Another day, another piece of bad news from Genzyme. The Cambridge, MA-based biotech giant (NASDAQ: GENZ) said today it is scrapping development of a next-generation drug for kidney disease after it was unable to beat its existing treatment on the market.

Genzyme’s experimental drug, called an advanced phosphate binder, didn’t appear any better at getting rid of excess phosphorus from the blood of kidney dialysis patients than Genzyme’s sevelamer carbonate (Renvela) in a clinical trial of 349 patients, the company said in a statement. Genzyme had been hoping that the new drug would be more potent.

Those following the Genzyme story know about what’s become a brutal year. Some of the big setbacks were the viral contamination at its Allston, MA factory in June that created shortages of its top-selling products, the failure to win FDA approval of large-scale manufacturing for a Pompe drug, and the rejection of a leukemia drug for elderly patients.

But this kidney drug failure is another serious body blow. The company generates $850 million a year in sales from its two phosphate binder treatments. Patents that protect that franchise from competition from cheaper generics will expire in September 2014, according to Christopher Raymond, an analyst with market research firm Robert W. Baird. The next-generation kidney drug was supposed to help the company extend the patent life of its kidney drug market, Raymond said.

The failure is “a very significant negative in our view,” Raymond said in a note to clients today. He added that he had been starting to warm up to the company, as it has been inching closer to resolving the Allston manufacturing problems, but he called today’s halting of the kidney program a “game-changer.” Raymond has a $54 price target for the stock.

Shares of Genzyme dropped 1.3 percent to $49.65 at 9:39 am Eastern time today after the news was announced. The stock has dropped 25 percent this year.

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Cambridge,MA-based biotech Alkermes said this morning that its long-acting naltrexone injection (Vivitrol) was more effective in treating opioid drug dependence than placebo in a Phase III clinical trial. The 250-patient clinical trial compared urine samples of patients who took the firm’s once-per-month injection of naltrexone versus those who took placebo over the last 20 weeks of a 24-week treatment period. Alkermes (NASDAQ:ALKS) said that it plans to file an application with the FDA for approval of its drug for treating opioid dependence in the first half of 2010. The FDA approved the drug for treating alcohol dependence in 2006.

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Cambridge, MA-based Ironwood Pharmaceuticals has some new evidence out this morning that its lead drug candidate is an effective treatment for patients with chronic constipation.

Ironwood and its partner, New York-based Forest Laboratories (NYSE: FRX) said last night that the drug, linaclotide, passed the test in a pair of pivotal clinical trials. A significant percentange of patients who got a once-daily dose of the drug experienced relief from their constipation, the primary goal of the studies, as well as relief from bloating and abdominal discomfort. The most common adverse events in patients on the Ironwood drug were diarrhea, flatulence and abdominal pain; 7.4 percent of patients taking the drug dropped out of the studies, compared to 4.2 of the patients who were on a placebo.

This result means that Ironwood and Forest essentially have two down, two to go with their clinical-trial plan for the new drug. About 1,270 patients were enrolled in these two constipation trials, and the companies expect results from two more pivotal trials among 1,600 more patients who have irritable bowel syndrome with constipation. Results from those trials are expected in the second half of 2010, the companies said. Forest has been quite bullish on this drug’s prospects, noting that estimated 26 million Americans have chronic constipation, there are few available therapies for it, and the Ironwood drug has a novel mode of action. The U.S. health care system spends more than $25 billion a year on treating irritable bowel syndrome, according to Forest Laboratories’ annual report. If approved, linaclotide could generated $572 million in annual sales by 2015, according to Tom Russo, an analyst with Robert W. Baird.

The results “confirm the potential for linaclotide to bring relief to the millions of patients suffering from many of the symptoms associated with chronic constipation,” said Forest CEO Howard Solomon in a statement. Ironwood CEO Peter Hecht noted that the company was glad to see the pivotal study results reproduce what was observed in smaller studies.

Ironwood has been able to raise more than $306 million since its founding, and accumulate more than $100 million in the bank now, thanks largely to having a drug with an unusual profile targeted at a common condition that’s tough to treat. Linaclotide is a peptide, and while most peptides are injected, Ironwood turned its drug into an oral pill that can withstand stomach acids, work its way into the intestines, and do its job there, without being absorbed into the bloodstream, where it can cause side effects, Hecht has said. The drug is thought to act by stimulating secretions of fluids into the intestines, which softens stool and helps people have easier bowel movements.

