Register for free to join our community of investors and share your ideas. You will also get access to streaming quotes, interactive charts, trades, portfolio, live options flow and more tools.
Register for free to join our community of investors and share your ideas. You will also get access to streaming quotes, interactive charts, trades, portfolio, live options flow and more tools.
Agreed. Hard to believe worldwide insulin sales will more than double in only four years even with very high growth in emerging markets.
do you mean a doubling isn't enough. If one of the attributes of this process is reducing the cost of production wouldn't that make it the insulin of choice in India and China
"The Satraplatin debacle has finally reached its logical conclusion as GPCB has withdrawn its European application. This is the typical MO in Europe when it becomes clear that an application will be rejected."
Ceplene(R) Receives Positive European Opinion for Approval From CHMP
Friday July 25, 5:44 am ET
Marketing Authorization Normally Anticipated within 67 Days in the EU
Conference Call to be Held July 28 at 9:00 a.m. Eastern Time
TARRYTOWN, N.Y.--(BUSINESS WIRE)--Regulatory News:
EpiCept Corporation (Nasdaq and OMX Nordic Exchange: EPCT) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMEA) has issued a positive opinion regarding the marketing authorization for Ceplene® (histamine dihydrochloride), for the remission maintenance and prevention of relapse in adult patients with Acute Myeloid Leukemia (AML) in first remission. Ceplene is to be administered in conjunction with low-dose interleukin-2 (IL-2). This positive opinion was issued following a request made by EpiCept to have the initial negative opinion of March 2008 re-examined by the CHMP. Ceplene has been designated as an orphan medicinal product, and as such is entitled to 10 years of marketing exclusivity in the EU.
ADVERTISEMENT
EpiCept attended an oral explanation hearing at the CHMP’s plenary meeting on July 22, 2008. Following this oral explanation, the CHMP recommended that Ceplene be granted a full marketing authorization under the provision of Exceptional Circumstances.
As part of granting of the marketing authorization under Exceptional Circumstances, EpiCept has agreed to perform two post-approval clinical studies. One of the studies seeks to further elucidate the clinical pharmacology of Ceplene by assessing certain biomarkers in AML patients in first remission. The other study will assess the effect of Ceplene/IL-2 on the development of minimal residual disease in the same patient population. EpiCept is entitled to seek further guidance on the design of such studies from the EMEA through the protocol assistance procedure.
“We are thrilled with the positive opinion reached by the CHMP and are pleased by the overwhelming support for Ceplene we received from key opinion leaders in hematology across Europe during this successful re-examination effort,” stated Jack Talley, President and CEO of EpiCept. “Ceplene, in conjunction with IL-2, is the first therapy shown to significantly prolong leukemia-free survival and prevent relapse in AML patients in first remission.”
The CHMP’s recommendation will now be forwarded to the European Commission for issuing a marketing authorization in the form of a Commission Decision, which normally occurs within 67 days. The marketing authorization with unified clinical usage for Ceplene granted under the Centralized Procedure will be valid for the entire European Union as well as in Iceland, Liechtenstein and Norway.
“We are highly optimistic about the commercial prospects for Ceplene and we intend to pursue its commercial introduction as expeditiously as possible,” continued Mr. Talley. “We are evaluating all of our strategic options for the marketing of Ceplene, and will continue to work towards realizing the drug’s potential to fulfill an important unmet medical need for AML patients in Europe.”
Conference Call
EpiCept announced that it will host a conference call to discuss this opinion and the Ceplene program on Monday, July 28, 2008 at 9:00 a.m. Eastern Daylight Time.
To listen to the conference call, please dial:
(877) 494-5472 (United States and Canada)
(706) 758-9407 (International)
The access code for the call is 57724791
A web cast of the conference can be accessed at www.epicept.com. The webcast will be archived for 90 days.
A playback of the call will be available for one week and may be accessed by dialing:
(800) 642-1687 (United States and Canada)
(706) 645-9291 (International)
Please reference reservation number 57724791
About Acute Myeloid Leukemia (AML)
The closest he came to an effective treatment was Iplex, a growth hormone developed by Insmed to treat dwarfism in children. Within days of taking it, he wrote last November, “I experienced rapid improvements in walking, speaking, appetite, swallowing and—critically—breathing.” But after a patent infringement claim by Genentech, manufacturers of rival drug Increlex, which Byer found less effective, Iplex was pulled from the market. Byer’s parents fought for its return to no avail.
I guess he should either move to Italy or go to Insmed's offices and steal it from their freezer. Actually I am sure if they offer to send 30 grand to a Geoff Alan's swiss bank account that greedy POS will get him some Iplex
the main problem with insmed is
Geoffrey Allan is a terrible ceo that only is interested in his salary.
shareholders are at the end of the list
i should have joined you.
I don't understand what made you buy the stock at 20 cents after they raised money at a penney.
what even made you think it would go up, much less double or triple. do you follow some pump and dump message board somewhere. It can't just be luck.
The Fed chairman said Treasury should take a lead in this process and that one option was a structure allowing federal regulators to set up a “bridge bank” to help a securities firm – a strategy used for failing commercial banks.
“A bridge bank authority is an important mechanism for minimising public losses from government intervention while imposing losses on shareholders and unsecured creditors, thereby limiting moral hazard and mitigating any adverse impact of government intervention on market discipline,” Mr Bernanke said.
===================================
If this is the reason that financial stocks went up then I am really confused. Investors looked at the comment that Bernanke made about the bridge bank as being positive for stocks. It looks like the reverse. They will bail out the financial institutions so there won't be a run on the bank but it would impose losses on the shareholders.
Comments please
I didn't want to be cruel
genta raised 20 million dollars at a penney a share.
do you think this should have a market cap of over a billion dollars?
GENTA INC(OTC BB: GNTA.OB)
Last Trade: 0.7490
Trade Time: 3:59PM ET
Change: 0.0990 (15.23%)
Prev Close: 0.65
Open: 0.66
Bid: 0.7400 x 2500
Ask: 0.7490 x 2500
1y Target Est: N/A
Day's Range: 0.6200 - 0.7700
52wk Range: 0.10 - 1.86
Volume: 1,257,274
Avg Vol (3m): 679,872
Market Cap: 27.52M
P/E (ttm): N/A
EPS (ttm): -0.871
Div & Yield: N/A (N/A)
With $100 Million Influx, MiddleBrook CEO Is Out
By Kendra Marr
Washington Post Staff Writer
Thursday, July 3, 2008; D01
http://www.washingtonpost.com/wp-dyn/content/article/2008/07/02/AR2008070203151_pf.html
Edward M. Rudnic, MiddleBrook Pharmaceuticals's chief executive, plans to leave the company he founded in exchange for an outside investment of $100 million.
MiddleBrook has been openly searching for a buyer over the past year. In the meantime, it agreed to the investment from Equity Group Investments, founded by Chicago billionaire Sam Zell, in an attempt to save its once-daily amoxicillin treatment for strep throat called Moxatag.
The Germantown biotech had previously considered a sale to another company, hoping for one that had a large sales force targeting primary-care physicians so that it could widely market the recently approved Moxatag.
The news sent the Germantown biotech's stock plummeting 51 percent, to close at $1.52, as the company deviated from investor's buyout expectations.
"This is a classic case of investor disappointment," said John T. McCamant, editor of the Medical Technology Stock Letter.
MiddleBrook announced late Tuesday that Equity Group Investments would install a new commercially focused senior management team when the deal closes in late August or early September. In addition to Rudnic, Chief Financial Officer Robert C. Low will leave.
Rudnic is the second executive to announce his departure from a local biotech in a week, following David M. Mott's decision to leave MedImmune, based in Gaithersburg, at the end of the month. Mott presided over the sale of Maryland's most successful biotech to British drug giant AstraZeneca.
Rudnic, who has weathered several ups and downs at MiddleBrook, has been the face and voice of the region's biotech industry. If there's a biotech meeting, Rudnic is usually speaking. If there's a task force or council, he is on the board.
"He is absolutely a pillar of this community," said Julie Coons, chief executive of the Tech Council of Maryland. "He is a leader in the biotech industry. Not only emerging, but accomplished executives look to him as a model of how to grow a company."
Three of MiddleBrook's largest stockholders -- HealthCare Ventures, Rho Ventures and Deerfield -- have agreed to vote in favor of the $100 million investment. "This is the alternative we thought would be best for the company going forward," MiddleBrook spokesman Robert Bannon said.
MiddleBrook began actively investigating its next move after winning Food and Drug Administration approval in January for Moxatag, which delivers the drug in time-released bursts, instead of requiring patients to swallow several doses a day.
Within 24 hours of the approval, the company announced that it would raise $21 million in a private placement of stock. In February, it brought on Morgan Stanley to help it explore strategic alternatives, which included selling MiddleBrook. Instead, the deal was made for the investment.
"This is an alternative," McCamant said, "but not the alternative the street wanted."
MiddleBrook will sell 30.3 million common shares at $3.30 each to Equity Group and issue a five-year warrant to purchase 12.1 million shares at an exercise price of $3.90 each.
The company has also agreed to buy back its Keflex assets, an anti-infection drug sold last year to Deerfield Management, for about $11 million.
Two Equity Group managing directors, along with former Adams Respiratory Therapeutics senior executive John Thievon, will join MiddleBrook's current board, which will expand to nine members. Thievon replaces Rudnic as chief executive. David Becker, another former Adams executive, will replace Low as chief financial officer.
Equity Group's investment allows MiddleBrook to move forward with Moxatag and continue development of drugs using the same time-release technology, called Pulsys.
The new management, who will work to launch Moxatag into the marketplace early next year, puts a powerful sales and marketing team behind MiddleBrook. While with Adams, another recipient of Equity Group investment, Thievon launched the Mucinex brand and built the franchise -- best known for commercials featuring Mr. Mucus, an animated green ball of phlegm -- into more than $350 million in annual revenue.
"What the incoming management is looking to do is replicate the success they had previously at Adams," Bannon said.
Both Rudnic and Low have long-term consulting agreements to work with MiddleBrook.
"They'll continue to be very close with the company," Bannon said.
Still, the investment marks the beginning of the end of Rudnic's MiddleBrook drama.
Nine years ago, Rudnic, who had left a senior drug company job and the accompanying $3.5 million in stock options, sketched out the beginnings of MiddleBrook at a friend's dining room table.
In 2005, its Pulsys technology failed, forcing the company to lay off about half of its 100 or so employees, including senior executives. It also cost the company a partnership with big drug company Par Pharmaceutical.
The next year, another attempt narrowly failed. MiddleBrook had burned through cash to get its product through the final stage of development. It reduced its workforce again.
The company, which first sold shares to the public in 2003, has yet to post a profit.
And a future sale remains a possibility.
"I don't think this rules it out," Bannon said.
91-Year-Old Woman Stuck Under Car for 2 Days After Looking for Keys
I wouldn't want to be a pedestrian anywhere around her while she is driving, or in a car in front, in back or to the side of her.
Please take away her license.
Middlebrook mbrk
http://washington.bizjournals.com/washington/stories/2008/06/30/daily44.html
Wednesday, July 2, 2008 - 3:34 PM EDT
MiddleBrook Pharmaceuticals secures $100M investment
Washington Business Journal - by Vandana Sinha Staff Reporter
MiddleBrook Pharmaceuticals Inc. has announced a $100 million private equity investment to help bring its lead drug to market — a milestone for the company, but disappointing for investors who expected a buyout.
Thwarting immediate plans to be the area’s next biotech to be sold to a pharmaceutical firm, MiddleBrook said it has ended its strategic review of its future options by accepting a private placement deal from Equity Group Investments LLC, a 40-year-old Chicago investment firm founded by entrepreneur Sam Zell that has dabbled in finance, energy, pharmaceuticals, transportation, media, telecommunications and real estate.
MiddleBrook plans to use that cash to hire a new sales and marketing team to launch Moxatag, its once-a-day strep throat tablet, in the first half of next year and restart other clinical trials that had been on hold while the company scraped together otherwise waning funds.
“At the end of the day, the decision of the board was that this was clearly the best alternative available to the company,” said Bob Bannon, vice president of investor relations. “Having this cash allows us to move forward with the sales and marketing team to bring Moxatag to market.”
Under that agreement, Equity Group will buy 30.3 million shares of MiddleBrook stock at $3.30 apiece, a 23 cent premium over the local company’s closing price on Tuesday. The move makes Equity Group the 30-person company’s largest shareholder, owning roughly 35 percent of its stock.
In the process, Equity Group has also asked to replace two of MiddleBrook’s top executives, Chief Executive and founder Ed Rudnic and Chief Financial Officer Bob Low, with leaders from one of the equity firm’s previous life sciences investments. Rudnic will make way for John Thievon, former executive vice president of sales and corporate accounts for Chester, N.J.-based Adams Respiratory Therapeutics Inc., while Low will be replaced by David Becker, former CFO at Adams.
Germantown-based MiddleBrook has won the blessing for the deal from its three largest shareholders, HealthCare Ventures, Rho Ventures and Deerfield Management, who said they would vote in favor of it. If other stockholders back the move, the companies hope to close on the investment by early September.
But investors have not been nearly as welcoming of the news. By midday Wednesday, MiddleBrook’s share prices had sunk by 55 percent after investors swapped shares at a stunning 24 times the average trading volume.
Wall Street, and MiddleBrook executives themselves, had pointed to the likelihood of a company sale in a biotech buying binge that has boasted some of the most generous premiums in recent history. MiddleBrook had been exploring strategic alternatives for the past year and, bolstered by federal approval of Moxatag in January -- a climactic event for biotech companies -- had hired Morgan Stanley & Co. Inc. in February to start sorting through those proposals.
“That was definitely one of the possible outcomes,” said Bannon, who declined to comment on any proposals or potential offers that resulted from its review. “Depending on the price of a transaction, it was possibly a preferred outcome. Of all of the available alternatives, this was clearly the best.”
