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The drug industry is threatening to end its support for President Barack Obama's health overhaul effort
so they are admitting that they supported a bill based on one part of the bill, which only benefits their industry, even though the bill as a whole is not good for 90 percent of the country's citizens.
It is pretty clear that at this point, except for bladerunner, the only people that support the bill have been bribed to do so.
the unions supported the bill until their member insurance plans would be taxed, then they would not support the bill. So they get bribed that their cadillac plans would be exempt from the tax so now they support the bill.
who will be bribed next, and what group will see that bribe and say, if you are giving them that deal, I want the same deal.
These people should all be in jail.
another pump and dump from Garza
NWCI "Buyout in the Works" After Company is Awarded Key Patent PDF | Print | E-mail
Written by M.E.Garza
Thursday, 14 January 2010 23:49
Another buyout rumor is engulfing the biotech sector. This one comes on the heels of Thursday morning's key patent news announcement for small cap, NewCardio (NWCI.OB).
This Silicon Valley based company with a very small share float wanted "very badly" to meet with us for a one-on-one interview session at the OneMedForum conference in San Francico. Suddenly, they dropped off the face of the earth and no one could be reached for comments, much less an interview.
Why? What happened?
On the last day of the conference, rumors began to fly that the company was in deep negotiations for a buy out. After chasing the story for the last 24 hours, and seeing the key patent announcement hit the news wires on Thursday morning, it is my opinion that if the buyout occurs, it will likely send shares of NewCardio somewhere between $10 and $14.
Newcardio, Inc.
1.75 +0.22 (+14.38%)
Intraday | 3 Month | 6 Month | 1 Year
Quotes delayed at least 20 mins.The company has a hot new technology that is shaping up to be not only the next great diagnostics tool in cardiovascular medicine, but could also turn into "the ultimate medical phone app." There is talk about a hardware/software combo that would allow anyone to hold a smart phone-like device up to their chest in order to interpret/communicate the readings directly to a doctor.
Designers of this technology (who come from a tech background designing chips and microprocessors) have figured out that a great number of doctors simply do not know how to interpret heart monitor signals either correctly or accurately. They feel that EKG technology is "under utilized" as a diagnostics tool and see a multi-billion dollar market for their products.
Thursday morning, the company announced news about a key patent (which needed to be in place before the rumored sale) and the news sent shares flying.
If the rumors of the buyout are true, (sources confirmed to BioMedReports on Thursday afternoon that discussions are definitely underway) then shares could easily continue to climb from Thursday's already unusual high trading/volume levels.
According to an upbeat presentation at the OneMedForum in San Francisco earlier this week, NewCardio is said to be entering the commercial phase as a cardiac diagnostic and services company.
The company's proprietary software platform technologies provide higher accuracy to, and increase the value of, the standard 12-lead electrocardiogram (ECG). NewCardio's three-dimensional ECG software platform reduces the time and expense involved in assessing cardiac status while increasing the ability to diagnose clinically significant conditions which were previously difficult to detect. NewCardio's software products and services significantly improve the diagnosis and monitoring of cardiovascular disease, as well as cardiac safety assessment of drugs under development.
The video below is from the OneMedForum presentation which took place 3 days ago.
cycc should have raised more if they could. If they run trials their burn will go up. this doesn't do much for them
Soligenix mentioned on seeking alpha
http://seekingalpha.com/article/180656-biotechs-2009-stealth-small-cap-rally
Gastrointestinal Disease: 3 Hits, 3 Misses
First, the good:
Both Salix Pharmaceuticals, Inc. (SLXP) and Santarus, Inc. (SNTS) appear in the list of top ten biotechnology gainers for 2009 with triple-digit returns due to favorable regulatory progress reported during the year [see Table 1]. In September, Salix announced the successful outcome of two Phase 3 trials to evaluate the efficacy and safety of Xifaxan® [rifaximin] for the treatment of non-constipation irritable bowel syndrome. Salix is planning an NDA submission for the first half of 2010. In December, Santarus announced that the FDA approved the company’s New Drug Application [NDA] for its prescription tablet product for all of the indications being sought, including for the treatment of heartburn and other symptoms associated with gastroesophageal reflux disease.
While not a member of either major biotechnology index, shares of Soligenix, Inc. (SNGX.OB) increased 317% during 2009. In January, the company reached agreement with the FDA on the design of a confirmatory, pivotal Phase 3 clinical trial evaluating its lead product orBec® for the treatment of acute gastrointestinal Graft-versus-Host Disease [GVHD]. The following month, Soligenix announced a potential $30 million North American partnership agreement with Sigma-Tau Pharmaceuticals for orBec and in October 2009 initiated patient enrollment in the confirmatory Phase 3 trial that is expected to complete with clinical data available in the first half of 2011.
Next, the bad:
As discussed in our December 2009 article “Graft Versus Host Disease: Failures and Future Opportunities,” Osiris Therapeutics, Inc. (OSIR) recently reported preliminary results from two Phase 3 trials evaluating its Prochymal product candidate for the treatment of acute GVHD. Unfortunately, neither trial reached its primary endpoint, sending shares from $14 to a 52-week low of $5.35 by November 2009, earning the company a spot in the top ten decliners for the year [see Table 2].
cycc has been moved by the same group that moved snss to 3 dollars before it came down
it doesn't matter that it is worth 3 it won't stay up here
all the momo guys caught holding the bag will force it down
cgen was a dollar in June.
I don't think any of those shareholder now are complaining.
if they still own it
If you own it now then you should consider that management should be looking to price a deal as fast as they can
20 tons of safflower is not tons of commercial grade product
Thus, even if another company can make the drug better or cheaper, they’ll have to cut a deal with MDCO unless they have their own set of clinical data.
I guess the point that I am trying to make is that mdco is not really much further away from getting the drug approved than anyone else. In fact they could start trials and have manufacturing problems and could fall behind a company with a better, easier process
Then can make it and probably others. I wouldn't buy mdco for the milano drug because they are no further ahead then anyone else that can make it.
look for companies that will be able to produce apo milano efficiently and not owe pfizer anything
Each infusion of the product needs 5 or six grams of protein and the protein is very difficult to separate from the endotoxins used in the e coli process. I am sure when Pfizer bought Esperion they didn't realize the difficulties that they encountered.
