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Jav
every analyst and his brother and multiple hedge fund managers are saying get into jav they are going to get a european approval any day.
1.unless it's an orphan drug, european approvals don't generate higher stock prices because the reimbursement rates are low.
2. the approval is already built into the price. Where are all these funds going to go when they want to sell on the news
Countrywide to Cut Workforce 20%
i think the ceo should be the first on the list.
he is dispicable
rex
Canadian companies do bought deals. I have noticed that they halt the stock when that is done
Applera Executes on $600 Million of $1.2 Billion Share Repurchase Authorization
Friday August 31, 7:00 am ET
there are many buyout rumors with this company. If they had any offers could they buy back stock? I believe the management is using every weapon in there arsenal to raise the stock so they can cash out of their options. It is a slow growth business
Applied Biosystems Reports Fiscal 2007 Fourth Quarter Results and Fiscal 2007 Results
Wednesday July 25, 7:00 am ET
-- Q4 net revenues increased by approximately 7% to $557.3 million
-- Q4 fully diluted GAAP EPS from continuing operations of $0.42
-- Q4 non-GAAP EPS of $0.38 from continuing operations, excluding specified items
-- Mark P. Stevenson promoted to Executive Vice President
FOSTER CITY, Calif.--(BUSINESS WIRE)--Applied Biosystems Group (NYSE: ABI - News), an Applera Corporation business, today reported net revenues of $557.3 million for the fourth quarter of fiscal 2007, an increase of approximately 7% over the prior year quarter of $523.1 million. The net effect of foreign currency on net revenues was a favorable impact of approximately 2% as compared to the prior year quarter.
For the fourth quarter of fiscal 2007, the Group reported income from continuing operations of $79.3 million, or $0.42 per share, compared to $76.7 million, or $0.41 per share, for the prior year quarter. Results in both periods were affected by the specified items described in the reconciliation below. Earnings per share (EPS) from continuing operations on a non-GAAP basis, excluding these items, were $0.38, an increase of approximately 9% compared to $0.35 for the prior year quarter. Earnings in the fiscal 2007 fourth quarter were affected by higher investments in marketing and selling functions and in research and development programs compared to the prior-year quarter. The net effect of foreign currency was a benefit of approximately $0.03 per share compared to the prior year quarter. All per share amounts refer to Applera Corporation-Applied Biosystems Group Common Stock.
Net income for the fourth quarter of fiscal 2007 was $87.8 million, compared to $76.7 million for the prior year quarter. Net income for the fourth quarter of fiscal 2007 included an after-tax benefit of $8.5 million for discontinued operations related to the settlement of German tax audits.
"Our businesses continued to perform well during the fourth fiscal quarter, sustaining the improved performance we have seen throughout this fiscal year," said Tony L. White, Chief Executive Officer, Applera Corporation. "An important development was the shipment of our first next-generation sequencing systems to early-access customers. Based on positive customer feedback and progress in our manufacturing process, we are accelerating plans to expand production and commence a full commercial release in October."
Today, the Group also announced the promotion of Mark P. Stevenson, president of the Molecular and Cell Division, to Executive Vice President. In this new position, he will continue to manage the Group's portfolio of DNA analysis systems and consumables for the research markets while assuming responsibility for the Applied Markets and Services Divisions and additional functions, including the European and Japan territories. As part of the new leadership structure, the Molecular and Cell Biology Division will be divided into two divisions: MCB Systems and MCB Consumables.
"This decision reflects Mark's growing contributions across a number of global functions and businesses over more than a decade with the company, in Europe, Asia and the U.S.," said Mr. White. Mr. Stevenson will continue to report to Mr. White, who will remain interim President of the Group. "Mark's strong leadership skills and focus on customer applications and markets are critical to driving growth and winning in today's life science industry."
During the fourth quarters of both fiscal 2007 and 2006, the Group recorded items that affected the comparability of results. For the fourth quarter of fiscal 2007, the Group recorded a pre-tax gain of $3.5 million due to the receipt of past royalties under new and newly amended patent licenses. The Group also recorded amortization expense of $2.8 million related to acquired intangibles. Additionally, the fourth quarter of fiscal 2007 included a net tax benefit of $6.9 million primarily related to foreign tax settlements and the reduction of foreign valuation allowances.
During the fourth quarter of fiscal 2006, the Group recorded items that increased income before taxes by a net $14.0 million. These items included a pre-tax gain of $16.9 million from the sale of a company-owned facility and amortization expense of $2.9 million related to acquired intangibles. The fourth quarter of fiscal 2006 also included a $1.4 million favorable tax rate adjustment.
The following table summarizes the impact of these items on EPS calculations:
Reconciliation of GAAP amounts to Non-GAAP amounts
(Dollar amounts in millions)
Three months ended Three months ended
June 30, 2007 June 30, 2006
------------------------ ------------------------
GAAP Non-GAAP GAAP Non-GAAP
amounts Adj. amounts amounts Adj. amounts
------- ------- -------- ------- ------- --------
Operating income $ 95.6 $ 0.7 $ 94.9 $ 102.1 $ 14.0 $ 88.1
Income before income
taxes 103.5 0.7 102.8 106.8 14.0 92.8
Provision for income
taxes 24.2 (6.5) 30.7 30.1 3.2 26.9
Income from
continuing
operations 79.3 7.2 72.1 76.7 10.8 65.9
Earnings per share
allocations(1) (0.1) (0.1) 1.3 1.3
Adjusted income from
continuing
operations for
earnings per share $ 79.2 $ 7.1 $ 72.1 $ 78.0 $ 12.1 $ 65.9
Total diluted
earnings per share
from continuing
operations $ 0.42 $ 0.04 $ 0.38 $ 0.41 $ 0.06 $ 0.35
(1) Represents allocation of intercompany sales of assets to adjust
net income for purposes of calculating earnings per share.
Quarterly Financial Highlights
Revenues by source and the change relative to the prior year quarter were: $242.6 million for Instruments, a 3% increase; $221.4 million for Consumables, an 11% increase; and $93.3 million for Other Sources, including service and support, royalties, licenses, and consulting, a 5% increase.
Revenues for major geographic regions and their change relative to the prior year quarter were: $231.9 million in the United States, a 1% increase; $210.4 million in Europe, a 14% increase including a favorable impact from foreign currency of approximately 5%; $50.5 million in Japan, an increase of less than 1% including an unfavorable impact from foreign currency of approximately 3%; and $40.8 million in Other Asia Pacific countries, a 9% increase including a favorable foreign currency impact of approximately 1%.
Gross margin in the fourth quarter of fiscal 2007 was 55.4 %, versus 54.3% in the prior year quarter. The increase in gross margin was primarily attributable to improved vendor pricing related to enzymes and the favorable impact of foreign currency.
Selling, general, and administrative (SG&A) expenditures in the fourth quarter of fiscal 2007 increased 7% to $160.2 million, or 28.7% of revenues, compared to $149.9 million, or 28.7% of revenues, in the prior year quarter. The increase in SG&A was driven primarily by certain strategic investments to support growth, the unfavorable effects of foreign currency, costs related to our acquired businesses and higher employee related costs.
Research, development, and engineering (R&D) expenditures in the fourth quarter of fiscal 2007 increased 16% to $53.5 million, or 9.6% of revenues, compared to $46.3 million, or 8.9% of revenues, in the prior year quarter. The increase in R&D expenditures was due to investments in the SOLiD(TM) System for next generation sequencing developed from the Agencourt Personal Genomics technology acquired in July 2006, the Ambion product line following the Ambion acquisition in March 2006, and other projects.
The tax rate for the fourth quarter of fiscal 2007, excluding the specified items described above, was approximately 30%.
The Group repurchased 3.3 million shares of Applera Corporation-Applied Biosystems group common stock during the quarter at a cost of $100.1 million.
Cash flow from operations was $135.3 million and capital expenditures were $16.5 million for the fourth quarter of fiscal 2007. Depreciation and amortization was $20.3 million. As of the end of the quarter, cash and short term investments were $494.5 million, up from $448.5 million as of March 31, 2007. This increase was largely the result of cash flow from operations partially offset by the repurchase of stock. Accounts receivable were $446.8 million, representing 58 days sales outstanding, and inventory was $132.1 million, representing 2.7 months of inventory on hand.
Recent Business Developments
In July, the Group announced the availability of a standardized TaqMan® Array for stem cell research. The Array uses gene expression markers discovered and validated by the International Stem Cell Initiative using the Group's technology.
In June, the Group opened a new Application Support Center in Foster City to drive closer relationships between the Group's scientists and its life-science customers through the creation of best-in-class applications support.
In June, the Group announced the start of its early-access program for its next-generation sequencing platform (SOLiD(TM) System), including shipment of the first systems.
Fiscal 2007 Highlights
For fiscal 2007, the Group reported net revenues of $2.09 billion, an increase of approximately 10% over fiscal 2006 revenues of $1.91 billion. Revenues for the year include the favorable impact of 2.3% from the sale of Ambion products. The net effect of foreign currency increased net revenues in fiscal 2007 by approximately 2%, as compared to the prior year.
Income from continuing operations in fiscal 2007 was $170.9 million, including $114.3 million expense for the write-off of the value of acquired in-process R&D related to the Agencourt Personal Genomics technology, as compared to $275.1 million for the prior year. EPS from continuing operations for fiscal 2007 were $0.90, a 37% decrease compared to $1.43 for the prior year. The net effect of foreign currency increased EPS by approximately $0.11 as compared to the prior year. EPS from continuing operations on a non-GAAP basis, excluding the specified items described in the reconciliation schedule below, were $1.41, a 14% increase compared to $1.24 for the prior year. Net income for fiscal 2007 included an after-tax benefit of $8.5 million for discontinued operations discussed above. Cash flow from operations for fiscal 2007 was $366.1 million compared to $375.3 million for fiscal 2006.
Revenues by source and the change relative to the prior year were: $889.3 million for Instruments, a 6% increase; $842.0 million for Consumables, a 15 % increase; and $362.2 million for Other Sources, including service and support, royalties, licenses, and consulting, a 6% increase. Fiscal year revenues include $66.6 million of Ambion-related revenues, compared to $20.4 million in fiscal 2006, which included Ambion only for the final four months of the fiscal year
During both fiscal 2007 and 2006, the Group recorded items that affected the comparability of results. For fiscal 2007, these items decreased income before taxes by $123.3 million. These items included: the previously-mentioned charge of $114.3 million to write off the value of acquired in-process research and development in connection with the Agencourt acquisition; net gains of $2.2 million related to the resolution of legal disputes; and amortization expense of $11.2 million related to acquired intangibles. Fiscal 2007 also included favorable tax adjustments of $23.8 million primarily related to foreign tax settlements and a reduction to foreign valuation allowances.
During fiscal 2006, the Group recorded pre-tax items that decreased income before taxes by approximately $19.1 million, including amortization expense of $4.8 million related to acquired intangibles. Fiscal 2006 also included net tax benefits of $50.2 million.
The following table summarizes the impact of these items on EPS calculations:
Reconciliation of GAAP amounts to Non-GAAP amounts
(Dollar amounts in millions)
Twelve months ended Twelve months ended
June 30, 2007 June 30, 2006
------------------------- ------------------------
GAAP Non-GAAP GAAP Non-GAAP
amounts Adj. amounts amounts Adj. amounts
------- -------- -------- ------- ------- --------
Operating income $ 237.1 $(123.3) $ 360.4 $ 297.0 $(19.1) $ 316.1
Income before
income taxes 259.0 (123.3) 382.3 317.2 (19.1) 336.3
Provision for
income taxes 88.1 (26.4) 114.5 42.1 (55.4) 97.5
Income from
continuing
operations 170.9 (96.9) 267.8 275.1 36.3 238.8
Earnings per share
allocations(1) 0.3 0.3 0.1 0.1
Adjusted income
from continuing
operations for
earnings per share $ 170.6 $ (97.2) $ 267.8 $ 275.2 $ 36.4 $ 238.8
Total diluted
earnings per share
from continuing
operations $ 0.90 $ (0.51) $ 1.41 $ 1.43 $ 0.19 $ 1.24
(1) Represents allocation of interperiod taxes and intercompany sales
of assets to adjust net income for purposes of calculating earnings
per share.
Applied Biosystems Outlook
The Group believes that its fiscal year 2008 outlook and financial performance could be affected by a number of factors, including: the introduction and adoption of new products; the level of commercial investments in life science R&D the level of government funding for life science research; legislation and funding for applications in the applied markets; the outcome of pending litigation matters; and competitive product introductions and pricing. Subject to the inherent uncertainty associated with these factors, Applied Biosystems has the following expectations for fiscal year 2008:
Fiscal 2008 Revenue Growth Rate: The Group expects mid to high single digit growth assuming current exchange rates. Revenues are expected to increase for both Instruments and Consumables.
Fiscal 2008 Growth by Segment: The Group anticipates revenue growth in the DNA Sequencing, Real-Time PCR/Applied Genomics, and Mass Spectrometry categories and revenue declines in the Core PCR and DNA Synthesis category and in the Other Product Lines category. Quarterly year-over-year revenue changes may be different from our annual expectations due to a variety of factors, including the timing of customer orders and disbursements of government funding.
Fiscal 2008 Margins and Expenses: The Group expects continued gross margin expansion in fiscal 2008 compared to the fiscal 2007 gross margin of 55.3%. SG&A as a percent of total revenues is expected to be slightly higher than the prior year level of 28.3%. R&D as a percentage of total revenues is expected to be approximately equal to or slightly below the prior year level of 9.7%. The Group expects an increase in operating margin in fiscal 2008 compared to the operating margin of 17.2% in the prior year, excluding special items in both fiscal years as described in the Use of Non-GAAP Financial Information section below.
Fiscal 2008 Effective Tax Rate: The Group expects the effective annual tax rate used to calculate non-GAAP financial measures to be approximately 31%, compared to approximately 30% in fiscal 2007.
Fiscal 2008 non-GAAP Earnings Per Share Growth: The Group expects non-GAAP EPS to increase faster than the annual revenue growth rate. This includes the incremental impact of stock based compensation and the effective tax rate. The total impact of these items on fiscal 2008 non-GAAP EPS is expected to be approximately $0.05.