An acquisition hasn’t been written out of MiddleBrook’s future, Bannon said. That prospect, he said, may in fact look more promising if the company can successfully launch Moxatag, estimated to enter a roughly $300 million market.
“It certainly doesn’t preclude us from doing any kind of strategic transaction down the road,” he said. “It might make sense to do that.”
MiddleBrook officials pointed to the Equity Group’s track record with Adams Respiratory, where the firm was an early investor. While with Adams, Thievon launched the Mucinex brand of nasal congestion and cough relievers, building it up to a $350 million revenue-generator each year before he and Becker helped sell Adams Respiratory to British-based Reckitt Benckiser Group PLC late last year for $2.3 billion.
Thievon, along with Equity Group Managing Directors William Pate and Mark Sotir, will join MiddleBrook’s board, bringing its total to nine members. Rudnic will step down from the board, but he and Low will hold long-term consulting contracts with MiddleBrook.
Rudnic will be leaving a company he built from the ground up eight years ago as founder and developer of MiddleBrook’s once-a-day, extended-dosing Pulsys technology. He had shepherded the company through a roller-coaster ride in the past several years, when MiddleBrook, formerly Advancis Pharmaceutical Inc., received failed results from its initial phase III clinical trials for Moxatag and even had to change its name after losing a trademark lawsuit to Sanofi Aventis.
After cash continued to run short, MiddleBrook had sold off rights to its Keflex franchise to investor Deerfield Management last year -- which it will buy back in an $11 million deal and redeem $8 million worth of warrants after this private placement with Equity Group closes. The local company also plans to renew clinical trial plans for its once-a-day Pulsys version of Keflex and pediatric version of Moxatag.
MiddleBrook (NASDAQ: MBRK) shares, which had peaked at $4.89 in the past year, had fallen from the previous day’s closing price of $3.07 to $1.46 roughly an hour before closing bell on Wednesday.
Feurstein comments on Middlebrook in early June
it is now trading at 1.50
Alex S. asks for an update on the auction process for MiddleBrook Pharmaceuticals (MBRK - commentary - Cramer's Take):
"I was hoping you could give me some quick input with regards to MiddleBrook Pharmaceuticals. They have been for sale a long time and only recently hired Morgan Stanley to assist in an ongoing strategic review. I understand these things take time, but how much longer do you think this could take? They have enough cash on hand to fund operations through 2008 so their cash position should not be an issue."
My sources tell me that the bidding process for MiddleBrook is on track, with multiple bids submitted in the first round. A second and final round of bids is expected by mid to late June, so hopefully, a deal gets announced soon.
When I first wrote about MiddleBrook putting itself up for sale back in January, I predicted the company could go for $6 to $8 a share. From what I hear, that's still in the ballpark.
Aradigm Reports Successful Top-line Phase 2 Data with Inhaled Liposomal Ciprofloxacin for Cystic Fibrosis
this is very good data
Monday June 30, 7:00 am ET
Study demonstrates significant reduction in bacterial infection and improvement in lung function following two weeks of treatment. Once daily dosing regimen may bring added convenience to patients
HAYWARD, Calif.--(BUSINESS WIRE)--Aradigm Corporation (OTCBB:ARDM - News) (the “Company”) today announced positive results from an open-label, two week efficacy and safety study of its once daily inhaled liposomal ciprofloxacin in patients with cystic fibrosis (CF). The study conducted at leading CF centers in Australia and New Zealand enrolled a total of 22 patients.
The primary efficacy endpoint in this Phase 2 study was the change from baseline in the sputum Pseudomonas Aeruginosa colony forming units (CFU), an objective measure of the reduction in pulmonary bacterial load. Data analysis in 21 patients who completed the study, demonstrated that the Pseudomonas CFU decreased by a mean 1.43 log over the 14-day treatment period (p<0.0001). Evaluation one week after study treatment was discontinued showed that the Pseudomonas bacterial density in the lung was still reduced by 1.02 log CFU from the baseline without additional antibiotic use. Pulmonary function testing as measured by the forced expiratory volume in one second (FEV1) showed a significant mean increase of 6.86 % from baseline after 14 days of treatment (p=0.04). The study drug was well tolerated, and there were no serious adverse events reported during the trial.
“Reduction of the burden of treatment with more conveniently administered products would be most welcomed by the cystic fibrosis community. It was therefore important in this study that, in addition to the successful clinical efficacy and safety outcomes, the patients found the once daily dosing regimen very convenient. This is a very attractive feature of the new product candidate,” said Dr. John Wilson of The Alfred Hospital, Melbourne, Australia, a CF physician and the lead investigator on the study.
“We are excited with the magnitude of the efficacy response seen in this proof-of-concept study,” said Tunde Otulana, M.D., Aradigm’s Chief Medical Officer. “The size of the reduction in bacterial density and the improvement in FEV1 represent clinically meaningful changes in these CF patients. Our results compare well with data with other inhaled antibiotics following 14-day treatment in similar subpopulations of CF patients where more frequent dosing was required.”
“We are very pleased with these Phase 2 data. The trial results will enable Aradigm to advance the development of our liposomal ciprofloxacin product for cystic fibrosis in the U.S., as well as pursue our strategy for other disease indications including bronchiectasis, and other geographical locations,” said Igor Gonda, Ph.D., the Company’s President and CEO.
Ciprofloxacin is a widely prescribed antibiotic to treat infections of the lung frequently experienced by cystic fibrosis patients. It is often preferred because of its broad-spectrum anti-bacterial action. The available oral and intravenous formulations of the drug are used to treat episodes of acute exacerbations of lung infections in CF. Aradigm’s once a day novel inhaled formulation of ciprofloxacin delivered in liposomes is to be used for chronic maintenance therapy as it is expected to achieve higher antibiotic concentration at the site of infection and relatively low systemic antibiotic concentrations to minimize side-effects.
Aradigm will provide more details on the data from this Phase 2 study in a business update conference call scheduled for July 16, 2008. Further information regarding the call will be announced soon.
About Aradigm
Aradigm is an emerging specialty pharmaceutical company focused on the development and commercialization of a portfolio of drugs delivered by inhalation for the treatment of severe respiratory diseases by pulmonologists. Current activities include partnered and self-initiated development programs addressing the treatment of cystic fibrosis, bronchiectasis, pulmonary hypertension, asthma and COPD, inhalation anthrax infections and smoking cessation.
More information about Aradigm can be found at www.aradigm.com.
Forward-Looking Statements
Except for the historical information contained herein, this news release contains forward-looking statements that involve risk and uncertainties, including the advancement of product development, as well as the other risks detailed in from time to time in Aradigm Corporation's Securities and Exchange Commission (SEC) Filings, including the Company's Annual Report on Form 10-K, and quarterly reports on Form 10-Q. Aradigm and the Aradigm Logo are registered trademarks of Aradigm Corporation.
Contact:
Aradigm
Investor Relations, 510-265-8850 x9370
or
Investors
Lippert/Heilshorn & Associates
Don Markley/Bruce Voss, 310-691-7100
dmarkley@lhai.com
why didn't lundbeck give me the 100 million instead, that is the question.
COPENHAGEN, June 30 (Reuters) - Danish pharmaceutical group Lundbeck (LUN.CO: Quote,
Profile, Research, Stock Buzz) said on Monday a Phase III trial with Alzheimer's drug
Flurizan failed to meet its two primary endpoints. "The clinical phase III data do
not correspond to the data observed in clinical phase II," Lundbeck said. Lundbeck
bought the European rights for the Myriad Genetics (MYGN.O: Quote, Profile, Research,
Stock Buzz) experimental drug last month for $100 million. (Reporting by Gelu Sulugiuc)
From a Prominent Death, Some Painful Truths
By DENISE GRADY
Apart from its sadness, Tim Russert’s death this month at 58 was deeply unsettling to many people who, like him, had been earnestly following their doctors’ advice on drugs, diet and exercise in hopes of avoiding a heart attack.
Mr. Russert, the moderator of “Meet the Press” on NBC News, took blood pressure and cholesterol pills and aspirin, rode an exercise bike, had yearly stress tests and other exams and was dutifully trying to lose weight. But he died of a heart attack anyway.
An article in The New York Times last week about his medical care led to e-mail from dozens of readers insisting that something must have been missed, that if only he had been given this test or that, his doctors would have realized how sick he was and prescribed more medicine or recommended bypass surgery.
Clearly, there was sorrow for Mr. Russert’s passing, but also nervous indignation. Many people are in the same boat he was in, struggling with weight, blood pressure and other risk factors — 16 million Americans have coronary artery disease — and his death threatened the collective sense of well-being. People are not supposed to die this way anymore, especially not smart, well-educated professionals under the care of doctors.
Mr. Russert’s fate underlines some painful truths. A doctor’s care is not a protective bubble, and cardiology is not the exact science that many people wish it to be. A person’s risk of a heart attack can only be estimated, and although drugs, diet and exercise may lower that risk, they cannot eliminate it entirely. True, the death rate from heart disease has declined, but it is still the leading cause of death in the United States, killing 650,000 people a year. About 300,000 die suddenly, and about half, like Mr. Russert, have no symptoms.
Cardiologists say that although they can identify people who have heart disease or risk factors for it, they are not so good at figuring out which are in real danger of having an attack soon, say in the next year or so. If those patients could be pinpointed, doctors say, they would feel justified in treating them aggressively with drugs and, possibly, surgery.
“It’s the real dilemma we have in cardiology today,” said Dr. Sidney Smith, a professor of medicine at the University of North Carolina and a past president of the American Heart Association. “Is it possible to identify the group at higher short-term risk?”
What killed Mr. Russert was a plaque rupture. A fatty, pimplelike lesion in a coronary artery burst, and a blood clot formed that closed the vessel and cut off circulation to part of the heart muscle. It was a typical heart attack, or myocardial infarction, an event that occurs 1.2 million times a year in the United States, killing 456,000 people.
In Mr. Russert’s case, the heart attack led to a second catastrophe, an abnormal heart rhythm that caused cardiac arrest and quickly killed him. An electric shock from a defibrillator might have restarted his heart if it had been given promptly when he collapsed at his desk. But it was apparently delayed.
Dr. Smith and other cardiologists say the main problem is that there is no way to figure out who has “vulnerable plaques,” those prone to rupture. Researchers are trying to find biomarkers, substances in the blood that can show the presence of these dangerous, ticking time-bomb plaques. So far, no biomarker has proved very accurate.
Mr. Russert’s heart disease was a mixed picture. Some factors looked favorable. There was no family history of heart attacks. Though he had high blood pressure, drugs lowered it pretty well, said his internist, Dr. Michael A. Newman. His total cholesterol was not high, nor was his LDL, the bad type of cholesterol, or his C-reactive protein, a measure of inflammation that is thought to contribute to plaque rupture. He did not smoke. At his last physical, in April, he passed a stress test, and his heart function was good. Dr. Newman estimated his risk of a heart attack in the next 10 years at 5 percent, based on a widely used calculator.
On the negative side, Mr. Russert had low HDL, the protective cholesterol, and high triglycerides. He was quite overweight; a waist more than 40 inches in men increases heart risk. A CT scan of his coronary arteries in 1998 gave a calcium score of 210, indicating artery disease — healthy arteries do not have calcium deposits — and a moderate to high risk of a heart attack. An echocardiogram in April found that the main heart pumping chamber had thickened, his ability to exercise had decreased slightly, and his blood pressure had increased a bit. Dr. Newman and his cardiologist, Dr. George Bren, changed his blood pressure medicines, and the pressure lowered to 120/80, Dr. Newman said.
Another blood test, for a substance called apoB, might have been a better measure of risk than LDL, some doctors say. Others disagree.
Some doctors say people like Mr. Russert, with no symptoms but risk factors like a thickened heart, should have angiograms, in which a catheter is threaded into the coronary arteries, dye is injected, and X-rays are taken to look for blockages. Some advocate less invasive CT angiograms. Both types of angiogram can identify plaque deposits, and if extensive disease or blockages at critical points are found, a bypass is usually recommended. But the tests still cannot tell if plaques are likely to rupture, Dr. Smith and other cardiologists say. And Mr. Russert’s doctors did not think that an angiogram was needed.
An autopsy found, in addition to the plaque rupture, extensive disease in Mr. Russert’s coronary arteries, enough to surprise his doctors, they said. Had they found it before, Dr Newman said, a bypass would have been recommended. Dr. Bren differed, saying many cardiologists would still not have advised surgery.
Given all the uncertainties, what’s a patient to do?
“You want to be sure your blood pressure and lipids are controlled, that you’re not smoking, and you have the right waist circumference,” Dr. Smith said.
Statins can reduce the risk of dying from a heart attack by 30 percent, he said.
“But what about the other 70 percent?” Dr. Smith asked. “There are other things we need to understand. There’s tremendous promise, but miles to go before we sleep.”
Acorda's drug is detrimental to MS patients because it has side effects, causes seizures, does nothing to slow the disease pogression, and when the drug is stopped the patients are worse off than before they started the treatment.
I don't think the risk/reward benefit of the drug makes any sense.
OPXA - I would appreciate comments on these results from anybody familiar with MS and in particular anybody who has knowledge of the "typical" relapse rates in these populations. Comments on the individualized T-Cell vaccine approach (and MOA)also appreciated. I note that a 150 patient phase IIb double-blind, placebo controlled trial will be reported on soon.
======================================================
I believe this data is excellent but the company doesn't have cash so whenever it has good news it gets sold down for the upcomming financing.
It is sick that this company that truly has a good benefit for people with MS trades with this market cap while Acorda which actually has a detrimental effect on the progression of MS but lets MS patients walk faster has a market cap of over a billion dollars.
It is insane
New Hints Seen That Red Wine May Slow Aging
By NICHOLAS WADE
http://www.nytimes.com/2008/06/04/health/research/04aging.html?_r=1&ei=5087&em=&en=08579ec468456085&ex=1212811200&adxnnlx=1212674400-xNKD%20gpr8xA3/ux442ZFnA&pagewanted=print
Red wine may be much more potent than was thought in extending human lifespan, researchers say in a new report that is likely to give impetus to the rapidly growing search for longevity drugs.
