Vertex Pharmaceuticals, the Cambridge, MA-based developer of treatments for hepatitis C, said today that investors who hold about $109 million worth of debt securities have converted those holdings into equity stakes. The deal doesn’t leave Vertex debt free, because the company still has to pay back $35 million in principal, at a 4.75 percent interest rate, in 2013. The debtholders who are accepting stock will now get 4.8 million shares of Vertex (NASDAQ: VRTX). The company’s shares have more than doubled in value to $40.69 at today’s close, compared with the 52-week low of $19.87.

Comments | Reprints | Share:        

Alder Biopharmaceuticals has just struck one of the biggest biotech partnerships of the year, both in the Seattle area and nationally. The Bothell, WA-based developer of faster and cheaper technology for making antibody drugs has formed a collaboration with Bristol-Myers Squibb (NYSE: BMY), focused on the development of Alder’s experimental rheumatoid arthritis drug, that could be worth more than $1 billion.

Under the deal, Alder will grant a worldwide exclusive license to Bristol to develop ALD518 for all potential uses except cancer; Bristol will get an option to market the drug as a treatment for cancer outside the U.S. In return, Alder will get $85 million in upfront cash, as much as $764 million in payments for reaching development and regulatory milestones, sales-related milestone payments that could exceed $200 million, and an undisclosed royalty rate on product sales. Alder may also require Bristol to make a $20 million equity investment to support an initial public offering, if it chooses to go that route.

Alder’s big idea is to manufacture antibody drugs in yeast instead of in traditional mammalian cells. This enables the company to make “fast-follower” drugs that are faster and cheaper to produce, and that also may have improved properties like less frequent dosing, Alder CEO Randy Schatzman has told Xconomy. In the case of ALD518, Alder has developed an antibody to follow Roche’s tocilizumab (Actemra). The Roche drug, the first in its class, is an antibody designed to block the receptor of an inflammatory protein called IL-6 that hammers the joints of rheumatoid arthritis patients. This approach could offer doctors an alternative to the $10 billion-a-year class of drugs that block a different protein called TNF, like Amgen’s etanercept (Enbrel) and Abbott Laboratories’ adalimumab (Humira). Analysts have predicted sales of the Roche drug could capture a tidy portion of this huge market, with sales of $2 billion a year.

Alder has designed its antibody to block the IL-6 protein target. The Alder drug, ALD518, is designed to last longer in the bloodstream, so it can be given less frequently, possibly as little as three or four times a year instead of once a month, Schatzman has said. It is much more potent, so it can be given in one-tenth the dose, which is one reason it should be much cheaper to manufacture. The drug is also engineered to avoid dangerous immune system reactions that other antibodies can sometimes provoke.

“With its novel mechanism of action, ALD518 has the potential to offer an exciting new option for patients with rheumatoid arthritis,” said Brian Daniels, Bristol’s senior vice president, global development and medical affairs in a statement. “We are pleased to have the opportunity to develop this novel monoclonal antibody.”

Alder has completed a Phase II trial with about 120 patients who got ALD518 for rheumatoid arthritis, although it hasn’t made the results of that study public. But the company was actively deal-hunting at the American College of Rheumatology conference in Philadelphia last month.

The deal with Bristol is that company’s second big partnership with a Seattle biotech company this year, after an alliance with ZymoGenetics that it formed in January. That collaboration could also be worth more than $1 billion over time, to co-develop and market a new interferon drug with fewer side effects for hepatitis C patients. It’s also the second partnership for Alder this year, after it agreed in June to develop new antibodies for Schering-Plough, which wants to test them for a neurological disease.

Alder was founded in 2004 after Celltech R&D closed its doors in Washington and a core scientific group stuck together to start something new. It is now one of the better-financed development-stage biotechs in Seattle. It pulled in a $40 million Series C round in January 2008 from Delphi Ventures, TPG Biotech, Sevin Rosen Funds, Ventures West, HIG Ventures, and WRF Capital.

Comments (1) | Reprints | Share:        

TEDMED had Martha, Goldie, and other celebrity speakers, but San Diego-based Amylin Pharmaceuticals broke this week’s big news when it signed up a big Japanese partner to develop its line of obesity drugs. It’s all part of your regular dose of San Diego biotech news, and it’s ready now:

—Amylin Pharmaceuticals, the San Diego-based diabetes drug specialist, announced that it has formed a partnership with Japan’s Takeda Pharmaceuticals, which agreed to carry most of the development costs for Amylin’s weight-loss drugs. In return, Takeda gets a worldwide exclusive license to eventually commercialize Amylin’s experimental obesity drugs, including the combination of pramlintide and metreleptin, and davalintide.