The total pre-tax impact of FAS 123R (accounting for stock based compensation) in fiscal 2008 is expected to be approximately $22.5 million, with an EPS impact of approximately $0.08.
Fiscal 2008 Capital Spending: The Group expects capital spending to be in the range of $70-75 million.
The rate of revenue growth in the first quarter of fiscal 2008 is expected to be lower than the revenue growth rate for the full fiscal year due to particularly strong sales growth in the first quarter of fiscal 2007, which was 15% over the prior-year quarter. Non-GAAP earnings per share in the first quarter of fiscal 2008 are expected to be modestly higher than the $0.29 earned in the prior year, which included a $5 million ($0.02 per share) benefit from a previously-disclosed reversal of a litigation-related accrual. The Group also anticipates increased R&D costs in the first quarter of fiscal 2008 related to the SOLiD (TM) next-generation sequencing systems.
Other risks and uncertainties that may affect Applied Biosystems' financial performance are detailed in the "Forward-Looking Statements" section of this release.
The comments in the Outlook section of this press release reflect management's current outlook. Applera does not have any current intention to update this outlook and plans to revisit the outlook for its businesses only once each quarter when financial results are announced.
Use of Non-GAAP Financial Measures
This press release contains non-GAAP financial measures, both historical and forward-looking, and including earnings per share and operating margin adjusted to exclude some costs, expenses, gains and losses and other specified items. These measures are not in accordance with, or an alternative for, generally accepted accounting principles, or GAAP, and may be different from non-GAAP financial measures used by other companies. Among the items included in GAAP earnings but excluded for purposes of determining adjusted earnings or other non-GAAP financial measures that we present are: gains or losses from sales of operating assets and investments; restructuring charges, including severance charges; charges and recoveries relating to significant legal proceedings; asset impairment charges; amortization of acquired intangibles; and tax adjustments, including settlements and the impact of new tax legislation. In addition, for non-GAAP financial measures, we have also excluded the allocation of interperiod taxes and intercompany sales. We believe the presentation of non-GAAP financial measures provides useful information to management and investors regarding various financial and business trends relating to our financial condition and results of operations, and that when GAAP financial measures are viewed in conjunction with non-GAAP financial measures, investors are provided with a more meaningful understanding of our ongoing operating performance. In addition, these non-GAAP financial measures are among the primary indicators we use as a basis for evaluating performance, allocating resources, setting incentive compensation targets, and planning and forecasting future periods. Non-GAAP financial measures are not intended to be considered in isolation or as a substitute for GAAP financial measures. To the extent this release contains historical non-GAAP financial measures, we have also provided corresponding GAAP financial measures for comparative purposes. However, in the case of forward-looking non-GAAP financial measures, we have not provided corresponding forward-looking GAAP financial measures because these measures are not accessible to us. We cannot predict the occurrence, timing, or amount of all non-GAAP items that we exclude from our non-GAAP financial measures but which could potentially be significant to the calculation of our GAAP financial measures for future fiscal periods.
Conference Call & Webcast
A conference call with Applera Corporation executives will be held today at 11:00 a.m. (ET) to discuss these results and other matters related to the businesses. The call will be formatted to focus on each of the Applera businesses separately. The Applied Biosystems Group portion of the call will start at 11:00 a.m. (ET). The Celera Group portion of the call will start at 11:45 a.m. (ET), or immediately following the end of the Applied Biosystems portion of the call, if later.
During each segment, the management team will make prepared remarks and answer questions from securities analysts and investment professionals. Investors, securities analysts, representatives of the media and other interested parties who would like to participate should dial 617.213.4868 and enter passcode 49372399 at any time from 10:45 a.m. until the end of the call. This conference call will also be webcast. Interested parties who wish to listen to the webcast should visit the "Investors & Media" section of www.applera.com, www.celera.com, or www.appliedbiosystems.com. A digital recording will be available approximately two hours after the completion of the conference call on July 25 until August 8, 2007. Interested parties should call 617.801.6888 and enter passcode 24256462.
Applera also encourages stockholders to submit questions for management consideration by e-mail in advance of today's conference call. Such questions, which should be brief and reasonably related to the releases, may be submitted to inna.kats@applera.com. While management cannot commit to answer all such submissions, it will endeavor to do so during the available time of the conference call.
August 29, 2007 Expert Voices
Tracing the Path from DNA to Dementia
By IRENE M. WIELAWSKI
Dr. Marcelle Morrison-Bogorad is director of the neuroscience and neuropsychology of aging program at the National Institute on Aging. In collaboration with the Alzheimer’s Association, the institute runs the Alzheimer’s Disease Genetics Initiative, which is collecting and banking genetic and cellular material from families in which multiple relatives have late-onset Alzheimer’s, the most common form of the disease.
Q. How do researchers today differentiate between the brain effects of normal aging and Alzheimer’s disease?
A. This is really fundamental to one of our major problems in Alzheimer’s research.
We’re used to saying that there is memory loss and a decline in brain function as we age. Things get a bit slower. You don’t remember quite as readily. But laid on these are changes in cognition that aren’t part of normal aging and that indicate that at some point later in time, the person is going to develop Alzheimer’s disease.
As the brain ages, inflammation and oxidative damage caused by free radicals increase. Free radicals are very unstable molecules that attack different components of cells, like DNA and protein and lipids, and transform them into oxidized products that do not work as well. It is a sort of brain rusting, to give you an analogy.
There are also age-related changes in the activity of particular genes. When we are young, we have in every cell of our body a rate of production which is very specific for every single one of the 30,000 proteins that we need to keep life going. As aging progresses, the levels of certain of these proteins change, with biological consequences. Specifically, in the brain there are changes in the production of neurotransmitters, which are the molecules responsible for connecting neuronal circuits — the basis for memory. The connections between neurons, called synapses, get weaker in certain areas of the brain.
It turns out to be very tricky to differentiate between changes that are normal aging and changes that are due to Alzheimer’s disease. Normal aging makes you even more susceptible to developing the pathology of Alzheimer’s disease, such as amyloid plaques and neurofibrillary tangles, and dementia.
Q. What do we know about the genetic risk of Alzheimer’s disease?
A. We know most about early-onset Alzheimer’s disease, which you can get as early as age 30, ranging up to age 65. That’s because about 10 percent of early-onset disease is inherited in an autosomal dominant fashion, which means that if your mother or father has the disease, you have a 50 percent chance of getting it, too. The rest of early-onset disease is not inherited in such an all-or-nothing fashion, but certainly the disease tends to run in families.
As for late-onset disease, it looks like quite a large percentage runs in families as well. It therefore has a genetic component, but it is more like having a risk factor gene for heart disease. Having a family where Alzheimer’s occurs certainly increases your chances of developing it, perhaps about threefold in some estimates, although that is a very soft number. But it certainly doesn’t mean that you yourself will get it.
Q. What gene mutations are involved in Alzheimer’s disease?
A. Three autosomal dominant genes have been identified for early-onset disease. The first one is the amyloid precursor protein (APP) gene, located on chromosome 21. Mutations in the APP gene lead to an increase in the production of amyloid, which is the major component of the plaques that develop in the brains of Alzheimer’s patients. The mutation also results in a more sticky form of amyloid.
The other two genes in early-onset disease are called presenilin 1 (PS1) and presenilin 2 (PS2). Mutations in these genes cause them to act as molecular scissors that help cut the amyloid out of the precursor protein. They cut it more rapidly, and they cut it in a form which is more toxic. These mutations account for only perhaps 1 percent of the total number of Alzheimer’s cases, but they gave us very important clues as to where to focus our research.
For late-onset Alzheimer’s, the only risk factor gene known right now is ApoE-4. You can have one or two copies, or no copies, of the gene. According to how many copies you have, your risk of getting Alzheimer’s increases. If you have one copy, you usually get the disease earlier. If you have two copies, then perhaps much earlier.
Q. Is it useful for someone who, say, has older relatives with late-onset Alzheimer’s to seek genetic testing?
A. There is a great difference in the value of genetic testing between early-onset and late-onset disease. In genetic tests of people with a family history of early-onset disease, testing can determine pretty much for sure that they will or will not get it. In terms of late-onset disease, it is much trickier. All the genetic tests can tell patients is that their risk of getting Alzheimer’s is increased, nothing more.
Perhaps when we find a few more late-onset genes and you can do a mix of them, then the predictive value might be higher, but we are not at that stage yet. We don’t know for sure any other late-onset genes, although there are several promising candidates. That’s why we have the A.D. Genetics Initiative: to try to find other late-onset genes that are risk factors for Alzheimer’s.
Q. Why are biological markers, whether gene mutations or pathological brain changes, important to the development of effective treatments for Alzheimer’s disease?
A. At the moment, all that we have to measure efficacy in clinical trials are psychological tests: how well you are doing compared to how well you did a year ago. There is quite a lot of variability in Alzheimer’s patients as to how they are doing mentally. You can do a test one day and get one result, and the next day you might get a different result. In order to account for these differences, you have to do the trial for quite a long period — years. We’re hoping that one or two of the biomarkers, whether through imaging or biochemical studies, will give better, more accurate indications whether a drug has an effect or not.
Q. What do scientists hope to learn from the Alzheimer’s Disease Genetics Initiative?
A. Just as the genes for early-onset disease gave us clues about how important amyloid is, identifying those for late-onset Alzheimer’s will give us clues about other pathways which are important. That will give us new avenues for potential therapies, so that when a patient comes to a doctor’s office, the doctor can do a test and say, “Well, you’ve got the ApoE-4 gene, so you are at risk. But you don’t have any of the other risk factor genes, so you aren’t very much at risk.”
The ApoE-4 gene turns out to be a very strong risk factor for late-onset Alzheimer’s. Other risk factor genes that we don’t know about yet probably have much smaller effects, but there might be quite a few of them. In order to find genes with much smaller effects, we have to analyze many thousands of cases. Our genetics initiative in large part is a way of collecting enough DNA samples from controls and diseased individuals that geneticists can find the remaining risk factor genes.
Q. How close are scientists to finding effective treatments for Alzheimer’s disease?
A. Not as far along as we would like. Unfortunately, there are no clinical trials whose results say this drug stops the disease or this drug will prevent it. But there are drugs which are being tested in the preclinical phases, some being developed from basic science findings and some being tested in humans right now. These include drugs that attack different parts of the process of plaque formation, including blunting the scissors that cut the amyloid and preventing amyloid aggregation. Another approach currently being tested is immunotherapy, which is like giving a vaccine to protect against infection. The idea is to induce the body to develop antibodies against amyloid. Clinical trials are underway, but there are no results yet. If we’re lucky we could have something in five years. If we’re unlucky, who knows?
One of the reasons it is hard to develop drugs for Alzheimer’s is that the brain is the most complicated organ in the body by many, many fold. Because Alzheimer’s pathology begins to develop before the patient shows any signs, drugs for prevention would need to be taken for many years. So these drugs need to be targeted very specifically, and they need to be very safe with minimal side effects. It’s going to take a lot of work to get it just right.
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you want to dive even further into the sequencing of your genome, start-up 23andMe, which got a major investment from Google, might be worth a look. The company has been fairly mum about exactly what products it plans to offer, but the hints have been related to helping customers understand their genome -- sounds like personalized medicine to me.
the article doesn't mention that one of the google founder's wife founded and runs 23andme
I heard the same about Geoff Alan
couldn't happen to a nicer guy
Now--for the issue where we are slightly more in agreement. I looked back over the past six months of financings for US traded, neuro-oriented companies without any products on the market--the best comparator group, and the only one I know.
this was your mistake. A better comparator is to look at Rodman pipes. they are usually 25 percent below market. The warrants here were actually less than their usual amount. they are usually 50 to 100 percent warrant coverage. The hedgies in the deal were probably told about it in june when it was trading near 3 with volume.
That is how Rodman operates
What would be the opinion on INSM (Insmed Inc.)? Does the company have a good opportunity to bounce back from the current price decline, or could NASDAQ delisting actually happen?
I believe Iplex could be one of the biggest biotech drugs. the problem is that investors cannot make money in a company that Geoffrey Alan is ceo of. his purpose is to collect a fat salary and have other people do the work.
if they do get good data from Iplex (which they should becaus e the drug works) in other indications, tercica and Dna have the right to the drug. I would bet that insmed will be trading at under a dollar and the buyout would be at a cheap price with Geoffrey Alan the recipiant of a golden parachute. he will be well rewarded
Neuro - Start here - PPHM. Worst deal of them all and came right after the CFO said:
"I want to emphasize to everyone that we are not in any discussions to sell
a single share of stock to investors. Let me say this again. We are not in
any discussions to sell a single share of stock."
As far as I can tell this was a true statement. they didn't sell a single share of stock, they sold a hell of a lot of stock
I saw one RS at a meeting declare that Lucentis will bankrupt medicare. he forgot to give the researchers their proper due for discovering the 1st drug to ever improve sight in AMD.
you and most of the people on this board give Genentech too much credit for their technology and their ethics.
As far as lucentis they figured out a way to charge more for a drug (avastin) that is similar in efficacy. Not exactly ethical.
avastin. The drug was basically a failure but the NiH did studies that worked, after Genentech had pretty much given up on the drug after initially poor results.
Did genentech come out with idea to use avastin/lucentis for AMD or did they follow what Macugen had done, and say to themselves, wait a minute this crap on the shelf works the same way?
The typical sales and installation cycle is 12 to 18 months in duration and involves multiple steps, which may include pre-selling activity, execution of a letter of intent, or LOI, execution of contracts for the purchase or acquisition of the CyberKnife system and multiyear service plans, and installation of the CyberKnife system. Prior to installation, a purchasing institution must typically obtain a radiation device installation permit, and in some cases, a certificate of need, both of which must be granted by state and local government bodies. In addition, the purchasing institution must
and multiyear service plans
they put the above 4 words in the paragraph discussing backlog but it is the largest reason the backlog is so high. I know of no other company that takes the full value of 5 year service contracts and includes them in backlog. A perfect example of that would be software companies. They usually sell sofware with maintenance contracts but they don't include the maintenance contracts going past one year as backlog.
As far as the tattoo, can you send pictures?
