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FierceBiotech's 2008 Fierce 15  

2009-03-04 17:20:46|  分类: Bio-Pharma |  标签: |举报 |字号 订阅

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The 2008 Fierce 15 is brought to you by Quintiles

If you want to know what the future of drug discovery looks like, check out the Fierce 15 Class of 2008. 

At this stage, biotechnology has produced a legion of scientists, entrepreneurs and investors who know how to evaluate a technology's potential and organize a new company pointed to proof-of-concept data. That's never an easy task. As one biotech executive recently told me, launching a new program these days means not just factoring in the drugs that are on the market, but also the ones that are in development. 

It's no wonder that this year's group includes several that early on adopted a stealth mode of operation. The competition in the industry is fierce, and providing too close a look too early can spark competition. And there's always plenty of that to go around. 

As in prior years, many of the companies on the list are closing in on the kind of mid-stage data that can make or break them. They've adopted technologies that are often suited to rapid advancement in the clinic - at least until the program is ready to be partnered out. And they have carefully calculated how much investment capital is needed to reach the next level. 

But there are also a selection of companies that have relied in large part on the determination of a tiny coterie of scientists and financiers whose belief in their program could never be matched by the size of their bank accounts. A few on the list are just getting started, some are sizing up their chances in Phase III. All have outsized hopes for what the future holds. 

As in prior years, of course, not everyone on the list will prove to be as successful as they desire to be. The odds are always steep in biotechnology, even when you think you have all the risks carefully removed. But at a time when big pharma is more reliant than ever on emerging biotech companies to offer promising new pipeline candidates as well as the technology and tools they need to streamline their own discovery engines, you'll also see a few of these companies make headlines as future acquisition targets or IPO-stage companies with commercial opportunities to pursue.   

FierceBiotech will be right there, bringing you the latest news on the industry in a timely fashion. In a matter of weeks we'll pass the 70,000 mark on subscribers, a key milestone for us. Like the Fierce 15, though, we're just getting started.

1. BIND Biosciences

Based: Cambridge, MA
Founded: 2006
Website: www.bindbio.com 

The Scoop: Anyone familiar with the work of MIT scientist Robert Langer knows just how prolific he has been, translating cutting edge science into biotech startups. Bind Biosciences is one of his latest brainchildren, and a familiar group of venture backers are betting that he's helped create another biotech winner--this time using advances in nanotechnology to improve drug delivery.  

What makes it Fierce: For an MIT scientist, Robert Langer has great friends in the venture field. Polaris Ventures has been a regular champion of various Langer ventures, hitting a home run with Momenta. This time around Langer has contributed his scientific know-how to developing new technology that marries polymers and existing therapeutics to deliver drug payloads at key targets. One clinical success here could spell a string of programs. 

Polaris and Flagship combined on a small seed round early on and were joined in

A second, $16 million round by NanoDimension and Arch, another venture firm with a pronounced taste for early-stage ventures exploring advanced technology. That doesn't add up to a tremendous amount of money, by drug development standards, but it should be plenty to get this technology to proof-of-concept. 

What to look for: Bind's science is built for partnering with drug companies that can see an opportunity to further develop proved drugs. That kind of licensing cash can help the founders keep their equity as Bind explores a future that may one day lead to an IPO. 

2. BrainCells

Based: San Diego, CA
Founded: 2003
Website: www.braincellsinc.com

The Scoop: With some impressive venture backing, BrainCells is pushing its lead therapy into a mid-stage study and making a convincing case that it can find therapies that spur the creation of new brain cells--targeting CNS diseases without some of the major side effects that accompany SSRI drugs. The right kind of proof-of-concept data would position BrainCells for quick growth.

What makes it Fierce: Fueled by $50 million in Series B money, BrainCells has taken its lead therapy into a Phase IIa trial, testing its theory that spurring the development of a fresh supply of brain cells can address key CNS conditions--without some of the side effects associated with SSRI drugs. 

