Concert Pharmaceuticals, the Lexington, MA-based company that chemically modifies existing drugs to make them more attractive, has started human testing of an HIV medication which it hopes will help GlaxoSmithKline wrestle back market share it has been losing to Gilead Sciences, the world’s largest maker of HIV drugs.

Concert will receive a $12 million payment from GlaxoSmithKline for starting the trial of a drug it calls CTP-518. It’s one small piece of the collaboration announced in June that could be worth more than $1 billion over time to the smaller company. I heard about this bit of news, and what it means strategically to Concert, when I visited CEO Roger Tung at the company’s office last week.

The basic idea is to take a common protease inhibitor, Bristol-Myers Squibb’s atazanavir (Reyataz), and swap out a few hydrogen atoms on the molecule with deuterium atoms. This is supposed to retain the drug’s viral killing punch, while making it last longer in the body. That’s desirable because it could allow doctors to quit prescribing a booster drug from Abbott Laboratories called ritonavir that adds cost, complexity, and hassles, Tung says. If the Concert drug is able to remain potent long enough in the bloodstream on its own, then it could be combined with other antivirals to create a convenient once-daily pill to compete with Gilead Sciences’ efavirenz emtricitabine tenofovir (Atripla), a drug that has helped Gilead surpass Glaxo as the world’s leading maker of HIV drugs the past couple of years.

“Doctors don’t like prescribing ritonavir, and patients don’t like taking it,” Tung says. “Our hope is to take a great drug and make it more tolerable and easier to take. The idea is to be the most tolerable, best-in-class HIV inhibitor.”

Without going too far into how this works, Concert says the deuterium atoms it is swapping into CTP-518 are supposed to form stronger bonds to the rest of the molecule than the traditional hydrogen bonds. By creating a more bulletproof package that stands up to the usual digestive processes, a greater amount of the drug and desirable metabolic byproducts are supposed to make it through the intestines and into the bloodstream, Tung says. That means the Concert drug should cause less irritation in the gut that can lead to diarrhea and nausea, and that it can use lower doses to get the same amount of drug into the bloodstream.

Getting rid of the ritonavir booster drug is important, Tung says, because …Next Page »

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Combination therapy has been a central component of treatment for certain viral diseases for more than 15 years. The benefits of combination therapy can arise from activation of multiple host pathways, suppression of mutational variants that can lead to viral escape, or perhaps both.

In HIV, the benefit of combination therapy is due to suppression of viral resistance, which is the result of using multiple agents acting at distinct sites within the virus life cycle. In hepatitis C (which I’ll abbreviate as HCV) the addition of ribavirin to interferon turned what was primarily an on-treatment lowering of viral titers into the first significant rate of viral clearance that persisted even after therapy was stopped. This sustained virological response, known as SVR, has become the primary measurement of clinical benefit in HCV. The mechanism underlying the dramatic effect of combining ribavirin with interferon is not clear. The benefit could be due to a pharmacologic interaction between pathways activated by interferon and pathways activated by ribavirin, or it may be the result of a modest ribavirin antiviral effect added to an “antiviral state” induced by interferon.

Just this year, companies in the HCV field began exploring the use of direct antiviral combinations. It is hoped that by appropriately choosing complementary targets, benefits of combination similar to what was seen in HIV may soon be seen in HCV therapy. Whether or not the combination of direct antivirals will permit the elimination of interferon and/or ribavirin remains unknown at this time, and is perhaps the most highly anticipated answer in the HCV field today.

Combinations of antivirals today

Three companies have moved into the clinical stage of exploring direct antiviral combinations for HCV.

Roche is most advanced in combination studies of direct antivirals with its INFORM-1 study. In this study, HCV patients were treated for 14 days with various dose levels of two drug candidates that inhibit different parts of the virus life cycle. These drug candidates are RG7128, a nucleoside polymerase inhibitor licensed from Pharmasset, and RG7227, a protease inhibitor licensed from Intermune. Data from the first several dosing cohorts was disclosed this past April at the annual meeting of the European Association for the Study of the Liver. Additional data, including responses at higher doses and in patients who previously failed interferon/ribavirin, will is being reported at the annual meeting of the American Association for the Study of Liver Diseases conference in Boston.

The INFORM-1 study clearly shows that two antiviral agents can act in concert to produce a greater antiviral effect over 14 days than either agent produced alone. At the same time, critical questions remain for longer studies — Can direct antivirals alone retain viral titers at undetectable levels over longer periods of treatment? Even more important, will a state of virus negativity elicited by a direct antiviral combination afford the same rate of SVR once therapy is stopped as when virus negativity is induced by the interferon/ribavirin combination? Is there anything special …Next Page »

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Seattle has more than its share of brilliant biologists for a medium-sized American city. But if people and ideas are going to properly mix to create a thriving local biotech industry over the next two decades, we could use a few more common places with intellectual sparks—like bars. At least according to one of the region’s top life sciences entrepreneurs.

When in Boston and San Francisco, Stephen Friend says he gets most of his work done by socializing with scientific colleagues he meets for coffee or a drink. Not so here.

“The way Seattle is currently set up, I don’t have a place I go for a coffee or a drink. A place where I run into the people who are ready to try a new idea. That’s an important anchoring ingredient that we’re missing, and should be happening,” says Friend, the former senior vice president of cancer research at Merck, and co-founder of Seattle’s Rosetta Inpharmatics.

(Coffee itself is certainly easy to find, but Stephen may want to check out Xconomy’s handy Greater Seattle Coffee Cluster guide for the spots that are best known as innovation mixing pots.)

That was one of the many insights that emerged during Seattle Life Sciences 2029, a sold-out event Xconomy organized on Monday night at Seattle Biomedical Research Institute (see the photo gallery here). This event brought together a group of industry visionaries who have rarely, if ever, appeared on the same stage in front of a local audience: Ben Shapiro, the former executive vice president of basic research at Merck; Steve Gillis, the managing director of Arch Venture Partners who co-founded Immunex and Corixa; and Friend. The panel was moderated by Carl Weissman of Accelerator and OVP Venture Partners, and biotech pioneer Leroy Hood offered his thoughts in a video message on how Seattle can make strides to get bigger and better at biotech over the coming two decades.

