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Thanks for the messages. Some day we'll all meet at the CTIX millionaires party (well, I'll settle for the several hundred thousandaires party). If I can convince my wife to jump in the deep end with me then maybe the millionaires.
Lee
Thanks to all responders. Easy and Gov, I appreciate your support of free speech. I didn't feel ridiculed-I expected feedback, both positive and negative. I think we all have a potentially fantastic opportunity here. When I have my money invested I feel free to make comments if I think I have some justification for my observations. I think most here would agree that at times we have had certain expectations here based on LE or KM comments, and sometimes those expectations have not been realized, or the original principles' statements have been subject to interpretation. We're all not going to see any of this exactly the same, nor do we individually have the same expectations and goals. While some responses can be somewhat snarky, I still appreciate all legitimate viewpoints, as I would expect others to respect mine. I have caused several friends and investors to buy CTIX and I feel a sense of added responsibility to keep myself informed, and I don't want to pass on misinformation or misguided expectations to my fellow investors. I like to deal in facts, and I like open, honest and realistic communication from the companies I invest in. For the most part I am very pleased with the CTIX communication, but there are some times where I do question the process. I wish us all the best--the next twelve months should be very exciting!
Lee
Thanks Progressive for asking for, receiving and posting that information. Now, why the heck couldn't Leo have made this information available to all of us, and, at an earlier time? Why does this very pertinent info have to come out as an email reply to one individual, and, somewhat after the fact of the occurrences? I don't get it. If the company can release specific information, they can release it to the public, and in an official manner. While I appreciate Leo's enthusiasm I keep seeing examples that this seems like his first rodeo. I, for one, would appreciate a more consistent and professional approach to information dissemination. This may seem petty to some, but in order to make educated investing decisions (guesses), we are, to a certain extent, only as good as the data provided to us. That data should be presented in a timely, concise and professional fashion. Not unhappy here, just confused about the communication process.
Lee
So does anybody know what actually happened to this deal?
Lee
I may have misunderstood your statement to mean that safety was the only thing to expect from this Phase 1. In rereading it I think you were implying that it is probably too soon in this test for anything like efficacy to show up. I am assuming you would still think that P21 can show up before this trial is over? (By the way--what I posted is a straight copy from: http://www.cancer.gov/clinicaltrials/search/view?cdrid=738782&version=HealthProfessional&protocolsearchid=11293870
Lee
I disagree--there is more than one objective in this Phase 1 study:
The primary objectives are the following:
•To determine the maximum tolerated dose (MTD) of Kevetrin.
•To determine the dose limiting toxicities (DLT) of Kevetrin.
•To establish a safe dose level of Kevetrin that can be used for future studies.
The secondary objectives are to determine the following:
•The pharmacokinetics of Kevetrin in humans.
•Observe for evidence of antitumor activity following administration of Kevetrin.
•If Kevetrin induces changes in the biomarker p21 in peripheral blood lymphocytes.
•If there is a pharmacodynamic relationship between the plasma concentrations of Kevetrin and a clinical or cellular effect.
It just makes me want to throw up that I get excited about a move from .10 to .11. Having said that, I still have visions of Aethlon actually becoming a commercially viable company someday!
Lee
CytoSorbents Announces FDA Approved US Air Force Funded Human Trauma Pilot Study for Rhabdomyolysis 06/17 05:00 AM
--------------------------------------------------------------------------------
MONMOUTH JUNCTION, N.J., June 17, 2013 (GLOBE NEWSWIRE) -- CytoSorbents Corporation (CTSO:$0.126,0$0.001,00.80%) , a critical care focused company using blood purification to treat life-threatening illnesses in the intensive care unit, announced today that the United States Food and Drug Administration (FDA) has granted approval to begin a U.S.-based human pilot study using CytoSorb® for the treatment of rhabdomyolysis as a result of trauma under an Investigational Device Exemption (IDE). Rhabdomyolysis is caused by the massive release of myoglobin from severely injured skeletal muscle that can lead to kidney failure. The study was initiated by and will be funded by the U.S. Air Force. CytoSorbents is the official sponsor of the study, and expects the study to begin this year.
