Vertex VX950 good news

[willows]

Why can I never find the button to make my links live?  Anyway, I was cruisin thru some forums I read everyday and found these abstracts....if I am to believe the poster of the info, these are for the big Liver wing ding in Boston this month and what they all four said was....VX950 rocks.  Very little mutation and when the nasty virus does mutate, the second variant is but a wimpy copy of itself and STILL sensitive to IFN.  They also had some GREAT luck with VX950 as a monotherapy, but have decided the IFN was needed for mutation.  Now, I hope this info is current and valid, I am still checkin around to make sure it is good.  Although it was a hard read, the results were so very worthwhile.  


http://messages.finance.yahoo.com/Healthcare/Biotechnology_and_Drugs/threadview?m=tm&bn=19596&tid=20261&mid=20280&tof=1&rt=1&frt=2&off=1

[niceguy2007]

so how does vx compare with ribavirin?

no anemia/ no gastric troubles? no rashes?

[Kalio1]

they don't know that yet because the people taking VX950 are also taking ribivirin and interferon in the trial.

[niceguy2007]

kalio...i think that particular link mentions vx and inf without riba/?

also, its interesting that a 14 day treatment resulted in a 3 month RNA negative period.  seems like SVR to me.

[NYgirl]

niceguy as far as we know all the trials are vertex AND interferon AND ribavirin.

It's an ADD on not a substitute.

[willows]

I am still trying to find the EXACT source of the article, so before I start celebrating with another scone this morning, has anyone found the info anyplace else?  I am a real BIG skeptic of anything I find on the stock boards, but my doctor just sent me a letter, telling me about the same news.  But still......how did the poster find the news?  I'm still looking, looking hard too.  I'm stage 3, and after a lot of blood work, doc says I am resistant to IFN?  So VX950 is about all I have to look forward too.  So, if anyone can verify the info, please let me know.  If it's true, I may leave the house for the first time in a month and go shopping to celebrate!

[niceguy2007]

ok yeah nygirl is right... they gave vx and inf for 14 days, then started regular therapy with riba.

anyhow, vx is a great hope for nonresponders. we will all beat this virus yet.

[jmjm530]

Currently VX-950 is being tested in the U.S. with Peg and ribavirin. In Europe, however, it's also being tested with Peg only -- no ribavirin. Should the European trials without ribavirin work out, no doubt ribavirn may be dropped from some future American trials. The other thing to keep in mind is that even when VX-950 is used with the current drugs, treatment time is potentially shorter, i.e. less exposure to the toxic drugs.

What we know at this point is that VX-950 knocks the virus down VERY fast with almost anyone who takes it. What we don't know is if it will produce SVR (cure). That information should be available within a year. Another reason for people to wait who can afford it.
If things work out in the trials, Vertex potentially will be on the market in about 3 years with trial opportunities for many sooner.

-- Jim

[Upbeat]

3 years?  No way.  This is on fast track and if it works as hoped it will be on the market as soon as early 08.  It is very possible it will work as a stand alone drug as well. Only time will tell.

                                                   Ron

[NYgirl]

If you are resistant to that kind of IFN why isn't he thinking to try you on the consensus interferon (Infergen)?  While both Pegs are the same type the consensus is different.

Vertex is not a stand alone drug, it relies on regular old Peg too.  Maybe eventually they will be working on mixing it with something else but for now...it's not really a plan for you I don't think

However Infergen IS indicated especially for non-responders and relapsers.

Have you discussed this?

[jmjm530]

I was trying to be conservative. Yes, if all works out it may be available sooner -- that's why I said "about". Your moniker serves you well :)

All the best.

-- Jim

[Alady1620]

This is wonderful news.  I am on NM283 and pegasys. I expect to relapse as soon as I stop taking the meds bc the virus came back once at small levels.  I don't want to treat again right away with Ribavin so this gives me hope that there will be something better in the future.  I was beginning to feel very depressed about my situation.  Thank you for posting this.

[couldn't think of a nickname]

I would point out that there are abstracts in there from the 2 week 950/IFN trial that did NOT use ribavirin.
In that trial, side effects seen were those mostly associated with the known side effect profile of IFN.

