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Geron announced today that the FDA has lifted a clinical hold placed on the company's Investigational New Drug (IND) application, allowing the Phase I clinical trial of GRNOPC1 in patients with acute spinal cord injury to proceed. Geron release

Results from COR-I, one of the Phase III trials supporting the NDA for Orexigen Therapeutics' (NASDAQ: OREX) weight-loss drug Contrave, show that patients taking the drug were two to three times more likely to lose at least 5 or 10 percent of their body weight compared with those taking placebo. The study results have been published online in the journal Lancet.  

Treatment with Contrave also resulted in significant improvements versus placebo in waist circumference, insulin resistance, HDL cholesterol, triglycerides and hsCRP, which are accepted measures of cardiometabolic risk. Patients taking Contrave also showed significant improvements in patient-reported control of eating, the company says in a statement.

But, as Reuters points out, only half the volunteers finished the trial, dropping out for various reasons. "The people in the placebo group seemed to drop out more frequently because they were dissatisfied with the lack of weight loss. People in the drug arm seemed to drop out more because of side-effects. Overall, the groups seemed to be equal," Frank Greenway, who led the trial, tells Reuters. The most frequent adverse event in participants taking Contrave was nausea. Headache, constipation, dizziness, vomiting and dry mouth were also more frequent in those taking the drug than in the placebo group, according to the study results.

Orexigen is one of three company trying to get weight-loss drugs to the market. Earlier this month, an expert panel recommended against approval of Vivus' (NASDAQ: VVUS) competing drug Qnexa. The vote was six in favor and ten against, with many of the no voters citing the need for more safety data.  As Bloomberg points out, some of the panelists' safety concerns have plagued other weight-loss drugs. For example, Abbott Laboratories pulled Meridia from the European market over concerns about heart risks in January.

Orexigen's other rival, Arena Pharmaceuticals (NASDAQ: ARNA), recently released promising data for its own obesity drug, lorcaserin. Results showed that just about half of the patients taking the drug in a two-year trial lost at least five percent of their body weight--roughly twice the rate of weight loss registered in the placebo group.

The FDA has tentatively scheduled a Division of Metabolic and Endocrine Drug Products Advisory Committee meeting for Dec. 7, and the Prescription Drug User Fee Act action date has been set for Jan. 31, 2011. 

- see the Orexigen release
- here is a summary of the study in the Lancet
- read the Reuters story
- get more from Bloomberg

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Three biotechs race for Holy Grail of drug development

Sanofi CEO Chris Viebacher (photo) wants his company to avoid the peaks and valleys that come with blockbuster drugs such as Lovenox, a $2 billion-a-year anti-clotting drug that's now facing generic competition. Instead, he's looking at emerging markets, consumer health products and generic medicines to help boost revenue, as well as focusing more on deals for drugs aimed at smaller patient groups. That may help explain Sanofi's interest in Genzyme--a biotech company that makes treatments for a variety of rare diseases. Such drugs are rarely susceptible to generic competition. "Above all, what I'm looking for is businesses that are not dependent on patents," Viehbacher told the AP in an interview. "This is my fourth patent cliff in my career and I'm looking to avoid a fifth."

- read the AP report for more

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Susan Molineaux (photo) helped guide cancer drug developer Proteolix from early stage multiple myeloma research through Phase II trials. The developer was snapped up by Onyx Pharmaceuticals, which last year paid $276 million up front for the company, leaving Molineaux free to concentrate on a new project. "I'm a risk taker," Molineaux tells the San Francisco Business Times. "There's probably no super-safe program I've ever been associated with."

The entrepreneur has now turned her attention to South San Francisco-based Calithera Biosciences, which earlier this month landed a $40 million Series A round to ramp up development work on a new group of cancer drugs discovered in the lab of Jim Wells, chair of the Department of Pharmaceutical Chemistry in the University of California, San Francisco School of Pharmacy. Wells was impressed by Proteolix's ability to find the correct dosing, formulation and potency for its cancer drug--a challenge he suspects Calithera will face in its work. Wells' compounds activate procaspases and trigger apoptosis (cell death) in cancer cells.

Wells and Chris Christoffersen, managing partner at Morgenthaler Ventures (one of Calithera's VC backers), agreed that Molineaux was the right choice to head up Calithera. They were impressed with her ability to bridge the gap between science and business. During Molineaux's interview for the CEO spot, "we talked serious science and candidly and openly with great strategic clarity about businesses she's been involved with," Christoffersen tells the San Francisco Business Times. "I walked out of the interview saying, 'That's impressive.'"