The study is based on dosing mice with resveratrol, an ingredient of some red wines. Some scientists are already taking resveratrol in capsule form, but others believe it is far too early to take the drug, especially using wine as its source, until there is better data on its safety and effectiveness.
The report is part of a new wave of interest in drugs that may enhance longevity. On Monday, Sirtris, a startup founded in 2004 to develop drugs with the same effects as resveratrol, completed its sale to GlaxoSmithKline for $720 million.
Sirtris is seeking to develop drugs that activate protein agents known in people as sirtuins.
“The upside is so huge that if we are right, the company that dominates the sirtuin space could dominate the pharmaceutical industry and change medicine,” Dr. David Sinclair of the Harvard Medical School, a co-founder of the company, said Tuesday.
Serious scientists have long derided the idea of life-extending elixirs, but the door has now been opened to drugs that exploit an ancient biological survival mechanism, that of switching the body’s resources from fertility to tissue maintenance. The improved tissue maintenance seems to extend life by cutting down on the degenerative diseases of aging.
The reflex can be prompted by a faminelike diet, known as caloric restriction, which extends the life of laboratory rodents by up to 30 percent but is far too hard for most people to keep to and in any case has not been proven to work in humans.
Research started nearly 20 years ago by Dr. Leonard Guarente of the Massachusetts Institute of Technology showed recently that the famine-induced switch to tissue preservation might be triggered by activating the body’s sirtuins. Dr. Sinclair, a former student of Dr. Guarente, then found in 2003 that sirtuins could be activated by some natural compounds, including resveratrol, previously known as just an ingredient of certain red wines.
Dr. Sinclair’s finding led in several directions. He and others have tested resveratrol’s effects in mice, mostly at doses far higher than the minuscule amounts in red wine. One of the more spectacular results was obtained last year by Dr. John Auwerx of the Institute of Genetics and Molecular and Cellular Biology in Illkirch, France. He showed that resveratrol could turn plain vanilla, couch-potato mice into champion athletes, making them run twice as far on a treadmill before collapsing.
The company Sirtris, meanwhile, has been testing resveratrol and other drugs that activate sirtuin. These drugs are small molecules, more stable than resveratrol, and can be given in smaller doses. In April, Sirtris reported that its formulation of resveratrol, called SRT501, reduced glucose levels in diabetic patients.
The company plans to start clinical trials of its resveratrol mimic soon. Sirtris’s value to GlaxoSmithKline is presumably that its sirtuin-activating drugs could be used to treat a spectrum of degenerative diseases, like cancer and Alzheimer’s, if the underlying theory is correct.
Separately from Sirtris’s investigations, a research team led by Tomas A. Prolla and Richard Weindruch, of the University of Wisconsin, reports in the journal PLoS One on Wednesday that resveratrol may be effective in mice and people in much lower doses than previously thought necessary. In earlier studies, like Dr. Auwerx’s of mice on treadmills, the animals were fed such large amounts of resveratrol that to gain equivalent dosages people would have to drink more than 100 bottles of red wine a day.
The Wisconsin scientists used a dose on mice equivalent to just 35 bottles a day. But red wine contains many other resveratrol-like compounds that may also be beneficial. Taking these into account, as well as mice’s higher metabolic rate, a mere four, five-ounce glasses of wine “starts getting close” to the amount of resveratrol they found effective, Dr. Weindruch said.
Resveratrol can also be obtained in the form of capsules marketed by several companies. Those made by one company, Longevinex, include extracts of red wine and of a Chinese plant called giant knotweed. The Wisconsin researchers conclude that resveratrol can mimic many of the effects of a caloric-restricted diet “at doses that can readily be achieved in humans.”
The effectiveness of the low doses was not tested directly, however, but with a DNA chip that measures changes in the activity of genes. The Wisconsin team first defined the pattern of gene activity established in mice on caloric restriction, and then showed that very low doses of resveratrol produced just the same pattern.
Dr. Auwerx, who used doses almost 100 times greater in his treadmill experiments, expressed reservations about the new result. “I would be really cautious, as we never saw significant effects with such low amounts,” he said Tuesday in an e-mail message.
Another researcher in the sirtuin field, Dr. Matthew Kaeberlein of the University of Washington in Seattle, said, “There’s no way of knowing from this data, or from the prior work, if something similar would happen in humans at either low or high doses.”
A critical link in establishing whether or not caloric restriction works the same wonders in people as it does in mice rests on the outcome of two monkey trials. Since rhesus monkeys live for up to 40 years, the trials have taken a long time to show results. Experts said that one of the two trials, being conducted by Dr. Weindruch, was at last showing clear evidence that calorically restricted monkeys were outliving the control animals.
But no such effect is apparent in the other trial, being conducted at the National Institutes of Health.
The Wisconsin report underlined another unresolved link in the theory, that of whether resveratrol actually works by activating sirtuins. The issue is clouded because resveratrol is a powerful drug that has many different effects in the cell. The Wisconsin researchers report that they saw no change in the mouse equivalent of sirtuin during caloric restriction, a finding that if true could undercut Sirtris’s strategy of looking for drugs that activate sirtuin.
Dr. Guarente, a scientific adviser to Sirtris, said the Wisconsin team only measured the amount of sirtuin present in mouse tissues, and not the more important factor of whether it had been activated.
Dr. Sinclair said the definitive answer would emerge from experiments, now under way, with mice whose sirtuin genes had been knocked out. “The question of how resveratrol is working is an ongoing debate and it will take more studies to get the answer,” he said.
Dr. Robert E. Hughes of the Buck Institute for Age Research said there could be no guarantee of success given that most new drug projects fail. But, he said, testing the therapeutic uses of drugs that mimic caloric restriction is a good idea, based on substantial evidence.
ONGLYZA(TM) (saxagliptin) Demonstrated Significant Reductions in Key Measures of Glucose Control in Treatment Naive People with Type 2 Diabetes
Saturday June 7, 1:03 pm ET
should this drug need an outcomes trial? If it does than Merck has a monopoly on the DPP-4 class because they got to the market before the fda made an arbitrary decision.
Statins have done a lot of good as the outcome trials are showing but they wouldn't have gotten approved if you needed an outcomes study.
SAN FRANCISCO--(BUSINESS WIRE)--Results from a 24-week Phase III study presented at the 68th American Diabetes Association Annual Scientific Sessions demonstrated that saxagliptin, a selective, reversible inhibitor of the dipeptidyl peptidase (DPP-4) enzyme in development by Bristol-Myers Squibb Company (NYSE: BMY - News) and AstraZeneca (NYSE: AZN - News), produced significant reductions in key measures of glucose control (glycosylated hemoglobin level [A1C], fasting plasma glucose [FPG] and postprandial glucose [PPG]) in treatment naïve people with type 2 diabetes compared to placebo (PBO). Over 24 weeks, saxagliptin had an adverse event profile that appeared similar to placebo. The companies have proposed the trade name ONGLYZA™ for saxagliptin if approved by the U.S. Food & Drug Administration.
“Diabetes is a serious and chronic condition that affects nearly 21 million people in the U.S. and, unfortunately, this number is only going to rise,” said Julio Rosenstock, MD, Director of Dallas Diabetes & Endocrine Center at Medical City, and also Clinical Professor of Medicine at University of Texas Southwestern Medical Center, Dallas, USA. “New therapies need to be researched and developed to help treat this growing epidemic.”
About the Study
The data represent findings from a multicenter, randomized, double-blind, placebo-controlled, parallel-group study of 401 people with type 2 diabetes (ages 18-77) who were treatment naïve and whose A1C level was greater than or equal to 7 percent and less than or equal to 10 percent. Individuals were randomized to one of four separate treatment arms: saxagliptin 2.5 mg (n=102), 5 mg (n=106) or 10 mg (n=98) or placebo (n=95), once daily.
The primary endpoint of the study was the change from baseline to Week 24 in A1C. The secondary endpoints of the study included achievement of A1C of less than 7 percent and changes from baseline in FPG (a measure of a person’s blood glucose after at least 8 hours of fasting). PPG (a measure of a person’s blood glucose, measured during an oral glucose tolerance test [OGTT]) was also evaluated.
Study Results
After 24 weeks, individuals receiving ONGLYZA™ (saxagliptin) 2.5 mg, 5 mg and 10 mg demonstrated a significant mean change in A1C from baseline of -0.4 percent, -0.5 percent and -0.5 percent, respectively, which resulted in a placebo-adjusted change of -0.6 percent, -0.6 percent and -0.7 percent, respectively (p-value less than 0.0001). Reductions in A1C levels were seen as early as four weeks after initiation of saxagliptin treatment, the first scheduled A1C measurement point.
The percentage of individuals receiving saxagliptin 2.5 mg, 5 mg and 10 mg or placebo who achieved the American Diabetes Association’s recommended A1C of less than 7 percent at Week 24 was 35 percent, 38 percent and 41 percent, respectively, compared to 24 percent of individuals treated with PBO (p-value not significant at the 2.5 mg dose; p-value at the other doses less than 0.05 versus PBO). Saxagliptin demonstrated significant reductions versus placebo in FPG and PPG at all doses from baseline to Week 24.
Over 24 weeks, saxagliptin had an adverse event profile that appeared similar to placebo. Across all treatment groups, the percentage of individuals who experienced at least one adverse event was slightly higher in individuals treated with saxagliptin (75.5 percent) compared to PBO (71.6 percent). The most commonly reported (greater than or equal to 5 percent) adverse events from the saxagliptin treatment arms and PBO, respectively, were: upper respiratory tract infection [8.8 percent (27/306), 11.6 percent (11/95)]; headache [8.2 percent (25/306), 7.4 percent (7/95)], urinary tract infection [6.9 percent (21/306), 4.2 percent (4/95)], nasopharyngitis [5.9 percent (18/306), 6.3 percent (6/95)] and sinusitis [5.6 percent (17/306), 3.2 percent (3/95)].
No cases of confirmed hypoglycemia (symptoms of hypoglycemia with a fingerstick glucose less than or equal to 50 mg/dL) were reported. The proportion of reported hypoglycemic events was similar in the saxagliptin-treatment arms (5.2 percent) and PBO (6.3 percent).
Saxagliptin was not associated with weight gain in any of the treatment groups. Mean change from baseline in body weight at Week 24 was -1.2 kg, -0.1 kg and -0.1 kg at the 2.5 mg, 5 mg and 10 mg doses, respectively, and -1.4 kg for PBO.
About ONGLYZA™ (saxagliptin)
Saxagliptin, a DPP-4 inhibitor, is an investigational drug under joint development by Bristol-Myers Squibb and AstraZeneca for the treatment of type 2 diabetes. Saxagliptin is being studied in clinical trials as a once-daily therapy to determine its efficacy and safety. Saxagliptin was specifically designed to be a selective, reversible inhibitor of the DPP-4 enzyme, with dual routes of clearance. Phase III data has previously been presented in combination with metformin, the most commonly prescribed OAD, and further Phase III data, including saxagliptin added to a sulfonylurea, a thiazolidinedione and as initial combination therapy with metformin, will be disclosed later this year. The Companies plan to submit a New Drug Application (NDA) in the United States in mid 2008.
About DPP-4 Inhibitors
DPP-4 inhibitors are a class of compounds that work by affecting the action of natural hormones in the body called incretins. Incretins decrease elevated blood sugar levels (glucose) by increasing the body’s utilization of sugar, mainly through increasing insulin production in the pancreas, and by reducing the liver’s production of glucose.
About Type 2 Diabetes
Diabetes (diabetes mellitus) is a chronic disease in which the body does not produce or properly use insulin. Insulin is a hormone that is needed to convert sugar, starches (carbohydrates) and other nutrients into energy needed for daily life. The cause of diabetes continues to be investigated, and both genetic and environmental factors such as obesity and lack of exercise appear to play a role. Diabetes is associated with long-term complications that affect almost every part of the body. The disease may lead to blindness, heart and blood vessel disease, stroke, kidney failure, amputations, and nerve damage.
There are two primary underlying causes associated with type 2 diabetes: the body does not produce enough insulin (insulin deficiency), or the cells ignore the insulin (insulin resistance). Symptoms of type 2 diabetes develop gradually, and their onset is not as sudden as in type 1 diabetes. Symptoms may include fatigue, frequent urination, increased thirst and hunger, weight loss, blurred vision, and slow healing of wounds or sores. Some people, however, have no symptoms.
Type 2 diabetes is most often associated with older age, obesity, family history of diabetes, previous history of gestational diabetes, physical inactivity and certain ethnicities. People with type 2 diabetes often are characterized with: insulin resistance, abdominal obesity, a sedentary lifestyle, having low HDL-C (“good”) cholesterol levels and high triglyceride levels and hypertension.
Type 2 diabetes accounts for approximately 90 to 95 percent of all diabetes. This equates to roughly 221 million people with type 2 diabetes globally, and 18.7 million people in the U.S. alone.
The American Diabetes Association recommends a hemoglobin A1C measurement of less than 7 percent for most people with type 2 diabetes. Hemoglobin A1C is a measurement of a person’s average blood glucose level over a two-to-three month period and is considered an important marker of long-term glucose control. Other important markers for type 2 diabetes include fasting plasma glucose, a measure of a person’s blood glucose after at least 8 hours of fasting and postprandial glucose, a measure of a person’s blood glucose after a meal.
Bristol-Myers Squibb and AstraZeneca Partnership
Bristol-Myers Squibb and AstraZeneca entered into a collaboration in January 2007 to enable the companies to research, develop and commercialize two investigational drugs for type 2 diabetes – ONGLYZA™ (saxagliptin) and dapagliflozin. The Bristol-Myers Squibb/AstraZeneca Diabetes collaboration is dedicated to global patient care, improving patient outcomes and creating a new vision for the treatment of type 2 diabetes.