Forest Labs shares climbed almost 2 percent to $27.93 at 11 am Eastern today.

Digging a little further into the data, this drug doesn’t appear to help everybody. Both studies …Next Page »

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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 »

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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.

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Some unexpected bad news out of Seattle Genetics is driving down its stock 14 percent this morning. The Bothell, WA-based developer of antibody drugs for cancer (NASDAQ: SGEN) said a mid-stage clinical trial of an experimental lymphoma drug was halted midway through enrollment after an independent panel of data monitors determined the experimental drug was unlikely to reach its goal of beating the control group.

The Seattle Genetics antibody drug, called dacetuzumab or SGN-40, was being studied in a trial of 224 patients with diffuse large B-cell lymphoma, in combination with rituximab (Rituxan) and standard chemotherapies. Patients in the control group got the same treatment, except a placebo instead of the Seattle Genetics drug. About two-thirds of the patients were enrolled in the randomized, blinded trial before it was closed. Seattle Genetics didn’t say anything in its statement today about the safety profile of its drug.

The trial failure is a setback for Seattle Genetics, which has been developing this drug since January 2007 in partnership with Genentech and now Roche. The new antibody is designed to hit a different target found on cells, called CD40, which is found on many types of malignant B cells and is supposed to be complementary to the standard rituximab antibody that hits another target called CD20. Rituximab, the pioneering antibody for cancer, generated $2.59 billion in U.S. sales last year as it has become a standard lymphoma treatment. Yet as good as it is, patients eventually see their disease spread, and relapse after getting rituximab. Seattle Genetics pointed out in its release that it has four other smaller clinical trials of its dacetuzumab treatment for non-Hodgkin’s lymphoma and multiple myeloma, a cancer of the bone marrow.

“We are disappointed that the interim analysis of the phase IIb clinical trial resulted in discontinuation of the study, especially given the unmet medical need for DLBCL patients,” said Clay Siegall, CEO of Seattle Genetics, in a statement. “In collaboration with Genentech, we will conduct a detailed analysis of data from the SeaGen MARINER trial.”

Seattle Genetics shares fell $1.79, or about 14 percent, at the opening bell this morning to $11.83.

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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.

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ZymoGenetics (NASDAQ: ZGEN), the Seattle-based biotech company, said today in a regulatory filing that the atacicept drug it developed and licensed to Merck KGaA has failed to reach its goal of controlling rheumatoid arthritis in a pair of mid-stage clinical trials. The study confirmed the biological effect of atacicept, and researchers saw no unexpected side effects. Studies are still ongoing of atacicept for two other autoimmune diseases—lupus and multiple sclerosis, the company said.

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CareFusion, a medical technology company, got its official start earlier this week and is already one of the largest companies in San Diego, with 15,000 employees and $3.7 billion in annual revenue. Find out how that happened and read up on other local biotech news.

—Shares of San Diego’s Acadia Pharmaceuticals (NASDAQ: ACAD), which had run up nearly six-fold since late April on hopes for its lead experimental drug, collapsed after the biotech said Tuesday that the anti-psychosis drug, pimavanserin, had failed in a trial. Acadia’s announcement also was a blow to Biovail, the Toronto-based collaborator that had provided $30 million in funding to help develop pimavanserin for Parkinson’s patients.

—CareFusion, a San Diego medical technology company spun off by Ohio-based Cardinal Health (NYSE: CAH), officially became an independent company with shares traded on the New York Stock Exchange (NYSE: CFN). CareFusion’s business, which includes technologies created by San Diego’s Pyxis and Alaris, has about 15,000 employees and $3.7 billion in annual sales. The company’s stock was included in the S&P 500 index.

—Carlsbad, CA-based Medsphere Systems says it raised $12 million in venture funding to expand sales of its OpenVista electronic health record technology. The healthcare IT company’s announcement was among several funding deals in the San Diego region, according to a series of regulatory filings with the Securities and Exchange Commission. Those deals included:

—Aubrey, the Carlsbad, CA-based developer of advanced biosynthetic dressings for treating wounds and burns, raised nearly $3.9 million from investors and wants to raise another $2.1 million.