- here's the San Francisco Business Times article

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Shareholder opposition has forced Charles River to call off its $1.6 billion plan to purchase China's WuXi PharmaTech. Despite CEO James Foster's assurances that the deal would bring up to $100 million in potential operational savings, several investors were never able to overcome their concerns that the deal wasn't in the company's best interests. Charles River has been expanding its Chinese operations for the past several years and had hoped to increase the size of its footprint in China with the WuXi deal.

Investors Jana Partners, Relational Investors and Neuberger Berman were among the firms concerned that Charles River was overpaying for the Chinese CRO, and that the company would face significant challenges integrating the two businesses. The termination agreement provides for Charles River to pay WuXi a $30 million breakup fee. Additionally, the company announced that its board of directors has authorized the repurchase of up to $500 million of Charles River stock.

"We believed that this transaction, which would have created the premier early-stage contract research organization, would have resulted in long-term strategic benefits for our business and our shareholders," said James in a statement. "We also value our stockholders' views and given their concerns about the proposed transaction, and our commitment not to proceed without their support, we have decided that terminating the transaction is the appropriate action to take."

- see Charles River's release
- here's the WSJ report

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GlaxoSmithKline (NYSE: GSK) has obtained an exclusive licence to develop and commercialize an antibiotic derived from Anacor's boron chemistry platform. As a result, Anacor will receive $15 million and is eligible for further milestone payments and royalties on any future product sales. GSK is assuming responsibility for further development of the compound and any resulting commercialization.

In early stage studies, GSK2251052 (GSK '052) has shown robust activity against multi-resistant gram-negative bacteria with no cross resistance to existing classes of antibiotics. GSK '052 is being looked at as a potential treatment for complicated urinary tract infection, complicated intra-abdominal infections and hospital/ventilator-associated pneumonia (HAP/VAP).

"GSK '052 has an entirely novel mechanism of action with the potential to be the first new class antibacterial to treat serious hospital gram-negative infections in 30 years," says David Payne, VP of GSK's anti-bacterial drug discovery unit. "Our collaboration with Anacor has enabled the rapid progression of GSK '052, and we are excited about the opportunity to address the growing need for new treatments for serious hospital acquired infections."

GSK and Anacor entered into a worldwide strategic alliance for the discovery, development and commercialization of novel medicines for viral and bacterial diseases in October 2007. The alliance grants GSK access to Anacor's proprietary boron-based chemistry for use in four target-based project areas. Contingent on achieving certain milestones, Anacor is eligible to receive development and regulatory milestone payments of up to $84 million as well as commercial milestones and tiered double-digit royalties up to the mid-teens, which are dependent on sales achieved.

- read the Anacor release

ALSO: Colgate-Palmolive hasasked a federal court to rule that its new "Triple Action" toothpaste doesn't infringe trademarks of GSK's competing product, Aquafresh. Report

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In what's described as an early-stage clinical trial, Hospira (NYSE: HSP) has unveiled plans to take its biosimilar of Amgen's anemia drug Epogen directly into a head-to-head study designed to swiftly pivot into a large, late-stage study.

The Chicago-based company plans to recruit hemodialysis patients at 20 different centers across the U.S. But unlike a typical Phase I study, researchers will immediately study both safety and efficacy of the therapy, Retacrit, in comparison with the market-leading drug. If it's successful, the study will vault into a Phase III trial next year.

"This trial marks a key milestone for Hospira's biosimilars program," says Sumant Ramachandra, a senior vice president at Hospira. "Congressional approval of a regulatory pathway for biosimilar drugs in the U.S. set the stage for our development of a U.S. biosimilar EPO. We hope to leverage our leadership in generics and our biosimilars experience in Europe to make affordable, safe and effective biosimilars available to U.S. patients and their healthcare providers once patents expire over the next several years."

Retacrit is already available in Europe, where it now has a more than 50 percent share of the "total short-acting EPO biosimilar market." This study illustrates the new approach that developers will take on clinical trials for biosimilars. Jumping past much of the early- and mid-stage research required for new chemical entities, companies can move quickly into Phase III, which requires deep pockets.

- here's the press release
- here's the story from the Chicago Tribune

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Athersys (ATHX) has benefited from some upbeat buzz about its positive--though small--early-stage results for a prospective new stem cell therapy for victims of a heart attack. Patients who received a medium and high dose of its MultiStem therapy--a stem cell treatment derived from patients' bone marrow cells--demonstrated significantly improved heart function in a Phase I trial. A total of 19 patients were treated with the experimental therapy.

