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Ghost Dancer

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PALS
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12/2008
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UK
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Aberdeen
Hi

I came across this information in the Wall Street Journal. Does anyone know anything beyond this slightly optimistic press release (Biogen Agrees to Develop Experimental ALS Drug

Biogen Idec expects to initiate Phase 3 program in the first half of 2011
Equity investment and potential license payments of $345 million.

Biogen and Knopp Neurosciences today announced they have entered into an exclusive, worldwide license agreement under which Biogen Idec will develop and commercialize KNS-760704 (dexpramipexole) for the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, and potentially other indications.

KNS-760704 is a novel oral neuroprotective therapy under development. In a Phase 2 study of ALS patients conducted by Knopp, the compound achieved its primary endpoint evaluating safety and tolerability and showed favorable dose-related effects in preserving motor function and extending survival. KNS-760704 has received orphan drug designation from the U.S. Food and Drug Administration and the European Commission for the treatment of patients with ALS, as well as Fast Track designation from the FDA. Biogen Idec expects to initiate a Phase 3 program of the compound in the first half of 2011.

Under the terms of the agreement, Biogen Idec will lead the development of KNS-760704 for ALS and its potential commercialization in global markets, with Knopp providing development support and conducting certain U.S. commercialization activities under the direction of Biogen Idec. As part of the transaction, Biogen Idec will purchase $60 million of Knopp stock, provide an up-front payment of $20 million and additional payments of up to $265 million based on the achievement of development, regulatory, and sales milestones. Biogen Idec will also pay tiered, double-digit royalties to Knopp on worldwide sales.
 
In April, Knopp released results of a phase 2 trial that showed some promising slowing of motor neuron degeneration and ALS progression in a dose dependent manner. They promised a phase 3 trial to get underway this year. Apparently the sale of the rights to Biogen has delayed this. The FDA has given it fast-track development but for PALS it is more like glacial-track.
 
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Wish we could take it right now. My quality of life is starting to suck! lol!
 
It does sound promising, I just wish for everyone's sake the Pharmaceutical companies would have more of a sense of urgency!
 
Hopefully they will put it on the market early enough or at least perform a trial on a compassionate basis. At least thats what im hoping for my father's sake.
 
I am doing 2 MS biogen studies at the moment. It usually takes a year or so to set up the sites and depending on the trial lenghth, usually they run for at least 1 year (MS usually 2 years) then there is the recruitment time . Our MS trial took 2 years to recruit the numbers worldwide, then the data gets all put together, so unfortunately It often takes 4 years to get it all done, sometimes even longer.

The best thing is to hope that your centre or neuro does the study and you can participate if you fulfil the Inclusion criteria.
 
I agree! "Hurry up and wait" isn't helpful in this situation!
 
kns-760704 is scheduled for phase III studies beginning in early 2011.

http://www.knoppneurosciences.com/materials/patientFaq2010.pdf

TedH - the issue is not the pharma companies, its typical gov't red tape thanks to the FDA. The benefit of this drug is it has received "fast-track" approval; from my understanding it could get thru phase III in a quick manner [circa 1yr?]. The pharma companies are interested in one thing...Profit. The faster to market the faster they can recoup costs and drive profit.

keep in mind this drug is intended to slow progression via relief of oxidative stress. I don't believe it is intended to "restore" function...call me greedy, but I want my arms/hands back!

I spoke with my neuro regarding kns-760704; at this time Univ. of Colorado is not interested in the study...unbelievable!

Dan
 
I just saw my Neurologist today, and seems I'm progressing in my right foot... sucks...

We talked about a few options, and he thinks that this drug is the most exciting news to come along in a while. I've copied some info I came across below, and will try to make it a bit more concise. BTW, the company that is developing this drug was started by Walter Knopp, who died of ALS in 2004.

Safety, Tolerability, and Pharmacokinetics of KNS-760704 (Dexpramipexole) in Healthy Adult Subjects

1. Michael E. Bozik
1. Knopp Neurosciences, Inc

1. James L. Mather
1. Knopp Neurosciences, Inc

1. William G. Kramer
1. Kramer Consulting LLC

1. Valentin K. Gribkoff
1. Knopp Neurosciences, Inc

1. Evan W. Ingersoll [email protected]
1. Knopp Neurosciences, Inc

Abstract

Dexpramipexole (KNS-760704; [6R]-4,5,6,7-tetrahydro-N6-propyl-2,6-benzothiazole-diamine) is a novel synthetic amino-benzothiazole in development for the treatment of amyotrophic lateral sclerosis (ALS). Preclinical studies have shown that dexpramipexole is neuroprotective in vitro and in vivo, is highly orally bioavailable and water soluble, and rapidly achieves and maintains high central nervous system concentrations relative to plasma. Two phase 1 clinical studies were conducted to assess the safety, tolerability, and pharmacokinetics (PK) of single and multiple doses of dexpramipexole in 54 healthy male and female adults. The effect of food on the single-dose PK of dexpramipexole was also evaluated. Single doses (50 mg, 150 mg, or 300 mg) and multiple doses (50 mg twice daily, 100 mg twice daily, or 150 mg twice daily) of dexpramipexole over 4.5 days were safe and well tolerated. Dexpramipexole was rapidly absorbed, with time to maximum plasma concentration ranging from 1.8 to 2.6 hours and half-life ranging from 6.4 to 8.1 hours under fasted conditions, and was mostly eliminated in urine as unchanged parent drug (84%-90% of dose). Food had no effect on the single-dose PK of dexpramipexole. These findings support the ongoing development of dexpramipexole for the treatment of ALS and further evaluation of the compound’s therapeutic potential in other neurodegenerative diseases.
 
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