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I am not a tax expert but I believe it isn't a coincidence that the company they made the deal with is domiciled in Ireland.
I believe this was done more for the Irish domain to lower their effective tax rate than to get access to this drug.
jq1234 Thursday, 04/24/14 11:01:32 AM
Re: ghmm post# 177242
Post # of 177264
Bold move by CELG to expand its autoimmune/inflammation space: ORAL ASO drug!!! 7-day treatment, 100% clinical response, 80% clinical remission, small sample size, but I assume CELG had seen larger ph2 randomized placebo controlled trial result, could be a big drug. There must be multiple bidders to get to such high upfront.
GED0301 ph1 results:
www.nature.com/mt/journal/v20/n4/pdf/mt2011290a.pdf
http://www.ncbi.nlm.nih.gov/pubmed/22971085
But what I really want to know is how they deal with folks with multiple issues - those unfortunate patients that were both sucked into a jet engine and bitten by their parrot. :)
They made need a code for being sucked into a jet engine while being bitten by a parrot. There should be some kind of discounted rate for that.
Code V97.33XD: sucked into a jet engine, subsequent encounter. (Subsequent encounter? You mean someone actually didn't learn his lesson the 1st time?
I think you are joking but I am not sure. If you are sucked into an engine there couldn't be a next time.
OPHT/REGN—An interesting slide [from OPHT’s R&D Day] concerned an 11,000-patient study showing that after two years of vegf injections the patient's vision is actually below the baseline of where they were at the start of treatments.
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The studies in OPHT’s slide set were observational, not randomized controlled trials; thus there may have been bias in selecting the databases to include in those studies. Moreover, some patients in the observational studies received little or no treatment beyond two years, so it’s not surprising that mean visual acuity declined to worse than baseline at some point after stopping treatment. No one has asserted that Lucentis/Avastin/Eylea enable a patient to retain the best visual acuity achieved during treatment after treatment is stopped.
Quote:
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I knew that many patients stopped responding to treatment after two years, but did not know that it was most of them.
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There’s nothing medically special about the two-year time point—it’s simply the duration of VEGF treatment commonly used in practice.
Quote:
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look at your response. you are saying that it is no surprise that the treatment didn't work after treatment stopped after two years. Is it reasonable to you that the doctor who makes money everytime he gives an injection and the patient who would go blind, would voluntarily stop the treatments if they were working.
Doesn't it make more sense the the treatment stops working at about two years, so the doctor gives up and the patient figures that it doesn't make sense to keep coming in to the office for an eye injection if the treatment isn't working.
Saying there is nothing special about the two-year time point, it is just the duration of treatment used in practice, doesn't make sense unless the treatment only works for two years.
I don't think people wake up after two years and say, I delayed blindness for two years, I don't mind going blind now.
found this article regarding pdgf role in amd
http://bmctoday.net/retinatoday/pdfs/0313RT_SF_Dugel.pdf
opht analyst day
Very interesting. The docs were all promoting the fact that it is great to have a choice of the vegf inhibitior to use with Ophthotech's drug.
They were saying they could use avastin with it, because all the VEGF's were basically the same.
An interesting slide concerned an 11 thousand patient study showing that after two years of vegf injections the patient's vision is actually below the baseline of where they were at the start of treatments.
After listening to many doc and company discussions on the subject this was never brought out.
I knew that many patients stopped responding to treatment after two years, but did not know that it was most of them.
I wonder that if that is why REGN was down yesterday, and not really the pcsk9.
On a different day regn could have gone up on the news relating to the loss of memory because they didn't see it in any of their trials, so it was probably a competitor. It doesn't have to be a class effect
arql
what are your thoughts. It seems cheap here. What do you think their liver cancer trial will show
Regarding image studies for pb2, Amarin also did studies in Huntingtons and had excellent image studies with their omega 3 drug
my big problem with the trial is the rate of decline in the placebo group. The progression of huntingtons can take 10 to 20 years. I am amazed the the placebo group declined so rapidly in just six months.
Richard III was King of England for two years, from 1483 until his death in 1485 in the Battle of Bosworth Field. He was the last king of the House of York and the last of the Plantagenet dynasty. His defeat at Bosworth Field, the last decisive battle of the Wars of the Roses, symbolizes the end of the Middle Ages in England. He is the subject of the play Richard III by William Shakespeare. At the time of the death of his father and older brother, at the Battle of Wakefield in 1460, Richard, who was eight years old, was sent by his mother, the Duchess of York, to the Low Countries, accompanied by his elder brother George. At this time Richard was named Duke of Gloucester as well as being made a Knight of the Garter and a Knight of the Bath. Richard was then sent to Warwick's estate at Middleham for his knightly training. Richard stayed at Middleham until early 1465, when he was twelve. During his adolescence, he developed idiopathic scoliosis.
Richard became involved in the rough politics of the Wars of the Roses at an early age. His brother, Edward appointed him the sole Commissioner of Array for the Western Counties in 1464, when he was 11. By the age of 17, he had an independent command. At 18 years, Richard played crucial roles in battles that resulted in Edward's restoration to the throne in spring 1471.
Polydore Vergil and Thomas More write about King Richard, emphasizing Richard's outward physical deformities as a sign of his inwardly twisted mind. More describes him as “little of stature, ill-featured of limbs, crook-backed and hard-favored of visage“. Vergil also says he was “deformed of body with one shoulder higher than the right“. Both emphasize that Richard was devious and flattering, while planning the downfall of both his enemies and supposed friends. Richard's good qualities were his cleverness and bravery. All these characteristics are repeated by Shakespeare, who portrays him as having a hunch, a limp and a withered arm.
