Alnylam Pharmaceuticals has been saying for a while that it needs hard data from clinical trials to prove its skeptics wrong, and today it’s coming out with an early hint of effectiveness with its RNA interference-based treatment for lowering cholesterol.

Cambridge, MA-based Alnylam (NASDAQ: ALNY) is announcing today that it has gotten some encouraging results from a study of 20 patients who got a single shot of Alnylam’s experimental drug called ALN-PCS. This drug, designed to block the activity of a protein known as PCSK9, was able to reduce the protein by an average of 60 percent after four days when given at the highest of five doses studied. That knockdown of the specific protein translated into an average drop of 39 percent in low-density lipoprotein, the so-called “bad” form of cholesterol that clogs arteries and is known to raise people’s risk of heart attack, stroke and death.

The new drug was well-tolerated at a variety of doses, and no one has dropped out of the study because of side effects, although a mild rash was observed in patients on the treatment, the company said. The study is still ongoing, and Alnylam plans to continue examining the safety of higher doses, but the company is communicating the progress before it’s all wrapped up. Preliminary data is being presented today at the Brigham & Women’s Hospital in Boston, and more details are expected to be presented in the first half of 2012, Alnylam said. The company also plans to discuss the results in more detail on a conference call with investors today at 8:30 am Eastern time.

The data is still from the first of three phases of clinical trials normally required for FDA approval, so this drug still has years of rigorous studies to complete if it is ever going to become a new treatment for lowering cholesterol. But the market for cholesterol-lowering drugs was worth $36.4 billion worldwide last year, according to research firm IMS Health, largely because of the huge success of statin drugs. While many of those drugs are losing patents and facing cheap generic rivals, many big biotech and pharma companies (including Amgen and Sanofi) are focusing on a promising new molecular target in PCSK9, which Robert Langreth of Bloomberg News described in a November feature. Carlsbad, CA-based Isis Pharmaceuticals (NASDAQ: ISIS) also has an anti-PCSK9 product in preclinical development with Bristol-Myers Squibb.

Alnylam is wagering that while other companies are focusing on hitting this protein with genetically engineered antibodies, it will have an effective option that works through RNA interference, which specifically silences the RNA that enables the body to make the PCSK9 protein. If Alnylam can continue to show intriguing data from clinical trials (like it did last fall for a rare condition called TTR amyloidosis), it could help revive some of the enthusiasm for RNAi. Excitement for the technique once ran high because of its new ability to molecular targets that have been considered “undruggable” in the past, but enthusiasm has waned in the past couple years as big companies like Roche and Merck have curtailed their RNAi drug development activities.

The data so far for Alnylam’s PCSK9 program is still quite preliminary. Researchers randomly assigned 20 patients with … Next Page »

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Saddle Brook, NJ-based CircuLite announced today that it has raised $30 million in an oversubscribed Series D financing round. The company said in a statement that it will use the proceeds to support the anticipated 2012 European launch of a device it dubs “the world’s smallest surgically implanted blood pump,” which is used to treat patients with heart failure. The device, called Synergy, is a pump roughly the size of a AA battery that boosts the heart’s ability to circulate blood to vital organs. The funding will also be used to support the U.S. development of Synergy, the company said.

The financing round was led by new investor MacAndrews & Forbes Holdings and founding VC Forbion Capital Partners. CircuLite’s other investors include Crédit Agricole Private Equity, Saints Capital, Foundation Medical Partners, and SB Life Science Ventures. CircuLite raised $69 million in three previous funding rounds.

CircuLite was founded in 2004 with a focus on developing minimally invasive devices to treat patients with life-threatening heart conditions. The company is working on next-generation Synergy micro-pumps, and it has a grant from the National Institutes of Health to collaborate with the University of Maryland School of Medicine on devices to treat children with heart conditions.

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There’s been a deluge of venture capital news over the past week, and a flash flood of VC cash during the second quarter went mostly to San Diego’s life sciences sector. Our briefing begins now.

