PhIII failure blights Shire’s effort to crack new market for bowel drug

Posted under Blog, Clinical Trials, Companies, Diagnostics, diverticulitis, Funding, Lialda, Medical Devices, Medical Supply, Pharmaceuticals, R&D, Shire Pharmaceuticals, Startups, Universities, Videos by john

Shire's ($SHPGY) hopes to open up a big new market for its bowel drug Lialda were blighted today by the news that the drug had failed a late-stage study. Mesalamine, the active ingredient in Lialda, proved no better than a placebo after two years of treatment for diverticulitis. The disappointing readout leaves one more late-stage study underway, but Shire says that it has no plans to file for an approval, regardless of the outcome.

Investigators recruited patients in 10 countries for the lengthy study, expecting to find that the anti-inflammatory mesalamine could prevent a recurrence of diverticulitis between treatments. And while the company didn't outline the data, investigators say the drug failed both the primary and secondary endpoint. News of the study failure triggered a quick 4% slide in company shares.

"PREVENT2, a large, well-controlled trial, provided us with important information regarding diverticulitis." said Dr. Jeffrey Jonas, Shire's senior vice president of R&D for specialty and regenerative meds. "We will continue to analyze these data and those of the second study, PREVENT1, which was similar in design to PREVENT2 and will report later in the year. Although the results of the second trial are pending, it is our current intention not to pursue a regulatory filing for this indication for MMX mesalamine."

Lialda, an oral drug, is approved in the U.S. and Europe for ulcerative colitis.

- here's the release

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Growing popularity of virtual biotechs promises boon to outsourcers

Posted under Blog, Companies, Diagnostics, FerroKin Biosciences, Funding, Medical Devices, Medical Supply, outsourcing, Pharmaceuticals, Shire Pharmaceuticals, Startups, Universities, Videos, Virtual model by John Carroll

The recent news that tiny FerroKin BioSciences earned a big buyout deal with Shire ($SHPGY) has helped spotlight the growing popularity of the virtual biotech model. And that has big implications for everyone in the outsourcing industry.

Like other virtuals--such as Stromedix, recently acquired by Biogen Idec ($BIIB)--FerroKin had only a handful of employees working full-time for the company. CROs, CMOs and other outsourcers created a network of support vendors that carried out much of the heavy lifting in drug research. And with some proof of concept data in hand, FerroKin made a tantalizing morsel for an acquirer looking to build up its pipeline without having to acquire a sizable research infrastructure it didn't need.

There's little doubt that a number of venture companies are behind this trend, as several made clear to me at BIO-Europe Spring in Amsterdam earlier this week. Investors like CMEA, Index and others believe that virtual companies can quickly accelerate a drug program to PoC, giving them a chance to cash out relatively quickly with a sale to Big Pharma. And the trend hasn't escaped the attention of the outsourcing industry as a whole.

"There's a herd mentality. If there's a string of successes, then the herd will follow," says Celtic Pharma's lead counsel Allan Cohen, according to a story in Outsourcing-Pharma.

- here's the story from Outsourcing-Pharma

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Shire nabs Bay Area’s FerroKin, orphan drug in $325M buyout

Posted under Blog, Companies, Diagnostics, FerroKin Biosciences, Funding, Medical Devices, Medical Supply, Mergers and Acquisitions, Pharmaceuticals, Shire Pharmaceuticals, Startups, Universities, Videos by John Carroll

Fresh off a disappointing decision to jerk its marketing application for Replagal, Shire ($SHPGY) bounced right back with its announcement that it has scooped up the Bay Area biotech FerroKin BioSciences in a $325 million deal. Shire is paying $100 million upfront for FerroKin, with the rest offered in potential milestones.

That's a big payday for a "backyard" biotech that won national attention in a 2011 feature in The Atlantic. Founder and CEO Dr. Hugh Young Rienhoff operated the company from his house, with a virtual staff of 7 and 60 vendors offering outsourced support. Rienhoff told The Atlantic he had raised a total of $27 million for the company.

The biotech fits snugly into Shire's orphan drug strategy. FerroKin has been gathering proof-of-concept data for FBS0701, which is designed to counter the iron overload anemic patients can suffer from after repeated transfusions of red blood cells. FBS0701 has won orphan status in both Europe and the U.S. In the summer of 2010 the biotech announced it had nailed down a $12 million financing round from current investors Burrill & Company, Clarus Ventures and MP Healthcare Venture Management, with Healthcap Ventures of Stockholm coming in as a new investor.

"There remains a significant unmet need for a once-a-day, oral iron chelator in a convenient dosage form for the treatment of transfusional iron overload with a better safety profile than currently available treatments," explained Shire hematology chief Ross Murdoch. "We believe FBS0701 has the potential to meet that need.  We hope to use our expertise in hematology coupled with our proven ability to progress products through the development pipeline to bring FBS0701 to the global marketplace. This acquisition marks an important step for Shire in building a business that serves the growing needs of specialty hematologists and their patients."

The deal comes after FerroKin displayed some of its mid-stage data at ASH a few months ago. FerroKin completed four Phase I studies before launching its dose-ranging Phase II trials. Rienhoff will provide consulting services to Shire during the transition period.

- here's Shire's release

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GSK lassos rare disease program in Angiochem tie-up

Posted under Angiochem, Blog, Companies, Diagnostics, Funding, Geron, GlaxoSmithKline, Medical Devices, Medical Supply, Pharmaceuticals, rare diseases, Shire Pharmaceuticals, Startups, Universities, Videos by Ryan McBride

GlaxoSmithKline ($GSK) has grabbed rights to biotech startup Angiochem's early-stage program for treating a lysosomal storage disease. The deal comes as the London-based drug giant and its large pharma peers plunge headlong into the rare diseases field.

Angiochem, headquartered in Montreal, expects to get up to $31.5 million in upfront, research and additional fees from GSK if the drugmaker decides to secure rights to more of the small company's rare disease treatments. Depending on a lot of things going its way in its rare diseases program, and the partnership expanding, Angiochem said that it could bring in more than $300 million from its work with GSK.

GSK, which develops orphan disease drugs internally and via several partnerships, has made treating rare diseases in the brain a main research priority. With Angiochem's technology, GSK hopes to be able to develop at least one enzyme therapy for a lysosomal storage disease that can be smuggled through the blood-brain barrier into the central nervous system--where no current treatments against such rare diseases can reach.  

Developers of rare disease drugs are trying to figure out how to get their protein treatments into the brain, including Shire's ($SHPGY) Human Genetic Disease unit, which is working on a device that administers the treatments into the cerebrospinal fluid. Angiochem develops drugs that are linked to a peptide that homes in on the LRP-1 receptor to sneak drugs into the brain, and last year the company began testing an enzyme-replacement drug that is linked to its brain barrier-penetrating peptide, company CEO Dr. Jean-Paul Castaigne told FierceBiotech. His company's research has gone as far as to show that its tech can deliver enzyme drugs for lysosomal storage diseases into the brains of mice.

Venture-backed Angiochem had some early success in using its tech to carry cancer drugs into the brain to attack tumors, and Geron ($GERN) snagged rights to its Phase II brain cancer candidate in 2010 for $35 million. GSK is the small company's first major partner in the rare diseases arena, and Angiochem hopes that success in the partnership will lead to deals with other drugmakers. The CEO said that the company's enzyme replacements for rare diseases are intended to treat tissues in the brain and the rest of the body. 

"[The deal] means a lot," Castaigne said. He added that "the fact that we may bring drugs to the market with Glaxo that address the CNS symptoms of these diseases makes all the people in the lab very proud."