drug candidates

Acadia shares plunge on mid-stage trial failure

John Carroll — Mon, 16 Jun 2008 09:26:07 -0400

Shares of Acadia Pharmaceuticals went into free fall this morning after the company announced that one of its schizophrenia drug candidates failed a mid-stage trial. Researchers said that ACP-104 was no better than a placebo in relieving symptoms of schizophrenia. And the company said that they would probably shutter the development program as a result of the data.

That news sent Acadia's stock into a nose dive, with shares losing 56 percent of their value this morning. Acadia does have another schizophrenia drug--pimavanserin--in mid-stage development.

"We clearly are disappointed in the results of this study," said Uli Hacksell, Ph.D., the CEO of Acadia. "While we will thoroughly analyze the data to understand the outcome, we currently do not anticipate conducting further studies with ACP-104."

- check out the press release
- read the AP report

Alexza panic drug fails proof-of-concept trial

John Carroll — Tue, 10 Jun 2008 11:56:02 -0400

One of Alexza Pharmaceuticals' drug candidates failed a key proof-of-concept trial, sending its stock into a swoon. The Phase IIa trial failed to meet either of its primary endpoints for AZ-002: its effect on the incidence of a doxapram-induced panic attack and on the duration of a doxapram-induced panic attack compared with placebo. The drug is being developed in collaboration with Symphony Capital. Shares of Alexza slid 8.5 percent in after-hours trading.

"The failure to reach statistical significance on the primary endpoints is disappointing, but this proof-of-concept clinical trial was designed to capture data and measurements for a number of clinical parameters in this clinical setting," said James V. Cassella, PhD, senior vice president, research and development at Alexza. "There is no FDA-approved therapy for the acute treatment of a panic attack or an established clinical development pathway for this potential new indication. We plan to work with our colleagues at Symphony Allegro and our external clinical experts to further analyze the data from this study to determine our next steps with AZ-002."

- check out Alexza's release
- read the report from the San Jose Mercury News

ALSO: Alexza Pharmaceuticals has completed enrollment of its first Phase III clinical trial with AZ-004, an inhalation product candidate being developed for acute agitation in patients with schizophrenia or bipolar disorder. Release

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Four promising companies to watch
Alexza gains $50M

SPOTLIGHT: Anesiva inks $114M licensing deal

Thu, 05 Jun 2008 06:59:53 -0400

Transcription Factor Therapeutics has licensed the worldwide rights to Anesiva's NF-kB Decoy program, which includes the clinical drug candidate Avrina. NF-kB Transcription Factor Decoy inhibits NF-kappa B, a protein implicated in the inflammatory cascade in diseases such as inflammatory bowel disease (IBD), eczema, rheumatoid arthritis, and asthma. Anesiva gets an upfront fee and up to $114 million in milestone payments if two products are developed. Release

SPOTLIGHT: Sunesis cuts 60 jobs

Wed, 04 Jun 2008 06:59:53 -0400

Sunesis Pharmaceuticals is realigning to focus on the development voreloxin (SNS-595), its lead drug candidate. The company is expanding activities around late-stage development of the drug and reduced its workforce by 60 percent. The plan includes de-emphasizing research work. Sunesis said the move concentrates the company's assets "on the strongest path to potential near-term value creation for the company's stockholders." Release

Lexicon cuts staff, focuses on R&D

Wed, 28 May 2008 06:59:57 -0400

Lexicon is reorganizing and cutting staff as it pursues its aggressive strategy to build its pipeline. The cuts will allow Lexicon to devote its resources to its strategic, business, and clinical development initiatives. Lexicon didn't discuss numbers but said facilities in Texas and New Jersey would feel the brunt of the cuts. Lexicon expects the move to reduce its expenses by $4 million for the balance of 2008.

"This reorganization is a significant milestone in the process we began in early 2007 to transform ourselves into a biopharmaceutical company with a pipeline of innovative drug products that derive from our unique insights into the human genome,'' said CEO Arthur Sands. Lexicon's 10 to 10 program is an initiative with the goal of advancing 10 drug candidates into human clinical trials by 2010. He added, "This action will reduce operating expenses associated with our research and discovery efforts and allow us to deploy these resources into key clinical development areas, including our four programs currently in human clinical trials.''

