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mcbio
you have been following ACHN very closely. what is your opinion at the present time.
I believe a grade 5 AE is not important if Wallstarb owns the stock because the chart says it is going higher
the stock has gone from 6 cent to over 15 dollars in less than a month
Vermillion, Inc. (VRMLQ.PK) At 3:59PM ET: 15.50 0.50 (3.12%)
has this stock ever been mentioned on the board
MORE ON VRMLQ.PK
Date Open High Low Close Volume Adj Close*
6-Oct-09 16.39 17.00 14.41 16.00 268,800 16.00
5-Oct-09 17.00 17.05 16.39 16.50 192,500 16.50
2-Oct-09 15.20 16.90 14.00 16.50 235,000 16.50
1-Oct-09 13.45 16.25 12.50 15.74 358,600 15.74
30-Sep-09 10.05 13.50 9.10 13.50 522,300 13.50
29-Sep-09 13.75 14.00 8.25 11.00 792,300 11.00
28-Sep-09 10.40 13.80 9.10 13.50 423,900 13.50
25-Sep-09 8.49 9.90 7.50 8.45 727,000 8.45
24-Sep-09 3.85 6.00 3.82 5.95 408,800 5.95
23-Sep-09 4.05 4.05 3.78 3.90 120,300 3.90
22-Sep-09 4.05 4.25 3.70 4.00 269,700 4.00
21-Sep-09 3.25 4.05 3.18 3.99 472,300 3.99
18-Sep-09 3.30 3.69 3.10 3.44 324,000 3.44
17-Sep-09 2.24 3.38 1.87 3.20 669,000 3.20
16-Sep-09 2.21 2.30 1.36 1.93 768,500 1.93
15-Sep-09 3.30 3.30 2.44 2.44 425,900 2.44
14-Sep-09 2.00 4.79 1.99 3.08 2,819,800 3.08
11-Sep-09 0.05 1.29 0.05 1.29 1,942,500 1.29
10-Sep-09 0.05 0.05 0.05 0.05 1,700 0.05
9-Sep-09 0.05 0.05 0.04 0.05 1,700 0.05
8-Sep-09 0.04 0.04 0.04 0.04 18,500 0.04
4-Sep-09 0.05 0.05 0.04 0.04 12,700 0.04
3-Sep-09 0.04 0.05 0.04 0.05 3,300 0.05
2-Sep-09 0.05 0.05 0.04 0.04 7,500 0.04
1-Sep-09 0.05 0.05 0.04 0.04 900
can you explain why you think that is the better way to own it.
Sandip Guptal from Calcutta will remove your apendix for $18 an hr.
You are being generous. Dr. Sandip will want to increase his revenues so he may charge 18dollars an hour but he will sub out the procedures to his ten kids that have gotten their experience by playing video games.
you are correct.
getting a heart attack after using viagra is telling me that the drug is effective
don't buy it and when it goes up you won't make money
are you saying the Allos product has great response rates with long durability.
look at that market cap
what bar is set too high, Tasigna is contra indicated in t315i patients
tasigna is an oral agent with more side effects.
to avoid relapses you would give yourself a shot. it isn't IV.
I don't understand your pessimism
and you said 10 percent cytogenic response is not impressive, but these are after other drugs failed. Earlier in the process, or in combo they would get higher responses
in what respect was it short. the people in the chonic phase had long responses. People with accelerated phase and blast phase had shorter responses. One of the people in blast phase which is an end of life scenario lived over a year.
four out of forty patients had no trace of cancer. so it would make sense that the earlier Omacetaxine is give the more chance there would be to rid the patient of the disease but it is only given in people that have failed the other drugs.
the big problem with chemgenex is that there is no coverage in the US so there is no one to enlighten US investors to the drugs positives.
the analyst in Australia that covers it thinks that ANDA's get approved in six months.
that means nothing ignore 84086
they do not mention response rates. The trial is how old?
you can relapse and still live for two years so that fact that they haven't had deaths doesn't mean much.
chemgenex has an 85 percent response rate in the t315i mutation trial and an 80 percent response rate in the trial that people have failed at least 2 tki's and 51 percent of the patients failed all three, and everyone poo-poohed the data.
Chemgenx filed their NDA and will be approve in the first quarter. then it becomes the standard of care. How will ariad be able to enroll an open label study even if the FDA lets them, if Omacetaxine becomes the standare of care early next year.
Thats "on board", not "on the board"
then this is a different problem. If he isn't on the board, then he hasn't seen the data, so how can he say it is outstanding.
If he has seen the data then he has inside information, whether he is on the BoD or not.
Dr. Ron Garren, a biotech newsletter writer and a practising oncologist, is also on board with ARIA. He says the 534 results are outstanding and it is sure to find a niche market in resistant CML
Let me get this straight. He is on the board of Ariad, and he is telling his subsribers that the results of the 534 trial are outstanding. That sounds like inside information to me. I think he is smarter than that.
rkrw was being sarcastic when he said cvm was a once in a lifetime opportunity.