I think there tends to be confusion, as they have run several different trials. As Jim pointed out, in the US, it is being tested CURRENTLY (not talking about the past or future) with IFN and riba, while in Europe, there will be arms without riba.
You can infer from the abstracts that 950 still works as a mono, but at a slower rate than with IFN, which should be expected.

IMHO, the riba issue is being done to placate the FDA. It doesn't seem there is any evidence that shows riba is going to do a whole lot. After all, it gets 4 logs in 2 weeks by itself, 5.5 logs in 2 weeks with IFN only.

When the time comes, I guarantee I will opt out of the riba, and it won't be an issue as far as I am concerned. But, that is a personal opinion, and no one else should necessarily take that advice for themselves. With Thalassemia minor, I don't see the point of taking riba when my baseline hgb would be around the 12 area.

[Kalio1]

Unless they have a crystal ball, no one can say WHEN VX950 will be available with any certainty. A statement like "within the decade is reasonable but that is about it. It is speculation and wishful thinking to think it will be available in the next two years. The idea that a drug that isn't even through phase III trials will be available in less than two years flies in the face of logic. We are still waiting on SVR data from the FIRST small group of people that were given the drug and that won't be available until 2007!
It does look very promising and could be the "cure" we all hope for but it is extremely unlikely if not impossible for it to be available in " 2 years"

[Upbeat]

With fast track they wont need phase III trials before approval from the FDA.  Vertex still maintains that they will be at market in early 08. They are going all out with their manufacturing process so they will be ready for 08. As soon as these ongoing trials are complete ( by the end of the year ) we will know for sure. The end of the year is comming quick.
                                                            Ron

[GrandOak]

I may be mistaken but I believe the initial trail for VX950 was a monotherapy trial and that all subsequent trials were combined with IFN/Riba.  So, the possibility of it being simple a monotherapy treatment has not been fully explored.

[Mister beagle bailey]

Hi, Can do Man just sent me your post and I to am waiting for the VX950 without the riba.  I didn't know you also have Thalassemia minor like me.  My HGB baseline is around 11.0 and I relapsed after finishing meds in June.  If you hear of a trial for this drug without the riba let me know, I'm game too.

Beagle

[GrandOak]

Like to article where VX950 was tested as a monotherapy in Phase IB:

http://www.vrtx.com/Pressreleases2005/pr111105.html

[Mister beagle bailey]

Thanks for the site.  I just read it and saved it in my favorite place.  

Beagle

[Kalio1]

One thing to remember is VX950 needs IFN due to the mutation issue. It will still leave patients that can not tolerate IFN out of the loop. Until they are also able to access a "cure" only a portion of HEP C patients will be helped.


My hope is that they can find a solution that helps ALL victims of HCV.

[niceguy2007]

could someone please post information about VX trials in europe? thanks.

[couldn't think of a nickname]

To set the record straight, VRTX  has said they anticipate filing for approval in 2008 (mid at the earliest I think), and it would be subject to expedited review, which is 6 months, instead of the normal year that is taken. That would take place after phase 3 is completed.

Further, the subject has come up in web casts with analysts (who have raised the issue) about whether or not they could file based on phase 2 data.
In the past, they have said that it is possible, but the data would have to be "transformational", and they mentioned 75% SVR, or something like that IIRC.
They have also said their phase 2 constitutes a filable package. Meaning, it is done like a phase 3 would be. Part of the proof of that is this is a 1000 patient trial, which is the size of many phase 3's.
So, yes, there is a chance (and the company doesn't want to focus on that based on prior web calls).

And yes, there is a timeline for launch. Analysts have put that in their numbers for the company. Can timelines change? Yes, but this has been the basic timeline with minor adjustments for a while.
Keep in mind, this phase 2 was supposed to start earlier than it did, but kept getting delayed. Now we know the reason. Instead of 200 patients for a normal phase 2, this is 1000 patients.