- read the announcement from Lexicon

Emergent BioSolutions buys flu vaccine maker

Tue, 27 May 2008 06:59:56 -0400

Emergent BioSolutions has inked a deal to acquire the Protein Sciences Corporation assets for up to $75 million. The move is part of Emergent's strategy to expand its product portfolio with a drug candidate focused on infectious disease. Currently, most of Emergent's business comes from government contracts for biodefense. Emergent will acquire PSC's ongoing operations, including FluBlok, a Phase III recombinant influenza vaccine candidate, and certain other assets. "The acquisition of FluBlok is consistent with Emergent's strategy of expanding its product portfolio with a vaccine candidate focused on a major infectious disease," said Fuad El-Hibri, Emergent's CEO. The company recently bought rival VaxGen's anthrax vaccine business.

- check out Emergent's release
- read the Washington Post article

Antisoma buys Xanthus for $52M

Fri, 16 May 2008 06:59:56 -0400

Looking to boost its pipeline, the U.K.'s Antisoma is paying $52 million for Boston oncology company Xanthus Pharmaceutical. Antisoma gains three drug candidates:

Xanafide, which is in Phase III trial in secondary acute myeloid leukemia,
Oral fludarabine, which has been submitted to the FDA for marketing approval for the second-line treatment of chronic lymphocytic leukemia, and
the Flt3 program, which is in preclinical testing for non-oncology indications. Antisoma says it may out-license this drug.

The deal lessens Antisoma's dependence on ASA404, a non-small cell lung cancer treatment. Last year the company announced it would pit the drug against Roche and Genentech's Avastin in a late-stage trial. Glyn Edwards, CEO of Antisoma, said: "This is a transforming deal. Combining Antisoma and Xanthus produces a company with the critical mass and mature pipeline needed to become a major player in oncology."

- here's the release on the deal
- read the MarketWatch article for more

SPOTLIGHT: Sangamo targets gene manipulation

Thu, 15 May 2008 06:59:53 -0400

Forbes has highlighted the development work of Sangamo BioSciences, which has a "hefty" pipeline of drug candidates in development that use zinc-finger DNA binding proteins to manipulate genes for a number of diseases which currently can't be treated. Report

Merck turns to Ranbaxy for $100M R&D effort

Mon, 12 May 2008 06:59:58 -0400

India's Ranbaxy has flexed its research muscles once again, this time signing a five-year pact with Merck to develop new anti-infectives. Ranbaxy, which received an undisclosed upfront payment, says it stands to earn more than $100 million from each new therapy developed in the five-year collaboration, which follows a similar deal it signed with GlaxoSmithKline. Ranbaxy researchers will advance therapies into Phase II, at which point Merck will take over the development program. Ranbaxy shares jumped more than five percent on the news.

This new deal signals an ongoing effort by big pharma companies to reduce their expenses by shifting development programs into China and India. Eli Lilly has also aggressively pursued an Asian strategy for its R&D work as well.

"We are going to start work on a minimum of two (drug candidates), and we can then add to it later," Ranbaxy CEO Malvinder Mohan Singh told Dow Jones.

- see this release on the deal
- read the report from Dow Jones

Spectrum sells off generic drugs

Fri, 09 May 2008 06:59:55 -0400

Specturm Pharmaceuticals has sold its abbreviated NDAs for its injectable generic drugs, ondansetron, carboplatin, fludarabine, and mitoxantrone, to Sagent Pharmaceuticals. Sagent, which made our list of the top 20 VC deals of 2007, brings finished pharmaceutical therapies onto the U.S. market and is focused on injectable products. "The sale of these non-core assets provides additional non-dilutive funding which will be used to further fund the advancement of our late stage clinical programs," said Rajesh C. Shrotriya, Spectrum's CEO. Spectrum has 12 drug candidates in the clinic and three more in preclinical development.

- check out Spectrum's release

Roche expands Memory pact

Fri, 02 May 2008 06:59:55 -0400

Roche has exercised its option to further develop and commercialize Memory Pharmaceuticals' drug candidate, MEM 3454, for various neurological and psychiatric disorders. Memory gets a $6 million milestone payment and will be eligible for a $17 million once an ongoing Phase IIa study in cognitive impairment associated with schizophrenia (CIAS) is completed. Back in November, Memory Pharmaceuticals announced a positive Phase II proof-of-concept trial of MEM 3454 in 80 patients with Alzheimer's disease.

"We are excited that Roche shares our enthusiasm for MEM 3454 and its potential to address the cognitive deficits associated with debilitating CNS disorders," stated Vaughn M. Kailian, Memory's CEO. "We have aggressively advanced MEM 3454 through early-stage clinical trials, including a positive Phase IIa trial in Alzheimer's disease. We believe that Roche's continued commitment to the program, together with its expertise in later-stage clinical development and commercialization, will provide the support and capabilities to realize the full potential of this compound."

- check out this release

ALSO NOTED: Brandt to helm Noven; Cougar launches Phase III; Wyeth cuts 1,200 jobs; and much more...