Reality is that it is a once in a lifetime opportunity for the ceo and his father who get paid over a million dollars a year each, to write letters to shareholders to pump the stock.
other favorites of M.E. Garza
Heb CVM and Ctic among others
http://seekingalpha.com/author/m-e-garza/articles/latest
David Bupp also said five years ago that he would be able to file Riggs without a phase 3 based on data that he already had.
He also said that he could get Lymphoseek approved based on one successful phase 3 trial but the stock went up when he announced that he would do a second phase 3 trial.
Dutton received their info from David Bupp the former CFO.
Unrelated cord blood transplant improves survival in fatal congenital diseases
By Martha Kerr
Published on Sep 16, 2008
--------------------------------------------------------------------------------
Article Tools:
Clinical
Unrelated cord blood transplant improves survival in fatal congenital diseases
Last Updated: 2007-12-13 16:13:52 -0400 (Reuters Health)
By Martha Kerr
NEW YORK (Reuters Health) - Infants with lysosomal and peroxisomal disorders (LSD), such as Krabbe disease and Hurler syndrome, which are often fatal, can be managed with umbilical cord blood transfusions from unrelated donors.
Transfusions of selected stem cells can replace the enzymes that cause the organ failure that accompanies these diseases, principal investigator Dr. Vinod Prasad of Duke University in Durham, North Carolina, told Reuters Health just after his presentation at the annual meeting of the American Society of Hematology in Atlanta.
Dr. Prasad presented the findings of umbilical cord blood transplantation received from unrelated donors by 159 children with LSD transplanted between 1995 and 2007. The children's median age was 1.5 years. Their performance status was below 80% and approximately 20% were cytomegalovirus (CMV)-positive.
The patients received pre-transplant conditioning and the cord blood was screened for the enzyme that the children lacked.
After transplantation, neutrophil and platelet engraftment was 87.1% at a median of 42 days and 71.0% at a median of 87 days.
The probability of acute grade III/IV acute graft-versus-host disease (GvHD) was 10.3% at 100 days. Chronic GvHD developed in 25 children (18%), with extensive disease in 11 children.
Overall survival was 79% at 6 months, 71.8% at 1 year, 62.7% at 3 years and 58.2% at 5 years. Higher performance status at baseline, higher concentration of enzyme infused and matched ethnicity were predictors of a better outcome after transplantation. With optimal matching, 5-year overall survival was 79.5%.
The primary problem with the management of these children, Dr. Prasad said, is "we (the transplant team) don't make the diagnosis. We only treat the child after referral to our center."
"We need to raise the awareness of healthcare providers about these diseases, and to make them aware that this treatment is available."
"It takes approximately 34 or 35 days to first diagnosis, and approximately 50 days until referral to us. It may take as long as 90 days until the transplant is performed. This is a significant delay and can have a big impact on outcome," Dr. Prasad stressed. "For diseases such as Krabbe, weeks matter in treatment success...It is ideal to do the cord blood transplant in asymptomatic patients."
"We need to improve the identification of patients for treatment and to develop more effective treatment techniques," the Duke investigator said.
Micheal Becker was the ceo of cytogen and made all kinds of outlandish statemnent of potential sales of his drugs.
Check out his bio. He is a high school graduate.
allos
fda.gov/downloads/AdvisoryCommittees/CommitteesMeeti ngMaterials/Drugs/OncologicDrugsAdvisoryCommittee/UCM18 0634.pdf
8. Summary
This NDA submission is based on overall tumor response rate from a single arm
phase 2 trial. The sponsor reported an overall response rate of 27% according to
IWC (n = 29). Nine patients (8%) achieved a CR and 20 patients (18%) achieved
a PR. The median duration of response was 287 days. However, 15 of 29
(51.7%) responders had their responses adjudicated because of the
disagreement between central readers 1 and 2 of the independent imaging
review committee (IRC).
According to FDA’s evaluation based on IWC, duration of response (DOR) in 13
of the 29 responders was < 14 weeks. Sixteen of these 29 responders had
confirmatory scans after initial designation of response. Thirteen of these 16
(12% of 109 evaluable patients) had a DOR of at least 14 weeks with 6 CR (5%),
1 CRu (1%) and 6 PR (5%). Median duration of response cannot be assessed in
these 16 patients due to few events and data censoring.
FDA seeks the advice of ODAC regarding the clinical significance of the overall
response rate and duration of response as well as the benefit:risk ratio for
pralatrexate treatment in patients with relapsed or refractory PTCL.