During Richard's reign, the historian John Rous praised him as a “good lord“ who punished “oppressors of the commons“, adding that he had “a great heart“. After his death, Richard's image was tarnished by propaganda fostered by his Tudor successors (who sought to legitimize their claim to the throne). Richard's reputation as a promoter of legal fairness persisted, however. William Camden in his Remains Concerning Britain (1605) states that Richard, “albeit he lived wickedly, made good laws“. Francis Bacon also states that he was “a good lawmaker for the ease and solace of the common people“. Despite this, the image of Richard as a ruthless power-grabber remained dominant in the 18th and 19th centuries.
Richard's Council of the North, greatly improved conditions for Northern England, as commoners of that region were formerly without any substantial economic activity independent of London. Its descendant position was Secretary of State for the Northern Department. In December 1483, Richard instituted what later became known as the Court of Requests, a court to which poor people who could not afford legal representation could apply for their grievances to be heard. He also introduced bail in January 1484, to protect suspected felons from imprisonment before trial and to protect their property from seizure during that time. He founded the College of Arms in 1484, he banned restrictions on the printing and sale of books, and he ordered the translation of the written Laws and Statutes from the traditional French into English.
Richard III's remains received burial without pomp, but the original tomb is believed to have been destroyed during the Reformation, and the remains were lost for more than five centuries. In 2012, an archaeological excavation was conducted on a city council car park using ground-penetrating radar on the site once occupied by Greyfriars, Leicester. The University of Leicester confirmed on 4 February 2013 that a skeleton found in the excavation was, beyond reasonable doubt, that of Richard III. This conclusion was based on a combination of evidence from radiocarbon dating, comparison with contemporary reports of his appearance, and a comparison of his mitochondrial DNA with that of two matrilineal descendants of Richard III's eldest sister, Anne of York. The genomes of King Richard III and one of his family's direct living descendants are to be sequenced in a project funded by the Wellcome Trust, the Leverhulme Trust and Professor Sir Alec Jeffreys. The project will be led by Dr Turi King of the Department of Genetics at the University of Leicester. The aim is to shed new light on the ancestry and health of the last king of England to die in battle, and provide a complete archive of information that historians, scientists and the public will be able to access and use. His remains and any samples taken from them are scheduled to be reinterred and for this reason, Dr Turi King and colleagues plan to sequence his genome and make it freely accessible as a resource to researchers wishing to analyze and interrogate its genetic information.
Richard III will be one of only a small number of ancient individuals to have had their genomes sequenced. Others include Otzi the Iceman, Neanderthal specimens, a Denisovan and a Greenlandic Inuit and a hunter gatherer from Spain. Richard III will be the first ancient individual of known identity to have his genome sequenced. This will be carried out in collaboration with Professor Michael Hofreiter at the University of Potsdam.
Analysis of Richard III's genome will allow insight into his genetic make-up, including susceptibility to certain diseases, hair and eye color, and as the genetic basis of other diseases becomes known, these too can be examined for. It is also expected to shed light on his genetic ancestry and relationship to modern human populations. In addition, next generation sequencing technologies will allow the researchers to detect DNA from other organisms such as pathogens. Whole genome sequencing from Otzi the Iceman found the first known human infection with Lyme disease, for example.
Dr King says: “It is an extremely rare occurrence that archaeologists are involved in the excavation of a known individual, let alone a king of England. At the same time we are in the midst of a new age of genetic research, with the ability to sequence entire genomes from ancient individuals and with them, those of pathogens that may have caused infectious disease. Sequencing the genome of Richard III is a hugely important project that will help to teach us not only about him, but foment discussion about how our DNA informs our sense of identity, our past and our future.“ In addition to sequencing the remains of Richard III, Dr King and colleagues will also sequence one of his living relatives, Michael Ibsen. An initial analysis of the DNA of his mitochondria -- the batteries that power the cells in our bodies -- which is passed down the maternal line, confirmed the genealogical evidence that Ibsen and Richard III shared the same lineage. A more detailed analysis is due to be published shortly. This new project will allow researchers to look for any other segments of DNA that these distant relatives share.
Researchers based at the University of Cambridge and the University of Leicester have also uncovered evidence that Richard III suffered from a roundworm (Ascaris lumbricoides) infection, according to a Clinical Picture published in The Lancet. A team of researchers led by Dr Piers Mitchell, of the Department of Archaeology and Anthropology at the University of Cambridge, UK, used a powerful microscope to examine soil samples taken from the skeleton's pelvis and skull, as well as from the soil surrounding the grave. The microscope revealed multiple roundworm eggs in the soil sample taken from the pelvis, where the intestines would have been situated in life. However, there was no sign of eggs in soil from the skull, and very few eggs in the soil that surrounded the grave, suggesting that the eggs found in the pelvis area resulted from a genuine roundworm infection during his life, rather than from external contamination by the later dumping of human waste in the area.
Roundworms are parasitic nematodes, which infect humans when people ingest their eggs via contaminated food, water, or soil. Once eaten, the eggs hatch into larvae, which migrate through the tissues of the body to the lungs where they mature. They then crawl up the airways to the throat to be swallowed back into the intestines, where they can grow into adults around a foot long. Roundworm infection is thought to be one of the commonest health conditions in the world, affecting up to a quarter of all people globally, although it is rare in the UK today.
According to Dr Mitchell, “Our results show that Richard was infected with roundworms in his intestines, although no other species of intestinal parasite were present in the samples we studied. We would expect nobles of this period to have eaten meats such as beef, pork and fish regularly, but there was no evidence for the eggs of the beef, pork or fish tapeworm. This may suggest that his food was cooked thoroughly, which would have prevented the transmission of these parasites.“ Dr Jo Appleby, Lecturer in Human Bioarchaeology at the University of Leicester, UK, said: “Despite Richard's noble background, it appears that his lifestyle did not completely protect him from intestinal parasite infection, which would have been very common at the time.“
.