Dr. Phillip Chan, MD, PhD, Chief Executive Officer stated, "This major announcement is a significant milestone for CytoSorbents (CTSO:$0.126,0$0.001,00.80%) for many reasons:
This will be the first human CytoSorb® study to begin in critically ill patients in the United States. We are confident that CytoSorb® treatment will be easy to implement, after now having safely completed more than 1,000 human treatments, mostly in Europe, including a number of treatments in trauma patients.
The U.S. Air Force is funding this study, a major benefit to the Company. The trial will be conducted by experienced clinical researchers from both the U.S. Air Force and Army. Along with our previously established Trauma Advisory Board, we will have a wealth of experience to draw upon for this trial.
This collaboration expands our relationship with the U.S. Department of Defense to now include the U.S. Air Force, the U.S. Army, and the Defense Advanced Research Projects Agency (DARPA). Our collective goal is to develop our technologies for the treatment of life-threatening conditions such as sepsis, burn injury, trauma and rhabdomyolysis, to protect our wounded warfighters and civilians alike. We greatly appreciate the ongoing interest and support of these respective agencies.
As part of the IDE application process, the FDA has reviewed the CytoSorb® treatment safety data from the European Sepsis Trial. We believe this early review of the European Sepsis Trial safety data will help streamline our future discussions with the FDA about a planned U.S. pivotal trial in sepsis.
Finally, this study will expand the CytoSorb® clinical trial experience to now include trials in sepsis and in trauma patients with rhabdomyolysis. If successful, the trauma and rhabdomyolysis application could represent yet another avenue to seek CytoSorb® approval in the U.S. CytoSorb® is currently approved in the European Union and has been used successfully in treating a variety of conditions, including sepsis, lung injury, influenza, burn injury, trauma, liver failure, and others."
Based on statistics from the National Trauma Institute, trauma accounts for 42 million emergency department visits, 2 million hospital admissions, and more than 170,000 deaths in the U.S. each year. In severe trauma patients, crush injury and ischemia can lead to rhabdomyolysis - the destruction and breakdown of muscle tissue, releasing intracellular myoglobin into the blood. High levels of myoglobin are toxic to the kidneys and can precipitate acute kidney injury and kidney failure, which significantly increases the risk of death in trauma injury. CytoSorb® has demonstrated the ability to efficiently remove myoglobin in pre-clinical testing and will be evaluated on its ability to reduce myoglobin levels in human trauma patients with rhabdomyolysis when used with standard of care therapy, compared to standard of care therapy alone.
About CytoSorbents Corporation (CTSO:$0.126,0$0.001,00.80%)
CytoSorbents (CTSO:$0.126,0$0.001,00.80%) is a critical care focused therapeutic device company using blood purification to modulate the immune system and fight multiple organ failure in life-threatening illnesses. Its purification technology is based on biocompatible, highly porous polymer beads that can actively remove toxic substances from blood and other bodily fluids by pore capture and adsorption. CytoSorb®, the Company's flagship product, is approved in the European Union as a safe and effective extracorporeal cytokine filter, designed to reduce the "cytokine storm" that could otherwise cause massive inflammation, organ failure and death in common critical illnesses such as sepsis, burn injury, trauma, lung injury, and pancreatitis. These are conditions where the mortality is extremely high, yet no effective treatments exist. CytoSorbents has numerous products under development based upon the same underlying blood purification technology including HemoDefend™, ContrastSorb, DrugSorb, and others. Additional information is available for download on the Company's website: http://www.cytosorbents.com.
Please keep in mind that just because the Docs think they will get orphan status it does not mean they will. I hope Denguecide does receive it, let's please be realistic, though. Also, some posters here keep getting ahead of the whole process by thinking that the PRV is a given, as is the perceived value of the PRV. Denguecide has to become an FDA approved drug in order for NNVC to get the PRV, and there are differing opinions on what the value of a PRV may be. Once again, I hope it comes to pass that Nanoviricides does get Denguecide approved and does qualify for a PRV which they can utilize or sell to another company. But it will probably be years before Denguecide is approved and a PRV granted. Flucide should be the current driver of value, with the PPS moving up as current goals/milestones are accomplished, such as tox testing, lab completion, Flucide batches manufactured, human testing somewhere in the world, etc. I am intrigued by the new board member's connection with EKC and I wonder if we will see news about that part of the program being resurrected.
