Drug Development Pipeline

We’re attacking CF from every angle.

Click on any drug in the interactive pipeline below to learn more about CF drugs in development or already in use by patients.

Restore CFTR Function
- Pre-clinical
- Phase One
- Phase Two
- Phase Three
- To Patients
Mucociliary Clearance
- Pre-clinical
- Phase One
- Phase Two
- Phase Three
- To Patients
Anti-Inflammatory
- Pre-clinical
- Phase One
- Phase Two
- Phase Three
- To Patients
Anti-Infective
- Pre-clinical
- Phase One
- Phase Two
- Phase Three
- To Patients
Nutritional-GI-Other
- Pre-clinical
- Phase One
- Phase Two
- Phase Three
- To Patients

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Drug Descriptions

Restore CFTR Function

Elexacaftor + tezacaftor + ivacaftor (Trikafta®)

Elexacaftor + tezacaftor + ivacaftor (Trikafta^®) is a combination therapy combining three CFTR modulators. Elexacaftor and tezacaftor are CFTR correctors, a type of modulator designed to fix the defective CFTR protein so that it can move to the proper place on the cell surface. Ivacaftor is a potentiator. Once CFTR protein reaches the cell surface, potentiators help facilitate the opening of the chloride channel to allow chloride and sodium (salt) to move in and out of the cell.
Ivacaftor (Kalydeco®)

Ivacaftor (Kalydeco^®) is an oral medication that was the first drug available that targeted the underlying cause of CF – the defective CFTR protein. Ivacaftor helps facilitate the opening of the chloride channel on the cell surface to allow chloride and sodium (salt) to move in and out of the cell.
Lumacaftor + ivacaftor (Orkambi®)

Lumacaftor and ivacaftor (Orkambi^®) is a combination therapy combining lumacaftor, which is designed to fix the defective CFTR protein so that it can move to the proper place on the cell surface, with ivacaftor, which helps improve the function of the protein as a chloride channel on the cell surface.
Tezacaftor + ivacaftor (Symdeko®)

Tezacaftor + ivacaftor (Symdeko^®) is a combination therapy combining tezacaftor, a compound designed to move the defective CFTR protein to the proper place in the airway cell surface, with ivacaftor, which helps facilitate the opening of the chloride channel on the cell surface to allow chloride and sodium (salt) to move in and out of the cell.
Ataluren

Ataluren (Translarna™) is a novel, small molecule compound designed to enable production of a full-length and functional CFTR protein in individuals with CF who have nonsense mutations. The compound aims to help the body override a premature signal to stop production of the CFTR protein.
VX-659 + tezacaftor + ivacaftor

This program tested VX-659 in combination with tezacaftor and ivacaftor. VX-659 and tezacaftor (VX-661) are CFTR correctors. Correctors are drugs designed to fix the defective CFTR protein so that it can move to the proper place on the cell surface. Once CFTR protein reaches the cell surface, ivacaftor helps facilitate the opening of the chloride channel to allow chloride and sodium (salt) to move in and out of the cell.
QBW251

QBW251 is a type of CFTR Modulator called a “potentiator”. Similar to the drug ivacaftor, this drug would help to facilitate the opening of the chloride channel on the cell surface. This compound is administered through an oral pill.
ABBV-2222 (formerly GLPG2222)

ABBV-2222 (formerly GLPG2222) is a type of CFTR modulator called a corrector. Correctors are drugs designed to fix and restore the function of the defective CFTR protein. The corrected CFTR then moves to the cell surface, where it functions as a chloride channel and helps maintain the right balance of fluid in the airways.
ABBV-3067

ABBV-3067 is a type of CFTR modulator called a potentiator. Potentiators help facilitate the opening of the chloride channel on the cell surface to allow chloride and sodium (salt) to move in and out of the cell.
ELX-02

ELX-02 is a compound designed to restore CFTR function in people with CF who have nonsense mutations. Nonsense mutations (also known as “x” or “stop” mutations) in the CFTR gene cause the production of CFTR protein to stop prematurely. ELX-02 is intended allow lung cells to override these premature stop signals and make full-length, functional CFTR protein.
Nesolicaftor (PTI-428)

