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ChemGenex Announces Publication Confirming Activity of Ceflatonin® in T315I-Positive CML
this is what their current pivotal study is all about. No drug has done this before
Overview
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ChemGenex announced the publication of a report in the prestigious Nature publication Leukemia confirming the positive clinical activity of the company’s lead compound, Ceflatonin® against Gleevec®-resistant, chronic myeloid leukemia (CML) associated with the T315I Bcr-Abl mutation.
The publication by Dr. Laurence Legros of the Hôpital Archet, Nice, France and colleagues describes the treatment with Ceflatonin (homoharringtonine, HHT) of a Gleevec-resistant, chronic-phase CML patient with the T315I Bcr-Abl mutation. The authors report that the patient experienced a 50 percent reduction of T315I Bcr-Abl levels within 2.5 months of treatment initiation, and the complete disappearance of the mutation within 5.5 months of treatment. Complete hematological response (CHR) was achieved after three cycles of therapy.
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Publication Details:
BCR-ABLT315I transcript disappearance in an Imatinib-resistant CML patient treated with Homoharringtonine: A new therapeutic challenge? Leukemia advanced online publication May 31, 2007.
Laurence Legros1, Sandrine Hayette2, Franck E. Nicolini3, Sophie Raynaud4, Kaddour Chabane2, Jean-Pierre Magaud2, Jill-Patrice Cassuto1, Mauricette Michallet3
1 Hematology Department, Hôpital Archet 1, 06202 Nice, France;
2 Laboratory for cytogenetics and molecular biology and EA3737, Centre Hospitalier Lyon Sud, 69495 Pierre-Bénite, France;
3 Hematology Department, Hôpital Edouard Herriot, 5 place d’Arsonval 69437 Lyon, France;
4 Hematology Laboratory, Hôpital Archet 2, 06202 Nice, France.
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For full press release please go to
www.chemgenex.com
drbio, re CRTX:
I think that you're right re the seller being just about done here. Selling pressure down to a trickle here @ 2.44. I expect to see an analyst reco on CRTX very soon. With the seller out, we should see a big bounce soon.
according to sec filings he is down to 589000 thru friday.
He killed the stock. I am pissed I didn't look to buy a day later.
my timing is impeccable
Response Biomedical Files for US FDA 510(k) Market Clearance of Rapid Influenza A+B Test
Monday June 11, 8:30 am ET
VANCOUVER, June 11 /CNW/ - Response Biomedical Corporation (TSX-V: RBM, OTCBB: RPBIF) announced today that it has filed a US Food and Drug Administration (FDA) 510(k) submission seeking clearance to market a rapid Influenza A+B test.
The test manufactured by Response will run on the RAMP(R) platform and will be marketed and sold exclusively by 3M Health Care as the 3M(TM) Rapid Detection Flu A+B Test. It is a qualitative immunochromatographic assay indicated for use as an in vitro diagnostic product used with the 3M(TM) Rapid Detection Reader (manufactured by Response) to identify the presence of Influenza A and Influenza B (Flu A and Flu B) nucleoprotein antigen in nasal wash, nasal swab, nasopharyngeal aspirate and nasopharyngeal swab specimens. Measurement of Influenza A and Influenza B aids in the rapid differential diagnosis of influenza viral infections through this point-of-care (POC) test.
POC or "near-patient" testing allows for diagnostic assays to be performed at the site of patient care delivery. POC testing provides for rapid clinical decision-making by reducing the time spent ordering tests, collecting and transporting samples, as well as retrieving data.
"Once again, RAMP has demonstrated exceptional performance," said Bill Radvak, President and CEO. "Attendance at several recent trade shows has seen lots of excitement about the RAMP Reader's ability to not only give a positive indication of flu but also delineate between Flu A and B. This is seen by many as being superior to our competitors' manual strips." Both companies hope to make this important test commercially available before the next U.S. flu season.
About Flu
Flu is a contagious disease caused by the influenza virus. Influenza viruses are classified into types A, B and C. Type A is a reportable disease, and is the most common and usually causes the most serious epidemics. Type B outbreaks also can cause epidemics, but the disease it produces is generally milder than that caused by type A. Type C viruses have never been connected with a large epidemic. Flu A is most prevalent and causes 87 percent of infections with the remaining 13 percent of reported cases being Flu B.
Every year, 5 percent to 20 percent of the U.S. population suffers from flu. Approximately 36,000 people infected with the flu die each year, and over 200,000 are admitted to hospital. During an average flu season, flu and flu related complications are the sixth leading cause of death in the United States.
Although most people who get the flu recover within a week, some of the medical complications caused by flu infection include bacterial pneumonia, dehydration and worsening of chronic medical conditions, such as congestive heart failure, asthma or diabetes. Children may develop sinus problems and ear infections as complications from flu infections. It is these complications, rather than the symptoms of flu themselves that lead to serious concerns with rapid diagnosis of infected individuals. The Centers for Disease Control (CDC) recommend that any person at high risk for serious complications of influenza and who is within the first two days of illness onset should be treated with antiviral medications.
About Response Biomedical
Response Biomedical develops, manufactures and markets rapid on-site diagnostic tests for use with its portable RAMP Platform for clinical and environmental applications. RAMP represents a new paradigm in diagnostics that provides high sensitivity and reliable information in minutes. It is ideally suited to both point-of-care testing and laboratory use. The RAMP system consists of a portable fluorescent reader and single-use disposable test cartridges, and has the potential to be adapted to more than 250 medical and non-medical tests currently performed in laboratories. RAMP clinical tests are commercially available for the early detection of heart attack and congestive heart failure.
In late 2006, the Company formed a strategic alliance with 3M Company to commercialize rapid infectious disease tests.
In the non-clinical market, RAMP Tests are currently provided for the environmental detection of West Nile Virus, and Biodefense applications including the rapid on-site detection of anthrax, smallpox, ricin and botulinum toxin. Several other product applications are under development. The Company has achieved CE Marking and its Quality Management System is registered to ISO 13485: 2003 and ISO 9001: 2000.
Response Biomedical is a publicly traded company, listed on the TSX Venture Exchange under the trading symbol "RBM" and quoted on the OTC Bulletin Board under the symbol "RPBIF". For further information, please visit the Company's website at www.responsebio.com.
About 3M Health Care
Since inventing Ioban(TM) antimicrobial incise surgical drapes more than 30 years ago, 3M has been a worldwide leader in developing health care products and services that address infection control. 3M Health Care, one of 3M's six major business segments, is dedicated to improving the practice, delivery and outcome of patient care and is a leading provider of solutions for medical, oral care, drug delivery and health information markets.
Ioban is a trademark of 3M.
The TSX Venture Exchange has not reviewed and does not accept
responsibility for the adequacy or accuracy of this release.
Statements contained in this press release relating to future results, events or developments, for example, statements containing the words "believes," "may," "could", "plans," "will," "estimate," "continue," "anticipates," "intends," "expects", "goal" and similar expressions, are "forward-looking statements" or "forward-looking information" under applicable United States and Canadian securities laws. Forward-looking statements or information may involve, but are not limited to, comments with respect to our planned activities, business plan and strategies and their future implementation, and our expectations for our financial condition and the results of, or outlook for, our business operations generally. Forward-looking statements or information are subject to the related assumptions made by us and involve known and unknown risks, uncertainties and other factors that may cause actual results, events or developments to be materially different from those expressed or implied by such statements or information.
Many of such risks, uncertainties and other factors form part of our underlying assumptions, and include, among other things, our need for substantial additional funding to conduct research and development and commercialization activities; our ability to establish, and our dependence upon, relationships with strategic alliance partners to develop and commercialize products; technological changes that impact our existing products or our ability to develop and commercialize our products; our ability to obtain and enforce timely patent and other intellectual property protection for our technology and products; our ability to obtain and maintain rights to technology from licensors; commercialization limitations imposed by patents owned or controlled by third parties; our ability to retain, and our reliance upon, third party suppliers, manufacturers and distributors; our ability to attract and retain qualified personnel; our ability to effectively and efficiently manage the planned growth of our operations; our ability to obtain, and the timing of, necessary regulatory approvals; our ability to profitably sell our products at prices that would be acceptable to third-party reimbursement programs; market acceptance of our products and the size of our markets; changes in business strategy or development plans; changes in, or the failure to comply with, governmental regulations; and other factors referenced in our annual report on Form 20-F and other filings with Canadian and United States securities regulatory authorities.