7.3 Safety
Safety assessments were performed on 111 enrolled patients who had received at least
one dose of pralatrexate. Mucositis (70%) and thrombocytopenia (41%) were the most
common AEs. AEs were the reason for dose reductions in 31%, dose omission in 69%
and treatment withdrawal in 22% of the patients.
There were a total of 49 Serious Adverse Events (SAEs) reported and those reported in
≥ 3 patients were pyrexia (8 patients), mucosal inflammation (6 patients), febrile
neutropenia (5 patients), sepsis (5 patients, 1 septic shock), and thrombocytopenia (3
patients).
Eight deaths were reported within 30 days of their last dose of pralatrexate. Seven were
attributed to PD and 1 was due to cardiopulmonary arrest (possibly related to
pralatrexate).
The FDA granted the drug a priority review (Acorda had not requested that). So the FDA finds the drug to be addressing an unmet medical need, and/or being a significant improvement over the current SOC.
could you provide a link to the fact that the company did not ask for priority review?
Entire market rally has been achieved based on a simple accounting rule change that Steve Forbes was proposing in 2008, prior to the bailout of the banks.
Read how the change in the rule below was the start of the rally. Also remember when reading this that the bad loans and derivatives on the banks books are still there. The only difference is that the banks aren't marking the to their value and because of that, they have been able to show profits and raise money from private funds to increase their capital requirements.
On March 10, 2009, In remarks made in the Council on Foreign Relations in Washington, Federal Reserve Chairman Ben Bernanke said, "We should review regulatory policies and accounting rules to ensure that they do not induce excessive (swings in the financial system and economy)". Although he doesn't support the full suspension of basic proposition of Mark to Market principles, he is open to improving it and provide "guidance" on reasonable ways to value assets to reduce their pro- cyclical effects.[18]
On March 16, 2009, FASB proposed allowing companies to use more leeway in valuing their assets under "mark-to-market" accounting, a move that could ease balance-sheet pressures many companies say they are feeling during the economic crisis. On April 2, 2009, after a 15-day public comment period, FASB eased the mark-to-market rules. Financial institutions are still required by the rules to mark transactions to market prices but more so in a steady market and less so when the market is inactive. To proponents of the rules, this removes the unnecessary "positive feedback loop" that can result in a deeply weakened economy.[19]
Companies can use the new guidance when issuing their first-quarter financial statements.[20] Such changes could significantly boost banks' statements of earnings and losses[21]. The FASB changes, however, are for acceptable accounting standards applicable to a broad range of derivatives, not just banks holding mortgage-backed securities.
The DJIA hit a market low of 6,443.27 on March 6, 2009.[6] The bear market reversed course on March 9, 2009, as the DJIA rebounded more than 20% from its low to 7924.56 after a mere three weeks of gains.[7] Despite the technical achievement of the DJIA and S&P 500 having increased 20% in value from March 9 through early May, many economists and traders still consider this rally to be a powerful secular bear market rally due to the fact that the fundamentals of the economy are still in decline. However, many other economists are forecasting an end to the recession as early as Summer 2009. As the stock market historically recovers several months before the economy begins to improve, the numbers are agnostic with respect to the economic outcome.
In Cortex's case, the FDA was correct in accepting the NDA for Phase 1 and the Phase 2a studies, since the shorter term animal tox data supporting those trials was clean.
The tox problem only occured in a longer term 3 month dosing animal tox study, which was submitted to support Cortex's proposed Phase 2b. That was the stage where the FDA appropriately red-flagged Cortex's drug.
The fda accepted Cortex's IND, not nda and they were correct in doing that. I was only using Cortex as an example to show that the fda is concerned about preclinical tox. So I can't understand why they accepted Heb's nda for filing if they never did the tox work. They would never have been able to raise the money to contiues the scam.
>The Cortex (COR) and Hemispherx (HEB) cases have nothing in common as far as I can tell. COR’s drug was in phase-2 when a tox problem showed up, and the FDA put the drug on clinical hold for the given indication (ADHD). What was unusual about this case was that the tox may have been an artifact of laboratory processing rather than a real pathology; however, in all other respects, this case was routine. <
I wasn't saying it was similar, I was saying that the fda is very concerned about this tox work even in early stage development and I used Cortex as an example of this. What I was saying was that the fda should not have accepted the nda filing at all.
You said you aren't sure it was accepted but I am 99 percent sure that it was. Carter keeps the con going by being very clever in the way he words his releases, but if he ever said the filing was accepted when it wasn't I believe the fda or sec would go after him. It isn't the same as getting a complete response letter and not reporting it, because since he never thought it would get approved, he can make the determination that, when it came it wasn't material. A ligitimate company wouldn't do that, but HEB would.
Question for the board
A lot od comapanies run into problems in their animal tox studies. A problem there pretty much destroyed Cortex.
Wouldn't you think there would be some kind of checklist that the FDA would have before they accept an NDA or Bla filing from a sponsor that would include whether the company had finished their tox studies.
