From: Beall, Bob
Sent: Monday, October 20, 2008 8:40 AM
To: All Chapter Employees; All Main Office Staff
Subject: Positive News About VX-770 and CF Patient Assistance Foundation
To All National and Chapter Employees:
Good morning. Given all of the turbulence in the world lately, it is always nice to be able to share good news. Today is going to be a remarkable day for our Foundation and Cystic Fibrosis Services. We have two important announcements that we want to share with you. Neither would be possible without your commitment and focus on our shared goals.
First is some very exciting news regarding the development of VX-770, a promising therapy being developed and tested by Vertex Pharmaceuticals with support from the CF Foundation. This morning Vertex announced positive results from the second part of a Phase 2a clinical trial of VX-770. Patients who took the investigational drug over a longer 28-day period had significant improvement in lung function, sweat chloride and nasal potential difference -- important indicators of CF.
These results reinforce the positive results reported last March from the first part of the trial and validate that with oral "CFTR modulators" we are on the right path to developing new therapies that treat the basic defect in CF. Based on this positive data, Vertex Pharmaceuticals plans to work with regulatory agencies, including the U.S. Food and Drug Administration, to move VX-770 forward in the approval process. The company is targeting the first half of 2009 to initiate a pivotal trial that could lead to drug approval.
The second piece of exciting news today is not science, yet is as critically important for those with CF and their families. Today, we will officially launch the Cystic Fibrosis Patient Assistance Foundation (CFPAF). The CFPAF will provide financial assistance to people with cystic fibrosis who cannot afford certain high-cost medications and medical devices.
While 99 percent of people with CF have health insurance, more and more patients find their coverage is inadequate. In the past year alone, data suggests that one in four CF patients skipped doses of their medicine or took smaller doses because of cost, and nearly one in five delayed seeking care for the same reason.
All funding for the CFPAF will be provided exclusively by pharmaceutical manufacturers. No dollars donated to the CF Foundation will be used for patient assistance. In fact, the CFPAF was specifically designed to avoid any conflicts or confusion. CF Foundation donations, as always, will continue to support research for a cure.
We all hope that this new entity, in conjunction with our drug development efforts, will provide a platform for creating innovative programs that will help patients have access to the new therapies that have been approved.
In the beginning, this program is geared to a select (and relatively small) group of patients who have inadequate insurance and is based on specific eligibility guidelines. Please refer families to the Website for more information (<a target=_blank class=ftalternatingbarlinklarge href="http://www.cfpaf.org/),">http://www.cfpaf.org/),</a> or have them call 1-888-315-4154 for personal consult. For those of you in the chapters, you will receive more details on this entire program via a teleconference briefing this afternoon.
In addition to the above, on Wednesday, we will begin to host more than 3,700 scientists and caregivers, from around the world, at our 22nd Annual North American CF Conference in Orlando, Florida. This represents an increase in attendance of more than 20 percent from last year -- setting the stage for what is certain to be one of the most important conferences in our history.
None of these exciting developments would be possible without your hard work and support.
As you know, we are also facing some real economic challenges that will require us in the coming weeks to look at every aspect of how we do business. I am fully confident that, working together, we will meet these challenges just as we have met so many other obstacles in the past.
Thank you for all you do each day to help bring us closer to a cure and for helping to improve the quality of life for all of those with CF.
We are grateful for the team we have in place. Together, we are making a difference.
Robert J. Beall, Ph.D.
President and CEO
Cystic Fibrosis Foundation
6931 Arlington Road
Bethesda, MD 20814
Sent: Monday, October 20, 2008 8:40 AM
To: All Chapter Employees; All Main Office Staff
Subject: Positive News About VX-770 and CF Patient Assistance Foundation
To All National and Chapter Employees:
Good morning. Given all of the turbulence in the world lately, it is always nice to be able to share good news. Today is going to be a remarkable day for our Foundation and Cystic Fibrosis Services. We have two important announcements that we want to share with you. Neither would be possible without your commitment and focus on our shared goals.
First is some very exciting news regarding the development of VX-770, a promising therapy being developed and tested by Vertex Pharmaceuticals with support from the CF Foundation. This morning Vertex announced positive results from the second part of a Phase 2a clinical trial of VX-770. Patients who took the investigational drug over a longer 28-day period had significant improvement in lung function, sweat chloride and nasal potential difference -- important indicators of CF.
These results reinforce the positive results reported last March from the first part of the trial and validate that with oral "CFTR modulators" we are on the right path to developing new therapies that treat the basic defect in CF. Based on this positive data, Vertex Pharmaceuticals plans to work with regulatory agencies, including the U.S. Food and Drug Administration, to move VX-770 forward in the approval process. The company is targeting the first half of 2009 to initiate a pivotal trial that could lead to drug approval.
The second piece of exciting news today is not science, yet is as critically important for those with CF and their families. Today, we will officially launch the Cystic Fibrosis Patient Assistance Foundation (CFPAF). The CFPAF will provide financial assistance to people with cystic fibrosis who cannot afford certain high-cost medications and medical devices.
While 99 percent of people with CF have health insurance, more and more patients find their coverage is inadequate. In the past year alone, data suggests that one in four CF patients skipped doses of their medicine or took smaller doses because of cost, and nearly one in five delayed seeking care for the same reason.
All funding for the CFPAF will be provided exclusively by pharmaceutical manufacturers. No dollars donated to the CF Foundation will be used for patient assistance. In fact, the CFPAF was specifically designed to avoid any conflicts or confusion. CF Foundation donations, as always, will continue to support research for a cure.
We all hope that this new entity, in conjunction with our drug development efforts, will provide a platform for creating innovative programs that will help patients have access to the new therapies that have been approved.
In the beginning, this program is geared to a select (and relatively small) group of patients who have inadequate insurance and is based on specific eligibility guidelines. Please refer families to the Website for more information (<a target=_blank class=ftalternatingbarlinklarge href="http://www.cfpaf.org/),">http://www.cfpaf.org/),</a> or have them call 1-888-315-4154 for personal consult. For those of you in the chapters, you will receive more details on this entire program via a teleconference briefing this afternoon.
In addition to the above, on Wednesday, we will begin to host more than 3,700 scientists and caregivers, from around the world, at our 22nd Annual North American CF Conference in Orlando, Florida. This represents an increase in attendance of more than 20 percent from last year -- setting the stage for what is certain to be one of the most important conferences in our history.
None of these exciting developments would be possible without your hard work and support.
As you know, we are also facing some real economic challenges that will require us in the coming weeks to look at every aspect of how we do business. I am fully confident that, working together, we will meet these challenges just as we have met so many other obstacles in the past.
Thank you for all you do each day to help bring us closer to a cure and for helping to improve the quality of life for all of those with CF.
We are grateful for the team we have in place. Together, we are making a difference.
Robert J. Beall, Ph.D.
President and CEO
Cystic Fibrosis Foundation
6931 Arlington Road
Bethesda, MD 20814