Lee
"And let's not forget Dengue. I was all excited about Eva Harris' work but then the radio went dead on that one too. Now they're quite certain they will get orphan drug status and may come into a voucher valued somewhere between $200-300 million. As if that's some sort of consolation."
You are getting a little ahead of yourself on the building issue. It's going to be a fairly long time still (it's all relative) before the building is "operational"--Of course, one person's "nearly" is another person's "interminable"!! The main thing I like is that there is cash available which enables everything to keep progressing.
Lee
I am just shocked that so many shareholders put so much faith in Leo's "expertise" and then he reveals that he is "relieved" that their pace is "normal"--how can anything he says or predicts be trusted? I am not questioning his veracity or intent, just his overall knowledge of the process. I have no problem believing his statements of fact, as in reporting on certain tangible developments, but, for me, any predictions he makes or statements about the future must be taken with a grain of salt. He is coming across to me as a neophyte in this whole trials process, when I thought or assumed or hoped he knew what was going on. Scares me a bit to rely on his communications to us. I still hope that CTIX will be a success and I have added shares on several occasions, but I will wait for actual tangible disclosure of successful testing to add any more shares.
Lee
I had to check to see if it was halted--I was hoping for an FDA good news announcement, but no such luck!
Lee
Couldn't get the video to load--anything important from Dr. Seymour? Thanks.
Lee
Okay, that's it--everybody has to go into a "timeout"! I'm guessing we're all a little on edge here, hoping that just maybe this time, this month, we'll actually get some tangible good news. The posters on this board are a good lot and I think we all want the same thing. PhilB, thanks for your protective nature, your enthusiasm and your willingness to set the record straight. Cardswin, I appreciate your enthusiasm, too and thanks for posting. The 350,000 at .103 was a big deal, in my opinion and it was worth posting. Guess we'll have to wait to see what it actually might mean. Right now, each morning and day has some potential and at least that gives me a little more hopeful feeling when checking the news. Good luck to everybody here, and keep posting whatever you think is relevant.
Lee
I believe certain types of grants may be available from the government for "orphan" drugs.
Lee
10-20-30 would represent a 100% increase on the second round and then 30 would represent a 50% increase on the third round. Not sure what the fourth round is scheduled to be, but if it is a 50% increase then it would be "45". Using the 50% increase method then the next round would be at 67.50 and so on. I'm not sure if Leo or Menon has provided us details in the past of the projected percentage increases on the cohorts. I've seen several different projections, and it's possible we had an "official" answer to that some time back. Does anyone remember specifically or can anyone provide the message number of that info?
Lee
Nanopatent--Thanks for the response and the link!
Lee
I can't find any reference to the 60 days response time that you stated--could you please send me a link to that info? I have looked at FDA sites and others and the only process I see is that the application goes to/through three sets of reviewers before a response is given. But I don't see a calendar related response notation. And, in answer to your other question about how long for FDA approval after orphan status is given--from what I have researched getting the orphan status doesn't seem to help with the approval timeframe--all the same other applications and testing processes must take place. In return for the Orphan designation, the company can receive a 7-year marketing exclusion for the approved product plus protocol assistance, tax credits and research grants. It can still take years to reach FDA approval. The main thing that NNVC has going for it on the Dengue case is its involvement with Timothy Cote--this is a major plus for the company. I wonder what it costs them for his assistance, and I still wonder why we weren't told about this connection when they have trumpeted other consulting firm hookups for the Flucide.
Lee
FDA approves four-strain Sanofi influenza vaccine
ReutersBy Ransdell Pierson | Reuters – 3 hrs ago..
By Ransdell Pierson
(Reuters) - French drugmaker Sanofi SA said the U.S. Food and Drug Administration had approved its four-strain influenza vaccine, which is meant to provide better protection from infection than traditional three-strain flu vaccines.
The Fluzone Quadrivalent vaccine is licensed for adults, adolescents and children 6 months and older, and will be available in the upcoming 2013-2014 flu season, the company said on Monday.
The 2013 influenza season will be the first when quadrivalent influenza vaccines will be available in the United States.
