Notes from BIO: Vaccine Applications to FDA Booming

The Food & Drug Administration expects to receive as many biological license applications for new vaccine products in a four-month period later this year as the agency has ever received in a full year.

The onslaught of applications suggest a big year for vaccine approvals in 2010.

“It is going to be a busy year” for the agency’s Center for Biologics Evaluation & Research, the head of the agency’s biologics review management, Robert Yetter, told an FDA Town Hall meeting here at BIO. FDA is planning for the uptick in applications based on conversations with sponsors and indications from the sponsors of projected filing dates.

The ramp up in applications is tangible evidence of the past five years of accelerated activity in the vaccine field. In 2008, FDA approved three new vaccines: two were new mutli-component products Kinrix (DTaP with polion from GlaxoSmithKline) and Pentacel (DTaP with polio and Haemophilus B from Sanofi-Pasteur); GSK also received approval for its rotavirus product (Rotarix).

The bolus of review applications will hit FDA at an awkward time as FDA deals simultaneously with efforts to support the preparations for a potential return of the H1N1 flu with the next northern hemisphere flu season.

Yetter reported that CBER is already looking at ways to move license applications and supplements for pandemic vaccines “as quickly as we can.” He noted that the agency has developed experience with dealing with emergency licensing procedures for vaccines in recent years. FDA licensed a Sanofi-Pasteur avian flu vaccine in 2007 to allow the federal government to purchase it for the national stockpile. Yetter said that the agency will “use every pathway” to speed vaccines for the H1N1 outbreak.

Yetter pointed out, however, that FDA is patently aware that it cannot take risks or appear to take short-cuts on vaccine approvals in an emergency. He noted that the agency has to be able to convince the public that a vaccine is safe or they went take it even if FDA gets it out for use.

At least two major vaccine manufacturers, Novartis and Wyeth, have important vaccine projects under review which also could get caught up in the increase of workflow at CBER. Novartis has its first meningitis application for Menveo for people 11-55 pending from last August. Wyeth has the key generation shift for the pneumoccoal conjuate franchise active at FDA in a BLA for Prevnar-13 since March 31. The application was okayed for a priority review in early May.

Notes From BIO: Josh Boger Goes Back to Merck

Okay, no, this isn't a news flash. We don't have a scoop on what Vertex' outgoing CEO Josh Boger plans to do once he officially leaves the company he founded at the end of this week. And we certainly aren't predicting that he will return to Merck, where he began his career in pharmaceutical R&D before leaving to found Vertex in 1989.

But we do know what Boger did when it was time to step down as chairman of the Biotechnology Industry Organization: he returned to the legendary vision statement offered by George Merck in 1950, which served as the touchstone for Merck's vision of leadership for the rest of the 20th Century.

George Merck's famous advice--usually paraphrased as "putting patients first"--is not just a slogan, Boger observed, but a business plan, complete with the assertion that the "better" industry remembers that medicine is "for the people...not for the profits," the better the profit ultimately is.

It is also a mission statement for the future: "We cannot rest until the way has been found to bring our finest achievements to everyone."

Notes from BIO: Swag Review

Although overall it's probably true that companies have toned down their exhibit hall shenanigans this year at BIO, there are still some stand-outs in the giveaways department.

Late yesterday afternoon (after a long day of working very hard, of course) we ventured into the Exhibits for the first time. We know you're curious, so here goes.

Re Louisiana: "The beer coozy technology they're developing down there really surpasses anything I could have possibly imagined," said one biotech CEO. We couldn't agree more.

Novo Nordisk was giving away drumsticks. Not the chicken-leg kind either. Proper drumsticks. We didn't pick up on why, but this will certainly make a lot of children happy and a lot of attendees eventually wonder: why on earth did i pick those up?

Argentina takes top honors for the best metaphorical exhibit, running tango demonstrations that drew large crowds. After all, everyone at BIO is interested in learning how to dance in lock-step with an attractive partner.

We couldn't pass Haiwaii by, of course. Who among us wouldn't rather our business be based there. Greeters offering leis amid the lovely strains of the hula made it seem so possible--but the gentleman in native garb looked positively frigid in the overcooled Atlanta exhibit hall.

Hungary was offering some kind of apricot cocktail. Is there a laetrile angle there?

Finally we should mention that Merck-Serono's booth lacks tchotchkes entirely--they're making a $5 donation to the National MS Society for each person who drops by their booth. Any other companies doing something similar?

We'll try to stop by the exhibit hall again today, so let us know what we missed. Also swing by the lovely Elsevier Business Intelligence booth (1737) where you can pick up some magazines and get a database lesson or two ... OK yes we're giving away puzzles.

