Re: CMT 1A: Pharnext Pleodrug PXT3003 Phase 2 Trials BEGIN

December 3, 2010

This is VERY BIG NEWS for those with CMT 1A!

>

> PHARNEXT Receives Clinical Trial Authorization for a Phase II Study with the

First Drug Generated by Its PleotherapyTM Technology

>

http://www.benzinga.com/press-releases/10/12/b665794/pharnext-receives-clinical-\

trial-authorization-for-a-phase-ii-study

>

> Pharnext SAS, a biopharmaceutical company specializing in the development of

innovative treatments for severe neurological diseases and other major unmet

medical needs, today announced that the French Agency for Healthcare Product

Safety (AFSSAPS) has authorized a Phase II clinical trial with the first drug (a

Pleodrug™) generated by the company's Pleotherapy™ technology platform in

patients suffering from Charcot-Marie Tooth disease type 1A (CMT1A) - a chronic,

severe, invalidating and currently incurable neuromuscular disease which affects

3 million people worldwide.

>

> Pharnext's first Pleodrug™ is a composed of three generic drugs in a new

formulation (a syrup) and which have already received marketing approval for

therapeutic indications other than CMT1A. Given that the three compounds are

present in the Pleodrug™ at 10- to 100-fold lower doses than in their usual

indication, the clinical development programme in humans has not necessitated

the performance of a Phase I safety trial and has enabled Pharnext to obtain a

Phase II clinical trial authorization directly after just 3 years (rather than

the 8 years required on average for most drugs).

>

> The forthcoming Phase II randomized, comparative, placebo-controlled clinical

trial will evaluate the safety, efficacy, pharmacodynamics and pharmacokinetics

of PXT3003 in patients suffering from CMT1A. The trial will be performed in 5

investigating centres (Timone University Hospital in Marseilles, Salpêtrière

Hospital in Paris, Limoges University Hospital, Lyons Sud University Hospital

and Pierre Swynghedauw Hospital in Lille) and will be initiated first at Timone

University Hospital (coordinating investigator: Shahram Attarian MD) in

December.

>

> " This first clinical trial authorization constitutes key proof of the

efficiency of our Pleotherapy™ technology. We are continuing to screen for

Pleodrugs™ for six other serious diseases (including Alzheimer's disease); our

model means that we can hope to make Pleodrugs™ available to patient more

rapidly " , emphasized Pharnext founder and CEO Professor Cohen. " We are

delighted to be able to start the first clinical trial of our first Pleodrug™

for the treatment of Charcot-Marie-Tooth disease " , he concluded.

>

> Pharnext has designed a novel, proprietary platform (Pleotherapy™) based on

network pharmacology. The company's discovery engine identifies rational drug

combinations (Pleodrugs™) made up of individual Pleocompounds™. This innovative

approach could provide a solution to the ongoing and worrying decline in

pharmaceutical pipeline productivity.

>

> About Pharnext

> Pharnext is a biopharmaceutical company focused on discovering, developing and

licensing new pharmaceutical treatments based on intelligent Pleocompounds™ that

form the basis of Pleodrugs™. Pharnext was founded in Paris, France in April,

2007, by Professor Cohen, MD, PhD, his research group (composed of

pioneers in genome science and technology) and Philippe Pouletty, MD (General

Partner at Truffle Capital). Thanks to Truffle Capital's founding investment,

strong support from OSEO, a grant from the French Muscular Dystrophy Association

(AFM) and the benefits of France's research tax credit scheme, Pharnext has

developed a novel, proprietary platform (PleotherapyTM) based on network

pharmacology and which may provide a solution for the current decline in

pharmaceutical pipeline productivity. Pharnext is advised by La Compagnie

Financière Edmond de Rothschild.

>

December 4, 2010

I wonder how long the clinical trial will take. Anyone have any ideas?

In a message dated 12/3/2010 4:02:29 P.M. Pacific Standard Time,

gfijig@... writes:

This is VERY BIG NEWS for those with CMT 1A!