"We are encouraged by the results of this Phase I study, and based on these results, will work with our partner, Angiotech Pharmaceuticals, on plans for a phase II trial to further evaluate safety and assess improvement in cardiac function" said Athersys CEO Gil Van Bokkelen. "This study is an important step and provides additional validation of the clinical potential and therapeutic profile of MultiStem as an off-the-shelf, allogeneic stem cell therapy, and we look forward to building off these results."

That sounded pretty good to investors, who drove the stock up 18 percent on the news. Late last year Pfizer inked a $111 million deal to license rights to develop Athersys' cell therapy for inflammatory bowel disease. But only $6 million of that was paid upfront.

- here's the Athersys release
- read the story from MedCity News
- check out the Dow Jones article

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Shares of Dendreon (NASDAQ: DNDN) soared into the stratosphere in the lead-up to Provenge's approval--and then promptly started tumbling fast as all sorts of questions arose about supply issues, cost and efficacy. But the company is likely to benefit enormously from the publication of its biggest study of the innovative prostate cancer vaccine in the prestigious New England Journal of Medicine, which carries biblical weight with providers. Story

Faced with a rising tide of red ink, Vertex (NASDAQ: VRTX) says it has begun rolling out its NDA for the potential blockbuster telaprevir and expects to wrap the app later this year. Banking on a treasure trove of positive Phase III results already in hand, Vertex is clearly hopeful about its chances with the closely watched hepatitis C drug. And it's laying the groundwork for another FDA campaign next year for its cystic fibrosis program.

Vertex will need to start ginning revenue quickly, assuming it wins an approval for telaprevir. The company projects a $750 million loss for 2010--significantly more than earlier estimates.

"In the first half of this year, we established a seasoned commercial leadership team with broad experience in the area of infectious diseases, and we continue to bolster the internal infrastructure needed to support a field-based sales force for telaprevir and other potential future medicines," says Vertex CEO Matthew Emmens in a statement that lays out the biotech's development strategy.

"Our Phase III registration program in cystic fibrosis is now fully enrolled, positioning us for the planned submission of a New Drug Application for VX-770 in the second half of 2011. There is an urgent need for new and more effective therapies in cystic fibrosis, and we are committed to working toward improving the lives of people affected by this disease."

- here's the press release on Vertex's pipeline strategy
- here's the story from Reuters

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Genzyme (GENZ) is in play. After days of fevered speculation over what Genzyme would be worth to a company like Sanofi-Aventis, the big pharma outfit appears to have set its sights on the low side of the spectrum. According to a flurry of news reports, Sanofi CEO Chris Viehbacher and the board think that $70 a share--that's $18.7 billion total--would be a fair price to pay. But any bidding could start under that level. 

Significantly, Sanofi appears ready to move past the discussion stage and lay its cards out on the table in a formally presented letter which spells out its terms, Bloomberg reports, quoting "three people with knowledge of the situation." The New York Times, meanwhile, said that the pharma company would likely issue a "bear hug" letter, indicating it would like to keep the bidding process friendly but would go hostile if necessary - a ploy that could help win over some of the board members who have been on the fence in recent days.

Genzyme, though, isn't likely to leap at any $70 bid that comes its way. Market analysts have been quick to target $80 as a good premium over the current trade, which has soared 30 percent on the back of all the rumors that have been floating around.

"My guess is that Genzyme would turn them down," Cowen & Co's Phil Nadeau told the business news wire, "do some negotiations, and the price would go up a little bit, maybe to $73 or $75."  

True, Genzyme has been damaged by its recent manufacturing snafus, but that's unlikely to intimidate a player like Sanofi, which could use Genzyme's storehouse of biotech knowledge. And a rich M&A deal could be just the way Genzyme CEO Henri Termeer would like to cap his long-running career.

While Sanofi's board has been leaking like an old wooden ship, Viehbacher publicly is keeping a tight lip on the deal. "We are under no pressure to do a deal," Viehbacher told analysts on a call, vowing to stay disciplined on its M&A strategy.

- here's the story from Bloomberg
- here's the report from the New York Times
- here's the report from Reuters

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AstraZeneca scored a big win in front of an FDA advisory panel on Wednesday afternoon, gaining solid support for its blockbuster blood thinner Brilinta in a seven-to-one vote in favor of approval.