Given these uncertainties, assumptions and risks, readers are cautioned not to place undue reliance on such forward-looking statements or information. We disclaim any obligation to update, or to publicly announce any revisions to, any such statements or information to reflect future results, events or developments.
For further information
Response Biomedical Contacts: Bill Wickson, Manager, Investor Relations, Response Biomedical Corporation, Tel (604) 456-6073, Email: bwickson@responsebio.com
Brian Korb, Vice President, The Trout Group LLC, Tel: (646) 378-2923, Email: bkorb@troutgroup.com
Pipex Pharmaceuticals' Oral TRIMESTA Initiates Enrollment of Phase II/III Clinical Trial for Multiple Sclerosis
Monday June 11, 7:45 am ET
$5 Million Grant From National Multiple Sclerosis Society, With Support From NIH
Previous Phase II Demonstrated 79% Reduction in Gadolinium Enhancing Brain (MS Lesion) Volumes (p=0.02) and Exceptional 14% Increase in Cognitive Function (p=0.04)
ANN ARBOR, MI--(MARKET WIRE)--Jun 11, 2007 -- Pipex Pharmaceuticals, Inc. (OTC BB:PPEX.OB - News), a specialty pharmaceutical company developing innovative late-stage drug candidates for the treatment of neurologic and fibrotic diseases, announced today that TRIMESTA (oral estriol), its proprietary therapy for multiple sclerosis (MS) has entered a multi-center, Phase II/III clinical trial for the treatment of women with relapsing-remitting MS. This clinical trial has received a $5 million grant from the National Multiple Sclerosis Society (NMSS) in partnership with the National MS Society's Southern California chapter, with support from the National Institutes of Health (NIH).
Previous Phase II Clinical Trial Results in Relapsing Remitting Multiple Sclerosis
TRIMESTA (oral estriol) has completed an initial 22 month, single-agent, crossover phase II clinical trial in the U.S. for the treatment of MS in relapsing remitting patients, with highly encouraging results. The results showed the total volume and number of enhancing pathogenic myelin lesions (established neuroimaging measurements of disease activity in MS) decreased during the treatment period as compared to a six-month pretreatment baseline period. The median total enhancing lesion volumes decreased by 79 percent (p=0.02) and the number of lesions decreased by 82 percent (p=0.09) within the first three months of treatment with TRIMESTA.
Following a three month drug holiday during which the patients weren't on any drug therapies, TRIMESTA therapy was reinitiated during a retreatment phase of this clinical trial. The relapsing-remitting MS patients again demonstrated a decrease in enhancing lesion volumes of 88 percent (p=0.008) and a decrease in the number of lesions by 48 percent (p=0.04) compared with original baseline scores (1),(2).
Improvement in Cognitive Testing Scores
During this phase II clinical trial, a 14 percent improvement in Paced Auditory Serial Addition Test ("PASAT") cognitive testing scores (p=0.04) was observed in these MS patients at six months of therapy. PASAT is a routine cognitive test performed in patients with a wide variety of neuropsychological disorders such as MS. The PASAT scores were expressed as a mean percent change from baseline and were significantly improved in the relapsing-remitting group.
Dr. Rhonda Voskuhl, Professor of Neurology at the University of California, Los Angeles and inventor of TRIMESTA, commented, "Due to its rapid onset of action, oral activity and high response rate seen in relapsing-remitting MS patients in the initial Phase II clinical trial, TRIMESTA may have an important clinical advantage over current injectable MS treatments. As a neurologist, the improvement in cognitive testing scores may also represent a new paradigm in treating MS patients as well as other neurodegenerative diseases with TRIMESTA."
Dr. John R. Richert, Executive Vice President of Research and Clinical Programs at the National MS Society, stated, "We're encouraged that the National MS Society's funding of the original pilot trial of estriol, which emerged from targeted research on gender differences in MS, has led to this new trial. We are pleased that Pipex and Dr. Voskuhl are committed to advancing the development of this novel approach which could potentially lead to an affordable, oral disease-modifying therapy for this debilitating disease."
Dr. Charles Bisgaier, Pipex's President, stated that, "The active ingredient in TRIMESTA has been marketed for over 40 years in Europe and Asia for the treatment of post-menopausal hot flashes. As we enter this Phase II/III clinical trial of TRIMESTA for treatment of MS, the product's known historical efficacy and tolerability coupled with the promising Phase II clinical trial results in MS patients an exciting new approach to treating this devastating disease with a targeted orally active drug candidate."
Dr. Bisgaier went onto say, "The exceptional increase in cognitive function together with the rapid reduction in lesion volumes and numbers positions TRIMESTA to become therapy of choice in the $10 billion global MS market. We are grateful for this $5 million grant and the support of the NMSS/NIH. We are excited about working with the National MS Society, Dr. Voskuhl and her team in furthering this promising new approach."
About the Phase II/III Clinical Trial
The clinical study is a double-blind, placebo-controlled trial that will take place at seven sites in the U.S., enrolling up to 150 female MS patients. Investigators will administer TRIMESTA to women between the ages of 18-50 who have been recently diagnosed with relapsing-remitting MS. TRIMESTA will be given in combination with subcutaneously injected Copaxone®, a standard treatment for MS. The team is evaluating effects of the treatment combination on relapse rates using several clinical and magnetic resonance imaging measures of disability progression.
The study sites include the University of California, Los Angeles (UCLA), Ohio State University (OSU), Rutgers University (UMDNJ), Washington University, St. Louis, University of Chicago, University of Utah and Wayne State University. For further information on this Phase II/III clinical trial, please visit www.clinicaltrials.gov/.
Pregnancy and MS
Doctors have known for decades that women often experience a sharp drop in MS disease symptoms during the course of pregnancy, specifically in the third trimester when the levels of estriol is being produced at their highest level by the placenta. The list of autoimmune diseases that improve during pregnancy includes multiple sclerosis, rheumatoid arthritis, thyroiditis, uveitis, juvenile rheumatoid arthirits, ankylosing spondylitis with peripheral arthritis and psoriatic arthritis.
A landmark clinical study published in the New England Journal of Medicine, known as the PRIMS study (Pregnancy in Multiple Sclerosis) followed 254 women with MS during 269 pregnancies and for up to one year after delivery. The PRIMS study demonstrated that relapse rates were significantly reduced by 71 percent (p < 0.001) through the third trimester of pregnancy from prebaseline levels and relapse rates then increased by 120 percent (p < 0.001) during the first three months postpartum before returning to prepregnancy rates (3).
About TRIMESTA
TRIMESTA is an orally active, immunomodulatory and anti-inflammatory molecule the has been approved and marketed throughout Europe and Asia for approximately 40 years for the treatment of post-menopausal hot flashes, but has never been introduced in North America. Estriol, the active ingredient in TRIMESTA, is a weak estrogenic-based molecule that is produced in the placenta by women during pregnancy. Estriol is considered to play an important role in the immunologic privilege offered to the fetus during pregnancy and is also thought to be responsible for the spontaneous remission of Th1-mediated autoimmune diseases of women (such as multiple sclerosis and rheumatoid arthritis) during pregnancy, especially during the third trimester. Pipex has an exclusive worldwide license with UCLA (through the Regent of the University of California) to the intellectual property rights surrounding TRIMESTA.
About Multiple Sclerosis (MS)
MS is a chronic, usually progressive disease of the central nervous system in which the immune system attacks and destroys the structure, and therefore degrades the function, of nerve cells. Approximately 400,000 Americans have MS, and virtually every hour someone is newly diagnosed. Most are between the ages of 20 and 50, and women are affected two to three times more often than men. Worldwide, MS may affect 2.5 million individuals.
According to the National MS Society, the economic cost of care for MS patients in the United States including medical and non-medical care, production losses, and informal care exceeds $23 billion annually, or more than $57,000 per U.S. patient per year. Complications from MS may make it harder for people to work and may interfere with their ability to perform common, daily activities. During 2006, combined sales estimates of FDA-approved injectable MS therapies, which include Avonex®, Betaseron®, Copaxone®, and Rebif®, totaled approximately $5.0 billion.
For most people with MS, the disease slowly progresses with a series of unpredictable flare-ups, also called relapses or exacerbations. But for some, the progression of the disease is rapid. Relapses often lead to increasing disabilities such as walking impairment, muscle weakness or speech or vision impairments.