>

> PHARNEXT Receives Clinical Trial Authorization for a Phase II Study with

the First Drug Generated by Its PleotherapyTM Technology

>

_http://www.benzinga.com/press-releases/10/12/b665794/pharnext-receives-clinical\

-trial-authorization-for-a-phase-ii-study_

(http://www.benzinga.com/press-releases/10/12/b665794/pharnext-receives-clinical\

-trial-authorization-fo

r-a-phase-ii-study)

>

> Pharnext SAS, a biopharmaceutical company specializing in the

development of innovative treatments for severe neurological diseases and other

major

unmet medical needs, today announced that the French Agency for Healthcare

Product Safety (AFSSAPS) has authorized a Phase II clinical trial with the

first drug (a Pleodrugâ„¢) generated by the company's Pleotherapyâ„¢

technology platform in patients suffering from Charcot-Marie Tooth disease type

1A

(CMT1A) - a chronic, severe, invalidating and currently incurable

neuromuscular disease which affects 3 million people worldwide.

>

> Pharnext's first Pleodrugâ„¢ is a composed of three generic drugs in a new

formulation (a syrup) and which have already received marketing approval

for therapeutic indications other than CMT1A. Given that the three compounds

are present in the Pleodrugâ„¢ at 10- to 100-fold lower doses than in their

usual indication, the clinical development programme in humans has not

necessitated the performance of a Phase I safety trial and has enabled Pharnext

to obtain a Phase II clinical trial authorization directly after just 3

years (rather than the 8 years required on average for most drugs).

>

> The forthcoming Phase II randomized, comparative, placebo-controlled

clinical trial will evaluate the safety, efficacy, pharmacodynamics and

pharmacokinetics of PXT3003 in patients suffering from CMT1A. The trial will be

performed in 5 investigating centres (Timone University Hospital in

Marseilles, SalpÃªtriÃ¨re Hospital in Paris, Limoges University Hospital, Lyons

Sud

University Hospital and Pierre Swynghedauw Hospital in Lille) and will be

initiated first at Timone University Hospital (coordinating investigator:

Shahram Attarian MD) in December.

>

> " This first clinical trial authorization constitutes key proof of the

efficiency of our Pleotherapyâ„¢ technology. We are continuing to screen for

Pleodrugsâ„¢ for six other serious diseases (including Alzheimer's disease); our

model means that we can hope to make Pleodrugsâ„¢ available to patient more

rapidly " , emphasized Pharnext founder and CEO Professor Cohen. " We

are delighted to be able to start the first clinical trial of our first

Pleodrugâ„¢ for the treatment of Charcot-Marie-Tooth disease " , he concluded.

>

> Pharnext has designed a novel, proprietary platform (Pleotherapyâ„¢) based

on network pharmacology. The company's discovery engine identifies

rational drug combinations (Pleodrugsâ„¢) made up of individual

Pleocompoundsâ„¢.

This innovative approach could provide a solution to the ongoing and worrying

decline in pharmaceutical pipeline productivity.

>

> About Pharnext

> Pharnext is a biopharmaceutical company focused on discovering,

developing and licensing new pharmaceutical treatments based on intelligent

Pleocompoundsâ„¢ that form the basis of Pleodrugsâ„¢. Pharnext was founded in

Paris,

France in April, 2007, by Professor Cohen, MD, PhD, his research

group (composed of pioneers in genome science and technology) and Philippe

Pouletty, MD (General Partner at Truffle Capital). Thanks to Truffle Capital's

founding investment, strong support from OSEO, a grant from the French

Muscular Dystrophy Association (AFM) and the benefits of France's research tax

credit scheme, Pharnext has developed a novel, proprietary platform

(PleotherapyTM) based on network pharmacology and which may provide a solution

for the current decline in pharmaceutical pipeline productivity. Pharnext is

advised by La Compagnie FinanciÃ¨re Edmond de Rothschild.

>

December 4, 2010

,

I hope to have this info soon.

Gretchen

> >

> > PHARNEXT Receives Clinical Trial Authorization for a Phase II Study with

> the First Drug Generated by Its PleotherapyTM Technology

> >

>

_http://www.benzinga.com/press-releases/10/12/b665794/pharnext-receives-clinical\

-trial-authorization-for-a-phase-ii-study_

>

(http://www.benzinga.com/press-releases/10/12/b665794/pharnext-receives-clinical\

-trial-authorization-fo

> r-a-phase-ii-study)

> >

> > Pharnext SAS, a biopharmaceutical company specializing in the

> development of innovative treatments for severe neurological diseases and

other major

> unmet medical needs, today announced that the French Agency for Healthcare

> Product Safety (AFSSAPS) has authorized a Phase II clinical trial with the

> first drug (a Pleodrugâ„¢) generated by the company's Pleotherapyâ„¢

> technology platform in patients suffering from Charcot-Marie Tooth disease

type 1A

> (CMT1A) - a chronic, severe, invalidating and currently incurable

> neuromuscular disease which affects 3 million people worldwide.