AstraZeneca's biggest obstacle was the relatively small contingent of 1,413 American patients in a large international Phase III who failed to benefit from the drug. But the 17,000 patients who participated in the study in 42 other countries demonstrated a sharp drop in deaths and heart attacks, leading the panelists to conclude that the anti-clotting therapy should be effective in a broad patient population.

"The overall results are so striking," said the University of Minnesota's James Neaton, who supported the application. Neaton, though, also reflected some general anxiety about the lack of U.S.-related efficacy data, which AstraZeneca has attempted to blame on a regular use of aspirin among the patients recruited in this country. "We are confident in the [effectiveness] of ticagrelor when used with low-dose aspirin," said AstraZeneca's Jonathan Fox, according to a report by Dow Jones.

The vote puts AstraZeneca on the path to full FDA approval--something that is likely given the lopsided vote in Brilinta's favor. If it does win full approval later this year, analysts project that the drug will quickly rack up more than a billion dollars in sales during its first year and has the potential to hit mega-blockbuster status with $4 billion in peak annual sales. With its patent on Nexium set to expire soon, a big regulatory win here is just what the pharma company needs most.

- here's the story from Reuters
- here's the report from Dow Jones

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Shares of Antigenics (AGEN) got some much-needed help after the developer reported that its vaccine/adjuvant combo to guard against genital herpes looked promising in a small, early-stage trial.

The Lexington, MA-based biotech recruited 35 patients for the Phase I study, dividing them into four different groups. One group got the vaccine AG-707 and Antigenics' adjuvant with the other three groups getting either the vaccine alone, the adjuvant alone or a placebo. The biotech noted that all seven patients who were evaluable for immune response and received AG-707 with the QS-21 adjuvant showed a statistically significant CD4+ T cell response.

"I believe these data represent the first finding of their kind in genital herpes treatments-showing a vaccine, AG-707, elicits both CD4+ and CD8+ T-cell responses in humans," said David Koelle, study investigator and professor of Medicine, Laboratory Medicine and Global Health Medicine at the University of Washington. "We are very encouraged by these results. Recent data suggest both of these arms of immunity are needed for successful treatment of genital herpes."

Investors liked the sound of that, pushing Antigenics' shares up about 20 percent to near the $1 mark. Antigenics, though, has had a tough time gaining much market respect, with its cancer vaccine Oncophage experiencing rough treatment at the hands of regulators outside of Russia.

- here's the Antigenics release
- read the story from the Boston Business Journal

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Over the last two years the EMA and the FDA have approved a total of 39 new drugs. But 11 of those drugs are only available in Europe, where regulators also scored significantly faster approval times, shaving 97 days off the FDA average. And those stats spurred a think tank called the Pacific Research Institute to suggest that the U.S. would be far better off if it allows pharma companies the right to go ahead and start marketing drugs here once they get an approval over there.

"Clearly, Congress's grant of a regulatory monopoly to the FDA is creating a significant obstacle to Americans' timely access to new medicines," the institute says. The think tank argues it would like to get some healthy regulatory competition in play and probably save lives to boot. The Pacific Research Institute also thinks that Rep. Diane Watson's proposal to make experimental meds available to very sick patients with no other options is a tonic solution, notes The Hill.

It's a long shot at best, though, that this idea will ever get any kind of serious consideration. Lawmakers have shown little appetite for getting the FDA to actually hustle on approvals. Europe's reputation for faster, more aggressive regulatory action is likely to remain unchallenged, with the FDA's regulatory "monopoly" likely to continue indefinitely.

- read the report from the Pacific Research Institute
- here's the story from The Hill

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After first getting unexpectedly stiff-armed by the FDA, Merck KGaA says that its app for the oral MS therapy cladribine has now been granted a priority review, putting it on track for a fourth-quarter decision. The German pharma company has been trailing Novartis recently in the race to the market with the world's first oral MS drug. An expert panel has already voted to endorse Novartis' Gilenia. Story

With an eye on the growing market for genetic lab products, Illumina today unveiled a deal to buy Helixis for up to $105 million. The genetic analysis equipment maker will now gain control of Helixis' real-time polymerase chain reaction system, which can be used to track the efficacy of cancer drugs or provide biologists with a unique insight on DNA activity.

Illumina, which is paying $70 million up front and $35 million more based on certain contingencies, has some tough competition in the field. According to the San Diego Union-Tribune, the acquisition will make Illumina more competitive with Life Technologies and others in the market.

Xconomy explains that Helixis got started with technology hatched at CalTech, developing a new PCR technology that's conveniently sized to sit on a biologist's lab bench while costing only a fraction of the competing products now on the market. Helixis started selling its new technology last year and had already lured Illumina CEO Jay Flatley to the board.