FDA Approves NVS’ Reclast for Osteoporosis
[One injection works for as long as three years, but the approved dosing schedule is yearly. Reclast was previously approved for Paget’s disease. The active ingredient is the same as the one in NVS’ blockbuster, Zometa.]
these drugs have side effects. what if you experience the side effect right after the dose is given?
aray
it seems interesting. I think the backlog includes between two to three hundred million in serivicing business which takes 60 months to accrue. I don't think that should be included in backlog.
technology seems fine. they could probably make a synergistic aquisition with the cash.
That flower dripping blood is a turn off
Dor post ODAC panel vs. Dendreon post Panel
since dor panel meeting they have had positive interaction with the FDA. After Dendreon's panel meeting they had very little fda interaction.
From the Dendreon transcript the day after receiving the approvable letter.
Q U E S T I O N S A N D A N S W E RS
Operator
Thank you.(OPERATOR INSTRUCTIONS.)Charles Duncan,JMP Securities.
Charles Duncan -JMP Securities -Analyst
Hi,guys.I wanted to ask you,Mitch,about the history of collaborative discussions you've had with the FDA.How did that kind
of change in the few weeks since the panel meeting?
Mitch Gold -Dendreon Corporation -President and CEO
I think what was most surprising to us after the panel meeting --I wouldn't say that our collaborative discussions with the FDA
have changed.I think what was surprising to us after the panel meeting was the very limited amount,if any,discussions that
we had with the FDA.There was very litte interaction between the Company and the agency between the panel meeting and
when we received the complete response letter.
Charles Duncan -JMP Securities -Analyst
And then moving on to some of the efficacy data,have you had any conversations with them,or what do you anticipate having
to be able to provide them?Is it survival data,or do you think the interim would be sufficient?
Mitch Gold -Dendreon Corporation -President and CEO
We have had some preliminary discussions with the FDA since the complete response letter has come out.And let me just
reiterate that we know that the current ongoing study,the IMPACT Study,is a binding written agreement looking at survival,
and that there's an interim analysis there and a final analysis,as I've said.
Is Sentinel Management Group invested in bio tech?
Sentinel Freezes $1.5 Billion Fund
Tuesday August 14, 2:01 pm ET
By Dan Seymour, AP Business Writer
Sentinel Management Group Freezes $1.5 Billion Fund Amid Global Liquidity Crisis
NEW YORK (AP) -- Sentinel Management Group Inc. on Tuesday froze a $1.5 billion fund, saying too many investors are trying to withdraw their money.
Sentinel Management Group is just the latest investment manager engulfed in a worldwide exodus from risk. Stung by decaying credit quality, the world's investors last month began to pull back from all kinds of risky instruments, from corporate bonds to credit-default swaps.
The Northbrook, Ill.-based fund told clients in a letter obtained by The Associated Press that "fear has overtaken reason" and most investments have no buyers. Sentinel Management Group said its own clients have joined in the panic by trying to bail out of the fund.
The fund cannot meet investors' requests to withdraw their money without selling investments at a steep discount, the fund said. Sentinel did not respond to calls for comment.
The firm sent a request to the Commodity Futures Trading Commission for permission to stop investors from cashing out. The CFTC said it plays no role in approving suspension of redemptions.
Sentinel Management, which boasts on its Web site that no client has ever lost money in its fund, makes money mainly by betting on overnight interest rates outpacing yields on short-term Treasury bonds.
According to CME Group, the fund manages $1.5 billion in assets. The company's investments include short-term debt and high-quality bonds.
The fund is available to corporations, institutional investors and wealthy people. The company claims that since its founding in 1979, the fund has never so much as been late redeeming an investor, even during the stock market crash of 1987.
"We have never experienced a situation quite like this one," Sentinel Management said. "Liquidity has dried up all over the Street."
While liquidity has for months been draining from riskier investments like mortgages backed by bad credit, Sentinel Management's investments appear to be much higher quality. The fund said that because of the dearth of buyers "investors dump names like General Electric at Tyco-like prices."
CME Group, which operates the Chicago Mercantile Exchange and Chicago Board of Trade, said in a statement none of Sentinel's $1.5 billion is on deposit with the exchanges' clearing agent. A "limited" number of the exchanges' members are clients of the fund, CME Group said.
MF Global Ltd., a brokerage, said in a statement it has no exposure to Sentinel Management.
Sentinel's letter was one of the factors dragging the stock market Tuesday, with the Standard & Poor's 500 Index and Dow Jones industrials both down more than 1 percent in afternoon trading.
AP Business Writer Alan Zibel in Washington contributed to this report.
Pipex Pharmaceuticals (PP) sounds like a scam outfit
The ceo made a comment about amd that he shouldn't have made, that doesn't make the company a scam
insider buys are a lot more than cox's
INSIDER TRANSACTIONS REPORTED - LAST TWO YEARS
Date Insider Shares Type Transaction Value*
25-Jul-07 KANZER STEVE H
Officer 14,300 Direct Purchase at $7.09 - $7.42 per share. $104,0002
24-Jul-07 KANZER STEVE H
Officer 8,000 Direct Purchase at $7 - $7.24 per share. $57,0002
23-Jul-07 KANZER STEVE H
Officer 29,700 Direct Purchase at $6.58 - $6.9 per share. $200,0002
17-Jul-07 KANZER STEVE H
Officer 25,000 Direct Purchase at $5.10 - $5.47 per share. $132,0002
16-Jul-07 BISGAIER CHARLES PHD
Officer 10,000 Direct Purchase at $4.64 per share. $46,400
16-Jul-07 KANZER STEVE H
Officer 81,000 Direct Purchase at $4.49 - $5.24 per share. $394,0002
11-May-07 KANZER STEVE H
Officer 26,500 Direct Purchase at $5 - $5.4 per share. $138,0002
2-Nov-06 KANZER STEVE H
Officer 7,086,379 Indirect Purchase at $0.67 per share. $4,747,873
2
this drug is not a scam
television snip
http://abclocal.go.com/kabc/story?section=health&id=5227091
data
ESTRIOL MAY EASE RELAPSING-REMITTING MS IN WOMEN
BALTIMORE—Administration of oral estriol, the predominant estrogen of pregnancy, to nonpregnant women with relapsing-remitting multiple sclerosis increased protective immune responses and decreased the number and volume of gadolinium-enhancing lesions on monthly cerebral magnetic resonance imagings (MRIs), a phase I study has shown.
“To our knowledge, this is the first time a pregnancy hormone has been given at a pregnancy dose to [patients] in an attempt to ameliorate a putative Th1-mediated autoimmune disease by using highly sensitive subclinical markers of disease activity as an indicator,” reported Rhonda Voskuhl, MD, and colleagues in the October Annals of Neurology. Dr. Voskuhl is Associate Professor of Neurology at the David Geffen School of Medicine at the University of California, Los Angeles (UCLA).
“This novel treatment strategy of using pregnancy doses of estriol in multiple sclerosis has relevance to other autoimmune diseases that also improve during pregnancy,” the authors noted, including rheumatoid arthritis, uveitis, thyroiditis, and a number of other arthritides.
HORMONES AND MS
Two widely accepted clinical observations implicate hormones in multiple sclerosis. First, “women are clearly more susceptible than men to multiple sclerosis; second, women with multiple sclerosis improve transiently during pregnancy,” particularly during the last trimester, said Dr. Voskuhl at the 7th Annual Meeting of the Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) and the 18th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS).
Estriol, produced by the fetal placental unit, is not detectable at appreciable amounts until pregnancy, when it progressively increases. Previous studies have found that estriol administered to mice with experimental autoimmune encephalomyelitis resulted in amelioration of multiple sclerosis, with a favorable shift in immune response similar to that seen during pregnancy.
Dr. Voskuhl, who is also a research scientist at UCLA’s Brain Research and Neuropsychiatric Institutes, explained that estriol at pregnancy doses has been given in Europe and Asia in the form of hormone replacement therapy for symptoms of menopause, “which means we didn’t have to reinvent the wheel on assessing toxicity. It was very well known how this estriol preparation would be tolerated. Therefore, we could go straight to a phase I clinical trial focusing on multiple sclerosis–related disease measures.”
Noting that all currently available anti-inflammatory therapies for multiple sclerosis are injections, Dr. Voskuhl reported that the purpose of the trial “was to test a noninjectable, oral anti-inflammatory hormonal treatment” in an attempt “to recapitulate the beneficial effect of pregnancy.” The study included a six-month baseline pretreatment period, six months of treatment with oral estriol 8 mg/d, and a six-month posttreatment evaluation period. Patients with relapsing-remitting multiple sclerosis received an additional four months’ retreatment with oral estriol 8 mg/d with progesterone, she added.
Twelve women with clinically definite multiple sclerosis were included in the study; six with relapsing-remitting and six with secondary progressive disease. Patients were excluded if they had been taking interferon-beta or glatiramer acetate for six months or steroid therapy for three months prior to enrollment.
“We did an MRI every month throughout—we followed them in the clinic every three months,” Dr. Voskuhl said. A delayed-type hypersensitivity (DTH) response to tetanus and candida was performed, once in the pretreatment period and once in the treatment period, as was Paced Auditory Serial Additional Task (PASAT) cognitive testing.
All six patients with relapsing-remitting multiple sclerosis and four with secondary progressive multiple sclerosis completed the study. One patient was disqualified because of concurrent steroid treatment “and the other did not wish to go untreated in the posttreatment period,” the investigators noted. Mean age of those who completed the study was 44 years (range, 28 to 50). For all patients, mean Expanded Disability Status Scale score was 3.3 (range, 1.0 to 6.5); for those with relapsing-remitting disease it was 2.2 and for secondary progressive, 5.0.
ENHANCING RESULTS
Compared with the six-month pretreatment baseline period, the total volume and number of enhancing lesions for all 10 patients decreased during the treatment period, the investigators found, with cognitive improvement seen entirely in the relapsing-remitting group. In this group, median total enhancing lesion volumes decreased by 79% and number of lesions by 82% within the first three months. Over the next three months, lesion volumes decreased by 82% and numbers by 82%.
During the six-month posttreatment period, “median total enhancing lesion volumes and numbers became variable in the first three months off treatment, before returning to near-baseline levels in the last three months,” the researchers remarked.
Patients retreated over four months had a decrease in enhancing lesion volumes of 88% and in numbers of 48% compared with original baseline scores. “Changes in median new enhancing lesion volumes and numbers followed similar patterns as median total lesion numbers and volumes,” they observed.
For the entire group, median T2 lesions were 15.3 cm3 (range, 6.1 to 33.8); no significant differences were seen between the two subgroups. In the relapsing-remitting group, median T2 lesion volumes showed no change during the six-month treatment period, a 7.4% increase during the six-month posttreatment period, and a 2.0% decrease in the four-month retreatment period.
After six months of estriol treatment, patients’ DTH responses to tetanus decreased significantly compared with month 3 (pretreatment baseline), whereas responses to candida “were decreased less drastically and more variably,” the researchers found. In relapsing-remitting patients, interferon-gamma levels were significantly decreased after six months of treatment compared with baseline. No decrease was seen in the patients with secondary progressive disease. Similarly, in the relapsing-remitting group, PASAT cognitive testing scores improved significantly and no change was seen in the secondary progressive group.
During treatment and retreatment, serum estriol levels approximated “those observed in women who were six months pregnant but were lower than those who were 8.5 months pregnant,” the authors wrote. Few relapses were observed during the study, and the estriol was found to be well tolerated.
Since the study was submitted for publication, Dr. Voskuhl reported that additional immunologic studies performed on the six patients with relapsing-remitting disease showed an increase in IL-5 and a decrease in TNF with estriol treatment. A double-blind placebo-controlled study is now being planned in patients with relapsing remitting multiple sclerosis only, with the goal “to reproduce what we did in the pilot—that is, to get a decrease in gadolinium-enhancing lesions, a favorable shift toward cytokine production, and an improvement in PASAT,” Dr. Voskuhl concluded.
NR
—Debra Hughes
Return to table of contents
o/tAccredited Lenders, Lone Star Deal Off
it didn't take long for the other shoe to drop. I bet Cramer was able to get a lot of his buddies out in the 7 to 8 dollar range pumping this deal
POSTED: Friday, August 10, 2007
FROM BLOG: Word on the Street - Word on the Street; Financial feed with daily updated stock blog alerts.
The following blog post is from an independent writer and is not connected with Reuters News. The opinions and views expressed herein are those of the author and are not endorsed by Reuters.com.
So much for speculation. Out tonight is a filing from Lone Star informing Accredited Home Lenders (NASDAQ: LEND) that in light of the drastic deterioration in the financial and operational condition of the company, among other things, as of today, the company would fail to satisfy the conditions to the closing of the tender offer. Accordingly, Lone Star does not expect to be accepting Accredited shares tendered at of the end of the current offer period ending on August 14. As of 6:00 PM EST, the stock is surprisingly only lower by $.15, or 1.74% to $8.75, but that is sure to change quickly as the news gets around. With the deal dead, the "balls of steel" trade is officially dead as well.
o/t Accredited Buyout Gets Regulatory Approval
Andrew Farrell, 08.10.07, 5:00 PM ET
aside from the fact that cramer and his cronies say the deal is going to go through, I cannot fathom why it will. Can someone enlighten me
Investors have fretted the private equity buyout of Accredited Home Lenders could collapse because of mounting mortgage problems. They were calmed Friday by a key regulatory approval that moves the deal closer to completion.
Accredited Home Lenders said Friday that states where it makes nearly all of its loans have approved its acquisition by the private equity fund Lone Star. The approvals satisfy one of the primary conditions required to close a tender offer by Lone Star for all of Accredited's 25.3 million outstanding shares. Accredited shares jumped 45.3%, or $2.77, to $8.90 on the news.
Accredited shares, however, still traded well below Lone Star's offer of $15.10 per share. Concerns that Lone Star could back out of the deal have kept Accredited stock down. The lender is struggling with rising losses because of growing mortgage defaults that forced it to delay filings its annual report. (See: "Does Lone Star Still Want Crumbling Accredited?")
The company has even warned it could head to bankruptcy. Accredited said in SEC filings it could go bankrupt if the value of its mortgage products does not stop declining and it cannot find fresh liquidity. Rising defaults have forced over 50 lenders into bankruptcy this year.
Morningstar analyst Erin Swanson says the problems could tank the deal. "If Accredited is unable to renegotiate the terms of its loans, its ability to operate as a going concern will be extremely questionable," says Swanson. "As a result, the recently extended tender offer from Lone Star would be terminated."