About the National MS Society
The National MS Society is committed to building a movement by and for people with MS that will move us closer to a world free of this disease. The Society funds more MS research, provides more services to people with MS, offers more professional education and furthers more advocacy efforts than any other MS organization in the world. The Society has approximately 500,000 members, including more than 300,000 people with MS, and over 460,000 volunteers who carry out the Society's mission. For further information on the National MS Society, please visit www.nationalmssociety.org.
About Pipex Pharmaceuticals, Inc.
Pipex Pharmaceuticals Inc. is a specialty pharmaceutical company that is developing proprietary, late-stage drug candidates for the treatment of neurologic and fibrotic diseases. Pipex's strategy is to exclusively in-license proprietary, clinical-stage drug candidates and complete the further clinical testing, manufacturing and regulatory requirements sufficient to seek marketing authorizations via the filing of New Drug Applications (NDAs) with the FDA in the US and Marketing Application Authorizations (MAAs) with the European Medicines Evaluation Agency (EMEA). For further information please visit www.pipexpharma.com.
(1) Sicotte NL, Liva SM, Klutch R, Pfeiffer P, Bouvier S, Odesa S, Wu TC, Voskuhl RR. Treatment of multiple sclerosis with pregnancy hormone estriol. Ann Neurol. 2002 Oct. 52(4):421-8.
(2) Soldan SS, Alvarez Retuerto AI, Sicotte NL, Voskuhl RR. Immune modulation in multiple sclerosis patients treated with pregnancy hormone estriol. J Immunol. 2003 Dec 1:171(11):6267-74.
(3) Confavreux, C., Hutchinson, M., Hours, M.M., Cortinovis-Tourniaire, P., and Moreau, T. Rate of pregnancy-related relapse in multiple sclerosis. 1998. Pregnancy in Multiple Sclerosis Group. N Engl J Med 339:285-291.
Copaxone® is a registered trademark of Teva Pharmaceuticals.
This press release contains forward-looking statements, within the meaning of Section 21E of the Securities Exchange Act of 1934, that reflect Pipex Pharmaceuticals, Inc. (the "Company," "we" or "our" "Pipex") current expectations about its future results, performance, prospects and opportunities, including statements regarding the potential use of TRIMESTA (TM) for the treatment of Multiple Sclerosis. Where possible, the Company has tried to identify these forward-looking statements by using words such as "anticipates," "believes," "intends," or similar expressions. These statements are subject to a number of risks, uncertainties and other factors that could cause actual events or results in future periods to differ materially from what is expressed in, or implied by, these statements, including the risks set forth in our most recent filing on Form 10-QSB and Form 10-KSB filed with the Securities and Exchange Commission. We cannot assure you that we will be able to successfully develop or commercialize products based on our technologies, including COPREXA(TM), TRIMESTA(TM), Anti-CD4 802-2, CORRECTA(TM), EFFIRMA(TM) and SOLOVAX(TM) particularly in light of the significant uncertainty inherent in developing, manufacturing and conducting preclinical and clinical trials of new pharmaceuticals and obtaining regulatory approvals, that our technologies will prove to be safe and effective, that our cash expenditures will not exceed projected levels, that we will be able to obtain future financing or funds when needed, that product development and commercialization efforts will not be reduced or discontinued due to difficulties or delays in clinical trials or due to lack of progress or positive results from research and development efforts, that we will be able to successfully obtain any further grants and awards, maintain our existing grants which are subject to performance, that we will be able to patent, register or protect our technology from challenge and products from competition or maintain or expand our license agreements with our current licensors, or that our business strategy will be successful. All forward-looking statements made in this press release are made as of the date hereof, and the Company assumes no obligation to update the forward-looking statements included in this news release whether as a result of new information, future events, or otherwise.
Contact:
For Further Information Contact:
Steve H. Kanzer, CPA, Esq.
Chairman and Chief Executive Officer
Tel (734) 332-7800
Or
Thomas Redington (investor relations)
Redington, Inc.
Tel: (203) 222-7399
http://www.redingtoninc.com
--------------------------------------------------------------------------------
Details of the comparison between ambrisentan and sitaxsentan (thelin) that I wonder about but never hear discussed:
1. sitaxsentan trials enrolled patients with connective tissue disease which is a more severe form of PAH; how did that affect trial outcome, especially with regards to liver failure rates? My understanding is that the liver dysfunction is partially related to the disease which is why you see liver enzyme increases in the placebo group.
2. the original concept behind ERAs was to counterbalance the high endothelin levels seen in PAH patients and heart failure patients which is thought to cause constriction by binding to the Endothelin A receptor. Thein has a greater specificity for the A receptor than the B receptor and is the only ERA shown in the clinic to reduce endothelin levels. Does this cause the drug to be more effective with long term dosing?
3. Recent cc with Encysive, company stated that 40% of tracleer patients stop the drug due to no efficacy -- is this because of point 2?
I agree that at this point, the street sees ambrisentan as the better drug....I suspect that many docs who treat PAH have the same idea. What I wonder is if that is a valid assumption.
it appears that Myogen learned a lot about the data from the way Encysive ran their trials. they kept out patients that wouldn't would show the best drug effect. they also changed the definition of liver elevations which seemed to fool analysts.
the thing that has bothered me is that the FDA has held up encysives drug for over a year so there could be data about the safety of the drug that we are unaware of.
Thelin also didn't get priority review and ambrisentan did. that has weighed in my mind.
As investors we do not get all the data that the fda does
I understand the patent on Zyflo expires in 2012. Is that correct? If true, would that alter your favorable outlook for CRTX?
Look at the market cap and 4 years of sales in decent quantities still favors the stock at being cheap. Look what happened to viopharam getting a drug that was losing it patent's sales up.
they are working on an R-isomer which would estend patent situation for them
Can anybody comment on any example where approval was granted post issuance of this 483 letter? Looking for indication of minimum time required.
You usually need the 483 cleaned up before you can get approval. Staar Surgical had a lens held up for almost 2 years
Question: shouldn't you be buying more CRTX stock while there is overhang from "prospect partners" which is keeping the price lower? Or do you expect the selling to drive the shares down ever lower to $2 before the seller is done? After the seller is gone the price will bounce up and I'd hate to chase the price as it rises quickly[/]
I bought a boatload just before he filed that he sold half of his position because I felt it had gotten too cheap. I did that without the facts. Now with my eyes open I can't buy more knowing he has more to sell.
I believe there was a 300 thousand share block towards the end of the day and 238 thousand after hours. It is possible he has sold out on Friday, it traded almost 2 million shares. I am sure the company is working on trying to find funds to take him out.
As you remember Staar Surgical took awhile to get their 483's resolved but it was always disclosed.
this is a big deal.
Regeneron Pharmaceuticals (REGN 1-Overweight, Sector 2-Neutral)
* REGN reported positive phase II data for its VEGF TRAP,
aflibercept, in patients with platinum resistant ovarian cancer as well
as Tarceva resistant non small cell lung adenocarcinoma (NSCLA) at this
weekend's American Society of Clinical Oncology (ASCO) annual meeting.
With responses rates in the range of that with Avastin, VEGF TRAP
remains a legitimate competitor with timelines on track for 2008 BLA
submission.
* REGN's VEGF TRAP had interim data reported this weekend from
a randomized phase II study comparing low and high dose VEGF TRAP in 162
patients with platinum resistant ovarian cancer. Blinded results
combining both doses yielded a 10% response rate in the 1st stage of the
study with 8% RR after randomization of 162 patients. Of note, in 23
patients with malignant ascites, 7 patients (29%) experienced complete
resolution.
* While there has been some suggestion that these results fall
below the 16% response rate reported for Avastin at last year's ASCO we
would highlight a response rate range of 3-18% for Avastin and note
fewer patients with resistant disease. VEGF TRAP results may be diluted
by early assessment and low dose inclusion but overall are promising.
Please see the attached report for further detail and full disclosures.
<<REGNASCOWeekend.pdf>>
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I took a copy of the last 45 minutes of the panel meeting to a
biotech analyst at a brokerage firm.
He saw Pazdur's remarks to Hussein and he said that was a very strong statement about orbec's efficacy.
He asked me if he could make a copy of the DVD.
I am pretty sure he will be showing to investors
Can you tell us more about why you like CRTX so much? I know they have an approved product with a large sales force, but the pipeline seems awfully thin. I'm looking at it, but didn't know the Prospect Partners. so thanks for that.
Zyflo is a drug that needs to be taken four times a day for asthma that Abbott developed. Singular needs to be taken once a day.
Zyflo works for severe asthma singulair only works for mild to moderate.