Hemispherx would have never been able to continue the scam if the Ampligen filing would have been refused on this basis.
I am actually amazed that all the law firms are willing to start this litigation because there isn't any cash to pay off a claim
insurance doesn't cover much,
FDA panel backs limited use of Amgen bone drug
>certainly not what the analysts following this in a breathless manner were looking for.<
FDA panel gives mixed endorsement to Amgen drug for osteoporosis patients
On Thursday August 13, 2009, 3:28 pm EDT
Buzz up! 0 Print.Companies:Amgen Inc.
WASHINGTON (AP) -- Federal health experts say a drug from Amgen Inc. benefits patients with osteoporosis, but its use should be limited because of long-term safety questions.
A Food and Drug Administration panel unanimously voted that Amgen's injectable drug denosumab benefits women with osteoporosis. However, panelists voted 12-3 against using the drug as a preventive measure for women who are at risk for osteoporosis because of low bone density.
Panelists say they want to see more safety data on the drug before recommending it to millions of patients with weakening bones.
The FDA is not required to follow the group's advice, though it usually does.
Denosumab represents a significant market opportunity for Amgen, with sales projections over $1 billion.
>But if this is the best that pharma can boast of getting from Washington, then it must figure its future is pretty bleak. Not only can Congress change the terms of this deal at any moment, but when spending on ObamaCare runs off the rails, where does Pfizer CEO Jeff Kindler think Congress will go for the money?<
As with the financial industry execs, of three years ago, the pharma companies are playing ball so the current management can get out in two or three years with their options excersized, before the sh-t hits the fan. the fallout from all of this will be someone else's problem.
this is a greater diservice. who at the fda recieved a paper bag with five million dollars to approve Vanda's drug?
One other Vanda related question -- who is the mystery "regulatory consultant" paid $5 million by Vanda for his assistance in getting Fanapt approved by the FDA?
Vanda, Oculus, Sequenom: Biotech Notebook
Adam Feuerstein
08/11/09 - 07:00 AM EDT
BOSTON, Mass. (TheStreet) -- Vanda Pharmaceuticals' (VNDA Quote) buyout partner remains missing in action. No more improper drug marketing claims from Oculus Innovative Sciences (OCLS Quote). Sequenom (SQNM Quote) walks back expectations for its Downs syndrome gene test -- these are some of the random notes and observations from second-quarter earnings.
Vanda Pharmaceuticals
Vanda reported a higher-than-expected loss for the second quarter Monday, but it's what the company didn't report -- a buyout or marketing partnership for its schizophrenia drug Fanapt -- which disappointed investors the most.
Three months have passed since the U.S. Food and Drug Administration's approval of Fanapt surprised everyone (including Vanda, from what I hear), yet the company seems no closer to launching the drug.
Vanda bulls are hoping the company is acquired outright, or at the very least, Vanda manages to license Fanapt to a larger drug company in exchange for a hefty sales-based royalty. Monday, however, the company said it was still evaluating all its options, which includes launching the drug on its own.
Vanda CEO Mihael Polymeropoulos said Monday that the company's plan is to have Fanapt available to patients in the fourth quarter. Yet if Vanda tries to sell Fanapt on its own, the stock will get crushed.
One other Vanda related question -- who is the mystery "regulatory consultant" paid $5 million by Vanda for his assistance in getting Fanapt approved by the FDA?
Vanda shares closed down 10.2% to $13.93 on Monday.
Drugs work best when swine flu found early - study
Mon Jul 27, 2009 5:47pm EDT
* Study shows need to diagnose cases quickly
* Findings contradict some policies
By Maggie Fox, Health and Science Editor
WASHINGTON, July 27 (Reuters) - Health officials trying to make best use of antiviral drugs to fight the new H1N1 flu need to do a better job of tracking and treating cases quickly, Italian researchers said on Monday.
Having stockpiles of drugs does little good if people do not get them within the 24 to 48 hour window when they work best, Stefano Merler of the the Bruno Kessler Foundation in Trento, Italy and colleagues reported.
"Our study also highlights the importance of the early detection of cases," they wrote in the BioMed Central journal BMC Infectious Diseases.
"In fact, great effort should be made in order to establish a surveillance system able to detect and treat cases as soon as possible since a delay of more than 24 hours could make both antiviral treatment and prophylaxis very inefficient."
But H1N1 swine flu has spread so fast that the World Health Organization has stopped counting cases, which in turn may encourage national health authorities to cut back on testing.
Merler's team found that if governments can track cases, quick use of drugs could help a great deal.
They said their mathematical models showed giving antiviral drugs to the elderly to protect them from infection would not save many lives, but treating younger people would.
Only three quick tests are licensed in the United States for diagnosing H1N1 flu -- one commercial test from Quest Diagnostics (DGX.N: Quote, Profile, Research, Stock Buzz) and two government tests.
