New England Journal of Medicine

ALSO NOTED: Dyax to close R&D facility;Nastech sells shares; and much more...

Mon, 28 Apr 2008 06:59:50 -0400

> Dyax says it plans to close its research facility in Belgium as it focuses on commercializing DX-88 to treat hereditary angioedema. Dyax report

> Nastech Pharmaceutical plans to sell $8 million in shares to advance its RNAi programs. Report

> North Park Aesthetics says it's reached an agreement with Biophysica, Inc. and Interpharma Praha for the exclusive worldwide rights to develop and commercialize products containing fluridil, the primary ingredient in NP-619, North Park's Phase II product for pattern baldness. Release

> FDA officials say that drugmakers needed to bear the responsibility for drug safety. FDA report

> Will all medical schools bar the door to pharma giveaways? The Association of American Medical Colleges hopes so. Pharma gift report

> In another blow to the Merck/Schering-Plough joint venture, the FDA rejected a proposed allergy and asthma treatment that would have combined Schering's Claritin and Merck's Singulair into one pill. Report

> The Brazilian government recently deemed the anti-HIV med Tenofovir a "public interest." That's code for, "We're thinking about issuing a compulsory license for this drug." Brazil report

> One of the things media love to do is get two people to go head-to-head on an issue. Today's topic: Allowing drugmakers to distribute peer-reviewed journal articles in support of off-label uses for their products. On the pro side is Scott Gottlieb (photo), an ex-FDA deputy commissioner who's now at the conservative American Enterprise Institute. Former New England Journal of Medicine editor Jerome Kassirer is on the con side. Report

And Finally... Indonesia has begun to drill health workers on responding to a human outbreak of bird flu. Article

Cardiome's CBO discusses changes at FDA

Fri, 28 Mar 2008 07:59:56 -0400

During a conference call with investors yesterday, Cardiome's President and Chief Business Officer, Doug Janzen, had some interesting thoughts on the slow approval process at the FDA:

"The FDA has actually come out and said it will be missing (PDUFA) dates... I think everyone is in a brand new environment... actually, some of the old-timers in the industry are talking about we're back in the old environment where the FDA just doesn't deal with timelines. They're missing PDUFA dates, they're missing type-C meetings, they're missing meetings to discuss protocol..."

Janzen's comments stem from the approval delay of Cardiome's intravenous drug vernakalant for atrial fibrillation. Despite being approved by an expert committee, the company has been awaiting FDA approval since the drug January 19th PDUFA date. His observations highlight the ongoing problems at the FDA. The agency is struggling to meet PDUFA dates, ensure food and drug safety, and maintain enough staff to deal with it's ever-growing list of responsibilities.

On the subject of PDUFA dates, however, it seems that an approval delay may be preferable to a hastily-made decision. Just yesterday a Harvard analysis found that that drugs approved in the two months leading up to the deadline are four to five times more likely to be withdrawn or require serious safety warnings. So is it better for the FDA to scramble to meet deadlines--and possibly miss safety problems--or miss the approval date that companies pay hefty fees for the FDA to meet?

- read the Motley Fool piece
- check out Cardiome's financial results

ALSO: Speaking of that study, the FDA says that it can't replicate the Harvard study's numbers. The agency is sending a detailed letter disputing the results to the New England Journal of Medicine, which published the research. Report

Related Articles:
Study: Last-minute FDA approvals less safe. Report
Developers: Politics behind slow approval process. Report
PDUFA debate highlights drug safety issues. Report
The FDA: Caught between a rock and a hard place. Report
FDA quick to reject drugs as it gets more cautious. Report

ALSO NOTED: CFOs are pharma's new power players; Progen axes staff in restructuring; MethylGene inks collaboration deal;and muc

Thu, 27 Mar 2008 07:59:50 -0400

> Australia's Progen is laying off workers in a restructuring that includes outsourcing the commercial manufacture of PI-88. Release

> Who's pharma's power forward these days? The CFO. Or so concludes Ernst & Young after slicing and dicing two global surveys. As companies shift focus from driving sales growth to managing returns, CFOs will have to be increasingly proactive. Report