About Bristol-Myers Squibb
Bristol-Myers Squibb is a global biopharmaceutical company whose mission is to extend and enhance human life. For more information, visit www.bms.com.
Bristol-Myers Squibb Forward-Looking Statement
This press release contains "forward-looking statements" as that term is defined in the Private Securities Litigation Reform Act of 1995 regarding product development. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual outcomes and results to differ materially from current expectations. No forward-looking statement can be guaranteed. Among other risks, there can be no guarantee that an NDA submitted to the FDA will be approved or that the timing of any NDA submission will occur as described in this release. Forward-looking statements in this press release should be evaluated together with the many risks and uncertainties that affect Bristol-Myers Squibb's business, including those identified in Bristol-Myers Squibb's Annual Report on Form 10-K for the year ended December 31, 2007, particularly under "Item 1A. Risk Factors". Bristol- Myers Squibb undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise.
About AstraZeneca
AstraZeneca is a major international healthcare business engaged in the research, development, manufacturing and marketing of meaningful prescription medicines and supplier for healthcare services. AstraZeneca is one of the world's leading pharmaceutical companies with healthcare sales of $29.55 billion and is a leader in gastrointestinal, cardiovascular, neuroscience, respiratory, oncology and infectious disease medicines. In the United States, AstraZeneca is a $13.35 billion dollar healthcare business with 12,200 employees committed to improving people’s lives. AstraZeneca is listed in the Dow Jones Sustainability Index (Global) as well as the FTSE4Good Index.
For more information about AstraZeneca, please visit www.astrazeneca-us.com.
AstraZeneca Forward-Looking Statement
The statements contain herein include forward-looking statements. Although we believe our expectations are based on reasonable assumptions, any forward-looking statements, by their very nature, involve risks and uncertainties and may be influenced by factors that could cause actual outcomes and results to be materially different from those predicted. The forward-looking statements reflect knowledge and information available at the date of the preparation of this press release and the Company undertakes no obligation to update these forward-looking statements. Important factors that could cause actual results to differ materially from those contained in forward-looking statements, certain of which are beyond our control, include, among other things, those risk factors identified in the Company's Annual Report/Form 20-F for 2007. Nothing contained herein should be construed as a profit forecast.
ONGLYZA™ is a trademark of the Bristol-Myers Squibb Company
Contact:
Media:
Bristol-Myers Squibb
David M. Rosen, 609-252-5675
Cell: 609-721-6155
david.rosen@bms.com
or
AstraZeneca
David Albaugh, 302-886-7098
Cell: 609-304-3445
david.albaugh@astrazeneca.com
or
Investors:
Bristol-Myers Squibb
John Elicker, 212-546-3775
john.elicker@bms.com
or
AstraZeneca
Mina Blair, 44-20-7304-5084
mina.blair@astrazeneca.com
--------------------------------------------------------------------------------
Not just larger - but to outcomes trials. Vs surrogate of HbA1C. The FDA was already moving that way but it will be interesting to see how fast the move takes place. Surprisingly there has only been one large outcomes trial before the one earlier this year.
This is good news.
If may think this is good news, but this isn't good news if you invest in biotech or want beneficial drugs to hit the market.
what will be the endpoint and size of such trials. will you need to have trials which include 10 thousand people that will have to show survival benefits. How many companies will be able to invest in those trials.
Four Japanese gang figures got liver transplants at UCLA
I heard that Osama Bin Laden is still alive because UCLA gave him the organ transplant that he needed because they don't make moral judgements. They just try to save lives (except if you are an uninsured american citizen). The crack about OBL was a joke but I wouldn't put it passed them.
Japanese Police
Tadamasa Goto received a life-saving liver transplant at UCLA Medical Center. Goto is one of Japan's most powerful gang bosses, which experts describe as vindictive and at times brutal.
The recipients included one of Japan's most powerful crime bosses. Some in the medical community worry the revelation will have a chilling effect on organ donations.
By John M. Glionna and Charles Ornstein, Los Angeles Times Staff Writers
May 30, 2008
»
UCLA Medical Center and its most accomplished liver surgeon provided a life-saving transplant to one of Japan's most powerful gang bosses, law enforcement sources told The Times.
In addition, the surgeon performed liver transplants at UCLA on three other men who are now barred from entering the United States because of their criminal records or suspected affiliation with Japanese organized crime groups, said a knowledgeable law enforcement official who spoke on condition of anonymity.
Four Japanese gang figures got liver transplants at UCLA
Japan's flamboyant crime syndicates
After livers, cash to UCLA
The four surgeries were done between 2000 and 2004 at a time of pronounced organ scarcity. In each of those years, more than 100 patients died awaiting liver transplants in the Greater Los Angeles region.
The surgeon in each case was Dr. Ronald W. Busuttil, executive chairman of UCLA's surgery department, according to another person familiar with the matter who also spoke on condition of anonymity. Busuttil is a world-renowned liver surgeon who co-edited a leading text on liver transplantation and is one of the highest-paid employees in the University of California system.
There is no evidence that UCLA or Busuttil knew at the time of the transplants that any of the patients had ties to Japanese gangs, commonly called yakuza. Both said in statements that they do not make moral judgments about patients and treat them based on their medical need.
U.S. transplant rules do not prohibit hospitals from performing transplants on either foreign patients or those with criminal histories.
The most prominent transplant recipient, Tadamasa Goto, had been barred from entering the U.S. because of his criminal history, several current and former law enforcement officials said. Goto leads a gang called the Goto-gumi, which experts describe as vindictive and at times brutal.
The FBI helped Goto obtain a visa to enter the United States in 2001 in exchange for leads on potentially illegal activity in this country by Japanese criminal gangs, said Jim Stern, retired chief of the FBI's Asian criminal enterprise unit in Washington.
Goto got his liver, Stern said, but provided the bureau with little useful information on Japanese gangs.
"I don't think Goto gave the bureau anything of significance," Stern said. Goto "came to the States and got a liver and was laughing back to where he came from. . . . It defies logic."
Although Stern was not involved with the deal, he said he learned the details when he became unit chief in 2004 and continues to be troubled by what happened.
After the transplant, Goto was again barred from reentering the U.S., said the first law enforcement official, who was not authorized to discuss the matter publicly and therefore requested anonymity.
But Goto continued to receive medical care from Busuttil in Japan. The doctor traveled there and examined Goto on more than one occasion, said Goto's Tokyo-based lawyer, Yoshiyuki Maki -- and evaluated Goto while he was in custody in 2006.
Busuttil's medical opinion was cited in a successful court petition to have Goto released for medical care at a Tokyo hospital, Maki said.
The Times is not naming the other three transplant recipients in this article because neither they nor their lawyers could be reached.
Several transplant experts and bioethicists contacted by The Times said they were troubled by the transplants, especially because organs are in such short supply in this country. In the year of Goto's surgery, 186 people in the Los Angeles region died waiting for a liver, U.S. transplant statistics show.
Some, but not all, of the experts said a transplant center has an obligation to determine whether a patient would be a worthy custodian of an organ and to protect potential donors' faith in the system.
"If you want to destroy public support for organ donation on the part of Americans, you'd be hard pressed to think of a practice that would be better suited," said Arthur Caplan, a bioethicist at the University of Pennsylvania.
In a statement, the UCLA Health System said it could not comment on specific cases because of federal patient privacy laws. Generally, it said it complies with all the rules and regulations of the United Network for Organ Sharing, the federal contractor charged with ensuring the safety and fairness of the U.S. transplant system. Last year, UCLA performed more liver transplants than any other U.S. hospital.
"UCLA's processes for evaluating a patient -- both for mental and physical suitability for organ transplants -- are the same regardless of whether the individual is a U.S. citizen or a foreign national," the statement said.
Hospitals and doctors in the U.S. have the final say on which patients get added to their waiting lists and have the discretion to refuse patients with unhealthy lifestyles that could compromise the transplant's success. Patients may be refused on other grounds as well, including an inability to pay.
At the time of Goto's 2001 transplant, liver allocations were made based on both a patient's medical status and waiting time. Since 2002, livers have been allocated to patients based almost entirely on how sick they are.
It is unclear when Goto joined UCLA's waiting list. He had been in the United States two months when he received a new liver. Overall, 34% of the patients added to UCLA's liver waiting list between January 1999 and December 2001 received a new liver within three years of being listed, national transplant statistics show.
Busuttil, a former president of the American Society of Transplant Surgeons who has testified before Congress on who should receive priority for transplants, released his own statement this week. He did not directly address the transplants of the Japanese patients but said in part:
"As a surgeon, it is not my role to pass moral judgment on the patients who seek my care. . . . If one of my patients, domestic or international, were in a situation that could be life-threatening, of course I would do everything in my power to assure that they would receive proper care.
"I consider that to be part of my responsibility and obligation as a physician."
'A serious player'
On May 18, 2001, Tadamasa Goto boarded Japan Airlines Flight 0062 at Narita International Airport, bound for Los Angeles with his son Masato.
Goto, now 65, had hepatitis C and was worried it would develop into cancer, Maki, Goto's lawyer, said in an interview last week in his Tokyo office. Because Japan has an extreme shortage of organ donors, many sick patients feel they need to go abroad to seek treatment.
The FBI did not help Goto arrange his surgery with UCLA but did help him gain entry to this country, Stern said. The agency had long been frustrated by the reluctance of Japanese law enforcement to share information on yakuza members in the United States.
"For American law enforcement, it's been like pulling teeth to get criminal intelligence from Japanese authorities," said David Kaplan, a journalist who co-wrote the book "Yakuza: Japan's Criminal Underworld," published in 2003 by the University of California Press.
In his book, Kaplan describes Goto's gang, the Goto-gumi, as an offshoot of the largest Japanese organized crime group, the Yamaguchi-gumi. In an interview, Kaplan said Goto is "a serious player in the yakuza. His gang is known for being particularly ruthless and violent."
A senior member of the group and an affiliated gang member were sentenced to prison for the 1992 slashing of a Japanese director whose film portrayed the yakuza as violent thugs, according to a story in the Japan Times. Goto was not personally implicated in the case.
Goto underwent a successful transplant in July 2001. He received the liver of a young man who died in a traffic accident, Maki said. "Goto is over 60 now, but his liver is young," he said.
Several years after the transplant, in May 2006, Goto was arrested in Japan on suspicion of real estate fraud.
Maki said he and other lawyers worried that their client was not well enough to be interrogated. In addition to his liver problem, Goto was suffering from heart disease, high blood pressure and diabetes.
The lawyers asked that Goto be released immediately, but authorities rejected the request, Maki said. He said the lawyers asked that Goto be given his medication at precise times, but that did not happen either. "Goto lost his appetite, had a terrible headache, scratched his arm until it started to get infected, and he was throwing up," Maki said.
Maki used the interview to vent against Japanese prosecutors, saying he believes they were attempting to exploit his client's poor health to obtain a conviction on what Maki considered groundless charges.
He said Busuttil, along with doctors from Tokyo University Hospital and Showa University Hospital in Tokyo, examined Goto and recommended that he be released for outside medical treatment.
On May 24, 2006, some 16 days after he was arrested, the court temporarily released Goto and he entered the hospital.
Goto was acquitted of the charges in March of this year.
the first twelve jurors were found accidently hung in the hotel room duing deliberations. The twelve supplemental jurors aquitted him in less than five minutes. A new record. Goto was holding 200 feet of rope in his hands and threw it in the air in celebration. The rope matched that used in the hangings but the police said people accidently hang themselves in their hotel rooms all the time.
"The UCLA doctor [Busuttil] examined Goto during his detention and again one week after he received his not-guilty ruling," Maki said.
The law enforcement official who spoke on condition of anonymity said Goto's criminal history includes prison time. But Maki said that his client's last conviction was three decades ago, for assault, and that his previous convictions were as a youth.
Court records in Japan are kept by prosecutors who generally do not share them with anyone not party to a case.
Jake Adelstein, a former reporter at Japan's largest daily newspaper, Yomiuri Shimbun, said he received a tip about the circumstances surrounding Goto's liver transplant in 2005. Within days of making inquiries, however, Adelstein was visited by men who told him: "Erase the story or be erased," he said in an interview.
Adelstein did not pursue the story but mentioned the incident in a recent opinion piece in the Washington Post. He said he would elaborate on it in a forthcoming book.
Dealing with scandals
Word of the surgeries at UCLA comes as the U.S. transplant system is slowly recovering from scandals that forced the closure of three transplant programs in California. In one of those, St. Vincent Medical Center in Los Angeles moved a Saudi national up a liver waiting list, bypassing dozens of others, and then covered it up by falsifying paperwork, officials there have acknowledged.
Overseers of the U.S. transplant system say they are unaware of other cases in which hospitals have provided organs to foreign criminals. But some hospitals, including Stanford University Medical Center, have performed transplants on U.S. prisoners -- often controversial because taxpayers foot the bill.
According to the ethics committee of the United Network for Organ Sharing, "one's status as a prisoner should not preclude them from consideration for a transplant."
The network encourages transplant programs to give foreign recipients less than 5% of organs from deceased donors each year, but that is not a hard-and-fast rule. At one point, in the 1980s, the threshold was 10%, but it was lowered after Congress considered banning transplants for foreign nationals entirely.
Centers that exceed the 5% guideline are asked for an explanation in writing, but none has been sanctioned publicly. In 2001, the year Goto received his transplant, UCLA slightly exceeded the guideline.
Typically, transplant experts say, foreigners cannot receive transplants at U.S. centers unless they are willing to pay the full cost of the procedure out of pocket -- without the substantial discounts given to insurers. Charges for a liver transplant and immediate follow-up care generally exceed $523,000, according to an April report by Milliman Inc., an actuarial firm.
It could not be determined how much UCLA and Busuttil were paid for the Japanese transplants.
Tom Mone, chief executive of OneLegacy, the group responsible for procuring and distributing organs in much of Southern California, said transplants for foreign criminals are "an unfortunate result of a system that's magnanimous to the world."
