CFF In The News

“Patient Power”
Forbes showcased the Foundation’s leadership and influence in driving drug development for a rare disease. The story highlighted the Foundation’s progress through the eyes of one CF patient, Emily Schaller. She said of the Foundation: “They’re the reason I’m alive.”
Forbes, September 15, 2008

“Breathing Easier”
A cover story in Chemical and Engineering News highlighted the Foundation’s drug development
efforts. The story notes that a stream of new drugs that treat the core defect in CF could reach patients in the next few years, thanks to the ambitious efforts of the Foundation.
C&EN, September 1, 2008

“PTC Therapeutics Announces $25 Million Award From CFFT for Development of PTC124”
PTC Therapeutics, Inc. and Cystic Fibrosis Foundation Therapeutics, Inc., the nonprofit affiliate
of the CF Foundation, announced the expansion of an existing collaboration to support the development of PTC124, a new investigational drug for the treatment of CF. PTC will receive up to $25 million from CFFT in support of clinical trials.
CTP Therapeutics, July 16, 2008

“Transave Announces Positive Phase II Results for Once daily ArikaceTM in the Treatment of CF Patients Who Have Pseudomonas Lung Infections”
Transave, Inc., announced positive Phase 2 results for its lead investigational drug, Arikace™.
The inhaled compound is being developed for the treatment of CF patients who have lung infections due to the bacterium Pseudomonas aeruginosa. Cystic Fibrosis Foundation Therapeutics, Inc., provided a $1.7 million award to support the development of Arikace.
Transave, June 13, 2008

“Inspire Announces Positive Results From Phase III Clinical Trial of Denufosol”
Inspire Pharmaceuticals, Inc., announced positive results from a Phase 3 clinical trial of denufosol, an inhaled therapy for the treatment of CF. Denufosol is designed to improve chloride
transport in the airways and enhance mucus hydration and clearance. The data from the trial is
particularly encouraging because it brings the CF community one step closer to a novel treatment that addresses the basic defect in CF.
Inspire, June 6, 2008

“Drug Makers Turning to Nonprofits for Cash”
The Boston Globe featured a story on the front-page of its business section about the Foundation’s efforts to develop effective therapies for CF. It mentioned the Foundation has awarded $300 million to biotech companies for this purpose, and announced an expanded $37.7 million collaboration with EPIX Pharmaceuticals.
The Boston Globe, April 7, 2008

“A Healthy Gamble for CF”
An article in BusinessWeek online showcased the Foundation’s unique business model and promising investments in biotech companies. The story highlighted the positive early results of an ongoing clinical trial of VX-770, an oral drug that targets the core defect in CF being developed by Vertex Pharmaceuticals.
BusinessWeek, April 3, 2008

“Vertex Reports Positive Results for Phase II Clinical Trial of VX-770”
Data from a Phase 2a clinical trial in CF patients who carry the G551D gene mutation showed that an oral dose of VX-770 for 14 days resulted in improved lung function, improved nasal potential difference measures, and sweat chloride levels. Vertex reported that, “while these are early data, it is unprecedented for an investigational oral compound for the treatment of CF
to have such a marked effect on multiple measures of CF disease activity.”
Forbes, March 27, 2008

“Landmark Clinical Trials Initiated for Drugs Designed to Repair Defective CF Protein”
In a major milestone for the Foundation, two drugs that target the core defect in CF have entered clinical trials. Vertex Pharmaceuticals, in collaboration with the Foundation, began a Phase 2 trial for VX-770 in late 2007, and in early 2008, began evaluating VX-809 in healthy volunteers. The two drugs were developed as a result of an historic collaboration, which harnesses high-throughput screening for drug discovery.
Vertex, January 2008

“Breath of Fresh Air for Cystic Fibrosis Drug Pipeline”
Pharmaceutical Approvals Monthly, a leading biotech publication, ran a story on the success of the Foundation’s drug discovery and development pipeline. The outlook for CF drug development is “a very different story than even five years ago,” said Robert J. Beall, Ph.D., president and CEO of the Foundation.
Pharm Approvals Monthly, December 2007

“Foundation Launches New Collaboration With AMRI”
CFFT, the nonprofit affiliate of the CF Foundation, announced a four-year research collaboration with AMRI, worth up to $23.7 million. The project aims to identify novel drugs that address the core CF defect.
AMRI, October 4, 2007

“Venture Philanthropy on the Rise”
An article in CenterWatch Monthly reported on the trend of venture philanthropy nationwide and identified the CF Foundation as the charitable organization spending the most on drug research and development. In fact, the Foundation will spend $48 million on drug research in 2007, three times more than any other disease foundation in the country.
CenterWatch, August 2007

“$22M to Help FoldRx Develop Drug for Cystic Fibrosis”
An article in The Boston Globe featured the CF Foundation’s new $22 million biotech collaboration with FoldRx Pharmaceuticals to discover and develop new compounds to treat the core defect in CF. In 2007 alone, the story said, the Foundation has earmarked $48 million for drug research with biotech companies as it seeks to stock its drug pipeline with promising treatments.
The Boston Globe, August 13, 2007

“Gilead Announces Expanded Access Program for Aztreonam Lysine for Inhalation for Patients With Cystic Fibrosis”
Gilead Sciences, Inc. announced the launch of an early access program Aug. 1, 2007 to provide its investigational therapy, aztreonam lysine for inhalation, to patients with cystic fibrosis and Pseudomonas aeruginosa infection who have limited treatment options and are at risk for disease progression.
Gilead, July 9, 2007

“A Different Sort of Venture”
The Foundation was prominently featured on National Public Radio’s Marketplace program. The
show, heard by 4.5 million listeners, credited the Foundation for bringing promising CF drugs to
market and helping people with CF live longer.
NPR, June 8, 2007