Usually when a company undergoes a study they say how many people will I have to treat for what period of time, to show efficacy. Esperion said how many vials of Milano do I have, and what is the minimum number of people can I treat in the shortest amount of time to perhaps a get a signal, with the number of vials we have in our refrigerator because we can't make any more.
That is why there was such amazement that the study worked.
This isn't an orphan disease that they will be able to charge 400 thousand a person, like Alexion. The cost to produce the protein will have to make economic sense.
apoA1 Milano
I have always felt that is a drug that can change cardiovascular care. I just do not believe that Pfizer had made strides in the manufacturing, and that is what has always been the issue.
The company that can manufacture it efficiently and at a reasonable cost will have a blockbuster.
In that case insurance rates will skyrocket because I won't be the only one smart enough to figure this out. So only the sickest people will have a insurance policy.
People take out insurance "in case" a catastrophic event happens. In this case you will only have insurance if you are hit with a catastrophic event. This is insane.
but we already knew that
INSURANCE INDUSTRY RESTRICTIONS: Starting in 2014: no denial of coverage based on pre-existing conditions. No higher premiums allowed for pre-existing conditions or gender. Limits on higher premiums based on age and family size. Starting upon enactment of legislation: children up to age 26 can stay on parents insurance; no lifetime limits on coverage.
does this mean that in 2013 I can pay the fine and save 20 thousand in medical insurance because in 2014 I won't be denied insurance. and not pay for years until someone in the family gets sick?
Biotech scams
I believe Stephen Kanzer did not set up Pipex as a scam. He believed that the drug for Wilson's disease would be approved. Unlike the CEO's of biotech scams he lost millions of dollars buying the stock on the open market before the NDA filing.
So as far as shareholders are concerned, a loss is a loss, but I would put Kanzer's efforts as gross mismanagement more than a scam. He depended on advice of consultants
Novartis Shifts Focus to Rare Diseases
CEO Vasella Sees Value in Niche Drugs That Can Have Broader .By JEANNE WHALEN
Like most pharmaceutical companies' CEOs, Daniel Vasella, chairman and chief executive of Switzerland's Novartis AG, is facing a large cocktail of problems.
Insurers, governments and others who pay for health care—called "payers" in the industry lingo—are increasingly refusing to cover expensive new drugs that aren't substantially better than older, cheaper treatments. Regulators have become more safety conscious and less willing to approve new products for sale. And through a series of large mergers, pharmaceuticals giants have found bureaucracy creeping in and hampering their development of new drugs.
But there are still a number of opportunities, and Dr. Vasella says he is trying to reposition Novartis to capture them. Advances in genetics are making it more possible to decipher and fix the biological flaws that cause disease. And there are still plenty of difficult diseases that badly need treatments, such as Alzheimer's and multiple sclerosis.
"There are lots of areas where there is a very clear need" for innovation, he said in an interview.
Dr. Vasella became the head of Novartis when it was formed through the 1996 merger of Sandoz and Ciba-Geigy. Early in his career he was a physician in his native Switzerland, but he joined Sandoz when his wife's uncle was the company's chairman and rose quickly through the ranks. Dr. Vasella says he made the switch to business because he believed his work could benefit "not one or 100 but thousands" of patients.
In past interviews he has also said he prefers business to medicine because he can be more openly aggressive. Asked about this, he said aggression "is something which is not only legitimate, but necessary to compete successfully in the marketplace."
Novartis is now one of the world's biggest drug companies, known for multibillion-dollar drugs such as Diovan for blood pressure and Gleevec for cancer.
Dr. Vasella has carried out a wide overhaul of Novartis's drug research in an effort to pump out better drugs. In 2002, he hired a genetics expert from Harvard—Mark Fishman—to run the company's research from a new set of labs in Cambridge, Mass. Their mantra: Focus on diseases that truly need treatments, and whose biological causes can be decoded and fixed.
There have been some initial signs of success, Dr. Vasella says. "We have increased substantially...the number of compounds in the pipeline, and the number of positive proof-of-concept trials in man," he says, referring to the small human studies that first show whether a drug is working.
The first drug to emerge from this new research approach was recently approved for sale in the U.S. Called Ilaris, it treats a rare inflammatory disease affecting only a few thousand people world-wide. Novartis focused on this disease because its genetic causes were clear, making it feasible to develop a drug. The same genetic triggers may underlie other inflammatory diseases, and Novartis is testing the drug in these disorders, as well.
"We've developed [the drug] in a very targeted way and are now expanding into additional areas," a model the company intends to employ with other drugs, too, Dr. Vasella says. But the jury is still out on whether the drug will work in other diseases. It's also still unclear how many other drugs from Dr. Fishman's labs will make it to market, because it can take 10 or more years to develop a new treatment.
Developing drugs for rare diseases—and attempting to expand them to larger ones with similar genetic triggers—marks a departure from the way Novartis used to develop drugs. In years past, Novartis and most drug makers tried to develop treatments for common diseases that offered a vast market of patients: hypertension, depression and cholesterol, for instance. It didn't matter whether the new drug was much better than older treatments, because health-care payers—particularly in the U.S.—would pay for most new drugs the industry launched, and hefty marketing campaigns drove demand. These "me-too drugs" generated "quite good returns" for the industry for years, Dr. Vasella says.
But that approach is faltering. Financially strapped payers increasingly compare a drug's cost to its clinical benefits and agree to pay for it only if the benefits justify the cost. Government health systems in Europe are leading this drive, but U.S. insurers are adopting the approach, too.
"Payer awareness of what they buy and why they buy is not going to diminish. And if they have good alternatives which are less expensive, they will go to the less expensive alternatives," Dr. Vasella says.
This increases the pressure on drug makers to focus on diseases for which there are few treatments. Novartis in coming weeks plans to request regulatory approval for a new drug for multiple sclerosis, a disease that badly needs new treatments. Novartis's drug, called FTY-720, would have an advantage over existing drugs because it is a pill that can be taken orally. Existing drugs are all given by injection or infusion, making them more cumbersome for patients. But it isn't clear whether Novartis's drug will pass regulatory reviews for safety and efficacy.
