The Food and Drug Administration, often the center of attention for the U.S. biotech and medical device industries, came under the spotlight a bit more this week amid talk of changing the agency’s mission statement. There also was news from Amylin that should address at least some concerns FDA regulators have raised about its once-a-week treatment for diabetes.

—Shares of San Diego’s Amylin Pharmaceuticals (NASDAQ: AMLN) have gained more than 13 percent since Friday, when the company said its analysis of an earlier trial found no connection between its once-weekly injectable version of exenatide (Bydureon) and prolonged heart rhythms in 148 patients. At the American Diabetes Association’s annual conference in San Diego, Amylin also said another study showed that an earlier version of the drug, the twice-daily injectable version of exenatide (Byetta), was associated with a lower risk of heart failure. At the FDA’s request, the company is conducting another trial to specifically measure the drug’s effect on the QT interval, the time the heart’s electrical system takes to recharge after each beat. Amylin shares closed yesterday at $13.37, gaining $1.54 since closing last week at $11.83.

—San Diego-based Vital Therapies recently raised $22.6 million toward a possible $56.8 million Series D financing that combines equity, warrants and securities, according to a regulatory filing. The company has developed technology to stabilize liver function in patients with life-threatening, acute liver failure, through a process akin to dialysis that uses a proprietary filter to help regenerate healthy liver cells. Since the company was founded in 2003, Vital Therapies has raised about $40 million through three financing rounds. Versant Ventures is the lead investor.

—Independa, a wireless health startup founded in San Diego two years ago, said it’s making its integrated telecare app available for beta testing. The tablet-based system enables friends, relatives, and other caregivers to monitor an elderly loved one. The technology also uses … Next Page »

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San Diego’s Conatus Pharmaceuticals, which specializes in developing drugs for liver disease and cancers, said it closed on a $20 million block of equity funding, which was led by a new investor, AgeChem Venture Fund of Montreal. Existing investors Aberdare Ventures, Advent Venture Partners, Bay City Capital, Gilde Healthcare Partners, and the Roche Venture Fund also participated. In a statement Friday, Conatus said it plans to use the proceeds to advance development of a new drug for treating hepatitis C. Conatus, which was founded in 2005, said the Series B financing will remain open as other potential investors study the deal.

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San Diego’s Ligand Pharmaceuticals said today it has licensed two liver drug candidates to Chiva Pharmaceuticals, a Los Altos Hills, CA, affiliate of Hainan Kaihua Pharmaceutical. Ligand says the deal, which gives Chiva rights to develop and market the drugs in China, could be worth more than $100 million in milestone payments and royalties. Ligand also has the potential to get a 10 percent stake in Chiva as well as sublicensing revenue. Ligand acquired drugs a year ago through its acquisition of Metabasis Therapeutics. One drug is a potential treatment for hepatitis B and the other is being tested as a treatment for hepatocellular carcinoma, the most common type of liver cancer.

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Conatus Pharmaceuticals, a San Diego-based drug developer targeting liver disease and cancer, announced it has acquired Pfizer subsidiary Idun Pharmaceuticals for an undisclosed sum. Conatus was launched in 2005 by Idun execs after Pfizer acquired the firm, which at the time was developing liver disease treatments and oncology drugs, and held intellectual property in the area of apoptosis. “Conatus plans to leverage its scientific and drug development expertise and resources to continue to advance potentially important therapeutic candidates in liver disease and oncology,” said Conatus CEO Steven Mento of the company’s acquisition of Idun.

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San Diego’s biotech news picked up in pace following the Thanksgiving holiday, with Phenomix announcing a major partnership, Santarus benefitting from FDA approval of its over-the-counter heartburn drug, and Receptos getting a VC infusion. Our Xconomy post-holiday news feast is ready, so come and get it:

—After forming a partnership last year with New York-based Forest Laboratories, San Diego’s Phenomix says Italian pharmaceutical company Chiesi Farmaceutici has agreed to help commercialize its diabetes drug in Europe and elsewhere. Chiesi has agreed to pay Phenomix as much as $163 million if all milestones are met, including $28 million in near-term cash and equity.

—Prominent algae biologist Stephen Mayfield, a co-founder of San Diego’s algae-to-biofuels startup Sapphire Energy, told Denise that algae could become just as important to the biotech industry as it is now with the renewable energy industry. Mayfield, who also founded Rincon Pharmaceuticals, showed earlier this year that algae could be used to produce an anti-anthrax antibody—and other projects are testing the feasibility of using algae to produce anti-tumor drugs and even a malaria vaccine.