—San Diego venture firms invested a total of $198.2 million in 29 local startups during the second quarter that ended June 30, according to a breakout of regional data from the MoneyTree Report. The single biggest deal was the $50.4 million for San Diego’s Sangart, but in fact, 24 of the 25 deals were categorized as life sciences deals, accounting for roughly $193.1 million—or more than 97 percent of the $198.2 million total invested here.

—San Diego’s Zogenix (NASDAQ: ZGNX) said it signed an agreement with Durect (NASDAQ: DRRX) of Cupertino, CA, to develop Durect’s long-lasting reformulation of the anti-psychotic drug risperidone (Risperdal) for use with the Zogenix needle-free injector. Zogenix says its collaboration with Durect is a good example of its unfolding strategy, and could lead to the first long-lasting formulation of the anti-psychotic drug available in a needle-free injector for once-a-month use.

—Intellikine, a San Diego startup developing new anti-cancer drugs, hired former Celgene vice president Greg Berk as chief medical officer responsible for overseeing Intellikine’s clinical trials. Intellikine CEO Troy Wilson says he’s encouraged by the company’s progress in developing INK128, a compound that blocks a mutated kinase linked to a variety of cancers from wreaking its havoc in what he calls the PI3K/mTOR signaling pathway.

—In his BioBeat column this week, Luke makes his case for resisting the pressure for life sciences companies to rush into mergers and acquisition deals with Big Pharma. He argues that … Next Page »

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San Diego-based Celladon, which is developing drug candidates that target a key enzyme deficiency apparent in heart failure, has secured $400,000 in a debt and securities financing, according to a recent regulatory filing. In late June, Celladon said a clinical study of its lead candidate, which is intended to restore a key enzyme that regulates calcium cycling and contractility in the heart, met its primary safety and efficacy endpoints at six months. The company has raised more than $60 million, according to Dow Jones VentureWire. On its website, Celladon identifies its current investors as as Enterprise Partners Venture Capital, Venrock, and Johnson & Johnson.

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In case you haven’t noticed, heart disease remains a debilitating illness for millions. Cancer, despite some recent advances, has not been cured. Drug resistant strains of bacteria are spreading across the globe. There are still no effective treatments for Parkinson’s disease, amyotrophic lateral sclerosis, muscular dystrophy, and a huge number of other illnesses.

The number of new medicines being brought to market has been declining in recent years at a time when we desperately need new treatments. A recent analysis of drugs moving through FDA trials in 2003-2010 found that the overall success rate was only about 14 percent for primary indications, and a dismal 3 percent success rate for secondary indications. The number of new drugs launched annually has fallen 44 percent since 1997, according to CMR International.

A variety of reasons have been put forth to explain this deterioration, as described below. As a nation, we should be doing all that we can to encourage innovators to find effective treatments for these diseases. Developing medications to successfully treat human diseases is an expensive enterprise, so it makes sense to ask the question: where will the new medicines come from? What financial resources are available now to biomedical researchers to fund their work, and who’s going to pay for future drug development?

A good place to start to answer these questions is to have an understanding of where our present drugs have come from. Two recent studies illustrate nicely that modern medicines originate from a variety of sources, and help explain the expanding number of partnerships between industry and academia. Robert Kneller examined every drug approved by the FDA over the past 10 years, and determined for each one whether it originated with pharmaceutical companies, from biotechs, or sprang forth from the labs of academic investigators. His analysis showed that about half of all new drugs arise from pharmaceutical companies, while the other half arise from the efforts of biotech firms and academic investigators. In a separate study, other researchers identified which medicines resulted from public sector research over the past 40 years. Their analysis identified some 153 products that were discovered or co-discovered by 75 different academic institutions. These drugs accounted for some 13 percent of all FDA approvals over that period. Having roughly quantified where our drugs come from, let’s shift our focus to how this drug discovery work gets funded. What monetary sources are available to biomedical researchers in the public and private sectors? Here’s what my informal survey found:

Pharma and Biotechs Still Lead the Charge (Funding Level: $65.3 billion in 2009)

There is no doubt that pharma and biotech companies are the major source of new drug discoveries. However, both groups find themselves under significant fiscal pressures as a result of troubles in the financial markets, recent patent expirations, and their diminishing number of drug approvals in the past decade. Why have these numbers been declining? The primary problem faced by industry is the challenge of translating basic biological discoveries (e.g. genomic sequences; RNA interference; mutated genes) into functional drugs. Although scientists have determined the causes of many diseases over the past quarter century, they haven’t figured out yet how to fix many of them. Other urgent industry concerns include pricing pressures, reimbursement issues, regulatory hurdles, generic competition, and investor expectations. Big Pharma’s response to these challenges have varied: some companies responded by either maintaining or increasing their research and development commitments (e.g. Merck), while others took the opposite approach and slashed their research dollars (e.g. Pfizer, Glaxo).