> >

> > Pharnext's first Pleodrugâ„¢ is a composed of three generic drugs in a new

> formulation (a syrup) and which have already received marketing approval

> for therapeutic indications other than CMT1A. Given that the three compounds

> are present in the Pleodrugâ„¢ at 10- to 100-fold lower doses than in their

> usual indication, the clinical development programme in humans has not

> necessitated the performance of a Phase I safety trial and has enabled

Pharnext

> to obtain a Phase II clinical trial authorization directly after just 3

> years (rather than the 8 years required on average for most drugs).

> >

> > The forthcoming Phase II randomized, comparative, placebo-controlled

> clinical trial will evaluate the safety, efficacy, pharmacodynamics and

> pharmacokinetics of PXT3003 in patients suffering from CMT1A. The trial will

be

> performed in 5 investigating centres (Timone University Hospital in

> Marseilles, SalpÃªtriÃ¨re Hospital in Paris, Limoges University Hospital,

Lyons Sud

> University Hospital and Pierre Swynghedauw Hospital in Lille) and will be

> initiated first at Timone University Hospital (coordinating investigator:

> Shahram Attarian MD) in December.

> >

> > " This first clinical trial authorization constitutes key proof of the

> efficiency of our Pleotherapyâ„¢ technology. We are continuing to screen for

> Pleodrugsâ„¢ for six other serious diseases (including Alzheimer's disease);

our

> model means that we can hope to make Pleodrugsâ„¢ available to patient more

> rapidly " , emphasized Pharnext founder and CEO Professor Cohen. " We

> are delighted to be able to start the first clinical trial of our first

> Pleodrugâ„¢ for the treatment of Charcot-Marie-Tooth disease " , he concluded.

> >

> > Pharnext has designed a novel, proprietary platform (Pleotherapyâ„¢) based

> on network pharmacology. The company's discovery engine identifies

> rational drug combinations (Pleodrugsâ„¢) made up of individual

Pleocompoundsâ„¢.

> This innovative approach could provide a solution to the ongoing and worrying

> decline in pharmaceutical pipeline productivity.

> >

> > About Pharnext

> > Pharnext is a biopharmaceutical company focused on discovering,

> developing and licensing new pharmaceutical treatments based on intelligent

> Pleocompoundsâ„¢ that form the basis of Pleodrugsâ„¢. Pharnext was founded in

Paris,

> France in April, 2007, by Professor Cohen, MD, PhD, his research

> group (composed of pioneers in genome science and technology) and Philippe

> Pouletty, MD (General Partner at Truffle Capital). Thanks to Truffle

Capital's

> founding investment, strong support from OSEO, a grant from the French

> Muscular Dystrophy Association (AFM) and the benefits of France's research

tax

> credit scheme, Pharnext has developed a novel, proprietary platform

> (PleotherapyTM) based on network pharmacology and which may provide a

solution

> for the current decline in pharmaceutical pipeline productivity. Pharnext is

> advised by La Compagnie FinanciÃ¨re Edmond de Rothschild.

> >

>

December 5, 2010

How many trial phases are needed to make sure the treatment works and cause no

harm to health?

Thank you very much.

May you be well and happy

Caroline

December 5, 2010

I believe this is very big news for all people with CMT!

Although this trial is for CMT1A specifically I would speculate that this

company would use the same technology to create other medications for all forms

of CMT.

With this announcement there are now two avenues of drug research for CMT - Star

and pleodrugs - providing a very good possibility of significant treatments for

all forms of CMT in the next few years

Take care

December 5, 2010

Thank you for sharing this - made my day : )

________________________________

From: cmt_forum <cmt_forum@...>

Sent: Sun, December 5, 2010 12:31:37 PM

Subject: Re: CMT 1A: Pharnext Pleodrug PXT3003 Phase 2 Trials BEGIN

I believe this is very big news for all people with CMT!

Although this trial is for CMT1A specifically I would speculate that this

company would use the same technology to create other medications for all forms

of CMT.

With this announcement there are now two avenues of drug research for CMT - Star

and pleodrugs - providing a very good possibility of significant treatments for

all forms of CMT in the next few years

Take care

December 6, 2010

I also believe treatments for other CMT types will come out soon. As far as

I know, different types are mutations from each other.

