Novartis Pauses Study of GenVec's Gene Therapy CFG166 (UPDATE May 2016: Trial Is Again Continuing)

[Anonymous2929]

January 2016

Can't believe this. At this rate, there won't be a cure or an therapy. Putting money first as usually....

http://www.biocentury.com/dailynews...novartis-pauses-study-of-genvecs-gene-therapy


May 2016

Trial is going to continue.

http://ir.genvec.com/press-releases...vides-update-on-hearing-loss-clinical-program

 

[Joan A.]

Big pharma/industries need us. Antidepressants, antiseizures, benzodiazepines, muscle relaxants, sleeping pills, maskers device, hearing aids, "natural" remedies, and only the Lord know what others....

Don't be sorry. Fortunately, if a drug can't be developed due to a matter of money and time, the ways of neuromodulations/stimulations, targeting directly the brain's areas overactive or hypoactive, are going on and better.

We will win

 

[tomytl]

Can't believe this. At this rate, there won't be a cure or an therapy. Putting money first as usually....

http://www.biocentury.com/dailynews...novartis-pauses-study-of-genvecs-gene-therapy

What it exactly means is unsure. It shows in the first, that genvec lost 70% of it's stock value.
So, it will be difficult for them to gain some new money.
I guess, this deals are highly complex between Novartis and Genvec. Maybe all this has tactical reasons, who knows.

As I already mentioned in some earlier posts, most other experts didn't see this Atoh 1 approach as a cure.
Once a inner ear researcher told ..." it would be a success if nobody get harmed by this approach"

So it doesn't sound very promising in case of hearing regeneration, more likely to proof that they can transfer
something in the inner ear.

Maybe and I hope I am wrong and it's really just a pause......

 

[skoupidis]

Well, not revealing the reason for the pause is not a good sign... At least they didn't call it off altogether.

First Autifony, now this. Things don't look good right now, however, there are a lot of players on the table.
Let's hope for the best!

 

[Anonymous2929]

Big pharma/industries need us. Antidepressants, antiseizures, benzodiazepines, muscle relaxants, sleeping pills, maskers device, hearing aids, "natural" remedies, and only the Lord know what others....

Don't be sorry. Fortunately, if a drug can't be developed due to a matter of money and time, the ways of neuromodulations/stimulations, targeting directly the brain's areas overactive or hypoactive, are going on and better.

We will win

When I read this post, it just got me emotional. Just a few hours before, I had an meeting with my cochlear implant doctor. I have been on hell-bent about removing my implant. He said there will not be a therapy for hearing loss in his life time and he seems quite young. He also said by removing my implant I will not be able to receive newer techonogly or it might be impossible to do something for my ear after the implant is removed.

He was kind and asked me what is bothering me. And I just broke down in tears. I recalled my childhood growing up deaf, being bullied, the discrimination etc.

It is a first time I had been personal with any doctor, usually its the speech therapists. He said he wish he could do more... So I won't remove my implant but have stopped using my processor. It is very dangerous for me as if I lose the remaining 10% hearing left in my left ear; I will profound in both ears then

I think I can related to deaf and hard of hearing people

 

[Anonymous2929]

What it exactly means is unsure. It shows in the first, that genvec lost 70% of it's stock value.
So, it will be difficult for them to gain some new money.
I guess, this deals are highly complex between Novartis and Genvec. Maybe all this has tactical reasons, who knows.

As I already mentioned in some earlier posts, most other experts didn't see this Atoh 1 approach as a cure.
Once a inner ear researcher told ..." it would be a success if nobody get harmed by this approach"

So it doesn't sound very promising in case of hearing regeneration, more likely to proof that they can transfer
something in the inner ear.

Maybe and I hope I am wrong and it's really just a pause......

I think the funding is really low in terms of research. Not just this trial but other potential therapies. Then you have the strictest criteria to start clinical trials. Even past that stage, there could be problems like we just found out.

 

[Anonymous2929]

Well, not revealing the reason for the pause is not a good sign... At least they didn't call it off altogether.

First Autifony, now this. Things don't look good right now, however, there are a lot of players on the table.
Let's hope for the best!