—Animal Cell Therapies, the San Diego company developing methods to produce and store animal stem cells, has raised $1.1 million of a planned …Next Page »

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San Diego-based Acadia Pharmaceuticals stock price collapsed today on news that its drug for psychosis related to Parkinson’s disease failed in a late-stage clinical trial. It’s a big setback for the small firm because it has no products on the market, and the Parkinson’s drug was the candidate furthest along in its development pipeline.

The company (NASDAQ:ACAD) reported this morning that its experimental drug, pimavanserin, was unable to significantly reduce the number of hallucinations and delusions associated with Parkinson’s disease when compared with a placebo in a study of 298 patients. While two groups in the trial that were treated with pimavanserin showed a reduction in psychotic events, there was also an “unexpected” reduction in such occurrences in the patients who took placebo, according to the company. Acadia’s stock price lost more than two-thirds of its value on the news, and shares traded as low as $1.87 just before noon Eastern time.

As Denise wrote back in May, Acadia had been hoping to silence its critics on Wall Street by reporting positive results in this trial. Also, this clinical trials setback is a blow to Parkinson’s disease patients because there are no approved treatments for psychosis related to their illness. Parkinson’s is a chronic neurodegenerative disease that affects about 1.5 million people in the U.S., according to the American Parkinson Disease Association, and an estimated 40 percent of patients suffer from psychosis. This is also bad news for Acadia’s collaborator Biovail, the Canadian biotech firm that forked over $30 million upfront this spring as part of deal to develop pimavanserin for Parkinson’s patients.

Still, Acadia is awaiting results of a second late-stage clinical trial of pimavanserin and is also developing the drug to treat Alzheimer’s disease, according to Alan Carr, a biotech analyst at investment banking and research firm Needham & Company in New York. “While we expect the stock to trade at… $1.78 [per] share … for the foreseeable future, we believe there is modest long-term potential for pimavanserin in another [central nervous system] indication,” Carr wrote in a note to investors this morning. His firm is maintaining a “hold” rating for Acadia’s stock.

Acadia has other drugs in earlier stages of clinical development, including an experimental treatment for chronic pain and another for glaucoma in partnership with the California-based healthcare products firm Allergan (NYSE:AGN). And at least as of May, when Denise last covered Acadia, the firm had enough cash in the bank to support operations into 2011.

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Resolvyx Pharmaceuticals, the company that develops derivatives from omega-3 fish oils into new drugs, is announcing today that the first therapy from this class passed its first clinical trial, helping alleviate symptoms for patients with chronic dry eye.

This was a big hurdle for Bedford, MA-based Resolvyx to clear, partly because the company set the initial bar unusually high. Most initial clinical trials enroll only a dozen or so patients, assess safety at a number of low doses, then start thinking about effectiveness. But Resolvyx cut to the chase. It is reporting today that its proprietary eye drop was able to achieve a statistically significant reduction in the dryness, stinging, burning, and grittiness sensations in a trial of 232 patients who were randomly assigned to get the drug or a placebo. There were no serious side effects.

This is certainly a big step for Resolvyx, because it provides some validation to the company’s claim that its class of compounds, called resolvins, can have potent anti-inflammatory effects. Resolvyx plans to develop oral pill formulas that can be used broadly in common inflammatory diseases like asthma or inflammatory bowel disease. While dry eye doesn’t sound nearly as serious as those conditions, it is one of the most common eye conditions treated by doctors, and is estimated to affect 25 million to 30 million people in the U.S..

“There is an urgent need for new treatment options in dry eye and the results of this Phase 2 study are as strong as any I have seen,” said Stephen Pflugfelder, an expert in dry eye at Baylor College of Medicine, in a company statement. “Based both on these clinical results and on its unique mode of action, I am confident that RX-10045 can be an important new treatment.”

Full details haven’t yet been presented in a peer-reviewed journal, although the results are expected to be presented at the Association for Research in Vision and Opthalmology meeting next year, the company says.

For the biostatistical sticklers out there, don’t worry, I had a chance to comb through the details with Resolvyx’s chief operating officer James Nichols. But for those who just want the gist, here goes: Resolvyx can’t go quite so far as to say this was an absolute grand slam, but the results are certainly good enough to go ahead with final stage clinical trials, estimated to begin in the first half of 2010. It’s also enticing enough that Resolvyx will now hit the fundraising trail to seek another round from investors, and discuss partnership possibilities with big drugmakers, Nichols says.