Many Big Pharma companies have been slowly transforming themselves over the past few years to be more like biotechs. They have taken note of the fact that the success rate for getting biologics (e.g. recombinant growth factors, monoclonal antibodies) approved is twice that seen with their small molecule drugs. Generic competitors of their blockbuster small molecule drugs are continuing to poke enormous … Next Page »

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Forget stealth mode—these days the coolest pre-launch Silicon Valley companies are in ninja mode. That’s how Sutha Kamal, the CEO of San Francisco-based Massive Health, describes his situation in a blog post today announcing the company’s $2.25 million seed funding round.

Luckily, I got Kamal and co-founder Aza Raskin to come far enough out of their ninja-turtle shells to tell me a little bit about the company and its plans, which center on using mobile apps to help people with chronic conditions like diabetes, obesity, hypertension, and heart disease take charge of their own health. The company expects to release its first app sometime this year, Kamal said.

Certainly, these guys have plenty of street cred on the software, wireless, and product design sides. Kamal managed mobile strategy and product design at TransGaming Technologies, a Canadian iPhone game developer, and ran a mobile content portal called Ambient Vector. Raskin was the head of user experience at Mozilla Labs and ultimately became creative lead for the Firefox browser. After leaving Mozilla in December to start Massive Health, Raskin blogged that “healthcare needs to have its design renaissance,” and said he wanted to apply his experience building products that are “disruptively easier and more enjoyable to use” to the challenge of helping people stay healthy.

But exactly how a team of three product-and-programming guys (Kamal and Raskin are joined by engineering lead Doug Soo, formerly of Linden Lab) plans to disrupt the notoriously complex, messy, and intractable economy of U.S. healthcare isn’t clear yet. Judging from Kamal’s post, the company’s apps will incorporate all the fashionable elements you might expect—crowdsourcing, game mechanics, the social graph, data analysis, and “tight feedback loops”—but in ways that aren’t reminiscent of today’s social-networking apps. “We’re not proposing giving you a badge for eating your broccoli or letting you check-in and become duke of ranch dressing,” Kamal writes, in a cheeky dig at Foursquare.

That seems to have been enough to convince investors. The unusually large seed round—which is almost Series A-sized—comes from an all-star team of venture investors, including Mohr Davidow Ventures, Greylock Partners’ Discovery Fund, Andreessen Horowitz, Aydin Senkut’s Felicis Ventures, Charles River Ventures, and the Collaborative Fund, as well as a group of “amazing angels” that the company hasn’t identified.

Now that the company has some money to throw around, it says it’s hiring—current job listings call for a “Front-End(ish) Engineer” and a “Back-End(ish) Engineer.” Applicants should be able to “run with the ball without guidance but know when to pass or assist,” the startup’s site says, but winningly adds, “Getting sports analogies shouldn’t be your strong point.”

Here’s a writeup of my brief conversation today with Kamal and Raskin.

Xconomy: You guys have managed to stay pretty deep in stealth mode. What are you up to, and what will the new funding help you do?

Aza Raskin: We looked at the state of the world and realized that 90 percent of U.S. healthcare costs go to 15 percent of patients—people with diabetes, weight loss problems, hypertension, heart disease. We are trying to create fantastically user-centric products that tackle these really hard problems that are going to be the pandemics of our times. The reason we raised as much as we did, $2.25 million as a seed round, was to give us the leeway to … Next Page »

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Minnow Medical, which we profiled just over a year ago, has raised a total of $4.7 million through two separate financings, according to a statement yesterday. The lead investor in both transactions was Neomed Management of Switzerland, according to Minnow, which has a new CEO, Raymond W. Cohen, who has replaced founding CEO Tom Steinke. Minnow also has moved its headquarters from San Diego to Laguna Hills, CA, in Orange County.