This came up during a freewheeling audience Q&A, which covered a lot of ground. I’m not going to recap everything here—I think with some of the jokes, you probably just had to be there—but here were some of the best little acorns that that I squirreled away with my digital recorder, which are edited as always for length and clarity:

Steve Gillis

Steve Gillis on what entrepreneurs need to do to adapt in today’s financial climate: “Entrepreneurs need to be open to combining their ideas with existing organizations. Or combining those ideas with like-minded new proposals from other folks, as opposed to always wanting to have what I call their own pie. In the world in which we live, in which money is quite tight, people have to be open to joining forces earlier in evolution. That will result in everyone having a smaller slice of the pie, but it will also result in the possibility of someday eating pie. Instead of just dreaming about it. It’s a fundamental mind-set that needs to change.”

Stephen Friend on how universities need to relax rules on how inventive faculty spend their time: “The concept that you are either inside or outside of the university, and that some fraction of your time has to be spent inside the university, and that some percentage of your time has to be spent in your role as a tenured professor at the university, that has to go away. It’s the concept that someone could teach courses, and can be a role model for education, has to be kept separate from someone who’s actually running a company. The best example I know of is what MIT is trying to do. That organization, the person who leads it, she [Susan Hockfield] feels the university has to become, in a broad sense, the incubator. I don’t think many people, particularly at some universities in this town, are ready to think that creatively. It’s a mistake.”

Ben Shapiro on how Seattle ranks as a hub for life sciences innovation: “It’s worth …Next Page »

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[Editor's Note: this piece was co-authored by Hari Kumar of San Diego-based Amira Pharmaceuticals.]

The benefits of partnerships between emerging biotech companies and major drug developers are numerous and well known. The smaller biotech company gets cash to support its innovative R&D, and the Big Pharma company gets hot new patented drug candidates to replace its aging blockbusters as they start to face competition from cheaper generics. However, obtaining these partnerships is becoming increasingly difficult for biotech companies in the current climate of increased economic pressures and mergers and acquisitions between major drug developers.

In recent months, major drug developers such as Genentech and Schering-Plough, who were previously partnering targets, are now going through the process of mergers (with Roche, and Merck, respectively). This means it is likely that there will be a continued gradual decrease in the number of potential partners looking to acquire co-ownership of new drugs going forward.

As big drugmakers continue to merge, the consolidated portfolios will also change. At first glance it would seem like the mergers and acquisitions would allow the combined company to expand its portfolio. However, these mergers often result in management trimming off ‘excess’ to increase focus on the most profitable areas. Therefore, not only is the number of companies decreasing, but the number of disease indications being pursued by these companies is also likely to continue to decrease.

Consequently, as small companies have ever decreasing choices, major drug developers have more options from which to choose when selecting a partner. It is no longer enough for a start-up biotech to be first-in-class. Instead, it is more important than ever for biotechs to see their own programs from the perspective of major drug developers and create fully enabled programs that are attractive to potential partners in multiple ways.

Identifying a Target

The discovery and the identification of game changing therapeutics have been central to the success of the likes of Genentech and Amgen. Others, such as Gilead Sciences, emerged as a leader in their field through the demonstration of being the best-in-class.

The right decisions at the earliest stages of target selection are instrumental in eventually attracting partners. One of the first questions to ask is, “If we have a program in the clinic in 3 or 4 years, will there be a partner across the table from us?” Or, simply ask, “Is this therapy going to make any difference to the treatment paradigm?”

Companies need to identify targets that will be appealing in the future, which may not necessarily be attractive now. This selection process should include …Next Page »

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Scientists have been trying for years to solve the mystery of why a few rare individuals get infected with HIV, yet somehow retain immune defenses so they never get sick. Today, researchers at a small Seattle biotech company, Theraclone Sciences, and collaborators at San Diego’s Scripps Research Institute say they have found a new vulnerability in the virus that could lead the way to new treatments or possibly a vaccine.

By studying rare blood samples from HIV-resistant people in the lab, scientists have found two weak spots on the virus, and were able to genetically engineer two new antibodies that broadly neutralize many variations of the virus circulating around the world, according to research being published this week in Science. Besides Theraclone and Scripps researcher Dennis Burton, this effort included collaborators from South San Francisco-based Monogram Biosciences (NASDAQ: MGRM) and the International AIDS Vaccine Initiative (IAVI) in New York.

This effort is still in its early days, and nobody knows yet for sure if these new antibodies will even work in lab animals. But this is the first time in more than a decade that scientists have discovered antibodies with broad neutralizing capability that can stand up to multiple strains of the wily virus in the lab. Plus, they were found in blood samples from donors in developing countries, where most of the new infections occur.

While HIV is largely considered a chronic disease in wealthy countries like the U.S. where there are 32 FDA-approved antiviral drugs, the discovery of neutralizing antibodies is potentially groundbreaking. The antibodies could be critical ingredients used to develop the first HIV vaccine, which would be most useful in poor countries. More than 30 million people around the world are thought to be living with HIV, and the disease is still thought to kill 2 million people a year.

“These new antibodies, which are more potent than other antibodies described to date while maintaining great breadth, attach to a novel, and potentially more accessible site on HIV to facilitate vaccine design,” said Burton, a professor of immunology and microbial science and scientific director of the IAVI Neutralizing Antibody Center at The Scripps Research Institute in La Jolla, CA, in a statement. Burton is also a member of the newly-formed Ragon Institute, a collaboration of Massachusetts General Hospital, MIT, and Harvard.

We first wrote about this HIV work in April based on an interview with Theraclone CEO David Fanning. I caught up with Fanning again by phone to talk about the business implications of getting such big recognition in one of the world’s top two scientific journals.

Theraclone CEO David Fanning

This publication—and all the global media attention it is bound to attract—is definitely going to attract the interest of prospective partners in Big Pharma and biotech, and funding agencies like the National Institutes of Health, that Theraclone needs to help pay the bills for its research program. When Fanning called me, it was 4 a.m. in Japan, where he has been meeting with potential partners. “I’m not here for vacation, you can put it that way,” he says.

“This really validates our technology in the eyes of people that we want to see start using it,” Fanning says. “Instead of us being a small private biotech that may or may not be doing something interesting, we’ve now made a mark very rapidly in one of the biggest challenges of all infectious disease.”

Still, the work is clearly just beginning. Theraclone, through ongoing financial support …Next Page »

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Billionaire investor Carl Icahn made a relatively softball argument a year ago about why shareholders should throw out the management slate for the Biogen Idec (NASDAQ: BIIB) board and vote for his nominees instead. It didn’t work. This time, he’s launching a blistering attack that accuses the company of botching its 2003 merger with Idec Pharmaceuticals, and for having “failed leadership” that leaves it poorly positioned for the future.