Notes from BIO: Meet Merck-Serono's Portfolio Guru

While Big Pharma grapples with its business model dilemma and R&D structures, some of its mid-sized competition is busy reaching out in an effort to boost externalization at the early stages of the research continuum.

On Tuesday afternoon we talked with Merck-Serono's executive vice president of portfolio development Vince Aurentz, also a member of the company's executive board, who is essentially tasked with making sure the company gets value from the R&D investments it makes.

It's a unique role. "I don’t think anyone else in industry has this job description," says Aurentz, who is responsible for all business development and licensing, as well as mergers and acquisitions. If that wasn't enough, all R&D project heads report into Aurentz as well.

He has been busy. Merck-Serono has been reviewing its pipeline and deciding what to prune. Of the recent agreement to end a collaboration with NovImmune, for example, Aurentz says "These compounds just didn’t fit where we were going. We’re trying to be a good partner, and sometimes that means you need to untangle relationships." Overall he says that Merck-Serono has looked at trimming about forty ongoing projects since 2007 as part of a portfolio review.

There has also been a decided shift toward earlier stage opportunities. Merck-Serono has in the past few months announced two separate corporate venture capital initiatives and is busy funding R&D within academia as well.

The timing was right to start the corporate venture funds, says Aurentz, partly because of the lack of early-stage funding for projects that aren't quite at the licensing stage. It also reflects a dearth of quality clinical assets available for in-licensing.

"Part of it is because of the lack of [later-stage] opportunities. But it is also because it doesn't make sense for us to build these capabilities out internally," in terms of research infrastructure, says Aurentz. At this point at least 60% of the company's projects are externally sourced, a figure that is only likely to grow.

In fact, he says, "we're passed the point as an industry where recreating all these research capabilities in-house makes sense. It's inefficient. Right now it’s hard not to view business development as the natural source of our product candidates."

Other companies are coming to similar conclusions. Bayer-Schering has also redoubled its academic outreach efforts, with a grant program that is part of a broader effort to drive innovation. Though in our interview with that company's BD head Michael Yeomans on Monday he noted that Bayer's shift toward accessing earlier stage assets hasn't extended to the venture capital arena. “We don't have a corporate VC fund,” says Yeomans. “But we do make investments as a limited partner in certain cases."

Notes from BIO: BIO, Meet Sanofi; Sanofi, Meet BIO

Sanofi-Aventis, which now calls itself a US and European company, is searching aggressively for partners, according to remarks made at a press conference on Tuesday.

“The message we want to pass on at this BIO conference is we are partnering more and we wish to do more,” said Jean-Claude Muller. Muller is VP for R&D, prospective and strategic initiatives in CEO Chris Viehbacher’s plan to grow the proportion of Sanofi’s business that comes from external relationships - currently at 27 percent. The announcement was a first for Sanofi at BIO.

“Our key message is the proportion [of business] will grow through partnering because value comes from partnerships,” Muller said. With a partnering word cloud like this (see pic) it's hard to argue isn't it?

Muller and Philippe Goupit, who is VP for business development and corporate license, are charged by Viehbacher with reshaping the company’s R&D to make Sanofi a diversified global leader in health care. Viehbacher “is really turning the page and entering into the 21st century,” said Goupit, who also testified the CEO has pledged to spend 20 percent of his time on personally exploring new business opportunities. --Shirley Haley

Notes From BIO: Treating the Government as a Partner

"You need to be thinking about a sustained relationship with the federal government in all of its arms." That was the advice of Amgen Executive Director-Global Public Affairs Andy Swire yesterday to biotech companies during a lively panel discussion on "The Reimbursement Policy Disaster" facing biotech.

Amgen has one of the largest (and, for our money, most capable) policy and advocacy staffs in Washington DC, and we thought a lot of what Swire had to say resonated at a time when all biopharma companies are preparing for an era of health care reform. So here is some of his advice for companies trying to survive and thrive in a challenging policy climate.

Swire began by contrasting a policy approach that stresses a "sustained relationship" with one that may be more "transactional." Since policy issues seldom disappear, he suggested, it is a mistake to approach them with the latter mindset.

Sponsors have to be trusted by those they interact with. "Its not that 'trust' means they have to like you," he said. It means "they have to trust that you are giving a comprehensive view" of the issue in question, even if it is one where there are deep disagreements.

In addition, "you need to be able to speak the language of the government." Focus on things like patient safety. Government officials are "not typically concerned about the profitability of an innovator company." Sponsors need to understant the intersection of their priorities with those of the agencies and officials they interact with.

Communication should be consistent. "Don't cherrypick only the good news," he said.

Last but not least: engage. "If you don't engage, they will be reading the press clippings and making conclusions based on that."