But this company is also capable of plenty of surprises. BrainCells recently launched a new, six-week mid-stage study of a combination therapy of two drugs, neither of which had been approved for treating depression. And the company did it without any fanfare, quietly initiating a new program as it steadily builds momentum. 

BrainCells has an intriguing platform for drug development. The technology finds therapies that have the ability to spur the production of new brain cells; neural stem cells that can have a therapeutic affect for a variety of illnesses. Exploring existing drugs for new indications is a rich field in biotechnology these days. And the technology has also been deployed for partners like Lundbeck, which has been scouting novel targets in the field. Organon went to BrainCells to test the neurogenic capacity of their drugs. 

Its venture backing provides the capital needed to in-license more therapies, building up a pipeline of new drug prospects. 

"The asset we've brought in from Taisho (last fall), we would move into the clinic next year," says CEO Jim Schoeneck. "That's the next set of milestones."

What to look for: Schoeneck likes to describe BrainCells as opportunistic. Key trial milestones lie ahead, but look for some surprises along the way. There's also plenty of potential for new partnerships as BrainCells explores the territory. Proof-of-concept data from a mid-stage trial will be a crucial milestone in the company's development. 

3. Cellular Dynamics 

Based: Madison, WI
Founded: 2005
Website: www.cellular-dynamics.com 

The Scoop: Cellular Dynamics has set out to do something unique in the stem cell field: Make money. Working with the scientific know-how of stem cell pioneer Jamie Thomson at the University of Wisconsin, the company is run by a group of Midwestern pragmatists who believe that stem cells can blaze a new path toward more efficient drug development, offering an early look at how human cells will respond to an experimental therapy. And they've actively begun marketing products in a breakthrough for the entire stem cell field.  

What makes it Fierce: Cellular Dynamics is taking a common sense approach to a field dominated by blue-sky thinking. Rather than focus on the remarkable long-term potential that stem cells have for growing new organs or curing some of the world's most complex diseases, the company has begun marketing stem cells as a tool for drug discovery. And that approach can start making money now, rather than years into the future. 

"We're from the Midwest," explains Chief Technology Officer Nick Seay. "We're practical." 

Starting off with funds from the state of Wisconsin, the company is using its stem cell technology to use human cells for drug screening. Instead of relying on animals, whose cells may not react to an experimental therapy the way a human cell would, CDI is offering a smart way for developers to get an early, more accurate look at the safety and effectiveness of their drug candidates. One of its technologies specializes in assessing the potential cardiac risks presented by a new drug program. And the company was quick to adopt IPS cells as soon as their potential was realized.  

"We hope to convince developers to use human cells in vitro in large part in lieu of animal studies," says Seay. 

Helping Seay make the case are some of the top scientists in the field.  

UW-Madison's Jamie Thomson, the scientific founder of the company, was present at the dawn of the human embryonic stem cell revolution 10 years ago. And he's gone into business with two other noted UW-Madison researchers: Craig January and Timothy Kamp. Their stem cell tools are helping advance new programs, giving developers a chance to identify a smarter, faster way to assess their chances in the clinic.  

What to look for: A key collaboration deal with industry-leader Roche should produce important data for the company in just about a year's time. Cellular Dynamics show-me-the-money practicality also has the potential to win over more developers willing to see how the technology can improve their performance in the clinic.

4. Concert Pharmaceuticals 

Based: Lexington, MA
Founded: 2006
Website: www.concertpharma.com 

The Scoop: Armed with a simple understanding that strategically replacing hydrogen molecules in therapeutics with deuterium can improve a drug's performance, Roger Tung created Concert Pharmaceuticals and raised $95.5 million in venture funds in less than two years, making believers of some of the most successful start-up artists in biotechnology. Now it's time to deliver the data. 