Mone also said hospitals do not have the resources to investigate their patients. "The enforcement should be at the borders, not at the hospital," he said.
In recent years, nonresident foreign nationals have accounted for less than 1% of all transplant recipients nationwide, transplant statistics show.
Dr. Mark Fox, associate director of the Oklahoma Bioethics Center, said the UCLA transplants may create pressure to eliminate transplants for foreign nationals entirely, which Fox said he does not support.
"For some people, there are misgivings for transplanting foreign nationals at all. For some people, there are misgivings about transplanting criminals at all," he said. "When you put those two together, it is certainly reasonable to expect that a certain portion of the population would say, 'This is not what I expected when I signed my donor card.' "
john.glionna@latimes.com
charles.ornstein@latimes.com
The Times' Tokyo bureau and staff writer Teresa Watanabe in Los Angeles contributed to this report.
Erbitux slightly boosts survival in cancer study
By MARILYNN MARCHIONE 05.31.08, 1:34 PM ET
I was listening to a conference call a couple of weeks ago and the doc was looking for the control group to live between 7 to 9 months. In this trial the control group had a median overall survival of 10.1 months. He was looking for a survival benefit of 20 to 25 percent and it only had a 10 percent survival benefit.
CHICAGO - Adding the novel cancer drug Erbitux to standard chemotherapy helped advanced lung cancer patients live just a month longer than chemo alone, a study found.
Although this is the first study to find a survival benefit from a newer, more targeted cancer drug as initial treatment for lung cancer patients, the results left doctors mostly disappointed.
"It's a very small benefit. No one should try to make any more of it than that," said Dr. Roy Herbst, lung cancer chief at the University of Texas M.D. Anderson Cancer Center in Houston.
He consults for Erbitux's makers but had no role in the study, which is to be presented Sunday at a meeting in Chicago of the American Society of Clinical Oncology.
Results were released Saturday because a news release was inadvertently published early.
Lung cancer is the world's top cancer killer, claiming 1.3 million lives each year. In the United States, 215,000 new cases and 162,000 deaths from the disease are expected this year.
Most are non-small cell - the type in the new study. Five-year survival is only 15 percent, mostly because the cancer usually has already spread by the time it is diagnosed.
Erbitux is already used to treat advanced colon cancer, and is best known for embroiling homemaking queen Martha Stewart in an insider trading scandal several years ago.
The new study tested it in 1,125 people with lung cancer that had already spread widely. Average survival was just over 11 months for those given Erbitux on top of standard chemotherapy, versus just over 10 months for those on chemo alone.
The study was led by Dr. Robert Pirker at the University of Vienna in Austria. It was sponsored by Germany's Merck (nyse: MRK - news - people ) KGaA, which markets Erbitux with the drug's initial developer, ImClone Systems Inc. (nasdaq: IMCL - news - people ), and Bristol-Myers Squibb Co. (nyse: BMY - news - people )
The companies announced last fall that the study had met its main goal of improving survival, but no numbers were released until now.
Several other targeted drugs are used to treat advanced lung cancer but not as initial treatment. Erbitux is another possibility "in a field that needs all the help it can get," because the cancer is so lethal, said Dr. Nancy Davidson, a cancer specialist at Johns Hopkins University who is president of the oncology society.
Recently, California-based Genentech (nyse: DNA - news - people ) announced that its targeted drug Avastin failed to improve survival in advanced lung cancer patients like those in the Erbitux study.
Heparin fix leads to new concerns
www.baltimoresun.com/news/health/bal-te.heparin15may15,0,581890.story
baltimoresun.com
Heparin fix leads to new concerns
Shortage after recall has led to rationing, fear of dosing errors
By Jonathan D. Rockoff
Sun reporter
May 15, 2008
WASHINGTON
Efforts to make the blood thinner heparin safer - and to replace supplies that were depleted by a major recall this year - have meant unintended and fresh safety concerns for hospitals, heart clinics and dialysis centers that use it.
The drug, a staple of medical care prescribed tens of millions of times a year, was recalled in February after contamination during production in China led to as many as 81 deaths in the United States. Its leading maker, Baxter International Inc., has suspended manufacture of most of its heparin products.
The resulting shortage of heparin - the least expensive and most commonly used drug of its type - means heparin from new suppliers is arriving in different quantities and strengths than medical staffs are accustomed to, and pharmacists and others worry that patients may be vulnerable to receiving improper doses.
Even before the drug's recall, proper administration was a major concern. Heparin is one of the five drugs most commonly associated with errors in hospitals, an issue publicly highlighted last year when the actor Dennis Quaid's newborn twins were mistakenly given 1,000 times the intended dosage.
Neither the Food and Drug Administration nor the private Institute for Safe Medication Practices has received reports of medication errors since the recall, but some medical centers are establishing extra precautions.
"I would be very concerned," said Michael Cohen, president of the Institute for Safe Medication Practices near Philadelphia, which is planning to warn about the higher risk of medication error in the next newsletter it sends to the country's 6,000 hospitals. "I wouldn't be surprised if it has already happened."
The problem, pharmacists and others say, is that doctors and nurses may be unfamiliar with new packaging for heparin and could easily give a patient a more potent dose than intended. Unlike the single-dose products typically distributed by Baxter, many of the vials now contain larger or more potent quantities.
"It can lead to dosing errors ... that could harm the patient," said John R. DiBona, director of pharmacy at LifeBridge Health, the parent company of Northwest and Sinai hospitals in the Baltimore area.
At Northwest and Sinai hospitals, DiBona said, pharmacists have taken several steps to prevent medication errors, such as reminding nurses to draw the drug from a vial instead of using a pre-filled syringe. The hospitals have removed the syringes so nurses won't make a mistake while switching between two different products.
Similarly, Children's Hospital in New Orleans, which has been forced to buy heparin products with half the potency it used to get, has given nurses extra training in administering the drug. The hospital also keeps the entire supply in its pharmacy so that nurses are automatically reminded to use a higher concentration when picking up the medication, said Helen M. Calmes, assistant director of pharmacy.
"You just don't want to put them out there for someone to grab," Calmes said.
Heparin supply was disrupted after hundreds of people suffered allergic-type reactions from contaminated heparin. Baxter was forced to recall most of its heparin products. In the company's absence, two smaller makers have significantly increased production, and federal health officials insist that supplies are sufficient.
But the American Society of Health-Systems Pharmacists, representing 30,000 pharmacists at hospitals and other medical facilities, lists heparin among the drugs in short supply. And pharmacists at several hospitals around the country said in interviews that their suppliers were rationing heparin products due to the shortages.
"They're scrambling to make do with whatever heparin products they can get their hands on," said Ronald A. Hartmann, vice president of the pharmacy division at MedAssets Supply Chain Systems in St. Louis, which negotiates drug purchases on behalf of 1,000 hospitals, dialysis centers, surgery clinics, nursing homes and doctor's offices.
Northwest and Sinai hospitals have been able to muster just a third of their normal supply because of rationing by their supplier, DiBona said. Their supplier, McKesson Corp., declined to comment.
To fill the gaps, some hospitals have stepped up use of alternative drugs, including a more purified form of heparin, that works just as well at preventing blood clots. But the alternatives are far more expensive, and hospitals have scrounged for whatever dose sizes and concentrations of heparin they can get their hands on.
For the University of Utah Hospitals and Clinics, that has meant buying heparin in 20-dose vials rather than in syringes filled with a single dose that a nurse could readily administer, said Erin R. Fox, a pharmacist at the Salt Lake City hospitals, which monitors drug shortages for the American Society of Health-System Pharmacists.
The hospitals, which were using 5,000 of these syringes a month before the recall, have been able to meet their needs by buying the larger vials, Fox said, but nurses must draw individual doses into syringes and sometimes refrigerate them before administering the drug.
"Smaller hospitals may not be equipped with the right sterile area to prepare batches of syringes. They may not have the manpower either," Fox said. Her hospitals and clinics are training nurses in drawing the right dose and posting reminders to prevent medication errors.
Pharmacists and industry officials proposed a variety of explanations for the heparin shortages, starting with hospitals' need to replace most of the existing supply from scratch. Also slowing the path to market, pharmacists and officials said, is a new requirement that heparin products must undergo sophisticated testing for the contaminant before being sold.
As supplies are replenished, industry officials see the high demand weakening. U.S. Pharmacopeia, a private group that sets the standards for drug production and quality in the United States, is helping heparin makers prepare to conduct in-house testing for the contaminant soon to speed up production, said Dr. Darrell Abernathy, chief science officer.
Johns Hopkins Hospital has already seen improvement, said Brian Pinto, a drug information specialist at the Baltimore hospital.
After the recall, Hopkins was getting less than half of its normal heparin supply, Pinto said. Within the past few weeks, its supply has returned to almost pre-recall levels, but the hospital is not getting the drug in the form it had normally used. "Those are in short supply," he said.
jonathan.rockoff@baltsun.com
Copyright © 2008, The Baltimore Sun
every should be allowed to have their dreams come true
video
http://a11news.com/92/sheyla-hershey/
From the Mannkind call
Alfred Mann
Thank you, Peter. Matt provided an update on first quarter financials and has reported that we are on plan, even somewhat better than plan. Hakan and Peter have spoken about our clinical program, our progress with the manufacturing facility, some of our product pipeline opportunities, and recent events about EXUBERA and Inhaled Insulin.
I would like to take a few moments to make some additional comments about that EXUBERA report, and about the market's reactions to the announcement by Pfizer of a numerical imbalance in their clinical trials in the number of primary lung cancer cases among EXUBERA treated patients versus controlled patients.
It is an understatement to say that we were dismayed that from a Pfizer report, so many of the analysts were quick to write off Technosphere Insulin MannKind and that investors have greatly devalued our stock in the wake of that announcement. It was almost as if we had announced a numerical imbalance of lung cancer patients for our own product or that we even acknowledge a genuine safety signal for EXUBERA.
The reaction was that strong, but that is not the case. First of all, as Peter noted while there was an imbalance in the Pfizer experience and in the two cohorts. The number of cases of lung cancer in the primary patients did not of itself signal a safety risk for EXUBERA. The actual incidence of lung cancer was consistent with statistics within the general population and that incidence was actually low because every one of those patients was a smoker. According to Pfizer and the FDA, EXUBERA remains a safe and effective medicine.
As to Technosphere Insulin, we have seen no lung cancer signaled in our clinical data. Our independent data safety monitoring board has consistently recommended that our trials continue without change. Even after Pfizer’s announcement, the DSMB found no basis to discontinue or otherwise modify our trial.
Let me also remind you that both Lilly and Nova Nordisk announced that there were no safety issues with their products. Both Abbott and Aradigm have announced that they have not seen any cancer signals in the data related to their inhaled insulin programs.
So what does this all mean for the future of inhaled insulin? After all three major companies have withdrawn from this market. But none of those products delivered any clinical advantages over existing standard of care rapid acting insulin analogs. Without a valid value proposition reimbursement was a serious obstacle for all those products.
One might even ask why Lilly and Novo Nordisk ever pursued those products and what really led to the termination of Ara and AERx. Consider the business rational for Lilly and Novo, for both company's injectable rapid acting insulin or analogs are major blockbuster products.
Why cannibalize those substantial profit drivers with a product directed to the same market but with lower margins. For example Lilly's air system had an effective bioavailability of only 7% to 8%, thus requiring 13 times more insulin, as well as all the processing costs and device costs and also the payments to Alkermes. As a consequence the economics made no sense for them. Lilly also decided the regulatory environment as part of the rational for termination. But in reality surely the economic issue dictated termination.
In the case of Novo Nordisk the reason was similar and even clearer. While pulmonary delivery of rapid acting insulin made no sense, Novo Nordisk is continuing to pursue an inhalable lung acting insulin. Why? I say because Novo Levemir twice daily injectable basal insulin is not competing well against Lantus from sanofi-aventis's.
So, what can we incur from all this after the market failure of EXUBERA both Lilly and Novo re-examined their inhaled insulin programs. It seems evident that both those companies were pursing this route of delivery only for defensive reasons. I say that after the demise of EXUBERA, the termination of both Ara and AERx were best cases where Lilly and Novo Nordisk simply found those products not to be economically justified within their product portfolio and found no reason, no defensive reason to continue them.
On the other hand, our product has much better viability than the others and clear clinical advantages over today's standard of care. With our data to-date we have never had any difficulties explaining our value proposition to physicians and why Technosphere Insulin become a valuable addition to the diabetes treatment option and why it should be prescribed for broad array of patients.
We're also finding encouragement from peers for re-imbursement, after all ours is not just another inhaled insulin, instead we have tried to define our product as the first in a new class of also ultra rapid insulin and the insulin that most closely mimics normal physiologic release by a healthy pancreas.
This is not just my view, this was firstly tested by Jay Skyler a world leader in diabetology and his description has since been embraced by other key opinion leaders. No other product has ever come close to TI's kinetics and those kinetics are absolutely needed to avoid the complication of diabetes.
Let me explain. Glucose of course is a fuel to the body, but must be controlled or serious consequences can ensue. A person is faced with two independent sources of glucose, that which is ingested in meals and that which is provided by the liver to fuel the body between meals. Basic control science dictates that to control the system with two independent variants require use of two separate and independent controls. Even nature realizes this. When a person eats a meal, the pancreas reacts with the meal by releasing a surge of insulin within minutes and that turns off delivered during meal digestion.
The normal body does feel the mealtime glucose load separately from fasting glucose loads. In contrast to the insulin released by the normal pancreas of a healthy person which reaches peak plasma concentrations in less than 10 minutes, subcutaneous prandial insulin peak in two to three hours, for regular insulin about 50 to 80 minutes or so for rapid analogs and the other inhaled insulins.
Moreover, the activity of those insulins continues for many hours long after the meal has already been digested. Compares this to Technosphere Insulin peaks in 12 to 14 minutes, almost as fast as in normal pancreas and with the insulin activity closely synchronized to meal digestion.