Rival drugamker GlaxoSmithKline Plc in December won U.S. marketing approval for the first four-strain seasonal flu vaccine, called Fluarix Quadrivalent. The injectable vaccine, approved to immunize adults and children age 3 and older against flu virus subtypes A and B, is also slated for introduction in the 2013-14 flu season.
Three-strain flu vaccines are currently administered to help protect against the two most common A virus strains, but against just one B strain expected to be predominant in a given year.
Since 2000, however, two B virus strains have circulated to varying degrees each season, meaning patients infected with the B virus not contained in the vaccine were not immunized.
Epidemics of influenza B occur every two to four years in all age groups and are associated with pneumonia and other respiratory illnesses, Sanofi said. It added that up to 44 percent of flu-related deaths in recent years among children and adolescents were due to influenza B.
Some 226,000 people in the United States are hospitalized each year for influenza, and the number of deaths - depending on virus severity during the flu season - may range from 3,000 to 49,000, Sanofi said.
The U.S. Centers for Disease Control and Prevention recommends vaccination for everyone six months of age and older in the United States.
Shares of Sanofi were down less than 0.1 percent in Paris, while Glaxo was up 0.5 percent in London.
(Reporting by Ransdell Pierson in New York; Additional reporting by Dominique Vidalon in Paris; Editing by Daniel Magnowski and Lisa Von Ahn)
Check out this informative posting about the inception of the PRV program and more recent developments. This is written by one of the original designers of the PRV program. About halfway down, under the "Pipeline" heading there is a very interesting reference to Nanoviricides! By the way, I was wrong on NNVC having to have Flucide approved first; I was remembering our earlier discussions about when the PRV was attainable and some posters at the time thought that the PRV could be used for the Denguecide and I had pointed out that they had to get the Denguecide approved in order to qualify for the PRV which could then be used on another drug application. Do check out this link:
https://faculty.fuqua.duke.edu/~dbr1/voucher/
I'm pretty sure we'll have to see approval of Flucide first, then approval of Dengue then the PRV. Right now they are only applying for Orphan Designation--we are still years away from a Denguecide approval. I think it's great the Dengue program is still alive, but I am confused why this relationship with Cote hasn't been mentioned before. I think we'll see a share price pop then as reality settles in we'll drift back a bit, or a lot, while awaiting all the developments we have been awaiting, such as building and lab development, tox studies commencement, tox results, etc.
Lee
No, I think you have it backwards. Guess we'll have to look it up later. Too busy right now.
Lee
I think that Flucide has to be approved first, then development of the Dengue drug would allow NNVC to qualify for a PRV. Unless the rules have changed, a company has to have an approved drug already in hand before they can then go about getting a second drug approved which does qualify for a PRV which can then be used for development of another drug. It's early and I am going from memory--so we need to check all this out. But it's still exciting to see anything still happening with Dengue and NNVC.
Lee
A thought I just had about patients progressing through multiple cycles. Perhaps some of the patients chose not to continue the Kevetrin treatments. Not necessarily because of any adverse reactions, but maybe they went into these trials expecting or hoping for some sort of miracle reaction and when it did not happen, maybe they decided to discontinue, not having the patience to stay on it or wait for increased dosage. Maybe there was another trial they had learned about and in order to qualify for that they had to be free from other treatments for a period of time. I don't know if the patients agree to keep going when they sign up for the Kevetrin trial, or if they always have the option of discontinuing. Just something to think about. I think what we are all learning is that these trials take much longer that we anticipate, and it is very slow going. I would think that these Stage 4 patients are constantly willing to grasp at any straw of hope, and they may not be committed to the time involved for testing new drugs. Maybe they are really disappointed when there is no tumor regression showing up in the first month, or second month, etc. For that matter, I don't know if they are informed instantly if regression or tumor arrest does show up in the scans or if the biomarkers do show up in the lab tests. So maybe they get discouraged fairly quickly and throw in the towel before efficacy can be noted or discovered. Just wondering.
Lee
Gilead’s Hepatitis C Drug to Get Priority Review by FDA
By Andrew Pollack - Jun 7, 2013 2:48 PM MT Facebook Share Tweet LinkedIn Google +1 0 Comments
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Gilead Sciences Inc. (GILD), the world’s largest maker of HIV medicines, said its experimental hepatitis C pill, sofosbuvir, will receive a priority marketing review by U.S. regulators.