In addition to abdominal infection, Richard III may have undergone painful medical treatments for his spinal curvature, according to research from a University of Leicester researcher. The remains of Richard III discovered by University of Leicester archaeologists revealed that the King suffered from severe scoliosis, which he probably developed in early adolescence. Scoliosis -- a lateral or side-to-side curvature of the spine -- can be a very painful condition to live with. Dr Mary Ann Lund, of the University's School of English, has carried out research into the kinds of scoliosis treatments available at the time Richard III was alive. But some of the treatments practiced in the late medieval period would have themselves caused sufferers a lot of anguish. Among the “cures“ practiced was traction -- the same principle on which “the Rack“ worked as an instrument of torture. The patient would be tied under the armpits and round the legs. The ropes were then pulled at either end, often on a wooden roller, to stretch the patient's spine. This treatment would probably have only been available to those who could afford it. Richard III would certainly have been able to afford the highest levels of medical care available -- and his physicians would have been well aware of the standard “traction“ methods for treating the condition.
Dr Lund charted the influence of Greek philosopher Hippocrates -- who developed early prototype methods of dealing with spinal disorders -- to the 11th century Persian polymath Avicenna, who utilized handed down treatments from the Greeks. These treatises on medicine and philosophy were well regarded in Medieval Europe. Avicenna's documents on using traction in scoliosis treatment would have been widely read and practiced by doctors in Richard III's lifetime. Avicenna also advocated the massage techniques practiced in Turkish baths, and herbal applications, as treatments for back disorders. In the longer term, patients might wear a long piece of wood or metal in an attempt to straighten their back. Dr Lund said: “Scoliosis is a painful illness, and Richard would have been in quite a lot of pain on a daily basis. These methods could also have been very painful -- but people would have expected treatments to be unpleasant. Medical practices could exacerbate conditions rather than improving them. These treatments would have only been open to people in the upper echelons. Richard would have probably received these treatments because he was a member of the nobility.
Later methods of treatment for scoliosis included the orthosis, which was developed by French physician Ambroise Pare in the late 16th century. This was a tightly fitting metal corset for treating scoliosis made by an armourer, which would have been worn by patients to brace the skeleton in an attempt to correct the curvature of the spine. Source: The University of Leicester; ScienceDaily.com
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Journal Reference: Piers D Mitchell, Hui-Yuan Yeh, Jo Appleby, Richard Buckley. The intestinal parasites of King Richard III. The Lancet, 2013; DOI: 10.1016/S0140-6736(13)61757-2
rtrx from Jim Birchenoff of BMO
Questions have arisen recently regarding potential competition to Intercept's obeticholic acid (OCA) from other bile acid therapeutics, following Retrophin's acquisition Manchester Pharmaceuticals of Chenodal (chenodeoxycholic acid or CDCA a synthetic bile acid). With approval of Chenodal in the US for treatment of gallstones and use in cerebrotendinous xanthomatosis (CTX), the question is whether Chenodal could be studied successfully in primary biliary cirrhosis (PBC) and non-alcoholic steatohepatitis (NASH). Recall, that ICPT has completed two successful randomized phase 2 studies and is set to report phase 3 POISE data in 2Q14, with early stoppage of a large phase 2B study by the NIH for efficacy at improving NAFLD activity scores.
Our View:
* We see limited competitive risk from synthetic bile acids like CDCA in PBC and NASH.
* We would note that CDCA levels are normal in PBC and NASH, as opposed to a deficiency state in CTX, and use of exogenous CDCA could be limited by dose-limiting detergent effects on the liver.
* Current use of Urso in PBC is specifically designed to displace CDCA and prevent detergent toxicity that occur in conditions of cholestasis and use of CDCA in gallstones is associated with elevated liver enzymes.
* Ultimately, OCA is the most potent FXR agonist in development for PBC and NASH, and with 100x greater potency than CDCA, we expect OCA to be the bile acid therapeutic of choice for cholestatic liver disease.
It was started by a fellow named Gene Inger. He also used to say the best investment a person could make was buying beachfront property in Florida because once it was gone, it was gone.
he was a smart guy
CNDO,
Regarding the press release, they mention positive results in 3 outcome measures, they may have measured 10 outcome measures and the other 7 measures may have favored the placebo, but that is left out.
I am not saying that is what happened, but it could have. Why didn't they mention what happened with the other outcome measures?
In the first 5 patients that completed the study, there was a statistically significant separation from placebo in favor of TSO on three measures of disease: the Montefiore-Einstein Rigidity Scale (MERS), the Repetitive Behavior Scale-Revised (RBS-R) Sameness Scale, and the Social Responsiveness Scale (SRS)-Repetitive Behaviors Scale. The treatment was well tolerated.
There has been nothing in their data to indicate that the product works
when ACAD put out the their original failed data I believe they mentioned that it worked in parkinson's patients but it wasn't a believeble subgroup, but it has shown to work there.
Coronado Biosciences Announces IND Submission for TSO for the Treatment of Moderate to Severe Chronic Plaque Psoriasis
It may work as well as the placeobo
DDXS. people do not know why they own stocks
Gsk's trial took all comers. The pl-pla2 test is more important if it works in the pre defined subgroup of patients with high levels of pl-pla2. This is a 30,000 patient phase 3 trial, if 10.000 patients with high levels of pl-pla2 show statistical significance GSK would submit an NDA using the diagnostic as a test to be used before the drug is prescribed.
It is better for diadexus that the trial failed if the subgroup works than if the trial was successful
diaDexus, Inc. Provides Update Related to Topline Lp-PLA2 Inhibitor Data Reported by GlaxoSmithKline
--STABILITY Trial Did Not Meet Primary Endpoint; Reduction in Some Secondary Endpoints Observed--
--Company Believes PLAC® Test Business Remains Strong--
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- diaDexus, Inc. (OTCQB:DDXS), today provided an update to their PLAC® Test business related to the topline Phase 3 STABILITY trial results announced by GlaxoSmithKline (GSK). GSK reported in a press release this morning that their trial evaluating the Lp-PLA2 inhibitor, darapladib, did not meet its primary endpoint measure, which was time to first occurrence of any major adverse cardiovascular event (MACE). GSK has indicated that patient sub-group analyses of the STABILITY trial will be forthcoming. diaDexus' patent protected research use only activity assay was used in the study to measure patient Lp-PLA2 activity at baseline and during follow-up visits.