[couldn't think of a nickname]

Oh, and to answer the question of the first response, we do know how 950 compares to riba.
It doesn't.
They don't have the same mechanisms of action, therefore, there is actual efficacy and direct effect from 950. As far as symptoms, the side effect profile has been covered in detail in the trial results, it does not cause anemia, or many of riba symptoms, and has generally been very well tollerated with no SAE's.

Here is an excerpt from the trial with 950 and IFN ONLY:

"A preliminary safety review has been conducted that indicates that the treatment was well tolerated. All patients completed dosing and no serious adverse events were reported. All adverse events in the patients receiving VX-950 alone were reported as mild. Typical interferon-related side effects, of mild to moderate severity, were reported in the patients that received peg-IFN along with VX-950 or placebo. Laboratory-related adverse events of neutropenia in one patient and thrombocytopenia in one patient were reported among the patients who received peg-IFN. Neutropenia and thrombocytopenia have previously been reported in patients receiving peg-IFN alone. It is not known if the two patients in whom these events occurred were also receiving VX-950, because the full safety database has not yet been unblinded. A complete safety analysis will be conducted once the study is fully unblinded."


The sides have been addressed in other publications in greater detail.

[couldn't think of a nickname]

Sorry man, I missed your post 3 times, and didn't even find it at first just now.
Sorry to hear about your relapse. I really am not expecting 950 to be tested without riba in the states, but then again, no one knows what phase 3 would look like.
I really think that if/when this gets approved, it will be with riba. I would choose not to take riba on my own, even if my doc disagrees.
My hgb is normally around 12, was once as high as just under 14, which I hear is unusual for Thal. minor. But, it is usually in the 12-13 range.

Good luck to you, I remembered from a while ago you were treating and had TM, but I was unaware you relapsed, as I miss many threads.

[mremeet]

I'm no "riba rash" expert, nor have I heard any convincing evidence that there's a scientifically accepted term for riba rash. Riba rash appears to simply be a one size fits all internet lore/description for those who are under SOC and have a rash of one type or another. When in fact many rashes that occur during SOC can have a wide range of causes (and consequences). Most of the SOC rashes I have heard about appear to nothing more than very dry skin, which definitely happens as a result of the extremely drying effects of interferon/riba treatment. I think that's why moisturizing lotions are often touted as working by many who swear by them. And again, for those that simply have really dry skin, that's what they would call their "riba rash". Others may have more serious afflictions, which may require more aggressive treatments like steroidal creams. And again, they'd call their psoriatic flareups or IFN reactions "riba rashes". But apparently most of the people who get these rashes do not have them head to toe, and they don't really rise to the level of terminating treatment. And when I say head to toe, I mean from your feet, up your legs, on your crotch, belly, back, chest/torso, arms, hands, neck, face, scalp and even in the ears. The vast majority of SOC rashes I've heard of are usually localized to certain areas or regions of the body. Plus, they come and go and are largely tolerable (comparatively speaking). If someone had a rash like I had (which was getting worse before going on the pred), I can promise you you're only option would have been to outright discontinue.

As far as why no rashes were reported from the shorter duration trials: both myself and the other participant who have the rash, had no real rash at 28 days. Only little bumps that just itched a little, that's all. It took longer than that to flower into what it is now. And again, please don't misinterpret this. I most certainly DO NOT know that VX is causing this, nor am I stating that. Just pointing out that just because no rashes or other assorted side effects were not noted in shorter term trials, that doesn't preclude the possibility that something might show up in a trial duration three times longer. (which is true with ANY experimental drug of course) And again, I know of some people in the trial who are not getting the rash and they have been taking it longer than myself. So even if it did cause a rash, it may only be in some people, not everyone.

[couldn't think of a nickname]

All good points, and another thought I had, was I was wondering if it was an autoimmune-type response. We have all heard of what IFN can do as far as autoimmune consequences, which is one reason why most want/wish a tx without IFN as well.