Former Domain Associates partner Olav Bergheim, Minnow’s board chairman, Christopher Weil & Co., and several private investors also participated in the financings. Minnow plans to use the proceeds to pay off its bank debt, for working capital, and to fund European clinical trials of its ZCath System, the proprietary angioplasty balloon catheter combined with controlled radiofrequency energy. The design is intended to prevent restenosis, a recurrence of the narrowing of a blood vessel, in peripheral vascular stents.

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Ryan McBride

Burlington, MA-based InfraReDx said today it has raised $21 million in a Series D round of equity financing from new and existing backers. The cash infusion will enable the firm to start U.S. sales of its latest-generation coronary imaging system, which gained FDA clearance this summer.

The 12-year-old firm says that its “LipiScan IVUS” system is the first to combine near infrared light and a type of intravascular ultrasound to provide detailed images of a type of fatty plaque in the arteries. Last year, the firm released its first version of the system, which doesn’t seem to include the ultrasound technology. (Here’s more on InfraReDx‘s infrared technology from our previous coverage.)

For the commercial release of its latest product, the company plans to target top catheterization labs in the U.S. and expand its sales to Europe in 2011. Catheterization labs are where doctors perform stenting procedures to prop open a patient’s artery after blockages are removed. The firm says that its imaging system can enable precise placement of stents, limit complications of stenting procedures, and help doctors to figure out the best treatments for a patient’s heart disease.

“The LipiScan IVUS system will enable interventional cardiologists to accurately and rapidly identify the lipid core plaques that complicate stenting and are believed to be the major cause of heart attacks,” said James Muller, the company’s founder and CEO, in the announcement.

InfraReDx, which has now raised more than $100 million from investors, named only its previous backer Sanderling Ventures of San Diego and San Mateo, CA in this latest round. The company declined to release the names of its new investors.

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San Diego’s Sequel Pharmaceuticals has raised almost $1.4 million in a combination of debt, rights, and securities in a round intended to raise more than $1.9 million for the three-year-old specialized drug developer, according to a recent regulatory filing. Sequel is focused on developing a drug candidate for treating an abnormal heart rhythm known as atrial fibrillation. As Luke reported last October, founding CEO Randall Woods started Sequel within 24 hours after selling his previous biotech, NovaCardia, to Merck for $350 million. Sequel’s financing was reported by VentureWire, which noted the startup previously raised $3 million just over a year ago, and raised $20 million when it was founded in 2007 from investors that include Domain Associates, Forward Ventures, InterWest Partners, Montreux Equity Partners and Skyline Ventures.

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Insilicos, a Seattle-based developer of biomedical software and diagnostics, has secured a $1.2 million grant from the National Institutes of Health, through the Small Business Innovation Research program, to study a potential new test for people with chest pain. The test will be given to 400 patients at the University of Washington with cardiovascular disease, and will attempt to predict whether they will suffer heart attacks. “People who can’t be diagnosed can’t benefit from treatment. For this and other reasons, diagnostics are, in my opinion, the greatest need and greatest opportunity in cardiovascular disease, and it feels great to take a big step towards doing something about that,” Insilicos president Erik Nilsson says in an e-mail.

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Genzyme has been all over the news in the past week because of takeover speculation, but today when fundamental news came out about one of its most important assets for the future, investors yawned.

Cambridge, MA-based Genzyme (NASDAQ: GENZ) and its partner, Carlsbad, CA-based Isis Pharmaceuticals (NASDAQ: ISIS) said today that their drug for people with severely high cholesterol passed a pair of clinical trials. The drug, mipomersen, has now passed a total of four pivotal clinical trials, and the companies are preparing to seek clearance in early 2011 from regulators to start selling the drug in the U.S. and Europe.

Isis, which relies more heavily on this product for its future financial success, saw its shares drop 5 percent to $9.49 at 11 am Eastern time, while Genzyme stock dropped 1.6 percent to $69.07.