What makes it Fierce: Roger Tung isn't the only scientist to explore the potential for deuterium. But he's shown just how much an individual with keen scientific insight--along with a few good contacts in the venture field--can accomplish. He has swiftly banked enough money to get to the proof-of-concept stage, developed a platform approach to drug development that can spawn new clinical candidates as well as partnerships, and pushed hard to get it all up and running in a short period of time. 

In many ways Tung represents the potential biotechnology has to swiftly breed new companies and advance fresh ideas. He's taken an approach that allows the company to develop a portfolio of drug candidates, rather than put all of its eggs in one basket. And he's recruited more than 40 staffers to make it a reality. 

Concert's lead therapy is a deuterium-enhanced drug for hot flashes, a potential blockbuster in a field that has been roiled by safety issues now surrounding hormone replacement therapy. Phase I data from an initial trial should be in by the end of this year with Phase Ib data on drug-drug interactions arriving around early 2009.

What to look for: Concert Pharma got off to a fast start by focusing on simple alterations that can deliver significant upgrades in therapeutics. Now it can start to partner with other developers to advance a variety of programs as it stays focused on delivering data for its own clinical candidates.

5. Constellation Pharma 

Based: Cambridge, MA
Founded: 2008
Website: www.constellationpharma.com 

The Scoop: Constellation Pharmaceuticals is a new player in an embryonic field: epigenetics. This new class of drugs promises to play a major role in regulating gene expression. With three of the top scientists in the field providing the brain trust, a solid lineup of leading venture investors supplying the money and a well-known biotech entrepreneur at the helm, Constellation is off to a solid start. 

What makes it Fierce: Mark Levin knows a thing or two about starting a biotech company. 

His resume includes a slate of start-ups that includes the founding CEO role at Millennium Pharmaceuticals, where he added to his reputation as an extraordinary deal-maker. Takeda ended up paying $8.8 billion for Millennium. 

Now a partner at Third Rock Ventures, Levin is taking his expertise in biotechnology and employing himself as the founding CEO of Constellation Pharmaceuticals, which bills itself as the one and only independent biotech now mining the potential of epigenetics. 

The technology is still in the early stages, but a few big pharma companies have been exploring the science; examining the role certain enzymes play in orchestrating the way that DNA activates genes. By shutting target genes on or off, the scientists can play a key role in controlling the mechanism of disease, and Constellation is out to gin a lineup of preclinical candidates. 

Constellation has all the hallmarks of the kind of biotech company that Levin likes: A big platform technology that promises to work as a "very broad product engine." 

Oncology is the first disease category on the radar. 

"Cancer is an area that allows us to do preclinical work and get to the clinic more rapidly," says Levin. "And all the pharma companies are interested in it." With a start-up staff of nine, the company has already selected 10 targets and started screening small molecules that can go forward in the clinic. By the end of this year, Levin will recruit a whole team of senior and junior staffers to take the company forward, using the next two or three years for animal studies and planning to be in the clinic in three to five years. 

Academic collaborations will be struck for much of the biology work that needs to be done. "And then we have begun discussions with pharma companies; these are just beginning right now. Pharma companies have had a tremendous interest," he adds, predicting that the fledgling outfit can forge its first pharma partnership next year. 

Levin is quick to confirm that an early collaboration is likely to produce less money than he would see for a mid-stage program, but collaborations now can gin money without diluting the founders' equity and it also offers an early avenue to tackling other promising disease categories. Meanwhile, the company has $32 million in the bank from a Series A--Third Rock Ventures, The Column Group and Venrock co-led the round--that Levin estimates will fund operations for three years. 

An impressive figure in biotech, Levin has surrounded himself with a circle of equally impressive venture and scientific co-founders. Danny Reinberg, Ph.D, professor of biochemistry at the New York University School of Medicine; Yang Shi, Ph.D., professor of pathology at the Harvard Medical School and David Allis, Ph.D., professor and head of the laboratory of chromatin biology at The Rockefeller University are all on board. 

What to look for: Constellation is a start-up; everything lies ahead. This year, it builds staff and pursues talks about collaborations that may bear fruit next year. But Levin says he's building the company for the long run, so the biotech either makes it big--or not. 