—After a five-year hiatus, TEDMED founder Richard Saul Wurman, and president, Marc Hodosh (who also is an Xconomist), brought the conference on medical technology, entertainment and design to San Diego’s Hotel del Coronado. TEDMED announced during the conference, which included presentations by Boston Scientific co-founder (and Xconomist) John Abele, Martha Stewart, and Goldie Hawn, that the conference will return to the same location next October.

—I only had time to attend a fraction of the presentations at TEDMED last week. One of my favorites talks, though, was delivered by Bill Davenhall, who leads the health and human services marketing team at ESRI, the Redlands, CA, giant in geographic information systems. Davenhall talked about the importance of including patients’ “place histories” as part of their medical records and raised an interesting question: Will the electronic health record systems being created today have the capability to add data in new categories—such as “geo-medicine”–that aren’t typically included in today’s patient records?

—New York-based Xenomics filed a lawsuit against San Diego-based Sequenom that alleges Sequenom misrepresented the progress in its development of a prenatal test for Downs syndrome. Xenomix says it would not have licensed its patents to Sequenom had it known the truth.

—Denise profiled San Diego-based Sirigen, an early stage medical diagnostic company that is developing technology that uses light-emitting polymers to detect bits of DNA. Sirigen founder Brent Gaylord developed the technology at UC Santa Barbara, extending the significance of UCSB physicist and Nobel laureate Alan Heeger’s discovery of conductive polymers.

—The FDA told San Diego-based Amylin Pharmaceuticals (NASDAQ: AMLN) and its partner Eli Lilly the companies can now market exenatide (Byetta) as a frontline, standalone therapy for diabetes. The drug was previously approved for use with other drugs, or as a fallback option when other tretments failed.

—Vertex (NASDAQ: VRTX), the Cambridge, MA, biotech with operations in San Diego, said the latest trial of its telaprevir treatment for hepatitis C was able to attain the clinical definition of a cure in more than 80 percent of patients who got the drug. The finding is part of the mounting evidence Vertex is gathering on its quest to develop the first-of-its-kind protease inhibitor for the chronic liver disease.

Comments | Reprints | Share:        

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 »

Comments | Reprints | Share:        

Vertex Pharmaceuticals wants to make sure doctors walk away from this weekend’s big liver disease research meeting with two main ideas stuck in their heads. No. 1—The company’s experimental drug is showing that it can cure even the toughest-to-treat patients with hepatitis C. No. 2—it may work just as well in a twice-daily dose as it does when given three times a day.

The latest batch of clinical trial data from Cambridge, MA-based Vertex (NASDAQ: VRTX) shows that its telaprevir treatment was able to attain the clinical definition of a cure in more than 80 percent of patients who got the drug. That’s regardless of whether they got the drug three-times-a-day, or a more convenient twice-daily dose. Side effects were similar for each dosage. Results from the mid-stage 161-patient study, known as C208, are being presented this weekend at the American Association for the Study of Liver Diseases, being held in Boston.

This latest finding is part of the mounting evidence Vertex is gathering on its quest to develop the first-of-its-kind protease inhibitor for hepatitis C, a chronic liver disease. The drug is currently being tested as an oral pill that patients take in combination with the standard treatments—pegylated interferon alpha and ribavirin. Vertex is hoping to more than double the cure rate for patients who are new to treatment, and make it so most patients only need to take the standard treatments for 24 weeks instead of 48, which is important because those drugs cause flu-like symptoms that make most patients feel miserable. The new drug is expected to trigger an outpouring of patients seeking treatment if teleprevir reaches the market on schedule in 2011, and it could generate $2.3 billion in U.S. sales by 2013, according to an analysis by investment firm Cowen & Company.

While Vertex is considered to be at the front of the pack when it comes to treating hepatitis C, there is a growing crowd of competitors following fast behind with an estimated 40 different drugs in clinical trials. That includes drug candidates from deep-pocketed rivals like Schering-Plough and Roche. Vertex has sought to fend off the threats to future market share a couple of ways. One is by showing that its drug can eradicate the virus—and cause a clinical cure—for the majority of the toughest patient population—those who didn’t respond at all to a prior round of standard treatment. That result was shown earlier this week in a small study known as ‘107, and Vertex is attempting to prove that point on a larger scale in the ongoing trial of 650 patients called Realize.

While it’s important to be seen as the most potent drug on the block, it’s also vital to at least stay even with the other guys when it comes to convenience. Vertex’s original mid-stage trials looked at three doses per day of 750 milligram telaprevir pills. That means patients who work during the day need to take their pills with them, and take them after a snack or lunch, says Vertex chief medical officer Bob Kauffman. By going to twice-a-day, with 1,125 milligrams—the same total amount of telaprevir in a given 24-hour cycle—researchers hoped they could achieve the same antiviral activity without seeing extreme peaks or valleys in blood concentration that might lead to increased side effects, or weak potency that could allow the virus to bounce back over time, Kauffman says.