I have given up...25 years of deafness. My life has flashed before my eyes. All i have left now is this ringing sounds! And you know what, it don't bother me at all!

 

[Nucleo]

It seems kind of likely to me that the DSMB (Data and Safety Monitoring Board) of Novartis paused the trial for safety concerns. Maybe patients showed signs of unwanted side effects? In any case it's not like this technology was anywhere close to market.

 

[RB2014]

Its says he hopes the study will resume in a few months. I'm guessing, but this might just be a pre-cautionary measure. If something bad had happened, they would stop it completely without hopes of continuing the tests anytime in the near future. I'm still hopeful. There are hundreds of animals that this was tested on and there were no side effects that anyone noted.

I feel like a cure to deafness is at least 25 years away the way things are progressing. It still won't stop me from joining a clinical study when it gets bad enough so there is hope. Hang in there, you are not alone. Life would be better without the ringing, but I don't let it bother me either.

 

[Aaron123]

The information from clinicaltrials.gov says

Detailed Description:
The current study will evaluate the safety, tolerability, and potential efficacy of CGF166 and the associated delivery procedures in patients with severe-to-profound bilateral hearing loss. Eligible patients are required to have documented, non-fluctuating hearing loss. Part A will include a safety and tolerability cohort (N=3). Patient dosing will be staggered; dosing the next patient in a cohort will be based on a safety review of all available data through 4 weeks post-dose of the previously dosed patient(s). Part B includes a volumetric escalation design to evaluate infusion volumes of the same CGF166 concentration (5.0 x 10E11vp/mL) in 4 cohorts of patients (n=3/cohort; total of 12 patients). Part C is an expansion cohort of the highest safe and tolerable dose identified in Part B, for further assessment of efficacy.

And the news story about the press release says

President and CEO Douglas Swirsky told BioCentury the per-protocol review followed a trio of three-patient cohorts in the dose escalation trial that will inform a dose for the fourth cohort. He declined to discuss the DSMB's specific reason for recommending the pause or the board's safety criteria for the trial, but said GenVec hopes the study will restart enrollment within "a few months" and generate data by 2017, as the company has previously guided.

Assuming the press release is right and they have 3 three person cohorts, they are half way through part B. It could be that, as the CEO says this is "per-protocol" though it wouldn't seem to warrant a press release. It seems possible that they had one or more adverse events during the "escalation".

It is also interesting that Genvec got the okay from Norvantis to resume research on hearing issues in the last few days (http://www.bizjournals.com/washingt...eturns-to-hearing-loss-research-with-new.html)

 

[tomytl]

The information from clinicaltrials.gov says
And the press release says

Assuming the press release is right and they have 3 three person cohorts, they are half way through part B. It could be that, as the CEO says this is "per-protocol" though it wouldn't seem to warrant a press release. It seems possible that they had one or more adverse events during the "escalation".

It is also interesting that Genvec got the okay from Norvantis to resume research on hearing issues in the last few days (http://www.bizjournals.com/washingt...eturns-to-hearing-loss-research-with-new.html)

Once I wrote, that Novartis invested about 500 Mio Dollars in Atoh1 research...
http://journals.lww.com/thehearingj...ser_to_a_Gene_Therapy_for_Hearing_Loss.6.aspx

I don't know what all that means. I think, for Genvec the reactions on the stock market must be clear.
And yes there must be a new agreement between this two companies...

Anyway, I hope other approaches will be tested in the nearer future.
Audion Therapeutics, Sound Pharmaceuticals or Decibeltx

I just need a perspective...

 

[linearb]

hey @Telis, @Anonymous2929, I am reading this very differently than you are... from the phrasing in the article, it sounds to me like the timing here was as follows:

First, Novartis told GenVac that they were halting further enrollment in the study because they want to conduct a safety review because the Data and Safety Monitoring Board (DSMB) overseeing the study recommended this.

Second, GenVac disclosed this information in a regulatory filing, and their stock lost a lot of value as a result.

Sorry for the bolding, just trying to emphasize.