The WHO has advised countries to stockpile enough antivirals to treat 25 percent of their populations.
These include zanamivir, made by GlaxoSmithKline (GSK.L: Quote, Profile, Research, Stock Buzz) under license from Biota Inc. (BTA.AX: Quote, Profile, Research, Stock Buzz) and sold under the brand name Relenza, and oseltamivir, made by Roche AG (ROG.VX: Quote, Profile, Research, Stock Buzz) under license from Gilead Sciences (GILD.O: Quote, Profile, Research, Stock Buzz) and sold under the brand name Tamiflu.
Both drugs can reduce the risk of death among people at serious risk of flu complications -- the very old, the very young, people with asthma and other chronic diseases and pregnant women. But they must be given within the first day or two -- which makes it important to diagnose people promptly.
Both can also be used to prevent infection. Most health experts say it is important to do this only for people who are both at high risk of complications and who have been in close contact with someone confirmed to be infected.
Some policies call for using these drugs as prophylactic agents in nursing homes, for example, where viruses can sweep through and kill many frail residents.
In a pandemic, with the drugs in high demand, this strategy may not save enough lives, the researchers said.
"Our work demonstrates that even in countries where the antiviral stockpile is not sufficient to treat 25 percent of the population, the minimum level suggested by the WHO, it is possible to reduce morbidity and excess mortality by prioritizing the use of antivirals by age," Merler said in a statement.
He said Italy, for example, only had enough drugs to treat 12 percent of its population.
Their model included data from the last three flu pandemics, in 1918, 1957 and 1968. In 1918, the virus affected more young adults and older children than usual -- just as has been seen with the H1N1 pandemic.
The only way to save lives with a small stockpile of drugs, they said, was to treat everyone who was infected, and give the drugs prophylactically to younger people. (Editing by Paul Simao)
© Thomson Reuters 2009. All rights reserved. Users may download and print extracts of content from this website for their own personal and non-commercial use only. Republication or redistribution of Thomson Reuters content, including by framing or similar means, is expressly prohibited without the prior written consent of Thomson Reuters. Thomson Reuters and its logo are registered trademarks or trademarks of the Thomson Reuters group of companies around the world.
Thomson Reuters journalists are subject to an Editorial Handbook which requires fair presentation and disclosure of relevant interests.
This seems like very interesting news in the current flu frenzy that is going on. If one dose of this is comparable to Tamiflu that would certainly make compliance a non issue with this treatment.
BTA has announced positive Phase III results in adults for a single
inhaled dose of its long acting version of Relenza (now named
laninamivir). The Phase III trial showed that a single inhaled dose of
laninamivir was shown to be as effective as oseltamivir (Tamiflu)
administered orally twice daily for 5 days. Together with its partner
Daiichi Sankyo (Japan's largest pharmaceutical company), laninamivir
will seek approval from the Japanese regulatory authorities, with a
submission in March 2010. A licensing partner will be sought outside
Japan. Given the significant interest in influenza, will believe there
is a strong chance that a licensing agreement could be achieved over the
next six months.
One thing I find a bit strange about this board in this debate is that no one here has put forward any ideas and/or numbers on what it will mean for pharma/bio investors when 40-50 million people are added to the health insurance rolls. More people diagnosed with infectious diseases and millions more prescriptions for them? (Gilead must be drooling at the possibility.) My hesitant assumption is that healthcare reform in this country means huge increased revenues for BP and BB..
do you think that the vast majority of people being treated with very expensive drugs for hep c and HIV are on insurance?
Just because the 40 or 50 million people do not have insurance does not mean they aren't getting medical care.
Sorry, I see a couple of other people already responded to this point.
Is this true. I hope it isn't
I’m not looking to make friends either. However, I do welcome input from other posters, which is what I meant by stopping by to say hello.
I realize that, I was kidding
it’s called The Global Demographic Tailwind and it can be accessed at #board-15787. Please stop by and say hello if you find this subject of interest.
In the words of Cramer, who I can't stand, I'm not here to make friends I am just here to make money.
Advanced Life Sciences Holdings, Inc. (OTC Bulletin Board: ADLS - News) announced that it received a complete response letter today from the U.S. Food and Drug Administration (FDA) regarding its New Drug Application (NDA) for Restanza(TM) (cethromycin) for the outpatient treatment of adults with mild-to-moderate community acquired pneumonia (CAP). In its letter, the FDA indicated that they cannot approve the application for Restanza in its current form and that, to gain approval, additional clinical data is required to demonstrate efficacy. Advanced Life Sciences submitted the NDA in September, 2008 based on the results of 53 clinical studies including 2 pivotal Phase 3 studies in CAP and a safety database of over 5,000 patients.