No one is going to take the drug 4 times a day. they make fill a prescription and try to use it twice a day but it won't work
on May 31 the recieved approval for the twice a day drug and if you have severe asthma I think you would take it to avoid asthma attacks. they just signed a deal with Dey which was just bought by Mylan. Dey will have 200 salespeople co promoting the drug but Critical will recognise 65 percent of the revenues. Critical will have 40 sales people. They pay for their own salses people so the economics are good for Critical. Dey received approval on a COPD drug in Early May sot critical will also be getting sales from that. Probably enough to pay for their sales force.
I don't need a deep pipeline because the stock is cheap and I think in their severe asthma maket it can be a 100 to 200 million dollar drug.
They also have an IV version of Zileuton which is going into trials for people going to the emergency room with asthma attacks. that has a chance to be a really nice drug.
regn is cheap if vegf trap is super avastin
I don't know if it is but doctors at memorial sloan think it might be
mannkind vs novo
what is the patent situation. you should be an expert by now from the insmed days.
Critical Therapeutics is very cheap and I own a lot of it
problem is the prospect partners invested in a pipe in 2005 at over 6 dollars a share. the manager wanted a seat on the board. He was blacked out the day they recieved approval and I guess he is tired of the story. He sold over a 1.1 million shares and still owns over a million shares. I would say he has telegraphed to the market that he wants to get out of the stock so until his block is cleaned out he is now an overhang.
He should have resigned from the board last month, then he could have sold out the day of approval and he could have been out of the stock.
I would say when he is out the stock should take off. I am holding but will buy more when he is out.
Rbm has more upside than camh
I was at a meeting with the ceo of Standard Diagnostics (SDIX).
Believing that he would probably know the diagnostics industry pretty well I asked him a question about the money being spent on Biosite and the bidding war between Beckman Coulter and Inverness. The analysts are all saying that the companies want Biosite's distribution so that is the way I asked the following question.
I asked him if the reason that the companies want Biosite so badly was because they wanted Biosite's distribution? He said the only reason they wanted Biosite was because they wanted to own the rapid BNP and cardiovascular market.
Lumpy, you have been following Response Biomedical and you are aware that RBM's data puts Biosite's to shame. What will Response be worth when they will have the preferred test?
the 3 hour window with tpa is because tpa beyond 3 hours causes hemorhages and other neurotoxicities.
Desmoteplase didn't have those issues
How would spending more money on this trial have helped?
first of all the assumption they made going into the trial was that they would get a placebo rate of 25 percent because that has been the historical response.
the cerovive trial had 35 percent placebo rate and that trial ended before this one did. they should have asked the FDA to extend the amount of people the trial because maybe in the last couple of years standard of care has changed.
Paion did reperfusion studies in their prior trials that showed that the clot dissolved within 15 minutes to 30 minutes from the drug infusion.
If you are scratching you head trying to determine if the drug worked that data would have helped. Forest didn't do the reperfusion analysis to save money.
That wouldn't have changed the trial result but it would have answered the question about whether the drug is effective. If you can dissolve the clot within 15 minutes to a half hour that is the most you can hope for. No drug could do better without causing hemmoraghes
there has never been a stroke trial with a 50 percent placebo response rate
renovis had a 35 percent rate
that is what happens when you do small trials on the cheap.
you run larger trials to remove issues like this
they didn't even check reperfusion rates, which they did do in the phase 2.
Paion spent more on their phase 2 trial than Forest paid for the phase 2/3. stupidity
drbio et al re Paion:
The phase-3 results are so awful they simply can’t be whitewashed by claiming bad luck, imbalance between arms, or what have you. Not only was there no efficacy, the safety results were not great. From today’s PR:
>>
The rate of symptomatic intracranial bleeding within 72 hours after study drug administration was 0% in the placebo group, 3.5% in the 90 mcg/kg dose group and 4.5% in the 125 mcg/kg group.
<<
Although these numbers are better than the 6% bleeding risk on tPA, they have to be a big disappointment.
In spite of the half-hearted encouragement in the PR, I doubt that Desmoteplase has any future.
-when i first looked at the p/r I thought the same thing as you. The bleeding rate was actually less than I thought it would be.
9 people died from non neurological reasons more than 2 weeks after the stroke. They are treated as total failures on the stroke scale.
If you take them out, which I know you can't do for the fda I bet the response rate of the rest of the patients are in the 70 percent range.
I never considered the 90 ug dose more than a placebo and that is how it worked out in the trial. so you have 2 placebo arms with over 40 percent responses.
I think they changed something in the inclusion criteria.
stroke trials don't usually have more then 25 percent responses and that is with 3 hour windows, this went out 9 hours.
this was forest treating a pivotal trial like proof of princple
that hedge fund manager is insane
the cypress offering was 1.5 dollars discount to 17 dollars a share
bioenvision did a raise that brought the stock down from over 4 to 3.20 so that genzyme could claim that they paid a 50 percent premium.
quite a difference
from the Paion data
Ten of the 14 deaths in the 125 mcg/kg dose group were considered by the investigators as not related to the drug, 9 of which occurred 14 or more days after stroke and were from non-neurological causes.
It seems to me that with 10 deaths not related to the drug for non neurological causes it would have been pretty damn hard to get a good outcome because all these patients were consider total failures because the died.
forest did a 186 patient trial. sound cheap to me
look what they are doing with cypb. first trial failed with 880 patients. so they increase next trial to 1200. the 3rd phase 3 has 800 patients. why not do another 1200 patient trial. It doesn't make sense
the three drugs they got approved were done before they got cheap
good luck to you
I own a stock in Australia named chemgenex
Sent By: surf1944 Date: 5/31/2007 8:54:50 AM
If you like I'll update the AVNR IHUB with links/charts.
surf
surf's up......crikey
contact the ihub administrator and tell him I am no longer moderating the board and he can take away my manage rights
The next ph iii would probably come in before the FDA rules on the NDA. Logic doesn't fully hold.
Now that is the answer. I was wondering when you were going to get to that. I knew that was the scenario but don't like the fact that they weren't honest about it. They know it can't get approved on the data they have, and I think you know it also. They will file half-assed results, but if the next phase 3 works they will submit the data before the pdufa date.
Of course since forest is cheap they are only putting 800 people in the next phase 3 even though the trial that worked had 1200 people. the trial that failed had 880 people.
Now that sounds dumb to me
Find me a drug for a non-fatal disease that was submitted with two trials - one with p<0.01 and one with p between 0.05 and 0.10 - that was rejected in the last 3 years. Then I'll admit you might have a strong case.
the fda will not approve a drug that has met its primary endpoint in only one phase 3 trial for the indication of Fibromyalgia. end of story.
forest always does things the cheap way and this is another example. I believe what they want to do is have the drug rejected and when cypress gets clobbered they will buy it at a discount.
Acorda Therapeutics
Rating: Sell
Acorda Files $150mm Mixed Shelf; Reiterate Sell Rating and $15 PT
Investment Highlights:
This morning, Acorda filed a $150mm mixed shelf to sell securities, which include common and preferred stock, debt securities, warrants and units, or any combination thereof. In addition, Acorda also registered for re-sale up to 1.37mm shares of common stock from three selling stockholders - the venture capital funds Saints Capital, Cross Atlantic Partners, and Vector Fund Management, all of which are poised to sell essentially their entire ownership of ACOR shares. We also remind investors that another holder, Third Point, LLC, which had previously advocated the sale of the company to large pharma, reduced its stake in Acorda by about 59% (from 9.9% to 4.1%).As of the end of 1Q07, the company had a balance of $45.2mm in cash, cash equivalents, and short-term investments, which according to management, is sufficient through 1Q08.
Our concerns regarding lead compound Fampridine-SR. We continue to view the overall response to Fampridine-SR as unimpressive (2.54 sec average improvement in previous trial) and is, at the very least, similar to what can be achieved using non-pharmacologic methods. Additionally, we remain concerned with the safety of Fampridine-SR, as its use has been associated with an increased risk of seizures. The FDA is also requiring that the company complete a QT/QTc prolongation safety study. Given the mechanism of action of Fampridine-SR (K+ion channel blocker), we believe that there is a significant risk that the drug may prolong the QTc interval. The company plans to initiate such a study this year.
We reiterate our Sell rating and our price target of $15. As we highlighted in our last note (May 23, 2007), we continue to believe that as a result of Acorda's need to raise additional capital, combined with the lack of any meaningful catalysts for at least the next 12-months, the company's shares could face significant downside pressure.