Icahn made his case today in a 52-page PowerPoint presentation he’s delivering to shareholders, filed with the Securities and Exchange Commission. It’s a key part of the strategy for swaying stockholders to vote in favor of his four nominees to the 13-member Biogen board, before the votes are tallied at the company’s annual shareholder meeting on June 3. The company needs an overhaul, Icahn argues, so badly that his directors would consider splitting it into two operations—one of which concentrates on neurology, the other on cancer.

For starters, Icahn argues that the November 2003 merger of Biogen and Idec was a failure. It never generated the kind of rich returns shareholders have gotten from peer companies like Genentech, Gilead Sciences, and Celgene. The Biogen executives never delivered the cost savings advertised at the time of the Idec merger; in fact, operating expenses have shot up to be more than $711 million beyond the company’s target at the time of the union, Icahn said. As this played out over the years, CEO James Mullen was “paid well, despite poor performance and failures across many dimensions,” Icahn said. Part of the root problem, Icahn said, is that “management turnover makes consistent strategy and execution difficult.”

Last year, Icahn rolled out a relatively vague four-point plan for Biogen based mainly on boosting spending on R&D, improving employee morale, and mending relationships with partners. The goal, for a time, was to sell the company to a larger drugmaker. Last year, Icahn didn’t attempt to deliver a knockout blow like today’s proxy filing.

Not surprisingly, Biogen Idec wasn’t thrilled to answer the latest round of tougher charges.

“This appears to be nothing more than an 11th hour tactic by Mr. Icahn to win votes,” said Biogen spokeswoman Naomi Aoki. “Since last year’s shareholder vote, he’s made no suggestions on how to better run the business. I find it curious that he only makes suggestions on the eve of the shareholder vote, even though he’s had a year to make suggestions.”

Alex Denner, a portfolio manager for Icahn who is one of his nominees for a board seat at the upcoming annual meeting, couldn’t be reached for comment tonight.

But Icahn’s team has obviously spent a lot of time and research digging into every aspect of Biogen Idec’s business, and it heaps criticism in every major department.

On the commercial product side, Icahn singled out shortcomings in marketing of interferon beta (Avonex) for multiple sclerosis. That drug has lost the lead in U.S. market share to Teva Pharmaceuticals’ glatiramer acetate (Copaxone), which now has 37 percent of the market, compared to Avonex’s 28 percent. The Biogen product has lost 14 percentage points of market share since April 2005, Icahn said. Another Biogen drug for severe psoriasis, alefacept (Amevive), “never lived up to management hype.” That product generated just $12 million in sales in 2006 instead of the $500 million projected three years earlier, Icahn said. Then there are the sales projections for natalizumab (Tysabri), its fastest-growing product, which were “overly aggressive” Icahn said.

On business development, the company has also “done very little” to defend or build up strength …Next Page »

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Genzyme is getting to the point where the law of large numbers start to look really daunting. The Cambridge, MA-based biotech company has been so successful for so long, it gets harder every year to sustain the breakneck sales and profit growth that investors in the company have come to expect.

So what to do? The company has assembled an R&D portfolio that includes its traditional strength in treating rare genetic diseases, and expands into the high-stakes, highly competitive markets of cardiovascular disease, cancer, and multiple sclerosis. The company’s executives, led by CEO Henri Termeer, offered glimpses yesterday into how this strategy is evolving at the annual investor day.

Before diving too deep into the details, Genzyme executive vice president Peter Wirth showed an intriguing slide on how far the company has come. Back near the peak of the high tech bubble in January 2000, Genzyme (NASDAQ: GENZ) ranked No. 14 among all biotech companies in stock market valuation, with a relatively puny $3.8 billion. Nine years later, Genzyme has more than quadrupled in value to about $16 billion, and now ranks behind only Amgen, Gilead Sciences, and Celgene by that measurement. Interestingly, Genzyme is one of only seven of the top 15 biotech companies from those heady days at the turn of the millennium that have had the staying power to remain independent. (The slide is in the Section 1 presentation if you’d like to see the full list for yourself.)

It’s an impressive record, but like I said, it’s not easy to boost revenues as much as 20 percent a year when you’re working from a baseline of $5.15 billion to $5.35 billion—which is Genzyme’s ballpark forecast for this year. To keep the pipeline moving, the company is going to carefully sock away a lot of the $5.6 billion in cash it expects to generate between now and the end of 2011. It plans to spend about one-third of this—$1.9 billion—to expand and maintain global infrastructure, another $1 billion on share buybacks to boost the stock price, and another $700 million for milestone payments to potential partners with promising drugs in early stages of development. Keeping the pipeline stocked with multiple drugs with decent sales potential, rather than relying on one or two megahits, is clearly a key to the plan.

“All products have life cycles, and you need multiple products, and multiple life cycles, to create a sustainable future for a company,” Wirth said.

So which ones are the innovative new medicines that Genzyme thinks will help patients, and the company’s bottom line? Here are highlights of what the company had to show investors:

—Genzyme is clearly pumped about its new oral pill for Gaucher’s disease (pronounced go-SHAYZ). The new drug, called GENZ-112638, was designed to be a more convenient way to maintain treatment of patients who rely on Cerezyme, an injectable drug that is Genzyme’s biggest seller. But it turns out that a Phase II trial showed it might be better than just a maintenance therapy. Patients on the oral drug …Next Page »

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One of the great riddles of modern medicine is why a few rare individuals who get infected with HIV retain a strong enough immune defense to naturally fight off the virus in all its nefarious strains. Some of these people are prostitutes in Africa, they never take an expensive cocktail of antiviral drugs, and they never need them because they never get sick.

What if scientists could pinpoint exactly what’s going on in their immune system that keeps the virus at bay, put it in a bottle, and market it as a treatment for the vast majority of people who aren’t so genetically gifted? That’s exactly what Seattle-based Theraclone Sciences (formerly known as Spaltudaq) is attempting to do at its lab on First Hill. This effort is still in its very early days, but CEO David Fanning told me about some intriguing progress in recent months, just a few steps away from our Xconomy Seattle office.