"We are excited to launch the Eco Real-Time PCR System, which we believe will set new standards for performance, simplicity and affordability," said Christian Henry, senior vice president and general manager of Illumina's life sciences division.

- check out the Illumina release
- here's the Xconomy report
- see the story from the San Diego Union-Tribune

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The first half of 2010 has drawn to a close, so it's time to look back on the biggest biotech venture capital deals so far this year. At the halfway point the leader is Archimedes Pharma, and England-based developer that has raised $100 million to support the planned roll-out of PecFent, a fentanyl nasal spray designed to treat breakthrough cancer pain. Overall, the biotech industry has raised $2.1 billion in 238 deals through the end of June 2010, according to the National Venture Capital Association. See the top 5 >>

Ambit Biosciences has completed a thorough executive search and appointed Alan Lewis as chairman and CEO of the company. Release

Ablynx Chief Scientific Officer Debbie Law has resigned from her position at Ablynx. Release

iBio has named Terence (Terry) Ryan as senior VP, business development. Release

Roche has announced that Karen Lackey has been named VP and head of medicinal chemistry, effective immediately. Release

Gentris has appointed Karen Weck as a consulting medical director. Dr. Weck replaces the previous medical director to continue Gentris' CLIA program and help expand opportunities to develop new biomarkers. Release

NexMed has announced that Mohamed Hachicha has been appointed to the position of VP, R&D for NexMed USA, effective immediately. Release

Celsion has announced the appointment of Sid Taubenfeld as director of communications and strategic initiatives, effective immediately. Release

Patrick Zenner has been elected to Par Pharmaceutical's board of directors. Zenner was also appointed to the compensation and management development committee and the corporate development review committee. Release

Dynavax Technologies has appointed Daniel Kisner to its board of directors. The addition of Kisner to the Dynavax board brings its membership to ten. Release

Alexza Pharmaceuticals has named Joseph Turner to its board. Release

BioVex has named Kapil Dhingra to its board of directors as a non-executive. Release

Nile Therapeutics has appointed Richard Brewer as executive chairman.  Release

Biotech IPO watchers will get a new player this week with San Diego-based Trius Therapeutics--a 2008 emerging drug developer--making its long-delayed debut on the market. Trius will attempt to sell six million shares for $12 to $14 each, worth up to $84 million. Depending on the underwriters, Trius could net anywhere from $71 million to $82 million from the offering.

A success here would attract a considerable amount of attention. The biotech company has no products on the market and is setting the stage for a Phase III study of a new antibiotic for serious gram-positive infections like the dread MRSA. That makes it more of an old-style IPO play, which depends on investors willing to take a high-risk leap of faith. So far this year, though, investors have been distinctly cool toward risk, particularly in the biotech field.

Trius plans to use more than $55 million of its IPO take for R&D purposes, with its lead product taking the lion's share of the cash. Citigroup Global Markets, Piper Jaffray & Co., Canaccord Genuity, and JMP Securities are underwriting the offering, which was delayed to accommodate new FDA rules.

Trius won an important endorsement from the Defense Threat Adjustment Agency two months ago, when the group provided a contract worth up to $29.5 million to advance new antibiotics for gram-negative bacterial pathogens.

- here's the AP report

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Regeneron's growing antibody expertise is paying off with a rich, newly renegotiated discovery deal with Japan's Astellas. The pharma company has decided to scrap its three-year-old collaboration--under which it paid Tarrytown, NY-based Regeneron $20 million a year to find promising new drug candidates--and agreed to hand over $165 million up front with a potential $130 million check due eight years from now.

Provided Astellas is happy with the first half of the deal and re-ups for the second, the discovery pact will stretch out to 2023. And Regeneron will be in line for "mid-single-digit" royalties on any products it has fed to Astellas. Regeneron (REGN), meanwhile, announced a better-than-expected quarterly loss--$25.5 million--this morning, with licensing payments from Sanofi-Aventis limiting the amount of red ink it had to report. Regeneron's revenue jumped 29 percent to about $116 million.

The developer, which has its own pipeline in play, has also been successful at striking a more ambitious discovery deal with Sanofi. The two companies decided to restructure their deal late last year, opting for a new pact that calls for four to five new clinical-stage antibody development programs each year. They now have five programs in the clinic, with a sixth NDA due on an angiogenesis drug by the end of this year. Regeneron has another discovery pact in place with AstraZeneca.

- check out the Regeneron release
- here's the story from Reuters

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