“We think this is a big deal,” Kauffman says. “For people who work, this means you can just take the drug in the morning before you go to work, and take another one when you come home in the evening. It is more patient-friendly.”

Side effects were the big concern, and the results from the C208 study suggest they are “similar” for the twice-daily and thrice-daily regimens, Kauffman says. Eight of the 161 patients dropped out of the study because of serious side effects, mostly because of rash (4 patients), and anemia (3 patients), Vertex said. It didn’t say how many of the serious side effects were in the twice-daily dosing group.

All of Vertex’s three pivotal studies—known as Advance, Realize, and Illuminate—are testing the three-times-a-day dosing regimen, so that’s where the bulk of the evidence is going to be in the FDA approved prescribing information, if telaprevir is cleared for sale. The company is talking with regulators about whether this result from C208 might be sufficient to include language in the label that says twice-a-day is OK, Kauffman says.

Comments | Reprints | Share:        

Shares of Cambridge, MA-based Idenix Pharmaceuticals plummeted in early trading this morning after Novartis declined an option to further develop an Idenix drug for the liver disease hepatitis C.

Idenix (NASDAQ: IDIX) dropped 14 percent to $2.15 shortly after the opening bell this morning after the announcement, and the stock was downgraded by the investment firm Caris & Co. The stock has lost about two-thirds of its value now since the beginning of the year, when it started at $5.79.

Idenix will now retain full worldwide commercial rights to IDX184, and it will look for another partner to support further development, said Jean-Pierre Sommadossi, Idenix’s chairman and CEO, in a statement. But any other company is certainly going to ask a lot of questions about the drug, given the closeness of Idenix’s relationship with Novartis. The Switzerland-based pharmaceutical giant held a majority ownership stake in Idenix, 53 percent, on April 1, according to Idenix’s most recent proxy statement, and Novartis markets another drug Idenix developed telbivudine (Tyzeka) for patients with hepatitis B.

IDX184 is designed to be a once-daily pill that’s active specifically in the liver, where the hepatitis C virus causes damage. It is Idenix’s lead drug candidate for hepatitis C, and it passed a preliminary clinical trial that demonstrated it was safe and had anti-viral activity in a three-day study, Idenix has said. Idenix said today in a separate statement that it plans to release more detailed results from that trial this weekend at the American Association for the Study of Liver Diseases, the big annual meeting for hepatitis C drugs which is being held this year in Boston.

Comments | Reprints | Share:        

We had a little something for everybody on the life sciences beat this week, with news on biopharmaceuticals, devices, and healthcare software.

—Seattle-based Omeros (NASDAQ: OMER) had potential to be a bellwether for the U.S. biotech sector earlier this month, as the first true biopharmaceutical company to go public since February 2008. But its stock has tanked, making it the worst-performing IPO of the year, according to data from Renaissance Capital. Not surprisingly, no other biotech company has stepped up to put its name on the IPO calendar since.

—ZymoGenetics‘ former chief medical officer, Jan Ohrstrom, told me about his latest passion to develop a dry powder drug for excess bleeding that could be conveniently used by paramedics, and might be a superior alternative to liquid formulations like the one made by his former employer. He has recruited a former Zymonite to work for him at ProFibrix, and he’s set up a new office not far away from his old stomping grounds on Seattle’s Eastlake Avenue.

—Microsoft has been leading the way for a couple years now to make medical records go electronic, and put individuals in charge of them, but the HealthVault software is still a long way from gaining mainstream acceptance. My colleague Ryan McBride reported on all the challenges Microsoft is facing following a talk by Peter Neupert, the vice president of Microsoft’s Health Solutions Group, at the Connected Health Symposium last week in Boston.

—The Washington Biotechnology & Biomedical Association’s big annual meeting in March is being revamped with a new name, new look, and a new program, as we heard in this guest editorial from Valerie Carricaburu of UW TechTransfer and biotech consultant Neile Grayson. The event, formerly known as Invest Northwest, is now going to be called Life Science Innovation Northwest, and will seek to entice not just investors, but also corporate and strategic partners with the wherewithal to support innovation in the Northwest. Valerie and Neile are seeking ideas from the community on how to improve the event, so feel free to post a note at the bottom of this story.

—Seattle-based Kineta, the developer of treatments for autoimmune diseases, formed a collaboration with Matawan, MI-based MPI Research, to support animal studies that will enable clinical trials to start in 2010. Terms weren’t disclosed.

—Bothell, WA-based SonoSite (NASDAQ: SONO) said its sales dropped by 13 percent in the quarter ending Sept. 30, and that it recorded an operating loss of $240,000 in the period.