6. CytImmune Sciences

Based: Rockville, MD
Founded: 1988
Website: http://www.cytimmune.com/ 

The Scoop: Nothing about this company fits the mold for a successful start-up. Its research relies on a toxic material to fight cancer. The biotech has been largely funded by grants or small investors. It has a tiny staff and huge aspirations. But Lawrence Tamarkin, PhD, has never given up, and now he's preparing for a Phase II trial. If he's right, CytImmune's nano-strategy is golden.

What makes it Fierce: CytImmune is all about using gold nanoparticles to deliver a payload of a very toxic cancer therapy--tumor necrosis factor--right in the tumor, where it can do the most good and the least harm. 

"The doses we were able to give in clinical trial exceed the previous maximum tolerated does by threefold," says Tamarkin about his lead program, CTT-6091. "Even more interesting, the circulating exposure to TNF is 30-fold higher to what was previously experienced with TNF alone with no toxicity." 

Tamarkin has good reason to be excited by the positive end of Phase I and the prospective launch of Phase II in September. CytImmune has been heading to this point on a shoestring budget, its tiny staff fuelled largely by angel investors, grants and his own money. While the headlines in the industry focus on major collaborations and monied partnerships, Tamarkin has subsisted largely on his own belief that he could be on to something big.   

"A lot of people have given up on TNF," says Tamarkind. "This is a very potent therapy that has some serious questions about its safety." But that's all the more reason to be cheered by a successful safety study.  

There's very little space between success and failure for CytImmune. Rededicated as a therapeutics company in the mid-90s, CytImmune now is closing in on a major milestone in its development. 

"A year from now," says the CEO, "we should have a reasonable sense of whether we have something that dramatically changes the way we treat cancer, or not." 

What to look for: Phase IIb will be a big influence on CytImmune's future. Tamarkin says that good data at the mid-stage level will set up partnerships for Japan and Europe. New money could propel the company forward, eventually leading to a possible IPO.

7. Genocea Biosciences 

Based: Cambridge, MA
Founded: 2006
Website: www.genocea.com 

The Scoop: Working out of his lab, Harvard Medical School's Dr. Darren Higgins set out to create a faster, cheaper, better way to develop new vaccines--mimicking the human immune system in vivo in order to swiftly zero in on the antigens most likely to elicit an immune response. Higgins is one of the scientific co-founders of Genocea Biosciences, which in a breathtakingly short time has won a series of kudos from experts in the field and may well change the way vaccines are developed.  

What makes them fierce: Genocea is all about speed. 

"The core value proposition," says President Robert Paull, is to take an average time period for finding the right antigen for a vaccine--which now can take years--and do the work in a matter of weeks or months. 

The technology lends itself to the collaborative approach, while giving the company a chance to develop its own pipeline of products. And the CEO says that Genocea is also likely to partner with delivery and adjuvant companies as well. 

There's also a lot he's not telling. The head count is "in the double digits" and the lead program is being kept under wraps for now. But the company is still gearing up, backed by Lux capital and Polaris Venture Partners. Paull is a Managing Partner at Lux.  

He is not thinking small.  

"Genocea a great IPO candidate," he says. "It's the only game beyond Intercell in Austria (another vaccine developer). And Intercell is a $1.5 billion publicly traded company. 

"Pandemics aren't a focus, but the technology is suited to rapid response to infectious disease outbreaks," adds Paull. "You can screen a diverse population to tease out antigens that are the most common to them. The technology is suited to rapid response," he adds, and that could have a lot of applications for the defense industry and beyond.  

What to look for: Genocea will double its staff in 18 months and probably raise another venture round in a year's time. Look for licensing deals and proof its technology is as fast as they claim it is. 

