Affymax shocked the world when an FDA advisory committee recommended last month that its anemia drug was good enough to earn a spot on the U.S. market. Now the Palo Alto, CA-based company (NASDAQ: AFFY) is making all sorts of moves behind the scenes that will determine how big a bite it will take out of Thousand Oaks, CA-based Amgen’s multi-billion dollar anemia drug monopoly.

Decisions about the product’s price, who to hire to sell it, and how to negotiate with Medicare and dialysis clinics are all high on the list of tasks facing the Affymax executive team. It’s part of the process of getting ready for March 27, the deadline the FDA has to complete its review of peginesatide, the experimental anemia drug from Affymax and its partner, Takeda Pharmaceuticals. If Affymax can get the green light from the FDA, it will be allowed to sell its product to about 400,000 patients in the U.S. on dialysis treatment, and tap into a market that generated $2.5 billion in sales last year for Amgen’s epoetin alfa (Epogen.)

Medicare has spent tens of billions of dollars on Amgen anemia products over the years, and members of Congress at various points have been critical of how much taxpayer money goes to one company making one drug.

“I don’t want to say there’s pent-up demand, but there’s a lot of interest in the renal community,” in the new product, says Affymax CEO John Orwin. Without disclosing the price, he suggested that Affymax plans to offer a lower-cost alternative. “Healthcare providers are under a lot of pressure to find overall cost-lowering solutions. So we think we’re coming into a favorable environment.”

Given the FDA advisory panel’s lopsided 15-1 vote in favor of its drug, the odds are Affymax will soon be in position to start selling the first alternative to Amgen’s epoetin alfa (Epogen), which has had a monopoly since it was first cleared for sale in 1989. The FDA previously approved a rival from Roche called epoetin beta (Mircera) in 2007, but that drug has been blocked from the U.S. market, at least until mid-2014, because of an intellectual property dispute that Amgen won. Affymax is able to sidestep that minefield because its drug helps stimulate production of oxygen-carrying red blood cells, like Amgen’s, but it is a peptide that is chemically distinct, and works through a different biological mechanism.

Even though Affymax will likely be in position to sell its product soon, it is a classic underdog in this story. Amgen (NASDAQ: AMGN), the world’s biggest biotech company, has contracts in place to provide its anemia drug to Fresenius Medical Care and DaVita, which together control about 70 percent of the dialysis market, analyst Christopher Raymond of Robert W. Baird said in a December 28 note to clients.

Raymond rates Affymax a “buy” with a price target of $13, but he sees many obstacles ahead. Small biotechs often … Next Page »

Comments (3) | Reprints | Share:          

Cambridge, MA-based Acceleron Pharma announced today that it has raised $30 million in a private financing round. One of the participants in this round was Summit, NJ-based Celgene (NASDAQ: CELG), which has a partnership with Acceleron to develop two drugs to treat anemia.

The two companies expanded their partnership most recently in August, when Celgene granted Acceleron an up-front payment of $25 million and the opportunity to earn $217 million in milestone payments for rights to the second anemia compound. The companies are aiming to grab a piece of the $5 billion worldwide market for anemia now dominated by Thousand Oaks, CA-based Amgen (NASDAQ: AMGN).

The funding round also included contributions from Advanced Technology Ventures, Bessemer Venture Partners, Flagship Ventures, and all of Acceleron’s other existing investors. The company plans to use the proceeds to advance its pipeline, which also includes experimental compounds to treat head and neck cancer, duchenne muscular dystrophy, and bone loss.

Comments | Reprints | Share:          

Wall Street didn’t give Affymax much of a chance at success today in front of an FDA advisory committee, but the Palo Alto, CA-based company clearly proved its skeptics wrong.

Affymax (NASDAQ: AFFY) received a 15-1 positive recommendation from an expert panel of FDA advisors today, who said the benefits of its experimental anemia drug outweigh its risks to the heart. The FDA isn’t required to follow the advice of its advisory panels, although it usually does, especially when the vote is so lopsided in favor of a new product being allowed on the U.S. market.