[Kalio1]

Since you are taking VX950 I think your info. is highly pertinent. The fact that you are not the only one with this all over rash is concerning too.
I am so sorry to hear you are coping with this all over rash issue. o the doctors have no clue? What is it like? I know it is hard to describe a rash, but it sounds like all over re bumps that itch? Do I have that right? Does it hurt? Cause any other symptoms? I agree with you and was told something similar by my doctor that there is no "riba rash" it is a catch all for various skin issues that crop up but it is not one particular rash cause by riba. How simply awful this must be for you. I hope the prednisone works out and you get some relief soon.
thanks so much for volunteering to be a "guinea pig" for us all.

[Mister beagle bailey]

Did you hear about the other drug called Tribavirin(viramidine) by Valeant Pharm?  It has already completed phase 3 of trial.  It will take the place of riba when they do release it to market.  My dr said maybe next year, it all depends on how fast the FDA moves but they are using it in Japan and Europe.

www.valeant.com

With my thalessemia minor there were times in the past that my HGB was 14 but the norm of the last few years appears to be 11.0-12.0.  Before starting tx it was 11.0.  For me the anemia was the worse sxs and having 2 transfusions to stay on tx.  The procrit didn't really help me until near the end when I was taking 60,000 units every 5 days.  Can't wait to hear you got to SVR, it will prove there is a cure for people like us.

Beagle

[NYgirl]

Don't bother wasting your time.......it's not worth it.

[mremeet]

I'm not going to start any rumors or attempt to downplay the significance of VX950. But both myself and another VX950 study participant are struggling with bad systemic rashes during our treatment. So far we don't know what is causing the rash, but from what I've heard so far, serious systemic head to toe rashes are apparently quite rare with SOC drugs. And yet out of a handful of known participants (to me) I already know of two people who appear to be experiencing this head to toe systemic rash (although I haven't heard of anyone else getting the rash so far).

What does it mean? Anyone's guess so far, it may not be caused by VX at all. But so far no one has been able to explain to me what's going on with this rash, or what it's likely origin is. Bottomline is that VX *IS* and remains an experimental drug, don't forget that. I've fully come to realize I'm a guinea pig, make no mistake about it. Maybe a willing and carefully monitored guinea pig, but a GP nonetheless. So extreme enthusiasm based on VX's performance in trials leading up to the current trial should be tempered with patience and realism. No one knows for certain yet what this drug is going to do in terms of improving SVR odds OR if it will be largely tolerable by most people for periods of time longer than 28 days of dosage. This rash has really been something. I never knew what true suffering was until I had this, and believe me I've been in some miserable spots before. And I had to go on prednisone to control it, which lowers the effectiveness of IFN/riba, and now am left hanging wondering if I'm immunologically "standing on my shoelaces" as a consequence of that. Anyway, everyone should keep it in mind before we start counting all our chickens before they're hatched. I'm still optimistic about VX950, and it's only natural for all of us to be so hopeful, but please just try and reserve over exuberance and temper your expectations with what we all know as "the real world".

[couldn't think of a nickname]

I thought those types of rashes were quite common with riba, which is what everyone in the trial is getting. Wouldn't it seem that if those are part of the sides from riba, especially since in the earlier trial without riba, that wasn't reported?

[NYgirl]

Oh man I'm so sorry to hear all of this.  In fact people completely and totally gloss over the "experimental" part of the whole thing and rarely stop and think of what you might be going through.

I had no idea that you were suffering that much.  Truthfully I feel like quite the baby because I made a HUGE deal of the RibaRash when it developed (I am still scarred from it so it wasn't nothing but it certainly wasn't anything as bad as you have).

I just have to say good luck on your journey one more time.  I hope that nothing else comes up and that they do figure something out. I'm sorry that they had to put you on the Pred - that totally totally SUCKS - has it at least helped at all?

Debby

[aiuta]

I went to a seminar on Hep C given by the American Liver Foundation and with top Southern Cal hepatologists last week.  One Dr. said that the FDA won't let them do serious mono-therapy testing with Vertex, not because it doesn't work, but because it's too risky (since they already know for sure Inf & Riba DO work to some extent).  He said that what will probably happen is that first the FDA will approve Vertex added to Inf/Riba and then these two drugs will slowly get phased out.  He was hoping in 10 years Int might be no longer used as a therapy.  He said that government approval takes a long time. Great thread. All my best, Aiuta

[Mister beagle bailey]

I also thought that the trials are VX950/riba.  I would think your rash is becuase of the riba as that is a common sx of riba.

couldn't think of a nickname, I posted to you in this thread about 4 - 6 posts above, about your thalaasemia.

