Biotech, Pharma, & VCs Offer Rare Disease Patient Groups Some Advice

Today’s issue examines the surge of interest in rare disease drugs, which in the past few years have attracted significant interest from biotech firms, big pharma, and venture capitalists alike. In addition to exploring the business and policy drivers behind increased investment in orphan drugs, the multi-part story looks at the critical role patient organizations play in drawing attention to rare diseases. As such, it seemed worth highlighting advice from various stakeholders on what patient groups can do to entice drug developers to work on their disease: —Organize yourselves. Find as many patients as possible, and establish a registry that will make it easy for a drug firm to begin a clinical trial. “Beginning to identify people, getting them into a registry, and collecting natural history data is one of the most valuable things a developer can have when they’re thinking about a program,” says Genzyme’s CEO David Meeker. “Among the most helful things that patient advocates can do is to help us to understand the natural history of disease,” agrees Kevin Lee, CSO of Pfizer’s rare disease unit. “Without that understanding of how the disease progresses, and what the endpoints can be, its almost impossible to do drug development.” –Find a way to collaborate with one another. In even the smallest of diseases, patient groups tend to proliferate. And while its natural and understandable for advocates to want to do all they can to help their own child or family member, it can lead to duplicative efforts. The disparate groups can also make it tougher for drug developers to access. “We all need to give everybody a lot of space here to do what they think is best, but in an optimal world, there are tremendous advantages to being coordinated,” Meeker says. —Be connectors. Patient organizations have the amazing ability to bring together academics who had previous not collaborated. “What I have found over and over again is that patient advocates know the investigators in their field far better than the investigators themselves do,” says Christopher Austin, director of NIH’s National Center for Advancing Translational Science (NCATS). “They can be instrumental there.” —Get the right researchers interested. Often only a handful of academic researchers are working on a given rare disease, and drug developers say attracting new scientists into the field, while also giving careful consideration about who to fund is key. Patient groups should look for someone who can use advocacy funds to attract larger grants. “If they can get some grant support, you’ll get more done,” says Emil Kakkis, CEO of Ultragenyx. “If they can’t get any grant support, you’ll have to wonder if it was just...

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Pharma & Biotech Job Cuts Mount in 2012

For those keeping track, yesterday’s layoffs at AstraZeneca add to an already substantial list of cuts in the pharma and biotech industries since the beginning of the year. By our tally, nearly 13,000 job cuts, many in R&D, have been announced so far–and we’re barely into February. Here’s where we’re at (and do let us know if we’ve missed any): –AstraZeneca is chopping 7,300 jobs, including 2,200 R&D positions, by 2014. Neuroscience research is being revamped and focused on external partnerships; the company’s Montreal R&D site will be shuttered, and research activities ended at its Södertälje site in Sweden. –Genzyme gave the pink slip to an unspecified number of R&D scientists this week. The layoffs come as Sanofi integrates its big biotech acquisition. –Alnylam is trimming 61 jobs, or 33% of its workforce, in order to save roughly $20 million this year. –BioSante Pharmaceuticals is shedding 25% of its staff, or 21 employees and contractors, after disappointing Phase III results for its female sexual dysfunction treatment LibiGel. –Takeda is axing 2,800 jobs, or 9% of its workforce, following its acquisition of Swiss drugmaker Nycomed. The bulk of the layoffs, which cut across R&D, commercial, operations, and administrative positions, will occur in Europe. –Novartis unveiled plans to shed some 1,960 positions in the U.S. as it braces for generic competition for Diovan, a blood pressure medicine that brought in more than $6 billion in 2010, and an expected drop in demand for its renin inhibitor Rasilez following questions about the drug’s safety. –Human Genome Sciences said it would cut 150 jobs, or about 14% of its workforce, in a move that affects manufacturing, R&D, and administrative activities. –Xoma is shedding 84 workers, or 34% of its staff, as it shifts to outsourcing late-stage and commercial manufacturing, as well as some research. –SkyePharma is cutting 20% of the 101 employees at its site in Muttenz, Switzerland. –Sanofi plans to layoff 100 workers at its Monteal site as part of an overhaul of its Canadian operations. –J&J will trim 126 workers as it closes its Monreal R&D...

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Genzyme R&D Layoffs Today

The other shoe has dropped at Genzyme, which last year was acquired by Sanofi, but had yet to experience the kind of major research restructuring that typically accompanies the integration of two pharma companies. Today, Genzyme scientists were told whether their job was being shed or moved. Here’s an excerpt from the official statement: As part of the integration process between Sanofi and Genzyme, R&D activities were reviewed and assessed. On January 31, 2012, the results of the review of U.S. R&D Genzyme activities were announced, including synergies that unfortunately make some positions redundant. All US R&D Genzyme employees impacted by the integration received notice regarding whether their position would be relocated or eliminated. The job cuts are separate from the latest round of R&D layoffs at Sanofi. As readers might recall, Sanofi announced last November that it was closing its Bridgewater R&D site, and move discovery and early development activities to Boston. A Sanofi spokesperson tells the Haystack that despite today’s cuts at Genzyme, the company is committed to its presence in Massachusetts, and to maintaining a stable level of jobs there. While R&D is falling under the axe, the company is hiring in manufacturing and multiple sclerosis, she says. The company has not provided details on how many R&D scientists will be shed, but once more information comes to light, we’ll update...