—ZymoGenetics (NASDAQ: ZGEN) said it has started a mid-stage clinical trial of its pegylated interferon lambda drug for hepatitis C, which triggered a $70 million milestone payment from its partner, Bristol-Myers Squibb.

Comments | Reprints | Share:        

Vertex Pharmaceuticals (NASDAQ: VRTX) is vying to set a new standard of care for hepatitis C in some of the toughest patients to treat, and today it is reporting some surprisingly strong evidence that suggests the drug is working like researchers hoped it would over the long haul.

The Cambridge, MA-based biotech company offered an interim peek at results from 117 patients who took its telaprevir compound after they failed to fully respond to standard therapy. Vertex looked at “null responder” patients who didn’t respond at all to prior therapy, and found that 16 out of 28 of them, or 57 percent, achieved a clinical cure after they got telaprevir in combination with another round of standard treatment. The clinical cure, known formally as sustained viral response, is achieved when the hepatitis C virus disappears from the blood for a full 24 weeks after the course of treatment.

Telaprevir had about the same effectiveness rate for patients who partially responded to an earlier round of treatment (55 percent achieved clinical cure), while that rate shot up to 90 percent for patients who initially were helped by standard therapy, but ended up relapsing later. These results were from a study known as ‘107. Comparing separate clinical trials can be a dubious exercise because of apples-to-oranges issues, but the clinical cure rates Vertex is reporting today are higher than what it announced in April at a conference of the European Association for the Study of the Liver, in a separate study that also enrolled tough-to-treat patients, called Prove 3.

Side effects from this latest batch of results were consistent with what Vertex has seen in prior studies. Eight of the 117 patients quit taking their medication because of side effects, including four who dropped out because of rash, and one who cited anemia.

For those who are new to the Vertex story, here’s a quick refresher on why this all matters. Telaprevir is aiming to be a first-of-its-kind protease inhibitor against hepatitis C, a chronic liver disease. If it can deliver in the final stage of clinical trials, it will change the standard of treatment for the disease just as an earlier generation of antivirals did for HIV infection. The market is potentially huge, because an estimated 3.2 million people in the U.S. have hepatitis C infections, and about 650,000 have failed …Next Page »

Comments (1) | Reprints | Share:        

Seattle-based ZymoGenetics (NASDAQ: ZGEN) will receive a $70 million milestone payment from its partner, Bristol-Myers Squibb, for starting a mid-stage clinical trial of pegylated interferon lambda for hepatitis C, according to a statement. The trial, called Emerge, will initially enroll 50 patients on a variety of doses, then expand to 500 patients who are randomly assigned to get the ZymoGenetics drug or Roche’s peginterferon-alfa 2a (Pegasys). ZymoGenetics’ pegylated interferon lambda, sometimes called IL-29, is designed to have potent anti-viral activity without the nasty flu-like symptoms that force many patients to quit taking other interferon drugs.

Comments | Reprints | Share:        

Vertex Pharmaceuticals said today it will bring in $155 million in cash through a pair of deals related to the potential commercialization of its lead hepatitis C drug in Europe, the company said this afternoon. In both transactions, investors are betting on telaprevir, a projected multibillion-dollar seller, to win European approval.

The Cambridge, MA-based drug developer (NASDAQ:VRTX), which also has a significant presence in San Diego, is receiving about $120 million in cash from the sale of corporate debt that must be paid back by October 31, 2012. The notes are secured by $155 million in milestone payments Vertex is eligible to receive from Janssen Pharmaceutica for the European commercialization of telaprevir. In the second transaction, the firm will be paid $35 million in cash for the rights to potential milestone payments Vertex is in line to receive from Janssen for the European market introduction of telaprevir. And Vertex won’t have to repay the $35 million if it doesn’t earn the milestone payments.

Vertex, which is counting on telaprevir to be its first big moneymaking product, has been burning through huge amounts of cash this year as it goes through the final phase of drug development. The company reported a second-quarter net loss of $171.3 million, and said it had $754 million of cash left in the bank on June 30, in its most recent quarterly report. The new debt financing, and a $105 million payment from Japanese partner Mitsubishi Tanabe Pharma will bolster the company’s reserves, so that Vertex should finish this year with $800 million in the bank, according to a separate statement issued today. The company plans to continue to burn through cash as it pursues FDA approval for telaprevir in the second half of 2010.

Vertex spokeswoman Jane Kramer said that the investors in both deals included hedge funds and other firms, but the names of the investors will not be disclosed.

Vertex has retained full commercial rights to telaprevir in the U.S. market, while it has partnered with Janssen, a Johnson & Johnson subsidiary, and Mitsubishi Tanabe to commercialize the potential blockbuster drug in Europe and Asia, respectively.

