Biogen Idec bets it can succeed on a risky Phase III ALS gamble

Posted under ALS, Biogen Idec, Blog, Companies, dexpramipexole, Diagnostics, Funding, George Scangos, Lou Gehrig's disease, Medical Devices, Medical Supply, Pharmaceuticals, Pipeline, Startups, Universities, Videos by john

Bloomberg biotech scribe Meg Tirrell has been plumbing Biogen Idec's ($BIIB) late-stage drug for ALS and finds some good reasons to believe that they are on to something big--perhaps even blockbuster-sized.

First, dexpramipexole had to stand up to George Scangos' review process when he took the helm a couple of years ago. Anxious to trim away any low percentage shots at an approval, Scangos was won over by the data and market profile. The drug--in-licensed from Knopp Biosciences--did well in Phase II, demonstrating a significant slowing in disease progression after a brief 12 weeks of treatment at the highest dose of 300 mg daily. A later stage of the trial tracked a significant mortality benefit comparing the high and the low dose. And if the data hold up in the late-stage trial, that could translate into a sizable market of a billion dollars-plus, considering the woeful absence of any effective therapy for patients.

But Scangos had been skeptical for a good reason. A whole slate of prospective treatments has been shot down in the clinical trial process, underscoring how little is known about Lou Gehrig's disease and what needs to be done to slow or stop it. But it's late-stage study--testing 150 mg twice daily--enrolled patients in just a few months. Now late-stage data is due out later this year. A win here would help further burnish Scangos' reputation for turning around the one-time biotech laggard. 

"This is far from a long shot," CEO Scangos told investors at the annual J.P. Morgan confab in San Francisco in January. "We are genuinely hopeful that we will be able to provide meaningful therapy for the thousands, tens of thousands, of ALS patients who right now have very little to help them."

- here's the story from Bloomberg

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Biogen Idec races to FDA and EMA with a blockbuster app for BG-12

Posted under BG-12, Biogen Idec, Blog, Companies, Diagnostics, Funding, George Scangos, Gilenya, Lemtrada, Medical Devices, Medical Supply, multiple sclerosis, Novartis, Pharmaceuticals, Startups, Universities, Videos by John Carroll

Biogen Idec has filed an NDA on BG-12, taking another step forward in its blockbuster quest to gain an approval on its best near-term hope for a game-changing new treatment. Biogen ($BIIB) racked up promising late-stage data on the program, giving the company good odds for achieving an ultimate OK from the FDA. And the Cambridge, MA-based biotech is putting the final touches on an EMA submission aimed at redefining the market for MS drugs in Europe.

BG-12 is a "real potential blockbuster in the space," Biogen R&D chief Doug Williams tells FierceBiotech. "The safety profile is really quite good, and the benefit/risk profile is really positive."

Biogen is hoping to land a priority review for the treatment, says Williams, which would shave four months off the standard 10-month regulatory cycle. But even if it doesn't, the developer can be upbeat about its prospects for an approval in late 2012 or early 2013. For Biogen Idec, which plans to spend about a quarter of its revenue on R&D this year, the ability to look forward to major league approvals gives the big biotech company some critical leeway with analysts.

"We think BG-12 is going to be a very successful drug in the marketplace," says Williams. "In this business, you have to make consistent investments in R&D over a period of time. That has paid off."

Speed is of the utmost importance right now. Novartis ($NVS) made it to the MS market first with Gilenya. Now the analysts expect that BG-12 will move in to snag significant market share. And if Biogen does nail down key approvals early, they'll continue to cast a shadow over Sanofi's ($SNY) big plans for Lemtrada, the experimental MS drug obtained in the Genzyme buyout.

Williams notes that while there's no head-to-head data with Gilenya, BG-12 can compete on data demonstrating "extremely strong disease control activity across a broad range of parameters."

- here's the press release

Special Report: BG-12 - 15 top blockbuster contenders

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Biogen Idec scores lung disease drug in $562.5M buyout of Stromedix

Posted under Biogen Idec, Blog, Companies, Diagnostics, Funding, George Scangos, Medical Devices, Medical Supply, Pharmaceuticals, Startups, Stromedix, STX-100, Universities, Videos by Ryan McBride

Biogen Idec ($BIIB) has scooped up a biotech very close to home. The Weston, MA-based biotech power has bought nearby Stromedix, whose lead fibrosis drug STX-100 was licensed from Biogen. The deal brings Cambridge, MA-based Stromedix's investors, which have invested $38 million in the company, $75 million upfront and up to $487.5 million in potential milestones.