Not faring as well as Accredited Friday was Countrywide Financial. The largest mortgage lender in the U.S. warned that disruptions in the credit and secondary mortgage markets would hurt its short-term financial pictures. Countrywide shares fell 2.7%, or 80 cents, to $27.86. (See: "U.S. Stocks Slide Despite Fed Aid")
VPHM/ Another HCV blowup...Potential Safety Issue Identified in Ongoing Phase 2 Clinical Study of HCV-796
In most cases I don't there are many news leaks. I have to say in this case there had to be a lot
-Aug-07 11.55 11.62 10.15 10.22 6,238,600 10.22
8-Aug-07 12.85 12.93 11.49 11.55 4,759,200 11.55
7-Aug-07 13.58 13.79 12.85 13.00 2,248,900 13.00
6-Aug-07 13.69 13.84 13.44 13.64 1,739,300 13.64
3-Aug-07 13.92 14.20 13.51 13.55 2,038,800 13.55
2-Aug-07 14.50 14.85 13.75 13.90 2,205,300 13.90
1-Aug-07 13.33 14.38 13.30 14.38 3,234,100 14.38
31
India's generic companies doing their bid to reduce the Indian population explosion.
Sanofi Drug Hits New Hurdle With Indian Knockoffs (Update1)
By Jason Gale and Angela Cullen
Slimona brand Rimonabant diet pills Aug. 9 (Bloomberg) -- Sanofi-Aventis SA's Acomplia weight- loss pill, linked to suicide, is becoming popular in generic form in India. That may end the product's chances of ever reaching the U.S., where it's been delayed by regulators.
Cipla Ltd. and Ranbaxy Laboratories Ltd. are among six drugmakers exploiting a loophole in India patent laws, selling copies of the medicine under names like Slimona and Defat. The pills are sold without prescription for as little as 12 cents.
Should the knockoffs, used without supervision, lead to an increase in suicides, the U.S. Food and Drug Administration's opposition may stiffen. The drugmaker's shares, near the lowest in two years, won't rise anytime soon because the company has few medicines to replace Acomplia's lost sales, analysts said. Sanofi had predicted Acomplia would generate $3 billion a year.
``This is going to be potentially disastrous,'' said Jeffrey Mechanik, an endocrinologist at Mt. Sinai Hospital in New York. ``People are going to be over-dosing'' if generics flood the market and people take them inappropriately, he said.
Sanofi's earnings have dropped for four straight quarters. The drugmaker is losing patent protection on older medicines like the sleep-pill Ambien.
The Paris-based company's shares fell 76 cents, or 1.2 percent, to 60.59 euros at 10:39 a.m. in Paris, and have dropped 14 percent since reaching this year's high of 71.66 euros May 31. Sanofi withdrew its U.S. marketing application for Acomplia on June 29 after the FDA raised safety concerns.
Slower Growth
Growth at the maker of the Plavix blood thinner probably will remain slower than that of other European drugmakers, according to Alexandra Hauber, a Bear Stearns & Co. analyst in London.
Under Indian intellectual property law, pharmaceutical companies can use a process called reverse engineering to manufacture drugs patented before 1995. The patent on Acomplia, which regulates hunger impulses, dates to 1994.
Sanofi hasn't heard from Indian authorities on some of the company's patent applications filed after 2000, spokesman Jean- Marc Podvin said.
Sanofi received approval to sell Acomplia in India in May, the same month as the generic-drug makers. The company hasn't decided whether to sell its branded version there. ``We're evaluating our options,'' Podvin said. ``Of course, it's a concern.''
A pharmacy in New Delhi's commercial center sells the pills for the equivalent of 22 cents apiece. At another in Mumbai, the tablet can be bought for 5 rupees (12 cents).
No Warning
The Indian regulator approved rimonabant, or generic Acomplia, requiring patients get a prescription and medical advice on its risks. Those include depression and anxiety --side effects that were serious enough to prompt an FDA panel of advisors to reject the pill. Both stores sold the medicine without an explanatory leaflet or a doctor's note.
European regulators, who approved the medicine last year, tightened prescribing rules last month to say Acomplia shouldn't be used by those taking antidepressants or who have depression.
``There is a risk that if you can just buy it over the counter and really want to lose weight for that wedding, you may end up committing suicide before you get married,'' said Stephen Bloom, a professor of metabolic medicine at London's Imperial College. ``The rest of the world will watch. It's very kind of the Indian nation to be testing drugs for us like this.''
India is growing obese. Almost a third of women and more than a fifth of men living in urban areas are considered overweight, according to a government survey last year.
Obesity and Diabetes
Obesity can lead to high blood pressure and diabetes, to which South Asians have a genetic predisposition. Indian men are three to four times more likely than East Asian, African American, Hispanic or Caucasian men to develop insulin resistance that leads to diabetes, according to a study last year in the Proceedings of the National Academy of Sciences.
Unregulated use of the medicine that leads to reports of serious side effects could spell trouble for Sanofi, researchers say.
``Doctors should be very careful because in all the clinical trials, there was a slight increase in anxiety and depression,'' said Andre Scheen, head of diabetes, nutrition and metabolic disorders at the University of Liege, Belgium, who led a clinical trial on Acomplia.
Sanofi says it still plans to make the drug available in the U.S. In a statement Aug 1, the drugmaker said the drug has a ``positive risk benefit ratio'' when used in the ``appropriate population.''
Selected Patients
Torrent Pharmaceuticals Ltd. started selling its version, Rimoslim, two months ago and aims to sell 100 million rupees worth within 12 months. Rimoslim is ``an extremely affordable therapy for the masses,'' the Ahmedabad-based company said in a statement on its Web site in May. Asked via e-mail whether there were concerns about patient safety, Ruchir Modi, Torrent's vice president of marketing, said ``we can only wait and see how this unfolds'' with the FDA.
S.K. Wangnoo, a senior consultant endocrinologist at Indraprastha Apollo Hospital in New Delhi, says the medicine should only be used by ``a selected group of patients who are not able to control their appetite despite all efforts by diet counselors, behavior therapy or psychotherapy.''
Those patients ``must be told about the side effects and be checked by a doctor every month,'' according Wangnoo, who says he's cautious about prescribing the medicine, especially after it wasn't approved by the FDA.
G.R. Sumathi, who runs Cash Pharmacy in downtown Bangalore, says he's selling at least one box of Defat every day.
To contact the reporters on this story: Jason Gale in Singapore at j.gale@bloomberg.net ; Angela Cullen in Frankfurt at acullen8@bloomberg.net
Last Updated: August 9, 2007 04:43 EDT
Sure feels tempting to put a few bob on that horse
I have more than a few bob on the horse. There is absolutely no approval premium priced into the stock. As you saw Pazdur started out saying that he would entertain a treatment Ind. doesn't the stock go up if they just get that?
I think they get some kind of approval
CV Therapeutics (CVTX 1-Overweight, Sector 2-Neutral)
RANEXA prescriptions were reported by IMS Health today for the week ended July 27 (72nd week). Of note, the RANEXA launch was announced on March 24, 2006. Total prescriptions (TRx) of 5,881 for the week were down 0.3% from 5,901 in the prior week. New prescriptions (NRx) of 2,355 were up 2.3% from 2,283 in the prior week. Based on current prescription trends, we are estimating 3Q07 sales of $16.0M.
right now the sales are a big disappointment if you own the stock. I don't think docs think the drug is worth much
Nastech Pharmaceutical Company to Host Webinar on its Strategy for Development of RNAi Therapeutics for Treatment of Influenza
another way of Steven Quay to say, I need to put out a press release where I can get RNAI into the headline
· IOMI: Better-than-Expected Results from Traveler’s Diarrhea Vaccine Phase II Clinical Trial; Reiterate Buy
Iomai Corp. (b)
(IOMI)
Better-than-Expected Results from Traveler’s Diarrhea Vaccine Phase II Clinical Trial
Reiterate Buy
Price: $ 1.72
Market Cap (M): $44
FY07 Revenue Estimate (M): Previous: No Change
Current: $14,000
FY07 EPS Estimate: Previous: No Change
Current: ($1.57)
FY08 Revenue Estimate (M): Previous: No Change
Current: $26,600
FY08 EPS Estimate: Previous: No Change
Current: ($0.93)
· Investment thesis. The recent announcement of positive Phase II travelers’ diarrhea vaccine efficacy data, in our view, provides a strong catalyst for increased IOMI share value by: 1) increasing the probability of success for the travelers’ diarrhea vaccine program, 2) enhancing Iomai’s ability to negotiate partnering agreements, 3) providing scientific support for other clinical programs at Iomai, 4) defining a timeline for late-stage clinical development of the travelers’ diarrhea vaccine, and 5) greatly increasing the potential for revenue generation. Since current IOMI share value does not adequately account for the recent Phase II success, potential partnering agreements, or ultimate clinical program successes, we recommend that investors aggressively accumulate IOMI shares at these levels.
· Travelers’ diarrhea vaccine provides statistically significant efficacy in Phase II field study. Iomai’s needle-free, patch-based vaccine for enterotoxigenic E. coli (ETEC) bacteria reduced the incidence, frequency, and duration of diarrhea. Travelers vaccinated with two doses prior to traveling to Mexico or Guatemala showed a 75% reduced incidence of moderate or severe diarrhea from any cause (p<0.01), and an 84% reduced incidence of severe diarrhea (p<0.05) as compared to placebo controls, without any vaccine-related serious adverse events. Conducted in collaboration with the Johns Hopkins Bloomberg School of Public Health and the University of Texas School of Public Health, the randomized (111 placebo-treated vs. 59 vaccinated travelers) double-blind field study evaluated vaccine safety, ETEC incidence, vaccine-preventable outcomes, immunogenicity, and stool frequency. Of particular note were findings that the vaccine provided a major reduction in the duration of disease, and that vaccine efficacy was greater than expected given that the trial was designed in part to gain data for subsequent ETEC field studies. Iomai plans to submit detailed trial data for publication in a peer-reviewed manuscript and will consider presenting the data at a leading scientific conference.
· Market potential for a travelers’ diarrhea vaccine. According to the CDC, travelers’ diarrhea affects up to 20-50% of international travelers, with between 10-20M yearly cases of travelers’ diarrhea. The worldwide market for a travelers’ diarrhea vaccine is estimated to exceed $400M annually, without taking into account potential military applications and utilization within developing countries. Currently, there is no approved vaccine for travelers’ diarrhea in the United States.
· Milestones for travelers’ diarrhea vaccine program. With respect to the travelers’ diarrhea program, we expect Iomai at this time to enter into ETEC program partnering discussions, conduct a small required final-formulation study (expected completion 1Q08), and hold discussions with the FDA to establish a Phase III trial design (mid-2008). We expect Iomai to be ready for a pivotal Phase III field study during the 2009 travel season.
· Additional pipeline activity in the 12-18 months include data from a Phase I elderly influenza IS vaccine booster patch study (3Q07), IND filing for a dose-sparing IS pandemic influenza patch (2H07), and initiation of a Phase I/II safety and immunogenic study for dose-sparing IS pandemic influenza patch (2H07).
· We reiterate our Buy rating and recommend the aggressive purchase of IOMI shares, given that IOMI’s current share price does not adequately account for the Phase II success, potential partnerships, potential success of the company’s needle-free travelers’ diarrhea vaccine patch program, a potential governmental pandemic influenza stockpiling contract for the company’s dose-sparing patch, or potential development of the company’s immunostimulant elderly influenza patch program.
-- Brian McCarthy, PhD (646) 292-1457 bmccarthy@mcfco.com
Dorb excerpts of ODAC transcript
Dr. Pazdur asks the panel to discuss whether another trial is feasible to run
1 large treatment, single-arm trial.
2 Here again, one of the questions that I have,
3 if that would be ongoing, would people even consider
4 doing another trial? Would another trial be feasible to
5 do? That’s another issue.
6 CHAIRPERSON HUSSAIN: Is this an issue you
7 want us to discuss now, or this is an issue that we want
8 to discuss after the vote?
9 DR. PAZDUR: Probably after the vote or it can
10 be discussed during the vote.
11 (General laughter.)
12 DR. PAZDUR: It’s really going to be part and
13 parcel of a decision here.
14 CHAIRPERSON HUSSAIN: Yes. Just a point of
15 clarification. If someone gets an IND for a specific
16 treatment, who pays for the cost of the drug?
17 DR. PAZDUR: Usually, the Sponsor would assume
18 the cost of that. Under the treatment IND mechanism,
19 however, which is a special type of expanded access
20 program, there can be some cost recovery on the part of
21 the Sponsor. We would entertain in a situation such as
22 this that type of program.
Mcdonal explains why trials using the drug for acute gvhd would be hard to run, because the doctors are formulating a drug themselves that isn't as good as Orbec but is better than placebo
1 in this group of patients for ethical and practical
2 reasons.
3 CHAIRPERSON HUSSAIN: Could you not consider
4 doing a trial, not placebo-controlled but with
5 equivalent doses of steroids to make the point that your
6 agent has a superior profile with regard to infections,
7 complications of steroids, and things of that sort? It
8 doesn’t have to be necessarily a survival-driven trial
9 but an efficacy trial?
10 DR. McDONALD: I think that is a
11 consideration, but actually there are some data that
12 bears on the question. If you look at the placebo arm
13 of 875, you will discover that 41 percent of people
14 required only 10 days of prednisone. If you look at the
15 placebo arm of the pivotal trial, 55 percent required
16 only 10 days of prednisone.
17 I think it is very difficult to argue that a
18 high-dose prednisone regiment would benefit any of those
19 patients who required only 10 days of prednisone. I
20 think the same ethical issues exist in doing a very
21 high-dose prednisone versus this approach.
22 We have already proved, I think, that the
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1 minimum prednisone approach protected by BDP has far
2 superior results to our 25-year standard of care. Again,
3 it is not the GVH that is killing people; it is the
4 treatment for GVH that’s doing the job.
5 I think we have adequately proved that BDP has
6 a steroid-sparing effect. I’m not sure I can go back 25
7 years in time and grab our old regimens. I am quite
8 convinced. I think it might well be unethical to do
9 that kind of a comparison.