Comments | Reprints | Share:        

More bad news out of ZymoGenetics (NASDAQ: ZGEN) this morning. The Seattle-based biotech company disclosed in a regulatory filing today that its partner, Germany-based pharmaceutical giant Merck KGaA, has halted studies of their atacicept drug candidate for multiple sclerosis after concluding the drug’s benefit wasn’t worth the risk.

One trial showed that multiple sclerosis patients actually had more flare-ups and brain lesions when they were given atacicept, compared with a placebo. Two studies are still ongoing of atacicept, one for rheumatoid arthritis and one for lupus, and no comparable issues have been seen in those studies, ZymoGenetics said.

The multiple sclerosis failure is the third big flop of the past year for the atacicept (pronounced Uh-TACK-ee-sept) program, once one of the shining stars in ZymoGenetics’ pipeline. The drug failed to control rheumatoid arthritis in a pair of mid-stage clinical trials last month. Last October, ZymoGenetics said a trial of the drug for patients with lupus of the kidneys was halted after it appeared to raise the risk of severe infections.

ZymoGenetics had been developing the product jointly with Merck KGaA until a year ago, when it decided to conserve cash by handing over full responsibility for development costs, and settled for a smaller share of future royalties from the product. That move was designed to save ZymoGenetics $200 million in development costs as atacicept was scheduled to move into the final, most expensive phases of clinical trials.

The ZymoGenetics drug was largely forgotten by many analysts at that point, but it got some renewed attention in July, when Rockville, MD-based Human Genome Sciences (NASDAQ: HGSI) delivered the first convincing proof that doctors can successfully treat lupus by blocking a specific inflammatory protein known as BLyS (pronounced bliss). This caused Human Genome Sciences’ stock to quadruple, and gave ZymoGenetics a smaller boost, because its drug is made to block not just BLyS, but another inflammatory protein called APRIL, that the Human Genome Sciences drug doesn’t hit. Since multiple sclerosis is an autoimmune disease, in which the immune system goes haywire and starts attacking the normal fatty coating around nerve cells, researchers hoped that atacicept’s ability to dampen the inflammation would help patients with that condition like with other autoimmune diseases.

Since last September, when ZymoGenetics handed over atacicpet rights to Merck KGaA, it has concentrated more of its efforts on a new pipeline project, pegylated interferon lambda for hepatitis C, which is the subject of a new partnership with Bristol-Myers Squibb that’s potentially worth more than $1 billion.

ZymoGenetics stock fell 8 cents to $6.07 after the opening bell on the news.

Comments | Reprints | Share:        

Santaris Pharma, the Danish company that has pushed the first microRNA therapy into clinical trials, is opening a U.S. subsidiary in San Diego to take advantage of the talent pool of people familiar with cutting-edge RNA-based therapies. As it turns out, that includes Art Levin, a former leader of drug development at Carlsbad, CA-based Isis Pharmaceuticals (NASDAQ: ISIS).

Santaris, founded in 2003, has recruited Levin to serve as president of U.S. operations and chief development officer for the Denmark-based parent company. Levin tells me his job will be to usher Santaris’ drug candidates through development, and build a team of about 8 to 10 people in San Diego to help raise the company’s profile with prospective partners and investors in the U.S. and Asia. The San Diego operation isn’t expected to do basic research, which will remain in Denmark, he says.

“There’s a wealth of science and RNA drug expertise here in San Diego, and Santaris understands this is a great place to do business,” Levin says.

RNA-based therapies have long excited scientists because they are supposed to offer greater ability to target the root causes of many diseases than conventional chemical compounds or genetically engineered proteins. MicroRNAs offer another new frontier because they can affect not just one gene or protein in isolation, but rather networks of genes. That might be useful in treating complex diseases like diabetes or heart failure, where multiple genes are thought to be out of whack. Isis and Regulus Therapeutics, a company Isis created with Alnylam Pharmaceuticals, are leaders in the field, but Santaris has been gaining visibility for its work. The Danish company has struck new collaborations this year with U.K.-based Shire and Madison, NJ-based Wyeth to co-develop RNA-based therapies.

When I asked if Santaris is also hoping to recruit some of Levin’s friends and former colleagues from Isis and Regulus, he said that would not be appropriate. He noted that biotech can be a “small world” and that he has a lot of friendships in San Diego he wants to keep.

But, he quickly added, “If people knock on my door, that’s great.”

Levin spent about a dozen years at Isis before he left in 2007 to do some consulting for drugmakers on RNA-based therapies. He said he did expert consulting on “all the major players” in RNA based therapies, and decided to take the Santaris job because he likes the chemistry Santaris is using the best.

The company’s advantage, he says, is that it can make RNA-based compounds that have higher affinity for a given target, which means they bond more tightly with the molecules that make up their target. They also appear to be more specifically aimed at targets, which means that they might be more effective at lower doses than competitors, he says.