Novartis hasn't completely stopped developing drugs for common ailments. Last year, it began selling Tekturna for hypertension, a disease for which there are already many effective generic drugs. Tekturna generated sales of $202 million during the first nine months of this year, which some analysts describe as disappointing. Some say the drug hasn't found favor with payers or doctors, because they have so many generics to choose from. Dr. Vasella says Novartis is working on clinical studies that it hopes will show Tekturna has benefits beyond what older therapies can offer. "But we will have to demonstrate in the ongoing trials that the outcome is very positive," he says. "People will not just believe it because you say it."
Write to Jeanne Whalen at jeanne.whalen@wsj.com
ogxi
The way the deal is structured and back loaded the trial has to work or the company isn't worth anything. If they had gotten more up front and didn't have to fund part of the trial they could have developed something else.
The fact that they couldn't get a better deal probably means you are correct in your view about ogx-11
snss
Frankly I think the stock go touted on some of these penny stock boards at the same time and caused tremendous volume, then they looked for an excuse as to why it went up. I think guys in the financing get the stocks pumped so they unwind a lot of their positions.
It happens a lot after financings
do you know why snss has such volume and price movement?
Osteologix Inc. Announces Plans to File for European Marketing Authorization for Proprietary Osteoporosis Drug
Press Release
Source: Osteologix Inc.
On 8:15 am EST, Wednesday December 9, 2009
GLEN ALLEN, Va.--(BUSINESS WIRE)--Osteologix Inc. (OLGX.OB) announced today that the company anticipates that it will be prepared to file a marketing application in 2011 in the European Union for its proprietary second-generation strontium therapy, NB S101 (strontium malonate). If successful, this will be Osteologix' first marketing approval of NB S101 for the treatment of postmenopausal osteoporosis.
Following the successful completion of the company's initial Phase 2 study comparing NB S101 (strontium malonate) to Protelos® (strontium ranelate), which is marketed by Les Laboratoires Servier (Servier), Osteologix sought feedback on its development program from various regulatory authorities. As a result of these discussions, Osteologix has determined that the company will be positioned to pursue a European Marketing Application for NB S101 as a treatment for post-menopausal osteoporosis. The company expects that it can complete the additional development work in 2010 and will be prepared to file a marketing application during the first half of 2011. This pathway is in keeping with the Company's overall strategy of developing its once-daily strontium tablet for the osteoporosis market. Osteologix has initiated discussion with potential regional and global partners and believes that the selected partner(s) will be able to begin marketing the novel product by 2012, providing them with at least 12 years of patent protection in the European Union.
"Following the regulatory meetings, we now believe we are well positioned to leverage our existing data package combined with new clinical data that we expect to generate in the next year to obtain European approval for our lead product. Our strategy to obtain approval for NB S101 is in keeping with current regulatory directives," said Philip J. Young, President and CEO of Osteologix. Young continued: "We are well on our way to achieving a significant milestone for Osteologix and our stakeholders. We believe that our product will be a significant addition to the osteoporosis market, and we anticipate that our product will benefit the millions of women with osteoporosis who are in search of a safe and tolerable therapy.”
As previously reported during the 35th European Symposium on Calcified Tissue, the Phase 2 study comparing NB S101 to Protelos demonstrated that NB S101 was safe and well tolerated in the target patient population with the frequency and types of adverse events comparable to those with the active comparator, Protelos (strontium ranelate). NB S101 induced a significant dose-dependent reduction in bone resorption relative to placebo. In addition, all three doses of NB S101 significantly increased bone mineral density (BMD) as measured at the total hip and lumbar spine.
Strontium ranelate, marketed in Europe since 2004 by Servier as Protelos or Osseor®, is the only prescription strontium-based therapy for the treatment of osteoporosis. This year the product is projected to reach sales of more than $300 million, with approximately 80% of this revenue being generated in Europe. Unlike Protelos (strontium ranelate), which is formulated as a powder and packaged in a sachet, NB S101 (strontium malonate) is manufactured as a tablet. Young further states, "We believe our tablet will be preferred over a powder formulation, and should rapidly garner significant market share. We expect the overall strontium market to grow as NB S101 establishes itself as a viable alternative for the treatment of osteoporosis.”
About Osteologix
Osteologix develops proprietary therapeutics for the treatment of unmet medical needs in bone disease and women's health. The Company's lead product, NB S101, is being evaluated in a phase II clinical trial for the treatment of osteoporosis. Based on the Company's own data as well as data from phase III clinical trials conducted on a similar drug that is approved for sale in Europe, Osteologix believes that NB S101 increases formation of new bone and decreases loss of existing bone. Osteologix further believes that NB S101 will provide patients with greater convenience and fewer side effects than drugs currently approved for osteoporosis in the United States, Europe and elsewhere. For more information please visit, www.osteologix.com.
Forward Looking Statements
Certain of the statements set forth in this press release constitute "Forward looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include, without limitation, any statement that may predict, forecast, indicate, or imply future results, performance or achievements, and may contain the words "anticipate," "believe," "estimate," "expect," "forecast," "intend," "may" "project," "plan," "will," "should," "could," "would," or words or expressions of similar meaning, including without limitations statements about Osteologix’ plans for regulatory approval of NB S101, clinical trial plans, uses and benefits of NB S101 and plans to enter into an agreement with a global or regional partner for the development and commercialization of NB S101. All such forward looking statements involve risks and uncertainties, including, but not limited to Osteologix’s ability to: enroll and conduct required clinical trials to support regulatory approval of NB S101, obtain regulatory approval for NB S101, raise additional capital necessary to fund its clinical trials and operations; maintain, protect and enforce its intellectual property rights; identify and negotiate agreements with third parties for the continued development and commercialization of NB S101 and retain and hire key personnel. Additional risk and uncertainties include the identification of potential adverse side-effects or toxicities involving NB S101, the effect of competition and proprietary rights of third parties and other factors affecting the health care industry in general, including pricing pressure and regulatory and reimbursement changes. There can be no assurance that such forward-looking statements will prove to be accurate and Osteologix undertakes no obligation to update any forward-looking statements.
Contact:
Osteologix Inc.Investor and Media Contact:Baxter Phillips, IIISr. Director, Corporate Development804.747.6026
There was no way from that data you could predict the data they just announced
yes,
the data looks very good. I just don't know how everyone on this board seemed to know it would be good before it was presented
that was birchenoff of Barclays
the data is very good
ARIAD Pharmaceuticals: -534 Data Impresses at ASH
We are reiterating our 1-OW on ARIA following presentation of Phase I data for pan Bcr-Abl inhibitor -534 in advanced hematological cancers at ASH. Data presented in heavily pretreated CML pts including pts with the T315I mutation were impressive and supportive of a partnership and future regulatory and commercial potential.