Expectations have been high for this drug to become a hit for a couple years now, as the next big thing for cholesterol after the patents expire on multi-billion dollar statin drugs such as Pfizer’s atorvastatin (Lipitor). Genzyme was said to have outhustled more than a dozen rivals that wanted to co-develop mipomersen, and it paid a whopping $325 million in upfront cash, plus $1.9 billion in potential milestone payments, (and potentially much more in future profit-sharing), to get a piece of ownership in this drug back in January 2008.

There’s so much interest for a few reasons. While statins have been used by millions of people to lower cholesterol for more than a decade, there’s a need for stronger cholesterol-lowering drugs for patients who can’t get their levels under control with conventional statins. The new drug is also based on new science, in that it’s designed to use specially engineered strands of RNA drugs to block a problematic protein in the body, which often can’t be hit by conventional small-molecule drugs. In this case, mipomersen is engineered to block the production of a protein called apoB that carries the so-called “bad” LDL cholesterol in the bloodstream. Isis CEO Stanley Crooke told me last October that mipomersen represents the third “remarkable” advance he’s seen in his 30-year career in drug development.

So what did today’s results actually tell us? The first study enrolled 58 patients who were randomly assigned to get a once-weekly injection of mipomersen, or a placebo, for about six months. The patients entered the study with severely high cholesterol—a median LDL count of 276 milligrams per deciliter of blood, even though they were already taking the highest tolerable dose of statins. By the end of the 26-week study, patients on mipomersen saw their cholesterol score drop 36 percent to a more manageable 175 milligrams per deciliter.

Still, not everybody in the trial was motivated to stick with the mipomersen regimen. Of the 39 patients who got the drug, eight dropped out of the study because of adverse events, Isis and Genzyme said. For comparison, just one of the 19 placebo patients dropped out because of an adverse event, the companies said. Researchers also saw increased levels of liver enzymes in mipomersen patients, which can be a sign of liver damage, although there were no reported cases of actual liver damage.

The second study enrolled 158 patients who weren’t as sick—they were classified merely as having a high risk of developing heart disease. More than half of the patients also had diabetes. Patients entered the study with a median LDL cholesterol score of 123 milligrams per deciliter, and when they got mipomersen, they saw that figure drop 37 percent on average to 75 milligrams per deciliter. Half of the patients got to below 70 milligrams, which is a treatment goal for high-risk heart disease patients, the companies said.

“We are pleased with the robust efficacy of mipomersen across all four phase 3 trials. These data, along with the emerging safety profile, support our focused approach on patients at highest cardiovascular risk who are in the greatest need of new treatments,” said Genzyme senior vice president John Butler, in a statement.

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In the last two years, biotechnology companies, especially the smaller ones, have gone through a dramatic restructuring of priorities and focus. Like so many industries which have prioritized their businesses to the immediate, the cash efficient, the safe, the low risk, and the cash conserving, biotech has done the same. Some biotech companies have done it out of necessity, and some have done it out of fiscal prudence, but almost all biotech companies have reduced or eliminated early stage research, early stage development programs—essentially all efforts other than their latest stage development efforts. Research labs have shrunk considerably or been closed, and all development programs other than the key lead program have been shut down or suspended.

About 10 years from now, roughly the time it takes to develop a new drug, it is likely that the medical community will be lamenting the fact there is a dearth of new medicines to treat the range of diseases we will be grappling with. Cancer, heart disease, diabetes, autoimmune diseases, and many others—many of the potentially new treatments in the labs and early development programs just a few years ago are now shelved, shut down, or left to falter as research and early development programs are being killed. If we think the number of new molecular entities, the number of truly innovative new medicines making it through the tortuous FDA approval process is low today, it will likely be even painfully lower a decade from now.