Lp-PLA2 levels aid in cardiovascular disease (CVD) risk assessment independent from other biomarkers—providing insight into the relationship between inflammation, atherosclerosis and CVD outcomes. GSK will continue investigations into the role of Lp-PLA2 inhibition as they analyze the data from STABILITY, including evaluation of the patient sub-groups and await the results of a second Phase 3 study of darapladib in acute coronary syndrome, termed SOLID-TIMI 52 expected to be available in 2014.
"We look forward to the anticipated sub-group analysis of the STABILITY study and the SOLID-TIMI 52 study results. There is a high unmet need in coronary heart disease and we continue to believe there is an important role for Lp-PLA2 evaluation. Our FDA-cleared PLAC® Test ELISA Kit continues to be a critical tool in assessing cardiovascular disease risk for millions of patients," said Brian Ward, Ph.D., diaDexus' president and chief executive officer. "Measurement of Lp-PLA2 with the PLAC Test is increasing among primary care physicians, and we will continue to educate the physician community on the importance of Lp-PLA2 and the risks of elevated Lp-PLA2 levels."
"Many Americans do not realize that risk factors other than cholesterol levels contribute to major cardiac events, but in fact, 50 percent of all heart attacks and strokes occur in people with normal lipid levels," said Robert Schott, M.D., MPH, FACC, diaDexus' vice president of Medical Affairs. "Lp-PLA2 as a CVD biomarker is well-studied and has been characterized as a risk factor for coronary heart disease. Identifying active cardiovascular inflammatory disease, indicated with increased levels of Lp-PLA2, can make a difference in patient care. Because of this, the PLAC Test remains an important, actionable tool and a valuable guide for patient management."
About GSK's STABILITY and Phase 3 program
The STABILITY trial (STabilisation of Atherosclerotic plaque By Initiation of darapLadIb TherapY) is a GSK sponsored global Phase 3 cardiovascular outcomes study designed to evaluate the clinical safety and efficacy of treatment with darapladib versus placebo, when added to the standard of care, on the incidence of MACE in high-risk patients with chronic coronary heart disease (CHD).
A second GSK sponsored Phase 3 study, SOLID-TIMI 52 (The Stabilisation of pLaque Using darapladib -Thrombolysis In Myocardial Infarction 52), evaluating the efficacy of darapladib in men and women with acute coronary syndrome, is also ongoing.
About diaDexus, Inc.
diaDexus, Inc., based in South San Francisco, California, develops and commercializes proprietary cardiovascular diagnostic products addressing unmet needs in cardiovascular disease. The company's PLAC® Test ELISA Kit is the only blood test cleared by the FDA to aid in predicting risk for both coronary heart disease and ischemic stroke associated with atherosclerosis, the #1 and #3 causes of death, respectively, in the United States. The company's PLAC® Test for Lp-PLA2 Activity, a CE-marked test, is an indicator of atherosclerotic cardiovascular disease, the #1 cause of death in Europe. diaDexus is ISO 13485 certified and manufactures the PLAC Test for Lp-PLA2 Activity on-site. For more information, please visit the company's website at www.diaDexus.com.
Forward-Looking Statements
This release contains forward-looking statements based on current expectations and assumptions which entail various risks and uncertainties that could cause actual results to differ materially from those expressed in such forward-looking statements. Important factors known to diaDexus that could cause actual results to differ materially from those expressed in such forward-looking statements include whether the results of the STABILITY trial, including any subgroup analysis, will negatively impact the current or future growth of the PLAC Test ELISA Kit, whether levels of Lp-PLA2 will continue to play a role in the evaluation of coronary heart disease; whether GSK will continue support of PLAC Test, whether future studies may support the scientific results observed in the LIPID study; the timing of the submission of the PLAC Test for Lp-PLA2 Activity and whether, following submission, it can achieve FDA clearance and if even if cleared, the ability to gain acceptance in the marketplace; and the timing of data from a longitudinal cardiovascular disease study necessary to support submission for clearance to the FDA. Additional factors that could cause diaDexus' results to differ materially from those described in the forward-looking statements can be found in diaDexus' most recent quarterly report on Form 10-Q and other reports filed with the Securities and Exchange Commission, and available at the SEC's web site at www.sec.gov. The information set forth herein speaks only as of the date hereof, and except as required by law, diaDexus disclaims any intention and does not assume any obligation to update or revise any forward looking statement, whether as a result of new information, future events or otherwise.
diaDexus, Inc.
Brian E. Ward, Ph.D., 650-246-6400
CEO
Jean-Frédéric Viret, Ph.D., 650-246-6400
CFO
investors@diadexus.com
or
Media / Investor Relations:
BrewLife
Nicole Foderaro, 415-946-1058
nfoderaro@brewlife.com
If you read the newsletter, it isn't just one patient.
we will know in May how it turns out. I am not betting the farm but I would rather see this than not. I felt I should point it out to the board but it really doesn't matter to me whether you believe it or not.
Ohrp. this is the page that was taken down
Clinical Trial Patient Success Story - Florida Eyewww.fleyedocs.com/clinical-trial-patient-success-story/?CachedOct 31, 2013 - Currently, over his five months of check-ups, Bob did not require a single Lucentis injection. “The drops are really doing what they are ...
They have one patient that recieved the first injection and has gone five months without another one.
It was on his site but it was taken down. the following is not very common
The results thus far have been amazing, as many of the study participants have been able to forego monthly injections as a result of the drug combination used in the nine month study.”
ohrp...I do not think this is a placebo effect
http://www.fleyedocs.com/current-upcoming-clinical-trials/
From Dr. Randy Katz:
We have one study for wet AMD for sub responders to Lucentis, Avastin, Eyelea or Macugen. This study is for patients who have received 3-10 injections in the past year with the last 3 treatments being with the same drug consistently.