So, I don't think this is cash motivated, if anything, it's contrary to the financial interests of GenVac. I think it's more likely that as @Aaron123 suggests, there was some kind of adverse effect. That does not altogether shock me, since this is a pretty dramatic and new thing to be doing in humans. I also would not necessarily think that having some kind of serious, adverse effect on one or more subjects is a sign that the technology is doomed.

This kind of thing is why I'm sort of glad I did not sign up to have UCLA put an experimental deep brain implant in me to treat tinnitus; being first in line for this sort of thing is often not a great idea.

 

[Atlantis]

This kind of thing is why I'm sort of glad I did not sign up to have UCLA put an experimental deep brain implant in me to treat tinnitus; being first in line for this sort of thing is often not a great idea.

What's that? The same thing they use for Parkinsons disease? Do you know if results for tinnitus have been published?

 

[linearb]

What's that? The same thing they use for Parkinsons disease? Do you know if results for tinnitus have been published?

as far as I know the study is still underway. It is indeed what's used for parkinson's. Three asides:

* there was one study which found 4 cases of people with parkinson's who had tinnitus go away or get better following a DBI procedure that was intended to treat their parkinson's.

* i found another, unrelated story of an ENT who had tinnitus for decades, who had it go away after he had a stroke. the part of his brain which was damaged in the stroke is the same part of the brain that they have to cut through to put in a parkinson's implant, which begs the question for me, what causes the tinnitus improvement, is it the implant itself or is it just that the brain damage from putting the implant in messes up the tinnitus pathways?

* much more worryingly, I found another analysis which found fifteen cases of people with parkinson's who did not have tinnitus, who developed tinnitus after the deep brain implant procedure

the last point there makes me think it's probably a good thing I wasn't desperate enough to get my skull cracked open; until there is a much better understanding of how this works, it seems like a huge gamble.

 

[Atlantis]

the last point there makes me think it's probably a good thing I wasn't desperate enough to get my skull cracked open; until there is a much better understanding of how this works, it seems like a huge gamble.

Yes I agree your decision was wise. Isn't the implantation procedure reversible though so it can be removed if needed?

Thanks for the info!

 

[linearb]

Yes I agree your decision was wise. Isn't the implantation procedure reversible though so it can be removed if needed?

Thanks for the info!

It can be removed, but, they cut through your brain to put the thing in -- that's not reversible, and based on my (non-doctorly) understanding of this, it seems like the brain damage might be the thing which impacts tinnitus.

I wish there were a non-surgical way to temporarily blank out parts of the brain. Like, if they could "numb" that region of my brain so I could see what impact that had on the tinnitus, then I could say "hey, it's better, take it out!" or "woah! it's worse! leave it in there!". No way to do that, though. TMS can do this to a very limited degree, but only to regions of the brain which are pretty close to the skull, as far as I know. Those parkinson's implants are way in there.

 

[Reinier]

I posted this on another thread, (denver-man-gets-gene-therapy-to-restore-hearing) but what I understand is that it has something to do with stock.

http://finance.yahoo.com/news/shareholder-alert-bronstein-gewirtz-grossman-175500268.html
quote:
"This investigation concerns whether GenVec and certain of its officers and/or directors have violated the Federal Securities Laws under the Securities Exchange Act of 1934 (the "Exchange Act")."
End quote.

When I looked up the Securities Exchange Act of 1934 (the "Exchange Act")."
I conclude it has something to do with stock trading and not with the gen technology.

After this news on Monday, stock price opened 35% lower than closing price on Friday.

 

[lapidus]

What's that? The same thing they use for Parkinsons disease? Do you know if results for tinnitus have been published?

https://www.tinnitustalk.com/threads/deep-brain-stimulation-dbs-for-tinnitus.3625/

 

[Aaron123]

I posted this on another thread, (denver-man-gets-gene-therapy-to-restore-hearing) but what I understand is that it has something to do with stock.

http://finance.yahoo.com/news/shareholder-alert-bronstein-gewirtz-grossman-175500268.html
quote:
"This investigation concerns whether GenVec and certain of its officers and/or directors have violated the Federal Securities Laws under the Securities Exchange Act of 1934 (the "Exchange Act")."
End quote.]