(Logo: http://www.newscom.com/cgi-bin/prnh/20080218/ALSLOGO)
Advanced Life Sciences intends to comply with the Agency's recommendations and continue to pursue approval of Restanza in CAP. The Company believes that an additional well-controlled clinical study designed to demonstrate efficacy in a more severe CAP population will likely be required for the approval of Restanza. In parallel, the Company will continue to advance Restanza as a biodefense agent against anthrax, plague and tularemia.
On June 2, 2009, the FDA Anti-Infective Drugs Advisory Committee (AIDAC) reviewed the Restanza NDA. The AIDAC voted that Restanza demonstrated safety for the outpatient treatment of adults with mild-to-moderate CAP, but voted that Restanza did not demonstrate efficacy in the treatment of CAP. The committee's negative vote on the drug candidate's efficacy followed a discussion that the Restanza NDA included data on patients with mild-to-moderate disease and that the new draft guidance for developing treatments for CAP, released in March, 2009, requires the enrollment of moderate-to-severe CAP patients for approval in the mild-to-moderate CAP indication. Advanced Life Sciences' pivotal Phase 3 program included in the NDA was designed and conducted under prior FDA guidance and before the new draft guidance was released.
Advanced Life Sciences is moving expeditiously to develop a protocol for an additional Phase 3 trial that is designed to satisfy the FDA's request for additional efficacy information. The Company intends to meet with and work closely with the Agency to design and establish a protocol under a Special Protocol Assessment. The company anticipates that initiation of the additional Phase 3 program could take place in the first half of 2010. Advanced Life Sciences is working collaboratively on these clinical plans with Wyeth, its development and commercialization partner in the Asia Pacific region.
"Advanced Life Sciences appreciates the FDA's response to the Restanza NDA and is committed to complying with the Agency's requirements to conduct a clinical program that could lead to approval in CAP," said Dr. Michael Flavin, Chairman and CEO of Advanced Life Sciences. "We are, however, disappointed that the timing of the new draft CAP guidance issuance had an unfortunate impact on the outcome of our NDA. Looking ahead, we intend to design a study that complies with the new draft CAP guidance and we will work with the Agency to determine the optimal pathway to registration."
Dr. Flavin continued, "We remain encouraged by the supportive comments made by AIDAC committee members in June relative to their positive view of the safety profile of Restanza, the therapeutic need for more effective treatments for CAP and their interest in seeing the Company pursue further development. We continue to receive strong support from our commercial partner Wyeth and intend to work in close collaboration with them as we implement our clinical program. We believe that Restanza has significant therapeutic and commercial potential as a novel agent to combat CAP as well as other lethal pathogens that represent serious bioterror threats to public health, and we intend to explore all avenues that can lead to success. We expect key data to be available and reported from our ongoing pivotal studies in plague and tularemia by the end of 2009. We plan to submit an NDA amendment seeking marketing approval for the biodefense indications in the first quarter of 2010."
In addition to developing Restanza as a potential treatment for CAP, Advanced Life Sciences has received a $3.8 million contract from the Department of Defense, via the Defense Threat Reduction Agency (DTRA), to fund NDA-enabling studies to evaluate Restanza's efficacy against bioterror agents such as tularemia, plague and melioidosis. In June, the company announced that Restanza demonstrated a 100% survival rate against an inhaled lethal dose of anthrax following a 14-day course of treatment in a second non-human primate study. The study was supported by the National Institute of Allergy and Infectious Diseases (NIAID).
Conference Call to Discuss the Restanza CAP NDA
Advanced Life Sciences will host a conference call and live webcast at 8:30 a.m. Eastern Time on Monday, August 3, 2009 to discuss the Restanza CAP NDA. The conference call will be webcast simultaneously over the Internet. Please visit the Investor Relations section of the Advanced Life Sciences corporate website www.advancedlifesciences.com. Alternatively, callers may participate in the conference call by dialing 866.362.4832 (domestic) or 617.597.5364 (international). The passcode for the conference call is 61915444. A replay of the conference call will be available until August 10, 2009. Callers may access the telephone replay by dialing 888-286-8010 (domestic) or 617-801-6888 (international), passcode 67060945. Investors are advised to dial into the call at least ten minutes prior to the call to register.
About Restanza
My gut instinct is usually a very good starting place, and one has to think this is going to be an issue for market acceptance. A pill beats a shot, but not if the shot is every 3 months. Depends on how often the pill needs to be taken.
A pill can be stopped during the menstrual cycle not the depot. They would want a drug holiday during their cycle.
chemgenex I believe has good efficacy and is a sub q injection not infusion. If it lets you live longer you will take a shot.
as far as becoming obsolete chemgenex was able to do an open label phase 2 pivotal study because nothing worked in the t315i mutation. Once omacetaxine gets approved early next year other drugs will have a higher hurdle to get approval.