With primary focus on efficacy, in 20 evaluable chronic phase (CP) pts, -534 achieved 25% complete and 45% major cytogenetic responses (CyR's) with a 83% complete hematologic response (CHR). In 12 evaluable CP pts with the T315I mutation, -534 achieved a CHR in all pts and a major CyR in 43% of pts. 28 pts still remain on drug in the study including 9/10 CP pts with the T315I mutation. While there may be a focus on the AE profile and in particular 3 pts with QTc prolongation, we would note that 2nd line agent Sprycel has a QTc warning while Tasigna has a boxed warning in the label.
ARIA has suggested initiation of a registration study in 2H10. With Phase III SUCCEED data for Ridaforolimus in sarcoma also expected in 1Q/3Q10, ARIA could potentially establish itself as a company with two late-stage assets in 2010.
Ariad Pharmaceuticals Inc (ARIA.O) said interim data from an early-stage trial of its experimental cancer drug showed promise in treating patients with advanced blood cancers who are resistant to other therapies.
Initial data presented by Ariad at the annual meeting of the American Society of Hematology showed the drug, AP24534, was well-tolerated and had beneficial anti-tumor activity.
The trial is studying patients who failed prior inhibitor therapy for the treatment of chronic myeloid leukemia (CML), which is characterized by an excessive and unregulated production of white blood cells by the bone marrow due to a genetic abnormality.
"We are in the process of planning with the regulators and investigators to structure a single-armed mid-stage trial ... and hope the trial will be up and running by the middle of 2010," Frank Haluska, vice president of clinical affairs at Ariad told Reuters.
Ariad is also in the process of early discussion with its partners to form a marketing collaboration for the drug, Haluska added.
It doesn't say that they are in discussions with the regulators for a single armed pivotal trial.
I don't see anything about qt prolongation in the ariad press release
the bottom of my post says just kidding
Ariad's data looks very good. I just couldn't understand how every was saying it would be, before they saw it.
the high dose seems to have a pretty high rate of severe side effects so we should probably see if the lower doses show side effects after people have been on if for awhile.
Merck had a tki that showed some good efficacy but had to be discontinued because of side effects.
did you look at my entire post or were you in too big a rush
ariad
data doesn't look that good to me
just kidding
what makes you think that just because the tki is oral it doesn't have side effects.
sometimes oral drugs have more side effects than injection.
first see the data.
why do you say approval is a question?
I know nothing is a sure thing but what do you know of that would be a reason not to approve the drug.
I do not see how a chemo drug that requires injection can beat an oral TKI
this isn't RA or MS, this is life and death.
Omapro is currently conducting a pivotal trial in all comers with an 85 percent response rate in patients that have failed Gleevec, Sprycel and Tasigna.
At this point it doesn't matter. Ariad was looking for a quick way on to the market so they were speaking about doing a pivotal trial for the t315i mutation.
IF they get to do an open label study in the mutation, fine, I am tired of writing about it. Omapro is not just targeting the t315i, they just did that to get on the market quicker, and they were able to do an open label phase 2 study because nothing else worked.
Once Omepro is on the market, Ariad can't go to the fda and say that nothing else works, can we run an open label study?
Pivotal Data on ChemGenex's Omapro Highlighted at ASH Press Conference
- Highest Response Rates Yet for T315I + CML Clinical Trial -
- Findings Suggest Omapro Represents a New Potential Therapy for CML Patients with the T315I Resistance Mutation -
Press Release
Source: ChemGenex Pharmaceuticals Limited
On 10:04 am EST, Saturday December 5, 2009
MELBOURNE, Australia, & MENLO PARK, Calif.--(BUSINESS WIRE)--ChemGenex Pharmaceuticals Limited (ASX:CXS - News) announced today updated clinical data showing that Omapro™ (omacetaxine mepesuccinate) produced durable hematologic and cytogenetic responses in chronic myeloid leukemia (CML) patients who have failed treatment with imatinib and who have developed the Bcr-Abl T315I mutation. New data was presented at a pre-conference press showcase at the 51st Annual American Society of Hematology Annual Meeting in New Orleans, Louisiana.
At the press conference titled "Advances in Diagnosing and Treating Leukemia and Myeloproliferative Disorders" Dr. Jorge Cortes, MD, Professor of Medicine and Deputy Chair in the Department of Leukemia at The University of Texas, MD Anderson Cancer Center, a lead investigator in the study, presented data on behalf of a team including investigators from ChemGenex and leading U.S. and European clinical research centers. Completing his presentation, Dr Cortes concluded that Omapro represents a new potential therapy for patients with T315I+ CML.
Data were presented from 81 CML patients: 49 in chronic phase, 17 in accelerated phase and 15 in blast phase. Highlights of the data were:
Complete hematologic responses (CHR) in 86% of chronic phase patients, median response duration 9 months
Total cytogenetic response rate of 41% in chronic phase patients, with major cytogenetic response (MCyR) rate of 27%
Overall hematologic responses in 35% of accelerated phase patients (median duration 7 months)
Overall hematologic responses in 47% of blast phase patients (median duration 2 months)
Investigators reported that omacetaxine is safe for self-administration, is well tolerated, and that reversible and manageable myelosuppression is the most common side effect
“We are delighted with the positive data presented today that continues to show that Omapro can provide clinical benefit to patients in this very difficult to treat sub-set of CML where there are no other approved treatment options,” said Greg Collier, Ph.D., Managing Director and Chief Executive Officer of ChemGenex. “We would like to thank Dr. Cortes and all of our investigators for their efforts to produce this data. These results support our regulatory filings, and we look forward to working with the agencies in the U.S. and Europe over the next several months as we seek approval for Omapro in 2010.”
Applications for marketing approval for Omapro are currently under review by the U.S. Food & Drug Administration (priority review), and the European Medicines Evaluation Agency.