10 DR. RODELL: Excuse me. Dr. Sullivan from
11 Duke was actually not involved in the trial.
12 CHAIRPERSON HUSSAIN: I just have another
13 question, though, please. If you say you don’t want to
14 go back to the past and grab old regimens, and if your
15 drug is currently not available on the market, and we
16 have heard that people use the oil-mixed product with
17 something, is that what people are currently using? Is
18 that what the alternative out there is?
19 DR. McDONALD: The alternative is what I call
20 the ad-hoc way of formulating something that is similar
21 to what we are using in these trials, that is, to use
22 the corn oil combined with budesonide.
the panel votes no
1 overwhelmingly we were against something and the FDA
2 approved it anyway.
3 (General laughter.)
4 CHAIRPERSON HUSSAIN: They don’t necessarily
5 always listen to us. Thank you.
6 If there are no other burning questions or
7 comments, I’m going to try to ask that we go ahead to
8 the vote.
9 Put the question up again, please.
10 (Staff complies.)
11 CHAIRPERSON HUSSAIN: We will begin around the
12 table. No description of why one is voting yes or no,
13 just the vote yes or no. Identify please yourself and
14 speak clearly into the microphone.
15 Dr. Link, can we begin with you, please?
16 DR. LINK: Michael Link. I vote no that it
17 has not shown substantial evidence.
18 MS. HAYLOCK: Haylock, no.
19 DR. HARRINGTON: No.
20 DR. MORTIMER: Mortimer, no.
21 CHAIRPERSON HUSSAIN: Hussain, no.
22 DR. RICHARDSON: Richardson, no.
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1 DR. PERRY: Perry, no.
2 DR. SPORTES: Sportes, yes.
3 DR. FLATAU: Flatau, yes.
4 CHAIRPERSON HUSSAIN: Two yes, seven no.
5 Dr. Pazdur, do you want us to go into the
6 second issue of design, of study design?
7 DR. PAZDUR: We’ve already started the
8 discussion.
9 CHAIRPERSON HUSSAIN: Yes, but I thought it
10 was for discussion, not for a vote.
11 DR. PAZDUR: We’ve already started the
12 discussion.
13 CHAIRPERSON HUSSAIN: In terms of what ideal
14 study design is?
15 DR. PAZDUR: Yes.
16 CHAIRPERSON HUSSAIN: Maybe I can begin and
17 ask the sponsor first, when you look at your data and
18 you look at what you started with and then what you got,
19 what do you think, like in your gut with yourself
20 looking at this, where do you think your strongest point
21 was? Because that seems to me where you should then go
22 back to look at that area because that may be where
Hussein explains that you couldn't run a trial in this population that could be stratified equally.
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1 between treatment arms, it would require, roughly, 160
2 deaths and a true hazard ratio of 0.55 would require,
3 roughly, 120 deaths. The accrual would be quite
4 substantial.
5 CHAIRPERSON HUSSAIN: Any words of wisdom from
6 committee members as far as study designs or suggestions
7 to the Sponsor or to the FDA with regard to what they
8 should advise them to do?
9 Sir?
10 DR. FLATAU: Yes. I would like to think at
11 least a secondary endpoint of overall survival at least
12 at one year, if not longer, would be interesting.
13 CHAIRPERSON HUSSAIN: I guess the only concern
14 I have about survival is unless you make the population
15 entering very uniform with the same diseases, the same
16 regimen, the same everything, you are never going to be
17 able to interpret the results, it would seem to me,
18 unless you stratify very clear where you include several
19 groups of people clearly stratified equal in each arm.
20 I can’t see how you would do it if you argue that we
21 don’t have a huge population.
Dr. Sportes the only transplant doctor on the panel and one of the positive votes explained that the 80 day endpoint which was met clinically means more than the 50 day endpoint
22 DR. SPORTES: Actually, I was wondering and I
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1 should have asked earlier why the secondary endpoint was
2 not the primary endpoint.
3 It seems to me from a transplant perspective
4 that a followup at 80 days is probably the strongest
5 endpoint, which is why I voted yes is because I think to
6 me conceptually this is much more a reflection of the
7 entire intervention. Why was that not chosen? I just
8 seek some explanation.
9 DR. RODELL: May I respond to that?
10 CHAIRPERSON HUSSAIN: Yes.
11 DR. RODELL: I think it’s important to
12 recognize the history of how this drug was developed.
13 The initial study, the initial efficacy study was Study
14 875 that was done essentially on a completely open
15 field, that is, this type of study had never been done
16 before and so the 30-day endpoint was selected in that
17 study, essentially, arbitrarily.
18 Once the results of that study were know, Dr.
19 McDonald spoke with and talked to the FDA, shared with
20 them the results of that study and began to design the
21 02 Study.
22 The two components with respect to efficacy
Dr. Link explains that the first 10 days on Orbec should be a guarantee period and is surprised that they didn't show this slide before the vote.
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1 The practicality, even without getting into
2 the economics of doing another study, is going to be a
3 significant issue in terms of the future development of
4 the drug.
5 CHAIRPERSON HUSSAIN: Thank you.
6 Any other comments?
7 (No verbal response.)
8 CHAIRPERSON HUSSAIN: Well, we will begin with
9 Dr. Link, then, and we will go around the table.
10 DR. LINK: I have two. The first is a
11 question of what if you actually -- the mistake maybe
12 was that you started the clock too soon in terms of you
13 started on the day of randomization. Either if you if
14 you had randomized after the 10 days and only randomized
15 responders -- Dave, you’re not going to shoot me here,
16 are you?
17 The second things is to actually do an
18 analysis, start the clock when they achieve remission
19 and throw out people who didn’t achieve remission, which
20 is often done in transplant studies. That would be one
21 suggestion.
22 What does it look like? Does it look better?
1 Does it look like you had achieved your goal, just out
2 of curiosity?
3 DR. RODELL: Yes, let me ask Mr. Cruickshank
4 to address that.
5 (PowerPoint presentation is in progress.)
6 MR. CRUICKSHANK: Going back to that question
7 about, what is the effect of those early failures, we
8 did do an analysis to look at the effect whereby
9 patients who failed during the 10-day high-dose
10 induction period of prednisone.
11 If you sensor the patients at the time of
12 treatment failure, you can see here that we lose the
13 effect of the crossing Kaplan-Meier curves. We have a
14 fairly clear, sustained difference between the arms at
15 day 50 as well as day 80.
16 DR. LINK: You should have presented that.
17 Second of all, I was impressed with the ability of the
18 bone marrow transplant community to do study with soft-19
tissue endpoints, a lot of patients, a “New England
20 Journal of Medicine” article, very prompt approval.
21 I’m just wondering why you’re so discouraged
22 about doing a trial like this? Admittedly, not
Page 148 Dr. Mcdonald from the Fred Hutchinson Center in Seattle again explains why it would be difficult to run the trials in acute GVHD, and explains that a prophylaxis trial would be the proper trial to run, but the FDA doesn't like mixing endpoints. Dr Pazdur chimes in, that he is open to negotiation
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1 everybody gets bowel GVH. But if you’re transplanting
2 60-year-olds, you probably will get a lot of it. I’m
3 just wondering why you’re so pessimistic about getting
4 it done? We’re going to do it in pediatrics, and we
5 don’t have that many patients.
6 DR. McDONALD: I’m pessimistic about doing a
7 placebo-controlled trial because I don’t think we can
8 get our IRB to approve one based on these data. I think
9 there are other options.
10 I mean, we have thought about a prophylaxis
11 trial, for example, but that’s a different indication,
12 though. This indication is for treatment of acute
13 graft-versus-host disease. A prophylaxis trial is for
14 prophylaxis of acute graft-versus-host disease.
15 From the FDA’s comments, you don’t like
16 merging of different kinds of trials to come to a
17 conclusion. That would take, what, two placebo-18
controlled prophylaxis trials for that --
19 DR. PAZDUR: We are always open for
20 negotiations.
21 (General laughter.)
22 DR. McDONALD: All right. That’s one thought,
Page 149 to 152 is basically the panel members all admitting that a prophlyaxis trial would be the way to go. Of course how does the drug get approved in acute GVHD if you run a prophylaxis trial.
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1 everybody gets bowel GVH. But if you’re transplanting
2 60-year-olds, you probably will get a lot of it. I’m
3 just wondering why you’re so pessimistic about getting
4 it done? We’re going to do it in pediatrics, and we
5 don’t have that many patients.
6 DR. McDONALD: I’m pessimistic about doing a
7 placebo-controlled trial because I don’t think we can
8 get our IRB to approve one based on these data. I think
9 there are other options.
10 I mean, we have thought about a prophylaxis
11 trial, for example, but that’s a different indication,
12 though. This indication is for treatment of acute
13 graft-versus-host disease. A prophylaxis trial is for
14 prophylaxis of acute graft-versus-host disease.
15 From the FDA’s comments, you don’t like
16 merging of different kinds of trials to come to a
17 conclusion. That would take, what, two placebo-18
controlled prophylaxis trials for that --
19 DR. PAZDUR: We are always open for
20 negotiations.
21 (General laughter.)
22 DR. McDONALD: All right. That’s one thought,
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1 that is, if this drug is as highly effective as a
2 topical therapy for treatment, it should be equally
3 effective and relatively nontoxic as prophylaxis. I
4 think that is one potential option.
5 CHAIRPERSON HUSSAIN: Gee, prophylaxis sounds
6 like very attractive. Why wait until an event happens?
7 I mean, that would be a clinically meaningful endpoint.
8 DR. McDONALD: I agree. Plus, it’s easier to
9 enroll patients who aren’t sick.
10 CHAIRPERSON HUSSAIN: Correct, correct.
11 DR. McDONALD: You can get a larger accrual.
12 Seven thousand patients should provide enough to enroll.
13 The hope there is that one would with effective
14 prophylaxis take what would ultimately be Stage IV and
15 turn them into Stage III, take Stage III and turn them
16 into Stage II, take Stage II and make them end up being
17 Stage I. I think that is a feasible thing. I can’t
18 speak for the company.
19 I think there are some financial issues. This
20 is a relatively small company, and I think that is a
21 consideration. In addition to the ethical and practical
22 issues, I think there are financial issues.
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1 DR. SCHABER: Dr. McDonald -- Chris Schaber,
2 president of Dor Bio Pharma -- that is in fact the
3 issue. For a company our size, we’ve put a lot of time,
4 effort, money, and resource into this study to support
5 Dr. McDonald and the rest of the investigators to go
6 after an indication that, quite honestly, big pharma
7 didn’t want to touch because it is a very small patient
8 population.
9 Although, as was outlined here and the experts
10 have spoken, we did not achieve the primary endpoint
11 without maybe the right sensoring or guarantee period.
12 With regard to the direct correlation of 80-
13 day as well as survival, which as Dr. Sullivan has
14 clearly stated in all of the trials that have been done
15 has never been seen, this is really an important trial
16 for us. To move forward from here and start over is
17 really economically not feasible for us with this
18 product.
19 CHAIRPERSON HUSSAIN: Dr. Mortimer.
20 DR. MORTIMER: Yes. My suggestion was going
21 to be a prophylactic study, but also it seems to me that
22 the biggest selling point of this drug is that it
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1 minimizes toxicity of steroids.
2 However the study is designed, a comparison of
3 this agent against steroids, if you demonstrate an
4 improved toxicity profile, I can’t but imagine that it
5 would move farther up in line for the indication
6 indicated here today.
7 CHAIRPERSON HUSSAIN: Okay. If there are no
8 other questions, I think that the FDA -- I’m sorry, Ms.
9 Haylock.
10 MS. HAYLOCK: I just wanted to suggest that
11 since some of the side-effects that were of concern to
12 people, for example, fatigue and then also some of the
13 other psychosocial issues, that some of our public
14 presenters brought up, there are quite a few
15 psychosocial tools or instrumentation available that
16 could be pretty easily incorporated into any trial. I
17 would suggest doing that. I think that would help as
18 well.
19 MR. SCHABER: May I say one more thing,
20 please?
21 CHAIRPERSON HUSSAIN: (Chairperson moving head
22 up and down.)
When has anyone ever seen Pazdur make a positive comment like this about a drug when there is a negative vote.
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1 in survival, and we’ve seen some survival advantages but
2 nothing spectacular, barring something like that, what
3 would be the point in bringing these discussions
4 forward?
5 DR. PAZDUR: I think to get public input on
6 and for discussion of points that may be missed by the
7 FDA reviewers. I am very thankful for the discussion
8 that we had today.
9 Personally, this drug because of this
10 discussion, irrespective of the vote, has a much
11 different impact in my mind. We will have discussions
12 internally on this drug and discuss the points that were
13 presented here.
14 CHAIRPERSON HUSSAIN: Thank you.
Pazdur will have the largest input on whether the drug gets approved. I think from the comments he made he likes the drug.
Dorb excerpts of ODAC transcript
Dr. Pazdur asks the panel to discuss whether another trial is feasible to run
1 large treatment, single-arm trial.
2 Here again, one of the questions that I have,
3 if that would be ongoing, would people even consider
4 doing another trial? Would another trial be feasible to
5 do? That’s another issue.
6 CHAIRPERSON HUSSAIN: Is this an issue you
7 want us to discuss now, or this is an issue that we want
8 to discuss after the vote?
9 DR. PAZDUR: Probably after the vote or it can
10 be discussed during the vote.
11 (General laughter.)
12 DR. PAZDUR: It’s really going to be part and
13 parcel of a decision here.
14 CHAIRPERSON HUSSAIN: Yes. Just a point of
15 clarification. If someone gets an IND for a specific
16 treatment, who pays for the cost of the drug?
17 DR. PAZDUR: Usually, the Sponsor would assume
18 the cost of that. Under the treatment IND mechanism,
19 however, which is a special type of expanded access
20 program, there can be some cost recovery on the part of
21 the Sponsor. We would entertain in a situation such as
22 this that type of program.
Mcdonal explains why trials using the drug for acute gvhd would be hard to run, because the doctors are formulating a drug themselves that isn't as good as Orbec but is better than placebo
1 in this group of patients for ethical and practical
2 reasons.
3 CHAIRPERSON HUSSAIN: Could you not consider
4 doing a trial, not placebo-controlled but with
5 equivalent doses of steroids to make the point that your
6 agent has a superior profile with regard to infections,
7 complications of steroids, and things of that sort? It
8 doesn’t have to be necessarily a survival-driven trial
9 but an efficacy trial?