In the open label phase 2 pivotal trial in all comers it is important to note that over 50 percent of the patients not only failed gleevec but also sprycel and tasigna so the omacetaxine results I believe are pretty good considering that.
here is the company slide presentation if you care
http://www.chemgenex.com/pdf/CXSClinicalupdateJune2009.pdf
tspt wallstarb
everyone always respond to his ideas. he accumulates thinly traded shares and says he is buying and posts on many boards.
believe me he will not be in the stock at the pdufas date
lupus expert conference call
The expert panel included:
• Dr. David Isenberg, Professor of Rheumatology at University College London, past
President of the British Society of Rheumatology, member, founder, and recent chair of
the Systemic Lupus Erythematosus International Collaborating Clinics, founding editor of
the journal "Lupus", author of the text "Lupus" and one of the
creators of the BILAG tool for assessing lupus activity. He is also chair of the British
Society's For Rheumatology Biologic's Register committee and Chair of the Arthritis
Research Campaign's autoimmune rheumatic disease clinical trials sub-committee.
• Dr. Jan Hillson, Medical Director of Clinical Research, Immunology and Inflammation
at ZymoGenetics. Dr. Hillson served as Clinical Associate Professor of
Medicine/Rheumatology at University of Washington Medical Center, and as Division Head of
Rheumatology for Virginia Mason Medical Center, consulting rheumatologist for Group
Health of Puget Sound and Assistant Professor of Medicine for University of Washington
Medical Center Division of Rheumatology. She is an editorial reviewer for Lupus and is on
the Medical Advisory Board for the Lupus Foundation.
The following are excerpts from the call.
Q. How do you read BLISS-52 trial?
HILLSON. It's a milestone trial. It demonstrated that one can design a successful lupus
trial. It validated the BLyS pathway. It's good news for lupus patients.
ISENBERG. It demonstrated a lupus drug can meet endpoints. It's a psychological positive
to clinicians and companies who are developing lupus drugs.
Q. Any red flags from the data?
ISENBERG. Don't see much from the top line data available so far. In terms of adverse
effects, there are no untoward side effects. Efficacy -- clearly met end points.
HILLSON. Endorse that. Safety profile so far looks favorable. The drug can add safely to
standard of care.
Q. Benlysta worked in 58.3% of the patients, versus standard of care 45%. Is this a
meaningful clinical benefit?
ISENBERG. The results are highly statistically significant. Need to see more details.
Need to see which organs and systems are improved. It may turn out one biologic is better
to treat one system or another.
HILLSON. Lupus is a very heterogeneous disease. We will learn if patients who responded
are the ones likely to respond. Some analysts say the results indicate 14% of the
patients will benefit. That's too simplistic.
Q. The trial excluded patients who are serologically negative. What does that mean in
the real world?
ISENBERG. Normally, there are only 4% of lupus patients who are serologically negative.
In Benlysta's Phase 2 trial, there were 20% of the patients who were serologically
negative. That's a little puzzling.
HILLSON. This may demonstrate the difficulty in standardizing the diagnosis of lupus.
Q. BLISS-52 appears to have missed the BILAG end point.
ISENBERG. If they separate A flare and B flares, there might be a different conclusion.
HILLSON. In order to achieve the flare end point, there is a need for sufficient time
for flare to occur. Patients were on steroids for the first 40 weeks. There is not enough
time in a 52 week trial.
Q. Better chance to achieve flare end point in the 76 week trial?
HILLSON. Don't want to put it so strongly. The trial is powered to achieve treatment
effect. Not flare.
Q. Any thoughts on the geographic effects on the two trials?
ISENBERG. Lupus is more severe in certain ethnic groups. For example, African Americans,
Asians tend to have a more severe disease course than Caucasians. It will be interesting
to see.
Q. Does concurrent medication interfere with the results?
ISENBERG. Principal reason Rituxan failed. If sustained high dose steroids were not the
case, the Rituxan trial may have succeeded.
HILLSON. As in oncology drug development, initially one needs to layer on the
experimental drug to current standard of therapy. Later, one aims to minimize the
concurrent meds.
Q. Would you care to give an odds ratio that BLISS-76 trial will do better or worse than
BLISS-52 trial?
ISENBERG. Not really. If the drug works in 52 weeks, it should work in 76 weeks.
HILLSON. Curves between placebo and drug separated early. They separated again later in
the study, after steroids were tapered. That adds confidence the drug may work in 76
weeks.
Q. Can you lay out the competitive landscape?
ISENBERG. Current medication is not satisfactory. There is about 15% infection risk.
Biologics are more targeted. Rituxan had encouraging early results. Disappointed in the
large trial. If we have a better designed trial, it may work. Anti-IL6 antibody is
interesting. Atacicept study can also be positive.