Many investors wrote off Affymax in June 2010, as I described in a feature as a couple of weeks ago ahead of this important FDA advisory meeting. That’s because a quartet of pivotal clinical trials that enrolled 2,600 patients showed that kidney patients who aren’t yet on dialysis had an increased rate of heart-related serious adverse events—such as heart attack, stroke, congestive heart failure, and death—when they got the Affymax drug instead of the usual treatment from Amgen. About 21.6 percent of patients on the Affymax drug had those serious adverse events, compared with 17.4 percent on the Amgen drug.

But Amgen’s drug has been associated with some well-documented safety issues as well, and Affymax’s drug, peginesatide, showed comparable safety and effectiveness in trials that strictly limited enrollment to patients on kidney dialysis. When the total set of evidence was considered, FDA staff said that some of the patients on the Affymax drug may have had worse outcomes because they were sicker when they entered the studies. The FDA has a deadline of March 27 to complete its formal review of the Affymax drug, and if it gives the green light, then peginesatide will be the company’s first marketed product, and the first drug ever to directly challenge Amgen’s 22-year monopoly in treating anemia of kidney dialysis patients.

Affymax hasn’t yet settled on a pricing strategy for its anemia drug, but CEO John Orwin suggested in an interview last month that his company could be in a position to compete by offering a lower-priced, fewer-hassles, yet highly effective alternative. This will be one of the key dynamics to watch as Affymax gets closer to its FDA decision date, and it will be fascinating to see what Amgen does, if anything, to respond.

Comments | Reprints | Share:          

[Corrected: 3:48 pm, 12/7/11] Investors have had pretty low expectations about the prospects for Affymax’s anemia drug, which seeks to challenge Amgen’s 22-year monopoly in the field. But the expectations are starting to rise today, as Affymax approaches its make-or-break moment.

Palo Alto, CA-based Affymax (NASDAQ: AFFY) saw its shares rise more than 20 percent this morning after staff from the FDA posted online briefing documents in advance of an advisory committee on Wednesday. The staff comments were made about peginesatide, an experimental anemia drug Affymax and its partner Takeda Pharmaceuticals hope to market to patients on kidney dialysis. The shares climbed to $6.34, up about 20 percent, at 10:19 am Eastern time.

Many investors wrote off Affymax in June 2010, when the company announced the results from a quartet of pivotal clinical trials that enrolled 2,600 patients. The studies showed that kidney patients who aren’t yet on dialysis had an increased rate of heart-related serious adverse events, such as heart attack, stroke, congestive heart failure, and death, when they got the Affymax drug instead of the usual Amgen treatment. About 21.6 percent of patients on the Affymax drug had those serious adverse events, compared with 17.4 percent on the Amgen drug.

Affymax, however, went ahead with its application to the FDA, saying the safety profile of its drug appeared similar to the Amgen drug in the sicker population of kidney dialysis patients. Today’s FDA document raises the safety concern about the Affymax drug, but does so in a balanced way that provides some context about well-known safety issues with Amgen’s drug as well. The FDA noted that while Affymax’s drug appeared worse “numerically” in the pre-dialysis population, there wasn’t a statistically significant difference between it and the Amgen drug.

The FDA also noted that despite efforts to randomly assign patients to one drug or the other in the trial, patients who were sicker at the start ended up getting the Affymax drug, putting it at a disadvantage.

Given that context, the FDA staff said it plans to ask the advisory committee not just for its opinion about Affymax’s drug for dialysis patients, but for the potentially larger group of patients that have chronic kidney disease that doesn’t yet require dialysis.

“FDA is asking the committee’s advice about the benefit risk profile of peginesatide for patients with CKD [chronic kidney disease], given the safety findings and in light of the prior history with agents that stimulate the erythropoietin receptor,” the FDA staff said.