You asked for one such drug in a non-life threatening disease in the last 3 years. Those two limitations (non-life threatening and in the last 3 years) severely limit the size of the database. Almost certainly less than 10. So the chance that one of those, say, 8, would have threaded the needle is small. Not sure what more I can say that might make it clearer why your question as stated is unrealistic.
the fact that drugs aren't approved on that basis doesn't make the question unrealistic, it makes Forest's and Cypb's statements that the drug might be approved with a failed phase 3 unrealistic.
they will need the third phase 3 data, which is still enrolling, to to be positive for the drug to be approved
arixa
I must be nuts. I looked for arixa and couldn't find it, and then went to drugs.com and couldn't find it.
I don't know why the question was unfair. Cypb and Forest are saying they will file for approval on Milnacipran with one trial that worked and one that didn't and all I asked for, was a case where a drug in a non-life threatening disease was approved under the same circumstances
dorb. Steven Kanzer. He was passionate but if they want the drug approved in some form he had to go
On May 28, 2007, Steven H. Kanzer resigned as a member of the Company’s board of directors (the “Board”). Mr. Kanzer was Vice-Chairman of the Board and a member of the audit, compensation and nominating committees of the Board. A copy of Mr. Kanzer’s letter of resignation is filed herewith as Exhibit 99.1.
Item 9.01. Financial Statements and Exhibits.
(c) Exhibits.
Exhibit No. Title
99.1 Letter of Resignation dated May 28, 2007.
SIGNATURE
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
DOR BIOPHARMA, INC.
By: /s/ Christopher J. Schaber
Name: Christopher J. Schaber
Title: Chief Executive Officer
Date: May 28, 2007
May 28, 2007
James S. Kuo M.D. MBA
Chris Schaber Ph.D.
Evan Myrianthopoulos
Board of Directors
DOR BioPharma, Inc.
1101 Brickell Avenue
Miami, FL 33131
Dear Fellow Board Members:
As you know, I have voiced my opinion that at the May 9, 2007 meeting of the Oncology Drug Advisory Committee convened by the FDA to consider the efficacy of orBec®, six (6) of the ten (10) committee members participating, including five (5) of the seven (7) committee members voting against orBec®, had in my opinion financial conflicts of interest as defined under the Federal Advisory Committee Act and the regulations thereunder relating to their imputed financial interest in the four investigational products and programs that represent the closest directly competitive products for the unmet medical need that, if approved, orBec® would be intended to treat. Attachments (1)(2)(3). Had all of the committee members having such financial conflicts of interest been appropriately excluded from participating in the meeting, as I believe they should have been, the outcome of the meeting would have been a 2-2 vote, a very different outcome.
As you are also aware, my interest as well as that of the American public in the topic of reducing financial conflicts of interest in FDA advisory committee meetings transgresses that of DOR. This topic was the subject of proposed legislation in the form of Senate Amendment 1034: To reduce financial conflict of interest in FDA Advisory Panels, that was debated and voted upon by the full Senate resulting in 47 Senators voting in favor and 47 Senators voting against on May 9, 2007.
In order that my opinion and interest in this subject not distract from the FDA’s decision as to whether to grant approval to orBec®, I hereby resign from the board of directors effective immediately and request that you file this letter with the Form 8-K disclosing my resignation as soon as practicable together with the attachments hereto as you are so required under Item 5.02(a)(2) of Form 8-K.
It has been a pleasure to serve on this board for these last 11 years. I wish you all the success in gaining approval for this life saving drug so that it may be made available to patients as well as for the future success of the company.
Sincerely,
/s/ Steve H. Kanzer
Steve H. Kanzer, CPA, Esq.
paion
you can't give them any value for enecandin because they have no data
but hopefully desmoteplase will have good data
cxs.ax cxsp adr chemgenex
Waiting in the wings
Next week, CXS will present Phase IIa clinical trial results for its
second clinical-stage programme, Quinamed, under development to
treat solid tumours. As yet we assign no value to this programme
and consider it a future partnering opportunity. Buy.
Quinamed - don't overlook second clinical stage programme
Quinamed is CXS' second clinical-stage programme under development to treat solid
tumours. The metabolism of Quinamed is affected by the patient's genotype. Through
genetic profiling, CXS is able adjust the dose patients receive for their rate of
metabolism, thereby reducing toxicity issues. A Phase IIa programme trial with
dosing based on NAT-2 genotype in prostate, breast and ovarian cancer cases that
have failed chemotherapy has been completed. Results are expected to be released
at the American Society of Clinical Oncology (ASCO) conference to be held 1-5 June
2007. If, as expected, positive results are released, a Phase IIb clinical trial is
expected to commence in 2HCY07. This is an important milestone for CXS.
Potential partnering opportunity
To determine our valuation to date we have taken the conservative approach of not
applying a value to the Quinamed programme. Following the release of the Phase IIa
results and the commencement of the Phase IIb trial, we expect to assign value to
this project. We note that it also represents a near-term partnering opportunity.
Ceflatonin - still a key focus
Lead-compound Ceflatonin is being tested in registration-directed Phase II/III clinical
trials in chronic myeloid leukaemia for patients who have developed resistance to
current treatments of Gleevec® and have one of the most common point mutations,
T315I. Interim results are expected mid-CY07. Patient recruitment is then expected
to be completed by late-CY07 with results due shortly thereafter.
Outlook - key catalysts due mid CY07, Buy recommendation maintained
We have made no changes to our forecasts and maintain our A$1.36 per share DCF
valuation on the stock (WACC 15.1%, terminal growth rate 3.0%, risk-free rate
5.75%). Our A$0.95 price target has been set at a 30% discount to our valuation to
reflect where we expect the stock will trade in six months assuming key clinical trial
results are reported as expected. Downside risks to our price target include any
delays in the progress of clinical trials.
Produced by: ABN AMRO
Better working relationship helping industry, study says
By Craig D. Rose
UNION-TRIBUNE STAFF WRITER
http://www.signonsandiego.com/news/business/20070523-9999-1b23biotech.html
May 23, 2007
A biotechnology industry survey released yesterday found that most companies believe the Food and Drug Administration has gotten better at spelling out its expectations and approving new products.
The study – which was released by Biocom, San Diego's biotech industry group, and the consulting firm PricewaterhouseCoopers – found that 81 percent of companies said the FDA was performing better since a landmark overhaul in 1997 that levied additional fees on companies to speed up product reviews.
“This study is very important because the working relationship between the life sciences industry and the FDA has a critical bearing on the health of Americans,” said Joseph Panetta, chief executive officer of Biocom. “The good news is that these relationships have greatly improved.”
The survey, which was released one day after a popular diabetes drug was alleged to raise the risk of heart attacks, also found that most companies see the monitoring of drugs after they reach the market as a key issue.
About half the companies said the FDA and the industry should do more to monitor the effects of drugs already approved for sale, which the industry calls pharmacovigilance, on the consumers who take them.
The survey polled about 70 companies nationwide and sought a representative sample of employment levels and revenue.
The survey found that 73 percent said the FDA's improved guidance had led to a better understanding of the regulator's expectations.
But many companies said the FDA had changed positions during their drug or device reviews, which the industry says can delay the review process. And 61 percent of the companies agreed or strongly agreed that personnel changes at the FDA resulted in a break of continuity for their product reviews.
Half the companies said that target schedules for reviews had caused the FDA to reject products because the staff ran out of time to resolve issues.
The survey comes as the FDA is drawing criticism for approving drugs that turn out to be dangerous.
On Monday, the New England Journal of Medicine reported a study alleging that Avandia, an FDA-approved diabetes drug, increases the risk of cardiovascular death by 64 percent.
In recent years the painkiller Vioxx and the diabetes drug Rezulin, among other drugs, have been pulled from the market because they were implicated in causing harm to patients or death.
In response, the Senate and House have separately passed drug safety bills and are in the process of reconciling the two. The Senate bill would double the number of government scientists to monitor risky side effects from drugs already approved for sale.
Dr. Sidney Wolfe, who monitors the industry for Public Citizen, a Washington-based watchdog group, said the FDA's performance in protecting public health over the past decade has gotten “significantly worse.”
He blamed an increasing reliance on funding the FDA from the industry and a decline in congressional oversight of the regulator. More than half the FDA's drug approval budget is now coming from the industry, Wolfe said.
“The same Congress that has abdicated funding of the FDA to industry has not done a fraction of the oversight that it used to do,” said Wolfe, who is a physician. “The FDA is getting closer and closer to the industry.”