Let us keep hope.

On Mon, Dec 6, 2010 at 6:27 AM, christine mcguire <

zchristinemcguire@...> wrote:

>

> Thank you for sharing this - made my day : )

>

> ________________________________

> From: cmt_forum <cmt_forum@... <cmt_forum%40.ca>>

> <%40>

> Sent: Sun, December 5, 2010 12:31:37 PM

> Subject: Re: CMT 1A: Pharnext Pleodrug PXT3003 Phase 2 Trials BEGIN

>

> I believe this is very big news for all people with CMT!

>

> Although this trial is for CMT1A specifically I would speculate that this

> company would use the same technology to create other medications for all

> forms

> of CMT.

>

> With this announcement there are now two avenues of drug research for CMT -

> Star

> and pleodrugs - providing a very good possibility of significant treatments

> for

> all forms of CMT in the next few years

>

> Take care

>

December 6, 2010

another thought, I forgot to add, is I have read, on occasion drug trials will

be shortened if medications are showing very positive results and are then

distributed to the general public. Whether this happens with this trial will yet

to be determined

Take care

>

> >

> > Thank you for sharing this - made my day : )

> >

> > ________________________________

> > From: cmt_forum <cmt_forum@... <cmt_forum%40.ca>>

> > <%40>

> > Sent: Sun, December 5, 2010 12:31:37 PM

> > Subject: Re: CMT 1A: Pharnext Pleodrug PXT3003 Phase 2 Trials BEGIN

> >

> > I believe this is very big news for all people with CMT!

> >

> > Although this trial is for CMT1A specifically I would speculate that this

> > company would use the same technology to create other medications for all

> > forms

> > of CMT.

> >

> > With this announcement there are now two avenues of drug research for CMT -

> > Star

> > and pleodrugs - providing a very good possibility of significant treatments

> > for

> > all forms of CMT in the next few years

> >

> > Take care

> >

December 7, 2010

Hi in Manitoba,

This is exactly what Pleodrugs are about. Using a combo of medications already *

on the market * (for other diseases that work) mixtures of chemicals can be put

together for extra positive effect, or so that is the idea.

By using medications already on the market, it has allowed Pharnext to skip

Phase 1 trials and advance to human trials (Phase 2) quickly.

Gretchen

December 7, 2010

I have passed along this press release (well the one on the same subject)

to my doctors at the National Hospital for Neurology in the UK, as Dr

Reilly is one of the most active researchers into CMT.

From what Gretchen says the trials are designed to use existing drugs and at

this stage to slow progress of CMT.

If I find out more from the researchers here I will pass it along to the list.

Donna from London

www.beatinglimitations.com

December 8, 2010

In Taiwan, we have national health insurance and laws to help purchase

orphan drugs for rare disorders, that is, government will pay.

But I am not sure whether the treatments will be marketed or sold as

ordinary drugs or orphan drugs......

I really hope CMT treatments ,a miracle, can appear on the earth. I have

waited all my life for this miracle........

On Tue, Dec 7, 2010 at 8:57 PM, Donna <ddh49@...> wrote:

>

> I have passed along this press release (well the one on the same

> subject) to my doctors at the National Hospital for Neurology in the UK, as

> Dr Reilly is one of the most active researchers into CMT.

>

> From what Gretchen says the trials are designed to use existing drugs and

> at this stage to slow progress of CMT.

>

> If I find out more from the researchers here I will pass it along to the

> list.

>

> Donna from London

> www.beatinglimitations.com

>

--

May you be well and happy

Caroline

December 9, 2010

I'm sorry if I missed this, but could someone give examples of 'pleodrug'

therapy, e.g. the Rx drugs being used, etc.?

And - I'm not being facetious here - why are we so excited about this new

therapy, i.e. specifically, what can we point to that gives us concrete hope?

December 9, 2010

Augold,

If you go back to Dec. 3 posts, you'll find the original and my comments after.

But one more time, a " pleodrug " is a combination of medications already 'on the

market' - example maybe Rebif, Embrel and Neurontin. (THESE ARE ONLY EXAMPLES)

The scientists develop chemical 'mixtures' of medications formerly used for

other diseases - like how Nerontin first started - as you remember, it's first

use is an anti-epleptic. Then scientists discovered it had positive effects on

CFS, then it had positive effects on Diabetic Neuropathy, then it made it's way

to be used to help the peripheral nerve pain of CMT.