8. GlobeImmune 

Based: Louisville, CO
Founded: 2003
Website: www.globeimmune.com 

The Scoop: "This year will put everyone at GlobeImmune to a crucial test. It has three mid-stage trials underway, unusual for any small biotech. But a major venture round last fall--its third--has provided the biotech enough money to see if its drug platform--engineering a T-cell attack on diseased cells--can produce the kind of definitive data that big pharma is after."

What makes it Fierce: GlobeImmune's third round last fall was its biggest: $41.2 million that brought its total venture haul to $88 million. Wexford capital led the round and Celgene, Boston Life Science Venture, Morgenthaler Ventures and Biogen Idec all participated. 

The money takes the company to a crucial inflection point: Phase II data that determines if the company's platform technology can deliver the kind of T-cell response it is looking for. The biotech has three mid-stage studies underway to determine if its genetically modified recombinant yeast can induce T-cells to attack pancreatic cancer and hepatitis C. The therapeutic potential of T cells are a major focus right now in biotechnology.  

GlobeImmune has been developing Tarmogens--Targeted Molecular Immunogens--based on scientific research in-licensed from the University of Colorado. And its development programs in divergent areas like hep C and cancer underscores just how broad a range the company could have. One successful program could lead to several more. Enrollment for the hepatitis C Phase II was wrapped in May. The study for pancreatic cancer is underway and a Phase IIa in non-small cell lung cancer--enrolling patients with a ras mutation--was launched in March.  

What to look for: The proof is in the data. A successful mid-stage trial sets the stage for broadening the company's research activities, is likely to excite new partnership interest and also moves the company one step closer to evaluating its worth in a potential IPO. 

9. NanoBio Corp.

Based: Ann Arbor, MI
Founded: 2000
Website: www.nanobio.com  

The Scoop: The University of Michigan's Dr. James R. Baker Jr. established a reputation long ago for groundbreaking scientific research. Now he's gaining a reputation as a biotech entrepreneur, building NanoBio around his nanoelmulsion technology and advancing new therapies for everything from cold sores and nail fungus to methicillin-resistant Staphylococcus aureus.  

What makes it Fierce: For a scientist, James Baker certainly knows how to whet someone's interest in clinical data. 

"Our clinical data looks better than any other topical that's ever been developed," says Baker as he discusses the mid-stage results for NanoBio's lead products. This fall the rest of the world will get a detailed look at just how far advanced the company has taken its nanoemulsion technology. In the meantime, NanoBio is making it clear that the small University of Michigan-spinout is hitting its stride.   

In March, NanoBio wrapped a $30 million financing round from Perseus after hitting goals for demonstrating the safety and efficacy of its two lead therapeutics and successfully completing an animal study on its influenza vaccine. That new money came after NanoBio had raised $31 million in angel investments and grants to get started. 

NanoBio is developing topical lotions -- NB-001 for herpes labialis (cold sores) and NB-002 for onychomycosis (nail fungus).  Phase IIa and IIb trials for NB-001 are complete and a mid-stage trial of NB-002 is scheduled to wrap up in the first quarter of next year. An interim analysis revealed "promising results," but NanoBio is still keeping the data confidential. And safety data has confirmed that high concentrations of the lotion can be applied safely. 

The University of Michigan spinout uses nano-scale particles to create therapies that can fight bacteria, viruses and fungi while the same technology platform offers a new, intranasal approach to such diseases as anthrax, influenza and hepatitis. 

"We are planning to initiate a Phase III for the herpes product by the end of the year," says Baker. "The data is so robust that a single Phase III could potentially lead to an NDA. We could have an NDA submitted by late 2009." 

What to look for: Partnerships make a lot of sense for NanoBio and should come relatively quickly. Baker says that Perseus is ready to supply funds as needed, and the founders are looking to grow the company as a standalone biotech. 

10. OncoMed Pharmaceuticals 

Based: Redwood City, CA
Founded: 2004
Website: www.oncomed.com 

The Scoop: OncoMed is focused on one of the hottest areas of oncology--preventing cancer stem cells from causing deadly havoc. A huge pharma pact with Glaxo could set the stage for a number of follow-up deals for a company that has already fueled its growth with more than $100 million in venture funds. OncoMed is shedding its stealth mode and has a lot to talk about.

