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Archive for the ‘hemophilia’ Category

Feb
01

Sangamo spikes on $13M discovery pact with Shire

Posted under Blog, Companies, Diagnostics, Funding, hemophilia, Medical Devices, Medical Supply, Pharmaceuticals, Sangamo BioSciences, Shire Pharmaceuticals, Startups, Universities, Videos by John Carroll

Shares of Sangamo BioSciences spiked 22% this morning after the Richmond, CA-based biotech announced plans to team up with Shire on a discovery pact to find new treatments for hemophilia and other monogenic ailments. Sangamo will bank $13 million upfront, as well as an unspecified set of milestones and a royalty on any approved treatments that emerge from the work.

Sangamo ($SGMO) also gains R&D support from Shire in the deal.

The deal marks an encouraging endorsement for Sangamo's zinc finger DNA-binding protein technology. ZFPs can be engineered to recognize a specific DNA sequence within a gene, making Sangamo a valuable partner as Shire explores new drugs for hematology and lysosomal storage disorders.

"Sangamo's ground-breaking ZFP gene-editing technology will enable us to expand our therapeutic pipeline into therapies for other genetic disorders such as hemophilia," said Sylvie Gregoire, president of Shire's Human Genetic Therapies business.

Sangamo needed a shot in the arm. The biotech was forced to scuttle its lead drug program last fall after it flunked both primary and secondary endpoints. An unexpectedly strong placebo response surged past the drug arm results.

- here's the press release

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Sangamo scuttles lead drug program after it fails PhIIb

Jan
12

Biogen CEO Scangos aims to patch weak points in pipeline

Posted under BG-12, Biogen Idec, Blog, Companies, Diagnostics, Funding, George Scangos, hemophilia, Medical Devices, Medical Supply, multiple sclerosis, Pharmaceuticals, Startups, Universities, Videos by Ryan McBride

After two years of sweeping change at Biogen Idec ($BIIB), CEO George Scangos (photo) and his team have some work to do on plugging some of the holes in the company's early-stage pipeline, from which many programs were cut during the company's exit from oncology and cardiovascular drug research.

"We didn't want to fund projects that were unlikely to generate value," Scangos told Dow Jones Newswires' Peter Loftus at the J.P. Morgan Healthcare Conference in San Francisco. There were 17 programs axed from the company's pipeline in 2010 and 2011, leaving the company with a wealth of late-stage programs, notably the potential blockbuster multiple sclerosis pill BG-12, but short on early-stage programs.

Steven Holtzman, Biogen's chief dealmaker, told FierceBiotech in an interview that he didn't view the shortage of early-stage programs as a problem for the company, but rather as an opportunity. The company, which is the world's top provider of MS drugs, is most interested in beefing up its pipeline in the areas of neurology, autoimmune diseases and hematology.

Hematology is one area in which the imbalance of late- versus early-stage compounds is particularly acute. Biogen expects later this year to report pivotal data from its late-stage hemophilia programs, including long-acting factor VIII and factor IX treatments, but the company has nothing in Phase I or Phase II to follow those products if they get approved.

"Hematology is an area we have not been in before. We're very interested in adding to that area, but we have to be thoughtful about when is the right time, because we have nothing to anchor it yet," Holtzman told FierceBiotech. "But we will have the data from the hematology products later this year, and if those look good, you might think about building out that franchise."

But the bottom line is that Biogen has an enviable late-stage pipeline, and there's no shortage of biotechs in the universe that are interested in finding partners for early-stage programs. So don't expect Scangos and Holtzman to lose sleep over the lack of Phase I programs.

- here's the Dow Jones article

Special Report: Biogen Idec - Biotech's Biggest Spenders 2011

Related Articles:
Biogen CEO Scangos sees room for improvement in 2012
After the R&D shakeout, Biogen aims to beef up drug pipeline

Dec
22

UPDATED: Ipsen-backed Inspiration Biopharma leaving California for Boston area

Posted under Blog, Boston, Companies, Diagnostics, Funding, hemophilia, Inspiration Biopharmaceuticals, Ipsen, Medical Devices, Medical Supply, Pharmaceuticals, Startups, Universities, Videos by Ryan McBride

Score one for the Boston-area biotech scene. With a newly appointed chief executive from Genzyme, Inspiration Biopharmaceuticals is relocating from Laguna Niguel, CA, to the biotech-rich Kendall Square section of Cambridge, MA. And the move comes amid the company's planned transformation from a development-stage outfit to a commercial provider of bleeding-disorder drugs.

