Also I do not understand why they cant merge the two ENCORE PH and LF and do just one trial startin in H2 2016 , big enough to qualify as ph3, using MELD and HGPV as approval endpoints for NASH Cirrhosis. Why do they need to do two different trial 6 months one after the other?
Sometimes it is better to do just one trial for one indication , get the drug approved and then do sNDA and/or other trials. All these long and expensive trials risk to disperse into too many targets the limited resources and also eat into Emricasan patents without any sales for quite some time. Considering the fast track in Nash LC I wonder why it takes one semester to meet with the FDA to decide about the LC trial design. Also I do not understand the purpose of the ENCORE XT trial. Either the ones already done are good enough to get the drug approved or you just need to do a ph3.
The whole program to me does not make much business sense, and I appreciated the FDA who gave them guidance to focus on Nash Cirrhosis, but now the company needs to help themselves working hard on the proposed ph3 for Nash LC trying to start it asap.
Background: Mortality due to cirrhosis has tripled over the last 30 years in the UK. However, we lack adequate, contemporary, population-based estimates of the excess mortality patients who are at risk compared with the general population.
Aim: To determine the overall survival in patients with cirrhosis compared with the general population taking into account the effects of severity and aetiology of disease and comorbidity.
Methods: In a cohort study, we identified 4537 people with cirrhosis and a control cohort of 44 403 patients, matched by age, sex and general practice from the UK General Practice Research Database between June 1987 and April 2002.
Results: Patients with compensated cirrhosis had a nearly five-fold [hazard ratio (HR) 4.7, 95% confidence interval (CI) 4.4–5.0] increased risk of death, while those with decompensated cirrhosis had a near 10-fold (HR 9.7, 95% CI 8.9–10.6) increased risk compared with the general population. Alcoholic cirrhosis conferred a worse prognosis than non-alcohol-related cirrhosis both in the first year following diagnosis and subsequently.
Conclusion: Having a diagnosis of cirrhosis confers a substantial increased mortality risk compared with the general population, even for those with compensated disease, with 5-year survival between that seen for breast and colorectal cancer.
Today's global death toll for LC is estimated at above 1M deaths annually. Capturing even a small fraction of those 50 M people suffering from LC would mean huge annual revenue. 1M people (only 2%) at $30K-$40K per annum each would mean $30-$40B or $200B in market cap. If Emricasan finally proves in ph3 capable of reversing or controlling LC, it could make CNAT the new GILD
Global prevalence of cirrhosis from autopsy studies ranges from 4.5% to 9.5% of the general population 5, 6, 7. Hence, we estimate that more than fifty million people in the world, taking the adult population, would be affected with chronic liver disease. Globally, alcohol, NASH and viral hepatitis currently are the most common causative factors. Prevalence of cirrhosis is likely to be underestimated as almost a third of the patients remain asymptomatic. With the use of non-invasive tests like transient elastography, a more realistic picture could emerge in the near future. During 2001, the estimated worldwide mortality from cirrhosis was 771,000 people, ranking 14th and 10th as the leading cause of death in the world and in developed countries, respectively 8. Deaths from cirrhosis have been estimated to increase and would make it as the 12th leading cause of death in 2020 9.
Doctors need to have “full and frank” discussions with patients about the potential risks and benefits of using direct-acting antivirals to treat hepatitis C in late-stage liver disease, especially among those waiting for a liver transplant, liver specialists said at the 2016 International Liver Congress in Barcelona on Thursday.
Experts were commenting on a large Spanish study of people with advanced liver disease who have received direct-acting antivirals since 2013.
Although direct-acting antivirals (DAAs) have produced high cure rates in people with hepatitis C who have advanced fibrosis and cirrhosis, there is still caution regarding their use in people with decompensated cirrhosis who are candidates for liver transplant. Use of ribavirin alongside DAAs in these patients is considered risky, and the extent to which DAAs can improve liver function in advanced liver disease in people awaiting a liver transplant is unclear.
MORE FROM THE GLOSSARY
Spanish researchers presented findings from patients enrolled in the Hepa-C registry, governed by the Spanish Association for the Study of the Liver and the Networked Biomedical Research Centre for the Study of the Liver and Digestive Diseases, Spain. The registry recorded treatment outcomes in 843 people who demonstrated clinical symptoms of advanced liver disease, had liver cirrhosis and had not received a liver transplant during or within 12 weeks after treatment.
The study cohort consisted of people with Child-Pugh A (compensated) cirrhosis and 175 people with Child-Pugh B or C (decompensated) cirrhosis. Those with Child-Pugh A had a median MELD score of 8; those with Child-Pugh B or C cirrhosis had a median MELD score of 15. MELD score predicts the risk of liver disease in patients with end-stage liver disease and helps to prioritise allocation of livers for transplant. The score ranges from 6, for those with less severe disease, to 40, for those who are critically ill. The score determines how urgently someone requires a transplant within three months and takes into consideration results from three routine lab tests.