Nesolicaftor is a new type of modulator called an amplifier. Amplifiers increase the amount of CFTR protein in the cell. This makes more CFTR protein available for other therapies, such correctors and potentiators, to work on.
Posenacaftor (PTI-801)

Posenacaftor is a type of CFTR modulator called a corrector. Correctors are drugs designed to fix and restore the function of the defective CFTR protein. The corrected CFTR then moves to the cell surface, where it functions as a chloride channel and helps maintain the right balance of fluid in the airways.
Dirocaftor (PTI-808)

Dirocaftor is a type of CFTR modulator called a potentiator. Potentiators help facilitate the opening of the chloride channel on the cell surface to allow chloride and sodium (salt) to move in and out of the cell.
VX-121

VX-121 is a CFTR corrector. Correctors are drugs designed to fix and restore the function of the defective CFTR protein. The corrected CFTR then moves to the cell surface, where it functions as a chloride channel and helps maintain the right balance of fluid in the airways.
VX-561 (formerly CTP-656)

VX-561 is an altered form of the potentiator ivacaftor (Kalydeco®). Potentiators are drugs that facilitate the opening of the chloride channel on the cell surface to allow chloride and sodium (salt) to move in and out of the cell. VX-561, or deuterated ivacaftor, may be more stable in the body than regular ivacaftor, which would allow it to be taken once a day.
Cavosonstat (N91115)

Cavosonstat (N91115) is a compound that modulates the function of the defective CFTR protein and decreases inflammation in the lung. Cavosonstat is the first of a new class of compounds that increase levels of an important signaling molecule in the body, called S-nitrosoglutathione or GSNO. Levels of GSNO have been shown to be decreased in people with CF. These novel compounds have been shown to increase the amount of CFTR protein that reaches the cell surface and to stabilize CFTR protein so that its function can be improved.
FDL169

FDL169 is a CFTR corrector. Correctors are drugs designed to fix and restore the function of the defective CFTR protein. The corrected CFTR then moves to the cell surface, where it functions as a chloride channel and helps maintain the right balance of fluid in the airways.
Riociguat

Riociguat is a novel therapy that stimulates sGC, an enzyme in the cardiopulmonary system and the receptor for nitric oxide (NO). Preclinical data has shown evidence that riociguat can result in improved function of the CFTR protein as a chloride channel, moving salt and fluids in and out of cells.
VX-152 + tezacaftor + ivacaftor

This program is testing VX-152 in combination with tezacaftor and ivacaftor. VX-152 and tezacaftor (VX-661) are new CFTR correctors. Correctors are drugs designed to fix the defective CFTR protein so that it can move to the proper place on the cell surface. Once CFTR protein reaches the cell surface, ivacaftor helps facilitate the opening of the chloride channel to allow chloride and sodium (salt) to move in and out of the cell.
VX-440 + tezacaftor + ivacaftor

This program is testing VX-440 in combination with tezacaftor and ivacaftor. VX-440 and tezacaftor (VX-661) are new CFTR correctors. Correctors are drugs designed to fix the defective CFTR protein so that it can move to the proper place on the cell surface. Once CFTR protein reaches the cell surface, ivacaftor helps facilitate the opening of the chloride channel to allow chloride and sodium (salt) to move in and out of the cell.
Eluforsen (QR-010)

Eluforsen (QR-010) is an oligonucleotide designed to repair CFTR-encoded mRNA, which could result in a normal CFTR protein in people who have one or two copies of the F508del mutation. It is delivered via inhalation.
ABBV-191

This program is studying ABBV-191, a type of CFTR modulator called a potentiator.