We are now enrolling for treatment naïve wet AMD patients for Eyelea. Phase IV, participants are guaranteed to get Eyelea. No AMD in the fellow eye allowed.
Our second study for wet AMD using eye drops that potentially will cut down on the need for monthly injections is still enrolling participants. The study combines Lucentis and the study eye drops Squalamine Lactate, administered topically. “The hope is that with the administration of these eye drops twice daily, the frequency of intravitreal injections will be greatly reduced,” said Dr. Katz. “The results thus far have been amazing, as many of the study participants have been able to forego monthly injections as a result of the drug combination used in the nine month study.”
The study is placebo controlled (for the drops). All receive Lucentis but need to be covered by insurance to pay for the Lucentis. Participants need to come for 12 visits over 38 weeks.
tgtx
You may not like to hear this but I own it also. I added yesterday.
The CD 20 drug has had some great data.
The company raised 30 million dollars at 6.05 three months ago. The drop doesn't make sense in this bull market.
psti,,,take a look at tigenix
if it was trading here it would have a much higher market cap
http://www.tigenix.com/
Late stage pipeline
Six minute walk is a rediculous way to measure benefit in the disease
proof of that is the two children that became non ambulatory even though their dystrophin levels increased
They can't walk but they aren't entering the olympics. They die of heart failure because their heart muscle dies. Chances are the increased dystrophin will allow them to live longer, but I guess they are no longer on the drug so they will die.
from the Intrexon s-1 what has made the company worth twice what is was three months ago when they raised money at 7.88 share
(i) On or about February 19, 2010, we sold 5,250,083 shares of our Series D preferred stock at a purchase price per share of $3.38 for
an aggregate purchase price of $17,745,281.
(ii) On or about October 29, 2010, we sold 5,990,711 shares of our Series D preferred stock at a purchase price per share of $3.38 for
an aggregate purchase price of $20,248,603.
(iii) On or about January 6, 2011, we sold 5,313,629 shares of our Series D preferred stock at a purchase price per share of $3.38 for an
aggregate purchase price of $17,960,074.
(iv) Between February 18, 2011 and February 25, 2011, we sold 3,249,262 shares of our Series D preferred stock at a purchase price
per share of $3.38 for an aggregate purchase price of $10,982,502.
(v) On or about May 26, 2011, we sold 19,047,619 shares of our Series E preferred stock at a purchase price per share of $5.25 for an
aggregate purchase price of $100,000,000 less $2,617,235 paid to Perella Weinberg Partners as placement agent.
(vi) On or about January 10, 2012, we sold 9,523,810 shares of our Series E preferred stock at a purchase price per share of $5.25 for an aggregate purchase price of $50,000,000.
(vii) On or about April 12, 2012, we sold 4,761,905 shares of our Series E preferred stock at a purchase price per share of $5.25 for an aggregate purchase price of $25,000,001.
(viii) Between October 26, 2012 and November 13, 2012, we sold 4,761,905 shares of our Series E preferred stock at a purchase price per share of $5.25 for an aggregate purchase price of $25,000,001.
(ix) On or about March 1, 2013, we sold 8,178,964 shares of our Series F preferred stock at a purchase price per share of $7.88 for an aggregate purchase price of $64,409,342 less $1,199,433 paid to Barclays as placement agent and $300,000 to White Rock Capital, Inc. as client referral fees.
(x) On or about April 30, 2013, we sold 10,868,655 shares of our Series F preferred stock at a purchase price per share of $7.88 for an aggregate purchase price of $85,590,658 less $100,000
first of all, what the fda says at a meeting doesn't mean very much. Most companies wait for the written transcript from the meeting before issuing guidance to the street with a conference call or a press release.
I am not sure why they had a conference call the day after the meeting.
No company we know filed accelerated approval without FDA's explicit yes for filing which is why very few NDA/BLA filed under subpart H regulation.
I think it was pretty clear from the conference call that SRPT did not have an explicit yes
charles duncan is looking for the next banking deal
Sarepta will probably announce that they will file the NDA
Almost all companies that have meetings like this say that they were given guidance by the FDA that they can file.
The problem is that the actual transcripts of the meetings are never made public so ninety percent of the time the fda probably says you can file the NDA but that doesn't mean the drug will be approved
Hemispherix has filed many NDA's. Sometimes their NDA's have been approved for filng, but when it comes time for the approval the answer is no.
if they submit a copy of the letter as an exhibit or quote from the letter I will accept it, otherwise I think approval is a long shot.
Glazo's drug has much more data. This drug hasn't been in enough patients for the fda to know if there is some kind of toxicity that hasn't shown up yet.
Now I know why Guantanamo is still open
If we can't perform non human primate tests on monkeys the residents of Guantanamo will have to do.
1. Fewer chimps to be used in NIH-backed biomedical research
By Emily Mullin Comment | Forward | Twitter | Facebook | LinkedIn
The National Institutes of Health announced Wednesday its plans to significantly reduce the number of chimpanzees it uses in NIH-funded biomedical research.
The decision means that NIH will retire most of its chimps--about 310--to sanctuaries over the next few years. It will keep but not breed up to 50 chimpanzees for future research projects that meet standards set by a 2011 Institute of Medicine (IOM) report. The advisory committee submitted its final recommendations to NIH in January, which proposed closing 16 current research projects, including 6 of 9 biomedical projects in immunology, bioterrorism, infectious agents and hepatitis.
NIH Director Dr. Francis Collins accepted most of the recommendations put forth by an independent advisory council and those outlined in the IOM report, published in December 2011. Collins said new scientific methods and technologies largely negate the need for chimpanzee subjects in biomedical experiments. He also cited ethical considerations as another reason for the policy change.
"After extensive consideration with the expert guidance of many, I am confident that greatly reducing their use in biomedical research is scientifically sound and the right thing to do," Collins said in a statement.