What makes it Fierce: Up until recently, OncoMed has specialized in keeping its scientific progress under wraps. A huge pact with GlaxoSmithKline last December only partially introduced the developer. But with a leading antibody ready for the clinic later this year and a large amount of IP work completed, the biotech is starting to come out of its shell. 

Stealth mode has been essential, says CEO Paul Hastings. "We've been consciously remaining stealth," he notes. "The company is only three-and-a-half years old, but we were in such a hot area."  

The company's scientific founder, Michael Clarke, has been a pioneer in cancer stem cell research, offering key insights into the critical role they play in metastasis. Finding antibodies that could interfere in that process could create a slate of blockbuster therapies that have the potential to have an impact in a variety of cancers.  

GlaxoSmithKline got a close look at the company's work and wound up forging a $1.4 billion pact to win licensing rights to four antibodies. OncoMed never has revealed just how much of that was upfront, but Hastings tells FierceBiotech it was "multiple" millions.  

The company won't be short of money. A Series A netted $17 million. But Hastings is now ready to discuss a Series B--undisclosed at the time--which brought in a whopping $86 million. And Glaxo's not the only big pharma company to come calling. 

"Last year, when GlaxoSmithKline partnered with us, there were many others interested in that. I think we're going to have lots of options." 

"I would love to take OncoMed all the way," says Hastings. In the meantime, there are lots of ways to create value at the company, and that's part of his job description. 

What to look for: An IND later this year will complete the company's coming out. After that, the nature of its antibody work will create new partnership opportunities. Hastings says that the company now has the financing in place to continue its work into 2011, and that will give OncoMed plenty of time to asses new partnerships - perhaps even enough time for the public markets to turn around. 

11. Relypsa

Based: Santa Clara. CA
Founded: 2007
Website: www.relypsa.com 

The Scoop: An experienced, focused team of developers has put Relypsa back on the same track Ilypsa was on before it was bought out by Amgen. The blueprint calls for a rapid turnaround on a new set of mid-stage data and the kind of compelling data that made Ilypsa attractive.

What makes it Fierce: The Relypsa team, now headed by Gerrit Klaerner, PhD, is experienced at this work and focused on gaining rapid results.  

After Amgen bought out Ilypsa and cherry-picked its lead therapy, the same team that made the company worth $420 million was spun out into a look-alike, sound-alike  biotech named Relypsa. And the new company hopes to advance much the way Ilypsa did, moving a lead therapy into Phase IIa (expected in the third quarter) while another program moves into the clinic. Within two years, with the right kind of mid-stage data in a program ready for late-stage testing, Relypsa could look a lot like Ilypsa did before Amgen bought the company out.   

"We have met or exceeded expectations on our lead program," says COO Gerrit Klaerner, PhD, who says the development team got into the clinic ahead of schedule.   

Relypsa is developing nonabsorbed polymeric drugs for renal and cardiovascular diseases. Because the drugs move through the GI tract and are cleared from the body before hitting the blood stream, the therapies can eliminate side effects seen in existing drugs. The lead program--RLY5016--is a potassium binder for hyperkalemia that offers a safer cardiovascular profile. And the developer says it can also advance these new therapies at a faster pace than the average biotech program.  

Simple. But then, what's wrong with simplicity? 

"I take simple any day," says Klaerner. So do the venture capitalists. Relypsa re-launched with $33 million from 5AM Ventures, New Leaf Venture Partners, Sprout Group, Delphi Ventures, CMEA Ventures and Mediphase Venture Partners. 

A Phase IIb trial next year would test the theory on a population of CHF patients. 

What to look for: In 18 to 24 months, if all goes according to plan, Klaerner says that Relypsa would be on the verge of a Phase III trial and have another program in the clinic. That's a "one-two punch" that the COO sees setting up a knockout "liquidity event." Depending on the size of the IIb trial, Relypsa may look for a relatively small financing round next year. 