Notes from BIO: Adimab Pulls Back the Curtain

Adimab, the yeast-platform antibody discovery play we wrote about here, has essentially been quiet about the specifics of its highly anticipated platform technology. Until today.

During a panel here at BIO founder Tillman Gerngross unveiled the details this morning: Adimab is re-engineering yeast cells to create antibody factories. The technology is completely separate from the GlycoFi yeast manufacturing system that Gerngross sold to Merck in 2006 for $400 million.

"In essence we’ve created a synthetic human immune system in yeast, and the yeast will do what a normal B cell does, which is create an antibody," Gerngross explained during his presentation. Those antibodies can then be optimized and eventually manufactured in a variety of expression systems: yeast, CHO, whatever. He claims the Adimab libraries will have a diversity that is more functional and more human than other ‘fully human’ antibody technologies, because of the way they're created.

The proof of Adimab's success will come when it executes on deals for the technology, Gerngross said in an interview. The company is currently working on three customer programs--a kind of 'try-before-you-buy' arrangement that will allow companies to kick the tires. “We want potential partners to convince themselves that it works. You send us an antigen and we either send you 100 antibodies in eight weeks or we don’t.” Gerngross promises more news on the partnering front "in the not too distant future." Given his and co-founder Dane Wittrup's track records, we have a feeling the news will be interesting.

Validation will not be coming from in-house drug discovery programs, he says. “We have no intention of becoming a drug discovery company,” says Gerngross. Why not? “Because we’re not good at it! Let everyone else do it, everyone else certainly wants to. We’re providing what we think will be the best tool to do it.”

We'll have more on Adimab's platform technology in the next issue of START-UP.

image from flickr user danagraves used under a creative commons license.

Notes From BIO: The Pathology of Personalized Medicine

Translating the vision of personalized medicine into reality is no simple matter, but here's an obstacle we hadn't considered before: the need to update the 19th century science of tumor pathology to address 21st century demands for biomarkers.

Pathologists rely on microscopic analysis of formalin-fixed, paraffin-embedded dead tumor cells--an analytical process that is logistically complicated and involves destroying most of the biological information contained in living tumor cells. For biopharma companies hoping to develop more targeted cancery therapy, there has to be a better way.

That, in a nutshell, is the business plan for Baltimore-based Biomarker Strategies, a 2007 start-up formed by Johns Hopkins professor of pathology and oncology Douglas Clark. Clark described the problem--and his company's proposed solution--during a panel discussion this morning on Integrating Genetic Markers in Drug Development.

Biomarker Strategies is developing an ex-vivo life tumor cell biomarker identification device--called SnapPath--which, Clark says, can help identify appropriate biomarkers and guide treatment choices, essentially at the patient's bedside.

We have no idea if SnapPath is a viable solution, but we appreciate the case study in the complexity involved in making targeted medicine a commercial reality. The science is revolutionary, but it will work only if there are also revolutionary changes in the practice of medicine to enable their use in the real world.

Notes from BIO: Delicious Drug Delivery

We took part in a media meet and greet this morning billed as a 'speed-dating' session. Now maybe it's been a while since we were playing the field but as we sat down at our first table to give the IN VIVO spiel we were surprised by one aspect of the modern dating ritual.

Did we get flowers, a heavily perfumed come-on? No, we were offered some peanut brittle. And then told said peanut brittle was in Phase III development for type-1 type-2 [thanks to the commenter who pointed out the error] diabetes. Is it the real deal? We don't pretend to know (and to be fair the 'drug candidate' isn't the brittle itself, but an ingredient, already FDA approved as a food additive). But that's gotta be the best drug delivery pitch we've seen in some time.

image from flickr user u m a m i used under a creative commons license.

Notes from BIO: Not Just Another Report

Has it really been just 18 months since a subcommittee of FDA's Science Board released its report, "FDA's Science and Mission at Risk?"

As Gail Cassel (Lilly VP-Scientific Affairs and chair of the committee that produced the report) pointed out in an update we checked out this afternoon, the report's message: "The demands on FDA have soared; resources have not," was "against the grain of thought at the time."

An industry spokesman, Cassel noted, wondered aloud after the report was released, whether FDA really needed more funding. That "attitude certainly has changed," Cassel noted. Industry is very much on board with advocacy for more funding for the agency; FDA's budget is now almost 50% higher from appropriated funds (based on FY 2010 proposals) than it was before the report came out.

The session included updates from several Science Board committee members, as well as former FDA Chief Scientist Frank Torti (now back at Wake Forest after a year at the agency). Torti's discussion of FDA's progress in implementing the recommendations of the report garnered praise from Cassel for "the massive change you've been able to bring about."