The IOM report did not call for an outright ban on chimpanzee research, but it endorsed putting a stop to the use of chimps for most invasive medical research and laid out a set of criteria as to when, if ever, current and future medical studies should use chimps.
In a telephone press conference, Collins said to reporters that the agency's decision represents "a compassionate era" and one that "recognizes the importance of adapting medical research."
The decision was long expected by the research community, but some scientists have noted that studies involving a potential vaccine for hepatitis C might still require the use of chimps. Collins said in the conference call that noninvasive research, including behavioral and genomics research, could still be done with chimps under the new guidelines.
What's not yet known is where the retired chimps will live out the rest of their days. Currently, NIH is legally limited as to the amount of financial resources the agency may allocate toward retiring chimpanzees and caring for them in the Federal Sanctuary System, the home of federally owned chimps since 2002
Shark Eye Drops To Cure Blindness?
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http://www.medicaldaily.com/articles/16656/20130619/shark-eye-drops-ohr-pharmaceuticals-shark-liver-squalamine-blindness-cure.htm
Shark Eye Drops To Cure Blindness? Pharma Companies Test Squalamine, Chemical Found In Liver, To Treat AMD
Researchers from OHR Pharmaceuticals have developed shark eye drops from the spiny dogfish's liver that could defend against blindness.
BY JUSTIN CABA | JUN 19, 2013 11:13 AM EDT
The medical research and development company OHR Pharmaceutical Inc. is currently in the middle of clinical trials to test shark eye drops derived from the liver of spiny dogfish. Researchers are confident a molecule found in the animal's organ may help treat and prevent wet age-related macular degeneration (AMD), both the U.S. and Britain's leading cause of blindness.
Wet AMD occurs after abnormal blood vessels behind the retina begin to grow under the macula causing the outflow of blood and fluid. The result is swelling and possible scarring around the retina. Wet AMD is regarded as more severe compared to dry AMD and affects around 10 percent of all people with age-related macular degeneration, the National Institutes of Health reports.
The molecule that has helped OHR develop this unique solution is known as squalamine, and it comes from the stomach and liver of the spiny dogfish shark. The chemical has been accredited with the shark's essential ability to ward off bacterial infection.
The research team at OHR believes squalamine is able to block certain proteins in the body that facilitate the growth of abnormal blood vessels. Similar to current treatments for AMD, the synthetic squalamine solution was originally administered by way of injections directly into the eye's bloodstream. However, during further testing, the compound was reformulated into a more convenient eye drop.
According to a statement on OHR's website, "Squalamine had previously been evaluated for the treatment of wet-AMD using an intravenous formulation in over 250 patients. The trials demonstrated that the molecule had biologic effect and maintained and improved visual acuity outcomes, with both early and advanced lesions responding."
"The program had been taken into to a phase III study with a special protocol assessment and fast track status from the US FDA, but had been discontinued due to the commercial challenges of a chronic weekly IV infusion for this patient population." Less
its saving grace is that in the clinical trial patients on tobi were probably taking it two times a day and in the real world there is a lot of non compliance
so the arikace was probably better than tobi once a day.
. Scientists dispute PTC's claim on muscular dystrophy drug mechanism
By John Carroll
The clause that I put in bold print was an absolutely rediculous statement by the writer. If they hadn't hid the information the IPO wouldn't have gotten done and they may have gone out of business. They are better off defending the stock price than the alternative. The investment banks should get sued.
Right on the heels of a $125 million IPO, PTC Therapeutics ($PTCT) has another public relations problem on its hands. The Scientist is reporting that a group of investigators have completed an in vitro study of their lead treatment and came to a startling conclusion: It doesn't work the way PTC has said it does.
As The Scientist explains, PTC Therapeutics has said that ataluren--or PTC124--can effectively treat a subset of cystic fibrosis or Duchenne muscular dystrophy patients by correcting the translation of specific proteins, a read-through process deemed critical in treating these diseases.
"From a basic mechanistic standpoint, it doesn't look like [PTC124 is causing] translational read-through, certainly in our assays," Stuart McElroy, a scientist at the University of Dundee in Scotland and an author of the paper, tells The Scientist.
The company, however, defended its work.
"Numerous independent laboratories have provided confirmation of our results, demonstrating ataluren's read-through activity in studies using reporters as well as multiple animal and cell-based nonsense mutation disease models," notes a company statement.
David Bedwell at the University of Alabama, who's done consultancy work for PTC, also said that mouse studies have shown that there's a read-through.
PTC has already had to contend with some discomforting revelations in its S-1. After touting the fact that PTC had filed for a European approval late last year, company officials stayed mum when regulators came back to say that their first review led them to conclude that the company didn't have the necessary efficacy data. And the company has had to concede that a Phase IIb as well as a Phase III study both failed primary endpoints, leaving the biotech at work on new Phase III trials for CF and DMD.
This isn't the first time investigators have failed to repeat key research work backing a lead program. But for PTC--which now has a stock price to defend--it comes at a particularly bad time.
- here's the article from The Scientist
Stem Cell Therapeutics (SSS.V) (SCTPF) is a completely new story to hit the United States. It is one of only three public companies developing therapeutics targeting cancer stem cells. The other companies are Verastem (VSTM) and Stemline (STML). Each of those companies have a market capitalization of well over two hundred milliion dollars.
SCTPF has two portfolio products. One is based on the science generated by Dr. Aaron Shimmer, a clinician scientist from the University of Toronto and Prince Margaret Hospital. In his highly awarded career he has prioritized repurposing currently marketed drugs for their activity against cancer stem cells. The company has licensed his clinical stage asset, a reformulated version of Pfizer's drug Tigecycline, for which clinical data is expected in early Q3 2013.