"We're building a fully integrated company,'' Inspiration CEO John Butler, who was previously president of the rare genetic disease unit of Cambridge-based Genzyme, said in an interview with The Boston Globe. "Ultimately, we're a bricks-and-mortar company as of Jan. 1. Going forward, Cambridge is our address. We expect to have two products on the market in the second half of 2013.''

Ipsen's partnership with Inspiration, which is 40% owned by the French biotech, played a role in the relocation to Massachusetts as well, according to the Globe's article. Inspiration recently filed for approval of its experimental factor IX product for hemophilia B patients in Europe, where Ipsen has rights to market the drug. The company is also planning an FDA app for approval of the drug early next year. Ipsen has set up a bioprocessing operation in Massachusetts where the treatment will be made, and the Paris-based biotech has an option to acquire Inspiration which is tied to whether the developer'' two lead bleeding-disorder drugs pan out, the Globe reported.

Inspiration's move to Massachusetts, of course, won't really improve its chances of getting market green lights for its hemophilia products. Yet there's no shortage of biotech talent walking the streets of Kendall Square, where Novartis ($NVS), Biogen Idec ($BIIB), Pfizer ($PFE) and others are building up their existing hubs and adding to their teams of scientists and business groups. And Ipsen recently revealed plans to plow $45 million into and add 100 workers to its R&D operation in Milford, MA. Inspiration is unlikely to have problems beefing up its own work force as it readies for potential commercial releases of its products.

- here's the company's release
- get more in the Globe article

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Dec
15

Ipsen plans new hires, $45M R&D expansion as it relocates U.S. HQ

Posted under Blog, Companies, Diagnostics, Funding, hemophilia, Ipsen, Medical Devices, Medical Supply, Pharmaceuticals, Startups, Universities, Videos by John Carroll

Paris-based Ipsen is reorganizing its U.S. operations, shifting its American headquarters  from California to New Jersey, hiring 100 employees and investing $45 million in an upgrade of its R&D facilities in Milford, MA. The moves follow Ipsen's $663 million deal to buy Tercica, an endocrine health biopharma company, in Brisbane, CA back in 2008. Now that operation is being renamed Ipsen as part of the reorganization and expansion in the U.S.

Ipsen already has a big presence in Milford, where its peptide and toxin R&D work, as well as manufacturing, is handled. One of its two buildings on the site will be renovated while a third building goes up to expand their work there. Its R&D staff is working on the hemophilia treatment OBI-1, which Ipsen describes as "a breakthrough B-domain depleted, recombinant porcine factor VIII in late stage development with Inspiration Biopharmaceuticals." Researchers are now recruiting patients for two late-stage trials.

The move from California to New Jersey makes a lot of sense, says Sean McKercher, the president of Ipsen North America. Ipsen decided to undertake a geographic review of its operations, and determined that it wanted to focus on the U.S., among other regions. "The U.S." McKercher tells FierceBiotech, "is an emerging market for us." And New Jersey is a lot closer to the Paris hub, making it easier to coordinate activates with closer time zones.

Ipsen currently sells a pair of products in the U.S. The expansion in New Jersey will focus on adding sales positions as well as new members for the executive team, but McKercher emphasized that its sales force in the field will not be affected at all by the move. He also expects to work closely with Ipsen's business development team on any U.S. pacts, which are likely to be in either the neurology or endocrinology arenas.

"We are confident that investing $45 million in this unique site, in such close proximity to the impressive concentration of scientific skills and talent in Massachusetts, will enable Ipsen to expand its bio-therapeutic technological platforms and pipeline," said Cynthia Sylvestre, who runs the Milford operation.

- here's the Ipsen release

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