edjohnson
Active member
- Joined
- Nov 18, 2011
- Messages
- 48
- Reason
- PALS
- Diagnosis
- 10/2011
- Country
- US
- State
- nc
- City
- emerald isle
Dear ALSTUN Supporters:
We in the Steering Committee wanted to give you all a quick update on the current state of our progress and what our next steps are.
"If you ever want to hear God laugh, tell Him your plans for the future."
1. Neuraltus' NP001. NP001 very recently finished its Phase 2 trial and the preliminary results weren't quite as positive as many of us anticipated. However, there were some encouraging results, especially when compared against historical controls. Neuraltus is going ahead with a Phase 3 but because it is not well funded it requires a partner. Therefore, the company doesnt believe it currently has the capability for an Expanded Access Program. Additionally, it is our view that the current efficacy data would have to be tortured a little bit to get past FDA - although this is potentially a minor task. However, even with full FDA blessing, there is no chance for production until after Neuraltus obtains sufficient funding for the Phase 3. We don't anticipate Neuraltus having a substantial delay in getting the Phase 3 going early next year. Unfortunately, the reality of the Universe is that at this point, until Neuraltus obtains a partner with existing production capability and a willingness to pursue other options, we have to wait for the commencement of the Phase 3 before we can go move forward with the EAP issue. Fortunately, we are primed to act very quickly once that happens.
2. Cytokinetics' Tirasemtiv. Tirasemtiv has so far only gone through very preliminary testing in a few people for a few weeks. The company position is that even though the efficacy appears quite potent but because it has a real fundamental effect on muscle (and remember that the heart is a muscle), it must be evaluated for safety for a longer time in more people. Right now Tirasemtiv will never get past FDA safety requirements. The experts we have consulted on this have high hope, but in their view the safety exercise (which Cytokinetics is now doing) simply must be completed. We recently offered assistance in filling/populating the trial, and will post updates on the status soon. Fortunately, the current trial is just a few months. ALSTUN continues to have dialogue with Cytokinetics and we will act when the company satisfies the FDAs minimum safety requirements.
3. So where are we? Some would call it a holding pattern while required activities of others play out. While we little to no input in these activities, trying to impress ourselves into them would most likely seriously jeopardize the relationships and progress we have already made. The one good thing about these waiting periods is that it gives us an opportunity to get ourselves prepared to immediately act when the opportunity for a large scale EAP presents itself. We feel strongly that we have a very positive path forward and the Steering Committee is excited about the prospects for accelerating access to potentially efficacious drugs/treatments. It starts with our successful meeting with the FDA in August 2012 where we came away with a clear view of the FDA's position on EAPs, and the real need to focus on the drug companies.
The FDA is pro-ALS EAP. The message was clear. Get a drug company to come to us with a drug that is proven safe and has demonstrated efficacy, and the FDA will support an EAP as the drug moves forward. Based on that meeting, and subsequent meetings with drug developers we have established a primary line of operation to achieve that goal through the ALSTUN EMERGENCY TREATMENT FUND. The solution isn't easy, requires a lot of hard work, and is centered on a multi-faceted campaign that has room for many more volunteers. Our success in fund raising is directly linked to our ability to get a drug company to agree to an large scale EAP. It's a unique partnering solution that's never been tried before, but one that has a real chance of success. Join us, work with us and learn more.
Thank you all for your continued support.
We in the Steering Committee wanted to give you all a quick update on the current state of our progress and what our next steps are.
"If you ever want to hear God laugh, tell Him your plans for the future."
1. Neuraltus' NP001. NP001 very recently finished its Phase 2 trial and the preliminary results weren't quite as positive as many of us anticipated. However, there were some encouraging results, especially when compared against historical controls. Neuraltus is going ahead with a Phase 3 but because it is not well funded it requires a partner. Therefore, the company doesnt believe it currently has the capability for an Expanded Access Program. Additionally, it is our view that the current efficacy data would have to be tortured a little bit to get past FDA - although this is potentially a minor task. However, even with full FDA blessing, there is no chance for production until after Neuraltus obtains sufficient funding for the Phase 3. We don't anticipate Neuraltus having a substantial delay in getting the Phase 3 going early next year. Unfortunately, the reality of the Universe is that at this point, until Neuraltus obtains a partner with existing production capability and a willingness to pursue other options, we have to wait for the commencement of the Phase 3 before we can go move forward with the EAP issue. Fortunately, we are primed to act very quickly once that happens.
2. Cytokinetics' Tirasemtiv. Tirasemtiv has so far only gone through very preliminary testing in a few people for a few weeks. The company position is that even though the efficacy appears quite potent but because it has a real fundamental effect on muscle (and remember that the heart is a muscle), it must be evaluated for safety for a longer time in more people. Right now Tirasemtiv will never get past FDA safety requirements. The experts we have consulted on this have high hope, but in their view the safety exercise (which Cytokinetics is now doing) simply must be completed. We recently offered assistance in filling/populating the trial, and will post updates on the status soon. Fortunately, the current trial is just a few months. ALSTUN continues to have dialogue with Cytokinetics and we will act when the company satisfies the FDAs minimum safety requirements.
3. So where are we? Some would call it a holding pattern while required activities of others play out. While we little to no input in these activities, trying to impress ourselves into them would most likely seriously jeopardize the relationships and progress we have already made. The one good thing about these waiting periods is that it gives us an opportunity to get ourselves prepared to immediately act when the opportunity for a large scale EAP presents itself. We feel strongly that we have a very positive path forward and the Steering Committee is excited about the prospects for accelerating access to potentially efficacious drugs/treatments. It starts with our successful meeting with the FDA in August 2012 where we came away with a clear view of the FDA's position on EAPs, and the real need to focus on the drug companies.
The FDA is pro-ALS EAP. The message was clear. Get a drug company to come to us with a drug that is proven safe and has demonstrated efficacy, and the FDA will support an EAP as the drug moves forward. Based on that meeting, and subsequent meetings with drug developers we have established a primary line of operation to achieve that goal through the ALSTUN EMERGENCY TREATMENT FUND. The solution isn't easy, requires a lot of hard work, and is centered on a multi-faceted campaign that has room for many more volunteers. Our success in fund raising is directly linked to our ability to get a drug company to agree to an large scale EAP. It's a unique partnering solution that's never been tried before, but one that has a real chance of success. Join us, work with us and learn more.
Thank you all for your continued support.