TI is gone before there is any excess insulin problem. These kinetics result in tremendous advantages. TI therapy can produce exception control of both post-prandial glycemic excursion, with very little if any risk of hypoglycemia. No need for complete meal titration and no weight gain, even weight loss for prior insulin users.
I believe that Technosphere Insulin is a truly disruptive technology that can change the way diabetes is treated. Independent key opinion leaders have postulated that TI even has the potential to stop or at least to slow the progression of type 2 diabetes. I believe this product has a tremendous future and I refused for it to be defined by the failure of others that do not have viable value propositions.
I do understand that we now have to overcome new marketing obstacles, but I say that these obstacles are based on perceptions, not on facts. Those of you who know me and my history will know that I understand what it takes to pioneer disruptive technology and I tell you I am up to this challenge.
To illustrate this point, let me remind you of remarkably similar crisis exploded in the early days of insulin pump therapy when I was at MiniMed. Eli Lilly, Novo Nordisk and Baxter were early competitors in the insulin pump along with MiniMed.
Another major European company was conducting a trial of instant insulin pump and in that trial several people died from severe hypoglycemia. There was uproar about the dangers of insulin pump therapy and the experts offered that the insulin pump market was dead. One by one Novo, Lilly and Baxter terminated their insulin pump programs. Lilly even paid MiniMed to supply replacement pumps for its patients, but MiniMed did not follow the herd.
We believe that our device was superior and different from the other offerings and so we continued. Ultimately, we prospered. MiniMed went public in 1995 and was later acquired. MiniMed's value escalated from the early price of $1.75 per share to its split adjusted $192 at the time when Medtronic acquisition in 2001. That was the return to the early investments over $100 for every dollar invested.
I want to state that MiniMed's past performance is no guarantee of MannKind's future results. Yet the parallel with MannKind situation today to that of MiniMed is quite interesting. Even two of the three players are the same, but please understand I am not being critical of Lilly or Novo for having abandoned their pump program back then or their inhaled insulins now. Those companies have always had multiple products under diabetes franchises.
So, for them it has simply been a matter of managing their product portfolio. The situation for both companies back them is not so different for that and believe is driving their decision about inhaled insulin today. On the other hand, innovators like MiniMed and MannKind focus on unique, singular qualities of their superior technology without worrying about balancing the profitability of the mixed product, some of which are potentially competitive with each other.
So, the situation we find ourselves in today is that all the other major players have removed their inhaled insulin products from their portfolio and have left the field open to MannKind. While we might deal with the perceptions of the current doubters, in the end this should be an important positive for MannKind. I want to reiterate my own conviction about Technosphere Insulin as well as that of the company and the entire team. We are all fully committed and remain so fully committed to take this for insulin. At all levels of our step, there is wide spread confidence in our future.
We believe Technosphere Insulin be the most effective means, indeed the only current means for safely and effectively controlling prandial glucose excursions and it does this without the historically problems of insulin therapy. I assert that Technosphere Insulin has the capacity to increase both the safety and efficacy of diabetes therapy in very significant ways that today are poorly met with available treatments. MannKind is much more that just a company with Technosphere Insulin.
Until now we have not spoken much about our pipeline even though early it is very strong, because we are so focused on TI all the other products are still in early stages and years before commercialization. But exciting are these other products, our focused over this next period will continue to be on Technosphere Insulin. Our intention has been defined a partner that shares our vision and is fully committed as we are to the value of Technosphere Insulin.
But given the reaction to the latest EXUBERA issue, we decided to reassess our partnership talks at least until our pivotal data is available. We view the increase in our challenge for commercialization to be caused not by anything real but by the perceptions raised by the exits of the other players and by the latest EXUBERA scare.
In summarizing, let me say that Mannkind is continuing its development of Technosphere Insulin that we are in course to file our NDA that we have not to-date seen any reason to be concerned about safety or efficacy of these products that the issues with EXUBERA may have led to certain hasty and I believe in accurate perceptions that personally none of EXURBERA problems have been seen with Technosphere Insulin, that we believe Technosphere Insulin is the most effective means to control prandial glucose excursions, that prandial excursions must be controlled if we hope to safely bring most diabetes patients to a normal A1c's. That our factory is nearing completion, and that I and the entire management team at MannKind continue to be committed to this project and to its success.
Arixtra(R) Significantly Reduced Major Bleeding Compared To Lovenox(R) In A Study Of Patients With Acute Coronary Syndromes
10 Apr 2006
why does everyone give these outlandish valuations to mnta because of its generic lovenox when arixtra will take business away from lovenox when they get their label expanded?
Data published today in the New England Journal of Medicine from OASIS 5, one of the largest clinical trials ever conducted in patients with acute coronary syndromes (ACS),demonstrated that treatment with the antithrombotic medicine, ARIXTRA(R) (fondaparinux sodium) significantly reduced major bleeding by nearly half (48 percent) as compared to Lovenox(R) at day 9. The resultsshowed ARIXTRA, manufactured by GlaxoSmithKline, demonstrated comparable efficacy to Lovenox(R) at day 9.
Previous studies have shown that blood thinners can substantially reduce the risk of heart attacks in patients with ACS; however, this may be accompanied by an increased risk of major bleeding. Therefore, there is a need for effective therapies in ACS with a lower incidence of major bleeding.
"These data are very encouraging," said Dr. Salim Yusuf, principal investigator of the study and professor of medicine, McMasterUniversityand Hamilton Health Sciences, Ontario, Canada. "This patient population needs treatments that have the benefits of standard therapies while also reducing the bleeding risk."
ARIXTRA is not approved for use in any country in patients with ACS.
About the Study: Organization to Assess Strategies for Ischemic Syndromes (OASIS) 5
The OASIS 5 program is an international, randomized, double-blind, controlled study that included more than 20,000 patients at 576 sites across 41 countries. Patients in the study were randomized to receive either ARIXTRA 2.5 mg once daily up to eight days or Lovenox(R)1 mg/kg twice daily for two to eight days. Study medications were administered in addition to standard medical care, such as aspirin,clopidogrel, glycoprotein IIb/IIIa inhibitors and revascularization procedures.The primary efficacy objective of the study was to evaluate whether ARIXTRA was as effective as Lovenox(R) in preventing death, myocardial infarction (MI) or refractory ischemia in the acute treatment of ACS patients with unstable angina or non-ST-segment elevation MI (NSTEMI). The primary safety objective was to evaluate major bleeding events up to day 9.
Unstable angina (potentially life-threatening chest pain) and acute MI (heart attack) caused by inadequate blood supply to the heart muscle are part of a complex group of cardiac diseases called ACS. NSTEMI is a type of heart attack typically caused by a severely, but not completely, blocked heart artery.
Prespecified secondary outcomes included the following: death or myocardial infarction; death, myocardial infarction, or refractory ischemia; and the individual components of these composite outcomes at 30 days and at the end of the study.
Headline Results:
ARIXTRA was as effective as Lovenox(R) for the primary composite endpoint of preventing death, myocardial infarction, and refractory ischemia (5.8 percent and 5.7 percent incidence, respectively) at nine days. ARIXTRA was also associated with a 48 percent (p<0.001) reduction in major bleeding versus Lovenox (2.2 percent and 4.1 percent incidence, respectively) in this study.
The study also showed mortality rates at one month were 2.9 percentin the patient group receiving ARIXTRA and 3.5 percentin the patient group receiving Lovenox, representing a 17 percent reduction (p=0.02) in favor of ARIXTRA. All-cause mortality rates at six months were 5.8 percentamong patients receiving ARIXTRA and 6.5 percentin the Lovenox group (p=0.05).
"New antithrombotic therapies for patients with ACS are needed, and GSK looks forward to submitting these data to regulatory agencies worldwide for review so that we may bring this important therapy to physicians and patients," said Dr. Lawson Macartney, senior vice president, Cardiovascular and Metabolic Medicine Development Center, GlaxoSmithKline.
Acute Coronary Syndromes
ACS account for about 2.5 million hospital admissions worldwide and are a major cause of mortality and morbidity in Western countries.1 There are three main cardiac diseases that make up ACS conditions: unstable angina or chest pain, non ST-segment elevation myocardial infarction (NSTEMI), and STEMI; the latter two are also known as heart attacks.2,3 STEMI is a severe heart attack in which there is irreversible myocardial damage as a result of insufficient blood supply to the heart muscle (or myocardial ischemia).3
ARIXTRA
ARIXTRA is the first in a class of antithrombotics that selectively inhibits Factor Xa, a central protein in the coagulation process. In the treatment of thrombosis, Factor Xa plays a central role in the generation of thrombin, a protein in blood that facilitates blood clotting.
For more information about ARIXTRA, please visit http://www.gsk.com.
ARIXTRA is the first selective inhibitor of Factor Xa, a protein central to the coagulation process. ARIXTRA is approved in the United Statesfor the prevention of VTE, which includes DVT and PE, in patients undergoing surgery for hip fracture (including extended prophylaxis), knee replacement, hip replacement, and in abdominal surgery patients who are at risk for thromboembolic complications. Additionally, ARIXTRA is indicated for the treatment of acute DVT when administered in conjunction with warfarin sodium and for the treatment of acute PE when administered in conjunction with warfarin sodium, when initial therapy is administered in the hospital.
Important Safety Information
Contraindications
In Europeand the United States, ARIXTRA is contraindicated in patients with severe renal impairment. In the United States, ARIXTRA is also contraindicated in patients weighing less than 50 kg (less than 110 lbs) who are undergoing major surgery of the lower limbs and abdominal surgery. ARIXTRA is contraindicated in patients with active major bleeding, bacterial endocarditis, and patients with hypersensitivity to fondaparinux sodium.
Warnings
When epidural/spinal anesthesia or spinal puncture is employed, patients anticoagulated with low-molecular-weight heparins, heparinoids or fondaparinux sodium are at risk of developing an epidural or spinal hematoma, which can result in long-term or permanent paralysis. The risk of these events may be higher with postoperative use of indwelling epidural catheters or concomitant use of drugs affecting hemostasis. Spinal/epidural anesthesia should not be used concurrently with ARIXTRA for the treatment of VTE (See BOXED warning in US Prescribing Information).
ARIXTRA is not intended for intramuscular administration.
ARIXTRA should be used with caution in all patient groups with increased risk of bleeding. This includes the elderly, and patients with moderate renal or severe hepatic impairment. In the EU, ARIXTRA should be used with caution in those patients weighing less than 50kg (less than 110lbs). ARIXTRA should not be co-administered with drugs that may increase the risk of bleeding.
The efficacy and safety of ARIXTRA in patients with heparin-induced thrombocytopenia type II has not been studied. Thrombocytopenia can occur during a treatment with ARIXTRA and if the platelet count falls below 100,000/mm3, ARIXTRA should be discontinued.
About GlaxoSmithKline 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.
Cautionary statement regarding forward-looking statements
Under the safe harbor provisions of the US Private Securities Litigation Reform Act of 1995, the company cautions investors that any forward-looking statements or projections made by the company, including those made in this announcement, are subject to risks and uncertainties that may cause actual results to differ materially from those projected. Factors that may affect the Group's operations are described under 'Risk Factors' in the Operating and Financial Review and Prospects in the company's Annual Report on Form 20-F for 2005.
References
1. Mehta S.R. Efficacy and safety of fondaparinux compared to enoxaparin in 20,000 high risk patients with ACS without ST elevation; the OASIS 5 Michelangelo programme. Presentation at the European Society of Cardiology, 5 September 2005(http://www.escardio.org/knowledge/OnlineLearning/slides/ ESC_Congress_2005/Mehta-FP1332).
2. An International Randomized Double-Blind Study Evaluating the Efficacy and Safety of Fondaparinux versus Enoxaparin in the Acute Treatment of Unstable Angina / Non ST-Segment Elevation MI Acute Coronary Syndromes. Population Health Research Institute (http://www.cardio.on.ca/oasis5/).
3. ARIXTRA Prescribing Information, GlaxoSmithKline, 2005.
*Lovenox(R) is a registered trademark of Sanofi-Aventis.
http://www.gsk.com
"There is uncertainty regarding the terms of the Isis-Genzyme collaboration, which was announced in January 2008 but has not been signed, sealed and delivered," Monane added.
How often are agreements announced without being signed. As usual I don't believe he knows what he is talking about. Genzyme may want to change the terms and Isis would probably have to let them because most partners would not want to touch this now, but I am sure the deal has been signed
the auction rate securities should not be written off. I don't understand that accounting.
interests in synthetic collateralized debt obligations referencing portfolios of corporate bonds
actually I don't consider these auction rate securities. they are much more risky and I don't know what they were thinking
never mind what I am saying below because that refers to true auction rate securities
As long as the interest is still being paid, in fact at a higher rate, the asset is still good.
It should be reclassified from a short term to long term investment.
Most of these securities are issued by Municipalities that aren't going anywhere
The FDA have given a thumbs down to Genzyme's request for permission to sell its Pompe disease drug Myozyme manufactured in an Allston, MA plant.
someone sent me this, it seems like it was from the Boston Globe but I am not sure.
Genzyme is already manufacturing the drug in a smaller Framingham-based plant and wants to ramp up production at the larger Allston location. But the FDA ruled that the Allston-produced Myozyme must "be classified as [a different product] because of differences in the carbohydrate structures of the molecules." The FDA said Genzyme has to submit a separate BLA to gain approval for Myozyme produced on a larger scale.
This rejection underscores the difficulty biosimilars face in the United States. "The FDA decision...suggests that regulators may be reluctant to approve any generic versions of biologic drugs...without clinical data proving the drugs are at least as safe and effective as the originals if there are even slight differences in the compounds," observes the Boston Globe. Currently, the U.S. has no pathway to quickly approve biosimilar drugs.