—San Diego’s Santarus (NASDAQ: SNTS) said the FDA approved a request by its corporate partner, the Schering-Plough unit of Merck, to start selling an over-the-counter version of its heartburn drug omeprazole (Zegerid). Approval of the lower-dose version of the heartburn drug means that Santarus can collect a $20 million milestone payment, and is eligible to get additional payments based on sales.

—Receptos, a San Diego-based biotech with a new way of taking the guesswork out of drug discovery, is the recipient of $25 million in venture capital from Arch Venture Partners, Flagship Ventures, Lilly Ventures, and Venrock Associates. Receptos also got something else from Venrock: Bill Rastetter, a Venrock partner who was the CEO of Idec Pharmaceuticals before it merged with Cambridge, MA-based Biogen, has joined Receptos as CEO.

—San Diego-based Ocera Therapeutics may have a drug for treating hepatic encephalopathy, a complication among people with cirrhosis of the liver that causes ammonia levels to build up in the bloodstream, eventually causing cognitive disorders. The company has raised $62 million in three rounds of financing from a group of prominent VC firms—including Domain Associates, Sofinnova Ventures, InterWest Partners, and Thomas, McNerney & Partners.

—Luke has been extremely enthusiastic about the speakers he’s recruited to speak about biotech innovators and innovation at Xconomy’s Dec. 14 event in San Diego. The afternoon session includes talks by Caltech’s David Baltimore, the Nobel Laureate and a co-founder of Carlsbad, CA-based Helixis, and John Maraganore, the CEO of Cambridge, MA-based Alnylam Pharaceuticals (NASDAQ: ALNY). Check out the full list of speakers, and register online here. It will be a great networking opportunity and we want to see you there.

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The liver is hot in the biotech world. Cambridge, MA-based Vertex Pharmaceuticals (NASDAQ: VRTX) has made headlines this year with an antiviral drug that it hopes will do for hepatitis C—a chronic liver-damaging condition affecting millions of people—what a previous generation of treatments did for HIV.

But away from the spotlight, a small San Diego biotech company called Ocera Therapeutics says it has built up some preliminary evidence that it may have an interesting new drug to treat hepatic encephalopathy, a common complication among people with cirrhosis of the liver. The company has raised $62 million in three rounds of financing from a group of prominent venture capitalists—including Domain Associates, Sofinnova Ventures, InterWest Partners, and Thomas, McNerney & Partners.

The lead drug candidate at Ocera is designed to treat a condition that sometimes afflicts people with damaged livers and that causes them to lose the ability to fully break down toxins like ammonia. This means that ammonia levels build up in the bloodstream, and eventually penetrate into the brain, causing cognitive defects like a lack of concentration and slow reaction times, says Ocera CEO Laurent Fischer. Estimates vary widely on how many people really suffer from hepatic encephalopathy, partly because it’s hard to diagnose. But the figure is somewhere between 30 and 80 percent of the 1 million people in the U.S. with cirrhosis, Fischer says. If Ocera is on the right track, its drug will get rid of some of that ammonia circulating through the bloodstream, and this will help people with liver damage to stay mentally sharp.

“People with mild hepatic encephalopathy have more car accidents, they’re more likely to lose their job, suffer falls, and have a poor quality of life,” Fischer says.

Laurent Fischer

Ocera is still very much in the early stages of building a medical case, so it’s too early to say that the drug is really effective. But the company did present some data last month at the American Association for the Study of Liver Disease in Boston—known as The Liver Meeting—so I figured it was worth catching up on its story.

The company was started in January 2005 by Fischer and Eckard Weber, a partner with Domain Associates in San Diego. The idea was to take a product that was being marketed in Japan by Kureha as a treatment for kidney dialysis patients. The drug (AST-120) is an oral pill that’s designed to stay in the gut, without being absorbed into the bloodstream, which makes it less likely to have a surprising side effect. The drug is what’s called an adsorbent, loaded with carbon microspheres that are made to bind efficiently with toxins like ammonia and other metabolic byproducts in the gut, while avoiding common digestive enzymes or vitamins, Fisher says.

“It acts like a sponge in the gastrointestinal tract,” he says.

Ocera sees lots of potential opportunities for a drug that works this way, especially … Next Page »

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More data is rolling in from Vertex Pharmaceuticals on its lead drug for hepatitis C, and it looks like the medicine is continuing to live up to its own high expectations. The Cambridge, MA-based company said today that telaprevir’s effect on killing the virus is remaining durable on follow-up analysis, and the drug appears to just about as good in a more convenient twice-daily pill form instead of its standard three-times-a-day dosing schedule.