FDA briefing documents can often be harsh toward new products, but the early read of the Affymax documents was positive on Wall Street. “We believe the probability of approval for the dialysis indication is high and that the FDA may have left the door open to approval” in the pre-dialysis population, said William Tanner, an analyst with Lazard Capital Markets, in a note to clients this morning. His comment was reported earlier today by Bloomberg News.

The FDA’s Oncologic Drugs Advisory Committee will review the evidence presented by regulators and Affymax at a meeting on Wednesday. [Corrected FDA deadline] The FDA has a deadline of March 27 to make its decision on whether to allow the Affymax drug on the U.S. market.

Comments (2) | Reprints | Share:          

Take one look at Affymax’s stock chart and you’d never guess the most important day in its history, a classic make-or-break moment, is coming up Dec. 7. It looks like nobody is giving the company a snowball’s chance in you-know-where to succeed.

The big event coming up for Palo Alto, CA-based Affymax (NASDAQ: AFFY) will be on Dec. 7 when an advisory committee to the FDA will meet. The panel will weigh the risks and benefits of Affymax’s peginesatide as a new anemia treatment for patients on kidney dialysis. If the panel likes what it sees, and the FDA ultimately clears the product for sale by its March 27 deadline, then the company could start marketing its first drug in the U.S. If that happens, Affymax will be in position to fight for a share of a $2.5 billion dollar annual market, and it will be the first direct challenger in the U.S. to a 22-year-old monopoly held by Thousand Oaks, CA-based Amgen.

Amgen is the big guy in this story, the world’s most valuable biotech company with a market valuation of about $49 billion. Affymax, with a stock price of $4.89 at yesterday’s close, is a relative pipsqueak, worth about $175 million. Given that Affymax had about $116 million in the bank at the end of September, investors are basically saying the company’s technology is only worth a piddling $60 million—hardly an expression of confidence that it’s about to wrestle away market share from Amgen. And even though Affymax’s stock is already low at below $5, an increasing number of investors are betting that Affymax will fall further, according to data on short-selling compiled by NASDAQ.

Affymax CEO John Orwin, who started at the company in January, isn’t making any predictions, but he says the efficacy of his company’s drug is “well-established” and that his team is ready to field questions about the safety of its drug candidate. “This was the largest and most comprehensive clinical trial program ever done,” in this class of anemia drugs he says.

Affymax CEO John Orwin

Most investors wrote off Affymax in June 2010, when the company announced the results from a quartet of pivotal clinical trials that enrolled 2,600 patients. The studies showed that kidney patients who aren’t yet on dialysis had a increased rate of heart-related serious adverse events, such as heart attack, stroke, congestive heart failure, and death, when they got the Affymax drug instead of the usual Amgen treatment. About 21.6 percent of patients on the Affymax drug had those serious adverse events, compared with 17.4 percent on the Amgen drug. Affymax stock crashed immediately, and hasn’t recovered.

Making matters worse, regulators have been on high-alert for years about serious adverse events with anemia drugs. Amgen suffered through its worst year ever in 2007, losing $29 billion in market value, as studies showed heart risks associated with its anemia drugs when used in high doses. That damaging information emerged as Affymax was beginning its quartet of clinical trials. The new evidence about the Amgen drugs prompted the FDA to insist on a rigorous set of studies that were designed to look for heart troubles from the start—unlike other studies which discovered heart trouble through retrospective analysis, which can introduce bias into clinical results.

Given the heightened anxiety at the FDA about heart risks with anemia drugs, many investors obviously concluded that Affymax’s drug would be dead on arrival. But Affymax, and its partner Takeda Pharmaceuticals, have continued their push to bring the new drug to the market.

The reason, Orwin says, is that when you look … Next Page »

Comments (1) | Reprints | Share:          

Amgen became the biotech industry’s biggest company largely because of a drug it developed in the 1980s for treating anemia. Plenty have tried, and failed, to knock Amgen off that pedestal ever since. But now a deepening alliance between Summit, NJ-based Celgene (NASDAQ: CELG) and Cambridge, MA-based Acceleron Pharma is taking one, and possibly two, more serious shots at the industry giant.