All of this work is in really early-stages of development. Santaris will get a better feel for what it has when it sees results from its first trial of a drug to block a microRNA target called miR-122, which is being tested as a potential therapy for hepatitis C. The drug, SPC3649, is scheduled to start a mid-stage clinical trial in hepatitis C patients in the second half of this year, according to the company’s website.

Comments | Reprints | Share:        

Arena Pharmaceuticals (NASDAQ: ARNA) plans to announce results of a 4,000-patient clinical trial of its obesity drug lorcaserin this month. But Northern California rival Vivus (NASDAQ: VVUS) announced results of its own obesity drug first. Get the skinny on all this and the rest of San Diego’s life sciences news.

—San Diego’s Arena Pharmaceuticals is set to release the results of a major study that was designed to see whether a high or low dose of its experimental drug lorcaserin can help patients lose weight. As Luke reported, Arena has a lot riding on the outcome. The company has raised almost $1 billion and spent 12 years developing its drug.

—Meanwhile, Mountain View, CA-based Vivus set high expectations yesterday when it reported that a high dose of its drug, which is a combination of phentermine and topiramate, helped patients lose an average of more than 10 percent of their body weight.

—After six years of development, San Diego’s Minnow Medical is working to commercialize technology that founding CEO Tom Steinke describes as an exciting advance in balloon catheters, which are used in angioplasty. After raising $22 million and expanding Minnow Medical to 28 employees, Steinke is seeking additional capital from prospective investors.

—Cambridge, MA-based Genzyme (NASDAQ: [[ticker: GENZ]]) paid $7 million to acquire a lot of patents and proprietary know-how in gene therapy from Seattle’s Targeted Genetics (NASDAQ: [[TICKER:GENZ]]). Targeted Genetics delivered the intellectual property earlier this week to San Diego, where Genyzme operates a gene therapy facility.

—San Diego’s Metabasis Therapeutics (NASDAQ: [[ticker: MBRX]]) said it has received a Nasdaq delisting notice, which may be the least of its worries. The ailing biotech said last week it hired a financial firm to evaluate its strategic options. Metabasis also announced the resignation of Mark Erion, the company’s CEO, chief scientific officer, and director. Chairman David Hale is overseeing what appears to be the company’s end game.

—San Diego’s Anadys Pharmaceuticals (NASDAQ: [[ticker: ANDS]]) said it has started a mid-stage clinical trial of its lead drug candidate for patients with hepatitis C. The company said results of the experiment should be available by year-end, with more follow-up data to come in 2010.

Comments | Reprints | Share:        

San Diego-based Anadys Pharmaceuticals (NASDAQ: ANDS) said today it has started a mid-stage clinical trial of its lead drug candidate, ANA598, for patients with hepatitis C. The study will enroll 90 patients on the drug or a placebo, in combination with the standard treatments—pegylated interferon alpha and ribavirin. Initial results on the anti-viral activity of the Anadys drug should be available by year-end, with more follow-up data to come in 2010, the company said. This ambitious trial design is part of what has brought Anadys back from some dark days earlier this year.

Comments | Reprints | Share:        

If there were a prize for the whiplash-inducing roller coaster story of the year in San Diego biotech, Anadys Pharmaceuticals would have to be a contender. But if CEO Steve Worland has his way, some of that stomach-turning drama of the past eight months will soon just be a memory.

“You might say we’ve been stabilized, but I’m not sure we were really unstable before,” Worland says. “We’ve turned an important corner.”

Anadys got to this position, talking about stability, after it dropped bombshells on investors twice this year—once in a good way, and once not so good. The positive one came in January, when the company (NASDAQ: ANDS) shocked Wall Street by dribbling out data from the first eight patients with hepatitis C who got the lowest dose of its experimental drug, ANA598. The patients had 99 percent of their virus wiped out within the first 72 hours, which was a far better anti-viral punch than any other drug in its class. The stock, which had been on practically no one’s trading screen the day before, with just 68,000 shares changing hands, rocketed on the news from $1.91 to $4.10 on volume of more than six million shares.

This was just the beginning. The preliminary results were from the lowest of three doses studied in a clinical trial, and Anadys suggested the data would only look better a couple months later, at a key research meeting, the European Association for the Study of the Liver. Anticipation was in the air: Anadys engaged in talks with potential partners about the data. The hepatitis C space was hot. Cambridge, MA-based Vertex Pharmaceuticals built a legion of fans on Wall Street for its industry-leading drug, and then Vertex paid more than $375 million in March to acquire another hepatitis C drug developer at a similar stage of development as Anadys. Shortly after, Worland talked about how his company and others were changing the paradigm for treating hepatitis C, following the cocktail-drug approach pioneered by HIV treatments.