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Haystack 2010 Year-In-Review

This Friday, we’re looking back at 2010’s big news in pharma and biotech, both the good and the bad. Check out our picks and be sure to weigh in on what you think we missed. 1. Provenge Approved In April, Dendreon’s Provenge became the first approved cancer immunotherapy. Dendreon CEO Mitch Gold called it “the dawn of an entirely new era in medicine.” And while prostate cancer patients are excited for a new treatment option, the approval is perhaps most exciting for its potential to reignite interest in cancer immunotherapy research. There’s a lot of room for improving the approach—Provenge is, after all, expensive and highly individualized. Now that immunotherapy have been proven to work, there’s hope that the lessons learned in both its discovery and clinical development will aid scientists in inventing even better cancer vaccines. 2. Obesity Field Slims The obesity drug race played out in dramatic fashion in 2010, with three biotech companies-Vivus, Arena, and Orexigen, each making their case for its weight-loss medication before FDA. As of this writing, Orexigen’s drug Contrave seems to be on the surest footing to approval, but longtime obesity-drug watchers know that caution seems to rule the day at FDA, so nothing is a sure bet. Orexigen’s Contrave and Vivus’s Qnexa are both combinations of already-approved drugs, whereas Arena’s Lorqess is a completely new molecule. When C&EN covered the obesity race in 2009, it seemed that Lorqess (then going by the non-brand-name lorcaserin) had the cleanest safety profile, but Qnexa was best at helping patients lose weight. But FDA’s panels didn’t always play out the way folks expected. There were safety surprises- notably the worries about tumors that cropped up in rats on high doses of Lorqess, and the extensive questioning about birth defect risks from one of the ingredients in Vivus’ Qnexa. The fact that FDA’s panel voted favorably for Orexigen’s Contrave, a drug that’s thought to have some cardiovascular risks, generated discussion because FDA pulled Abbott’s Meridia, a diet drug with cardiovascular risks, from the market in October. The dust still hasn’t fully settled. Arena and Vivus received Complete Response Letters from FDA for Lorqess and Qnexa. Vivus has submitted additional documentation and a followup FDA meeting on Qnexa is happening in January. Also to come in January is the agency’s formal decision on Contrave. And if you’re interested in learning about the next wave of obesity drugs coming up in clinical trials, read this story in Nature News. 3. Sanofi & Genzyme: The Neverending Story Speaking of drama, Sanofi’s pursuit of Genzyme has been in the headlines for months now, and promises to stretch well into 2011. The...

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This Week in Pharma Job Cuts…

As we know, its tough out there for scientists working in the pharmaceutical and biotech industries. This week brought yet another spate of drug industry cutbacks that seemed worth chronicling. For those keeping track: Abbott Laboratories is getting rid of 3% of its workforce, or about 3,000 jobs, as it folds Solvay’s pharmaceuticals business into its operations. Most of the cuts will come from Europe: 300 jobs will be trimmed in Hannover, Germany; 500 in Weesp, the Netherlands; and Solvay’s U.S. headquarters in Marietta, Ga., will be shuttered. According to SEC filings, certain R&D programs will be discontinued (perhaps they can be auctioned off?). Alnylam Pharmaceuticals, a leader in RNAi technology, is shedding 25-30% of its employees after Novartis decided not to opt-in to a technology licensing clause worth $100 million at the end of a five-year pact between the companies. The biotech raked in $125 million in funding during the lifetime of the pact, and had 25 full-time employees working on projects for Novartis. An interesting tidbit from a conference call held this morning from Alnylam: According to Alnylam’s CEO John Maraganore, Novartis now has 100 people working on RNAi technology at its site in Cambridge, Mass. Clearly they made a commitment to the field with their five-year pact with Alnylam, but somehow the sheer numbers were surprising. Alnylam expects to end the year with $320 million in cash on hand. Bristol-Myers Squibb is cutting 3% of its workforce, estimated at around 800 jobs, as generic competition on its blood thinner Plavix looms. The company had already unveiled plans in 2008 to eliminate about 10% of its workforce, or about 3,700 jobs, by the end of this year. A 2007 cost-savings program had separately targeted 4,350 jobs. Genzyme said last week it would eliminate 1,000 jobs over the next 15 months as it tries to right its manufacturing operations and combat takeover attempts. Lundbeck is shedding 50 R&D jobs amid a reorganization of its R&D operations in Denmark and the U.S. The specialty pharma firm is shifting its focus to three main areas of biology– neurodegeneration, neuroinflammation and synaptic transmission—related to brain diseases, and will rely more on external partnerships. Merck, meanwhile, appears to be having a tough time making cuts in the Netherlands, where it is essentially ridding itself of Organon’s R&D operations. According to Radio Netherlands, the big pharma firm is now negotiating with the Dutch government over the thousands of jobs slated to be lost in the...

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