Celgene, the world’s third biggest biotech by market valuation, said earlier this week that it had agreed to expand its partnership with Acceleron to get the right to co-develop not just one, but also a second experimental drug the smaller company has designed against anemia. If the challengers are successful, they will prove to physicians that they can boost the body’s production of oxygen-carrying red blood cells to help patients fight anemia, without some of the safety concerns that have dogged Amgen’s products the past five years. And if that can be proven, the rivals will be in position to grab a piece of a market that, despite the safety worries, still generated $5 billion in worldwide sales for Amgen last year.

Celgene has had a remarkable growth run of its own the past decade with treatments for various blood cancers, but companies of that size need to aim high to sustain fast growth. That’s what has led it to zero in on a big new opportunity like anemia, says Acceleron CEO John Knopf. “When you’re a $25 billion company, you need a big hit,” Knopf said on a recent visit to his company’s offices.

How big is the anemia drug business exactly? Amgen lost $29 billion in stock market value in 2007 after its two anemia drugs were tied to higher risk of heart attack, stroke, and death when used at high doses, prompting the FDA to slap tight restrictions on their use. The worldwide market for anemia drugs generates more than $10 billion in sales each year, for patients with various states of kidney disease, and cancer patients who get anemic from chemotherapy that kills off otherwise healthy red blood cells just like tumors.

A little bit of science is required to understand how the new products are being made differently. Amgen’s drugs are genetically engineered forms of a hormone called erythropoietin that stimulates the production of red blood cells that carry oxygen through the blood, which people need to feel vigorous. One product is primarily designed for treating patients who are on kidney dialysis (Epogen), while the other (Aranesp) is a longer-lasting form that requires fewer injections, and is positioned for patients with less severe kidney disease and the chemotherapy-induced anemia.

Acceleron’s first alternative to this is … Next Page »

Comments | Reprints | Share:          

Cambridge, MA-based Acceleron Pharma is broadening its anemia research partnership with Celgene (NASDAQ: CELG), the Summit, NJ-based biotech giant, according to a press release. The companies, which have been collaborating since 2008, have formed a new joint development and commercialization deal for ACE-536, a compound that treats anemia. Celgene will make an upfront payment of $25 million to to Acceleron, which will also be eligible to receive up to $217 million in milestone payments and double-digit royalties should the product make it to market.

Comments | Reprints | Share:          

Palo Alto, CA-based Affymax (NASDAQ: AFFY) and its partner, Japan-based Takeda Pharmaceuticals, said today that the FDA has agreed to review their application to market a new anemia drug in the U.S. If the FDA agrees to clear peginesitide (formerly known as Hematide) for sale in the U.S., it would be the first once-monthly anemia drug for patients with kidney dialysis, and would compete with products from Amgen.

Comments | Reprints | Share:          

Affymax (NASDAQ: AFFY), the Palo Alto, CA-based developer of an anemia drug, said today it has raised $50 million in gross proceeds through a stock offering. The company sold about 8.47 million new shares at $5.90 apiece, about an 8.5 percent discount from yesterday’s closing stock price of $6.45. Lazard Capital Markets and Stifel Nicolaus Weisel managed the offering.

Comments | Reprints | Share:          

The biotech year doesn’t really start until the JP Morgan Healthcare Conference gets underway in San Francisco next week, but there were a few small news items that hit this week. See you next week in Union Square.

—Affymax (NASDAQ: AFFY), the Palo Alto, CA-based developer of an experimental anemia drug, said it has hired John Orwin as its new CEO, replacing Arlene Morris. Job No. 1 for Orwin will be getting the company’s lead compound approved by the FDA—despite the doubts that linger in the stock market about whether this is possible.