Theraclone’s effort has a lot stacked against it from the get-go. There are already 33 FDA-approved drugs for HIV, according to this list compiled by the agency. These various cocktails of antivirals have basically turned HIV in wealthy countries from a death sentence into a chronic disease to be managed. The drugs have made fortunes for companies like Gilead Sciences and GlaxoSmithKline. But one drawback is these pills need to be taken diligently every day. Patients, some of whom are indigent or drug users, sometimes struggle to stick with this disciplined medication schedule for life.In doing this, they run the risk of letting the virus develop resistance to these treatments. So Theraclone’s idea is to develop a potent genetically engineered antibody drug that could be given intravenously under a doctor’s supervision once a month, or maybe even less frequently.

No one has come close to doing this, partly for the same reason nobody has been able to develop a successful HIV vaccine. The virus, a wily character, has always been able to mutate into multiple strains that have dodged these kind of antibody-based attackers in past studies. No companies are pursuing a neutralizing antibody approach to HIV, according to the International AIDS Vaccine Initiative, or IAVI. But Theraclone, along with partners at IAVI, thinks it may have found a rare type of antibody in the blood of people innately immune to HIV with superb viral-killing ability. If they’re right, it could lay the groundwork for a promising new alternative form of therapy, and maybe even a vaccine far in the future. The implications of that are obvious—more than 30 million people around the world are estimated to be living with HIV, and it is still thought to kill 2 million people a year.

“We’d like to investigate two or three complementary antibodies to put sufficient pressure on the virus, so you don’t enable it to escape,” Fanning says. “That’s the key.”

Theraclone reported some intriguing data on its program a week ago at the HIV Immunobiology Meeting in Keystone, CO, sponsored by the Bill & Melinda Gates Foundation. Based on blood samples from these rare HIV neutralizer patients, obtained from IAVI, Theraclone scientists discovered two new antibodies that were able to neutralize a broad array of HIV strains in the lab.

Theraclone made these antibodies using standard genetic engineering techniques, and showed in a lab dish that they can kill HIV in a much more potent, or lower dose, than the other four antibodies scientists have identified over the past 15 years against HIV.

This sort of exploration for novel antibodies is right up Theraclone’s alley. The company’s scientists …Next Page »

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Biomedical research funded by Uncle Sam is hot, but publicly traded biotech companies that seek to develop those discoveries in the Northwest are cold. Here’s a recap of the week’s ups and downs in the local life sciences scene:

—President Obama is proposing a whopping $10 billion addition to the National Institutes of Health’s budget as part of his economic stimulus plan, and this has researchers in the Northwest scrambling to turn in their applications. The University of Washington, already the nation’s largest public research center with $1 billion a year in sponsored research, may capture as much as another $300 million in federal stimulus grants, says Linden Rhoads, vice provost of UW TechTransfer. Still, she says that’s no excuse for state lawmakers to start whacking away at the university’s state support.

—Yet even with basic biomedical research on the rise, many public biotech companies are struggling. Based on my review of the quarterly financial filings of the 12 public companies in the Northwest, six were looking pretty vulnerable with less than $20 million in available cash as they headed into 2009.

—Healionics, the Redmond, WA-based company that spun out of the University of Washington bioengineering lab of Buddy Ratner, took its first step this week toward becoming a commercial business. It shipped its first product, a biomaterial coating for shunts that are made to relieve pressure behind the eyes of dogs with glaucoma. If it works by helping allow fluid to pass more easily through the shunt, it could set an important precedent for using the biomaterial in human beings with glaucoma—a more lucrative potential market.

—Halosource, the Bothell, WA-based maker of technology to purify drinking water, won certification from the U.S. Environmental Protection Agency for its technique. This is no big deal in the U.S., where we take clean drinking water for granted, but it’s an important validation to partners Halosource is working with in India, China, Brazil and other parts of the world that are looking at its cheap, gravity-fed system for ridding water of viruses and bacteria. This technology is now providing clean water for 2 million people in India, double the number who were using it nine months ago.

—MDRNA (NASDAQ: MRNA), the Bothell, WA-based developer of RNA interference drug technology, received a bit of a lifeline this week when it received $7.25 million from Novartis in exchange for a non-exclusive worldwide license to its technology. But easy come, easy go. The company has to use $870,000 to write a severance check to its former CEO, Steven Quay.

—Nanostring Technologies CEO Perry Fell has resigned his post, and is being replaced …Next Page »

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Gilead Sciences, the Foster City, CA-based biotech company that has a research center in Seattle, said a scientific committee of the European Union adopted a negative opinion of its aztreonam lysine drug. The treatment, an inhalable antibiotic for cystic fibrosis developed by Seattle-based Corus Pharma, was turned down by the FDA last fall. EU regulators said Gilead needs more evidence about long-term benefits, repeated treatments in different age groups, and risk of bacterial resistance, said Thomas Russo, an analyst with Robert W. Baird.

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People with pulmonary fibrosis have a pretty raw deal. Scientists don’t really know what causes this lung-damaging disease, and there’s no really effective FDA-approved treatment. The disease makes it hard to breathe, and kills an estimated 40,000 people a year. That’s a bigger killer than some far better known culprits like prostate cancer.

A few drug companies have tried to fill this void with little to show for it yet. But San Diego-based Amira Pharmaceuticals says it is hot on the trail of creating a new small-molecule drug against a new target that it thinks could represent a big step forward. This program is in very early stages—too early to say anything about potential effectiveness—but I figured I’d listen because this company’s scientific team worked together at Merck’s former San Diego operation and played important roles in developing montelukast sodium (Singulair), the asthma drug that generates $4 billion a year in revenue.

Amira was started in 2005 by a trio of scientists—Peppi Prasit, Jilly Evans, and John Hutchinson—who worked together for years at Merck until that company closed its San Diego operation. I got the update on what’s new from CEO Bob Baltera, who came to Amira after a 17-year run at Amgen, while that company became the world’s largest biotech. He surprised me a bit, because instead of talking about the company’s two lead programs in development-including one in clinical trials for respiratory diseases that’s in a partnership with GlaxoSmithKline—Baltera mostly wanted to talk about a promising new protein target for pulmonary fibrosis characterized by Andrew Tager and colleagues at Massachusetts General Hospital. The Amira team is at work developing the best drug candidate it can to hit this new target.

“This is a grievous illness, and Amira is out at the forefront,” Baltera says.

There’s still a lot of work to do, maybe 12 to 18 months’ worth, before Amira will have a drug candidate that can enter clinical trials, Baltera says.

Tager’s research was described in January 2008 in Nature Medicine. Scientists found that mice who lack a certain protein called LPA1 were protected from fibrosis and death after being exposed to likely triggers of the disease.