Once CFTR protein reaches the cell surface, potentiators help facilitate the opening of the chloride channel to allow chloride and sodium (salt) to move in and out of the cell. This helps maintain the right balance of salt and water on the airway surface.
MRT5005

MRT5005 is a new drug designed to restore CFTR function by delivering correct copies of CFTR-encoded mRNA to the lungs. mRNA is a molecule that contains genetic instructions to make proteins. Delivery of CFTR-encoded mRNA would allow the lung cells to create normally functioning CFTR protein, regardless of an individual’s specific CFTR gene mutation. This drug is delivered via inhalation.
Arcturus

This program is developing a potential inhaled therapy to deliver normal CFTR messenger RNA to the lungs. Delivery of CFTR-encoded mRNA would allow the lung cells to create normally functioning CFTR protein, regardless of an individual’s specific CFTR gene mutation.
Icagen

This program is working to identify potential therapies for people with CF who have nonsense mutations. Nonsense mutations (also known as “x” or “stop” mutations) in the CFTR gene cause the production of CFTR protein to stop prematurely. Overriding these premature stop signals would allow full-length, functional CFTR protein to be made.
Reata

This program is working to expand the number of therapies designed to fix the defective CFTR protein in people who have the F508del mutation.
ReCode Therapeutics

This program is developing suppressor tRNA delivery to treat nonsense mutations. Nonsense mutations (also known as “x” or “stop” mutations) in the CFTR gene cause the production of CFTR protein to stop prematurely. Delivery of suppressor tRNA would allow lung cells to override these premature stop signals and make full-length, functional CFTR protein.
Southern Research Institutes

This program is working to identify potential therapies for nonsense mutations in the cystic fibrosis-causing gene.
Spirovant Sciences

This program is working to advance inhaled adeno-associated virus (AAV) delivery of the CFTR gene to the lungs. This would allow the lung cells to create normally functioning CFTR protein, regardless of an individual’s specific CFTR gene mutation.
4D Molecular Therapeutics

This program is working to advance a gene delivery vehicle that targets cells in the lung. 4D-710 is a customized adeno-associated virus (AAV) vector designed to deliver a healthy CFTR gene specifically to cells in the lungs of people with CF. This would allow the lung cells to create normally functioning CFTR protein, regardless of an individual’s specific CFTR gene mutation.

Mucociliary Clearance

Dornase Alfa (Pulmozyme®)

Dornase alfa (Pulmozyme^®) is an inhaled medication that thins and loosens mucus in the airways of people with CF.
Hypertonic Saline

Hypertonic saline as an inhaled therapy is believed to increase hydration of airway surface liquid in people with CF, thereby improving mucus clearance.
Inhaled Mannitol (Bronchitol®)

Bronchitol^® is an inhaled dry powder form of mannitol, a naturally occurring osmotic agent, which works by drawing water into the airways. This helps to moisten and thin the sticky mucus found in the lungs of people with CF, making it easier to cough it out.
BI 1265162

BI 1265162 is a compound designed to block the function of the sodium (Na+) channel found in the lungs. These channels move sodium and water away from the airway surface. Blocking these channels may help maintain fluid within the airways to improve mucus clearance.
IONIS-ENaC-2.5

IONIS-ENaC-2.5_Rx is a compound designed to block the function of the sodium (Na+) channel found in the lungs. These channels move sodium and water away from the airway surface. Blocking these channels may help maintain fluid within the airways to improve mucus clearance.
OligoG

OligoG is a dry powder drug that has been shown to decrease the thickness of mucus in the lungs and may help individuals with cystic fibrosis clear mucus easier. OligoG may also help improve the effectiveness of some antibiotics. It is administered using a dry powder inhaler and also developed as a liquid for use with a nebulizer.
QBW276

QBW276 is a compound designed to block the function of the sodium (Na+) channel found in the lungs. These channels move sodium and water away from the airway surface. Blocking these channels may help maintain fluid within the airways to improve mucus clearance.
SPX-101

SPX-101 is a compound designed to block the function of the sodium (Na+) channel found in the lungs. These channels move sodium and water away from the airway surface. Blocking these channels may help maintain fluid within the airways to improve mucus clearance.
VX-371 formerly (P-1037)

VX-371 is a compound designed to block the function of the sodium (Na+) channel found in the lungs. These channels move sodium and water away from the airway surface. Blocking these channels may help maintain fluid within the airways to improve mucus clearance.
AZD5634