The yahoo.com link is labelled as an "Attorney Advertisement". Thus, while there may indeed be something to investigate, this seems more like a law firm looking for possible clients. At least at the moment, it isn't as if the Justice department or the SEC is investigating. I don't know anything about this sort of thing, but I could imagine law firms doing similar advertising any time some "bad" news comes out and a stock price falls.

 

[RB2014]

I know we are all speculating, but if there really was some type of adverse affect on one of the participants, I think they would have indefinitely stopped the entire experiment. Maybe someone had something happen and they are determining if it is related to the gene therapy. I wish we knew more. I'm pretty sure they don't mess around with this gene therapy stuff given what happened in the initial experiments where some people died many years ago.

The last article I read was from one of the participants, Amanda, and she was making progress and regaining some lost hearing.

 

[Nick Pyzik]

I know we are all speculating, but if there really was some type of adverse affect on one of the participants, I think they would have indefinitely stopped the entire experiment. Maybe someone had something happen and they are determining if it is related to the gene therapy. I wish we knew more. I'm pretty sure they don't mess around with this gene therapy stuff given what happened in the initial experiments where some people died many years ago.

The last article I read was from one of the participants, Amanda, and she was making progress and regaining some lost hearing.

I actually added one of the participants on their facebook and was chatting with them a few months ago. They had noticed a little bit of changes in their one ear but not to much. I just contacted them again yesterday to see how progress has been and they replied with no changes at all since a month or two ago. Although the procedure was only done in one ear, it seems like there hasn't been a whole lot of signs showing that the experimental procedures were working well. This participant also received the highest dose out of everyone else in the clinical trials. Maybe there just wan't enough signs of change coming out of the doses being given to the patients. They could stopping to fix any minor issues from the drug so that it works better when they resume clinical trials.

 

[RB2014]

As tomytl mentioned, they invested 500 million in additional research. Maybe they found something like you mentioned to tweak the forumula? but I would think that they would have just finished the clinical trial to see what happened and started another. All they have are the animal models right now. I remember reading that some of the animals that got too high a dose showed less improvement than the ones that got a lower dose. Maybe they found that point and are adjusting dosage back down?

 

[tomytl]

I actually added one of the participants on their facebook and was chatting with them a few months ago. They had noticed a little bit of changes in their one ear but not to much. I just contacted them again yesterday to see how progress has been and they replied with no changes at all since a month or two ago. Although the procedure was only done in one ear, it seems like there hasn't been a whole lot of signs showing that the experimental procedures were working well. This participant also received the highest dose out of everyone else in the clinical trials. Maybe there just wan't enough signs of change coming out of the doses being given to the patients. They could stopping to fix any minor issues from the drug so that it works better when they resume clinical trials.

It's very interesting to read about outcomes of the trial.
As from what I learned from different talks with some of leading hearing researchers, all of them
didn't expect a relevant increase of hearing... but some of them mentioned, if they could proof
save delivery of drugs this way would be good option.
Since Novartis licensed some of Genvec's hearing programm, nobody excatly knows
what they might have change in theirs labs.

I hope they will get back to trials and nobody of the people participating got harmed.

 

[Nick Pyzik]

It's very interesting to read about outcomes of the trial.
As from what I learned from different talks with some of leading hearing researchers, all of them
didn't expect a relevant increase of hearing... but some of them mentioned, if they could proof
save delivery of drugs this way would be good option.
Since Novartis licensed some of Genvec's hearing programm, nobody excatly knows
what they might have change in theirs labs.

I hope they will get back to trials and nobody of the people participating got harmed.

Yes exactly. I believe I read on one of their powerpoints that a minimal amount of increased hearing abilities would be excellent with this new drug delivery. I hope that no one had any side effects from this procedure too. All we can do is wait.

 

[Reinier]

The last article I read was from one of the participants, Amanda, and she was making progress and regaining some lost hearing.

I assume this is the same person?
http://www.islandpacket.com/news/business/technology/article47711735.html

I am sure that on this forum more than ones a link to this article or similar ones have been posted.