As Genzyme points out, Myozyme production has already been scaled up in 40 other countries. "Based on the global clinical experience of nearly 900 patients of all ages currently receiving Myozyme produced at the larger scale...Genzyme believes that Myozyme produced at both the 160L and 2000L scales is clinically effective and safe," the company said in a statement. The company expects the FDA to act on the application by the end of the year.
New drug offers hope to leukemia victims
April 1, 2008 - 2:07PM
http://news.smh.com.au/new-drug-offers-hope-to-leukemia-victims/20080401-22w3.html#
Advertisement
A new drug under development by a listed Australian company could soon bring hope to sufferers of chronic myeloid leukemia (CML), one of the deadliest forms of cancer.
Biopharmaceutical company ChemGenex says it has received encouraging results from the last phase of ongoing trials of the drug omacetaxine mepesuccinate (formerly known as Ceflatonin) on patients in the chronic phase of the disease who have already failed other forms of chemotherapy.
"Of those patients, about 86 per cent or 87 per cent of patients are responding well and benefiting from treatment," ChemGenex chief operations officer Dr James Campbell told AAP Tuesday.
"This is a very, very exciting development.
"Our goal is obviously to improve the outcome for patients and we see omacetaxine as another tool that would go into the armory of effective therapeutics for doctors."
Dr Campbell said the latest results from an ongoing trial of 34 patients in the United States and Europe were released Tuesday to the New Directions in Leukaemia Research Conference on the Sunshine Coast, and to the Australian Stock Exchange.
ChemGenex will early next year seek approval for its as yet unnamed new drug with the US regulatory agency, the Food and Drug Administration (FDA), and then with Australia's Therapeutic Drugs Administration (TGA).
CML has been treated for around seven years with the drug Glivec in a global market estimated to be worth around $US3 billion a year, Dr Campbell said.
"That's been a very successful drug but what is happening over time is that an increasing number of patients are becoming resistant to the drug," he said.
"Our drug is basically targeting patients who are becoming resistant to Glivec."
Omacetaxine uses chemically-modified extracts of leaves from the Japanese yew tree grown in a plantation in China.
The drug is self-administered by the patient via injection twice a day for one week each month.
It is less invasive than conventional forms of chemotherapy, Dr Campbell said.
"It's not like going into hospital and being set up on a drip and having a constant infusion for 24 hours," he said.
Dr Campbell expects the world-wide market for the new drug will be around $200 million "in the early days".
"But that will grow," he said.
The drug, which is likely to be manufactured in Switzerland, was also expected to boost the company's bottom line.
"We believe very passionately that omacetaxine is very exciting and obviously from a management point of view, it's very positive for the outlook for the company," Dr Campbell said.
If ChemGenex received FDA approval next year, omacetaxine was likely to be available in Australia in 2010.
But it won't be cheap.
Dr Campbell said new drugs such as omacetaxine traditionally cost patients around $50,000 a year.
"It could cost around that figure," he said.
"But whilst $50,000 sounds like a lot of money if you are paying for it yourself, with insurance rebates and PBS (Pharmaceutical Benefits Scheme) coverage, obviously it would not be of that magnitude."
Dr Campbell said ChemGenex would be seeking PBS listing once the drug had been granted regulatory approval in Australia.
© 2008 AAP
Epix follows a new course in developing CF program
http://www.fiercebiotech.com/special-reports/emerging-drug-developer-epix-pharmaceuticals?utm_medium=nl&utm_source=internal
For most investors, the sweet spot in any biotech play is right around proof-of-concept data on a new therapy for a disease that afflicts a large population of patients. That’s the point when a big pharma company can size up the commercial opportunities along with late-stage development costs and step in and make an offer to partner up.
It’s also the reason why emerging drug developers like Epix focus on diseases like Alzheimer’s and obesity, where current therapies often don’t work very well and the unmet medical need and market potential is greatest.
But two years ago the Cystic Fibrosis Foundation made Kauffman an offer that made him disregard the traditional business model and move into a disease category that otherwise wouldn’t have made much economic sense. The foundation put up $12 million to fund a program at Epix that put the biotech’s technology to work exploring a new therapy for cystic fibrosis. Rather than look for a market payoff, the foundation’s primary objective is to advance the research far enough along that the commercial opportunity comes within reach -- and the biotech can advance on its own to commercial development.
Last week, after Epix hit its fourth milestone in that development pact, the foundation came back and added a very big carrot in the form of an additional $37 million in fresh research funds.
“This funding goes through Phase IIa, early stage clinical proof-of-concept,” says Epix CEO Michael Kauffman, M.D., Ph.D. “If we see an effect in the clinic by then, I would stand by this. We would be very excited about this mechanism.” The foundation, he adds, “wants to see this disease treated. They want to take it to a point of top priority. There’s a different late-stage focus for the foundations and nonprofits.”
Epix’s success in the field comes from its expertise in developing computer models of the 3-D structures of drug targets – in this case the cystic fibrosis transmembrane conductance regulator, or CFTR. The most common mutation of the CFTR gene is the Delta F508 mutation, which is responsible for key symptoms of CF in about 70 percent of the patients who suffer from the disease. The body eliminates the abnormal protein made from the mutated gene, and the reduced levels of CFTR protein spur the creation of a sticky mucous material that causes lung infections and disrupts digestion in people who suffer from the disease.
“What happens is that the Delta F508 protein is made just fine,” says Kauffman. But the correct 3-dimensional folding process that normally follows goes awry. The body in turn detects the misfolded CFTR proteins and discards them as it normally does to protect the body from abnormal proteins. “The problem is that less than five percent of the Delta F508 ever makes it to the surface, so people with CF have a level of CFTR maybe 20- to 50-fold lower than normal, which is why you get the disease. The little bit that gets to the surface also doesn’t function as well as the normal protein.”
Any new therapy doesn’t have to be 100 percent effective, he adds. Even a 20 percent to 25 percent increase in CFTR would probably trigger a considerable improvement in patients.
Understanding the structure of CFTR gives Epix an inside track in developing a therapy that can make the protein work properly, eliminating those symptoms.
“We create our own computer-derived model we think is right,” says Kauffman. “Essentially, we’re trying to replace X-ray crystallography.”
To illustrate what he’s talking about, Kauffman frequently uses an analogy of examining a lock – the drug target -- to come up with a key – the drug. “If you know the structure inside the lock, it’s far easier to make a key.” That’s a far different approach than high-throughput screening technology, where you try a few hundred thousand keys to see which ones best fit the lock.
Epix developed the technology around G protein-coupled (GPCR) receptors that play a role in some ‘big’ diseases like Alzheimer’s. And the company has developed partnerships with the likes of GlaxoSmithKline and Amgen to advance new therapies into mid-stage clinical development.
The Cystic Fibrosis Foundation “came to us and said, Can you modify your technology so you can come up with a 3D structure of what this ion channel protein looks like and use this structure to come up with new drugs to treat cystic fibrosis?” recalls Kauffman. “Ion channels have a different structure than GPCRs, but both traverse the cell membrane, which makes them very difficult to crystallize. But they do have certain properties in common.”
Over the past 10 years, the Foundation has pumped $300 million into research programs.
And it isn’t hard to find other examples of non-profits stepping in to help fund early-stage research work. The Boston Globe recently concluded that the disease foundations provided $75 million to drug developers last year, up from only $7 million in 2000.
Aside from the CF program, Epix has a number of irons in the fire as it pursues a total of five clinical development programs.
Kauffman just killed off a mid-stage development program in depression after failing to come up with the kind of compelling data needed to justify continued clinical work. That program is up on the auction block. And while Kauffman points to its added potential in pain and memory loss on top of depression, he also is realistic about its potential following a mid-stage trial failure.
But Kauffman believes the company can easily overshadow that setback with a new review of its crucial Vasovist Phase III program, an imaging agent already approved in 33 countries but long stymied at the FDA. Essentially, says Kauffman, the FDA handed the company a “non-decision” in an approvable letter. It took some time, but Epix got the clarification it needed to provide specific data to the FDA and Kauffman says its amended NDA for the FDA could come mid-year with a decision by the agency at the end of this year.
Epix already has a profit-sharing deal on Vasovist. But Kauffman says the company “will likely monetize the asset. We won’t wait for a profit share to pay off; near term cash is more important.”
ChemGenex Reports Oral Bioavailability of Omacetaxine and Expands on Mechanism of Action
Monday April 14, 7:00 pm ET
MELBOURNE, Australia & MENLO PARK, Calif.--(BUSINESS WIRE)--ChemGenex Pharmaceuticals Limited (ASX:CXS - News) (NASDAQ:CXSP - News) announced today that pre-clinical data characterizing the bioavailability and mechanism of action of omacetaxine mepesuccinate (formerly known as Ceflatonin®) were presented on Monday local time at the American Association of Cancer Research (AACR) 99th Annual Meeting in San Diego, California.
Data from ChemGenex researchers and collaborators were presented across three sessions. Highlights include:
Omacetaxine can be delivered orally in animals with oral bioavailability of approximately 75% compared with subcutaneous administration. (Abstract 2275).
Omacetaxine reduced the number of leukemic stem cells in the bone marrow by more than 80% in an animal model of chronic myeloid leukemia (CML). Significantly, the tyrosine kinase inhibitor (TKI) imatinib mesylate did not reduce leukemic stem cell number. (Abstract 2275).
Omacetaxine causes a rapid and dose-dependent reduction in the level of the key regulatory protein Mcl-1 in a cell model of CML. Mcl-1 is a key target protein in several types of leukemias and other cancers. Mcl-1 levels were not reduced in the short term by imatinib mesylate. (Abstract 2350).
“Following the clinical update presented last week showing hematologic and/or cytogenetic responses in 86% of chronic phase patients on trial, this is a timely confirmation of the significant advances we have made in understanding the mechanism of action of omacetaxine,” said Dr. Greg Collier, ChemGenex’s Managing Director and Chief Executive Officer. “In addition, we now have independent confirmation that omacetaxine has excellent oral bioavailability, opening a range of development and potential commercialization opportunities into the future.”
Dr. Collier confirmed that the strategic positioning of omacetaxine is strongly supported by the new mechanism of action data emerging from the laboratories of ChemGenex, its collaborators and independent researchers. “We can reiterate that Mcl-1 is down-regulated by omacetaxine in CML, and that in an animal model the drug acts directly on leukemic stem cells in the bone marrow. We are seeing a range of biological effects of omacetaxine that clearly differentiate it from the TKIs, supporting its clinical development both in CML patients who fail TKI therapy and in other forms of leukemia.”
Ceflatonin® is a registered trademark of ChemGenex Pharmaceuticals Limited.
About ChemGenex Pharmaceuticals Limited (http://www.chemgenex.com)
ChemGenex Pharmaceuticals is a pharmaceutical development company dedicated to improving the lives of patients by developing personalized oncology medicines. ChemGenex harnesses the power of genomics both to discover novel targets and drug compounds, and in clinical trials to develop more individualized treatment outcomes. ChemGenex’s lead compound, omacetaxine mepesuccinate (formerly known as Ceflatonin®), is currently in phase 2/3 clinical trials for chronic myeloid leukemia (CML). ChemGenex has a second anticancer compound, amonafide dihydrochloride (formerly known as Quinamed®) which is in phase 2 clinical development for various solid cancers, and a portfolio of assets in pre-clinical development. ChemGenex currently trades on the Australian Stock Exchange under the symbol "CXS" and on NASDAQ under the symbol "CXSP".
Safe Harbor Statement
Certain statements made herein that use the words “estimate”, “project”, “intend”, “expect”, “believe” and similar expressions are intended to identify forward-looking statements within the meaning of the US Private Securities Litigation Reform Act of 1995. These forward-looking statements involve known and unknown risks and uncertainties which could cause the actual results, performance or achievements of the company to be materially different from those which may be expressed or implied by such statements, including, among others, risks or uncertainties associated with the development of the company’s technology, the ability to successfully market products in the clinical pipeline, the ability to advance promising therapeutics through clinical trials, the ability to establish our fully integrated technologies, the ability to enter into additional collaborations and strategic alliances and expand current collaborations and obtain milestone payments, the suitability of internally discovered genes for drug development, the ability of the company to meet its financial requirements, the ability of the company to protect its proprietary technology, potential limitations on the company’s technology, the market for the company’s products, government regulation in Australia and the United States, changes in tax and other laws, changes in competition and the loss of key personnel. These statements are based on our management’s current expectations and are subject to a number of uncertainties that could change the results described in the forward-looking statements. Investors should be aware that there are no assurances that results will not differ from those projected.
Contact:
ChemGenex Pharmaceuticals Limited
Dr. Greg Collier
Cell – Australia: +61 419 897 501
Cell – USA: +1-650-200-8145
CEO and Managing Director
gcollier@chemgenex.com
or
Buchan Consulting
Rebecca Wilson, +61 2 9237 2800
Cell: + 61 (0) 417 382 391
Media Relations – Australia
rwilson@bcg.com.au
or
Kureczka/Martin Associates
Joan Kureczka, +1-415-821-2413
Media Relations – USA
Jkureczka@comcast.net
--------------------------------------------------------------------------------
Source: ChemGenex Pharmaceuticals Limited
Targeted Genetics Acquires Rights to siRNA Development Program in Huntington's Disease From Sirna Therapeutics
Monday April 7, 7:30 am ET
wasn't this and the als program the lead programs that Sirna had when taken over by Merck. It couldn't have been worth much
SEATTLE, WA--(MARKET WIRE)--Apr 7, 2008 -- Targeted Genetics Corporation (NasdaqCM:TGEN - News) today announced that it has acquired full exclusive rights to its preclinical Huntington's disease (HD) program from Sirna Therapeutics, a wholly owned subsidiary of Merck & Co., Inc.
ADVERTISEMENT
In exchange for these rights, which include a license to intellectual property (IP) that Targeted Genetics may find necessary to develop and commercialize an HD product, Targeted Genetics will pay Sirna an undisclosed royalty on future sales. Sirna has also assigned to Targeted Genetics a licensing agreement it has with the University of Iowa that covers certain IP developed by Dr. Beverly Davidson's laboratory related to RNA interference (RNAi) including Adeno-Associated Virus (AAV) expressed RNAi.