Gilead applied to gain approval of the drug in combination with ribavirin, a current treatment, as an oral therapy for patients with two types of the virus and with ribavirin and interferon for patients with other types of the infection who have never received treatment. The Food and Drug Administration has set a target review date of Dec. 8, Foster City, California-based Gilead said today in a statement.
Gilead is competing with drugmakers including Bristol-Myers Squibb Co. (BMY) and AbbVie Inc. (ABBV) to gain approval for oral hepatitis C treatments that don’t involve injections of interferon, which can cause flu-like symptoms. Hepatitis C attacks the liver and can lead to liver cancer. The virus affects about 150 million people worldwide and the market for new pills is estimated at $20 billion.
A priority review, granted to drugs that may provide major advances in treatment, speeds FDA evaluation to eight months, rather than usual 12.
Gilead gained 3 percent to $52.89 at the close in New York before the announcement. The stock has more than doubled in the past 12 months.
To contact the reporter on this story: Andrew Pollack in San Francisco at apollack1@bloomberg.net
To contact the editor responsible for this story: Reg Gale at rgale5@bloomberg.net
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57 in Phoenix
Lee
Get ready for competition for EKC
Nonresistant antibiotic in multiple late-stage trials
NovaBay Pharmaceuticals (“NovaBay” or the “Company”) is a biotechnology company focused on addressing the large unmet therapeutic needs of the global anti-infective market with its two distinct categories of compounds, Aganocides® and NeutroPhase® Skin and Wound Cleanser. The Company's four core business units, DermaBay, UroBay, EyeBay and MediBay, are developing treatments that tackle infections in the dermatology, urology, ophthalmology, and wound care.
Investment Highlights
•Aganocides provide a reduced likelihood of resistance as compared to currently existing antibiotics; 2013 is a crucial year to prove efficacy, with Phase 2b trials for viral conjunctivitis and impetigo and a Phase 2a trial for urology.
•Phase 2b results for impetigo due in 2H13; Phase 2a results show a clinical success rate of 92% and microbiological success rate of 95%, vs. approximately 30% - 50% success rate for standard of care.
•Phase 2b results for viral conjunctivitis due in 2H13; Phase 2a results showed 92% of epidemic keratoconjunctivitis (EKC) adenoviral patients experienced blurred vision clearing (vehicle: 50%), and 69% of the EKC patients had sustained clinical cure (vehicle: 54%).
•NovaBay has begun marketing its FDA-cleared NeutroPhase® skin and wound cleaner in SE Asia.
•Results from Phase 2a urology trial due mid-year 2013.
•As of December 31, 2012, cash, cash equivalents and short-term investments totaled $16.9 million.
Jeff--It will be a great press release whether it relates to HepC or HepC and Cancer as long as the word "approved" is included!
Lee
With Stage 4 cancer patients, I imagine it is tough to determine if an incident that qualifies as a significant event was caused by the dose of Kevetrin. These are, for the most part, some very ill people and I would think they are subject to all sorts of significant events. I can see where determining the cause could certainly slow down the entire evaluation and dose escalation process.
Lee
Maybe I haven't seen the latest Red Chip interview--what is the date and do you have a link? I still stand by this IDE just for the HepC ten-patient trial. Thanks.
Lee
I have addressed this multiple times as well. In one of his presentations, and possibly in the one that had a Q and A, JJ clearly stated that this IDE is for the HepC trial and then, after they got approval for that trial, they would look several months later at working towards an IDE filing for the exosome-removal trial. As I recall, at the time he said this, they were hoping for the FDA go-ahead on the current IDE in the Mar-Jun time-frame and he said, and I will paraphrase from memory, "... towards the end of the year..." as far as hoping to be able to submit an IDE for the exosome work. I would guess that is now delayed until 2014 at best, but I hope I am wrong and they could still find trial partners and be able to work up a submission before this year is over.
Lee
Read the last line in the first paragraph of your post--"...in the coming weeks.." I doubt we'll see anything relating to P21 for a while. Hope I am wrong.