12. Regulus Therapeutics 

Based: Carlsbad, CA
Founded: 2007 
Website: www.regulusrx.com 

The Scoop: Spawned by two powerhouses in the RNA field--Isis Pharmaceuticals and Alnylam Pharmaceuticals--Regulus Therapeutics got started at an advanced stage in the emerging microRNA field. And the company was quick to capitalize on its hefty IP with a $600 million pact with GlaxoSmithKline. Anyone tracking microRNA drug development should pay close attention. 

What makes it Fierce: For a small, newly minted biotech company, Regulus has some very big allies. Alnylam Pharmaceuticals and Isis Pharmaceuticals spawned the mcroRNA developer and gifted it not only with years of IP but a backup group that can lend assistance to Regulus as it advances new therapies in the pipeline. 

That's something that GlaxoSmithKline understood early on; another reason why the pharma giant was willing to commit to a $600 million development deal with the startup. 

"It surprised even me that the deal went so quickly," says CEO Kleanthis Xanthopoulis, who joined the company last December. "I don't recall a company that was formed and so early on was able to conclude a $600 million deal. It reflects the excitement in the pharma industry in a very new company with years of research." 

About 500 microRNAs, a new category of RNA, have been identified that regulate the expression of some 30 percent of all human genes. Understanding how that works has the potential to deliver a range of new therapies covering multiple disease categories. 

Regulus' lead therapy targets miR-122 in the quest for a new anti-viral drug for hepatitis C. Other programs are being developed for cancer, metabolic diseases and inflammatory conditions. The collaboration with Glaxo is focused on developing four new therapies for rheumatoid arthritis and other inflammatory conditions. 

The founders are clearly set on creating a new player in the biotech industry with an independent future. 

"We want to build a company that discovers, develops and selectively commercializes microRNA therapeutics," says Xanthopoulos. "That's the bigger goal. We are not building the company for an acquisition." 

What to look for: Regulus says the Glaxo deal was just the first. More pacts are expected in the next year or two.  At the same time, Regulus plans to add two more development programs to its pipeline. That expansion will drive more hires, as the company grows to up to 45 employees by the end of '09. In 12 to 18 months, look for a new financing round. 

13. Resolvyx Pharmaceuticals 

Based: Bedford, MA
Founded: 2005
Website: www.resolvyx.com 

The Scoop: Hoping to take a page from Sirtris' successful play book, Resolvyx is taking an active ingredient from nature and engineering it into a therapeutic with a long lineup of potential applications. And it has some of the world's top scientists advising it. Researchers are off to a fast start on the way to proof-of-concept data.  

What makes it Fierce: Platform technologies are favored by a whole host of venture capital firms, and that's one reason why Resolvyx has successfully captured a lineup of A-list investors that includes Biogen Idec.  

The biotech company is advancing the scientific work of Charles Serhan, an investigator at Brigham and Women's Hospital. His insight that resolvins, derived from omega-3 fatty acids, could play a role as a key mediator for inflammatory conditions has earned $42 million in the first two rounds for the company. And the tonic nature of resolvins has been compared to resveratrol in red wine, which has made Sirtris' fortune. 

Proof-of-concept data is expected to start flowing this year, and the company has the capital to keep it pumping into the third quarter of next year, when it will have plenty of insight into the efficacy of resolvins for eye disease and systemic inflammation. And researchers have highlighted early indications of efficacy for wet, age-related macular degeneration, raising the possibility of a topical application.  

Success at this stage would open up new research opportunities in a host of disease categories, including programmed cell death. 

What to look for: Mid-stage data. If Resolvyx can make a convincing case for resolvins, the versatility of the drug platform opens up new prospects for collaborations and keeps investors coming back for more. There could even be an IPO in the cards, if the markets and the data can align. 