10 DR. McDONALD: I think that is a
11 consideration, but actually there are some data that
12 bears on the question. If you look at the placebo arm
13 of 875, you will discover that 41 percent of people
14 required only 10 days of prednisone. If you look at the
15 placebo arm of the pivotal trial, 55 percent required
16 only 10 days of prednisone.
17 I think it is very difficult to argue that a
18 high-dose prednisone regiment would benefit any of those
19 patients who required only 10 days of prednisone. I
20 think the same ethical issues exist in doing a very
21 high-dose prednisone versus this approach.
22 We have already proved, I think, that the
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1 minimum prednisone approach protected by BDP has far
2 superior results to our 25-year standard of care. Again,
3 it is not the GVH that is killing people; it is the
4 treatment for GVH that’s doing the job.
5 I think we have adequately proved that BDP has
6 a steroid-sparing effect. I’m not sure I can go back 25
7 years in time and grab our old regimens. I am quite
8 convinced. I think it might well be unethical to do
9 that kind of a comparison.
10 DR. RODELL: Excuse me. Dr. Sullivan from
11 Duke was actually not involved in the trial.
12 CHAIRPERSON HUSSAIN: I just have another
13 question, though, please. If you say you don’t want to
14 go back to the past and grab old regimens, and if your
15 drug is currently not available on the market, and we
16 have heard that people use the oil-mixed product with
17 something, is that what people are currently using? Is
18 that what the alternative out there is?
19 DR. McDONALD: The alternative is what I call
20 the ad-hoc way of formulating something that is similar
21 to what we are using in these trials, that is, to use
22 the corn oil combined with budesonide.
the panel votes no
1 overwhelmingly we were against something and the FDA
2 approved it anyway.
3 (General laughter.)
4 CHAIRPERSON HUSSAIN: They don’t necessarily
5 always listen to us. Thank you.
6 If there are no other burning questions or
7 comments, I’m going to try to ask that we go ahead to
8 the vote.
9 Put the question up again, please.
10 (Staff complies.)
11 CHAIRPERSON HUSSAIN: We will begin around the
12 table. No description of why one is voting yes or no,
13 just the vote yes or no. Identify please yourself and
14 speak clearly into the microphone.
15 Dr. Link, can we begin with you, please?
16 DR. LINK: Michael Link. I vote no that it
17 has not shown substantial evidence.
18 MS. HAYLOCK: Haylock, no.
19 DR. HARRINGTON: No.
20 DR. MORTIMER: Mortimer, no.
21 CHAIRPERSON HUSSAIN: Hussain, no.
22 DR. RICHARDSON: Richardson, no.
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1 DR. PERRY: Perry, no.
2 DR. SPORTES: Sportes, yes.
3 DR. FLATAU: Flatau, yes.
4 CHAIRPERSON HUSSAIN: Two yes, seven no.
5 Dr. Pazdur, do you want us to go into the
6 second issue of design, of study design?
7 DR. PAZDUR: We’ve already started the
8 discussion.
9 CHAIRPERSON HUSSAIN: Yes, but I thought it
10 was for discussion, not for a vote.
11 DR. PAZDUR: We’ve already started the
12 discussion.
13 CHAIRPERSON HUSSAIN: In terms of what ideal
14 study design is?
15 DR. PAZDUR: Yes.
16 CHAIRPERSON HUSSAIN: Maybe I can begin and
17 ask the sponsor first, when you look at your data and
18 you look at what you started with and then what you got,
19 what do you think, like in your gut with yourself
20 looking at this, where do you think your strongest point
21 was? Because that seems to me where you should then go
22 back to look at that area because that may be where
Hussein explains that you couldn't run a trial in this population that could be stratified equally.
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1 between treatment arms, it would require, roughly, 160
2 deaths and a true hazard ratio of 0.55 would require,
3 roughly, 120 deaths. The accrual would be quite
4 substantial.
5 CHAIRPERSON HUSSAIN: Any words of wisdom from
6 committee members as far as study designs or suggestions
7 to the Sponsor or to the FDA with regard to what they
8 should advise them to do?
9 Sir?
10 DR. FLATAU: Yes. I would like to think at
11 least a secondary endpoint of overall survival at least
12 at one year, if not longer, would be interesting.
13 CHAIRPERSON HUSSAIN: I guess the only concern
14 I have about survival is unless you make the population
15 entering very uniform with the same diseases, the same
16 regimen, the same everything, you are never going to be
17 able to interpret the results, it would seem to me,
18 unless you stratify very clear where you include several
19 groups of people clearly stratified equal in each arm.
20 I can’t see how you would do it if you argue that we
21 don’t have a huge population.
Dr. Sportes the only transplant doctor on the panel and one of the positive votes explained that the 80 day endpoint which was met clinically means more than the 50 day endpoint
22 DR. SPORTES: Actually, I was wondering and I
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1 should have asked earlier why the secondary endpoint was
2 not the primary endpoint.
3 It seems to me from a transplant perspective
4 that a followup at 80 days is probably the strongest
5 endpoint, which is why I voted yes is because I think to
6 me conceptually this is much more a reflection of the
7 entire intervention. Why was that not chosen? I just
8 seek some explanation.
9 DR. RODELL: May I respond to that?
10 CHAIRPERSON HUSSAIN: Yes.
11 DR. RODELL: I think it’s important to
12 recognize the history of how this drug was developed.
13 The initial study, the initial efficacy study was Study
14 875 that was done essentially on a completely open
15 field, that is, this type of study had never been done
16 before and so the 30-day endpoint was selected in that
17 study, essentially, arbitrarily.
18 Once the results of that study were know, Dr.
19 McDonald spoke with and talked to the FDA, shared with
20 them the results of that study and began to design the
21 02 Study.
22 The two components with respect to efficacy
Dr. Link explains that the first 10 days on Orbec should be a guarantee period and is surprised that they didn't show this slide before the vote.
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1 The practicality, even without getting into
2 the economics of doing another study, is going to be a
3 significant issue in terms of the future development of
4 the drug.
5 CHAIRPERSON HUSSAIN: Thank you.
6 Any other comments?
7 (No verbal response.)
8 CHAIRPERSON HUSSAIN: Well, we will begin with
9 Dr. Link, then, and we will go around the table.
10 DR. LINK: I have two. The first is a
11 question of what if you actually -- the mistake maybe
12 was that you started the clock too soon in terms of you
13 started on the day of randomization. Either if you if
14 you had randomized after the 10 days and only randomized
15 responders -- Dave, you’re not going to shoot me here,
16 are you?
17 The second things is to actually do an
18 analysis, start the clock when they achieve remission
19 and throw out people who didn’t achieve remission, which
20 is often done in transplant studies. That would be one
21 suggestion.
22 What does it look like? Does it look better?
1 Does it look like you had achieved your goal, just out
2 of curiosity?
3 DR. RODELL: Yes, let me ask Mr. Cruickshank
4 to address that.
5 (PowerPoint presentation is in progress.)
6 MR. CRUICKSHANK: Going back to that question
7 about, what is the effect of those early failures, we
8 did do an analysis to look at the effect whereby
9 patients who failed during the 10-day high-dose
10 induction period of prednisone.
11 If you sensor the patients at the time of
12 treatment failure, you can see here that we lose the
13 effect of the crossing Kaplan-Meier curves. We have a
14 fairly clear, sustained difference between the arms at
15 day 50 as well as day 80.
16 DR. LINK: You should have presented that.
17 Second of all, I was impressed with the ability of the
18 bone marrow transplant community to do study with soft-19
tissue endpoints, a lot of patients, a “New England
20 Journal of Medicine” article, very prompt approval.
21 I’m just wondering why you’re so discouraged
22 about doing a trial like this? Admittedly, not
Page 148 Dr. Mcdonald from the Fred Hutchinson Center in Seattle again explains why it would be difficult to run the trials in acute GVHD, and explains that a prophylaxis trial would be the proper trial to run, but the FDA doesn't like mixing endpoints. Dr Pazdur chimes in, that he is open to negotiation
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1 everybody gets bowel GVH. But if you’re transplanting
2 60-year-olds, you probably will get a lot of it. I’m
3 just wondering why you’re so pessimistic about getting
4 it done? We’re going to do it in pediatrics, and we
5 don’t have that many patients.
6 DR. McDONALD: I’m pessimistic about doing a
7 placebo-controlled trial because I don’t think we can
8 get our IRB to approve one based on these data. I think
9 there are other options.
10 I mean, we have thought about a prophylaxis
11 trial, for example, but that’s a different indication,
12 though. This indication is for treatment of acute
13 graft-versus-host disease. A prophylaxis trial is for
14 prophylaxis of acute graft-versus-host disease.
15 From the FDA’s comments, you don’t like
16 merging of different kinds of trials to come to a
17 conclusion. That would take, what, two placebo-18
controlled prophylaxis trials for that --
19 DR. PAZDUR: We are always open for
20 negotiations.
21 (General laughter.)
22 DR. McDONALD: All right. That’s one thought,
Page 149 to 152 is basically the panel members all admitting that a prophlyaxis trial would be the way to go. Of course how does the drug get approved in acute GVHD if you run a prophylaxis trial.
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1 everybody gets bowel GVH. But if you’re transplanting
2 60-year-olds, you probably will get a lot of it. I’m
3 just wondering why you’re so pessimistic about getting
4 it done? We’re going to do it in pediatrics, and we
5 don’t have that many patients.
6 DR. McDONALD: I’m pessimistic about doing a
7 placebo-controlled trial because I don’t think we can
8 get our IRB to approve one based on these data. I think
9 there are other options.
10 I mean, we have thought about a prophylaxis
11 trial, for example, but that’s a different indication,
12 though. This indication is for treatment of acute
13 graft-versus-host disease. A prophylaxis trial is for
14 prophylaxis of acute graft-versus-host disease.
15 From the FDA’s comments, you don’t like
16 merging of different kinds of trials to come to a
17 conclusion. That would take, what, two placebo-18
controlled prophylaxis trials for that --
19 DR. PAZDUR: We are always open for
20 negotiations.
21 (General laughter.)
22 DR. McDONALD: All right. That’s one thought,
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1 that is, if this drug is as highly effective as a
2 topical therapy for treatment, it should be equally
3 effective and relatively nontoxic as prophylaxis. I
4 think that is one potential option.
5 CHAIRPERSON HUSSAIN: Gee, prophylaxis sounds
6 like very attractive. Why wait until an event happens?
7 I mean, that would be a clinically meaningful endpoint.
8 DR. McDONALD: I agree. Plus, it’s easier to
9 enroll patients who aren’t sick.
10 CHAIRPERSON HUSSAIN: Correct, correct.
11 DR. McDONALD: You can get a larger accrual.
12 Seven thousand patients should provide enough to enroll.
13 The hope there is that one would with effective
14 prophylaxis take what would ultimately be Stage IV and
15 turn them into Stage III, take Stage III and turn them
16 into Stage II, take Stage II and make them end up being
17 Stage I. I think that is a feasible thing. I can’t
18 speak for the company.
19 I think there are some financial issues. This
20 is a relatively small company, and I think that is a
21 consideration. In addition to the ethical and practical
22 issues, I think there are financial issues.
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1 DR. SCHABER: Dr. McDonald -- Chris Schaber,
2 president of Dor Bio Pharma -- that is in fact the
3 issue. For a company our size, we’ve put a lot of time,
4 effort, money, and resource into this study to support
5 Dr. McDonald and the rest of the investigators to go
6 after an indication that, quite honestly, big pharma
7 didn’t want to touch because it is a very small patient
8 population.
9 Although, as was outlined here and the experts
10 have spoken, we did not achieve the primary endpoint
11 without maybe the right sensoring or guarantee period.
12 With regard to the direct correlation of 80-
13 day as well as survival, which as Dr. Sullivan has
14 clearly stated in all of the trials that have been done
15 has never been seen, this is really an important trial
16 for us. To move forward from here and start over is
17 really economically not feasible for us with this
18 product.
19 CHAIRPERSON HUSSAIN: Dr. Mortimer.
20 DR. MORTIMER: Yes. My suggestion was going
21 to be a prophylactic study, but also it seems to me that
22 the biggest selling point of this drug is that it
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1 minimizes toxicity of steroids.
2 However the study is designed, a comparison of
3 this agent against steroids, if you demonstrate an
4 improved toxicity profile, I can’t but imagine that it
5 would move farther up in line for the indication
6 indicated here today.
7 CHAIRPERSON HUSSAIN: Okay. If there are no
8 other questions, I think that the FDA -- I’m sorry, Ms.
9 Haylock.
10 MS. HAYLOCK: I just wanted to suggest that
11 since some of the side-effects that were of concern to
12 people, for example, fatigue and then also some of the
13 other psychosocial issues, that some of our public
14 presenters brought up, there are quite a few
15 psychosocial tools or instrumentation available that
16 could be pretty easily incorporated into any trial. I
17 would suggest doing that. I think that would help as
18 well.
19 MR. SCHABER: May I say one more thing,
20 please?
21 CHAIRPERSON HUSSAIN: (Chairperson moving head
22 up and down.)
When has anyone ever seen Pazdur make a positive comment like this about a drug when there is a negative vote.
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1 in survival, and we’ve seen some survival advantages but
2 nothing spectacular, barring something like that, what
3 would be the point in bringing these discussions
4 forward?
5 DR. PAZDUR: I think to get public input on
6 and for discussion of points that may be missed by the
7 FDA reviewers. I am very thankful for the discussion
8 that we had today.
9 Personally, this drug because of this
10 discussion, irrespective of the vote, has a much
11 different impact in my mind. We will have discussions
12 internally on this drug and discuss the points that were
13 presented here.
14 CHAIRPERSON HUSSAIN: Thank you.
Pazdur will have the largest input on whether the drug gets approved. I think from the comments he made he likes the drug.
exas
I think their test is full of sh-t
OT- Biopharm this will be my last statement on the topic of Miller. I wasn't defending him. I was trying to make the point that his treatment on this board was deplorable.
The very fact that he has a subscription service, and a large following means (to me, at least) that he deserves a modicum of respect here, even if one does not agree with all (or any for that part) his statements or points.
