E2 it didn't sound like Joanne had much choice in the decision but I agree if she wasn't doing that well Tefferi maybe had to discontinue her use. If her earlier improvement digressed from a PR to a marginal CI or no improvement and that was the cut off point he may have had no choice given the parameters the FDA set. Tefferi doesn't sound like a guy that would intentionally direct patients off a trial for the sake of better results. We're still unclear as to what cut off point was used, CI or PR's. Furthermore she may have had other issues like the patient that had died with Hep complications....without direct knowledge this becomes a guessing game.
maybe they still will with some elaboration, but this is all good news regardless especially the 3 yr MDS time frame measurement. Heck a bald guy on IMETcould grow more hair than Rapunzel given that time frame.
why would INCY patients talk about a drug that treats only symptoms but offers little efficacy against the cancer? By the side effects listed on Rux, they're probably too sick to follow any board talk. I'm sure INCY has a 20 pg. disclosure contract they were all forced to sign bfr being treated. If you don't know of any alternative treatment even if it's experimental you would assume that's all there is. The MPN forum is great at confusing and obfuscation.
This was my response to that thread about the other trial patient Joanne
"Hard to tell if CI's were in the FDA's red line MF inclusion. I believe the FDA used "any improvement" CI, PR & CR as the criteria for continuing treatment in MF trials and anyone else in ET was taken off. Clinical improvements I would think were included in this group. However Joane showed some benefits and yet was cut off so perhaps CI's were not included.
Ranchands report on Joanne's progress blog was sad. She seemed to be doing well on imetelstat for the last year and as he points out has deteriorated since being take off. Someone mentioned earlier that several patients may have switched back and forth from CI to PR to CR and visa versa. If CL's were where the FDA decided to draw it's line than it would be unfortunate as you pointed out to patients like Joan who had some benefits but were not deemed as significant PR or CR in order to remain on treatment but still may have benefited by being o the drug. "If her health has deteriorated since treatment stopped as a result of the clinical hold this clearly falls on the short sightedness of the FDA in their decision. Maybe if allowed to continue she could have switched to a PR given a longer time frame"
Of course Irish is real.....she gave me 7 shamrocks the other day.
this from Biopearl's Sept 6 post;
"Joanne? Her husband John kept a good blog and unfortunately she was taken off Imetelstat. I was always perplexed as to why. He said they talked for half and hour with Dr. T before she was taken off drug so the decision was not an easy one. Was there liver tox? Was she "only" a CI? She had an amazing response to Imetelstat initially and the blog quoted Dr T as calling her response a "miracle". I don't think she is doing too well now, as she could not tolerate hydroxyurea and is now on interferon. I wish the drug were still available to her. My suspicion is that she was "only" a CI and was taken off study during the partial hold for that reason but have no solid reason for thinking so other than Dr. Snyder's comments re only PRs and CRs remaining in the study."
where do get your 3 month updated tute data from? I have 3 different numbers
with no statistical quality of life improvements in their RELIEF trial, using their BAT designation they side step into an sNDA indication for PV using only a 'low bar' spleen reduction symptom response trial. I guess feeling more comfortable sitting in a lazy boy whilst being infused really gets attention around the FDA.
I can hardly wait for IMET and Jakafi to go neck n neck in a comparative RESPONSE study using efficacious endpoints criteria such as CIs, PRs, CRs and EL
E2W as Irish points out INCY's supplemental warning labeling for Jakafi(R) omits a large number of severe side effects listed under independent labeling. They've really stretched their marketing indications going after PV when in fact the drug has shown an extremely low level of efficacy with disappointing RELIEF trial data. No statistical significance over a placebo in their RELIEF trial did not meet their trial criteria endpoints. Although Jakafi’s NDA PV submission was based on the results from the RESPONSE (comfort) study, more patient subjectivity involved, this was the data they'll be using for expanding their PV indication application. The data from the RELIEF study should be a matter of concern for Incyte as far as Jakafi’s PV indication is concerned but the way they are going on about their NDA they talk like they have the FDA's approval already.
Am I missing something here?
You are right on in determining a potential SP value for GERN based on IMET's potential remissions and INCY's market share. As Irish pointed out maybe far more for treating male patterned baldness topically.
with low volume trading the 11 days left to cover for 27 mil shorts would create some squeeze, if the company was to announce some positive catalyst.
I like how they`ve approved themselves for their PV NDA indication by Dec. I guess they don`t require the FDA review committee panel, seeing how they own it.
WOW 7 shamrocks....Thanx Irish. That's the number of zeros I want behind the $ value of my GERN portfolio. With male patterned baldness Irish, I know we can do this. (2;=3) no more comb overs, hello bangs
funny I posted this on the INCY board earlier this morning, apologizing afterwards ;=3) but they removed it without me doing it. Now if that's not board courtesy for you.
" there is an approved drug in this space already" Gerney lets objectively not forget how low the bar was set for that FDA approved drug. No statistical significance for survival but using a "comfort" endpoint they are expanding their indications. INCY has pretty well given themselves approval for their NDA wrt PV this Dec. Seems to me if your in bed with the right people and influence enough regulators on the review committee you can get approval & sell anything, toxicity bar none.
well then here's some more;
"Common side effects of ruxolitinib:
Abnormal Liver Function Tests Severe
Decreased Blood Platelets Severe
Decreased Neutrophils a Type of White Blood Cell Severe
Bruise Less Severe
Dizzy Less Severe
Gas Less Severe
Head Pain Less Severe
High Cholesterol Less Severe
Urinary Tract Infection Less Severe
Weight Gain Less Severe
Infrequent side effects of ruxolitinib:
Rare side effects of ruxolitinib:
A Fungal Infection that Occurs Under Certain Circumstances Severe
Active Tuberculosis Severe
Progressive Disease in the White Matter of the Brain Severe"
in all fairness it was a cut'n paste job from INCY's company's PR site but who would refuse a good luck shamrock? ;=3)
"Jakafi can cause serious side effects including:
Low blood counts: Jakafi may cause your platelet, red blood cell, or white blood cell counts to be lowered. If you develop bleeding, stop taking Jakafi and call your healthcare provider. Your healthcare provider will perform blood tests to check your blood counts before you start Jakafi and regularly during your treatment. Your healthcare provider may change your dose of Jakafi or stop your treatment based on the results of your blood tests. Tell your healthcare provider right away if you experience unusual bleeding, bruising, fatigue, shortness of breath, or a fever.
Infection: You may be at risk for developing a serious infection while taking Jakafi. Tell your healthcare provider if you develop symptoms such as chills, nausea, vomiting, aches, weakness, fever, or painful skin rash or blisters.
The most common side effects of Jakafi include dizziness and headache"
All this doesn't sound very comforting to me