the biotech firms … Next Page »

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