Respect that sadly, I am not seeing here....
I agree with this statement. I do not usually agree with David but he has a following, and this board was lucky to have his comments because he is well thought of in the business, or he wouldn't have a business.
If I were him I certainly wouldn't participate in any further dialogue here. He has nothing to gain.
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MEDICURE INC. —MCU
CLOSE TO THE HEART
Overweight AMEX: $1.42 as of 7/31/07
Initiation Report
Key Data FY 2006 2007 2008
52-Week Range: $1 - 2 EPS
Market Cap. (mn): $165.1 1Q $(0.05)A $(0.03)A $(0.06)E
Shares Outstanding (mn): 116.3 2Q $(0.04)A $(0.06)A $(0.03)E
Average Daily Volume: 95,410 3Q $(0.03)A $(0.06)A $(0.02)E
Free Float (mn) 105.4 4Q $(0.03)A $(0.08)E $(0.02)E
Short Int. (% Free Float) 0.0 Year $(0.15)A $(0.23)E $(0.13)E
Fiscal Year End: 5/31 P/E NM NM NM
Revenue (mn)
Total Debt/Equity 34% 1Q $0.03A $0.25A $4.04E
TEV/TTM Sales: 41.0x 2Q $0.03A $1.26A $4.93E
Net Cash/Share: $0.13 3Q $0.05A $2.16A $5.72E
Book Value/Share: $0.27 4Q $0.15A $3.12E $6.29E
Price/Book Value: 5.4x Year $0.26A $6.79E $20.98E
Price Target $3.00 TEV/Sales NM 23.1x 7.5x
Source: Company reports, Thomson Financial and Thomas Weisel International estimates
Note: Medicure Inc. reports in CN$ we have used convenience translation into US$ equivalents. Price is as of the close on the date
indicated. Any price target displayed in the data box above represents either a specific price target or the midpoint of a range. EPS are pro
forma for stock-based compensation expense and one-time items.
• We are initiating research coverage of Medicure Inc. (MCU) with an Overweight rating and
a 12-month price target of $3.
• Growing Aggrastat sales; near-term phase III data on MC-1: Medicure is a cardiovascular drug
company. It has acquired and started marketing Aggrastat, a GPIIb/IIIa inhibitor. Lead pipeline
drug MC-1 is currently in a pivotal phase III study in coronary artery bypass graft (CABG) patients.
• Aggrastat set to recover; peak sales to cross $100mn: We believe that Aggrastat will recapture
significant market share in light of focused marketing efforts and an active salesforce. We estimate
peak sales to cross $100mn by 2015.
• MC-1 phase II data indicative of efficacy; likely to meet phase III endpoints: MEND-1 and
MEND-CABG phase II study results were clearly indicative of the cardio-protective properties of
MC-1. Based on that, we believe that the endpoints in the MEND-CABG II phase III studies are
likely to be met. We expect a mid-2009 launch of the drug and estimate that peak sales will reach
$700mn if additional indications of angioplasty and acute coronary syndrome are approved.
• Valuation: We obtained our 12-month price target of $3 by applying a 30x P/E multiple to our
2012 pro forma EPS estimate of $0.20 and discounting it back at 20%. There are always risks that
the price target for any security will not be realized. In addition to general market and
macroeconomic risks, for Medicure, Inc., these risks include, among other things, clinical trial
failure of its ongoing MC-1 development programs and competition from bigger pharmaceutical
companies.
August 1, 2007
HEALTHCARE
Biopharmaceuticals
Bino Pathiparampil
415.262.6365
Bpathiparampil@tweisel.com
August 1, 2007 Thomas Weisel International
2 Bino Pathiparampil 415.262.6365
CARDIOVASCULAR DRUG DEVELOPER WITH IMPRESSIVE PORTFOLIO
One product in market; high potential product in phase III; rich pipeline:
Medicure, Inc, located in Winnipeg, Manitoba, focuses on developing therapeutics for
the treatment of cardiovascular diseases. Aggrastat, Medicure’s sole product on the
market, was acquired from MGI Pharma in August 2006 and is used in patients
undergoing invasive cardiac procedures such as angioplasty. The company has one drug
currently in phase III studies to prevent ischemic injury in coronary artery bypass graft
(CABG) patients and two other clinical stage drugs—one in phase II for treatment of
hypertension in diabetics and another in phase I to treat hypertension in patients with
metabolic syndrome.
Aggrastat set to recover; peak sales could cross $100mn: Aggrastat (tirofiban
hydrochloride), a GPIIb/IIIa inhibitor, is used for the treatment of acute coronary
syndrome (ACS) and as an adjuvant to percutaneous transluminal coronary angioplasty
(PTCA) or atherectomy. After several years of languishing sales as a result of inadequate
marketing efforts, Aggrastat was acquired in the United States by Medicure. Its newly
formed, 25-person-strong salesforce is working actively and turning around the product.
We believe that Aggrastat can improve its market share significantly and we estimate that
peak annual sales will cross $100mn by 2015. Total U.S. sales for GPIIb/IIIa inhibitors,
including Reopro (abciximab) marketed by J&J and Integrilin (eptifibatide) marketed by
Schering Plough, totaled $430mn in 2006 (a 0.1% increase y/y), with Aggrastat
accounting for only $8.6mn. In the EU, however, where Merck actively markets the
product, Aggrastat has a significant market share and commands sales comparable to
those of Integrilin. Available data comparing the three GPIIb/IIIa inhibitors suggest that
Aggrastat is as efficacious as Reopro and Integrilin, with some added advantages.
Aggrastat is currently being evaluated in several clinical studies by Merck. Data from an
Italian study comparing Aggrastat with Reopro is expected in the spring of 2008. We
estimate Aggrastat FY07 U.S. sales of $6.79mn.
MC-1—fast-track status and SPA agreement with the FDA: MC-1, a vitamin B6
metabolite, has significant cardio-protective effects that can mitigate the effects of
reperfusion injury. Reperfusion injury occurs when blood flow to the heart muscle cells is
restored after a period of ischemia, leading to a flurry of cell-damaging immune
mediators. This typically happens in the setting of a coronary artery bypass graft
(CABG), PCTA and thrombolysis in ACS. MC-1 was granted fast-track status with the
FDA as a treatment to reduce cardiovascular and cerebrovascular events associated with
ischemic and/or ischemic reperfusion injury in patients undergoing angioplasty, CABG
surgery and in ACS.
$1bn potential opportunity; serving an unmet medical need: According to the
American Heart Association (AHA), approximately 400,000 CABG and 1.4mn
angioplasty procedures were conducted in the United States in 2006. There is currently
no cardio-protective drug available against reperfusion injury resulting from these
procedures. Assuming a per-patient cost of $600 for MC-1, this could represent a $1bn
opportunity, should the drug receive FDA approval.
August 1, 2007 Thomas Weisel International
3 Bino Pathiparampil 415.262.6365
MC-1 Phase II data suggestive of efficacy: In the 60-patient, MEND-1 phase II
study, MC-1 reduced the median area under the periprocedural CK-MB curve (AUK)
from 32.9ng/ml to 18.6ng/ml (a 43.5% reduction; p=0.038) on post-operative day 30
(POD30). It also showed a good safety profile. In the 900-patient, placebo controlled,
multi-dose MEND-CABG study, MC-1 achieved a 37.2% reduction in the composite
endpoint, with myocardial infarction (MI) defined as peak CK-MB ≥100ng/ml, in the
250mg arm versus placebo (p=0.028). The composite endpoint consisted of death, non-fatal
MI and non-fatal stroke. In addition, there was also a 46.9% reduction in the
incidence of non-fatal MI (peak CK-MB band ≥100ng/ml) in the 250mg MC-1 group.
MC-1 also achieved a 31.7% reduction in the composite end, with MI defined as peak
CK-MB ≥70ng/ml, in the 250mg arm versus placebo (p=0.035). The POD30 findings
of significant reduction in the composite endpoint continued to POD90; however, MC-1
could not establish significant improvement in the composite endpoint versus placebo
when MI was defined as peak CK-MB ≥50ng/ml. Also, the 750mg arm performed
worse than the 250mg arm; the company explains this as a result of the peaking of
plasma levels of MC-1 followed by accelerated excretion from the body. The compound
exhibited a good safety profile in the study.
MEND-CABGII phase III study ongoing; expect results in 1H08: MC-1 is
currently being studied in a 3,000-patient, double-blind, randomized, placebo-controlled
phase III trial, the MEND-CABG II study. This trial is being conducted in 120 centers
across the United States, Canada and Germany, and results are expected in 1H08. The
primary endpoint is a reduction in a composite of death and nonfatal MI (peak CK-MB
band ≥100ng/ml) up to POD30 compared to placebo. We believe that the trial has a
good chance of meeting its endpoint and expect a mid-2009 launch. We estimate that
peak annual sales could exceed $700mn if the other indications (PTCA and ACS) are also
approved. The company intends to conduct future label expansion studies in ACS and
stroke patients
RICH PIPELINE ADDS TO THE PROMISE
Satisfactory MC-4232 phase II data: phase III study pending: According to the
Centers for Disease Control and Prevention (CDC), more than 14mn Americans are
currently living with diabetes with another 6.2mn undiagnosed with the disease. Of those
diagnosed with diabetes, around 73% suffer from hypertension. MC-4232 is a
combination of MC-1 and lisinopril, an ACE inhibitor, and was recently evaluated in a
phase II trial, the MC-1 and ACE Therapeutic Combination for Hypertensive Diabetics
(MATCHED) study. Results from this study showed that MC-4232 reduced mean
daytime ambulatory systolic blood pressure (MDASBP) by 4.5mm Hg more when
compared with the use of lisinopril alone and reduced fasting serum glucose by
1.45mmol/L. No statistically significant decrease in HbA1c levels was seen, however.
The company intends to conduct a 1,500-patient phase III study, but further
development is currently on hold until results are available from the MEND-CABG II
study.
Good early stage drugs in MC-4262 and MC-4538: Other drugs in the pipeline
include MC-4262 (a combination of MC-1 and an angiotensin II receptor blocker (ARB))
currently in phase I for treatment of hypertension in individuals with metabolic
August 1, 2007 Thomas Weisel International
4 Bino Pathiparampil 415.262.6365
syndrome. Medicure is also developing a novel compound, MC-4538 which is currently
in preclinical evaluation for use as an anti-thrombotic agent.
MCU Pipeline
Products
Aggrastat ®
Acute Coronary Syndrome (ACS)
MC-1
Coronary Artery Bypass Graft (CABG)
Acute Coronary Syndrome
Stroke
MC-4232
Diabetic Hypertension
MC-4262
Hypertension Complicated
with Metabolic Syndrome
MC-45308
Thrombosis
MC-5422
Ischemia DISCOVERY
Source: Company reports
Approval Market Preclinical Phase I Phase II Phase III
COMPANY INSIGHTS
Experienced management: Dr. Albert Friesen, Medicure’s founder and CEO, has
been with the company since its inception in 1997. Prior to that, he held senior
management positions in several companies such as Viventia Biotech and Genesys
Pharma. He also played a significant role in the founding and successful sale of
companies including Rh Pharmaceuticals (acquired by Cangene) and ABI Biotechnology
(acquired by Apotex). Other senior management includes personnel with several years of
significant industry experience.
Good cash position: Medicure ended 3Q07 with more than $37mn in cash. Based on
our estimates, the company will be able to make it through late 2008 with the current
drug development plans. Medicure raised approximately $25mn in December 2006 via a
private placement of common stock at a price of $1.30 per share, in addition to the
issuance of approximately 4mn warrants.
Key shareholders: Major shareholders of Medicure include Columbia Wanger Asset
Management, L.P. (7.94%) and Dr. Albert Friesen (6.59%).
ESTIMATES
Our 4Q07 and FY07 revenue and pro forma estimates are $3.12mn and $(0.08) and
$6.79mn and $(0.23), respectively. Our FY08 revenue and pro forma estimates are
$20.98mn and $(0.13), respectively.
August 1, 2007 Thomas Weisel International
5 Bino Pathiparampil 415.262.6365
POTENTIAL UPSIDES TO OUR ESTIMATES
MC-1 label expansion; off-label use: Our current estimates for MC-1 only include
revenue from its use in CABG procedures. There could be significant upside potential to
our estimates should the drug be used for patients undergoing angioplasty procedures
and in ACS patients.
FDA approval of MC-4232: Our current revenue estimates do not include sales of MC-4232,
since further development of the drug is currently on hold. If results from the
planned 1,500-patient trial turn out positive and the drug receives FDA approval, there
would be significant upside to our revenue estimates.
POTENTIAL DOWNSIDES TO OUR ESTIMATES
Failure of MC-1 phase III trial: All of Medicure’s resources are currently directed
toward the MC-1 MEND-CABGII trial. Failure of MC-1 to demonstrate significant
benefit in the phase III trial would result in significant downside to our estimates.
Inability of the sales force to deliver: Medicure competes in the cardiovascular space
currently dominated by large pharmaceutical companies. Should the salesforce be unable
to gain a foothold in the market, this could result in lower revenue and downside to our
estimates.
Medicure, Inc - Near-Term Milestones
Drug Indication Milestone Timing
Aggrastat ACS Data from four ongoing clinical studies 2Q08
MC-1 CABG Phase III data 2Q08
MC-4232 Hypertension in Diabetics Initiation of phase III study 2H08
Source: Company reports and Thomas Weisel International estimates
RISKS
Drug development risks: All drug development programs carry an inherent risk of
failure by virtue of the unpredictable nature of clinical trial results and could result in
significant financial loss in terms of resources spent on the program.
Market risk: Medicure’s drug serves an unmet medical but will cater to the
cardiovascular market, which is highly dominated by large pharmaceutical companies.
Medicure could face intense competition from these firms with bigger salesforces,
especially in relation to Aggrastat.