Tue, 29 Apr 2008 06:59:50 -0400

> Peter Brandt, a senior VP at Pfizer, has been tapped as the new CEO at Noven Pharmaceuticals. Release

> Cougar Biotechnology has started enrolling patients in its Phase III clinical trial of the company's lead drug candidate, CB7630. Release

> And the ax is falling again. Wyeth notified employees last Friday than another 1,200 jobs will be slashed from the payroll. Wyeth report

> Alexza Pharmaceuticals announced positive results from a Phase I clinical trial of AZ-007, an inhalation product candidate for insomnia. Release

> Pfizer is back at the negotiating table with the Nigerian state of Kano in an attempt to finally settle claims about a meningitis drug trial the government contends was conducted illegally. Pfizer report

> Wanted: CEO who can find a new "cash cow" to push a Danish drugmaker past impending patent expirations. Found: Ulf Wiinberg, lately of Wyeth, to take over Lundbeck. CEO report

> Call it a new pricing strategy. Bristol-Myers Squibb is preparing to launch the cancer treatment Erbitux in Canada after a two-year wrangle over cost. Erbitux report

> Pfizer is providing $14.4 million to fund a research consortium involving four universities and Entelos, a company that does computer modeling for drug research work, in a drive to create a new class of diabetes drugs. Report

And Finally... Australian scientists say that targeting angiotensin converting enzyme (ACE) can be an effective way to spur weight loss and prevent diabetes. ACE inhibitor report

Argos garners $35.2M in third round

Wed, 23 Apr 2008 06:59:55 -0400

TVM Capital led a hefty $35.2 million third round of venture capital for Colorado's Argos Therapeutics. The funding will be used to support Argos' Phase II trial of Arcelis immunotherapy drug candidates in renal cell carcinoma and HIV. In addition, it will finance the company's CD83 protein immunology program through an initial human clinical trial in transplantation.

"We believe that the Company's many near-term data events, combined with its ability to move into additional cancer and infectious disease indications with its Arcelis platform technology, as well autoimmune disorders with its CD83 soluble protein, position Argos as a leader in the immunotherapy field," said Hubert Birner, Ph.D., General Partner of TVM Capital and Argos' Chairman. Argos was featured in FierceBiotech's weekly column on emerging drug developers.

- see this Argos release

ALSO NOTED: FDA approves UCB's Cimzia; MacroChem acquires Virium; and much more...

Wed, 23 Apr 2008 06:59:50 -0400

> The FDA has approved UCB's monoclonal antibody Cimzia for the treatment of moderate to severe Crohn's disease. Cimzia's approval was delayed last year when the FDA demanded that UCB conduct another trial of the drug. UCB release

> New York-based Emiliem has signed a licensing agreement with the NIH for the worldwide rights to develop and commercialize a series of compounds that modulate key biological pathways known to be important in the progression of cancer and other proliferative and inflammatory diseases. Emiliem release

> MacroChem has acquired Virium Pharmaceuticals and will issue 23 million shares of common stock to Virium shareholders. Virium has a number of clinical-stage oncology drug candidates. MacroChem release

> FDA Commissioner Andy von Eschenbach found himself in the hot seat again as Congress grilled him about foreign oversight and scolded him for safety lapses that occurred on his watch. Report

> Though drugmakers have been reporting increases to their bottom lines, investors are looking past the foreign-currency gains and one-time earnings boosts to the fundamental problems the industry faces. Report

> Now that its Pristiq antidepressant is finally approved, Wyeth is offering a cut-rate price to woo patients over from Effexor XR. Wyeth report

And Finally... By one estimate, CROs will see their contract research operations swell from $7 billion in 2006 to $19 billion in 2013 as more and more biotech companies turn to them to manage drug trials. But as the work of the CROs grows, so do concerns over the quality of the research work they manage. CRO report

Resolvyx rounds up $25M

Tue, 22 Apr 2008 06:59:55 -0400

Bedford, MA-based Resolvyx has garnered $25 million in a second round of venture capital. QVT Financial LP led the funding round. The money will be used to accelerate Resolvyx's preclinical pipeline development and to advance multiple resolvin drug candidates into clinical trials in 2008. Resolvins are a recently discovered family of naturally-occurring, small molecule lipid mediators that resolve inflammation.

"We are excited about the growing recognition of the value of our unique resolvin platform. Attracting investors of this caliber underscores the significant progress we have made toward breakthrough drug products," said Paul Rubin, M.D., President and CEO of Resolvyx. "This funding allows us to continue to advance our resolvin therapeutic candidates, RvE1 and RX-10045, and to further evaluate additional resolvin and protectin compounds." Resolvyx's first round occurred in 2005, when the company gained $17 million.