Wolfe also said turnover at the FDA had increased because scientists and doctors had left because their professional judgments are too often overruled by superiors in what he said was an agency that increasingly “kowtowed” to industry.
Panetta of Biocom also said the increased reliance on funding from industry fees was a problem. But he said FDA turnover was more likely a function of a high pressure work atmosphere and limited resources.
Panetta said public attitudes about approved drugs also need adjustment.
“Part of the breakdown in public confidence is due to the fact that there was and is a lack of understanding that all drugs carry a certain level of risk,” he said.
“Along with the benefits, there is also going to be the potential for side effects. But the survey found industry overwhelmingly said we need to increase pharmacovigilance.”
--------------------------------------------------------------------------------
Craig Rose: (619) 293-1814; craig.rose@uniontrib.com
Hospital-borne ailments face Medicare budget ax
Feds consider ending payment for common medical errors
By Daniel Lee
daniel.lee@indystar.com
May 22, 2007
http://www.indystar.com/apps/pbcs.dll/article?AID=2007705220351&template=printart
Medicare says it might no longer pay for many of the mistakes made by hospitals.
Late next year Medicare plans to stop paying hospitals for costs incurred from some of the most common and preventable medical errors suffered by patients.
It could be getting a bloodstream infection from a caretaker not thoroughly washing his or her hands. Or it could be developing a dangerous bed sore because a patient's skin was not inspected or cleaned as recommended.
The proposed change could have a far-reaching impact because Medicare is the largest single payer of medical bills in the nation. Commercial health insurers, such as Anthem Blue Cross and Blue Shield, often follow Medicare's lead when determining their own reimbursement policies.
"It is something we will want to look at and try to follow," said Eric Schmitz, director of provider contracting for Anthem. However, he added that Anthem is waiting to see how the final policy looks before making any decisions.
The federal Centers for Medicare and Medicaid Services, which operates Medicare, is taking public comment on the proposal through June. The changes are scheduled to take effect in October 2008.
Medical mistakes are deadly and expensive. Infections acquired in hospitals account for about 90,000 deaths and $4.5 billion in extra spending each year, according to the U.S. Centers for Disease Control and Prevention.
Preventing such problems is an ongoing effort among hospitals, doctors and organizations focused on health-care quality.
The Indiana Patient Safety Center was formed last year to help hospitals develop reliable systems to prevent harm to patients. Indiana also has a mandatory medical-error reporting system requiring that hospitals disclose certain mistakes, such as objects left inside a patient during surgery.
But unlike many other efforts, Medicare's proposal holds the potential to hit hospitals in their pocketbooks by changing the way they are paid for providing care.
Under the change, hospitals no longer would receive payment for costs stemming from certain complications if those conditions did not exist when the patient was admitted into the hospital.
Medicare is looking at 13 specific conditions that include everything from urinary-tract infections related to catheters to a patient receiving an incompatible blood type during a transfusion.
The CDC reports more than 561,600 catheter-associated urinary tract infections a year, according to Medicare's proposal. In 2006, 11,780 Medicare patients had the infection as a secondary diagnosis during a hospital stay.
According to Medicare, those infections add an estimated $676 to a hospital bill.
Some hospital-acquired conditions can be much more expensive.
The cost of treating pressure ulcers, or bed sores, can run anywhere from $500 to $40,000 a patient, according to the Institute for Healthcare Improvement. In 2006, 322,946 Medicare patients had a pressure ulcer as a secondary diagnosis.
St. Vincent Health, which operates hospitals across Indiana, said the proposed Medicare changes are in line with other initiatives to improve patient safety.
"We don't look at it from the negative side," said St. Vincent spokesman Johnny Smith.
The American Hospital Association, which represents almost 5,000 hospitals and other health-care providers, said it welcomes efforts by Medicare and others to reduce errors and improve health-care quality.
"There's not a doctor or nurse who comes to work in the morning and wants to make a mistake," said Carmela Coyle, the AHA's senior vice president of policy. "I don't see nonpayment as an incentive to try to do better."
She also said it is important to make sure that the conditions included in the Medicare policy change are indeed preventable and in control of the hospital.
stroke article from the NY Times
Many hospitals say they cannot afford to have neurologists on call to diagnose strokes, and cannot afford to have M.R.I. scanners, the most accurate way to diagnose strokes, for the emergency room.
most hospitals have perfusion CT which can give the same diagnosis. As far as a neurologist on call they could transmit the image over the internet to a neurologist at another hospital. The reading of the image doesn't have to be in the same hospital.
Although tPA was shown in 1996 to save lives and prevent brain damage, and although the drug could help half of all stroke patients, only 3 percent to 4 percent receive it. Most patients, denying or failing to appreciate their symptoms, wait too long to seek help — tPA must be given within three hours. And even when patients call 911 promptly, most hospitals, often uncertain about stroke diagnoses, do not provide the drug.
Demoteplase is being studied for up to nine hours after the stroke but time isn't as important as penumbra. If someone has a penumbra 15 hours after the stroke the drug could still be used. TPA actually causes neurotoxicty which is why it is acutally harmful beyond 3 hours and causes hemmorhage.
The drug had a serious side effect — it could cause potentially life-threatening bleeding in the brain in about 6 percent of patients. But the clinical trial demonstrated that the drug’s benefits outweighed its risks.
That 6 percent is only in the first 3 hours. If the drug is given later the percent goes up
There is a way to diagnose strokes more accurately — with a diffusion M.R.I., a type of scan that shows water moving in the brain. During a stroke, the flow of water slows to a crawl as dead and dying cells swell. In one recent study, diffusion M.R.I. scans found five times as many strokes as CT scans, with twice the accuracy. A diffusion M.R.I. “answers the question 95 percent of the time," Dr. Sorensen said.
this probably has more to do with the readers of the scans than the scans themselves. this is very sad if the scan readers do such a poor job. Maybe they were regular CT scans without the proper imaging software. It is hard to tell because the article doesn't go into why this happens and what could be done to correct it.
It is simply not practical to demand the scans at every hospital or even every stroke center, said Dr. Edward C. Jauch, an emergency medicine doctor at the University of Cincinnati and a member of the Greater Cincinnati/Northern Kentucky Stroke Team.
“If you made M.R.I. the standard of care before giving tPA, most centers would not be able to comply,” Dr. Jauch said. And if it takes more time to get a scan — as it often does — it might be better to forgo it and give tPA immediately if the patient’s symptoms seem unambiguous.
This is true because of the 3 hour window and most people will not be helped by the MRI diagnosis. Once the 3 hour window is increased to 9 hours or more the excuse about not having an MRI should go away. The difference would be people dying or living in a wheelchair for 30 or 40 years that otherwise would have been able to care for themselves.
It has still not been shown, though, that M.R.I. scans actually improve outcomes. It might depend on the circumstances and the hospital, said Dr. Walter J. Koroshetz, deputy director of the National Institute of Neurological Disorders and Stroke.
But some who use M.R.I. scans, and who have studied them in research, say the system has to change. They say enough is known about the scans to advocate having them at every major medical center that will treat stroke patients.
“All these problems could be solved if there was a will to do it,” Dr. Sorensen said. In his opinion, it comes down to old and outdated assumptions that there is not much to be done for a stroke, to financial considerations and to a medical system that resists change. But the most significant barriers, he said, are financial.
the biggest thing that needs to be changed is increasing the window passed 3 hours
When she arrived, Dr. Grotta asked if she was sure she wanted the drug. Did she want to risk bleeding in the brain? Dr. Fite did not hesitate. The stroke, she said, “was just so devastating that I would rather die of a hemorrhage in the brain than be left completely paralyzed in my right side.”
“In my horrible voice, I said, ‘Yes, I want the tPA,’ ” Dr. Fite said.
Within 10 to 15 minutes, the drug started to dissolve the clot.
“I had weird spasms as nerves started to work again,” Dr. Fite said. “An arm would draw up real quick, a leg would tighten up. It hurt so bad I was crying because of the pain. But it was movement, and I knew something was going on.”
She was lucky because she was a doctor and knew where to have the ambulance bring her. She was treated within 3 hours. Aside from the reduction of neurotoxicity of Desmoteplase vs. TPA the other advantage of Desmoteplase is it is 150 to 200 times more specific to fibrin than TPA. This allows less drug to be used causing less side effects.
We will know June 1st whether it worked in the phase 3 trial the way it worked in the earlier trials.