Beagle

[couldn't think of a nickname]

It is also possible he is in the 950 arm, but it isn't caused by 950. The point is, it is an ongoing trial, and no one knows for sure until results are released. It might be, it might not be. One just needs to be careful of speculation though.

[pln]

I am at week 10 vx-950 ,no rash here, but my riba was cut in half at week 3, I could also be getting placebo??? I just don't know!! Pam

[Kalio1]

I agree, I meant to say that in my post, that it could be caused by some other source too.
Ideally VX950 will not have side effects of any consequence but if it does, a rash isn't the end of the world. Unless you are the one that has the rash of course, then it can be consuming.

[couldn't think of a nickname]

The design of 950 and Schering's PI are such that they should not cause as many sides (different design than BILN). This was written about by Forbes before their very first trial. I believe the reason is that the drug targets proteins only found on the virus, and not normally in the human body IIRC.
However, I think every medicine there is has sides, because these chemicals are foreign to the body. I have been told certain meds (allergy for example) are well-tolerated and shouldn't cause symptoms. For me, they have. One a long time ago was supposed to cause drowsiness, instead, it wound me up. Sometimes I wish I had that allergy med in the house lol.

Just another reason to hope for as short as possible. After all, these are all chemicals, and I know no one wants to take them a day longer than they have to.

Having said all of the above, there are sides with 950, SGP's, and all of them.

[GoofyDad]

Floppy ears says: Thanks for the site. I just read it and saved it in my favorite place.

Don't worry buddy, we're not askin.....like Clinton's military -- Don't ask; Don't tell.  

[couldn't think of a nickname]

Kalio, being a VRTX trial, as far as the study is going, it is only pertinent IF caused by 950, and it may not be. I hope you are not jumping to a conclusion.
I don't know about your doctor, but skin issues have been mentioned in here for a very long time, with SOC.

Mr B,
I was once very hopeful about viramadine, but it now has had 2 trials where it didn't quite meet its endpoint.
I know it still does show activity, and may still be a player, but I think the trial results of late haven't been as anticipated. From a theoretical standpoint, the fewer the drugs needed the better of course. But, there is a lot of data that needs to be seen with everything, and the 950 trial in Europe without riba might shed some light on some questions.

[Kalio1]

Yes, I do understand he could be in the non VX950 arm. I doubt it though. His rash doesn't sound like the "riba rash" we hear so many have on SOC.

A rash doesn't necessarily mean anything at this point however he is in the trial and he knows of at least one other participant that has the same odd rash. Time will tell. If he is on VX950, his rash and need for prednisone will be noted in the data. I know everyone wants it to be problem free and available soon but as he points out, we don't know enough yet to say one way or the other. He makes a good point when he says that the rash wasnt seen in the other trial group BUT he has been on it longer than 28 days they were on it. Anyone who has taken SOC knows that it can take time before side effects rear their head.

[pln]

http://www.businessweek.com/investor/content/oct2006/pi20061002_781817.htm?chan=top+news_top+news+index_investing

[BNR]

I experienced the AWFUL rash you described at the end of my treatment with Pegasys/Rib. I had to go on prednisone also, plus Diflorasone Diacetate ointment, Desonide ointment, etc. It was truly unbearable. I had itching previously on Peg-Interferon and the old interferon/reba treatment also, but this was much much worse.
I now have Vertiligo (white patches on my skin) and it is spreading.  It may not be the VX950 that caused the rash -- something to consider.

[Kalio1]

I have heard narrow-band UVB therapy if very effective with mild or moderate cases of Vitiligo. Have you tried it? I have not heard of treatment causing it, but anything is possible.