- see this release for more

ALSO: Another Massachusetts-based company, Cambria Pharmaceuticals, has closed a $5.4 million Series A financing. Release

ALSO NOTED: FDA approves Orencia; Delay on Amgen clotting drug decision; Icahn files suit for Biogen Idec papers; and much more

Wed, 09 Apr 2008 06:59:50 -0400

> The FDA has approved Bristol-Myers Squibb's Orencia for juvenile arthritis. Release

> Amgen says that the FDA will delay a decision on its blood clotting drug by three months. A decision had been expected by April 23. Release

> Not satisfied with Biogen Idec's explanation of its failed effort to find a buyer, Carl Icahn has filed suit to get a hold of internal documents on the search. No way, responds Biogen Idec, which says it's just another ploy aimed at forcing a sale. Report

> Antigenics has raised $21 million in a private placement. The money will be used to market Oncophage, a cancer vaccine that just won regulatory approval in Russia. Release

> Potentia Pharmaceuticals has completed a $12 million round of financing. The funds will allow Potentia to complete Phase I and move into Phase II clinical development of Potentia's novel investigational drug candidate, POT-4. Release

> Schering-Plough has officially terminated its partnership with Novacea for its Asentar prostate cancer program. A clinical trial was halted earlier due to an imbalance of deaths between the two treatment arms. Release

> The FDA has rebuked GlaxoSmithKline for failing to regularly report post-marketing Avandia data. Report

> Scientists at Memorial Sloan-Kettering Cancer Center in New York City have identified the molecular process by which breast cancer cells spread to other tissue during metastasis. Report

> The heparin death toll is far worse than we originally thought. The FDA has tripled the number of deaths it's attributing to allergic reactions to the blood thinner since January 2007, to 62 from 19. Report

> Just why would Novartis pay $11 billion for one-fourth of eye-care Alcon company and promise another $28 billion for another big chunk? Report

> Expect the FDA to stay out of the Vytorin fray, at least for now. Report

> In the latter part of the 20th century, the conventional wisdom in the drug industry was simple: bigger is better. But now the Big-Big management model is cracking up. Report

And Finally... It turns out that hundreds of bacteria can actually 'eat' antibiotics, according to researchers at Harvard Medical School. Report

Keryx triggers big shakeup to counter trial fiasco

Mon, 07 Apr 2008 06:59:58 -0400

Less than a month after seeing its lead drug candidate go down in flames in a late-stage trial, Keryx Biopharmaceuticals announced plans to restructure, chopping its work force in half as it reduces the company's burn rate. Keryx announced that it will have 25 full- and part-time workers when the restructuring is complete.

Keryx's plans include continuing R&D for Zerenex but cutting 12 of 20 early-stage trials of KRX-0401, delaying a late-stage trial of KRX-0401, terminating a licensing deal and closing offices in San Francisco and Memphis as well as its manufacturing site in Wisconsin. And in pursuit of new capital, Keryx has begun to hunt out new partnerships, strategic alliances and "product-specific financing alternatives." Keryx ended the first quarter of this year with about $50 million.

Keryx's troubles crystallized last month with the late-stage failure of the diabetes drug Sulonex. Sulonex, which Keryx dumped at the end of March, worked no better than a placebo in a pivotal clinical trial.

- check out the press release

Related Articles:
Keryx shares in meltdown after late-stage failure. Report
Keryx inks $100M deal with Japan Tobacco. Report
Keryx sells shares. Report
Keryx drug best at low dose. Report

Quark Pharma snares $27M for RNAi work

Mon, 07 Apr 2008 06:59:55 -0400

Quark Pharmaceuticals has garnered $27 million in a new round of venture financing. The RNAi developer attracted the support of SBI Asset Management and SBI Investment, subsidiaries of the prestigious SBI Holding Inc.

"The additional funding we are announcing today will allow us to significantly expand our clinical program," says Quark CEO Daniel Zurr. "Between our licensee, Pfizer, and Quark, we anticipate having Quark drug candidates in five clinical programs by the second half of 2008."