> MethylGene will get $2 million up front and up to $50.5 million in milestones in its newly signed collaboration deal with Otsuka Pharmaceutical. MethylGene will be responsible for the design, synthesis, characterization and initial screening of kinase inhibitors. Otsuka will be responsible for and fund efficacy and toxicology studies, as well as preclinical and clinical development of compounds. Otsuka is also responsible for the global commercialization of any resulting product. Release

> When FDA reviewers cram, do they perform poorly on the test? A new study concludes that yes, when drugs are approved right on deadline, they're more likely to cause safety problems later. The Harvard analysis, published in this week's New England Journal of Medicine, concludes that drugs approved in the two months leading up to the deadline are four to five times more likely to be withdrawn or require serious safety warnings. Report

> The FDA will allow copper alloys to be sold as a weapon against bacteria and microbes. Report

> Japan's Ono Pharmaceutical has inked a discovery deal with Evotec. The collaboration applies Evotec's fragment-based drug discovery platform, EVOlution, to identify small molecular weight compounds active against a protease target. Release

> Novelos Therapeutics has raised $5 million in a private placement. Release

And Finally... People with larger stomachs in their 40s are more likely to have dementia when they reach their 70s, according to a new study. Release

Theratechnologies explores sale, other options

Tue, 29 Jan 2008 06:59:57 -0500

Theratechnologies has posted a "sale" sign on the company as it announced its intention to conduct a strategic review of its options. At the same time, the Canadian developer said that it is considering raising C$35 million in equity financing to fund its operations. One other point to make on the subject of maximizing shareholder value: Partnership and licensing deals are available for its lead compound--tesamorelin, a late-stage therapy for HIV-associated lipodystrophy.

"Our first Phase III results, as published in the New England Journal of Medicine, indicate that tesamorelin may offer an attractive combination of efficacy and safety with potential advantages over other approaches to treat HIV-associated lipodystrophy," touted CEO Yves Rosconi in a release.

- see this release
- here's the report from Canada East

Related Articles:
Theratechnologies reports positive Phase III results. Report
Late-stage HIV drug trial delivers positive data. Report

Studies inflating antidepressant efficacy?

Fri, 18 Jan 2008 06:59:54 -0500

Yesterday we reported on an article published in the New England Journal of Medicine that found studies with positive results were far more likely to be published than those with negative results. The study found that the FDA is working with raw data, whereas medical journals are not, which can contribute to misleading results about the efficacy of certain drugs, particularly antidepressants. And the folks over at the blog Clinical Psychology and Psychiatry have an in-dept look at exactly which antidepressants may have inflated benefits due to data suppression. The list includes Wellbutrin, Celexa, Cymbalta, Lexapro, Prozac, Remeron, Serzone, Paxil, Zoloft, and Effexor--every antidepressant approved between 1987 and 2004.

- see this blog entry for more

Related Article:
Positive data more likely to find its way to public. Report
Top 10 drug warnings and recalls of 2007: Antidepressants

Positive data more likely to find its way to public

Thu, 17 Jan 2008 06:59:56 -0500

There's been a growing understanding in the drug development world that studies boasting of positive data on a drug tend to get published in the scientific literature with great fanfare; negative studies are often silently allowed to sink out from sight. Now a new report in the New England Journal of Medicine concludes how that trend has played out in the antidepressant field. Ninety-four percent of studies with positive drug data showed up in print, compared to 14 percent of the studies with negative or inconclusive results. And that has skewed the reported effectiveness of the drugs. If you combine the negative studies on antidepressants they were able to outperform placebos by only a modest amount, not the 60 percent in published trials that demonstrated a positive response.

That's not likely to be news to practitioners in the field. Antidepressants have long been notoriously unreliable. But it's, well, depressing, to see developers have been gaming the system with such indifference to the scientific pursuit of the truth. Could they have been more interested in making a marketing case for a drug? Need you ask?

- read the article in the New York Times

Related Articles:
Study: Antidepressant warnings work. Report
Top 10 drug warnings and recalls of 2007: Antidepressants

Researcher calls FDA 'shortcuts' into question

Wed, 12 Sep 2007 06:59:58 -0400

A high-profile researcher has issued what is now one of many public challenges to the manner in which the FDA speeds approval of certain drugs. Researcher Dr. Clifford J. Rosen is concerned, in particular, about shortcuts the FDA is using to approve drugs treating type 2 diabetes, particularly in light of questions about the safety of diabetes drug Avandia. His remarks, which were published in a recent edition of the New England Journal of Medicine, are bringing additional attention to an issue which has recently become much higher-profile.