Then came the plunge. Anadys presented full results from the clinical trial, which showed that, as predicted, the company’s ANA598 product had even stronger anti-viral activity at higher doses. There were no serious side effects, no signs of patients developing drug resistance, or of the virus bouncing back.

Anadys CEO Steve Worland

What was the problem? A separate study of 24 healthy volunteers showed that three patients dropped out of the study because they had developed Grade 2 rashes, measured on a scale of one to four, with four being the most severe. The price of Anadys shares fell, even though hepatitis C patients on the drug had no severe rashes, other drugs in the class have the same side effect, and healthy volunteers essentially have no reason to stay in a study if they see any side effects at all. Such nuances were lost on the fast-money crowd. Anadys shares fell 40 percent that day, making it the biggest decliner on the Nasdaq, and shares kept falling. “People thought the rash was more severe than it was,” Worland says. “It was an extreme reaction.”

Regardless of which interpretation you prefer to believe, Anadys had to deal with the consequences. It wasn’t able to find a partner to help take ANA598 through the next phases of clinical development on its preferred terms, and cash began to run low. By June, Worland had to act, by shedding …Next Page »

Comments (1) | Reprints | Share:        

San Diego-based Anadys Pharmaceuticals (NASDAQ: ANDS) said today it has gotten the green light from the FDA to start a mid-stage clinical trial of its experimental hepatitis C drug, ANA598, in combination with the standard treatments, pegylated inferferon alpha and ribavirin. About 90 patients are expected to enroll, with the first joining the study within weeks, the company said. Anadys, which hopes this drug candidate will alter the standard of care for hepatitis C, expects to receive the first results from this study as soon as the end of the year.

Comments (1) | Reprints | Share:        

Vertex Pharmaceuticals (NASDAQ:VRTX) is making its second move this month to raise non-dilutive cash from its previous agreements to commercialize its experimental hepatitis C drug telaprevir. The Cambridge, MA-based firm said this morning that it will receive an upfront sum of $105 million from Mitsubishi Tanabe Pharma in an amended deal between the two companies to commercialize the potential blockbuster drug in Asia.

Mitsubishi, one of the largest pharma outfits in Japan, is paying Vertex for rights to sell and manufacture telaprevir for treating HCV infections in combination with the existing drugs interferon and ribavirin in Japan and the Far East. Vertex is also eligible for between $15 million and $65 million in milestone payments—in lieu of royalties—for the approval and commercial launch of telaprevir in Japan, according to Vertex. This amends the two companies’ 2004 deal, in which Mitsubishi gained certain rights to telaprevir in Asia as a standalone HCV therapy.

The new agreement gives Vertex an immediate cash infusion as the firm moves telaprevir, a protease inhibitor drug, through expensive, late-stage clinical trials in the U.S., Europe, and Japan. Earlier this month, Vertex announced an interesting fund-raising plan to sell rights to receive $250 million in payments that the company is eligible to garner for the successful approval and launch of telaprevir in Europe from the Janssen Pharmaceutica unit of health products giant Johnson & Johnson (NYSE:JNJ). (Yes, that’s the rights to receive the payments, not the commercial rights themselves, which Janssen would still control if the payment rights were sold to another party.) Vertex still has exclusive commercial rights to telaprevir in North America, where analysts expect the drug to become a multibillion-dollar product if it is approved.

“The cash received [in this amended deal with Mitsubishi] strengthens our corporate financial position during an important period of investment and growth as we advance two Phase III programs in hepatitis C and cystic fibrosis,” said Kurt Graves, chief commercial officer of Vertex, in a prepared statement.

Telaprevir is an oral drug that is designed to attack an enzyme important to viral replication in HCV, a chronic liver disease that affects an estimated 170 million people worldwide, according to Vertex.

Comments | Reprints | Share:        

Regulus Therapeutics, its CEO admits, is a child of privilege. It was born two years ago with a silver spoon in its mouth—some hot intellectual property to make drugs based on emerging knowledge about microRNA biological networks. It has a couple of parents with wealth and pedigree: Cambridge, MA-based Alnylam Pharmaceuticals (NASDAQ: ALNY), and Carlsbad, CA-based Isis Pharmaceuticals (NASDAQ: ISIS).

Since we’re watching closely to see how innovation is affected during this economic downturn, I figured it seemed reasonable to check whether ideas on the real frontier of biology can still be sustained at a place like Carlsbad, CA-based Regulus, which we first profiled back in October. CEO Kleanthis Xanthopoulos says he’s always worried about financing—it’s the nature of being a biotech CEO—but enough positive things have happened this year that he says the company has still been able to grow.