Many of the first responders on Sept. 11, 2001 in New York are coming down with pulmonary fibrosis, so anything that’s remotely successful …Next Page »

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George Milstein is a survivor. Lots of peers in his line of work, biotech investment banking, have vanished from the scene with pink slips in hand. Yet after 15 years in the business, living through the mid-90s health reform bust, the genomics collapse of 2001, and now this mortgage-fueled catastrophe, Milstein is still doing what he has always done, flying in from San Francisco to scout emerging companies in the Northwest. Some of these people might need his merger-and-acquisition service, or maybe, someday when the clouds lift, even help with doing an IPO, he figures.

Milstein, head of healthcare M&A for Wedbush Pacific Growth Life Sciences, will be in Seattle tomorrow as one of the featured speakers coming to Invest Northwest, the area’s biggest annual life sciences investing conference of the year. I spoke to Milstein by phone on Friday to get a sense for how his firm is doing, and his outlook for the sector.

Pacific Growth Equities has longstanding ties to the Seattle area, namely through underwriting a couple of significant life sciences IPOs of the past—ZymoGenetics (NASDAQ: ZGEN) in 2002, and Trubion Pharmaceuticals (NASDAQ: TRBN) in 2006. Those deals have certainly inflicted pain (Zymo’s IPO price was $12, and Trubion’s was $13, while the stocks closed Friday at $3.83 and $1.60, respectively.) Even so, Pacific Growth had the relative good fortune —or foresight—of not getting wound up in mortgage-backed securities that have killed off bigger firms like Lehman Brothers.

Still, the investment bank is not completely independent anymore. In January, Pacific Growth was acquired by Los Angeles-based Wedbush Morgan Securities. To hear Milstein tell it, this deal has kept the firm “well-capitalized,” with no exposure to toxic mortgage-backed securities, and Wedbush said at the time it planned to keep all of the firm’s 75 bankers, analysts, traders, and staff. Milstein can’t support this activity from the once-lucrative fees generated from IPOs, so he’s positioning the firm to make a living on advising companies about mergers, acquisitions, divestitures, and other restructuring deals they will likely need to adopt to live through this recession. Plenty of companies will need to make moves—an estimated one-third of public biotechs are operating with less than six months of cash in the bank.

“We’re over the shock of it all,” Milstein says. “The long-term prospects for this industry are still there. We’re not making cars nobody wants to buy, we’re making drugs that improve peoples’ quality of life. I’m confident things will improve, it’s only a question of timing.”

He used two headline-grabbing deals from the past week to buttress his point. Swiss drug giant Roche agreed to buy the remaining minority stake in South San Francisco-based Genentech it didn’t already own for $95 a share, or about $46.8 billion. Down the road in Foster City, CA, Gilead Sciences agreed to acquire CV Therapeutics, a small maker of a drug for chest pain, for $1.4 billion in cash. The muscle being shown by Gilead, the world’s largest maker of HIV drugs, is remarkable, Milstein says. “Gilead was a small-to-mid-sized company when I was getting started,” he says. He sounded optimistic there will be more deals like this for him and his peers in biotech investment banking to stay busy.

There has been plenty of armchair quarterbacking …Next Page »

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(Updated) Angel investors and biotech companies don’t usually mix. Most biotech startups begin with university research, and embark on a decade-long quest of product development, all while inhaling several hundred million dollars of capital, and carrying the burden of a 90 percent failure rate. This is where deep-pocketed, risk-seeking venture capitalists and public equity investors tread, not angels who are long on experience and short on dough.

So I was curious why, in the teeth of a recession, and as many biotech companies are facing extinction, the Seattle-based Zino Society chose to organize its first Life Sciences Investment Forum. This event, held yesterday at the Pan Pacific Hotel in Seattle, drew 12 determined startup companies from around the country, developing drugs, diagnostics, or medical devices. They all lined up to make their best 7-minute pitch to attract angel investment bucks.

The event drew some of the pioneers of Seattle biotech, including Bruce Montgomery of Gilead Sciences, Bruce Carter, the former CEO of ZymoGenetics, and H. Stewart Parker, formerly the CEO of Targeted Genetics. They were there to listen and to offer advice to the entrepreneurs, and maybe more than anything, provide a pep talk for people willing to try to do this innovative work. In his keynote, Montgomery outlined four ways he sees businesses have historically made money in the past. The first was centralization of small businesses like coffee (Starbucks); second was becoming the low-cost provider (Wal-Mart); third was leveraging or securitizing assets (Wall Street); and fourth is through innovation. Guess which one he believes has a future?

“Innovation is the only way to have a true win-win for our society and make us competitive with foreign competitors,” Montgomery said. “Innovation still actually has a chance.”

Zino Society did its best to make this a bit fun, and a bit competitive, with awards. The award for best presenter, as voted by the audience of about 100 Seattle-area angel investors, was given to Douglas Turnquist of Salt Lake City-based ThermImage. The prize for best investment opportunity went to Malvern, PA-based Carbon Nanoprobes. One big winner will be selected in coming weeks by the Zino Society for a $50,000 investment, and the right to make a presentation next month on a bigger stage at Invest Northwest, the regional life sciences investing conference.

So what were some of these innovative companies about? Here were a few of the highlights:

—Carbon Nanoprobes CEO Brian Ruby had one of the best opening gambits in his presentation. “We take pictures of tiny things. We’re like the paparazzi of atoms and molecules,” he said. Essentially, his company’s nanotech materials are used to provide high-resolution images for customers who need to look at small things in the semiconductor, basic research, and biomedical research businesses. It has raised almost $1 million from about 30 investors in Seattle, and is moving into manufacturing its disposable product before the end of March. He said the company has sales leads from big-name customers like IBM, Intel, and Harvard University, among others, although I didn’t hear him say he had booked sales or delivered any just yet. “People are lining up to buy the product before it’s off production line,” Ruby said.

—Oasis Diagnostics. This Vancouver, WA-based company is attempting to develop precise diagnostic tests that extract tiny bits of DNA from saliva, instead of blood. CEO Paul Slowey showed a picture of a young girl grimacing after getting stuck with a needle, stacked next to a photo of her smiling while sticking what looks like a power toothbrush inside her cheek. “A picture paints a thousand words,” Slowey says.

OK, but he had more than just fluff in his quick talk. The saliva diagnostics technology has improved enough in recent years that it can detect minute amounts of a foreign invader, like HIV virus, which has 1,400 times less concentration in saliva than in blood. The company is developing tests for tuberculosis, anthrax immunity, diabetes, and breast cancer. Slowey has experience at two other saliva diagnostics companies, Saliva Diagnostic Systems and OraSure Technologies.