Lee
I guess we can start our own private club and our members will have a special designation at the AEMD Vegas party--the party only takes place when we hit $5.00 a share based on today's share count and valuation. We can call the club:
AIRFORCE
Aethlon
Idiots
Reaching
For
Our
Rightful
Chance to
Exceed
or
We can substitute the word "Investors" for "Idiots"
lol
Glad to see I am not the only one who has felt like an idiot at times during my roller coaster relationship with AEMD. Unfortunately, most of the time the car has been going down, and it was more scary than thrilling. I play that "what if" scenario in my mind a lot--and sometimes I vow to myself that on the next big upsurge, like a 50% move, that I will sell and rebuy on the inevitable drop. But, the thing that stops me is that for the past eight years I have been terrified of being out during the exact moment that some monumental news is released and the stock goes through the roof. An outsider would call me crazy--in fact, my wife often does. So I don't know if I am a masochist or stupid or an idiot or a dreamer or I can see what so few others can see. I have had numerous conversations with JJ and I always feel that there is something potentially of value here, so I stick with it. Back to your question--I think it would depend on the nature of the news and the force and speed of the share price rise. If we get the FDA nod to go ahead with the trials I think that news will provide a pop, and the actual initiation of the trials could support that--but if we get the nod, and then we are told that the trials won't commence for a year, we lose all the momentum. If the US trials are conducted and reported on in similar fashion to the India fiasco, then forget about it. But if we get timely communication, and positive reports, then I think we bump up along the way. I would love to see what kind of dollars are being talked about with the Battelle contract, and maybe we'll get a little more light shed on that with the annual report, or the next quarterly. If they can keep this business going, and get FDA approval to proceed, and then let us know they are working on another IDE with a cancer institute partner, then I think there is a future. I once asked JJ what keeps him up at night and he said worrying about not being able to produce enough HPs when the demand is created--that would be a good problem to have--could be a long time before we get to that stage, or maybe we never get to that stage. I am either very loyal or very foolish, or both. Don't know if my answer to your question was helpful but it let me vent a little--thanks!
Lee
Based on some dashed expectations, is it possible that Leo could be that much of a novice or foolishly optimistic that he would not be more on top of when the "lab results" will be available? To have even mentioned the possibility that they would be available by ASCO and then to officially announce they hope to have results in "the coming weeks" seems to be a communication or understanding of the process that doesn't align properly. If he was hoping for them by today, and then to possibly not have them for a few weeks is a big miss in my opinion. I don't quite understand how that can occur. Well, I still have faith and picked up more shares today. I didn't expect anything more out of the PR, as it had to coincide with the Poster presentation. I did/do anticipate more helpful information either leaking out from today's Q&A or being put out in an 8-K or follow-up news release. Keep in mind, that today's PR was probably still under the auspices and control of ASCO--at least I hope this is the case.
Lee
I doubt this will create any new buying pressure--hopefully not a big dropoff in current share price. Will be interesting to see if the Q & A can produce any new information that is subsequently PRd or filtered out to the public. Keeping in mind that the Poster presentation materials have been made in advance. I'm not sure if the PR contains the absolute latest and greatest info.
Lee
I am posting these articles as they show up on my Yahoo news page. The point of this exercise is to not only provide interesting and related information on other developments at ASCO and in the industry, but to show that the media is covering this event. I wonder (hope) if there will be any reporters who are able to lock onto the CTIX and Kevetrin story and then file a report which could hit the major wire services. So tomorrow could be beneficial for us for many reasons, including the possible public awareness meter being raised. Even if what is being reported is not new to CTIX shareholders, it may be newsworthy to certain members of the media. Guess we'll see in the next 48 hours.
Lee
Merck melanoma drug shrinks tumors in 38 percent of patients
ReutersBy Julie Steenhuysen | Reuters – 35 mins ago
By Julie Steenhuysen
CHICAGO (Reuters) - A Merck & Co drug designed to unmask tumor cells and mobilize the immune system into fighting cancer helped shrink tumors in 38 percent of patients with advanced melanoma in an early-stage study, U.S. researchers said on Sunday.