—Berkeley, CA-based Plexxikon said this week it has exercised its option to co-promote its experimental melanoma drug along with the U.S.-based Genentech unit of Roche. Plexxikon, which reported some startling early-stage clinical trial results with its drug over the summer, will have to pay some of the marketing expenses of this product, and stands to get an “enhanced royalty” because of its decision to set up its own sales force alongside Genentech’s.

—Genentech also crossed the wire earlier in the week when AMRI, the Albany, NY-based pharmaceutical service firm, said it licensed some antibacterial drug candidates to the biotech giant. This caught my eye partly because when I think of Genentech, I don’t think of antibiotics, so this could be an interesting new thing to watch.

—We’re always looking for innovative ideas at the intersection of biotech and IT, and I found one this week in something called the Cancer Commons. Internet commerce pioneer Marty Tenenbaum talked about how he’s pushing hard to create an open source data repository where cancer physicians and researchers can look at how individuals with certain tumor types are responding (or not) to certain treatments, and potentially see patterns that might not emerge in the usual setting of a controlled clinical trial.

—Xoma (NASDAQ: XOMA), the Berkeley, CA-based developer of antibody drugs, said this week it has secured a $35 million upfront payment under a partnership with France-based Les Laboratories Servier. Xoma also released some three-month interim clinical trial data for its drug, XOMA 052, although the final six-month results won’t be available until the second quarter of 2011.

—Those of you who follow the op-ed section of Xconomy have noticed that we’ve had a steady stream of interesting guest editorials the past couple weeks. Here are three that are probably of most interest to Bay Area life sciences readers:

BayBio’s Gail Maderis on Northern California’s Top 5 Biotech Innovation News Stories of 2010

Onyx Pharmaceuticals’ Tony Coles on Big Surprises of 2010 and Trends to Watch in 2011

Mohr Davidow’s Sue Siegel on Five Things to Look for In the Coming Year in Life Sciences

Comments | Reprints | Share:          

Affymax (NASDAQ: AFFY), the Palo Alto, CA-based developer of an experimental anemia drug, said today it has hired John Orwin as its new CEO, to replace Arlene Morris. Orwin was formerly the senior vice president of marketing and sales for Genentech before he joined Affymax last April as president and chief operating officer.

Morris, who became Affymax’s CEO in 2003, will continue to consult to the company until September. She leaves after a year in which Affymax stock took a big hit. Affymax, along with its partner Takeda Pharmaceuticals, said in June that clinical trials showed their anemia drug peginesatide (Hematide) was associated with a higher rate of heart-related adverse events, such as congestive heart failure and unstable angina, if patients had chronic kidney disease that hadn’t yet required dialysis.

The stock fell almost 70 percent on that news, and didn’t really recover from there, even though the company said the overall package of results was still encouraging enough for the company to seek FDA approval in a sicker patient population—kidney patients who are on dialysis.

I quoted one analyst back in August who said Affymax is “not dead yet.” Affymax confirmed in November that it planned to go ahead and seek FDA approval of its anemia drug candidate in the first half of 2011. That will be one of Orwin’s main objectives this year, no doubt. If Affymax can get its drug cleared for sale in the U.S., it will be in a position to compete with Amgen’s multi-billion dollar franchise drug, epoetin alfa (Epogen).

“We are now in final preparations of our new drug application and are on track to submit this application to the Food and Drug Administration in the second quarter of 2011,” Orwin said in a statement. “We believe we are well positioned to commercialize peginesatide upon potential approval for the treatment of anemia in dialysis patients, and look forward to our transition to a commercial organization.”

Comments (1) | Reprints | Share:          

Alnylam Pharmaceuticals (NASDAQ: ALNY), the Cambridge, MA-based developer of RNA-interference drugs, said today it has set a goal of moving five RNAi development programs into advanced clinical testing by 2015. Alnylam also named a new drug candidate for refractory anemia, which it hopes to move into clinical trials in 2012. The company said it hopes to form new partnerships, and that it expects to finish this year with more than $275 million of cash and investments left in the bank—meaning that it expects to reach its goals while burning less than $70 million of its existing cash reserves.