—Calcionics. This Seattle-based company, which spun out of technology developed at the University of Washington, is aiming to develop a drug to prevent and treat cardiovascular disease, particularly hardening of the arteries. CEO Chip Jacob is working with Cecilia Giachelli of the UW, who discovered osteopontin, a naturally occurring protein that breaks down calcified tissue that makes arteries harden. “It’s the body’s natural response to hardening of arteries, we augment it,” Jacob says. This drug has a long way to go through animal and human clinical trials, but Calcionics has no intention of commercializing it alone.

It plans to raise $35 million in three investment rounds over the next five years, and then cash out through getting acquired by a larger drug company for around $350 million. It would be tempting to scoff at this idea in the downturn, but Jacob pointed out it’s been done, and not long ago. Santa Clara, CA-based Ilypsa, which developed a phosphate binding drug for kidney-dialysis patients, raised $45 million before it was sold to Amgen for $420 million in 2007.

What Jacob didn’t mention is that when Amgen took the drug into further clinical trials, it failed. But that’s life in biotech—I’m pretty sure the people at Ilypsa were pretty happy with the returns, and willing to do it again, regardless of whether the drug ever made it on the market.

(Update: The original version of this story left out one of the finalists for the $50,000 investment prize, Inson Medical Systems. The finalists were Oasis Diagnostics, Calcionics, and Inson. Here’s a glimpse of what Inson had to say.)

—Inson Medical Systems, a Bellevue, WA-based is developing polymer-based materials from the lab of UW bioengineering professor Buddy Ratner. As CEO Tracy Klein explained, these materials are supposed to allow a constant dose of a drug over time. The first product is being developed as an eye implant to treat cataracts in animals.

Inson is going after the veterinary market before humans, because it’s easier and faster, Klein says.  But that doesn’t mean Inson has written off the human market. About 14.6 million people worldwide have cataract surgeries each year, and Inson says its technique could help many of them avoid infection, inflammation, and secondary cataracts that can crop up after surgery. If it can capture any significant piece of that market in the next few years, I’m sure this is a story we’ll hear more about.

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Vertex Pharmaceuticals has been around the block with biotech hedge funds. These are the people who aim to get rich trading volatile stocks second-to-second, and make big bets, long or short, on whether an experimental drug will work. Now that Vertex has passed some of the riskiest stages of drug development, the company figured it was time for steady, buy-and-hold investors to support the next phase, as it morphs into a commercial player.

That was one of the insights that I gathered yesterday in a conversation with Vertex chief financial officer Ian Smith. He was in a pretty good mood—as you might be too, if you’d just helped your company raise $320 million in a secondary stock offering. The financing is important because it gives Vertex (NASDAQ: VRTX) enough cash to operate until it introduces its lead drug to the market and starts generating positive cash flow, according to analyst Thomas Russo of Robert W. Baird & Co. Especially in the midst of a recession, that’s good news for Vertex’s 1,300 employees in Cambridge, MA, and 200 in San Diego.

But beyond the stability that comes with the dollars, Smith was just as enthused about being able to recruit the type of long-term shareholder he wants backing Vertex for the next five years. Vertex was able to attract these blue-bloods because of growing interest from investors in a pair of drugs that aim to transform the standard of care for patients with hepatitis C and cystic fibrosis. Since these drugs have continued to make important progress, and telaprevir, the hepatitis drug, is thought to be worth at least $2 billion a year in U.S. sales by 2013, Vertex saw an opportunity to invite in new investors who want to hold onto shares throughout that trajectory, not just between now and next Tuesday. To get some insight into the company’s thinking, look at its stock chart from 2007, when shares fell 37 percent, even though the company continued to make progress.

“We as a company have been trying to align our shareholder base with the progress of the company,” Smith says. “These are people who invest for the long haul.”

Smith wouldn’t give all the names yet of the new crew of investors, but this deal went to a small number of investors—three “anchor” funds that took the biggest stakes, two other “high-quality” funds, and a few others, Smith said. What they all have in common is that they like to invest in late-stage biotech companies that have cleared major risky steps, and need to get to the next level. These funds typically backed some of the industry’s bellwethers–Genentech, Gilead Sciences, Biogen Idec, and Celgene—when they reached this stage, Smith says. One of them is San Francisco-based Capital World Investors, although he said he couldn’t yet name the others.

Smith, as well as other Vertex leaders, have been cultivating relationships …Next Page »

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People in the life sciences business throw around the term “Big Biotech” as a way to distinguish the industry’s elites from the money-losing masses in the product-development stage. The “Big Biotechs” are the industry’s select few profitable companies with multi-billion dollar stock valuations, like Amgen, Genentech, Gilead Sciences, Celgene, and a duo from Cambridge, MA—Genzyme and Biogen Idec. Now another Cambridge neighbor, Alkermes, says it is pounding on the door of that club.

I gathered some of this insight into Alkermes’ thinking last month at an investor meeting in San Francisco, where I talked with chairman Richard Pops and chief financial officer Jim Frates. They were somewhat unnerved by the financial crisis, but said they took some comfort in being able to sell investors on that rarest of entities—a profitable biotech company with growth potential.

Alkermes (ALK-ur-meez) was founded in 1987, and like the usual biotech company, it lost money for its first 20 years. Alkermes’ calling card all those years was its expertise in making existing drugs more stable and longer-lasting in the bloodstream, allowing for less frequent dosing. It has sought help from partners along the way. That softened the R&D bills, lowered the company’s risk, and also limited its ability to reap the rewards from its work. But now that one of its partnered products with Johnson & Johnson, a long-acting version of the drug risperidone, marketed as Risperdal Consta for schizophrenia, has become a $1.3 billion-a-year hit, it has turned Alkermes into a consistently profitable company. That’s enabled the company to build all the capability it needs to develop drugs from start to finish, and market them on its own, just like the rest of the class in “Big Biotech.”

“Now we actually have an economic business,” Pops says. “As a biotech company, you spend the first decade or two as a science project. It’s not really a business. It’s funded by investors who are willing to take risk. Now we’re in a different place. The whole nature of the risk profile changes over time.”