AZD5634 is a compound designed to block the function of the sodium (Na+) channel found in the lungs. These channels move sodium and water away from the airway surface. Blocking these channels may help maintain fluid within the airways to improve mucus clearance.
ETD002

ETD002 is a compound designed to increase the activity of TMEM16A, a chloride channel found on the surfaces of airway cells. Similar to the CFTR channel, the TMEM16A channel helps regulate the amount of salt and fluids on the airway surface. Increasing the activity of TMEM16A may help increase the amount of fluid within the airways to improve mucus clearance.
Denufosol

This alternative chloride channel activator has been removed from the pipeline due to lack of efficacy.

Anti-Inflammatory

High dose ibuprofen for CF

Ibuprofen is an oral, non-steroidal, anti-inflammatory medication.
Lenabasum (JBT-101)

Lenabasum (JBT-101) is an oral drug that is aimed at promoting the resolution of inflammation. It is thought to increase production of anti-inflammatory molecules while reducing production of molecules that increase inflammation. Reduction of inflammation helps prevent permanent tissue damage in the lungs.
Acebilustat (CTX-4430)

Acebilustat (CTX-4430) is an oral anti-inflammatory drug that reduces production of leukotriene B4 (LTB4), a molecule that leads to inflammation and is known to be elevated in people with CF. Reduction of inflammation helps prevent permanent tissue damage in the lungs.
Alpha 1 Anti-Trypsin

Alpha 1 Anti-trypsin is an aerosolized protease inhibitor with anti-inflammatory properties, derived from human plasma.
Docosahexaenoic Acid (DHA)

Docosahexaenoic acid (DHA) is an omega-3 fatty acid with anti-inflammatory properties.
LAU-7b

LAU-7b is an oral compound. It is a form of the retinoid fenretinide. Retinoids are a group of compounds related to vitamin A. Fenretinide may help reduce the inflammatory response in the lungs of people with CF.
GS-5745

GS-5745 is an antibody that may help reduce inflammation in the lungs, leading to improved lung function in people with CF.
KB001-A

KB001-A is a humanized monoclonal Fab fragment that targets a Pseudomonas aeruginosa virulence factor (Type III secretion system).
Sildenafil

Sildenafil is a phosphodiesterase inhibitor. Researchers at National Jewish Health in Denver examined whether sildenafil can lower markers of airway inflammation in people with CF.
CB-280

CB-280 is an oral drug designed to increase the amount of arginine in the lungs. Arginine is a molecule that occurs naturally in the body, and is important for the lungs to produce nitric oxide, a gas that helps the lungs fight infection.

Sputum from people with CF has been shown to contain lower amounts of arginine and nitric oxide than normal. Lower nitric oxide levels are associated with worsened lung function and increased infection. Increasing arginine levels may increase the production of nitric oxide, which would strengthen the immune response, reduce inflammation and improve lung function.
Lonodelestat (formerly POL6014)

Lonodelestat is a compound designed to block the function of neutrophil elastase. Neutrophil elastase is a type of protein associated with inflammation in the lungs. Blocking the function of neutrophil elastase may minimize tissue destruction and decrease inflammation within the CF airway.
PUR 118

This compound is an inhaled dry powder formulation of a cationic airway liquid modulator. It has anti-inflammatory properties and increases mucociliary clearance.

Anti-Infective

Amikacin Liposome Inhalation Suspension (Arikayce®)

Amikacin liposome inhalation suspension (Arikayce^®) is an antibiotic to treat chronic lung infections caused by nontuberculous mycobacteria (NTM).
Azithromycin

Azithromycin is an oral antibiotic with anti-inflammatory properties.
Aztreonam (Cayston®)

Inhaled aztreonam (Cayston^®) is an antibiotic that has been approved for people with CF who are chronically infected with Pseudomonas aeruginosa.
Inhaled Tobramycin