The findings on the melanoma drug lambrolizumab were published in the New England Journal of Medicine and presented at the American Society of Clinical Oncology meeting in Chicago this weekend.
The research may heap pressure on market leader Bristol-Myers Squibb, maker of Yervoy - the only approved immune system drug for the treatment of advanced melanoma, the deadliest form of skin cancer.
Bristol-Myers is conducting three phase-three studies of its own drug called nivolumab in advanced melanoma, and is studying the drug's effect on a range of other cancers, including lung cancer.
Both nivolumab and lambrolizumab are part of a promising new class of drugs that disable programmed death 1 or PD-1, a protein that keeps the immune system from spotting and attacking cancer cells.
"Even though it's (lambrolizumab) the second player in the field and even though it's all early, it impressed me," said Dr. Antoni Ribas of the University of California Los Angeles' Jonsson Comprehensive Cancer Center, the lead author of the study.
Last month, the U.S. Food and Drug Administration deemed the treatment a "breakthrough therapy," a designation FDA cancer drugs chief Dr. Richard Pazdur described as "knock-your-socks-off therapies."
Results of an early-stage study of nivolumab in advanced melanoma released at the cancer meeting showed 31 percent of patients overall responded to different doses of the drug. Among those who took the 3 milligram per kilogram dose, 41 percent of patients responded. The drug response lasted an average of two years, and in many patients, the drug kept working even after patients stopped taking it.
Analysts expect the drugs to generate billions of dollars in sales. Nivolumab alone is forecast to have sales of $1.2 billion in 2017, according to Wall Street analysts tracked by Thomson Reuters Pharma.
MERCK'S STUDY
Merck's results are from the first clinical trial of lambrolizumab in advanced melanoma. They are based on analysis of 135 patients with metastatic melanoma who were divided into three groups with different treatment regimens.
Overall, lambrolizumab resulted in 38 percent of patients having confirmed improvement of their cancer across all dose levels given after 12 weeks of treatment. But there was a wide range among doses, with only a 25 percent rate among patients who got the lowest dose and 52 percent among those who got the highest dose. In the highest dose group, 10 percent had a complete response, meaning their tumors could not be detected on scans.
Side effects were generally mild and included fatigue, fevers, skin rash, loss of skin color and muscle weakness. More severe side effects were seen in 13 percent of patients, including inflammation of the lung or kidney and thyroid problems.
"This study is showing the highest rate of durable melanoma responses of any drug we have tested thus far in this cancer, and it is doing it without serious side effects in the great majority of patients," Ribas said.
Merck said it plans to start a late-stage randomized trial of the drug in melanoma and in non-small cell lung cancer in the third quarter of this year.
The company recently started a global, randomized mid-stage study of the drug versus standard chemotherapy in patients whose disease had progressed. And it is studying the drug as a treatment for triple negative breast, metastatic bladder and head and neck cancers.
Researchers at the meeting marveled at responses to new immune system treatments after decades of failed studies among patients with melanoma.
Only about one in five patients respond to Yervoy, approved in 2011 as the first immunotherapy to extend survival in patients with advanced melanoma. Yervoy works by blocking CTLA-4, a different molecule that also keeps the immune system from attacking cancer.
Ribas said he has followed one patient on Yervoy for 12 years now. "She's not supposed to be around, and she's alive and well and melanoma free. That is why we've been doing these immunotherapies," he said.
With Yervoy, Ribas said these types of responses were few and far between. With the new PD-1 drugs, they are much more common, with fewer side effects.
However, an early-stage Bristol-Myers' study released this month showed that 53 percent of patients who got a combination of Yervoy and nivolumab had at least a 50 percent reduction in tumor size, with fewer side effects.
Tim Turnham, executive director of the Melanoma Research Foundation, said combination treatments would make a major difference for patients because they help overcome cancer's "sneaky" ability to evade treatment. But, at this point, he said, "Nobody knows which one is better."
(Editing by Christopher Wilson)
FDA oncology chief Pazdur vows to accelerate 'breakthrough' drug R&D
May 31, 2013 | By John Carroll
CHICAGO--The FDA's oncology chief, Richard Pazdur, used his turn in front of the press mob at ASCO today to make some very public vows about his commitment to the newly launched breakthrough-drug program, that intriguing new classification for certain stellar therapeutic programs that will be extended VIP status at the agency.