The buyout comes after Biogen CEO George Scangos (pictured) remodeled the company's early- and midstage pipeline. He and his team have cut more than a dozen programs and exited oncology and cardiology drug development. With an interest in fibrosis and a need for midstage assets, Biogen's Stromedix acquisition gives it the startup's lead program in idiopathic pulmonary fibrosis (IPF). Stromedix CEO Michael Gilman, a former EVP of R&D at Biogen, said his company is now recruiting patients for the midstage IPF trial for the antibody STX-100. In addition, he and his team will become Biogen employees and lead fibrosis research at the company.

"They know the program, they understand it, and they love it as much as we do," Gilman told FierceBiotech. "That was important for me at the end of the day to find the right home for this program and for the team. And it doesn't really get much better than Biogen."

Stromedix has patiently advanced its STX-100 program in recent years as a number of its counterparts in the fibrosis field have been gobbled up. For example, Bristol-Myers Squibb ($BMY) bought San Diego-based Amira Pharmaceuticals last year in part to gain its experimental drug for IPF. Prior to that deal, Gilead Sciences ($GILD) boosted its stake in the fibrosis field through its buyout of Arresto. The market for drugs against IPF, which causes a buildup of scarring in the lungs that causes breathing problems and eventual organ failure, remains pretty wide open, as there are no FDA-approved treatments.

Stromedix's venture backers--which included New Leaf Venture Partners, Bessemer Venture Partners, Red Abbey Venture Partners, Atlas Venture and Frazier Healthcare Ventures--now can claim a victory in backing early-stage biotechs. Especially Atlas, where Gilman formed the startup as an entrepreneur-in-residence at the Cambridge, MA, venture firm after leaving Biogen.

"We weren't really trying to sell the [Stromedix]," Peter Barrett, a partner at Atlas, told FierceBiotech. "A lot of people were watching this and saying that it would be interesting after Phase II. But Biogen came along" with an offer that made sense.

Now Stromedix won't have to worry about raising additional funds to develop STX-100 for multiple types of fibrosis. It's still early in the development of the antibody, but if it makes it to market, Biogen could win big on its Stromedix bet.

- here's the release

Related Article:
Stromedix closing in on new venture round

George Scangos – The 25 most influential people in biopharma today

Posted under autoimmune disease, BG-12, Biogen Idec, Blog, Carl Icahn, Companies, daclizumab, Diagnostics, Funding, George Scangos, Medical Devices, Medical Supply, Pharmaceuticals, Startups, Universities, Videos by Ryan McBride

Setting the pace on prioritization

George Scangos
CEO
Biogen Idec

George Scangos has shown that he belongs in the small fraternity of the very top chief executives in biotech, bringing what appears to be a golden touch to one of the industry's most important companies, Biogen Idec ($BIIB). Biogen, which had lagged biotech peers in R&D productivity and stock price before Scangos' arrival in June 2010, has been returning to grace under his leadership.

The Weston, MA-based company's stock took off last year, fueled by positive data on key experimental therapies such as the multiple sclerosis pill BG-12 and the long-acting MS treatment daclizumab. Those programs predate Scangos' arrival, but he has refocused the company's R&D organization on the areas it knows best such as neurological and autoimmune diseases.

He's been decisive--cutting oncology and cardiovascular disease research from the company's R&D mix and shuttering its San Diego research outpost just months after taking the reins. He made changes and appointments on his executive team, surrounding himself with standout veterans of the biotech game. And he made the call last year to return the company's headquarters to Cambridge, MA--all as part of his larger leadership theme of improving communication and integrating efforts.

Abandon oncology research? It's almost unheard of in biopharma nowadays, but Scangos has set the tone among his peers for prioritization--which has become essential as drugmakers narrow their focus on their most valuable programs to get the best returns possible on their R&D dollars.

Scangos also hasn't hesitated to shrug off some big trends in the business. Early on Scangos' new team decided to get out of the corporate VC side of the business, happy to go their own way and rely on a seasoned team to provide the kind of insights on new companies and technologies expected from the corporate VC teams roaming the planet. He hasn't been emulated, yet.

The hundreds of workers who lost their jobs as a result of Scangos' house-cleaning in the fall of 2010 might disagree, but Scangos has even been regarded as a friendly leader who is liked among the rank and file. (It probably doesn't hurt that employees' company stock has skyrocketed with Scangos in charge.) Yet there's no doubt that Scangos exudes confident calm, something that was seriously lacking during the infamous charades at the '09 annual shareholder meeting, when management and Carl Icahn's camp were battling for as many board seats as each could get.

Scangos' first annual board meeting was perfectly boring, another sign of contentment among investors. How content? Icahn cashed out his shares in Biogen last year. A Scangos-led Biogen had finally delivered the billionaire investor what he wanted, a big profit. With BG-12 advancing, we'll soon see whether Scangos can lead Biogen to a feat it hasn't accomplished since 2004--an FDA approval of a new drug.