The complete oral presentation by Dr. Cortes detailing this study will take place:
Date/Time: Monday, December 7, 2009 at 4:45 p.m., U.S. Central Time
Abstract/Title: #644: Safety and Efficacy of Subcutaneous-Administered Omacetaxine Mepesuccinate in Imatinib-Resistant Chronic Myeloid Leukemia (CML) Patients Who Harbor the Bcr- Abl T315I Mutation – Results of An Ongoing Multicenter Phase 2/3 Study
Oral Session: Chronic Myeloid Leukemia - Therapy: Managing Resistance and Residual Disease
Location: Conference Auditorium AB (Ernest N. Morial Convention Center)
About the Study
The study was designed to evaluate the safety and efficacy of subcutaneously (SC) administered omacetaxine in patients with imatinib resistant T315I+ Philadelphia chromosome positive CML. Eligible patients include adult CML Patients in chronic, accelerated, or blast disease phase (CP, AP, BP) with a confirmed Bcr-Abl T315I mutation and resistance to imatinib therapy. Patients were given 1.25 mg/m2 SC omacetaxine twice daily for 14 days every 28 days until hematologic response for induction therapy. For maintenance therapy, patients were dosed 1.25 mg/m2 SC omacetaxine twice daily for 7 days every 28 days. Eighty one patients were described in this presentation (49 CP, 17 AP and 15 BP). The median age was 58 years (19-83) with a median CML disease duration of 54 months (5-286). All patients had failed prior imatinib therapy, and 79% had failed two or more prior TKIs. The presence of baseline T315I mutation was confirmed in all patients.
About Omapro™ (omacetaxine mepesuccinate)
Omacetaxine mepesuccinate is administered subcutaneously and acts differently from TKIs. It may have a therapeutic advantage for patients who have failed TKIs. Omacetaxine is currently in global phase 2/3 clinical trials for CML and has been granted Orphan Drug designations by the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMEA) as well as Fast Track status by the FDA.
Omacetaxine is a first-in-class cetaxine with demonstrated clinical activity as a single agent in a range of hematological malignancies. Omacetaxine has a novel mechanism of action, specifically binding to the ribosomal A-site cleft and inhibiting protein translation of short-lived oncoproteins that are upregulated in leukemic cells (particularly Cyclin-D1, Mcl-1 and c-Myc). In addition, pre-clinical research presented at the 14th Congress of the European Hematology Association (EHA) in Berlin, Germany this summer, demonstrated that omacetaxine kills human CML stem cells that are known to be insensitive to TKIs.
About Chronic Myeloid Leukemia (CML) and the Bcr-Abl T315I Mutation
Chronic myeloid leukemia (CML) is a cancer of the bone marrow with a worldwide prevalence of approximately 200,000 patients. The bone marrow is responsible for the production of specialized cells that constitute blood; these cells include red blood cells (to carry oxygen around the body), thrombocytes (to help stop bleeding) and certain white cells (part of the body’s defense system against infection). In patients with CML the cell production system is diseased and defective. Cells multiply uncontrollably and do not fully develop (differentiate) into functional blood cells.
The majority of CML patients initially respond well to treatments with drugs called tyrosine kinase inhibitors (TKIs). However, a significant proportion of patients fail, or become intolerant to, one or more TKIs. In many of these situations the cause of failure can be traced to the emergence of Bcr-Abl mutations. A common mutation called T315I renders CML resistant to all currently approved TKIs, and has created a significant unmet medical need in the management of CML.
About ChemGenex Pharmaceuticals Limited
ChemGenex is an oncology focused biopharmaceutical company developing small molecules with new mechanisms of action to treat malignancies with significant unmet medical needs. The company is developing omacetaxine, its lead product candidate, for the treatment of patients with Chronic Myeloid Leukemia (CML), Acute Myeloid Leukemia (AML), and Myelodysplastic Syndrome (MDS). A New Drug Application has been accepted by the U.S. Food and Drug Administration and a Marketing Authorisation Application has been validated by the European Medicines Agency for CML patients with the Bcr-Abl T315I mutation. The corporate strategy for ChemGenex is to commercialize omacetaxine independently in North America and to establish commercial partnerships in the rest of the world. ChemGenex currently trades on the Australian Stock Exchange under the symbol "CXS" For additional information on ChemGenex Pharmaceuticals, please visit the company’s website at http://www.chemgenex.com.
Details on the clinical trials can be accessed from the following websites: http://www.clinicaltrials.gov/ct2/show/NCT00375219?term=homoharringtonine&rank=9 and http://www.tkiresistantcmltrials.com.
Omapro™ is a trademark of ChemGenex Pharmaceuticals Limited
Safe Harbor Statement
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Contact:
ChemGenex InformationDr. Greg CollierCEO and Managing DirectorCell (Australia): +61 419 897501Cell (USA): +1 650 200 8145Email:gcollier@chemgenex.comorInvestor Relations – AustraliaKyahn WilliamsonBuchan ConsultingTel: +61 (0)3 9866 4722Cell: + 61 (0)401 018 828Email: kwilliamson@bcg.com.auorInvestor Relations – USARemy BernardaBlueprint Life Science GroupTel: +1.415.375.3340 x 2022Cell: +1.415.203.6386Email: rbernarda@bplifescience.comor
considering that the stock was trading at 50 cents in May I think people wouldn't be too upset about raising 45 million dollars at these prices.
they can now get to where they need to go.
Soligenix, Inc
http://seekingalpha.com/instablog/494183-steven-cordovano/37944-soligenix-inc
Investors are drawn to the biotechnology sector in search of outsized returns, but today’s speculative spirit seems to have matured somewhat as the quest for upside has been tempered with the additional requirement for less risk. It obviously takes a lot of work to root out biotech opportunities where there are large potential returns with greater chances of success. Soligenix (SNGX) is one of these special, special situations and one of the best kept secrets in biotech.
While not devoid of uncertainty, Soligenix is poised for substantial growth and success and an investment in the company--currently enrolling a confirmatory Phase 3 clinical trial, is analogous to having the exam questions the night before the test.
Recent developments include:
· Initiated enrollment in its confirmatory Phase 3 of orBec® for the treatment of acute GI GVHD
· 35% royalty generating North American partnership with Sigma-Tau Pharmaceuticals for orBec
· Corporate name change to Soligenix, Inc. from DOR BioPharma, Inc.
· Completed a $4.4 million financing with its partner Sigma-Tau and institutional investors.