14. Santaris Pharma

Based: Copenhagen, Denmark
Founded: 2003
Website: www.santaris.com 

The Scoop: Santaris has been breaking new ground with its advancement of a microRNA therapy into the clinic in late May. A successful proof-of-concept trial for hepatitis C is being billed as a potential medical breakthrough, offering a key to modulating networks of genes.  

What makes it Fierce: Before Regulus was born last fall, Santaris was already making headlines in the microRNA space. Its oligonucleotide drugs target both messenger RNA and microRNA. In '06 and '07 the Danish biotech inked deals with Enzon and GlaxoSmithKline which start small and wind up big. On top of that there was a $30 million Series C last December, which followed a $48 million Series B. 

The company is moving fast, given the complexity of the work. Santaris launched the first clinical trial of a microRNA therapy--SPC3649--in late May, with the same target, microRNA-122 for hepatitis C, that Regulus has focused on with its lead program.    

And the company is aggressively pushing forward into a Phase II trial of 2996 for Rituximab-relapsed lymphoma. 

Santaris now has three products in the clinic and expects to add a fourth by the end of this year, says CEO Keith McCullagh. Add to that two programs in preclinical development and 10 or 11 in discovery and you have a full plate for the more than 100 employees of the company. 

"Every drug is targeted at a precise RNA sequence in messenger or microRNA," says McCullagh. For McCullagh, the whole field offers an opportunity that can one day rival monoclonal antibodies. And that message has not been lost in the pharma world. 

"We are always talking to big pharma and other companies about collaborations," says McCullagh. "I'd be disappointed if we didn't do another deal this year. No biotech company can fund this work out of its own resources. Alliances are an important way to build that pipeline." A deal like the one they struck with Enzon also reserves marketing opportunities for the company. "We would love to do similar deals with other companies." 

What to look for: New collaborations are in the works. Phase II data will be a major milestone for Santaris as it advances a variety of trials.

15. Tolerx 

Based: Cambridge, MA
Founded: 2000
Website: www.tolerrx.com 

The Scoop: A rich partnership pact with GlaxoSmithKline last October kept Tolerx in the race to develop an anti-CD3 monoclonal antibody for autoimmune disease. That program is now headed into Phase III, and all the chips are on the table.

What makes it Fierce: Anyone looking to strike a partnership deal would do well coming even close to Tolerx. Within days of the news that MacroGenics had struck a billion-dollar deal with Eli Lilly for its program to develop a monoclonal antibody for immune disease, Tolerx struck back with a $760 million pact in the same space.    

Tolerx is at a crucial stage of its development. An end-of-Phase-II meeting with the FDA went well, setting up a late-stage trial for otelixizumab--a monoclonal antibody that binds to CD3--in new-onset type 1 diabetes.  

"In the meeting with the FDA March 20 we discussed not only the Phase I and II program, but also the requirements for a BLA filing," says CEO Douglas Ringler. "That all went extremely well and now we're actively enrolling in the U.S. We intend to get other European and Canadian sites up by the end of summer." 

Tolerx has good cause to be working with a sense of urgency. Ringler knows only too well that the company has strong competition for its development program. The deal with GlaxoSmithKline also has a crucial co-promotion arrangement for the U.S. market. And the company is working to create a commercial infrastructure as it expands its pipeline and starts making post-BLA plans. 

Tolerx also hit a key milestone in its development pact with Genentech when it announced in January that TRX1, an anti-CD4 monoclonal antibody, completed a Ib trial in subjects with refractory cutaneous lupus erythematosus. New MAbs are in preclinical development. 

When FierceBiotech spoke with Ringler in early June, the company had 55 staffers. That figure should rise to 75 by the end of the year, including a new hire for a senior marketing executive.  

What to look for: Late-stage data will mark a key turning point for Tolerx, which has enormous potential advancing a new diabetes drug. It's all about executing on the plan, says Ringler. So far, the company has attracted more than $118 million in financing from investors. A successful Phase III would offer a big reward. 
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