It is ironic that while corporate biotechnology has been cutting back all but the latest stage programs, massive federal dollars have been flooding into universities and research centers. Although a few recent articles have bemoaned the lack of real products emanating from our landmark work in sequencing the human genome, there is an extraordinary amount of early stage work being done at our university labs. Unfortunately, with the cutback in early stage biotech funding, there is little money, and fewer resources to pull these technologies out of the university environment and spend the “slogging dollars” required to move the research from the lab to the development environment. Given the long time lag in the biotech product cycle, it will take years for this diminished “pull” of academic research into the corporate development world to manifest itself as a striking and potentially alarming decrease in innovative treatments for critical diseases. This situation is compounded by the fact that many great advances, including many great new drugs, were discovered by serendipity. “Spurious” results seen on one indication turn out to be primary endpoints for another. Serendipity requires numbers—many programs, many efforts, increasing out research work, not decreasing it.

Is there a solution to this inevitable drop in new product development? Other than a miraculous near term turnaround in the economy (unlikely), what can all of the players do to keep the early stage research flames alight? Every player can make a contribution:

• Universities can alter licensing terms to ensure the rapid and risk adjusted movement of their technology from the institution to the company. We need lower up front/early payments and more risk sharing on the part of the institutions.
• Investors and VC’s will have to take the longer term view, recognizing that one program or one compound does not an industry make. Funded companies will have to generate more opportunities and move them to partnerships faster.
• Management teams will have to be willing to take on the risk of the early stage startup, partner early and often, and create as many “shots on goal” as possible.
• Pharmaceutical companies will have to learn to develop partnerships faster and more efficiently with biotechnology companies and early stage programs. Both biotech companies and pharma will have to develop a partnering model allowing for more dollars spread over more projects to help winners and weed out the losers. Pharma cannot continue to sit on the sidelines and wait for investors (and management teams) to take all the risk, and step in to partner only after key data emerges.

Will all of the players move consciously to ensure a robust future pipeline of new treatments? Will each institution make the difficult adjustments to their own business models? Can one group do it without the others moving as well? Will players be forced to move as the market pressures their business model? Or will they find other outlets (ie: green tech for investors, bio-similars for pharma) and possibly move away from discovery on a permanent basis? Let’s hope that all parties will find a way to make the discovery model work. The future of new treatments hangs in the balance.

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Many people have probably never heard of some of the diseases that venture capitalists and drug company executives are swooning over lately. But regardless of how obscure a rare illness like X-linked hypohidrotic ectodermal dysplasia is, investments in developing drugs for such diseases are growing in popularity.

In Boston, both venture firms and pharma executives are getting in on the act. Third Rock Ventures has funded or formed three biotech startups in Cambridge, MA over the past two years that are developing drugs for rare genetic disorders (which are in some cases called orphan diseases). One of those companies, Alnara Pharmaceuticals, counts among its founders Christoph Westphal, a Cambridge-based executive who scouts for external business opportunities for London-based drug giant GlaxoSmithKline (NYSE:GSK).

These companies are ripping a page or two from the battletested playbook at Cambridge-based Genzyme (NASDAQ:GENZ). The company’s three best-selling treatments are for rare genetic diseases that affect fewer than 10,000 patients each. Still, Genzyme has been profitable because it can command hundreds of thousands of dollars per year for each patient treated with some of its drugs, and for years it has faced very little or no competition in these niche markets.

But the party’s getting more crowded nowadays. New York-based Pfizer (NYSE:PFE), the world’s biggest drug company, inked a deal announced in December to partner with Israel-based Protalix Biotherapeutics to develop and market Protalix’s rival drug to Genzyme’s top seller, imiglucerase (Cerezyme), an enzyme-replacement therapy for patients with Gaucher’s disease. Genzyme’s Gaucher drug brought sales last year of $793 million, way less than Pfizer makes from its top sellers like the heart pill atorvastatin (Lipitor). Despite the smaller markets for rare disease treatments, major pharma companies are investing in them as many of their multi-billion dollar drug franchises face greater competition from generic knockoffs.

“Pharma and bigger biotech players need to … Next Page »

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Ascent Therapeutics, a Cambridge, MA-based pre-clinical stage drug development company, announced today that it has changed its name to Anchor Therapeutics to better reflect its technology. The company is developing drugs based on “pepducins,” which anchor in cell membranes to target molecules called G protein coupled receptors that are involved in ailments such as cancers, heart disease, inflammation, pain, and metabolic disorders. Anchor’s backers include HealthCare Ventures, Novartis Option Fund, and TVM Capital.