OK, back to Pleodrugs, so several medications are used for their * positive *

effects to see if a certain combination will help persons with neurogenerative

diseases slow or stop symptoms - here, Phase 2 trials have started in France.

Because the medications are already 'on the market', this allows Pharnext to

skip Phase 1 and therefore speed up trials to Phase 2 (testing on us, people

with CMT 1A in this case)

Let me make sure you and everyone else understands the 3 drugs I mentioned above

are only * an example * of a 'mix'. The Pharnext Trials is a double-blind,

placebo trials - that means NO ONE - not even the researchers - knows which

patient receives the 'mix' and which one doesn't. And this will all take 2

years, with more medications added along the way. Dr. Cohen explained to me we

'may have' a mid-trial indication Dec. 2011.

As for why this is exciting: Pharnext is a private, venture capital funded

biotech corporation with a whole lot of private funders. (includes some from

Silicon Valley even though Pharnext is in France) Having said this, money is

flowing steadily and rapidly in for this) This is opposed to non - profit

organizations who either have several hundred diseases to research, or CMT

organizations who are dependent on the government, holding special events and

their members to ante up the bucks. So Pharnext raises more capital for the

project and also stays true to their word about their timeline. Once Pharnext

completes Phase 2, the reasearch will also be turned over to Ibsen

Biopharmeceuticals for further research and a drug delivery system (pill,

liquid, patch, or something else)

Why is this exciting?! Because this is a BIG DEAL that venture capital is

backing CMT research, can form solid business partnerships and stay on timeline

track. And people with CMT 1A are 2 years away from some real help. And this

research may also provide clues to what may work for Type 2 and X.

Because of this, 2 years from right now, we may have a treatment 'mix' that

works to slow/stop CMT progression (think cocktail) The medications given in

this Phase 2 trial are in liquid form (syrup) which also hits the bloodstream

faster and may provide better results, with no side effects.

Gretchen

December 9, 2010

Hello Gretchen and All,

Speaking as an engineer, it is not only what individual drugs that make

up the mix but what amounts of each that are on trial. The initial

research has shown a beneficial effect, but is it viable over a broad

spectrum of people to the extent that it can become a standard

application. In addition, there is always the possibility that a

combination of drugs may react differently than the same drugs given

individually. I believe that ongoing research will lead to individual

combinations that will prove beneficial to individual sets of symptoms.

For many years now, I have taken a " combination " drug therapy of

Neurontin, and a muscle relaxant primarily to relieve night muscle

spasms, but I also take other drugs for heart conditions and metabolism

problems. In all it is 14 pills a day all of which contribute as a

mixture and quite possibly in total either beneficial or derogatory for

HNPP. It progresses slowly and I get more disabled with each passing

year. Is the rate of deterioration faster or slower than being without

them? No one knows and we have a long way to go to find out.

And, what about the recent protein application that has shown ability to

rebuild myelin sheath structures? And, what about the other recent

therapy applied to a spinal nerve injured war dog that took back fat

tissue and genetically altered it to rebuild the injured spinal nerves

which allowed the dog to regain use of it's hind quarters? These are

exciting possibilities and all that we can do for now is help to maintain

the flow of funds toward the research projects that will some day provide

relief. At 75 years, I may not get help from them but somebody will if we

persevere.

EdM from NH

December 9, 2010

Gretchen,

Thank you for clarifying that these are compounds, not just individual drugs.Â

It does make me curious about something.

If the medication was just a single approved drug, i.e. Neurontin (as an

imaginary example), then the cost would already be determined by the

marketplace.Â For example, if the medication was already available as a

generic, then it would stay available as a generic.

However, if the medication is actually a new compound formula of existing

medications, it may be possible for them to get new Brand Name protection for

their unique blend, even if the individual drugs are already available in

generic form. (similar example = Vytorin)Â Would that mean they could also get

Orphan drug protection as well?Â I'm not sure, but that may be possible as

well.

Certainly, the focus here is that this is GREAT news.Â Hopefully, it will also

be something that affordable for all who could benefit from it.Â Perhaps we'll

just have to wait and see...unless you have additional insight to add.

From: gfijig <gfijig@...>

Subject: Re: CMT 1A: Pharnext Pleodrug PXT3003 Phase 2 Trials BEGIN

Date: Thursday, December 9, 2010, 9:28 AM

Â