Researchers find big batch of breast cancer genes
By Maggie Fox, Health and Science Editor
WASHINGTON, May 27 (Reuters) - A genetic mutation that raises the risk of breast cancer is found in up to 60 percent of U.S. women, making it the first truly common breast cancer susceptibility gene, researchers reported on Sunday.
Reports from several teams around the world identified changes in four other genes that raise the risk of breast cancer significantly. Several are found in many men and women.
More than 60 percent of the women in the United States probably carry at least one of the mutations in one of the genes, called FGFR2, the researchers said.
"This is a truly landmark breakthrough for breast cancer research, because these genes are the first confirmed common genetic risk factors for breast cancer," said Jianjun Liu of the Genome Institute of Singapore, who took part in one of the studies.
The researchers, reporting in the journals Nature and Nature Genetics, said the discoveries are the most important genes associated with breast cancer since BRCA1 and BRCA2 were identified.
Women with faulty copies of BRCA1 or BRCA2 have a 50 percent to 85 percent chance of getting breast cancer in their lifetimes. But they are rare genes, and only account for 5 percent to possibly 10 percent of breast cancer cases.
Researchers have been testing women for other genes associated with breast cancer, to find its causes, to understand how and why it develops, and to make more effective treatments. Reuters 2007. All Rights Reserved.
Better techniques to analyze DNA, and the publication of the human genome, the map of all DNA in the body, have made this a much faster and easier process.
David Hunter of Harvard University and a team at the U.S. National Cancer Institute looked at more than 2,200 women of European ancestry.
COMMON GENE, BIG RISK
They found four common mutations in FGFR2 associated with the breast cancer in women after menopause who do not have known relatives with breast cancer.
The mutations raise the risk of breast cancer risk by 20 percent if they carry one copy of the gene and by 60 percent if they carry two copies. And close to 60 percent of the women they studied carried at least one copy.
The findings do not yet have any real relevance for women, Hunter stressed.
"It is premature to recommend screening women for these gene variants, at least until the scientific community has further combed through the genome-wide findings and found all the variants that are associated with increased risk," Hunter said in a statement.
Douglas Easton of Britain's University of Cambridge led a team of researchers around the world to look at tiny changes in the DNA code called single nucleotide polymorphisms or SNPs -- pronounced "snips" -- in the DNA of 21,860 people with breast cancer and 22,578 people without it.
They found mutations in four genes that were more common in the people with breast cancer -- FGFR2, TNRC9, MAP3K1 and LSP1.
FGFR2 may be a logical candidate for a breast cancer gene -- it is a receptor, a kind of molecular doorway, for compound called tyrosine kinase which is involved in several cancers.
In a third study, a team at deCODE genetics (DCGN.O: Quote, Profile , Research), the University of Nijmegen in the Netherlands and elsewhere studied 22,000 people to find two other gene mutations associated with breast cancer. One is also near TNRC9.
"DeCODE estimates that these two variants are contributing factors in one quarter of breast cancer cases in women of European origin," the company wrote in a statement.
Breast cancer kills 500,000 people a year globally according to the World Health Organization, and 1.2 million men and women are diagnosed with it every year.
© Reuters 2007. All Rights Reserved
A sure thing is all relative - but I'd give odds of pain label approval based upon the current trial data at greater than 95%. But if you feel there is something fishy, feel free to go short -g-.
I never said you implied that approval was a sure thing, I just asked you what made you or the company so sure it could get approved based on one trial that met the endpoint and one trial that missed.
Can you show me one other time in the last 3 years where the fda approved a drug with a missed phase 3 in a non-life threatening disease.
If you provide me with that I may not be so skeptical, not that my skepticism matters.
cypb------They do have to execute anothor phase 3 trial before seeking approval correct?
a) Pain Label - almost certainly not. They just barely missed the pain composite endpoint on the first ph 3 and were very stat sig on the one that just finished
This is not horseshoes. Barely missed is a miss. This is not a life threatening disease where one phase three could be sufficient. This is pain which is caused by ... that's right, no one knows.
I am unclear how they or you, can be so sure they can get the drug approved on one trial that worked. Something doesn't sound right to me.
paion
I own it. The way the drug works makes it better than TPA.
I guess we will know on June 1st
Pipex Pharmaceuticals Announces Presentation of Phase I/II Clinical Trial Results of COPREXA (Oral Tetrathiomolybdate) for the Treatment of Refractory Idiopathic Pulmonary Fibrosis (IPF)
9 minutes ago - Market Wire
Pipex Pharmaceuticals, Inc. (OTCBB: PPEX), a specialty pharmaceutical company developing innovative late-stage drug candidates for the treatment of neurologic and fibrotic diseases, announced today that the results of an open-label phase I/II clinical trial of COPREXA (oral tetrathiomolybdate) for the treatment of refractory Idiopathic Pulmonary Fibrosis (IPF) were presented at the American Thoracic Society (ATS) 2007 Annual Meeting in San Francisco by Kevin R. Flaherty, M.D., M.S., Associate Professor of Pulmonology at the University of Michigan. IPF is a fatal disorder with no FDA approved or effective therapy. This clinical trial was partially supported by a grant from the Coalition for Pulmonary Fibrosis, a non-profit organization.
Dr. Kevin Flaherty, the principal investigator of the clinical trial, stated, "Patients with IPF are in dire need for new potential effective therapies. COPREXA was easy to administer and well tolerated in a group of patients with progressive IPF, refractory to standard therapy. The pre-clinical work and patient tolerance of COPREXA makes us optimistic about this new treatment approach for IPF. We look forward to conducting a multi-center, double-blind, placebo controlled study of this agent."
COPREXA has completed two pivotal phase III trials for the treatment of neurologic Wilson's disease, an orphan genetic neurologic disease, and Pipex is preparing to file a New Drug Application (NDA) with the FDA for the indication of initially presenting neurologic Wilson's disease.
Clinical Trial Design
In a single-center, open-label, phase I/II clinical trial, 20 patients with IPF that had evidence of disease progression despite treatment with prednisone +/- cytotoxic therapy were treated for one (1) year with COPREXA (oral tetrathiomolybdate) 20mg capsules per oral twice a day with meals plus 20-40mg per oral at bedtime and adjusted as necessary to maintain a targeted serum ceruloplasmin range of 5-15mg/dl.
Patients were monitored for adverse events, change in pulmonary function as measured by percent predicted forced vital capacity (FVC), diffusing capacity of the lung for carbon monoxide (DLco) and walk distance (six minute walk test room air). When pre-treatment FVC values were available, the course of pre-treatment FVC (slope) was compared to the post-treatment FVC (slope) at 6 and 12 months.
The baseline characteristics of the patients entered into the clinical trial were as follows:
Variable mean + sd -------- --------- Age (years) 69 +/- 7.1 Onset symptoms (years) 3.3 +/- 2.4 Smoker (yes/no/NA) 16/2/2 Pack*years 42.1 +/- 19.5 FVC (% predicted) 62 +/- 12 DLco (% predicted) 39 +/- 11 FVC >/- 55% predicted (yes/no) 15/5 DLco >/- 35% predicted (yes/no) 11/9 SaO2 < 88% during 6MWT (yes/no) 13/7
Clinical Trial Results
Patients responses at one (1) year were as follows:
Patients Patients Patients Stable Improved Progressed Forced Vital Capacity (FVC) 60% (12/20) 10% (2/20) 30% (6/20) Diffusing capacity of the lung for carbon monoxide (DLco) 75% (15/20) 5% (1/20) 20% (4/20)
Definitions: Stable = Change in FVC less than 10%, DLCO less than 15%; Improved = Relative increase in FVC at least 10%, DLCO at least 15%; Progressed = Relative decrease in FVC at least 10%, DLCO at least 15%.
When pre-treatment forced vital capacity (FVC) values were available, the course of pre-treatment FVC (slope) was compared to the post-treatment FVC at 6 and 12 months. The change in the pre-treatment and post treatment FVC slopes demonstrated favorable trends in the rate of decline of FVC, at 6 months pre-treatment compared to 6 months post-treatment (p < 0.06) and at 12 months pre-treatment compared to 12 months post-treatment (p < 0.12).