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Ischemix is facing the biggest hurdle in its 11-year history. The Maynard, MA-based biotech firm is launching the first human study to test the effectiveness of its drug that’s supposed to protect heart tissue from injuries that often occur when blood flow is restored in cardiac surgeries.

The drug, called CMX-2043, is the only contender the company has that’s ready for clinical trials, says CEO Duffy DuFresne. Without backup clinical candidates, the success of the firm’s Phase IIa trial in treating ischemic-reperfusion injury is hugely important to the future of the company.

Success would be a major leap forward for both Ischemix and the treatment of ischemic-reperfusion injuries, for which there are no FDA-approved therapies despite many previous attempts. The closely held company—funded primarily by its chairman, and its medical director—has enough money to pay for the mid-stage clinical and not much else beyond that, according to DuFresne.

“The good news is that we’ve raised enough money to carry us to the end of this trial,” DuFresne says, “the bad news is that we’re not funded beyond that and additional funding is going to have to be determined by how encouraging our results are.”

The clinical trial is expected enroll 200 to 220 patients, many of whom are undergoing surgeries to unblock their arteries and implant stents to restore blood flow, according to DuFresne. The firm’s drug, which was previously tested for safety in healthy people, will need to prove that it’s both … Next Page »

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I’ve heard about serial entrepreneurs who start a company, build it up to a certain point, sell it to someone bigger, and then repeat the whole cycle again. But I had never heard of a true biotech sequel until I met Randall Woods a couple weeks ago.

Woods is the CEO of San Diego-based Sequel Pharmaceuticals, and a well-known entrepreneur who’s also the chairman of Biocom, the local biotech trade association. The two-year-old startup is literally the sequel to his previous company, Novacardia, a company that Woods led until it was sold for $350 million to Merck on Sept. 6, 2007.

Sequel came less than 24 hours later. The same nine employees, in the same office, with the same management team, and the same board (except for one), set their sights on a new goal. The idea was to take a drug in the early stage of development, steer it to the later stage of trials until the concept is more proven, and then sell it for a bundle to big drugmaker. Novacardia took a drug into pivotal studies for congestive heart failure, then passed the baton to Merck for the final phase of development. Sequel aspires to do the same thing with a different drug for a different heart ailment—atrial fibrillation.

“We didn’t even have a 24-hour break,” Woods says. “We just changed the sign on the door.”

This group of people clearly has skill in cardiovascular disease, so it knows something about the new problem. Atrial fibrillation is an abnormal heart rhythm that can cause acute attacks, or a chronic condition whose symptoms include shortness of breath, chest pain, or stroke.

Randall Woods

About 2.2 million people in the U.S. are estimated to be affected, and it caused 470,000 people to be hospitalized in 2003, according to the American Heart Association. The incidence is thought to be increasing as the Baby Boomers get older. There haven’t been many new developments in treatment either, except Sanofi-Aventis’ dronederone (Multaq), which first won FDA approval in July. That drug showed it could reduce hospitalizations from cardiovascular disease and deaths from all causes by 24 percent when compared to a placebo. Other than that, patients sometimes take beta-blockers to slow down their heart, or warfarin to thin their blood, Woods says. Another treatment from Vancouver, BC-based Cardiome Pharma is seeking FDA approval.

Sequel’s drug, called K201, is designed … Next Page »

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Doctors often prescribe a drug like clopidogrel (Plavix) or aspirin to help prevent their patients from suffering a heart attack, stroke, or even waxy plaque buildup along the inside of blood vessels. But how do they know if the dosage is correct, and that the drug prescribed is actually working as intended to prevent blood platelets from clumping together?

San Diego-based Accumetrics, a venture-backed medical diagnostics company, has a solution. “In a nutshell, we’re the leading company in the measurement of platelets,” says Accumetrics CEO Timothy Still.

Heart disease remains the leading killer of both men and women in the United States. And Still says Accumetrics sees a potential multi-billion-dollar market in helping doctors and patients calibrate the most widely used anti-clotting drugs. Still, who was named CEO last fall, joined Accumetrics with its fourth round of venture capital in 2007. After revamping the company over the past seven months, the CEO says he anticipates additional opportunities for Accumetrics if key regulatory developments unfold as he expects later this year.