· Awarded a $9.4 million grant from the NIH for RiVax and its biodefense business.
· Awarded a $500,000 NIH grant to support a clinical trial in radiation enteritis with its research compound SGX201 (oral BDP).
· Awarded Orphan Drug Designation for orBec in chronic GI GVHD.
Background
Previously known as DOR BioPharma, Inc., Soligenix is a biotechnology company developing products to treat the gastrointestinal side effects of cancer treatments and vaccines for bioterrorism. Its lead compound, orBec® is a topically active corticosteroid currently in a confirmatory Phase 3 clinical trial for the treatment of acute GI GVHD.
After having missed several projected NDA filing dates, Chris Schaber, PhD (formerly COO with Discovery Labs) was hired as a consultant to perform a regulatory review on the NDA because at the time, the filing was late and still incomplete.
Dr. Schaber was ultimately hired as CEO in August of 2006 and the Company subsequently filed the NDA after providing the FDA with additional long term mortality data on orBec. During his career, Dr. Schaber has overseen multiple Phase 3 clinical trials--all which were successful. In addition to his positive assessment of the prospects for approval of orBec, Dr. Schaber was drawn to Soligenix because his own father had recently been diagnosed with multiple myeloma and was facing the realities of helping him through the stem cell transplantation process, and was well aware of the potential risk of GI GVHD.
While the company has faced some tough years in the past, management has begun to work more creatively at minimizing dilution and the recently partnership with Sigma-Tau is confirmation of this. The company has a very simple capital structure with no debt or preferred stock.
orBec
Oral beclomethasone dipropionate (BDP), the active ingredient in orBec is a corticosteroid that works topically against inflammatory disorders at mucosal surfaces. The drug has demonstrated activity in acute Graft-Versus-Host-Disease (GVHD) which affects the entire GI tract and can occur after hematopoietic cell transplantation (HCT). Typically, the disorder manifests itself in the GI tract where orBec is beneficial because it reduces exposure to systemically delivered steroids which in turn reduces the incidence of infections while preserving the graft- versus-leukemia effect. This is a very important part of the orBec story. The graft versus-leukemia effect means that some GVHD is actually beneficial to the patient as donor lymphocytes assist in the killing of cancer cells.
orBec is a 2-pill system that contains 1 mg each of BDP. One pill is an immediate release tablet and the other is enteric coated for delayed release in the lower GI tract. Each 2-pill dose is taken 4 times per day for a total of 8 mgs per day. This 2-pill system delivers coverage to the entire GI tract from the stomach through the colon.
BDP, the active ingredient in orBec is a known compound and has been previously approved by the FDA for other indications such as asthma (Becloforte: Glaxo), rhinitis (Beconase: Glaxo) and psoriasis (Propaderm: Glaxo, others) in other dosage forms. BDP is a very potent topical steroid which gives unique properties in treating conditions in the gastro intestinal tract.
GI GVHD
Gastrointestinal Graft versus Host Disease is an unmet medical need and considered an Orphan Disease. It generally occurs in blood cancer patients who undergo bone marrow or stem cell transplantation. Symptoms can include anorexia, vomiting, bloody stool and necrosis of endothelial cells. The disease occurs when the immune cells from another person (the donor) are put into a patient whose own immune system can’t eliminate the foreign cells (allogeneic). This creates an inflammatory response that can damage epithelial cells of various organs in the body--typically in the GI tract, but also affects the skin and liver.
The current standard of care is treatment by systemic corticosteroids (either prednisone or methyl prednisone). There are various grades of the condition that are identified by the number of organ systems affected. Grade III and IV are the most severe and deadly as there can be significant gastrointestinal, bacterial translocation, intestinal fluid and loss of protein. Mortality is also prevalent in Grade II but is likely caused by the side effects of the treatment which suppresses the immune system and neutrophil function resulting in potential infections.
GI GVHD is commonly multi-episodic which under the current standard of care requires continuous treatments of immunosuppressant systemic corticosteroids.
It is very important to understand two things with regard to orBec:
1. A certain amount of graft versus-leukemia effect is desirous to eliminate the underlying malignancy but high dose prednisone inhibits this effect. And,
2. Due to its topical activity, orBec has been shown to preserve the graft-versus-leukemia effect due the prednisone sparing properties.
Market
There are over 10,000 allogeneic hematopoietic stem cell transplants each year in the US.. GI GVHD affects approximately 50% of allogeneic transplant patients and approximately 5% of autologous transplant patients. The associated mortality rate is approximately 25% at 200 days post transplant.
Phase 2 Trial
In the single-center randomized double-blind, placebo-controlled Phase 2 study, the primary endpoint was the clinically relevant determination of whether GI GVHD patients at Day 30 (the end of treatment) had a durable GVHD treatment response as measured by whether or not they were able to consume at least 70% of their estimated caloric requirement. The response rate at 30 days (the primary endpoint) was 71% in the orBec treatment arm versus 55% in the control group for a statistically significant p-value of 0.02. Also, at 40 days (10 days after end of orBec treatment) 52% of patients were meeting the caloric intake requirement versus only 17% in the control group. This result demonstrated a durable and lasting effect of orBec even beyond the treatment period.
Phase 3 Trial
Soligenix’s Phase 3 trial was a multicenter randomized, double blind, placebo controlled trial that treated 129 allogeneic HCT recipients with Grade II GI GVHD. The same basic protocol was followed as in the Phase 2 trial. Although orBec did not achieve statistical significance in the primary endpoint of this trial, namely the time-to-treatment failure through Day 50 (p-value 0.117), orBec did achieve statistical significance in other key secondary endpoints such as the proportion of patients free of GVHD at Day 50 (p-value 0.05) and Day 80 (p-value 0.005) and the time-to-treatment failure through Day 80 (p-value 0.022). These endpoints are more clinically relevant.
There was also a 66% reduction in mortality among patients randomized to orBec at 200 days post-transplant with only 5 patient (8%) deaths in the orBec group compared to 16 patient (24%) deaths in the placebo group (p-value 0.013).
Even more impressively, at one year after randomization in the pivotal Phase 3 trial, 18 patients (29%) in the orBec group and 28 patients (42%) in the placebo group died (p-value 0.04). This demonstrates that orBec not only had an effect on patients’ acute GI GVHD, but also had an effect on their underlying cancer. These data further support the hypothesis that orBec would in fact enhance the positive effects of that transplant (noted above as the graft-versus-leukemia effect).