The strategy, Pops says, was to build the company in a step-by-step manner. Alkermes never bet the company on a single drug, a strategy Pops calls “the classic biotech gamble.” Instead, Alkermes chose to hone its proprietary technology, he says. Despite a big setback last year when Eli Lilly pulled the plug on an inhaled insulin partnership, Alkermes steadily developed enough of a royalty stream off partners’ products to switch over into profitability from 2006 through 2008. It did this while building up enough in-house talent (610 employees as of last March) and proprietary resources (it has its own drug factory) to turn the company into an independent operation, rather than an R&D shop for big drugmakers. The company made an important strategic move in that direction in December by adding a 70-person sales and marketing staff, and acquiring 100 percent of the commercial rights from Frazer, PA-based Cephalon to naltrexone (Vivitrol) for alcohol dependence.

“We know how to develop drugs that get approved by FDA, we have a factory, we have proprietary technology, it’s patented, we have people who can do it. Why do it all for pharmaceutical companies anymore? Now we can do it ourselves,” Pops says.

Still, Alkermes’ business depends heavily on riding a wave of royalties from its big partner, Johnson & Johnson. The Alkermes technology was particularly useful for schizophrenia, as many schizophrenia patients struggle to take their pills every day, causing relapses. The Alkermes technology transformed a daily pill into an every-other-week injection. It also enables the medication to remain stable in the blood for a full two weeks, allowing patients to avoid the peaks and valleys of concentration in the bloodstream that can lead to spotty effectiveness, Pops says. (This week, the FDA delayed an application to expand the drug’s use to bipolar disorder.)

Since about 1.5 million people worldwide get diagnosed with schizophrenia each year, and patients depend on their medication, even in a downturn, this is not a bad pharmaceutical business to be in. …Next Page »

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Techies, with their cool iPhone apps and Internet software, get all the attention. So a couple of Seattle biotechies squawked at me a couple weeks ago when the Washington Technology Industry Association unveiled an academic study that traced the origins of the “Puget Sound Tech Universe” over the past three decades.

The WTIA study examined the career paths of company founders who started at a place like Microsoft or the University of Washington before they gained critical experience and inspiration to go off and create something new. The analysis purposely left out biotechs—after all, they had to draw the line somewhere at 711 companies. Nothing quite like this visual poster has ever been done for the Puget Sound’s life sciences community, but Sanjaya Joshi reminded me of an analysis of Seattle’s biotech roots that he drew up in a 2004 paper, and updated in 2007.

Joshi, president of Kirkland, WA-based Userspace, runs an IT services company for biotech organizations that have extreme data needs, like the Institute for Systems Biology in Seattle. His paper identified three critical anchors of biotech’s early days in the Puget Sound region, which have served as critical training grounds for biotech entrepreneurs: the Fred Hutchinson Cancer Research Center, the University of Washington, and Seattle-based Immunex. It shows how the “Hutch” served as a base of operations for immunologists Christopher Henney and Steve Gillis, who went on to co-found Immunex in 1981. Immunex veterans went on to start notable operations like Icos, Corixa, Dendreon, Targeted Genetics, Frazier Healthcare Ventures, and Trubion Pharmaceuticals.

Joshi’s boxy flowchart that shows these and many other connections looks kind of confusing to my eye, especially compared with the slick astronomy-inspired depiction of Washington’s tech companies.

Even though his chart can appear disorganized at times, Joshi makes some interesting observations based on his exercise in connecting the dots over a 25-year period. He did interviews with many prominent players in the region, including Bruce Carter, former CEO of ZymoGenetics, Tom Clement of Pathway Medical Technologies, Chris Elias of PATH, Bruce Montgomery of Gilead Sciences, and Clay Siegall of Seattle Genetics.

Based on those interviews and other research, Joshi concludes, Seattle has become a second-tier life sciences hub behind the leaders in Boston and the San Francisco Bay Area. He dubbed it the “BioForest.” The key ingredients that went into the mix for Seattle: excellent academic research centers, a critical mass of talented people and connections formed over the years, a mix of startups and mid-sized companies in different stages of development, and an attractive outdoor lifestyle that helps with recruiting.

This blend has led the region to build up strengths in immunology, cancer biology, ultrasound, and cardiac management technologies like defibrillators, Joshi says. He notes that his analysis isn’t fully up to date and, for example, doesn’t account for the loss of Merck’s Rosetta Inpharmatics division—which spun out of the founding efforts of Stephen Friend and Lee Hartwell at the Hutch, as well as Leroy Hood of the Institute for Systems Biology. Of course, the loss of Icos a couple years ago led to the creation of Seattle-based Calistoga Pharmaceuticals, and an intriguing TB Drug Discovery Initiative inside the Infectious Disease Research Institute. Sounds like it may be time for somebody to do an update on what important new projects will emerge from the scattering of the Rosetta talent pool.

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The recession will surely kill off some biotech companies that lack hard cash and the hard data that shows their experimental drugs can improve patients’ lives. But after I heard Seattle Genetics CEO Clay Siegall tell the story of how his company raised $55.8 million last week, I had to conclude it’s still possible to raise money in biotech as long as you have a drug that represents a big advance against a deadly disease.

Seattle Genetics (NASDAQ: SGEN) pulled off this small miracle on Jan. 27. How unusual is it for a money-losing development-stage biotech company to sell 5.74 million shares to raise $55.8 million? It was the first underwritten stock offering in the biotech industry since Aug. 7, according to data from Deloitte Recap. (Although Portland, OR-based AVI Biopharma was able to raise $16.5 million a couple days later as well.)

Siegall gave me this account of how the improbable deal went down. Seattle Genetics was in a strong position after having released stellar clinical trial results for SGN-35, the “empowered antibody” it is developing against Hodgkin’s disease and related lymphomas. It had just agreed with the FDA on the final-stage clinical trial design, which provides a clear path so Seattle Genetics bring it to the market in 2012. But the company was bound to burn significant cash over the next three years, and it was sitting on a thinning stockpile of only about 20 to 22 months of cash reserves, Siegall said.

Siegall, who had just gone through a marathon of 50 meetings during the JP Morgan Healthcare Conference in San Francisco, sensed there was demand for his company’s shares. He also felt a secondary offering wouldn’t flood the market and drive down the price. So without even bothering to go on a traditional road show, UBS Investment Bank “made a very reasonable offer” Siegall says, to buy all the shares in a single block purchase, at 4:01 pm Eastern time on Jan. 27. The deal was struck. After about two hours of putting UBS traders to work on the phones, they made a slight profit for their firm by selling all of the shares at a 7 percent discount to the $10.46 market closing price that day. The press release announcing the deal crossed the wire the next morning.