TOBI^®and BETHKIS^®are aerosolized forms of the antibiotic tobramycin, which is used to treat people with CF who are chronically infected with Pseudomonas aeruginosa.
Tobramycin Inhaled Powder (TOBI® Podhaler™)

The TOBI® Podhaler™ is the powder form of the inhaled antibiotic tobramycin and is used to treat people with CF who have Pseudomonas aeruginosa. It is taken with an inhaler, making it a convenient alternative to TOBI®, which must be taken using a nebulizer.
Inhaled Levofloxacin (Quinsair™)

Inhaled levofloxacin (Quinsair™) is a formulation of the antibiotic levofloxacin for the management of chronic lung infections caused by Pseudomonas aeruginosa and other bacteria.
Vancomycin Inhalation Powder (AeroVanc™)

Vancomycin inhalation powder (AeroVanc™) is an inhaled dry powder version of the antibiotic vancomycin for the treatment of methicillin-resistant Staphylococcus aureus (MRSA) airway infection in people with CF.
Intravenous Gallium

Gallium is a molecule, nearly identical to iron, that disrupts iron-dependent biological processes and has been shown to kill antibiotic-resistant strains of Pseudomonas aeruginosa in laboratory research. Gallium has already been approved by the FDA for intravenous use in people. It is currently being studied for its effectiveness in treating infections in people with CF.
AP-PA02

This program is studying AP-PA02, a type of phage therapy designed to fight Pseudomonas aeruginosa infections in people with CF. Bacteriophages are specialized viruses that kill very specific bacterial strains. They are found in the environment and are the predators of bacteria in nature. “Phage therapy” refers to the use of these bacteriophages to treat an infection in a person.

AP-PA02 is a mix of multiple different types of phages that is also known as an inhaled cocktail. In lab tests AP-PA02 was able to kill more than 80 percent of Pseudomonas strains from people with CF.
Inhaled Nitric Oxide

Nitric oxide is a gas with antimicrobial properties that may reduce lung infections when inhaled, leading to improved lung function in people with CF.
SPI-1005

SPI-1005 is an oral form of the drug ebselen. Ebselen is a molecule designed to mimic the function of a key enzyme found in the inner ear, glutathione peroxidase (GPx). Regular use of aminoglycoside antibiotics can cause damaging side effects in the inner ear. By mimicking the function of GPx, SPI-1005 may protect the inner ear from damage.
Inhaled Molgramostim

Inhaled molgramostim is a form of granulocyte macrophage-colony stimulating factor (GM-CSF). GM-CSF is a protein that occurs naturally in the human immune system and plays an important role in activating the immune system to kill bacteria and other infections. Because inhaled mogramostim helps to stimulate the part of the immune system that fights Nontuberculous mycobacteria (NTM), it may be able to help the body clear NTM infection.
ALX-009

ALX-009 is a combination of two substances found naturally in the body, hypothiocyanite (OSCN-) and lactoferrin. Both of these molecules are part of the body’s natural immune system, but they are deficient in the airway surface liquid of people with CF. By adding both OSCN- and lactoferrin to the airways, ALX-009 may increase the ability of people with CF to fight bacteria without harming their body’s own cells. ALX-009 is delivered via inhalation.
Inhaled Gallium

This program is studying an inhaled formulation of gallium. Gallium is a molecule, nearly identical to iron, that disrupts iron-dependent biological processes and has been shown to kill antibiotic-resistant strains of Pseudomonas aeruginosa in laboratory research.

Gallium can also be administered intravenously. Intravenous (IV) gallium has already been approved by the FDA for use in people and is now being studied for its safety and effectiveness in controlling infections in people with CF.
SNSP113

SNSP113 is a new inhaled glycopolymer, a type of molecule being developed to treat infection and inflammation in the lungs. When bacteria infect the lungs of people with CF, it can form structures called biofilms. Biofilms make bacteria more resistant to antibiotics. SNSP113 helps break up biofilms in the lungs, allowing antibiotics to fight bacteria more effectively. SNSP113 may also make mucus thinner and less sticky, reducing inflammation and improving airway clearance.
Inhaled Murepavadin