In 2005, Targeted Genetics and Sirna Therapeutics formed a collaboration to develop HD therapeutics using an AAV delivered RNAi approach to target the HD gene. This collaboration also involved Dr. Davidson's laboratory and was based on preclinical proof of concept established by University of Iowa researchers. AAV vectors express for long periods of time and allow for infrequent dosing, which is highly desirable when administering a therapeutic directly to the brain.
"This moves all of the key pieces of this program to Targeted Genetics and gives us exclusive rights and direct involvement with the University of Iowa to expedite the program," said H. Stewart Parker, president and chief executive officer of Targeted Genetics. "This program is our primary proof of concept in the area of expressed RNAi, which we believe could present multiple product opportunities to help patients who currently have little hope for treatment, such as those with HD."
"The results we have produced in animal models of HD are very exciting. We have identified and are currently evaluating lead candidates in preclinical studies to move forward into clinical trials," stated Beverly L. Davidson, Ph.D., Roy J. Carver professor of medicine and vice chair of research in the Department of Internal Medicine at the University of Iowa. "Targeted Genetics' approach of using AAV vectors for the delivery of expressed RNAi may have advantages over alternative RNAi delivery approaches due to AAV's proven long-term expression capabilities, stability and safety profile."
HD is a devastating, inherited, neurodegenerative disorder that results from a mutation in the gene that codes for the huntingtin protein. HD generally shows onset in mid-life and, according to the Huntington's Disease Society of America, one of out of every 10,000 Americans has HD and an additional 200,000 are at risk of onset. The disease-causing gene produces a defective huntingtin protein that is toxic to certain brain cells and the subsequent neuronal damage leads to the movement disorders, psychiatric disturbances and cognitive decline that characterize this disease.
"There is a serious unmet medical need for an effective treatment for HD," said Alan Sachs, M.D., Ph.D., vice president RNA therapeutics, Merck Research Laboratories. "This agreement provides Targeted Genetics with the freedom to advance this AAV vector program in the hope of developing a treatment that targets the genetic cause of this devastating condition, not just the symptoms."
Medicare Finds How Hard It Is to Save Money
my mother in law lives in florida. she went to the hospital with a bad cold. She was there for 3 days and they took all kinds of Mri's and CT scans. It is a wonder that they didn't find anything wrong with her. She glows in the dark now. on the forth day they asked her if she would want to stay another day and she said fine, she won't have to cook.
Is it a wonder medicare is going bankcupt?
The hospital made a bundle on her and until there is something to stop this from happening we are in deep doo doo.
ChemGenex Poised for Rebound With Merck-Beating Drug (Update4)
By Simeon Bennett
April 3 (Bloomberg) -- ChemGenex Pharmaceuticals Ltd., the Australian developer of cancer drugs, may beat Merck & Co. to the market with a novel leukemia treatment derived from the leaves of a Chinese evergreen tree.
The medicine is likely to win U.S. approval next year, according to ChemGenex, located in Geelong, Victoria. Merck, the Whitehouse Station, New Jersey-based drugmaker, and its partner, Vertex Pharmaceuticals Inc., stopped recruiting test subjects for their product in November because of side effects.
While ChemGenex has yet to make a profit, it's at least 18 months ahead of six competitors in a race to develop a therapy for an untreatable form of leukemia -- a market that may be worth more than $230 million next year. ChemGenex, down 23 percent on the Australian Stock Exchange this year, may rebound 53 percent by 2009 as the company completes its tests, says Scott Power, a health-care analyst at ABN Amro Morgans Ltd. in Brisbane.
``It's one of the rare companies, especially in the oncology field, that has a product that's nearing approval, and could really make a difference for a set of patients that don't have any other alternatives,'' said Daniel Janney, who manages about $2 billion in biotechnology and medical-device stocks at San Francisco-based venture capital firm Alta Partners. Alta holds about 20 percent of ChemGenex and Janney is on the board.
ChemGenex fell 5 Australian cents, or 5.8 percent, to 81 cents, valuing the company at A$152 million ($139 million). The shares have risen 65 percent since April 2005.
Philadelphia Chromosome
An estimated 4,830 Americans will be diagnosed this year with chronic myeloid leukemia, according to the American Cancer Society. The slow-progressing cancer develops mostly during middle age and is caused by a genetic flaw known as the Philadelphia chromosome that triggers the overproduction of white blood cells.
Novartis AG's best-selling cancer drug, Gleevec, is the standard treatment for the disease, and earned Europe's third- largest drugmaker $3 billion in sales last year. The Basel, Switzerland-based drugmaker declined to comment on ChemGenex.
A third of leukemia patients fail Gleevec after five years, and about 2 percent develop resistance because of a specific mutation in their cancer cells, said Stephen O'Brien, a leukemia researcher at the U.K.'s Newcastle University.
Mutations develop because of errors in the way cells copy DNA when they divide and multiply. The same so-called T315I mutation also makes patients unresponsive to alternative treatments, including Novartis's Tasigna and Bristol-Myers Squibb Co.'s Sprycel. About 15 percent of Sprycel patients developed the mutation within a median of seven months, according to a study in the journal Hematologica last year.
`Only Viable Option'
Merck has said it will soon begin testing another compound, said Michael Partridge, a spokesman for Cambridge, Massachusetts- based Vertex, which discovered the partnership's drugs.
ChemGenex's drug, omacetaxine, fights leukemia differently than Gleevec, Sprycel and Tasigna by stopping a protein responsible for the runaway growth of white blood cells. The drug is in the final of three stages of patient studies usually needed for a drug's regulatory review.
If approved, it may be the ``only viable option'' for patients with drug-resistant leukemia, said Jorge Cortes, who is leading a study of the drug at Houston's M.D. Anderson Cancer Center.
``It's still very early, but it is looking promising, particularly for patients in the more advanced stages of the disease,'' Cortes said in an interview.
Tests on 25 leukemia sufferers showed omacetaxine fought the cancer in 86 percent of them, ChemGenex said this week. In 9 out of 15 patients with an early stage of the disease, the drug lowered white blood cells to normal levels, and rid cancer cells from three patients.
Stem Cells
The medicine's main side effect was a severe, ``transient and reversible'' decrease in the bone marrow's ability to produce white blood cells, observed in 60 percent of patients, ChemGenex said.
Omacetaxine is derived from the yew tree known as Cephalotaxus harringtonia. Its medicinal properties were probed in a U.S. National Cancer Institute screening program in the 1970s funded after former President Richard Nixon declared war on cancer. The same project uncovered Bristol-Myers' breast-cancer drug, Taxol, derived from Pacific yew trees.
Studies on omacetaxine languished in the 1990s, when Gleevec entered late-stage trials and researchers didn't see a need for a similar treatment. When Gleevec was approved in 2001, ``everyone thought the disease was cured,'' said Greg Collier, ChemGenex's chief executive officer. Interest was rekindled by reports of Gleevec resistance in 2002, Collier said.
Fast-Track Status
ChemGenex said it expects omacetaxine to generate about $230 million in its first year of sales as a treatment for patients with the T315I mutation. The company was awarded fast-track status with the Food and Drug Administration in November 2006 and plans to file for U.S. approval by December.
``Once it's registered it will probably be further investigated for other indications,'' said Darren Grubb, a health-care analyst at Intersuisse Ltd. in Melbourne, who rates the stock a ``speculative buy'' and expects it to reach $1.31 within a year. Grubb doesn't own the shares.
ChemGenex's chief executive, Collier, said in November the company will seek partners this year to market its product, after identifying ``a number of companies that could be natural fits.'' Alternatively, ChemGenex may market omacetaxine independently, the company said.
The most likely suitor is ``probably Novartis,'' said ABN Amro analyst Power, who gives ChemGenex a 75 percent chance of gaining FDA approval for omacetaxine. Power owns the shares.
Novartis spokeswoman Beatrix Benz declined to comment on whether the pharmaceutical company is interested in buying ChemGenex.
To contact the reporter on this story: Simeon Bennett in Singapore at sbennett9@bloomberg.net
Last Updated: April 3, 2008 06:04 EDT
that article I posted was an april fools joke.
The Onion | 9/17/2003
FDA Approves Sale Of Prescription Placebo
WASHINGTON, DC—After more than four decades of testing in tandem with other
drugs, placebo gained approval for prescription use from the Food and Drug
Administration Monday.
"For years, scientists have been aware of the effectiveness of placebo in treating a
surprisingly wide range of conditions," said Dr. Jonathan Bergen of the FDA's Center
for Drug Evaluation and Research. "It was time to provide doctors with this often
highly effective option."
In its most common form, placebo is a white, crystalline substance of a sand
consistency, obtained from the evaporated juice of the Saccharum officinarum
plant. The FDA has approved placebo in doses ranging from 1 to 40,000 milligrams.
The long-awaited approval will allow pharmaceutical companies to market placebo
in pill and liquid form. Eleven major drug companies have developed placebo
tablets, the first of which, AstraZeneca's Sucrosa, hits shelves Sept. 24.
"We couldn't be more thrilled to finally get this wonder drug out of the labs and into
consumers' medicine cabinets," said Tami Erickson, a spokeswoman for
AstraZeneca. "Studies show placebo to be effective in the treatment of many
ailments and disorders, ranging from lower-back pain to erectile dysfunction to
nausea."
Pain-sufferers like Margerite Kohler, who participated in a Sucrosa study in March,
welcomed the FDA's approval.
"For years, I battled with strange headaches that surfaced during times of stress,"
Kohler said. "Doctors repeatedly turned me away empty-handed, or suggested that
I try an over-the-counter pain reliever—as if that would be strong enough. Finally, I
heard about Sucrosa. They said, 'This will work,' and it worked. The headaches are
gone."
Researchers diagnosed Kohler with Random Occasional Nonspecific Pain and
Discomfort Disorder (RONPDD), a minor but surprisingly pervasive medical
condition that strikes otherwise healthy adults.
RONPDD is only one of many disorders for which placebo has proven effective,
Bergen said.
"Placebo has been successful in the treatment of everything from lower-back pain
to erectile dysfunction to nausea," Bergen said. "That's the beauty, and the
2/2
mystery, of placebo. It's all-purpose. Think of it like aspirin, but without any of the
analgesic properties."
The FDA is expected to approve the drug for a wide range of mood disorders later
this year. According to Bergen, initial research has shown placebo to be effective in
the treatment of bipolar disorder, depression, dysthymia, panic disorder, post
traumatic stress disorder, seasonal affective disorder, and stress.
As industry analysts predict the drug's sales will top $25 billion in the first year, the
approval of placebo is expected to unleash one of the pharmaceutical industry's
biggest marketing battles to date.
Above: An advertisement for AstraZeneca's placebo Sucrosa.
GlaxoSmithKline expects to have two versions of placebo on the shelves in late
December. One, a 40-milligram pill called Appeasor, will be marketed to patients 55
and over, while the other, Inertra, designed for middle-aged women, is a liquid that
comes in a 355-milliliter can, and is cola-flavored. Eli Lilly plans a $3 million
marketing campaign for its 400-milligram tablet, Pacifex.
"All placebos are not the same," Eli Lilly spokesman Giles French said. "Pacifex is
the only placebo that's green and shaped like a triangle. Pacifex: A doctor gave it to
you."
Despite such ringing endorsements, some members of the medical community have
spoken out against placebo's approval, saying that the drug's wide range of side
effects is a cause for concern.
"Yes, placebo has benefits, but studies link it to a hundred different side effects,
from lower-back pain to erectile dysfunction to nausea," drug researcher Patrick
Wheeler said. "Placebo wreaked havoc all over the body, with no rhyme or reason.
Basically, whichever side effects were included on the questionnaire, we found in
research subjects."
Added Wheeler: "We must not introduce placebo to the public until we pinpoint
exactly how and why it works. The drug never should have advanced beyond the
stage of animal testing, which, for some reason, was totally ineffective in
determining its effectiveness."
In spite of the confusing data, drug makers say placebo is safe.
"The only side effect consistent in all test subjects was a negligible one—an almost
imperceptible elevation in blood-glucose levels," French said. "It's unfair to the
American people to withhold a drug so many of them desperately think they need.
(e) On March 5, 2008, the Registrant entered into an amendment to its employment agreement with its President, Charles L. Bisgaier, Ph.D. The amendment reduced the President's salary effective immediately from $295,000 to $0 through at least May 17, 2008 at which time the President may be awarded a base salary, at the discretion of the Registrant's compensation committee. The amendment also eliminated the President's guaranteed bonus of approximately $98,000 and the provision for severance upon a termination without cause. Since mid-February, the President has not been receiving any cash compensation.
http://biz.yahoo.com/e/080310/pp8-k.html
Actually this is someone paying for their mistakes. he was supposed to be the internal guy to review the NDA and he screwed up. should they have continued to pay him top dollar?
I read the article. arna did get mentioned in a sidebar. The brief comment was it's considered very risky since its structurally based on one of the fen-phen combination.
I think amln is the real sleeper, but one wart is it will be injectable.
I beleive it is quite a wart. I think they patients are taking injections four or five times a day
The CHMP conducts a nonbinding oral vote of the member-state representatives only in cases where the committee believes there is a substantial difference of opinion, i.e. when there are nearly even votes for acceptance and rejection. There is no evidence that this was done for GTCB’s ATryn application.
So what you are saying is, in the original denial of Atryn the CHMP was sure that there was going to be a denial, so it was rejected without one of those oral hearings?
It would seem to me that since it was rejected originally, they would have wanted to give the company an opportunity to present their case orally. It also makes sense that when it was rejected, it was by a narrow vote, which would mean that there should have been an oral hearing by your definition.
In other words I think the evidence points to the fact that there was an oral hearing when the company's stock crashed weeks before the rejection, but the company only made the response that they hadn't heard from the CHMP.
At least Epct was upfront about it.