Participants in the cohort received a variety of interferon-free regimens. Forty-five per cent received sofosbuvir and simeprevir, 22% sofosbuvir and daclatasvir, 16% sofosbuvir and ledipasvir, 10% ombitasvir, paritaprevir/ritonavir and dasabuvir, and the remainder received other DAA regimens. The majority of patients also received ribavirin.
Sustained virologic response data 12 weeks after completion of treatment (SVR12) were available for 595 cohort participants, and showed that people with Child-Pugh A cirrhosis were significantly more likely to be cured when analysed by intention to treat (i.e. the cure rate among all those who began treatment, regardless of whether they completed treatment or not). Ninety-four per cent of those with Child-Pugh A cirrhosis were cured (402/428), compared with 78% of those with Child-Pugh B or C cirrhosis (102/131). The difference was due to a higher rate of virologic relapse after treatment and a higher frequency of deaths in those with Child-Pugh B and C cirrhosis.
People with Child-Pugh B or C cirrhosis were also more likely to experience severe adverse events during treatment, including the development of liver decompensation, anaemia requiring transfusion or other adverse events. Severe adverse events during treatment and post-treatment mortality were predicted by a MELD score of 18 or above, such that only 68% of those with a MELD score of 18 or above were alive 36 weeks after starting treatment, compared to 97% of those with MELD scores below 18 (p
Dear Mr Engbring,
just wanted to touch base with CNAT progress regarding the LC registration trial discussions with the FDA. Has a meeting been scheduled? When more or less it will happen? The sooner the better, I hope the FDA will take in due consideration also the fast track and exepedit the ph3 (hopefully) planning.
I think we all know that LC, now Nash LC should be the main focus and yet we are quite far from the trial initiation.
There are hundreds thousands undeserved patients in NASH LC and many more in LC in general who desperately need a drug that can reverse or even halt their condition and I just feel that things are not proceeding as quickly as they should. In my opinion all efforts should be focussed to get Emricasan to the market for LC as quickly as possible, whereas I feel that you are dedicating your limited time and scarce resources to too many trials for conditions in which there seem already to be a lot of competition and more advanced drug candidates (such as Nash fibrosis for instance). And I think that is why also the price per share is very low.
That is what I have also been able to gather from other investors. They are all more or less disappointed by the too many, expensive, long trials in indications which are not the primary focus, whereas the one that is the primary focus has no trial and no money for it, and it won't before the next year that it will start.
A bit of paradoxical situation I would say. Sorry if I sound a bit too harsh, but as an investor I like to go to the heart of the problem , and try to give my input to solve it, if possible.
I would like to hear your comments, please.
Thanks and regards
They have just added PCC also in the CA4P arm : PCC + Bevacizumab + CA4P Versus PCC + Bevacizumab + Placebo for Subjects With Platinum Resistant Ovarian Cancer
Is this only the abstract? The PR hinTed at more info Regarding OS, number of progress free patients at the end of the regimen (double vs placebo), more efficacy for the patients with measurable lesions, more efficacy for patients with platinum resistant disease (double pfs vs placebo). Anyway to post the link ( or at least a disguiSed one since ymb doesnt allow?). Thks
R/s does not affect the value of your investment. You will have 5 times less shares But at 5 times higher price
I agree ROCHE should buy out OXGN. $4 just from cash and NOL, another $2 tip for the pipeline would make it ten times current PPS. Amazing how low current PPS is
Interesting note Slowdown. $4 a share and you even get the pipeline for free. That makes the current PPS so absurd.
pharma, any ideas when the Platinum resistant sub-group OS data will be reported? Do you think they were not reported as not availaible yet?
Aren't Avastin patents expiring in 2019 in US? If so, OXGN could even use a bioequivalent, couldn' they?
pharma, I think your market estimates are very conservative when it comes to pricing. anticancer drug sell at $150K pa. (see for instance Nivolumab). If the platinum resistant ovarian cancer patients are ca 10,000 in US , then we are tallking about $1.5B potential, The same for GBM
A Market price manipulation is evidently going on, the company pipeline has $5M negative value at the moment, considering cash on hand. Until these manipulators are taken to jail (I think they have automated programs selling small quantities of shares to each other to drive price down and buy larger quantities from retail investors) , we will keep suffering this BS price depression
$23M cash on hand, $18M market cap. What about reporting the MM (market manipulator) to the SEC? They are obviously criminals (because in addition to market price manipulation, they make it difficult to the company to fund R&D, and consequently get an effective drug to help many unfortunate patients as early as possible to the market).
Maybe if we all report them the SEC will do something about it.
Abg OS improvement is 2.6mths, but it will be likely more in recurrent cancer. Entering ph3 in ovarian cancer (and also other ph2 ongoing) and trading at less than cash with no debt.(ca $20M). LOL. How long they will manage to keep it down?
smalla correction. The strong improvement in MELD score after only 6 months was shown for patients whose MELD score at baseline was equal or above 15 (not 16). Basically all the ones that qualify for a transplant