“I was thrilled,” Siegall says.

Seattle Genetics could still pocket another $11.5 million by May if shareholders approve a related transaction that allows Baker Brothers Life Sciences, one of the company’s largest shareholders, to buy another 1.18 million shares at the same price.

The company plans to discuss its fourth-quarter financial report after markets close today, and it will provide guidance to investors on how this financing changes its outlook, Siegall says. But essentially, it adds another eight months of cash to its bank account, assuming shareholders approve the Baker Brothers deal.

But what’s even more interesting is what has happened to Seattle Genetics’ stock since this deal was done. Since secondary offerings increase the supply of available shares, they usually dilute the value of existing ones, and depress the price. Instead, Seattle Genetics stock has actually climbed …Next Page »

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Vertex Pharmaceuticals isn’t going to move its labs and offices anywhere, at least in the near future. The Cambridge, MA-based biotech company has extended leases at two main buildings in Cambridge until 2015.

The leases will keep Vertex’s headquarters at 130 Waverly Street, and in other space at 200 Sidney Street through 2015, according to a statement today from Vertex’s landlord, Biomed Realty Trust (NYSE: BMR).

Vertex (NASDAQ: VRTX) has grown to about 1,300 employees spread among nine buildings in Cambridge, plus 200 more employees in San Diego and a small group in the United Kingdom, says company spokesman Zach Barber. About 80 percent of its square footage is now leased through 2015 or beyond, he says.

Even though the leases are in place for six years, Vertex will keep its eyes out for real estate opportunities. “Our long-term goal is to consolidate all of our employees into one campus,” Barber says. That applies to the workers spread among nine buildings in Cambridge, not the employees in San Diego or the U.K., he added.

If Vertex’s ongoing clinical trials pan out, it will get quite a bit bigger before the leases expire. Vertex doesn’t have any major moneymaking product on the market yet, but it is in the final stage of development with a hepatitis C drug, telaprevir, that could generate $2.6 billion in U.S. sales in 2013, says Rachel McMinn, an analyst with Cowen & Co.

The company clearly has a bold vision. This week at the JP Morgan Healthcare Conference in San Francisco, Vertex CEO Josh Boger’s presentation featured slides that likened his company’s ongoing efforts to the trailblazing work by Genentech in the 1970s that led to the first genetically engineered medicines, and work by Gilead Sciences in the early 1990s that created HIV treatments that made it possible for patients to take one or two pills a day, instead of 15. Boger’s slides were even accompanied by some of the disco and grunge hits that were popular when those advances were being made—the Bee Gees “Stayin’ Alive” from the ’70s and Nirvana’s “Smells Like Teen Spirit” from the ’90s. No word yet on the anthem that will symbolize Vertex’s hopes for growth in the decade to come.

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Anadys Pharmaceuticals is skyrocketing today. Shares in the San Diego-based biotech shot up more than 80 percent after it reported its experimental hepatitis C drug was able to wipe out more than 99 percent of the virus from the blood in the first 72 hours. As we described today in an interview with CEO Steve Worland, this finding is only from the first eight patients, but it beats others in its class at this early phase of testing.

Anadys (NASDAQ: ANDS) climbed 84 percent to $3.49 at Noon Eastern time today after it released preliminary results. One of the analysts who covers the company, Reni Benjamin of Rodman & Renshaw in New York, told clients in a note the data was “impressive.” He reminded investors the Anadys drug produced its effects at the lowest of three doses it is testing in this Phase I clinical trial.

Results from those groups of patients could drive the stock further this year, and the data could entice big companies like Gilead Sciences or Roche to form a partnership with Anadys to get a piece of the drug, Benjamin says. “While the stock could be volatile in the coming months, based on the results obtained to date, we continue to believe that Anadys Pharmaceuticals represents an overlooked and undervalued company for the long-term, risk oriented investor,” Benjamin wrote.

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An early peek at data from Anadys Pharmaceuticals (NASDAQ: ANDS) suggests the company may have a promising new drug in the works for hepatitis C. The San Diego-based biotech is announcing results today from the first eight patients with the chronic liver infection, which shows its drug has more viral killing pop in the first three days of treatment than was seen in other drugs studied in its class, with minimal side effects.

The company found that its experimental medicine was able to wipe out 99 percent of the virus from the blood (known as a 2.5 logarithmic reduction) within 72 hours at the lowest dose tested in a Phase I clinical trial, says CEO Steve Worland. This finding was in the first group of patients who took a 200 milligram, twice-daily dose of ANA598. It is just the first slice of data available, and the trial is continuing to enroll patients at two higher doses, Worland says. The company hopes to present full data at the European Association for the Study of the Liver meeting in Copenhagen, Denmark in April.

Today’s news will at least give Worland a fresh talking point next week when he meets with a bunch of bummed-out investors at the JP Morgan Healthcare Conference in San Francisco. Worland will try to cheer them up by arguing his company’s drug is looking better at this early stage of development than others in its class from Gilead Sciences (NASDAQ: GILD) and Pfizer (NYSE: PFE). Drugs in this class—non-nucleoside polymerase inhibitors—aim to be fast followers in the race to improve treatment for patients with hepatitis C, behind a group of protease inhibitor meds.

Most researchers’ attention is now focused on a different class, protease inhibitors like ones from Vertex Pharmaceuticals and Schering-Plough, as well as nucleoside polymerase inhibitors like ones from Pharmasset and Roche. Since the hepatitis C virus is a chronic infection, like HIV, many scientists believe a cocktail of therapies with different viral killing mechanisms is the best way to keep it in check.

“This way you can attack the virus from multiple angles,” Worland says.

Anadys (Uh-nad-iss) showed its drug appeared safe in 48 healthy volunteers, at a variety of doses back in November. But of course, it still has a long way to go. The company’s plan is to move later this year to a mid-stage clinical trial, which looks at its drug in combination with pegylated interferon alpha and ribavirin, the standard anti-viral course. It’s possible Anadys could enroll as many as 100 patients in that trial, and design it to analyze effectiveness for 24 or 48 weeks, although the protocol hasn’t been finalized, Worland says. The company is also doing long-term toxicity studies in animals, and working on scaling up manufacturing, he says.

The FDA typically requires companies to first show an ability to quickly wipe out the virus in the first three days, like the study being announced today, before companies can move on to more rigorous long-term follow-up studies, Worland says. Anadys plans to hold a conference call with analysts today at 8:30 am Eastern time to discuss the findings and answer questions about what comes next.

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