This program is studying an inhaled version of murepavadin, an antibiotic that targets multi-drug resistant Pseudomonas aeruginosa infections in people with cystic fibrosis. An inhaled version could make it easier for someone with a Pseudomonas infection to take the drug from home. In addition, the drug, which targets the outer membrane of bacteria, has the potential to be a once-a-day treatment.
Matinas BioPharma

This program is studying MAT2501, an oral version of the drug amikacin. Amikacin is an antibiotic that targets nontuberculous mycobacteria (NTM) infections in people with cystic fibrosis. An oral formulation of the drug may reduce the risk of known amikacin side effects such as hearing loss and kidney problems. An oral version would also make it easier for someone with an NTM infection to take the drug from home.
Microbion

This program is studying pravibismane, a new inhaled antibiotic. In the lungs of people with CF, bacteria may form protective layers -- known as biofilms -- that make them more difficult to kill. Pravibismane may help break down biofilms and kill drug-resistant bacteria, such as multi-drug resistant Pseudomonas aeruginosa and nontuberculous mycobacteria.
Respirion

This program is developing and testing a new antibiotic therapy for cystic fibrosis. This therapy is a combination of two existing approved drugs: the antibiotic tobramycin and another compound, calcium EDTA. When bacteria infect the lungs of people with CF, they can form structures called biofilms. Biofilms make bacteria more resistant to antibiotics. The compound calcium EDTA may help break down biofilms in the lungs, allowing antibiotics to fight bacteria more effectively.
TB Alliance

This program will identify and test potential treatments for infections caused by Mycobacterium abscessus (M. abscessus) and Mycobacterium avium complex (MAC), two types of nontuberculous mycobacteria (NTM).

Nutritional-GI-Other

AquADEKs

AquaADEKs® is an oral antioxidant vitamin formulation specifically for people with CF.
Pancrelipase Enzyme Products

These are digestive enzymes that are prepared using pancreases from pigs.
RELiZORB®

RELiZORB^® is a digestive enzyme cartridge that connects to enteral tube feeding systems. It is designed to mimic normal pancreatic function by helping to break down the fats in enteral tube feeding formula. By breaking down these fats before the formula is ingested, RELiZORB^® may allow for delivery of increased absorbable calories and simplify the use of enzymes in overnight feedings.
Liprotamase

Liprotamase (Sollpura) is a pancreatic enzyme replacement that is not prepared from animal sources.
Burlulipase

Burlulipase is a liquid solution developed to treat pancreatic insufficiency caused by CF. It is a bacterial lipase that has been shown to be more resistant against inactivation by gastric acid, giving the drug the potential to be more effective than existing pancreatin products derived from pigs.
Glutathione

Glutathione is an antioxidant that is important to the normal functioning of the intestine and lungs. Glutathione levels have been shown to be lower in people with CF. Oral glutathione may improve growth and decrease gut inflammation in children with CF.
MS1819-SD

MS1819-SD is a non-porcine (not pig-derived) enzyme for individuals with CF who have exocrine pancreatic insufficiency. It is a man-made version of a lipase enzyme taken from the yeast Yarrowia lipolytica. This drug does not contain any animal products.
Synspira

This program is studying SNSP003, a non-porcine (not pig-derived) enzyme replacement therapy for individuals with CF who have exocrine pancreatic insufficiency. It is a man-made version of a lipase enzyme taken from bacteria, and it does not contain any animal products.

SNSP003 may improve dosing convenience by reducing pill burden compared to current porcine-based pancreatic enzyme replacement therapies (PERT). SNSP003 is also being developed in a formulation for pediatric and adult patients who are unable to swallow capsules.

To advance drug development and a search for a cure, the Cystic Fibrosis Foundation (CFF) has contracts with several companies to help fund the development of potential treatments and/or cures for cystic fibrosis. Pursuant to these contracts, CFF may receive milestone based payments, equity interests, royalties on the net sales of therapies, and/or other forms of consideration. Resulting revenue received by CFF is used in support of our mission. See “How Drugs Get on the Pipeline.” for more.