What Went Wrong?
On May 9, 2007 the Oncology Drug Advisory Committee discussed orBec for the treatment of GVHD. Based primarily on the missed primary end point, the panel voted against substantial evidence of efficacy 7 votes to 2. The FDA ensured the outcome of this vote by dictating to the panel their specific definition of substantial evidence of efficacy which is comprised of two successful controlled clinical trials. With the missed primary endpoint, the panel could not vote in favor of orBec despite all the efficacy and safety it had demonstrated.
This was further complicated by the attempt to overlay strict statistical methodology that in this case overemphasized the importance of a primary end point even though the primary endpoint was essentially clinically meaningless. Interestingly, only one of the doctors on the panel had experience with GVHD and he was one of the 2 votes in support of approval.
As the conversation between Advisory Panel Chairperson Maha Hussain and Nancy Scher--a Medical Officer with CDER, the Division of Oncology Drug Products demonstrates, the panel was concerned with “lowering the bar,” and regardless of the clinical outcomes, missing the primary end point in their minds tainted the results:
“CHAIRPERSON HUSSAIN: I have a question to the FDA. Either of the presenters could take it. Understanding that the day 80 time-to-failures or number of failures was a secondary endpoint, but in reality it is just an extension of time, so why is it not good enough?
DR. SCHER: I guess the answer is statistical.
CHAIRPERSON HUSSAIN: I guess I understand it’s statistical. I understand that party line of failing primary endpoint, therefore everything is exploratory otherwise.”*
*US Food and Drug Administration Center for Drug Evaluation and Research (CDER), Oncologic Drugs Advisory Committee Meeting Transcript, Wednesday May 9, 2007, pg 265.
Some have speculated that it might actually have been due to the fact that the agency was under fire from Dendreon interest groups because Provenge (a prostate cancer drug that also missed its primary endpoint in a Phase 3 trial) had been turned down the same day as the orBec panel meeting and that the agency was trying to send a message that a missed primary endpoint, regardless of any other meaningful data you have, is a fatal flaw.
Mortality Rates
Irrespective of why or what caused the company to miss its primary end point, orBec has demonstrated outstanding mortality data as published in the January 2007 issue of the peer-reviewed journal Blood. The percentage reduction in mortality as reported in the Phase 3 trial was 66% and 55% in the Phase 2. More astounding, orBec achieved a 94% reduction in mortality among mismatched donors at 200 days post transplant in the Phase 3 trial. In other words, only 4% of mismatched orBec patients died versus 42% of the patients who received the placebo. orBec is the only drug in over 3 decades of GVHD clinical research that has been shown to both treat GI GVHD’s symptoms and actually reduce mortality.
Rationale for Investment in Soligenix
Soligenix has obtained an SPA for a confirmatory Phase 3 pivotal trial (recently initiated enrollment) which will be almost the exact design as the previous Phase 3 but with the new primary end point being Treatment Failure Rate at Study Day 80 (previous p-value of .005). Additionally, Soligenix has agreement from the EMEA that the current confirmatory Phase 3 trial would suffice for European approval. The confirmatory Phase 3 is a multicenter trial with 166 patients enrolled and powered at 90%.
The design of this new confirmatory clinical trial and Soligenix’s choice of the new primary endpoint are the foundation for the investment rationale. In my opinion Soligenix has delivered to its shareholders a value proposition built around the absolutely correct design of the Phase 3 clinical trial. Importantly, Soligenix has anchored this design using extensive clinical data obtained from its previous Phase 3 study. The company has essentially eliminated the question they got wrong on the previous test and are now going into the final exam armed with questions that they have already answered correctly.
Furthermore, even putting aside orBec’s prior clinical success and the $7.5MM in cash on the balance sheet as of September 30th (and an additional $1MM received from Sigma-Tau for starting the Phase 3), at the current market cap of approximately $40MM the stock seems inexpensive for a company with an orphan drug in a fully funded Phase 3 trial with other indications beginning soon and a marketing deal already in place that has a 35% royalty.
Sigma-Tau Partnership
Soligenix has a signed marketing agreement in place with Sigma-Tau Pharmaceuticals, Inc., a U.S.-based, wholly owned subsidiary of the Sigma-Tau Group which is dedicated solely to the global development and commercialization of medicines for patients with rare diseases. Sigma-Tau will launch orBec in the North America and pay $10MM in milestone payments with $1MM now due that the enrollment of the first patient in the Phase III clinical trial has occurred. Because there are only around 15 major transplant centers that do 60% of all transplants in the US, investors should anticipate a very rapid launch post approval. The company also was able to negotiate a healthy 35% net royalty on US sales of orBec (and with a similar rate expected in the EU).
Other Indications for orBec/oral BDP
GVHD Prophylaxis
Soligenix is nearing completion on this 138 patient Phase 2 study and expects the announcement of results in the first half of 2010. Approval in this indication would effectively double the market for the orBec. This study is almost entirely funded by a National Institutes of Health (NIH) grant. However, as was speculated by members of the FDA panel meeting, if orBec gains approval for treatment of GI GVHD, it will be highly likely that it will be used off-label for prevention of GVHD.
Panel member Joanne Mortimer, M.D., Professor of Clinical Medicine and Medical Director at the Moores UCSD Cancer Center highlighted this when discussing the possibility of a prophylactic study as an alternative approach to another phase 3 trial for orBec:
“DR. MORTIMER: … My suggestion was going to be a prophylactic study, but also it seems to me that the biggest selling point of this drug is that it minimizes toxicity of steroids. However the study is designed, a comparison of this agent against steroids, if you demonstrate an improved toxicity profile, I can’t but imagine that it would move farther up in line for the indication indicated here today.”*
*US Food and Drug Administration Center for Drug Evaluation and Research (CDER), Oncologic Drugs Advisory Committee Meeting Transcript, Wednesday May 9, 2007, pgs 328-329.
Radiation Enteritis
Radiation Enteritis is a swelling of the inner lining of the small intestine that occurs after cancer radiation treatments commonly associated with cervical, uterine or rectal cancers given the proximity of the radiation treatment to the small intestine.