Rosen is suggesting that approvals should only go through if drug makers can offer proof that a drug saves lives and improves patient health, as measured by such impacts as reductions in stroke, heart attack or mortality rates. Where diabetes drugs are concerned, he suggests, FDA officials should prove that the drugs improve patient quality of life and cut risks for side effects.

- read the Boston Globe article
- and see this FierceHealthcare report

ALSO: Not only did a new study in the Journal of the American Medical Association find that the diabetes drug doubles the risk of heart failure and boosted heart-attack risk by 42 percent, another JAMA study showed that Actos, a rival drug, actually lowered the risk of heart attacks, strokes, and death (though risk of heart failure did rise). Report

Related Articles:
FDA emphasizing speed over safety? Report
Poll finds problems at the FDA. Report
FDA offers safety reforms to skeptical lawmakers. Report
IOM slams FDA, calls for major reforms. Report

What to do with Avandia sales force?

Maureen Martino — Tue, 14 Aug 2007 10:25:15 -0400

GlaxoSmithKline may cut or redeploy its Avandia sales force in the wake of safety concerns that have virtually destroyed the drugs sales. Avandia is GSK’s second bestselling drug, earning £1.4 billion (about $2.8 billion) in 2006. Earlier this year, a New England Journal of Medicine study caused a uproar when it pointed to previously undisclosed safety risks associated with the diabetes drug. However, an expert committee of the FDA recommended by an overwhelming margin that Avandia should stay on the market with heightened warnings regarding its risks.

According to the Times, “A spokewsoman for GSK said the company was in ‘wait-and-see mode’ ahead of a final decision from the FDA but said that it was normal for the group to appraise regularly its allocation of staff and resources.” Given the GSK has a number of drug in the pipeline, the company may choose to redeploy salespeople on new drugs rather than cut them loose entirely.

- see this article for more

Press Release: GlaxoSmithKline Receives Approvable Letter For New Indications For ARIXTRA

Maureen Martino — Fri, 02 Feb 2007 11:04:52 -0500

GlaxoSmithKline Receives Approvable Letter For New Indications For ARIXTRA(R)(Fondaparinux Sodium) Injection

PHILADELPHIA, Feb. 2 -- GlaxoSmithKline (GSK) announced today that the U.S. Food and Drug Administration (FDA) has issued an approvable letter for the once-daily anticoagulant, ARIXTRA (fondaparinux sodium) Injection, for the treatment of patients with

-- Unstable angina or non-ST segment elevation myocardial infarction (UA/NSTEMI) -- ST-segment elevation myocardial infarction (STEMI). UA/NSTEMI and STEMI are types of acute coronary syndromes (ACS).

The FDA priority review was completed in six months. GSK will continue to work with the FDA to provide the additional information requested to complete the assessment of ARIXTRA for these indications. The company looks forward to making ARIXTRA available as an additional treatment option for the care of a broad range of patients with acute coronary syndromes. Results of the clinical studies (OASIS 5 and OASIS 6) supporting these two New Drug Applications have been presented and published in New England Journal of Medicine and Journal of the American Medical Association, respectively.

GlaxoSmithKline is one of the world's leading research-based pharmaceutical and healthcare companies. GlaxoSmithKline is committed to improving the quality of human life by enabling people to do more, feel better and live longer. For company information visit http://www.gsk.com.

CONTACT: Jennifer Armstrong GlaxoSmithKline 919-483-2839

Press Release: Aastrom Receives Orphan Drug Designation from FDA for Dilated Cardiomyopathy

Maureen Martino — Thu, 01 Feb 2007 12:38:12 -0500

Aastrom Receives Orphan Drug Designation from FDA for Dilated Cardiomyopathy

ANN ARBOR, Mich., Feb. 1 -- Aastrom Biosciences, Inc., a company focused on the use of autologous cells for regenerative medicine, today announced that the Company's proprietary Tissue Repair Cells (TRCs) received an Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for use in the treatment of dilated cardiomyopathy (DCM), a severe chronic disease of the heart. In the U.S., Orphan Drug Designation provides a variety of incentives, including 7 years of market exclusivity, should TRCs receive FDA approval for this indication.