SCTPF's other cancer stem cell product has been developed by Dr. John Dick, Phd, who is credited with the initial identification of Leukemic Stem Cells. Our product is a CD47/SIRPa fusion protein. Dr. Irving Weissman of Stanford University is working on a monoclonal antibody targeting CD47, and he has validated the target. Dr. Weissman has just received a twenty million dollar grant from the California Institute for Regenerative Medicine to continue his research. Last year a private company, Inhibrx, entered into an option and license agreement with Celgene. The deal potential is in excess of five hundred million dollars. Evidently Celgene really wanted to get access to that antibody.
Both docs are on the company SAB and follow on products are expected to be collaborated on.
Of note, the Chairman of SCTPF, David Allan, is the person that brought these two assets into this company. David was the CEO of YMI at the time it acquired an Australian company by the name of Cytopia for twelve million dollars. YMI was sold three years later for over five hundred million dollars to Gilead.
By the way, most of Verastem's valuation is based on this drug in licensed from Pfizer. This was the terms of the licensing deal.
VS-6063 in-licensed from Pfizer
In July 2012, Verastem in-licensed the worldwide commercial rights from Pfizer for the FAK inhibitor VS-6063 (PF-04554878). Pfizer received a one-time cash payment of $1.5 million and 192,012 shares of VSTM common stock.
PFE is eligible to: Receive up to $2 million in developmental milestones
• Up to $125 million should certain regulatory and commercial sales milestones be met
• High single to mid double digit royalties on future net sales of VS-6063, for 10 years
Please review the attached company powerpoint presentation and get back to me if you would like to discuss this.
Stem Cell Therapeutics (SSS.V) (SCTPF) is a completely new story to hit the United States. It is one of only three public companies developing therapeutics targeting cancer stem cells. The other companies are Verastem (VSTM) and Stemline (STML). Each of those companies have a market capitalization of well over two hundred milliion dollars.
SCTPF has two portfolio products. One is based on the science generated by Dr. Aaron Shimmer, a clinician scientist from the University of Toronto and Prince Margaret Hospital. In his highly awarded career he has prioritized repurposing currently marketed drugs for their activity against cancer stem cells. The company has licensed his clinical stage asset, a reformulated version of Pfizer's drug Tigecycline, for which clinical data is expected in early Q3 2013.
SCTPF's other cancer stem cell product has been developed by Dr. John Dick, Phd, who is credited with the initial identification of Leukemic Stem Cells. Our product is a CD47/SIRPa fusion protein. Dr. Irving Weissman of Stanford University is working on a monoclonal antibody targeting CD47, and he has validated the target. Dr. Weissman has just received a twenty million dollar grant from the California Institute for Regenerative Medicine to continue his research. Last year a private company, Inhibrx, entered into an option and license agreement with Celgene. The deal potential is in excess of five hundred million dollars. Evidently Celgene really wanted to get access to that antibody.
Both docs are on the company SAB and follow on products are expected to be collaborated on.
Of note, the Chairman of SCTPF, David Allan, is the person that brought these two assets into this company. David was the CEO of YMI at the time it acquired an Australian company by the name of Cytopia for twelve million dollars. YMI was sold three years later for over five hundred million dollars to Gilead.
By the way, most of Verastem's valuation is based on this drug in licensed from Pfizer. This was the terms of the licensing deal.
VS-6063 in-licensed from Pfizer
In July 2012, Verastem in-licensed the worldwide commercial rights from Pfizer for the FAK inhibitor VS-6063 (PF-04554878). Pfizer received a one-time cash payment of $1.5 million and 192,012 shares of VSTM common stock.
PFE is eligible to: Receive up to $2 million in developmental milestones
• Up to $125 million should certain regulatory and commercial sales milestones be met
• High single to mid double digit royalties on future net sales of VS-6063, for 10 years
Please review the attached company powerpoint presentation and get back to me if you would like to discuss this.
David Allan was appointed chairman after sctpf's drug failed.
David and the ceo of the private company of Trillium Nicklas Stiernolm, worked together in the past, and I guess Nick felt that David had the contacts to bring the story to the US.
I think the science at this company compares favorably with that of Stemline and especially Verastem.
Stem Cell Therapeutics (SSS.V) (SCTPF) is a completely new story to hit the United States. It is one of only three public companies developing therapeutics targeting cancer stem cells. The other companies are Verastem (VSTM) and Stemline (STML). Each of those companies have a market capitalization of well over two hundred milliion dollars.
SCTPF has two portfolio products. One is based on the science generated by Dr. Aaron Shimmer, a clinician scientist from the University of Toronto and Prince Margaret Hospital. In his highly awarded career he has prioritized repurposing currently marketed drugs for their activity against cancer stem cells. The company has licensed his clinical stage asset, a reformulated version of Pfizer's drug Tigecycline, for which clinical data is expected in early Q3 2013.
SCTPF's other cancer stem cell product has been developed by Dr. John Dick, Phd, who is credited with the initial identification of Leukemic Stem Cells. The product is a CD47/SIRPa fusion protein. Dr. Irving Weissman of Stanford University is working on a monoclonal antibody targeting CD47, and he has validated the target. Dr. Weissman has just received a twenty million dollar grant from the California Institute for Regenerative Medicine to continue his research. Last year a private company, Inhibrx, entered into an option and license agreement with Celgene. The deal potential is in excess of five hundred million dollars. Evidently Celgene really wanted to get access to that antibody.
Both docs are on the company SAB and follow on products are expected to be collaborated on.
Of note, the Chairman of SCTPF, David Allan, is the person that brought these two assets into this company. David was the CEO of YMI at the time it acquired an Australian company by the name of Cytopia for twelve million dollars. YMI was sold three years later for over five hundred million dollars to Gilead.
By the way, most of Verastem's valuation is based on this drug in licensed from Pfizer. This was the terms of the licensing deal.
VS-6063 in-licensed from Pfizer
In July 2012, Verastem in-licensed the worldwide commercial rights from Pfizer for the FAK inhibitor VS-6063 (PF-04554878). Pfizer received a one-time cash payment of $1.5 million and 192,012 shares of VSTM common stock.