[TravelGrl]

I am in week 5 on the VX-950, and have been taken off the Riba for the time being...I broke out in an intense rash all over my body as well on day 4 of starting...we stopped the Riba and then restarted a week later at a lower dose...the rash started to appear again..Maybe it is a combination of the Riba and the VX-950?  Just a thought...Right now I am just on the VX and INF...Hopefully they will allow me to continue to participate.

I still am experienceing severe itching, especially after a shower which tends to last 20-30 minutes..but no real rash now.

[Kalio1]

Glad to hear the rash is improving, itching is so hard to deal with. It will be very interesting to see how it goes with you on VX an IFN with no riba. Maybe you got something there and it is the VX and Riba together that causes the rash problem with some, guess we have to wait to find out for sure.
I hope they let you stay on it without the Riba.
I personally hate Riba!
Best of luck to you.

[Mister beagle bailey]

Your right about the tribavirin not being as strongh as the riba but if you look at the trials done they were not given permission to do the trial with a high dose like the riba and therefore showing riba had a unfair advantage over tribavirin.  They are now asking for approval that the tribavirin be tested (trialed)at higher doses.

Beagle

[mremeet]

Don't despair, it may very well be some kind of VX-riba interaction that will no longer happen once the VX is stopped and riba is reintroduced without it. Plus, unless you have tried prednisone already, they may be able to permanently subdue the rash with a single tapered course of prednisone. It's no guarantee, but there are certainly options left to try before throwing in the towel at the end of the first 12 week "VX" cycle. And in the meantime, rest assured that the VX and IFN can kill the virus quite effectively even without the riba around. So you have plenty of hope for an SVR left, just hang in there and take it one day at a time. With this rash though, I know that's a LOT easier said than done. Lastly, from what I've read the Atarax may not always make you so tired once you get used to it. That seems to be happening to me now, it's not quite as much of a downer as it was initially. Plus letting it build up in your system will help keep the itch at bay much better than taking it sporadically.

Hope this helps, take care.

[mremeet]

Hey, great to hear from you. I've been wondering how the rest of the VX clan has been faring, especially in regard to this. I'm sorry to hear of your rash, but it's also interesting at the same time. So you developed a rash early on huh? And they removed the riba altogether, so you're just flying on VX and IFN alone? Hmm, well that's three of us now that I've seen who have had severe rash problems which have occurred within the first 1-2 months of treatment. I definitely think that's rare (per capita) in SOC, we may be seeing some kind of pattern developing here. And as you mention it may be a riba-VX interaction, as opposed to simple riba or VX allergic reaction.

If you're still experiencing the nightmarish itching, I'm taking Atarax which is a potent antihistamine commonly prescribed for these types of rashes. As long as I take it regularly, it seems to hold the creepy crawlies at bay...although it spaces you out pretty bad. But still, I'll take the spaciness over the itching any day. Hope you're doing well otherwise, please stay in touch and let us know how you're making out. Best of luck, and here's to an SVR for all of us.

[TravelGrl]

Yes, I am also taking the Atarax...20mg at night...I can't handle it during the day..along with prescription cortizone cremes.. the itching comes and goes now...but when I get it, it drives me CRAZY!! I am sure you know the feeling.. So for now I will be just doing the  VX with INF...as I said, I do not know how long they will allow me to continue.  It is not a compliance issue, I just could not tolerate the Riba in the mix...so hopefully I will be allowed to stay in the trial.  I will definitely keep you updated..and please do the same..  The other issue is that when the VX stops at week 12, what happens??  Continueing with just INF doesn't seem to make sense to me..but who knows...I guess time will tell.

Hope you continue to do well and we end up with SVR...I would love to be able to donate my liver to my beautiful daughter...she seems to be on the verge of transplant..

Good Luck,
Jodi

[niceguy2007]

CTOAN,

ribavirin is a broad spectrum antiviral that is also used for influenza.  unfortunately, hcv patients take it for much longer than do patients with other illnesses.  i guess thats what makse the side effects worse.

VX is a narrow spectrum antiviral thats supposed to work only for HCV.  naturally, a specialized medicatino will be more effective, and have lesser side effects.  

personally i believe it will be used without riba once it comes out.