So what about the details has gotten Resolvyx so excited? The trial looked at …Next Page »

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Rib-X Pharmaceuticals, the New Haven, CT-based developer of novel antibiotics, is announcing today that one of its experimental treatments passed a mid-stage clinical trial for patients with a common form of pneumonia. It’s looking to deal with a Big Pharma company with the money and resources to take the drug through the next steps needed for FDA approval.

The study looked at 160 patients with mild to moderate community-acquired pneumonia, who were randomly assigned to get one of three different doses of Rib-X’s oral pill version of radezolid (pronounced ruh-DAY-zo-lid). Researchers found that 78 to 92 percent of patients were considered clinically cured, while the most common side effect was a mild irritation in the intestines.

The Rib-X antibiotic was designed with the help of a proprietary computer system that identified points of vulnerability in bacteria, and ways in which they develop resistance to existing drugs, as Ryan reported in October. The company’s goal is to create two versions of radezolid, an oral and intravenous form, so that physicians can use them to treat a wide-range of bacterial invaders, both among hospitalized patients and those who can take an oral pill at home. If Rib-X can navigate the final stage of clinical trials to prove both of these versions are effective, it will be in position to compete with one of Pfizer’s fast-growing products, linezolid (Zyvox), which generated $1.1 billion in sales last year.

“We feel this is very promising data,” says Susan Froshauer, Rib-X’s CEO. “We’re not seeing the kind of bone marrow suppression that you see with other treatments in the class.”

Still, the data is from a mid-stage study, so it’s too early to conclude that this drug will be a winner. The point was to assess the lowest possible dose at which Rib-X could see an anti-bacterial effect, Froshauer says. Researchers saw comparable effectiveness at all three doses tested, …Next Page »

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Shire, the U.K.-based specialty pharmaceutical company, had some good news this morning for patients with Gaucher’s disease that might not be nearly as good for its competitor, Cambridge, MA-based Genzyme (NASDAQ: GENZ).

Shire said today that its experimental treatment for Gaucher’s, a rare genetic disorder, passed a key clinical trial in 25 patients. This is the first of three pivotal trials Shire is conducting, so this news might have gotten little notice except for one thing. The data is good enough that Shire persuaded the FDA to allow doctors to start prescribing the new treatment, velaglucerase alfa, before it’s ready for commercialization. Shire said it will provide free drug for patients who enroll in a special protocol.

This move clearly puts some additional heat on Genzyme, which is dealing with a shortage of its best-selling product, imiglucerase (Cerezyme), the market-leading enzyme replacement therapy for Gaucher’s. Genzyme’s Allston, MA-based manufacturing plant was plagued by a viral contamination in June, and the company shut the facility down for sanitizing, which created a shortage of Cerezyme production expected to last 6-8 weeks. This has created a serious concern for patients, who need to stay on steady medication to prevent the buildup of a fatty substance in their spleen, liver, lungs, and bones that can interfere with the function of those organs and lead to extreme fatigue, and death.

Other competitors besides Shire, like Switzerland-based Actelion, have sought to fill the void in the market during the Cerezyme shortage. Genzyme said in its second-quarter financial report that its sales of Cerezyme this year will likely be between $750 million to $1 billion, down from its previous forecast of $1.25 billion to $1.275 billion.

“With Allston supply issues front and center, it would be hard to imagine worse timing for Genzyme,” to face new competition from Shire, said analyst Christopher Raymond of Robert W. Baird & Co, in a note to clients today. This could erode Genzyme’s market share, especially if shortages continue, he said. “That said, we remain confident in Genzyme’s ability to bring Allston back up in a timely fashion.”

Genzyme shares fell 2 percent to $50.62 at 11:05 am Eastern.

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Oncothyreon (NASDAQ: ONTY), the Seattle-based developer of cancer treatments, said today its Stimuvax therapy showed a long-lasting response and was well-tolerated in a long-term follow-up study. Ten of the 16 patients with non-small cell lung cancer, who were followed two to 8.2 years, were still alive as of April with no evidence of their disease spreading after they got Stimuvax. The treatment, being co-developed with Germany-based Merck KGaA, was well-tolerated during long-term use, researchers said. Data were presented Saturday at the World Conference on Lung Cancer in San Francisco.