Accumetrics says more than 80 million Americans have been diagnosed with one or more types of cardiovascular disease. The company says about 50 million Americans regularly take aspirin for its renowned anti-clotting benefits, and another 29 million take clopidogrel. But Accumetrics says the effect of such drugs can vary—some people show an inherent resistance—so that as many as one-third of the patients taking anti-clotting drugs are not getting the full intended effect.

So exactly how much of the intended effect is a patient actually getting?

Accumetrics makes an automated diagnostic instrument called VerifyNow, and replaceable test kits that are used to measure a patient’s individual response to anti-clotting drugs. The cost of the desktop unit is about $8,000, according to Still—a daunting price. Test cartridges are an additional cost, although Still says that is reimbursable and codes have been assigned by the American Medical Association to facilitate billing Medicare and health providers. (Still says the reimbursement rate for the clopidogrel test is $63 a cartridge). Accumetrics also offers other assays for measuring the effectiveness of other specific drugs on platelets, including aspirin, abciximab (ReoPro), and eptifibatide (Integrillin).

Still says Accumetrics’ diagnostic tests can help doctors adjust the dosage of the anti-platelet drugs they are prescribing to reach … Next Page »

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Molecular Insight Pharmaceuticals (NASDAQ:MIPI) reports today it has a new president and CEO, Daniel Peters, who was previously president of the diagnostics division of GE Healthcare. Peters takes over the chief executive role from Molecular co-founder John Babich, who is staying with the Cambridge, MA-based firm as chief scientific officer in charge of research and development. Molecular is developing an imaging agent, Zemiva, for diagnosing lack of blood flow to the heart, or cardiac ischemia.

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Biotech firm Acusphere says that it plans to move out of its corporate headquarters in Watertown, MA, and cut 40 jobs or about two-thirds of its workforce to conserve cash as it pursues approval of its imaging agent perflubutane polymer microspheres (Imagify) for detecting coronary artery disease. The company plans to move all of its remaining employees into its manufacturing facility in Tewksbury, MA. The firm has also filed papers with the Securities and Exchange Commission to voluntarily suspend its reporting obligations to the agency. The firm’s job cuts have been added to our Boston Layoff Tracker.

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Isis Pharmaceuticals is getting closer to a milestone few biotechs ever reach-profitability. The Carlsbad, CA-based company, through its strategy of forming partnerships with pharmaceutical companies that want a piece of its inventions, was able to build up a war chest of $491 million at year’s end. Isis also trimmed its net loss last year to a relatively skinny $12 million.

The company has been around 20 years, long after many biotech companies wear out their welcome, and it still hasn’t gotten into the black permanently. But the financial picture at Isis looks quite a bit brighter than it did a year ago, based on the fourth-quarter and year-end report it issued this morning. Isis has more than twice as much cash as it did a year ago ($193.7 million) and narrowed its annual loss from the $136 million hit it took in 2007.

The big difference last year came via a huge partnership in January 2008 with Cambridge, MA-based Genzyme to co-develop a cholesterol-lowering drug called mipomersen. That deal brought in $325 million upfront, and it could generate a lot more if the drug has success in the final stage of clinical trials, which should start showing results this year. Another recent coup for Isis, the sale of its diagnostics subsidiary to Abbott Laboratories, brought in $175 million in cash last month, and will help keep the company’s losses down in the low-to-mid $20 million range for 2009. The company didn’t say what its financial models look like for 2010 and beyond, but simple math suggests means that its cash reserves ought to last a long time when its losses have been cut this low.

“We believe that we are now at the beginning phase of sustained financial strength,” said Lynne Parshall, Isis’ chief operating officer and chief financial officer, in a statement.

In the midst of a recession, that kind of growing stability has to be good news for the roughly 300 employees at Isis, as of its last official count in March. It’s certainly looking like a more solid place to be than a lot of other San Diego companies we’ve been writing about lately, who are making big layoffs to hold onto the cash they have as the long winter continues.

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