Pazdur stressed that any drug development team working on a breakthrough drug will have no trouble getting steady feedback from the agency, and in particular the oncology division he runs. Pazdur and the agency view these drugs as potentially "transformative" therapies capable of changing the course of patients' lives. And the agency is committed to spending time in an "iterative fashion," holding monthly meetings and teleconferences to advise teams on accelerating the whole development process, with a goal of quickening final marketing decisions.
This is going to be a "different communication structure," Pazdur promises, "with much more of a continuous dialogue with sponsors." Together, regulators and development teams can change endpoints, downsize trial sizes in light of the data they're seeing and expedite reviews--adaptive research with regulatory input.
For an industry that broadly views drug development as a 10-year process that can consume an average of a billion dollars per success story, this is big stuff.
But the breakthrough designation seems to have stirred up equal amounts of skepticism and hope in the biopharma community. Still in its early days, I've been studying each announcement for some sign of who's likely to get the backstage pass at the FDA and what it means in real terms. In drug development, and particularly in the cancer field, the focus now is on carving years out of the development process. So if Pazdur's serious--and there's no mistaking his public commitment--there's potential here to do some game-changing redesigns of the clinical trial process.
The FDA, though, can only help. This R&D revolution is being driven by major players, and they're already changing the way cancer drugs are developed. Earlier today I was talking to Dr. Nancy Valente, the senior clinician heading up Genentech's hematology franchise, who's been involved in the development of obinutuzumab or GA101, a fascinating new therapy that applied some intriguing glycoengineering technology to craft an antibody that could directly terminate cancer cells at the same time it spurs the immune system to send its own killer team to participate in the massacre. That kind of immunotherapy-plus process has produced some outstanding progression-free survival data, giving Roche ($RHHBY) a good shot at coming up with a superior successor to Rituxan, a drug that earned about $7 billion last year.
At the time Roche started its randomized Phase III study, they had data on only 33 patients, including some mid-stage single-arm results. Investigators started off by testing six patients to see if there were any big red safety flags that would prevent testing, then pushed ahead and essentially cut out the randomized Phase II study that has long been a standard trial feature. At the beginning of the clinical program, investigators paid especially close attention to side effects to make sure they hadn't overlooked anything in the abbreviated safety study.
Doing that cut anywhere from two to three years out of the development process, says Valente. The clinical program, from first dosing to its first regulatory filing, took less than 6 years.
It's no surprise GA101 is one of the 8 experimental cancer drugs to win the breakthrough designation. Of course, word of that only came down a couple of weeks ago, so no one on the Roche/Genentech side has had much of a chance to determine exactly how breakthrough status is going to change things. The drug has already been filed for an approval for CLL, but there are also follow-up studies underway for non-Hodgkin's lymphoma.
Roche is one of a number of major pharma players to win this designation, underscoring that Pazdur initially is going to be most comfortable handing out breakthrough passes to the deep-pocket outfits he's worked with for years. Trust goes a long way in any field. In drug development, it's crucial. And with Pazdur, it's critical. Anyone who follows the drug approval process knows Pazdur can make or break a drug, and he personally won't hesitate to come out swinging during a panel review if he's already made his decision to squash an application (just ask Aveo).
That kind of commitment indicates that Pazdur will keep his promises on shepherding the drugs he believes qualify as breakthrough drugs. Conversely, rival developers excluded from the VIP crowd are likely to view the designation as rank favoritism. And they may find it tougher than ever to win an audience with regulators, let alone an approval. That may be particularly worrisome for smaller companies looking to pioneer a new therapy, especially without that regular feedback that Pazdur's team is directing elsewhere.
What the FDA gives with one hand, it can take away with the other. And we'll keep looking to see how this plays out. -- John Carroll, Editor-in-Chief. Follow me on Twitter and LinkedIn.
Read more: FDA oncology chief Pazdur vows to accelerate 'breakthrough' drug R&D - FierceBiotech http://www.fiercebiotech.com/story/fda-oncology-chief-pazdur-vows-accelerate-breakthrough-drug-rd/2013-05-31#ixzz2V4KhBJBR
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