The data dump came in front of a prime audience of physicians attending the American Association for the Study of Liver Disease annual meeting today in San Francisco. The results appear to match up pretty consistently with what Vertex has reported previously, which I wrote about in this advance story.

To recap what’s at stake, telaprevir is aiming to be a first-in-class 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. An estimated 3.2 million people in the U.S. have hepatitis C infections, and about 170 million have it worldwide. Telaprevir must be taken with a pair of standard drugs, pegylated interferon alpha and ribavirin, which cause flu-like symptoms and must be taken for almost a year. The Vertex drug is highly anticipated, though, because it has shown in earlier trials that it can almost double the cure rate for patients when added to standard therapy, and in half the time. If approved, the product could generate $2.6 billion in U.S. sales in 2013, according to Rachel McMinn, an analyst with Cowen & Co. in San Francisco.

“These are strong and striking early results,” says Freda Lewis-Hall, Vertex’s executive vice president for medicines development.

In the spirit of helping you keep all this data straight, because there’s a lot of it, we’re breaking this down into bite-size chunks. .

—The ’107 study. This trial of patients who didn’t respond to previous treatment, produced data from more patients who were followed over a longer period than was previously reported. Researchers found that 68 of 104 patients, or about two-thirds, who got telaprevir along with standard drugs had no evidence of the virus in their blood after four weeks. That effectiveness appeared to be long-lasting. Researchers found that 41 of 71 patients (58 percent) followed for 24 weeks still had no evidence of the virus in the blood. Importantly, telaprevir is showing that it can wipe out the virus about 43 percent of the time in the toughest patients of all to treat—those who didn’t respond at all to a prior round of therapy.

The last time Vertex reported results from this trial, at a European meeting in April, it didn’t have any follow-up data that extended this long, or such strong data for what are called “null responder” patients. Of course, there were side effects, though. About 8 percent of patients dropped out of the study because of adverse events, with about half of them quitting because they developed a rash, the company said.

—The C208 study. This trial is about convenience for patients. … Next Page »

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Anadys Pharmaceuticals may not be first in a new class of emerging drugs for hepatitis C, but it’s aiming to show this weekend it’s a contender. The San Diego-based company is reporting today that its lead drug candidate was found safe at a variety of doses in a clinical trial of 48 healthy volunteers, and appears to have potential to be given as a once-daily or twice-daily pill.

San Diego-based Anadys (NASDAQ: ANDS) reported results on ANA598 at the American Association for the Study of Liver Disease meeting today in San Francisco. The company (pronounced Uh-nad-iss), found its drug appeared to be highly potent against the virus at the second-lowest dose tested, and that no serious side effects were reported among any patients, whether they took 400 milligrams, 3000 milligrams, or anything between, researchers said at the liver meeting.

“We’re comfortable predicting that we’re going to have an antiviral effect,” said CEO Steve Worland. “This puts our stake in the ground.”

This safety data is important to Anadys. Its stock was cut in half on June 26, 2006 after one of its other candidates, ANA975, was found to cause “intense immune stimulation” in animals. That drug was scrapped a year later when another animal study confirmed the unwanted effect, which caused a partnership with the drug giant Novartis to unravel.

Now Anadys is taking a new approach with ANA598, a polymerase inhibitor drugs against hepatitis C. These drugs could be added in combination treatments with protease inhibitor drug candidates now in later stages of development from Vertex Pharmaceuticals and Schering-Plough. They’re all chasing a big market opportunity, with 3.2 million people in the U.S. infected with hepatitis C, and an estimated 170 million worldwide.

The Anadys drug is going up against some tough players in its quest to develop a polymerase inhibitor for hepatitis C, namely Gilead Sciences (NASDAQ: GILD) and a partnership between Roche and Pharmasset. Those companies are further along in development than Anadys, Worland says, plus Abbott Laboratories and Pfizer are working in the field at earlier stages. They are drawn to this class of polymerase inhibitors because doctors see potential for them to add punch to protease inhibitors, since the different drugs attack different parts of the virus, Worland says.

“What people want is to use more than one antiviral in combination with each other. It’s building on the HIV paradigm,” Worland says.

The data from Anadys and its competitors is being watched carefully by the bigger players in heptatis C treatment. … Next Page »

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