Comments | Reprints | Share:          

[Updated: 3:57 pm Eastern] One of the hot privately-held biotech companies in the Boston area, Acceleron Pharma, has made some significant job cuts.

The Cambridge, MA-based company eliminated 57 jobs, or about 40 percent of its workforce, late last month, according to a story first reported today in the Boston Globe. Acceleron’s vice president of corporate development, Steve Ertel, confirmed in a phone interview that the company made the cuts a couple weeks ago after striking a partnership with U.K.-based Shire. Acceleron now has about 90 employees.

[Update with company comment.] “We decided to right-size the organization to take advantage of our position of strength,” and reduce the company’s cash burn rate, Ertel says. The cuts were made across departments, in order to maintain the company’s expertise in manufacturing, discovery research, protein engineering, and clinical development, Ertel says.

Acceleron’s cutbacks are surprising given that the company has made a lot of headway in the past couple years, striking partnerships with big companies like Summit, NJ-based Celgene (NASDAQ: CELG), and most recently with U.K.-based Shire. Acceleron, founded in 2004, has raised about $100 million in venture capital from a big-name VC crew that includes Advanced Technology Ventures, Flagship Ventures, OrbiMed Advisors, Venrock Associates, MPM Capital, Bessemer Venture Partners, Polaris Venture Partners, and Sutter Hill Ventures.

In late September, Acceleron CEO John Knopf told my colleague Ryan McBride about how he hoped the new partnership with Shire would enable the company to wean itself off any dependence on venture capital.

Acceleron has been putting its money to work building a pipeline of experimental drugs, and infrastructure to manufacture protein drugs in-house. Some of the more visible work is going on to combat Duchenne Muscular Dystrophy, cancer, and anemia caused by chemotherapy.

Comments | Reprints | Share:          

Taligen Therapeutics wants to apply its technology platform to treat rare diseases in which the immune system goes haywire and attacks healthy cells. The Cambridge, MA-based biotech startup says it has received $10 million from its investors to drive its first drug toward initial human studies against a rare autoimmune disease.

The new funding brings the total amount of the firm’s Series B funding round to $36 million. The latest $10 million installment came from Taligen’s previous investors Alta Partners, Clarus Ventures, High Country Venture, and Sanderling Ventures, according to the company. The first closing of the second-round financing was revealed in February 2008, when the company said investors committed to put in as much as $65 million in separate chunks if the firm met certain development goals.

Taligen CEO Abbie Celniker says the company got the money because it made it made “substantial progress” over the past year that enabled it to advance close to its first clinical trial.

The company, founded in 2004 with technology from the University of Colorado, has been developing protein drugs that target an immune reaction called the complement pathway. Its lead drug, TT30, prevents overactive complement reactions in certain diseases by replacing defective proteins. While the drug has the potential to treat a number of immune and inflammatory diseases, the company has decided to focus initially on rare diseases where there’s less competition such as atypical hemolytic uremic syndrome (aHUS), which affects one in 500,000 Americans, and paroxysmal nocturnal hemoglobinuria (PNH).

A potential advantage of Taligen’s drugs over existing treatments is that they aim to … Next Page »

Comments | Reprints | Share:          

Acceleron Pharma, a Cambridge, MA-based maker of genetically engineered drugs for treating conditions such as anemia and bone loss, has pulled in $8.4 million of a planned $11.5 million offering of equity, options, and warrants, according to an SEC filing.  Members of Advanced Technology Ventures, Venrock Associates, Flagship Ventures, OrbiMed Advisors, and Polaris Venture Partners are among those listed as Acceleron directors on the filing for the newest funding. Alkermes CEO Richard Pops is also listed as a member of the Acceleron board. Luke wrote about Acceleron back in December when the company gave him a tour of its Cambridge facilities, after it had invested in building its own manufacturing and development site rather than outsourcing the tasks.