DCM is a chronic cardiac disease that leads to enlargement of the heart and reduces pump function to a point that normal blood circulation cannot be maintained. Typically patients with DCM present with symptoms of congestive heart failure, including limitations in their physical activity and shortness of breath. DCM often represents the end stage of chronic ischemic heart disease in patients who have experienced multiple heart attacks. Patient prognosis depends on the stage of the disease but is characterized by a high mortality rate. Other than heart transplant, there are no effective long-term treatment options for end stage patients with this disease. The New England Journal of Medicine estimates that in the U.S. alone 120,000 people currently suffer from this disease; other sources report estimates of up to 150,000.

Scientific and early clinical evidence suggest that high doses of stem and progenitor cells may possibly slow down or reverse disease progression in the heart of DCM patients. It is intended that Aastrom's TRCs, a proprietary product containing large numbers of stem and progenitor cells derived from a small sample of the patient's own bone marrow, will be used as a therapeutic to induce heart tissue regeneration in these patients. If successful, TRC treatment may eliminate or delay the need for a heart transplant.

"We are pleased to receive an orphan drug designation from the FDA for our TRC-based product as a potential new treatment option for patients faced with this severe chronic heart disease," said George Dunbar, Chief Executive Officer and President of Aastrom. "Achieving this milestone is the first step in building the foundation for our clinical program in cardiac regeneration. The next anticipated milestone for our cardiac program is to initiate a clinical trial that treats patients with dilated cardiomyopathy."

The orphan drug designation is granted to development-stage novel therapeutics that offer potential value in the treatment of rare diseases and medical conditions. Above and beyond assistance from the Office of Orphan Products Development in furthering its TRC tissue regeneration program, Aastrom may receive other benefits. In particular, Aastrom may be entitled to an expedited FDA review, the reduction or elimination of filing fees, and the availability of possible tax credits.

About Aastrom Biosciences, Inc.

Aastrom Biosciences, Inc. develops autologous cell products for the repair or regeneration of multiple human tissues, based on its proprietary Tissue Repair Cell (TRC) technology. Aastrom's TRC-based products are a unique cell mixture containing stem and progenitor cell populations, produced from a small amount of bone marrow taken from the patient. TRC-based products have been used in over 230 patients, and are currently in clinical trials for bone regeneration (osteonecrosis of the femoral head, long bone fractures and spine fusion) and vascular regeneration (critical limb ischemia) applications. Aastrom has reported positive interim clinical trial results for TRCs suggesting both the clinical safety and the ability of TRCs to promote healing in bone regeneration applications. The Company is developing programs for TRC-based therapies to address cardiac and neural regeneration indications. TRCs have received Orphan Drug Designation from the FDA for use in the treatment of osteonecrosis of the femoral head and dilated cardiomyopathy.

For more information, visit Aastrom's website at http://www.aastrom.com/. (astmc)

This document contains forward-looking statements, including without limitation, statements concerning the timing of planned clinical trials, clinical trial strategies, product development objectives, potential advantages of TRCs, and potential product applications, which involve certain risks and uncertainties. The forward-looking statements are also identified through use of the words "intended," "may," "possible," "potential," "should," and other words of similar meaning. Actual results may differ significantly from the expectations contained in the forward-looking statements. Among the factors that may result in differences are potential patient accrual difficulties, clinical trial results, potential product development difficulties, the effects of competitive therapies, regulatory approval requirements, the availability of financial and other resources and the allocation of resources among different potential uses. These and other significant factors are discussed in greater detail in Aastrom's Annual Report on Form 10-K and other filings with the Securities and Exchange Commission.

CONTACTS: Kris M. Maly Cameron Associates Investor Relations Department Kevin McGrath Aastrom Biosciences, Inc. Phone: (212) 245-4577 Phone: (734) 930-5777 Deanne Eagle (Media) Phone: (212) 554-546
Photo: NewsCom: http://www.newscom.com/cgi-bin/prnh/20070117/CLW099LOGO
AP Archive: http://photoarchive.ap.org/
PRN Photo Desk, photodesk@prnewswire.com