High Resolution Computed Tomography (HRCT) was utilized in the study to reveal images of the lungs. A semi-quantitative approach to measuring interstitial inflammation (HRCT ground glass opacity pattern) and the extent of lung fibrosis (HRCT honeycomb) was utilized. The HRCT results were as follows:
High Resolution Baseline COPREXA Therapy P value Computed @ 12 month Tomography (HRCT) Ground Glass 1.31 + 0.81 1.03 + 0.67 0.02 Fibrosis 1.61 + 0.48 1.67 + 0.50 0.60
The results of the six minute distance walk test while on COPREXA at baseline and at (1) year for the patients that completed either or both of the walk tests were as follows:
Distance (feet) P value Baseline (n=15) 611 + 388 12 month (n=10) 735 + 461 Difference completed either (n=16) -123 + 477 0.32 completed both (n = 9) 86 + 426 0.56
The St. George's Respiratory Questionnaire (SGRQ) a standardized quantitative quality of life (QOL) measurement commonly utilized for lung disorder clinical trials showed a trend towards improvement, as follows:
Value P value Symptom 3.55 0.45 Impact -5.22 0.07 Activity -6.43 0.41 Total -4.41 0.17
During the study, patients were also monitored for adverse events. Dose-related nausea and cytopenias were the most common and expected side effects of COPREXA therapy. These side effects were reversible with drug holiday. Three patients did not complete the trial (patient preference n=2, anemia n=1), and 2 patients died during the trial, 1 of exacerbation/pneumonia at month 1 and 1 of IPF progression at month 6.
Overall, the results of this small pilot trial are believed to be encouraging given the requirement for progressive disease to enter the trial. These preliminary data give us enthusiasm for a larger placebo-controlled trial for patients with IPF to confirm that treatment with COPREXA will help stabilize the course of IPF. Later this year, Pipex plans to initiate a twelve month, multi-center, randomized, double-blind, placebo-controlled trial of COPREXA in IPF. A manuscript that further details the clinical trial and its results is in preparation and is expected to be submitted and published in a leading peer reviewed journal.
Preclinical Data of COPREXA in Animal Models in IPF
The scientific rationale for conducting this clinical trial was based on several published preclinical studies indicating that COPREXA favorably modulates the fibrotic response in numerous animal models including the well established bleomycin mouse model of pulmonary fibrosis. GJ Brewer et at, Inorg Biochem. 2004 Dec;98(12):2160-7 and GJ Brewer et al, J. Lab. Clin. Med. 2003, 141:210-6.
In these published studies, COPREXA demonstrated the ability to inhibit fibrosis in a number of well established animal models which is believed to be based upon its ability to sequester endogenous free copper and inhibit key fibrotric cytokines, including secreted protein acid rich in cysteine (SPARC), NF(Kappa)B, TGF-(Beta), FGF-2, IL-1, IL-6, IL-8 and connective tissue growth factor (CTGF).
In these preclinical models, COPREXA prevented inflammation and the fibrotic response of the lung to bleomycin. Bleomycin is an FDA approved agent which is known to cause lung fibrosis in humans. According to Dr. George Brewer, Professor Emeritus of Human Genetics at the University of Michigan, inventor of this utility for COPREXA and co-author of the clinical trial, "An effective treatment for IPF is a key medical goal since the outcome of patients is grim and the prevalence of this disease in the population is increasing. Our observations that COPREXA significantly inhibited the fibrotic pathway has led us toward a new potential paradigm for the treatment of this deadly disease. We are very excited about the potential of bringing this new potential oral therapy to the 124,000 American patients afflicted with IPF."
Acknowledgements
Kevin R Flaherty, M.D., M.S. acknowledged the assistance of the following collaborators, Douglas A. Arenberg, M.D., Eric S. White, M.D., Victor Thannickal, M.D., Adin-Cristian Andrei, Ph.D.**, Susan Murray, Sc.D.**, Thomas V. Colby, M.D.*, William D. Travis, M.D.+, Ella A. Kazerooni, M.D., Barry H. Gross, M.D, Robert Paine III, M.D., Galen B. Toews, M.D., George J. Brewer, M.D. and Fernando J. Martinez, M.D., M.S. of the University of Michigan Health System, Ann Arbor, MI; Mayo Clinic, Scottsdale, AZ (*), Memorial Sloan Kettering, New York, NY (+) and the Department of Biostatistics, University of Michigan School of Public Health (**).
About IPF
IPF is a fatal respiratory disease characterized by progressive loss of lung function due to extensive fibrosis of lung tissues that are essential for respiration and life. According to the Pulmonary Fibrosis Foundation and Coalition for Pulmonary Fibrosis, IPF affects an estimated 124,000 patients in the U.S., including 48,000 new patients per year, and results in approximately 30,000 deaths in the U.S. annually. This represents more deaths annually than deaths from either breast or prostate cancer.
Currently, there are no FDA approved therapies for IPF. Standard of care for IPF patients are high dose corticosteroids and immunosuppresants which have numerous side effects that increase patient morbidity.
About COPREXA
COPREXA is an oral, small-molecule, anti-copper agent that is highly specific for the reduction of free copper in serum, the most toxic form of copper in the body, and is thus ideally suited for the treatment of central nervous system (CNS) diseases in which abnormal serum and CNS copper homeostasis are implicated. COPREXA has completed pivotal clinical trials for the treatment of neurologic Wilson's disease for which we are planning to file a New Drug Application (NDA). We are also developing COPREXA for fibrotic disorders based upon the rationale that the fibrotic disease process is dependent upon the availability of free copper in the body. COPREXA has demonstrated the ability to inhibit fibrosis in a number of well established animal models through the sequestration of available copper and inhibition of key fibrotric cytokines, including secreted protein acid rich in cysteine (SPARC), NF(Kappa)B, TGF-(Beta), FGF-2, IL-1, IL-6, IL-8, and connective tissue growth factor (CTGF).
As such, COPREXA is also in a phase II clinical trial for the treatment of primary biliary cirrhosis (PBC), a fibrotic disease of the hepatic system.
About Pipex Pharmaceuticals, Inc.
Pipex Pharmaceuticals, Inc. ("Pipex") is a specialty pharmaceutical company that is developing proprietary, late-stage drug candidates for the treatment of neurologic and fibrotic diseases Pipex's strategy is to exclusively in-license proprietary, clinical-stage drug candidates and complete the further clinical testing, manufacturing and regulatory requirements sufficient to seek marketing authorizations via the filing of New Drug Applications (NDAs) with the FDA in the U.S. and Marketing Application Authorizations (MAAs) with the European Medicines Evaluation Agency (EMEA). For further information, please visit, www.pipexpharma.com.
This press release contains forward-looking statements, within the meaning of Section 21E of the Securities Exchange Act of 1934, that reflect Pipex Pharmaceuticals, Inc. and Pipex Therapeutics, Inc. ("we" or "our") current expectations about its future results, performance, prospects and opportunities, including statements regarding the potential use of COPREXA(TM) for the treatment of Idiopathic Pulmonary Fibrosis, Wilson's disease, inflammatory and fibrotic diseases, as well as its the prospects for regulatory filings in the treatment of neurologic Wilson's disease. Where possible, the Company has tried to identify these forward-looking statements by using words such as "anticipates," "believes," "intends," or similar expressions. These statements are subject to a number of risks, uncertainties and other factors that could cause actual events or results in future periods to differ materially from what is expressed in, or implied by, these statements. We cannot assure you that we will be able to successfully develop or commercialize products based on our technologies, including COPREXA(TM), TRIMESTA(TM), SOLOVAX(TM), EFFIRMA(TM) or Anti-CD4 802-2, particularly in light of the significant uncertainty inherent in developing, manufacturing and conducting preclinical and clinical trials of new pharmaceuticals, and obtaining regulatory approvals, that our technologies will prove to be safe and effective, that our cash expenditures will not exceed projected levels, that we will be able to obtain future financing or funds when needed, that product development and commercialization efforts will not be reduced or discontinued due to difficulties or delays in clinical trials or due to lack of progress or positive results from research and development efforts, that we will be able to successfully obtain any further grants and awards, maintain our existing grants which are subject to performance, that we will be able to patent, register or protect our technology from challenge and products from competition or maintain or expand our license agreements with our current licensors, or that our business strategy will be successful. All forward-looking statements made in this press release are made as of the date hereof, and the Company assumes no obligation to update the forward-looking statements included in this news release whether as a result of new information, future events, or otherwise. The forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those set forth or implied by any forward-looking statements.
For Further Information Contact: Steve H. Kanzer, CPA, Esq. Chairman and Chief Executive Officer (734) 332-7800 or Thomas Redington (Investor Relations) Redington, Inc. Tel: (203) 222-7399
SOURCE: Pipex Pharmaceuticals Inc.