Quote from this article:
Amanda James first noticed a difference early this year when she picked up a pill bottle at the Norfolk, Va., pharmacy where she worked and shook it next to her right ear.
For the first time, she could hear the pills rattling inside.
End quote.

I have been wondering about this news. This article is from December last year. Why wasn't this on the news in every other news program? I would have thought that any regeneration of hearing in humans is regarded as a break through. Is it expected this gain in hearing is temporary? I find it unlikely this being a placebo effect.

​

 

[frogatog]

Hi Everyone, I have not read all of the replies here, but I am involved in the stock market in my professional life.

This is a very small trial with a small number of people. The fact that it is paused ENROLLMENT for a safety reason could mean many things -- if even one person in the trial had a serious medical issue which may have been unrelated to the trial they might be required to do this. Keep in mind they said they were pausing enrollment; they didn't say anything about not continuing treatment for those involved.

Also keep in mind that GenVec as a company might not be financially viable, however if the drug itself has potential there is no question that Novartis will find a way to continue to develop it. It just might not be a great situation for GenVec shareholders.

I would say that given the very low value the market is putting on GenVec itself, it is saying that it is a long shot that the drug will reach market in its current form. However, if I were watching these trials as an observer, I would certainly not give up hope in the technology.

I personally bought some shares of GenVec after this announcement. Yes, it could go to zero, but as others have said, there may yet be something real here.

 

[Reinier]

Hi Everyone, I have not read all of the replies here, but I am involved in the stock market in my professional life.

I am curious.
Could you please comment on the fact that after the shareholders alert on Monday, stock went down considerably.
The share holders alert was a reaction on this quote , if I am not mistaken:
quote:
"This investigation concerns whether GenVec and certain of its officers and/or directors have violated the Federal Securities Laws under the Securities Exchange Act of 1934 (the "Exchange Act")."
End quote.[/QUOTE]
http://finance.yahoo.com/news/shareholder-alert-bronstein-gewirtz-grossman-175500268.html

I have been thinking about the fact this could be a good time to buy stock. I am a novice though and at the moment am not able to give it my undivided attention.
Just Imagen what would happen with Genvec stock prices if this therapy has a positive outcome.

I still believe this therapy could be a first important step. Time will tell.

 

[frogatog]

Any time a stock goes down a lot, there will be shareholder lawsuits.

GenVec's stock could easily go to zero, because they have under $10 million in cash and they are using over $4 million a year. Thus, a set back like this which slows them down a lot could mean that they have to raise new funds at a high cost. I would consider a stock like this more of a lottery ticket, where you could lose all of your money, or if you are lucky, make 10-20 times your investment, or even more. Caveat emptor!

 

[Lauren Morse]

I'm hoping there is a legitimate safety concern....I am very disappointed that they will not disclose the reason. Perhaps the entire project would be in jeopardy if negative information was released, but I still find all of this secrecy to be ridiculous when so many people are holding onto that hope.

I'm just hoping this new development isn't going to slowly suffocate the project. We know atoh-1 is working from anecdotal reports and restoring some noticeable hearing; no, it's not going to be the magic bullet but it's a very critical stepping stone to inspiring and fine-tuning future therapies. If this gets thrown under the bus think of all the discouragement that could spread to other endeavors...

In essence I believe there needs to be greater emphasis on the positive outcomes so far for this therapy, not all of these repeating negative stories and hidden information.

 

[Reinier]

@Lauren Morse.
I agree whole heartedly with your post.

no, it's not going to be the magic bullet but it's a very critical stepping stone to inspiring and fine-tuning future therapies.

Exactly my point of view.

Something new in my daily routine is checking stock prices of companies like Novartis, Genvec and Otonomy and hopefully more in the near future.

I would consider a stock like this more of a lottery ticket, where you could lose all of your money, or if you are lucky, make 10-20 times your investment, or even more. Caveat emptor!

Winning something in the lottery is highly unlikely. Are you saying it is highly unlikely Genvec will recover? I won't be buying shares and therefore am not asking advice in regards to this, but am interested in Genvec and their drug CGF-166