Comments | Reprints | Share:          

Affymax (NASDAQ: AFFY), the Palo Alto, CA-based developer of an experimental anemia drug, released some bad news today from clinical trials conducted with partner, Takeda Pharmaceuticals. While four pivotal studies of a combined 2,609 patients showed that peginesatide (Hematide) wasn’t inferior to competing products from Amgen in terms of boosting hemoglobin to carry oxygen in the blood, researchers did see a higher combined rate of adverse cardiac events like heart attack, stroke, heart failure, and death in a group of patients on the new drug. The cardiac effect was seen among anemia patients who aren’t on kidney dialysis, a potentially huge market for any anemia drug. Shares of Affymax plunged 65 percent to $8.06 by 9:15 am Eastern in pre-market trading.

Comments | Reprints | Share:          

Looks like the recent Boston sunshine helped plump up deal flow for the region’s software, Internet, and life sciences companies.

—Financial services software company SS&C Technologies Holdings, of Windsor, CT, set its IPO price at $15 a share, for a total of 10.725 million new shares. The price was at the high end of SS&C’s earlier proposed range. The initial public offering was worth nearly $161 million and put SS&C stock trading on the NASDAQ under the ticker symbol SSNC.

—Hangout Industries, a software platform provider for social games targeted toward teens, raised $2 million from existing investors, including Highland Capital Partners and Polaris Venture Partners. The Boston-based company’s CEO told Mass High Tech that another $3 million could be on the way, bringing Hangout’s funding total to $15 million.

—Lexington, MA-based Amag Pharmaceuticals (NASDAQ: AMAG) grabbed $60 million in upfront cash from a deal that gives Japan-based Takeda Pharmaceutical exclusive license rights to all therapeutic uses of ferumoxytol, Amag’s treatment for iron deficiency anemia, in Europe, former Soviet states, Asia Pacific countries (excluding China, Japan, and Taiwan), Canada, and Turkey.

—Cambridge, MA-based Cequent Pharmaceuticals and Bothell, WA’s MDRNA (NASDAQ: MRNA) announced they would merge in a $46 million, all-stock deal. The merger, expected to close in July, will … Next Page »

Comments | Reprints | Share:          

Looks like the region’s massive flooding didn’t get in the way of everything this week. We saw lots of headlines of partnerships for area drugmakers.

—Dicerna Pharmaceuticals, a Watertown, MA, developer of drugs designed to silence disease-related genes using RNA interference, announced its second big partnership this year, this time with the French biotech Ipsen. The deal won’t pull in a ton of cash for Dicerna, but will give the company 50-50 ownership rights to drugs developed through combining Dicerna’s RNAi technology with Ipsen’s strategy of using peptides as delivery vehicles in cells, Luke wrote. In January, Dicerna announced a partnership with Japan-based Kyowa Hakko Kirin for developing cancer drugs, which could be worth up to $1.4 billion in milestone payments.

—Ryan wrote about how the impending healthcare reform could mean big payoffs for Burlington, MA-based Nuance Communications (NASDAQ:NUAN), a speech recognition software company that’s advocating for the government to make rules that would standardize its technology alongside electronic medical records. Nuance has products that allow doctors to dictate patient information directly into electronic records systems and enables radiologists to more efficiently order imaging tests, helping to make healthcare customers the company’s biggest market.

—Avila Therepeutics announced it will receive as much as $3.2 million from The Leukemia & Lymphoma Society for the development of a drug that treats B-cell related cancers. The Waltham, MA-based company’s AVL-292 drug aims to form covalent bonds that hit targets on the B-cells in the immune system, and is set to enter its first clinical trial this year.

—Cambridge, MA-based Ascent Therapeutics announced it had changed its name to Anchor Therapeutics to better … Next Page »

Comments | Reprints | Share:          

[Updated, 4/01/10, 4:40 pm] Amag Pharmaceuticals has struck a deal to expand global marketing of its best-selling drug. The Lexington, MA-based company (NASDAQ:AMAG) reports this morning that it has licensed its iron deficiency anemia drug to Japanese drug giant Takeda Pharmaceutical Company in a deal that includes $60 million in initial fees.