The company has cleared their IND with the FDA and has been granted Fast Track status. The trial Phase 1/2 trial will begin enrollment sometime before the end of the year and is partially funded by a $500,000 NIH grant. The balance of this trial not covered by the NIH grant will be paid for by Sigma-Tau.
Chronic GI GVHD
The company is planning a Phase 2 protocol and expects to initiate the trial in the second half of 2010.
Crohn’s Disease
The single largest potential market for Soligenix could be Crohn’s Disease which is similar to GVHD in that it is an inflammatory disease that can affect any part of the gastrointestinal tract. It is actually classified as an inflammatory bowel disease, but unlike GVHD, Crohn’s is an autoimmune disease and not a complication of HCT. This indication will require an expansion of the company’s scientific advisory board and more extensive planning of dose ranging studies before Soligenix can formally inaugurate their Crohn’s program.
There are strong indications that oral BDP will have success in this indication given that topically applied Budesonide has already been shown to improve symptoms and is the standard of care. Since Crohn’s can present at various points in the entire intestinal tract, oral BDP’s advantage may be that it will have a two pronged effect on the disease--both topical and systemic (oral BDP is approximately 35% bioavailable) and can treat both the upper and lower GI.
Approximate Worldwide Market Sizes
Treatment of GI GVHD ~5,000 patients per year (US): $100MM per year
Prevention of GVHD ~10,000 patients per year (US): $200MM per year (inclusive of Treatment of GI GVHD)
Chronic GI GVHD ~2,500 patients per year (US): $50MM per year
Radiation Enteritis ~50,000 patients per year (US): $200MM per year
Crohn’s Disease~ 500,000 patients in the US with 100,000 pediatric cases per year: >$500MM per year.
Biodefense
RiVaxTM is Soligenix’s proprietary vaccine developed to protect against exposure to ricin toxin. With RiVax, Soligenix is the world leader in ricin toxin vaccine research. There currently are no vaccines available to prevent or treat ricin poisoning. One human Phase 1 clinical trial has been completed with and a second trial now in progress with results expected in the first half of 2010.
The potential use of ricin toxin as a biological weapon of mass destruction has been highlighted in an FBI Bioterrorism report released in 2007 entitled Terrorism 2002-2005, which states that, “Ricin and the bacterial agent anthrax are emerging as the most prevalent agents involved in WMD investigations” (http://www.fbi.gov/publications/terror/terrorism2002_2005.pdf).
Soligenix has recently been awarded a $9.4 Million grant from the NIH which will fund the development, formulation and manufacturing processes for vaccines, including RiVax, that are stable at elevated temperatures.
The company has received approximately $25 million in government funded development grants to date for RiVax. Soligenix’s biodefense business is cash flow positive and completely supported by the grants. This means that investors will realize all the benefits of potential advancements in its biodefense technology without having to suffer any dilution as a result of raising money to support the research.
Management
Christopher Schaber, PhD, CEO, has over 20 years of experience with companies such as Discovery Labs (COO), The Liposome Company and Wyeth Ayerst
Evan Myrianthopoulos, CFO, has 14 years of experience with CVL Advisors Group, Discovery Labs (CFO) and Paramount Capital.
Brian Hamilton, MD PhD, CMO, has worked for over 30 years with companies such as Astra, USA, Wyeth and Akermes.
Robert Brey, PhD, CSO, has over 27 years experience with Lederle-Praxis (American Cyanamid) and Vaxcel, Inc.
Valuation
While it is always difficult to value biotech companies prior to an approved indication, Soligenix had almost a direct comp in Osiris Therapeutics which was the only other public company with trials to treat Crohn’s disease and GVHD. Both of Osiris’ trials failed to show efficacy, but prior to the failures the market valued Osiris at approximately $600MM.
Using $100MM for the available worldwide market for the treatment of GI GVHD and applying a 3 multiple to the a blended 35% (US and expected EU) net royalty and discounting it back by 20% yields a valuation of $292MM or $1.22 per fully diluted shares (240 million). This assumes commercial sales beginning in 2 years and goes through 2019 when orBec's first patent expires.
Furthermore, this gives no value to all of the other indications which are currently in the works: Prevention of GVHD, Radiation Enteritis, Chronic GI GVHD, Crohn’s Disease and RiVax.
Conclusion
It is not that often that a company has 2 randomized, double-blind, placebo controlled trials demonstrating the effectiveness of a compound before entering a confirmatory Phase 3 trial. OrBec has unquestionably been shown to treat the side effects of GI GVHD after hematopoietic stem cell transplants and it has shown efficacy regardless of type of patient conditioning or the type of stem cell donor.
If that were not enough, it is highly likely in my opinion that the unique 2-pill formulation of orBec (which gives it the ability to reach the entire intestinal tract from the stomach to the rectum) gives it a great degree of probability to address the other intestinal inflammatory disorders--some of which, like Crohn’s Disease, possess market opportunities many times larger than GI GVHD.
One final consideration is that orBec actually has the potential to expand the overall market for stem cell transplantation. Because of the drug’s unique ability to treat GI GVHD effectively in the non-myeloablative and mismatched donor transplant groups (which tend to be older and sicker), doctors may now be more likely to recommend transplants for these patients that heretofore would not have been considered viable candidates. While this remains to be seen, an expansion of usage obviously has the potential to deliver further value to shareholders.
In summation, given orBec’s positive clinical history, investors can be somewhat reassured that Soligenix has very high odds of a successful outcome in the confirmatory Phase 3 trial. Now that they have worked out most of the issues with the FDA and ironed out the kinks from the previous pivotal trial, knowing the answers to the test questions should give Soligenix the strong likelihood of acing the final exam!
Disclosure: the author has a position in Soligenix, Inc.
Please give me an example of a cancer trial where there was an open label study in an indication where there was an approved drug?
They were not approved for the t315i mutation, in fact Tasigna is contraindicated in the mutation because it makes things worse. I am not sure if Sprycel is contra indicated
In other words you are saying that if Omapro is approved in the t315i mutation that ARIA will be given the opportunity to run a placebo controlled trial
Would it be ethical for the FDA to allow ARIA to put people on a placebo when Chemgenex has proven that their drug works and has been approved. It would be unethical to run a placebo controlled trial. If Omapro isn't approved then you would be right.
The fda is probably curious to find out if Omapro in addition to a tki would result in cures and a pivotal trial would be a good way for them to find out.