VALUATION
We obtained our 12-month price target of $3 by applying a 30x P/E multiple to our 2012
pro forma EPS estimate of $0.20 and discounting it back at 20%. There are always risks
that the price target for any security will not be realized. In addition to general market
and macroeconomic risks, for Medicure, Inc., these risks include, among other things,
clinical trial failure of its ongoing MC-1 development programs and competition from
bigger pharmaceutical companies.
it is nice that the Bear Stearns CFO would make these comments since it was cowboys in their hedge funds that went to zero that caused a lot of this panic
DACOGEN DETRACTION This morning, SuperGen’s main competitor Pharmion (PHRM, Not Rated) showcased very impressive survival data in MDS patients on Vidaza. Based on these results, we believe that Vidaza could recoup significant market share in the MDS space and potentially expand its reach. Dacogen sales could stagnate or decline, until the maturation of the EORTC study in mid-2008, evaluating Dacogen’s survival benefit. Until this study yields results, we believe SuperGen shares could be range-bound. Thus, we are downgrading SuperGen shares to Market Perform / Speculative Risk rating without a target price.
Elemir Piros had a 25 dollar target. Rodman is known for nutty targets and this helps to prove that point
VIDAZA IMPRESSIVE The survival study yielded a two-year survival rate of 50.8% for Vidaza vs. 26.2% for conventional care regimens. Median survival benefit was 9.4 months (74%) for patients on Vidaza compared to conventional care regimens. High statistical significance was achieved (p<0.0001 via stratified log-rank test), while the hazard ratio was 0.58, indicating that high-risk MDS patients on conventional care regimens were almost twice as likely to die as those on Vidaza. Vidaza is now the only agent to demonstrate survival benefit in MDS. This was the largest study conducted in high-risk MDS.
NEAR-TERM IMPACT The near-term effect of these results is likely to be neutral or negative for Dacogen sales trends, since SuperGen and its marketing partner MGI Pharma (MOGN, Not Rated) will not have an equivalent weapon in their arsenal until mid-2008. Previously, Dacogen was closing in on Vidaza in the market place. Dacogen achieved >30% share and MGI recorded $30MM in sales in 2Q07. In the best case, sales could remain flat. In the worst case, Dacogen’s market share could go down to the 10% range, yielding annualized sales of $40MM for the next 12 months. We previously estimated sales of nearly $150MM for the same period. We will continue to monitor Dacogen sales trends through Oncology Inc. and IMS Health in order to better gauge impact of the Vidaza survival data in coming months.
CLASS EFFECT? Based on information available up until today, we believed that Dacogen is a more potent drug vs. Vidaza. Furthermore, the impressively positive survival data for Vidaza may point to a class effect for hypomethylating agents in general. However, until Dacogen demonstrates a survival advantage in a clinical trial setting the marketing advantage will rest squarely with Pharmion. We note that SuperGen is also developing next-generation molecules that inhibit DNA methyltransferase 1 (DNMT1), which are expected to have improved side-effect profiles vs. the current hypomethylating agents.
pozn
A friend of mine told me it was her largest position. I told her to buy puts as a hedge because I believed there was something in the package that the companies weren't fully elucidating as with Encysive.
I didn't buy puts because I didn't want to root against her. I am surprised it is trading here.
I think it goes to between 6 and 7
it gave nuvello and the analysts something to speak about after alfimiprase died
iomi
everyone is looking for new to get out of stocks. I didn't sell any.
The stock was trading at over 4 when their prior news of their patch versus a needle failed. The purpose of the patch is to use as an adjuvant with a needle. This trial validates the entire program but it happened in a bad market.
the stock is cheap and they will be able to partner this off.
the pipeline is validated.
Lehman Brothers l Regarding Amgen
Jim Birchenough (415) 274-5393, jbirchen@lehman.com
Ajim Tamboli (415) 274-5337, Charles Whitesell (212) 526-6687
Nick Abbott (206) 542-2492, Hartaj Singh (415) 274-5242
Ryan Martins (415) 274-5335, Badri Rengarajan (415) 274-5264
Amgen (AMGN 2-Equal weight, Sector 2-Neutral)
* AMGN announced its response to CMS's final National Coverage
Decision (NCD) for erythrocyte stimulating agents (ESAs) in cancer late
yesterday after market close, characterizing CMS coverage restrictions
as having no scientific basis, being contrary to FDA approved labeling
and limiting availability to Medicare beneficiaries with cancer. Despite
AMGNs objections CMS posted it's own "Questions and Answers to the NCD
for ESAs in Cancer" late yesterday, highlighting the impending deadline
for implementation and the stringency of the rules. With the NCD inline
with recent revised guidelines by the National Comprehensive Cancer
Network (NCCN) we believe that likelihood of revision is low and
continue to anticipate downside risk for Aranesp sales in cancer.
* CMS issued late Monday it's National Coverage Decision (NCD) for
ESAs in oncology suggesting restrictions on ESA initiation thresholds,
dosing, length of therapy and associated conditions. Initiation of ESAs
is now restricted to patients with Hgb<10g/dl, qwith dose initiation at
labeled doses, suggested length of therapy no longer than 8 weeks
following completion of chemotherapy and with discontinuation
recommended for Hgb rise<1g/dl over 8 weeks or >1g/dl over 2 weeks.
Proposed limitations on use in MDS and in conjunction with Avastin have
not been maintained but were viewed with considerable skepticism before
hand.
* In it's Question and Answer briefing posted yesterday
(http://www.cms.hhs.gov/mcd/ncpc_view_document.asp?id=12) CMS indicated
that it is developing instructions for its local contractors, that
implementation would occur by September 30th and that the NCD would
override local Medicare contractors local contract decisions. The
document further indicates that with initial coverage of ESAs for 4
weeks after Hgb falling below 10g/dl that further coverage beyond 4
weeks would require documentation that Hgb criteria was still met (ie
<10g/dl) with dates of service beyond January 1, 2008 requiring
beneficiaries Hgb or hematocrit level.
* As suggested previously following a review of 46 CMS local
coverage decisions we believe that only 15% of local carriers recommend
ESA initiation at Hgb <10g/dl and that for 85% of carriers the final NCD
will represent a dramatic shift. We would note further that with
electronic medical record data indicating 28% of patients initiated at
thresholds of hgb<11g/dl we believe that at least 20% of Aranesp sales
are at risk going forward. With further risk to the ESA franchise from
FDA cardiorenal panel review in September and with potential Mircera at
risk launch in 4Q07 we would continue to reiterate our equalweight
rating.
Iomai Vaccine Confers Statistically Significant Protection Against Travelers' Diarrhea in Phase 2 Study
Wednesday August 1, 6:45 am ET
- Interim Data from Field Study Also Shows Patch Vaccine Limits Severity, Shortens Duration of Travelers' Diarrhea -
- Conference Call Scheduled for 8:30 a.m. Eastern Today -
this data is so good I have to go to the toilet
GAITHERSBURG, Md., Aug. 1 /PRNewswire-FirstCall/ -- Iomai Corporation (Nasdaq: IOMI - News) today announced that its patch-based vaccine for enterotoxigenic E. coli (ETEC) bacteria conferred statistically significant protection from travelers' diarrhea as compared to placebo, particularly for more severe cases, according to interim data from a double-blind Phase 2 field study. Vaccinated travelers were 75 percent less likely to suffer moderate or severe diarrhea from any cause (p<0.01) and 84 percent less likely to be afflicted by severe diarrhea (p<0.05). No vaccine-related serious adverse events were reported.
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In addition to the protective effects, investigators found that the frequency and duration of diarrhea in vaccinated subjects who did contract disease was significantly lower than in their unvaccinated peers.
"The results from this study strongly suggest that the use of Iomai's needle-free, patch-based vaccine can have a notable impact in reducing the chances of suffering from the ravages of travelers' diarrhea," said Herbert L. DuPont, M.D., professor and director of the Center for Infectious Diseases at The University of Texas School of Public Health and the principal investigator on the trial. "Right now, we are generally limited to administering antibiotics after the illness has begun. An effective, easy-to-use vaccine would be a vast improvement and have an immediate impact on travel medicine."
The Trek Phase 2 field study followed 170 subjects, 111 who received a placebo and 59 who received two doses of Iomai's patch-based vaccine before traveling to Mexico or Guatemala. These efficacy results were particularly notable as the trial was conducted largely to gather information on the logistics of conducting such a study in the field. The study was conducted in collaboration with researchers from the Johns Hopkins Bloomberg School of Public Health and the University of Texas School of Public Health.
Iomai's vaccine uses the company's novel transcutaneous immunization (TCI) technology, which allows the vaccine to be delivered to the immune system via a simple patch affixed to the skin. ETEC causes illness through the toxins it produces, including one known as heat-labile toxin or LT. Iomai's unique patch-based vaccine enables the safe administration of this potent immunogen into the skin to stimulate the immune response.
"These convincing results, demonstrating high levels of efficacy in a real-world setting, serve as validation of our TCI approach," said Dr. Gregory Glenn, M.D., Iomai's founder and chief scientific officer. "The data suggest that our TCI technology can be used to provide protection against other pathogens. We are moving as quickly as possible to complete our Phase 2 work so we can begin pivotal trials and bring our travelers' diarrhea vaccine candidate to market."
The study met its primary endpoints, which were designed to evaluate the safety of the vaccine and the incidence of ETEC. The secondary objectives included evaluation of vaccine-preventable outcomes, immunogenicity and stool frequency. The study also gathered data on the protective efficacy of the vaccine. Iomai plans to submit the full dataset from the trial for publication as soon as possible. The company intends to launch a Phase 3 program for the vaccine in 2008.
About Travelers' Diarrhea
This year, approximately 55 million international travelers will visit countries where bacteria that cause travelers' diarrhea are endemic, particularly Africa, Asia and Latin America, and about 20 million of those travelers will develop travelers' diarrhea. "Though we always caution travelers about the best ways to avoid food and drink that may harbor the bacteria, that advice is often not enough, and an effective means of vaccination could substantially reduce the burden of disease," said Robin McKenzie, M.D., an assistant professor at Johns Hopkins and an investigator on the trial.
ETEC, the most common cause of travelers' diarrhea, represents just under half of all cases of travelers' diarrhea for international travelers to areas where ETEC is common. A recently completed market study suggested that there is a large market for an effective ETEC vaccine, potentially exceeding $500 million annually. If approved, the Iomai vaccine would be the first vaccine for travelers' diarrhea available in the United States.
ETEC's impact goes beyond travelers. The World Health Organization estimates that children in the developing world suffer 210 million episodes of diarrhea caused by ETEC annually, causing 380,000 deaths each year.
Conference Call Details
To access the live conference call today at 8:30 a.m. Eastern Time via phone, please dial 866-510-0705 from the United States and Canada or 617-597- 5363 internationally. The conference ID is 26738905. Please dial in approximately ten minutes prior to the start of the call. A telephone replay will be available beginning approximately one hour after the call through Aug. 8 and may be accessed by dialing 888-286-8010 from the United States and Canada or 617-801-6888 internationally. The replay passcode is 85144095.
To access the live and subsequently archived webcast of the conference call, go to the Investor Relations section of the Company's website at www.iomai.com. Please connect to the web site at least 15 minutes prior to the call to allow for any software download that may be necessary. The webcast is also being distributed through the Thomson StreetEvents Network. Individual investors can listen to the call at www.earnings.com, Thomson's individual investor portal, powered by StreetEvents. Institutional investors can access the call via Thomson StreetEvents (www.streetevents.com), a password-protected event management site.
Single gene deletion boosts lifespan
I will lose my jeans right now
"Cyberknife is a robotic radio-surgery system used to treat cancer lesions that are too large or inaccessible for the Gamma Knife, as well as tumors in the lungs, liver, pancreas, prostate, kidney, adrenal gland and spine. The Cyberknife system allows many patients to avoid surgery for surgically complex tumors."
thanks
I don't din dis happened
http://www.minyanville.com/articles/Dendreon-Provenge-FDA-Amgen/Goat+to+genius+on+a+passed+note/inde....
On May 9 and 10, ODAC meets in DC. On May 9, a superficially similar NDA for a drug from pink sheet company DOR BioPharma (DORB) called orBec will be in front of the committee. Despite potentially compelling efficacy data, I believe Dr. Pazdur will take out his “Provenge frustration” on orBec.
The stock is priced like it happened but it didn't happen.
DOR BioPharma, Inc. Announces 3 Month Extension of FDA PDUFA Action Date for orBec(R) New Drug Application to Review Supplemental Data
Thursday July 19, 6:00 am ET
DOR to Host Investor Teleconference at 11AM EDT July 19, 2007
MIAMI, FL--(MARKET WIRE)--Jul 19, 2007 -- DOR BioPharma, Inc. (OTC BB:DORB.OB - News) (DOR or the Company) announced today that it has received notification from the United States Food and Drug Administration (FDA) that the action date for the FDA's review of the New Drug Application (NDA) for orBec® (oral beclomethasone dipropionate) for the treatment of gastrointestinal graft-versus-host disease (GI GVHD) has been extended to October 21, 2007. The original action date under the Prescription Drug User Fee Act (PDUFA) for the orBec® NDA was July 21, 2007. On or before the new PDUFA date, the FDA will notify DOR whether or not orBec® will be approved for marketing, or whether or not it may be approvable after fulfillment of additional regulatory conditions.
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The extension is the result of DOR's July 13, 2007 provision of supplemental information to the orBec® NDA. This information was requested by the FDA at a June 13, 2007 NDA review meeting. According to FDA policy, the submission of this supplemental information was classified as a major amendment, putting the new action date for the orBec® NDA at October 21, 2007.
On June 13, 2007 DOR met with the FDA to review and discuss the orBec® NDA. The discussion was productive and focused on ways to more fully elucidate the clinical effect and benefit of orBec® in the treatment of GI GVHD. Based on these discussions, the FDA requested that DOR review its existing clinical data from both its randomized, double-blind, placebo-controlled trials and highlight pertinent additional data that could provide a more comprehensive picture of orBec®'s clinical effect in treating acute GI GVHD. This new presentation of data formed the basis for the recently filed supplemental submission to the orBec® NDA. Following the FDA's review of this submission, it was determined that it constituted a major amendment. Given the close proximity of the submission of additional information relative to the July 21, 2007 PDUFA date, the FDA determined that a three month extension to the PDUFA date was necessary to allow for an adequate and complete review.
"We appreciate the FDA's willingness to allow us to provide supplemental data and for taking the additional time necessary to ensure a complete and high level review of the orBec® NDA," said Christopher J. Schaber, Ph.D., President and Chief Executive Officer of DOR. "We will continue to work with the FDA in every way possible to support their review so that it may be completed as expeditiously as possible."