HILLSON. Anti-CD20 drugs are promising. All three drugs in this category: Rituxan,
ocrelizumab and Arzara. Some immune suppression approaches could also work. The success
of Benlysta is encouraging for Atacicept. In addition to targeting BLyS pathway,
Atacicept also targets the APRIL pathway. It could be a better drug.
ISENBERG. I want to emphasize one point. HGSI did very well in executing the study. The
trial was adequately prepared. Physicians and nurses must be carefully trained. HGSI did
that. Lupus is a complicated disease. It's not easy to standardize the evaluation.
Q (from audience). Can you comment on the geographic effects again? Lupus is a
heterogeneous disease. Considering the degree of background therapy, is it fair for HGSI
to make a blanket statement that geography does not have a major effect on the trials?
ISENBERG. With internet use, internationally the practice is not as disparate.
Q (from audience). What makes you think a new trial of Rituxan will do better?
ISENBERG. Previous trial enrolled mostly patients with lung and skin symptoms. If you
give 40 mg of steroids, you don't need Rituxan to make a difference. The physician
training in the trial could also be improved.
Q (from audience). Is it possible Rituxan is more efficacious than Benlysta?
ISENBERG. It is very likely each drug will find a different patient subgroup. Rituxan
may work in patients who are resistant to multiple therapies and in patients with
hematological symptoms. Benlysta may work in patients susceptible to T and B cell
responses.
Q (from audience). Is 45% placebo effect typical?
ISENBERG. It's rather high. In RA trials, we see similar a phenomenon. I don't have a
good explanation.
HILLSON. I thought the placebo effect is rather high also. It tracks efficacy by time.
Lupus waxes and wanes. But again, I am a little surprised by the placebo effect.
Q (from audience). How effective is micofenelate?
ISENBERG. There is an approximate equivalence between micofenelate and cyclophosphamid.
About 50% response rates. It does appear the drug can be effective for other than for
renal.
HILLSON. The issue is toxicity. The emergence of latent viruses, including neurological
viruses. Biologics will not be a simple substitution to micofenenlate/cyclophosphamid.
Rather, they will be a step up.
Q (from audience). Any comment on Lupuzor?
ISENBERG. It's a peptide. So far results are quite interesting. It lowers antibodies to
DNA. It's quite behind Benlysta in development.
Q (from audience). If Benlysta gets approved, what percentage of the patients will
benefit?
ISENBERG. Difficult to answer. Consider the inadequacy of the current therapies.
Probably Benlysta will be used first in the set of patients for whom immune suppression
has failed. Also for patients who have failed steroids. Also consider the flare data. In
one year, 10% of the patients will have A flare. 50% will have B flare.
Q (from audience). Do you envision the drug to be used in a continuous basis or used
intermittently?
ISENBERG. In my experience in treating lupus patients, beneficial effects on kidneys can
last a year. On joints, symptoms can come back in a few weeks. So, it depends. It may be
used intermittently.
Q (from audience). If one looks at SLEDAI scale, which symptoms are more important?
ISENBERG. One would think death or more visible signs such as rash. But most often, what
bothers the patient is fatigue.
Q (from audience). If pain and fatigue are not impacted, do you see patients being
motivated to receive monthly infusions?
ISENBERG. Need to see data. For most patients with whom the physician has good rapport,
they will be motivated.
HILLSON. Many lupus medications make the patient feel worse. Nonetheless, patients can
be motivated to remain on those drugs. Understanding the consequences of failing therapy
makes them motivated.
Q (from audience). Do you have the relative number of lupus patients versus rheumatoid
arthritis patients who receive systemic therapies?
HILLSON. In the US, 1/200 people have RA, 1/2000 people have lupus. So, the disease
prevalence is 1 to 10. There are 330,000 lupus patients in the US, 350,000 lupus patients
in Europe. 2/3 of these patients are receiving some immune suppression meds. 60% suffer
flare in a given year. Look at it another way. 150,000 to 200,000 patients will be
candidates for biologics each year.
Q. Comments on Atacicept?
HILLSON. The drug is in a Phase 3 trial, under an SPA with the FDA. Full enrollment is
expected at the beginning of next year. Results a year later. Both Benlysta and Atacicept
work on the BLyS pathway. Atacicept may be more potent. Atacicept also inhibits a second
target on the B cell: APRIL. Hope to see additional data. It is subcutaneously
administrated.
Q. Are there differences in clinical trial design?
HILLSON. Benlysta trials are designed to treat active disease. Atacicept trial is
designed to prevent flare. Patients coming into the trials are very similar.
Q. The Atacicept trial randomizes only patients who have responded to initial high dose
steroid therapy. I assume this gives enough time to demonstrate flare?
HILLSON. The trial is powered to see flare. There is a 60% annual frequency of flare.
Out of 500 patients, we could expect to see 300 flare. This is an adequate size to
demonstrate a drug effect. An interim analysis to test the power has been included.