The deal gives Takeda an exclusive license to all therapeutic uses of Amag’s drug, ferumoxytol (Feraheme), in Europe, former Soviet states, Asia Pacific countries (excluding China, Japan, and Taiwan), Canada, and Turkey. Amag, which received FDA approval in June 2009 to market the drug as a treatment for iron deficiency anemia in adults with chronic kidney disease, will continue to control rights to the drug and handle sales of the product in the U.S. [Editor's note: The word "therapeutic" was inadvertently omitted from the paragraph above when this story was published this morning.]

For Amag, this deal provides cash to seek additional approvals for ferumoxytol as well as an organization to sell its lead product in the next big potential market for the drug, Europe. The company plans to file an application with the European Medicines Agency in mid-2010 for permission to market the drug in European Union countries for patients with chronic kidney disease. Takeda has agreed to pay Amag $220 million in milestone fees for meeting certain development goals with the drug as well as double-digit royalties on Takeda’s potential sales of the product in the territories included in the deal.

“Takeda’s global presence, their pipeline that includes complementary products to [ferumoxytol] and their strength in the marketing and commercialization of therapeutics across many specialties where iron deficiency anemia is present makes them the ideal partner,” Brian Pereira, Amag’s president and CEO, said in a statement.

Last year, Amag took in $15.8 million in revenue from sales of ferumoxytol. The iron-replacement drug, which is intended to boost the amount of oxygen-carrying proteins in blood, is given to patients intravenously. About $500 million in injected iron-replacement drugs are sold annually for the some 400,000 Americans with kidney failure, according to analysts at Robert W. Baird.

Comments | Reprints | Share:          

Stephen Seiler has been hunting for cash to advance an experimental therapy for sickle cell disease into initial human studies. And the veteran biotech executive, the former CEO of Cambridge, MA-based Idera Pharmaceuticals (NASDAQ:IDRA), has founded a startup in Newton, MA called AesRx to carry out his goal.

Seiler said he has provided an undisclosed amount of money from his own pocket to support the young startup, which was formed in November 2008. Now he’s looking to raise about $10 million in a Series A round of venture capital to advance the startup’s lead drug, called Aes-103, into a Phase I clinical trial next year for sickle cell disease. He’s making the case that the current treatments for sickle cell disease (sometimes called sickle cell anemia), which affects about 75,000 Americans, have left patients with far too few options to treat the genetic, lifelong illness that causes pain, infections, organ damage, and can often be deadly.

The firm’s drug is a small molecule called 5-hydroxymethyl-2-furfural, or 5HMF, which is derived from sugar and can be found in everything from caramel and cookies to prune juice. Based on preclinical studies, according to Seiler, there is evidence that the drug could be less toxic than previous treatments for sickle cell disease. For example, the standard treatment for the disease, called hyroxyurea, is typically prescribed only when patients are experiencing pain and other complications of sickle cell because it can cause side effects such as vomiting and lowered red blood cell production. And while bone marrow transplants have been able to cure the illness, there are challenges to making this therapy available to most patients because a matching donor is needed and it’s too expensive for them.

“Although it’s still pre-clinical, we know a lot about this drug,” Seiler said. “Therefore we believe our odds of success are better than a random pre-clinical compound.”

For now, the company is supporting some of its claims about the potential utility of its treatment with outside, peer-reviewed studies that validate the importance of the target that AesRx’s treatment binds with, as well as the drug’s intended mechanism of action. The compound was also studied extensively by researchers at Virginia Commonwealth University who first discovered its potential to treat sickle cell disease. (Seiler purchased the experimental drug after its former owner, New Jersey-based Xechem International, filed for Chapter 11 bankruptcy protection in fall 2008, he said.)

Sickle cell disease is caused by abnormal … Next Page »

Comments (2) | Reprints | Share: