The Cystic Fibrosis Foundation (CFF) and Cystic Fibrosis Foundation Therapeutics (CFFT) are non-profit organizations that fund research to cure cystic fibrosis. They invest $73.2 million annually in research. CFFT operates a drug development program that has brought 9 therapies to market and has 13 drugs in clinical trials. Funding comes primarily from donations and the sale of intellectual property rights, and is used to support research grants, fellowships, and drug discovery efforts through matching awards to researchers and a network of clinical centers. This model has yielded successful treatments for cystic fibrosis and billions in returns through drug approvals that are reinvested into further research.

1. Inception Date: 1955 (CFF), 2000 (CFFT)
Investment Size: $73.2 million annually (5 yr. avg)
Products in Market: 9
Products in Clinical Pipeline: 13
Overview
The Cystic Fibrosis Foundation (CFF) and Cystic Fibrosis Foundation Therapeutics (CFFT) are not-for-profit voluntary health organizations
whose mission is to cure cystic fibrosis by funding research and drug development, promoting individualized treatment, and ensuring access
to high quality, specialized care. The CFFT operates the Therapeutics Development Program, which supports activities related to cystic
fibrosis drug discovery through the many stages of drug development and clinical evaluation.
The Cystic Fibrosis Foundation
Investment Pipeline
• CFF and CFFT funds a CF research consortia focused on: CFTR folding, CFTR 3D structure, Mucocilliary clearance, and CF Biomarkers.
• The Foundation provides individual monetary awards to U.S. based clinicians and researchers tenable from 4 months to 5 years.
• Research is funded on a project-by-project basis through open annual calls for application. Applicants for funding must be U.S. citizens or
permanent residents and demonstrate access to a sufficient number of CF patients from CF Foundation-accredited care centers.
• Through the Therapeutics Development Program, CFFT offers matching research awards to scientists as well as access to a specialized
network of CF clinical research centers.
95%
4%
Sale of IP
Other (e.g. Mutual Funds)
Special Events (e.g. Marathons)
General Contributions
Multi-Year Commitments
Key Facts
Sources of Funding (2014)
$137.6 million
in public
donations
Sources of Funding
• The Foundation’s sources of funding are through public donations, sale of intellectual property rights, product royalties, as well as other
investments (e.g. mutual funds).
• In 2014, CFF recorded $3.42 billion in revenue, amongst which 95% was the result of sales of IP rights. Of the $137.6 million received
from public donations, $16.6 million were part of multi-year donor commitments for specific areas of CF advocacy.
Cystic Fibrosis Foundation Consolidated Financial Statements Dec 2014
Investment Pipeline
6
8
10
12
14
16
18
7.5 8 8.5 9 9.5
ResearchTeams
Clinical Centers
Mucocilliary Clearance
CFTR Folding
CFTR 3D Structure
CF Biomarker
Relative # of publications
Cystic Fibrosis Foundation CF Research Consortia

2. The Cystic Fibrosis Foundation
Track Record
• CFF funding has allowed for the development of 9 CF therapies that have since entered market: Kalydeco, Hypertonic Saline, Pulmozyme,
Ibuprofen, Inhaled Tobramycin, Azithromycin, Cayston, Tobramycin Powder, Aquadeks, and Pancrelipase.
• In 2014, 6 CFF funded drugs were in Phase-III of clinical testing: Ataluren, VX-809, Bronchitol, Levofloxacin, Arikace, and Liprotamase.
Exits
• CFF may forgo their future claims to Foundation-funded discoveries for a lump sum payment, although they do not actively seek exits
from their investments.
• In 2014, CFF recorded $3.27 billion from the sale of intangible rights for a CF drug under their medical discovery agreement. In 2014, the
Foundation received over $9.4 million in royalties.
73%
11%
10%
3% 3%
Therapeutics Development
Program awards
Research grants
Clinical research grants
Clinical and research
fellowship grants
Quality improvement
training program
Research Investment Distribution (2014)
Research Investments vs. Return, 2010-2013
$0
$50
$100
$150
$200
$250
$0
$20
$40
$60
$80
$100
2010 2011 2012 2013
Return($million)
Investment($million)
Quality improvement training program
Clinical and research fellowship grants
Clinical research grants
Research grants
Therapeutics Development Program awards
Annual Research Investment (left)
Annual Research Investment Return (right)
$98.3 million
invested
Investment Structure
• CFF and CFFT invests in medical research through the Therapeutic Development Program, basic science and clinical research grants,
fellowships, and quality improvement training. CFF and CFFT retain legal and beneficial rights to intellectual property developed under
certain scientific grants and drug discovery agreements.
• CFF maintains a venture philanthropy business model. CFFT has contractual agreements primarily with pharmaceutical companies such
as Vertex, Pfizer, and Genzyme. CFF will receive royalties in the event of the approval and/or sales of certain drugs — including Kalydeco,
VX-809 and ataluren — that are developed as a result of CFFT funding. Any royalties received are reinvested into the Foundation.
Cystic Fibrosis Foundation Consolidated Financial Statements Dec 2014
Cystic Fibrosis Foundation Consolidated Financial Statements 2010-2013