That’s no small thing, because companies pursuing microRNA know they are staring at what amounts to a 20-year vision to develop a new class of therapies, as Xanthopoulos pointed out in that first story in October. The key is showing people enticing bits of progress along the way. Regulus did that around Thanksgiving, when it published a scientific paper in Nature that was the first to demonstrate a therapeutic benefit in animals by blocking a strand of microRNA. The company raised $20 million from its corporate parents a couple months later, then scored an undisclosed sum of cash from its big partner, GlaxoSmithKline, back in May when it reached a milestone as part of a collaboration worth as much as $600 million. The company has almost doubled in size, from 22 employees last October, to almost 40 today, Xanthopoulos says.

“Am I concerned about financing? Always. I’m paranoid when it comes to financing,” Xanthopoulos says. “It’s a capital-intensive business, with long development timelines. But we’re really well-capitalized.” He has at least three years of operating cash on hand, and the company recently re-organized …Next Page »

Comments | Reprints | Share:        

Cambridge, MA-based Vertex Pharmaceuticals (NASDAQ:VRTX) revealed today its plans to raise cash through the sale of rights to $250 million in milestone payments it could receive for successfully developing and launching its experimental hepatitis C treatment, telaprevir, in Europe, according to a press release. No buyer of the rights was announced. Vertex gained rights to the payments through its 2006 collaboration with Janssen Pharmaceutica, a unit of Johnson & Johnson (NYSE:JNJ), which agreed to pay Vertex $100 million if telaprevir garners European approval and $150 million upon the launch of the drug in Europe in exchange for European marketing rights. Here is a story from the In Vivo Blog with some insights about why selling rights to these milestone payments may make sense for Vertex.

Comments (1) | Reprints | Share:        

There was a lot of life sciences news over the past week in San Diego, with much of it coming out of the American Diabetes Association annual meeting in New Orleans.

—At a time when San Diego’s Amylin Pharmaceuticals (NASDAQ: AMLN) doesn’t need more challenges, Denmark’s Novo Nordisk announced that liraglutide, its once-daily diabetes drug, is substantially better at controlling blood sugar in type 2 diabetes than Amylin’s exenatide, which is injected twice-daily.

—If Amylin Pharmaceuticals’ rivals are targeting the diabetes specialist, at least the San Diego biotech is a moving target. Amylin says the latest clinical data for the new formulation of its exenatide diabetes drug (which only has to be injected once weekly instead of twice daily) shows patients were able to maintain control over blood sugar for two years, helped them lose weight, and lowered their blood pressure.

—San Diego’s Orexigen Therapeutics (NASDAQ: OREX) posted new results of its late-stage drug for treating obesity, showing that 41.5 percent of patients on the drug lost 10 percent of their body weight. That compared with 20.2 percent who did as well taking a placebo.

—Another San Diego drug developer, Arena Pharmaceuticals, (NASDAQ: ARNA) says patients on its lorcaserin treatment for a year generally had reduced their risk of heart attacks.

—San Diego’s Anadys Pharmaceuticals (NASDAQ: ANDS) is cutting its workforce by 40 percent and focusing its remaining resources on its lead drug candidate for treating the hepatitis C virus. The company also says it’s raising $17.5 million.

—Trius Therapeutics, a San Diego biotech developing a new antibiotic against deadly MRSA bacterial infections, said a study showed 98 percent of patients on the lowest dose of its new compound achieved a clinical cure. 

—Raj Krishnan, a bioengineering Ph.D candidate at UC San Diego, began entering student entrepreneur and business contests as a way to raise money for Biological Dynamics, the cancer diagnostics company he co-founded. It worked. He has now won 13 awards, and his most recent win included a check for $40,000. Krishnan, 27, says his research is focused on technology to identify a key biomarker for almost every type of cancer.

—After getting a $20 million infusion from investors last month, San Diego’s Vical (NASDAQ: VICL) says it has enough resources to continue operating through the end of 2011, which should be enough time to complete its clinical study of an immune-stimulating therapy for metastatic melanoma. Vical also is developing a vaccine to prevent life-threatening cytomegalovirus infections in bone marrow transplant patients.

—Bothell, WA-based Sonosite (NASDAQ: SONO) says it’s acquiring San Diego’s CardioDynamics International (NASDAQ: CDIC) in a deal valued at about $10 million. Sonosite makes portable ultrasound machines and CardioDynamics makes noninvasive medical diagnostic tools for cardiovascular disease.

—San Diego-based Tocagen, which is developing gene therapy treatments for terminally ill cancer patients, was among three biotechs that raised cash recently. Tocagen has raised $10.8 million in an $11.2 million equity offering that began in September. The startup was founded in 2007 by Harry Gruber, who previously founded Gensia Pharmaceuticals, Viagene, Aramed, and Metabasis, among others.

Comments | Reprints | Share: