VERTEX is spending the $$$ for '09 launch.

[orleans]

Vertex CEO preparing for 09 hepatitis drug launch
Last updated:20June2008

June 19 (Reuters) - Vertex Pharmaceuticals will be prepared to launch its experimental hepatitis C drug next year even though U.S. regulators have not said whether they would review it based on a mid-stage trial, the company's chief executive said.

"That's Plan B," CEO Joshua Boger said late on Wednesday at an event at the BIO International Convention in San Diego.

He said Plan A continues to be completion of a pivotal trial of the drug, telaprevir, in hepatitis C patients not previously treated, with data expected in the first half of 2010. If positive, Vertex would then file for regulatory approval in the second half of 2010.

But the company "has to be ready," despite the expense, to launch in the third quarter of next year should the U.S. Food and Drug Administration agree to a faster timeline, Boger said.

Vertex last week reported positive interim results from a Phase 2b study of the drug in hard-to-treat patients who failed to respond to prior treatment, leading many observers to expect accelerated approval of the drug.

The interim results found 52 percent of the patients who received telaprevir as well as the standard interferon/ribavirin combination had undetectable levels of the hepatitis C virus after 36 weeks, compared with 30 percent of patients treated only with the standard therapy.

Boger said Vertex expects to take the final data from the trial to the FDA late this year or early next year. Should the agency agree to review it, he said the application would be only for patients who have stopped responding to other therapies.

Boger said preparing Vertex to scale up for a launch next year is expensive, but needs to be done because having telaprevir on the market as soon as possible will save lives.

Earlier this month Vertex Pharmaceuticals sold the rights to future royalties on two HIV drugs under a license agreement with GlaxoSmithKline to unnamed financial investors for a one-time cash payment of $160 million.

[redrodeo]

Thats good news. That FDA is so slow and politically driven. I know there are alot of people out there hoping it gets early approval.
     Red

[merryBe]

36 weeks is a long time to get under.

what are the clearance rates is the main question. Do more reach SVR and remain so.

even if TP gets people UND faster, it still doesn't help unless they stay that way at a higher rate as well.

I'm a little confused by the numbers because A. teleprevir was supposed to get people under faster...like in 4 weeks or less...
AND, SOC gets 50% UND in 12 wks on average...yet here they say only 30% cleared and it took 36 weeks? What doses were they using? this is not making sense to me.

mb.

[merryBe]

see here is part of my dilemna..look at their proof 3 trial stats

               Week 12 ,       Week 24-end of treatment    week 36-12 weeks post-treatment
Non-responders (n=66)  71%                            65%                                           41%
Relapsers (n=40)  88%                                    83%                                            73%
Breakthroughs (n=9)  44%                               44%                                            44%
Total (n=115) 75%                                          70%                                             52%

look at the non responders at 41% end treatment, not exactly stellar over SOC treating
the main results are not now you are at 12 weeks what matters is the end numbers.
certainly curing half that couldn't be cure earlier is good, but not the stellar numbers they were claiming would be coming,
mb  
If I was getting into a trial, I'd still say get alinia on board too....less problems but similar improvements over SOC.

[frijole]

First off, I would not suggest using TP for Telaprevier.  I read TP as transplant and I think most on the board do as well.  Maybe TV or something would work.

Secondly, I think most non responders are aware that they are the really really tough cookies.  A 41% chance at reaching UND may be pretty good odds.  When you are saying that 41% odds is not stellar for nonresponders compared to SOC you are comparing apples and oranges.  SOC odds for Geno 1 treatment naive are 50%.  As a nonresponder (one that does not respond to interferon)-, you are already in the bottom 50% - and worse off than a relapser.  So a 41% chance for the bottom 50% looks pretty good.

I, as a relapser think the 73% odds for relapsers are awesome.  I have been thinking about a trial, if they include relapsers in their proposed nonresponder trial, but if TV is on the market in as little as one year, may as well wait.  Thanks for the post, orleans

[frijole]

a little googling on the news.......

"Boger said Vertex expects to take the final data from the trial to the FDA late this year or early next year.

Should the agency agree to review it, he said the application would be only for patients who have stopped responding to other therapies."

So that is great news for us -- that is if "stopped responding" means relapsing too.  This would be new news to my hepatologist.  I asked her this very thing last week - in fact on 06/19, the very day this news was out.  GO VERTEX!

frijole

[nygirl7]

I took it the same way as Merry at first and am glad you reminded me Friole that indeed it is apples to oranges.

Are they talking about using it as a first course in SOC or predominantely FOR the non-responder, relapser if it's released early aka the "tough cookies"?

[Andiamo1]

If you make it apples to apples, 0% respond in the non responders to SOC and 41% with Telaprevir.  That's pretty good in my view!  At least that is what I think in my sleep deprived state :).

[Willy50]

Here's a slightly different take on the news.  Read the below article.  I also find it very noteworthy that in the trial they will try the same approach as Boceprevir and try lead in SOC before dosing the Telaprevir.  I personally think this could work well both with efficacy and possibly getting the viral load down and THEN hitting it with a new compound.  Investors may not be impressed but I think it's agreat idea particularly since there is evidence which suggests that there could be resistant virii produced through using PI's such as Telaprevir.  This means of treating could wring out a better response rate from an already successful treatment plan.  All that Vertex is doing is hedging their bets and continuing to try to wring out the best treatment plan before the drug gets approved.  IF the short course trip to approval doesn't come to pass Vertex will already be in trials with a trial which WILL meet FDA requirements.  To play it otherwise would be irresponsible.  Why pin all your hopes on something that might not happen?  
---Willy

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http://www.thestreet.com/story/10422784/1/vertex-investors-balk-at-new-study-plans.html

Vertex Investors Balk at New Study Plans
06/24/08 - 09:27 AM EDT


A speedier-than-expected regulatory filing for Vertex Pharmaceuticals' (VRTX - Cramer's Take - Stockpickr) hepatitis C drug appears less likely after the company disclosed plans Monday for a new phase III study of the drug.
Investors have been hoping that Vertex and partner Johnson & Johnson (JNJ - Cramer's Take - Stockpickr) could seek regulatory approval for telaprevir in 2009 based on data from existing clinical trials in treatment-resistant patients.

However, the companies posted details of a new phase III study of telaprevir in treatment-resistant hepatitis C patients on the ClinicalTrials.gov Web site Monday. The disclosure dampened investor outlook for a quick telaprevir filing.

If Vertex cannot file telaprevir with the U.S. Food and Drug Administration in 2009, the filing will likely come in the second half of 2010 after studies of the drug in treatment-naïve hepatitis C patients are completed.

Vertex shares fell 5% to $31.66 Monday as a result.

"We have learned that telaprevir's phase III study in treatment experienced hepatitis C patients is different from PROVE 3 and study 107, which in our view decreases the chances of an early filing," wrote Citibank analyst Yaron Werber in a note to clients Monday evening. PROVE 3 and study 107 refer to the existing telaprevir studies in treatment-resistant patients.

Werber has a hold rating on Vertex, and perhaps echoing the sentiment of his momentum and catalyst-driven hedge fund clients, he added that, "We are no longer warming up to the stock and believe the momentum has cooled off as we believe that the chances for an early approval on PROVE 3 and 107 are low."


Vertex spokesman Michael Partridge says Monday's disclosure of a new phase III telaprevir study is in line with the company's previous guidance and "not closely connected to a possible [FDA] filing based on PROVE 3 results."

Partridge added that Vertex and the FDA are still discussing the previously disclosed PROVE 3 data, but that "initiation of a new phase III study is a natural extension of the PROVE 3 results which we would have started no matter the path to a filing."

The new phase III study disclosed Monday will test two different regimens of telaprevir in patients who previously did not respond to conventional hepatitis C treatment with pegylated interferon and ribavirin.

The study differs from previous studies in that patients will be treated with two regimens of telaprevir for a full 48 weeks instead of the accelerated 24-week treatment cycle used for treatment-naïve patients.

In addition, one of the arms of the new study will see whether a "lead-in" treatment with interferon and ribavirin alone before dosing of telaprevir may improve overall cure rates.

The design of the new study is sufficiently different from the previous PROVE 3 and study 107 trials to suggest that regulators want more and different data on telaprevir's effect on treatment-resistant patients before considering the drug for approval.

Cowen & Co. analyst Rachel McMinn views Monday's selloff as an overreaction to expected news. She reiterated her outperform raring on Vertex.

"We believe that yesterday's [Vertex] sell off was driven by an over-reaction to two-week old information. Timelines for commercial launch of ... telaprevir (late 2010/early 2010) are unchanged, and we see 50% upside relative to the market on the basis of this timeframe. While probability of an early 2009 FDA approval is low, we see 100% upside relative to the market under this scenario over the next 6-12 months."

[orleans]

I would have to agree. This is why I am surprised more folks are not adding Alinia. It's easy to get, approved for children down to 1 year old, low sx, can be had cheap, and has shown promise. Just don't see alot of downside (though,you can never tell!) jerry

[springfever]

There is a clinical trial on Alinia in Atlanta, now.  Started March 2008, but not recruiting.  I'm glad you put me onto Alinia.  

[moahunter]

Depending on how it performs in serious studies, Alina must be considered a very serious threat to the viability of vertex (and other drugs in the pipeline), at least in terms f these drugs recovering their study costs, due to the very low price of Alina, and possibly low side effects.

[frijole]

It has hit the net today or yesterday!!!!   Hope for us nonresponder/relapsers.

http://www.clinicaltrials.gov/ct2/show/NCT00703118?term=VERTEX&recr=Open&rank=7&show_locs=Y#locn

I feel that Vertex is handling this right and doing additional testing no matter what the investors want.  Willy, I hope that starting the SOC first is the right option.  I think the sooner you clear the better and you would clear sooner with Vertex.  I had about 2680 IU/mL with SOC at 4 weeks .  If I were to be in the trial and fortunate enough to get Telaprevir but unfortunate enough to get it up front, I just don't know that I would clear.

Dallas is  test sight so I may be a shoe in (if I want it)
frijole

[merryBe]

thanks for clearing that up for me...yes on the surface it looks like not much improvement over SOC...but as Andy also pointed out, compared to no treatment, which is what many get is a refusal by ins.to retreat with SOC any odds are better.

I'm just praying for us all this doesn't get pulled like the Rochce stuff did.

take care.

mb

[geterdone]

Well, it’s about time Vertex got around to reversing the dosing of this cocktail. I will very interested in the outcome of this study. I have always thought that if they had put telaprevir after the initial start of SOC treatment that they would have stellar results and will see sometime in the future if this were true. I had seemed to me some time ago that if telaprevir did not work alone but did in combination with INF, Riba and telaprevir, in which telaprevir was administered first and killed the more resilient virions but allowed the newer mutating virions to learn and adapt to the new insurgent and become resistant to telaprevir as their DNA evolution evolved, therefore creating an even stronger resistant virion to both inf/riba and telaprevir. I would like to see a 24 week trial in which INF and Riba were administered for the first 12 weeks and then Telaprevir administered for the remaining 12 weeks.
jasper
In addition, one of the arms of the new study will see whether a "lead-in" treatment with interferon and ribavirin alone before dosing of telaprevir may improve overall cure rates.

http://www.thestreet.com/_yahoo/newsanalysis/biotech/10422784.html?cm_ven=YAHOO&cm_cat=FREE&cm_ite=NA

[Willy50]

Moa;  It's still a little early to tell for sure abou alinia but it sure looks like a good thing to me.  Pretty quick we'll see how geno 1's fare with it.  As far as I'm concerned all these drugs will possibly work together with each other so I'm already wondering how predosing with alinia would work with triple therapy.

Frijole:  I don't know how it will pan out.  Vertex tried and succeeded with their current method..... but I'd bet that Sherring-Plough has some pretty sharp guys working for them too.  If they decided to do it; why not try it also?  Might as well if you are already running a trial and have a 12&12 and a control arm
        The argument with preloading riba is that it takes some time to build up to optimum trough levels.  It's too nasty to take an opening mega dose of riba and so it may take several weeks before you've got the needed dose levels in the blood.  That means that in VERY early SOC one is essentially on interferon monotherapy.  One achilles heel that the PI's have is that they create resistant mutations.  I think the thought is why expose the virus to Telaprevir before the immune system is ramped up?  Predosing could get optimum riba levels in the bloodstream, allow the immune system to kick in, and possibly get a log drop before adding Vertex into the equation.  It could prove to be a very effective move, or it may not.  It sure will be interesting to see what happens.  I'd love to see an improved response rate.  Time will tell .

Merrybe, maybe I didn't follow but in my opinion this treatment looks way better than SOC.  On the vertex trials about 90% of the participants RVR.  Their SOC control arms have about a 10% RVR rate.
I believe that I heard regarding the treatment failures trial (prove 3) the overall response rate was about quadruple that of SOC at 12 weeks.  After treatment ends generally speaking very few triple therapy responders relapse.  We have to wait intil the trial is over to draw any conclusions but the preliminary results look very encouraging.

The Roche trial, if I we are talking about the R1626 trial that Trish and a few others are in, did not get pulled.  They did stop dosing of the compound however from what I heard here.  The trial participants continue on with SOC (please correct me if I'm wrong).  The trial is still ongoing to the best of my knowlege.

best,
Willy