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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.

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Several of San Diego’s biotech and life sciences companies raised money this past week, while others await the result of studies to see if the money they’ve already invested will pay off.

—Cytori Therapeutics (NASDAQ: CYTX), which is developing a device that can isolate and process regenerative cells from a patient’s fat tissue, has been bulking up its cash position to deal with the recession, Denise reported yesterday. After raising $15 million since March, and with a recent deal with Seaside 88 in June, San Diego-based Cytori will be viable at least through the end of next year. However, after losing the rights to a patent to produce stem cells from fat cells, the company is still a little shaky financially. Luckily for Cytori, this does not affect any of its current developments. The company’s fat isolation and regeneration device is already approved in Europe and is being tested for breast reconstruction after mastectomies as well as for helping blood flow in patients with angina.

—San Diego-based aTyr Pharma, which develops medical therapeutics from proteins, raised $12 million in venture capital to further its research and development, as I reported on Tuesday. The protein drugs aTyr hopes to make come from a group of enzymes that perform catalyzing reactions in the body, known as aminoacyl tRNA synthetases. The drugs could eventually treat a variety of ailments, including autoimmune diseases, metabolic disorders, and blood conditions. While some of the research is done in San Diego, the rest is through Pangu BioPharma, a majority-owned subsidiary at the Hong Kong University of Science and Technology.

—Bruce reported on Tuesday that Helicon Therapeutics has raised $50 million from undisclosed investors to develop memory enhancing drugs. Helicon, which is more than 10 years old although fairly new on the San Diego scene, is working on a gene called CREB to make drugs that will better convert short-term memories into long-term memories. The company is planning its second trial of one of its most recently developed drugs this year.

—Luke profiled Regulus Therapeutics, a company working to develop a new class of drugs based on microRNA technology. Regulus, which came out of Cambridge, MA-based Alnylam Pharmaceuticals (NASDAQ: ALNY) and Carlsbad, CA-based Isis Pharmaceuticals (NASDAQ: ISIS), started off strong, and despite financing worries, is continuing to grow. The research at Carlsbad-based Regulus has previously shown the effectiveness of microRNA in stopping heart failure in animals, while a recent study showed how a different type of microRNA seems to limit the spread of Hepatitis C.

—Good news may be coming for investors in Avanir Pharmaceuticals (NASDAQ: AVNR), as Denise reported, when the results of a trial for a drug to control involuntary laughter and tears are released. The drug, dextromethorphan/quinidine (also referred to as DMQ), treats a neurological condition sometimes known as pseudobulbar, but after trials three years ago showed negative cardiac side effects, Avanir took the drug back to the lab for reworking. Originally based in San Diego, Avanir is now located in Aliso Viejo, CA, where the results of the trial of the reformulated drug will be released. If the trial is successful, the company will begin marketing the drug under the name Zenvia.

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Ariad Pharmaceuticals, the Cambridge, MA-based developer of cancer drugs, said today that its lead experimental drug is likely to succeed in an ongoing mid-stage trial of the drug as a treatment for breast cancer. Shares of Ariad (NASDAQ: ARIA) shot up 29 percent, to $3.15 after the news crossed the wire.

The Ariad drug, ridaforolimus, was given in combination with the standard trastuzumab (Herceptin) treatment to women with an overactive form of the Her-2 gene whose cancer had developed resistance to trastuzumab alone. The trial needed to show tumor shrinkage for at least five patients (15 percent of the 33 patients planned to be included in the study) and it has reached that threshold after the first 28 patients have enrolled, Ariad said. The company didn’t describe the safety data obtained in the study, but said no new or unexpected safety concerns were observed. Abstracts describing more complete data have been submitted to major medical meetings later in the year.

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Avanir Pharmaceuticals (NASDAQ: AVNR) has been working for so long on a drug for an odd neurological syndrome that triggers involuntary laughter and tears, it’s almost enough to make some investors cry. Soon they will learn if the wait has been worth it.

In the next few weeks, the onetime San Diego biotech now just over the Orange County border in Aliso Viejo, CA, will announce results of a late-stage trial of a reformulated version of its experimental drug dextromethorphan/quinidine, or DMQ, to be marketed as Zenvia.