- read the release

Related Articles:
NovaCardia, Quark Biotech file for $86M IPOs. Report
A billion-dollar lesson in RNAi economics. Report

ALSO NOTED: Dendreon shares jump on investment deal; Iomai in pact with Merck; Foundation antes millions for MS leads; and much

Thu, 03 Apr 2008 07:59:50 -0400

> Schering-Plough has proven quick on the layoff trigger. The company announced a plan to cut costs by $1.5 billion by 2012 in the wake of the latest round of Vytorin controversy. Roughly 5,500 employees will lose their jobs, or about 10 percent of the company's workforce. Report

> Shares of Dendreon jumped seven percent on the news that an institutional investor will buy up 47 million in shares and warrants. That money is earmarked for Dendreon's prostate cancer drug Provenge and other drug candidates in its pipeline. Release

> Iomai has forged a deal with Merck to conduct proof-of-principle preclinical studies evaluating the use of the Iomai needle-free immunostimulant patch. Merck has first option to negotiate an exclusive license. These preclinical proof-of-principle studies will be conducted using an undisclosed Merck vaccine. Release

> The Myelin Repair Foundation has spent $10 million so far to identify 18 new drug leads for multiple sclerosis. Report

> Chutes & Ladders: Akers Biosciences has promoted Robert J. Paratore to chief financial officer. Report

> Two lawmakers asked the FDA yesterday to mandate that all TV ads for prescription drugs include contact info for patients to report serious side effects to the agency. The impetus? A Consumer Reports poll that showed 16 percent of respondents had a side effect serious enough to send them to the doctor or hospital--but 35 percent of respondents didn't know they could report side effects to the FDA. Report

> The U.K.'s National Health Service is spending around $3.9 billion annually to treat patients for adverse reactions to prescription drugs. Report

And Finally... A professor at the California Institute of Technology is walking away with the $1 million Gotham Prize for an original approach to killing cancer cells. Report

Tips for funding a biotech start-up

Maureen Martino — Wed, 19 Mar 2008 14:55:15 -0400

In case you missed it, last week FierceBiotech brought together three experts to discuss tips for the early-stage funding field. As many have noted, there’s been a slowdown in the amount of funding available for seed-stage companies. Despite the fact that biotech companies are snagging a growing share of the billions of dollars in venture capital, most of that is going toward late-stage developers who can offer investors a better bet at a near-term payback. Start-ups, on the other hand, are dealing with a challenging funding environment. Since 1995, early-stage funding has dropped from 45 percent of deals to 26 percent in 2007 (cite the source). And while some early stage money has come back into the market over the last two years, the market is still under-served.

Why the gap? For one thing, the average age of a company before it goes public has increased, which means that investors have to support them for a longer time. And even when they do go public, unfavorable market conditions mean they’re getting lower valuations. Many VCs now regard early-stage investments to be too great of a risk.

However, that doesn’t mean there isn’t a lot of great science coming out of early stage companies, and Big Pharma is starting to pay more attention to early stage development as it seeks to fill its depleted pipelines. And they’re willing to pay more to acquire those targets.

So what can a start-up do to attract much-needed seed money? Daphne Zohar, founder and managing partner of PureTech Ventures, Carl Weissman, president and CEO of the Accelerator Corporation and Barbara Schilberg, managing director and chief executive officer of BioAdvance, shared their tips for attracting funding.

Technology is the foundation of it all. Investors are looking for a unique product--not a “me-too” drug. When asked whether investors prefer funding technology platforms or drug candidates more, all three said they prefer to finance technology platforms that have produced drug candidates. Early-stage investors also look for investments that have the ability to hit a key milestone in one to three years.

There needs to be significant market potential for the drug. Investors are attracted to technologies that fulfill a significant unmet need, and will have a big impact on a large market. Additionally, the type of market matters a lot when it comes to securing funding. It’s very difficult for, say, a cancer drug company to gain venture capital before it’s completed human trials because VCs have been burned in the past by early-stage targets that don’t pan out. On the other hand, Alzheimer’s candidates do well with early-stage funding because Big Pharma companies often acquire early stage drugs in that space. If the development landscape for a company's drug is too crowded, investors will run the other way. But keep in mind that the market a company is aiming for must justify the cost of development. Start-ups must walk a fine line between finding market potential and developing a drug that suits it.

Another area Zohar, Weissman and Schilberg stressed was strong IP (intellectual property) protection. It’s a bad move to publish findings before locking down commercially-driven IP. Also, IP not covering key areas (such as the international market) isn’t attractive to investors, and will only create problems in the long run.

Having respected and recognized experts involved in a company often says more than even the best science. The best minds attract the best investors and management team. They also validate scientific ideas and reduce risk in the eyes of investors. And just as a great management team will validate a product, a lackluster team that lacks relevant experience will send up a warning flag with investors, so avoid bringing in the wrong people at all costs.

There's one final piece of the start-up puzzle, and that's having a back-up plan. The only way a company can insulate itself against the risk of one molecule or program not succeeding is to have a back-up plan. Only about one in 5,000 drugs will make it from discovery to approval. Having multiple programs is the only way for start-up to survive the potential pitfalls of the drug development process.