PFE is eligible to: Receive up to $2 million in developmental milestones
• Up to $125 million should certain regulatory and commercial sales milestones be met
• High single to mid double digit royalties on future net sales of VS-6063, for 10 years
Stem Cell Therapeutics (SSS.V) (SCTPF) is a completely new story to hit the United States. It is one of only three public companies developing therapeutics targeting cancer stem cells. The other companies are Verastem (VSTM) and Stemline (STML). Each of those companies have a market capitalization of well over two hundred milliion dollars.
SCTPF has two portfolio products. One is based on the science generated by Dr. Aaron Shimmer, a clinician scientist from the University of Toronto and Prince Margaret Hospital. In his highly awarded career he has prioritized repurposing currently marketed drugs for their activity against cancer stem cells. The company has licensed his clinical stage asset, a reformulated version of Pfizer's drug Tigecycline, for which clinical data is expected in early Q3 2013.
SCTPF's other cancer stem cell product has been developed by Dr. John Dick, Phd, who is credited with the initial identification of Leukemic Stem Cells. The product is a CD47/SIRPa fusion protein. Dr. Irving Weissman of Stanford University is working on a monoclonal antibody targeting CD47, and he has validated the target. Dr. Weissman has just received a twenty million dollar grant from the California Institute for Regenerative Medicine to continue his research. Last year a private company, Inhibrx, entered into an option and license agreement with Celgene. The deal potential is in excess of five hundred million dollars. Evidently Celgene really wanted to get access to that antibody.
Both docs are on the company SAB and follow on products are expected to be collaborated on.
Of note, the Chairman of SCTPF, David Allan, is the person that brought these two assets into this company. David was the CEO of YMI at the time it acquired an Australian company by the name of Cytopia for twelve million dollars. YMI was sold three years later for over five hundred million dollars to Gilead.
By the way, most of Verastem's valuation is based on this drug in licensed from Pfizer. This was the terms of the licensing deal.
VS-6063 in-licensed from Pfizer
In July 2012, Verastem in-licensed the worldwide commercial rights from Pfizer for the FAK inhibitor VS-6063 (PF-04554878). Pfizer received a one-time cash payment of $1.5 million and 192,012 shares of VSTM common stock.
PFE is eligible to: Receive up to $2 million in developmental milestones
• Up to $125 million should certain regulatory and commercial sales milestones be met
• High single to mid double digit royalties on future net sales of VS-6063, for 10 years
Squalamine in the Daily Mail
http://www.dailymail.co.uk/health/article-2343533/Shark-pills-save-sight-grow-older.html
Eye drops made from sharks that may prevent you from losing your sight as you grow older
By Pat Hagan
PUBLISHED:18:44 EST, 17 June 2013| UPDATED:20:31 EST, 17 June 2013
Eye drops made from a chemical originally found in sharks' livers could help to prevent blindness.
The drops contain squalamine, a compound produced by sharks to stop them getting infections.
Now a synthetic version of the compound is being trialled as a treatment for age-related macular degeneration (AMD) - Britain's leading cause of blindness, affecting more than 500,000 people.
As well as warding off deadly viruses, squalamine is able to prevent the abnormal growth of blood vessels. It does this by blocking the release of growth factors produced by the body, which help blood vessels grow.
The compound that makes the pill was extracted from the liver of the dogfish shark after scientists started to investigate why sharks rarely get ill
Abnormal growth of blood vessels is one of the things that helps cancerous tumours thrive.
While research on squalamine as a cancer treatment is at an early stage, studies on its ability to prevent sight loss are advanced.
The growth of unwanted blood vessels at the back of the eye is also one of the major reasons for blindness in AMD.
There are drugs to treat the problem, but they have to be injected into the eye once a month in hospital. Drops can be administered once a day by patients.
The condition develops after the age of 50 and is caused by the growth of new blood vessels over the macula, a small oval-shaped area at the back of the eye that helps us pick out visual details.
These blood vessels tend to be fragile and thin-walled, which means they leak fluid that causes scar tissue to form, destroying vision in the centre of the eye.
This makes it difficult to recognise faces, read or watch TV.
Around 90 per cent of cases involve dry AMD, a form of the disease that comes on slowly over several years and for which there is no treatment.
The rest involve wet AMD - because of the leaking of fluid in the eye - which can cause blindness in just three months.
'Squalamine looked so promising after being used on 250 patients with wet AMD that it was fast-tracked for approval by the U.S. Food and Drug Administration.'
Treatment for the wet form involves injections into the back of the eye every four to eight weeks.
Given early enough, these drugs - designed to curb the growth of abnormal blood vessels - can help prevent complete loss of vision, but can't reverse the disease.
Drugs injected into the eye can slow down progression of the disease in 90 per cent of patients and can increase vision in a third.
Squalamine looked so promising after being used on 250 patients with wet AMD that it was fast-tracked for approval by the U.S. Food and Drug Administration.
The compound was extracted from the liver of the dogfish shark after scientists started to investigate why sharks rarely develop illnesses, but is now synthesised in a lab.
It's thought the chemical protects sharks from illness by preventing the growth of bacteria and viruses.
A study at Indiana University, published in the journal Retina, showed the chemical reduced neovascularisation - the growth of unwanted blood vessels - by a third.
It works primarily by blocking the effects of certain proteins that stimulate that growth. But at the time it was being given as a weekly infusion that had to be injected into the bloodstream.
The technique was effective, but was not very convenient and was unpopular with patients.
Now, though, New York firm OHR Pharmaceutical has turned the treatment into eye drops.
It is in phase two trials in the U.S., which means it is being tested for safety and effectiveness
Commenting on the technology, Professor Bernie Chang, honorary secretary of the Royal College of Ophthalmologists, said: 'This could be one of the most exciting developments in treating AMD.
'If it works as well as current drugs, it would mean patients would not have to come to hospital for injections into their eyes. But we need to be sure it works and does not have major side-effects.'
what I am saying is his numbers are wrong