The drug’s original formulation reduced unwanted laughter and tears in neurological patients, but carried worrisome cardiac side effects and caused nausea and dizziness—a potential hazard in people with conditions that also affect balance, such as amyotrophic lateral sclerosis (Lou Gehrig’s disease). When the FDA raised concerns, Avanir agreed to develop and test a new version of the oral drug. That was three years ago.

“It has been a long road by our team,” CEO Keith Katkin tells me.

It could get longer still as Avanir begins to create a market for DMQ. Doctors may not recognize emotional outbursts in patients with neurological diseases or injuries as a behavioral syndrome with a potential treatment. Avanir calls the odd behaviors pseudobulbar affect, or PBA, but that name may not be familiar even to those who suffer from it. It isn’t described in the copies of the Merck Manual or Diagnostic and Statistical Manual of Mental Disorders I consulted to try to learn more about PBA. When I googled PBA, I didn’t immediately find pseudobulbar affect but pulled up numerous references to some better known PBAs, such as the Professional Bowlers Association.

Daniel Wynn, a Chicago neurologist who was involved in clinical trials for DMQ, tells me the symptoms of PBA go by different names—but the disorder is real, regardless of what it is called. He says there are parallels between PBA and overactive bladder or erectile dysfunction. There is little doubt that those conditions are real, he says, yet no one really used those terms until drugs came along to treat the disorders. Involuntary crying or laughing is distressing to patients, he says, who may withdraw from social situations or quit work because they can’t keep their reactions under wraps.

“Imagine if you shared an office with someone and you couldn’t stop crying,” Wynn says.

Still, there’s no question Avanir will need to do some serious marketing …Next Page »

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San Diego-based Orexigen Therapeutics dumped a boatload of information in the public domain this morning about clinical trials of its experimental obesity drug. Investors have been in a wait-and-see mode about this drug for months after arguing over the merits of one big trial, but they reacted positively to this new batch of data, sending the stock up 16 percent to $6.62 just after the opening bell.

Orexigen (NASDAQ: OREX) said today that all three of the big clinical trials of the drug in obese patients reached their goals in showing that the treatment helped people lose more weight than a placebo. The company also said that it found encouraging signs on secondary goals, like reducing cholesterol, and blood sugar levels, and helping patients develop slimmer waists. There wasn’t much in the initial announcement about side effects, although Orexigen said nausea was the most common side effect that caused patients to drop out of the studies, and that seven out of 4,500 patients in the trials had severe side effects that may have been related to the drug, including gallbladder inflammation, seizure, heart palpitations, and vertigo.

The drug, a longer-lasting form of two generic treatments known as buproprion and naltrexone, has now cleared enough hurdles that Orexigen plans to send off an application to the FDA in the first half of 2010 to start marketing it in the U.S. as Contrave. If the FDA agrees the drug is safe enough to reach the market, then it has a shot to tap into a market surely worth multi-billions in the U.S. Obesity rates in the U.S. have been skyrocketing for years, as a result of the deadly combo of the sedentary lifestyle and junk-food culture. About two-thirds of U.S. adults are now considered overweight or obese, putting them at risk for a litany of conditions such as heart disease, diabetes, high blood pressure, and arthritis. The cost to society of all this unhealthy weight gain is hard to measure because it’s intertwined with so many diseases, but a U.S. Surgeon General’s report in 2000 fingered it as responsible for about 9 percent of national healthcare spending.

“The results demonstrate the potential for Contrave to help patients in their battle against obesity,” said Mike Narachi, president and CEO of Orexigen, in a statement.

Before breathless commentators start turning too many cartwheels though, it’s worth a closer look at the data to see what kind of magnitude of weight loss researchers saw with Orexigen’s drug.

The FDA has given guidance to drugmakers that it typically likes to see …Next Page »

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Vical (NASDAQ:VICL) says early results from a mid-stage trial of its cytomegalovirus vaccine look promising. The San Diego-based company says that at four months, 30 percent of treated patients had infections compared to 50 percent in the untreated group. Viral load measurements and other data all favor the vaccinated group, according to Vical, which did not provide a statistical analysis of the results. Although complete results from the study are due in early 2010, Vical says it is encouraged enough to plan its late-stage trial of the vaccine and talk with potential development partners. But investors seem unimpressed. Vical shares dipped 23 cents, or almost 9 percent, to $2.44 in afternoon trading.

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